Voyager Therapeutics, Inc. Q2 FY2024 earnings call

At Voyager, leveraging human genetics to develop transformative neurological disease medicines. Made progress in 2024, including dosing first healthy volunteers in VY7523 Phase Ia trial in May; advancing CNS gene therapy pipeline with expected IND filings next year; completed pre-IND meeting and initiated GLP toxicology studies for VY9323; selected development candidate in GBA1 program triggering $3M milestone; appointed Nathan Jorgensen as CFO; team presented data at ASGCT 2024; ended Q2 with $371M cash position with runway through 2027.

Expect IND filings for three CNS gene therapy programs next year. VY7523 Phase Ia trial enrollment on track, topline safety/PK data expected in first half 2025; plan multiple ascending dose study in early Alzheimer's patients next year, initial tau imaging data in H2 2026. VY9323 expects IND filing mid-2025, initiate clinical trial in SOD1-ALS patients post-IND acceptance.

Forward-looking statements subject to risks and uncertainties; actual results may differ materially. Risks discussed in SEC filings, including those related to clinical trial outcomes, regulatory approvals, and market acceptance.

Q: Cadence of gene therapy programs entering clinic in 2025 and timeline for biomarkers in SOD1-ALS program A: Al mentioned wholly-owned SOD1 program expects IND mid-2025; Toby discussed biomarkers like CSF SOD1 and plasma neurofilament Q: Doses explored for VY7523 and tau therapy population A: Todd discussed dosing based on preclinical models to achieve tau spread reduction; Todd and Alfred discussed targeting Alzheimer's patients in specific stages Q: TRACER platform potential for multi-organ specificity A: Todd discussed evaluating capsids for different tissues and disease needs, focusing on detargeting liver and other off-targets Q: Competitive positioning of anti-tau therapy vs Biogen's program and gating factors for SOD1 IND A: Alfred discussed Biogen's BIIB080 program and competitive landscape; Toby discussed GLP toxicology studies as a key gating factor for VY9323 IND Q: Mechanism rationale for liver detargeting of TRACER capsid and ALPL binding A: Todd discussed capsid charge and selection criteria for liver detargeting; Alfred discussed testing proteins, antibodies, enzymes, and oligonucleotides for ALPL binding Q: Pros/cons of targeting extracellular vs intracellular tau and one-and-done tau silencing A: Alfred, Toby, and Todd discussed safety vs efficacy of extracellular targeting; Toby and Todd discussed benign preclinical data for tau knockdown Q: NHP data for siRNA program and combo strategy with antibody A: Todd discussed ongoing experiments for NHP data; Toby discussed prioritizing antibody program if positive Q: Patient selection for GBA1 and Friedreich's ataxia programs and 2024 milestones A: Toby discussed partner programs and not commenting on specific milestones