Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment
February 26 2024 - 7:00AM
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
Friedreich’s ataxia (FA) program. The candidate combines a frataxin
(FXN) gene replacement payload with an intravenously administered,
blood-brain barrier penetrant, novel capsid derived from Voyager’s
TRACER™ capsid discovery platform. The companies expect the program
to advance into first-in-human clinical trials in 2025.
Selection of the development candidate triggered a
$5 million milestone payment to Voyager, which the Company expects
to receive in the first 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 FA
marks an important step in our strategic collaboration with
Neurocrine, reflecting the power of combining Voyager’s TRACER AAV
capsids and payload design capabilities with Neurocrine’s expertise
in neuroscience and clinical development,” said Alfred W. Sandrock,
Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “While there
has been encouraging recent progress in the treatment of FA, it
remains a very challenging and eventually fatal disease for which
new therapeutic approaches are needed. We believe our strategy to
replace the defective frataxin gene could address the underlying
disease etiology of FA. We look forward to progressing this and our
other gene therapy programs, including our wholly-owned SOD1 ALS
program and our Neurocrine-partnered GBA1 Parkinson’s program,
towards clinical studies.”
The FA program is being developed under the 2019
strategic collaboration agreement between Voyager and Neurocrine
Biosciences for research, development, and commercialization of
certain AAV gene therapy products for programs targeting
Friedreich’s ataxia and two other undisclosed targets. Under the
terms of the 2019 collaboration agreement, Voyager is eligible to
receive up to $1.3 billion in potential development and commercial
milestone payments, tiered royalties on net sales, and program
funding, and Voyager could exercise an option for 60/40 cost- and
profit-sharing (Neurocrine/Voyager) in the U.S. for the FA program
following the determination by the joint steering committee of
proof of mechanism based on established milestones and metrics.
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,” “believe,” “could,” “future,” or
“potential,” and other similar expressions are intended to identify
forward-looking statements.
For example, all statements Voyager makes regarding
Voyager’s ability to advance its AAV-based gene therapy programs
into first-in-human clinical trials, including the FA program in
2025; the potential of the FA program to address the underlying
disease etiology of FA; and Voyager’s eligibility to receive
development and commercial milestone payments, tiered royalties on
net sales, and program funding under the 2019 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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