1. Presentations highlight progress in the development of losmapimod for FSHD –

    CAMBRIDGE, Mass., Sept. 20, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it will present multiple posters and presentations on losmapimod for facioscapulohumeral muscular dystrophy (FSHD) during the Virtual Congress of the World Muscle Society. These results highlight progress made in the development of losmapimod for the treatment of FSHD as well as imaging modalities for the management of FSHD.

    "FSHD is a serious, relentless and debilitating disease for which there are currently no approved therapies…

    Presentations highlight progress in the development of losmapimod for FSHD –

    CAMBRIDGE, Mass., Sept. 20, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it will present multiple posters and presentations on losmapimod for facioscapulohumeral muscular dystrophy (FSHD) during the Virtual Congress of the World Muscle Society. These results highlight progress made in the development of losmapimod for the treatment of FSHD as well as imaging modalities for the management of FSHD.

    "FSHD is a serious, relentless and debilitating disease for which there are currently no approved therapies," said Chris Morabito, MD, Fulcrum's chief medical officer. "The results from the Phase 2b ReDUX4 trial show clinically relevant benefit across multiple structural, functional and patient reported endpoints with losmapimod and represent a major advance for the potential treatment of FSHD. These data being presented support the benefit/risk of losmapimod as a disease-modifying therapy and its continued development. We remain on track to meet with regulators in the fourth quarter of this year and to provide an update on the clinical path forward in the first quarter of 2022."

    Fulcrum-Sponsored Symposium:

    Advances in Assessment of FSHD and Clinical Trial Results with Losmapimod

    Date/Time: Monday, September 20, 12:00pm – 1:30pm EDT

    Presenters:

    • Jeffrey Statland, MD, PhD – University of Kansas Medical Center
    • Michelle Mellion, MD – Fulcrum Therapeutics
    • Rabi Tawil, MD – University of Rochester Medical Center
    • Baziel van Engelen & Joost Kools – Radboud University Medical Center
    • Chris Morabito, MD – Fulcrum Therapeutics

    Oral Presentation:

    A Phase 2, Randomized, Double-blind, Placebo-controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4

    Date/Time: Thursday, September 23rd, 10:30am – 10:40am EDT

    Presenter:

    • Rabi Tawil, MD – University of Rochester Medical Center

    Poster Presentations:

    Date/Time: Thursday, September 23rd, Available for viewing starting at 11:30am EDT

    Posters will be available to registered conference attendees and will also be available in the "Publications" section of fulcrumtx.com.

    Title: An Open-Label Study of Losmapimod to Evaluate the Safety, Tolerability, and Biomarker and Clinical Outcome Assessment Changes in Subjects with FSHD1

    Author: Joost Kools – Radboud University Medical Center

    Poster ID: EP 168

    Title: Use of snRNA-seq to Characterize the Pathogenic Skeletal Muscle Microenvironment

    Author: Anu Raman, Ph.D. – Fulcrum Therapeutics

    Poster ID: EP 170

    Title: Whole body MRI Quantitative Muscle Analysis to Evaluate Efficacy of Losmapimod in a Phase 2 Placebo-controlled Study in Subjects with FSHD (ReDUX4)

    Author: Michelle Mellion, MD – Fulcrum Therapeutics

    Poster ID: EP 330

    Title: Quantitative Muscle Analysis in FSHD Using Whole-Body MRI: Composite Muscle Measurements for Cross-Sectional Analysis

    Author: Michelle Mellion, MD – Fulcrum Therapeutics

    Poster ID: EP 331

    Title: Revolutionizing Drug Discovery in Genetically Defined Muscle Disease Using Single-Cell and High Dimensional Datasets

    Author: Alejandro Rojas, Ph.D. – Fulcrum Therapeutics

    Poster ID: EP 321

    About FSHD

    FSHD is a serious, rare, progressive and disabling disease for which there are no approved treatments. FSHD is characterized by muscle degeneration and fat infiltration, initially affecting movement of the face and eventually the arms, trunk and legs. Disease progression results in accumulation of disability, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility. Impact on patients includes decreased ability to perform activities of daily living, maintain independence, and lost ability to function or work.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its proprietary product engine, FulcrumSeek™, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Losmapimod has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of the Company's product candidates and planned meetings with regulatory agencies. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  2. CAMBRIDGE, Mass., Sept. 10, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the grant of an inducement award outside of the Company's 2019 Stock Incentive Plan to Mel Hayes, the Company's newly appointed Chief Commercial Officer. The grant was approved by the Compensation Committee on August 4, 2021 as an inducement material to the employee entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    The inducement grant consisted of a nonstatutory option to purchase up to 140,000 shares of common stock. The option has an exercise price of $28.49 per…

    CAMBRIDGE, Mass., Sept. 10, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the grant of an inducement award outside of the Company's 2019 Stock Incentive Plan to Mel Hayes, the Company's newly appointed Chief Commercial Officer. The grant was approved by the Compensation Committee on August 4, 2021 as an inducement material to the employee entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    The inducement grant consisted of a nonstatutory option to purchase up to 140,000 shares of common stock. The option has an exercise price of $28.49 per share, the closing price per share of the Company's common stock as reported by Nasdaq on September 7, 2021. The option has a ten-year term and vests over four years, with 25% of the original number of shares vesting on the first anniversary of the employee's start date and an additional 6.25% of the shares vesting in equal quarterly installments over the twelve successive quarters following the first anniversary, subject to such employee's continued service with the Company through the applicable vesting dates.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. Fulcrum has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development.

    Contact:

    Investors:



    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

                    



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  3. CAMBRIDGE, Mass., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the appointment of Mel Hayes as Chief Commercial Officer, effective September 7, 2021. Mr. Hayes will be responsible for delivering an integrated global strategy that fully maximizes the potential of Fulcrum's transformative therapies.

    "We are very excited to welcome Mel to Fulcrum as we plan for important milestones with our development programs and FulcrumSeek™," said Bryan Stuart, president and chief executive officer. "Mel brings broad experience in building commercial organizations and launching products…

    CAMBRIDGE, Mass., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the appointment of Mel Hayes as Chief Commercial Officer, effective September 7, 2021. Mr. Hayes will be responsible for delivering an integrated global strategy that fully maximizes the potential of Fulcrum's transformative therapies.

    "We are very excited to welcome Mel to Fulcrum as we plan for important milestones with our development programs and FulcrumSeek™," said Bryan Stuart, president and chief executive officer. "Mel brings broad experience in building commercial organizations and launching products in markets around the world. His expertise positions Fulcrum well to build the commercial capabilities required to deliver transformative therapies that treat the root cause of genetically defined rare diseases."

    "With Fulcrum's recent progress with losmapimod and FTX-6058, the company is ideally positioned to transition from a research and development-stage organization to a commercial-stage organization," said Mr. Hayes. "I look forward to working with the experienced leadership team at Fulcrum, leveraging my extensive knowledge of commercialization strategies for therapies to treat rare diseases. I'm excited about the great work already in motion as we aim to bring new and innovative treatments to patients around the world."

    Mr. Hayes is an industry leader with more than 25 years of Global and U.S. experience in all areas of product commercialization including marketing, sales, new product planning, pricing and reimbursement, advocacy and patient engagement. Prior to Fulcrum, Mr. Hayes most recently served as Global Head Commercial, Vice President, Rare Blood Disorders at Sanofi-Genzyme where he led the global commercial organization for hemophilia and complement assets. He also served as U.S. Vice President Hemophilia and Global Head, Hematology Rare Blood Disorders at Bioverativ (acquired by Sanofi-Genzyme). Prior to Bioverativ, he served as Global Vice President, Head of Global Marketing and Launch Excellence at Shire and at Baxalta (acquired by Shire) as Global Vice President Hemophilia. Prior to Baxalta, Mr. Hayes spent 10 years and nine years at Bayer and Bristol Myers Squibb respectively in progressive commercial leadership roles where he was responsible for launching products in Diabetes, Cardiovascular Disease, Neurology, Rheumatology, Multiple Sclerosis and Parkinson's Disease.

    Mr. Hayes earned dual bachelor's degrees in Business and Communications from Southern Methodist University and an MBA from Columbia University.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The Company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development. Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of Fulcrum's product candidates and the Company's ability to transition from a research and development-stage organization to a commercial organization. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  4. CAMBRIDGE, Mass., Sept. 02, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will be participating in the following upcoming investor conferences:

    • Morgan Stanley 19th Annual Global Healthcare Conference
      Thursday, September 9, 2021 at 4:15 p.m. ET
    • H.C. Wainwright 23rd Annual Global Investment Conference
      Monday, September 13, 2021 at 7:00 a.m. ET
    • Cantor Virtual Global Healthcare Conference
      Tuesday, September 28, 2021 at 3:20 p.m. ET

    Live audio webcasts will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations

    CAMBRIDGE, Mass., Sept. 02, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will be participating in the following upcoming investor conferences:

    • Morgan Stanley 19th Annual Global Healthcare Conference

      Thursday, September 9, 2021 at 4:15 p.m. ET
    • H.C. Wainwright 23rd Annual Global Investment Conference

      Monday, September 13, 2021 at 7:00 a.m. ET
    • Cantor Virtual Global Healthcare Conference

      Tuesday, September 28, 2021 at 3:20 p.m. ET

    Live audio webcasts will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for 30 days.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The Company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development. 

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  5. CAMBRIDGE, Mass., Aug. 16, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced closing of its previously announced underwritten public offering of 7,590,000 shares of its common stock at a public offering price of $19.00 per share, which includes 990,000 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares of common stock at the public offering price, less underwriting discounts and commissions. The total gross proceeds of the offering were $144.2 million, before deducting underwriting discounts and commissions and offering expenses…

    CAMBRIDGE, Mass., Aug. 16, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced closing of its previously announced underwritten public offering of 7,590,000 shares of its common stock at a public offering price of $19.00 per share, which includes 990,000 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares of common stock at the public offering price, less underwriting discounts and commissions. The total gross proceeds of the offering were $144.2 million, before deducting underwriting discounts and commissions and offering expenses payable by Fulcrum. All of the shares in the offering were sold by Fulcrum.

    SVB Leerink, Piper Sandler & Co. and Stifel acted as joint bookrunning managers for the offering. H.C. Wainwright & Co. acted as lead manager for the offering.

    The shares were offered by Fulcrum pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC") and a related registration statement that was filed with the SEC on August 11, 2021 pursuant to Rule 462(b) under the Securities Act of 1933, which became automatically effective upon filing.

    This offering was made only by means of a prospectus and prospectus supplement that form a part of the registration statements. A final prospectus supplement relating to and describing the terms of the offering has been filed with the SEC and is available on the SEC's website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting: SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, Massachusetts 02109, by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@svbleerink.com; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, by telephone at (800) 747-3924, or by email at prospectus@psc.com; or Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at 415-364-2720 or by email at syndprospectus@stifel.com.

    This press release shall not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The Company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development. 

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  6. CAMBRIDGE, Mass., Aug. 11, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 6,600,000 shares of its common stock at a public offering price of $19.00 per share, for total gross proceeds of approximately $125 million, before deducting underwriting discounts and commissions and offering expenses payable by Fulcrum. All of the shares in the offering are being sold by Fulcrum. In addition, Fulcrum has granted the underwriters a 30-day option to purchase up to 990,000 additional shares of its common stock at the public offering price…

    CAMBRIDGE, Mass., Aug. 11, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 6,600,000 shares of its common stock at a public offering price of $19.00 per share, for total gross proceeds of approximately $125 million, before deducting underwriting discounts and commissions and offering expenses payable by Fulcrum. All of the shares in the offering are being sold by Fulcrum. In addition, Fulcrum has granted the underwriters a 30-day option to purchase up to 990,000 additional shares of its common stock at the public offering price, less the underwriting discount and commissions. The offering is expected to close on August 16, 2021, subject to customary closing conditions.

    SVB Leerink, Piper Sandler & Co. and Stifel are acting as joint bookrunning managers for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering.

    The shares are being offered by Fulcrum pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC") and a related registration statement that was filed with the SEC on August 11, 2021 pursuant to Rule 462(b) under the Securities Act of 1933 (and became automatically effective upon filing).

    This offering is being made only by means of a prospectus and prospectus supplement that form a part of the registration statements. A preliminary prospectus supplement relating to and describing the terms of the offering has been filed with the SEC and is available on the SEC's website at www.sec.gov. A final prospectus supplement relating to the offering will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting: SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, Massachusetts 02109, by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@svbleerink.com; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, by telephone at (800) 747-3924, or by email at prospectus@psc.com; or Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at 415-364-2720 or by email at syndprospectus@stifel.com.

    This press release shall not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeekTM, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The Company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development.  

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, such as those, among others, relating to the Company's plans to consummate the public offering. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include, without limitation, risks and uncertainties related to the satisfaction of customary closing conditions related to the public offering and the impact of general economic, industry or political conditions in the United States or internationally. There can be no assurance that the Company will be able to complete the public offering on the anticipated terms, or at all. You should not place undue reliance on these forward-looking statements. Additional risks and uncertainties relating to the offering, the Company and its business can be found under the caption "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, the Company's preliminary prospectus supplement filed with the SEC on August 10, 2021, and other filings that the Company may make with the SEC in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  7. CAMBRIDGE, Mass., Aug. 10, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has commenced an underwritten public offering of $100 million of shares of its common stock. All of the shares are being offered by Fulcrum. In addition, Fulcrum expects to grant the underwriters a 30-day option to purchase up to an additional $15 million of shares of its common stock.

    SVB Leerink, Piper Sandler & Co. and Stifel are acting as joint book-running managers for the offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering…

    CAMBRIDGE, Mass., Aug. 10, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has commenced an underwritten public offering of $100 million of shares of its common stock. All of the shares are being offered by Fulcrum. In addition, Fulcrum expects to grant the underwriters a 30-day option to purchase up to an additional $15 million of shares of its common stock.

    SVB Leerink, Piper Sandler & Co. and Stifel are acting as joint book-running managers for the offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    The shares are being offered by Fulcrum pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC").

    This offering is being made only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement relating to and describing the terms of the offering is expected to be filed with the SEC and, if and when filed, copies of the preliminary prospectus supplement relating to the offering may be obtained for free by visiting the SEC's website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus may also be obtained by contacting: SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, Massachusetts 02109, by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@svbleerink.com; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, by telephone at (800) 747-3924, or by email at prospectus@psc.com; or Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, or by telephone at (415) 364-2720. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

    This press release shall not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.  

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The Company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, such as those, among others, relating to the Company's plans to consummate its proposed public offering. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include, without limitation, risks and uncertainties related to whether or not the Company will be able to raise capital through the sale of shares of common stock, the final terms of the proposed offering, market and other conditions, the satisfaction of customary closing conditions related to the proposed public offering and the impact of general economic, industry or political conditions in the United States or internationally. There can be no assurance that the Company will be able to complete the proposed public offering on the anticipated terms, or at all. You should not place undue reliance on these forward-looking statements. Additional risks and uncertainties relating to the proposed offering, the Company and its business can be found under the caption "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, the Company's preliminary prospectus supplement expected to be filed with the SEC on August 10, 2021, and other filings that the Company may make with the SEC in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com 



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  8. – Reported positive interim results from ongoing Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease demonstrating proof of mechanism and proof of biology –

    – Company plans to initiate enrollment in a Phase 1b clinical trial in sickle cell patients in 4Q 2021 –

    – Cash runway into 1Q 2023 –

    – Conference call scheduled for 8:00 a.m. ET today –

    CAMBRIDGE, Mass., Aug. 10, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the second quarter of 2021.

    "This quarter was marked by meaningful…

    – Reported positive interim results from ongoing Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease demonstrating proof of mechanism and proof of biology –

    – Company plans to initiate enrollment in a Phase 1b clinical trial in sickle cell patients in 4Q 2021 –

    – Cash runway into 1Q 2023 –

    – Conference call scheduled for 8:00 a.m. ET today –

    CAMBRIDGE, Mass., Aug. 10, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the second quarter of 2021.

    "This quarter was marked by meaningful progress, particularly as we furthered our efforts with our lead candidates for FSHD and sickle cell disease," said Bryan Stuart, president and chief executive officer. "We made groundbreaking progress with losmapimod and reported clinical data from ReDUX4 demonstrating slowed disease progression and improved function in FSHD. We are also pleased with the dose proportional target engagement data, fetal hemoglobin (HBG) mRNA induction and F-reticulocyte increases that we observed in our ongoing Phase 1 trial in healthy adult volunteers with FTX-6058, demonstrating proof of mechanism and proof of biology. These results, along with the safety, tolerability and pharmacokinetics observed to date, reinforce our belief in FTX-6058's potential to be a best-in-class treatment for select hemoglobinopathies. Our team continues to optimize the potential of FulcrumSeek™, our proprietary product engine. Driven by our focus on the patient and our ability to rapidly identify novel, high quality targets that modulate the root cause of genetically defined rare diseases, we are on track to submit two investigational new drug applications (INDs) by the end of the first quarter of 2023. With these important advances and cash runway that takes us into the first quarter of 2023, we believe we are well positioned to continue to build on our momentum to bring important therapies to patients with genetically defined rare diseases."

    Recent Business Highlights

    • Reported positive interim results today from the ongoing single- and multiple-ascending dose (SAD and MAD) Phase 1 trial in healthy adult volunteers with FTX-6058. FTX-6058 is an investigational, potent and selective small molecule inhibitor of EED designed to induce expression of fetal hemoglobin (HbF) with the potential to treat hemoglobinopathies, such as sickle cell disease and beta-thalassemia.
      • Results from the MAD portion of the trial demonstrated proof of biology as evidenced by a dose proportional induction in HBG mRNA and accompanying increases in HbF-containing reticulocytes (F-reticulocytes). At 10mg, the highest dose studied to date, the mean changes were 4.5-fold and 4.2-fold, respectively. The increases in F-reticulocytes indicate that the HBG mRNA increases observed with FTX-6058 treatment are translating to HbF protein production.
      • All FTX-6058 doses from the MAD portion of the trial achieved maximal target engagement.
      • FTX-6058 was generally well-tolerated with no serious adverse events observed to date.
      • Fulcrum plans to initiate enrollment in a Phase 1b clinical trial in sickle cell patients with FTX-6058 in the fourth quarter of 2021.
      • Fulcrum plans to submit an IND in non-sickle cell disease hemoglobinopathies (e.g., beta thalassemia) by year-end 2021.
    • Data reported in June from ReDUX4, a Phase 2b trial of losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor, demonstrated slowed disease progression and improved function in FSHD.
      • Results showed clinically relevant and nominally statistically significant benefits versus placebo on multiple measures of structural and functional FSHD disease progression and patient reported outcomes at 48 weeks. Consistent with the previously reported interim analyses, the primary endpoint was not met.
      • Losmapimod was generally well-tolerated, with no drug-related serious adverse events reported.
      • Fulcrum plans to meet with health authorities, including the U.S. Food and Drug Administration (FDA), in the second half of 2021 to determine the regulatory path for losmapimod in FSHD.
    • Presented the medicinal chemistry strategy for FTX-6058 at the First Time Disclosure Session at the American Chemical Society (ACS) Spring 2021 National Meeting. The presentation included initial pharmacokinetic data from the SAD cohort of the Phase 1 trial in healthy adult volunteers.
    • Advanced FulcrumSeek discovery efforts and strategic collaborations with Acceleron and MyoKardia, a wholly owned subsidiary of Bristol-Myers Squibb Company.
    • Mani Sundararajan, Ph.D., joined Fulcrum in July 2021 as Senior Vice President, Technical Operations.
    • Christopher J. Morabito, M.D. joined Fulcrum in May 2021 as Chief Medical Officer.
    • Judith A. Dunn, Ph.D. joined Fulcrum in April 2021 as President of Research and Development.

    Second Quarter 2021 Financial Results

    • Cash Position: As of June 30, 2021, cash, cash equivalents, and marketable securities were $125.6 million, as compared to $112.9 million as of December 31, 2020. Based on current plans, the company expects that its existing cash, cash equivalents, and marketable securities will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2023.



    • Collaboration Revenue: Collaboration revenue was $4.4 million for the second quarter of 2021, as compared to $2.0 million for the second quarter of 2020. The increase in collaboration revenue was due to the execution of the company's collaboration and license agreement with MyoKardia in July 2020, as well as due to an increase in collaboration revenue associated with the company's collaboration and license agreement with Acceleron.



    • R&D Expenses: Research and development expenses were $17.4 million for the second quarter of 2021, as compared to $12.8 million for the second quarter of 2020. The increase of $4.6 million was primarily due to increased costs to support the company's ongoing and planned clinical trials.



    • G&A Expenses: General and administrative expenses were $6.7 million for the second quarter of 2021, as compared to $5.1 million for the second quarter of 2020. The increase of $1.6 million was primarily due to increased employee-compensation costs to support the growth of the organization, including increased stock-based compensation expense, as well as increased professional services costs.



    • Net Loss: Net loss was $19.6 million for the second quarter of 2021, as compared to a net loss of $15.7 million for the second quarter of 2020.

    Conference Call and Webcast

    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the Company's second quarter 2021 recent business highlights and financial results. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 3291056

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 3291056

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Losmapimod has been evaluated in more than 3,600 subjects in clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary product engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum is conducting a Phase 1 trial with FTX-6058 in healthy adult volunteers.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.



    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of Fulcrum's product candidates, initiation and enrollment of clinical trials and availability of clinical trial data, the Company's planned meetings with regulatory agencies, the Company's ability to submit an IND by the end of 2021 and the Company's ability to submit two INDs by the end of the first quarter of 2023, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Fulcrum Therapeutics, Inc.

    Selected Consolidated Balance Sheet Data

    (In thousands)

    (Unaudited)

     June 30,

    2021
      December 31,

    2020
     
    Cash, cash equivalents, and marketable securities$125,550  $112,914 
    Working capital(1) 106,001   92,785 
    Total assets 137,879   129,577 
    Total stockholders' equity 110,890   95,181 

    (1)    We define working capital as current assets minus current liabilities.

    Fulcrum Therapeutics, Inc.

    Consolidated Statements of Operations

    (In thousands, except per share data)

    (Unaudited)

     Three Months Ended

    June 30,
      Six Months Ended

    June 30,
     
     2021  2020  2021  2020 
    Collaboration revenue$4,381  $2,000  $9,170  $2,750 
    Operating expenses:               
    Research and development 17,378   12,775   33,712   27,257 
    General and administrative 6,685   5,149   12,183   10,213 
    Total operating expenses 24,063   17,924   45,895   37,470 
    Loss from operations (19,682)  (15,924)  (36,725)  (34,720)
    Other income, net 34   239   78   583 
    Net loss$(19,648) $(15,685) $(36,647) $(34,137)
    Net loss per share, basic and diluted$(0.60) $(0.66) $(1.14) $(1.47)
    Weighted-average common shares outstanding, basic and diluted 32,636   23,854   32,076   23,287 

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881 



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    • Achieved dose proportional pharmacodynamic changes in HBG mRNA and F-reticulocytes in whole blood
      • Mean 4.5-fold induction in HBG mRNA at 10mg after 14 days of once-daily dosing
      • Mean 4.2-fold increase in F-reticulocytes at 10mg after 14 days of once-daily dosing

    • Achieved maximal target engagement by day seven in 6mg and 10mg cohorts

    • FTX-6058 was generally well tolerated with no serious adverse events observed to date

    • Company plans to initiate enrollment in a Phase 1b clinical trial in sickle cell patients in 4Q 2021

    • Company plans to submit Investigational New Drug (IND) application in non-sickle cell hemoglobinopathies (e.g., beta-thalassemia) by year-end 2021

    • Company to review clinical results on second quarter earnings call today at 8:00am
    • Achieved dose proportional pharmacodynamic changes in HBG mRNA and F-reticulocytes in whole blood

      • Mean 4.5-fold induction in HBG mRNA at 10mg after 14 days of once-daily dosing
      • Mean 4.2-fold increase in F-reticulocytes at 10mg after 14 days of once-daily dosing



    • Achieved maximal target engagement by day seven in 6mg and 10mg cohorts



    • FTX-6058 was generally well tolerated with no serious adverse events observed to date



    • Company plans to initiate enrollment in a Phase 1b clinical trial in sickle cell patients in 4Q 2021



    • Company plans to submit Investigational New Drug (IND) application in non-sickle cell hemoglobinopathies (e.g., beta-thalassemia) by year-end 2021



    • Company to review clinical results on second quarter earnings call today at 8:00am ET



    CAMBRIDGE, Mass., Aug. 10, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced positive interim results from the ongoing single- and multiple-ascending dose (SAD and MAD) Phase 1 trial with FTX-6058 in healthy adult volunteers. FTX-6058 is an investigational, potent, and selective small molecule inhibitor of EED designed to increase the expression of fetal hemoglobin (HbF) with the potential to treat hemoglobinopathies, such as sickle cell disease and beta-thalassemia. Results from the MAD portion of the trial demonstrated proof of biology as evidenced by a dose proportional induction in HBG mRNA and accompanying increases in HbF-containing reticulocytes (F-reticulocytes). At 10mg, the highest dose studied to date, the mean changes were 4.5-fold and 4.2-fold, respectively. The increases in F-reticulocytes indicate that the HBG mRNA increases observed with FTX-6058 treatment are translating to HbF protein production.

