Voyager Therapeutics Begins Dosing in VY-TAU01 Alzheimer's Trial

Voyager Therapeutics, Inc., a biotechnology firm committed to advancing neurogenetic medicines, has announced the commencement of a Phase 1a single ascending dose (SAD) trial for VY-TAU01. This investigational anti-tau antibody is designed to inhibit the spread of pathological tau proteins in Alzheimer’s disease. The trial aims to evaluate the safety and pharmacokinetics of VY-TAU01 among healthy adult volunteers. The study, which is randomized, double-blind, and placebo-controlled, is being conducted at a single location in the United States and plans to enroll around 48 participants across multiple cohorts.

The data obtained from this SAD trial will inform the design of a subsequent Phase 1b multiple ascending dose (MAD) trial. Voyager Therapeutics expects to initiate the MAD trial in 2025, focusing on patients with early-stage Alzheimer’s disease. Preliminary tau PET imaging data from the MAD trial is anticipated in the latter half of 2026, potentially indicating whether VY-TAU01 can slow down the progression of pathological tau in the brain.

The initiation of the VY-TAU01 clinical development is a significant milestone for Voyager Therapeutics. Toby Ferguson, M.D., Ph.D., Chief Medical Officer of Voyager, emphasized the importance of this development, highlighting the company's commitment to advancing treatments for neurological diseases. Ferguson noted the considerable unmet need within the Alzheimer's disease patient community, despite recent progress in the field. He expressed optimism about the preclinical data, which showed that VY-TAU01 could significantly slow the spread of tau proteins, and looked forward to assessing its therapeutic potential in clinical settings.

VY-TAU01 is administered intravenously and is a recombinant, humanized IgG4 monoclonal antibody. It targets a specific C-terminal epitope of tau, differing from previous N-terminal directed anti-tau antibodies, which showed no efficacy in clinical trials. Preclinical models have demonstrated VY-TAU01's robust ability to inhibit the spread of pathological tau and its favorable pharmacokinetic profile.

Alzheimer’s disease is a progressive neurodegenerative condition affecting an estimated 6 million individuals in the United States and up to 416 million globally. The disease leads to memory loss and can progress to significant impairments in independence, communication, and physical control, often accompanied by behavioral disorders such as paranoia and anxiety. The financial burden of caring for individuals with Alzheimer’s and other dementias in the U.S. was estimated at $345 billion in 2023.

Voyager Therapeutics, Inc., listed on Nasdaq under the ticker VYGR, harnesses human genetics to modify the course of neurological diseases. Their pipeline includes programs targeting Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, among other central nervous system disorders. Voyager's TRACER™ AAV capsid discovery platform is central to their research, enabling the development of novel capsids with high brain penetration potential following intravenous dosing. Their programs are a mix of wholly-owned initiatives and collaborations with partners such as Alexion, AstraZeneca Rare Disease, Novartis Pharma AG, Neurocrine Biosciences, Inc., and Sangamo Therapeutics, Inc.

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