1. 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 ; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924, or by email at ; 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 .

    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

     



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  2. 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 ; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924, or by email at ; 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 . 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



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  3. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881



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  4. 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



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  5. 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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  6. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

     



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  7. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.

     

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881



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  8. 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

    Investors:

    Michelle Avery

    Pandion Therapeutics

    857-273-0444



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  9. 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



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  10. 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



    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881 

    Primary Logo

    View Full Article Hide Full Article
  11. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.

     

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  12. 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

    Primary Logo

    View Full Article Hide Full Article
  13. 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



    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  14. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations

    kgallagher@berrypr.com

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  15. - 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



    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

    Primary Logo

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  16. 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



    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Primary Logo

    View Full Article Hide Full Article
  17. 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



    617-651-8664

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  18. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

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  19. 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.



    212-362-1200

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  20. 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

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  21. 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 
     
      
    Hannah Deresiewicz (investors)Stephanie Ascher (investors)
    Stern Investor Relations, Inc.Stern Investor Relations, Inc.
    212-362-1200212-362-1200
      
    Julie Normant (media)Kaitlin Gallagher (media)
    W2OBerry & Company Public Relations
    628-213-3754212-253-8881
      

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  22. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.

     

    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

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  23. 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

    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  24. 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



    Stephanie Ascher

    Stern Investor Relations, Inc.



    212-362-1200

    Media:

    Kaitlin Gallagher

    Berry & Company Public Relations



    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  25. 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

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  26. 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

    617-651-8664

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  27. 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

    Primary Logo

    View Full Article Hide Full Article
  28. 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

    Primary Logo

    View Full Article Hide Full Article
  29. 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

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  30. 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

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  31. – 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

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  32. 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

    617-651-8664

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

     

    Primary Logo

    View Full Article Hide Full Article
  33. 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
     
    650-483-1970

    Stephanie Ascher
    Stern Investor Relations, Inc.
     
    212-362-1200

    Media Contact:
    Lynn Granito
    Berry & Company Public Relations
     
    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
    • 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

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media:
    Kaitlin Gallagher
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  34. 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

    Primary Logo

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  35. 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

    Primary Logo

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  36. 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 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.

    Investor Contacts:
    Christi Waarich
    Director, Corporate Communications and Investor Relations

    617-651-8664

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  37. CAMBRIDGE, Mass., Jan. 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 the appointment of Katina Dorton to the Company's board of directors and as chair of the audit committee.

    "We are excited to have Katina join the team," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "Her appointment reflects our commitment to ensuring we have a broad mix of skills and perspectives on Fulcrum's board. We look forward to benefiting from Katina's guidance and leadership as we continue to make important progress with losmapimod and expand our pipeline."

    Ms. Dorton…

    CAMBRIDGE, Mass., Jan. 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 the appointment of Katina Dorton to the Company's board of directors and as chair of the audit committee.

    "We are excited to have Katina join the team," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "Her appointment reflects our commitment to ensuring we have a broad mix of skills and perspectives on Fulcrum's board. We look forward to benefiting from Katina's guidance and leadership as we continue to make important progress with losmapimod and expand our pipeline."

    Ms. Dorton most recently served as Executive Vice President and Chief Financial Officer at Repare Therapeutics, an oncology company focused on DNA damage repair and synthetic lethality, and prior to that, Chief Financial Officer at AVROBIO, a gene therapy company focused on rare disease. Previously, Ms. Dorton spent more than 15 years as an investment banker, advising companies and their Boards on capital markets, fund raising, mergers and acquisitions and other strategic transactions. She was a Managing Director at Morgan Stanley and a Managing Director at Needham & Co. She also served as an attorney in private practice at Sullivan & Cromwell. Ms. Dorton currently is on the Board of Directors at US Ecology, an environmental services company. Ms. Dorton holds a J.D. from the University of Virginia School of Law, an M.B.A. from George Washington University and a B.A. from Duke University.

