Amylyx Pharmaceuticals (AMLX) investor relations material

Pipeline and development focus

• Advancing three novel therapies: Avexitide for post-bariatric hypoglycemia (PBH), AMX0035 for Wolfram syndrome, and AMX0114 for ALS, all targeting diseases with high unmet needs.

• All programs have received FDA designations (Breakthrough, Orphan, or Fast Track) and are supported by robust intellectual property portfolios.

• Clinical development is led by an experienced team with a strong track record in rare disease commercialization.

Avexitide for post-bariatric hypoglycemia (PBH)

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

• Phase 3 LUCIDITY trial began dosing in April 2025, with topline data expected in 1H 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., is life-altering, and currently has no approved treatments.

• Avexitide demonstrated significant improvement in glucose stabilization and reduction of insulin hypersecretion in both clinical and preclinical studies.

AMX0035 for Wolfram syndrome

• AMX0035 is a fixed-dose combination targeting ER stress and mitochondrial dysfunction, central to Wolfram syndrome pathology.

• Phase 2 HELIOS trial met its primary endpoint of improved pancreatic function at Week 24, with continued stabilization or improvement at Week 48.

• Long-term data suggest benefit across multiple outcomes, including glycemic control, vision, and symptom burden, with a favorable safety profile.

• Orphan Drug Designation granted in both the U.S. and EU; Phase 3 trial design to be informed by ongoing FDA discussions.

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