Amylyx Pharmaceuticals announces publication of CENTAUR survival data in Muscle and Nerve journal demonstrating a significant survival benefit from AMX 0035 in ALS patients.

Amylyx Pharmaceuticals, Inc.,announced the publication of an overall survival analysis from the CENTAUR trial evaluating AMX 0035 in people with ALS in the neuromuscular disease journal Muscle & Nerve . As previously reported in the New England Journal of Medicine, the CENTAUR study also met its primary efficacy end point of slowing ALS as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) versus placebo over a six-month period. AMX 0035 was generally well tolerated with a manageable safety profile. These sister publications highlight that administration of AMX 0035 resulted in both functional and survival benefits in people with ALS. Survival Results : Survival analyses compared time to death (all-cause mortality) between participants originally randomized to AMX 0035 and those originally randomized to placebo, with the longest follow-up 35 months after randomization. There was a 44% risk reduction for death over the course of follow-up (HR, 0.56; 95% CI, 0.34–0.92; P=0.023). Survival benefit of AMX 0035 was independent of baseline use of riluzole, and/or edaravone, or both together. Participants in CENTAUR were given the option after the trial to enroll in an OLE study to receive AMX 0035. 92% of participants who completed CENTAUR elected to enroll in the extension study. Long-term functional and safety data from the CENTAUR and OLE studies are also being submitted to a peer reviewed journal and planned for publication in the near future. Interim data from the ongoing extension study will be presented in 2020. See-"Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS"-Timothy Miller, M.D., Ph.D., Merit Cudkowicz, M.D., et al.July 9, 2020.N Engl J Med 2020; 383:109-119 DOI: 10.1056/NEJMoa2003715. See-"LongTerm Survival of Participants in the CENTAUR Trial of Sodium Phenylbutyrate-Taurursodiol in ALS"- Sabrina Paganoni MD, PhD Suzanne Hendrix PhD Samuel P. Dickson PhD Newman Knowlton MS et al. First published: 16 October 2020 https://doi.org/10.1002/mus.27091 (Muscle and Nerve).