Release Date: August 06, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Voyager Therapeutics Inc (VYGR, Financial) dosed the first healthy volunteers in the Phase 1a single ascending dose trial of VY7523, their anti-tau antibody for Alzheimer's disease.
- The company completed a pre-IND meeting with the FDA and initiated GLP toxicology studies for VY9323, their SOD1 silencing gene therapy program for ALS.
- Voyager Therapeutics Inc (VYGR) selected a development candidate in their GBA1 gene therapy program partnered with Neurocrine, triggering a $3 million milestone payment.
- The company presented significant data at ASGCT 2024, showcasing their second-generation TRACER capsids and their potential in CNS gene therapy.
- Voyager Therapeutics Inc (VYGR) ended the second quarter with a strong cash position of approximately $371 million, providing runway through multiple clinical data readouts into 2027.
Negative Points
- The company’s tau silencing gene therapy program is still in preclinical stages, with an IND filing not expected until 2026, indicating a longer timeline to potential market entry.
- There are uncertainties regarding the efficacy and safety of their anti-tau antibody, VY7523, as previous N-terminal directed antibodies have been unsuccessful in clinical trials.
- The competitive landscape for anti-tau therapies is crowded, with multiple companies pursuing similar approaches, potentially impacting market share and differentiation.
- The success of their gene therapy programs is contingent on overcoming significant delivery challenges posed by the blood-brain barrier, which remains a high-risk factor.
- Voyager Therapeutics Inc (VYGR) relies heavily on partnerships for several of their programs, which may limit their control over development timelines and strategic decisions.
Q & A Highlights
Q: Can you discuss the cadence of the three gene therapy programs expected to enter the clinic in 2025?
A: The SOD1 gene therapy program is expected to file an IND in mid-2025. The other two programs are partnered, and while they are also expected to file INDs in 2025, the exact timing is less certain. (Alfred Sandrock, CEO)
Q: How quickly can we see early indications of efficacy from the SOD1-ALS gene therapy?
A: Changes in neurofilament and CSF SOD1 levels can be observed as early as 4 weeks, with more definitive changes by 12 weeks. (Toby Ferguson, CMO)
Q: How many dose levels are being explored for VY7523, and how many doses are needed to hit an active dose?
A: Multiple doses are being explored in the single-ascending dose trial. Based on preclinical models, we believe we understand the exposures needed to inhibit tau spread. (Toby Ferguson, CMO; Todd Carter, CSO)
Q: Are the two tau-targeting programs aimed at the same Alzheimer's population, or do they target different subpopulations?
A: The tau antibody program targets early-stage patients where tau spread has just started. The tau silencing gene therapy will likely follow the BIIB080 program model, targeting a broader population. (Alfred Sandrock, CEO; Toby Ferguson, CMO)
Q: Can you discuss the potential of the TRACER platform to yield capsids with multi-organ specificity?
A: TRACER-derived capsids can potentially target multiple tissues, including the brain and heart, which is beneficial for diseases like Friedreich's ataxia and DM1. (Alfred Sandrock, CEO; Todd Carter, CSO)
Q: What is the competitive positioning of your anti-tau therapies versus Biogen's program?
A: Biogen's BIIB080 program is ahead in clinical development. Our anti-tau antibody targets a different epitope, and our gene therapy approach offers a one-time IV administration. (Alfred Sandrock, CEO; Toby Ferguson, CMO)
Q: What are the remaining gating factors for the SOD1 silencing gene therapy to reach IND filing?
A: The main next step is completing the toxicology programs. (Toby Ferguson, CMO)
Q: What are the pros and cons of targeting extracellular versus intracellular tau?
A: Targeting extracellular tau is likely safer but may be less effective. Knockdown approaches affect all forms of tau, which could be more effective but come with higher safety risks. (Alfred Sandrock, CEO; Toby Ferguson, CMO; Todd Carter, CSO)
Q: When can we expect NHP data for the siRNA program, and what are your thoughts on a combo strategy with the antibody?
A: We have ongoing experiments but no specific timeline for NHP data. If the antibody program shows positive data, we would move forward with it first. (Todd Carter, CSO; Toby Ferguson, CMO)
Q: What type of patients will you target for the GBA1 and Friedreich's ataxia programs, and what milestones should we watch for in 2024?
A: These are partnered programs, and patient selection will follow established paths. We do not comment on specific milestones. (Toby Ferguson, CMO; Alfred Sandrock, CEO)
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
