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

Preparations Underway to Initiate PROVIDE Phase 1/2 Trial of PR001 for Treatment of Type 2 NeuronopathicGaucher 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 TherapeuticsInc. (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 Parkinsons 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:

Full Year 2019 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 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 Parkinsons with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to PrevailStatements 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 Phase 1/2 clinical trials of PR001 in PD-GBA and in nGD and Prevails 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: 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 September 30, 2019, filed with the SEC on November 12, 2019, the Companys 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)

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

Media Contact:Mary CarmichaelTen Bridge Communicationsmary@tenbridgecommunications.com

Investor Contact: investors@prevailtherapeutics.com

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Prevail Therapeutics Reports Full Year 2019 Financial Results and Business Highlights - BioSpace

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