Voyager Therapeutics Receives FDA Clearance of IND Application for Gene Therapy Candidate VY-HTT01 for Treatment of Huntingto...
April 26 2021 - 8:00AM
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a clinical-stage gene
therapy company developing life-changing treatments for severe
neurological diseases, today announced the U.S. Food and Drug
Administration (FDA) has removed its clinical hold on the company’s
Investigational New Drug (IND) application for VY-HTT01, a gene
therapy candidate for the treatment of Huntington’s disease (HD),
and confirmed that the company may proceed with its planned Phase
1/2 clinical trial. The decision was made following a comprehensive
review of the Chemistry, Manufacturing and Controls information
previously submitted to the FDA. Voyager plans to initiate VYTAL, a
Phase 1/2 clinical trial of VY-HTT01, this year.
“The decision by the FDA regarding our IND application for
VY-HTT01 for Huntington’s disease represents an important milestone
for Voyager and is the result of years of commitment to developing
an impactful new therapy to address this devastating disease,” said
Andre Turenne, President and CEO of Voyager.
Currently there are no disease-modifying therapies approved for
the treatment of Huntington’s disease, a fatal, inherited
neurodegenerative disease caused by toxic gain-of-function
mutations in the huntingtin, or HTT, gene. HD affects over 30,000
people in the U.S. alone, with symptom onset commonly appearing
between the ages of 30 to 50. The disease is marked by progressive
decline of motor and cognitive functions and a range of behavioral
and psychiatric disturbances.
VY-HTT01 is a gene therapy designed to reduce the expression of
huntingtin, thereby altering disease progression. VY-HTT01 is
comprised of an adeno-associated virus capsid (AAV1) and a
proprietary transgene that harnesses the canonical RNA interference
pathway to selectively knock down levels of HTT mRNA. Preclinical
data in non-human primates demonstrated robust and durable
reduction of HTT mRNA and protein and widespread distribution of
VY-HTT01 across the striatum and cortex, which are core areas of
disease pathology.
“Our investigational gene therapy has been designed to achieve
broad knockdown of HTT mRNA throughout the brain via a one-time
MRI-guided neurosurgical delivery,” said Omar Khwaja, CMO and Head
of Research and Development of Voyager. “We are thrilled to be
collaborating with leading experts in Huntington’s disease and
neurosurgical delivery of gene therapies as we begin the planned
clinical evaluation of our promising candidate.”
Voyager’s VYTAL Phase 1/2 clinical trial is a dose escalation
study to evaluate the safety and tolerability of VY-HTT01 in
patients with early manifest Huntington’s disease. Secondary
endpoints include disease biomarkers and clinical outcome
measures.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene therapy company
focused on developing life-changing treatments for severe
neurological diseases. Voyager is committed to advancing the field
of AAV gene therapy through innovation and investment in vector
engineering and optimization, manufacturing, and dosing and
delivery techniques. Voyager’s wholly owned and partnered pipeline
focuses on severe neurological diseases for which effective new
therapies are needed, including Parkinson’s disease, Huntington’s
disease, Friedreich’s ataxia, and other severe neurological
diseases. For more information on Voyager Therapeutics, please
visit the company’s website at www.voyagertherapeutics.com or
follow @VoyagerTx on Twitter and LinkedIn.
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 “may,” “might,” “will,” “would,” “should,”
“expect,” “plan,” “anticipate,” “believe,” “estimate,”
“undoubtedly,” “project,” “intend,” “future,” “potential,” or
“continue,” and other similar expressions are intended to identify
forward-looking statements. For example, all statements Voyager
makes regarding the initiation, timing, progress, activities and
goals of the VYTAL clinical trial of VY-HTT01, the ability of
VY-HTT01 to achieve broad knock down of HTT mRNA throughout the
brain, the collaboration by Voyager with leading Huntington’s
disease and neurosurgical experts, and Voyager’s intentions and
programs for the development of a gene therapy for Huntington’s
disease are forward looking statements.
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 ability to arrange for the initiation of
the VYTAL clinical trial at clinical sites, including the ability
to receive the review by and approval of Institutional Review
Boards and other necessary institutional approvals at proposed
clinical sites, the ability to recruit and engage researchers,
physicians and patients to participate in the VYTAL clinical trial,
the ability to agree to terms, and to sign and implement clinical
trial agreements and associated agreements with VYTAL clinical
trial collaborators, the ability to identify Huntington patients
who meet the eligibility requirements for the VYTAL clinical trial,
and the ability for Voyager to conduct a clinical trial in a timely
manner under pending Covid-19 pandemic protocols. These statements
are also subject to a number of material risks and uncertainties
that are described in Voyager’s Annual Report on Form 10-K filed
with the Securities and Exchange Commission, as updated by its
subsequent filings 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.
Investors:
Investors@voyagertherapeutics.com
Media:
Sheryl Seapy
W2Opure949-903-4750sseapy@purecommunications.com
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