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

Key program updates and strategic initiatives

• Three major catalysts this year: tau-targeted therapies, capsid proof-of-concept (POC), and NeuroShuttle platform advancements.

• Tau programs include an antibody (VY7523) with Q4 data expected and a gene therapy (VY1706) using a BBB-penetrant capsid, with first patient enrollment this year and acute safety data in Q1 next year.

• Neurocrine, a partner, is also entering the clinic with a capsid-based program for Friedreich's ataxia, with data expected next year.

• NeuroShuttle leverages novel BBB receptors (notably ALPL) to deliver diverse modalities to the brain, with animal and safety data to be presented in December.

• Multiple partnerships are in place, including with Neurocrine, Novartis, and Alexion, with ongoing business development for further collaborations.

Tau program insights and industry context

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

• J&J's mid-domain tau antibody failed, while bepranemab showed efficacy in slowing tau spread; these serve as benchmarks for ongoing antibody development.

• The VY7523 antibody program aims to match or exceed bepranemab's efficacy; if not, the program will be discontinued.

• VY1706 gene therapy uses a potent, liver-de-targeted capsid, aiming for 60-70% tau knockdown in NHPs, with CSF tau data expected next year and PET imaging data later.

• Both antibody and gene therapy assets are intended for partnership after proof-of-concept data is generated.

Clinical development and future milestones

• VY7523 multiple ascending dose study will benchmark tau PET imaging against bepranemab, with a go/no-go decision based on results.

• VY1706 will provide acute safety data in Q1 next year and CSF tau data later in the year; PET imaging data will follow after 12-18 months.

• External safety monitoring committees will oversee dose escalation in clinical trials.

• NeuroShuttle platform will present new animal pharmacology and safety data in December, focusing on ALPL and other undisclosed receptors.

• Ongoing and future partnerships are expected to accelerate clinical translation and expand CNS applications.