First Patient Treated in Phase 2 EDG-7500 Trial for Obstructive HCM

Edgewise Therapeutics, a prominent biopharmaceutical firm specializing in muscle diseases, has initiated the Phase 2 CIRRUS-HCM trial for EDG-7500, marking a significant step in the treatment of hypertrophic cardiomyopathy (HCM). The trial's first patient has received the novel oral medication, which is a selective cardiac sarcomere modulator. EDG-7500 is designed to decelerate the early pace of heart contraction and improve the impaired relaxation of the heart muscle, a common issue in HCM and other diastolic dysfunction conditions.

The Phase 2 study will focus on the safety, tolerability, pharmacokinetics, and pharmacodynamics of EDG-7500 in individuals with obstructive HCM. It is divided into two parts: Part A will examine the effects of a single dose, while Part B will look into the impact of multiple doses over a period of 28 days.

Marc Semigran, M.D., Edgewise's Chief Development Officer, expressed optimism based on clinical and preclinical data, highlighting the drug's potential to alleviate the left ventricular outflow tract gradient while maintaining normal heart contractility. In non-obstructive HCM models, EDG-7500 has shown promising results in enhancing ventricular filling and diastolic function.

Kevin Koch, Ph.D., President and CEO of Edgewise, emphasized the advancement of EDG-7500 as a milestone, reflecting the company's dedication to developing treatments for severe muscle diseases. The company anticipates reporting data from the single dose arm and a Phase 1 trial involving healthy volunteers in the third quarter of 2024. Additionally, plans are in place to initiate a 28-day trial for both obstructive and non-obstructive HCM patients later in 2024, followed by an open-label extension trial in the fourth quarter.

The CIRRUS-HCM trial is an open-label, multi-center study set to take place at approximately 20 U.S. clinical sites, enrolling up to 30 adults with obstructive HCM. Participants will receive a single oral dose of EDG-7500, with the option to continue with multiple doses over 28 days.

HCM, the most prevalent genetic heart disease, affects an estimated one in 200-500 people. It is characterized by abnormal proteins leading to excessive cardiac contraction, causing the heart's main pumping chamber to thicken and become less compliant. This results in decreased heart function and an increased risk of heart failure, stroke, atrial fibrillation, and sudden cardiac arrest. Despite existing treatments, there remains a significant need for new therapeutic approaches.

EDG-7500's unique properties have shown potential in preclinical models to provide effective clinical responses with a low risk of reducing left ventricular ejection fraction. The company plans to explore fixed-dose regimens to minimize the need for intensive safety monitoring.

Edgewise Therapeutics is at the forefront of developing innovative treatments for muscular dystrophies and serious cardiac conditions. With a robust pipeline, including Sevasemten for Becker and Duchenne muscular dystrophies, the company is committed to improving the lives of those affected by muscle diseases.