Voyager says to increase size of RESTORE-1 Phase 2 trial following FDA feedback

Voyager Therapeutics announced an update to its VY-AADC clinical program for Parkinson’s disease. In December 2018, the company held a Type B meeting with the U.S. FDA to discuss the overall development program for VY-AADC. Based on the meeting discussion and subsequent written feedback from the FDA, Voyager plans to submit a revised trial protocol that will include an increase in the target number of patients in the RESTORE-1 Phase 2 trial, resulting in 75 to 100 total patients in the trial, and to conduct a staggered-parallel Phase 3 trial of similar size and design to RESTORE-1. These updates incorporate guidance from the FDA from the Type B meeting to conduct two adequate and well-controlled clinical trials for a large patient population such as Parkinson’s disease. “Our recent meeting with the FDA was informative and helps to clarify the expected regulatory pathway for VY-AADC,” said Andre Turenne, president and CEO of Voyager Therapeutics. “We look forward to continuing to engage with the FDA and other regulators as we advance our clinical development program and our work to bring VY-AADC to patients in need.” The RESTORE-1 Phase 2 trial is currently enrolling patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary. The primary efficacy endpoint of RESTORE-1 is ON time without troublesome dyskinesia, or good ON time, as measured by a validated self-reported patient diary at 12 months. Voyager expects RESTORE-1 will take approximately 15 to 21 months to enroll. Voyager plans to begin enrolling patients in RESTORE-2 in both active Phase 2 sites and additional sites globally in the first half of 2020. Voyager anticipates that, if positive, results from RESTORE-1 and RESTORE-2 could potentially form the basis for submission of a biologics license application to the FDA for VY-AADC for the treatment of Parkinson’s disease.

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