Amylyx Pharmaceuticals (AMLX) FY Conference Transcript

Summary of Amylyx Pharmaceuticals (AMLX) FY Conference June 10, 2025 Company Overview - Amylyx Pharmaceuticals has undergone significant changes in the past year, focusing on three different assets across four ongoing clinical trials [1][2] Key Assets and Clinical Trials 1. Avexatide - A GLP-1 receptor antagonist aimed at treating post-bariatric hypoglycemia (PBH) - Currently in a pivotal study with enrollment completion targeted by the end of 2025 and data expected in the first half of 2026 [3][6] - Previous studies supported FDA Breakthrough Therapy Designation, with five prior studies demonstrating its efficacy [3][19] 2. AMX-35 - A small molecule targeting endoplasmic reticulum (ER) stress and mitochondrial dysfunction - Trials ongoing for Wolfram's syndrome and progressive supranuclear palsy (PSP) - Positive data from the HELIOS trial for Wolfram's syndrome was recently announced, showing increased C-peptide levels, indicating improved insulin production [4][5] 3. AMX-114 - An antisense oligonucleotide targeting calpain-2 for ALS and other neurodegenerative diseases - A dose-escalating placebo-controlled study has commenced, with safety and early biomarker data expected by the end of 2025 [5][6] Market Insights and Patient Population - The estimated addressable patient population for PBH is around 160,000, based on studies indicating that approximately 8% of bariatric surgery patients develop PBH [10][11] - Symptoms of PBH can severely disrupt patients' lives, leading to dizziness, loss of consciousness, and confusion, making effective treatment critical [9][10] - Adult endocrinologists primarily manage PBH symptoms, with many clinics reporting a significant number of patients under care [13][14] Clinical Data and Efficacy - Phase II trials of Avexatide showed a 53% reduction in level II hypoglycemic events and a 66% reduction in level III events, both statistically significant [23][24] - The Phase III trial is designed to maintain consistency with Phase II, focusing on patients experiencing at least one hypoglycemic event per week [25][28] Regulatory and Commercial Considerations - The FDA's Breakthrough Therapy Designation facilitates more frequent interactions and support for the pivotal study [35] - Pricing strategies will be informed by market research and comparisons with other rare endocrine launches, with a focus on demonstrating the unmet need and efficacy to payers [37][39] Intellectual Property and Collaborations - Avexatide has a base case patent extending to 2037, with additional method of use and formulation patents [41] - A collaboration with Gubra aims to develop a long-acting GLP-1 receptor antagonist, with potential applications in other hypoglycemia-related conditions [43][44] Future Directions - The company is exploring additional indications for Avexatide, including congenital hyperinsulinism and hypoglycemia resulting from other surgeries [50][51] - Ongoing discussions with regulators will focus on the design of the Phase III trial for Wolfram's syndrome, aiming to establish key endpoints and study duration [61][64] Financial Position - Amylyx ended the last quarter with $204 million in cash, providing a runway through the end of 2026, covering pivotal studies and ongoing programs [74][75]