Neurocrine Biosciences, Inc. (Nasdaq: NBIX) and Voyager
Therapeutics, Inc. (Nasdaq: VYGR) today announced data from
PD-1101, a Phase Ib open-label, three-year efficacy and safety
study, demonstrating that a one-time treatment with investigational
gene therapy, NBIb-1817 (VY-AADC), showed sustained improvement in
motor function including greater “On” time without troublesome
dyskinesia, reduction in Unified Parkinson’s Disease Rating Scale
(UPDRS) Part III scores, and reduction in the amount of medications
in patients with Parkinson’s disease. In the PD-1101 study,
NBIb-1817 reduced average “Off” time by up to -1.91 hours and
improved average “On” time without troublesome dyskinesia by up to
+2.23 hours in patients with advanced Parkinson’s disease after
three years across three cohorts. In addition, 14 out of 15
patients treated with NBIb-1817 continued to show an improvement in
disease staging after three years, as assessed by the modified
Hoehn & Yahr scale. These new data, along with two-year data
from another open-label Phase Ib trial, PD-1102, were presented
today at the MDS Virtual Congress 2020, September 12–16, 2020
(www.mdscongress.org/Congress/Registration.htm).
In data from the three-year PD-1101 trial, the one-time
treatment with NBIb-1817 showed sustained reduction in diary “Off”
time by an average of -0.15 to -1.91 hours (baseline 4.28 to 4.93
hours) and improved diary “On” time without troublesome dyskinesia
by an average of +0.26 to +2.23 hours (baseline 10.32 to 10.46
hours) across the cohorts as reported by 15 patients with advanced
Parkinson’s disease. NBIb-1817 also showed sustained improvement in
motor function after three years, as measured by UPDRS Part III off
medication scores, by -10.2 to -19.0 points (baseline 35.8 to 38.2
points) across the cohorts, per clinician assessment. Requirements
for Parkinson’s disease medications were also reduced in cohorts 2
and 3 (daily levodopa-equivalent dose reductions, average of -322.0
and -441.2 mg/day, respectively; baseline 1507.0 and 1477.0 mg/day,
respectively). Two-year data from the PD-1102 trial for 7 patients
showed that NBIb-1817 reduced diary “Off” time by an average of
-3.2 hours and increased diary good “On” time by +2.1 hours
(baselines 9.3 hours and 6.6 hours, respectively). In this study,
NBIb-1817 showed sustained improvement in motor function after two
years, with improved UPDRS Part III off medication scores of -12.0
points (baseline 34.4). Requirements for Parkinson’s disease
medications were also reduced (daily levodopa-equivalent dose
reduction, average pf -439.5 mg/day; baseline 1500.9 mg/day).
Preliminary safety data from both studies suggest that NBIb-1817
was well-tolerated, with no study drug-related serious adverse
events (SAEs) reported. The most common adverse events reported
were headache, hypoesthesia, and musculoskeletal pain (PD-1101),
and upper respiratory tract infection, headache, nausea, and
depression (PD-1102).
“It is promising to see that after three years, a single
administration of one-time investigational gene therapy treatment
NBIb-1817 showed sustained reduction in “Off” time, as well as
improvement in “On” time without troublesome dyskinesia and other
measures of motor function in patients with Parkinson’s disease,”
said Chad Christine, M.D., primary author, a lead investigator of
the study and Professor of Neurology at the University of
California, San Francisco (UCSF) Weill Institute for Neurosciences.
“Parkinson’s disease patients’ motor function would be expected to
worsen over three years, making these results very encouraging. The
standard of care for advanced Parkinson’s disease has not
significantly changed in decades and it is our hope that NBIb-1817
has the potential to become the first gene therapy for Parkinson’s
disease.”
Parkinson's disease is a chronic, progressive and debilitating
neurodegenerative disorder that affects approximately one million
people in the U.S. and six million people worldwide. It is
characterized by a loss of dopamine from neuronal degeneration,
with a concomitant loss of the aromatic L-amino acid decarboxylase
(AADC) enzyme required to synthesize dopamine in the brain, leading
to associated impairment in motor, neuropsychiatric, and autonomic
functions.
“We are pleased that the results from these studies show that
one-time treatment with investigational NBIb-1817 may help restore
the brain’s ability to convert levodopa into dopamine,” said Eiry
Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences.
“Our hope is that NBIb-1817 will help patients experience less
“Off” time and more “On” time and improve motor symptom control. We
plan to re-initiate enrollment in our registrational RESTORE-1
clinical trial with NBIb-1817 this year and look forward to further
evaluating NBIb-1817 in patients with Parkinson’s disease.”
NBIb-1817 is an investigational recombinant adeno-associated
viral serotype 2 vector encoding the gene for human AADC that is
designed to help produce the AADC enzyme in brain cells where it
can convert levodopa to dopamine.
“We are encouraged by the congruence of long-term data,
including clinician- and patient-reported clinical outcomes in our
clinical studies,” said Omar Khwaja, M.D., Ph.D., Chief Medical
Officer and Head of Research and Development at Voyager
Therapeutics. “These results are promising and show that the
approach has the potential to transform the treatment of
Parkinson’s disease, and help improve the lives of patients and
their families.”
Additional information about PD-1101 and PD-1102 will be
available on demand for registered participants through October 1,
2020 on the MDS meeting website
(www.mdscongress.org/Congress/Registration.htm).
