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Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA Trial
- AMX0114 was generally well-tolerated, with no treatment-related serious adverse events - Amylyx will proceed with opening enrollment of second cohort -
About this update from Amylyx Pharmaceuticals, Inc.
[{"type":"text","content":"\n- AMX0114 was generally well-tolerated, with no treatment-related serious adverse events\n\n- Amylyx will proceed with opening enrollment of second cohort\n\n- Data are being presented at the 36th International Symposium on ALS/MND\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAmylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the presentation of early safety and tolerability data from its Phase 1 LUMINA trial of AMX0114 and results from ongoing work characterizing biomarkers of AMX0114 target engagement at the 36th International Symposium on ALS/MND (MNDA) held from December 5-7 in San Diego, California. AMX0114 was generally well-tolerated in LUMINA trial participants enrolled in cohort 1 (n=12), with no treatment-related serious adverse events (SAEs). Based on these data, Amylyx expects to begin enrolling the second cohort of participants in Canada later this month and in the U.S. in January.\n\n“LUMINA is a first-in-human study, and we are encouraged by these data as we continue to advance AMX0114 as a potential treatment for this rapidly progressive disease with high unmet need. The safety and tolerability analysis allows LUMINA to proceed with its next cohort of participants, which is critical given that this community has no time to wait,” said Sabrina Paganoni, MD, PhD, principal investigator of the LUMINA trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General Brigham, and member of the Scientific Advisory Board of the Network of Excellence for ALS (NEALS).\n\nThe LUMINA trial (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial of AMX0114 – an investigational, potent antisense oligonucleotide (ASO) targeting calpain-2 – in people living with amyotrophic lateral sclerosis (ALS). LUMINA is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS and assessing both novel and broadly researched ALS biomarkers, including change from baseline in neurofilament light chain (NfL) levels. Amylyx expects biomarker data from the first cohort of LUMINA participants (n=12) in the first half of 2026.\n\n“We appreciate the partnership with LUMINA sites and participants to achieve complete enrollment of the first cohort. In addition, we are pleased that to dat...