November 10, 2025 — Leads & Copy — Cytokinetics, Incorporated (Nasdaq: CYTK) presented additional data from its MAPLE-HCM trial at the Hypertrophic Cardiomyopathy Medical Society Scientific Sessions and the American Heart Association Scientific Sessions 2025 in New Orleans, LA.
The data, which compared aficamten to metoprolol in patients with hypertrophic cardiomyopathy (HCM), was presented in three Late Breaking Science sessions. Two of the presentations were simultaneously published in the Journal of the American College of Cardiology.
According to Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development, the additional analyses from MAPLE-HCM expand on the primary finding that aficamten is superior to metoprolol on exercise capacity, with new insights into the overall treatment effect of aficamten as well as its effect on symptoms and biomarkers in comparison to metoprolol. He noted that patients treated with aficamten achieved a significantly greater number of clinical response categories compared with metoprolol, and that nearly 40% achieved significant improvements in patient reported symptoms.
One presentation by Andrew Wang, M.D., Cardiologist and Professor of Medicine, Duke University School of Medicine, detailed a pre-specified responder analysis from MAPLE-HCM. The analysis evaluated five clinically relevant measures of disease burden: complete hemodynamic response, symptom improvement, cardiac biomarker response, enhanced exercise capacity and favorable cardiac remodeling. These results were also simultaneously published in the Journal of the American College of Cardiology.
The responder analysis showed that after 24 weeks of treatment, aficamten was associated with greater improvements than metoprolol in all outcome measures (all p<0.001). The proportion of patients who had a positive response (improvement in three or four clinical parameters) or a complete response (improvement in all five clinical parameters) was 78% in those receiving aficamten vs. 3% for those receiving metoprolol (p<0.001).
Another presentation by Michael E. Nassif, M.D., Cardiologist, Saint Luke’s Mid America Heart Institute, Associate Professor of Medicine, University of Missouri Kansas City, presented results from a pre-specified sub-study of two patient reported outcome instruments in MAPLE-HCM: the Kansas City Cardiomyopathy Questionnaire (KCCQ) and Seattle Angina Questionnaire Summary Score (SAQ). The results from this analysis were also simultaneously published in the Journal of the American College of Cardiology.
At 24 weeks, treatment with aficamten resulted in significantly greater improvements than metoprolol in both KCCQ Overall Summary Score (KCCQ-OSS) (16.6 points vs. 8.9 points, respectively; between-group difference = 7.8 points [95% CI: 3.3 to 12.3; p=0.001]) and KCCQ Clinical Summary Score (KCCQ-CSS) (15.8 points vs. 8.7 points, respectively; between-group difference = 6.9 points [95% CI: 2.6 to 11.2; p=0.002]).
The study also found that Aficamten was also associated with statistically significant improvements across all KCCQ domains (p<0.05). Patients on aficamten more frequently reported very large improvements in KCCQ-OSS, defined as ≥20-point improvement (38.6% vs. 18.4%; number needed to treat (NNT)=4.9), and were significantly less likely to experience a worsening in KCCQ-OSS than those treated with metoprolol (6.8% vs 18.4%; number needed to harm (NNH)=8.6).
A third presentation by Neal K. Lakdawala, M.D., Cardiovascular Medicine, Brigham and Women’s Hospital, Harvard Medical School, showed results from a pre-specified supplemental analysis from MAPLE-HCM of the impact of treatment with aficamten compared to metoprolol on the cardiac biomarkers NT-proBNP and high sensitivity cardiac troponin I (hs-cTnI).
The analysis showed that after treatment for 24 weeks, aficamten was associated with a 73% reduction (p<0.001) from baseline in NT-proBNP compared to an increase of 42% observed in patients receiving metoprolol (-81% treatment effect, p<0.001). Similarly, aficamten was associated with a 43% reduction in hs-cTnI, compared to a 17% decrease for metoprolol (-28% treatment effect, p=0.001).
Aficamten is an investigational selective, small molecule cardiac myosin inhibitor designed to reduce myocardial hypercontractility associated with HCM. It is currently under regulatory review in the U.S, where the FDA is reviewing a New Drug Application (NDA) for aficamten with a Prescription Drug User Fee Act (PDUFA) target action date of December 26, 2025. Additionally, the European Medicines Agency (EMA) is reviewing a Marketing Authorization Application (MAA) for aficamten, and The Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) is reviewing an NDA for aficamten with Priority Review.
HCM is a disease in which the heart muscle becomes abnormally thick, leading to reduced exercise capacity and symptoms including chest pain, dizziness, shortness of breath, or fainting during physical activity.
Cytokinetics is a specialty cardiovascular biopharmaceutical company advancing a pipeline of potential new medicines for patients suffering from diseases of cardiac muscle dysfunction. Cytokinetics is readying for potential regulatory approvals and commercialization of aficamten, following positive results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy (HCM). Aficamten is also being evaluated in additional clinical trials enrolling patients with obstructive and non-obstructive HCM. In addition, Cytokinetics is developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), ulacamten, a cardiac myosin inhibitor with a mechanism of action distinct from aficamten, for the potential treatment of heart failure with preserved ejection fraction (HFpEF) and CK-089, a fast skeletal muscle troponin activator with potential therapeutic application to a specific type of muscular dystrophy and other conditions of impaired skeletal muscle function.
Diane Weiser, Senior Vice President, Corporate Affairs, can be reached at (415) 290-7757.
Source: Cytokinetics, Incorporated
