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Prevail Therapeutics Provides Program Update on PR001 in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher DiseaseNEW 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 ased 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 Forward-Looking Statements Media Contact: Investor Contact: |