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Home / Health / ALS Drug AMX0114 Shows Early Promise in Safety Trial

ALS Drug AMX0114 Shows Early Promise in Safety Trial

8 Dec

•

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.
ALS Drug AMX0114 Shows Early Promise in Safety Trial

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.

Disclaimer: This story has been auto-aggregated and auto-summarised by a computer program. This story has not been edited or created by the Feedzop team.
The initial 12 participants in the LUMINA study showed AMX0114 was well-tolerated, with no treatment-related serious adverse events reported.
Amylyx plans to start enrolling the second cohort of the LUMINA study in Canada in December 2025 and in the US in January 2026.
AMX0114 is designed to inhibit calpain-2, a key factor in axonal degeneration and ALS disease progression, by reducing its protein levels.

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