Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company
dedicated to breaking through barriers in gene therapy and
neurology, today announced that it has selected a lead development
candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic
lateral sclerosis (ALS) gene therapy program. The Company
anticipates filing an investigational new drug (IND) application
with the Food and Drug Administration for the candidate in
mid-2025.
Voyager’s SOD1-ALS lead candidate combines a highly potent siRNA
construct to decrease the expression of SOD1 with an IV-delivered,
blood-brain barrier-penetrant TRACER™ capsid. In a non-human
primate study, the candidate demonstrated 73% reduction of SOD1 in
cervical spinal cord motor neurons following a single intravenous
dose in cynomolgus macaques. The candidate demonstrated robust
knockdown of SOD1 across all levels of the spinal cord and motor
cortex. Further, the candidate demonstrated an ability to transduce
both neurons and astrocytes, two cell types thought to play an
important role in ALS. Voyager expects to present additional data
on the candidate at an upcoming scientific conference. In data
previously shared at the 2022 American Society of
Gene and Cell Therapy (ASGCT) annual meeting,
another Voyager SOD1-ALS preclinical candidate increased survival
by a median of 152 days in a G93A mouse model, with survival in
some animals exceeding 430 days.
“Our SOD1-ALS program is one of several TRACER™-powered gene
therapy programs we expect to see advance to IND in 2025,” said
Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of
Voyager. “We believe our development candidate could represent a
significant advancement in the treatment of SOD1-mutated ALS by
offering the potential for durable SOD1 knockdown with a single IV
administration. SOD1 is a validated target, and we plan to utilize
established cerebrospinal fluid and plasma biomarkers in early
clinical studies to efficiently achieve potential proof of
concept.”
About Amyotrophic Lateral Sclerosis
(ALS)Amyotrophic Lateral Sclerosis (ALS) is a progressive
neurodegenerative disease in which the motor neurons atrophy and
die, resulting in loss of the ability to speak, move, eat and,
eventually, breathe. ALS is typically fatal within approximately
two to five years of symptom onset1,2. The disease is estimated to
impact approximately 20,000 people in the U.S.2 Multiple genes have
been implicated in ALS; mutations in the superoxide dismutase
1 (SOD1) gene are estimated occur in approximately 2-3% of ALS
cases, or up to 600 people in the U.S.2,3,4 There are multiple
treatments available for ALS and its symptoms, but there is no
cure5.
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 (Nasdaq: VYGR) is a biotechnology company dedicated to
breaking through barriers in gene therapy and neurology. The
potential of both disciplines has been constrained by delivery
challenges; Voyager is leveraging cutting-edge expertise in capsid
discovery and deep neuropharmacology capabilities to address these
constraints. Voyager’s TRACER™ AAV capsid discovery platform has
generated novel capsids with high target delivery and blood-brain
barrier penetration at low doses, potentially addressing the narrow
therapeutic window associated with conventional gene therapy
delivery vectors. This platform is fueling alliances with Alexion,
AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine
Biosciences, Inc. and Sangamo Therapeutics, Inc., as well as
multiple programs in Voyager’s own pipeline. Voyager’s pipeline
includes wholly-owned and collaborative preclinical programs in
Alzheimer’s disease, amyotrophic lateral sclerosis (ALS),
Parkinson’s disease, and other diseases of the central nervous
system, with a focus on validated targets and biomarkers to enable
a path to rapid potential proof-of-biology. For more information,
visit www.voyagertherapeutics.com.
Voyager Therapeutics® is a registered trademark, and TRACER™ is
a trademark, of Voyager Therapeutics, Inc.
Forward-Looking StatementsThis 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 “anticipate,” “expect,” “plan,” “believe,” 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, including
the preclinical development of its potential development candidates
and timing of IND filings; Voyager’s expected presentation of
additional data from its SOD1 ALS gene therapy program at an
upcoming scientific conference; the potential for the development
candidate to achieve durable SOD1 inhibition following IV delivery
or address ALS manifestations beyond the CNS by enabling broad CNS
knockdown of SOD1; and Voyager’s ability to utilize established
cerebrospinal fluid and plasma biomarkers to efficiently achieve
potential proof-of-biology for the development candidate 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; 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 the exercise of
development, commercialization, license and other options under
the Alexion, AstraZeneca Rare Disease and Novartis license
option agreements and Neurocrine collaborations; 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.
References:
1 ALS Association. ALS Symptoms and Diagnosis. Available at:
https://www.als.org/understanding-als/symptoms-diagnosis. Accessed:
December 2023.
2 Mehta P., et al. Prevalence of amyotrophic lateral sclerosis
in the United States, 2018. Amyotrophic Lateral Sclerosis and
Frontotemporal Degeneration. 2023 Aug 21:1-7. doi:
10.1080/21678421.2023.2245858. Epub ahead of print. PMID:
37602649.
3 Brown C., et al. Estimated Prevalence and Incidence of
Amyotrophic Lateral Sclerosis and SOD1 and C9orf72 Genetic
Variants. Neuroepidemiology. 2021;55(5):342-353. doi:
10.1159/000516752. Epub 2021 Jul 9.
4 Ricci C., et al. A Novel Variant in Superoxide Dismutase 1
Gene (p.V119M) in Als Patients with Pure Lower Motor Neuron
Presentation. Genes (Basel). 2021 Sep 29;12(10):1544. doi:
10.3390/genes12101544.
5 National Institute of Neurological Disorders and Stroke.
Amyotrophic Lateral Sclerosis (ALS). Available at:
https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als.
Accessed: December 2023.
Contacts
InvestorsInvestors@vygr.comAndrew
Funderburkafunderburk@kendallir.com
MediaTrista Morrisontmorrison@vygr.comPeg
Rusconiprusconi@vergescientific.com
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