Edgewise Therapeutics Inc. (EWTX) announced encouraging topline data from its Phase 1 and 2 trials of EDG-7500, a potential treatment for obstructive hypertrophic cardiomyopathy (HCM). This heart condition occurs when the wall between the left and right ventricles of the heart thickens, hindering blood flow from the left ventricle to the body’s main artery.
In the Phase 1 trial, healthy subjects received single doses of EDG-7500 ranging from 5 to 300 mg. The drug was well-tolerated, with no significant changes in vital signs, blood tests, or electrocardiograms. Importantly, the left ventricular ejection fraction (LVEF), a measure of heart strength, remained unchanged across various doses.
The Phase 2 CIRRUS-HCM trial focused on patients with obstructive HCM. In the single-dose arm, patients received 50, 100, or 200 mg of EDG-7500. The results were impressive: a significant reduction in resting LVOT pressure gradient (LVOT-G), which indicates the severity of the blood flow obstruction, was observed in patients receiving 100 and 200 mg doses. This reduction was also seen when patients performed a Valsalva maneuver, which simulates the conditions that often trigger HCM symptoms. Notably, these reductions in LVOT-G occurred without impacting LVEF.
Furthermore, the 200 mg dose of EDG-7500 led to a 64% reduction in NT-proBNP levels, a key biomarker for heart failure. This suggests that EDG-7500 might not only alleviate the symptoms of HCM but also address the underlying disease process.
The consistency of these positive results across both Phase 1 and 2 trials, coupled with the drug’s favorable safety profile, highlights EDG-7500’s potential as a promising treatment for obstructive HCM. Investors responded enthusiastically, sending EWTX stock soaring by 39.3% on the news.
