Amylyx Pharmaceuticals (AMLX) Corporate presentation summary

Pipeline overview and strategy

• Focus on developing novel therapies for serious neurodegenerative and endocrine diseases with high unmet needs, leveraging a team experienced in rare disease commercialization.

• Pipeline includes avexitide (GLP-1 receptor antagonist) for PBH and congenital hyperinsulinism, AMX0035 for Wolfram syndrome, AMX0114 for ALS, and AMX0318 for PBH and other rare diseases.

• Multiple programs have received FDA Breakthrough Therapy, Orphan Drug, or Fast Track designations.

• Strong intellectual property portfolio with patent protection for lead assets through at least 2037.

Avexitide for post-bariatric hypoglycemia (PBH)

• Avexitide is a first-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy and Orphan Drug designations for PBH.

• Phase 3 LUCIDITY trial is underway, with topline data expected in Q3 2026 and potential commercial launch in 2027.

• Prior Phase 2 trials showed >50% reduction in hypoglycemic events, with consistent, dose-dependent effects and favorable safety profile.

• PBH affects ~160,000 people in the U.S., with no approved treatments and significant impact on quality of life.

• Avexitide demonstrated robust efficacy in reducing both Level 2 and Level 3 hypoglycemic events and was generally well-tolerated.

AMX0035 for Wolfram syndrome

• AMX0035 is an oral fixed-dose combination targeting ER stress and mitochondrial dysfunction, key drivers of neurodegeneration in Wolfram syndrome.

• Phase 2 HELIOS trial met its primary endpoint, showing improvement in pancreatic function and stabilization or improvement in glycemic control, vision, and symptom burden at 24 and 48 weeks.

• AMX0035 was generally well-tolerated, with most adverse events mild or moderate and no serious drug-related events.

• Wolfram syndrome is a rare, fatal disorder with no approved therapies, affecting ~3,000 people in the U.S.