Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene
therapy company focused on developing life-changing treatments for
severe neurological diseases, today announced dosing of the first
patient in RESTORE-1, a Phase 2, randomized, double-blind,
placebo-controlled trial evaluating the safety and efficacy of
VY-AADC for the treatment of Parkinson’s disease in patients with
motor fluctuations that are refractory to medical management.
“Patients with Parkinson’s disease need new
therapeutic options, especially as the disease progresses and there
is less AADC enzyme in parts of the brain where it is needed to
convert levodopa to dopamine,” said Mark Richardson, M.D., Ph.D.,
Associate Professor, Director of Epilepsy and Movement Disorders
Surgery at the University of Pittsburgh Medical Center and
principal investigator in the RESTORE-1 trial. “Voyager’s VY-AADC
is an experimental gene therapy that is designed to deliver the
AADC gene into brain cells where the enzyme can be produced to
increase dopamine production. We are excited to contribute to this
Phase 2, placebo-controlled trial of VY-AADC in patients with
Parkinson’s disease.”
About the RESTORE-1 Phase 2 trial of
VY-AADC for Parkinson’s Disease
The Phase 2 RESTORE-1 trial will enroll patients
who have been diagnosed with Parkinson’s disease for at least four
years, are not responding adequately to oral medications, and have
at least three hours of OFF time during the day as measured by a
validated self-reported patient diary. Patients who meet the
eligibility criteria will be randomized (1:1) to one-time
administration of VY-AADC (for a total dose of up to 2.5×1012
vector genomes) or placebo surgery.
The primary endpoint of RESTORE-1 is ON time
without troublesome dyskinesia, or good ON time, as measured by a
self-reported patient diary at 12 months. Secondary endpoints
include diary OFF time, other motor function and quality of life
measures from the United Parkinson’s Disease Rating Scales
(UPDRS-II,-III scores), the Parkinson’s Disease Questionnaire
(PDQ-39), and patient’s global function as measured by the
proportion of participants with improvement on the Clinical Global
Impression (CGI) score. The trial will also measure non-motor
symptoms from the Non-Motor Symptom Scale (NMSS), as well as
safety.
Biomarker data include measurements of the
coverage of the specific region of the brain (putamen) targeted
with VY-AADC and measurements of AADC enzyme expression and
activity in the putamen measured by positron emission tomography
(PET) using fluorodopa F-18. Changes in patients’ daily doses of
oral levodopa and related medications will also be recorded.
For additional information regarding Voyager’s
RESTORE-1 Phase 2 clinical trial with its gene therapy VY-AADC for
the treatment of Parkinson’s disease, please use the following link
or email Voyager at clinicaltrials@vygr.com.
About Voyager’s VY-AADC Gene Therapy for
Parkinson’s Disease
VY-AADC is an investigational gene therapy
product designed to deliver the AADC gene directly into neurons of
the putamen where dopamine receptors are located, bypassing the
substantia nigra neurons and enabling the neurons of the putamen to
produce the AADC enzyme to convert levodopa into dopamine. With
this approach, VY-AADC has the potential to durably enhance the
conversion of levodopa to dopamine and provide clinically
meaningful improvements by restoring motor function in patients and
improving symptoms following a single administration.
The FDA granted Regenerative Medicine Advanced
Therapy (RMAT) designation for VY-AADC for the treatment of
Parkinson’s disease in patients with motor fluctuations that are
refractory to medical management. RMAT designation is an expedited
program for the advancement and approval of regenerative medicine
products, including gene therapy products. RMAT designation was
granted based on clinical data from the Phase 1b trial with VY-AADC
in patients with Parkinson’s disease. During this trial, one-time
administrations of VY-AADC demonstrated robust and durable
improvements in patients’ motor function along with substantial
reductions in use of daily oral levodopa and other Parkinson’s
disease medications. Infusions of VY-AADC have been well-tolerated
in this trial with no vector-related serious adverse events
reported to date.
About Parkinson’s Disease
Parkinson’s disease is a chronic, progressive
and debilitating neurodegenerative disease that affects
approximately 1,000,000 people in the U.S.1 and seven to 10 million
people worldwide2. While the underlying cause of Parkinson’s
disease in most patients is unknown, the motor symptoms of the
disease arise from a loss of neurons in the midbrain that produce
the neurotransmitter dopamine. Declining levels of dopamine and the
enzyme needed to convert levodopa to dopamine in this region of the
brain, the putamen, leads to the motor symptoms associated with
Parkinson’s disease including tremors, slow movement or loss of
movement, rigidity, and postural instability. Additional motor
symptoms during the advanced stages of the disease include falling
and difficulty with speech and swallowing, with patients often
requiring the daily assistance of a caregiver.
There are currently no therapies that
effectively slow or reverse the progression of Parkinson’s disease.
Levodopa remains the standard of care treatment, with its
beneficial effects on symptom control having been discovered over
40 years ago3. Patients are generally well-controlled with oral
levodopa in the early stages of the disease but become less
responsive to treatment as the disease progresses. Patients
experience longer periods of reduced mobility and stiffness termed
OFF time, or the time when medication is no longer providing
benefit, and shorter periods of ON time when their medication is
effective.
The progressive motor symptoms of Parkinson’s
disease are largely due to the death of dopamine neurons in the
substantia nigra, a part of the midbrain that converts levodopa to
dopamine, in a single step catalyzed by the enzyme AADC.
Neurons in the substantia nigra release dopamine into the putamen
where the receptors for dopamine reside. In Parkinson’s disease,
neurons in the substantia nigra degenerate and the enzyme AADC is
markedly reduced in the putamen, which limits the brain’s ability
to convert levodopa to dopamine4. The intrinsic neurons in the
putamen, however, do not degenerate in Parkinson’s disease5,6.
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 pipeline focuses on severe
neurological diseases in need of effective new therapies, including
Parkinson’s disease, a monogenic form of ALS called SOD1,
Huntington’s disease, Friedreich’s ataxia, neurodegenerative
diseases related to defective or excess aggregation of tau protein
in the brain including Alzheimer’s disease and severe, chronic
pain. Voyager has strategic collaborations with Sanofi Genzyme and
AbbVie. 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.
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 gene therapy platform, its ability to develop
manufacturing capability for its products and successfully
transition its manufacturing process, its ability to perform under
existing collaborations with, among others, Sanofi Genzyme and
AbbVie and to add new programs to its pipeline, its ability to
enter into new partnerships or collaborations, the sufficiency of
its cash resources and the regulatory pathway of, and the timing or
likelihood of its regulatory filings and approvals for, any of its
product candidates, 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, the initiation and conduct of
preclinical studies and clinical trials; the availability of data
from clinical trials; the expectations for regulatory
communications, submissions and approvals; the continued
development of the gene therapy platform; Voyager’s scientific
approach and general development progress; and the availability or
commercial potential of Voyager’s product candidates. 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.
1 Willis et al, Neuroepidemiology.2010;34:143–151
2 www.pdf.org/en/parkinson_statistics
3 Poewe W, et al, Clinical Interventions in
Aging.2010;5:229-238.
4 Lloyd, J Pharmacol Exp Ther. 1975;195:453-464, Nagatsu, J
Neural Transm Suppl.2007
5 Cold Spring Harb Perspect Med 2012;2:a009258
6 Braak et al, Cell Tissue Res.2004;318:121-134
Investor Relations:
Matt Osborne
Vice President of Corporate Affairs, Communications and Investor Relations
857-259-5353
mosborne@vygr.com
Media:
Sheryl Seapy
W2O Group
949-903-4750
sseapy@w2ogroup.com
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