Prevail Therapeutics Provides Program Update on PR001 in Parkinson’s Disease with GBA1 Mutations and Neuronopathic Gaucher ...
September 10 2019 - 4:05PM
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 based 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 TherapeuticsPrevail 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.
Forward-Looking StatementsStatements 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 likelihood of its interactions with the FDA to
support Prevail’s clinical development plans; the anticipated
timing of Prevail’s Phase 1/2 clinical trials of PR001 in each of
its two indications; and the ability of PR001 to slow or stop
disease progression in patients with PD-GBA or neuronopathic
Gaucher disease. 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: Prevail’s novel
approach to gene therapy makes it difficult to predict the time,
cost and potential success of product candidate development or
regulatory approval; PR001 or Prevail’s other gene therapy programs
may not meet safety and efficacy levels needed to support ongoing
clinical development or regulatory approval; and the regulatory
landscape for gene therapy is rigorous, complex, uncertain and
subject to change. These and other risks are described more fully
in Prevail’s filings with the Securities and Exchange Commission
(SEC), including the “Risk Factors” section of the Company’s
Quarterly Report on Form 10-Q for the period ended June 30, 2019,
filed with the SEC on August 14, 2019, 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.
Media Contact:Katie Engleman1ABkatie@1abmedia.com
Investor Contact:investors@prevailtherapeutics.com
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