Voyager Therapeutics, Inc. Q1 FY2024 earnings call

• Alfred Sandrock thanked the Voyager team for advancing genetic medicines, mentioned obtaining IND clearance for VY-TAU01, pipeline progress in gene therapy programs, appointment of Toby Ferguson as CMO, and collaboration with Novartis. Also, highlighted data presentations at AD/PD and ASGCT meetings. • Toby Ferguson discussed wholly owned and partnered programs, focus on tau-targeted programs, and progress in gene therapy programs, including VY-TAU01 and programs for GBA1 and Friedreich's Ataxia. • Todd Carter talked about data on TRACER capsids at ASGCT, manufacturability assessment of gene therapies, and identification of ALPL as a receptor for blood-brain barrier delivery.

No specific financial performance data for product segments provided in the transcript.

• Robust pipeline with 4 wholly owned and 13 partnered programs, with first expected to enter clinical trials soon and potential for 3 more in 2025. • Cash runway extended into 2027 with $200 million from Novartis collaboration and public offering, enabling multiple clinical data readouts. • Expectation of dosing first subject in VY-TAU01 single ascending dose trial in coming weeks and IND filings in 2025 for SOD1-ALS, GBA1, and Friedreich's Ataxia programs.

• Uncertainties in clinical trial outcomes, including whether therapies will meet efficacy endpoints. • Regulatory approval challenges for new genetic medicines. • Competition in the neurogenetic medicine space, which could impact market share.

Q: Mehdi Goudarzi asked about using ALPL receptor knowledge for delivering other modalities to CNS, specifically if antisense oligos and LMPs could see similar transcytosis as AAVs.

A: Al Sandrock and Todd Carter responded that they are looking at conjugating ligands to macro molecules to enable transcytosis of various molecules including proteins, oligos, etc.

Q: Jack Allen inquired about optimizing manufacturing of gene therapies and COGS benefits.

A: Alfred Sandrock and Todd Carter discussed using HEK293 cells for manufacturing, assessing manufacturability during development candidate selection, and considering payload impact on manufacturability.

Q: Patrick Trucchio asked about donanemab advisory committee meeting impact on Alzheimer's programs and tau silencing gene therapy data.

A: Alfred Sandrock mentioned watching the advisory committee for outcome measures, and Todd Carter discussed tau knockdown program data showing mRNA and protein reduction in mouse models.

Q: Philip Nadeau asked about doses for VY-TAU01 trial and frataxin candidate details.

A: Toby Ferguson discussed single ascending dose trial details and Alfred Sandrock mentioned Neurocrine program criteria for development candidates are confidential.

Q: Ry Forseth asked about market opportunity expansion with gen-3 technology and preexisting neutralizing antibodies.

A: Alfred Sandrock and Toby Ferguson talked about modifying capsids to evade preexisting antibodies and using enzymes to lower antibody levels.

Q: David Hoang asked about anti-tau antibody vs tau silencing gene therapy and ALPL receptor expression.

A: Toby Ferguson and Todd Carter explained different mechanisms of action (antibody requires regular infusions, gene therapy is once-and-done) and ALPL is expressed across vasculature with significant cross-species activity.

Q: Chung asked about ALPL expression variability and therapeutic modality development.

A: Todd Carter discussed ALPL expression changes with age and Alfred Sandrock mentioned internal and collaborative efforts for development.

Q: Sumant Kulkarni asked about SOD1-ALS program and NFL reduction relevance.

A: Toby Ferguson and Alfred Sandrock talked about lessons from tofersen, biomarker application, and SMA treatment model relevance.

Q: Yanan Zhu asked about ALPL receptor expression on other tissues and vectorized antibody platform.

A: Todd Carter explained ALPL expression across body but key for brain delivery, and Alfred Sandrock and Todd Carter discussed vectorized antibody potential and preclinical plans.

Q: Laura Chico asked about milestones and strategic focus with VY-TAU01.

A: Robin Swartz mentioned not providing guidance on milestones, and Alfred Sandrock discussed potential partner involvement and vectorization options for VY-TAU01.

Q: Joon Lee asked about vectorized antibody platform expression and preclinical data cadence.

A: Alfred Sandrock and Todd Carter talked about vectorized antibody expression, constitutive expression in CNS, and interim data release plans.