    Additionally, all FTX-6058 doses in the MAD portion of the trial achieved maximal target engagement as evidenced by decreases in trimethylation at lysine 27 of histone H3 (H3K27me3), a downstream target of the polycomb repressive complex 2 (PRC2). These proof of mechanism results were achieved with once-daily, oral administration of FTX-6058 for 14 consecutive days, which was also generally well-tolerated in all SAD and MAD cohorts completed to date.

    "These results with FTX-6058 are very encouraging," said Julie Kanter, MD, co-director, Lifespan Comprehensive Sickle Cell Center at the University of Alabama at Birmingham. "Tremendous unmet need exists for many people with sickle cell disease and the availability of an effective, tolerable, oral, once-daily treatment option could represent a significant advancement."

    "We are very pleased with the interim results from this clinical trial of FTX-6058, which demonstrated compelling results across all primary, secondary, and exploratory endpoints included in this study," said Bryan Stuart, Fulcrum's president and chief executive officer. "We are excited to have been able to demonstrate proof of mechanism and proof of biology, as evidenced by maximal target engagement and increases in fetal hemoglobin parameters, including HBG mRNA and F-reticulocytes. These PD effects were further supported by predictable pharmacokinetics and being generally well tolerated."

    "Preclinically, we demonstrated consistent 2-3-fold induction of HBG mRNA and HbF protein both in vitro and in vivo," continued Mr. Stuart. "These clinical results reported today not only underscore the consistency observed preclinically, but also demonstrate the first evidence that FTX-6058 can achieve or exceed these preclinical thresholds predicted to provide meaningful clinical benefits to individuals with SCD. We look forward to moving this program forward into a trial in people living with sickle cell disease in the fourth quarter of 2021."

    FTX-6058 Phase 1 Healthy Volunteer Trial to Assess Safety, Tolerability, and Pharmacokinetics

    The Phase 1 randomized, double-blind, placebo-controlled trial is designed to evaluate the safety, tolerability, and pharmacokinetics of FTX-6058 (NCT04586985). In the SAD portion of the trial, healthy volunteers have received to date a single oral dose of either placebo or 2, 4, 10, 20, 30 or 40mg of FTX-6058. In the MAD portion of the trial, healthy volunteers have received to date a single oral dose of placebo or 2, 6, or 10 mg of FTX-6058 daily for 14 consecutive days. Safety assessments are performed regularly throughout the trial. The trial is also collecting secondary pharmacokinetic measurements, including bioavailability and half-life assessments. Exploratory measures were included to assess target engagement, HBG mRNA changes and F-reticulocyte changes. Target engagement of FTX-6058 was assessed as a change from baseline in H3K27me3/Total Histone H3 ratio in circulating monocytes. Pharmacodynamic parameters assessed include changes in HBG mRNA and F-reticulocytes. Subjects were seen seven to 10 days after the conclusion of study drug or placebo for a safety follow-up.

    Results from the MAD cohorts showed maximal target engagement as evidenced by 70% – 80% reduction in baseline H3K27me3 levels. The 10mg dose showed a mean 4.5-fold induction in HBG mRNA at day 14 and mean 4.2-fold increases in F-reticulocytes at the safety follow-up, indicating increased HbF protein expression. The kinetics observed across the target engagement and pharmacodynamic endpoints are consistent with the process of erythropoiesis in healthy individuals. These results demonstrated a time- and dose-dependent relationship between target engagement, mRNA induction and F-reticulocyte increases.

    HBG mRNA Mean Fold Induction for FTX-6058 versus Placebo

     2mg6mg10mg
     Mean Fold InductionP-valueMean Fold Induction P-valueMean Fold InductionP-value
    Day 71.560.18732.29 0.01792.340.0157
    Day 141.67 0.11203.28 0.00084.50<0.0001
    Safety Follow-up (Day 21-24)1.44 0.12143.25 <0.00013.74<0.0001

    F Reticulocyte Mean Fold Increase for FTX-6058 versus Placebo

     2mg6mg10mg
     Mean Fold IncreaseP-valueMean Fold IncreaseP-valueMean Fold IncreaseP-value
    Day 70.780.48361.560.21681.270.5032
    Day 140.740.47931.110.80861.980.1076
    Safety Follow-up (Day 21-24)0.630.12531.750.03204.23<0.0001



    There were no serious adverse events reported and no discontinuations. All treatment-emergent adverse events (TEAEs) deemed at least possibly related were mild (Grade 1 or 2) in both the SAD and MAD cohorts. There was one Grade 4 TEAE in the 10mg MAD cohort, which was determined to be unrelated to FTX-6058. No clinically significant changes in safety-related laboratory tests were reported during treatment periods for any of the FTX-6058 dose cohorts included in the analysis.

    Fulcrum anticipates presenting additional results from all Phase 1 dose cohorts at an upcoming medical meeting in the fourth quarter of 2021, pending abstract acceptance.

    Based on the results reported today, Fulcrum anticipates initiating enrollment in a clinical trial in sickle cell patients in the fourth quarter of 2021. The Phase 1b trial will be an open-label multi-dose trial starting at 6mg once-daily dosing and will include a treatment period of up to three months. This trial could provide the opportunity to demonstrate HbF protein induction in people living with sickle cell disease and will be used to help inform a potential Phase 2/3 trial. These results also support the initiation of a clinical trial in non-SCD hemoglobinopathies (e.g., beta-thalassemia) and Fulcrum plans to submit an IND by year-end 2021.

    Conference Call Information

    Fulcrum will host a conference call and webcast today at 8:00 am ET to discuss these results and the company's second quarter financial results. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 3291056

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 3291056

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary product engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum is conducting a Phase 1 trial with FTX-6058 in healthy adult volunteers.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of the Company's product candidates, initiation and enrollment of clinical trials and availability of clinical trial data, the timing of planned clinical trials and the Company's ability to submit an IND by year end. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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    • Primary endpoint, change in DUX4-driven gene expression which was included as an experimental biomarker, was not met
    • Losmapimod showed statistically significant* (p0.05) and clinically relevant benefit across multiple structural, functional and patient reported endpoints
      • Decreased Muscle Fat Infiltration
      • Improved Reachable Workspace
      • Improved Patient Global Impression of Change
    • Losmapimod was well tolerated with no treatment related discontinuations or treatment related serious adverse events
    • Positive benefit/risk supports losmapimod's potential to be a transformative therapy for the treatment of FSHD
    • Fulcrum expects to engage with health authorities, including the FDA, in H2 2021
    • Fulcrum to host conference call today at 8:00am ET; Full data
    • Primary endpoint, change in DUX4-driven gene expression which was included as an experimental biomarker, was not met
    • Losmapimod showed statistically significant* (p<0.05) and clinically relevant benefit across multiple structural, functional and patient reported endpoints
      • Decreased Muscle Fat Infiltration
      • Improved Reachable Workspace
      • Improved Patient Global Impression of Change
    • Losmapimod was well tolerated with no treatment related discontinuations or treatment related serious adverse events
    • Positive benefit/risk supports losmapimod's potential to be a transformative therapy for the treatment of FSHD
    • Fulcrum expects to engage with health authorities, including the FDA, in H2 2021
    • Fulcrum to host conference call today at 8:00am ET; Full data to be presented at FSHD International Research Congress today at 1:33pm ET

    CAMBRIDGE, Mass., June 24, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced results from the company's Phase 2b trial, ReDUX4, in people with facioscapulohumeral muscular dystrophy (FSHD). Results being presented with losmapimod at the FSHD International Research Congress today showed clinically relevant and statistically significant* benefits versus placebo on multiple measures of structural and functional FSHD disease progression and patient reported outcomes at 48 weeks. Losmapimod was generally well-tolerated, with no drug-related serious adverse events reported. Consistent with the previously reported interim analyses the primary endpoint was not met. Changes in DUX4-driven gene expression, which were included in the trial as an experimental biomarker endpoint, could not be demonstrated, the Company believes due to several technical and biologic variables with the endpoint. Based on today's results, the Company plans to meet with health authorities, including the U.S. Food and Drug Administration (FDA), in the second half of 2021 to determine the regulatory path for losmapimod in FSHD.

    "These results provide strong support that treatment with losmapimod has a meaningful clinical benefit in relevant measures of FSHD disease progression, despite the challenges of measuring DUX4," said Rabi Tawil, MD, ReDUX4 principal investigator and professor of neurology at University of Rochester Medical Center. "I am enthusiastic about the potential for losmapimod to offer meaningful improvements in preserving muscle function and patient quality of life."

    "People living with FSHD experience a progressive loss of function," said Fran Sverdrup, Ph.D., FSHD parent and FSHD researcher and associate professor of biochemistry and molecular biology at Saint Louis University. "The ReDUX4 trial with losmapimod is the first trial to demonstrate a positive benefit in several measures of FSHD, including slower disease progression and patient reported improvement over time. I am excited about today's findings and hopeful for the many patients who suffer from this devastating disease."

    *Nominally statistically significant means the p-value of a test is ≤ 0.05, calculated without regard to the test procedures in the trial's statistical analysis plan.

    FSHD is a serious, rare, progressive and disabling disease for which there are no approved treatments. FSHD is characterized by muscle degeneration and fat infiltration, initially affecting movement of the face and eventually the arms, trunk and legs. Disease progression results in accumulation of disability, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility. Impact on patients includes decreased ability to perform activities of daily living, maintain independence, and lost ability to function or work.

    "We are thrilled with the data reported from ReDUX4," said Bryan Stuart, Fulcrum's president and chief executive officer. "These results showing consistent reduction in fat infiltration and correlating benefit on multiple measures with losmapimod represent a major advance for the potential treatment of this devastating disease. Our broader molecular insights confirm the variability in DUX4 expression in skeletal muscle and can play an important role in guiding future research, while our structural and functional results show statistically significant* differences in several novel and established measures of disease progression between losmapimod and placebo at 48 weeks. I would like to thank the patients, caregivers and clinicians who participated in ReDUX4 for their important contributions and dedication."

    "These structural, functional, and patient-reported results observed in ReDUX4 are unprecedented in FSHD," continued Mr. Stuart.   "In particular, we are pleased by the fact that we observed these results within a 48-week time period. We plan to meet with health authorities, including the FDA, in the second half of the year to determine the regulatory path for losmapimod in FSHD."

    About ReDUX4

    ReDUX4 was an equally randomized, double-blind, placebo-controlled multicenter international Phase 2b clinical trial in 80 participants with FSHD designed to investigate the efficacy and safety of oral administration of losmapimod 15 mg twice per day. As a result of the COVID-19 pandemic, Fulcrum announced in May 2020 that the trial had been extended from 24 to 48 weeks to ensure the safety of participation during the pandemic. This extension also enabled the collection of safety and efficacy data over a longer time period. Over the course of the trial, there were three discontinuations, none of which were assessed to be related to study drug. Following the completion of the trial, 99% of eligible participants elected to continue in the Open Label Extension trial.

    The following data describe key results from the primary, secondary and exploratory endpoints showing clinically relevant and nominally statistically significant benefits versus placebo on multiple measures of structural and functional FSHD disease progression and patient reported outcomes at 48 weeks.

    DUX4-Driven Gene Expression

    The primary endpoint, change from baseline in DUX4-driven gene expression in affected skeletal muscle at Week 16 or Week 36, was not met. Reduction in DUX4-driven gene expression was included as an experimental biomarker endpoint because the Company believed it would correlate with downstream clinical improvements in patients with FSHD.

    Losmapimod reduced DUX4-driven gene expression in preclinical in vivo and in vitro experiments.  ReDUX4 was the first interventional clinical trial to test whether changes in intramuscular DUX4-driven gene expression could be assessed in patients with FSHD.  FSHD is a highly heterogenous disease, and DUX4 expression in each patient's muscle is heterogeneous and stochastic.  In the losmapimod treatment arm, repeat muscle needle biopsies at Week 16 or 36 did not demonstrate a difference in DUX4 activity, including from the prespecified subgroup analyses by DUX4-expressing quartiles. The ability to detect changes in DUX4-driven gene expression was confounded by significant variability across biopsies at baseline and upon repeat biopsy in both the placebo and losmapimod groups. The Company believes the sources of the variability include the stochastic nature of DUX4 expression in which biopsy samples showed a dynamic state of expression (over 1,000-fold variation), the scarcity of DUX4 positive myonuclei (~1/1000), as well as the relative imprecision in the needle biopsy procedure across multiple clinical trial sites.

    While a reduction in the molecular biomarker of DUX4-driven gene expression was not observed, the Company believes that benefits on muscle health, function and patient benefit observed in the clinical trial were associated with a reduction of DUX4-driven gene expression.  

    Muscle Fat Infiltration (MFI)

    • Losmapimod-treated participants showed decreased progression in the treatment efficacy composite measure of muscle fat infiltration as measured in intermediate muscles, those most likely to change (p=0.01*). Normal appearing muscles appeared to be preserved in the losmapimod group versus placebo based on a post hoc analysis.

    Muscle fat infiltration is a measure of diffuse fatty infiltration in lean muscle tissue that is correlated with disease severity in FSHD.   Participants in ReDUX4 trial were assessed with a quantitative whole body musculoskeletal magnetic resonance imaging (WB-MSK-MRI) which provides a holistic evaluation of skeletal musculature with the ability to volumetrically measure fat replacement of skeletal muscle in FSHD. Prior clinical trials have demonstrated that the amount of muscle fat replacement correlates with muscle function in many neuromuscular diseases, including FSHD. Furthermore, changes in fat content are correlated with changes in function. Taken together, ReDUX4 demonstrated that this MRI technology has sufficient sensitivity to detect FSHD relevant disease progression.  

    Reachable Workspace (RWS)

    • Treatment with losmapimod was shown to slow the rate of decline and improve accessible surface area in Reachable Workspace (RWS) measures (p<0.05*).

    RWS is a measure of upper extremity range of motion and function. Prior studies have shown that RWS correlates with changes in the ability of patients to independently perform activities of daily living. Based on published results, reachable workspace is an important measure of disability. The disease tends to progress from the upper body down, and loss of shoulder movement leads to loss of mobility. Participants in the losmapimod group showed improvements of up to 1.5% from baseline in the accessible surface area when using a 500g weight on their wrist compared to placebo. Participants in the placebo group were able to access 2 to 4% less total surface area (with and without 500g weights) measured by RWS after 48 weeks.

    Patient Global Impression of Change (PGIC)

    • Participants reported feeling better when treated with losmapimod compared to placebo through the Patient Global Impression of Change assessment (p=0.02*)

    PGIC, a measure of self-reported change in how a patient feels and functions, showed that participants were able to recognize improvements after 48 weeks of treatment. More participants in the losmapimod group reported improvement at 48 weeks compared to placebo. Four times more losmapimod participants reported improvement over time as compared to participants treated with placebo. Importantly, fewer losmapimod participants reported worsening as compared to placebo, and no losmapimod participants reported being "much worse" as compared to more than 13% of placebo participants, who reported that their disease had become "much worse."

    Additional Secondary and Exploratory Endpoints, Pharmacokinetics and Target Engagement

    Additional secondary and exploratory endpoints measuring disease progression and function demonstrated differences between losmapimod and placebo at week 48. In a post hoc analysis, dynamometry, which measures muscle strength, demonstrated that participants in the losmapimod group showed non-statistically significant trends of slower progression, as well as meaningful improvements (12-27%) in the strength of bilateral shoulder abductors and ankle dorsiflexors, two muscle groups particularly affected in FSHD, compared to placebo. Functional scales including RWS and TUG showed improvements in limb function consistent with dynamometry results. Two recently designed scales, (FSHD TUG, and FSHD Health Index) did not demonstrate changes from baseline in either group or differences between losmapimod and placebo groups, suggesting that these tests are not sensitive to change over the 48-week time period. Motor function measure also showed no changes in either group or differences between the groups over 48 weeks. There was no difference in muscle fat fraction or lean muscle volume between losmapimod and placebo groups at 48 weeks in intermediate muscles. The Company believes it may observe statistically significant differences in these measures with more time. Additional MRI data will be presented during the IRC meeting. Fulcrum will continue to analyze data from each endpoint to determine their viability for future trials. Blood concentrations and target engagement in muscle were consistent with previous studies and were within the expected range for clinical efficacy.

    Overall Safety and Tolerability

    Safety and tolerability data were consistent with previously reported results with no drug-related serious adverse events reported. Losmapimod was generally well-tolerated and the majority of treatment emergent adverse events were deemed unlikely related or not related to study drug by the investigator. There were three serious adverse events (post-op wound infection, alcohol poisoning and a suicide attempt) reported in two participants in the losmapimod group, each assessed as unrelated to losmapimod. There were no deaths or discontinuations due to adverse events. Losmapimod has now been evaluated in over 3,600 subjects in clinical trials across multiple indications, including FSHD.

    Fulcrum will present detailed results from the ReDUX4 trial during the FSHD International Research Congress today at 1:33pm ET.

    Conference Call Information

    Fulcrum will host a conference call and webcast today at 8:00 am ET to discuss the ReDUX4 data. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 3005948

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 3005948

    About FSHD

    FSHD is a serious, rare, progressive and disabling disease for which there are no approved treatments. FSHD is characterized by muscle degeneration and fat infiltration, initially affecting movement of the face and eventually the arms, trunk and legs. Disease progression results in accumulation of disability, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility. Impact on patients includes decreased ability to perform activities of daily living, maintain independence, and lost ability to function or work.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its proprietary product engine, FulcrumSeek, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Losmapimod has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of the Company's product candidates planned meetings with regulatory agencies and availability of clinical trial data. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  9. CAMBRIDGE, Mass., June 21, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced multiple presentations at the 28th Annual FSHD Society International Research Congress (IRC).

    June 24, 2021 IRC Virtual Presentations

    • 10:43 am ET - Use of snRNA-seq to characterize the skeletal muscle microenvironment during pathogenesis in FSHD
    • 1:33 pm ET - A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) with Open Label Extension (OLE): ReDUX4
    • 1:58 pm ET

    CAMBRIDGE, Mass., June 21, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced multiple presentations at the 28th Annual FSHD Society International Research Congress (IRC).

    June 24, 2021 IRC Virtual Presentations

    • 10:43 am ET - Use of snRNA-seq to characterize the skeletal muscle microenvironment during pathogenesis in FSHD
    • 1:33 pm ET - A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) with Open Label Extension (OLE): ReDUX4
    • 1:58 pm ET - Fulcrum Panel Q&A
    • Poster - Evaluating DUX4 Activity in a Phase 2, Randomized, Double-Blind, Placebo-Controlled,​ 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD
    • Poster - Quantitative Muscle Analysis in FSHD Using Whole-Body MRI: Composite Muscle Measurements for Cross-Sectional Analysis

    The sessions will be available to registered conference attendees. The posters and presentation will also be made available in the "Events & Presentations" section of fulcrumtx.com.

    Management will host a conference call on June 24, 2021 at 8:00 am ET to discuss the results of the ReDUX4 trial.

    Conference Call Details

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 3005948

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 3005948

    An audio webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    About FSHD

    FSHD is a serious, rare, progressive and disabling disease for which there are no approved treatments. FSHD is characterized by muscle degeneration and fat infiltration, initially affecting movement of the face and eventually the arms, trunk and legs. Disease progression results in accumulation of disability, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility. Impact on patients includes decreased ability to perform activities of daily living, maintain independence, and lost ability to function or work.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its proprietary product engine, FulcrumSeek, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Losmapimod has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine, FulcrumSeek, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  10. CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to losmapimod for the potential treatment of facioscapulohumeral muscular dystrophy (FSHD).

    "There are no approved therapies to treat patients with FSHD, and losmapimod is currently the only drug in clinical development for this serious and debilitating disease," said Judith Dunn, Ph.D., Fulcrum's president of research and development. "We are pleased that the FDA has granted Fast Track designation, which we believe demonstrates…

    CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to losmapimod for the potential treatment of facioscapulohumeral muscular dystrophy (FSHD).

    "There are no approved therapies to treat patients with FSHD, and losmapimod is currently the only drug in clinical development for this serious and debilitating disease," said Judith Dunn, Ph.D., Fulcrum's president of research and development. "We are pleased that the FDA has granted Fast Track designation, which we believe demonstrates the potential for losmapimod to address unmet medical needs for people living with FSHD."

    Fast Track Designation is intended to facilitate development and expedite the review of drugs that treat serious conditions so an approved product can reach the market expeditiously. It enables the company to have more frequent interactions with the FDA throughout the drug development process and allows for eligibility for priority review and accelerated approval in certain cases, as well as a rolling review.

    Fulcrum is on track to report full data from ReDUX4, a Phase 2b randomized, double-blind, placebo-controlled trial of losmapimod in FSHD patients, at the virtual FSHD International Research Congress taking place June 24-25, 2021. Data will include the primary endpoint, reduction from baseline of DUX4-driven gene expression, as well as a pre-specified sensitivity analysis assessing biopsies with the highest pre-treatment level of DUX4-driven gene expression. Additional data to be reported include secondary endpoints evaluating disease progression via skeletal muscle MRI, exploratory endpoints assessing muscle function measures and patient reported outcomes.

    Losmapimod previously received Orphan Drug Designation for FSHD.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,600 subjects in clinical trials across FSHD and multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of Fulcrum's product candidates and availability of clinical trial data. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  11. CAMBRIDGE, Mass., May 07, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will present a corporate overview at the BofA Securities 2021 Virtual Health Care Conference on Thursday, May 13, 2021 at 11:00 a.m. ET.

    A live audio webcast will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. An archived replay will be available on the Company's website for 90 days.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients…

    CAMBRIDGE, Mass., May 07, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will present a corporate overview at the BofA Securities 2021 Virtual Health Care Conference on Thursday, May 13, 2021 at 11:00 a.m. ET.

    A live audio webcast will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. An archived replay will be available on the Company's website for 90 days.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  12. On track to present data from Phase 2b ReDUX4 trial with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) at virtual FSHD International Research Congress in June 2021

    On track to report data from Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease in mid-2021

    Conference call scheduled for 8:00 a.m. ET today

    CAMBRIDGE, Mass., May 06, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the first quarter of 2021.

    "Fulcrum has continued to make great progress in advancing our…

    On track to present data from Phase 2b ReDUX4 trial with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) at virtual FSHD International Research Congress in June 2021

    On track to report data from Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease in mid-2021

    Conference call scheduled for 8:00 a.m. ET today

    CAMBRIDGE, Mass., May 06, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the first quarter of 2021.

    "Fulcrum has continued to make great progress in advancing our business and progressing our pipeline in the first quarter," said Bryan E. Stuart, president and chief executive officer. "We are on track to report data from ReDUX4 in FSHD later this quarter. Our Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease continues to move forward with the initiation of the multiple ascending dose cohort. Our team continues to optimize the potential of FulcrumSeek™, our proprietary product engine, driven by our focus on the patient and our ability to rapidly identify novel, high quality targets that modulate the root cause of genetically defined rare diseases. In addition, we have expanded our leadership team to include Dr. Judith Dunn, President of Research and Development, and today announced that Dr. Chris Morabito has been appointed Chief Medical Officer. With these important advances and cash runway that takes us into the fourth quarter of 2022, we believe we are well positioned to continue to build on our momentum to bring important therapies to patients with genetically defined rare diseases."

    Recent Business Highlights

    • On track to present data from ReDUX4, a Phase 2b trial of losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor, in FSHD at the virtual FSHD International Research Congress taking place June 24-25, 2021.
      • Data will include the primary endpoint, reduction from baseline of DUX4-driven gene expression, as well as a pre-specified sensitivity analysis assessing biopsies with the highest pre-treatment level of DUX4-driven gene expression. Additional data to be reported include secondary endpoints evaluating disease progression via skeletal muscle MRI, exploratory endpoints assessing muscle function measures and patient reported outcomes.
      • Continued evaluation of the Phase 2 Open Label Study.
    • On track to report data from the Phase 1 trial in healthy adult volunteers with FTX-6058 in development for sickle cell disease (SCD) in mid-2021, and to begin dosing patients with SCD in a clinical trial by year end.
      • FTX-6058, a highly potent small molecule EED inhibitor, is designed to induce expression of fetal hemoglobin (HbF) in red blood cells following oral administration to compensate for the mutated adult hemoglobin associated with hemoglobinopathies, including SCD and beta-thalassemia.
      • Preclinical data generated in CD34+ cells from healthy and SCD donors with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, indicating the potential to have a significant impact on patients with sickle cell disease.
      • US patent 10,973,805 issued, providing composition of matter coverage for FTX-6058 until 2040.
    • Multiple scientific meeting presentations
      • Presented new data related to the use of imaging biomarkers and clinical outcome assessments for FSHD at the virtual Muscular Dystrophy Association Clinical and Scientific Conference. Presentations included development of the whole-body musculoskeletal MRI (WB-MSK-MRI) protocol. Data showed that WB-MSK-MRI can capture heterogeneity and provide important information about disease severity. Meaningful composite MRI measurements demonstrated correlation with relevant FSHD clinical endpoints.
      • Presented the medicinal chemistry strategy for FTX-6058 at the First Time Disclosure Session at the American Chemical Society (ACS) Spring 2021 National Meeting. The presentation included initial pharmacokinetic data from the single ascending dose cohort of the Phase 1 trial in healthy adult volunteers.
    • Advanced FulcrumSeek discovery efforts and strategic collaborations with Acceleron and MyoKardia, a wholly owned subsidiary of Bristol-Myers Squibb Company.
    • Senior management updates:
      • CSO transition: On January 19, 2021, Fulcrum announced that Christopher Moxham, Ph.D. was promoted to chief scientific officer and Owen Wallace, Ph.D. stepped down from his role as chief scientific officer, each effective February 5, 2021. Dr. Wallace was appointed to Fulcrum's Scientific Advisory Board.
      • CEO transition: On March 4, 2021, Fulcrum announced that Bryan E. Stuart was promoted to president and chief executive officer and was appointed to Fulcrum's Board of Directors following the retirement of Robert J. Gould, Ph.D., each effective March 31, 2021. Dr. Gould continues to serve on Fulcrum's Board and was appointed to the Scientific & Technology committee.
      • President R&D: On March 24, 2021, Fulcrum announced that Judith A. Dunn, Ph.D. was appointed President of Research and Development, effective April 1, 2021.
      • Chief Medical Officer: On May 6, 2021, Fulcrum announced that Christopher J. Morabito, M.D. has been appointed Chief Medical Officer, effective May 10, 2021.

