Neurocrine Biosciences, Inc. (NASDAQ: NBIX) and Voyager
Therapeutics, Inc. (NASDAQ: VYGR) today announced Phase I trial
results for VY-AADC from eight patients with Parkinson’s disease
who participated in the open-label trial to evaluate the safety and
efficacy of VY-AADC and to further assess the posterior (i.e., from
the back of the head) surgical delivery approach. These Phase I
results are being presented today as a poster presentation at the
2019 American Academy of Neurology (AAN) Annual Meeting.
Parkinson's disease is a chronic, progressive and debilitating
neurodegenerative disorder that affects approximately one million
people in the U.S.1
Treatment with VY-AADC improved good ON time (ON time without
troublesome dyskinesia) by 1.7 hours from baseline and reduced OFF
time by 2.2 hours at 12 months from baseline in patients with
Parkinson’s disease. Exploratory analyses in four of the eight
patients with low or no dyskinesia or absence of impulse control
disorder (ICD) at baseline demonstrated a greater improvement in
motor function including a 3.2-hour improvement in good ON time
from baseline to 12 months. Infusions of VY-AADC were well
tolerated with no serious adverse events (SAEs) reported. These
Phase I results show that the posterior trajectory is an additional
surgical delivery route in patients with Parkinson’s disease.
“The results from this Phase I trial in patients with
Parkinson’s disease provide further evidence that VY-AADC
administration can allow neurons in the brain to convert levodopa
to dopamine and improve motor function,” said Eiry Roberts, M.D.,
Chief Medical Officer at Neurocrine Biosciences. “The results from
this trial confirm previous data from a separate, ongoing Phase I
study demonstrating that increased coverage of the putamen with
VY-AADC leads to an increase in AADC enzyme activity and
improvements in motor function and quality of life in patients with
Parkinson’s disease – with less need for oral levodopa
medication.”
VY-AADC Motor Function Results from the Phase I
(PD-1102) Trial
The PD-1102 trial includes eight patients with advanced
Parkinson’s disease. On average, patients’ baseline characteristics
in PD-1102 were consistent with patients’ baseline from a separate,
ongoing Phase Ib trial (PD-1101) employing a frontal (i.e., from
the top of the head) surgical delivery approach. Two patients in
PD-1102 were identified as having impulse control disorder while no
patients were identified as having impulse control disorder in
PD-1101. At baseline, patients’ mean good ON time was 9.1 hours and
mean OFF time was 6.8 hours.
Administration of VY-AADC with the posterior trajectory resulted
in a mean coverage of the putamen of 54% and reduced the infusion
time by approximately two hours (from 5.2 hours to 3.1 hours)
compared to PD-1101. In PD-1102, treatment with VY-AADC increased
AADC enzyme activity in the putamen as measured by positron
emission tomography (PET) using [18F] fluorodopa (or 18F-DOPA) by
85%. AADC enzyme activity in the putamen as measured by PET using
18F-DOPA reflects the capacity of neurons in the brain to convert
levodopa to dopamine.
Treatment with VY-AADC improved patients’ motor function from
baseline to twelve months across multiple assessments. These
assessments include patient self-reported diary entries of ON and
OFF times (including good ON time), Unified Parkinson’s Disease
Rating Scales, and activities of daily living measures. In
addition, patients’ reported an ability to maintain motor function
with less Parkinson’s disease medication, as patients’ reported a
mean 28% reduction in the dosage of Parkinson’s disease medication
(measured as levodopa equivalents) at six months and at 12 months
from a baseline mean of 1,500 mg/day.
Treatment with VY-AADC improved patients’ good ON time by 1.7
hours from baseline and reduced OFF time by 2.2 hours from baseline
to 12 months. Exploratory analyses in PD-1101 suggested that
patients with high dyskinesia or an ICD at baseline may show
different outcomes, especially in patient-reported diary measures.
A clinical assessment of the subgroup of patients (n=4) with no or
low baseline dyskinesia as measured by the Unified Dyskinesia
Rating Scale score (≤ 30) and absence of ICD at baseline as
determined by the investigator, indicated that VY-AADC improved
good ON time from baseline by 3.2 hours and reduced OFF time by 3.2
hours in patients at 12 months.
In addition to motor function, VY-AADC improved patients’
quality of life as measured by the patient-reported 39-item
Parkinson’s Disease Questionnaire (PDQ-39). For PDQ-39, VY-AADC
improved (reduced) patients’ score by a mean change from baseline
to 12 months of -7.6. Infusions of VY-AADC have been well tolerated
in the eight patients treated in PD-1102 with no serious adverse
events (SAEs) reported.
About the Phase II RESTORE-1 Clinical
TrialBased on the results from PD-1101 and PD-1102,
Voyager initiated RESTORE-1, a Phase II, 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 the first quarter of 2019,
Neurocrine Biosciences and Voyager Therapeutics entered into 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.
About Parkinson’s Disease and VY-AADC
Parkinson’s disease is a chronic, progressive and debilitating
neurodegenerative disease that affects approximately one million
people in the U.S. and six 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 Ib 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 BiosciencesNeurocrine
Biosciences (Nasdaq: NBIX) is a neuroscience-focused,
biopharmaceutical company with more than 25 years of experience
discovering and developing life-changing treatments for people with
serious, challenging and under-addressed neurological, endocrine
and psychiatric disorders. The company's diverse portfolio
includes FDA-approved treatments for tardive dyskinesia and
endometriosis* and clinical development programs in multiple
therapeutic areas including Parkinson’s disease, congenital adrenal
hyperplasia and uterine fibroids*. Headquartered in San Diego,
Neurocrine Biosciences specializes in targeting and interrupting
disease-causing mechanisms involving the interconnected pathways of
the nervous and endocrine systems. For more information,
visit neurocrine.com, and follow the company
on LinkedIn. (*in collaboration with AbbVie)
About Voyager TherapeuticsVoyager 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 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 is headquartered in
Cambridge, Massachusetts. For more information on Voyager, please
visit the company’s website at www.voyagertherapeutics.com or
follow @VoyagerTx on Twitter and LinkedIn.
Voyager Therapeutics® is a registered trademark of Voyager
Therapeutics.
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. 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 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 quarterly report on Form 10-Q for
the quarter ended March 31, 2019. Neurocrine disclaims any
obligation to update the statements contained in this press release
after the date hereof.
Voyager Forward-Looking StatementsThis 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 Sanofi
Genzyme, 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
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, 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; the continued
development of the gene therapy platform; Voyager’s scientific
approach and general development progress; the sufficiency of cash
resources; the possibility or the timing of the exercise of
development, commercialization and license options under
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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