Prevail Therapeutics Reports First Quarter 2020 Financial Results and Business Highlights – GlobeNewswire

May 14, 2020 07:00 ET | Source: Prevail Therapeutics

Phase 1/2 Trial of PR001 for Parkinsons Disease with GBA1 Mutations Ongoing; Study Startup Activities Progressing for Phase 1/2 Trials of PR001 for Type 2 NeuronopathicGaucher Disease and PR006 for Frontotemporal Dementia with GRN Mutations

Data Presentations Highlight Potential of AAV Gene Therapy Approach toSlow 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 Parkinsons 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:

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

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 Parkinsons 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 Parkinsons 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 Prevails ongoing and planned clinical trials, business and operations; the potential of Prevails gene therapies to modify the course of neurodegenerative diseases; the anticipated timing of Prevails clinical trials of PR001 in PD-GBA and in nGD and Prevails 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 Prevails Phase 1/2 clinical trial of PR001; and expectations regarding Prevails 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: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails 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 Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on or about May 14, 2020, the Companys 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)

Balance Sheets (in thousands, except share and per share data)

Media Contact:Mary Carmichael Ten Bridge Communications mary@tenbridgecommunications.com 617-413-3543

Investor Contact: investors@prevailtherapeutics.com

New York, New York, UNITED STATES

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Prevail Therapeutics Reports First Quarter 2020 Financial Results and Business Highlights - GlobeNewswire