Trial Evaluating Reldesemtiv for ALS Ends After Missing Endpoints

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The trial met the criteria for futility and is being discontinued.

Cytokinetics has decided to discontinue the phase 3 COURAGE-ALS trial evaluating reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS).

Reldesemtiv is an investigational, selective, small molecule fast skeletal muscle troponin activator designed to slow the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers. This then sensitizes the sarcomere, a functional unit of skeletal muscle, to calcium, leading to an increase in muscle contractility.

The multicenter, double-blind, randomized, placebo-controlled COURAGE-ALS trial (ClinicalTrials.gov Identifier: NCT04944784) was expected to enroll 555 patients with ALS.  At the interim analysis, approximately 460 patients had been randomly assigned 2:1 to receive reldesemtiv 300mg or placebo orally twice daily for 24 weeks; over 200 patients had reached the 24-week assessment.

A decision was made to end the trial due to futility. A review of data by the Data Monitoring Committee showed no evidence of effect in patients treated with reldesemtiv based on the primary endpoint of change from baseline to 24 weeks in ALS Functional Rating Scale – Revised (ALSFRS-R) vs placebo. The study also missed its key secondary endpoints.

The Company plans to notify all regulatory agencies and clinical trial investigators of the trial findings. Full data will be presented at an upcoming medical meeting.

References:

Cytokinetics announces COURAGE-ALS met criteria for futility at second interim analysis. News release. Cytokinetics. Accessed April 6, 2023. https://ir.cytokinetics.com/news-releases/news-release-details/cytokinetics-announces-courage-als-met-criteria-futility-second.