Cytokinetics announced the presentation of new data on its investigational drug candidate, aficamten, at the European Society of Cardiology Congress 2025 in Madrid, Spain. The data, published in the Journal of the American College of Cardiology and Heart Rhythm, further supports aficamten‘s potential for treating hypertrophic cardiomyopathy (HCM).
A pre-specified analysis from the MAPLE-HCM trial, comparing aficamten to metoprolol, showed that aficamten significantly improved diastolic function and reduced the likelihood of mitral valve issues contributing to left ventricular outflow tract (LVOT) obstruction. While aficamten led to a significant decrease in maximal wall thickness, the reduction in left ventricular mass index (LVMI) wasn’t significantly different from metoprolol. A minimal number of patients experienced a transient reduction in left ventricular ejection fraction (LVEF).
Another presentation highlighted a new analysis of atrial fibrillation (AF) incidence in patients with obstructive HCM treated with aficamten in the FOREST-HCM study, published in Heart Rhythm. Researchers found the annual incidence rate of AF to be 1.5%, consistent with expected rates in HCM patients. The clinical impact of any new-onset AF was minimal.
Further data from FOREST-HCM, an open-label extension study, confirmed the long-term safety and efficacy of aficamten. Long-term treatment showed sustained hemodynamic and clinical benefits, with a low incidence of new-onset AF and LVEF below 50%. The study also revealed significant improvements in symptoms and biomarkers of cardiac stress. Only a small percentage of patients required dose adjustments or discontinued treatment due to adverse events.
An integrated safety analysis across multiple clinical trials, including REDWOOD-HCM, SEQUOIA-HCM, MAPLE-HCM, and FOREST-HCM, provided further evidence of aficamten‘s safety and tolerability. The analysis showed a low incidence of LVEF below 50% and AF, comparable to placebo and metoprolol. No permanent treatment discontinuations were attributed to aficamten.
Cytokinetics will host an investor webcast on September 2, 2025, to discuss these results. Aficamten is currently under regulatory review in the U.S., Europe, and China. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of December 26, 2025. Aficamten remains an investigational drug and its safety and efficacy have not been fully established.
HCM, a common inherited cardiovascular disorder, causes thickening of the heart muscle, leading to reduced pumping function and various symptoms. Patients with HCM are at increased risk of serious complications, including AF, stroke, and sudden cardiac death. Aficamten‘s potential to improve exercise capacity and alleviate symptoms in HCM patients is currently under investigation.
Cytokinetics is a biopharmaceutical company focused on developing treatments for cardiovascular diseases. The company is also pursuing development of other drug candidates.
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