Voyager Therapeutics Announces First Participants Dosed in Single Ascending Dose Trial of VY-TAU01 for the Treatment of Alzheimer’s Disease

Voyager Therapeutics, Inc. recently announced that the first participants were dosed in a Phase 1a single ascending dose (SAD) trial of VY-TAU01, an investigational anti-tau antibody developed to inhibit the spread of pathological tau in Alzheimer’s disease.

The objective of the randomized, double-blind, placebo-controlled, SAD trial is to evaluate the safety and pharmacokinetics of VY-TAU01 in healthy adult volunteers. The study is being conducted at a single site in the US and is expected to enroll approximately 48 patients in multiple cohorts. Voyager anticipates that the data from the SAD trial will inform the design of a Phase 1b multiple ascending dose (MAD) trial in patients with early Alzheimer’s disease, which Voyager expects to initiate in 2025. The MAD study has the potential to generate initial tau PET imaging data in the second half of 2026, which may indicate if VY-TAU01 can slow the spread of pathological tau in the brain.

“The initiation of clinical development of VY-TAU01 for the treatment of Alzheimer’s disease is an important milestone for Voyager; it demonstrates the executional abilities of our neurology drug development team, which will be central to our advancement of three wholly-owned and partnered neurology gene therapies towards IND filings next year,” said Toby Ferguson, MD, PhD, Chief Medical Officer of Voyager Therapeutics. “Alzheimer’s disease remains an area with tremendous unmet patient need, despite recent advances. We are encouraged by our preclinical data demonstrating the ability of VY-TAU01 to significantly slow tau spreading, and we look forward to evaluating the therapeutic potential of VY-TAU01 in the clinic.”

VY-TAU01 is an IV-administered, recombinant, humanized IgG4 monoclonal antibody developed to inhibit the spread of pathological tau, which is closely correlated with disease progression and cognitive decline in Alzheimer’s disease. In contrast to previous N-terminal directed anti-tau antibodies that did not show efficacy in clinical studies, VY-TAU01 targets a distinct C-terminal epitope of tau and has demonstrated robust in vivo inhibition of the spread of pathological tau in a preclinical model. Additional preclinical studies have demonstrated that VY-TAU01 was well-tolerated and demonstrated a favorable pharmacokinetic profile following IV administration.

Alzheimer’s disease is a progressive neurodegenerative disease estimated to affect 6 million people in the US and up to 416 million people globally. The disease causes memory loss and may escalate to decreased independence, communication challenges, behavioral disorders such as paranoia and anxiety, and lack of physical control. In 2023, the total cost of caring for people living with Alzheimer’s and other dementias in the U.S. is estimated at $345 billion.

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com.