    First Quarter 2021 Financial Results

    • Cash Position: As of March 31, 2021, cash, cash equivalents, and marketable securities were $143.9 million, as compared to $112.9 million as of December 31, 2020. Based on current plans, the company expects that its existing cash, cash equivalents, and marketable securities will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the fourth quarter of 2022.
    • Collaboration Revenues: Collaboration revenue was $4.8 million for the first quarter of 2021, as compared to $0.8 million for the first quarter of 2020. The increase in collaboration revenue was due to the execution of the company's collaboration and license agreement with MyoKardia in July 2020, as well as due to an increase in collaboration revenue associated with the company's collaboration and license agreement with Acceleron.
    • R&D Expenses: Research and development expenses were $16.3 million for the first quarter of 2021, as compared to $14.5 million for the first quarter of 2020. The increase of $1.8 million was primarily due to increased costs to support the company's ongoing and planned clinical trials.
    • G&A Expenses: General and administrative expenses were $5.5 million for the first quarter of 2021, as compared to $5.1 million for the first quarter of 2020. The increase of $0.4 million was primarily due to increased employee-compensation costs to support the growth of the organization, including increased stock-based compensation expense.
    • Net Loss: Net loss was $17.0 million for the first quarter of 2021, as compared to a net loss of $18.5 million for the first quarter of 2020.

    Conference Call and Webcast

    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the Company's first quarter 2021 recent business highlights and financial results. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 5767008

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 5767008

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Losmapimod has been evaluated in more than 3,600 subjects in clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary product engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum is conducting a Phase 1 trial with FTX-6058 in healthy adult volunteers.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of Fulcrum's product candidates, initiation and enrollment of clinical trials and availability of clinical trial data, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.



    Fulcrum Therapeutics, Inc.

    Selected Consolidated Balance Sheet Data

    (In thousands)

    (Unaudited)

     March 31,

    2021
      December 31,

    2020
     
    Cash, cash equivalents, and marketable securities$143,856  $112,914 
    Working capital(1) 124,356   92,785 
    Total assets 156,476   129,577 
    Total stockholders' equity 127,754   95,181 

    (1)    We define working capital as current assets minus current liabilities.



    Fulcrum Therapeutics, Inc.

    Consolidated Statements of Operations

    (In thousands, except per share data)

    (Unaudited)

     Three Months Ended

    March 31,
     
     2021  2020 
    Collaboration revenue$4,789  $750 
    Operating expenses:       
    Research and development 16,334   14,482 
    General and administrative 5,498   5,064 
    Total operating expenses 21,832   19,546 
    Loss from operations (17,043)  (18,796)
    Other income, net 44   344 
    Net loss$(16,999) $(18,452)
    Net loss per share, basic and diluted$(0.54) $(0.81)
    Weighted-average common shares outstanding, basic and diluted 31,510   22,719 



    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



    Primary Logo

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  13. CAMBRIDGE, Mass., May 06, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced Christopher J. Morabito, M.D. has been appointed Chief Medical Officer, effective May 10, 2021. Dr. Morabito will oversee the development of Fulcrum's lead programs in facioscapulohumeral dystrophy (FSHD) and select hemoglobinopathies, including sickle cell disease. He brings more than two decades of experience as a physician-scientist, treating patients, driving key research, and advancing clinical programs across a wide range of disease areas.

    "Chris brings to Fulcrum an impressive range of industry and operational…

    CAMBRIDGE, Mass., May 06, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced Christopher J. Morabito, M.D. has been appointed Chief Medical Officer, effective May 10, 2021. Dr. Morabito will oversee the development of Fulcrum's lead programs in facioscapulohumeral dystrophy (FSHD) and select hemoglobinopathies, including sickle cell disease. He brings more than two decades of experience as a physician-scientist, treating patients, driving key research, and advancing clinical programs across a wide range of disease areas.

    "Chris brings to Fulcrum an impressive range of industry and operational experience that will provide critical support for advancing our current and future clinical programs," said Bryan E. Stuart, president and chief executive officer. "His clinical vision and proven leadership, as well as his extensive medical and research experience will be instrumental as we work toward our goal of treating rare diseases at their root cause."

    "It's an exciting time to be joining Fulcrum, and I'm looking forward to contributing to the FSHD and select hemoglobinopathy trials as well as future clinical programs," said Dr. Morabito. "Fulcrum is increasingly being recognized for its scientifically rigorous and patient-centric approach to drug development. I am very much looking forward to guiding the clinical development of Fulcrum's pipeline as we advance promising new treatments for rare diseases."

    Dr. Morabito has extensive leadership experience at biotechnology and pharmaceutical companies, advancing drug discovery and clinical programs, including for numerous Phase 1, proof-of-concept and pivotal studies. Most recently, he served as Chief Medical Officer at Cardurion Pharmaceuticals where he oversaw all drug development activities. Prior to Cardurion, he served in several roles at Takeda, including program leadership, pipeline strategy, most recently serving as Senior Vice President and Head of Research and Development for plasma-derived protein therapies. In this role, he established a complete R&D organization and built a pipeline of medicines for patients with rare and complex, difficult-to-manage diseases. Before Takeda, he led a number of programs at Sanofi, from early development stage through regulatory submission. He began his career in industry at Merck & Co, leading development of multiple cardiovascular assets.

    Before joining industry, Dr. Morabito was the Chief of the Division of Neonatology at Lehigh Valley Hospital and Health Network. He trained initially as a neonatologist at the University of California, San Francisco, where he focused on neonatal cardiology and was an NIH-funded scientist interrogating mechanisms of cardiomyocyte development. He earned his B.A. in Biology from Franklin and Marshall College, and his M.D. from Penn State University College of Medicine.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



    Primary Logo

    View Full Article Hide Full Article
  14. CAMBRIDGE, Mass., April 29, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its first quarter 2021 financial results will be released on Thursday, May 6, 2021 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 5767008

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 5767008

    An audio…

    CAMBRIDGE, Mass., April 29, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its first quarter 2021 financial results will be released on Thursday, May 6, 2021 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 5767008

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 5767008

    An audio webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  15. CAMBRIDGE, Mass., April 09, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today presented the medicinal chemistry strategy for FTX-6058 at the First Time Disclosure Session at the American Chemical Society (ACS) Spring 2021 National Meeting. FTX-6058 is a highly potent orally bioavailable small molecule EED inhibitor for the potential treatment of select hemoglobinopathies, including sickle cell disease and β-thalassemia. The validation of EED as a fetal hemoglobin (HbF) inducer target for sickle cell disease was conducted using FulcrumSeek, Fulcrum's proprietary product engine.

    "We are pleased…

    CAMBRIDGE, Mass., April 09, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today presented the medicinal chemistry strategy for FTX-6058 at the First Time Disclosure Session at the American Chemical Society (ACS) Spring 2021 National Meeting. FTX-6058 is a highly potent orally bioavailable small molecule EED inhibitor for the potential treatment of select hemoglobinopathies, including sickle cell disease and β-thalassemia. The validation of EED as a fetal hemoglobin (HbF) inducer target for sickle cell disease was conducted using FulcrumSeek, Fulcrum's proprietary product engine.

    "We are pleased to report progress on our development of FTX-6058 including the first publication of the structure of this compelling EED inhibitor," said Chris Moxham, Ph.D., Fulcrum's chief scientific officer. "We believe that this oral, once-a-day therapy with an impressive preclinical pharmacological profile has the potential to provide a meaningful therapeutic benefit to patients with sickle cell disease and β-thalassemia. We are also excited to report initial PK results from the SAD cohort and that our Phase 1 trial in healthy volunteers continues to progress with initiation of the multiple ascending dose cohorts. We expect to report the full data from this Phase 1 trial mid-year."

    FTX-6058 inhibits PRC2 via binding to EED, which induces robust HbF protein expression in both cell and murine models. Increasing HbF has the potential to prevent or reduce disease-related pathophysiology and reduce the risk of recurring events such as vaso-occlusive crises and hemolysis. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, demonstrating the potential to have a significant impact in patients with sickle cell disease. Human genetic data further indicate that individuals with the sickle cell mutation and high HbF levels may have asymptomatic disease, underscoring the protective effect of increased HbF.

    Fulcrum's Phase 1 trial in healthy volunteers is evaluating the safety, tolerability and pharmacokinetics of FTX-6058. Dosing has been initiated in the randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) cohorts of the trial. Dosing continues in the single ascending dose (SAD) portion. The company anticipates sharing data from this Phase 1 trial in mid-2021 and initiating a clinical trial in sickle cell patients by the end of 2021.

    Today's presentation, titled "Discovery of clinical candidate FTX-6058: a potent, orally bioavailable upregulator of fetal hemoglobin for treatment of sickle cell disease", will be available in the "Publications" section of fulcrumtx.com.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary product engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy adult volunteers.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of Fulcrum's product candidates, initiation and enrollment of clinical trials and availability of clinical trial data, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  16. CAMBRIDGE, Mass., March 24, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that Judith A. Dunn, Ph.D. has been appointed President of Research and Development. Dr. Dunn has held multiple leadership roles in global research and drug development spanning from discovery through commercialization. Most recently, she supported the development of new companies targeting areas of unmet need in neuroscience as an Entrepreneur in Residence at Atlas Venture.

    "We are honored to have Dr. Dunn join Fulcrum to expand our R&D leadership team, particularly as we move toward significant advancements…

    CAMBRIDGE, Mass., March 24, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that Judith A. Dunn, Ph.D. has been appointed President of Research and Development. Dr. Dunn has held multiple leadership roles in global research and drug development spanning from discovery through commercialization. Most recently, she supported the development of new companies targeting areas of unmet need in neuroscience as an Entrepreneur in Residence at Atlas Venture.

    "We are honored to have Dr. Dunn join Fulcrum to expand our R&D leadership team, particularly as we move toward significant advancements with our losmapimod and FTX-6058 development programs," said Bryan E. Stuart, Fulcrum's incoming president and chief executive officer. "Our product engine and industry collaborations position us to advance many new opportunities in discovery and drug development in the months and years ahead. Judy's outstanding experience with over 25 years in drug discovery and clinical development will be an important resource for us as we work to advance these programs."

    "I am thrilled to take on this role at Fulcrum," said Dr. Dunn. "I was very impressed with the productivity and potential of Fulcrum's product engine, which has already generated two important programs for FSHD and select hemoglobinopathies, including sickle cell disease. I'm also excited for the advances in research and development, including bringing forward two INDs over the next 24 months. FulcrumSeek's ability to generate and integrate functional, morphological and transcriptional data with great precision enables rapid identification of novel targets across a wide variety of diseases. I look forward to working with the team to continue to advance programs to treat the root causes of genetically defined rare diseases."

    Prior to Atlas, Dr. Dunn served as Vice President of Clinical Development at Roche and was previously head of the Roche Innovation Center in New York City. She also supported the establishment of the Empire Discovery Institute, a New York State initiative to support progression of early stage research to clinical application. She has additionally held positions in both the research and commercial divisions of Pfizer and led clinical programs in psychiatry at Sunovion (Sepracor). Dr. Dunn received a postdoctoral fellowship in new drug development from Pfizer, a training fellowship in neuropharmacology from the Center for Brain Research, a Doctor of Philosophy, Developmental Neurobiology from Wesleyan University and a Bachelor of Science, Neurobiology from the University of Rochester.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  17. – Demonstrated Whole-Body MRI captures heterogeneity and provides key disease severity and progression information correlated with FSHD clinical endpoints

    – Demonstrated potential of FSHD-TUG and Emerald in-home assessments as accurate, low-burden clinical assessments of mobility for FSHD patients

    – Company on track to report data from Phase 2b ReDUX4 trial with losmapimod in FSHD in late-2Q 2021 –

    CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the presentation of new data related to the use of imaging biomarkers and clinical outcome…

    – Demonstrated Whole-Body MRI captures heterogeneity and provides key disease severity and progression information correlated with FSHD clinical endpoints

    – Demonstrated potential of FSHD-TUG and Emerald in-home assessments as accurate, low-burden clinical assessments of mobility for FSHD patients

    – Company on track to report data from Phase 2b ReDUX4 trial with losmapimod in FSHD in late-2Q 2021 –

    CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the presentation of new data related to the use of imaging biomarkers and clinical outcome assessments for facioscapulohumeral muscular dystrophy (FSHD) at the 2021 Muscular Dystrophy Association (MDA) virtual Clinical and Scientific Conference. Presentations included evaluation of disease severity and progression with whole body musculoskeletal magnetic resonance imaging (WB-MSK-MRI), FSHD-TUG, a modified Timed Up and Go (TUG) assessment for FSHD patients, and in-home passive measurements of mobility and sleep. The presentation and posters can be found on Fulcrum's website at https://www.fulcrumtx.com/pipeline/#publications.

    "There is a critical need for accurate, low patient burden assessments that can effectively track disease severity and progression, and correlate with clinical outcomes in FSHD," said Michelle Mellion, M.D., Fulcrum's senior medical director. "The Whole Body-MSK MRI can capture the heterogeneity and provide important information about disease severity as it correlates with FSHD relevant clinical endpoints. This protocol is currently being used in our Phase 2 clinical trials of losmapimod. WB-MSK-MRI also may enable an individualized assessment of disease progression, offering a more efficient screening of potential therapies and better facilitate decisions in the development of new treatments. Additionally, Emerald and FSHD-TUG help capture key metrics in FSHD patients."

    Fulcrum and AMRA Medical have developed a quantitative WB-MSK-MRI protocol and analysis algorithms to volumetrically measure fat replacement of skeletal muscle in FSHD to use in multi-site clinical trials. WB-MSK-MRI is non-invasive and captures a holistic evaluation of the skeletal musculature, identifying small quantitative changes in muscle health that correlate with functional measures in FSHD patients and enable an assessment of disease heterogeneity. In the study being presented, the protocol was performed and standardized at six sites where patients were screened, biopsies were taken between 1-4 weeks and 5-12 weeks, and a final MRI scan was conducted between weeks 5-12.

    WB-MSK-MRI was shown to capture heterogeneity and provide important information about disease severity and progression in 17 patients. Of 618 muscles, 478 were analyzed. Good reproducibility was found across all muscles, with higher reproducibility in larger muscles. Results also showed strong cross-sectional correlation between Regional Composite Measurement, TUG, FSHD-TUG and Reachable Work Space (RWS) assessments.

    Fulcrum has also identified FSHD-TUG as a potential clinical outcome assessment of mobility in FSHD patients. In a separate study presented at the MDA meeting, FSHD-TUG demonstrated a correlation with clinical severity and patient reported physical function and lower extremity function. Existing assessments largely focus on walking parameters as a test of function, but most FSHD patients report difficulty getting up from lying down position. The FSHD-TUG was optimized to also include evaluation of sit to supine (laying on back) and the reverse.

    The study was conducted to determine the reliability and validity of TUG, a traditional measure of mobility, and FSHD-TUG, over a one-year period. Twenty-two FSHD patients and twenty healthy volunteers were enrolled. Patients were screened and stratified into groups, and each group performed two trials of the classic TUG and FSHD TUG on two separate visits one week apart. A total of four trials over two separate visits were recorded for each participant. On average, FSHD subjects took approximately twice the time to complete TUG, FSHD-TUG, and components of the FSHD-TUG compared to healthy volunteers. These results support the reliability and validity of FSHD-TUG as a potential clinical assessment of mobility for ongoing and future clinical trials.

    Study results also highlight the use of in-home monitoring from Emerald, a contactless radio-wave-based home monitoring system, to enable a large number of passively derived measurements of clinical progression including gait speed, time in bed, sleep and vital signs. Ten FSHD patients were observed in their homes for three months. In addition to in-home gait speed, novel metrics including assessments of sleep schedule variability and eTUG (the time from motion initiation within the bed to moving two meters away from the bed edge) were derived. Emerald's in-home measurements were strongly correlated with clinical metrics. As the number of measurements increased, Emerald's metrics became increasingly sensitive and were shown to detect smaller fluctuations in disease progression.

    "As we advance our clinical development program for losmapimod for the treatment of FSHD, we are also continually working to improve our ability to assess disease progression based on the most clear and effective outcome measures," said Chris Moxham, Ph.D., Fulcrum's chief scientific officer. "Assessments based on musculoskeletal MRI and clinical outcomes may be key to demonstrating patient benefit in this population. We expect full data from our Phase 2b ReDux4 trial late in the second quarter of this year, which will provide additional insights to inform the path forward for losmapimod in the treatment of FSHD."

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of Fulcrum's product candidates, initiation and enrollment of clinical trials and availability of clinical trial data, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  18. – Company on track to present data from Phase 2b ReDUX4 trial with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) in late-2Q 2021 –

    – On track to report results from Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease in mid-2021 –

    – Company to discontinue Phase 3 COVID-19 trial (LOSVID) –

    Extended cash runway into 4Q 2022; raised $50.6 million in gross proceeds from January 2021 public offering

    Conference call scheduled for 8:00 a.m. ET today

    CAMBRIDGE, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business…

    – Company on track to present data from Phase 2b ReDUX4 trial with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) in late-2Q 2021 –

    – On track to report results from Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease in mid-2021 –

    – Company to discontinue Phase 3 COVID-19 trial (LOSVID) –

    Extended cash runway into 4Q 2022; raised $50.6 million in gross proceeds from January 2021 public offering

    Conference call scheduled for 8:00 a.m. ET today

    CAMBRIDGE, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the fourth quarter and full year of 2020.

    "In 2020, we made meaningful progress in advancing our pipeline despite the extraordinary challenges brought on by COVID-19," said Robert J. Gould, Ph.D., president and chief executive officer. "We have laid the foundation to achieve several key milestones in 2021, including a comprehensive assessment of our Phase 2 ReDUX4 trial with losmapimod in facioscapulohumeral muscular dystrophy late in the second quarter and completing our Phase 1 trial in healthy adult volunteers with FTX-6058, a highly potent small molecule EED inhibitor in development for the treatment of select hemoglobinopathies, including sickle cell disease and beta-thalassemia. Additionally, after careful consideration and a strategic review of the COVID-19 landscape, we are discontinuing our LOSVID trial. This enables us to focus on rare diseases. I would like to thank the patients and investigators who participated in this trial and the Fulcrum team who worked tirelessly to rapidly design and launch the LOSVID trial during a global pandemic."

    "Furthermore, we have made great progress with our next-generation product engine including new levels of validation in our internal research efforts and externally through our strategic collaborations," continued Dr. Gould. "With the additional capital from our recent public offering, we have extended our cash runway into the fourth quarter of 2022 and we believe that we are well positioned to continue progress on our goal to advance therapies to improve the lives of patients with genetically defined rare diseases."

    Recent Business Highlights

    • On track to report data from ReDUX4, a Phase 2b trial of losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor, in FSHD late in the second quarter of 2021.
      • Data will include the primary endpoint, reduction from baseline of DUX4-driven gene expression, as well as a pre-specified sensitivity analysis assessing biopsies with the highest pre-treatment level of DUX4-driven gene expression. Additionally, secondary endpoints, including skeletal muscle MRI and exploratory endpoints, including clinical outcome assessments and patient reported outcomes will also be reported.
      • Continued evaluation of the Phase 2 Open Label Study.
    • On track to report results from the Phase 1 trial in healthy adult volunteers with FTX-6058 in development for sickle cell disease (SCD) in mid-2021, and to begin dosing patients with SCD by year end.
      • FTX-6058, a highly potent small molecule EED inhibitor, is designed to induce expression of fetal hemoglobin (HbF) in red blood cells following oral administration to compensate for the mutated adult hemoglobin associated with hemoglobinopathies, including sickle cell disease and beta-thalassemia.
      • Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, indicating the potential to have a significant impact on patients with sickle cell disease.
    • After careful consideration, Fulcrum is discontinuing LOSVID, a Phase 3 trial with losmapimod for hospitalized subjects with COVID-19, due to significant changes in the COVID-19 treatment paradigm, including new therapeutic options and emerging vaccines.
      • The company has decided to redeploy its resources to other clinical programs and discovery efforts, with a continued focus on rare diseases.
      • Losmapimod was generally well tolerated in LOSVID, and an independent data safety monitoring board did not identify any safety concerns related to losmapimod.
    • Multiple scientific meeting presentations
      • Presentation on FulcrumSeek, the company's next generation product engine approach, at the Society for Laboratory Automation and Screening annual meeting in January 2021.
      • Presented multiple posters supporting the potential of FTX-6058 in sickle cell disease at the 62nd American Society of Hematology (ASH) annual meeting, December 5-8, 2020.
      • Presented target engagement and good tolerability with FTX-6058 in multiple preclinical rodent models with once-a-day oral dosing at the 14th Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting, September 25, 2020.
      • Presented multiple posters supporting Fulcrum's integrated approach to the evaluation of FSHD patients during the 25th International Congress of the World Muscle Society (WMS), October 1, 2020.
    • Advanced FulcrumSeek discovery efforts and strategic collaborations with Acceleron and MyoKardia, a wholly owned subsidiary of Bristol-Myers Squibb Company.
    • Raised gross proceeds of $50.6 million from a public offering in January 2021.
      • The underwritten public offering of 4,600,000 shares of the company's common stock at a public offering price of $11.00 per share included 600,000 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares at the public offering price.
    • Key management updates
      • CEO transition: On March 4, 2021, Fulcrum announced that Bryan E. Stuart will be promoted to president and chief executive officer and Robert J. Gould, Ph.D. will retire from his role as president and chief executive officer, each effective March 31, 2021. Mr. Stuart has also been appointed to Fulcrum's Board of Directors. Dr. Gould will continue to serve on Fulcrum's Board and has also been appointed to the Scientific & Technology committee of the Board. Additionally, Mark Levin, Fulcrum's Board chair, will assume the role of executive chair effective upon Dr. Gould's retirement.
      • CSO transition: On January 19, 2021, Fulcrum announced that Chris Moxham, Ph.D. was promoted to chief scientific officer and Owen Wallace, Ph.D. stepped down from his role as chief scientific officer, each effective February 5, 2021. Dr. Wallace has been appointed to Fulcrum's Scientific Advisory Board.

    Fourth Quarter and Full Year 2020 Financial Results

    • Cash Position: As of December 31, 2020, cash, cash equivalents, and marketable securities were $112.9 million, as compared to $96.7 million as of December 31, 2019. Based on current plans, the company expects that its cash, cash equivalents, and marketable securities as of December 31, 2020, together with the net proceeds of $46.4 million from the sale of its common stock in a public offering on January 22, 2021, will be sufficient to enable Fulcrum to fund operating expenses and capital expenditure requirements into the fourth quarter of 2022.
    • Collaboration Revenue: Collaboration revenue was $4.2 million for the fourth quarter of 2020, as compared to no revenue recognized during the fourth quarter of 2019. The increase in collaboration revenue was due to the execution of the company's collaboration and license agreements with Acceleron and MyoKardia in December 2019 and July 2020, respectively.



      Collaboration revenue was $8.8 million for the year ended December 31, 2020, as compared to no revenue recognized during the year ended December 31, 2019. The increase in collaboration revenue was due to the execution of the collaboration and license agreements with Acceleron and MyoKardia in December 2019 and July 2020, respectively.
    • R&D Expenses: Research and development expenses were $16.1 million for the fourth quarter of 2020, as compared to $12.1 million for the fourth quarter of 2019. The increase of $4.0 million was primarily due to increased costs to support the company's ongoing and planned clinical trials and increased personnel-related costs to support the growth of Fulcrum's research and development organization, including increased stock-based compensation expense.



      Research and development expenses were $59.0 million for the year ended December 31, 2020, as compared to $71.1 million for the year ended December 31, 2019. Research and development expenses for the year ended December 31, 2019 include $25.6 million of one-time costs incurred associated with the issuance of Series B convertible preferred stock under the company's license agreement with GSK for losmapimod and $2.5 million of one-time costs incurred associated with the achievement of a milestone under the company's license agreement with GSK for losmapimod. Excluding these one-time costs, the increase of $16.0 million was primarily due to increased costs to support the company's ongoing and planned clinical trials and increased personnel-related costs to support the growth of Fulcrum's research and development organization, including increased stock-based compensation expense.
    • G&A Expenses: General and administrative expenses were $5.9 million for the fourth quarter of 2020, as compared to $4.4 million for the fourth quarter of 2019. The increase of $1.5 million was primarily due to increased costs associated with operating as a public company and increased personnel-related costs to support the growth of the organization, including increased stock-based compensation expense.



