Amylyx Pharmaceuticals Resumes AMX0114 ALS Clinical Trial

Amylyx Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Phase 1 trial of AMX0114, an antisense oligonucleotide targeting calpain-2 for amyotrophic lateral sclerosis (ALS). This milestone enables Amylyx to proceed with U.S. site activations for screening, enrollment, and dosing. The Phase 1 LUMINA trial, set to begin in Canada in early 2025, will evaluate the safety and biological activity of AMX0114.

The multicenter, placebo-controlled trial aims to enroll 48 ALS patients, assessing safety and ALS biomarkers like neurofilament light (NfL) levels. “Calpain-2 plays a critical role in axonal degeneration, and we’re optimistic about AMX0114’s potential for ALS and other neurological diseases,” said Dr. Camille L. Bedrosian, Chief Medical Officer of Amylyx. The company is eager to collaborate with North American clinical sites to advance research for people living with ALS.