Amylyx Pharmaceuticals has dosed the first subject in the randomised Phase I LUMINA trial, investigating AMX0114, a potential treatment for amyotrophic lateral sclerosis (ALS).
This double-blind, multinational, placebo-controlled trial aims to assess the therapy’s pharmacodynamics, safety, tolerability, and pharmacokinetics in ALS subjects.
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Expected to enrol 48 subjects across North America, the trial will explore the ALS biomarkers, particularly the change from baseline in neurofilament light chain levels.
Subjects will be administered the therapy or a placebo through intrathecal injections, a process of delivering medication into the spinal canal, also referred to as lumbar puncture, once every four weeks for up to four doses.
The trial will monitor the occurrence of serious adverse events, dose-limiting toxicities, and other potential abnormalities in clinical lab evaluations, physical and neurological exams, vital sign checks, and electrocardiograms.
Early cohort data from this trial is anticipated by the company this year.
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By GlobalDataAmylyx Pharmaceuticals chief medical officer Camille Bedrosian said: “AMX0114 targets calpain-2, which is an important contributor to axonal degeneration and studied over decades of research as a potential target for the treatment of ALS and other neurodegenerative diseases.
“We are excited to progress AMX0114 into the clinic for people with ALS as we work to advance a potential therapy for this relentlessly progressive, fatal disease.”
Designed by the company, AMX0114 targets a calcium-activated protease, calpain-2. The investigational antisense oligonucleotide aims at this fatal neurodegenerative condition, which leads to the death of motor neurons in the brain and spinal cord.
This announcement follows the positive topline outcomes from the Phase II HELIOS trial reported in October 2024, which showed sustained improvements with the company’s AMX0035 in adults with Wolfram syndrome.
