Voyager Therapeutics Highlights Lead Program VY-AADC for Advanced Parkinson’s Disease, AAV Novel Capsid and Delivery Optimi...
November 16 2017 - 04:45PM
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company focused on developing life-changing treatments for
severe neurological diseases, today at its R&D Day highlighted
recent progress and plans for VY-AADC for advanced Parkinson’s
disease, and progress with testing novel adeno-associated virus
(AAV) capsids and delivery optimization efforts, along with its
ALS, Huntington’s disease, Friedreich’s ataxia, anti-Tau antibody
and severe chronic pain preclinical programs.
“Results from our primary market research of
physicians and patients presented today at our R&D day reflect
the excitement surrounding our VY-AADC program and its one-time
treatment, unique mechanism of action, and potential for marked and
durable improvements in patients’ motor function,” said Steve Paul,
M.D., president and chief executive officer of Voyager
Therapeutics. “In addition, based on these attributes, neurologists
indicated an interest to recommend VY-AADC to patients even before
they reach an advanced stage of disease. Our global, pivotal
program for VY-AADC in advanced Parkinson’s disease remain on
track, and we are excited to consider this and other areas of
Parkinson’s disease with this program. Also at the R&D day, we
described our innovative work optimizing capsid and vector delivery
and moving only highly-targeted and differentiated candidates into
the clinic.”
Senior executives from Voyager Therapeutics’
scientific, clinical and management team, provided the following
highlights at the R&D day:
- Results from primary market research indicating a strong
interest among neurologists, movement disorder specialists,
neurosurgeons and patients for VY-AADC for advanced Parkinson’s
disease. The primary market research was conducted following the
recent Phase 1b results with VY-AADC that demonstrated durable,
dose-dependent and time-dependent improvements across multiple
measures of patients’ motor function after a one-time
administration, and with meaningfully lower doses of oral levodopa.
Key drivers of interest for VY-AADC among these physicians and
patients included its one-time treatment, lack of in-dwelling
hardware used in deep brain stimulation that often requires
replacement and revisions, unique mechanism of action, and
magnitude and durability of improvement in patients’ motor
function. In addition, based on these attributes, neurologists
indicated an interest to recommend VY-AADC to patients earlier
during the course of Parkinson’s disease, before they reached the
advanced stage of the disease.
- Plans to begin the global, pivotal Phase 2-3 program for
VY-AADC before year-end 2017 and dose the first patient during the
second quarter of 2018. Voyager remains on track to file an
investigational new drug (IND) application before year-end 2017
with product for VY-AADC manufactured using its baculovirus/Sf9
manufacturing process to support this pivotal program.
- A review of VY-AADC clinical data including the Phase 1b
results, historical placebo rates for past gene therapy trials in
advanced Parkinson’s disease, and a posterior trajectory approach
designed to further optimize the intracranial delivery of VY-AADC.
Investigators have now dosed a total of six patients in this
separate Phase 1 trial designed to further optimize the
intracranial delivery of VY-AADC. Administration of VY-AADC with
this posterior approach has been well-tolerated in all six patients
dosed since the start of the trial and no serious adverse events
have been reported. The posterior approach resulted in greater
average putaminal coverage (approximately 50%) and reduced average
surgical times by two to three hours compared with the transfrontal
approach of Cohorts 1 through 3 of the ongoing Phase 1b clinical
trial. Voyager continues to expect to enroll additional patients in
this trial prior to the start of the pivotal Phase 2-3 program and
will continue to follow patients from Cohorts 1 through 3, and
those in this posterior trajectory trial, and plans to report
updated results from these trials during the first quarter of 2018,
prior to the start of patient enrollment in the pivotal program.
