1. INDIANAPOLIS and NEW YORK, Dec. 15, 2020 /PRNewswire/ -- Eli Lilly and Company (NYSE:LLY) and Prevail Therapeutics Inc. (NASDAQ:PRVL) today announced a definitive agreement for Lilly to acquire Prevail for $22.50 per share in cash (or an aggregate of approximately $880 million) payable at closing plus one non-tradable contingent value right ("CVR") worth up to $4.00 per share in cash (or an aggregate of approximately $160 million), for a total consideration of up to $26.50 per share in cash (or an aggregate of approximately $1.040 billion). The CVR is payable (subject to certain terms and conditions) upon the first regulatory approval of a product from Prevail's pipeline as set forth in more detail below. Prevail is a biotechnology company developing…

    INDIANAPOLIS and NEW YORK, Dec. 15, 2020 /PRNewswire/ -- Eli Lilly and Company (NYSE:LLY) and Prevail Therapeutics Inc. (NASDAQ:PRVL) today announced a definitive agreement for Lilly to acquire Prevail for $22.50 per share in cash (or an aggregate of approximately $880 million) payable at closing plus one non-tradable contingent value right ("CVR") worth up to $4.00 per share in cash (or an aggregate of approximately $160 million), for a total consideration of up to $26.50 per share in cash (or an aggregate of approximately $1.040 billion). The CVR is payable (subject to certain terms and conditions) upon the first regulatory approval of a product from Prevail's pipeline as set forth in more detail below. Prevail is a biotechnology company developing potentially disease-modifying AAV9-based gene therapies for patients with neurodegenerative diseases.

    The acquisition will establish a new modality for drug discovery and development at Lilly, extending Lilly's research efforts through the creation of a gene therapy program that will be anchored by Prevail's portfolio of clinical-stage and preclinical neuroscience assets. Prevail's lead gene therapies in clinical development are PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN). Prevail's preclinical pipeline includes PR004 for patients with specific synucleinopathies, as well as potential gene therapies for Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and other neurodegenerative disorders.

    "Gene therapy is a promising approach with the potential to deliver transformative treatments for patients with neurodegenerative diseases such as Parkinson's, Gaucher and dementia," said Mark Mintun, M.D., vice president of pain and neurodegeneration research at Lilly. "The acquisition of Prevail will bring critical technology and highly skilled teams to complement our existing expertise at Lilly, as we build a new gene therapy program anchored by well-researched assets. We look forward to completing the proposed acquisition and working with Prevail to advance their groundbreaking work through clinical development."

    "Lilly is an established leader in neuroscience drug development and commercialization who shares our commitment to patients with neurodegenerative diseases, and I'm excited for Prevail to join the Lilly family," said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Prevail. "I'm incredibly proud of the Prevail team, who have made great progress advancing our pipeline of gene therapy programs for patients with these devastating disorders. In just over three years, Prevail has advanced two first-in-class gene therapy programs into clinical development for PD-GBA, nGD, and FTD-GRN, established two manufacturing platforms, and developed a broad pipeline with great potential to impact patients in need of disease-modifying treatment options.  With its global scale and resources, Lilly will be the ideal organization to maximize the potential of our pipeline and accelerate our ability to bring these therapies to as many patients as possible. We look forward to working together to advance our shared mission."

    Under the terms of the agreement, Lilly will commence a tender offer to acquire all outstanding shares of Prevail Therapeutics Inc. for a purchase price of $22.50 per share in cash (or an aggregate of approximately $880 million) payable at closing plus one non-tradeable CVR. The CVR entitles Prevail stockholders to up to an additional $4.00 per share in cash (or an aggregate of approximately $160 million) payable (subject to certain terms and conditions) upon the first regulatory approval for commercial sale of a Prevail product in one of the following countries: United States, Japan, United Kingdom, Germany, France, Italy or Spain. To achieve the full value of the CVR, such regulatory approval must occur by December 31, 2024. If such regulatory approval occurs after December 31, 2024, the value of the CVR will be reduced by approximately 8.3 cents per month until December 1, 2028 (at which point the CVR will expire). There can be no assurance any payments will be made with respect to the CVR. The transaction is not subject to any financing condition and is expected to close in the first quarter of 2021, subject to customary closing conditions, including receipt of required regulatory approvals and the tender of a majority of the outstanding shares of Prevail's common stock. Following the successful closing of the tender offer, Lilly will acquire any shares of Prevail that are not tendered in the tender offer through a second-step merger at the same consideration as paid in the tender offer.

    The purchase price payable at closing represents a premium of approximately 117 percent to the 60-day volume-weighted average trading price of Prevail's common stock ended on December 14, 2020, the last trading day before the announcement of the transaction. Prevail's Board of Directors unanimously recommends that Prevail's stockholders tender their shares in the tender offer. Additionally, certain Prevail stockholders, beneficially owning approximately 51 percent of Prevail's outstanding common stock, have (subject to certain terms and conditions) agreed to tender their shares in the tender offer.

    Upon closing, the impact of this transaction will be reflected in Lilly's 2021 financial results according to Generally Accepted Accounting Principles (GAAP). There will be no change required to Lilly's 2021 financial guidance being issued today for research and development expense or non-GAAP earnings per share as a result of this transaction.

    For Lilly, Lazard is acting as sole financial advisor and Weil, Gotshal & Manges LLP is acting as legal counsel. For Prevail, Centerview Partners LLC is acting as sole financial advisor, Ropes & Gray LLP is acting as legal counsel, and Cooley LLP also provided legal counsel.

    Prevail Therapeutics Pipeline

    • PR001 is being developed as a potentially disease-modifying, single-dose AAV9-based gene therapy for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD), delivered by intra-cisterna magna injection. The PROPEL trial, a Phase 1/2 clinical trial of PR001 for the treatment of PD-GBA patients, is ongoing. The PROVIDE trial, a Phase 1/2 clinical trial of PR001 for the treatment of Type 2 Gaucher disease patients, is now recruiting. The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for PR001 for the treatment of PD-GBA and for the treatment of nGD. It has also granted Orphan Drug Designation for PR001 for the treatment of Gaucher disease, and Rare Pediatric Disease Designation for the treatment of nGD.
    • PR006 is being developed as a potentially disease-modifying, single-dose AAV9-based gene therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN), also delivered by intra-cisterna magna injection. The PROCLAIM trial, a Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN patients, is currently ongoing and the first patient was dosed in December 2020. The FDA and the European Commission have granted orphan designation for PR006 for the treatment of FTD, and the FDA has granted Fast Track Designation for PR006 for FTD-GRN.
    • PR004 is a gene therapy in preclinical development for patients with certain synucleinopathies. PR004 utilizes an AAV9 vector to deliver the GBA1 gene, which encodes glucocerebrosidase (GCase), and a molecule that suppresses expression of α-Synuclein.
    • Prevail is developing a broad pipeline of additional AAV gene therapies for the treatment of Alzheimer's disease, ALS, Parkinson's disease, and other neurodegenerative disorders. Preclinical development of these potential therapies is currently ongoing.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. Prevail is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA, and OrbiMed, and is headquartered in New York, NY.

    About Eli Lilly and Company

    Lilly is a global healthcare leader that unites caring with discovery to create medicines that make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at www.lilly.com.  C-LLY   

    Cautionary Statement Regarding Forward-Looking Statements

    This press release contains forward-looking statements about Lilly's proposed acquisition of Prevail Therapeutics Inc. ("Prevail"), regarding potential contingent consideration amounts and terms, regarding the anticipated occurrence, manner and timing of closing of the proposed transaction, regarding Prevail's product candidates and ongoing preclinical development, regarding Lilly's development of a potential gene therapy program, and regarding Lilly's expected 2021 financial guidance and the impact of the proposed acquisition on research and development expense and non-GAAP earnings per share. It reflects current beliefs and expectations; however, as with any such undertaking, there are substantial risks and uncertainties in consummating the proposed transaction, in drug research, development and commercialization, and in Lilly's evaluation of its estimated financial results for 2021 and the impact of the proposed acquisition. Actual results could differ materially due to various factors, risks and uncertainties. Among other things, there can be no guarantee that the proposed transaction will be completed in the anticipated timeframe or at all, that the conditions required to complete the proposed transaction will be met, that Lilly will realize the expected benefits of the proposed transaction, that product candidates will be approved on anticipated timelines or at all, that Lilly will be successful in building a gene therapy program, that any products, if approved, will be commercially successful, that all or any of the contingent consideration will become payable on the terms described herein or at all, that Lilly's financial results will be consistent with its expected 2021 guidance or that Lilly can reliably predict the impact of the proposed acquisition on its 2021 financial guidance and results. For further discussion of these and other risks and uncertainties, see Lilly's and Prevail's most recent Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission (the "SEC"). Except as required by law, neither Prevail nor Lilly undertakes any duty to update forward-looking statements to reflect events after the date of this press release.

    Additional Information about the Acquisition and Where to Find It

    The tender offer for the outstanding shares of Prevail referenced in this communication has not yet commenced. This announcement is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell shares of Prevail, nor is it a substitute for the tender offer materials that Lilly and its acquisition subsidiary will file with the SEC upon commencement of the tender offer. At the time the tender offer is commenced, Lilly and its acquisition subsidiary will file tender offer materials on Schedule TO, and thereafter Prevail will file a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC with respect to the tender offer. THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER TENDER OFFER DOCUMENTS) AND THE SOLICITATION/RECOMMENDATION STATEMENT WILL CONTAIN IMPORTANT INFORMATION. HOLDERS OF SHARES OF PREVAIL ARE URGED TO READ THESE DOCUMENTS CAREFULLY WHEN THEY BECOME AVAILABLE (AS EACH MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME) BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION THAT HOLDERS OF PREVAIL SHARES SHOULD CONSIDER BEFORE MAKING ANY DECISION REGARDING TENDERING THEIR SHARES. The Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents, as well as the Solicitation/Recommendation Statement, will be made available to all holders of shares of Prevail at no expense to them. The tender offer materials and the Solicitation/Recommendation Statement will be made available for free at the SEC's web site at www.sec.gov

    In addition to the Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents, as well as the Solicitation/Recommendation Statement, Lilly and Prevail file annual, quarterly and special reports and other information with the SEC.  You may read and copy any reports or other information filed by Lilly or Prevail at the SEC public reference room at 100 F Street, N.E., Washington, D.C. 20549. Please call the Commission at 1-800-SEC-0330 for further information on the public reference room.  Lilly's and Prevail's filings with the SEC are also available to the public from commercial document-retrieval services and at the website maintained by the SEC at www.sec.gov.

    Refer to:   

    Mark Taylor; ; (317) 276-5795 (Lilly Media)   



    Kevin Hern; ; (317) 277-1838 (Lilly Investors)



    Gina Nugent; ; (617)460-3579 (Prevail Media)



    ; (Prevail Investors)

     

    Eli Lilly and Company logo. (PRNewsfoto/Eli Lilly and Company)

     

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  2. NEW YORK, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the first patient has been dosed in the Phase 1/2 PROCLAIM clinical trial evaluating PR006, an investigational AAV9 gene therapy delivering the GRN gene, for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN).

    "Dosing the first patient in our PROCLAIM clinical trial marks an important milestone in our efforts to advance a potentially disease-modifying treatment for patients with frontotemporal dementia with GRN mutations," said Asa Abeliovich, M.D., Ph.D., Founder and Chief…

    NEW YORK, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the first patient has been dosed in the Phase 1/2 PROCLAIM clinical trial evaluating PR006, an investigational AAV9 gene therapy delivering the GRN gene, for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN).

    "Dosing the first patient in our PROCLAIM clinical trial marks an important milestone in our efforts to advance a potentially disease-modifying treatment for patients with frontotemporal dementia with GRN mutations," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "We are excited to progress clinical development of PR006 and to bring forward a much-needed therapy for this rapidly progressing neurodegenerative disease."

    The PROCLAIM trial is a Phase 1/2 open-label trial investigating the safety and tolerability of PR006 as well as key biomarkers and exploratory efficacy endpoints. The Company expects to enroll up to 15 patients, and it currently anticipates it will provide a biomarker and safety analysis on a subset of patients enrolled in the PROCLAIM trial in 2021.

    "Frontotemporal dementia is a devastating condition, with no disease-modifying therapeutic options available," said Dr. Jonathan Rohrer, principal research fellow at the University College London Queen Square Institute of Neurology. "PROCLAIM is an important clinical study which could further increase our understanding of frontotemporal dementia due to mutations in the progranulin gene, and help demonstrate the potential of gene therapy to correct the underlying genetic cause of this condition, potentially slowing or stopping disease progression."

    PR006 has been granted Orphan Drug designation for the treatment of FTD and Fast Track designation for the treatment of FTD-GRN by the U.S. Food and Drug Administration, as well as orphan designation for the treatment of FTD by the European Commission.

    About Frontotemporal Dementia with GRN Mutations

    Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer's disease. FTD affects 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union. FTD-GRN represents 5-10% of all patients with FTD. FTD results from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language. In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation and neurodegeneration. There are no approved treatments for FTD or FTD-GRN.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for PR006 to be a disease-modifying gene therapy to patients with FTD-GRN; the potential benefits of Fast Track and Orphan Drug designation by the FDA and orphan designation by the FDA and the European Commission; the anticipated timing of enrollment and of reporting of interim data on a subset of patients from the PROCLAIM trial; and the potential for the PROCLAIM trial to increase understanding of frontotemporal dementia and help demonstrate the potential of gene therapy to correct the underlying genetic cause of this condition, potentially slowing or stopping disease progression. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" sections of the Company's most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:

    Lisa Qu

    Ten Bridge Communications



    678-662-9166

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  3. NEW YORK, Nov. 30, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the European Commission has granted orphan designation for PR006 for the treatment of frontotemporal dementia (FTD). PR006 is an investigational AAV9 gene therapy delivering the GRN gene and is being developed as a potential therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

    "The European Commission's decision to grant orphan designation for PR006 is an important step in helping to advance this potential therapeutic option for patients with frontotemporal dementia with GRN

    NEW YORK, Nov. 30, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the European Commission has granted orphan designation for PR006 for the treatment of frontotemporal dementia (FTD). PR006 is an investigational AAV9 gene therapy delivering the GRN gene and is being developed as a potential therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

    "The European Commission's decision to grant orphan designation for PR006 is an important step in helping to advance this potential therapeutic option for patients with frontotemporal dementia with GRN mutations," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "We are excited to progress clinical development of PR006 as part of our mission to deliver a potentially disease-modifying gene therapy to these patients as quickly as possible."

    The Company expects to initiate enrollment in the Phase 1/2 PROCLAIM trial of PR006 in the fourth quarter of 2020, and it currently anticipates it will provide a biomarker and safety analysis on a subset of patients in the PROCLAIM trial in 2021.

    The U.S. Food and Drug Administration has also granted Orphan Drug designation for PR006 for the treatment of FTD and Fast Track designation for FTD-GRN.

    About European Commission Orphan Designation

    Orphan designation is granted by the European Commission to encourage development of medicines intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the European Union. Orphan designation by the European Commission provides companies with certain benefits and incentives, including protocol assistance, reduced regulatory fees and 10 years of market exclusivity following regulatory approval.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's mission to deliver a potentially disease-modifying gene therapy to patients with FTD-GRN as quickly as possible; the potential benefits of orphan designation by the European Commission; and the anticipated timing of enrollment and of reporting of interim data on a subset of patients from the PROCLAIM trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" sections of the Company's most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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    Lisa Qu

    Ten Bridge Communications



    678-662-9166

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  4. NEW YORK, Nov. 18, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) on November 17, 2020 issued a composition of matter patent, U.S. Patent No. 10,837,028, with claims directed to the AAV vector used in PR001, Prevail's experimental gene therapy program for the treatment of Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). The base patent term extends until October 3, 2038, excluding patent term extensions or coverage in additional related patent filings.

    "We are excited to make…

    NEW YORK, Nov. 18, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) on November 17, 2020 issued a composition of matter patent, U.S. Patent No. 10,837,028, with claims directed to the AAV vector used in PR001, Prevail's experimental gene therapy program for the treatment of Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). The base patent term extends until October 3, 2038, excluding patent term extensions or coverage in additional related patent filings.

    "We are excited to make important progress this year with PR001, which is being evaluated in the Phase 1/2 PROPEL trial for patients with Parkinson's disease with GBA1 mutations and in the Phase 1/2 PROVIDE trial for patients with Type 2 Gaucher disease," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "We are advancing clinical development of PR001 to make a potentially transformative difference for these patients who currently have no approved treatment options."

    The Company recently announced that patient dosing has continued in the Phase 1/2 PROPEL clinical trial of PR001 for PD-GBA patients, and it expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021. The Company expects to initiate enrollment of the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease patients in the fourth quarter of 2020 and currently anticipates it will provide the next update on PR001 biomarker and safety data for nGD in 2021.

