Prevail Therapeutics Receives U.S. FDA Fast Track Designation for PR006 to Slow the Progression of Frontotemporal Dementia with a GRN Mutation

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.