- Data in adult non-human primates with novel AAV gene therapy
capsids that crossed the blood-brain barrier after a single,
intravenous administration resulting in widespread, enhanced gene
transfer to the brain and spinal cord. One month after dosing of a
transgene encoding both a therapeutic protein (frataxin) and a
reporter gene (HA) to facilitate molecular and immunohistochemical
measurements, substantial levels of frataxin-HA were expressed in
the CNS, including motor neurons throughout the length of the
spinal cord, the brain stem, pyramidal neurons in the motor cortex,
and neurons in the substantia nigra, and cerebellar dentate
nucleus.
- Preclinical data from its Friedreich’s ataxia (FA) program in a
transgenic mouse model of FA, with a one-time intravenous (IV)
dosing of an AAV vector composed of a novel capsid and a frataxin
transgene, together with intracerebral dosing of an AAV vector with
the same transgene, that led to a rapid halting and reduction of FA
disease progression in multiple functional tests of motor behavior.
Additional preclinical studies are underway at Voyager including
steps to identify a lead clinical candidate for the treatment of FA
during 2018.
- An overview of its capsid and delivery optimization efforts
resulting in lead candidate selections of VY-SOD101 for the
treatment of amyotrophic lateral sclerosis (ALS) caused by
mutations in the superoxide dismutase 1 gene (SOD1) and VY-HTT01
for Huntington’s disease. During the third quarter of 2017, Voyager
conducted preclinical studies with additional routes of
administration in large animal models, including studies with its
novel AAV capsids yielding markedly improved bio-distribution and
pharmacology with the potential for substantially improved
efficacy. Voyager plans further investigation of novel capsids and
additional routes of administration for VY-SOD101 and, together
with the ongoing efforts on the Huntington’s disease and
Friedreich’s ataxia programs, expects that these efforts will
support two IND filings from these programs during 2019.
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. The company’s pipeline focuses on severe
neurological diseases in need of effective new therapies, including
advanced Parkinson’s disease, a monogenic form of ALS, Huntington’s
disease, Friedreich’s ataxia, frontotemporal dementia, Alzheimer’s
disease and severe, chronic pain. Voyager has broad strategic
collaborations with Sanofi Genzyme, the specialty care global
business unit of Sanofi, and the University of Massachusetts
Medical School. Founded by scientific and clinical leaders in
the fields of AAV gene therapy, expressed RNA interference and
neuroscience, Voyager Therapeutics is headquartered in Cambridge,
Massachusetts. For more information, please visit
www.voyagertherapeutics.com.
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
and reporting of results of its preclinical programs and clinical
trials and its research and development programs, its ability to
advance its AAV-based gene therapies into, and successfully
initiate, enroll and complete, clinical trials, the potential
clinical utility of its product candidates, its ability to continue
to develop its product engine, its ability to develop manufacturing
capability for its products, its ability to add new programs to its
pipeline, its ability to enter into new partnerships or
collaborations, its expected cash, cash equivalents and marketable
debt securities at the end of a fiscal period and anticipation for
how long expected cash, cash equivalents and marketable debt
securities will last, and the timing or likelihood of its
regulatory filings and approvals, are forward looking. All
forward-looking statements are based on estimates and assumptions
by Voyager’s management that, although Voyager believes 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,
those related to the initiation and conduct of preclinical studies
and clinical trials, the availability of data from clinical trials
and the expectations for regulatory submissions and approvals; the
continued development of the product engine; Voyager’s scientific
approach and general development progress; the availability or
commercial potential of Voyager’s product candidates; the
sufficiency of cash resources; and need for additional financing.
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, as updated by its subsequent filings with the
Securities and Exchange Commission. Any forward-looking statement
speaks only as of the date on which it was made. Voyager
undertakes no obligation to publicly update or revise any
forward-looking statement, whether as a result of new information,
future events or otherwise, except as required by law.
Investor Relations:
Matt Osborne
Vice President of Investor Relations & Corporate Communications
857-259-5353
mosborne@vygr.com
Media:
Katie Engleman
Pure Communications, Inc.
910-509-3977
Katie@purecommunicationsinc.com
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