SAN DIEGO and CAMBRIDGE, Mass., Sept.
11, 2020 /PRNewswire/ -- 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 of -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 Disease
Poster # 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 Disease
Poster # 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 Trial
Paused 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 Trial
Preparations 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 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; 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 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
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.
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SOURCE Neurocrine Biosciences, Inc.