Voyager Therapeutics Reports Robust Preclinical Activity in Tau Silencing Gene Therapy Program for Alzheimer’s Disease; Advances Program Into Late Research

Voyager Therapeutics, Inc. recently announced new data from its two preclinical programs targeting pathological tau for the treatment of Alzheimer’s disease. Data on VY-TAU01, Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau silencing gene therapy program will be presented at the upcoming 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD 2024), taking place March 5-9, 2024, in Lisbon, Portugal.

The data demonstrate that a single intravenous (IV) administration of one of Voyager’s tau silencing gene therapy candidates in mice expressing human tau resulted in broad AAV distribution across multiple brain regions and dose-dependent reductions in tau messenger RNA (mRNA) levels of up to 90%, which were associated with robust reductions in human tau protein levels across the brain. Voyager’s tau silencing gene therapy program combines vectorized tau-targeted siRNA with a proprietary, blood-brain barrier (BBB)-penetrant capsid derived from the Company’s TRACER™ discovery platform. Based on these data, Voyager has advanced this program into late research and expects to file an investigational new drug (IND) application in 2026.

Voyager also will report new preclinical data demonstrating that VY-TAU01, the company’s lead antibody targeting pathological tau, and Ab-01, its murine surrogate, were well-tolerated and showed favorable pharmacokinetic profiles following IV administration in NHPs and P301S transgenic mice expressing pathological human tau. Voyager continues to anticipate an IND filing for VY-TAU01 in the first half of 2024.

“The Voyager team is increasingly excited about the potential for treatments targeting pathological tau to play an important role in improving clinical outcomes for patients suffering with Alzheimer’s disease,” said Todd Carter, PhD, Chief Scientific Officer of Voyager Therapeutics. “Recent third-party data with tau-targeted investigational therapies have demonstrated reduced spread of pathological tau, as measured by tau PET imaging, and favorable trends in cognition. We look forward to continuing to advance our two programs targeting tau, and observing anticipated data read-outs from others in this space that will play an important role in further validating this target.”

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’s TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs). In preclinical studies, TRACER-generated capsids have demonstrated widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach, while de-targeting the liver and dorsal root ganglia. As part of its external partnership strategy, Voyager has established multiple collaboration agreements providing access to its next-generation TRACER capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.

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.