Voyager Shifts Focus In Gene Therapy For Amyotrophic Lateral Sclerosis, Maintains Strong Financial Outlook

Voyager Therapeutics Inc., a biotechnology company, announced the decision made to investigate different payload options within its superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) gene therapy program. Unprocessed data from three-month non-human primate trials indicate the need for a different payload to fulfil the required product performance. The company will maintain the current design of the novel capsid component. Three-month NHP testing of the same capsid delivered targeted performance results while showing good tolerance in the VY1706 (tau silencing) gene therapy program. Voyager has stopped planning to submit VY9323 for investigational new drug filing during the mid-2025 timeframe. Voyager now estimates its financial resources will last until mid-2027 without considering any possible payments that could come from existing alliance collaborations.

Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager, commented, “Emerging preclinical data indicate the siRNA payload component of VY9323 does not meet our high standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window. While we are disappointed that the development candidate VY9323 will not advance, we hope that we may be able to identify an alternate payload and find a path forward for this program, given the unmet need in ALS. We are encouraged that our novel TRACER capsids continue to perform consistently across multiple programs, and we believe they have the potential to transform gene therapy for CNS diseases. We continue to expect IND filings in 2025 for our gene therapy candidates for GBA1 and FA and in 2026 for VY1706.”

Despite this shift, Voyager’s other gene therapy programs remain on track. IND filings are still expected in 2025 through its partnership with Neurocrine Biosciences for GBA1-related Parkinson’s disease and other GBA1-mediated conditions, along with a program targeting Friedreich’s ataxia. Voyager also plans to submit an IND for VY1706 in 2026. Financially, Voyager’s cash runway now extends into mid-2027, excluding any potential milestone payments from current partnerships. The company will present additional updates at the Oppenheimer 25ᵗʰ Annual Healthcare Life Sciences Conference today at 2:40 p.m. ET.