Key takeaways:
- Aficamten became the first cardiac myosin inhibitor to show benefit in patients with nonobstructive hypertrophic cardiomyopathy.
- In the ACACIA-HCM trial, aficamten improved HF symptoms and peak VO2.
Aficamten was associated with improvement in heart failure symptoms and exercise capacity in patients with nonobstructive hypertrophic cardiomyopathy, according to topline results of the ACACIA-HCM trial.
The results mean that aficamten (Myqorzo, Cytokinetics) is the first cardiac myosin inhibitor to show benefit in patients with nonobstructive HCM. As Healio previously reported, the ODYSSEY-HCM trial did not show a benefit for mavacamten (Camzyos, Bristol Myers Squibb) in this population.
“Patients with nonobstructive HCM have no therapies approved to treat the underlying hypercontractility associated with the disease. We hope that will change with ACACIA-HCM, which is the first clinical trial to demonstrate statistically significant improvements in exercise capacity and symptom burden in patients with nonobstructive HCM,” Fady I. Malik, MD, PhD, executive vice president of research and development at Cytokinetics, said in a press release issued by Cytokinetics. “We believe that the totality and consistency of evidence favoring aficamten across multiple patient-reported and physician-assessed endpoints of symptom improvement and physical function are clinically meaningful for patients with nonobstructive HCM.”
For the ACACIA-HCM trial, researchers enrolled 516 participants with nonobstructive HCM and randomly assigned them to aficamten or placebo.
Each patient in the aficamten group received up to four doses, starting at 5 mg, and were escalated based on echocardiographic findings at weeks 2, 4 and 6, the company stated in the release. The dose was escalated if left ventricular ejection fraction was 60% or more and deescalated if LVEF was less than 50%, according to the release.
Aficamten met its dual primary endpoints, with significant improvement compared with placebo at 36 weeks in both Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (least square mean difference = 3; 95% CI, 0.5-5.5; P = .021) and peak VO2 (least square mean difference = 0.67; 95% CI, 0.22-1.1; P = .003), according to the release.
The company reported significant improvement vs. placebo in secondary endpoints including NYHA functional class, composite z score of ventilatory efficiency and peak VO2, and N-terminal pro-B-type natriuretic peptide (P for all < .001), and no new safety signals.
“We look forward to presenting the results from ACACIA-HCM at an upcoming medical meeting, as well as discussing them with the U.S. FDA and other regulatory authorities,” Malik said in the release.
As Healio previously reported, the FDA approved aficamten in December 2025 to improve exercise capacity and HF symptoms in adults with symptomatic obstructive HCM.
Sources/Disclosures
Source:
Press Release
Disclosures:
Malik reports being an employee of Cytokinetics.
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