Amylyx Pharmaceuticals (AMLX) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic focus and pipeline overview

• Focus remains on cell death and degeneration, especially in neurodegenerative diseases, with clinical programs in Wolfram syndrome, PSP, and a new ALS candidate entering trials later this year.

• Recent PHOENIX trial results in ALS led to organizational reprioritization and pipeline refocus.

• Strong cash position of $373 million, with runway expected into 2026, supporting all current milestones.

• Business development is considered, but current priority is advancing existing programs with high unmet need and clear mechanistic rationale.

• Multiple key milestones expected: full Wolfram data in fall, PSP interim results mid-2025, and initial ALS cohort data later next year.

Wolfram syndrome program

• Wolfram syndrome is a rare, monogenic, progressive neuroendocrine disease with severe unmet need and no approved therapies.

• Lead candidate AMX0035 targets endoplasmic reticulum stress and mitochondrial dysfunction, addressing the root pathophysiology.

• Early clinical data show improved C-peptide response and unexpected visual acuity gains in adults, with further data on all 12 patients expected in the fall.

• Regulatory engagement with FDA is underway, aiming for accelerated approval using C-peptide as a primary endpoint.

• Plans to expand to pediatric studies and ongoing work on pediatric-friendly formulations.

Progressive supranuclear palsy (PSP) program

• PSP is a rapidly progressive tauopathy with high unmet need and at least 20,000 U.S. patients, often underdiagnosed.

• Phase 3 trial initiated based on strong biomarker data from Alzheimer’s studies showing significant tau reduction.

• Interim analysis planned for mid-2025, with a seamless study design to allow for rapid data sharing and continuation.