Amylyx Pharmaceuticals (AMLX) Corporate presentation summary

Pipeline overview

• Pipeline spans avexitide for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism, AMX0035 for Wolfram syndrome, AMX0114 for ALS, and AMX0318 for PBH and other rare diseases.

• Strategy emphasizes high-unmet-need diseases with mechanistic rationale, measurable clinical outcomes and biomarkers, and rigorous preclinical evidence.

Avexitide for PBH

• Phase 3 LUCIDITY met its primary endpoint: 55% reduction in the composite rate of Level 2 and Level 3 hypoglycemic events vs. placebo at Week 16; rate ratio 0.45, p=0.000003.

• All secondary endpoints were met, with reductions in Level 2 events by SMBG and CGM and Level 3 events; safety was generally favorable, with no treatment-related serious adverse events.

• Potential first FDA-approved PBH therapy; ~160,000 people in the U.S. live with PBH, for which no approved treatment options are available.

• NDA submission to the FDA is expected by end-2026; commercial launch is anticipated in 2027, if approved.

• Patent rights extend through 2037, with potential patent-term extension; the portfolio includes granted Orphan Drug designation and NCE exclusivity positioning.

AMX0035 for Wolfram syndrome

• Phase 2 HELIOS met its primary efficacy outcome, improvement in pancreatic function at Week 24; mean C-peptide response increased from baseline at Weeks 24 and 48 (N=11 and N=10).

• At Week 48, multiple measures showed improvement or stabilization, including pancreatic function, glycemic control, vision and symptom burden; 9 of 11 participants reported improvement in at least one symptom.

• AMX0035 targets ER stress and related mitochondrial dysfunction; FDA and EU Orphan Drug designations have been granted for Wolfram syndrome.

• A focused pivotal Phase 3 trial is planned, pending alignment with the FDA.

• HELIOS safety was generally well tolerated; all treatment-emergent adverse events were mild or moderate, with no serious events related to treatment reported.