    The U.S. Food and Drug Administration has granted Fast Track designations for PR001 for the treatment of PD-GBA and nGD. In addition, the FDA granted PR001 Rare Pediatric Disease designation for the treatment of nGD, and Orphan Drug designation for the treatment of patients with Gaucher disease.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to make a transformative difference for patients with neurodegenerative diseases; the expected timing of reporting additional interim data on a subset of patients from the PROPEL trial; and the anticipated timing of enrollment of and the next update on data from the PROVIDE trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" sections of the Company's most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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    Lisa Qu

    Ten Bridge Communications



    678-662-9166

    Investor Contact:

     



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  5. Patient Dosing Continues in the Phase 1/2 PROPEL Trial of PR001 for Parkinson's Disease with GBA1 Mutations

    Phase 1/2 PROVIDE Trial of PR001 for Type 2 Gaucher Disease and Phase 1/2 PROCLAIM Trial of PR006 for Frontotemporal Dementia with GRN Mutations Expected to Initiate Enrollment in Fourth Quarter of 2020

    PR001 Receives U.S. FDA Fast Track Designation for Neuronopathic Gaucher Disease

    NEW YORK, Nov. 13, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reviewed recent clinical and business updates and reported financial results for the third quarter ended September 30, 2020.

    Patient Dosing Continues in the Phase 1/2 PROPEL Trial of PR001 for Parkinson's Disease with GBA1 Mutations

    Phase 1/2 PROVIDE Trial of PR001 for Type 2 Gaucher Disease and Phase 1/2 PROCLAIM Trial of PR006 for Frontotemporal Dementia with GRN Mutations Expected to Initiate Enrollment in Fourth Quarter of 2020

    PR001 Receives U.S. FDA Fast Track Designation for Neuronopathic Gaucher Disease

    NEW YORK, Nov. 13, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reviewed recent clinical and business updates and reported financial results for the third quarter ended September 30, 2020.

    "We're pleased to be making significant progress across our pipeline as we seek to develop urgently needed disease-modifying gene therapy treatments for patients with neurodegenerative diseases," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "We are encouraged by the continuation of patient dosing in our Phase 1/2 PROPEL trial of PR001 for Parkinson's disease with GBA1 mutations, and we are excited to advance our PROVIDE and PROCLAIM clinical trials for Type 2 Gaucher disease and frontotemporal dementia with GRN mutations, respectively, this year."

    Recent Business Updates

    Patient Dosing Continues in Phase 1/2 PROPEL Trial of PR001 for Parkinson's disease with GBA1 mutations (PD-GBA): Enrollment in the Phase 1/2 PROPEL clinical trial for PD-GBA has resumed following implementation of modifications to the clinical protocol. As previously announced, Prevail elected to modify the immunosuppression regimen in the clinical protocol for PROPEL and has adapted the trial design to be open-label. The Company expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021.

    Phase 1/2 PROVIDE Trial Expected to Initiate Enrollment in Fourth Quarter of 2020: Initiation of patient enrollment remains on track for the fourth quarter of 2020 for the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease. The optimized immunosuppression regimen used in the amended PROPEL trial will also be implemented in the PROVIDE trial. The Company currently anticipates it will provide the next update on PR001 biomarker and safety data for neuronopathic Gaucher disease (nGD) in 2021.

    Phase 1/2 PROCLAIM Trial Expected to Initiate Enrollment in Fourth Quarter of 2020: Initiation of patient enrollment remains on track for the fourth quarter of 2020 for the Phase 1/2 PROCLAIM clinical trial of PR006 for frontotemporal dementia with GRN mutations (FTD-GRN). The optimized immunosuppression regimen used in the amended PROPEL trial will also be implemented in the PROCLAIM trial. The Company currently anticipates it will provide a biomarker and safety analysis on a subset of patients in the PROCLAIM trial in 2021.

    PR001 Granted U.S. FDA Fast Track Designation for nGD: The U.S. Food and Drug Administration (FDA) granted Fast Track designation for PR001 for the treatment of nGD. The FDA previously granted PR001 Rare Pediatric Disease designation for the treatment of nGD, and Orphan Drug designation for the treatment of patients with Gaucher disease. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of PD-GBA.

    Strengthened Leadership with Board Appointment: Prevail has appointed William H. Carson, M.D., to its Board of Directors. Dr. Carson was most recently the President and CEO of Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC), leading the development and regulatory approvals of Otsuka's global compounds. Before joining Otsuka, he held several roles in the CNS Research and Development department at Bristol Myers Squibb. Dr. Carson currently serves as Chairman of the Board of Directors of OPDC and is also the Chairman of the Board of the Sozosei Foundation, a newly established Otsuka charitable organization with a main focus on decriminalization of mental illness. He is a Board Member of Excision Biotherapeutics and Trustee of the non-profit Internet2. He is a Distinguished Fellow of the American Psychiatric Association, the National Medical Association and the Executive Leadership Council. Prior to joining the pharmaceutical industry, Dr. Carson, a board-certified psychiatrist, was an Associate Professor in the Department of Psychiatry and Behavioral Sciences at the Medical University of South Carolina.

    Favorable Decision Received in Alector Arbitration: Prevail announced a favorable decision in the arbitration proceeding brought in 2019 by Alector Inc. against Prevail's Founder and Chief Executive Officer, Asa Abeliovich, M.D., Ph.D. The arbitrator rejected Alector's claims against Dr. Abeliovich that Alector confidential information was used in connection with his work on behalf of Prevail and that Alector had rights to Prevail's patents and patent applications. The arbitrator found that Dr. Abeliovich did not breach his confidentiality obligations to Alector under his consulting agreement. Prevail was not a party to this arbitration.

    Third Quarter 2020 Financial Results

    • Cash Position: Cash, cash equivalents and investments were $114.3 million as of September 30, 2020, as compared to $131.2 million and $168.1 million as of June 30, 2020 and December 31, 2019, respectively. The Company continues to anticipate that its cash runway will extend into the first half of 2022.

       
    • R&D Expenses: R&D expenses were $12.3 million for the third quarter of 2020 compared to $16.8 million for the third quarter in 2019. The decrease was primarily due to a decrease of $3.9 million in external manufacturing costs due to the timing of production of clinical and preclinical supply, a decrease of $1.5 million in direct clinical trial costs, and a decrease of $0.5 million related to external preclinical studies. These decreases were partially offset by an increase of $1.4 million in employee-related costs, resulting from an increase in research and development employees hired to execute the development of our clinical-stage product candidates and preclinical pipeline.

       
    • G&A Expenses: G&A expenses were $6.3 million for the third quarter of 2020, compared to $4.5 million for the third quarter of 2019. The increase was primarily due to a $1.3 million increase in employee related costs, resulting from an increase in general and administrative employees to support our expanded operations and establish capabilities to operate as a public company, a $0.8 million increase in legal fees, offset by a decrease of $0.2 million in other professional services and facilities cost.

       
    • Net Loss: Net loss was $18.6 million, or $0.55 loss per share, for the third quarter of 2020, compared to $20.3 million, or $0.62 loss per share, for the third quarter of 2019.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to modify the course of neurodegenerative diseases; the anticipated timing of Prevail's clinical trials of PR001 in PD-GBA and in Type 2 Gaucher disease and Prevail's clinical trial of PR006 in FTD-GRN; the expected timing of reporting of additional interim data for a subset of patients from the PROPEL trial; and expectations regarding Prevail's cash runway. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.



    Prevail Therapeutics Inc.

    Statements of Operations

    (Unaudited)

    (in thousands, except share and per share data)

      Three Months Ended September

    30,
        Nine Months Ended

    September 30,
     
      2020    2019    2020    2019 
    Operating Expenses:                      
    Research and development $12,321    $16,836    $36,681    $37,202 
    General and administrative  6,303     4,452     23,373     10,050 
    Total operating loss  (18,624)    (21,288)    (60,054)    (47,252)
    Other income            210      
    Interest income, net  37     989     582     1,905 
    Total other income  37     989     792     1,905 
    Net loss $(18,587)   $(20,299)   $(59,262)   $(45,347)
    Other comprehensive income  5          4      
    Comprehensive loss $(18,582)   $(20,299)   $(59,258)   $(45,347)
    Net loss per share, basic and diluted $(0.55)   $(0.62)   $(1.77)   $(1.68)
    Weighted average shares outstanding, basic and diluted  33,636,651     32,864,156     33,457,768     26,950,854 
     
     

    Prevail Therapeutics Inc.

    Balance Sheets

    (Unaudited)

    (in thousands, except share and per share data)

      September 30,

    2020
      December 31,

    2019
     
             
    ASSETS        
    CURRENT ASSETS:        
    Cash and cash equivalents $81,732  $168,051 
    Investments  9,755    
    Prepaid expenses and other current assets  4,839   6,410 
    Total current assets  96,326   174,461 
    Property and equipment, net  2,746   2,549 
    Investments  22,861    
    Operating lease right-of-use assets  9,023   10,001 
    Other long-term assets  3,068    
    Restricted cash  91   91 
    TOTAL ASSETS $134,115  $187,102 
    LIABILITIES AND STOCKHOLDERS' EQUITY        
    CURRENT LIABILITIES:        
    Accounts payable $2,597  $5,162 
    Accrued expenses and other current liabilities  8,651   5,330 
    Operating lease liabilities  1,500   1,341 
    Total current liabilities  12,748   11,833 
    Long-term operating lease liabilities  8,787   9,927 
    TOTAL LIABILITIES  21,535   21,760 
    COMMITMENTS AND CONTINGENCIES (Note 13)        
    STOCKHOLDERS' EQUITY        
    Preferred stock - $0.0001 par value, 10,000,000 shares authorized as of September 30, 2020 and December 31, 2019, respectively; no shares issued as of September 30, 2020 and December 31, 2019, respectively      
    Common stock - $0.0001 par value, 200,000,000 shares authorized as of September 30, 2020 and December 31, 2019, respectively, 34,245,433 and 34,138,750 shares issued and outstanding as of September 30, 2020 and December 31, 2019, respectively  3   3 
    Additional paid-in capital  255,937   249,441 
    Accumulated deficit  (143,364)  (84,102)
    Accumulated other comprehensive income  4    
    Total stockholders' equity  112,580   165,342 
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $134,115  $187,102 
             



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  6. NEW YORK, Nov. 10, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will participate in the following virtual investor conferences in November and December:

    • Guggenheim Healthcare Talks Idea Forum 2nd Annual Neuro/Immunology Day on Monday, November 16, 2020.

    • Stifel 2020 Virtual Healthcare Conference on Wednesday, November 18, 2020 at 10:40-11:10 a.m. ET.

    • Piper Sandler 32nd Annual Virtual Health Care Conference on Tuesday, December 1, 2020.

    Webcasts of the presentations will be available under "Events…

    NEW YORK, Nov. 10, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will participate in the following virtual investor conferences in November and December:

    • Guggenheim Healthcare Talks Idea Forum 2nd Annual Neuro/Immunology Day on Monday, November 16, 2020.



    • Stifel 2020 Virtual Healthcare Conference on Wednesday, November 18, 2020 at 10:40-11:10 a.m. ET.



    • Piper Sandler 32nd Annual Virtual Health Care Conference on Tuesday, December 1, 2020.

    Webcasts of the presentations will be available under "Events and Presentations" in the Investors and Media section of the Company's website at ir.prevailtherapeutics.com following the presentations.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Media Contact:

    Lisa Qu

    Ten Bridge Communications



    678-662-9166

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  7. NEW YORK, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced a decisive victory in the arbitration proceeding brought in 2019 by Alector Inc. against Prevail's founder and Chief Executive Officer, Asa Abeliovich, M.D., Ph.D.

    The arbitrator rejected all of Alector's principal claims against Dr. Abeliovich, including all claims alleging misappropriation or misuse of trade secrets. Significantly, the arbitrator rejected Alector's claims that Dr. Abeliovich used Alector trade secrets or confidential information in connection with his work on behalf of Prevail, as well as…

    NEW YORK, Nov. 02, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced a decisive victory in the arbitration proceeding brought in 2019 by Alector Inc. against Prevail's founder and Chief Executive Officer, Asa Abeliovich, M.D., Ph.D.

    The arbitrator rejected all of Alector's principal claims against Dr. Abeliovich, including all claims alleging misappropriation or misuse of trade secrets. Significantly, the arbitrator rejected Alector's claims that Dr. Abeliovich used Alector trade secrets or confidential information in connection with his work on behalf of Prevail, as well as Alector's claim that it has rights to Prevail's patents and patent applications. Prevail was not a party to this arbitration.

    "We appreciate the arbitrator's thorough and independent review of this matter. This decision vindicates Dr. Abeliovich and rules in his favor on all significant issues," said Francois Nader, M.D., Non-Executive Chairman of Prevail. "With this positive outcome, Prevail is looking forward to advancing our pipeline for the benefit of patients."

    "I am pleased with the decision, which confirms Prevail's ownership of its intellectual property," said Dr. Abeliovich. "Our team continues to advance our pipeline and remains committed to our critical mission of developing novel, urgently needed therapies for patients with neurodegenerative disorders such as Parkinson's disease and frontotemporal dementia."

    The only relief granted to Alector by the arbitrator was permission to seek reimbursement for a portion of Alector's out-of-pocket costs and fees in connection with certain document retention-related claims. Alector will need to provide sufficient proof of such amounts.

    About Prevail Therapeutics

    Prevail is a clinical stage gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the amount of costs and legal fees that Prevail may be required to reimburse. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, filed with the SEC on August 11, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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  8. NEW YORK, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company's experimental gene therapy program, PR001, for the treatment of neuronopathic Gaucher disease (nGD). The Company expects to initiate enrollment of the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 neuronopathic Gaucher disease patients in the second half of 2020.

    "FDA Fast Track designation for PR001 for the treatment of neuronopathic Gaucher disease underscores the significant unmet medical need for…

    NEW YORK, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company's experimental gene therapy program, PR001, for the treatment of neuronopathic Gaucher disease (nGD). The Company expects to initiate enrollment of the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 neuronopathic Gaucher disease patients in the second half of 2020.

    "FDA Fast Track designation for PR001 for the treatment of neuronopathic Gaucher disease underscores the significant unmet medical need for this devastating condition, for which there are no currently approved therapies," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "Type 2 Gaucher disease involves rapidly progressive neurodegeneration leading to death in infancy or early childhood, and Type 3 Gaucher disease is associated with significant neurological manifestations including seizures and motor abnormalities. We believe PR001 has the potential to serve as a much-needed therapeutic option for these patients as enzyme replacement therapies approved for Type 1 Gaucher disease cannot cross the blood brain barrier to address neurological symptoms."

    The FDA previously granted PR001 Rare Pediatric Disease Designation for the treatment of nGD, and Orphan Drug Designation for the treatment of patients with Gaucher disease. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson's disease with GBA1 mutations.

    About Fast Track Designation

    The FDA's Fast Track designation is a process designed to expedite or facilitate the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It may also allow for priority or rolling review of a company's Biologics License Application (BLA).

    About Neuronopathic Gaucher Disease

    Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease. Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as seizures and gaze and motor abnormalities. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential advantages of Fast Track designation and the potential for PR001 to serve as a potentially disease-modifying therapeutic option for patients with neuronopathic Gaucher disease. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, filed with the SEC on August 11, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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    Lisa Qu

    Ten Bridge Communications



    678-662-9166

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  9. NEW YORK, Sept. 25, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the Company will participate in the following virtual investor conferences in October:

    • Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will participate in the Jefferies Gene Therapy/Editing Virtual Summit, including a fireside chat at 11:30 a.m. ET and a panel discussion at 3:00 p.m. ET on Thursday, October 1, 2020.
    • Dr. Abeliovich will present at the Chardan's 4th Annual Genetic Medicines Conference on Tuesday, October 6, 2020 at 2:00 p.m. ET.

    Webcasts of the presentations…

    NEW YORK, Sept. 25, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the Company will participate in the following virtual investor conferences in October:

    • Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will participate in the Jefferies Gene Therapy/Editing Virtual Summit, including a fireside chat at 11:30 a.m. ET and a panel discussion at 3:00 p.m. ET on Thursday, October 1, 2020.
    • Dr. Abeliovich will present at the Chardan's 4th Annual Genetic Medicines Conference on Tuesday, October 6, 2020 at 2:00 p.m. ET.

    Webcasts of the presentations will be available under "Events and Presentations" in the Investors and Media section of the Company's website at ir.prevailtherapeutics.com following the presentations.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, filed with the SEC on August 11, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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  10. NEW YORK, Sept. 18, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will present at the virtual Oppenheimer Fall Healthcare Life Sciences & MedTech Summit on Tuesday, September 22, 2020 at 2:30 p.m. ET.

