Phase 3 study with cardiac myosin inhibitor in obstructive hypertrophic cardiomyopathy meets primary endpoint
The VALOR-HCM study met its primary endpoint at week 16.
VALOR-HCM is a phase 3, randomized, double-blind, placebo-controlled, multicenter study that evaluates the effect of the cardiac myosin inhibitor mavacamten in patients with symptomatic, obstructive hypertrophic cardiomyopathy (NYHA class III-IV) who meet guideline criteria for septal reduction therapy (SRT) and have been referred for an invasive procedure.
The study randomized more than 100 patients in a 1:1 ratio to receive mavacamten or placebo. The study has three treatment periods: a 16-week placebo-controlled period, a 16-week active treatment period where all patients will receive mavacamten and a 96-week long-term extension period where all patients will continue to receive mavacamten.
The primary endpoint is the composite outcome of the number of patients who decide to proceed with SRT prior to or at week 16 and the number of patients who remain SRT-guideline eligible (LVOT gradient of ≥50mmHg and NYHA Class III-IV) at week 16 in the mavacamten group compared with the placebo group. Key secondary endpoints are the impact on exercise gradient LVOT, NYHA Class, Kansas City Cardiomyopathy Questionnaire (KCCQ) and biomarkers at week 16.
Mavacamten is an investigational therapy. It is a selective cardiac myosin inhibitor that targets the underlying pathophysiology of obstructive hypertrophic cardiomyopathy. Mavacamten has previously been shown to reduce cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance.
Results from VALOR-HCM will be presented at the American College of Cardiology 71st Annual Scientific Session & Expo in April 2022.