AMX0114 - (LUMINA)

June 23, 2026: Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) announced it will present first-in-human, dose-ranging Phase 1 LUMINA trial data for AMX0114 in amyotrophic lateral sclerosis (ALS) at the European Network to Cure ALS (ENCALS) Meeting 2026 (June 24–26, 2026; Madrid). In Cohort 1 (12.5 mg), AMX0114 showed no drug-related serious adverse events and no serious neurological adverse events; ALS-related biomarkers (including SBDP-145, neurofilament light, and pNFH) remained near baseline. The company said these findings support continued evaluation at higher doses in the multiple ascending dose study.

LUMINA (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled multiple ascending dose trial evaluating safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarkers; participants receive intrathecal dosing every four weeks for up to four doses (3:1 AMX0114:placebo). Cohorts 1 (12.5 mg) and 2 (25 mg) are fully enrolled; Cohort 3 (50 mg) is enrolling; four dose levels are planned (~48 participants total). Poster details: “Advancing AMX0114 for ALS: Updates from the LUMINA Phase 1 Study” (PO119), presented June 24, 2026, 5:20–7:00 p.m. CEST. AMX0114 has FDA Fast Track designation for ALS.