Phase III ORARIALS-01 trial of arimoclomol fails to meet endpoints in amyotrophic lateral sclerosis.- Orphazyme

Orphazyme announced that the phase III ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial. The randomized, placebo-controlled Phase III trial was conducted among 245 patients at 29 sites in 12 countries in North America and Europe. Participants were randomized (2:1 ratio) to receive either arimoclomol (248 mg three times daily) or placebo for up to 76 weeks. The primary endpoint was to determine the efficacy of chronic treatment with arimoclomol compared to placebo in participants with ALS as assessed by the combined assessment of function and survival (CAFS). Secondary endpoints included survival, change in ALSFRS-R, and slow vital capacity (SVC). Topline data will be presented at the upcoming virtual European Network to Cure ALS (ENCALS) meeting, May 12-14, and complete data from the study will be published later this year.