Cytokinetics presents results from COURAGE-ALS study

Cytokinetics announced that the full results of COURAGE-ALS, were presented at the 34th International Symposium on ALS/MND. At the second interim analysis the Data Monitoring Committee recommended the discontinuation of the clinical trial due to futility. Subsequently, the Company concluded study conduct in March 2023 and discontinued treatment with reldesemtiv in all patients including those in the open-label extension study, COURAGE-ALS OLE. Development of reldesemtiv has been terminated. At the time of the discontinuation of COURAGE-ALS, 486 patients had started treatment with reldesemtiv or placebo and 276 had completed dosing through 24 weeks. Treatment with reldesemtiv for 24 weeks had no effect on the primary efficacy endpoint measure of change from baseline up to Week 24 in the ALS Functional Rating Scale Revised. Patients treated with reldesemtiv declined 5.3 points per month while patients treated with placebo declined 4.8 points per month. No pre-defined patient subgroup favored treatment with reldesemtiv. Patients with a faster disease progression rate did not experience a greater treatment effect from reldesemtiv, contrary to analyses conducted post hoc from FORTITUDE-ALS, the Phase 2 clinical trial of reldesemtiv, which had suggested that treatment effects were more evident in patients with a faster disease progression rate. Reldesemtiv also demonstrated no effect on key secondary endpoints including change from baseline to Week 24 in in-clinic percent predicted forced vital capacity, ALS Questionnaire 40 and handgrip strength. The incidence of serious adverse events in COURAGE-ALS was similar between the placebo group and the reldesemtiv group. The most common treatment-emergent adverse events were in system organ classes including respiratory, thoracic and mediastinal disorders, gastrointestinal disorders and infections and infestations. Overall, non-serious TEAEs also occurred at similar frequency between the placebo group and the reldesemtiv group. The only non-serious TEAE that differed between the placebo and reldesemtiv groups was abnormal laboratory investigations driven primarily by elevated transaminases, which occurred in 18.8% of patients treated with reldesemtiv and 11.8% of patients treated with placebo.