Voyager Therapeutics Decides to Assess Alternate Payloads for SOD1 ALS Program

On February 11, 2025, Voyager Therapeutics, Inc. (NASDAQ: VYGR) announced through an 8-K filing that it has chosen to evaluate alternative payloads related to its superoxide dismutase 1 silencing gene therapy program for amyotrophic lateral sclerosis (ALS), known as the SOD1 ALS Program. According to the statement, recent three-month non-human primate (NHP) data has indicated that an alternate payload may be required to meet the company’s desired product specifications for the SOD1 ALS Program.

The filing also noted that there are no planned modifications to the novel capsid component of VY9323, which was previously the leading development candidate for the SOD1 ALS Program. Consequently, there are no expectations for the Company to submit an investigational new drug (IND) application or a Canadian clinical trial application for VY9323 by mid-2025 due to this decision. It was clarified that this determination has no bearing on the company’s other gene therapy initiatives.

Despite the adjustments to the SOD1 ALS Program, Voyager Therapeutics anticipates Neurocrine Biosciences, Inc. (Neurocrine) to file INDs in 2025 for the glucocerebrosidase 1 gene therapy for Parkinson’s disease and other GBA1-mediated disorders, along with the gene therapy program for Friedreich’s ataxia. Furthermore, an IND is expected in 2026 for VY1706, the primary development candidate of its tau silencing gene therapy program, based on positive results from preclinical studies similar to those conducted for VY9323.

Regarding financial matters, based on its current operating plan, the Company estimates that its existing cash reserves, alongside anticipated reimbursements from collaborations with Neurocrine and Novartis Pharma AG, and interest income, will be adequate to support operational expenses and capital needs until mid-2027. However, the Company acknowledges that the timing of these assumptions may vary, potentially leading to the exhaustion of capital resources earlier than expected.

The filing, in compliance with the safe harbor provisions of securities laws, recognized the inherent unpredictability associated with forward-looking statements. The Company’s management, although optimistic, emphasizes that actual results may deviate from projections due to various risks and uncertainties. Voyager Therapeutics is consistently cautious, as demonstrated in the document, and vows to promptly communicate any updates or changes as necessary, aligning with legal stipulations.

The report concludes with the formal signature of Alfred Sandrock, M.D., Ph.D., the Chief Executive Officer, President, and Director of Voyager Therapeutics, verifying the authenticity of the report filed with the Securities and Exchange Commission.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Voyager Therapeutics’s 8K filing here.

Voyager Therapeutics Company Profile

Voyager Therapeutics, Inc, a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial.