Voyager Therapeutics (VYGR) Oppenheimer 36th Annual Healthcare Life Sciences Conference summary

Strategic pillars and financial position

• Focused on three pillars: tau-targeting therapies, gene therapy assets entering the clinic, and the NeuroShuttle platform for brain drug delivery.

• Two gene therapy programs, including a partnered Neurocrine asset and a wholly owned program, are set to enter clinical trials this year.

• NeuroShuttle platform aims to deliver diverse drugs into the brain using novel receptor-based shuttles.

• Well-capitalized with cash runway into 2028.

Tau-targeting programs and industry context

• Tau is a validated target in Alzheimer's, with its spread correlating more closely with clinical decline than amyloid.

• Recent trials (e.g., UCB's bepranemab) showed mixed results, highlighting the importance of epitope selection for antibody efficacy.

• VY7523, an anti-tau antibody, completed SAD and MAD enrollment, showing favorable safety and pharmacokinetics.

• MAD study is powered to detect effects on tau PET imaging, the primary biomarker for efficacy.

• Plans to seek a partner for Phase 3 and possibly Phase 2 due to the scale of Alzheimer's trials.

Gene therapy and tau silencing advancements

• VY1706 gene therapy targets 50–70% tau knockdown at low doses, with improved safety by reducing liver exposure.

• IND filing for VY1706 expected in Q2, with first patient dosing in H2 this year.

• VY1706 offers broad CNS distribution via IV, unlike intrathecal antisense approaches, and will use tau PET imaging as a key endpoint.

• BIIB080’s strong clinical and biomarker effects set a benchmark for tau silencing therapies.