Amylyx Reports Q4 and Full-Year 2024 Financial Results
Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) recently released its financial results for the fourth quarter and full year ending December 31, 2024, showcasing significant advancements in its research and development pipeline, as well as key financial and operational milestones.
Strategic Progress in 2024 and Outlook for 2025
Amylyx made substantial progress in 2024, setting the stage for critical milestones in the years ahead, particularly in orphan markets with high unmet medical needs. The company successfully advanced its lead investigational asset, avexitide, into a pivotal Phase 3 clinical trial. It anticipates topline results from this trial in the first half of 2026. Additionally, 2025 is expected to yield Week 48 data from the AMX0035 program in Wolfram syndrome, interim analysis data from the AMX0035 program in progressive supranuclear palsy (PSP), and early cohort data from the AMX0114 program in amyotrophic lateral sclerosis (ALS).
According to Joshua Cohen and Justin Klee, Co-CEOs of Amylyx, “We made significant progress in 2024 across our pipeline, setting the stage for important milestones in the year ahead. With a strong balance sheet, strengthened by our recent financing that extended our expected cash runway through the end of 2026, we believe we are well positioned to achieve our goals.”
Fourth Quarter and Recent Updates
Avexitide Phase 3 LUCIDITY Trial Initiation
Amylyx launched the pivotal Phase 3 LUCIDITY clinical trial to evaluate avexitide, a glucagon-like peptide-1 (GLP-1) receptor antagonist, for the treatment of post-bariatric hypoglycemia (PBH). The trial, which commenced recruitment in February 2025, is designed as a randomized, double-blind, placebo-controlled study involving approximately 75 participants across 20 sites in the U.S. The study will assess avexitide’s efficacy and safety in PBH patients who have undergone Roux-en-Y gastric bypass (RYGB) surgery. Dosing is anticipated to begin in March or April 2025.
Avexitide has already demonstrated consistent, dose-dependent effects in prior PBH trials, showing statistically significant reductions in hypoglycemic events and improved glucose control. These promising results position avexitide as a potential first-approved therapy for PBH.
Executive Leadership Expansion
Amylyx appointed Dan Monahan as Chief Commercial Officer in January 2024. Monahan, who brings over 20 years of commercialization experience from Otsuka, Novartis, and Sanofi, will spearhead the company’s commercialization strategy, particularly in preparation for avexitide’s potential launch.
Strategic Collaboration with Gubra A/S
Amylyx announced a collaboration with Gubra A/S to develop a novel long-acting GLP-1 receptor antagonist. The companies plan to identify a lead development candidate for Investigational New Drug (IND)-enabling studies, reinforcing Amylyx’s commitment to innovation in metabolic and neurological diseases.
Phase 1 LUMINA Trial for AMX0114 in ALS
The company initiated the Phase 1 LUMINA clinical trial in February 2025 to evaluate AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for ALS treatment. The multicenter, randomized, placebo-controlled, multiple ascending dose trial will assess safety and biological activity, including changes in neurofilament light (NfL) levels, an ALS biomarker. The trial plans to enroll 48 participants, randomized 3:1 to receive AMX0114 or placebo through intrathecal administration every four weeks, for up to four doses. Dosing is expected to commence in March or April 2025.
Public Offering and Financial Stability
Amylyx successfully closed an underwritten public offering of 19.7 million shares of common stock, raising approximately $65.5 million. This financing extends the company’s expected cash runway through the end of 2026.
Upcoming Expected Milestones in 2025-2026
- Phase 3 LUCIDITY Trial: Completion of enrollment is expected in 2025, with data readout in the first half of 2026. If approved, commercial launch is anticipated in 2027.
- AMX0035 in Wolfram Syndrome: Week 48 data from the Phase 2 HELIOS trial will be available in the coming months, guiding the design of a future Phase 3 trial.
- AMX0035 in PSP: Unblinded interim analysis of the Phase 2b portion of the ORION trial is expected in Q3 2025. Data from 139 participants through Week 24 will inform a go/no-go decision for the Phase 3 portion.
- AMX0114 in ALS: Early cohort data from the Phase 1 LUMINA trial will be available in 2025. Trial sites in Canada have already begun participant recruitment, with U.S. sites expected to follow soon.
Financial Results for the Fourth Quarter and Full Year 2024
Research & Development (R&D) Expenses
R&D expenses for Q4 2024 were $22.9 million, compared to $44.9 million in Q4 2023. For the full year 2024, expenses totaled $104.1 million, down from $128.2 million in 2023. The decrease is attributed to reduced spending on AMX0035 for ALS, lower payroll costs, and decreased preclinical development activities.
Selling, General & Administrative (SG&A) Expenses
SG&A expenses for Q4 2024 were $17.1 million, significantly lower than $52.2 million in Q4 2023. For the full year, SG&A expenses totaled $114.3 million, down from $188.4 million in 2023. The decrease primarily resulted from reductions in payroll, consulting, and professional services expenses.
Net Loss
Amylyx reported a net loss of $37.5 million ($0.55 per share) for Q4 2024, compared to net income of $4.7 million ($0.07 per share) in Q4 2023. The full-year 2024 net loss was $301.7 million ($4.43 per share), compared to net income of $49.3 million ($0.70 per share) in 2023.
Cash Position
As of December 31, 2024, Amylyx held cash, cash equivalents, and short-term investments totaling $176.5 million, compared to $234.4 million on September 30, 2024. The company also raised $65.5 million in January 2025, securing funding through the end of 2026. The cash position reflects payments of $31.0 million in Q4 2024 related to revenue returns, rebates, and settlement of purchase commitments for AMX0035 following the voluntary discontinuation of RELYVRIO®/ALBRIOZA™ in April 2024. The company expects to pay the remaining residual obligations of $7.8 million through 2025.
Investor Conference Call
Amylyx management will host a conference call on March 4, 2025, at 8:00 a.m. ET to discuss financial results and corporate updates. Interested parties can access the call by dialing +1 (800)-836-8184 (U.S. & Canada) or +1 (646)-357-8785 (international). A live audio webcast will be available on the company’s investor relations website, where it will remain accessible for 90 days.
