Summary of Amylyx Pharmaceuticals FY Conference Call Company Overview - **Company**: Amylyx Pharmaceuticals (NasdaqGS:AMLX) - **Focus**: Clinical-stage biotech developing therapies for neurodegenerative and endocrine diseases [1][2] Key Programs and Developments - **Avexitide**: - Late-stage asset focused on post-bariatric hypoglycemia (PBH) - Phase 3 LUCIDITY study ongoing, with expected enrollment completion by the end of 2025 and data release in the first half of 2026 [3][20] - First-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy Designation for PBH [5][6] - Mechanism: Blocks GLP-1 to reduce insulin production, addressing hypoglycemia caused by hyperinsulinemia [6][8] - Phase 2 studies showed a 64% reduction in level 3 hypoglycemic events and a 53% reduction in level 2 events [13][14] - Safety profile: Generally well tolerated with mild to moderate side effects [15] - **Wolfram Syndrome**: - Rare, progressive disease with no current treatment - Using AMX0035, showing positive outcomes in C-peptide and HbA1c levels [21][24] - Estimated 3,000 to 3,500 patients in the U.S. [25] - **ALS Program**: - Targeting axonal degeneration with AMX0114, an antisense oligonucleotide aimed at Calpain 2 - First patient data expected by the end of 2025 [26] Financial Position - Recently completed financing of $175 million, extending runway into 2027 - Cash balance of $180 million as of June 30, 2025 [27] Market Opportunity - Over 160,000 individuals in the U.S. suffer from persistent hypoglycemia post-bariatric surgery, a significant unmet medical need [18][20] - Over 2 million bariatric surgeries performed in the U.S. over the last decade, with 8% developing PBH [18] Regulatory and Patent Status - Patent rights for avexitide extend through 2037, with orphan designation granted for PBH [20][21] Conclusion - Amylyx Pharmaceuticals is actively advancing multiple programs in rare diseases with significant unmet needs, backed by a strong financial position and promising clinical data [27]