Edgewise Therapeutics Reports Positive Progress in EDG-7500 Hypertrophic Cardiomyopathy Trials

Edgewise Therapeutics Reports Progress on EDG-7500 Trials

Edgewise Therapeutics, a biopharmaceutical leader in muscle disease treatment, revealed significant advancements in its ongoing Phase 2 clinical trial, CIRRUS-HCM, aimed at evaluating EDG-7500. This novel oral medication is designed as a selective cardiac sarcomere modulator, specifically targeting hypertrophic cardiomyopathy (HCM).

The latest updates indicate that EDG-7500 was well tolerated among participants, with no clinically meaningful reductions observed in left ventricular ejection fraction (LVEF). Notably, participants from the trial, which comprises multiple parts—including Parts B and C—exhibited various improvements in key markers associated with HCM. These markers included N-terminal pro-B-type natriuretic peptide (NT-proBNP), Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, and the New York Heart Association (NYHA) class.

Summary of Trial Findings

The CIRRUS-HCM trial is designed to evaluate EDG-7500 in both obstructive and non-obstructive forms of HCM. Previous results from Parts B and C demonstrated positive outcomes: participants receiving EDG-7500 showed notable enhancements in NT-proBNP levels and KCCQ scores. Furthermore, the trial confirmed that treatment did not lead to any significant changes in LVEF nor increased risks of atrial fibrillation, known dangers often associated with other treatments for the condition.

In addition to previously administered doses of 50 mg and 100 mg, the trial also assessed a lower 25 mg fixed dose, revealing beneficial results in different markers of HCM throughout the study groups. Notably, the lower dose still upheld a favorable safety profile, which is crucial in establishing the viability of EDG-7500 as a potential treatment option.

Future Steps in Development

The ongoing Part D of the trial is aimed at exploring further correlations between dosage and participant response, with a focus on biomarker-guided dose optimization. This aspect is crucial for informing future Phase 3 trials, scheduled to begin by late 2026. Over a 12-week active treatment period in Part D, over 40 participants have been enrolled, which successfully met the enrollment goals set for this phase.

With strong safety data indicating that the drug maintains heart function without adverse events, Edgewise is strategically positioned to refine its approach for EDG-7500, which could deliver significant advancements in the management of HCM.

Challenges and Unmet Needs

Hypertrophic cardiomyopathy is a prevalent genetic heart disease affecting approximately 1 in 500 individuals. Symptoms can severely diminish a patient's quality of life, manifesting through episodes of breathlessness, irregular heartbeats, or even fainting due to excessive heart muscle thickening. Despite some treatment advancements, there persists a critical unmet need for effective management solutions that do not compromise heart function.

Conclusion

Edgewise Therapeutics remains determined to provide a comprehensive analysis of Phase 2 results in the second quarter of 2026, bolstering preparations for a prospective Phase 3 trial. The company's commitment to advancing therapeutic solutions for HCM underlines its focus on addressing significant gaps in treatment options and enhancing patient outcomes effectively. As Kevin Koch, President and CEO, stated, “The lack of clinically meaningful drops in ejection fraction is encouraging, and we look forward to refining our strategy in 2026 for EDG-7500's development.”

For ongoing information and updates regarding this promising approach to HCM treatment, follow Edgewise therapeutics on their official website or on LinkedIn and X.