Amylyx Pharmaceuticals has presented early tolerability and safety outcomes from its Phase I LUMINA study of AMX0114, along with biomarker characterisation findings in amyotrophic lateral sclerosis (ALS).
The data was presented at the 36th International Symposium held from 5-7 December 2025 in San Diego, California, US, on ALS / motor neurone disease (MND).
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In the first cohort of the LUMINA trial, which included 12 participants, AMX0114 was generally well-tolerated and no treatment-related serious adverse events were reported.
Based on these results, Amylyx plans to start enrolling the second cohort in Canada later in December and in the US in January.
LUMINA is a randomised, double-blind, multinational, multiple ascending, placebo-controlled dose trial. It is investigating AMX0114, an antisense oligonucleotide targeting calpain-2, in individuals with ALS.
The study is designed to assess the tolerability, safety, pharmacodynamics, pharmacokinetics, and ALS biomarkers, including neurofilament light chain (NfL) changes. Amylyx anticipates biomarker results from cohort one in the first half of 2026.
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By GlobalDataAmylyx chief medical officer Camille Bedrosian said: “We appreciate the partnership with LUMINA sites and participants to achieve complete enrolment of the first cohort. In addition, we are pleased that to date no drug-related SAEs or dose-limiting toxicities were observed, which represent important early steps in this study.
“AMX0114 is designed to inhibit calpain-2, a calcium-activated protease that is one of the fundamental drivers of axonal degeneration and consequent disease progression in ALS.
“Preclinical studies have demonstrated that treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in calpain-2 protein levels, translating to improved neuronal survival and reductions in extracellular NfL levels.”
In July 2025, Amylyx presented new exploratory analyses from the Phase II PREVENT and Phase IIb trials of the investigational GLP-1 receptor antagonist, avexitide, for the treatment of post-bariatric hypoglycaemia (PBH).
