CAMBRIDGE, Mass.— Amylyx Pharmaceuticals has officially begun dosing participants in its Phase 1 clinical trial of AMX0114, a potential new treatment for amyotrophic lateral sclerosis (ALS). The announcement marks a key milestone in the LUMINA study, a multinational, double-blind, placebo-controlled trial that will assess the safety and biological effects of the experimental drug.
The LUMINA trial will enroll around 48 ALS patients across North America, with participants receiving intrathecal doses of AMX0114 or a placebo every four weeks for up to four doses. Early cohort data from the trial is expected later this year.
AMX0114 is a novel antisense oligonucleotide (ASO) therapy designed by Amylyx to inhibit calpain-2, an enzyme linked to the degeneration of axons — the long nerve fibers that transmit signals from neurons to muscles or other neurons. By targeting this pathway, researchers hope AMX0114 could slow the progression of ALS, a fatal neurodegenerative condition that currently has few effective treatment options.
“In preclinical studies, AMX0114 demonstrated improved neuron survival and reduced levels of neurofilament light chain, a key biomarker of neurodegeneration,” said Dr. Camille L. Bedrosian, Chief Medical Officer at Amylyx. “We are excited to bring this therapy into the clinic for people with ALS.”
The LUMINA trial will also track changes in neurofilament light chain (NfL) levels — a biomarker associated with ALS progression — to further evaluate the drug’s potential impact.
Dr. Sabrina Paganoni, principal investigator for the trial and a researcher at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, emphasized the significance of the trial’s launch: “ALS is a devastating disease with limited treatment options. AMX0114 targets one of the underlying mechanisms driving progression, and dosing the first participant in LUMINA brings us closer to a potential new therapy.”
The study is registered under clinical trial number NCT06665165.
