Core Insights - Cytokinetics' MYQORZO® (aficamten) has received approval from the China National Medical Products Administration (NMPA) for treating adults with New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (oHCM), aimed at improving exercise capacity and symptoms [1][2] Company Overview - Cytokinetics is a biopharmaceutical company focused on developing treatments for cardiac muscle dysfunction, with over 25 years of experience in muscle biology [9] - The company is advancing a pipeline that includes MYQORZO® (aficamten) and other investigational drugs targeting various heart conditions [9] Collaboration with Sanofi - Under a collaboration agreement, Sanofi holds exclusive rights to develop and commercialize MYQORZO in Greater China, triggering a $7.5 million milestone payment to Cytokinetics upon approval [2][11] - Cytokinetics is eligible for additional milestone payments up to $142.5 million and royalties in the low-to-high teens on future sales of MYQORZO in Greater China [2] Product Details - MYQORZO® (aficamten) is a selective cardiac myosin inhibitor designed to reduce myocardial hypercontractility associated with HCM by binding to cardiac myosin [4] - The drug has shown potential in improving exercise capacity and relieving symptoms in patients with HCM, as evidenced by the positive results from the SEQUOIA-HCM Phase 3 clinical trial [5] Regulatory Status - MYQORZO is currently approved only in China, while aficamten is under regulatory review in the U.S. with a target action date of December 26, 2025 [3] - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for aficamten in the EU, with a final decision expected in Q1 2026 [3] Hypertrophic Cardiomyopathy (HCM) Overview - HCM is characterized by abnormal thickening of the heart muscle, leading to reduced exercise capacity and various symptoms [7] - It is the most common inherited cardiovascular disorder, with approximately 280,000 diagnosed patients in the U.S., and an estimated 400,000-800,000 undiagnosed cases [7][8]

Core Insights - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending marketing authorization for MYQORZO® (aficamten) for treating symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults, with a final decision expected from the European Commission in Q1 2026 [2][3]. Company Overview - Cytokinetics is a biopharmaceutical company focused on developing treatments for cardiac muscle dysfunction, with over 25 years of experience in muscle biology [15]. - The company is preparing for potential regulatory approvals and commercialization of aficamten following positive results from the pivotal Phase 3 clinical trial SEQUOIA-HCM [15]. Clinical Trial Results - The CHMP recommendation is based on the SEQUOIA-HCM trial, which demonstrated significant improvements in exercise capacity and safety for patients treated with MYQORZO compared to placebo [5][6]. - MYQORZO treatment for 24 weeks resulted in a peak oxygen uptake (pVO2) increase of 1.8 ml/kg/min compared to a 0.0 ml/kg/min change in the placebo group, with a statistically significant difference (p=0.000002) [6]. - The treatment was well-tolerated, with serious adverse events occurring in 5.6% of MYQORZO patients versus 9.3% in the placebo group [7]. Drug Mechanism and Development - Aficamten is a selective cardiac myosin inhibitor designed to reduce myocardial hypercontractility associated with hypertrophic cardiomyopathy (HCM) by binding to cardiac myosin [8]. - The drug has received Breakthrough Therapy Designation from the FDA and the NMPA in China for treating symptomatic HCM [10]. Ongoing Research - Aficamten is also being evaluated in additional clinical trials, including ACACIA-HCM for non-obstructive HCM and CEDAR-HCM for pediatric patients with oHCM [11].