    "Fulcrum's unique approach to developing small molecule therapies that target the root cause of serious genetic disorders offers great promise to patients and their families," said Ms. Dorton. "I look forward to working with the board and company leadership as Fulcrum advances its pipeline."

    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. 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

    650-483-1970

    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

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  38. Fulcrum to receive $10 million upfront payment and be eligible for future milestone payments

    Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, and Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced they have entered into a collaboration and license agreement to identify small molecules designed to modulate specific pathways associated with a targeted indication within the pulmonary disease space.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191230005047/en/

    Fulcrum to receive $10 million upfront payment and be eligible for future milestone payments

    Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, and Fulcrum Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced they have entered into a collaboration and license agreement to identify small molecules designed to modulate specific pathways associated with a targeted indication within the pulmonary disease space.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191230005047/en/

    "This collaboration brings together Fulcrum's skill in identifying drug targets based on modulation of genetic pathways associated with disease and Acceleron's deep expertise in TGF-beta superfamily signaling in an effort to generate potentially disease-modifying therapeutics," said Habib Dable, Chief Executive Officer of Acceleron Pharma. "With this agreement, along with the advancement of the Acceleron-discovered assets sotatercept—in Phase 2 trials in pulmonary arterial hypertension—and ACE-1334, we underscore our growing commitment to the development of novel therapies for patients with pulmonary diseases of high unmet medical need."

    Under the agreement, Acceleron will have access to Fulcrum's unique, proprietary product engine and target identification platform with the potential to identify small molecules that control the expression of genes known to impact specific pathways associated with a pulmonary disease of interest. Acceleron and Fulcrum will collaborate on the identification of therapeutic targets and small molecule drug candidates for those targets. Acceleron will be responsible for all development and commercialization activities for any potential therapeutics identified via this platform. Fulcrum will receive a one-time, upfront payment of $10 million as well as reimbursement for relevant R&D costs. Fulcrum will also be eligible to receive research, development and commercial milestone payments of up to $295 million for a first product commercialized and up to a maximum of $143.5 million in additional milestone payments for all subsequent products commercialized. Fulcrum will additionally receive tiered royalty payments in the mid-single-digit to low double-digit range on net sales.

    "We are very pleased to partner with Acceleron on this important research initiative," said Robert J. Gould, Ph.D., Chief Executive Officer of Fulcrum Therapeutics. "This collaboration builds on and extends the proven potential of our platform to identify therapies that can address the root cause of diseases, including our progress with losmapimod, currently in a Phase 2 clinical trial for FSHD and extensive pre-clinical and early stage research targeting other genetically defined diseases. This new opportunity to screen and identify pulmonary disease-specific therapies is another reflection of the broad potential applications of the Fulcrum platform in gene modulation."

    About Acceleron

    Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.

    Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol-Myers Squibb, are co-promoting newly approved REBLOZYL® (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States and are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes and myelofibrosis. Acceleron is also advancing its neuromuscular program with ACE-083, a locally-acting Myostatin+ agent in Phase 2 development in Charcot-Marie-Tooth disease and is conducting a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension.

    For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

    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. Fulcrum 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.

    Acceleron Forward-Looking Statements

    This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development and commercialization of Acceleron's compounds, the timeline for clinical development and regulatory approval of Acceleron's compounds, the expected timing for reporting of data from ongoing clinical trials, and the potential success of a collaboration with Fulcrum Therapeutics. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "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 could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, that preclinical testing of Acceleron's compounds and data from clinical trials may not be predictive of the results or success of ongoing or later clinical trials, that the results of any clinical trials may not be predictive of the results or success of other clinical trials, that regulatory approval of Acceleron's compounds in one indication or country may not be predictive of approval in another indication or country, that the development of Acceleron's compounds will take longer and/or cost more than planned, that Acceleron will be unable to successfully complete the clinical development of Acceleron's compounds, that Acceleron may be delayed in initiating, enrolling or completing any clinical trials, that Acceleron's compounds will not receive regulatory approval or become commercially successful products, and that Acceleron's collaboration with Fulcrum Therapeutics will not be successful or result in any successful development candidates. These and other risks and uncertainties are identified under the heading "Risk Factors" included in Acceleron's most recent Annual Report on Form 10-K, and other filings that Acceleron has made and may make with the SEC in the future.

