Amylyx Pharmaceuticals (AMLX) Update / Briefing Transcript

Summary of Amylyx Pharmaceuticals (AMLX) Update / Briefing July 13, 2025 Company Overview - Company: Amylyx Pharmaceuticals (AMLX) - Focus: Development of Avexatide, a potential GLP-1 receptor antagonist for treating post-bariatric hypoglycemia (PBH) with Breakthrough Therapy Designation from the FDA Industry Context - Condition: Post-bariatric hypoglycemia (PBH) is a serious metabolic condition that occurs in patients after bariatric surgery, characterized by severe hypoglycemic episodes - Prevalence: Approximately 400,000 individuals in the U.S. experience clinically important hypoglycemia after bariatric surgery, with around 166,000 requiring medical management [42][43] Key Points and Arguments 1. Patient Experience: A patient shared her struggles with PBH, highlighting the debilitating nature of the condition, which severely limits daily activities and quality of life [8][9][15][16] 2. Medical Need: There is a significant unmet medical need for effective treatments for PBH, as current dietary modifications and off-label medications often fail to provide relief [39][40] 3. Mechanism of PBH: PBH is caused by an exaggerated GLP-1 response post-surgery, leading to hyperinsulinism and subsequent hypoglycemia. This condition can develop 2-3 years after surgery, but may occur anytime from 2 to 20 years post-operation [19][41] 4. Current Treatments: Existing treatments are limited and often ineffective, with no FDA-approved medications specifically for PBH. Dietary modifications and off-label medications have high failure rates and significant side effects [22][39] 5. Avexatide's Mechanism: Avexatide targets the GLP-1 receptor to normalize insulin secretion and stabilize glucose levels, addressing the underlying pathophysiology of PBH [49][50] 6. Clinical Trials: The Phase II and Phase IIb studies showed that Avexatide significantly reduced the frequency of Level II and Level III hypoglycemic events by over 50% [57][72] 7. FDA Breakthrough Therapy Designation: Avexatide is the only drug in development for PBH to receive this designation, indicating its potential to address a serious condition with high unmet medical need [57] 8. LUCIDITY Phase III Trial: The ongoing Phase III trial will evaluate the efficacy of Avexatide at a dose of 90 mg once daily, focusing on reducing hypoglycemic events [78][81] Additional Important Content - Patient Education: There is a critical need for increased awareness and education about PBH among healthcare providers and patients, as many patients are misdiagnosed or experience delays in receiving appropriate care [38][41] - Quality of Life Impact: The unpredictable nature of hypoglycemic events leads to social isolation and significant emotional distress for patients, emphasizing the need for effective treatment options [66][68] - Future Outlook: The company is actively recruiting for the LUCIDITY trial, with data expected in the first half of 2026, aiming to bring a much-needed therapy to market for PBH patients [85][86]