Amylyx Pharmaceuticals (NasdaqGS:AMLX) FY Conference Transcript

Summary of Amylyx Pharmaceuticals FY Conference Call (September 08, 2025) Company Overview - Company: Amylyx Pharmaceuticals (NasdaqGS:AMLX) - Focus: Development of Avexitide for post-bariatric hypoglycemia (PBH) and other assets in the pipeline including AMX0035 for Wolfram syndrome and AMX0114 for ALS Key Points on Avexitide and PBH - Acquisition of Avexitide: Acquired over a year ago, Avexitide is a competitive inhibitor of the GLP-1 receptor, which lowers insulin secretion and raises glucose levels, crucial for treating hyperinsulinemic hypoglycemia [4][6] - PBH Prevalence: Approximately 160,000 people in the U.S. suffer from PBH, a debilitating condition that can lead to severe hypoglycemic events, including confusion, loss of consciousness, and seizures [5][11] - Clinical Trials: Avexitide has shown strong data in five prior trials, leading to FDA breakthrough therapy designation. A phase 3 pivotal study is currently underway, with enrollment expected to complete by the end of 2025 and data anticipated in the first half of 2026 [6][26] - Reduction in Hypoglycemic Events: In previous studies, Avexitide demonstrated a 53% reduction in level 2 hypoglycemia and a 66% reduction in level 3 hypoglycemia, indicating its potential effectiveness [20][25] - Breakthrough Therapy Designation: This designation signifies that Avexitide has substantial benefits over existing treatments for a high unmet medical need, allowing for more frequent FDA interactions and expedited review processes [27][28] Other Pipeline Assets - AMX0035: A combination therapy for Wolfram syndrome, which has shown stabilization or improvement in symptoms during initial trials. The company is working with the FDA on a potential phase 3 design [42][45] - AMX0114: An antisense oligonucleotide targeting Calpain II for ALS, currently in a multiple ascending dose study with initial safety data expected by the end of the year [49][52] Market Insights and Commercial Readiness - Market for PBH: The company is focusing on building market insights for PBH, recognizing it as a large orphan disease with significant unmet needs. Education and targeted strategies will be crucial for commercialization [30][31] - Partnership with BIBRA: A collaboration aimed at developing a longer-acting version of Avexitide, leveraging BIBRA's expertise in peptide drug development [38][39] Future Milestones - Upcoming Data: Key milestones include the completion of enrollment for the phase 3 study of Avexitide by the end of 2025 and the release of data in the first half of 2026. Updates on Wolfram syndrome and AMX0114 are also expected [56][57] Conclusion - Commitment to Unmet Needs: The company emphasizes its mission to address significant unmet medical needs, particularly in PBH, and is optimistic about the potential impact of Avexitide on patients' lives [57]