Amylyx’s confident data from its First-in-human LUMINA Trial

Announcement

Amylyx Pharmaceuticals, Inc., a leading company, has showcased the presentation of early tolerability and safety data from the combined of results from the running work characterizing biomarkers of AMX0114 and its Phase 1 LUMINA trial of AMX0114, a focused deal at the 36^th International Symposium on ALS/MND (MNDA) in San Diego, California, started from Decemebr 5-7 2025.

The AMX0114 was basically a tolerable one in LUMINA trial participants who participated in cohort 1 (n=12), with the absence of treatment-based serious adverse events (SAEs). Based on these data, the company hopes to start the enrolment process of the second cohort of candidates in Canada by this month and by January next year in the U.S.

Member of the Scientific Advisory Board of the Network of Excellence for ALS (NEALS), and MD, PhD, principal investigator of the LUMINA trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General Brigham, Sabrina Paganoni, said, “LUMINA is a first ever human study and we are empowered by these promising data as we move forward to modernize AMX0114 as a capable treatment for this emerging active disease with no as such treatment reached to every corner.”

“The tolerability and safety assessment enables LUMINA to continue with its next group of participants, who have already mentioned that the community has no time to wait.”

LUMINA Trial

The LUMINA trial is a shuffled, double blind, multinational, placebo-balanced and diversified ascending dose clinical trial of AMX0114. It’s an investigational, capable antisense oligonucleotide (ASO) focusing on calpain-2 in amyotrophic lateral sclerosis (ALS) individuals. The trial is assessing the pharmacodynamics, tolerability, safety and pharmacokinetics of AMX0114 biomarkers.

Including the noted fluctuation from the root line in neurofilament light chain (NfL) levels. The company is hoping to complete as much of the trial process and get the biomarker data from its first group of LUMINA candidates (n=12) by the first half of 2026.

The MD, Chief Medical Officer at Amylyx, Camille L. Bedrosian, said, “We kindly applaud this collaboration with LUMINA participants and sites to complete the enrollment process of the first cohort. Additionally, we are proud that till now no dose-limiting toxicities or drug-based SAEs have been found. This shows a major early step in this well-potent study. AMX0114 is engineered to restrain calpain-2, a calcium booster protease that is one of the important drivers of axonal degeneration and resulting disease progression in ALS.”