Cytokinetics Presents Additional Data from MAPLE-HCM at the Hypertrophic Cardiomyopathy Medical Society Scientific Sessions and American Heart Association Scientific Sessions 2025

Core Insights - The MAPLE-HCM trial presented additional data indicating that aficamten is superior to metoprolol in improving exercise capacity and patient-reported outcomes in patients with hypertrophic cardiomyopathy (HCM) [1][2][3] Group 1: Treatment Efficacy - Aficamten demonstrated significantly greater improvements than metoprolol across all outcome measures after 24 weeks of treatment, with a positive response rate of 78% for aficamten compared to 3% for metoprolol [4] - Nearly 40% of patients treated with aficamten reported significant improvements in symptoms, highlighting its overall treatment effect [2][4] - Aficamten was associated with a 73% reduction in NT-proBNP levels, while metoprolol showed a 42% increase, indicating a substantial difference in cardiac biomarker response [12] Group 2: Patient-Reported Outcomes - Treatment with aficamten resulted in a KCCQ Overall Summary Score improvement of 16.6 points compared to 8.9 points for metoprolol, with a statistically significant between-group difference of 7.8 points [9] - Aficamten also showed significant improvements in the KCCQ Clinical Summary Score, with a difference of 6.9 points compared to metoprolol [9][10] - Patients on aficamten reported a higher frequency of very large improvements in KCCQ-OSS, defined as a ≥20-point improvement, at 38.6% compared to 18.4% for metoprolol [10] Group 3: Safety Profile - The rate of adverse events was similar between aficamten and metoprolol, with 73.9% and 75.9% of patients reporting at least one treatment-emergent adverse event, respectively [5][7] - The most common adverse event in the aficamten group was hypertension (10.2%), while dizziness was more prevalent in the metoprolol group (17.2%) [7] Group 4: Regulatory Status and Future Development - Aficamten has received Breakthrough Therapy Designation from the FDA for the treatment of symptomatic HCM and is currently under regulatory review in the U.S. and Europe [15][17] - The drug is being evaluated in multiple ongoing clinical trials, including ACACIA-HCM and CEDAR-HCM, targeting both obstructive and non-obstructive HCM [16][19]