Cytokinetics presented new Phase 3 and long-term extension data for MYQORZO (aficamten) at ESC Heart Failure 2026, showing improvements in exercise capacity, LVOT gradients, and cardiac biomarkers in obstructive hypertrophic cardiomyopathy patients, while reinforcing the therapy’s long-term safety and durability profile.
Written By: Nikita Jha, BPharm
Reviewed By: Pharmacally Editorial Team
Cytokinetics presented new clinical and real-world evidence for MYQORZO at the ESC Heart Failure 2026 Congress, reinforcing the therapy’s efficacy, safety, and durability in obstructive hypertrophic cardiomyopathy (oHCM). MYQORZO, a cardiac myosin inhibitor, is approved in the United States, European Union, and China for symptomatic oHCM.
New analyses from the Phase 3 MAPLE-HCM trial (NCT05767346) showed that aficamten outperformed metoprolol, a commonly used beta-blocker therapy for oHCM, across treatment doses. Patients treated with aficamten experienced improvements in exercise capacity, reductions in left ventricular outflow tract (LVOT) gradients, and lower N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels. Metoprolol did not show similar benefits regardless of dose. A separate analysis showed consistent improvements in women and men, despite women entering the study with more severe baseline disease characteristics. Both groups achieved comparable gains in peak oxygen consumption, quality-of-life scores, and cardiac biomarkers.
Data from the FOREST-HCM long-term extension (NCT04848506) further supported the safety profile of aficamten. Among 122 patients with ambulatory electrocardiogram data, treatment for up to 96 weeks did not increase arrhythmia incidence, including in patients who discontinued beta-blocker therapy. Rates of non-sustained ventricular tachycardia remained stable, with no increase in atrial fibrillation episodes or newly identified subclinical atrial fibrillation. Investigators noted that this represents the first prospective ambulatory ECG analysis conducted in patients receiving a cardiac myosin inhibitor.
Additional extension data from Chinese patients with symptomatic oHCM showed aficamten was well tolerated through 48 weeks. No serious or severe treatment-emergent adverse events, reductions in left ventricular ejection fraction below 50%, or treatment discontinuations were reported. Patients also experienced durable reductions in resting and Valsalva LVOT gradients, alongside improvements in New York Heart Association functional class, Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, and NT-proBNP levels.
Expanded findings from the SEQUOIA-HCM pivotal study (NCT05186818) showed that aficamten significantly improved left atrial reservoir and conduit strain compared with placebo, while also reducing left atrial volume index. Researchers said the findings suggest aficamten may improve atrial mechanics in addition to relieving LVOT obstruction.
The company also presented real-world studies highlighting the burden of hypertrophic cardiomyopathy. A multinational survey found that patients with obstructive HCM reported greater activity impairment than those with non-obstructive disease. Another U.S. analysis showed that symptom burden, healthcare use, and quality-of-life impairment increased with worsening New York Heart Association class.
Stephen Heitner, Senior Vice President of Clinical Research and Development at Cytokinetics, said the findings expand scientific understanding of oHCM and further support the clinical profile of MYQORZO in clinical practice.
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About the Writer
Nikita Jha, BPharm (LinkedIn) a pharmacy graduate specializing in medical writing, with a strong ability to interpret complex medical and regulatory information and translate it into clear, accurate, and evidence-based healthcare content. Known for her attention to detail and precision, she focuses on delivering high-quality scientific communication that supports drug safety and informed decision-making.