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Cytokinetics announced August 28, 2026, that the pivotal Phase 3 ACACIA-HCM trial of aficamten in symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM) met both of its dual primary endpoints, with results presented in a Hot Line session at the European Society of Cardiology Congress 2026 in Munich and published simultaneously in the New England Journal of Medicine. Ahmad Masri, MD, MS, of Oregon Health & Science University presented the findings.
The trial compared the cardiac myosin inhibitor against placebo in patients with symptomatic nHCM, a common form of hypertrophic cardiomyopathy for which no approved therapy targets the underlying disease. According to the company, aficamten produced statistically significant improvements from baseline to week 36 versus placebo on both the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, a patient-reported measure of symptoms and function, and peak oxygen uptake on cardiopulmonary exercise testing, a physician-assessed measure of maximal exercise capacity. The company reported that results for the dual primary endpoint analysis consistently favored aficamten across the prespecified subgroups studied, making ACACIA-HCM the first Phase 3 trial to demonstrate significant improvement on both patient-reported and physician-assessed endpoints in this population.
Aficamten, marketed as Myqorzo, is currently approved in the United States, China, the European Union, and the United Kingdom for adults with symptomatic obstructive hypertrophic cardiomyopathy, where excess contraction of the heart muscle obstructs blood flow out of the left ventricle. Nonobstructive disease produces similar symptoms without outflow obstruction, and sarcomere-directed therapy had not previously demonstrated benefit in that setting in a pivotal trial.
Cytokinetics stated that it plans to submit a supplemental New Drug Application to the US Food and Drug Administration in the fourth quarter of 2026 seeking to extend the indication to nHCM. If accepted and approved, the filing would bring the first disease-modifying option to a patient group managed to date with symptom-directed therapy, and coverage policies built around the obstructive indication would face a broader eligible population.
