Voyager Therapeutics Announces Selection of Development Candidate for GBA1 Program in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment
16 Abril 2024 - 6:00AM
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company
dedicated to advancing neurogenetic medicines, today announced that
the joint steering committee with its collaborator Neurocrine
Biosciences has selected a lead development candidate in the GBA1
gene therapy program for the potential treatment of Parkinson’s
disease and other GBA1-mediated diseases. The candidate combines a
GBA1 gene replacement payload with an intravenously administered,
blood-brain barrier penetrant, novel capsid derived from Voyager’s
TRACER™ capsid discovery platform. The companies expect to file an
Investigational New Drug (IND) application with the FDA for the
program in 2025.
Selection of the development candidate triggered a $3 million
milestone payment to Voyager, which the Company expects to receive
in the second quarter of 2024. Voyager is eligible to receive
additional future development and commercialization milestone
payments based on the further advancement of this program.
“The nomination of this development candidate in the GBA1
program, following the recent nomination of a development candidate
in the Friedreich’s ataxia program, demonstrates the productivity
of the collaboration between Voyager and Neurocrine to advance gene
therapies for neurological diseases,” said Alfred W. Sandrock, Jr.,
M.D., Ph.D., Chief Executive Officer of Voyager. “We now see the
potential for three gene therapies leveraging our novel TRACER
capsids to enter the clinic in 2025: the Neurocrine-partnered GBA1
and FA programs, and our wholly-owned SOD1 ALS program.”
The GBA1 program is being developed under the 2023 strategic
collaboration agreement between Voyager and Neurocrine Biosciences
for research, development, manufacture, and commercialization of
certain AAV gene therapy products for programs targeting
Parkinson’s disease and other GBA1-mediated diseases and three
other undisclosed programs to address central nervous system
diseases or conditions associated with rare genetic targets. Under
the terms of the 2023 collaboration agreement, Voyager is eligible
to receive up to $1.5 billion in potential development milestone
payments, as well as additional commercial milestone payments,
tiered royalties on net sales, and program funding. Voyager
maintains an option to elect 50/50 cost- and profit-sharing in
the U.S. for the GBA1 program following topline data from
the first Parkinson’s disease clinical trial.
About the TRACER™ Capsid Discovery
PlatformVoyager’s TRACER™ (Tropism Redirection of AAV by
Cell-type-specific Expression of RNA) capsid discovery platform is
a broadly applicable, RNA-based screening platform that enables
rapid discovery of AAV capsids with robust penetration of the
blood-brain barrier and enhanced central nervous system (CNS)
tropism in multiple species, including non-human primates (NHPs).
TRACER™ generated capsids have demonstrated superior and widespread
gene expression in the CNS compared to conventional AAV capsids as
well as cell- and tissue-specific transduction, including to areas
of the brain that have been traditionally difficult to reach, while
de-targeting the liver and dorsal root ganglia. As part of its
external partnership strategy, Voyager has established multiple
collaboration agreements providing access to its next-generation
TRACER™ capsids to potentially enable its partners’ gene therapy
programs to treat a variety of diseases.
About Voyager TherapeuticsVoyager
Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company
dedicated to leveraging the power of human genetics to modify the
course of – and ultimately cure – neurological diseases. Our
pipeline includes programs for Alzheimer’s disease, amyotrophic
lateral sclerosis (ALS), Parkinson’s disease, and multiple other
diseases of the central nervous system. Many of our programs are
derived from our TRACER™ AAV capsid discovery platform, which we
have used to generate novel capsids and identify associated
receptors to potentially enable high brain penetration with genetic
medicines following intravenous dosing. Some of our programs are
wholly owned, and some are advancing with partners including
Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine
Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more
information, visit www.voyagertherapeutics.com.
Voyager Therapeutics® is a registered trademark, and TRACER™ is
a trademark, of Voyager Therapeutics, Inc.
Forward-Looking Statements
This press release contains forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
The use of words such as “expect,” “future,” or “potential,” and
other similar expressions are intended to identify forward-looking
statements.
For example, all statements Voyager makes regarding filing an
IND on the GBA1 program and the potential to advance Voyager’s
AAV-based gene therapy programs into the clinic in 2025, and
Voyager’s eligibility to receive development and commercial
milestone payments, tiered royalties on net sales, and program
funding under the 2023 Neurocrine collaboration agreement are
forward-looking.
All forward-looking statements are based on estimates and
assumptions by Voyager’s management that, although Voyager believes
such forward-looking statements to be reasonable, are inherently
uncertain. All forward-looking statements are subject to risks and
uncertainties that may cause actual results to differ materially
from those that Voyager expected. Such risks and uncertainties
include, among others, the continued development of Voyager’s
technology platforms, including Voyager’s TRACER™ platform and its
antibody screening technology; the ability to initiate and conduct
preclinical studies in animal models; the development by third
parties of capsid identification platforms that may be competitive
to Voyager’s TRACER™ capsid discovery platform; Voyager’s ability
to create and protect intellectual property rights associated with
the TRACER™ capsid discovery platform, the capsids identified by
the platform, and development candidates for Voyager’s pipeline
programs; the initiation, timing, conduct and outcomes of Voyager’s
preclinical studies; the possibility or the timing of Voyager’s
receipt of program reimbursement, development or commercialization
milestones, option exercise, and other payments under Voyager’s
current licensing or collaboration agreements; the ability of
Voyager to negotiate and complete licensing or collaboration
agreements with other parties on terms acceptable to Voyager and
the third parties; the ability to attract and retain talented
directors, employees, and contractors; and the sufficiency of cash
resources to fund its operations and pursue its corporate
objectives.
These statements are also subject to a number of material risks
and uncertainties that are described in Voyager’s most recent
Annual Report on Form 10-K filed with the Securities and
Exchange Commission. All information in the press release is as of
the date of this press release, and any forward-looking statement
speaks only as of the date on which it was made. Voyager undertakes
no obligation to publicly update or revise this information or any
forward-looking statement, whether as a result of new information,
future events or otherwise, except as required by law.
ContactsTrista Morrison, NACD.DC,
tmorrison@vygr.com Investors: Adam Bero, Ph.D., abero@kendallir.com
Media: Brooke Shenkin, brooke@scientpr.com
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