Mitsubishi Tanabe Pharma America Inc. announced 48-week results from the global Phase 3 open-label, multi-center clinical trial (MT-1186-A01) assessing the safety and tolerability of RADICAVA ORS® (edaravone) in patients with amyotrophic lateral sclerosis (ALS). Details about the findings will be presented at the European Network to Cure ALS (ENCALS) Meeting 2022, being held in Edinburgh, Scotland, from June 1-3. RADICAVA ORS was approved by the U.S. Food and Drug Administration (FDA) on May 12, 2022, as an oral treatment for ALS.1 The FDA approval of RADICAVA ORS was supported by several studies, including 24-week results from the global Phase 3 trial (MT-1186-A01) demonstrating the safety and tolerability profile of the treatment in 185 ALS patients (aged =18 years to 75 years) across 50 sites in the U.S., Canada, Europe and Japan. According to the 48-week results presented at ENCALS, treatment-emergent adverse events (TEAEs) reported by =10% of patients were fall (22.2%), muscular weakness (21.1%), constipation (17.8%), dyspnea (10.8%), dysphagia (10.3%) and back pain (10.3%).

No serious TEAEs considered to be treatment-related by investigators were reported. Sixteen subjects (8.6%) discontinued the study due to TEAEs. The most common TEAEs leading to discontinuation were respiratory failure, muscular weakness and pneumonia, consistent with the disease state.

There were 12 deaths during the 48-week study period, and none of the deaths were related to the study drug (respiratory failure, worsening of ALS, pneumonia, acute respiratory failure, lung disorder, diabetic ketoacidosis, feeding disorder and suicide). In addition, the loss of physical function was evaluated as an Exploratory Endpoint and measured by the ALS Functional Rating Scale–Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disease in patients with ALS.3 The changes from baseline to all post-baseline visits until Week 48 in ALSFRS-R score were estimated using a mixed model for repeated measures (MMRM) analysis from 139 subjects who completed the study. At the beginning of the study, patients had an average ALSFRS-R score of 40 (SD 4.5).

At Week 48, the average change from baseline in ALSFRS-R score was -11.3 (95% CI -12.6, -10.1).