Bristol-Myers Squibb(US:BMY) Xin Lang Cai Jing·2025-12-18 14:26Core Insights - Hypertrophic Cardiomyopathy (HCM) is the most common single-gene hereditary heart disease, characterized by asymmetric hypertrophy of the myocardium, with obstructive HCM having a lower survival rate [2][23] - Mavacamten, a first-in-class myosin inhibitor, targets the core pathophysiological mechanisms of HCM, representing a significant advancement in treatment options [2][17] Group 1: Disease Overview - HCM affects approximately 0.2% to 0.5% of the global population, with an estimated 2 million patients in China [2][23] - Patients with obstructive HCM experience symptoms such as dyspnea, fatigue, chest pain, and syncope, which severely limit their daily activities and mental well-being [2][23] Group 2: Mavacamten Development - Mavacamten is the first drug to provide targeted intervention at the pathophysiological level, differing from traditional symptomatic treatments like beta-blockers and calcium channel blockers [4][25] - The EXPLORER-HCM trial demonstrated that Mavacamten significantly improves exercise capacity, left ventricular outflow tract (LVOT) obstruction, and overall health status compared to placebo [6][27] Group 3: Clinical Trials in China - The EXPLORER-CN trial, led by Professor Zhang Shuyang, is the first randomized, double-blind, placebo-controlled study specifically for symptomatic obstructive HCM patients in China [12][28] - Results from EXPLORER-CN showed significant improvements in LVOT peak pressure gradient and other clinical parameters after 30 weeks of treatment with Mavacamten [13][32] Group 4: Safety and Efficacy - Mavacamten treatment resulted in consistent benefits across various subgroups, including gender, age, and baseline NYHA classification, with no new safety signals identified [13][28] - The drug demonstrated positive effects on cardiac structure and diastolic function, as evidenced by reductions in left ventricular mass index and wall thickness [14][34] Group 5: Global Contribution and Future Prospects - Chinese clinical data and research have enriched the global evidence base for Mavacamten, highlighting the role of Chinese researchers in advancing scientific understanding of HCM [16][37] - Mavacamten was approved for use in China in April 2024, marking a significant milestone in providing a root-cause treatment option for obstructive HCM patients [18][17]