    The webcast will be available in the Events and Presentations section of the Company's website at ir.prevailtherapeutics.com. The webcast will be archived for 90 days following the presentation.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics…

    NEW YORK, Sept. 18, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will present at the virtual Oppenheimer Fall Healthcare Life Sciences & MedTech Summit on Tuesday, September 22, 2020 at 2:30 p.m. ET.

    The webcast will be available in the Events and Presentations section of the Company's website at ir.prevailtherapeutics.com. The webcast will be archived for 90 days following the presentation.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, filed with the SEC on August 11, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:

    Mary Carmichael

    Ten Bridge Communications



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  11. NEW YORK, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment of William H. Carson, M.D., to its Board of Directors.

    "Bill knows how to drive the development of innovative treatments — including drugs for a wide range of diseases that affect the brain — from concept to commercialization," said Francois Nader, M.D., Non-Executive Chairman of Prevail's Board of Directors. "We are thrilled that he will bring that expertise to bear at Prevail as we continue to develop gene therapies for patients suffering from devastating neurodegenerative diseases, including…

    NEW YORK, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment of William H. Carson, M.D., to its Board of Directors.

    "Bill knows how to drive the development of innovative treatments — including drugs for a wide range of diseases that affect the brain — from concept to commercialization," said Francois Nader, M.D., Non-Executive Chairman of Prevail's Board of Directors. "We are thrilled that he will bring that expertise to bear at Prevail as we continue to develop gene therapies for patients suffering from devastating neurodegenerative diseases, including Parkinson's disease, frontotemporal dementia and Gaucher disease."

    Dr. Carson was most recently the President and CEO of Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC), leading the development and regulatory approvals of Otsuka's global compounds including Abilify Maintena®, Rexulti®, Samsca® and Jynarque®. He also oversaw the approval of Abilify MyCite®, the first FDA-approved digital medicine. Dr. Carson joined Otsuka as Vice President of the Princeton Aripiprazole Unit in 2002, establishing the company's presence there. Before joining Otsuka, he rose through the ranks in the CNS Research and Development department at Bristol-Myers Squibb. Prior to joining the pharmaceutical industry, Dr. Carson, a board-certified psychiatrist, was an Associate Professor in the Department of Psychiatry and Behavioral Sciences at the Medical University of South Carolina.

    Dr. Carson currently serves as Chairman of the Board of Directors of OPDC, and is also the Chairman of the Board of the Sozosei Foundation, a newly established Otsuka charitable organization with a main focus on decriminalization of mental illness. He is a Board Member of Excision Biotherapeutics and Trustee of the non-profit Internet2. He is Board Chair Emeritus of the Sphinx Organization, which advocates for parity and inclusion in the arts. Dr. Carson holds an A.B. Degree from Harvard College and a M.D. degree from Case Western Reserve University. He is a Distinguished Fellow of the American Psychiatric Association, the National Medical Association and the Executive Leadership Council. Dr. Carson was named to Savoy Magazine's 2018 list of the Top 100 Most Influential Blacks in Corporate America.

    "Dr. Carson is an outstanding and important addition to our Board of Directors," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "I know that he will bring both deep medical expertise and compassion for patients to the role, as well as his stellar track record of advancing therapies through clinical development, FDA approval and commercialization."

    "I am honored to join Prevail at this exciting time for the company," said Dr. Carson. "Prevail's programs are moving forward quickly, and promising data are beginning to emerge. I am looking forward to helping the company continue to advance its mission of developing potentially disease-modifying treatments for patients with neurodegenerative disorders."

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop potentially disease-modifying treatments for patients with neurodegenerative disorders. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; initial data or other preliminary analyses or results of early clinical trials may not be predictive of final trial results or of the results of later clinical trials; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on Prevail's business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, filed with the SEC on August 11, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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    Mary Carmichael

    Ten Bridge Communications



    617-413-3543

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  12. NEW YORK, Sept. 02, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the Company will participate in the following upcoming virtual investor conferences in September:

    • Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will participate in a panel presentation at the Citi 15th Annual BioPharma Conference on Thursday, September 10 at 3:20 p.m. ET. The panel is titled "Disease-Modifying Therapies for Neurodegenerative Diseases."
       
    • Dr. Abeliovich will participate in a fireside chat at the Morgan Stanley 18th Annual Global Healthcare Conference on Monday…

    NEW YORK, Sept. 02, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the Company will participate in the following upcoming virtual investor conferences in September:

    • Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will participate in a panel presentation at the Citi 15th Annual BioPharma Conference on Thursday, September 10 at 3:20 p.m. ET. The panel is titled "Disease-Modifying Therapies for Neurodegenerative Diseases."

       
    • Dr. Abeliovich will participate in a fireside chat at the Morgan Stanley 18th Annual Global Healthcare Conference on Monday, September 14 at 12 p.m. ET.

       
    • Dr. Abeliovich will participate in a fireside chat at the Cantor Fitzgerald Global Healthcare Conference on Tuesday, September 15 at 4:40 p.m. ET.

    Webcasts of the presentations will be available under "Events and Presentations" in the Investors and Media section of the Company's website at ir.prevailtherapeutics.com following the presentations.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, filed with the SEC on August 11, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:

    Mary Carmichael

    Ten Bridge Communications



    617-413-3543

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  13. Preliminary Data Demonstrates Normalization of CSF GBA1 Enzyme Activity in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease Patients

    Company Modifies Protocol for PROPEL Trial of PR001 for Parkinson's Disease Patients with GBA1 Mutations; Expects to Continue Enrollment in Second Half of 2020

    PROVIDE Trial of PR001 for Type 2 Gaucher Disease and PROCLAIM Trial of PR006 for Frontotemporal Dementia Patients with GRN Mutations Expected to Initiate Enrollment in Second Half of 2020

    $75.0 Million At-The-Market Equity Program

    Conference call and live webcast today at 7:30 a.m. ET

    NEW YORK, Aug. 11, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying…

    Preliminary Data Demonstrates Normalization of CSF GBA1 Enzyme Activity in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease Patients

    Company Modifies Protocol for PROPEL Trial of PR001 for Parkinson's Disease Patients with GBA1 Mutations; Expects to Continue Enrollment in Second Half of 2020

    PROVIDE Trial of PR001 for Type 2 Gaucher Disease and PROCLAIM Trial of PR006 for Frontotemporal Dementia Patients with GRN Mutations Expected to Initiate Enrollment in Second Half of 2020

    $75.0 Million At-The-Market Equity Program



    Conference call and live webcast today at 7:30 a.m. ET

    NEW YORK, Aug. 11, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reviewed recent clinical and business updates and reported financial results for the second quarter ended June 30, 2020.

    "We are making strong progress advancing our lead program, PR001, for both Parkinson's disease patients with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) patients — two devastating neurodegenerative disorders with no disease-modifying treatments available," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "The first patients dosed demonstrated normalization of CSF enzyme activity in response to PR001 administration, which is very encouraging and a critical first step in establishing PR001 as a potential new therapeutic approach for patients in need of effective treatment options."

    Dr. Abeliovich added, "We are incredibly proud of the many achievements made by our team as we continue to advance our gene therapy programs into the clinic, including activation of the IND and clinical preparations for PR006 for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). Their ongoing dedication to Prevail's goal of developing groundbreaking gene therapies to help as many patients as quickly as possible is evident in our many accomplishments this year."

    "There are no effective treatment options available for patients with neuronopathic Gaucher disease," said Ari Zimran, M.D., founder, Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem. "The fact that PR001 was able to increase GCase enzyme activity to normal levels in these two patients is incredibly encouraging, and we look forward to future updates."

    Recent Business Updates:

    • Clinical Administration of PR001 Yields Early Data from Two Patients: To date, two patients have been enrolled in the Phase 1/2 PROPEL trial of PR001 for PD-GBA — one who received PR001 and another who received a sham procedure. Additionally, the company has received initial data from a Type 2 Gaucher disease patient treated with PR001 under a previously disclosed compassionate use request.



      In both treated patients, administration of PR001 resulted in normalization of glucocerebrosidase (GCase) enzyme activity levels measured in the cerebrospinal fluid (CSF) at 3 to 4 months after administration.

                   
      • The Type 2 Gaucher disease patient demonstrated an increase in CSF GCase enzyme activity from an undetectable level at baseline to 1.0 μmol/L/d at month 1 and 4.7 μmol/L/d at month 4 following PR001 administration (adult normal range: 1.1 – 8.1 μmol/L/d).

         
      • The PD-GBA patient is also diagnosed with Gaucher disease and thus has GBA1 mutations in both chromosomal copies. This patient demonstrated an increase in CSF GCase enzyme activity from an undetectable level at baseline to 3.0 μmol/L/d at month 3 following PR001 administration.

    PR001 was observed to be well tolerated in the Type 2 Gaucher disease patient, and no adverse events related to PR001 treatment have been reported. The patient is clinically stable and no apparent worsening of the patient's neurological symptoms has been observed since PR001 administration. Follow-up clinical assessments are planned.

                 

    In the case of the PD-GBA patient, approximately three months following PR001 administration, the patient experienced severe adverse events (SAEs) that are presumed to have been caused by an immune-mediated response to the AAV9 viral vector. The patient received additional immunosuppressive treatment and the SAEs have markedly resolved.



    Based on these initial efficacy and safety findings, Prevail has elected to modify the clinical protocol for the PROPEL trial in order to optimize the immunosuppression regimen, and has adapted the trial design to be open-label. The modifications have been endorsed by the independent data monitoring committee and discussed with and submitted to the FDA.

                 

    Taking into account the prior impact of COVID-19 on trial enrollment as well as this protocol amendment, the Company expects to continue enrollment in PROPEL in the second half of 2020, and to provide the next biomarker and safety analysis on a subset of patients enrolled in the PROPEL trial by mid-2021. 

    • Planning Continues for Phase 1/2 Clinical Trial for nGD: Study startup activities are continuing for the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 Gaucher disease patients, and the Company expects to initiate enrollment in the second half of 2020. The optimized immunosuppression regimen to be used in the amended PROPEL trial will also be implemented in the PROVIDE trial. In addition, the initiation of the PROGRESS Phase 1/2 clinical trial of PR001 for Type 3 Gaucher disease will be postponed until additional clinical data from the PROPEL and PROVIDE trials is available to inform the clinical development strategy for this indication.

       
    • Second Compassionate Use Patient Dosed: The Company has granted a second compassionate use request for the administration of PR001 to a child with nGD, following approval by an international regulatory authority. The second patient was recently dosed, and the procedure was well tolerated.

       
    • PROCLAIM Trial of PR006 for FTD-GRN Scheduled to Initiate Enrollment in Second Half of 2020: Study startup activities are also continuing for the PROCLAIM Phase 1/2 clinical trial of PR006 for FTD-GRN patients. The optimized immunosuppression regimen to be used in the amended PROPEL trial will also be implemented in the PROCLAIM trial.

       
    • Composition of Matter Patent Granted: On June 23, the United States Patent and Trademark Office (USPTO) issued a composition of matter patent, U.S. Patent No. 10,689,625, with claims directed to the AAV vector used in PR006, Prevail's experimental gene therapy program for the treatment of FTD-GRN. The base patent term extends until October 2038, excluding patent term extensions or coverage in additional related patent filings.

       
    • Data Presented at Annual Alzheimer's Association International Conference (AAIC): Prevail presented three poster presentations at the 2020 AAIC meeting in July. The data underscored the robust preclinical evidence in support of Prevail's AAV-based gene therapy approach, and highlighted the Company's strategy to validate these data in the planned PROCLAIM clinical trial evaluating PR006 for FTD-GRN.

       
    • Leadership Team Strengthened with Addition of General Counsel: Kira Schwartz, J.D., joined Prevail on June 1 as the Company's General Counsel. In this new role, she leads all aspects of the Company's legal organization. Prior to joining Prevail, Ms. Schwartz served as Senior Vice President, Associate General Counsel and Assistant Secretary at Allergan plc (formerly Actavis plc), where she led a legal group supporting business development, corporate governance, finance, human resources, supply chain and real estate functions.

       
    • $75 Million At-The-Market Equity Program: The Company has established an at-the-market equity program under which it may offer and sell up to $75.0 million of shares of its common stock.

    Second Quarter 2020 Financial Results

    • Cash Position: Cash, cash equivalents and investments were $131.2 million as of June 30, 2020, as compared to $149.6 million and $168.1 million as of March 31, 2020 and December 31, 2019, respectively. The Company continues to anticipate that its cash runway will extend into the first half of 2022.

       
    • R&D Expenses: R&D expenses were $12.9 million for the second quarter of 2020, compared to $12.0 million for the second quarter of 2019. The increase was primarily due to an increase of $2.6 million in direct clinical trial costs related to the PROPEL, PROVIDE, and PROCLAIM clinical trials and other trial startup costs and a $1.8 million increase in employee-related costs. These increases were partially offset by decreases of $2.6 million in direct manufacturing and process development cost due to the timing of production of clinical and preclinical supply and $1.7 million in license fees related to the options exercised under a license agreement with REGENXBIO during the three months ended June 30, 2019.

       
    • G&A Expenses: G&A expenses were $9.2 million for the second quarter of 2020, compared to $3.7 million for the second quarter of 2019. The increase was primarily due to a $4.4 million increase in legal fees, primarily related to costs associated with the ongoing arbitration matter, intellectual property patent costs and costs to operate as a public company.

       
    • Net Loss: Net loss was $22.1 million, or $0.66 loss per share, for the second quarter of 2020, compared to $15.1 million, or $0.58 loss per share, for the second quarter of 2019.

    Conference Call and Webcast Information

    Prevail will host a conference call and webcast today at 7:30 a.m. ET to discuss its second quarter 2020 financial results and other clinical and business updates.

    The webcast will be available under "Events and Presentations" in the Investors and Media section of the Company's website at ir.prevailtherapeutics.com. The conference call can be accessed by dialing 1 (866) 996-7201 (U.S. domestic) or +1 (270) 215-9495 (international) and referring to conference ID 7058186. A replay of the webcast will be archived on the Prevail Therapeutics website following the presentation.

    About Prevail Therapeutics

    Prevail is a clinical stage gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential impact of COVID-19 on Prevail's ongoing and planned clinical trials, business and operations; the potential of Prevail's gene therapies to modify the course of neurodegenerative diseases; the anticipated timing of Prevail's clinical trials of PR001 in PD-GBA and in nGD and Prevail's clinical trial of PR006; the expected timing of reporting of additional interim data for a subset of patients from the PROPEL trial; the modifications to the clinical trial protocols for PR001, PR004 and PR006 and the FDA's feedback thereon; and expectations regarding Prevail's cash runway. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises.

    These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020 and its other documents subsequently filed with or furnished to the SEC, including its Quarterly Report on Form 10-Q for the period ended June 30, 2020. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

               

    Prevail Therapeutics Inc.

    Statements of Operations

    (Unaudited)

    (in thousands, except share and per share data)

      Three Months Ended June 30,    Six Months Ended June 30, 
      2020    2019    2020    2019 
    Operating Expenses:                      
    Research and development $12,943    $11,955    $24,360    $20,366 
    General and administrative  9,208     3,713     17,070     5,598 
    Total operating loss  (22,151)    (15,668)    (41,430)    (25,964)
    Other income            210      
    Interest income, net  51     565     545     916 
    Total other income  51     565     754     916 
    Net loss $(22,100)   $(15,103)   $(40,675)   $(25,048)
    Other comprehensive loss  (1)         (1)     
    Comprehensive loss $(22,101)   $(15,103)   $(40,676)   $(25,048)
    Net loss per share, basic and diluted $(0.66)   $(0.58)   $(1.22)   $(1.05)
    Weighted average shares outstanding, basic and diluted  33,467,346     26,212,356     33,367,344     23,945,198 



    Prevail Therapeutics Inc.


    Balance Sheets

    (Unaudited)

    (in thousands, except share and per share data)

      June 30,

    2020
      December 31,

    2019
     
             
    ASSETS        
    CURRENT ASSETS:        
    Cash and cash equivalents $111,065  $168,051 
    Investments  6,458    
    Prepaid expenses and other current assets  4,620   6,410 
    Total current assets  122,143   174,461 
    Property and equipment, net  2,698   2,549 
    Investments  13,674    
    Operating lease right-of-use assets  9,355   10,001 
    Other long-term assets  2,730    
    Restricted cash  91   91 
    TOTAL ASSETS $150,691  $187,102 
    LIABILITIES AND STOCKHOLDERS' EQUITY        
    CURRENT LIABILITIES:        
    Accounts payable $1,981  $5,162 
    Accrued expenses and other current liabilities  9,435   5,330 
    Operating lease liabilities  1,447   1,341 
    Total current liabilities  12,863   11,833 
    Long-term operating lease liabilities  9,173   9,927 
    TOTAL LIABILITIES  22,036   21,760 
    COMMITMENTS AND CONTINGENCIES (Note 13)

    STOCKHOLDERS' EQUITY
            
    Preferred stock - $0.0001 par value, 10,000,000 shares authorized

    as of June 30, 2020 and December 31, 2019, respectively;

    no shares issued as of June 30, 2020 and December 31, 2019, respectively
          
    Common stock - $0.0001 par value, 200,000,000

    shares authorized as of June 30, 2020 and December 31, 2019,

    respectively, 34,214,851 and 34,138,750 shares issued and outstanding as of June 30, 2020 and December 31, 2019, respectively
      3   3 
    Additional paid-in capital  253,430   249,441 
    Accumulated deficit  (124,777)  (84,102)
    Accumulated other comprehensive loss  (1)   
    Total stockholders' equity  128,655   165,342 
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $150,691  $187,102 



    Media Contact:

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    Ten Bridge Communications



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  14. NEW YORK, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will present at the virtual 2020 Wedbush PacGrow Healthcare Conference on Tuesday, August 11, 2020 at 1:45 p.m. ET.

