Cytokinetics Announces Discontinuation Of ALS Drug Trial
The biopharmaceutical company says it is “extremely disappointed” with the results of the COURAGE-ALS study, which failed to meet primary or secondary endpoints.
Despite all of the attention that amyotrophic lateral sclerosis (ALS) receives in the general population—most notably from the popular “ALS ice bucket challenge” from a few years ago—no cure has been found, and even general treatment is difficult because scientists and doctors are still largely unsure of ALS’s root causes.
Approximately 6300 individuals in the United States are diagnosed with the degenerative muscle disease each year, with a majority of those patients living fewer than 5 years after they are diagnosed.
A potential treatment for ALS, a drug called reldesemtiv, showed promise in phase 1 and phase 2 trials, but met the criteria for futility in a phase 3 trial, leading to the study’s discontinuation, biopharma company Cytokinetics said in a press release.1
“We are extremely disappointed with this outcome,” said Cytokinetics CEO and President Robert I. Blum in a statement. “And would like to thank the people with ALS, caregivers, investigators and clinical trial staff for their participation in COURAGE-ALS.”
According to Bio Space2 and past Cytokinetics press releases3, the drug also failed to reach endpoints in phase 2 of its progression—2019’s FORTITUDE-ALS trial—but was pushed through to phase 3 due to positive post hoc analysis.
During phase 3, it was shown that reldesemtiv “could not significantly improve disease severity” so a data monitoring committee recommended study be discontinued, a recommendation that Cytokinetics will honor. The trial consisted of 555 patients with ALS, and was “randomized 2:1 to receive 300 mg of reldesemtiv or matching placebo dosed orally twice daily for 24 weeks, followed by a 24-week period in which all patients received 300 mg of reldesemtiv twice daily.” All participants were within 2 years of their original sign of muscle weakness, the most common symptom of ALS.
The intended goal of reldesemtiv was “to slow the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers,” in order to increase muscle contractility and control.
This news comes on the heels of Cytokinetics’ candidate for heart failure, omecamtiv mecarbil, being rejected for a New Drug Application by the FDA due to lack of evidence of efficacy.
According to its website, Cytokinetics is “a late-stage biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which muscle performance is compromised.”
Currently, the most common drug used for ALS treatment is riluzole, a liquid formulation taken twice a day. It has been effective in prolonging the life expectancy of patients with ALS, but no treatment has been discovered that will stop or reverse ALS and its symptoms.
“Cytokinetics has been committed to the ALS community for more than a decade and recognizes the urgency to bring new potential medicines to the forefront for this grievous disease,” Cytokinetics said. “In the coming months, we will assess next steps relating to our neuromuscular development programs.”
References
1. Cytokinetics announces COURAGE-ALS met criteria for futility at second interim analysis. Press release. Cytokinetics. March 31, 2023. Accessed April 5, 2023. https://ir.cytokinetics.com/news-releases/news-release-details/cytokinetics-announces-courage-als-met-criteria-futility-second
2. Phase III failure leaves Cytokinetics with one last late-stage hopeful. Bio Space. April 3, 2023. Accessed April 5, 2023. https://www.biospace.com/article/phase-iii-failure-leaves-cytokinetics-with-one-last-late-stage-hopeful/#:~:text=Reldesemtiv%20is%20a%20small%20molecule,calcium%20and%20improve%20muscle%20contractility.
3. Cytokinetics announces results of FORTITUDE-ALS, a phase 2 clinical trial of reldesemtiv in patients with ALS, presented at American Academy of Neurology Annual Meeting. Press release. May 6, 2019. Accessed April 5, 2023. https://ir.cytokinetics.com/news-releases/news-release-details/cytokinetics-announces-results-fortitude-als-phase-2-clinical
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