Release Date: August 08, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Amylyx Pharmaceuticals Inc (AMLX, Financial) expanded its late-stage pipeline with the acquisition of Avexitide, a Phase 3 ready asset with FDA breakthrough therapy designation.
- Avexitide has shown significant reductions in hypoglycemic events in clinical trials, with a 66% reduction in level three hypoglycemic events in a Phase 2B trial.
- The company has a strong cash position, with $309.8 million in cash and investments, expected to support operations into 2026.
- Amylyx Pharmaceuticals Inc (AMLX) is on track to initiate a pivotal Phase 3 program for Avexitide in post-bariatric hypoglycemia (PBH) in Q1 of next year.
- The company is actively engaging with the FDA and planning for a single Phase 3 clinical trial for AMX0035 in Wolfram syndrome, with promising data on glycemic control and other outcomes.
Negative Points
- Net product revenue was negative $1 million for the second quarter due to adjustments to gross to net revenue reserve estimates.
- The company recorded a net loss of $72.7 million for the second quarter, including restructuring charges.
- Cost of sales increased to $7.4 million for Q2, primarily related to estimated losses on firm commitments under commercial manufacturing and supply agreements.
- Research and development expenses were $23.3 million for the quarter, reflecting a decrease due to restructuring and the outcome of the Phoenix trial.
- Selling, general, and administrative expenses were $21.6 million for Q2, a decrease from the previous year, primarily due to a decline in payroll and personnel-related costs.
Q & A Highlights
Q: Can you provide more details on the PSP data expected next year and the potential for adjustments based on interim results?
A: Camille L Bedrosian, Chief Medical Officer, explained that the Phase 2B study will analyze about 100 participants at 24 weeks. If the results are positive, they will seamlessly proceed into the Phase 3 portion, determining the final sample size based on these data.
Q: How does the Wolfram syndrome opportunity in Europe compare to the US?
A: Camille L Bedrosian noted that while they are aware of a significant Wolfram population in Europe, their current focus is on the US. Justin Klee, Co-CEO, added that Wolfram syndrome is a global unmet need due to its monogenic nature.
Q: What do prior studies in PSP suggest for placebo and natural history population at the 24-week time point?
A: Camille L Bedrosian stated that previous Phase 3 studies in PSP showed a consistent rate of deterioration, which helps in understanding upcoming data. Justin Klee added that the placebo rate is strikingly consistent, usually just under a point a month.
Q: Will the Wolfram syndrome Phase 3 trial need to be placebo-controlled?
A: Camille L Bedrosian mentioned that they are engaging with stakeholders, including the FDA, and will share finalized plans. They will present data on all 12 participants at 24 weeks and longer-term data for some participants later this fall.
Q: What type of data will be presented at the updated Helios data presentation?
A: Camille L Bedrosian confirmed that they will provide longer-term data for individuals reported earlier this year, as well as data through 24 weeks for all 12 participants, covering top-line data, primary endpoint, and key secondary endpoints.
Q: What is the strategy for R&D expenses moving into 2025, and how will the pipeline be built?
A: James Frates, CFO, explained that restructuring and the winding down of the Phoenix Phase 3 trial open up room for new spending. They expect cash expenses for R&D and SG&A to be $30 million to $40 million per quarter.
Q: For the PSP interim analysis, does completing enrollment by the end of this year seem feasible?
A: Camille L Bedrosian stated that enrollment is progressing well, and they are on track to have data by mid-2025, with more information to be provided as they approach that timeframe.
Q: Are there plans for more acquisitions following the recent acquisition of Avexitide?
A: Justin Klee mentioned that they are continuously evaluating assets that fit their pipeline and have a clear path forward. While they are focused on current assets, they remain open to adding more.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
