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Voyager Therapeutics Provides Update on NBIb-1817 (VY-AADC) Gene Therapy Program
Voyager’s understanding is that Neurocrine’s decision to terminate the NBIb-1817 (VY-AADC) program was based on a portfolio review and prioritization of its current pipeline assets. Voyager plans to support Neurocrine, the IND holder and sponsor of the RESTORE-1 Phase 2 clinical trial, on any ongoing matters related to additional imaging and clinical assessments requested by the Data Safety & Monitoring Board (DSMB) and other information that may be requested by the
Voyager is evaluating the complete financial impact of the termination and the future of the Parkinson’s program and expects to provide a subsequent update.
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.
Voyager Therapeutics® is a registered trademark of
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 progress, activities, goals and reporting of results of its clinical trials, the potential benefits and future operation of its collaboration with Neurocrine and the activities thereunder, Voyager’s ability to perform its obligations under the collaboration agreement with Neurocrine, 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, the regulatory pathway of, and the timing or likelihood of its regulatory filings and approvals for, any of Voyager’s product candidates, and its anticipated financial results, including the financial impact of the termination of the NBIb-1817 (VY-AADC) program, 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 and its TRACER system; Voyager’s scientific approach and general development progress; the sufficiency of its cash resources; the availability or commercial potential of Voyager’s product candidates; and 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
Source: Voyager Therapeutics, Inc.