Amylyx Pharmaceuticals (AMLX) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Pipeline and clinical development updates

• Four clinical programs are ongoing, including AMX35 in PSP and Wolfram syndrome, AMX0114 in ALS, and the newly acquired avexitide for post-bariatric hypoglycemia (PBH).

• Interim data for AMX35 in PSP expected mid-next year; Wolfram syndrome program showed encouraging interim results with further data in October.

• AMX0114, an antisense oligonucleotide targeting calpain-2 for ALS, will begin a phase 1 multiple ascending dose study by year-end, with cohort-level data in 2025.

• Avexitide, a first-in-class GLP-1 antagonist for PBH, is phase III ready, with pivotal trial initiation planned for Q1 2025 and data readout in 2026.

Avexitide acquisition and clinical rationale

• Avexitide addresses PBH, a severe orphan disease affecting about 160,000 people in the U.S., with an estimated 16,000 new cases annually.

• Five clinical trials support avexitide's efficacy in reducing hypoglycemic events, with a 66% reduction in Level 3 events at the 90 mg dose.

• FDA has granted breakthrough status for both PBH and congenital hyperinsulinism; only one pivotal trial required for approval.

• The phase III trial will be a 12-week, randomized, placebo-controlled study with 90 participants, focusing on Level 2 and Level 3 hypoglycemia as the primary endpoint.

• Strong IP position with 11 issued patents, composition of matter patent to 2037, and potential for patent term extension.

Market opportunity and competitive landscape

• No approved therapies for PBH; current management relies on diet and off-label medications with limited efficacy.

• One other GLP-1 antagonist in early development; avexitide is the most advanced in the field.

• PBH is a significant orphan disease, with a large, easily identifiable patient population due to the prevalence of bariatric surgery.

• The need for PBH treatment is expected to persist despite trends in obesity management and bariatric surgery rates.