BMS Reports Positive Phase 3 SCOUT-HCM Results for Camzyos in Teens with oHCM
Bristol Myers Squibb today announced positive topline results from SCOUT-HCM, a Phase 3 trial evaluating Camzyos (mavacamten) in the first study of a cardiac myosin inhibitor (CMI) in adolescents (ages 12 years to <18 years) with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The trial met its primary endpoint, demonstrating a statistically significant reduction from baseline in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 versus placebo, indicating Camzyos was effective in improving LVOT obstruction. Statistical significance was also met for multiple secondary endpoints, including those for clinically meaningful aspects of the disease.
Safety results in the trial were consistent with the established safety profile of Camzyos in adults, and no new safety signals were reported in this new, younger population. The study continues with active treatment and long-term extension periods.
“Adolescent oHCM is a serious, rare disease associated with substantial morbidity and mortality. The SCOUT-HCM topline results highlight the potential for Camzyos to be the first cardiac myosin inhibitor for adolescent patients with oHCM,” said Cristian Massacesi, MD, executive vice president, Chief Medical Officer and Head of Development, Bristol Myers Squibb. “Camzyos has redefined the treatment paradigm for symptomatic oHCM in adults, with over 20,000 patients started in the U.S. alone, and we look forward to the potential opportunity to transform clinical care in the adolescent patient population.”
“The SCOUT-HCM study is important for patients and the field of pediatric cardiology, being one of the very few pediatric cardiology randomized and placebo-controlled clinical trials that has generated positive Phase 3 results,” said Joseph Rossano, MD, Principal Investigator and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia. “Treatment options for adolescents with oHCM are currently limited to medical symptom management or invasive surgery. As a clinician who has cared for patients in this field for decades, I am very excited about the potential opportunity that a therapy like this could hold for the patient population if approved by the FDA.”
Bristol Myers Squibb will work with key investigators to present detailed results at an upcoming medical congress, as well as discuss these data with health authorities.
Camzyos is supported by the largest global clinical trial and real-world evidence base in the CMI class, reinforcing its role in transforming care for symptomatic oHCM in adults by reducing hypercontractility through inhibition of excess myosin-actin cross-bridges in the sarcomere.
Bristol Myers Squibb thanks the patients and their caregivers, investigators, and trial sites who participated in this clinical trial.
About the Phase 3 SCOUT-HCM Trial
SCOUT-HCM (NCT06253221) is a Phase 3 randomized, double-blind, placebo-controlled international trial that enrolled 44 adolescent patients (12 years to <18 years old) with symptomatic oHCM. The trial includes three treatment periods totaling up to 200 weeks: a 28-week placebo-controlled period, followed by a 28-week active-treatment period (when patients randomized to placebo crossed over to Camzyos) and an open-label long-term extension period for up to 144 weeks.
The primary endpoint is change from baseline to Week 28 in Valsalva LVOT gradient. Secondary endpoints include efficacy parameters of resting and post-exercise LVOT gradients, peak oxygen consumption, symptoms, and health status, plus safety and pharmacokinetic parameters.
Joseph Rossano, MD, is a paid consultant to Bristol Myers Squibb and served as the site principal investigator for the SCOUT-HCM Study at Children’s Hospital of Philadelphia.
About Obstructive Hypertrophic Cardiomyopathy (oHCM) in Adolescent Patients
Hypertrophic cardiomyopathy (HCM) is a primary cardiac disorder that may result from known or suspected genetic defects in sarcomeric proteins of the cardiac myocyte or be due to unknown reasons (idiopathic). Adolescents with obstructive HCM suffer substantial morbidity largely related to reduced exertional tolerance. Though available treatments can lead to improvement in symptoms, they have significant limitations for adolescent patients (e.g., side effects from beta-blockers and risks of invasive procedures).
About CAMZYOS® (mavacamten)
CAMZYOS® (mavacamten) is the most extensively studied cardiac myosin inhibitor (CMI), approved by regulatory bodies in more than 50 countries and regions across five continents worldwide. In the U.S., CAMZYOS is indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. In the European Union, CAMZYOS is indicated for the treatment of symptomatic (NYHA, class II-III) oHCM in adult patients.
A selective, reversible, allosteric inhibitor of cardiac myosin, CAMZYOS targets the pathophysiology of HCM by reducing the dynamic LVOT obstruction and improving cardiac filling pressures in oHCM patients. These effects translate to improvement in symptoms for patients with symptomatic oHCM, enabling them to be more active in their daily lives. CAMZYOS can be used with or without background therapies, including for newly diagnosed patients.
CAMZYOS is supported by the largest body of worldwide evidence in the CMI treatment class, demonstrating an ability to reduce obstruction, improve symptoms, and impact cardiac structure, reinforced by long-term extension data through nearly four years of follow-up. CAMZYOS has been prescribed by more than 4,000 HCPs to more than 20,000 patients in the U.S. alone.
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