Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company
dedicated to advancing neurogenetic medicines, today announced new
data from its two preclinical programs targeting pathological tau
for the treatment of Alzheimer’s disease. Data on VY-TAU01,
Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau
silencing gene therapy program will be presented at the upcoming
2024 International Conference on Alzheimer’s and Parkinson’s
Diseases and Related Neurological Disorders (AD/PD™ 2024), taking
place March 5-9, 2024, in Lisbon, Portugal.
Tau Silencing Gene Therapy Program:
- Intravenous administration of BBB-penetrant AAV containing
primary artificial microRNA targeting tau reduces tau broadly and
robustly in hTau mouse brain. Wencheng Liu, Ph.D., Poster
P0783/#2852.
The data demonstrate that a single intravenous (IV)
administration of one of Voyager’s tau silencing gene therapy
candidates in mice expressing human tau resulted in broad AAV
distribution across multiple brain regions and dose-dependent
reductions in tau messenger RNA (mRNA) levels of up to 90%, which
were associated with robust reductions in human tau protein levels
across the brain. Voyager’s tau silencing gene therapy program
combines vectorized tau-targeted siRNA with a proprietary,
blood-brain barrier (BBB)-penetrant capsid derived from the
Company’s TRACER™ discovery platform. Based on these data, Voyager
has advanced this program into late research and expects to file an
investigational new drug (IND) application in 2026.
VY-TAU01 Anti-Tau Antibody:
- Pharmacokinetics and tolerability of VY-TAU01, an anti-tau
antibody for the treatment of Alzheimer’s disease, in P301S mouse
and nonhuman primate. Wencheng Liu, Ph.D., Poster P0795/#1303.
Voyager also will report new preclinical data demonstrating that
VY-TAU01, the Company’s lead antibody targeting pathological tau,
and Ab-01, its murine surrogate, were well-tolerated and showed
favorable pharmacokinetic profiles following IV administration in
NHPs and P301S transgenic mice expressing pathological human tau.
Voyager continues to anticipate an IND filing for VY-TAU01 in the
first half of 2024.
“The Voyager team is increasingly excited about the potential
for treatments targeting pathological tau to play an important role
in improving clinical outcomes for patients suffering with
Alzheimer’s disease,” said Todd Carter, Ph.D., Chief Scientific
Officer of Voyager Therapeutics. “Recent third-party data with
tau-targeted investigational therapies have demonstrated reduced
spread of pathological tau, as measured by tau PET imaging, and
favorable trends in cognition. We look forward to continuing to
advance our two programs targeting tau, and observing anticipated
data read-outs from others in this space that will play an
important role in further validating this target.”
About Alzheimer’s DiseaseAlzheimer’s disease is
a progressive neurodegenerative disease estimated to affect 6
million people in the U.S.i and up to 416 million people
globallyii. The disease causes memory loss and may escalate to
decreased independence, communication challenges, behavioral
disorders such as paranoia and anxiety, and lack of physical
controliii. In 2023, the total cost of caring for people living
with Alzheimer’s and other dementias in the U.S. is estimated at
$345 billioniv.
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).
In preclinical studies, TRACER-generated capsids have demonstrated
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 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 “will,” “expect,” “continue,” “anticipate,” 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 tau silencing gene therapy program and
anti-tau antibody program, including expectations for Voyager’s
achievement of preclinical and clinical development milestones for
its potential development candidates such as IND filings; the
potential for treatments targeting pathological tau to improve
clinical outcomes for patients suffering with Alzheimer’s disease;
and the potential for additional third-party data read-outs to
provide further validation for pathological tau as a target 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: Adam Silverstein, adam@scientpr.com
____________________________
i Alzheimer’s Association. 2023 Alzheimer’s Facts and Figures.
Available at:
https://www.alz.org/media/Documents/alzheimers-facts-and-figures.pdf.
Accessed February 15, 2024.ii Gustavsson A, Norton N, Fast T,
et al. Global estimates on the number of persons across the
Alzheimer's disease continuum. Alzheimer's Dement. 2023; 19:
658–670. doi: 10.1002/alz.12694. iii Penn Medicine. The 7 Stages of
Alzheimer’s Disease. Available at:
https://www.pennmedicine.org/updates/blogs/neuroscience-blog/2019/november/stages-of-alzheimers.
Accessed February 15, 2024.iv USAgainstAlzheimer’s. The Alzheimer’s
Disease Crisis – By the Numbers. Available at: The Alzheimer’s
Disease Crisis – By the Numbers | UsAgainstAlzheimer's
(usagainstalzheimers.org). Accessed: February 15, 2024.
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