Release Date: November 07, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Amylyx Pharmaceuticals Inc (AMLX, Financial) reported positive top-line data from the Phase II HELIOS trial of AMX0035 in Wolfram syndrome, showing improvement in pancreatic function and stabilization across secondary endpoints.
- The company is on track to initiate a Phase III trial for avexitide in post-bariatric hypoglycemia (PBH) in Q1 2025, with FDA breakthrough therapy and orphan drug designations.
- Amylyx Pharmaceuticals Inc (AMLX) has a strong financial position with a cash runway expected to last into 2026.
- The ORION trial of AMX0035 in progressive supranuclear palsy (PSP) is recruiting well, with interim analysis data expected in mid-2025.
- The company has received clearance from Health Canada for a Phase I trial of AMX0114 in ALS, with early cohort data expected in 2025.
Negative Points
- The FDA has placed a clinical hold on the AMX0114 trial in the US, citing concerns over the proposed starting dose, which could delay US-based trials.
- Net product revenues were only $400,000 for the third quarter, while the cost of sales was $800,000, indicating a financial loss from discontinued commercial sales.
- The company recorded a net loss of $72.7 million for the third quarter, highlighting ongoing financial challenges.
- Research and development expenses decreased, primarily due to a decline in clinical expenses and restructuring, which may impact future innovation and development.
- The company faces challenges in balancing cash among its lead program avexitide and other programs, which may have a lower probability of success.
Q & A Highlights
Q: How should we think about the market size for PBH patients that are officially managed by existing therapy? And what are the gating factors to initiating the phase III PBH study?
A: Joshua Cohen, Co-CEO, explained that there are approximately 160,000 people living with PBH, based on literature suggesting 20% to 40% of bariatric surgery patients show abnormal glycemic control. About 8% of these patients have persistent PBH. Camille L. Bedrosian, Chief Medical Officer, added that they are actively working with clinical sites to initiate the trial in Q1 2025, with significant enthusiasm from the clinical community.
Q: What are the considerations for the phase III trial design for PBH, and is there potential for an earlier readout in 2026?
A: Camille L. Bedrosian, Chief Medical Officer, stated that the trial will use a 90 mg dose with a primary endpoint of composite level II and III hypoglycemia. The study is expected to begin in Q1 2025, with data anticipated in 2026. The design is based on previous significant results in phase II studies.
Q: How do you address the risk of hyperglycemia with avexitide, and why did the FDA deem the 12.5 mg dose of AMX0114 too high?
A: Camille L. Bedrosian, Chief Medical Officer, clarified that avexitide is a GLP-1 receptor antagonist, returning glycemic control to homeostasis without causing hyperglycemia. Regarding AMX0114, they are confident in its safety profile and are addressing FDA concerns, with plans to proceed with trials in Canada.
Q: Why include level II hypoglycemia in the primary endpoint for the PBH trial?
A: Camille L. Bedrosian, Chief Medical Officer, explained that including level II hypoglycemia captures all clinically meaningful events. The composite endpoint is well-established in anti-diabetic drug trials and provides a comprehensive measure of efficacy.
Q: How do you plan to balance cash among your lead program and other projects?
A: James Frates, CFO, emphasized that avexitide is their lead asset due to its clinical data and market opportunity. They are committed to advancing all programs but will prioritize avexitide, ensuring cash runway through its phase III data.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
