Edgewise Therapeutics Inc. (NASDAQ:EWTX) on Tuesday released topline results from the 12-week Part D cohort of its Phase 2 CIRRUS-HCM trial.
Patients received doses ranging from 25 mg to 150 mg.
EDG-7500 demonstrated clinically meaningful improvements in patients with both obstructive and 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.
The company said the oral cardiac sarcomere modulator improved key measures of heart function, symptoms, and biomarkers without compromising systolic function.
Strong Improvements Seen In Obstructive HCM Patients
The Part D cohort enrolled 53 patients, including 20 with obstructive HCM (oHCM) and 33 with nonobstructive HCM (nHCM). The 12-week study was designed to help inform a future Phase 3 trial.
Among oHCM patients, EDG-7500 produced significant reductions in left ventricular outflow tract gradient (LVOT-G) at rest and after the Valsalva maneuver.
According to the company, 90% of patients showed improvement in hemodynamic measures.
The treatment also reduced heart failure severity markers. Nearly three-quarters of oHCM patients either achieved normal NT-proBNP levels or recorded at least a 50% reduction from baseline.
Patient-reported outcomes improved as well. Additionally, 70% of patients improved by at least one New York Heart Association functional class.
Nonobstructive HCM Patients Show Similar Benefits
In nHCM patients, EDG-7500 generated an average 65% reduction in NT-proBNP levels.
The company said 88% of patients achieved either normalized NT-proBNP levels or at least a 50% reduction from baseline.
Patients also reported symptom improvements, with a 13-point mean increase and 64% achieving at least one class improvement in NYHA status.
Diastolic function measures improved as well.
Safety Profile Remains Favorable
Edgewise said EDG-7500 was generally well tolerated, with no new safety signals identified among the 53 patients evaluated. Most adverse events were mild to moderate in severity.
The company reported no meaningful changes in left ventricular ejection fraction and no cases where ejection fraction fell below 50%. Two cases of new-onset atrial fibrillation were reported, though investigators determined both events were unrelated to the study drug.
Just two weeks ago, the biopharmaceutical company agreed to sell its muscular dystrophy business to Servier for $1.55 billion in upfront cash consideration and up to $1.1 billion in additional milestone payments.
The transaction meaningfully strengthens Edgewise’s balance sheet and sharpens the company’s strategic focus on its cardiovascular pipeline.
EWTX Price Action: Edgewise Therapeutics shares were down 2.58% at $34.29 at the time of publication on Tuesday, according to Benzinga Pro data.
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