Shares of Edgewise Therapeutics EWTX soared 30% in the pre-market trading on Thursday after it reported positive top-line data of EDG-7500 from an early-stage study in healthy subjects and Part A (single-dose arm) of a mid-stage study in patients with obstructive hypertrophic cardiomyopathy (HCM).

EWTX’s EDG-7500 Shows Superior Efficacy in Both Studies

Per the data readout from the phase I study in healthy adults, no meaningful changes in left ventricle ejection fraction (LVEF), an important predictor of mortality, were observed for all single and multiple ascending dose (SAD and MAD) subjects treated with a broad range of EDG-7500 doses. Dose-proportional increases in exposure were generally seen in both SAD and MAD cohorts.

The candidate was also overall well tolerated in the SAD and MAD cohorts of the phase I study and demonstrated a favorable safety profile, with no clinically meaningful changes or trends in vital signs, clinical chemistry, hematology or electrocardiograms.

In Part A of the phase II CIRRUS-HCM study, treatment with the 100 and 200 mg single doses of EDG-7500 resulted in a 67% mean reduction inresting left ventricular outflow tract (LVOT) pressure gradientand a 55% mean reduction in provokable (Valsalva) LVOT pressure gradient. Interestingly, gradient reduction was achieved without a meaningful change in LVEF.

Year to date, shares of Edgewise have rallied 74.3% compared with the industry’s 0.5% growth.

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In the 200 mg cohort, it was observed that treatment with the candidate also led to a 64% mean reduction in NT-proBNP, a key biomarker of heart failure. Per Edgewise, such robust outcomes underscore the potential of EDG-7500’s unique mechanism of action to treat diastolic dysfunction diseases, including non-obstructive HCM.

EWTX further reported that no patients in the phase I or the CIRRUS-HCM studies experienced an LVEF reduction below 50% across all administered doses of EDG-7500.

EWTX’s EDG-7500 Developmental Plans for Heart Diseases

Based on the encouraging clinical and preclinical data reported to date, Edgewise has started dosing the first patients with obstructive and non-obstructive HCM, in the 28-day Part B of the phase II CIRRUS-HCM study of EDG-7500, where there is a huge unmet medical need.

The study will also continue to evaluate the tolerability and PK of the candidate and its effects on LVOT pressure gradient, LVEF, biomarkers and measures of feel and function in the obstructive and non-obstructive HCM patient population.