Amylyx Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial Results
Amylyx Pharmaceuticals, Inc. today reported financial and business results for the fourth quarter and full year ended December 31, 2025. 2025 was a year of meaningful advancement for Amylyx’s pivotal avexitide program in post-bariatric hypoglycemia, as well as progress across our broader pipeline,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “In 2026, our primary focus is on our Phase 3 LUCIDITY trial of avexitide in PBH. With recruitment of LUCIDITY complete, we are on track to complete enrollment this month and continue to expect topline data in Q3 2026.
We designed LUCIDITY with the goal of replicating and building on the robust body of evidence demonstrating statistically significant reductions in hypoglycemic events observed in five prior clinical trials of avexitide in PBH. We continue to be encouraged by the high participant interest and broad engagement seen across the LUCIDITY trial sites.
“Looking ahead, we have a clear trajectory toward potentially delivering the first FDA-approved therapy to the PBH community. Supported by a cash runway extending into 2028, we remain focused on disciplined execution as we continue to actively prepare for a regulatory submission and build our commercial infrastructure to support potential commercialization in 2027,” continued Mr. Cohen and Mr. Klee.
Fourth Quarter and Recent Updates:
- Amylyx announced the selection of AMX0318, a novel glucagon-like peptide-1 (GLP-1) receptor antagonist for long-acting administration, as a development candidate for post-bariatric hypoglycemia (PBH) and other rare diseases. AMX0318 was selected as a development candidate after demonstrating robust preclinical and chemical properties, including a favorable pharmacokinetic profile that may support long-acting administration, a robust chemical stability profile, strong in vitro potency, evidence of in vivo activity and tolerability, and high solubility. AMX0318 was identified through a research collaboration with Gubra A/S, a company specializing in peptide-based drug discovery and preclinical contract research services.
- Amylyx presented early safety and tolerability data from Cohort 1 of its Phase 1 LUMINA trial of AMX0114 at the 36th International Symposium on ALS/MND (MNDA) in December 2025. AMX0114 is an investigational antisense oligonucleotide (ASO) targeting calpain-2 with U.S. Food and Drug Administration (FDA) Fast Track Designation for the potential treatment of amyotrophic lateral sclerosis (ALS). AMX0114 was generally well-tolerated in LUMINA trial participants enrolled in Cohort 1 (n=12), with no treatment-related serious adverse events (SAEs).
Upcoming Expected Milestones:
- Recruitment of the pivotal Phase 3 LUCIDITY trial of avexitide in PBH is complete. Amylyx continues to expect to randomize and dose the last eligible participants this month with topline data expected in Q3 2026, and, if approved, a commercial launch in 2027. Avexitide is an investigational, first-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy Designation in PBH.LUCIDITY is a 16-week, multicenter, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of avexitide in approximately 75 participants with PBH following Roux-en-Y gastric bypass (RYGB) surgery. LUCIDITY is evaluating the FDA-agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Participants who complete the 16-week double-blind period are eligible to enter a 32-week open-label extension (OLE) period.
- Completion of enrollment of Cohort 2 (n=12) of the Phase 1 LUMINA clinical trial of AMX0114 in ALS is expected in March 2026 and presentation of biomarker data from Cohort 1 (n=12) is expected in the first half of 2026. The LUMINA trial is a randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial of AMX0114 in people living with ALS, with Cohort 1 investigating the first and lowest of four doses being evaluated in the trial. LUMINA is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS and assessing both novel and broadly researched ALS biomarkers, including change from baseline in neurofilament light chain (NfL) levels.
- Investigational New Drug (IND)-enabling studies for AMX0318 are underway with an IND filing targeted for 2027. AMX0318 has completed extensive preclinical evaluation, including stability, solubility, potency, in vivo pharmacokinetics and pharmacodynamics, and in vivo tolerability studies.
About Amylyx Pharmaceuticals
At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on four investigational therapies across several endocrine conditions and neurodegenerative diseases in which we believe they can make the greatest impact.
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