    The webcast will be available in the Events and Presentations section of the Company's website at ir.prevailtherapeutics.com. The webcast will be archived for 90 days following the presentation.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal…

    NEW YORK, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, will present at the virtual 2020 Wedbush PacGrow Healthcare Conference on Tuesday, August 11, 2020 at 1:45 p.m. ET.

    The webcast will be available in the Events and Presentations section of the Company's website at ir.prevailtherapeutics.com. The webcast will be archived for 90 days following the presentation.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:

    Mary Carmichael

    Ten Bridge Communications



    617-413-3543

    Investor Contact:

    Primary Logo

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  15. NEW YORK, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the company will host a conference call and live webcast on Tuesday, August 11, 2020, at 7:30 a.m. ET to discuss its second quarter 2020 financial results and other business updates.

    The webcast will be available in the Events and Presentations section of the Company's website at ir.prevailtherapeutics.com. The conference call can be accessed by dialing +1 (866) 996-7201 (U.S. domestic) or +1 (270) 215-9495 (international) and referring to conference ID 7058186. A replay of the webcast will be archived…

    NEW YORK, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the company will host a conference call and live webcast on Tuesday, August 11, 2020, at 7:30 a.m. ET to discuss its second quarter 2020 financial results and other business updates.

    The webcast will be available in the Events and Presentations section of the Company's website at ir.prevailtherapeutics.com. The conference call can be accessed by dialing +1 (866) 996-7201 (U.S. domestic) or +1 (270) 215-9495 (international) and referring to conference ID 7058186. A replay of the webcast will be archived on the Prevail Therapeutics website following the presentation.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:

    Mary Carmichael

    Ten Bridge Communications



    617-413-3543

    Investor Contact:

    Primary Logo

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  16. NEW YORK, July 27, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) on June 23, 2020 issued a composition of matter patent, U.S. Patent No. 10,689,625, with claims directed to the AAV vector used in PR006, Prevail's experimental gene therapy program for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). The base patent term extends until October 2038, excluding patent term extensions or coverage in additional related patent filings.

    "At Prevail, we are working continuously to bring…

    NEW YORK, July 27, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) on June 23, 2020 issued a composition of matter patent, U.S. Patent No. 10,689,625, with claims directed to the AAV vector used in PR006, Prevail's experimental gene therapy program for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). The base patent term extends until October 2038, excluding patent term extensions or coverage in additional related patent filings.

    "At Prevail, we are working continuously to bring innovative treatments to patients with neurodegenerative diseases," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "FTD-GRN is a devastating condition, with no disease-modifying therapeutic options available. We are excited about the possibility of making a significant impact with PR006."

    In March of this year, Prevail announced an active IND for the Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN. The U.S. Food and Drug Administration (FDA) has granted the therapy Fast Track Designation.

    PR006 is being developed as a potential one-time gene therapy for FTD-GRN, a progressive neurodegenerative disease caused by mutations in the GRN gene that reduce production of progranulin, a protein critical for lysosomal function, neuronal survival and normal microglial activities. The progranulin deficiency leads to lysosomal dysfunction, ineffective protein degradation and recycling, neuroinflammation, and ultimately neurodegeneration and death, typically within three to ten years of diagnosis.

    PR006 is designed to increase progranulin levels in the brains of FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

    About Frontotemporal Dementia with GRN Mutations

    Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer's disease. FTD affects 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union. FTD-GRN represents 5-10% of all patients with FTD. FTD results from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language. In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation, and neurodegeneration. There are no approved treatments for FTD or FTD-GRN.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. In addition to PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN), the company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease, and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:

    Mary Carmichael

    Ten Bridge Communications



    617-413-3543

    Investor Contact:

    Primary Logo

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  17. Preclinical Data Demonstrate Potential of Prevail's AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

    Company Provides Overview of Planned Phase 1/2 PR006 PROCLAIM Clinical Trial for FTD-GRN Patients

    Company to Host a Panel Discussion and Q&A Session on FTD-GRN
    with Jonathan Rohrer, Ph.D., MRCP, an Expert in Frontotemporal Dementia (FTD) and its Genetic Causes

    NEW YORK, July 22, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced three upcoming poster presentations at the Alzheimer's Association International Conference (AAIC) 2020…

    Preclinical Data Demonstrate Potential of Prevail's AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

    Company Provides Overview of Planned Phase 1/2 PR006 PROCLAIM Clinical Trial for FTD-GRN Patients

    Company to Host a Panel Discussion and Q&A Session on FTD-GRN

    with Jonathan Rohrer, Ph.D., MRCP, an Expert in Frontotemporal Dementia (FTD) and its Genetic Causes

    NEW YORK, July 22, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced three upcoming poster presentations at the Alzheimer's Association International Conference (AAIC) 2020. These data underscore the robust preclinical evidence in support of Prevail's AAV-based gene therapy approach, and highlight the Company's strategy to validate these data in the planned PROCLAIM clinical trial evaluating PR006 for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). The conference will be held virtually July 27-31, 2020.

    "Our novel gene therapy candidates have the potential to transform the treatment of patients with FTD-GRN and other devastating neurodegenerative diseases," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "An increased understanding of genetically defined forms of neurodegenerative diseases, including the role that GRN mutations can play in frontotemporal dementia, has opened exciting possibilities to stop or slow disease progression using a gene therapy approach."

    PR001

    Prevail will highlight preclinical data demonstrating the effect of PR001 treatment on key disease biomarkers and functional parameters. These data provide the basis for the Company's clinical trials for Type 2 neuronopathic Gaucher disease (nGD) patients and Parkinson's disease with GBA1 mutations (PD-GBA) patients.

    Poster title: PR001 Gene Therapy Improved Phenotypes in Models of Parkinson's Disease with GBA1 Mutation

    Session date and time: Monday, July 27, 12:00 a.m. - 11:59 p.m. CDT

    PR006



    Prevail will present the design of the PROCLAIM Phase 1/2 clinical trial for the treatment of FTD-GRN patients and preclinical data demonstrating the effect of PR006 treatment on progranulin expression, lysosomal dysfunction and inflammation in the CNS.

    Poster title: Preclinical Development of PR006, a Gene Therapy for the Treatment of Frontotemporal Dementia with Progranulin Mutations

    Session date and time: Monday, July 27, 12:00 a.m. - 11:59 p.m. CDT

    Poster title: Design of a Phase 1/2 Study of an AAV9-Based Gene Therapy for Fronto-Temporal Dementia Patients with Pathogenic GRN Mutations (PROCLAIM Trial)

    Session date and time: Wednesday, July 29, 12:00 a.m. - 11:59 p.m. CDT

    FTD-GRN Panel Discussion and Q&A Session

    In addition to its presentations at AAIC, on Wednesday, July 29 at 2:30 p.m. EDT, management will be hosting a panel discussion and Q&A session on FTD-GRN by Jonathan Rohrer, Ph.D., MRCP, an expert in frontotemporal dementia (FTD) and its genetic causes. A live webcast of the event and replay following its conclusion will be available on the Events and Presentations section of the Company's website at https://ir.prevailtherapeutics.com/events.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail's gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:

    Mary Carmichael

    Ten Bridge Communications



    617-413-3543

    Investor Contact:

    Primary Logo

    View Full Article Hide Full Article
  18. NEW YORK, June 04, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment of Kira Schwartz, J.D., as the Company's General Counsel. In this new role, Ms. Schwartz will lead all aspects of the Company's legal organization.

    "We are excited to welcome Kira to Prevail as our general counsel and latest member of our executive team," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "Prevail will greatly benefit from Kira's significant expertise as a senior legal counselor within the biopharmaceutical industry. She will play a key role in…

    NEW YORK, June 04, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment of Kira Schwartz, J.D., as the Company's General Counsel. In this new role, Ms. Schwartz will lead all aspects of the Company's legal organization.

    "We are excited to welcome Kira to Prevail as our general counsel and latest member of our executive team," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "Prevail will greatly benefit from Kira's significant expertise as a senior legal counselor within the biopharmaceutical industry. She will play a key role in shaping our internal legal function and advising the Board and leadership team on all legal and corporate governance issues as we continue to advance our AAV gene therapy-based pipeline through the clinic for patients with neurodegenerative diseases."

    "Prevail is well-positioned to be a leader in developing transformative treatments for patients with neurodegenerative disorders, and I look forward to joining such a mission-driven team at an important point in the Company's growth and development, with anticipated clinical advancements in the year ahead," said Ms. Schwartz.

    Prior to joining Prevail, Ms. Schwartz served as Senior Vice President, Associate General Counsel and Assistant Secretary at Allergan plc (formerly Actavis plc), where she led a legal group supporting business development, corporate governance, finance, human resources, supply chain and real estate functions. As Vice President, Associate General Counsel at Actavis, she led Actavis' $70.5 billion acquisition of Allergan, Inc. Previously, she held senior leadership positions at Forest Laboratories, Inc. (acquired by Actavis), where she oversaw a variety of projects, including business development, manufacturing and supply chain, R&D and more, and was senior corporate counsel in Pfizer Inc.'s business transactions group. Ms. Schwartz started her career at Cleary, Gottlieb, Steen & Hamilton LLP. She received her J.D. from Yale Law School and a B.A. in economics from Tufts University.

    About Prevail Therapeutics

    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications

    617-413-3543

    Investor Contact:

    Primary Logo

    View Full Article Hide Full Article
  19. NEW YORK, May 26, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer, will present a company overview at the upcoming Jefferies Virtual Global Healthcare Conference on Tuesday, June 2, 2020 at 3:30 p.m. ET. 

    A live webcast of the presentation will be available under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com. The webcast will be archived for 90 days following the event.

    About Prevail Therapeutics
    Prevail is a…

    NEW YORK, May 26, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer, will present a company overview at the upcoming Jefferies Virtual Global Healthcare Conference on Tuesday, June 2, 2020 at 3:30 p.m. ET. 

    A live webcast of the presentation will be available under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com. The webcast will be archived for 90 days following the event.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications

    617-413-3543

    Investor Contact:

     

    Primary Logo

    View Full Article Hide Full Article
  20. CEO, CTO to Discuss Company's Manufacturing and CMC Strategy and Overall Industry Trends

    NEW YORK, May 21, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, and Yong Dai, Ph.D., Chief Technology Officer of Prevail will participate in a virtual fireside chat at the Chardan 4th Annual Genetic Medicines Manufacturing Summit on Tuesday, May 26, 2020.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing…

    CEO, CTO to Discuss Company's Manufacturing and CMC Strategy and Overall Industry Trends

    NEW YORK, May 21, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail, and Yong Dai, Ph.D., Chief Technology Officer of Prevail will participate in a virtual fireside chat at the Chardan 4th Annual Genetic Medicines Manufacturing Summit on Tuesday, May 26, 2020.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications

    617-413-3543

    Investor Contact:

    Primary Logo

    View Full Article Hide Full Article
  21. Phase 1/2 Trial of PR001 for Parkinson's Disease with GBA1 Mutations Ongoing; Study Startup Activities
    Progressing for Phase 1/2 Trials of PR001 for Type 2 Neuronopathic
     Gaucher Disease and PR006 for
    Frontotemporal Dementia with GRN Mutations

    Data Presentations Highlight Potential of AAV Gene Therapy Approach to
    Slow or Stop Neurodegenerative Disease Progression in Preclinical Models

    NEW YORK, May 14, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reviewed recent business highlights and reported financial results for the first quarter ended March 31, 2020.

    "We are excited to continue…

    Phase 1/2 Trial of PR001 for Parkinson's Disease with GBA1 Mutations Ongoing; Study Startup Activities
    Progressing for Phase 1/2 Trials of PR001 for Type 2 Neuronopathic
     Gaucher Disease and PR006 for
    Frontotemporal Dementia with GRN Mutations

    Data Presentations Highlight Potential of AAV Gene Therapy Approach to
    Slow or Stop Neurodegenerative Disease Progression in Preclinical Models

    NEW YORK, May 14, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reviewed recent business highlights and reported financial results for the first quarter ended March 31, 2020.

    "We are excited to continue the clinical development of PR001 and are on track to report interim data for a subset of patients from our Phase 1/2 clinical trial of PR001 for Parkinson's disease with GBA1 mutations (PD-GBA) later this year. In addition, we are advancing our AAV gene therapy-based pipeline, with the planned mid-year initiation of Phase 1/2 clinical trials of PR001 for Type 2 neuronopathic Gaucher disease (nGD) and PR006 for frontotemporal dementia with GRN mutations (FTD-GRN)," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "In addition, at ASGCT and AAT-AD/PD, we presented or will present data that validate the potential of these products for neurodegenerative disease patients with urgent unmet needs, and detailed our ongoing and planned clinical trials."

    Recent Business Highlights and Updates:

    • Data and Clinical Trial Designs Presented or to be Presented for PR001 in nGD and PR006 in FTD-GRN at the 2020 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting: In separate presentations, Prevail described the design of two planned clinical trials: the PROVIDE Phase 1/2 clinical trial of PR001 for the treatment of patients with Type 2 nGD, and the PROCLAIM Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN. Preclinical data serving as the basis for the company's clinical programs, including PR001 for nGD and PD-GBA and PR006 for FTD-GRN, will also be presented.
    • Data and Clinical Trial Design Presented for PR001 in PD-GBA and PR006 in FTD-GRN at the AAT-AD/PD Focus Meeting 2020: Advances in Alzheimer's and Parkinson's Therapies: Presentations at the AAT-AD/PD Focus Meeting included details on the design of Prevail's PROPEL Phase 1/2 clinical trial to evaluate the safety and efficacy of PR001 in patients with PD-GBA, and preclinical data related to PR001 for PD-GBA and PR006 for FTD-GRN.
    • Clinical Development of PR001: As announced in March 2020, Prevail initiated dosing for the PROPEL Phase 1/2 trial of PR001 for patients with PD-GBA. As previously reported, in response to the COVID-19 pandemic, trial sites had temporarily suspended patient screening and enrollment activity. Prevail now expects patient screening to resume by the end of the second quarter of 2020 at certain clinical sites. The Company remains on track to report interim data on a subset of patients in the second half of 2020.

      In addition, study startup activities are continuing for the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 nGD, and the Company intends to initiate dosing in mid-2020. Prevail also continues to expect to initiate the PROGRESS Phase 1/2 clinical trial of PR001 for Type 3 nGD in the second half of 2020. The timelines for PR001 are subject to any delays related to the COVID-19 pandemic.

      Clinical Development of PR006: Study startup activities are also underway for the PROCLAIM Phase 1/2 clinical trial of PR006 for FTD-GRN patients, which is planned to initiate in mid-2020, subject to any delays related to the COVID-19 pandemic.

    First Quarter 2020 Financial Results

    • Cash Position: Cash and cash equivalents were $149.6 million as of March 31, 2020, as compared to $168.1 million as of December 31, 2019. As previously guided, the Company expects its current cash position to be sufficient to fund operating expenses into the first half of 2022.
    • R&D Expenses: R&D expenses were $11.4 million for the three months ended March 31, 2020, compared to $8.4 million for the three months ended March 31, 2019. The increase was primarily related to the Company's development programs, specifically as a result of increased clinical trial activities, and headcount.
    • G&A Expenses: G&A expenses were $7.9 million for the three months ended March 31, 2020, compared to $1.9 million for the three months ended March 31, 2019. The increase was primarily due to an increase in professional services fees associated with the ongoing arbitration matter, personnel costs resulting from increased headcount, and other corporate and public company-related expenses.
    • Net Loss: Net loss was $18.6 million, or $0.56 loss per share, for the three months ended March 31, 2020, compared to $9.9 million, or $1.73 loss per share, for the three months ended March 31, 2019.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential impact of COVID-19 on Prevail's ongoing and planned clinical trials, business and operations; the potential of Prevail's gene therapies to modify the course of neurodegenerative diseases; the anticipated timing of Prevail's clinical trials of PR001 in PD-GBA and in nGD and Prevail's clinical trial of PR006, including resuming of delayed trials and initiation of new trials; the expected timing of reporting of interim data for a subset of patients from Prevail's Phase 1/2 clinical trial of PR001; and expectations regarding Prevail's cash runway. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises.

