medicalxpress.com
Mavacamten, a drug originally designed to treat hypertrophic cardiomyopathy (HCM), is showing potential for another challenging cardiac condition: heart failure with preserved ejection fraction (HFpEF). A recent study has highlighted its ability to reduce biomarkers of heart stress in HFpEF patients, offering new hope for a population that often struggles with limited treatment options.
In a preliminary Phase IIa study called EMBARK-HFpEF, researchers found that mavacamten significantly reduced cardiac stress biomarkers in HFpEF patients with a left ventricular ejection fraction (LVEF) of 60% or higher. The open-label study, conducted at 20 sites, included 30 symptomatic patients treated with mavacamten for 26 weeks. The drug led to a 26% reduction in N-terminal pro-B-type natriuretic peptide (a marker of heart stress) and notable decreases in high-sensitivity troponin T and I levels. These results suggest that mavacamten could relieve some of the cardiac wall stress and injury experienced by these patients.
HFpEF affects nearly half of all heart failure patients, and those with LVEF of 60% or more typically show poor response to conventional heart failure treatments. Mavacamten’s ability to reduce key biomarkers of heart stress could represent a significant therapeutic breakthrough for this population. Additionally, 41.7% of patients in the study experienced an improvement in heart failure symptoms, further underscoring the drug’s potential to alleviate the burden of HFpEF. While the safety profile appeared generally favorable, more extensive studies are needed to validate these early results.