    The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.

    Fulcrum Therapeutics 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 whether the collaboration will yield any targets, potential milestone payments or royalty payments in connection with the collaboration and the potential benefits of the collaboration. All statements, other than statements of historical facts, contained in this press release, including statements regarding Fulcrum'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 each party's ability to perform its obligations under the agreement, the sufficiency of Fulcrum's cash resources to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements on its expected timeline and other important factors discussed in the "Risk Factors" sections contained in Fulcrum's quarterly and annual reports on file with the Securities and Exchange Commission. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum'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 Fulcrum's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Fulcrum's views as of the date hereof and should not be relied upon as representing Fulcrum's views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum's views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so.

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  39. CAMBRIDGE, Mass., Dec. 02, 2019 (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 members of the management team will provide a business overview and update at the 31st Annual Piper Jaffray Healthcare Conference at 10:50 a.m. ET on Tuesday, December 3, 2019 in New York, NY.

    A live webcast of the presentation will be available through the investor relations section of the Company's website at 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…

    CAMBRIDGE, Mass., Dec. 02, 2019 (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 members of the management team will provide a business overview and update at the 31st Annual Piper Jaffray Healthcare Conference at 10:50 a.m. ET on Tuesday, December 3, 2019 in New York, NY.

    A live webcast of the presentation will be available through the investor relations section of the Company's website at 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. Please visit www.fulcrumtx.com.

    Investor Contact:
    Stephanie Ascher
    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  40. – Announced preliminary results of a Phase 1 clinical trial of losmapimod in FSHD patients –

    – Initiated IND-enabling studies for FTX-6058 for the potential treatment of sickle cell disease and beta-thalassemia and announced pre-clinical data showing increase in HbF levels to ~30% of total hemoglobin –

    CAMBRIDGE, Mass., Nov. 14, 2019 (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 2019.

    "This past quarter, we made significant progress in advancing our pipeline and executing on our goal of creating therapeutics targeted…

    – Announced preliminary results of a Phase 1 clinical trial of losmapimod in FSHD patients –

    – Initiated IND-enabling studies for FTX-6058 for the potential treatment of sickle cell disease and beta-thalassemia and announced pre-clinical data showing increase in HbF levels to ~30% of total hemoglobin –

    CAMBRIDGE, Mass., Nov. 14, 2019 (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 2019.

    "This past quarter, we made significant progress in advancing our pipeline and executing on our goal of creating therapeutics targeted at genetically defined rare diseases. We presented encouraging Phase 1 clinical results from our lead program, losmapimod in patients with FSHD, which support the design and dose selection of our ongoing Phase 2 clinical trials. Additionally, we announced our plans to advance our second program, FTX-6058 for the potential treatment of sickle cell disease and beta-thalassemia," said Robert J. Gould, Ph.D., Fulcrum's president and chief executive officer. "We look forward to advancing these two programs through our ongoing Phase 2 trials with losmapimod and our IND-enabling studies with FTX-6058 and utilizing our product engine to identify and validate drug targets to address diseases caused by the mis-expression of certain genes."

