Bristol Myers Squibb announced positive topline data from the Phase 3 SCOUT-HCM trial evaluating Camzyos (mavacamten) in adolescents aged 12 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The study is the first to test a cardiac myosin inhibitor in this pediatric population and met its primary endpoint, showing a clinically meaningful and statistically significant reduction from baseline in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 versus placebo, with a least-squares mean difference of −48.0 mm Hg (95% CI: −67.7, −28.3; P < 0.0001).
Camzyos also demonstrated meaningful improvement over placebo in multiple secondary endpoints at 28 weeks, including LV obstruction, diastolic function, maximal left ventricular wall thickness, New York Heart Association class, and mitral valve dysfunction. Improvements were seen in resting and post-exercise LVOT gradients, with LS mean differences of −47.0 mm Hg (nominal p < 0.0001) and −41.7 mm Hg (nominal p < 0.0001), respectively. Positive structural changes included reductions in maximal LV wall thickness and E/e’ ratio.
The safety profile of Camzyos in adolescents was similar to that established in adults, with no new safety signals. Treatment-emergent adverse events and serious adverse events were comparable between Camzyos and placebo groups, and there were no treatment discontinuations, deaths, cases of atrial fibrillation or symptomatic heart failure, or left ventricular ejection fraction below 50%.
Investigators noted the lack of approved therapies for pediatric oHCM and highlighted the results as a significant advance with the potential to deliver the first targeted pharmacological option for adolescents if approved. Bristol Myers Squibb plans to present 56-week data from the ongoing trial at a future medical congress.
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