Neurocrine Biosciences, Inc. (NASDAQ: NBIX) and Voyager
Therapeutics, Inc. (NASDAQ: VYGR) today announced the publication
of interim results from the Phase 1b trial of VY-AADC, an
investigational gene therapy treatment for Parkinson’s disease, in
the peer-reviewed journal, Annals of Neurology. The publication
titled “Magnetic Resonance Imaging-Guided Phase 1 Trial of
Putaminal AADC Gene Therapy for Parkinson's Disease,” can be
accessed online. The publication reports data from patients in this
trial who were followed for up to three years (Cohort 1), two years
(Cohort 2), or 18 months (Cohort 3) as reported in November 2018.
“The interim results from this Phase 1b trial
demonstrated that administration of VY-AADC to the putamen using a
novel technique, which included intraoperative monitoring with
magnetic resonance imaging guidance, facilitated targeted delivery
of the investigational gene therapy,” said Chad Christine, M.D.,
Professor of Neurology, University of California, San Francisco and
Investigator in the Phase 1b trial of VY-AADC. “Additionally,
administration of VY-AADC resulted in dose-dependent increases in
AADC enzyme expression and improvements in clinical measures and
has been well-tolerated to date.”
About the Phase 1b trial of
VY-AADC
The Phase 1b, open-label trial included 15
patients with Parkinson’s disease and disabling motor fluctuations,
treated with a single administration of VY-AADC. The primary
objectives of the trial were to assess the safety, tolerability and
distribution of ascending doses of VY-AADC administered under
magnetic resonance imaging (MRI) guidance to the putamen, a region
of the brain associated with impaired motor function in Parkinson’s
disease.
Secondary objectives included assessment of AADC
enzyme expression and activity in the putamen measured by positron
emission tomography (PET) using [18F] fluorodopa (or 18F-DOPA),
which reflects the capacity to convert levodopa to dopamine. Other
secondary measures included assessments of motor function and
activities of daily living, as measured by the Unified Parkinson’s
Disease Rating Scale (UPDRS-III and UPDRS-II, respectively),
quality of life, and a patient-completed Hauser diary. Daily
requirements for anti-Parkinsonian medications were also
measured.
About the Phase 2 RESTORE-1 Clinical
Trial
Based on the results from this Phase 1b trial
and a separate Phase 1 trial administering VY-AADC with a posterior
infusion trajectory, Voyager initiated RESTORE-1, a Phase 2,
randomized, placebo-surgery controlled, double-blinded,
multi-center, clinical trial to evaluate the safety and efficacy of
VY-AADC in 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.
For more information about the RESTORE-1
clinical trial, including eligibility criteria, please visit
restore1study.com.
About Neurocrine Biosciences and Voyager
Therapeutics Strategic Collaboration
In January 2019, Neurocrine Biosciences and
Voyager Therapeutics announced a strategic collaboration focused on
the development and commercialization of gene therapy programs,
VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s
ataxia, as well as rights to two programs to be determined. This
collaboration combines Neurocrine Biosciences’ expertise in
neuroscience, drug development and commercialization with Voyager’s
innovative gene therapy programs targeting severe neurological
diseases. The collaboration became effective in March 2019
following the expiration of the waiting period under the
Hart-Scott-Rodino Antitrust Improvements Act of 1976.
About Parkinson’s Disease and
VY-AADC
Parkinson’s disease is a chronic, progressive
and debilitating neurodegenerative disease that affects
approximately 1 million people in the U.S. and 6 million people
worldwide1. Parkinson’s disease is characterized by a loss of
dopamine and its function. Dopamine is a chemical “messenger” that
is produced in the brain and is involved in the control of
movement. Dopamine is made in the brain when the enzyme AADC
(Aromatic l-amino acid decarboxylase) converts the chemical
levodopa to dopamine. Levodopa, AADC, and dopamine are each present
at normal levels in healthy people. As Parkinson’s disease worsens,
there is less AADC enzyme in parts of the brain where it is needed
to convert levodopa to dopamine. When this happens, patients’ motor
function may worsen with a less predictable response to
medications.
VY-AADC, an investigational gene therapy, is
designed to put the AADC enzyme into brain cells where it can
convert levodopa to dopamine. To do this, the AADC gene is
delivered inside a transporter called “adeno-associated viral
vector” (AAV). Interim results from an open-label Phase 1b trial
demonstrated that administration of VY-AADC to the putamen using
intraoperative monitoring with MRI facilitated targeted delivery of
the investigational gene therapy with dose-dependent increases in
AADC enzyme expression and improvements in clinical measures and
has been well-tolerated to date.
