Voyager Therapeutics Provides Update on NBIb-1817 (VY-AADC) Gene Therapy Program
December 22 2020 - 4:17PM
Voyager Therapeutics, Inc. (Nasdaq: VYGR) today announced that the
U.S. Food and Drug Administration (FDA) has notified Neurocrine
Biosciences (Nasdaq: NBIX) that it has placed a clinical hold on
the RESTORE-1 clinical trial of NBIb-1817 (VY-AADC). As previously
announced, trial sites participating in RESTORE-1 had not been
screening, enrolling or dosing patients as a result of the COVID-19
pandemic and more recently, as a result of the independent Data
Safety Monitoring Board (DSMB)’s request to pause dosing pending
its review of additional data. The DSMB has requested additional
patient level data from the trial and now plans to review these
data in early 2021. The clinical hold follows the submission by
Neurocrine Biosciences of an IND Safety Report related to the
observation of MRI abnormalities in some RESTORE-1 study
participants. The clinical implications of this observation are
currently unknown and are being evaluated.
RESTORE-1 is a Phase 2 clinical trial of NBIb-1817 (VY-AADC), an
intracerebral AAV-based investigational gene therapy, in
development for the treatment of Parkinson’s disease. The RESTORE-1
DSMB has been informed of the clinical hold, as have the study
investigators and central and local ethics committees. Neurocrine
Biosciences and Voyager will work closely with the FDA and the DSMB
to determine the next steps for the RESTORE-1 clinical trial.
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 ten
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 (VY-AADC) 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
(VY-AADC) is administered into the brain using intraoperative
monitoring with magnetic resonance imaging (MRI)-facilitated
targeted delivery.
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 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
Therapeutics, 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, Inc.
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
makes regarding the ability of Neurocrine and Voyager to gather
additional information to further characterize the safety profile
of NBIb-1817 (VY-AADC) and to work with the FDA to determine the
next steps for the RESTORE-1 clinical trial.
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, the ability of Neurocrine and Voyager to
complete their evaluation and to meet the information requests of,
and to resolve questions raised by, the FDA required to bring an
end to the clinical hold on the RESTORE-1 clinical trial. These
statements are also subject to a number of material risks and
uncertainties that are described in Voyager’s 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. 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 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 Information
Voyager Therapeutics
Investors: Investors@voyagertherapeutics.com
Media: Sheryl
Seapy W2Opure949-903-4750sseapy@purecommunications.com
Neurocrine Biosciences (NASDAQ:NBIX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Neurocrine Biosciences (NASDAQ:NBIX)
Historical Stock Chart
From Apr 2023 to Apr 2024