Bristol Myers Squibb has reported positive topline results from its Phase 3 SCOUT-HCM trial evaluating Camzyos (mavacamten) in adolescents aged 12 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
The study met its primary endpoint, demonstrating a statistically significant reduction in left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo, indicating improved cardiac blood flow in treated patients.
The trial also achieved statistical significance across multiple secondary endpoints, including measures linked to clinical status, exercise capacity, symptoms, and health-related quality of life. Safety findings in adolescents were consistent with the established profile of Camzyos in adults, with no new safety concerns identified.
The results position Camzyos to potentially become the first cardiac myosin inhibitor approved for adolescent patients with oHCM, a rare but serious condition associated with reduced exercise tolerance and long-term cardiovascular risk.
SCOUT-HCM is a randomized, double-blind, placebo-controlled international study that enrolled 44 adolescents with symptomatic oHCM. The trial includes a 28-week placebo-controlled phase, followed by a 28-week active-treatment crossover period and a long-term open-label extension lasting up to 144 weeks.
The primary endpoint was the change in Valsalva LVOT gradient from baseline to Week 28, with secondary endpoints covering resting and post-exercise LVOT gradients, peak oxygen consumption, symptoms, health status, safety, and pharmacokinetics.
Camzyos is already approved for adult patients with symptomatic oHCM and is supported by a large global clinical and real-world evidence base. It works by reducing excessive heart muscle contractility through inhibition of abnormal myosin-actin interactions in the cardiac sarcomere.
Bristol Myers Squibb plans to present detailed SCOUT-HCM data at an upcoming medical meeting and engage with regulatory authorities to discuss next steps for potential use of Camzyos in the adolescent population.
