Impressive Phase III results from Valor-HCM study of mavacamten

US pharma major Bristol Myers Squibb (NYSE: BMY) has announced impressive results from the Phase III VALOR-HCM study, which showed the addition of mavacamten, an investigational, first-in-class cardiac myosin inhibitor, significantly reduced the need for septal reduction therapy (SRT) in patients with severely symptomatic obstructive hypertrophic cardiomyopathy (obstructive HCM) who had been appropriate for SRT per the 2011 American College of Cardiology/American Heart Association (ACC/AHA) Guidelines at baseline.

Study participants were on maximally tolerated background regimens when they entered the trial and remained on them through the duration of the study. These data were presented on Saturday as a late-breaking clinical trial at the American College of Cardiology’s 71st Annual Scientific Session.

At 16 weeks the primary and all secondary endpoints were met. Of patients treated with mavacamten, 82% had not proceeded with SRT and no longer met the criteria for SRT according to the 2011 ACC/AHA Guidelines compared to 23% of patients receiving placebo. Patients in the mavacamten arm also demonstrated reduction in left ventricular outflow tract (LVOT) gradients, improvement in New York Heart Association (NYHA) Classification, improvement in quality-of-life measures and improvement in cardiac biomarkers at a high degree of statistical significance compared to the placebo arm. No new safety signals were observed.