Prevail Therapeutics Announces IND Active for Phase 1/2 Trial of its Gene Therapy PR001 to Treat Parkinson’s Disease Patients with GBA1 Mutations

NEW YORK–(BUSINESS WIRE)–Prevail Therapeutics Inc. (Prevail), a gene therapy company developing
AAV-based gene therapies for patients with neurodegenerative diseases,
today announced the U.S. Food and Drug Administration (FDA) has accepted
the Company’s Investigational New Drug (IND) application for its lead
gene therapy program, PR001. PR001 is a potentially disease-modifying,
single-dose, AAV9-based gene therapy being developed for the treatment
of Parkinson’s disease patients with a GBA1 mutation (PD-GBA).

“We are pleased that the FDA has accepted the IND for our first program,
which we believe has the potential to transform the lives of patients
with Parkinson’s disease with a GBA1 mutation,” said Asa Abeliovich,
M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “At
Prevail, our goal is to halt the progression of serious
neurodegenerative diseases by applying precision medicine to the
development of gene therapies. Our active IND brings us a step closer to
achieving that goal, and we look forward to entering this new phase as a
clinical-stage company.”

The active IND allows Prevail to initiate a Phase 1/2 clinical trial
that will investigate the safety and tolerability of PR001, and will
also measure key biomarkers and exploratory efficacy endpoints, in
patients with PD-GBA. The Company plans to begin dosing patients in the
trial this year.

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 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.

Contacts

Katie Engleman
1AB
[email protected]