• Cash: $332 million as of the end of 2024.
• Potential Future Milestone Payments: $8.2 billion from partnerships.

Release Date: March 11, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Voyager Therapeutics Inc (NASDAQ:VYGR) has a robust pipeline with 4 wholly owned and 13 partnered programs, providing multiple opportunities for clinical data generation.
• The company reported $332 million in cash at the end of 2024, with potential future milestone payments totaling $8.2 billion, indicating strong financial health.
• Voyager's partnerships have been a significant source of non-diluted revenue, contributing to its financial stability.
• The tau silencing gene therapy program, VY1706, has advanced into IND enabling studies, showing promising data with 50% to 73% knockdown of tau messenger RNA in non-human primate studies.
• Voyager's anti-tau antibody, VY7523, performed well in a single ascending dose study, showing no serious adverse events and consistent pharmacokinetics with approved Alzheimer's treatments.

Negative Points

• The SOD1 silencing gene therapy program has moved back into the research stage due to the payload not meeting the target profile, requiring a new payload to be identified.
• Despite progress, the field of gene therapy continues to face setbacks, which could impact Voyager's pipeline and commercial viability.
• The company's reliance on partnerships for revenue could pose a risk if future collaborations do not materialize as expected.
• Voyager's tau silencing program is still in preclinical stages, with IND filing not expected until 2026, indicating a long timeline before potential market entry.
• The competitive landscape in the tau space is intense, with multiple external readouts expected, which could impact Voyager's positioning and strategy.

Q & A Highlights

• Warning! GuruFocus has detected 5 Warning Signs with VYGR.

Q: Can you provide additional insights into the upcoming tau-related data expected at the AD/PD conference? A: Alfred Sandrock, CEO, mentioned that they expect to see data from bepranemab, focusing on exposure PD relationships and the decrease in tau spreading necessary for a clinically relevant effect. They are also interested in subgroup efficacy, such as the impact of ApoE e4 carrier status and initial tau burden. Other companies, like ASI, are sharing data on fluid-based biomarkers, and Biogen's tau-silencing approaches are also of interest.

Q: Regarding the financial outlook, how do upcoming milestones from partnerships like Neurocrine impact your cash runway? A: Nathan Jorgensen, CFO, stated that there are $2.9 billion in developmental milestones, with total potential biobucks exceeding $8 billion. These milestones are not included in the current cash runway guidance, which extends to mid-2027, representing potential upside.

Q: What are the key steps needed before filing the IND for the tau gene silencing program? A: Alfred Sandrock, CEO, explained that completing the GLP tox study and ensuring a therapeutic window are crucial. The initial non-human primate study showed 50% to 73% knockdown, which is within the desired range, but safety must be confirmed to allow for the right dosing.

Q: How does the ALPL shuttle compare to other blood-brain barrier shuttles, and what are its potential applications? A: Alfred Sandrock, CEO, noted that the ALPL shuttle allows for broad CNS delivery, potentially transporting proteins, enzymes, antibodies, and oligonucleotides. It offers a wide array of possibilities for treating various diseases, including spinal cord and cerebral cortex conditions.

Q: How do you differentiate between tau silencing and targeting antibodies, and what are the potential benefits of each approach? A: Toby Ferguson, CMO, highlighted that tau silencing offers broader bio-distribution and a one-time IV administration, potentially benefiting patients and healthcare systems. The antibody approach aims to impede tau spread, targeting populations where tau has not yet spread. Both approaches have unique advantages and may be used in combination or sequence.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.