- Christine CW, Richardson RM, Van Laar AD, et al. Three-Year
Safety and Clinical Outcomes from the PD-1101 Trial of AADC Gene
Therapy for Advanced Parkinson’s DiseasePoster # 879: Update on
Genetics of Movement Disorders, September 13, 2020, 10:30–12:30pm
EST (10-minute prerecorded presentation)
- Factor SA, Van Laar AD, Richardson RM, et al. AADC Gene Therapy
Administered via a Posterior Approach: 24-Month Results from the
PD-1102 Trial in Advanced Parkinson’s DiseasePoster # 889: Poster
Tour, launches on-demand on September 11, 2020 8:00am EST (5-minute
prerecorded presentation)
About Parkinson’s Disease and NBIb-1817
(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
worldwide. It is characterized by a loss of dopamine and neuronal
degeneration with a concomitant loss of the aromatic L-amino acid
decarboxylase (AADC) enzyme required to synthesize dopamine in the
brain, leading to associated impairment in motor, neuropsychiatric,
and autonomic functions. Dopamine is a chemical “messenger” that is
produced in the brain and is involved in the control of movement.
It is made when AADC converts the chemical levodopa to dopamine. As
Parkinson’s disease progresses, there is less AADC enzyme in parts
of the brain where levodopa is converted to dopamine.
NBIb-1817 is an investigational recombinant adeno-associated
viral (AAV) serotype 2 vector encoding the gene for human AADC that
is designed to help produce the AADC enzyme in brain cells where it
can convert levodopa to dopamine. NBIb-1817 is administered into
the brain using intraoperative monitoring with magnetic resonance
imaging (MRI)-facilitated targeted delivery.
About the RESTORE-1 Clinical TrialPaused
temporarily in April 2020 due to the COVID-19 pandemic, Neurocrine
Biosciences and Voyager Therapeutics plan to re-initiate RESTORE-1,
a Phase 2, randomized, placebo-surgery controlled, double-blinded,
multi-center clinical trial, to evaluate the safety and efficacy of
NBIb-1817 in patients who have been diagnosed with Parkinson's
disease for at least four years 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
clinicaltrials.gov and restore1study.com.
About the RESTORE-2 Clinical TrialPreparations
are ongoing for the RESTORE-2 global registrational trial that will
include clinical sites within and outside the U.S.
About Neurocrine Biosciences and Voyager Therapeutics
Strategic Collaboration In 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 Neurocrine Biosciences Neurocrine
Biosciences is a neuroscience-focused, biopharmaceutical company
with 28 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, Parkinson’s disease, endometriosis* and
uterine fibroids*, with three pivotal and five mid-stage clinical
programs in multiple therapeutic areas. 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 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 partnered pipeline focuses on severe neurological
diseases for which effective new therapies are needed, including
Parkinson’s disease, Huntington’s disease, Friedreich’s ataxia, and
other severe neurological diseases. 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
StatementsIn 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; the
impact of the COVID-19 pandemic and efforts to mitigate its spread
on our business; risks and uncertainties associated with the scale
and duration of the COVID-19 pandemic and resulting global,
national, and local economic and financial disruptions; risk and
uncertainties related to any COVID-19 quarantines, shelter-in-place
and similar government orders that are currently in place or that
may be put in place in the future, including the impact of such
orders on our business operations and the business operations of
the third parties on which we rely; risks related to the
development of our product candidates; 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
June 30, 2020. Neurocrine disclaims any obligation to update the
statements contained in this press release after the date
hereof.
Voyager Therapeutics 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 Therapeutics makes regarding the potential
impact or significance of the long-term medical data for patients
treated in the PD-1101 and PD-1102 clinical trials; the
re-initiation of RESTORE-1 Phase 2 clinical trial prior to
year-end, the initiation of the RESTORE-2 Phase 3 clinical trial
during the first half of 2021; the initiation, timing, progress,
activities, goals and reporting of results of other activities
associated with the PD program, and the potential benefits, timing
and future operation of the collaboration with Neurocrine
Biosciences 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, 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
now being evaluated in clinical trials; the impact of the COVID-19
pandemic and efforts to mitigate its spread on our clinical trials
and our business generally; risks related to the initiation and
conduct of preclinical studies and clinical trials; the sufficiency
of preclinical and clinical data to support applications for
additional studies and marketing approval of our PD drug
development candidates; changes in expectations from the FDA and
other regulatory authorities as to the requirements for obtaining
product approvals; the decisions of the FDA and other regulatory
authorities in response to applications we file in connection with
our product candidates under our PD program and otherwise in our
conduct of PD drug development activities; the priorities,
capabilities, diligence and efforts of Neurocrine Biosciences, our
collaboration partner for the PD program, and other collaborators
and vendors supporting our PD program; and the commercial
potential of PD product candidates that may be developed as part of
our PD program. These statements are also subject to a number of
material risks and uncertainties that are described in Voyager
Therapeutics’ Annual Report on Form 10K, Voyager Therapeutics’
Quarterly Reports on Form 10-Q and other reports filed by Voyager
Therapeutics with the Securities and Exchange Commission, as
may be updated by its subsequent filings with the Securities
and Exchange Commission. All information in the press release is as
of the date of this press release, and any forward-looking
statement speaks only as of the date on which it was made. Voyager
Therapeutics undertakes no obligation to publicly update or revise
this information or any forward-looking statement, whether as a
result of new information, future events or otherwise, except as
required by law.
Contact: Neurocrine
BiosciencesNavjot Rai
(Media)858-617-7623media@neurocrine.com
Todd Tushla
(Investors)858-617-7143ir@neurocrine.com
Contact: Voyager TherapeuticsPaul Cox
(Investors)857-201-3463pcox@vygr.com
Sheryl SeapyW2Opure949-903-4750sseapy@purecommunications.com
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