      General and administrative expenses were $21.4 million for the year ended December 31, 2020, as compared to $13.1 million for the year ended December 31, 2019. The increase of $8.3 million was primarily due to increased costs associated with operating as a public company and increased personnel-related costs to support the growth of the organization, including increased stock-based compensation expense.
    • Net Loss: Net loss was $17.7 million for the fourth quarter of 2020, as compared to a net loss of $16.1 million for the fourth quarter of 2019.



      Net loss was $70.8 million for the year ended December 31, 2020, as compared to $82.7 million for the year ended December 31, 2019.

    Conference Call and Webcast

    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the company's fourth quarter and full year 2020 financial results and recent business highlights. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 6376419

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 6376419

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Losmapimod has been evaluated in more than 3,600 subjects in clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary product engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy adult volunteers.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of Fulcrum's product candidates, initiation and enrollment of clinical trials and availability of clinical trial data, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.





    Fulcrum Therapeutics, Inc.

    Selected Consolidated Balance Sheet Data

    (In thousands)

    (Unaudited)

     December 31,

    2020
      December 31,

    2019
     
    Cash, cash equivalents, and marketable securities$112,914  $96,713 
    Working capital(1) 92,785   87,943 
    Total assets 129,577   110,439 
    Total stockholders' equity 95,181   87,153 



    (1) We define working capital as current assets minus current liabilities.





    Fulcrum Therapeutics, Inc.

    Consolidated Statements of Operations

    (In thousands, except per share data)

    (Unaudited)

     Three Months Ended

    December 31,
      Year Ended

    December 31,
     
     2020  2019  2020  2019 
    Collaboration revenue$4,225  $  $8,823  $ 
    Operating expenses:               
    Research and development 16,145   12,087   59,042   71,072 
    General and administrative 5,867   4,403   21,392   13,145 
    Total operating expenses 22,012   16,490   80,434   84,217 
    Loss from operations (17,787)  (16,490)  (71,611)  (84,217)
    Other income, net 67   367   792   1,540 
    Net loss$(17,720) $(16,123) $(70,819) $(82,677)
    Cumulative convertible preferred stock dividends          (7,128)
    Net loss attributable to common stockholders$(17,720) $(16,123) $(70,819) $(89,805)
    Net loss per share attributable to common stockholders, basic and diluted$(0.64) $(0.71) $(2.79) $(8.13)
    Weighted average number of common shares used in net loss per share attributable to common stockholders, basic and diluted 27,537   22,610   25,354   11,046 





    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  19. Robert J. Gould, Ph.D. announces retirement; will continue to serve as board member and advisor

    Bryan E. Stuart promoted to president and chief executive officer effective March 31, 2021

    CAMBRIDGE, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that Bryan E. Stuart, the company's chief operating officer, will become Fulcrum's president and chief executive officer and will be appointed to the Board of Directors. Mr. Stuart will succeed current president and chief executive officer Robert J. Gould, Ph.D. who will be retiring as of March 31, 2021. Dr. Gould…

    Robert J. Gould, Ph.D. announces retirement; will continue to serve as board member and advisor

    Bryan E. Stuart promoted to president and chief executive officer effective March 31, 2021

    CAMBRIDGE, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that Bryan E. Stuart, the company's chief operating officer, will become Fulcrum's president and chief executive officer and will be appointed to the Board of Directors. Mr. Stuart will succeed current president and chief executive officer Robert J. Gould, Ph.D. who will be retiring as of March 31, 2021. Dr. Gould will remain as a member of the Board of Directors and will serve as an advisor to the company. Additionally, Mark Levin, Fulcrum's Board chair, will assume the role of executive chair effective upon Dr. Gould's retirement.

    "On behalf of the entire Board, I want to thank Robert for his leadership and dedication to Fulcrum," said Mark Levin. "Robert joined us as CEO in 2016 and was instrumental in ensuring Fulcrum's successful launch. His decades of experience and steadfast dedication have positioned Fulcrum to make significant and rapid progress in its goal to create therapies for genetically defined rare diseases. We wish Robert the best in his retirement and are grateful that he will continue to be a member of our board and serve as an advisor."

    "Bryan is the ideal candidate to lead Fulcrum through its next critical stage of growth," continued Mr. Levin. "He is a trusted and strategic leader and a natural successor to Robert. Bryan brings over 20 years of industry experience, predominantly in the rare disease space. With his proven track record of helping to successfully advance numerous rare disease programs from the clinic to commercialization, I'm confident he will bring significant value to Fulcrum in his new role."

    "I am honored to become Fulcrum's next CEO and to lead our exceptional team, especially at such an exciting time for the company," said Bryan Stuart. "Along with a strong bench of leadership talent and support from the Board, I am confident in our ability to continue to advance our clinical programs, external collaborations and Product Engine to develop and deliver therapies to people living with rare genetic diseases. We have a lot of important work ahead of us as we expect to report meaningful updates from our clinical programs in FSHD and sickle cell disease later this year."

    Bryan Stuart joined Fulcrum as chief operating officer in 2018. Prior to Fulcrum he served as president and chief executive officer of Yarra Therapeutics and president and chief executive officer of Kastle Therapeutics, both companies focused on developing therapies for rare diseases. Prior to Kastle, Bryan was chief business officer of Civitas Therapeutics (acquired by Acorda) and previously led business development, corporate development, and strategy at both EKR Therapeutics (acquired by Chiesi) and Ovation Pharmaceuticals (acquired by Lundbeck A/S). Bryan earned his MBA from the Kellogg School at Northwestern University and his bachelor's degree from the University of Illinois.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  20. CAMBRIDGE, Mass., Feb. 26, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the company's recognition of Rare Disease Day 2021.

    "While COVID-19 has had an impact on most people around the world, it has put a new spotlight on the importance of connectivity and community for people affected by rare diseases who are at a higher risk of feelings of isolation and can face significant challenges in accessing information and healthcare services," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "We have been privileged to work with many patients and caregivers…

    CAMBRIDGE, Mass., Feb. 26, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the company's recognition of Rare Disease Day 2021.

    "While COVID-19 has had an impact on most people around the world, it has put a new spotlight on the importance of connectivity and community for people affected by rare diseases who are at a higher risk of feelings of isolation and can face significant challenges in accessing information and healthcare services," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "We have been privileged to work with many patients and caregivers affected by genetically defined diseases including facioscapulohumeral muscular dystrophy and sickle cell disease. They have bravely shared their stories in efforts to help others better understand these diseases and access information and support that can help."

    Rare Disease Day (www.rarediseaseday.org) was established by EURODIS in 2008 and is held on the last day of February each year in an effort to build awareness of rare diseases and the impact they have on patients and their families. The theme for Rare Disease Day 2021 highlights the need to provide members of the rare disease community around the world with new opportunities to meet, share insights and support and join together in efforts in education and advocacy. This year will mark the first all-digital Rare Disease Day involving interactive online events planned by hundreds of international advocacy, research and patient care organizations.

    During February, Fulcrum Therapeutics supported a range of efforts to help build broader awareness of facioscapulohumeral muscular dystrophy (FSHD) and sickle cell disease (SCD) by presenting information from patients and caregivers about the challenges they face. The company will also invite employees to join a virtual meeting and Q&A session where members of the FSHD and SCD communities will talk about how they have addressed different challenges and the opportunities for collaboration between patients and companies working to develop new treatments. Company team members will also take part in the virtual 5k race sponsored by the National Organization for Rare Disorders (NORD). And Fulcrum is launching a new corporate Facebook page that will present perspectives from patients and caregivers about their experiences and hopes for the future.

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission.

    Contacts:

    Investors:

    Christi Waarich

    Director, Corporate Communications and Investor Relations

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  21. CAMBRIDGE, Mass., Feb. 26, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its fourth quarter and full year 2020 financial results will be released on Thursday, March 4, 2021 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 6376419

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID…

    CAMBRIDGE, Mass., Feb. 26, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its fourth quarter and full year 2020 financial results will be released on Thursday, March 4, 2021 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 6376419

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 6376419

    An audio webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  22. CAMBRIDGE, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • Virtual SVB Leerink 10th Annual Global Healthcare Conference
      Presentation on Thursday, February 25, 2021 at 10:40 a.m. ET.
    • Virtual H.C. Wainwright Global Life Sciences Conference
      Prerecorded Fireside Chat Available on Tuesday, March 9, 2021 at 7:00 a.m. ET.

    Audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived…

    CAMBRIDGE, Mass., Feb. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • Virtual SVB Leerink 10th Annual Global Healthcare Conference

      Presentation on Thursday, February 25, 2021 at 10:40 a.m. ET.
    • Virtual H.C. Wainwright Global Life Sciences Conference

      Prerecorded Fireside Chat Available on Tuesday, March 9, 2021 at 7:00 a.m. ET.

    Audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for 90 days after each conference.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

     



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  23. CAMBRIDGE, Mass., Jan. 22, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced closing of its previously announced underwritten public offering of 4,600,000 shares of its common stock at a public offering price of $11.00 per share, which includes 600,000 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares at the public offering price, less underwriting discounts and commissions. The total gross proceeds of the offering were $50.6 million, before deducting underwriting discounts and commissions and offering expenses payable by Fulcrum…

    CAMBRIDGE, Mass., Jan. 22, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced closing of its previously announced underwritten public offering of 4,600,000 shares of its common stock at a public offering price of $11.00 per share, which includes 600,000 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares at the public offering price, less underwriting discounts and commissions. The total gross proceeds of the offering were $50.6 million, before deducting underwriting discounts and commissions and offering expenses payable by Fulcrum. All of the shares in the offering were sold by Fulcrum.

    SVB Leerink, Piper Sandler & Co. and Credit Suisse acted as joint book-running managers for the offering. H.C. Wainwright & Co. acted as lead manager for the offering.

    The shares were offered by Fulcrum pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC").

    This offering was made only by means of a prospectus and prospectus supplement that form a part of the registration statement. A final prospectus supplement relating to and describing the terms of the offering has been filed with the SEC and is available on the SEC's website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting: SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by email at syndicate@svbleerink.com; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924, or by email at prospectus@psc.com; or Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037, or by e-mail at usa.prospectus@credit-suisse.com.

    This press release shall not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  24. CAMBRIDGE, Mass., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 4,000,000 shares of its common stock at a public offering price of $11.00 per share, for total gross proceeds of $44.0 million, before deducting underwriting discounts and commissions and offering expenses payable by Fulcrum. All of the shares in the offering are being sold by Fulcrum. In addition, Fulcrum has granted the underwriters a 30-day option to purchase up to 600,000 additional shares of its common stock at the public offering price, less the underwriting…

    CAMBRIDGE, Mass., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 4,000,000 shares of its common stock at a public offering price of $11.00 per share, for total gross proceeds of $44.0 million, before deducting underwriting discounts and commissions and offering expenses payable by Fulcrum. All of the shares in the offering are being sold by Fulcrum. In addition, Fulcrum has granted the underwriters a 30-day option to purchase up to 600,000 additional shares of its common stock at the public offering price, less the underwriting discount and commissions. The offering is expected to close on January 22, 2021, subject to customary closing conditions.

    SVB Leerink, Piper Sandler & Co. and Credit Suisse are acting as joint book-running managers for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering.

    The shares are being offered by Fulcrum pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC").

    This offering is being made only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement relating to and describing the terms of the offering has been filed with the SEC and is available on the SEC's website at www.sec.gov. A final prospectus supplement relating to the offering will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting: SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by email at syndicate@svbleerink.com; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924, or by email at prospectus@psc.com; or Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037, or by e-mail at usa.prospectus@credit-suisse.com.

    This press release shall not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development.  

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, such as those, among others, relating to the Company's plans to consummate the public offering. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include, without limitation, risks and uncertainties related to the satisfaction of customary closing conditions related to the public offering and the impact of general economic, industry or political conditions in the United States or internationally. There can be no assurance that the Company will be able to complete the public offering on the anticipated terms, or at all. You should not place undue reliance on these forward-looking statements. Additional risks and uncertainties relating to the offering, Fulcrum and its business can be found under the caption "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, the Company's preliminary prospectus supplement filed with the SEC on January 19, 2021, and other filings that the Company may make with the SEC in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com 



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  25. CAMBRIDGE, Mass., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has commenced an underwritten public offering of shares of its common stock. All of the shares are being offered by Fulcrum. In addition, Fulcrum expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of its common stock sold in the public offering.

    SVB Leerink, Piper Sandler & Co. and Credit Suisse are acting as joint book-running managers for the offering. The offering is subject to market and other conditions, and there can be no assurance as to whether…

    CAMBRIDGE, Mass., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has commenced an underwritten public offering of shares of its common stock. All of the shares are being offered by Fulcrum. In addition, Fulcrum expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of its common stock sold in the public offering.

    SVB Leerink, Piper Sandler & Co. and Credit Suisse are acting as joint book-running managers for the offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    The shares are being offered by Fulcrum pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC").

    This offering is being made only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement relating to and describing the terms of the offering is expected to be filed with the SEC and, if and when filed, copies of the preliminary prospectus supplement relating to the offering may be obtained for free by visiting the SEC's website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus may also be obtained by contacting: SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by email at syndicate@svbleerink.com; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924, or by email at prospectus@psc.com; or Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037, or by e-mail at usa.prospectus@credit-suisse.com. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

    This press release shall not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.  

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into Phase 1 clinical development.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, such as those, among others, relating to the Company's plans to consummate its proposed public offering. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include, without limitation, risks and uncertainties related to whether or not the Company will be able to raise capital through the sale of shares of common stock, the final terms of the proposed offering, market and other conditions, the satisfaction of customary closing conditions related to the proposed public offering and the impact of general economic, industry or political conditions in the United States or internationally. There can be no assurance that the Company will be able to complete the proposed public offering on the anticipated terms, or at all. You should not place undue reliance on these forward-looking statements. Additional risks and uncertainties relating to the proposed offering, Fulcrum and its business can be found under the caption "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, the Company's preliminary prospectus supplement expected to be filed with the SEC on January 19, 2021, and other filings that the Company may make with the SEC in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  26. CAMBRIDGE, Mass., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that Christopher Moxham, Ph.D., has been promoted to the role of chief scientific officer. In this role, Dr. Moxham will oversee the organization's full range of drug discovery and translational science research, including advancing the company's multiple preclinical collaborations and building and expanding Fulcrum's product engine. Dr. Moxham played a key role in the discovery of FTX-6058, Fulcrum's highly potent small molecule inhibitor of EED for the treatment of hemoglobinopathies, such as sickle cell disease…

    CAMBRIDGE, Mass., Jan. 19, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that Christopher Moxham, Ph.D., has been promoted to the role of chief scientific officer. In this role, Dr. Moxham will oversee the organization's full range of drug discovery and translational science research, including advancing the company's multiple preclinical collaborations and building and expanding Fulcrum's product engine. Dr. Moxham played a key role in the discovery of FTX-6058, Fulcrum's highly potent small molecule inhibitor of EED for the treatment of hemoglobinopathies, such as sickle cell disease and beta thalassemia. Dr. Moxham will join the executive leadership team and report to Robert Gould, Ph.D., Fulcrum's president and chief executive officer. Owen Wallace, Ph.D., will step down from his role as chief scientific officer, effective February 5, 2021 to pursue another opportunity. Dr. Wallace has been appointed to Fulcrum's Scientific Advisory Board and in that role will continue to provide counsel to Fulcrum on the company's research and development programs.

    "On behalf of the entire Fulcrum organization, I would like to express my profound thanks for Owen's contributions to Fulcrum's early-stage pipeline and clinical research, including building our world-class team of talented and experienced researchers leading our preclinical development efforts," said Robert Gould, Ph.D., president and chief executive officer. "We wish him all the best in the next phase of his professional career and now welcome his outstanding experience and insights as a member of our Scientific Advisory Board."

    "Chris's appointment to chief scientific officer reflects his strong scientific acumen and deep expertise in drug discovery," continued Dr. Gould. "I am confident that under Chris's leadership we will continue to build new levels of momentum in our strategy to advance the promising compounds in our pipeline as rapidly as possible and to build for the future with new and promising opportunities in our research and development programs. Chris has made significant contributions to Fulcrum and I look forward to continuing to work together as we advance FTX-6058 into sickle cell patients and evolve our product engine."

    Dr. Moxham joined Fulcrum in January 2019 after having spent over 20 years in drug discovery primarily at Eli Lilly and Company. In this role, he helped grow Lilly's oncology pipeline, developed critical biopharma and academic partnerships and established a state-of-the-art laboratory facility focused on preclinical and translational research. He has led teams that have delivered numerous small and large molecules into clinical development. Dr. Moxham serves on the external review boards of the Long Island Bioscience Hub and the Alzheimer's Drug Discovery Foundation. He received his B.S. in biological sciences from Cornell University and his Ph.D. in molecular and cellular pharmacology from the State University of New York at Stony Brook. He also conducted postdoctoral training at SUNY Stony Brook under the sponsorship of the National Institutes of Health.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates and the potential advantages and therapeutic potential of the Company's product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in later clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  27. CAMBRIDGE, Mass., Jan. 06, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in a fireside chat at the virtual H.C. Wainwright BioConnect Conference.

    A webcast will be available on-demand on Monday, January 11, 2021 at 6:00 a.m. ET through the H.C. Wainwright conference portal and on the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. An archived replay will also be available on the Company's website for 90 days after the conference.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage…

    CAMBRIDGE, Mass., Jan. 06, 2021 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in a fireside chat at the virtual H.C. Wainwright BioConnect Conference.

    A webcast will be available on-demand on Monday, January 11, 2021 at 6:00 a.m. ET through the H.C. Wainwright conference portal and on the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. An archived replay will also be available on the Company's website for 90 days after the conference.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  28. Strengthens senior leadership team with appointments of Donald Johns, M.D., as Chief Medical Officer and Katina Dorton, J.D., MBA, as Chief Financial Officer

    NodThera, a biotechnology company developing a new class of medicines that inhibit the NLRP3 inflammasome to treat chronic inflammation, today announced the expansion of its senior leadership team with the appointments of Donald Johns, M.D., as Chief Medical Officer and Katina Dorton, J.D., MBA, as Chief Financial Officer.

    Dr. Johns is an accomplished drug development leader and board-certified clinical neurologist who previously served as Chief Medical Officer and Executive Vice President of Medical and Scientific Affairs at Syntimmune, prior to the company's acquisition by Alexion…

    Strengthens senior leadership team with appointments of Donald Johns, M.D., as Chief Medical Officer and Katina Dorton, J.D., MBA, as Chief Financial Officer

    NodThera, a biotechnology company developing a new class of medicines that inhibit the NLRP3 inflammasome to treat chronic inflammation, today announced the expansion of its senior leadership team with the appointments of Donald Johns, M.D., as Chief Medical Officer and Katina Dorton, J.D., MBA, as Chief Financial Officer.

    Dr. Johns is an accomplished drug development leader and board-certified clinical neurologist who previously served as Chief Medical Officer and Executive Vice President of Medical and Scientific Affairs at Syntimmune, prior to the company's acquisition by Alexion Pharmaceuticals. Ms. Dorton is a recognized and internationally experienced financial executive, corporate director and public company CFO. Ms. Dorton's industry expertise includes roles in healthcare, life sciences and investment banking, including experiences at Repare Therapeutics and AVROBIO, Inc.

    NodThera is advancing a portfolio of potent and selective inhibitors of the NLRP3 inflammasome that reduce both IL-1β and IL-18, pro-inflammatory cytokines which are known to play a role in chronic inflammation underlying a wide range of diseases. The pipeline includes brain penetrant NLRP3 inhibitors for central nervous system (CNS) indications.

    "We welcome Don and Katina to the team as we continue to advance our portfolio of differentiated NLRP3 inhibitors to capitalize on the opportunity to exploit the well-understood, but still untapped therapeutic potential of the NLRP3 inflammasome across a broad spectrum of diseases," said Adam Keeney, Ph.D., President & Chief Executive Officer of NodThera. "Don is an accomplished clinician with an impressive track record who brings a wealth of innovative clinical development experience. Katina brings capital markets experience and has built financial, legal and operational functions to support companies through aggressive growth, including IPO preparation. Their combined contributions will add significant value as we continue to advance NodThera's portfolio through clinical development and additional financing rounds."

    "The NLRP3 inflammasome is one of the most exciting emerging areas of therapeutic science. NodThera's best-in-class molecules have great potential to address the unmet medical need in patients with a wide range of inflammatory disorders," said Dr. Johns. Ms. Dorton adds, "By leveraging innate immunity, NodThera's platform has the potential to blaze a new path forward in a wide range of disorders, and I am excited to join the rest of the experienced leadership team in this effort."

    Dr. Johns brings more than 25 years of experience in the development of novel treatments for serious diseases, including autoimmune and CNS disorders. Prior to his role at Syntimmune, Dr. Johns served in key leadership positions at Biogen and the Novartis Institutes for Biomedical Research (NIBR). He has contributed to numerous investigational new drug applications (INDs), first-in-human studies and conclusive proof-of-concept studies in a broad spectrum of CNS and autoimmune diseases, as well as four successful new drug applications (NDAs). Prior to joining the pharmaceutical industry, he was a neurologist clinician-scientist at Johns Hopkins and Harvard Medical School. Dr. Johns earned his B.A. from Vanderbilt University and his M.D. from the Yale University School of Medicine.

    Ms. Dorton currently serves on the board of directors for Fulcrum Therapeutics (NASDAQ:FULC), Pandion Therapeutics, Inc. (NASDAQ:PAND) and US Ecology (NASDAQ:ECOL). She most recently served as Chief Financial Officer of Repare Therapeutics, a synthetic lethality and DNA repair-focused oncology company. Prior to Repare, Ms. Dorton served as Chief Financial Officer of AVROBIO, a lentiviral gene therapy company. Earlier in her career, she served as a managing director in investment banking for Morgan Stanley and Needham & Company and as an associate attorney at Sullivan & Cromwell. Ms. Dorton received her J.D. from the University of Virginia School of Law, her MBA from George Washington University and her B.A. from Duke University.

    About NodThera

    NodThera is a biotechnology company developing a new class of potent and selective NLRP3 inflammasome inhibitors for the treatment of diseases driven by chronic inflammation. Led by an experienced management team, NodThera is leveraging new insights into inflammasome biology and chemistry to build a platform of highly differentiated small molecule NLRP3 inhibitors. The company was founded by Epidarex Capital and further financed by 5AM Ventures, Cowen Healthcare Investments, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera was founded in 2016 and maintains offices in Cambridge, UK, Seattle, WA and Boston, MA. For more information please visit www.nodthera.com.

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  29. Event will review the company's novel approach to inducing fetal hemoglobin

    Live webcast on December 15, 2020 at 8:30am ET

    CAMBRIDGE, Mass., Dec. 09, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it will host a Key Opinion Leader (KOL) meeting on Tuesday, December 15, 2020 from 8:30am – 10:00am ET to discuss the company's program with FTX-6058 for select hemoglobinopathies, including sickle cell disease and beta-thalassemia.

    Dr. Maureen Achebe and Dr. Gerd Blobel will join senior executives from Fulcrum in presenting and discussing sickle cell disease, the treatment landscape…

    Event will review the company's novel approach to inducing fetal hemoglobin

    Live webcast on December 15, 2020 at 8:30am ET

    CAMBRIDGE, Mass., Dec. 09, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it will host a Key Opinion Leader (KOL) meeting on Tuesday, December 15, 2020 from 8:30am – 10:00am ET to discuss the company's program with FTX-6058 for select hemoglobinopathies, including sickle cell disease and beta-thalassemia.

    Dr. Maureen Achebe and Dr. Gerd Blobel will join senior executives from Fulcrum in presenting and discussing sickle cell disease, the treatment landscape and the FTX-6058 program followed by a Question and Answer session. Maureen Achebe, MD is currently Clinical Director, Non-Malignant Hematology Clinic, Assistant Director, Brigham and Women's Hospital Outpatient Infusion Center, Director, Brigham and Women's Hospital Sickle Cell Program and Assistant Professor of Medicine, Harvard Medical School. Gerd Blobel, MD, PhD is currently Frank E. Weise III professor of pediatrics, University of Pennsylvania and Co-director Epigenetics Institute. He also holds the Frank E. Weise III Endowed Chair of Pediatrics at The Children's Hospital of Philadelphia and the Perelman School of Medicine.