    Third Quarter 2019 and Recent Business Highlights

    • In October 2019, Fulcrum presented Phase 1 data from its clinical trial of losmapimod in facioscapulohumeral dystrophy (FSHD) patients and related plans for using molecular biomarkers indicative of the root cause of disease in the Company's ongoing Phase 2 clinical trials of losmapimod at the International Annual Congress of the World Muscle Society in Copenhagen. The results showed that losmapimod was well tolerated with no serious adverse events (SAEs) reported, and that treatment with losmapimod demonstrated dose-dependent pharmacokinetics (PK) and target engagement (TE) in blood. These data support the selection of the 15 mg dose of losmapimod taken orally twice daily in the Company's ongoing Phase 2b placebo-controlled 24-week clinical trial, referred to as ReDUX4, as well as its ongoing Phase 2 open label-study, of losmapimod for the treatment of patients with FSHD.
    • In October 2019, Fulcrum announced progress in the Company's plan to advance the development program for FTX-6058 for the potential treatment of sickle cell disease and beta-thalassemia. Fulcrum has initiated IND-enabling studies for FTX-6058 and anticipates filing an IND in mid-2020. In pre-clinical research, treatment with FTX-6058 was shown to increase HbF levels to ~30% of total hemoglobin as measured by HPLC and mass spectrometry methods in human erythroid progenitor cells from multiple donors.
    • In September 2019, Fulcrum announced the appointment of Pamela Strode as senior vice president, regulatory affairs and quality assurance. She previously served as senior vice president, regulatory affairs and quality assurance at Epizyme, Inc.

    Third Quarter 2019 Financial Results

    • Cash Position: As of September 30, 2019, cash and cash equivalents were $101.6 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, including the proceeds from its July 2019 initial public offering, 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 $13.5 million for the third quarter of 2019, as compared to $7.0 million for the third quarter of 2018. The increase of $6.5 million was primarily due to a $2.5 million increase associated with the achievement of a milestone due under the right of reference and license agreement with GlaxoSmithKline plc during the third quarter of 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 due to additional headcount to support the growth of Fulcrum's research and development organization.
    • G&A Expenses: General and administrative expenses were $3.5 million for the third quarter of 2019, as compared to $2.1 million for the third quarter of 2018. The increase of $1.4 million was primarily due to increased personnel-related costs due to additional headcount, as well as increased consulting and professional fees.
    • Net Loss: Net loss was $16.5 million for the third quarter of 2019, as compared to a net loss of $8.9 million for the third quarter of 2018.

    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 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 Beta-thalassemia

    Beta-thalassemia is a rare blood disorder caused by genetic mutations in the HBB gene, which are associated with the absence or reduced production of beta-globin – one of the two proteins that comprise adult hemoglobin. This results in an abnormally low level of hemoglobin as well as an excess of alpha-globin chains, causing destruction of red blood cells.

    Beta-thalassemia has been clinically characterized into three forms, depending on disease severity: major, intermedia and minor. The most severe form is generally diagnosed shortly after birth and is characterized by life-threatening anemia. Pediatric patients do not grow and gain weight at the typical rates, and often have liver, heart and bone problems. Many patients with beta-thalassemia major require chronic blood transfusions due to severe anemia that results from low hemoglobin levels. Beta-thalassemia intermedia is a less severe form of the disease that results in mild to moderate anemia. These patients sometimes require blood transfusions depending on the severity of their symptoms. Patients with beta-thalassemia minor suffer from very mild anemia and generally do not require treatment.

    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. 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)

      September 30,
    2019 
      December 31,
    2018 
    Cash and cash equivalents $ 101,597     $ 72,797  
    Working capital(1)   96,637       69,866  
    Total assets   115,956       85,771  
    Convertible preferred stock         139,670  
    Total stockholders' equity (deficit)   102,128       (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
    September 30,
        Nine Months Ended
    September 30,
     