    These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on or about May 14, 2020, the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2019, filed with the SEC on March 26, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Prevail Therapeutics Inc.
    Statements of Operations
    (Unaudited)
    (in thousands, except share and per share data)

        Three Months Ended March 31,  
        2020     2019  
    Operating Expenses:                
    Research and development   $ 11,417     $ 8,411  
    General and administrative     7,862       1,885  
    Total operating loss     (19,279 )     (10,296 )
    Other income     210        
    Interest income     494       351  
    Total other income     704       351  
    Net loss   $ (18,575 )   $ (9,945 )
    Net loss per share, basic and diluted   $ (0.56 )   $ (1.73 )
    Weighted average shares outstanding, basic and diluted     33,267,342       5,740,874  


    Balance Sheets

    (in thousands, except share and per share data)

        March 31,
    2020
        December 31,
    2019
     
        (unaudited)          
    ASSETS                
    CURRENT ASSETS:                
    Cash and cash equivalents     149,580       168,051  
    Prepaid expenses and other current assets     4,909       6,410  
    Total current assets     154,489       174,461  
    Property and equipment, net     2,725       2,549  
    Operating lease right-of-use assets     9,682       10,001  
    Other long-term assets     1,817        
    Restricted cash     91       91  
    TOTAL ASSETS   $ 168,804     $ 187,102  
    LIABILITIES AND STOCKHOLDERS' EQUITY                
    CURRENT LIABILITIES:                
    Accounts payable     3,943     $ 5,162  
    Accrued expenses and other current liabilities     5,452       5,330  
    Operating lease liabilities     1,395       1,341  
    Total current liabilities     10,790       11,833  
    Long-term operating lease liabilities     9,553       9,927  
    TOTAL LIABILITIES     20,343       21,760  
    COMMITMENTS AND CONTINGENCIES (Note 12)
    STOCKHOLDERS' EQUITY
                   
    Preferred stock - $0.0001 par value, 10,000,000 shares authorized
    as of March 31, 2020 and December 31, 2019, respectively;
    no shares issued as of March 31, 2020 and December 31, 2019, respectively
               
    Common stock - $0.0001 par value, 200,000,000
    shares authorized as of March 31, 2020 and December 31, 2019,
    respectively, 34,196,456 and 34,138,750 shares issued and outstanding as of March 31, 2020 and December 31, 2019, respectively
        3       3  
    Additional paid-in capital     251,135       249,441  
    Accumulated deficit     (102,677 )     (84,102 )
    Total stockholders' equity     148,461       165,342  
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY   $ 168,804     $ 187,102  


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    Ten Bridge Communications

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  22. NEW YORK, May 05, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer, will participate in a fireside chat at the Bank of America Global Research Virtual Health Care Conference 2020 on Tuesday, May 12, 2020 at 11:40 a.m. ET. 

    A live webcast of the fireside chat will be available under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com. The webcasts will be archived for 90 days following the event.

    About Prevail Therapeutics

    NEW YORK, May 05, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer, will participate in a fireside chat at the Bank of America Global Research Virtual Health Care Conference 2020 on Tuesday, May 12, 2020 at 11:40 a.m. ET. 

    A live webcast of the fireside chat will be available under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com. The webcasts will be archived for 90 days following the event.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications

    617-413-3543

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  23. Preclinical Data Demonstrate Potential of Prevail's AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

    Company Provides Design Overview for Planned Phase 1/2 PR001 PROVIDE and PR006 PROCLAIM Clinical Trials

    NEW YORK, April 29, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced two upcoming oral presentations and two poster presentations at the virtual 2020 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. These data underscore the robust preclinical evidence in support of Prevail's AAV-based gene therapy approach, and highlight…

    Preclinical Data Demonstrate Potential of Prevail's AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

    Company Provides Design Overview for Planned Phase 1/2 PR001 PROVIDE and PR006 PROCLAIM Clinical Trials

    NEW YORK, April 29, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced two upcoming oral presentations and two poster presentations at the virtual 2020 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. These data underscore the robust preclinical evidence in support of Prevail's AAV-based gene therapy approach, and highlight the company's strategy to validate these data in multiple ongoing or planned clinical trials.

    "Patients living with neurodegenerative diseases are in urgent need of novel therapies with the potential to slow or stop the course of their disease," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "Our gene therapy candidates have the potential to improve outcomes for patients with genetically defined forms of Parkinson's disease, frontotemporal dementia and neuronopathic Gaucher disease."

    PR001

    Prevail will highlight the design of the planned PROVIDE Phase 1/2 trial for the treatment of Type 2 neuronopathic Gaucher disease (nGD) patients and preclinical data demonstrating the impact of PR001 treatment on key disease biomarkers and functional parameters. These data provide the basis for the company's clinical programs in nGD and Parkinson's disease with GBA1 mutations (PD-GBA).

    Poster title: Design of a Phase 1/2 Study to Evaluate the Safety and Efficacy of an AAV9-Based Gene Therapy in Infants with Type 2 Gaucher Disease (PROVIDE Trial)
    Session title: AAV Vectors - Clinical Studies
    Session date and time: Wednesday, May 13th, 5:30 p.m.-6:30 p.m. ET

    Presentation title: PR001 Gene Therapy Increased GCase Activity and Ameliorated GBA1-Associated Disease Phenotypes
    Session title: AAV Vectors Preclinical and Proof-of-Concept Studies in Systemic Diseases 
    Presentation date and time: Thursday, May 14th 3:45 p.m.-4:00 p.m. ET
    Presenter: Patty Sheehan, Ph.D., Senior Scientist, Prevail Therapeutics

    PR006

    Prevail will present the design of the PROCLAIM Phase 1/2 clinical trial for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN) and preclinical data demonstrating the impact of PR006 treatment on progranulin expression, lysosomal dysfunction and inflammation in the CNS.

    Poster title: Proposed Patient Population and Outcome Measures for a First in Human Study of PR006, an AAV9-Based Gene Therapy, for Fronto-Temporal Dementia Patients with Pathogenic GRN Mutations
    Session title: AAV Vectors - Clinical Studies
    Session date and time: Wednesday, May 13th, 5:30 p.m.-6:30 p.m. ET

    Presentation title: PR006, an AAV Gene Therapy Vector Expressing Progranulin, Improved FTD-GRN Phenotypes In Vitro and In Vivo
    Session title: AAV Vectors Preclinical and Proof-of-Concept Studies in CNS Disorders
    Presentation date and time: Friday, May 15th 10:15 a.m.-10:30 a.m. ET
    Presenter: Laura D. Heckman, Ph.D., Director, Preclinical Development, Prevail Therapeutics

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

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  24. Design of PROPEL Phase 1/2 Clinical Trial for Parkinson's Disease Patients with GBA1 Mutations to Be Presented

    Preclinical Data of PR001 for Parkinson's Disease with GBA1 Mutations and PR006 for Frontotemporal Dementia with GRN Mutations to Be Presented

    NEW YORK, March 31, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced three upcoming presentations at the AAT-AD/PD Focus Meeting 2020: Advances in Alzheimer's and Parkinson's Therapies, taking place virtually April 2-5, 2020. All presentations will be available on-demand for registered participants in the AAT-AD/PD™ Virtual…

    Design of PROPEL Phase 1/2 Clinical Trial for Parkinson's Disease Patients with GBA1 Mutations to Be Presented

    Preclinical Data of PR001 for Parkinson's Disease with GBA1 Mutations and PR006 for Frontotemporal Dementia with GRN Mutations to Be Presented

    NEW YORK, March 31, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced three upcoming presentations at the AAT-AD/PD Focus Meeting 2020: Advances in Alzheimer's and Parkinson's Therapies, taking place virtually April 2-5, 2020. All presentations will be available on-demand for registered participants in the AAT-AD/PD™ Virtual Meeting platform. The presentations include information about Prevail's PR001 program for Parkinson's disease patients with GBA1 mutations (PD-GBA), and Prevail's PR006 program for frontotemporal dementia patients with GRN mutations (FTD-GRN). 

    Preclinical data related to PR001 for PD-GBA and the design of PROPEL, Prevail's Phase 1/2 clinical trial to evaluate the safety and efficacy of PR001 in patients with PD-GBA, will be presented on Saturday, April 4, 2020:

    Presentation title: PR001 Gene Therapy Improves PD-GBA Phenotypes in Mouse Models by Increasing GCase Activity
    Symposium title: Emerging Therapies for PD Related to Glucocerebroside Mutations: From Bench to First in Human Trial
    Date: Saturday, April 4, 2020
    Presenter: Asa Abeliovich, M.D., Ph.D., Founder and CEO, Prevail Therapeutics

    Presentation title: Design of the First-in-Human Trial of Novel AAV9-based Gene Therapy for Parkinson's Disease with Pathogenic GBA1 Mutations
    Symposium title: Emerging Therapies for PD Related to Glucocerebroside Mutations: From Bench to First in Human Trial
    Date: Saturday, April 4, 2020
    Presenter: Olga Uspenskaya, M.D., Ph.D., Senior Medical Director, Prevail Therapeutics

    Preclinical data related to PR006 for FTD-GRN will also be presented on Saturday, April 4, 2020:

    Presentation title: PR006 Gene Therapy Improves FTD-GRN Phenotypes in Model Systems by Increasing Progranulin Levels
    Symposium title: ALS, FTD and PSP: Diagnosis and Treatment
    Date: Saturday, April 4, 2020
    Presenter: Laura D. Heckman, Ph.D., Director, Prevail Therapeutics

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

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  25. Dosing Initiated in PROPEL Phase 1/2 Trial of PR001 for Treatment of Parkinson's Disease with GBA1 Mutations

    Preparations Underway to Initiate PROVIDE Phase 1/2 Trial of PR001 for Treatment of Type 2 Neuronopathic Gaucher Disease in Mid-2020

    IND for PR006 for Treatment of Patients with Frontotemporal Dementia with GRN Mutation Active; Initiation of PROCLAIM Phase 1/2 Trial Planned for Mid-2020

    Cash Runway into First Half of 2022

    NEW YORK, March 26, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reported financial results for the full year ended December 31, 2019, and reviewed recent…

    Dosing Initiated in PROPEL Phase 1/2 Trial of PR001 for Treatment of Parkinson's Disease with GBA1 Mutations

    Preparations Underway to Initiate PROVIDE Phase 1/2 Trial of PR001 for Treatment of Type 2 Neuronopathic Gaucher Disease in Mid-2020

    IND for PR006 for Treatment of Patients with Frontotemporal Dementia with GRN Mutation Active; Initiation of PROCLAIM Phase 1/2 Trial Planned for Mid-2020

    Cash Runway into First Half of 2022

    NEW YORK, March 26, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reported financial results for the full year ended December 31, 2019, and reviewed recent business highlights.

    "2019 was an important year for Prevail as we continued to make important clinical and regulatory progress across our portfolio of novel AAV9-based gene therapy candidates for neurodegenerative diseases and brought the company public," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "Dosing has begun in our PROPEL clinical trial of PR001 for patients with Parkinson's disease with GBA1 mutations, or PD-GBA. Additionally, we are working to initiate three more Phase 1/2 clinical trials this year: the PROVIDE and PROGRESS trials of PR001 for the treatment of Type 2 and Type 3 neuronopathic Gaucher disease, or nGD, respectively, and the PROCLAIM trial of PR006 for the treatment of frontotemporal dementia patients with GRN mutation, or FTD-GRN — potentially the first gene therapy for this condition to enter clinical trials. Our ability to advance multiple candidates towards the clinic over the last year reflects the dedication of our team, the potential reach of our platform, and the urgent need for novel potentially disease-modifying therapies for patients with these genetically-defined neurodegenerative diseases."

    Recent Business Highlights and Updates:             

    • PROPEL trial for PD-GBA underway: Prevail has initiated dosing in its PROPEL Phase 1/2 trial of PR001 for patients with PD-GBA. In response to the COVID-19 pandemic, trial sites have temporarily suspended patient screening and enrollment activity. The Company still intends to report interim data on a subset of patients in the second half of 2020, however, this may be impacted if there is a prolonged suspension of enrollment. This randomized, double-blind trial is enrolling up to 16 moderate-to-severe PD-GBA patients to evaluate the safety and tolerability of two escalating doses of PR001, and will measure key biomarkers and exploratory efficacy endpoints. Currently, there are no treatments available to slow or stop disease progression for PD-GBA patients.
    • Clinical Development of PR001 for nGD Advancing: Prevail announced in December 2019 that its Investigational New Drug (IND) application for PR001 for the treatment of nGD has been accepted by the U.S. Food and Drug Administration (FDA) and is now active. Study startup activities are ongoing for the PROVIDE Phase 1/2 clinical trial for Type 2 nGD. The Company intends to initiate dosing in the PROVIDE trial in mid-2020 and in the PROGRESS Phase 1/2 clinical trial for Type 3 nGD in the second half of 2020, in each case, subject to any delays related to the COVID-19 pandemic. There are no effective treatments available to address the neurological manifestations associated with either type of neuronopathic Gaucher disease.
    • PR001 Granted Orphan Drug and Rare Pediatric Disease Designations: The FDA has granted Orphan Drug Designation for PR001 for the treatment of patients with Gaucher disease, and Rare Pediatric Disease Designation for the treatment of nGD, the most severe form of the condition. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity. Programs with Rare Pediatric Disease status may, upon marketing approval, qualify for a fully transferable priority review voucher applicable to a subsequent marketing application.
    • IND Active for PR006 for the Treatment of Patients with FTD-GRN: Prevail announced in March 2020 that the FDA accepted the Company's IND application for PR006 for the treatment of FTD-GRN. FTD-GRN is a fatal, rapidly progressive, early-onset form of dementia for which there are no FDA approved treatments. The Company believes that PR006, which is being developed as a potential one-time gene therapy, has the ability to slow or stop progression of FTD-GRN. Prevail plans to initiate the PROCLAIM Phase 1/2 clinical trial for FTD-GRN patients in mid-2020, subject to any delays related to the COVID-19 pandemic.
    • PR006 Granted Orphan Drug and Fast Track Designations: The FDA has granted Orphan Drug Designation for PR006 for the treatment of FTD and Fast Track Designation for FTD-GRN. Programs with Fast Track status receive accelerated approval or rolling review of a company's Biologics License Application (BLA). In addition, such a product candidate could be eligible for Priority Review if supported by clinical data at the time of BLA submission.
    • Preclinical Data Presented for PR001 in nGD and PR006 in FTD-GRN at WORLDSymposium: Prevail presented preclinical data at the 16th Annual WORLDSymposium in February 2020 demonstrating proof of concept in both nGD and FTD-GRN. Prevail also hosted a presentation and roundtable discussion during the WORLDSymposium featuring Professor Ari Zimran, M.D., a key opinion leader in the field of nGD. A recording of this event, including Dr. Zimran's presentation "Unmet Needs and Emerging Therapeutic Options for Neuronopathic Gaucher Disease," can be found under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com.
    • Strengthened Leadership with Board Appointment: Prevail appointed Morgan Sheng, Ph.D., to its Board of Directors. Dr. Sheng, who brings a wealth of expertise in neurodegenerative disease drug development, is a Core Institute Member and Co-Director of the Stanley Center for Psychiatric Research at the Broad Institute of MIT and Harvard, and a Professor in the Department of Brain and Cognitive Science at MIT. Previously, he was Vice President of Neuroscience at Genentech, where he was head of neuroscience research and drug discovery.             

    Full Year 2019 Financial Results

    • Cash Position: Cash and cash equivalents were $168.1 million as of December 31, 2019, as compared to $63.0 million as of December 31, 2018. Following a strategic review and prioritization of programs, the Company expects its current cash position to be sufficient to fund operating expenses into the first half of 2022, versus mid-2021 as previously guided.
    • R&D Expenses: R&D expenses were $48.8 million for the year ended December 31, 2019, compared to $14.1 million for the year ended December 31, 2018. The increase was primarily related to the Company's development programs, as a result of increased manufacturing-related spend, clinical and preclinical activities, and headcount.
    • G&A Expenses: G&A expenses were $17.0 million for the year ended December 31, 2019, compared to $4.7 million for the year ended December 31, 2018. The increase was primarily due to an increase in personnel costs resulting from increased headcount, professional services fees, and other corporate-related expenses.
    • Net Loss: Net loss was $63.2 million, or $2.22 loss per share, for the year ended December 31, 2019, compared to $19.1 million, or $3.71 loss per share, for the year December 31, 2018.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with a GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential impact of COVID-19 on Prevail's ongoing and planned clinical trials, business and operations; the potential of Prevail's gene therapies to modify the course of neurodegenerative diseases; the anticipated timing of Prevail's Phase 1/2 clinical trials of PR001 in PD-GBA and in nGD and Prevail's clinical trial of PR006; and the potential impacts of Orphan Drug and Rare Pediatric Disease Designations. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises.