    The live webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available on the website for up to 90 days.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of EED capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary Product Engine. The company's composition of matter patent covering FTX-6058 and related structures has been granted. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy volunteers.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia, into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates and the potential advantages and therapeutic potential of the Company's product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in later clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

     



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  30. CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that preclinical data with FTX-6058 for the treatment of sickle cell disease will be presented in three posters at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 5-8, 2020. FTX-6058 is a highly potent small molecule EED inhibitor that induces expression of fetal hemoglobin (HbF). Elevating HbF can compensate for the mutated adult hemoglobin that has been identified as the root cause of several hemoglobinopathies and can ameliorate or eliminate the symptoms…

    CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that preclinical data with FTX-6058 for the treatment of sickle cell disease will be presented in three posters at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 5-8, 2020. FTX-6058 is a highly potent small molecule EED inhibitor that induces expression of fetal hemoglobin (HbF). Elevating HbF can compensate for the mutated adult hemoglobin that has been identified as the root cause of several hemoglobinopathies and can ameliorate or eliminate the symptoms of sickle cell disease.

    "We are encouraged by the robust preclinical data package and unique mechanism of action of FTX-6058, which has the potential to be a transformative therapy for sickle cell patients," said Owen B. Wallace, Ph.D., Fulcrum's chief scientific officer. "Through internal research and discussions with key opinion leaders, we have identified areas within the sickle cell disease landscape where FTX-6058 has the potential to address significant unmet need. Enrollment has begun in our Phase 1 trial in healthy volunteers and we look forward to progressing FTX-6058 in clinical development."

    FTX-6058 Results at ASH

    Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, demonstrating the potential to have a significant impact in patients with sickle cell disease. FTX-6058 inhibits PRC2 via binding to EED, which induces a robust HbF protein expression in cell and murine models. Increasing HbF has the potential to prevent or reduce disease-related pathophysiology, resulting in reduction of recurring events such as vaso-occlusive crises and hemolysis. Human genetic data indicates that individuals with the sickle cell mutation but who have high HbF levels may have asymptomatic disease, underscoring the protective effect of increased HbF.

    Key highlights include:

    • Demonstrated potent target engagement and HbF induction in vivo in animal models at plasma concentrations reasonably expected to be achieved in the clinic.
    • Pharmacological activity in target cells can be readily monitored in the clinic since target engagement in bone marrow correlates with target engagement in peripheral monocytes in animals.
    • Demonstrated an impressive preclinical pharmacological profile with the potential to be a disease-modifying therapeutic for sickle cell patients.

    The posters will be available in the "Publications" section of fulcrumtx.com following the sessions.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of EED capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary Product Engine. The company's composition of matter patent covering FTX-6058 and related structures has been granted. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy volunteers.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture leading to anemia. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia, into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates and the potential advantages and therapeutic potential of the Company's product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in later clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com 

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  31. WATERTOWN, Mass., Dec. 03, 2020 (GLOBE NEWSWIRE) -- Pandion Therapeutics, Inc. (NASDAQ:PAND), a clinical-stage biotechnology company developing novel therapeutics designed to address the unmet needs of patients living with autoimmune diseases, today announced the appointment of Katina Dorton, J.D., M.B.A., to Pandion's board of directors and as chair of the audit committee. Ms. Dorton assumes the position of chair of the audit committee from Christopher Fuglesang, Ph.D., J.D., who will continue to serve as a member of the board and audit committee. Mitchell Mutz, Ph.D., resigned from the Company's board on December 2, 2020.

    "Ms. Dorton brings to Pandion over two decades of financial expertise, leading a multitude of financial transactions…

    WATERTOWN, Mass., Dec. 03, 2020 (GLOBE NEWSWIRE) -- Pandion Therapeutics, Inc. (NASDAQ:PAND), a clinical-stage biotechnology company developing novel therapeutics designed to address the unmet needs of patients living with autoimmune diseases, today announced the appointment of Katina Dorton, J.D., M.B.A., to Pandion's board of directors and as chair of the audit committee. Ms. Dorton assumes the position of chair of the audit committee from Christopher Fuglesang, Ph.D., J.D., who will continue to serve as a member of the board and audit committee. Mitchell Mutz, Ph.D., resigned from the Company's board on December 2, 2020.

    "Ms. Dorton brings to Pandion over two decades of financial expertise, leading a multitude of financial transactions for companies in the life sciences industry. We look forward to her contributions to the growth and value creation for Pandion as a newly public company," said Rahul Kakkar, M.D., Chief Executive Officer of Pandion Therapeutics. "We also sincerely thank Mitchell for his guidance as we brought Pandion from an idea through its first-in-human clinical trial and wish him the best in his future endeavors."

    "Pandion has the potential to bring about the next generation in autoimmune treatments with a unique focus on activating the body's natural immune control nodes. I am excited to be a part of the team, particularly as we look to the Phase 1a results for the Company's lead program, PT101, in early 2021," commented Katina Dorton.

    Ms. Dorton currently serves on the board of directors for Fulcrum Therapeutics (NASDAQ:FULC) and US Ecology (NASDAQ:ECOL). She most recently served as Chief Financial Officer of Repare Therapeutics, a synthetic lethality and DNA repair-focused oncology company. Prior to Repare, Ms. Dorton served as Chief Financial Officer of AVROBIO, a lentiviral gene therapy company. Earlier in her career, she served as a managing director in investment banking for Morgan Stanley and Needham & Company and as an associate attorney at Sullivan & Cromwell. Ms. Dorton received her J.D. from the University of Virginia School of Law, her M.B.A. from George Washington University and her B.A. from Duke University.

    About Pandion Therapeutics

    Pandion Therapeutics is developing novel therapeutics designed to address the unmet needs of patients living with autoimmune diseases. Pandion's TALON (Therapeutic Autoimmune reguLatOry proteiN) drug design and discovery platform enables the company to create a pipeline of product candidates using immunomodulatory effector modules, with the ability to also combine an effector module with a tissue-targeted tether module in a bifunctional format. Pandion's lead product candidate PT101, a combination of an interleukin-2 mutein effector module with a protein backbone, is designed to selectively expand regulatory T cells systemically, without activating proinflammatory cells, such as conventional T cells and natural killer cells, is currently in a Phase 1a clinical trial. Pandion is continuing to develop and expand its library of effector and tether modules as part of its earlier-stage research and discovery pipeline. For more information, please visit www.pandiontx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy and clinical development plans, timelines and prospects, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Pandion's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; initiate preclinical studies and clinical trials of PT101 and its other product candidates; advance PT101 and its other product candidates in preclinical research and clinical trials; replicate in clinical trials positive results found in preclinical studies; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Media:

    Kathryn Morris

    The Yates Network

    914-204-6412

    kathryn@theyatesnetwork.com

    Investors:

    Michelle Avery

    Pandion Therapeutics

    857-273-0444

    investors@pandiontx.com



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  32. CAMBRIDGE, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • Stifel Virtual 2020 Healthcare Conference
      Presentation on Wednesday, November 18, 2020 at 11:20 a.m. ET.
    • Piper Sandler 32nd Annual Virtual Healthcare Conference
      Prerecorded Fireside Chat Available on Monday, November 23, 2020 at 10:00 a.m. ET

    Audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be…

    CAMBRIDGE, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • Stifel Virtual 2020 Healthcare Conference

      Presentation on Wednesday, November 18, 2020 at 11:20 a.m. ET.
    • Piper Sandler 32nd Annual Virtual Healthcare Conference

      Prerecorded Fireside Chat Available on Monday, November 23, 2020 at 10:00 a.m. ET

    Audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for 90 days after each conference.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations

    and Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com



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  33. ReDUX4 trial progressing; full data with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) expected in 2Q 2021

    – On track to begin dosing Phase 1 trial with FTX-6058 for sickle cell disease before YE 2020

    – Cash runway into 2Q 2022

    – Key appointments to management team

    Conference call scheduled for 8:00 a.m. ET today

    CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the third quarter of 2020.

    "We've made significant progress in 2020, which will lead to…

    ReDUX4 trial progressing; full data with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) expected in 2Q 2021

    – On track to begin dosing Phase 1 trial with FTX-6058 for sickle cell disease before YE 2020

    – Cash runway into 2Q 2022

    – Key appointments to management team

    Conference call scheduled for 8:00 a.m. ET today

    CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the third quarter of 2020.

    "We've made significant progress in 2020, which will lead to a number of key data readouts as we move into 2021," said Robert J. Gould, Ph.D., president and chief executive officer. "We plan to announce full data from our ReDUX4 trial with losmapimod in facioscapulohumeral muscular dystrophy (FSHD), including the primary endpoint in the second quarter of 2021. We are encouraged by the interim analysis from ReDUX4, which assessed reductions in DUX4-driven gene expression, the root cause of the disease, via muscle biopsy. The full data will also contain a number of validated and novel endpoints and we look forward to presenting a comprehensive assessment of the data in its entirety. Our select hemoglobinopathy program, FTX-6058, a highly potent small molecule EED inhibitor, has made important progress as well, and we are currently screening healthy volunteers in our Phase 1 trial. We believe FTX-6058 has the potential to offer a transformative small molecule treatment option that could represent a significant development for sickle cell patients."

    "I am also pleased to report that Curtis Oltmans will be joining Fulcrum as Senior Vice President, General Counsel and Kim Hazen has been promoted to Senior Vice President, Human Resources," continued Dr. Gould. "In addition, Alan Ezekowitz, MBChB, D.Phil will take on the responsibility of clinical advisor as Diego Cadavid, MD is leaving his position as Senior Vice President, Clinical Development. Curt has an extensive record of legal and corporate development accomplishments. Kim has made important contributions since joining Fulcrum in 2017 and Alan has served on our Board of Directors since 2016. We are thrilled to have them on our leadership team. We thank Diego for his many contributions to Fulcrum."

    Third Quarter and Recent Business Highlights

    • Announced interim data for ReDUX4, a Phase 2b trial of losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor, in FSHD.
      • Encouraging data suggest that muscles with the highest DUX4-driven gene expression in pre-treatment biopsies showed large reductions in DUX4-driven gene expression following treatment with losmapimod compared to placebo.
      • Remain on track to report full data in the second quarter of 2021. Data will include the primary endpoint, reduction from baseline of DUX4-driven gene expression, as well as a pre-specified sensitivity analysis assessing biopsies with the highest pre-treatment level of DUX4-driven gene expression, in addition to secondary and exploratory endpoints.
      • Continued evaluation of the Open Label Study and ReSOLVE Natural History Study
    • On track to begin dosing healthy volunteers in a Phase 1 trial for sickle cell disease with FTX-6058 before year-end 2020.
      • FTX-6058, a highly potent small molecule EED inhibitor, is designed to induce expression of fetal hemoglobin (HbF) in red blood cells to compensate for the mutated adult hemoglobin for the potential treatment of hemoglobinopathies, including sickle cell disease and beta-thalassemia.
      • Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin showing the potential to have a significant impact on the sickle cell patient population.
      • Fulcrum's non-provisional composition of matter patent application covering FTX-6058 and related structures published.
    • Enrolling patients in the international, multicenter Phase 3 trial with losmapimod for hospitalized subjects with COVID-19 (LOSVID). The trial is designed to assess the safety and efficacy of losmapimod compared to placebo for 14 days on top of standard of care in approximately 400 patients who are at risk of progression to critical illness based on older age and elevated systemic inflammation.
      • Progress in opening trial sites has been slower than anticipated. Based on assessments of enrollment thus far we anticipate providing an update on the LOSVID trial in the first quarter of 2021.
    • Announced multiple scientific meeting presentations
      • Presented target engagement and good tolerability with FTX-6058 in multiple preclinical rodent models with once-a-day oral dosing at the 14th Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting, September 25, 2020.
      • Presented multiple posters demonstrating Fulcrum's integrated approach to the evaluation of FSHD patients during the 25th International Congress of the World Muscle Society (WMS), October 1, 2020.
      • Announced multiple posters demonstrating the potential of FTX-6058 accepted at the 62nd American Society of Hematology (ASH) annual meeting, December 5-8, 2020.
    • Executed strategic collaboration and license agreement in July 2020 with MyoKardia to identify therapeutics that control the expression of genes that are known to be underlying drivers of genetic cardiomyopathies.
      • Fulcrum is eligible to receive preclinical milestone payments, development milestone payments and sales milestone payments of up to $298.5 million for a first product to progress through development and commercialization and may be eligible for up to $150.0 million in milestone payments for additional targets, as well as reimbursement for the costs of the research activities.
      • Fulcrum may also be eligible to receive tiered royalty payments in the mid-single digit to low double-digit range on net sales for any products under the collaboration that are commercialized.
    • Key appointments to management team
      • Curtis Oltmans will join Fulcrum as Senior Vice President, General Counsel and Corporate Secretary, effective November 30, 2020.
        • Most recently, Mr. Oltmans served as Vice President, Head of Litigation for DaVita, Inc. where he was responsible for strategy and initiatives in the areas of law, including litigation, employee safety and insurance. Prior to DaVita, he served as General Counsel for Array BioPharma (acquired by Pfizer). Prior to Array BioPharma, Mr. Oltmans served as the US General Counsel for Novo Nordisk.  Mr. Oltmans earned his undergraduate degree in political science at the University of Nebraska and his Juris Doctorate Degree at the University of Nebraska College of Law.
      • Alan Ezekowitz, MBChB, D.Phil will take on the responsibility of clinical advisor.
        • Dr. Ezekowitz has served on our board of directors since December 2016 and has served as a venture partner at Third Rock Ventures, LLC since December 2019. Prior to Third Rock Ventures, Dr. Ezekowitz served as the president and chief executive officer of Abide Therapeutics, Inc. (acquired by H. Lundbeck A/S). Prior to founding Abide, Dr. Ezekowitz was the senior vice president and franchise head for disease areas including bone, respiratory, immunology, muscle, dermatology and urology at Merck. Dr. Ezekowitz received his medical training at the University of Cape Town in South Africa and received a Doctor of Philosophy degree from Oxford University.
      • Kim Hazen promoted to Senior Vice President, Human Resources.
        • Ms. Hazen has over 25 years of experience in human resources and has been an invaluable member of the leadership team since she joined Fulcrum in September 2017.

    Third Quarter 2020 Financial Results

    • Cash Position: As of September 30, 2020, cash, cash equivalents, and marketable securities were $127.0 million, as compared to $96.7 million as of December 31, 2019. Based on its current plans, the Company expects that its existing cash, cash equivalents, and marketable securities will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second quarter of 2022.



    • R&D Expenses: Research and development expenses were $15.6 million for the third quarter of 2020, as compared to $13.5 million for the third quarter of 2019. The increase of $2.1 million was primarily due to increased costs to support our ongoing and planned clinical trials and increased personnel-related costs to support the growth of Fulcrum's research and development organization, including increased stock-based compensation expense. Research and development expenses for the third quarter of 2019 include $2.5 million of one-time costs incurred associated with the achievement of a milestone under the Company's license agreement with GSK for losmapimod.



    • G&A Expenses: General and administrative expenses were $5.3 million for the third quarter of 2020, as compared to $3.5 million for the third quarter of 2019. The increase of $1.8 million was primarily due to increased costs associated with operating as a public company and increased personnel-related costs to support the growth of our organization, including increased stock-based compensation expense.



    • Net Loss: Net loss was $19.0 million for the third quarter of 2020, as compared to a net loss of $16.5 million for the third quarter of 2019.

    Conference Call and Webcast

    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the Company's third quarter 2020 recent business highlights and financial results. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 2408316

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 2408316

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Researchers at Fulcrum also believe that losmapimod has the potential to treat COVID-19 by reducing the acute exaggerated pro-inflammatory responses to SARS-CoV-2 infection and restoring the antigen-specific immune responses needed for viral clearance, potentially leading to improved clinical outcomes. Losmapimod has been evaluated in more than 3,600 subjects in clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the Company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD and a Phase 3 trial investigating the safety, tolerability, and efficacy of losmapimod to treat hospitalized patients with COVID-19.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary Product Engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy volunteers.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of our product candidates, initiation and enrollment of clinical trials and availability of clinical trial data, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.



    Fulcrum Therapeutics, Inc.

    Selected Consolidated Balance Sheet Data

    (In thousands)

    (Unaudited)

     September 30,

    2020
     December 31,

    2019
    Cash, cash equivalents, and marketable securities$127,025 $96,713
    Working capital(1) 105,657  87,943
    Total assets 142,215  110,439
    Total stockholders' equity 104,985  87,153

    _______________

    (1)    We define working capital as current assets minus current liabilities.





    Fulcrum Therapeutics, Inc.

    Consolidated Statements of Operations

    (In thousands, except per share data)

    (Unaudited)

     Three Months Ended

    September 30,
      Nine Months Ended

    September 30,
     
     2020  2019  2020  2019 
    Collaboration revenue$1,848  $  $4,598  $ 
    Operating expenses:               
    Research and development 15,640   13,496   42,897   58,985 
    General and administrative 5,312   3,510   15,525   8,742 
    Total operating expenses 20,952   17,006   58,422   67,727 
    Loss from operations (19,104)  (17,006)  (53,824)  (67,727)
    Other income, net 142   464   725   1,173 
    Net loss$(18,962) $(16,542) $(53,099) $(66,554)
    Cumulative convertible preferred stock dividends    (796)     (7,128)
    Net loss attributable to common stockholders$(18,962) $(17,338) $(53,099) $(73,682)
    Net loss per share attributable to common stockholders, basic and diluted$(0.70) $(0.97) $(2.16) $(10.33)
    Weighted average number of common shares used in net loss per share attributable to common stockholders, basic and diluted 27,261   17,785   24,621   7,133 



    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881 

    Primary Logo

    View Full Article Hide Full Article
  34. CAMBRIDGE, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it will present multiple posters on the company's IND-supporting preclinical studies with its Embryonic Ectoderm Development (EED) inhibitor, FTX-6058, for hemoglobinopathies such as sickle cell disease and β-thalassemia during the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, December 5-8, 2020.

    "We believe FTX-6058 has the potential to offer a differentiated approach for the treatment of hemoglobinopathies like sickle cell disease and β-thalassemia," said Owen B. Wallace…

    CAMBRIDGE, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it will present multiple posters on the company's IND-supporting preclinical studies with its Embryonic Ectoderm Development (EED) inhibitor, FTX-6058, for hemoglobinopathies such as sickle cell disease and β-thalassemia during the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, December 5-8, 2020.

    "We believe FTX-6058 has the potential to offer a differentiated approach for the treatment of hemoglobinopathies like sickle cell disease and β-thalassemia," said Owen B. Wallace, Ph.D., Fulcrum's chief scientific officer. "By demonstrating clinically desirable elevations of fetal hemoglobin up to approximately 30% of total hemoglobin in preclinical studies, these data, and those from our completed IND-enabling studies with FTX-6058, are incredibly exciting. We are pleased to present our preclinical proof-of-concept findings at this important scientific meeting and look forward to our clinical trial of FTX-6058 in healthy volunteers."

    Fulcrum will present three posters describing the potential of FTX-6058 to treat hemoglobinopathies during the following virtual sessions:

    Poster Title: In vivo characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell disease

    Presenter: Keqiang Xie, Ph.D., Senior Scientist at Fulcrum Therapeutics

    Session Name: 113. Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: Poster I

    Date and Time: Saturday, December 5, 2020 from 7:00 a.m. – 3:30 p.m. PT

    Poster Title: Induction of fetal hemoglobin by FTX6058, a novel small molecule development candidate

    Presenter: Christopher Moxham, Ph.D., Senior Vice President Discovery Research at Fulcrum Therapeutics

    Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I

    Date and Time: Saturday, December 5, 2020 from 7:00 a.m. – 3:30 p.m. PT

    Poster Title: In vitro characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell disease

    Presenter: Billy Stuart, Scientist II, Fulcrum Therapeutics

    Session Name: 113. Hemoglobinopathies, Excluding Thalassemia—Basic and Translational Science: Poster III

    Date and Time: Monday, December 7, 2020 from 7:00 a.m. – 3:30 p.m. PT

    The poster sessions will be available to registered conference attendees and the presentations will be published online in the November 2020 supplemental issue of Blood. The posters will also be made available in the "Publications" section of fulcrumtx.com following the sessions.

    About FTX-6058

    FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's proprietary Product Engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy volunteers.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture leading to anemia. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia, into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates and the potential advantages and therapeutic potential of the Company's product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in later clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com 

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  35. CAMBRIDGE, Mass., Nov. 03, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its third quarter 2020 financial results will be released on Tuesday, November 10, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 2408316

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 2408316

    An audio…

    CAMBRIDGE, Mass., Nov. 03, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its third quarter 2020 financial results will be released on Tuesday, November 10, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 2408316

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 2408316

    An audio webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Primary Logo

    View Full Article Hide Full Article
  36. CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) --  Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the Investigational New Drug application (IND) is now in effect for its Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease. FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.

    This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts. Part A will be a randomized, double-blind…

    CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) --  Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the Investigational New Drug application (IND) is now in effect for its Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease. FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.

    This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts. Part A will be a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study in up to six cohorts. Part B will be a randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) study in up to four cohorts dosed once daily for 14 days. Part C will be an open label pilot food effect study in subjects randomized to take FTX-6058 with and without a high-fat meal, and Part D will be an open label study to evaluate the potential of FTX-6058 to induce CYP3A (using midazolam).

    "We are pleased to leverage our expertise in the modulation of genetic drivers of disease to expand our clinical development efforts into a third area with sickle cell," said Robert J. Gould, Ph.D., president and chief executive officer of Fulcrum Therapeutics. "We believe FTX-6058 offers a differentiated approach to a potential treatment. A major unmet need remains for many sickle cell patients, and the availability of an effective and safe small molecule treatment option could represent a significant advancement. We are excited about the preclinical data that showed elevations of fetal hemoglobin up to 30% of total hemoglobin. Should this elevation be seen in sickle cell patients, it has the potential to address multiple symptoms, including painful crises and anemia."

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture leading to anemia. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19. Fulcrum is also advancing FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the Company's initiation of a clinical trial and evaluation, and the potential benefits, of FTX-6058 as a potential treatment for sickle cell disease, the development status of the Company's product candidates and the potential advantages and therapeutic potential of the Company's product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  37. CAMBRIDGE, Mass., Oct. 01, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it will present multiple posters on the company's ongoing studies in patients with facioscapulohumeral muscular dystrophy (FSHD) during the 25th International Congress of the World Muscle Society.

    "FSHD is a serious and debilitating disease for which there are currently no approved therapies," said Diego Cadavid, MD, Fulcrum's senior vice president, clinical development. "We are pleased to share these data at this important scientific meeting as we continue to pursue losmapimod as a potential treatment for…

    CAMBRIDGE, Mass., Oct. 01, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it will present multiple posters on the company's ongoing studies in patients with facioscapulohumeral muscular dystrophy (FSHD) during the 25th International Congress of the World Muscle Society.

    "FSHD is a serious and debilitating disease for which there are currently no approved therapies," said Diego Cadavid, MD, Fulcrum's senior vice president, clinical development. "We are pleased to share these data at this important scientific meeting as we continue to pursue losmapimod as a potential treatment for FSHD by addressing the root cause of the disease. We greatly appreciate the patients who have participated in our trials and the support we have received from key opinion leaders and investigators."

    During the Virtual Poster Session today, October 1, 2020 from 12:30pm – 2:30pm ET, Fulcrum will present four posters on its integrated approach to the evaluation of FSHD patients, highlighting the progress made in the development of imaging and molecular biomarkers in FSHD and the design of clinical trials to evaluate potential benefits of losmapimod in FSHD patients:

    • Development and Evaluation of a Whole-body MRI Protocol and Analysis Algorithms to Measure Changes in Skeletal Muscle in FSHD



    • A Biomarker of Aberrant DUX4 Activity to Evaluate Losmapimod Treatment Effect in FSHD Phase 2 Trials



    • Open-Label Pilot Study of Losmapimod in FSHD1 (NCT04004000)



    • A Phase 2, Randomized, Placebo-Controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Treating Subjects with FSHD: ReDUX4 (NCT04003974) Interim Analysis

    The poster sessions will be available to registered conference attendees. The posters will also be made available in the "Publications" section of fulcrumtx.com.

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD.  Researchers at Fulcrum also believe that losmapimod has the potential to treat COVID-19 by reducing the acute exaggerated pro-inflammatory responses to SARS-CoV-2 infection and restoring the antigen-specific immune responses needed for viral clearance, potentially leading to improved clinical outcomes. Losmapimod has been evaluated in more than 3,600 subjects in clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the Company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD and initiating a Phase 3 trial investigating the safety, tolerability, and efficacy of losmapimod to treat hospitalized patients with COVID-19.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates the initiation of a clinical trial in the fourth quarter of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, including the timing of initiation of a Phase 1 clinical trial for FTX-6058, and the potential advantages and therapeutic potential of our product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in later clinical trials positive results found in preclinical studies and /or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  38. - Fetal Hemoglobin expression in human cellular models increased up to ~30% by FTX-6058 for the potential treatment of sickle-cell disease

    - Company plans to initiate Phase 1 trial in healthy volunteers by year-end

    - Non-provisional composition of matter patent application covering FTX-6058 published

    CAMBRIDGE, Mass., Sept. 25, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced preclinical proof-of-concept data supporting the development of FTX-6058 to treat sickle cell disease and beta-thalassemia. FTX-6058, a small molecule designed to increase expression of fetal hemoglobin…

    - Fetal Hemoglobin expression in human cellular models increased up to ~30% by FTX-6058 for the potential treatment of sickle-cell disease

    - Company plans to initiate Phase 1 trial in healthy volunteers by year-end

    - Non-provisional composition of matter patent application covering FTX-6058 published

    CAMBRIDGE, Mass., Sept. 25, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced preclinical proof-of-concept data supporting the development of FTX-6058 to treat sickle cell disease and beta-thalassemia. FTX-6058, a small molecule designed to increase expression of fetal hemoglobin, demonstrated target engagement and good tolerability in multiple preclinical rodent models with once-a-day oral dosing. The Company presented these data today at the 14th Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting being held virtually. Slides from the presentation will be available on Fulcrum's website at ir.fulcrumtx.com/events-and-presentations.

