{"id":62862,"date":"2026-03-29T19:02:43","date_gmt":"2026-03-29T17:02:43","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/bristol-myers-squibb-presents-positive-results-from-phase-3-scout-hcm-trial-demonstrating-efficacy-and-safety-of-camzyos-mavacamten-in-adolescents-with-symptomatic-obstructive-hypertrophic-cardiomyo\/"},"modified":"2026-03-29T19:02:43","modified_gmt":"2026-03-29T17:02:43","slug":"bristol-myers-squibb-presents-positive-results-from-phase-3-scout-hcm-trial-demonstrating-efficacy-and-safety-of-camzyos-mavacamten-in-adolescents-with-symptomatic-obstructive-hypertrophic-cardiomyo","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/bristol-myers-squibb-presents-positive-results-from-phase-3-scout-hcm-trial-demonstrating-efficacy-and-safety-of-camzyos-mavacamten-in-adolescents-with-symptomatic-obstructive-hypertrophic-cardiomyo\/","title":{"rendered":"Bristol Myers Squibb Presents Positive Results from Phase 3 SCOUT-HCM Trial Demonstrating Efficacy and Safety of Camzyos (mavacamten) in Adolescents with Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM)"},"content":{"rendered":"
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\nTrial met its primary endpoint, demonstrating clinically meaningful and statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28<\/i><\/b><\/p>\n

\nResults demonstrate the potential for <\/i>Camzyos to be the first targeted pharmacological therapy for the treatment of oHCM in adolescents<\/i><\/b><\/p>\n

\nSafety profile of <\/i>Camzyos in adolescents was similar to established profile in adults, with no new safety signals and no patients experiencing left ventricular ejection fraction (LVEF) of <50%<\/i><\/b><\/p>\n

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY<\/a> #ACC<\/a>—Bristol Myers Squibb<\/a> (NYSE: BMY) today announced positive data from the Phase 3 SCOUT-HCM trial of Camzyos <\/i>(mavacamten), the first study of a cardiac myosin inhibitor (CMI) in adolescents (ages 12 years to <18 years) with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The SCOUT-HCM trial met its primary endpoint, demonstrating a clinically meaningful and statistically significant reduction from baseline in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 with Camzyos <\/i>versus placebo, with a significant least-squares (LS) mean difference (95% CI) at Week 28 of \u221248.0 (\u221267.7, \u221228.3) mm Hg; P < 0.0001.<\/p>\n

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\nAdditionally, Camzyos <\/i>showed meaningful improvement over placebo in multiple secondary endpoints at 28 weeks, and similar safety findings were observed in the Camzyos<\/i> and placebo groups. The data are being presented as a late-breaking clinical trial presentation at the American College of Cardiology\u2019s (ACC) Annual Scientific Session & Expo 2026, with simultaneous publication in The New England Journal of Medicine<\/i>.<\/p>\n

\n\u201cPediatric HCM is a rare cardiac disorder that is associated with severe, sometimes life-threatening, symptoms,\u201d said Joseph Rossano, MD, Principal Investigator of SCOUT-HCM and Chief of the Division of Cardiology at Children\u2019s Hospital of Philadelphia. \u201cWith 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.\u201d<\/p>\n

\nSCOUT-HCM evaluated Camzyos<\/i> compared to placebo in 44 patients aged 12 to <18 years old with symptomatic oHCM and New York Heart Association (NYHA) class II-III symptoms over a 28-week period. In addition to meeting the primary endpoint of reduction of Valsalva LVOT gradient, Camzyos<\/i> showed meaningful improvements over placebo in LV obstruction, diastolic function, maximal left ventricular wall thickness, NYHA class, and mitral valve dysfunction at 28 weeks.<\/p>\n

\n\u201cThe SCOUT-HCM results underscore the potential for Camzyos<\/i> 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,\u201d said Cristian Massacesi, MD<\/a>, executive vice president, Chief Medical Officer and Head of Development, Bristol Myers Squibb. \u201cWith these meaningful safety and efficacy data, we are excited about the potential to provide a paradigm-changing treatment for adolescents and their families.\u201d<\/p>\n

\nImprovements were seen in resting and post-exercise LVOT gradients with Camzyos<\/i> versus placebo, with an LS mean difference of \u221247.0 mm Hg (\u221262.7, \u221231.4); nominal p < 0.0001, and \u221241.7 mm Hg (\u221259.7, \u221223.7); nominal p < 0.0001, respectively. The primary endpoint was supported by the proportion of patients who achieved reduction in maximal LVOT gradient (resting or Valsalva) to <30 mm Hg, from baseline to 28 weeks.<\/p>\n

