Global pharma powerhouse Bristol Myers Squibb has reported positive Phase 3 results for Camzyos (mavacamten), marking the first successful trial of a cardiac myosin inhibitor (CMI) in adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
The SCOUT-HCM trial hit its primary endpoint, showing a clinically meaningful and statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28. Camzyos reduced the gradient by an LS mean difference of −48.0 mm Hg compared to placebo.
The drug also improved multiple secondary endpoints, including LV obstruction, diastolic function, left ventricular wall thickness, NYHA class, and mitral valve dysfunction, while demonstrating a safety profile comparable to placebo.
“Pediatric HCM is a rare cardiac disorder that is associated with severe, sometimes life-threatening, symptoms,” said Joseph Rossano, Principal Investigator of SCOUT-HCM and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia.
“With no approved therapies for pediatric patients with oHCM and current recommendations for pharmacological therapy primarily extrapolated from evidence obtained from adult studies, the positive results of this trial represent a significant advance in the field of pediatric cardiology and the potential for a meaningful new therapy for adolescent patients if approved by the FDA.”
The trial enrolled 44 patients aged 12 to <18 with symptomatic oHCM and NYHA class II-III symptoms over 28 weeks. Camzyos delivered significant improvements in both resting and post-exercise LVOT gradients and structural heart measures, including maximal LV wall thickness and E/e’ ratio.
“The SCOUT-HCM results underscore the potential for Camzyos to become the first CMI for adolescents, reinforcing our leadership in the CMI space and our role in reshaping the scientific understanding of oHCM and how the disease is diagnosed, evaluated and potentially treated,” said Cristian Massacesi, Executive VP, Chief Medical Officer and Head of Development, Bristol Myers Squibb.
“With these meaningful safety and efficacy data, we are excited about the potential to provide a paradigm-changing treatment for adolescents and their families.”
Safety outcomes were similar between Camzyos and placebo, with no treatment-related discontinuations or deaths, no atrial fibrillation or symptomatic heart failure, and no patient experiencing LVEF <50%.
The 28-week treatment phase is ongoing, with 56-week data expected to be presented at a future medical congress.