    "Despite newly approved therapies for sickle cell disease, a significant unmet need remains," said Martin H. Steinberg, MD, Professor of Medicine at Boston University School of Medicine. "An orally available small molecule therapeutic acting through a novel mechanism to induce increased pancellular HbF should be an important disease-modifying agent."

    Fetal Hemoglobin (HbF) is a key modulator of sickle cell disease. Increasing HbF levels has the potential to prevent or reduce disease-related pathophysiology, resulting in reduction of recurring events such as vaso-occlusive crises (VOCs) and hemolysis. In some cases, sickle cell patients with high HbF levels have asymptomatic disease, underscoring the protective effect of HbF. Fulcrum has identified FTX-6058, a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Additionally, Fulcrum believes that pharmacokinetics and human dose simulations support FTX-6058 may be given as a once daily oral compound.

    The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrum's Product Engine. Through inhibition of EED, Fulcrum has demonstrated the ability to modulate the activity of the Polycomb Repressive Complex 2 (PRC2), a key regulator of HbF expression, in preclinical studies. Fulcrum validated the role between EED binding/PRC2 modulation and HbF induction utilizing its proprietary CRISPR and chemical probe screening platform. Treatment of human CD34+-derived erythroid cells from healthy and sickle cell disease donors with FTX-6058 resulted in clinically desirable fetal hemoglobin levels (up to ~30% of total hemoglobin), demonstrating a superior globin profile relative to hydroxyurea and other small molecule compounds or mechanisms currently under development. In these preclinical studies, FTX-6058 also induced pancellular distribution of HbF similar to hereditary persistence of fetal hemoglobin.

    In vivo preclinical studies showed elevation of HbF at the mRNA and protein levels at plasma concentrations predicted by Fulcrum to be achievable in patients. FTX-6058 treatment led to, elevation of the human HBG1 mRNA and HbF protein in the Townes SCD mouse model. In a head-to-head in-vivo preclinical study, FTX-6058 demonstrated superior HbF induction over hydroxyurea in the Townes SCD mouse model.

    "We continue to demonstrate important progress with our Product Engine, developing a robust pipeline focused on treatments for rare diseases and areas of significant unmet need," said Owen Wallace, Fulcrum's chief scientific officer. "We are very encouraged by these in vitro and in vivo findings, as the preclinical data support our novel approach to treating hemoglobinopathies, such as sickle cell disease and beta-thalassemia. In addition to achieving robust fetal hemoglobin levels in cell and murine models, an extensive nonclinical safety package and off-target profile has been established for FTX-6058. We believe FTX-6058 has the potential to offer a durable and transformative therapy for people living with sickle cell disease."

    Fulcrum completed a comprehensive IND-enabling package, including preclinical safety studies and up to 28-day Good Laboratory Practices (GLP) toxicology studies, as well as Good Manufacturing Practices (GMP) material scale-up for its planned Phase 1 clinical trial. The Company remains on track to initiate a Phase 1 clinical trial by year-end. In addition, Fulcrum's non-provisional composition of matter patent application covering FTX-6058 and related structures has published.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has advanced losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates filing an IND in the third quarter with initiation of a clinical trial in the fourth quarter of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, including the timing of initiation of a Phase 1 clinical trial for FTX-6058, and the potential advantages and therapeutic potential of our product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

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  39. CAMBRIDGE, Mass., Sept. 08, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • Virtual H.C. Wainwright 22nd Annual Global Investment Conference
      Tuesday, September 15, 2020 at 10:00 a.m. ET
    • Morgan Stanley Virtual Global Healthcare Conference
      Wednesday, September 16, 2020 at 11:00 a.m. ET
    • Cantor Fitzgerald Virtual Global Healthcare Conference
      Thursday, September 17, 2020 at 4:40 p.m. ET

    Live audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations

    CAMBRIDGE, Mass., Sept. 08, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • Virtual H.C. Wainwright 22nd Annual Global Investment Conference

      Tuesday, September 15, 2020 at 10:00 a.m. ET
    • Morgan Stanley Virtual Global Healthcare Conference

      Wednesday, September 16, 2020 at 11:00 a.m. ET
    • Cantor Fitzgerald Virtual Global Healthcare Conference

      Thursday, September 17, 2020 at 4:40 p.m. ET

    Live audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for 90 days after each conference.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates filing an IND in the third quarter with initiation of a clinical trial in the fourth quarter of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Primary Logo

    View Full Article Hide Full Article
  40. Conference call scheduled for 8:00 a.m. ET today

    CAMBRIDGE, Mass., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the second quarter of 2020.

    "The team at Fulcrum not only delivered key progress across our pipeline this quarter, but also expanded our work into additional areas of critical unmet need," said Robert J. Gould, Ph.D., president and chief executive officer. "We are encouraged by the data from the ReDUX4 interim analysis announced earlier today, suggesting that muscles with the highest DUX4-driven gene expression in pre-treatment biopsies show greater reduction in DUX4-driven gene expression following treatment with losmapimod compared to placebo. Progress continues with FTX-6058 where we have seen an increase in HbF levels up to approximately 30% of total hemoglobin in preclinical assessment. Additionally, our recent collaboration with MyoKardia has expanded our research efforts into genetic cardiomyopathies following our previously announced collaboration with Acceleron. And while our focus remains steadfast on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need, our scientific team saw a compelling rationale to use losmapimod for the treatment of hospitalized patients with COVID-19, and we recently initiated a Phase 3 international trial, with topline data expected in the first quarter of 2021. In the midst of all this activity, we further strengthened our cash position through a private placement to support our expanding pipeline."

    Recent Business Highlights

    • Announced interim data for ReDUX4, a Phase 2b trial of losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor, in facioscapulohumeral muscular dystrophy (FSHD)
      • Results from the interim analysis in the first 29 randomized subjects indicated that DUX4-driven gene expression did not separate from placebo at 16 weeks.
      • In a pre-specified sensitivity analysis assessing biopsies with the highest pre-treatment level of DUX4-driven gene expression, treatment with losmapimod resulted in a 38-fold reduction in DUX4-driven gene expression as compared to the 5.4-fold reduction seen in the placebo arm.
      • Results indicate that muscle biopsies within the higher range of DUX4-driven gene expression may be needed to observe a reduction from baseline.
      • Secondary and exploratory endpoints were not assessed as part of this analysis.
      • Remain on track to share topline results on the primary endpoint in the first quarter of 2021 and full data, including all secondary and exploratory endpoints, in the second quarter of 2021.
    • Initiated international, multicenter Phase 3 trial with losmapimod for hospitalized patients with COVID-19 (LOSVID). The trial is designed to assess the safety and efficacy of losmapimod compared to placebo for 14 days on top of standard of care in approximately 400 patients who are at risk of progression to critical illness based on older age and elevated systemic inflammation.
      • Primary endpoint is the proportion of patients treated with losmapimod as compared to placebo who progress to death or respiratory failure by day 28, and additional secondary endpoints include clinical status on days seven and 14 as measured on the nine point WHO ordinal scale of COVID-19 severity, total number of study days free of oxygen supplementation, all-cause mortality, length of hospitalization and ICU stay, adverse events and viral clearance.
      • Trial site activation underway in the United States, Mexico and South America.
      • Expect to report topline data in the first quarter of 2021.
    • On track to initiate Phase 1 trial of FTX-6058 in the fourth quarter of 2020.
      • FTX-6058 is an oral small molecule therapeutic discovered by Fulcrum and designed to induce expression of fetal hemoglobin (HbF) in red blood cells to compensate for the mutated adult hemoglobin in sickle cell disease.
      • Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin.
      • On-track to submit investigational new drug (IND) application in Q3 2020.
    • Executed strategic collaboration and license agreement in July 2020 with MyoKardia to identify therapeutics that control the expression of genes that are known to be underlying drivers of genetic cardiomyopathies.
      • Fulcrum is eligible to receive preclinical milestone payments, development milestone payments and sales milestone payments of up to $298.5 million for a first product to progress through development and commercialization and may be eligible for up to $150.0 million in milestone payments for additional targets, as well as reimbursement for the costs of the research activities.
      • Fulcrum may also be eligible to receive tiered royalty payments in the mid-single-digit to low double-digit range on net sales for any products under the collaboration that are commercialized.
    • Presented multiple posters at the 2020 American Academy of Neurology (AAN) Science on losmapimod data.
      • Identified a set of stable DUX4-regulated gene transcripts that provide a pharmacodynamic biomarker endpoint to measure the treatment effect of losmapimod in FSHD.
      • Created a standardized whole-body magnetic resonance imaging (MRI) protocol to evaluate skeletal muscle composition in FSHD patients in collaboration with AMRA Medical.
    • Completed $68.5 million private placement in June 2020, with a mix of new and existing investors, including EcoR1 Capital, LLC, Alyeska Investment Group, L.P., Boxer Capital, Casdin Capital LLC, Perceptive Advisors LLC, Samsara BioCapital, Monashee Investment Management LLC and Foresite Capital, to fund research and development expenses and other general corporate purposes.

    Second Quarter 2020 Financial Results

    • Cash Position: As of June 30, 2020, cash, cash equivalents, and marketable securities were $131.7 million, as compared to $96.7 million as of December 31, 2019. Based on its current plans, the Company expects that its existing cash, cash equivalents, and marketable securities, including the $12.5 million received from MyoKardia in July 2020, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2022.
    • R&D Expenses: Research and development expenses were $12.8 million for the second quarter of 2020, as compared to $10.9 million for the second quarter of 2020. The increase of $1.9 million was primarily due to increased personnel-related costs to support the growth of Fulcrum's research and development organization, as well as increased costs related to the advancement of losmapimod for the treatment of FSHD.
    • G&A Expenses: General and administrative expenses were $5.1 million for the second quarter of 2020, as compared to $2.6 million for the second quarter of 2019. The increase of $2.5 million was primarily due to increased costs associated with operating as a public company, as well as increased personnel-related costs to support the growth of our organization.
    • Net Loss: Net loss was $15.7 million for the second quarter of 2020, as compared to a net loss of $13.2 million for the second quarter of 2019.

    Conference Call and Webcast

    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the Company's second quarter 2020 recent business highlights and financial results, as well as the ReDUX4 interim analysis. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 9625789

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 9625789

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD.  Researchers at Fulcrum also believe that losmapimod has the potential to treat COVID-19 by reducing the acute exaggerated pro-inflammatory responses to SARS-CoV-2 infection and restoring the antigen-specific immune responses needed for viral clearance, potentially leading to improved clinical outcomes. Losmapimod has been evaluated in more than 3,600 subjects in clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the Company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD and initiating a Phase 3 trial investigating the safety, tolerability, and efficacy of losmapimod to treat hospitalized patients with COVID-19.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates filing an IND in the third quarter with initiation of a clinical trial in the fourth quarter of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the potential advantages and therapeutic potential of our product candidates, the timing of regulatory filings, initiation and enrollment of clinical trials and availability of clinical trial data, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.





    Fulcrum Therapeutics, Inc.

    Selected Consolidated Balance Sheet Data

    (In thousands)

    (Unaudited)

      June 30,

    2020
      December 31,

    2019
     
    Cash, cash equivalents, and marketable securities $131,738  $96,713 
    Working capital(1)  120,841   87,943 
    Total assets  144,862   110,439 
    Total stockholders' equity  122,131   87,153 

    ____________

    (1)      We define working capital as current assets minus current liabilities.





    Fulcrum Therapeutics, Inc.

    Consolidated Statements of Operations

    (In thousands, except per share data)

    (Unaudited)

      Three Months Ended

    June 30,
      Six Months Ended

    June 30,
     
      2020  2019  2020  2019 
    Collaboration revenue $2,000  $  $2,750  $ 
    Operating expenses:                
    Research and development  12,775   10,860   27,257   45,489 
    General and administrative  5,149   2,634   10,213   5,232 
    Total operating expenses  17,924   13,494   37,470   50,721 
    Loss from operations  (15,924)  (13,494)  (34,720)  (50,721)
    Other income, net  239   325   583   709 
    Net loss $(15,685) $(13,169) $(34,137) $(50,012)
    Cumulative convertible preferred stock dividends     (3,291)     (6,332)
    Net loss attributable to common stockholders $(15,685) $(16,460) $(34,137) $(56,344)
    Net loss per share attributable to common stockholders, basic and diluted $(0.66) $(9.21) $(1.47) $(32.85)
    Weighted average number of common shares used in net loss per share attributable to common stockholders, basic and diluted  23,854   1,787  $23,287  $1,715 





    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    cwaarich@fulcrumtx.com

    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  41. Reduction in DUX4-driven gene expression observed in biopsies with highest baseline levels of DUX4-driven gene expression

    Topline data on-track for Q1 2021 with full data in Q2 2021

    Company to review clinical data on second quarter earnings call today at 8:00am ET

    CAMBRIDGE, Mass., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced results from a pre-specified interim analysis of the primary endpoint of the Phase 2 ReDUX4 trial in subjects with facioscapulohumeral muscular dystrophy (FSHD). The primary endpoint is the reduction from baseline of DUX4-driven gene expression…

    Reduction in DUX4-driven gene expression observed in biopsies with highest baseline levels of DUX4-driven gene expression

    Topline data on-track for Q1 2021 with full data in Q2 2021

    Company to review clinical data on second quarter earnings call today at 8:00am ET

    CAMBRIDGE, Mass., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced results from a pre-specified interim analysis of the primary endpoint of the Phase 2 ReDUX4 trial in subjects with facioscapulohumeral muscular dystrophy (FSHD). The primary endpoint is the reduction from baseline of DUX4-driven gene expression in affected skeletal muscle after subjects have been treated with losmapimod or placebo. Secondary and exploratory endpoints were not assessed as part of this analysis. Results from the interim analysis in the first 29 randomized subjects indicate that DUX4-driven gene expression did not show a separation from placebo at 16 weeks.  However, in a pre-specified sensitivity analysis, those with the highest pre-treatment DUX4-driven gene expression in their muscle biopsy sample showed a 38-fold reduction in DUX4-driven gene expression following treatment with losmapimod compared to a 5.4 fold reduction with placebo.

    FSHD is a rare, progressive and disabling disease for which there are no approved treatments.  FSHD is caused by aberrant expression of DUX4 in skeletal muscle, resulting in the inappropriate presence of the DUX4 protein, which causes the death of muscle and its replacement by fat. In preparatory studies, the range of DUX4 expression levels within affected muscles throughout a patient's body have been shown to be relatively stable over time at the site of a muscle biopsy.

    "Preliminary evidence from our interim analysis suggests that muscles with higher DUX4-driven gene expression in pre-treatment biopsies show greater reduction of DUX4-driven gene expression following treatment with losmapimod compared to placebo. These results, which provide evidence of the ability of losmapimod to reduce DUX4-driven gene expression, are very encouraging," said Robert J. Gould, Ph.D., president and chief executive officer. "This initial data represents the first time a treatment is being evaluated to impact the root cause of FSHD in a placebo-controlled trial and are helping to inform our longer-term clinical strategy for losmapimod.  We look forward to further leveraging the open label study to evaluate the long-term effects of losmapimod in additional FSHD subjects. We remain on track to share topline results on the primary endpoint in the first quarter of 2021 and full data, including all secondary and exploratory endpoints, in the second quarter of 2021."

    Interim Analysis Summary

    The interim results included an analysis of the first 29 subjects who completed their 16-week biopsy out of the 80 subjects enrolled. Pharmacokinetics, demographics and the primary endpoint were assessed.  Subjects were randomized to receive an oral dose of losmapimod 15mg (n=15) or placebo (n=14) twice per day. While results showed a significant reduction in DUX4-driven gene expression in the muscle biopsies of subjects whose baseline biopsy showed the highest levels of DUX4 gene expression (38-fold decrease with losmapimod, n=3, and 5.4 fold-decrease with placebo, n=5), the population level data analysis of the reduction in DUX4-driven gene expression from all 29 subjects did not show a separation of losmapimod from placebo  (3.7 fold increase with losmapimod, n=15, and 2.8 fold increase with placebo, n=14). Results indicate that muscle biopsies within the higher levels of DUX4-driven gene expression may be needed to observe a reduction from baseline.

    Interim Analysis Results



    (All Subjects)
    Interim Analysis Results



    (Highest Expressing Muscle Biopsies)*
     Sample SizeFold change (Δ CT)Sample SizeFold change (Δ CT)
    Losmapimodn=15+3.7n=3-38
    Placebon=14+2.8n=5-5.4

    * Highest expressing muscle biopsies represent the top quartile of biopsies assessed based on baseline DUX4-driven gene expression.

    Losmapimod was generally well tolerated with no serious drug-related adverse events (SAEs) reported. The interim analysis was not powered for statistical significance and did not include individual patient level data. ReDUX4 remains blinded.

    "One of the critical factors in patients with FSHD is that there can be significant variability in the magnitude of DUX4-driven gene expression at the site of each pre-treatment needle biopsy," said Diego Cadavid, MD, Fulcrum's senior vice president, clinical development. "The initial observation of greater reduction by losmapimod over placebo in DUX4-driven gene expression in the biopsied muscles with the highest baseline expression may represent the potential losmapimod has to treat the root cause of the disease. We're excited about the study progress and look forward to the final analysis."

    About ReDUX4

    ReDUX4 is a randomized, double-blind, placebo-controlled multicenter international Phase 2b clinical trial in 80 subjects with FSHD to investigate the efficacy and safety of oral administration of losmapimod 15 mg twice per day. The primary endpoint is to evaluate the reduction of DUX4-driven gene expression in affected skeletal muscle biopsies. The original design of ReDUX4 included a muscle biopsy at week 16 during the 24-week treatment period followed by an open label extension.

    As a result of the COVID-19 pandemic, Fulcrum announced in May 2020 that the trial had been extended from 24 to 48 weeks through a protocol amendment to ensure the safety of the subjects and to allow for the opportunity for a biopsy at week 16 as originally intended or at week 36.

    The extension from 24 to 48 weeks also allows for a longer assessment in a placebo-controlled design of the skeletal muscle MRI secondary endpoint and the various exploratory clinical endpoints, such as reachable workspace, FSHD-Timed Up and GO, muscle function measures and patient reported outcomes. Topline data from approximately 80 patients is expected in the first quarter of 2021. Approximately 68 subjects remain active in the randomized portion of the trial and 12 remain active in the open label extension.

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD.  Researchers at Fulcrum also believe that losmapimod has the potential to treat COVID-19 by reducing the acute exaggerated pro-inflammatory responses to SARS-CoV-2 infection and restoring the antigen-specific immune responses needed for viral clearance, potentially leading to improved clinical outcomes. Losmapimod has been evaluated in more than 3,600 subjects in clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the Company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD and initiating a Phase 3 trial investigating the safety, tolerability, and efficacy of losmapimod to treat hospitalized patients with COVID-19.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates filing an IND in the third quarter with initiation of a clinical trial in the fourth quarter of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, such as the planned timing of submission of the Company's IND and initiation of the Company's clinical trial for FTX-6058, the potential advantages and therapeutic potential of the Company's product candidates and the timing of availability of clinical trial data. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

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  42. CAMBRIDGE, Mass., Aug. 06, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • BTIG Virtual Biotechnology Conference 2020
      Tuesday, August 11, 2020 at 1:00 p.m. ET

    • Canaccord Genuity 40th Annual Growth Conference
      Thursday, August 13, 2020 at 1:30 p.m. ET

    Live audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for 90 days after each conference…

    CAMBRIDGE, Mass., Aug. 06, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following virtual investor conferences:

    • BTIG Virtual Biotechnology Conference 2020

      Tuesday, August 11, 2020 at 1:00 p.m. ET



    • Canaccord Genuity 40th Annual Growth Conference

      Thursday, August 13, 2020 at 1:30 p.m. ET

    Live audio webcasts of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for 90 days after each conference.

    About Fulcrum Therapeutics 

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates a regulatory filing in the second half of 2020 with FTX-6058 for the treatment of sickle cell disease. 

    Please visit www.fulcrumtx.com.

    Contact:

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

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  43. CAMBRIDGE, Mass., Aug. 05, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its second quarter 2020 financial results will be released on Tuesday, August 11, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 9625789

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 9625789

    An…

    CAMBRIDGE, Mass., Aug. 05, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its second quarter 2020 financial results will be released on Tuesday, August 11, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 9625789

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 9625789

    An audio webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates a regulatory filing in the second half of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Contact:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

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  44. BRISBANE, Calif. and CAMBRIDGE, Mass., July 21, 2020 (GLOBE NEWSWIRE) -- MyoKardia, Inc. (NASDAQ:MYOK), a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases and Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced today that they have entered into a strategic collaboration and license agreement to discover, develop and commercialize novel targeted therapies for the treatment of genetic cardiomyopathies.

    Under the agreement, MyoKardia will access Fulcrum's unique, proprietary target discovery engine to identify therapeutics that…

    BRISBANE, Calif. and CAMBRIDGE, Mass., July 21, 2020 (GLOBE NEWSWIRE) -- MyoKardia, Inc. (NASDAQ:MYOK), a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases and Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced today that they have entered into a strategic collaboration and license agreement to discover, develop and commercialize novel targeted therapies for the treatment of genetic cardiomyopathies.

    Under the agreement, MyoKardia will access Fulcrum's unique, proprietary target discovery engine to identify therapeutics that control the expression of genes that are known to be underlying drivers of genetic cardiomyopathies. The collaboration focuses joint discovery efforts on certain undisclosed genetic targets. MyoKardia will be responsible for all development and commercialization activities for, and will have global rights to, any potential therapeutics identified through this collaboration.

    Fulcrum will receive a payment of $12.5 million at the close of the transaction and may be eligible to receive research, development and commercial milestone payments and additional research reimbursement of up to $302.5 million for a first product to progress through development and commercialization. If MyoKardia chooses to develop and commercialize products directed to additional targets under the collaboration, Fulcrum may be eligible for up to $150.0 million in milestone payments. Fulcrum may also be eligible to receive tiered royalty payments in the mid-single-digit to low double-digit range on net sales for any products under the collaboration that are commercialized.

    MyoKardia intends to select targets for further exploration under this collaboration informed by its integrated research and development engine, which includes capabilities in translational research, proprietary and novel disease models, clinical development, and patient engagement and identification. Potential diseases associated with such undisclosed targets are expected to share common characteristics with indications currently being pursued by MyoKardia: strong and genetically-validated mechanistic rationale, high unmet patient need, potentially efficient pathway to approval, and synergy with the commercial organization that MyoKardia is building.

    "This partnership is a natural extension of MyoKardia's investments over the last eight years in building a world-leading cardiovascular research, translational, clinical and commercial organization. We believe this collaboration will enable us to leverage our unique strengths to expand thoughtfully in identifying new therapeutic candidates for the potential treatment of heritable cardiomyopathies," said Robert S. McDowell, Ph.D., MyoKardia's Chief Scientific Officer. "We have been impressed by Fulcrum's ability to discover new biology around genetic muscle disorders. By working together, we hope to further our mission to treat patients suffering from serious cardiovascular disease."

    "This collaboration highlights the broad applicability of our product engine to discover and develop new treatments in genetically defined rare diseases with high unmet need," said Robert J. Gould, Ph.D., Fulcrum's President and Chief Executive Officer.  "We are pleased to partner with MyoKardia, a leader in the field of precision cardiovascular medicine and look forward to leveraging their unique capabilities to rapidly advance potential treatments to serve patients in urgent need."

    About MyoKardia

    MyoKardia is a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases. The company is pioneering a precision medicine approach to its discovery and development efforts by (1) understanding the biomechanical underpinnings of disease; (2) targeting the proteins that modulate a given condition; (3) identifying patient populations with shared disease characteristics; and (4) applying learnings from research and clinical studies to inform and guide pipeline growth and product advancement. MyoKardia's initial focus is on small molecule therapeutics aimed at the proteins of the heart that modulate cardiac muscle contraction to address diseases driven by excessive contraction, impaired relaxation, or insufficient contraction. Among its discoveries are three clinical-stage therapeutics: mavacamten (formerly MYK-461); danicamtiv (formerly MYK-491) and MYK-224.