    These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2019, to be filed with the SEC on or about March 26, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.


    Prevail Therapeutics Inc.
    Statements of Operations
    (in thousands, except share and per share data)

        Year ended
    December 31,
    2019
        Year ended
    December 31,
    2018
     
    Operating Expenses:                
    Research and development   $ 48,798     $ 14,127  
    General and administrative     17,005       4,682  
    Operating loss     (65,803 )     (18,809 )
    Change in fair value of derivative liabilities           (781 )
    Other income           87  
    Interest income     2,615       887  
    Interest expense           (471 )
    Total other income (expense), net     2,615       (278 )
    Net loss   $ (63,188 )   $ (19,087 )
    Net loss per share                
    Basic and diluted   $ (2.22 )   $ (3.71 )
    Weighted average shares outstanding:                
    Basic and diluted     28,494,950       5,145,469  


    Prevail Therapeutics Inc.
    Balance Sheets
    (in thousands, except share and per share data)

        December 31,
    2019
        December 31,
    2018
     
    Assets                
    Current assets:                
    Cash and cash equivalents   $ 168,051     $ 63,014  
    Prepaid expenses and other current assets     6,410       563  
    Total current assets     174,461       63,577  
    Property and equipment, net     2,549       678  
    Operating lease right-of-use assets     10,001       8,534  
    Restricted cash     91       91  
    Total assets   $ 187,102     $ 72,880  
    Liabilities, redeemable convertible preferred stock, and stockholders' equity (deficit)                
    Current liabilities:                
    Accounts payable   $ 5,162     $ 1,241  
    Accrued expenses and other current liabilities     5,330       1,477  
    Operating lease liabilities     1,341       917  
    Total current liabilities     11,833       3,635  
    Long-term operating lease liabilities     9,927       7,952  
    Total liabilities     21,760       11,587  
    Commitments and contingencies (see Note 15)
    redeemable convertible preferred stock
                   
    Series Seed Preferred Stock - $0.0001 par value, 0 and 6,480,000 shares authorized, issued and outstanding as of December 31, 2019 and 2018, respectively           3,524  
    Series A Preferred Stock - $0.0001 par value, 0 and 9,072,000 shares authorized, 0 and 8,997,085 shares issued and outstanding as of December 31, 2019 and 2018, respectively           76,186  
    Stockholders' equity (deficit)                
    Common stock - $0.0001 par value, 200,000,000 and 28,398,600 shares authorized as of December 31, 2019 and 2018, respectively, 34,098,819 and 7,209,000 shares issued and outstanding as of December 31, 2019 and 2018, respectively     3       1  
    Additional paid-in capital     249,441       2,496  
    Accumulated deficit     (84,102 )     (20,914 )
    Total stockholders' equity (deficit)     165,342       (18,417 )
    Total liabilities, redeemable convertible preferred stock, and stockholders' equity (deficit)     187,102     $ 72,880  


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  26. NEW YORK, March 24, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company's experimental gene therapy program, PR006, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN). Prevail announced an active IND for the Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN earlier this month.

    "The FDA's decision to grant Fast Track Designation for PR006 is an important step forward in our mission to deliver a potentially disease-modifying gene therapy…

    NEW YORK, March 24, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company's experimental gene therapy program, PR006, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN). Prevail announced an active IND for the Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN earlier this month.

    "The FDA's decision to grant Fast Track Designation for PR006 is an important step forward in our mission to deliver a potentially disease-modifying gene therapy to FTD-GRN patients as quickly as possible," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "FTD-GRN progresses rapidly and there are currently no therapeutic options available. We believe PR006 has the potential to fill this unmet medical need and make a significant impact for patients."

    About Fast Track Designation
    The FDA's Fast Track designation is a process designed to facilitate the development and expedite the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It allows for accelerated approval or rolling review of a company's Biologics License Application (BLA). In addition, such a product candidate could be eligible for Priority Review if supported by clinical data at the time of BLA submission.

    About Frontotemporal Dementia with a GRN Mutation
    Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer's disease. FTD affects 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union. FTD-GRN represents 5-10% of all patients with FTD. FTD results from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language. In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation, and neurodegeneration. There are no approved treatments for FTD or FTD-GRN.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR006 for patients with frontotemporal dementia with a GRN mutation (FTD-GRN); PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases and slow the progression of FTD-GRN. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications

    617-413-3543

    Investor Contact:

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  27. NEW YORK, March 03, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, provided an update today on the clinical advancement of its gene therapy program PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA). Enrollment in the PR001 Phase 1/2 PROPEL clinical trial is progressing, patient dosing continues, and the Company is on track to report interim data on a subset of patients in the second half of 2020. 

    The Company will present on its clinical progress at the Cowen & Co. Annual Healthcare Conference in Boston today.

    "We believe the PROPEL trial makes PR001 the first potentially…

    NEW YORK, March 03, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, provided an update today on the clinical advancement of its gene therapy program PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA). Enrollment in the PR001 Phase 1/2 PROPEL clinical trial is progressing, patient dosing continues, and the Company is on track to report interim data on a subset of patients in the second half of 2020. 

    The Company will present on its clinical progress at the Cowen & Co. Annual Healthcare Conference in Boston today.

    "We believe the PROPEL trial makes PR001 the first potentially disease-modifying gene therapy for PD-GBA patients to enter clinical trials. Its ongoing progress brings us a step closer to new treatment options for patients living with PD-GBA," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "We are excited about the potential of PR001 to slow or stop disease progression for PD-GBA patients."

    The PROPEL trial is a randomized, double-blind Phase 1/2 clinical trial evaluating the safety and tolerability of two escalating dose levels of PR001 in up to 16 patients with moderate-to-severe PD-GBA. The trial also evaluates the effect of PR001 on biomarkers of disease activity and on Parkinson's disease clinical efficacy measures. Full trial details are available at clinicaltrials.gov.

    PR001 utilizes an AAV9 viral vector to deliver the GBA1 gene to a patient's cells, correcting the lysosomal enzyme deficiency caused by PD-GBA patients' GBA1 mutations. GBA1 encodes the lysosomal enzyme, beta-glucocerebrosidase, or GCase, which is required for the disposal and recycling of glycolipids. PD-GBA patients have a mutation in at least one chromosomal copy of GBA1.

    In addition to the PROPEL clinical trial for patients with PD-GBA, PR001 is also being developed for neuronopathic Gaucher disease, a devastating disorder that shares the same underlying genetic mechanism. In December 2019, the Company announced that its IND for PR001 for the treatment of neuronopathic Gaucher disease is active.

    About PD-GBA
    Parkinson's disease is a chronic, progressive neurodegenerative disorder that affects up to one million people in the United States and more than seven million people worldwide. PD-GBA affects 7% to 10% of the total Parkinson's disease population worldwide and an estimated 90,000 individuals in the United States alone. GBA1 encodes the lysosomal enzyme, beta-glucocerebrosidase, or GCase. Mutations in the GBA1 gene lead to a deficiency of GCase, resulting in lysosomal dysfunction in CNS cells, which we believe leads to the inflammation and neurodegeneration present in PD-GBA. GBA1 mutations impact the risk of developing Parkinson's disease as well as many other aspects of the disease course, including the severity, age of onset and rate of progression of disease and the likelihood of dementia. There are no treatments available that modify the progressive course or the underlying disease process of Parkinson's disease.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases, the continued progression of our PROPEL Phase 1/2 clinical trial, the continued dosing of patients and the expected timing for the delivery of interim data from a subset of patients from our PROPEL Phase 1/2 clinical trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications

    617-413-3543

    Investor Contact:


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  28. NEW YORK, March 02, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's Investigational New Drug (IND) application for its experimental gene therapy program, PR006, for the treatment of frontotemporal dementia patients with GRN mutation (FTD-GRN), and that the Company may proceed with the initiation of its Phase 1/2 clinical trial.

    PR006 is being developed as a potential one-time gene therapy for FTD-GRN, a progressive neurodegenerative disease caused by mutations in the GRN gene that reduce production…

    NEW YORK, March 02, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's Investigational New Drug (IND) application for its experimental gene therapy program, PR006, for the treatment of frontotemporal dementia patients with GRN mutation (FTD-GRN), and that the Company may proceed with the initiation of its Phase 1/2 clinical trial.

    PR006 is being developed as a potential one-time gene therapy for FTD-GRN, a progressive neurodegenerative disease caused by mutations in the GRN gene that reduce production of progranulin, a protein critical for lysosomal function, neuronal survival, and normal microglial activities. The progranulin deficiency leads to lysosomal dysfunction, ineffective protein degradation and recycling, neuroinflammation, and ultimately neurodegeneration and death, typically within three to ten years of diagnosis.

    PR006 is designed to increase progranulin levels in the brains of FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

    "FTD-GRN is a serious and progressive neurodegenerative disease for which there are no approved treatments," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "Now that the PR006 IND is active, we look forward to advancing clinical development of PR006, which has the potential to positively impact the lives of patients by slowing or stopping the progression of this devastating disease."

    The PROCLAIM Phase 1/2 clinical trial will investigate the safety and tolerability of PR006, and will also measure key biomarkers and exploratory efficacy endpoints in patients with FTD-GRN. The Company plans to begin dosing for PROCLAIM this year. We believe this makes PR006 the first gene therapy for FTD-GRN to enter clinical trials.

    The FDA has granted Orphan Drug Designation for PR006 for the treatment of patients with FTD.

    About Frontotemporal Dementia with a GRN Mutation
    Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer's disease. 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union are affected by FTD. Frontotemporal dementia with a GRN mutation (FTD-GRN) represents 5-10% of all patients with FTD. FTD results from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language. In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation, and neurodegeneration. There are no approved treatments for FTD or FTD-GRN.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases, the ability of PR006 to increase progranulin levels in the brains of FTD-GRN patients, and timing of enrollment and dosing of patients in the PROCLAIM trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications

    617-413-3543

    Investor Contact:

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  29. NEW YORK, Feb. 21, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that management will present at two upcoming conferences:

    • SVB Leerink 9th Annual Global Healthcare Conference on Thursday, February 27, 2020, fireside chat at 9:00 a.m. ET in New York, NY
       
    • Cowen and Company 40th Annual Healthcare Conference on Tuesday, March 3, 2020, presentation at 12:00 p.m. ET in Boston, MA

    The live webcast of each event will be available under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com

    NEW YORK, Feb. 21, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that management will present at two upcoming conferences:

    • SVB Leerink 9th Annual Global Healthcare Conference on Thursday, February 27, 2020, fireside chat at 9:00 a.m. ET in New York, NY
       
    • Cowen and Company 40th Annual Healthcare Conference on Tuesday, March 3, 2020, presentation at 12:00 p.m. ET in Boston, MA

    The live webcast of each event will be available under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com. The webcasts will be archived for 90 days following the events.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications
    617-413-3543

    Investor Contact:

     

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  30. Orphan Drug Designation Granted for Treatment of Gaucher Disease 

    Rare Pediatric Disease Designation Granted for Treatment of
    Neuronopathic Gaucher Disease

    NEW YORK, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company's investigational gene therapy, PR001, for the treatment of patients with Gaucher disease. The Company also announced that the FDA has granted Rare Pediatric Disease Designation for PR001 for the treatment of neuronopathic Gaucher…

    Orphan Drug Designation Granted for Treatment of Gaucher Disease 

    Rare Pediatric Disease Designation Granted for Treatment of
    Neuronopathic Gaucher Disease

    NEW YORK, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company's investigational gene therapy, PR001, for the treatment of patients with Gaucher disease. The Company also announced that the FDA has granted Rare Pediatric Disease Designation for PR001 for the treatment of neuronopathic Gaucher disease (nGD), the most severe form of the condition.

    "We are pleased to receive these important designations from the FDA, which underscore the critical nature of our work," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "These designations support our conviction that new gene therapies for Gaucher disease are urgently needed — especially for the severe, neuronopathic form of the disease, for which there are no FDA-approved therapies."

    PR001 is also being developed as a potentially disease-modifying, single-dose gene therapy for Parkinson's disease with GBA1 mutation (PD-GBA).

    Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.

    Rare Pediatric Disease Designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the United States, primarily those 18 years of age and younger. The sponsor of a drug with Rare Pediatric Disease Designation may, upon marketing approval, qualify for receipt of a priority review voucher applicable to a subsequent marketing application, which voucher is fully transferable.

    Prevail announced in December 2019 that its Investigational New Drug (IND) application for PR001 for the treatment of nGD is active. Prevail is proceeding with its Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020 under the same IND.

    About Neuronopathic Gaucher Disease
    Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease. Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as seizures and gaze and motor abnormalities. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases, the timing and progress of clinical trials, and the potential advantages of Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; despite receiving Rare Pediatric Disease designation, the Company may not be successful in obtaining a priority review voucher; gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:
    Mary Carmichael
    Ten Bridge Communications
    617-413-3543

    Investor Contact:

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  31. NEW YORK, Feb. 06, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the upcoming presentation of three abstracts at the 16th Annual WORLDSymposium™, taking place February 10-13, 2020, in Orlando, FL.  The abstracts include preclinical data from Prevail's programs PR001 for neuronopathic Gaucher disease and PR006 for frontotemporal dementia with GRN mutation.  

    Two abstracts will be presented during the poster session on Monday, February 10th from 4:30 p.m. to 6:30 p.m. ET.  Poster information is as follows:

    Abstract Title: Gene therapy PR006 increased…

    NEW YORK, Feb. 06, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the upcoming presentation of three abstracts at the 16th Annual WORLDSymposium™, taking place February 10-13, 2020, in Orlando, FL.  The abstracts include preclinical data from Prevail's programs PR001 for neuronopathic Gaucher disease and PR006 for frontotemporal dementia with GRN mutation.  

    Two abstracts will be presented during the poster session on Monday, February 10th from 4:30 p.m. to 6:30 p.m. ET.  Poster information is as follows:

    Abstract Title: Gene therapy PR006 increased progranulin levels and improved lysosomal related phenotypes in model systems (poster #52)
    Presenter: Laura Heckman, Ph.D.

    Abstract Title: Gene therapy PR001 increased GCase activity and improved neuronopathic Gaucher disease phenotypes in mouse models (poster #159)
    Presenter: Laura Heckman, Ph.D.

    One abstract will be presented during the Contemporary Forum session on Thursday, February 13th at 1:30 p.m. ET. Oral presentation information is as follows:

    Abstract Title: Gene therapy PR006 increased progranulin levels and improved lysosomal related phenotypes in model systems
    Presenter: Asa Abeliovich, M.D., Ph.D.

    In addition to its presentations at WORLDSymposium, on Wednesday, February 12th at 8:30 p.m. ET, management will be hosting a presentation by Professor Ari Zimran, M.D., a key opinion leader in the field of neuronopathic Gaucher disease, on "Unmet Needs and Emerging Therapeutic Options for Neuronopathic Gaucher Disease", with a roundtable discussion to follow.  A recording of this event and accompanying slides will be made available immediately following its conclusion and can be found under "Events and Presentations" in the Investors & Media section of the company's website, www.prevailtherapeutics.com. The webcasts will be archived for 90 days following the presentation.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:
    Katie Engleman
    1AB

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  32. PR001 Administered to International Type 2 Gaucher Disease Patient Via Compassionate Use Pathway

    Company Advancing its Phase 1/2 Clinical Trial for the Treatment of Neuronopathic Gaucher Disease and Expects to Initiate Patient Dosing in First Half of 2020

    NEW YORK, Jan. 09, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today provided an update on its investigational program, PR001, an AAV9-based gene therapy delivering the GBA1 gene, for the neuronopathic Gaucher disease (nGD) indication.

    Prevail has granted a compassionate use request for the administration…

    PR001 Administered to International Type 2 Gaucher Disease Patient Via Compassionate Use Pathway

    Company Advancing its Phase 1/2 Clinical Trial for the Treatment of Neuronopathic Gaucher Disease and Expects to Initiate Patient Dosing in First Half of 2020

    NEW YORK, Jan. 09, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today provided an update on its investigational program, PR001, an AAV9-based gene therapy delivering the GBA1 gene, for the neuronopathic Gaucher disease (nGD) indication.

    Prevail has granted a compassionate use request for the administration of PR001 to a single patient with Type 2 Gaucher disease via a compassionate use pathway, following approval by an international regulatory authority, and the patient was recently dosed. Type 2 Gaucher disease is the more severe form of nGD, which presents in infancy and involves rapidly progressing neurodegeneration leading to death in infancy or early childhood.

