Amylyx Pharmaceuticals disclosed that the first cohort of its Phase 1 LUMINA trial of the antisense oligonucleotide AMX0114 was well‑tolerated, with 12 participants enrolled and no treatment‑related serious adverse events reported. The data, cut off on November 11, 2025, were presented at the 36th International Symposium on ALS/MND in San Diego, California, which ran from December 5‑7, 2025.
The LUMINA study is a multinational, randomized, double‑blind, placebo‑controlled, multiple‑ascending‑dose trial designed to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114, an antisense oligonucleotide that targets calpain‑2, a protease implicated in axonal degeneration. The trial aims to enroll 48 participants across Canada and the United States, and the first cohort’s data provide a critical de‑risking milestone for the program.
Amylyx plans to begin enrollment of a second cohort in Canada later in December and in the United States in January. The company also expects to release biomarker data, including neurofilament light chain levels, from the first cohort in the first half of 2026. In addition to AMX0114, Amylyx’s pipeline includes avexitide for primary biliary cholangitis, underscoring a diversified therapeutic strategy.
Dr. Sabrina Paganoni, principal investigator of the LUMINA trial, said, “We are encouraged by these data as we continue to advance AMX0114 as a potential treatment for this rapidly progressive disease with high unmet need. The safety and tolerability analysis allows LUMINA to proceed with its next cohort of participants, which is critical given that this community has no time to wait.”
The positive safety profile, combined with the Fast Track designation from the FDA and the lifting of a clinical hold in January 2025, positions AMX0114 as a promising candidate in the ALS therapeutic landscape. The data support the continuation of the trial and lay the groundwork for future efficacy studies, potentially accelerating regulatory approval and providing a new treatment option for patients with ALS.
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