Cambridge, Mass.– Amylyx Pharmaceuticals has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for AMX0114, an investigational treatment targeting amyotrophic lateral sclerosis (ALS). The designation is intended to accelerate the development and review process for therapies that address serious conditions with unmet medical needs.
AMX0114 is an antisense oligonucleotide developed by Amylyx to inhibit calpain-2, a calcium-activated protease identified as a key driver in axonal degeneration—a major mechanism contributing to the progression of ALS. The drug is currently being evaluated in the Phase 1 LUMINA clinical trial, which began dosing its first participant in April. Early cohort data from the trial is expected later this year.
“Fast Track designation represents a meaningful step in our work to deliver promising new therapies for people living with ALS,” said Dr. Camille L. Bedrosian, Chief Medical Officer at Amylyx. “The FDA’s decision underscores the seriousness of ALS and the scientific rationale behind targeting calpain-2. We’re committed to advancing AMX0114 with urgency and scientific rigor.”
The Fast Track designation offers several regulatory benefits, including more frequent interactions with the FDA and eligibility for Priority Review if key criteria continue to be met.
AMX0114 has shown encouraging results in preclinical studies, where it improved neuronal survival and reduced levels of neurofilament light (NfL), a well-studied biomarker associated with neurodegeneration. The ongoing LUMINA trial is a multinational, randomized, double-blind, placebo-controlled study designed to assess the safety and biological activity of the drug. Approximately 48 participants with ALS will be randomized in a 3:1 ratio to receive either AMX0114 or a placebo through intrathecal injection once every four weeks, for up to four doses.
The trial will also monitor biomarker changes, including NfL levels, to evaluate the drug’s potential impact on disease progression.
Amylyx aims to move AMX0114 swiftly through development as part of its broader mission to bring novel therapies to patients facing neurodegenerative diseases with limited treatment options.