    MyoKardia's mission is to change the world for people with serious cardiovascular disease through bold and innovative science.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates a regulatory filing in the second half of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    MyoKardia ContactsFulcrum Contacts
    Michelle CorralChristi Waarich
    Executive Director, Corporate Communications and Investor RelationsDirector, Investor Relations and
    MyoKardia, Inc.Corporate Communications
    650-351-4690617-651-8664 
    ir@myokardia.comcwaarich@fulcrumtx.com 
      
    Hannah Deresiewicz (investors)Stephanie Ascher (investors)
    Stern Investor Relations, Inc.Stern Investor Relations, Inc.
    212-362-1200212-362-1200
    hannah.deresiewicz@sternir.comstephanie.ascher@sternir.com
      
    Julie Normant (media)Kaitlin Gallagher (media)
    W2OBerry & Company Public Relations
    628-213-3754212-253-8881
    jnormart@w2ogroup.comkgallagher@berrypr.com
      

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  45. CAMBRIDGE, Mass., June 24, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it received early notification from the U.S. Food and Drug Administration (FDA) that the company may proceed with initiating a Phase 3, randomized, double-blind, placebo-controlled trial of losmapimod in higher risk hospitalized adults with COVID-19. Losmapimod is an orally available selective p38α/β mitogen activated protein kinase (MAPK) inhibitor.

    The LOSVID trial is a Phase 3, international, multicenter trial designed to assess the safety and efficacy of a 15 mg twice per day oral dose of losmapimod…

    CAMBRIDGE, Mass., June 24, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it received early notification from the U.S. Food and Drug Administration (FDA) that the company may proceed with initiating a Phase 3, randomized, double-blind, placebo-controlled trial of losmapimod in higher risk hospitalized adults with COVID-19. Losmapimod is an orally available selective p38α/β mitogen activated protein kinase (MAPK) inhibitor.

    The LOSVID trial is a Phase 3, international, multicenter trial designed to assess the safety and efficacy of a 15 mg twice per day oral dose of losmapimod compared to placebo for 14 days on top of standard of care in approximately 400 patients hospitalized with COVID-19 and at risk of progression to critical illness based on older age and elevated systemic inflammation. The primary endpoint is the proportion of patients who progress to death or respiratory failure by day 28. Additional secondary endpoints include clinical status on days seven and 14 as measured on the nine point WHO ordinal scale of COVID-19 severity, total number of study days free of oxygen supplementation, all-cause mortality, length of hospitalization and ICU stay, adverse events and viral clearance.

    An interim analysis will be conducted in the fourth quarter of 2020 for futility and sample size re-estimation by an independent data monitoring committee when approximately 50 percent of subjects complete the 28-day visit. Topline data is expected to be reported in the first quarter of 2021.

    "We believe that losmapimod has the potential to be a differentiated treatment option in the global fight against COVID-19," said Robert J. Gould, Ph.D., president and chief executive officer. "The speed with which we have advanced this program reflects our deep understanding of the mechanism of action of losmapimod, the pressing need for treatment options and the commitment of our team to develop therapies that result in meaningful outcomes for patients. We are encouraged by the feedback we have received from investigators, and we expect to have all sites participating in the trial to be activated in the coming weeks."

    "We look forward to conducting this important trial in parallel with our ongoing clinical program for losmapimod as a potential treatment of facioscapulohumeral muscular dystrophy (FSHD)," continued Dr. Gould.  "We have several anticipated upcoming data readouts in the next six to 12 months from both the COVID-19 and FSHD trials and we look forward to the outcomes of these trials which will tell us more about losmapimod's impact in both these patient populations. Additionally, we continue our progress with FTX-6058 towards the clinic for the potential treatment of sickle cell disease."

    Based on its mechanism of action and preclinical and clinical studies, Fulcrum believes that inhibiting the p38 MAPK pathway with losmapimod has the potential to treat COVID-19 by reducing the acute exaggerated pro-inflammatory responses to SARS-CoV-2 infection and restoring the antigen-specific immune responses needed for viral clearance potentially leading to improved clinical outcomes. 

    "Studies in elderly people indicating that p38 inhibition can decrease inflammatory responses but enhance T-cell responses to a viral antigen suggest that losmapimod could ameliorate the pathology of COVID-19 in multiple ways," said Robert Finberg, MD, Professor of Medicine, University of Massachusetts Medical School. "Losmapimod is a promising treatment candidate that could address multiple key contributors to the pathogenesis of COVID-19 and already has an extensive amount of safety and tolerability data across multiple age groups, including the elderly. The rapid initiation of this pivotal trial reflects the data supporting this research and the pressing need for effective therapies that reduce the morbidity associated with COVID-19."

    The Potential Role of p38 Inhibition in the Treatment of COVID-19

    p38 MAPK is well known as an important mediator of acute response to stress, including acute inflammation.  Multiple preclinical and clinical studies have shown that activation of the p38 MAPK significantly contributes to the pathogenesis of coronavirus infections including COVID-19. In two clinical studies reported in the literature, an oral dose of 15 mg twice per day of losmapimod in older individuals decreased inflammatory responses and enhanced normal immune responses. Additionally, in prior human clinical trials predominantly in chronic inflammatory conditions, losmapimod had an immediate effect on a number of inflammatory biomarkers that have been associated with poor prognosis in COVID-19, including C-reactive protein (CRP) and interleukin-6 (IL-6).  p38 inhibition has also been demonstrated to reduce Ang II-induced endothelial and organ damage in several experimental models and may address the renin-angiotensin system imbalance that is believed to contribute to key morbidities in COVID-19 patients.



    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD.  Researchers at Fulcrum believe that losmapimod has the potential to treat COVID-19 by reducing the acute exaggerated pro-inflammatory responses to SARS-CoV-2 infection and restoring the antigen-specific immune responses needed for viral clearance, potentially leading to improved clinical outcomes. Losmapimod has been evaluated in more than 3,600 subjects in prior clinical research across multiple other indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD and initiating a Phase 3 trial investigating the safety, tolerability, and efficacy of losmapimod to treat hospitalized patients with COVID-19.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and is advancing losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates a regulatory filing in the second half of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the Company's initiation of a clinical trial and evaluation, and the potential benefits, of losmapimod as a potential treatment for COVID-19, the development status of the Company's product candidates, including the planned timing of submission of the Company's IND for FTX-6058, and the timing of availability of clinical trial data. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Company's product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com 

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

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  46. CAMBRIDGE, Mass., June 10, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC) today announced the closing of a $68.5 million private placement pursuant to a securities purchase agreement with a group of institutional investors and accredited investors.

    The private placement includes a mix of new and existing investors, including EcoR1 Capital, LLC, Alyeska Investment Group, L.P., Boxer Capital, Casdin Capital LLC, Perceptive Advisors LLC, Samsara BioCapital, Monashee Investment Management LLC and Foresite Capital. 

    SVB Leerink acted as the exclusive placement agent to the Company in connection with the private placement.

    In the private placement, the Company sold 4,029,411 shares of common stock at a price of $17.00…

    CAMBRIDGE, Mass., June 10, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC) today announced the closing of a $68.5 million private placement pursuant to a securities purchase agreement with a group of institutional investors and accredited investors.

    The private placement includes a mix of new and existing investors, including EcoR1 Capital, LLC, Alyeska Investment Group, L.P., Boxer Capital, Casdin Capital LLC, Perceptive Advisors LLC, Samsara BioCapital, Monashee Investment Management LLC and Foresite Capital. 

    SVB Leerink acted as the exclusive placement agent to the Company in connection with the private placement.

    In the private placement, the Company sold 4,029,411 shares of common stock at a price of $17.00 per share. The private placement priced on June 8, 2020.

    The Company expects to use net proceeds from the private placement to fund research and development expenses, including the ongoing clinical development of losmapimod for facioscapulohumeral muscular dystrophy (FSHD), the initiation of clinical development of losmapimod for COVID-19 pending review of the Company's recently filed investigational new drug application by the U.S. Food and Drug Administration, the advancement of its hemoglobinopathies program into clinical development and the advancement of both its discovery efforts and product engine capabilities, as well as working capital and other general corporate purposes.

    The securities sold in the private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other applicable jurisdiction's securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions' securities laws. The Company has agreed to file a registration statement with the U.S. Securities and Exchange Commission (the "SEC") registering the resale of the shares of common stock issued in the private placement no later than the 15th day after the date of the securities purchase agreement for the private placement.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any offer, solicitation or sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful. Any offering of the securities under the resale registration statement will only be made by means of a prospectus.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Cautionary Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the anticipated use of proceeds from the private placement; the filing of a registration statement to register the resale of the shares issued and sold in the private placement; and the Company's plans, strategies and prospects for its business, including the Company's plans to evaluate losmapimod as a potential treatment for COVID-19. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials, if at all; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

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  47. Investigational New Drug (IND) application submitted; Company preparing to initiate Phase 3 trial

    ReDUX4 interim analysis in facioscapulohumeral dystrophy (FSHD) on track for Q3 2020 readout; FTX-6058 Phase 1 trial on track for Q4 2020 initiation

    Conference call scheduled for 8:30 a.m. ET today

    CAMBRIDGE, Mass., June 10, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced plans to evaluate losmapimod as a potential treatment for patients with COVID-19. Fulcrum has submitted an investigational new drug (IND) application to support initiation of a randomized…

    Investigational New Drug (IND) application submitted; Company preparing to initiate Phase 3 trial

    ReDUX4 interim analysis in facioscapulohumeral dystrophy (FSHD) on track for Q3 2020 readout; FTX-6058 Phase 1 trial on track for Q4 2020 initiation

    Conference call scheduled for 8:30 a.m. ET today

    CAMBRIDGE, Mass., June 10, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced plans to evaluate losmapimod as a potential treatment for patients with COVID-19. Fulcrum has submitted an investigational new drug (IND) application to support initiation of a randomized, placebo-controlled, Phase 3 clinical trial in hospitalized patients in the United States following Pre-IND consultation via the Coronavirus Treatment Acceleration Program (CTAP).

    "The decision to advance this development program is a reflection of the compelling science that supports losmapimod's potential in COVID-19," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "Prior clinical trials have shown its ability in other disease states to reduce acutely the inflammatory cytokines, such as C-reactive protein and IL-6, that are associated with poor prognosis. This new planned Phase 3 program adds to our robust development portfolio including potential treatments for facioscapulohumeral dystrophy (FSHD) and sickle cell disease. We are excited to apply our insights and understanding of losmapimod in COVID-19 as a potential differentiated treatment option in the global fight against this virus. I am proud of the work by our team and collaborators to rapidly advance this planned clinical program and look forward to collaborating with regulators and our outstanding team of clinical investigators to move this important effort forward as quickly as possible."

    "An extensive body of data in the literature suggests that inhibiting p38 MAP kinase may be beneficial for patients with COVID-19," said Owen B. Wallace, Ph.D., Fulcrum's chief scientific officer. "Poor prognosis for COVID-19 patients has been attributed to an exaggerated inflammatory response following SARS-CoV-2 infection. Losmapimod has been shown in preclinical and clinical studies to reduce proteins associated with acute inflammatory stress. Additionally, p38 inhibition has been reported to reduce pathophysiology associated with an activated renin-angiotensin system and positively impact the innate-adaptive immune system imbalance, both of which have been linked to increased morbidity and mortality in COVID-19.  The p38 MAPK pathway has been implicated in other viral infections, and there may be the opportunity to explore losmapimod in additional serious infections."

    The Potential Role of p38 Inhibition in the Treatment of COVID-19

    Several lines of preclinical and clinical evidence indicate that activation of the p38 mitogen-activated protein kinase (MAPK) significantly contributes to the pathogenesis of coronavirus infections including COVID-19. p38 MAPK is well known as an important mediator of acute response to stress, including acute inflammation.

    In two clinical studies reported in the literature, an oral dose of 15 mg twice per day of losmapimod in older individuals restored the normal immune response to a viral antigen challenge and demonstrated resolution of acute inflammation,  a relevant observation because the majority of severe and fatal cases of COVID-19 occur in older individuals.  Additionally, in prior human clinical trials predominantly in chronic inflammatory conditions, losmapimod had an immediate effect on a number of inflammatory biomarkers that have been associated with poor prognosis in COVID-19, including C-reactive protein (CRP) and interleukin-6 (IL-6).  In previous trials in more than 3,600 subjects, losmapimod exhibited favorable safety and tolerability not significantly different from placebo. These trials have also indicated that losmapimod had good exposure after oral dosing and robust target engagement. 

    The Company believes that losmapimod has the potential to treat COVID-19 by reducing the acute exaggerated pro-inflammatory responses to SARS-CoV-1 infection and restoring the antigen-specific immune responses needed for clearance of SARS-CoV-2, potentially leading to improved clinical outcomes. Additionally, p38 inhibition has been demonstrated to reduce angiotensin II (Ang II)-induced endothelial and organ damage in several experimental models and may address the renin-angiotensin system imbalance that is believed to contribute to key morbidities in COVID-19 patients.

    Losmapimod for FSHD

    Fulcrum's clinical program in COVID-19 is not expected to impact the timing or prioritization of other key Company milestones, including the interim analysis of ReDUX4, a randomized, double-blind, placebo-controlled multicenter international Phase 2b clinical trial in 80 subjects with FSHD. The Company remains on track to report data from this interim analysis late in the third quarter of 2020. Additionally, Fulcrum believes that it has sufficient supply of losmapimod for ongoing clinical trials in patients with FSHD, as well as for the planned Phase 3 trial in patients with COVID-19. Fulcrum owns all worldwide development and commercialization rights to losmapimod.

    FTX-6058 in Sickle Cell Disease

    FTX-6058 is an oral small molecule therapeutic discovered by Fulcrum and designed to induce expression of fetal hemoglobin (HbF) in red blood cells to compensate for the mutated adult hemoglobin in sickle cell disease. Fulcrum has completed IND-enabling studies including toxicology work with FTX-6058 and remains on track to initiate a Phase 1 trial by the end of 2020.

    Financial Expectations

    The Company expects that its cash, cash equivalents, and marketable securities of $81.2 million as of March 31, 2020, together with the $68.5 million in gross proceeds from its private placement announced today, will be sufficient to fund its operating expenses and capital expenditure requirements into the first quarter of 2022.

    Conference Call and Webcast

    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:30 a.m. ET to discuss today's developments. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number

    U.S./Canada Dial-in Number: 800-527-6973

    International Dial-in Number: 470-495-9162

    Conference ID: 3895852

    Replay Dial-in Number: 855-859-2056

    Replay International Dial-in Number: 404-537-3406

    Conference ID: 3895852

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and plans to advance losmapimod to Phase 3 for the treatment of COVID-19. Fulcrum also anticipates filing an IND in the second half of 2020 with FTX-6058 for the treatment of sickle cell disease.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the Company's planned evaluation of losmapimod as a potential treatment for COVID-19 and the potential benefits of such treatment, the development status of the Company's product candidates, including the timing of submission of the Company's IND for FTX-6058, and the timing of availability of clinical trial data. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:

    Christi Waarich

    Director, Investor Relations and

    Corporate Communications

    617-651-8664

    cwaarich@fulcrumtx.com

    Stephanie Ascher

    Stern Investor Relations, Inc.

    stephanie.ascher@sternir.com

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  48. CAMBRIDGE, Mass., May 21, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced multiple presentations at the 2020 American Academy of Neurology (AAN) Science Highlights Virtual Platform. AAN's annual meeting was cancelled due to COVID-19 and the virtual platform allows for all accepted oral and poster presentations to be presented. The materials linked below will be available for approximately 10 months. 

    "We are pleased by the breadth of losmapimod data selected for presentation, which highlights the progress we've made to develop a treatment for patients with facioscapulohumeral muscular…

    CAMBRIDGE, Mass., May 21, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced multiple presentations at the 2020 American Academy of Neurology (AAN) Science Highlights Virtual Platform. AAN's annual meeting was cancelled due to COVID-19 and the virtual platform allows for all accepted oral and poster presentations to be presented. The materials linked below will be available for approximately 10 months. 

    "We are pleased by the breadth of losmapimod data selected for presentation, which highlights the progress we've made to develop a treatment for patients with facioscapulohumeral muscular dystrophy (FSHD) and our approach to treating the root cause of genetically defined rare diseases," said Diego Cadavid, M.D., Fulcrum's senior vice president, clinical development. "In particular, we have identified a set of stable DUX4-regulated gene transcripts that provide a pharmacodynamic biomarker endpoint to measure the treatment effect of losmapimod in FSHD. Additionally, in collaboration with AMRA Medical, we have created a standardized whole-body magnetic resonance imaging (MRI) protocol to evaluate skeletal muscle composition in FSHD patients.  I would like to thank the patients and their families who have participated in our trials, and who inspire us every day."

    2020 AAN Virtual Platform Presentation and Poster Details

    Phase 1 Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD): Safety, Tolerability, and Target Engagement

    https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2192

    Design of a Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD): ReDUX4

    https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2193

    Development of an Optimized Timed Up and Go (oTUG) for Measurement of Changes in Mobility Impairment in Facioscapulohumeral Muscular Dystrophy (FSHD) Clinical Trials

    https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/1638

    A Biomarker of DUX4 Activity to Evaluate losmapimod Treatment Effect in FSHD Phase 2 Trials

    https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2196

    An In-Home Study of Facioscapulohumeral Muscular Dystrophy (FSHD) Patients using Contactless Wireless Sensing and Machine Learning

    https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/1721

    Development and Evaluation of a Whole-body MRI Imaging Protocol and Analysis Algorithms to Measure Changes in Skeletal Muscle in FSHD

    https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2176

    About FSHD
    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms, and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod
    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements
    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:
    Christi Waarich
    Director, Investor Relations and
    Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com
    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations
    kgallagher@berrypr.com
    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  49. CAMBRIDGE, Mass., May 13, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the first quarter of 2020.

    "Despite these unprecedented times, the first quarter was a period of important progress for Fulcrum and the fundamentals of our business remain strong," said Robert J. Gould, Ph.D., president and chief executive officer. "I am proud of our continued commitment to patients and the dedication demonstrated by our employees in these challenging times. In the wake of COVID-19, a number of our clinical trial sites postponed their activities…

    CAMBRIDGE, Mass., May 13, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the first quarter of 2020.

    "Despite these unprecedented times, the first quarter was a period of important progress for Fulcrum and the fundamentals of our business remain strong," said Robert J. Gould, Ph.D., president and chief executive officer. "I am proud of our continued commitment to patients and the dedication demonstrated by our employees in these challenging times. In the wake of COVID-19, a number of our clinical trial sites postponed their activities. Our team acted swiftly to minimize the health risks to patients, families and healthcare professionals involved in our studies of losmapimod in the treatment of facioscapulohumeral dystrophy (FSHD). We amended our Phase 2b trial, ReDUX4, to extend the trial from 24 to 48 weeks, added an interim analysis for subjects who underwent their 16-week biopsy as originally planned, and added a 36-week biopsy for patients who cannot undergo their 16-week biopsy. We believe these changes will enable patients and investigators to continue participation in the trial and will allow us to collect the essential data needed to evaluate the potential efficacy and safety of losmapimod for the treatment of FSHD."

    Recent Business Highlights

    • Amended ReDUX4, a Phase 2b trial of losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor, to accommodate COVID-19 impact; extended the trial from 24 to 48 weeks, adding a 36-week biopsy for those subjects unable to undergo their 16-week biopsy, and included an interim analysis on patients who completed their 16-week biopsy as planned in the original protocol.
      • ReDUX4 enrollment completed.
      • ReDUX4 open label extension initiated.
      • Data from interim analysis expected in the third quarter of 2020.
    • Received U.S. and European Orphan Designation for losmapimod in FSHD.
    • Completed investigational new drug application (IND)-enabling studies, including toxicology work with FTX-6058.
      • Remain on track to initiate Phase 1 trial in the second half of 2020.
      • FTX-6058 is an oral small molecule therapeutic discovered by Fulcrum and designed to induce expression of fetal hemoglobin (HbF) in red blood cells to compensate for the mutated adult hemoglobin in sickle cell disease.
    • Presented evidence of dose-dependent target engagement observed in skeletal muscle with losmapimod 15 mg twice per day in a Phase 1 trial during the Muscular Dystrophy Association virtual clinical trials session.
      • Builds on previously announced dose-dependent pharmacokinetics and target engagement in blood.
    • Continued evolution of Fulcrum's proprietary product engine, which is designed to identify drug targets, programs and clinical development candidates in a broad range of genetically defined diseases (FulcrumSeek), and initiated research activities under the Acceleron collaboration.

    ReDUX4 Trial Amendment
    ReDUX4 is a randomized, double-blind, placebo-controlled multicenter international Phase 2b clinical trial in 80 subjects with FSHD to investigate the efficacy and safety of oral administration of losmapimod 15 mg twice per day. The primary endpoint is to evaluate the reduction of DUX4-driven gene expression in affected skeletal muscle biopsies. The original design of ReDUX4 included a muscle biopsy at week 16 during the 24-week treatment period followed by an open label extension. Twelve of the 80 subjects have completed the 24-week treatment period and rolled over to the open label extension portion of the trial.

    As a result of the COVID-19 pandemic, Fulcrum has extended the trial from 24 to 48 weeks through a protocol amendment to ensure the safety of the subjects and to allow for the opportunity for a biopsy at week 16 as originally intended or at week 36. Approximately 68 subjects who did not complete the original 24-week treatment period remain active in the randomized portion of the trial. An interim analysis of approximately 25 subjects who completed their 16-week biopsy will be conducted. The Company expects to report data from this interim analysis in the third quarter of 2020. The extension from 24 to 48 weeks also allows for a longer assessment in a placebo-controlled design of the skeletal muscle MRI secondary endpoint and the various exploratory clinical endpoints, such as reachable workspace, FSHD-Timed Up and GO, muscle function measures and patient reported outcomes. Topline data from approximately 80 patients is expected in the first quarter of 2021. Fulcrum believes the amendment to the trial provides flexibility to address the challenges presented by the COVID-19 pandemic and supports collection of efficacy and safety data to support continued discussions with health authorities regarding potential registration strategies.

    First Quarter 2020 Financial Results

    • Cash Position: As of March 31, 2020, cash, cash equivalents, and marketable securities were $81.2 million, as compared to $96.7 million as of December 31, 2019. Based on its current plans, the Company expects that its existing cash, cash equivalents and marketable securities will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the third quarter of 2021.
    • R&D Expenses: Research and development expenses were $14.5 million for the first quarter of 2020, as compared to $34.6 million for the first quarter of 2019. Research and development expenses for the first quarter of 2019 include $25.6 million of one-time costs incurred associated with the issuance of Series B convertible preferred stock under the Company's license agreement with GSK for losmapimod. Excluding these one-time costs, the increase of $5.5 million was primarily due to increased costs related to the advancement of losmapimod for the treatment of FSHD, as well as increased personnel-related costs to support the growth of Fulcrum's research and development organization.
    • G&A Expenses: General and administrative expenses were $5.1 million for the first quarter of 2020, as compared to $2.6 million for the first quarter of 2019. The increase of $2.5 million was primarily due to increased personnel-related costs to support the growth of our organization, as well as increased consulting and professional fees associated with operating as a public company.
    • Net Loss: Net loss was $18.5 million for the first quarter of 2020, as compared to a net loss of $36.8 million for the first quarter of 2019.

    Conference Call and Webcast
    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the Company's first quarter 2020 recent business highlights and financial results. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 8297069

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 8297069

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The Company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive preclinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the timing of availability of clinical trial data and initiation of clinical trials, and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.



    Fulcrum Therapeutics, Inc.

    Selected Consolidated Balance Sheet Data

    (In thousands)

    (Unaudited)

      March 31,
    2020
        December 31,
    2019
     
    Cash, cash equivalents, and marketable securities $ 81,207     $ 96,713  
    Working capital(1)   70,141       87,943  
    Total assets   94,856       110,439  
    Total stockholders' equity   70,631       87,153  
    ____________              
    (1)     We define working capital as current assets minus current liabilities.              
                   



     Fulcrum Therapeutics, Inc.

    Consolidated Statements of Operations

    (In thousands, except per share data)

    (Unaudited)

      Three Months Ended
    March 31,
     
      2020     2019  
    Collaboration revenue $ 750     $  
    Operating expenses:              
    Research and development   14,482       34,629  
    General and administrative   5,064       2,598  
    Total operating expenses   19,546       37,227  
    Loss from operations   (18,796 )     (37,227 )
    Other income, net   344       384  
    Net loss $ (18,452 )   $ (36,843 )
    Cumulative convertible preferred stock dividends         (3,041 )
    Net loss attributable to common stockholders $ (18,452 )   $ (39,884 )
    Net loss per share attributable to common stockholders, basic and diluted $ (0.81 )   $ (24.29 )
    Weighted average number of common shares used in net loss per share attributable to common stockholders, basic and diluted   22,719       1,642  
                   


    Contact:

    Investors:
    Christi Waarich
    Director, Investor Relations and
    Corporate Communications
    cwaarich@fulcrumtx.com
    617-651-8664

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com
    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations
    kgallagher@berrypr.com
    212-253-8881

    Primary Logo

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  50. CAMBRIDGE, Mass., May 11, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in a fireside chat at the virtual BofA Securities 2020 Health Care Conference on May 14, 2020 at 1:40 p.m. ET.

    A live audio webcast will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. An archived replay will be available on the Company's website for 90 days.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with…

    CAMBRIDGE, Mass., May 11, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in a fireside chat at the virtual BofA Securities 2020 Health Care Conference on May 14, 2020 at 1:40 p.m. ET.

