RARE Daily

Neurocrine Terminates Parkinson’s Gene Therapy Collaboration with Voyager Therapeutics

February 3, 2021

Rare Daily Staff

Voyager Therapeutics said Neurocrine Biosciences has terminated the Parkinson’s disease gene therapy portion of their collaboration agreement, effective August 2, 2021.

The Parkinson’s program was the focus of their potential $1.8 billion deal signed two years ago. However, the Friedreich’s ataxia program and two discovery programs that are also part of the agreement are not impacted and remain under active collaboration.

Voyager said 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 and other information that may be requested by the U.S. Food and Drug Administration.

NBIb-1817 (VY-AADC) is an experimental 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 MRI-facilitated targeted delivery.

In December 2020, the FDA notified Neurocrine that it had placed a clinical hold on the RESTORE-1 clinical trial of NBIb-1817. The FDA notification followed a request by the study’s independent DSMB for a pause in dosing pending the receipt of information about magnetic resonance imaging abnormalities observed in trial participants. In January 2021, the FDA informed Neurocrine of the information required to provide a complete response to the FDA in connection with the clinical hold, which includes an assessment of how the investigational product may have given rise to the adverse findings, a mitigation plan to manage the adverse findings, and supportive data to justify that a favorable benefit/risk profile remains for the product.

Voyager is evaluating the complete financial impact of the termination and the future of the Parkinson’s program, its only clinical asset, and expects to provide a subsequent update.

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