      2019     2018     2019     2018  
    Operating expenses:                              
    Research and development $ 13,496     $ 6,963     $ 58,985     $ 18,324  
    General and administrative   3,510       2,125       8,742       5,923  
    Total operating expenses $ 17,006     $ 9,088     $ 67,727     $ 24,247  
    Loss from operations   (17,006 )     (9,088 )     (67,727 )     (24,247 )
    Other income, net:                              
    Interest income, net   457       138       1,151       133  
    Other income   7       7       22       385  
    Net loss and comprehensive loss $ (16,542 )   $ (8,943 )   $ (66,554 )   $ (23,729 )
    Cumulative convertible preferred stock dividends   (796 )     (1,858 )     (7,128 )     (3,736 )
    Net loss attributable to common stockholders $ (17,338 )   $ (10,801 )   $ (73,682 )   $ (27,465 )
    Net loss per share attributable to common stockholders, basic and diluted $ (0.97 )   $ (8.08 )   $ (10.33 )   $ (23.28 )
    Weighted average number of common shares used in net loss per share attributable to common stockholders, basic and diluted   17,785       1,337       7,133       1,180  
                                   

    Contact

    Investors:
    Christina Tartaglia
    Stern IR, Inc.

    212-362-1200

    Media:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

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  41. CAMBRIDGE, Mass., Nov. 13, 2019 (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 members of the management team will provide a business overview and update at the Stifel 2019 Healthcare Conference at 8:00 a.m. ET on Wednesday, November 20, 2019 in New York, NY.

    A live webcast of the presentation will be available through the investor relations section of the Company's website at 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…

    CAMBRIDGE, Mass., Nov. 13, 2019 (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 members of the management team will provide a business overview and update at the Stifel 2019 Healthcare Conference at 8:00 a.m. ET on Wednesday, November 20, 2019 in New York, NY.

    A live webcast of the presentation will be available through the investor relations section of the Company's website at 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. Please visit www.fulcrumtx.com.

    Investor Contact:
    Christina Tartaglia
    Stern Investor Relations, Inc.

    212-362-1200

    Media Contact:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  42. CAMBRIDGE, Mass., Nov. 06, 2019 (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 live conference call and webcast at 8:00 a.m. ET on Thursday, November 14, 2019 to report its third quarter 2019 financial results and provide a corporate update.

    The live call may be accessed by dialing (800) 527-6973 for domestic callers or (470) 495-9162 for international callers and providing the conference ID number 1688051. An audio webcast of the call will be available on the Company's website at https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived…

    CAMBRIDGE, Mass., Nov. 06, 2019 (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 live conference call and webcast at 8:00 a.m. ET on Thursday, November 14, 2019 to report its third quarter 2019 financial results and provide a corporate update.

    The live call may be accessed by dialing (800) 527-6973 for domestic callers or (470) 495-9162 for international callers and providing the conference ID number 1688051. An audio webcast of the call will be available on the Company's website at 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. Please visit www.fulcrumtx.com.

    Investor Contact:
    Christina Tartaglia
    Stern Investor Relations, Inc.
    212.362.1200
      

    Media Contact:
    Kaitlin Gallagher
    Berry & Company Public Relations
     
    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  43. CAMBRIDGE, Mass., Oct. 31, 2019 (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) breakfast symposium focused on understanding Facioscapulohumeral Dystrophy (FSHD) genetics, biology, and clinical implications, on Thursday, November 7, 2019 from 8:30 a.m. to 10:30 a.m. ET in New York, NY. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    Guest speakers scheduled to present at the event include:

    • Kathryn Wagner, M.D., Ph.D., Professor of Neurology and…

    CAMBRIDGE, Mass., Oct. 31, 2019 (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) breakfast symposium focused on understanding Facioscapulohumeral Dystrophy (FSHD) genetics, biology, and clinical implications, on Thursday, November 7, 2019 from 8:30 a.m. to 10:30 a.m. ET in New York, NY. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.

    Guest speakers scheduled to present at the event include:

    • Kathryn Wagner, M.D., Ph.D., Professor of Neurology and Neuroscience at Johns Hopkins School of Medicine and Director of the Center for Genetic Muscle Disorders Kennedy Krieger Institute
    • Peter Jones, Ph.D., Mick Hitchcock, Ph.D. Endowed Chair in Medical Biochemistry and Associate Professor of Pharmacology at University of Nevada, Reno School of Medicine
    • Fran Sverdrup, Ph.D., Associate Professor of Biochemistry and Molecular Biology at Saint Louis University, School of Medicine

    A live webcast of the presentation will be available through the investor relations section of the Company's website at https://ir.fulcrumtx.com/events-and-presentations. Following the live webcast, an archived replay will also be available.

