Voyager Therapeutics (VYGR) TD Cowen 45th Annual Healthcare Conference summary

Key program updates

• Anti-tau antibody program completed a single ascending dose study in healthy volunteers with no safety events and initiated a multiple ascending dose study in early Alzheimer's patients, aiming for a tau PET readout in H2 2026.

• The anti-tau antibody demonstrated strong CSF penetration and preclinical efficacy in inhibiting tau spread, with plans to present data at a medical meeting in H2 2024.

• siRNA-mediated tau silencing program (VY1706) targets tau knockdown with an IND planned for 2026; preclinical studies showed no safety concerns with the capsid used.

• SOD1 program is paused pending identification of a suitable payload, with no set timeline for resumption.

Strategic direction and partnerships

• Transitioned from gene therapy-only to a multimodality neurotherapeutics approach, leveraging novel capsids that cross the blood-brain barrier and enable partnerships.

• Partnerships, especially those involving capsid licensing and co-development, have been a major driver of shareholder value and provide non-dilutive funding.

• Two partnered programs with Neurocrine (Friedreich's ataxia and GBA) are expected to file INDs in 2024, with opt-in rights for significant ownership after phase 1 data.

• Capsid engineering efforts focus on discovering new receptors (e.g., ALPL) and expanding delivery modalities, with ongoing discussions for LNP partnerships.

Clinical and development insights

• Multiple-ascending dose study in Alzheimer's will focus on safety and tau PET as a key biomarker, with learnings from recent anti-tau antibody trials informing design.

• Patient selection for trials may favor those with lower tau burden, as evidence suggests greater potential for biologic or clinical effects in this group.

• For gene therapy programs in rare diseases, biomarker-based endpoints may suffice for approval, while more common indications like Parkinson's will require larger trials.