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

Edgewise Therapeutics, Inc., EWTX on Wednesday reported topline data of EDG-7500 from the Phase 2 CIRRUS-HCM four-week trial in participants with obstructive or nonobstructive Hypertrophic Cardiomyopathy (HCM).

HCM is a genetic heart condition where the heart muscle thickens, making it harder for the heart to pump blood and potentially leading to complications like heart failure or sudden cardiac death.

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In September 2024, the company announced topline data from Part A of the trial showing that a single oral dose of EDG-7500 in participants with obstructive HCM demonstrated robust reductions in left ventricular outflow tract gradient (LVOT-G) without meaningful changes in left ventricular ejection fraction (LVEF).

Part B of CIRRUS-HCM included 17 participants with obstructive HCM, and Part C included 12 with nonobstructive HCM.

Both trial parts evaluated the safety and efficacy of once-daily doses of 50 or 100 mg of EDG-7500 for four weeks.

In participants with obstructive HCM, EDG-7500 demonstrated meaningful dose-dependent reductions in LVOT-G at rest and post-Valsalva.

• Participants receiving 100 mg experienced mean reductions from baseline of 71% and 58% in resting and provokable (Valsalva) gradients, respectively. Gradient reductions were achieved without meaningful changes in LVEF.
• Treatment with 100 mg of EDG-7500 also demonstrated a 62% mean reduction from baseline in NT-proBNP, a key biomarker of heart failure.
• Clinically meaningful improvements were also observed on the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS, which quantifies the frequency and burden of clinical symptoms in heart failure), with a substantial mean increase of 23 points observed at the 100 mg dose.
• Treatment with 100 mg of EDG-7500 over four weeks demonstrated improvements on the NYHA functional class score (classifies stages of heart failure).
• 78% of participants improved by ≥ 1 NYHA Class, and 67% improved to NYHA Class I (i.e. asymptomatic).

In participants with nonobstructive HCM, EDG-7500 administration resulted in a dose-dependent reduction in NT-proBNP, with a 42% mean decrease from baseline at 100 mg.

• Substantial improvements in KCCQ-OSS and KCCQ-Clinical Summary Scores, with substantial mean increases of 17 points and 22 points, respectively, were observed at the 100 mg dose after only four weeks.

The initial data read-out from Part D is expected in the second half of 2025, with Phase 3 initiation planned for the first half of 2026.

EWTX Price Action: Edgewise Therapeutics stock is down 25.34% at $15.02 at publication Wednesday.

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