Parkinson Disease Gene Therapy Program Loses Funding

A study of gene therapy VY-AADC for Parkinson disease (PD) (NCT01973543) being done by Voyager (Cambridge, MA) will no longer be funded by their partner Neurocrine (City, ST), although Neurocrine will continue partnering with them in other areas. This is because of the study being put on hold by the FDA for observed adverse events. Voyager has not determined whether or not they will try to continue the study.

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 FDA.  

In December 2020, Voyager announced that the FDA had notified Neurocrine that it had placed a clinical hold on the RESTORE-1 clinical trial of NBIb-1817 (VY-AADC). The FDA notification followed a request by the study’s independent DSMB for a pause in dosing pending the receipt of information about MRI 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. Information required by the FDA 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 PD gene therapy program and expects to provide a subsequent update.
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 MRI-facilitated targeted delivery.