Shares of Cytokinetics, Incorporated (CYTK) moved higher by 23% after the company announced positive topline results from ACACIA-HCM, its pivotal Phase 3 clinical trial evaluating Aficamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
The study met both dual primary endpoints, showing statistically significant improvements in symptoms and exercise capacity compared with placebo.
Non-obstructive HCM is a chronic cardiac condition in which the heart muscle becomes abnormally thick, impairing its ability to relax and fill properly. Unlike obstructive HCM, there are no approved therapies that directly target the underlying hypercontractility in nHCM.
ACACIA-HCM was a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial enrolling 516 patients with symptomatic nHCM. Participants received Aficamten or placebo for 36 weeks, with dose adjustments guided by echocardiography.
The trial met its co-primary endpoints, demonstrating statistically significant improvements from baseline to Week 36 in both:
- Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)
- maximal exercise performance (pVO2) compared with placebo.
Aficamten also achieved statistically significant improvements (p<0.001) across key secondary endpoints, including New York Heart Association (NYHA) functional class, ventilatory efficiency, pVO2 composite z-scores, and NT-proBNP levels. No new safety signals were identified.
Left ventricular ejection fraction (LVEF) <50% occurred in 10% of patients on Aficamten versus 1% on placebo, with a small number of treatment interruptions due to reduced LVEF. Two patients on Aficamten experienced serious adverse events of heart failure associated with LVEF <50%.
Cytokinetics said the results represent the first Phase 3 trial to demonstrate meaningful improvements in both exercise capacity and symptom burden for patients with non-obstructive HCM. The company plans to present full data at an upcoming medical meeting and engage with regulatory authorities regarding potential approval pathways.
Aficamten, marketed as MYQORZO for obstructive HCM, is already approved in U.S. EU, and China for adults with symptomatic obstructive hypertrophic cardiomyopathy. The ACACIA-HCM results may support expanding its use into the non-obstructive population, pending regulatory review.
CYTK has traded between $29.31 and $70.98 over the past year. The stock closed Monday's trading at $66.05, up 4.26%. In pre-market trading the stock is at $81.67, up 23.65%.
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