Release Date: May 13, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Voyager Therapeutics Inc (VYGR, Financial) announced IND clearance for VY-TAU01, an anti-tau antibody for Alzheimer's, with upcoming dosing in a trial.
- The company has expanded its collaboration with Novartis, enhancing its financial position with $200 million in total consideration, extending its cash runway into 2027.
- Voyager Therapeutics Inc (VYGR) presented robust data at AD/PD and ASGCT meetings, demonstrating significant progress in neurogenetic medicine.
- The company has a strong pipeline with 4 wholly owned and 13 partnered programs, with multiple expected to enter clinical trials soon.
- Voyager Therapeutics Inc (VYGR) has developed an industry-leading TRACER platform that addresses CNS delivery challenges, potentially transforming treatment for CNS diseases.
Negative Points
- The clinical success of VY-TAU01 and other programs is still uncertain, with critical data readouts expected only by 2026.
- Significant financial reliance on partnerships and milestone payments, which introduces variability and potential instability in funding.
- Challenges in manufacturing optimization and scale-up could impact the timely delivery and cost-effectiveness of gene therapies.
- Potential regulatory hurdles and the need for extensive clinical validation pose risks to advancing the pipeline to market.
- Competition in the gene therapy and CNS treatment landscape is intensifying, which could impact Voyager Therapeutics Inc (VYGR)'s market position and success.
Q & A Highlights
Q: Can you elaborate on the ways you are planning to use the knowledge of Receptor X, ALPL, for delivery of other modalities to CNS? Specifically, do you think antisense oligos and LMPs could see similar levels of transcytosis seen with AAVs?
A: Alfred W. Sandrock, President, CEO & Director of Voyager Therapeutics, explained that the receptors mediate the transport of large AAV capsids across the BBB. The company plans to make ligands against the receptor and conjugate them to various macromolecules to see if they can cross the BBB by transcytosis. The range of molecules could include protein therapeutics and oligonucleotides. Todd Carter, Chief Scientific Officer, added that they are looking at a variety of modalities including antibodies and oligos, and have identified some ligands.
Q: How are you thinking about optimizing manufacturing before you bring candidates into the clinic with your gene therapies? What do you expect the benefits will be as it relates to COGS of your more potent gene therapies?
A: Alfred W. Sandrock mentioned that they plan to use HEK293 cells for manufacturing, with a robust internal technical operations team assessing manufacturability. Todd Carter added that manufacturability is a key component of their assessment of novel capsids, and they expect substantial savings due to the potency of their novel capsids, which require much less material for delivery to the CNS.
Q: Can you discuss any potential read-through from the advisory committee meeting on donanemab in Alzheimer's disease to your Alzheimer's programs?
A: Alfred W. Sandrock stated that the advisory committee meeting might provide insights into important outcome measures for approval, which could be relevant for their programs. However, he noted that many questions might be unique to donanemab and may not pertain much to Voyager's programs.
Q: Have you disclosed what doses you are going to explore in the single ascending dose trial for VY-TAU01? How will you use the pharmacokinetic data to determine what doses should be explored in the MAD trial?
A: Toby Ferguson, Chief Medical Officer, mentioned that they haven't disclosed the doses yet but based on preclinical data, they expect to get safety and PK information to appropriately translate into doses for the MAD in mid-2025. Alfred W. Sandrock added that they anticipate the trial will inform the dose range for the upcoming multiple ascending dose trial.
Q: How does the shelf life of gene therapy products play a role when thinking about the commercial applicability of manufacturing scale?
A: Todd Carter explained that gene therapy products typically require specific cold temperatures for storage, which is part of the evaluation for stability in their manufacturability and process development.
Q: What percentage reduction on NFL is considered clinically relevant in the SOD1-ALS context?
A: Toby Ferguson noted that while it's not clear what constitutes a clinically relevant reduction, it has to be substantially greater than the variability of the assay and biological variability. The data from Biogen's tofersen informs the magnitude of necessary reduction.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
