VY-SOD101 Selected Based on Robust Delivery and
Knock-Down of Toxic SOD1 in Preclinical Models
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the central nervous system (CNS), today announced the
selection of VY-SOD101, a clinical candidate for the treatment of
ALS due to mutations in the superoxide dismutase 1 gene (SOD1).
Multiple studies have demonstrated that mutant SOD1 is toxic to
motor neurons, and leads to their progressive loss. VY-SOD101 is
composed of a proprietary adeno-associated virus (AAV) capsid and
transgene with a micro RNA (miRNA) expression cassette that
harnesses the RNAi pathway to selectively silence, or knock-down,
the production of SOD1 messenger RNA. With a single intrathecal
(IT) injection, VY-SOD101 has the potential to durably reduce the
levels of toxic mutant SOD1 protein in the CNS to slow the
progression of disease. Preclinical pharmacology and toxicology
studies are now underway to support filing of an investigational
new drug (IND) application for VY-SOD101 during the fourth quarter
of 2017.
“To select an AAV gene therapy candidate for a
particular disease, Voyager considers a number of features of the
overall candidate profile including optimization of the AAV vector
capsid, the transgene, and the dosing paradigm,” said Dinah Sah,
Ph.D, senior vice president of neuroscience at Voyager. “The
VY-SOD101 clinical candidate was selected after screening a series
of capsids, microRNA expression cassettes and encoded payloads.
Multiple rounds of optimization resulted in a candidate that is
potent and selective. In addition, many construct configurations
were evaluated toward the identification of one which would provide
excellent yield and genome integrity for manufacturing scale-up in
Voyager’s baculovirus AAV manufacturing system in insect-derived
cells. Preclinical data in large mammals demonstrated that a single
IT administration resulted in robust knock-down of SOD1 in motor
neurons, and based on these results, we are excited to progress
VY-SOD101 closer towards the clinic and to those living with this
devastating disease.”
“Voyager’s pipeline targeting severe, often
fatal, diseases of the CNS is rapidly progressing,” said Steven
Paul, M.D., president and chief executive officer at Voyager. “One
of the core competencies of our company is vector optimization, as
Dinah and her team have demonstrated with the selection of
VY-SOD101 for ALS. Importantly, vector delivery and manufacturing
at scale are additional core competencies, which will undoubtedly
facilitate the clinical development of VY-SOD101. During 2017, we
remain committed to progressing lead candidate selections for our
pipeline programs that are close behind VY-SOD101, including
VY-HTT01 for Huntington’s disease, and VY-FXN01 for Friedreich’s
ataxia.”
About Amyotrophic Lateral
Sclerosis
Amyotropic Lateral Sclerosis (ALS) is a rare,
rapidly progressive, fatal disease characterized by the
degeneration of nerve cells in the spinal cord and brain resulting
in severe muscle atrophy with loss of the ability to walk and
speak, and premature death. The median survival is approximately
three years, and 90 percent of people with ALS die within five
years of symptom onset.1 ALS affects approximately 20,000
people in the U.S., with less than 10,000 new cases identified each
year reflecting a high rate of mortality and short survival,
relative to other diseases with similar incidences.2
Patients with ALS typically develop weakness in
one body region (upper or lower limb or bulbar) and then develop
symptoms and signs of progressive dysfunction of motor neurons. The
majority of ALS cases occur sporadically and with unknown cause,
but in approximately 10 percent of patients, the cause is familial
and can be linked to an identifiable genetic defect. An estimated
20 percent of familial cases can be attributed to mutations in
SOD1, the first mutant gene discovered to be causal for the
development of ALS, through a toxic gain of function mechanism
leading to motor neuron pathogenesis.3 Riluzole is the only
drug approved by the U.S. Food and Drug Administration for the
treatment of ALS. In controlled trials, Riluzole delayed the time
to onset of tracheostomy or death by approximately two to three
months but did not improve muscle strength or neurological
function.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the CNS. 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 CNS diseases
in need of effective new therapies, including advanced Parkinson’s
disease, a monogenic form of ALS, Friedreich’s ataxia, Huntington’s
disease, 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. Follow Voyager on
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 law. The use of words such as “may,” “might,” “will,”
“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 complete, clinical
trials, its ability to continue to develop its product engine, its
ability to add new programs to its pipeline, its expected cash,
cash equivalents and marketable securities at the end of a fiscal
year and anticipation for how long expected cash, cash equivalents
and marketable 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. These statements are also subject to a number of material
risks and uncertainties that are described in Voyager’s most recent
Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission, as updated by its future 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 Sorenson EJ, et al. (2002) Neurology 59:280-282
2 www.alsa.org
3 Rosen D, et al. (1993) Nature 362:59-62
Investor Relations:
Matt Osborne
Head 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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