Voyager Therapeutics (VYGR) investor relations material

Company overview and platforms

• Focuses on multimodality neurotherapeutics, optimizing delivery across gene therapy and shuttle platforms.

• Gene therapy platform discovers capsids that cross the blood-brain barrier (BBB) after IV delivery, identifying both capsids and their target receptors.

• ALPL is the first validated receptor used for shuttling therapeutics across the BBB.

• Emphasis on developing ligands and conjugates for improved delivery of oligonucleotides and protein therapeutics.

• Partnerships with Neurocrine, Novartis, and AstraZeneca support multiple programs.

Alzheimer's disease and Tau programs

• Heavy focus on Alzheimer's, with a phase 1 anti-Tau antibody program in a multiple ascending dose study, readout expected next year.

• Other industry programs (J&J, Biogen) in Tau expected to read out in early to mid-next year, potentially providing read-through for their own programs.

• Their antibody showed 70% efficacy in blocking Tau spread in a humanized mouse model, compared to zero effect from failed N-terminal antibodies.

• Chose an antibody specific for pathological Tau, using empirical animal models to select candidates.

• Tau PET imaging prioritized over fluid biomarkers for clinical development due to better correlation with disease progression.

Clinical development and strategy

• Phase 1 trial is powered to detect effects on Tau spread using Tau PET imaging, with the goal to impede progression between Braak stages.

• Plans to seek a partner for phase 3 if phase 1 results are positive, as commercialization is not feasible independently.

• Tau silencing gene therapy (vectorized siRNA) is expected to enter the clinic next year, with strong read-through from positive results in similar Biogen programs.

• Uses low-dose, liver-detargeted capsids for gene therapy, achieving 50-80% knockdown across the brain in non-human primates.

• Safety considerations include minimal on-target risk for Tau knockdown, supported by knockout animal studies and ongoing monitoring in human trials.