    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 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 diseases in areas of high unmet medical need, with an initial focus on rare diseases. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. Please visit www.fulcrumtx.com.

    Investor Contact:
    Christina Tartaglia
    Stern Investor Relations, Inc.
    212.362.1200
     

    Media Contact:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

    Primary Logo

    View Full Article Hide Full Article
  44. CAMBRIDGE, Mass., Oct. 04, 2019 (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 preliminary results of a Phase 1 clinical trial of losmapimod to treat the root cause of facioscapulohumeral dystrophy (FSHD). Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod following Fulcrum's discovery that the inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD, a disease which is caused by the mis-expression of DUX4 in skeletal muscle. Results were presented today by Michelle…

    CAMBRIDGE, Mass., Oct. 04, 2019 (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 preliminary results of a Phase 1 clinical trial of losmapimod to treat the root cause of facioscapulohumeral dystrophy (FSHD). Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod following Fulcrum's discovery that the inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD, a disease which is caused by the mis-expression of DUX4 in skeletal muscle. Results were presented today by Michelle Mellion, MD, medical director at Fulcrum Therapeutics, in an oral presentation during the 24th International Annual Congress of the World Muscle Society in Copenhagen, Denmark. 

    "Losmapimod has previously been shown to have 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. Until now, losmapimod had not been tested in patients with FSHD, nor was it known if it was muscle-penetrant in humans," said Dr. Mellion. "The preliminary results from our Phase 1 clinical trial of losmapimod in patients with FSHD indicate 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."

    The primary objective of the 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) in FSHD patients. 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. 

    Losmapimod was well tolerated with no serious adverse events (SAEs) reported. Similar tolerability, safety and PK were observed in healthy volunteers and patients with FSHD. Treatment with losmapimod demonstrated dose-dependent PK and TE in blood. This was consistent with previously reported data from more than 3,500 patients treated with losmapimod across multiple other indications. FSHD patients treated with losmapimod also achieved dose-dependent concentrations in skeletal muscle, with a muscle to plasma exposure ratio of approximately 1:1. The losmapimod 15 mg dose taken orally twice daily demonstrated sustained drug concentrations that in preclinical models with human FSHD myotubes resulted in a robust reduction of DUX4-driven gene expression. Analysis of target engagement in muscle is ongoing. These data support the selection of the 15 mg dose of losmapimod taken orally twice daily in the Company's ongoing Phase 2b placebo-controlled 24-week clinical trial, referred to as ReDUX4, as well as its ongoing Phase 2 open label-study of losmapimod for the treatment of patients with FSHD.

    "There are currently no approved treatment options available for patients with FSHD. They face a lifetime of accumulating disability that can severely impact their day-to-day function and quality of life," said Diego Cadavid, MD, senior vice president of clinical development at Fulcrum Therapeutics. "We are very encouraged by these results and are working rapidly to advance our development program for losmapimod."

    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 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 diseases in areas of high unmet medical need, with an initial focus on rare diseases. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. 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.

    Contacts
    Media:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

    Investors:
    Christina Tartaglia
    Stern IR, Inc.

