Amylyx Pharmaceuticals (AMLX) investor relations material

Key program updates and clinical development

• Avexitide is in a pivotal Phase 3 trial for post-bariatric hypoglycemia (PBH), with enrollment completion expected in Q1 and top-line results in Q3 2026, targeting commercialization in 2027.

• Phase 2b data for avexitide showed a 64% reduction in severe hypoglycemic events, supporting FDA Breakthrough Therapy designation; Phase 3 is powered to detect a 35% effect size.

• Research is ongoing for a long-acting GLP-1 receptor inhibitor, with a candidate decision for IND-enabling studies expected in the coming months.

• Additional candidates include AMX0035 for Wolfram syndrome (Phase 3 planned for late next year) and AMX0114 for ALS, with early Phase 1 data and biomarker analysis underway.

• The company is leveraging prior rare disease launch experience to prepare for commercialization, focusing on patient journey, supply chain, and chronic treatment models.

Market opportunity and awareness

• PBH affects an estimated 160,000 people in the U.S., with high unmet need and no approved treatments; diagnosis is straightforward for endocrinologists.

• Growing awareness is supported by PBH inclusion in board exams, physician petitions for CGM coverage, and recent SNOMED code adoption; ICD-10 code decision expected in April.

• Market research and claims data confirm the patient population and support targeted commercialization strategies through endocrinology and bariatric centers.

• Educational partnerships with professional societies are planned to raise awareness and improve early diagnosis, especially as new treatments become available.

• The company anticipates that a meaningful treatment will drive earlier diagnosis and greater urgency among providers.

Pipeline expansion and future directions

• Avexitide's mechanism may be applicable to other forms of post-surgical hypoglycemia, including sleeve gastrectomy and gastrectomy for cancer, as well as congenital hyperinsulinism.

• A research partnership with Gubra is advancing long-acting GLP-1 receptor inhibitor development, with promising preclinical results.

• Regulatory strategy may leverage learnings from avexitide to expedite clinical development of new candidates.

• The company is preparing for potential label expansion and broader indications based on robust pharmacology and clinical outcomes.

• Market access strategies are informed by recent rare endocrine launches, supporting premium orphan drug pricing and patient support models.