Amylyx Pharmaceuticals, Inc. Q1 FY2025 earnings call

Key Points

• 2025 is a year of execution with 3 potential therapies across 4 clinical trials. Achieved milestones: dosed first participant in pivotal Phase 3 LUCIDITY trial of avexitide in post-bariatric hypoglycemia (PBH) and first participant in Phase 1 LUMINA trial of AMX0114 in ALS. Raised $65.5 million to extend cash runway through end of 2026.

Avexitide

• Lead asset, investigational GLP-1 receptor antagonist with FDA breakthrough therapy designation for PBH. Pivotal Phase 3 LUCIDITY trial ongoing, expected enrollment completion in 2025, top line data in H1 2026, potential commercial launch in 2027. Discussed mechanism, trial details, and upcoming presentations at ENDO 2025.

AMX0035

• Evaluated in Wolfram syndrome and progressive supranuclear palsy (PSP). Phase 2 HELIOS trial in Wolfram syndrome to present week 48 data next week; Phase 2b ORION trial in PSP completed enrollment in Jan 2025, expected Q3 data.

AMX0114

• Investigational antisense oligonucleotide for ALS. Phase 1 LUMINA trial dosed first participant last month, a multinational trial evaluating safety, tolerability, etc., with early cohort data expected later in 2025.

At the end of the first quarter, Amylyx had a cash position of $204.1 million, including approximately $65.5 million in net proceeds from a public offering. Total operating expenses for the quarter were $37.8 million, down 82% from Q1 2024. Research and development expenses were $22.1 million, and selling, general and administrative expenses were $15.7 million. The company believes it has the cash to deliver planned clinical milestones through the end of 2026.

• Anticipates top line data from Phase 3 LUCIDITY trial of avexitide in PBH in H1 2026.
• Expects week 48 data from HELIOS trial in Wolfram syndrome.
• Aims to report top line data from Phase 2b ORION trial in PSP in Q3.
• Looks forward to early cohort data from Phase 1 LUMINA trial of AMX0114 in ALS later in 2025.
• Believes it has the cash to advance pipeline and support commercial preparations for avexitide.

Q: On avexitide patient education and PBH population.

A: Justin Klee discussed that PBH is well recognized by adult endocrinologists but education is needed for rare disease awareness and to inform about avexitide's potential benefits if approved.

Q: On PSP interim data efficacy.

A: Josh Cohen stated the study has 80% power to detect a 30% effect on the PSP rating scale, and the decision on next steps will be driven by all data, not just the PSPRS.

Q: On PBH prevalent population and new incidents.

A: Justin Klee and Josh Cohen explained that PBH affects a single-digit percentage of bariatric surgery patients, with about 160,000 people in the US living with PBH, and 8% of bariatric surgery patients developing PBH within 1-3 years post-surgery.

Q: On HELIOS FDA trial design.

A: Camille Bedrosian mentioned the week 48 data from HELIOS will inform the Phase 3 program, and discussions with FDA on trial design are ongoing as there's no template for Wolfram syndrome trials.

Q: On avexitide subgroup analysis and payer access.

A: Josh Cohen stated all trial patients have frequent events, and no step therapy is anticipated in PBH as there are no currently approved therapies.

Q: On GLP-1 antagonist safety for avexitide.

A: Justin Klee noted avexitide has a good safety profile with generally well-tolerated data from preclinical and clinical studies.

Q: On ALS trial inclusion criteria and NfL.

A: Justin Klee said there's no inclusion criterion for high NfL levels in the ALS trial, as they didn't want to oversubset and miss important signals.

Q: On avexitide vs other drugs and placebo response.

A: Justin Klee and Josh Cohen compared avexitide's strong profile with prior trials showing differences, and mentioned no meaningful placebo effect seen in Phase 2 studies.