    A live audio webcast will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. An archived replay will be available on the Company's website for 90 days.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Contact:
    Christi Waarich
    Director, Investor Relations
    and Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Primary Logo

    View Full Article Hide Full Article
  51. CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its first quarter 2020 financial results will be released on Wednesday,  May 13, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 8297069

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 8297069

    An audio…

    CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its first quarter 2020 financial results will be released on Wednesday,  May 13, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 8297069

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 8297069

    An audio webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Contact:
    Christi Waarich
    Director, Investor Relations and
    Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Primary Logo

    View Full Article Hide Full Article
  52. CAMBRIDGE, Mass., April 13, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it is on track for submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the second half of 2020 for FTX-6058 following the completion of preclinical safety studies and Good Laboratory Practices (GLP) toxicology work.

    "Completed preclinical studies illustrate that FTX-6058, a small molecule upregulator of fetal hemoglobin, has the potential to provide distinct advantages over biologics and gene therapies currently being used or developed for the treatment…

    CAMBRIDGE, Mass., April 13, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced it is on track for submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the second half of 2020 for FTX-6058 following the completion of preclinical safety studies and Good Laboratory Practices (GLP) toxicology work.

    "Completed preclinical studies illustrate that FTX-6058, a small molecule upregulator of fetal hemoglobin, has the potential to provide distinct advantages over biologics and gene therapies currently being used or developed for the treatment of sickle cell disease," said Owen B. Wallace, Ph.D., Fulcrum's chief scientific officer. "Based on its attractive profile in preclinical in vitro and in vivo models and its preclinical safety profile, we believe that FTX-6058 has the potential to be an effective oral therapy for people living with sickle cell disease." 

    In pre-clinical studies, treatment with FTX-6058 was shown to significantly increase HbF levels up to approximately 30% of total hemoglobin as measured by HPLC and mass spectrometry methods in erythroid progenitor cells from multiple human donors. FTX-6058 also elevated HbF in vivo in efficacy models at plasma concentrations reasonably expected to be achieved in humans. Fulcrum believes these results indicate that FTX-6058 could play a role in alleviating the burden of disease in people living with this devastating disorder.

    About Sickle Cell Disease
    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements
    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, including the timing of submission of the Company's IND. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:
    Christi Waarich
    Director, Investor Relations and
    Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com
    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations
    kgallagher@berrypr.com
    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  53. CAMBRIDGE, Mass., April 02, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update in the context of the developing situation with the COVID-19 (coronavirus) pandemic.

    "During the unprecedented challenges we all face with the SARS-CoV-2 pandemic, we have recently implemented plans to protect the safety, health and well-being of the patients, families and healthcare professionals involved in our clinical development programs, as well as our employees," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "A number of our clinical trial sites are temporarily…

    CAMBRIDGE, Mass., April 02, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update in the context of the developing situation with the COVID-19 (coronavirus) pandemic.

    "During the unprecedented challenges we all face with the SARS-CoV-2 pandemic, we have recently implemented plans to protect the safety, health and well-being of the patients, families and healthcare professionals involved in our clinical development programs, as well as our employees," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "A number of our clinical trial sites are temporarily postponing trial-related activities in the wake of COVID-19. While we are fully focused on moving our clinical programs forward, Fulcrum supports this temporary reallocation of resources to ensure hospitals and healthcare workers can focus on meeting the needs of patients with COVID-19. I am confident in Fulcrum's ability to face this challenge with tenacity, humility, and spirit. FSHD is an area of tremendous unmet need, we remain as eager as ever to continue to advance a therapy that may impact patients."

    Losmapimod for Facioscapulohumeral Muscular Dystrophy (FSHD) Program Update

    • The Company's ReDUX4 trial is fully enrolled. As a result of the suspension of clinical-trial activity by a number of the Company's clinical trial sites stemming from the pandemic, the Company is currently assessing the impact to the ReDUX4 clinical trial, including whether it will have topline data for the primary endpoint by the end of the third quarter of 2020 as previously disclosed. Fulcrum plans to provide a further update when it has more clarity. ReDUX4 is an international, multicenter, randomized, Phase 2b double-blind, placebo-controlled, 24-week trial of losmapimod in 80 patients with genetically confirmed facioscapulohumeral muscular dystrophy (FSHD).

    Employee Safety

    • Fulcrum has instituted a mandatory work-from-home policy for the majority of its employees. The duration of this remote working arrangement will be guided by the direction of the Commonwealth of Massachusetts and actions and guidelines issued by the U.S. federal government, including the Centers for Disease Control and Prevention.
    • Due to the nature of Fulcrum's work, essential-work exemptions continue to permit critical research and development and laboratory activities for limited personnel. Those exemptions enable some continued discovery research and activities. For these employees, Fulcrum has established a set of safety guidelines to reduce close interactions, including limiting the number of people on-site.

    About FSHD
    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod
    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements
    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates and the timing of availability of clinical trial data. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks relating to the COVID-19 pandemic; risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:
    Christi Waarich
    Director, Investor Relations and
    Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com
    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations
    kgallagher@berrypr.com
    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  54. – Losmapimod achieves target engagement in muscle –

    – Oral presentation at Virtual Muscular Dystrophy Association (MDA) Clinical and Scientific Conference highlights safety, tolerability and pharmacokinetics of losmapimod –

    CAMBRIDGE, Mass., March 24, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced complete results from its Phase 1 clinical trial of losmapimod, which is being developed for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Results were presented by Michelle Mellion, M.D., the Company's senior medical director, in a live Virtual Clinical Trial Session of…

    – Losmapimod achieves target engagement in muscle –

    – Oral presentation at Virtual Muscular Dystrophy Association (MDA) Clinical and Scientific Conference highlights safety, tolerability and pharmacokinetics of losmapimod –

    CAMBRIDGE, Mass., March 24, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced complete results from its Phase 1 clinical trial of losmapimod, which is being developed for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Results were presented by Michelle Mellion, M.D., the Company's senior medical director, in a live Virtual Clinical Trial Session of the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference today. Presented material is available on Fulcrum's website at fulcrumtx.com. 

    The primary objective of the Phase 1 trial was to investigate the safety and tolerability of losmapimod in healthy volunteers and in FSHD patients. The secondary objective was to evaluate repeated dose pharmacokinetics (PK) and target engagement (TE), measured by phosphorylated and total HSP27, in FSHD patients in blood and muscle. In the first cohort, 10 healthy volunteers were randomized to a single oral dose of losmapimod (n=8) 7.5 mg followed by a single oral dose of 15 mg after a wash out period or to single oral dose placebo (n=2) in both dosing periods. In the second cohort, 15 FSHD patients were randomized and treated with placebo (n=3) or losmapimod 7.5 mg (n=6) or 15 mg (n=6) taken orally twice daily for 14 days. The third cohort was open label with five FSHD patients treated with losmapimod 15 mg twice daily for 14 days. Biopsies of normal appearing (second cohort) and actively involved (STIR+) muscle (third cohort) were performed at baseline and during treatment.

    Dose-dependent and sustained target engagement was observed in blood with losmapimod.  Evidence of target engagement was also observed in skeletal muscle biopsies. Consistent with previously reported safety data, losmapimod was well tolerated with no serious adverse events (SAEs). Similar tolerability, safety and PK were observed in healthy volunteers and patients with FSHD at the two doses examined. This safety data is consistent with previously reported data from more than 3,500 healthy volunteers and patients treated with losmapimod across multiple other indications.

    These data support the selection of the 15 mg dose of losmapimod taken orally twice daily in the Company's ongoing Phase 2b randomized, double-blind, placebo-controlled 24-week clinical trial, ReDUX4, as well as its Phase 2 open-label study. Both Phase 2 trials completed enrollment in February 2020.

    Fulcrum plans to hold a separate webcast event in the Spring to discuss the abstracts that were accepted for poster presentation at the Muscular Dystrophy Association meeting which was postponed due to the COVID-19 pandemic. Further details will be announced in the coming weeks.

    About FSHD
    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod
    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements
    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the timing of availability of clinical trial data and the Company's planned presentations. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:
    Christi Waarich
    Director, Investor Relations and
    Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com
    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations
    kgallagher@berrypr.com
    212-253-8881

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  55. CAMBRIDGE, Mass., March 05, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the fourth quarter and full year of 2019.

    "We made important progress at a rapid pace in 2019 as we transitioned from a private, discovery company into a public, clinical-stage company," said Robert J. Gould, president and chief executive officer. "In our losmapimod program for patients with facioscapulohumeral muscular dystrophy (FSHD), we completed enrollment in our Phase 2 clinical trials, including ReDUX4, the randomized, double-blind placebo-controlled…

    CAMBRIDGE, Mass., March 05, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the fourth quarter and full year of 2019.

    "We made important progress at a rapid pace in 2019 as we transitioned from a private, discovery company into a public, clinical-stage company," said Robert J. Gould, president and chief executive officer. "In our losmapimod program for patients with facioscapulohumeral muscular dystrophy (FSHD), we completed enrollment in our Phase 2 clinical trials, including ReDUX4, the randomized, double-blind placebo-controlled multicenter international Phase 2b clinical trial, and we continue to expect to announce topline data on the primary endpoint in the third quarter of 2020. We also plan to submit an IND for FTX-6058 for the treatment of sickle cell disease in the second half of this year. We look forward to building on this progress as we execute on our goal of advancing therapies focused on improving the lives of patients with genetically defined diseases."

    Recent Business Highlights

    • Evidence of dose-dependent target engagement observed in skeletal muscle with losmapimod in Phase 1.
      • Builds on previously announced dose-dependent pharmacokinetics and target engagement in blood.
    • Completed patient enrollment in ReDUX4 Phase 2b trial of losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor.
      • Phase 2 Open Label Study also completed enrollment
      • ReDUX4 open label extension initiated
      • Remain on track to report topline data in the third quarter of 2020
    • Received U.S. Orphan Drug Designation for losmapimod in FSHD.
    • Presented Phase 1 clinical data at the World Muscle Society meeting highlighting the safety, tolerability, and target engagement of losmapimod for the treatment of FSHD.
    • Plan to submit an investigational new drug application (IND) to the U.S. Food and Drug Administration for FTX-6058 in the second half of 2020.
      • FTX-6058 is an oral small molecule therapeutic discovered by Fulcrum and designed to induce expression of fetal hemoglobin (HbF) in red blood cells to compensate for the mutated adult beta hemoglobin in sickle cell disease.
    • Expanded patent portfolio.
      • U.S. patent 10,537,560 covers the use of other p38 kinase inhibitors for the treatment of FSHD. This is in addition to U.S. patent 10,342,786 which covers the method of using losmapimod for the treatment of FSHD. These two patents each provide protection through 2038.
    • Executed a pulmonary research and discovery collaboration agreement with Acceleron Pharma, Inc.
    • Multiple abstracts accepted at the Muscular Dystrophy Association and American Academy of Neurology Annual Meetings this Spring.
    • Continued evolution of Fulcrum's proprietary product engine to identify drug targets, programs and clinical development candidates in a broad range of genetically defined diseases (FulcrumSeek).
    • Appointed Katina Dorton to the Company's Board of Directors.

    Fourth Quarter and Full Year 2019 Financial Results

    • Cash Position: As of December 31, 2019, cash and cash equivalents were $96.7 million, as compared to $72.8 million as of December 31, 2018. Based on its current plans, the Company expects that its existing cash and cash equivalents will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the third quarter of 2021.

    • R&D Expenses: Research and development expenses were $12.1 million for the fourth quarter of 2019, as compared to $6.9 million for the fourth quarter of 2018. The increase of $5.2 million was primarily due to increased costs related to the advancement of losmapimod for the treatment of FSHD, including increased external costs to support Fulcrum's ongoing and planned clinical trials, as well as increased personnel-related costs due to additional headcount to support the growth of Fulcrum's research and development organization.

      Research and development expenses were $71.1 million for the year ended December 31, 2019, as compared to $25.2 million for the year ended December 31, 2018. The increase of $45.9 million was primarily due to a $28.1 million increase in one-time costs associated with the Company's license agreement with GSK, including $25.6 million of costs associated with the issuance of Series B convertible preferred stock to GSK during 2019 and a $2.5 million milestone paid to GSK during 2019, increased costs related to the advancement of losmapimod for the treatment of FSHD, including increased external costs to support Fulcrum's ongoing and planned clinical trials, as well as increased personnel-related costs associated with increased headcount to support the growth of Fulcrum's research and development organization.
    • G&A Expenses: General and administrative expenses were $4.4 million for the fourth quarter of 2019, as compared to $2.4 million for the fourth quarter of 2018. The increase of $2.0 million was primarily due to increased personnel-related costs associated with increased headcount to support the growth of our organization, as well as increased consulting and professional fees associated with operating as a public company, including costs for insurance premiums, legal services, investor relations, and accounting.

      General and administrative expenses were $13.1 million for the year ended December 31, 2019, as compared to $8.3 million for the year ended December 31, 2018. The increase of $4.8 million was primarily due to increased personnel-related costs associated with increased headcount to support the growth of our organization, as well as increased consulting and professional fees associated with operating as a public company, including costs for insurance premiums, legal services, investor relations, and accounting.
    • Net Loss: Net loss was $16.1 million for the fourth quarter of 2019, as compared to a net loss of $8.9 million for the fourth quarter of 2018. Net loss was $82.7 million for the year ended December 31, 2019, as compared to $32.6 million for the year ended December 31, 2018.

    Conference Call and Webcast
    Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the Company's fourth quarter and full year 2019 financial results and recent developments. The webcast will be accessible through the Investor Relations section of Fulcrum's website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 3683902

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 3683902

    About FSHD

    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod

    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Sickle Cell Disease

    Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The Company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates, the timing of availability of clinical trial data and the Company's ability to fund its operations with cash on hand. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Fulcrum Therapeutics, Inc.

    Selected Consolidated Balance Sheet Data

    (In thousands)

    (Unaudited)

        December 31,
    2019
        December 31,
    2018
     
           
        (in thousands)  
    Consolidated Balance Sheet Data:                
    Cash and cash equivalents   $ 96,713     $ 72,797  
    Working capital(1)     87,943       69,866  
    Total assets     110,439       85,771  
    Convertible preferred stock           139,670  
    Total stockholders' equity (deficit)     87,153       (63,670 )

     

    (1) We define working capital as current assets minus current liabilities.


    Fulcrum Therapeutics, Inc.

    Consolidated Statements of Operations and Comprehensive Loss

    (In thousands, except per share data)

    (Unaudited)

        Three Months Ended
    December 31,
        Year Ended
    December 31,
     
        2019     2018     2019     2018  
    Collaboration revenue   $     $     $     $  
    Operating expenses:                                
    Research and development     12,087       6,860       71,072       25,184  
    General and administrative     4,403       2,391       13,145       8,314  
    Total operating expenses     16,490       9,251       84,217       33,498  
    Loss from operations     (16,490 )     (9,251 )     (84,217 )     (33,498 )
    Other income, net:                                
    Interest income, net     360       385       1,511       518  
    Other income     7       7       29       392  
    Net loss and comprehensive loss   $ (16,123 )   $ (8,859 )   $ (82,677 )   $ (32,588 )
    Cumulative convertible preferred stock dividends           (2,823 )     (7,128 )     (6,559 )
    Net loss attributable to common stockholders   $ (16,123 )   $ (11,682 )   $ (89,805 )   $ (39,147 )
    Net loss per share attributable to common stockholders, basic and diluted   $ (0.71 )   $ (7.82 )   $ (8.13 )   $ (31.14 )
    Weighted average number of common shares used in net loss per share attributable to common stockholders, basic and diluted     22,610       1,493       11,046       1,257  

    Contact:

    Investors:
    Christi Waarich
    Director, Corporate Communications and Investor Relations
    cwaarich@fulcrumtx.com
    617-651-8664

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com
    212-362-1200

    Media:
    Lynn Granito
    Berry & Company Public Relations
    lgranito@berrypr.com
    212-253-8881

     

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  56. CAMBRIDGE, Mass., Feb. 28, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the company's support for Rare Disease Day 2020 and the importance of changing perceptions about rare diseases that continue to make patients and families feel isolated and unable to access information and support.

    "In our work developing therapies to treat genetically defined rare diseases, including facioscapulohumeral muscular dystrophy (FSHD) and sickle cell disease (SCD), we have met with so many patients and caregivers who share their challenges in accessing information and support for a rare disease…

    CAMBRIDGE, Mass., Feb. 28, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the company's support for Rare Disease Day 2020 and the importance of changing perceptions about rare diseases that continue to make patients and families feel isolated and unable to access information and support.

    "In our work developing therapies to treat genetically defined rare diseases, including facioscapulohumeral muscular dystrophy (FSHD) and sickle cell disease (SCD), we have met with so many patients and caregivers who share their challenges in accessing information and support for a rare disease," said Robert J. Gould, Ph.D., president and chief executive officer of Fulcrum Therapeutics. "We applaud EURORDIS, the organization who created Rare Disease Day, for highlighting the need for all of us to take steps to ‘Reframe Rare' in the years ahead."

    To commemorate Rare Disease Day 2020, Fulcrum sponsored a campaign throughout the month of February honoring patients and families and inviting them to share their perspectives about the best ways to change the dialogue about rare diseases. Their insights including photographs and quotations were shared on the company's Instagram, Twitter and LinkedIn profiles. Members of the Fulcrum team also presented their thoughts on the best ways to support patients and make a difference for people affected by rare diseases. In addition to the social media campaign, Dr. Gould and one of Fulcrum's patient advocate participants will participate in a panel discussion during the MassBio program on Rare Disease Day to be held at Omni Parker House in Boston.

    "People have so many misconceptions about rare diseases – if they know about them at all. The effort to build awareness and change attitudes about these diseases can have a very positive impact on patients and families all around the world," said Lexi Pappas, FSHD patient advocate. "I am glad to be able to share my experience with the team at Fulcrum and to join in their efforts to help reframe discussions about rare diseases in ways that can lead to better treatment and support."

    Established by EURORDIS in 2008, Rare Disease Day is recognized on the last day of February each year to raise awareness of rare diseases and their impact on patients, caregivers and communities. This year, Rare Disease Day falls on February 29 and will focus on reframing what it means to be rare by promoting the message that "rare is many, rare is strong and rare is proud."

     Fulcrum Therapeutics invites all people who support families affected by FSHD, SCD and other rare diseases to join in the effort to build awareness by using the hashtags #RareDiseaseDay, #ReframeRare, #FSHD, #SCD and #SickleCellWarriors on social media during Rare Disease Day.

    About Fulcrum Therapeutics

    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit http://www.fulcrumtx.com.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission.

    Investor Contacts:

    Christi Waarich
    Director, Corporate Communications and Investor Relations
    cwaarich@fulcrumtx.com 
    650-483-1970

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com 
    212-362-1200

    Media Contact:
    Lynn Granito
    Berry & Company Public Relations
    lgranito@berrypr.com 
    212-253-8881

    Primary Logo

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    • Topline data on track for 3Q 2020
    • Dosing Initiated in ReDUX4 Open Label Extension

    CAMBRIDGE, Mass., Feb. 26, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has completed patient enrollment in ReDUX4, its double blind, placebo-controlled, international Phase 2b trial evaluating the efficacy and safety of losmapimod in patients with facioscapulohumeral muscular dystrophy (FSHD). An open label extension for participants in ReDUX4 who have completed 24 weeks of dosing has also initiated, providing the opportunity for patients randomized to losmapimod to continue treatment and…

    • Topline data on track for 3Q 2020
    • Dosing Initiated in ReDUX4 Open Label Extension

    CAMBRIDGE, Mass., Feb. 26, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has completed patient enrollment in ReDUX4, its double blind, placebo-controlled, international Phase 2b trial evaluating the efficacy and safety of losmapimod in patients with facioscapulohumeral muscular dystrophy (FSHD). An open label extension for participants in ReDUX4 who have completed 24 weeks of dosing has also initiated, providing the opportunity for patients randomized to losmapimod to continue treatment and for patients randomized to placebo to initiate treatment with losmapimod.

    "We want to express our gratitude to all the patients, investigators and caregivers participating in this global trial," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "Completing patient recruitment for ReDUX4 is a testament to the commitment of the patient community, clinical investigators, our partners, and our Fulcrum team to address the critical needs of patients with FSHD and is a significant milestone. The root cause of FSHD is the aberrant activity of the DUX4 transcriptional program and we believe, as independent research indicates, that any reduction may be beneficial for patients. We remain on track to report topline data on the primary endpoint in the third quarter of this year."

    About ReDUX4
    ReDUX4 is an international, multicenter, randomized, Phase 2b double-blind, placebo-controlled, 24-week trial of losmapimod in approximately 80 patients with genetically confirmed FSHD. The primary endpoint is the change from baseline on DUX4 activity in affected skeletal muscle. The open label extension allows patients randomized to losmapimod to continue treatment and for patients randomized to placebo to initiate treatment with losmapimod.

    In addition, Fulcrum finished enrollment in the Phase 2, 52-week open label study.

    About FSHD
    FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.

    FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned "on" as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

    About Losmapimod
    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Forward-Looking Statements
    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the development status of the Company's product candidates and the timing of availability of clinical trial data. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contact:

    Investors:
    Christi Waarich
    Director, Investor Relations and
    Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Stephanie Ascher
    Stern Investor Relations, Inc.
    stephanie.ascher@sternir.com
    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations
    kgallagher@berrypr.com
    212-253-8881

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  57. CAMBRIDGE, Mass., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its fourth quarter and full year 2019 financial results will be released on Thursday, March 5, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 3683902

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID…

    CAMBRIDGE, Mass., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its fourth quarter and full year 2019 financial results will be released on Thursday, March 5, 2020 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an update on recent corporate developments.

    Dial-in Number
    U.S./Canada Dial-in Number: 800-527-6973
    International Dial-in Number: 470-495-9162
    Conference ID: 3683902

    Replay Dial-in Number: 855-859-2056
    Replay International Dial-in Number: 404-537-3406
    Conference ID: 3683902

    An audio webcast will be accessible through the Investor Relations section of the company's website https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Contact:
    Christi Waarich
    Director, Corporate Communications
    and Investor Relations
    617-651-8664
    cwaarich@fulcrumtx.com

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  58. CAMBRIDGE, Mass., Feb. 20, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following investor conferences:

    • Leerink Global Healthcare Conference
      Thursday, February 27, 2020 at 9:30 a.m. ET
      Location: New York

    • Cowen and Company Annual Health Care Conference
      Monday, March 2, 2020 at 3:30 p.m. ET
      Location: Boston, MA

    A live audio webcast of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for…

    CAMBRIDGE, Mass., Feb. 20, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that management will participate in the following investor conferences:

    • Leerink Global Healthcare Conference
      Thursday, February 27, 2020 at 9:30 a.m. ET
      Location: New York

    • Cowen and Company Annual Health Care Conference
      Monday, March 2, 2020 at 3:30 p.m. ET
      Location: Boston, MA

    A live audio webcast of the presentations will be available through the Investor Relations section of the Fulcrum website at https://ir.fulcrumtx.com/events-and-presentations. Archived replays will be available on the Company's website for 90 days after each conference.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and has completed extensive pre-clinical research for FTX-6058 for the treatment of sickle cell disease and beta-thalassemia.

    Please visit www.fulcrumtx.com.

    Contact:
    Christi Waarich
    Director, Investor Relations
    and Corporate Communications
    617-651-8664
    cwaarich@fulcrumtx.com

    Primary Logo

    View Full Article Hide Full Article
  59. CAMBRIDGE, Mass., Jan. 29, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum also announced the issuance of U.S. patent 10,537,560 with claims covering the use of other p38 kinase inhibitors for the treatment of FSHD.

    "We are pleased to have been granted Orphan Drug Designation for losmapimod in FSHD as it underscores the critical…

    CAMBRIDGE, Mass., Jan. 29, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to losmapimod, a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum also announced the issuance of U.S. patent 10,537,560 with claims covering the use of other p38 kinase inhibitors for the treatment of FSHD.

    "We are pleased to have been granted Orphan Drug Designation for losmapimod in FSHD as it underscores the critical need for treating this rare muscular dystrophy that has no approved therapies," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "We believe losmapimod represents a promising, novel approach to treat the known root cause of FSHD and remain on-track to announce data from the Phase 2b ReDUX4 clinical trial in the third quarter of 2020. Our recently issued patent also expands our intellectual property protection relating to the use of other clinical-stage p38 inhibitors for the treatment of FSHD, strengthening our position as a leader in the treatment of genetically defined diseases."

    In October 2019, the Company announced preliminary results from a Phase 1 clinical trial of losmapimod in patients with FSHD, which indicated that losmapimod was generally well tolerated and achieved dose-dependent concentrations in plasma and muscle believed to be adequate for efficacy based on preclinical pharmacology studies. Additionally, losmapimod has shown adequate safety and tolerability in over 3,500 patients and healthy volunteers across multiple indications, with no safety signals attributed to the drug in those trials.

    The patent announced today is in addition to U.S. patent 10,342,786, which covers the method of using losmapimod for the treatment of FSHD. These two patents each provide protection through 2038.

    About Orphan Drug Designation
    The FDA Office of Orphan Products Development grants orphan status to support development of medicines that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval if received, exemption of FDA application fees and tax credits for qualified clinical trials. For more information about orphan designation, please visit the FDA website at www.fda.gov.

    About Losmapimod
    Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed by Fulcrum Therapeutics following Fulcrum's discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clini