Edgewise Therapeutics Sees Meaningful HCM Treatment Gains With EDG-7500 In Phase 2 Study

Core Insights - Edgewise Therapeutics, Inc. reported topline data from the Phase 2 CIRRUS-HCM trial for EDG-7500, targeting obstructive and nonobstructive Hypertrophic Cardiomyopathy (HCM) [1][2] Group 1: Trial Results - In Part A of the trial, a single oral dose of EDG-7500 showed significant reductions in left ventricular outflow tract gradient (LVOT-G) without affecting left ventricular ejection fraction (LVEF) [2] - Part B included 17 participants with obstructive HCM, while Part C included 12 with nonobstructive HCM, both evaluating the safety and efficacy of once-daily doses of 50 or 100 mg of EDG-7500 for four weeks [3] - Participants receiving 100 mg experienced mean reductions of 71% in resting LVOT-G and 58% in provokable (Valsalva) gradients, achieved without meaningful changes in LVEF [6] Group 2: Biomarker and Clinical Improvements - In nonobstructive HCM participants, a 42% mean decrease in NT-proBNP was observed at the 100 mg dose [4] - Significant improvements in Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS) and Clinical Summary Scores were noted, with mean increases of 17 points and 22 points, respectively, at the 100 mg dose after four weeks [4] - Treatment with 100 mg of EDG-7500 resulted in a 62% mean reduction in NT-proBNP, a key heart failure biomarker, and 78% of participants improved by at least one NYHA Class, with 67% improving to NYHA Class I [6] Group 3: Future Outlook - Initial data from Part D of the trial is expected in the second half of 2025, with Phase 3 initiation planned for the first half of 2026 [4]