Amylyx Pharmaceuticals (AMLX) investor relations material

Clinical trial results and data insights

• Avexitide demonstrated a 55% reduction in level two and three hypoglycemia events in PBH patients, with high statistical significance across all primary and secondary endpoints and a favorable safety profile.

• The study confirmed the underlying biology of PBH as driven by excessive GLP-1 response post-bariatric surgery, and avexitide's GLP-1 antagonism directly addresses this mechanism.

• No avexitide-related weight gain or medical hyperglycemia was observed, and the benefit appears durable over the 16-week study period.

• Plans are in place to publish and present the full data set in high-profile formats to inform the medical community.

Regulatory and commercial plans

• NDA submission for avexitide is targeted by year-end, with launch preparations underway for a potential 2027 approval.

• The team is leveraging claims-based and survey data to identify approximately 160,000 PBH patients in the U.S., with ongoing efforts to build out commercial and field medical teams.

• Launch strategy focuses on physician education, patient access, and operational readiness, with continued hiring and infrastructure development.

• Breakthrough status for avexitide is expected to facilitate a priority review process.

Market opportunity and patient impact

• PBH is a chronic, disabling condition with no FDA-approved treatments; avexitide addresses a significant unmet need.

• Physicians report high demand for effective therapies, with strong feedback from both clinicians and patients on the clinical impact of reducing severe hypoglycemic events.

• The PBH patient population is expected to grow as bariatric surgery remains a mainstay for severe obesity, despite advances in weight loss drugs.