    Separately, as announced on December 26, 2019, Prevail's Investigational New Drug (IND) application for PR001 for the treatment of nGD is now active. The Company is proceeding with its Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020 under the same IND. Prevail intends for the Company's clinical studies to serve as the primary option for patients to receive access to any of Prevail's investigational drugs.

    "Our decision to grant a compassionate use request for this patient and our continued progress toward initiating clinical trials underscore our deep dedication to our mission of treating all eligible patients with neuronopathic Gaucher disease, the most severe form of Gaucher disease," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "We continue to believe that PR001 has the potential to be a disease-modifying therapy for the treatment of Parkinson's disease with GBA1 mutation and neuronopathic Gaucher disease, which share the same underlying genetic mechanism."

    Prevail is also developing PR001 for Parkinson's disease patients with a GBA1 mutation (PD-GBA). The Company has an active IND for PR001 for the treatment of PD-GBA and the PROPEL Phase 1/2 clinical trial for PD-GBA patients is now recruiting.

    About Neuronopathic Gaucher Disease
    Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology.  Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease.  Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as gaze and motor abnormalities and seizures.  There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases and the timing and progress of clinical trials. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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    Katie Engleman
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  33. NEW YORK, Dec. 26, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has notified Prevail that the Company's Investigational New Drug (IND) application for PR001 for the treatment of neuronopathic Gaucher disease (nGD) patients is now active and that Prevail may proceed with initiating its proposed clinical trial. As previously reported, Prevail's IND for PR001 for the treatment of nGD had been put on clinical hold by the FDA, and this clinical hold has now been removed.

    The Company's planned Phase…

    NEW YORK, Dec. 26, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or the "Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has notified Prevail that the Company's Investigational New Drug (IND) application for PR001 for the treatment of neuronopathic Gaucher disease (nGD) patients is now active and that Prevail may proceed with initiating its proposed clinical trial. As previously reported, Prevail's IND for PR001 for the treatment of nGD had been put on clinical hold by the FDA, and this clinical hold has now been removed.

    The Company's planned Phase 1/2 clinical trial for nGD patients will commence at a dose higher than originally proposed. Prevail submitted nonclinical data in which no PR001-related safety events or adverse findings were observed, supporting the initiation of the Phase 1/2 clinical trial at this higher dose.

    Prevail is activating a Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. Type 2 Gaucher disease is the more severe form of nGD, which presents in infancy and involves rapidly progressing neurodegeneration leading to death in infancy or early childhood. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020, under the same nGD IND.  Type 3 Gaucher disease is a form of nGD that typically presents in childhood and involves multiple neurological manifestations. 

    "We are pleased to now have an active IND for PR001 for the nGD indication and look forward to initiating a Phase 1/2 clinical trial in the first half of 2020," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "Patients with nGD have the most severe form of Gaucher disease and a significant unmet need for therapies to treat their neurological manifestations. We believe PR001 has tremendous potential to help patients suffering from this devastating disease."

    Prevail is also developing PR001 for Parkinson's disease patients with a GBA1 mutation (PD-GBA). The Company has an active IND for PR001 for the treatment of PD-GBA and the PROPEL Phase 1/2 clinical trial for PD-GBA patients is now recruiting.

    About Neuronopathic Gaucher Disease
    Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology.  Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease.  Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as gaze and motor abnormalities and seizures.  There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning timing of initiation of clinical trials for the treatment of nGD, the potential for PR001 to slow or stop disease progression in these patients and Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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    Katie Engleman
    1AB
     

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  34. NEW YORK, Dec. 16, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) for the company's gene therapy candidate, PR006, for the treatment of patients with frontotemporal dementia (FTD) with a GRN mutation (FTD-GRN). PR006 is designed to increase progranulin levels in FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

    Orphan Drug designation is granted by the U.S. Food and Drug Administration to drugs or biologics intended to treat a rare disease or condition, which is…

    NEW YORK, Dec. 16, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) for the company's gene therapy candidate, PR006, for the treatment of patients with frontotemporal dementia (FTD) with a GRN mutation (FTD-GRN). PR006 is designed to increase progranulin levels in FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

    Orphan Drug designation is granted by the U.S. Food and Drug Administration to drugs or biologics intended to treat a rare disease or condition, which is a disease or condition that affects fewer than 200,000 individuals in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.

    "FTD with a GRN mutation is characterized by progressive difficulties in decision-making, behavior and language," said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Prevail. "With no approved treatments for FTD, there is an urgent unmet need for therapies that slow or stop this disease. Orphan drug designation is an important milestone as we prepare to bring PR006 into the clinic in the first half of 2020." 

    About Frontotemporal Dementia with a GRN Mutation
    Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer's disease. 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union are affected by FTD. Frontotemporal dementia with a GRN mutation (FTD-GRN) represents 5-10% of all patients with FTD. FTD results from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language. In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation, and neurodegeneration. There are no approved treatments for FTD or FTD-GRN.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson's disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

    Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases and the ability to derive benefits from Orphan Drug designation. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:
    Katie Engleman
    1AB

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  35. Former CEO of NPS Pharmaceuticals brings broad expertise to support Moderna's progress towards late-stage development and commercialization

    Moderna, Inc., (NASDAQ:MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that François Nader, M.D., M.B.A. has joined its Board of Directors. Dr. Nader is an experienced biopharmaceutical executive and director with broad expertise across development, regulatory affairs and commercial at NPS Pharmaceuticals (acquired by Shire Plc, now part of Takeda (NYSE: TAK)), as well as Aventis-Pharma and the Pasteur Vaccines division of Rhone-Poulenc, now both part of Sanofi (NASDAQ…

    Former CEO of NPS Pharmaceuticals brings broad expertise to support Moderna's progress towards late-stage development and commercialization

    Moderna, Inc., (NASDAQ:MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that François Nader, M.D., M.B.A. has joined its Board of Directors. Dr. Nader is an experienced biopharmaceutical executive and director with broad expertise across development, regulatory affairs and commercial at NPS Pharmaceuticals (acquired by Shire Plc, now part of Takeda (NYSE: TAK)), as well as Aventis-Pharma and the Pasteur Vaccines division of Rhone-Poulenc, now both part of Sanofi (NASDAQ:SNY).

    Concurrently with Dr. Nader's appointment, Peter Barton Hutt, LL.M. has elected to step down after seven years of service to Moderna. Mr. Hutt will remain as a consultant and advisor to the Company.

    "We are focused on continuing to add leaders to our board who understand the complexity of pioneering breakthrough innovations to support the next phase of Moderna's growth," said Noubar Afeyan, Ph.D., Co-Founder and Chairman of Moderna, and CEO of Flagship Pioneering. "François is a value builder with deep experience in vaccine and rare disease therapeutic development and expertise in leading companies from early to late-stage development. As we welcome François, I would like to thank Peter Hutt for the invaluable strategic and regulatory guidance he provided while serving on the board over the last seven years."

    "I am energized by Moderna's culture, science and the impact I believe mRNA medicines will make for patients across a range of therapeutic areas," said Dr. Nader. "I look forward to working with my fellow directors and applying learnings to support Moderna as the team continues generating new clinical data, prepares for pivotal trials and scales to ensure it is ready for commercialization."

    "As Moderna prepares for late-stage development and commercialization of our CMV vaccine, we sought a director with diverse experience across clinical development, regulatory affairs and commercial. François' success in leading late-stage companies as well as his deep experience in infectious and rare diseases make him an important addition to the board as we continue to advance our pipeline," said Stéphane Bancel, Chief Executive Officer of Moderna. "François has an impressive track record of leadership and value creation in the biopharmaceutical sector and I know we will benefit from his perspective. It has been a pleasure to work closely with Peter Hutt during his seven years on the board and I would like to thank him for his contributions to the Company's success."

    Dr. Nader served as President, Chief Executive Officer and Executive Director of NPS Pharmaceuticals from 2008 until 2015, when the company was acquired by Shire. During his tenure as CEO, Dr. Nader transformed NPS Pharma into a leading global biotechnology company focused on delivering innovative therapies to patients with rare diseases. Prior to NPS, Dr. Nader was a venture partner at Care Capital, a venture capital firm. He previously served on Aventis Pharma's North America Leadership Team, holding a number of executive positions in integrated healthcare markets and medical and regulatory affairs. Dr. Nader previously led global commercial operations at the Pasteur Vaccines division of Rhone-Poulenc.

    In addition to serving on the Moderna board of directors, Dr. Nader currently serves as Chairman of the board of directors of Acceleron Pharma (NASDAQ:XLRN), Prevail Therapeutics (NASDAQ:PRVL) and Talaris Therapeutics. He also serves on the Board of Directors of Alexion Pharmaceuticals (NASDAQ:ALXN) and advisor for SVB-Leerink. Dr. Nader is the past Chairman of BioNJ, New Jersey's biotechnology trade organization, and previously served on the board of the Biotechnology Industry Organization (BIO), NPS Pharma (NASDAQ:NPSP), Advanced Accelerator Applications (NASDAQ:AAAP), Baxalta (NYSE:BXLT), Clementia Pharmaceuticals (NASDAQ:CMTA), Trevena (NASDAQ:TRVN) and Noven (NASDAQ:NOVN). In 2013, he was recognized as the Ernst and Young National Life Science Entrepreneur of the Year.

    Dr. Nader earned his French doctorate in medicine from St. Joseph University in Lebanon and a physician executive MBA from the University of Tennessee.

    About Moderna

    Moderna is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. mRNA medicines are designed to direct the body's cells to produce intracellular, membrane or secreted proteins that have a therapeutic or preventive benefit with the potential to address a broad spectrum of diseases. Moderna's platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, providing the Company the capability to pursue in parallel a robust pipeline of new development candidates. Moderna is developing therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases, independently and with strategic collaborators.

    Headquartered in Cambridge, Mass., Moderna currently has strategic alliances for development programs with AstraZeneca, Plc. (NASDAQ:AZN) and Merck, Inc. (NASDAQ:MRK), as well as the Defense Advanced Research Projects Agency (NASDAQ:DARPA), an agency of the U.S. Department of Defense and the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS). Moderna has been named a top biopharmaceutical employer by Science for the past five years. To learn more, visit www.modernatx.com.

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  36. NEW YORK, Nov. 18, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment of Morgan Sheng, Ph.D., to its Board of Directors.

    "Morgan is an esteemed leader who brings a wealth of expertise in neuroscience and specifically drug development in neurodegenerative diseases," said Francois Nader, M.D., Non-Executive Chairman of Prevail's Board of Directors. "He will be a great addition to Prevail's Board as we continue to develop meaningful therapeutic advances for patients suffering from devastating neurodegenerative diseases like Parkinson's disease, frontotemporal dementia…

    NEW YORK, Nov. 18, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment of Morgan Sheng, Ph.D., to its Board of Directors.

    "Morgan is an esteemed leader who brings a wealth of expertise in neuroscience and specifically drug development in neurodegenerative diseases," said Francois Nader, M.D., Non-Executive Chairman of Prevail's Board of Directors. "He will be a great addition to Prevail's Board as we continue to develop meaningful therapeutic advances for patients suffering from devastating neurodegenerative diseases like Parkinson's disease, frontotemporal dementia and Gaucher disease, among others."

    Dr. Sheng is a Core Institute Member and Co-Director of the Stanley Center for Psychiatric Research at the Broad Institute of MIT and Harvard. He is also a Professor in the Department of Brain and Cognitive Science at MIT. Previously, he was Vice President of Neuroscience at Genentech, where he was head of neuroscience research and drug discovery. Under his leadership at Genentech, multiple innovative programs for treatment of serious diseases of the nervous system advanced into clinical trials. Prior to joining Genentech, Dr. Sheng was the Menicon Professor of Neuroscience at MIT, as well as an Investigator of the Howard Hughes Medical Institute. He is a Fellow of the Royal Society in the U.K., the Academy of Medical Sciences in the U.K. and the American Association for the Advancement of Science and is the author of more than 200 peer-reviewed publications focused on the molecular cellular biology of brain synapses and the mechanisms of neurodegenerative diseases. Dr. Sheng holds a Ph.D. in molecular genetics from Harvard University, a B.A. from Oxford University, and obtained his medical degree and training in internal medicine at London University. Following his postdoctoral work in neuroscience at the University of California, San Francisco, he served on the faculty at Massachusetts General Hospital and Harvard Medical School before joining MIT.

    "As we transition to a clinical-stage company, we are thrilled to welcome Morgan to our Board of Directors," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "He will be instrumental to Prevail as we advance our pipeline of novel, urgently-needed gene therapies that target the major neurodegenerative disorders."

    "I am honored to join the Board at Prevail," said Dr. Sheng. "The Prevail team has made significant progress in developing potential treatments for patients with neurodegenerative disorders, with its first program entering the clinic soon, and I am looking forward to working with the team help push these innovative gene therapies forward."

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail's ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases and the anticipated initiation of Prevail's first clinical trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Media Contact:
    Katie Engleman
    1AB

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  37. Opened Enrollment of PROPEL Phase 1/2 Trial of PR001 to Treat Parkinson's Disease Patients with GBA1 Mutations

    PR001 for Neuronopathic Gaucher Disease and PR006 for Frontotemporal Dementia with GRN Mutations Progressing Towards Clinic

    Ongoing Collaboration with Lonza to Support the Manufacturing of our Pipeline of Novel AAV9-Based Gene Therapy Programs

    NEW YORK, Nov. 12, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reported recent business highlights and third quarter 2019 financial results.

    "Prevail made important progress throughout the third quarter in the advancement of our gene…

    Opened Enrollment of PROPEL Phase 1/2 Trial of PR001 to Treat Parkinson's Disease Patients with GBA1 Mutations

    PR001 for Neuronopathic Gaucher Disease and PR006 for Frontotemporal Dementia with GRN Mutations Progressing Towards Clinic

    Ongoing Collaboration with Lonza to Support the Manufacturing of our Pipeline of Novel AAV9-Based Gene Therapy Programs

    NEW YORK, Nov. 12, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reported recent business highlights and third quarter 2019 financial results.

    "Prevail made important progress throughout the third quarter in the advancement of our gene therapy programs for the treatment of neurodegenerative diseases for patients with urgent unmet medical needs. We activated the first sites for our PROPEL Phase 1/2 clinical trial of PR001 for patients with Parkinson's disease with GBA1 mutations, and we expect to begin dosing patients in the fourth quarter. This is an exciting step forward for patients suffering from this devastating disease," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "In addition, we are working diligently to finalize the IND submissions for PR001 for patients with neuronopathic Gaucher disease and PR006 for frontotemporal dementia patients with GRN mutations. We expect to initiate those Phase 1/2 trials in the first half of next year. Finally, our collaboration with Lonza is advancing our process development and manufacturing capabilities as we prepare to supply our late-stage clinical trials."

    Recent Business Highlights

    • Actively Recruiting for PROPEL Phase 1/2 Trial of PR001 for PD-GBA: Prevail previously announced that the U.S. Food and Drug Administration (FDA) accepted the company's Investigational New Drug (IND) application for its lead program, PR001 for the treatment of Parkinson's disease patients with GBA1 mutations (PD-GBA). The PROPEL trial is now open for enrollment. The trial will enroll up to 16 patients to investigate the safety and tolerability of PR001 and will also measure key biomarkers and exploratory efficacy endpoints. The company expects to initiate patient dosing this year.
       
    • Planning to Initiate the Phase 1/2 Trial of PR001 for Neuronopathic Gaucher Disease Patients in 1H 2020: Prevail is also developing PR001 for the potential treatment of neuronopathic Gaucher disease (nGD). In September, the company announced that, based on feedback from the FDA and findings from its preclinical studies, it is modifying the design of the Phase 1/2 clinical trial in nGD to commence at a dose higher than originally proposed. Based on this feedback, the IND was placed on clinical hold pending FDA review of an amendment to the nGD IND, which will detail this modification. To support the higher dose, Prevail recently completed a non-human primate (NHP) safety study in which no PR001-related safety events or adverse findings were observed. Prevail expects to initiate a Phase 1/2 clinical trial for patients with nGD during the first half of 2020.
       
    • Phase 1/2 Trial of PR006 for Frontotemporal Dementia Patients with GRN Mutations (FTD-GRN) Expected to Initiate in 1H 2020: Prevail has completed the necessary preclinical efficacy and safety studies and is targeting submission of an IND for PR006 at the end of the year. In a mouse model of FTD-GRN, PR006 increased progranulin expression, reduced markers of neuroinflammation, and reduced measures of lysosomal pathology and no PR006-related safety events or adverse findings were observed. In a GLP NHP safety study, PR006 treatment increased progranulin levels in the brain in a dose-dependent manner. An extremely minor degree of nerve fiber degeneration in spinal cord and glial cellularity in dorsal root sensory ganglia was observed and was not considered adverse. The company expects to initiate its Phase 1/2 clinical trial for FTD-GRN during the first half of 2020. Prevail believes PR006 has the potential to be a first-in-class, disease-modifying treatment for patients with FTD-GRN.
       
