Voyager Therapeutics, Inc. Q1 FY2023 earnings call

• Neurotherapeutics and Gene Therapy Renaissance: Noted recent approvals in Alzheimer's, Friedreich's ataxia, ALS, and gene therapy advancements, highlighting Voyager's position at the intersection of these fields.
• TRACER Capsid Discovery Platform: Engineered capsid libraries with over 20 novel variants of AAV9 and AAV5 capsids, showing over 100x higher transgene expression in the brain via IV delivery, with plans to share data at ASGCT.
• CNS Pipeline: Advancing four CNS gene therapy programs towards clinical trials, including wholly-owned anti-tau antibody and SOD1 gene therapy, and co-developed GBA1 and frataxin programs with Neurocrine.
• Partnerships: Completed agreements with Pfizer and Novartis, strategic collaborations with Neurocrine, and exploration of additional transactions.
• Pipeline Progress: Selected lead anti-tau antibody, received pre-IND feedback, updated timeline for SOD1 program, launched new Huntington's and Alzheimer's gene therapy programs, and welcomed George Scangos to the board.

In Q1 2023, collaboration revenue was $150.5 million, composed of $69.5 million from the 2023 Neurocrine collaboration agreement for the GBA1 program, $79 million from Novartis option exercises, and $2 million from the 2019 Neurocrine collaboration agreement activities. R&D expense was $18.6 million, an increase compared to Q1 2022. G&A expenses were $9 million. The company had net income of $124 million. Cash, cash equivalents, and marketable securities at the close of March 31, 2023, were $273.3 million, with pro forma totaling $298.3 million at quarter end. Deferred revenue increased by $18.7 million related to Neurocrine's upfront payment and Novartis option exercise recognition.

• Anticipate multiple IND filings across wholly-owned and collaborative programs by 2024-2025, leading to milestone payments and proof-of-concept in CNS indications.
• Potential for early biomarker-based evidence in CNS diseases, with opportunities for value creation through pipeline advancements.

• Dependence on successful clinical trials and regulatory approvals for pipeline programs.
• Challenges in CNS gene therapy delivery despite the TRACER platform.
• Impact of non-human primate availability on preclinical programs, with the field closely monitoring this issue.

Q: Jay Olson asked about optimizing tau therapy development strategy, monotherapy vs combination, and partnering.

A: Dr. Al Sandrock discussed multiple tau targeting approaches, proof-of-concept plans, and considerations for partnering, mentioning multiple approaches against tau, including passive antibody and vectorized knockdown, and weighing independent advancement vs partnering.

Q: Jack Allen inquired about tau therapy population enrollment and competitive landscape.

A: Dr. Al Sandrock and Dr. Todd Carter discussed population enrollment in early-stage Alzheimer's patients and the importance of epitope in tau antibodies, noting differentiated performance of their mid-domain antibodies.

Q: Philip Nadeau asked about tau antibody comparison and GLP tox timeline.

A: Dr. Todd Carter confirmed the tau antibody is a humanized version of prior data, and Dr. Al Sandrock explained the GLP tox timeline is based on FDA feedback and prior experience with humanized antibodies against pathological proteins.

Q: Yanan Zhu asked about tau therapy success criteria and TAU01 candidate delay.

A: Dr. Al Sandrock discussed success criteria based on tau PET scan reduction and payload/vector optimization for TAU01, while Dr. Todd Carter detailed payload optimization for the tau knockdown program.

Q: Laura Chico asked about reimbursement learnings for tau therapy and remaining milestones in 2023.

A: Dr. Al Sandrock discussed reimbursement implications for combination therapies, and Pete Pfreundschuh noted potential near-term milestones but no specific guidance on them.

Q: Joon Lee asked about Huntington's program strategy and tau program species targeting.

A: Dr. Al Sandrock and Dr. Todd Carter explained combining siRNA for Huntington's and the importance of targeting both extracellular and intracellular tau in the tau program.

Q: Sumant Kulkarni asked about preclinical models for SOD1 gene therapy with Tofersen and non-human primate availability.

A: Dr. Al Sandrock discussed preclinical model considerations and Dr. Todd Carter addressed non-human primate availability mitigation efforts.

Q: Jack Allen asked about non-human primate availability impact on preclinical programs.

A: Dr. Todd Carter noted the field is monitoring availability, with Voyager having agreements to mitigate risk.