Voyager Therapeutics (VYGR) Wells Fargo 21st Annual Healthcare Conference summary

Key program updates and clinical milestones

• Multiple tau-targeting programs are advancing, including an antibody (VY7523) with Q4 data expected and a gene therapy (VY1706) using a novel BBB-penetrant capsid entering the clinic this year.

• Acute safety data for VY1706 is anticipated in Q1 next year, with CSF tau data later in the year and gene expression confirmation by year-end; tau PET imaging data will follow after 2027.

• The antibody program will benchmark against bepranemab, with a go/no-go decision based on tau PET imaging efficacy; partnering is planned if results are positive.

• Neurocrine and Novartis are also advancing programs using the company’s capsids, with clinical data expected next year, providing proof of concept for the platform.

• The NeuroShuttle platform, leveraging novel BBB receptors like ALPL, will present new animal data and safety results at a December meeting, with ongoing business development discussions.

Industry insights and competitive landscape

• Recent data from BIIB080 and Novartis validate tau as a target in Alzheimer’s, showing strong cognitive efficacy but some safety concerns with intrathecal ASOs.

• Antibody approaches targeting tau have mixed results; the company’s candidate was selected for robust efficacy in preclinical models and will be benchmarked against bepranemab.

• BBB-penetrant capsids are seen as a major innovation, with both internal and partner programs racing to provide first-in-human proof of concept.

• The NeuroShuttle platform aims to differentiate from transferrin receptor-based shuttles, focusing on safety and versatility for delivering various modalities to the brain.

• Multiple partnerships are in place, including with Neurocrine, Novartis, and Alexion, with expectations of increased interest if clinical safety and efficacy are demonstrated.

Forward-looking statements and strategic plans

• Acute safety and gene expression data for VY1706 are expected by the end of next year, with tau PET imaging data to follow in 2027 or later.

• Both antibody and gene therapy assets are intended for partnering after proof of concept is established, given the scale of Alzheimer’s disease.

• The company is open to business development for the NeuroShuttle platform, aiming for non-dilutive revenue and broader CNS applications.

• If both antibody and gene therapy approaches prove effective, combination strategies may be considered, similar to treatment paradigms in SMA.

• Ongoing animal and safety data from the NeuroShuttle program will inform future clinical development and partnership opportunities.