Amylyx Pharmaceuticals (AMLX) TD Cowen 46th Annual Health Care Conference summary

Strategic outlook and key milestones

• 2026 is expected to be transformative, with top-line results from the LUCIDITY phase III trial for avexitide in PBH anticipated in Q3 2026.

• NDA submission for avexitide is planned following positive results, with commercialization targeted for 2027.

• Commercial preparations and NDA work are underway ahead of trial results.

• Cash runway extends into 2028, supporting ongoing development and commercialization plans.

Clinical development and pipeline updates

• LUCIDITY phase III trial for avexitide has completed screening and expects full enrollment this month.

• Avexitide previously showed 52% reduction in level 2 and 66% reduction in level 3 hypoglycemic events in phase II.

• Additional pipeline includes AMX0114 for ALS (dose-escalating study), AMX0035 for Wolfram syndrome, and a long-acting GLP-1 receptor antagonist in IND-enabling studies.

• AMX0114 for ALS is progressing, with biomarker data expected in the first half of the year.

Market landscape and unmet needs

• PBH affects about 160,000 people in the U.S., with no approved treatments; current management relies on medical nutrition therapy.

• Bariatric surgery rates remain robust despite GLP-1 agonist adoption, and PBH is a lifelong condition.

• Wolfram syndrome affects about 3,000 people in the U.S., with more cases internationally.