About Neurocrine
Biosciences
Neurocrine Biosciences, a San Diego based
biopharmaceutical company, is focused on developing treatments for
neurological and endocrine related disorders. The company
discovered, developed and markets INGREZZA® (valbenazine) capsules,
the first FDA-approved treatment for adults with tardive
dyskinesia, an involuntary movement disorder. Neurocrine also
discovered and led the Phase II clinical development of ORILISSA®
(elagolix), the first FDA-approved oral medication for the
management of endometriosis with associated moderate to severe pain
in over a decade, which is marketed by AbbVie as part of a
collaboration to develop and commercialize elagolix for women’s
health. Neurocrine’s clinical development programs include
opicapone, an adjunctive therapy to levodopa/DOPA decarboxylase
inhibitors for Parkinson’s disease patients, elagolix for uterine
fibroids (with AbbVie), valbenazine for the treatment of Tourette
syndrome, NBI-74788 for the treatment of congenital adrenal
hyperplasia (CAH), and early-stage gene therapies for neurological
disorders including Parkinson's disease and Friedreich's ataxia,
acquired through a collaboration with Voyager Therapeutics. For
more information and the latest updates from Neurocrine
Biosciences, please visit www.neurocrine.com.
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 collaborative
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,
Alzheimer’s disease, and other neurodegenerative diseases related
to defective or excess aggregation of tau and alpha-synuclein
proteins in the brain. Voyager has strategic collaborations with
Sanofi Genzyme, AbbVie and Neurocrine Biosciences. 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
on Voyager Therapeutics, please visit the company’s website at
www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and
LinkedIn.
Neurocrine Biosciences Forward-Looking
Statements
In addition to historical facts, this press
release contains forward-looking statements that involve a number
of risks and uncertainties. These statements include, but are not
limited to, statements related to: the potential benefits to be
derived from the Voyager Therapeutics collaboration agreement,
including any statements related to Voyager’s proprietary
CNS-focused gene therapy platform and Neurocrine’s ability to
leverage such platform; Neurocrine’s ability to expand its research
and development pipeline, and Neurocrine’s ability to develop
disease modifying and potentially curative treatments for diseases,
including Parkinson’s disease and Friedreich’s ataxia. Among the
factors and risks that could cause actual results to differ
materially from those indicated in the forward-looking statements
are risks that the benefits of the agreements with Voyager may
never be realized; risks that the product candidates licensed from
Voyager may not obtain regulatory approval from the FDA or other
regulatory agencies, or such approval may be delayed or
conditioned; risks that development activities related to the
product candidates licensed from Voyager may not be completed on
time or at all; risks associated with the Company's dependence on
Voyager for research, development and manufacturing activities;
risks that ongoing or future clinical trials may not be
successful or replicate previous clinical trial results, or may not
be predictive of real-world results or of results in subsequent
clinical trials; risks and uncertainties relating to competitive
products and technological changes that may limit demand for
product candidates licensed from Voyager; risks that the product
candidates licensed from Voyager may be precluded from
commercialization by the proprietary rights of third parties; and
other risks that are described in the Company’s periodic reports
filed with the Securities and Exchange Commission, including
without limitation the Company’s annual report on Form 10-K for the
year ended December 31, 2018. Neurocrine disclaims any obligation
to update the statements contained in this press release after the
date hereof.
Voyager 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, goals and reporting of results of its
preclinical programs and clinical trials and its research and
development programs, the potential benefits and future operation
of the collaboration agreements with AbbVie and Neurocrine,
including any potential future payments thereunder, 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 perform under
existing collaborations with, among others, Sanofi Genzyme, AbbVie
and Neurocrine and to add new programs to its pipeline, 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, those related to 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, including antitrust
approvals related to Voyager’s collaborations; the continued
development of the gene therapy platform; Voyager’s scientific
approach and general development progress; the sufficiency of cash
resources; the possibility of timing of AbbVie’s exercise of its
development and license options under its collaborations, 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 www.michaeljfox.org
Neurocrine Biosciences Media & Investor Relations:
Navjot Rai
Director, Corporate Communications
858-617-7623
IR@neurocrine.com
Voyager Therapeutics Media & Investor Relations:
Matt Osborne
Vice President of Corporate Affairs, Communications and Investor Relations
857-259-5353
mosborne@vygr.com
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