Clinical Trial Outcomes for Cardiac Myosin Inhibitors in oHCM Treatment


An expert in cardiology shares clinical trial findings of emerging and approved cardiac myosin inhibitors for the treatment of oHCM.

This is a video synopsis of a discussion involving James L. Januzzi, MD, providing insights from clinical trials investigating cardiac myosin inhibitors in obstructive hypertrophic cardiomyopathy (HCM). These trials have shown profound effects on left ventricular outflow tract (LVOT) gradient, a key mechanism contributing to symptoms in HCM patients.

The landmark EXPLORER-HCM trial examined mavacamten, the first cardiac myosin inhibitor approved for obstructive HCM. In this trial, individuals with an LVOT gradient ≥50 and heart failure symptoms (NYHA class II-III) were randomized to receive either mavacamten or placebo for 30 weeks. Mavacamten demonstrated significant improvements in peak oxygen consumption (a marker of exercise capacity), LVOT gradient reduction, symptom severity, and health status compared to placebo. Notably, safety profiles were favorable, with no exaggerated drop in ejection fraction observed.

The VALOR-HCM study further explored the benefits of mavacamten in patients referred for septal reduction therapy. Those treated with mavacamten showed a marked reduction in the perceived need for septal reduction therapy compared to placebo, emphasizing its potential as an alternative therapeutic option.

Additionally, the newer cardiac myosin inhibitor, aficamten, showed promising results in the Redwood trial, reducing LVOT gradients and improving symptoms without significant safety concerns. Its shorter half-life and lack of drug interactions may offer additional advantages for patients with obstructive HCM.

Overall, these trials underscore the efficacy and safety of cardiac myosin inhibitors in managing obstructive HCM, providing hope for improved outcomes and quality of life for affected individuals.

Video synopsis is AI-generated and reviewed by HCPLive® editorial staff.

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