Home / Health / ALS Drug AMX0114 Shows Early Promise in Safety Trial
ALS Drug AMX0114 Shows Early Promise in Safety Trial
8 Dec, 2025
Summary
- AMX0114, an investigational drug for ALS, was well-tolerated in the first cohort.
- No serious adverse events related to AMX0114 were reported in the early trial phase.
- Amylyx Pharmaceuticals is preparing to expand the LUMINA study to Canada and the US.
Amylyx Pharmaceuticals has presented initial tolerability and safety findings from the Phase I LUMINA study investigating AMX0114 for amyotrophic lateral sclerosis (ALS). The first cohort of 12 participants demonstrated that AMX0114 was well-tolerated, with no serious adverse events linked to the treatment reported to date. This successful early stage paves the way for the next phase of the trial.
The company is now poised to commence enrollment for the second cohort in Canada by the end of December, followed by the United States in January. The LUMINA trial is a multinational, randomized, double-blind, placebo-controlled study designed to thoroughly evaluate AMX0114's safety, pharmacodynamics, and pharmacokinetics, while also monitoring key ALS biomarkers.
AMX0114 targets calpain-2, a protease implicated in axonal degeneration and disease progression in ALS. Preclinical data indicated that AMX0114 effectively reduces calpain-2 levels, promoting neuronal survival and lowering neurofilament light chain levels, a critical biomarker for neuronal damage.
