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- AMX0114 / Amylyx
A Phase 1, multicenter, randomized, placebo-controlled multiple ascending dose study to evaluate the safety and tolerability of AMX0114 in Amyotrophic Lateral Sclerosis (LUMINA) (Poster Hall at the Palais de Congres Montreal; 517b/c) - Nov 7, 2024 - Abstract #ALSMND2024ALS_MND_578;
Secondary endpoints will be PK concentrations, including plasma and cerebrospinal fluid (CSF) levels of AMX0114. Tertiary endpoints include plasma and CSF bio-markers as well as functional measures of ALS progression.
- || AMX0114 / Amylyx
New P1 trial: LUMINA: AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - Oct 29, 2024
P1, N=48, Not yet recruiting, Sponsor: Amylyx Pharmaceuticals Inc.
- AMX0114 / Amylyx
Update on AMX0114: An Antisense Oligonucleotide Targeting Calpain-2 a Critical Effector of Axonal Degeneration (Event Halle, Messe Basel, Ground Floor) - Nov 7, 2023 - Abstract #ALSMND2023ALS_MND_485;
In kinetic profiling experiments, AMX0114 treatment resulted in a maximum CAPN2 mRNA knockdown of 94% on Day 0 (48 hours after gymnosis) with a slight decrease over time to 77% on Day 21. A stable knockdown of calpain-2 protein was also achieved compared to the negative control ASO.