Conduct a Phase 2 trial to evaluate the safety, dose response, and preliminary efficacy of reldesemtiv for 12 weeks in patients with amyotrophic lateral sclerosis (ALS). Reldesemtiv is a drug designed to enhance muscle contractility. Patients received one of three different drug dosages (150, 300, or 450 mg) versus one group that received a placebo.
Results showed that the drug was well tolerated by patients and side effects could be mitigated. The drug efficacy was not statistically significant in terms of the ALS Functional Rating Scale-Revised (ALSFRS-R), slow upright vital capacity (SVC) and muscle strength mega-score, all metrics for evaluating ALS disease progression. However, there were trends favoring reldesemtiv for each of the three measures. The impact of reldesemtiv on patients with ALS advocates a pivotal Phase 3 trial.