The first patients to complete the phase 2 clinical trial of AMX0035 (Amylyx; Cambridge, MA), a combination of phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), for amyotrophic lateral sclerosis (ALS) have elected to continue treatment in an open-label extension of the CENTAUR study. This extension will have no placebo comparator, and all patients who complete the main phase of the study are eligible to participate.
Both PB and TUDCA, used individually, have shown efficacy in cellular and animal models of ALS and met safety and tolerability endpoints in ALS clinical trials. Amylyx has demonstrated a synergistic effect between the 2 compounds in preclinical trials, suggesting that the combination may have improved efficacy compared to the individual agents.
The CENTAUR study is a phase 2 clinical trial that has enrolled 132 patients with ALS and has primary objectives of evaluating the safety and tolerability of AMX0035 and assessing the impact of the drug on progression of ALS as measured by the revised ALS functional rating scale (ALSFRS-R) over a 24-week period. It is also the first trial to use patient’s muscle strength as an objective measure using a device called accurate test of limb isometric strength (ATLIS). The CENTAUR study is also assessing potential blood and imaging biomarkers of ALS.
Amylyx CEO and co-founder, Joshua Cohen commented, “We are excited to offer this extension to the participants in this study and are so grateful for their commitment.” Justin Klee, President and co-founder added, “Thank you also to The ALS Association, ALS Finding a Cure, the Northeast ALS Consortium, the Neurological Clinical Research Institute at MGH, and the ALS community for their outstanding support in helping us offer this open label extension.”Next Story