    212-362-1200

    Primary Logo

    View Full Article Hide Full Article
    • Oral presentation to highlight Phase 1 data on safety, tolerability and target engagement of losmapimod in treatment of FSHD
    • Company also announces updated preclinical data on FTX-6058 for potential treatment of sickle cell disease; FTX-6058 showed an increase in HbF levels to ~30% total hemoglobin

    CAMBRIDGE, Mass., Oct. 01, 2019 (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 announces that the company will present data from multiple studies of losmapimod in facioscapulohumeral dystrophy (FSHD) patients during the International Annual Congress of the World Muscle Society being held from October…

    • Oral presentation to highlight Phase 1 data on safety, tolerability and target engagement of losmapimod in treatment of FSHD
    • Company also announces updated preclinical data on FTX-6058 for potential treatment of sickle cell disease; FTX-6058 showed an increase in HbF levels to ~30% total hemoglobin

    CAMBRIDGE, Mass., Oct. 01, 2019 (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 announces that the company will present data from multiple studies of losmapimod in facioscapulohumeral dystrophy (FSHD) patients during the International Annual Congress of the World Muscle Society being held from October 1-5 in Copenhagen, Denmark. 

    Michelle Mellion, MD, medical director at Fulcrum Therapeutics, will present data from the Phase 1 trial to assess the pharmacokinetics, safety and tolerability, and target engagement of losmapimod in FSHD patients during an oral presentation on Friday, October 4, 2019 from 10:30-12:30 CET. The company will highlight findings from the Phase 1 trial as well as information related to plans for using biomarkers in the ongoing Phase 2 clinical trials of losmapimod. A link to the Phase 1 data abstract is available here.

    "We are very pleased to have such a strong presence at the World Muscle Society meeting this year, a testament to our progress and the significance of our efforts thus far to develop a treatment targeting the root cause of FSHD," said Dr. Mellion. "We look forward to sharing these important insights with the research community during the meeting."

    Update on data related to Hemoglobinopathy Program

    Fulcrum also announces recent progress in the company's plan to advance the development program for FTX-6058, formerly referred to as FTX-HbF, for the potential treatment of sickle cell disease and beta-thalassemia. FTX-6058 is a novel upregulator of fetal hemoglobin. In pre-clinical research, treatment with FTX-6058 was shown to increase HbF levels to ~30% of total hemoglobin as measured by HPLC and mass spectrometry methods in human erythroid progenitor cells from multiple donors. FTX-6058 also elevated HbF in vivo in animal models at plasma concentrations reasonably expected to be achieved in humans. Fulcrum believes these results indicate that FTX-6058 could play a role in reducing the risk of crises in people living with sickle cell disease and may also help to address transfusion requirements in patients with beta-thalassemia as an oral therapeutic. Fulcrum has initiated IND-enabling studies for FTX-6058 and anticipates filing an IND in mid-2020.

    "Our progress with both losmapimod and FTX-6058 highlights the strength of our research platform with the potential to rapidly advance development programs from our research efforts," said Robert J. Gould, PhD, president and CEO of Fulcrum Therapeutics. "We look forward to providing updates on these important programs as we proceed."

    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 beta-thalassemia
    Beta-thalassemia is a rare blood disorder caused by genetic mutations in the HBB gene, which are associated with the absence or reduced production of beta-globin – one of the two proteins that comprise adult hemoglobin. This results in an abnormally low level of hemoglobin as well as an excess of alpha-globin chains, causing destruction of red blood cells.

    Beta-thalassemia has been clinically characterized into three forms, depending on disease severity: major, intermedia and minor. The most severe form is generally diagnosed shortly after birth and is characterized by life-threatening anemia. Pediatric patients do not grow and gain weight at the typical rates, and often have liver, heart and bone problems. Many patients with beta-thalassemia major require chronic blood transfusions due to severe anemia that results from low hemoglobin levels. Beta-thalassemia intermedia is a less severe form of the disease that results in mild to moderate anemia. These patients sometimes require blood transfusions depending on the severity of their symptoms. Patients with beta-thalassemia minor suffer from very mild anemia and generally do not require treatment.

    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 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.

    About Fulcrum Therapeutics
    Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial focus on rare diseases. Fulcrum's proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. 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.

    Contacts
    Media:
    Lynn Granito
    Berry & Company Public Relations

    212-253-8881

    Investors:
    Christina Tartaglia
    Stern IR, Inc.

    212-362-1200

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