Amylyx Pharmaceuticals (AMLX) investor relations material

Clinical trial results and data highlights

• Phase III trial of avexitide in post-bariatric hypoglycemia (PBH) met primary and all secondary endpoints with high statistical significance, showing a 55% reduction in severe hypoglycemic events and a strong safety profile.

• Consistent efficacy was observed across different measures, including self-monitored blood glucose and continuous glucose monitoring, with both Level 2 and Level 3 events significantly reduced.

• All eligible patients rolled into the open-label extension, reinforcing confidence in efficacy and safety.

• Full data presentation and publication are targeted before year-end to maximize medical community impact.

Regulatory and launch plans

• NDA submission for avexitide is planned before the end of 2026, with preparations underway for a potential 2027 launch.

• Label scope may depend on FDA decision; while the phase III trial focused on Roux-en-Y gastric bypass PBH, data suggest avexitide is effective regardless of surgery type.

• If limited to Roux-en-Y, about 120,000 of 160,000 U.S. PBH patients would be eligible, but efficient trials could expand the label.

Market opportunity and disease landscape

• PBH affects about 8% of bariatric surgery patients, estimated at 160,000 in the U.S., with high awareness among endocrinologists and significant unmet need.

• Multiple data sources, including literature, claims analysis, and clinic surveys, support the 160,000 patient estimate.

• PBH is a chronic, debilitating condition with frequent medical emergencies, and current management options are limited to dietary counseling.

• The disease is recognized on endocrinology board exams, and most endocrinologists report having PBH patients under care.