Amylyx will present data from its first-in-human, dose-ranging Phase 1 LUMINA trial of AMX0114 at the European Network to Cure ALS Meeting 2026, held 24-26 June in Madrid.
AMX0114 was generally well tolerated in Cohort 1 (12.5 mg) with no drug-related serious adverse events and no serious neurological adverse events, findings the company says support escalation to higher dose levels in the multiple ascending dose trial.
Biomarker measures, including spectrin breakdown product 145 (SBDP-145), neurofilament light (NfL) and phosphorylated neurofilament heavy (pNFH), remained near baseline in Cohort 1.
Cohorts 1 (12.5 mg) and 2 (25 mg) are fully enrolled, Cohort 3 (50 mg) is enrolling, and the study is anticipated to include four dose levels. LUMINA is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose trial planned to enroll approximately 48 adults, randomized 3:1 to AMX0114 or placebo with intrathecal dosing once every four weeks for up to four doses.
AMX0114 holds U.S. FDA Fast Track designation for ALS, and the company claims preclinical data showed dose-dependent reductions in CAPN2 mRNA and calpain-2 protein, improved neuronal survival, and reductions in extracellular NfL in disease models.
The poster (PO119) was presented on 24 June 2026, and the presentation will be posted on Amylyx’s events page.
