Voyager Therapeutics, Inc. Q4 FY2023 earnings call

• Defined as an emerging leader in neurogenetic medicine, with pipeline anticipating at least four wholly owned and partnered CNS programs in clinic by end 2025, including anti-tau antibody for Alzheimer's and SOD1 gene therapy for ALS.
• TRACER capsid discovery platform for CNS gene therapy delivery, demonstrating high transduction, liver detargeting, blood brain barrier penetrance.
• Partnerships: Received $100M from Novartis in Jan 2024 for Huntington's and SMA, total partnered programs 13 with potential $8.2B in milestones; triggered $5M milestone with Neurocrine for Friedreich's ataxia.
• Strong balance sheet with ~$431M cash as of Dec 31, 2023, runway into 2027.
• Progress on anti-tau antibody VY-TAU01: IND filing expected in first half 2023, plans for clinical studies in healthy volunteers and early Alzheimer's patients, aiming for proof of concept data in 2026 via PET imaging.
• Tau silencing gene therapy: Single IV administration in humanized tau mouse model showed 90% reduction in tau mRNA, presented at AD/PD conference, IND filing anticipated in 2026.
• SOD1 ALS gene therapy: Development candidate selected, non-human primate data showed 73-82% reduction in SOD1 mRNA in spinal cord, progressing to IND filing in mid-2025.

No detailed financial performance by product segments provided in the transcript.

• Anticipate at least four wholly owned and partnered CNS programs in clinic by end of 2025.
• Aim to generate clinical data in 2025 and 2026.
• Cash runway into 2027, enabling potential value-creating clinical data.
• Plan to initiate single ascending dose study of VY-TAU01 in healthy volunteers in 2023, multiple ascending dose study in early Alzheimer's patients in 2024, and proof of concept data via PET imaging in 2026 for tau program.
• SOD1 ALS gene therapy progressing to IND filing in mid-2025.

• Forward-looking statements are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ.
• Risks associated with clinical trial outcomes, regulatory approvals, and the success of pipeline programs.
• Uncertainties in translating preclinical results to clinical efficacy.

Q: How to think about vector selection across assets, preclinical model predictability?

A: Use TRACER capsid profiles based on disease and target, validate targets with human genetics/clinical trials, use non-human primate pharmacology for predictability.

Q: Collaboration revenues for 2024?

A: No specific guidance, but existing collaborations advancing, potential further revenue from capsid platform and milestones.

Q: TAU01 program and tau targeting strategy?

A: Epitope matters, chose C-terminal based on animal spreading assay, positive results underscore tau as target, negative results may be due to wrong epitope.

Q: FDA biomarkers for gene therapy approval?

A: Appreciate use of surrogate markers for accelerated approval, valid approach for diseases with severe outcomes.

Q: SOD1 program GLP tox timeline, TAU01 Phase 1b PD markers?

A: GLP tox on schedule, Phase 1a focuses on safety/PK, Phase 1b uses tau PET imaging and other biomarkers.

Q: Tau silencing candidate vector choice, C-terminal antibody uniqueness?

A: Chose vectorized siRNA for catalytic knockdown, C-terminal antibodies not unique but selected via in vivo spreading assay.

Q: GLP tox on anti-tau antibody?

A: Nuances in running studies due to targeting human pathological tau, on track to complete in first quarter, IND filing in first half 2023.