\nAdditionally, positive structural changes were observed with Camzyos <\/i>versus placebo, as shown by improvement in maximal LV wall thickness LS mean difference (95% CI) of \u22121.8 (\u22123.4, \u22120.2) mm; nominal p = 0.0269 and average E\/e\u2019 ratio (ratio between early mitral inflow velocity and mitral annular early diastolic velocity) of LS mean difference (95% CI) of \u22123.4 (\u22125.1, \u22121.6); nominal p = 0.0002.<\/p>\n

\nSimilar safety findings were observed in the Camzyos <\/i>and placebo groups, including treatment-emergent adverse events (TEAEs) (18 versus 17 participants experiencing at least 1 respectively), treatment-related TEAEs (2 versus 3, respectively), treatment-emergent serious adverse events (TESAEs) (2 for both groups), and treatment-related TESAEs (1 versus 0, respectively). During the 28-week study period, there were no TEAE\u2011related treatment discontinuations or deaths, no cases of atrial fibrillation or symptomatic heart failure, and no patient experienced a left ventricular ejection fraction (LVEF) of <50%. Overall, these findings demonstrate that the safety profile of Camzyos<\/i> treatment in symptomatic adolescents with oHCM was similar to that reported in adults, with no new safety signals identified.<\/p>\n

\nThe 28-week active treatment period of SCOUT-HCM is ongoing, and Bristol Myers Squibb will work with key investigators to present the 56-week data at an upcoming medical congress.<\/p>\n

\nAbout the Phase 3 SCOUT-HCM Trial
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<\/span><\/b>SCOUT-HCM (NCT06253221<\/a>) is a Phase 3 randomized, double-blind, placebo-controlled, international trial that enrolled 44 adolescent patients (12 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<\/i>), and an open-label long-term extension period for up to 144 weeks.<\/p>\n

\nThe 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.<\/p>\n

\nJoseph Rossano, MD, is a paid consultant to Bristol Myers Squibb and served as the site principal investigator for the SCOUT-HCM study at Children\u2019s Hospital of Philadelphia.<\/p>\n

\nAbout Obstructive Hypertrophic Cardiomyopathy (oHCM) in Adolescent Patients
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<\/span><\/b>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).<\/p>\n

\nAbout CAMZYOS\u00ae<\/sup> (mavacamten)
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<\/span><\/b>CAMZYOS\u00ae<\/sup> (mavacamten) is the most extensively studied cardiac myosin inhibitor (CMI), approved by regulatory bodies in more than 60 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.<\/p>\n

\nA selective, reversible, allosteric inhibitor of cardiac myosin, CAMZYOS targets hypercontractility, the source of oHCM. Reduction in cardiac contractility with CAMZYOS treatment leads to reduced LVOT obstruction, improved energy consumption, and lower cardiac filling pressures in oHCM patients. These effects translate to improvements 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.<\/p>\n

\nCAMZYOS is supported by the largest body of worldwide evidence in the CMI treatment class, with up to five years of follow up across multiple long-term evidence and real-world studies, demonstrating the consistent and sustained benefits of CAMZYOS to improve symptoms and impact cardiac structure. CAMZYOS has been prescribed by more than 4,500 healthcare providers (HCPs) to more than 22,000 patients in the U.S. alone.<\/p>\n

\nBristol Myers Squibb: Changing the Course of Cardiovascular Disease
\n
<\/span><\/b>Bristol Myers Squibb is inspired by a single vision \u2013 transforming patients\u2019 lives through science. Cardiovascular disease is the leading cause of death worldwide, and despite major advances in how we prevent and treat it, the human and societal burden continues to worsen each year. Whether a cardiovascular disease that affects millions of people around the world, or a rarer condition, the need is the same: new and better treatment options that allow people to continue to live their fullest lives.<\/p>\n

\nBristol Myers Squibb is committed to developing new treatments to address the global burden of cardiovascular disease. Building on our 70-year legacy of discovering and delivering paradigm-changing cardiovascular medicines, we are leveraging our experience and expertise to take cardiovascular research to the next level and deliver meaningful, life\u2011changing outcomes for patients.<\/p>\n

\nCAMZYOS U.S. IMPORTANT SAFETY INFORMATION<\/span><\/b><\/p>\n

\nWARNING: RISK OF HEART FAILURE<\/b><\/p>\n

\nCAMZYOS reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction.<\/b><\/p>\n

\nEchocardiogram assessments of LVEF are required prior to and during treatment with CAMZYOS. Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Interrupt CAMZYOS if LVEF is <50% at any visit or if the patient experiences heart failure symptoms or worsening clinical status.<\/b><\/p>\n

\nConcomitant use of CAMZYOS with certain cytochrome P450 inhibitors or discontinuation of certain cytochrome P450 inducers may increase the risk of heart failure due to systolic dysfunction; therefore, the use of CAMZYOS is contraindicated with the following:<\/b><\/p>\n