    • Established Collaboration with Lonza to Enable Large Scale Gene Therapy Production: In October 2019, Prevail and Lonza announced the establishment of a collaboration focused on GMP manufacturing utilizing the baculovirus/Sf9 expression system for Prevail's pipeline of novel AAV-based gene therapy programs for patients with neurodegenerative diseases. Under the terms of the agreement, Lonza will manufacture PR001 and PR006 at its gene therapy center of excellence in Houston, Texas. The collaboration also has the potential to extend to Prevail's future pipeline of AAV-based gene therapy programs.
       
    • Continued Expansion of Internal Expertise and Capabilities: Prevail continued to build out its team of over 50 employees and moved into a new office space in the Alexandria Center for Life Science in New York City. The new location includes approximately 12,000 square feet of lab space to support early-stage research and in-house process and analytical development utilizing both HEK293 and baculovirus/Sf9 AAV expression systems.

    Third Quarter 2019 Financial Results

    • Cash Position: Cash and cash equivalents were $183.1 million as of September 30, 2019, as compared to $202.1 million as of June 30, 2019.
       
    • R&D Expenses: R&D expenses were $16.8 million for the third quarter of 2019, compared to $4.6 million for the third quarter of 2018. The increase was primarily related to our development programs, as a result of increased manufacturing-related spend, clinical and preclinical activities, and headcount.
       
    • G&A Expenses: G&A expenses were $4.4 million for the third quarter of 2019, compared to $0.9 million for the third quarter of 2018. The increase was primarily due to an increase in personnel costs resulting from increased headcount, professional services fees, and other corporate-related expenses.
       
    • Net Loss: Net loss was $20.3 million, or $0.62 loss per share, for the third quarter of 2019, compared to $5.2 million, or $0.99 loss per share, for the third quarter of 2018.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for genetically defined sub-populations of patients with neurodegenerative diseases. Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential of Prevail's gene therapies to modify course of neurodegenerative diseases; the anticipated timing of Prevail's Phase 1/2 clinical trials of PR001 in PD-GBA and in nGD and Prevail's clinical trial of PR006; Prevail's ability to work with Lonza to supply Prevail's late stage trials and commercial production; and the applicability of the collaboration to Prevail's future pipeline of AAV-based gene therapy programs. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2019, filed with the SEC on August 14, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.


    Prevail Therapeutics Inc.
    Statements of Operations
    (Unaudited)
    (in thousands, except share and per share data)

        Three Months Ended September 30,     Nine Months Ended September 30,  
        2019     2018     2019     2018  
    Operating Expenses:                                
    Research and development   $ 16,836     $ 4,599     $ 37,202     $ 9,110  
    General and administrative     4,452       920       10,050       2,520  
    Total operating loss     (21,288 )     (5,519 )     (47,252 )     (11,630 )
    Change in fair value of derivative liabilities                       (781 )
    Interest income     989       320       1,905       543  
    Interest expense                       (471 )
    Total other income (expense), net     989       320       1,905       (709 )
    Net loss   $ (20,299 )   $ (5,199 )   $ (45,347 )   $ (12,339 )
    Net loss per share, basic and diluted   $ (0.62 )   $ (0.99 )   $ (1.68 )   $ (2.47 )
    Weighted average shares outstanding, basic and diluted     32,864,156       5,244,585       26,950,854       4,989,604  


    Prevail Therapeutics Inc.
    Balance Sheets
    (Unaudited)
    (in thousands, except share and per share data)

        September 30,
    2019
        December 31,
    2018
     
    ASSETS                
    CURRENT ASSETS:                
    Cash and cash equivalents   $ 183,074     $ 63,014  
    Prepaid expenses and other current assets     7,425       563  
    Total current assets     190,499       63,577  
    Property and equipment, net     2,527       678  
    Operating lease right-of-use assets     10,312       8,534  
    Restricted cash     91       91  
    TOTAL ASSETS   $ 203,429     $ 72,880  
    LIABILITIES, REDEEMABLE CONVERTIBLE PREFERRED
      STOCK, AND STOCKHOLDERS' EQUITY (DEFICIT)
                   
    CURRENT LIABILITIES:                
    Accounts payable   $ 4,952     $ 1,241  
    Accrued expenses and other current liabilities     5,089       1,477  
    Operating lease liabilities     1,134       917  
    Total current liabilities     11,175       3,635  
    Long-term operating lease liabilities     10,226       7,952  
    TOTAL LIABILITIES     21,401       11,587  
    COMMITMENTS AND CONTINGENCIES (Note 13) REDEEMABLE
      CONVERTIBLE PREFERRED STOCK
                   
    Series Seed preferred stock - $0.0001 par value, 0 and 6,480,000 shares
    authorized, issued and outstanding as of September 30, 2019 and
    December 31, 2018, respectively
              3,524  
    Series A preferred stock - $0.0001 par value, 0 and 9,072,000 shares
    authorized, 0 and 8,997,085 shares issued and outstanding as of
    September 30, 2019 and December 31, 2018, respectively
              76,186  
    STOCKHOLDERS' EQUITY (DEFICIT)                
    Common stock - $0.0001 par value, 200,000,000 and 28,398,600
    shares authorized as of September 30, 2019 and December 31, 2018,
    respectively, 34,098,819 and 7,209,000 shares issued and outstanding
    as of September 30, 2019 and December 31, 2018, respectively
        3       1  
    Additional paid-in capital     248,286       2,496  
    Accumulated deficit     (66,261 )     (20,914 )
    Total stockholders' equity (deficit)     182,028       (18,417 )
    TOTAL LIABILITIES, REDEEMABLE CONVERTIBLE
    PREFERRED STOCK, AND STOCKHOLDERS' EQUITY
    (DEFICIT)
      $ 203,429     $ 72,880  
                     

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  38. Strategic collaboration focuses on baculovirus/Sf9 expression system for Prevail's pipeline of novel AAV9-based gene therapy programs for patients with neurodegenerative diseases

    Companies to partner on process development, analytical development, and large-scale manufacturing for late-stage clinical and commercial supply

    Process development and manufacturing to be located in Lonza's cGMP facility in Houston, Texas

    BASEL, Switzerland and NEW YORK, Oct. 08, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, and Lonza (SWX: LONN) today announced that the companies have entered into a strategic…

    Strategic collaboration focuses on baculovirus/Sf9 expression system for Prevail's pipeline of novel AAV9-based gene therapy programs for patients with neurodegenerative diseases

    Companies to partner on process development, analytical development, and large-scale manufacturing for late-stage clinical and commercial supply

    Process development and manufacturing to be located in Lonza's cGMP facility in Houston, Texas

    BASEL, Switzerland and NEW YORK, Oct. 08, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, and Lonza (SWX: LONN) today announced that the companies have entered into a strategic collaboration.

    Lonza and Prevail have been working together since 2018, with an initial focus on process development, towards the GMP manufacturing of Prevail's two lead programs, PR001 and PR006. Under this collaboration, focused on the baculovirus/Sf9 production system for gene therapies, Lonza will manufacture Prevail's pipeline of novel AAV-based gene therapy programs for patients with neurodegenerative diseases at its gene therapy center of excellence in Houston, TX.

    "Prevail is a brilliant trailblazer and it is a privilege to partner with them," said Alberto Santagostino, SVP Head of Cell & Gene Technologies, Lonza Pharma & Biotech. "This strategic collaboration reflects both companies' commitment to fueling the innovation of novel medicines. The partnership combines Prevail's frontier science with Lonza's operational expertise to support the development and commercialization of potentially life-changing treatments. Our cell and gene therapy center of excellence in Houston, TX, will support Prevail in their journey as they develop and potentially bring to market AAV-based gene therapies for patients suffering from debilitating neurodegenerative diseases."

    Prevail's pipeline includes PR001, an AAV9-based gene therapy delivering GBA1, in development for Parkinson's disease patients with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease patients (nGD). Prevail plans to initiate a Phase 1/2 clinical trial of PR001 in PD-GBA in 2019. The company is also developing PR006, an AAV9-based gene therapy delivering GRN, for frontotemporal dementia patients with a GRN mutation (FTD-GRN). Prevail anticipates PR006 will enter the clinic in 2020. The collaboration also has the potential to extend to Prevail's future pipeline of AAV-based gene therapy programs.

    "We are pleased to partner with Lonza, a leader in the manufacturing of AAV gene therapy vectors, to enable large-scale production of our novel gene therapies that we believe hold potential to halt the course of neurodegenerative diseases for patient populations with urgent unmet needs," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer, Prevail Therapeutics. "Lonza and Prevail will work together closely on process development and scaling up production of PR001, our gene therapy for Parkinson's disease with GBA1 mutations and neuronopathic Gaucher disease, to supply late-stage clinical trials and for commercial production. In addition, we look forward to a collaborative relationship to support our PR006 gene therapy program for patients with frontotemporal dementia with GRN mutations, and for future gene therapy programs in our pipeline."

    Prevail has built in-house process and analytical development capabilities utilizing both HEK293 and baculovirus/Sf9 AAV expression systems. Under this collaboration, Prevail and Lonza will work closely together on process development, analytical development, and large-scale production using the baculovirus/Sf9 process for late-stage clinical and commercial supply at Lonza's GMP facility in Houston.

    About Lonza
    Lonza is an integrated solutions provider that creates value along the Healthcare Continuum®. Through our Pharma Biotech & Nutrition segment and our Specialty Ingredients segment businesses, we harness science and technology to serve markets along this continuum. We focus on creating a healthy environment, promoting a healthier lifestyle and preventing illness through consumers' preventive healthcare, as well as improving patient healthcare by supporting our customers to deliver innovative medicines that help treat or even cure severe diseases.

    Patients and consumers benefit from our ability to transfer our pharma know-how to the healthcare, hygiene and fast-moving consumer goods environment and to the preservation and protection of the world where we live.

    Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide at the end of 2018. The company generated sales of CHF 5.5 billion in 2018 with a CORE EBITDA of CHF 1.5 billion. Further information can be found at www.lonza.com.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements Related to Prevail
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential of Prevail's gene therapies to halt the course of neurodegenerative diseases; the anticipated timing of Prevail's Phase 1/2 clinical trial of PR001 in PD-GBA and Prevail's clinical trial of PR006; Prevail's ability to work with Lonza to supply Prevail's late stage trials and commercial production; and the applicability of the collaboration to Prevail's future pipeline of AAV-based gene therapy programs. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail's gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2019, filed with the SEC on August 14, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Additional Information and Disclaimer
    Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited ("SGX-ST"). Lonza Group Ltd is not subject to the SGX-ST's continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

    Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

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  39. NEW YORK, Oct. 01, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, today announced that management will present at three upcoming conferences in October:

    • 2019 Cell & Gene Meeting on the Mesa on Thursday, October 3, 2019 at 12:00 p.m. PT in Carlsbad, CA.
       
    • Chardan's Annual Genetic Medicines Conference on Tuesday, October 8, 2019 at 9:00 a.m. ET in New York, NY.
       
    • 2019 Jefferies Gene Editing Summit on Tuesday, October 8, 2019 at 1:10 p.m. ET in New York, NY.         

    The live webcast of all conferences will be available in the investor section of the company's…

    NEW YORK, Oct. 01, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, today announced that management will present at three upcoming conferences in October:

    • 2019 Cell & Gene Meeting on the Mesa on Thursday, October 3, 2019 at 12:00 p.m. PT in Carlsbad, CA.
       
    • Chardan's Annual Genetic Medicines Conference on Tuesday, October 8, 2019 at 9:00 a.m. ET in New York, NY.
       
    • 2019 Jefferies Gene Editing Summit on Tuesday, October 8, 2019 at 1:10 p.m. ET in New York, NY.         

    The live webcast of all conferences will be available in the investor section of the company's website at www.prevailtherapeutics.com. The webcast will be archived for 90 days following the presentation.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

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  40. NEW YORK, Sept. 19, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, today announced that it will be added to the Russell 2000 and Russell 3000 and Microcap Indexes, effective after the close of the U.S. markets on Friday, September 20, 2019.

    The Russell 2000® Index measures the performance of the small-cap segment of the U.S. equity market. The index is a subset of the Russell 3000® Index and represents approximately 10 percent of the total market capitalization of that index. The Russell Microcap Index represents 2,000 small cap and microcap stocks and captures…

    NEW YORK, Sept. 19, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL) ("Prevail" or "the Company"), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, today announced that it will be added to the Russell 2000 and Russell 3000 and Microcap Indexes, effective after the close of the U.S. markets on Friday, September 20, 2019.

    The Russell 2000® Index measures the performance of the small-cap segment of the U.S. equity market. The index is a subset of the Russell 3000® Index and represents approximately 10 percent of the total market capitalization of that index. The Russell Microcap Index represents 2,000 small cap and microcap stocks and captures the smallest 1,000 companies in the Russell 2000 in addition to 1,000 smaller U.S.-based listed stocks.

    Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked against Russell's U.S. indexes. Russell indexes are part of FTSE Russell, a leading global index provider.

    About Prevail Therapeutics
    Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

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    Katie Engleman
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  41. NEW YORK, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing AAV-based gene therapies for patients with neurodegenerative disorders, today provided an update on the Company's clinical development of PR001, a potentially disease-modifying, single-dose gene therapy in development for Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD).

    PR001 for Treatment of PD-GBA

    Prevail has an open Investigational New Drug Application (IND) for PR001 for the treatment of PD-GBA, the first indication the Company is pursuing. The Company is in the process of activating clinical sites for its Phase 1/2 clinical trial in PD-GBA and is on track to initiate patient…

    NEW YORK, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ:PRVL), a biotechnology company developing AAV-based gene therapies for patients with neurodegenerative disorders, today provided an update on the Company's clinical development of PR001, a potentially disease-modifying, single-dose gene therapy in development for Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD).

    PR001 for Treatment of PD-GBA

    Prevail has an open Investigational New Drug Application (IND) for PR001 for the treatment of PD-GBA, the first indication the Company is pursuing. The Company is in the process of activating clinical sites for its Phase 1/2 clinical trial in PD-GBA and is on track to initiate patient dosing this year. Approximately 7%-10% of Parkinson's disease patients harbor an underlying mutation in the GBA1 gene, and it is estimated that there are 90,000 PD-GBA patients in the U.S. alone. No therapies currently available have shown the ability to slow or stop the disease progression of PD-GBA. This randomized, double-blind, sham procedure-controlled, ascending dose Phase 1/2 clinical trial will enroll up to sixteen PD-GBA patients. Two escalating dose cohorts are planned. In each cohort, six patients will receive PR001, administered as a single injection, and two patients will receive a sham procedure as control. The trial will investigate the safety and tolerability of PR001 and will also measure key biomarkers and exploratory efficacy endpoints. 

    PR001 for Treatment of Pediatric nGD

    Prevail is also developing PR001 for pediatric nGD, a second indication with urgent unmet need.  The Company submitted an IND to the U.S. Food and Drug Administration (FDA) for PR001 for the treatment of pediatric patients with nGD. Following discussions with the FDA, and based on preclinical studies that demonstrated increased efficacy at a higher dose, Prevail is modifying the design of the Phase 1/2 clinical trial in nGD to commence at a dose higher than originally proposed. Prevail's IND for PR001 for the treatment of pediatric nGD has been placed on clinical hold pending FDA review of an amendment to the nGD IND, which will detail this modification. No safety or adverse events have been observed in any of Prevail's studies of PR001. The start of the Phase 1/2 trial in nGD is anticipated to be delayed approximately one quarter and to begin enrollment in the first half of 2020. The modification to the nGD Phase 1/2 trial design is not anticipated to delay the overall timeline to trial completion. 

    "Prevail is developing PR001 for two indications, both with urgent unmet needs," said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. "We are excited to begin dosing patients in our Phase 1/2 clinical trial for PD-GBA this year and are dedicated to developing PR001 for pediatric nGD, the most progressive form of Gaucher disease, which involves neurological manifestations that cause severe morbidity and mortality. We believe PR001 has tremendous potential to slow or stop disease progression in patients with PD-GBA or nGD, who currently have no disease-modifying therapeutic options."  

    About Prevail Therapeutics
    Prevail Therapeutics is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. Prevail was founded by Dr. Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.

    Forward-Looking Statements
    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the likelihood of its interactions with the FDA to support Prevail's clinical development plans; the anticipated timing of Prevail's Phase 1/2 clinical trials of PR001 in each of its two indications; and the ability of PR001 to slow or stop disease progression in patients with PD-GBA or neuronopathic Gaucher disease. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail's novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; PR001 or Prevail's other gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; and the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change. These and other risks are described more fully in Prevail's filings with the Securities and Exchange Commission (SEC), including the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2019, filed with the SEC on August 14, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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