Amylyx Pharmaceuticals (AMLX) investor relations material

Key program updates and clinical development

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

• Five prior trials supported FDA breakthrough therapy designation for Avexitide; phase 2B showed a 64% reduction in severe hypoglycemic events.

• A long-acting formulation of Avexitide is in preclinical development, with a candidate selection decision expected in the next few months.

• Additional candidates include AMX0035 for Wolfram syndrome and AMX0114 for ALS, with phase 3 for Wolfram planned for the second half of next year.

• AMX0114 phase 1 data in ALS will be presented, with biomarker analysis and dose escalation ongoing.

Market opportunity and commercialization strategy

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

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

• Commercialization plans focus on endocrinology and bariatric centers, leveraging rare disease launch experience and analogs from recent rare endocrine launches.

• Patient journey typically takes 1–3 years from symptom onset to diagnosis; earlier diagnosis is expected with the availability of treatment.

• Market research suggests daily subcutaneous injection is acceptable, but a long-acting version would be more convenient.

Expansion and future directions

• Avexitide may be expanded to sleeve gastrectomy and other upper GI surgeries, as well as congenital hyperinsulinism.

• Research partnership with Gubra is advancing long-acting GLP-1 receptor inhibitor development.

• Regulatory path for new formulations may leverage learnings from Avexitide to expedite development.

• Combination therapy is seen as the future for ALS, with AMX0114 targeting axon degeneration as a potential mainstay.

• Phase 3 trial for AMX0035 in Wolfram syndrome is pending FDA alignment, with initiation targeted for the second half of next year.