Dive Brief:

• Edgewise Therapeutics said its experimental heart disease drug hit the goals of a Phase 2 clinical trial, reducing signs of disease and biological markers in two types of cardiomyopathy and supporting advancement of the medicine into pivotal studies.
• The Colorado-based biotechnology company is testing its pill, called EDG-7500, in both the obstructive and non-obstructive forms of cardiomyopathy, seeking to improve blood flow and relieve heart failure symptoms in people whose hearts have thickened and weakened as their disease progressed.
• The trial data come two weeks after Edgewise wagered the company’s future on cardiovascular disease by selling off its experimental muscular dystrophy pipeline. In cardiomyopathy, Edgewise will have hefty competition, however, as it would face Bristol Myers Squibb’s Camzyos and Cytokinetics’ Myqorzo in the obstructive form of the disease.

Dive Insight:

Since Edgewise’s executive team fully committed to the company’s cardiovascular business, investor expectations ran high for the cardiomyopathy trial. The data may not have met those expectations, however, as shares fell by as much as 11% in early trading Tuesday, while Cytokinetics’ were up around 4%.

Wall Street analysts, conversely, viewed the data as positive, giving them a better view of the EDG-7500’s competitive profile against Camzyos, which failed in non-obstructive cardiomyopathy, and Myqorzo, which succeeded in non-obstructed patients.

The “Cirrus-HCM” trial was designed primarily as a safety test — as many mid-stage cardiovascular trials are — but it did include efficacy measures based around symptoms, quality of life and biological signs of cardiomyopathy.

In both forms of disease, in which the heart muscles have thickened enough to restrict blood flow from the heart, most enrollees experienced improved blood flow as well as a reduction or normalization of blood proteins that signal heart failure. The participants with obstructive disease saw a mean 24-point increase on a 12-item questionnaire on symptoms, and those with non-obstructive disease saw a mean 13-point increase.

On safety, EDG-7500 didn’t lead to a decrease in the left ventricular ejection fraction, or the amount of blood pumped into the aorta, which has happened with both Camzyos and Myqorzo. Edgewise said two cases of atrial fibrillation occurred, both of which trial investigators determined weren’t drug-related.

RBC Capital Markets analyst Leonid Timashev wrote in a client note that the data showed an “impressive safety profile and compelling efficacy,” pointing to its ejection fraction data and the improvements in symptomatic scores, in particular for people with obstructive cardiomyopathy.

Stifel analyst James Condulis acknowledged the data in obstructive disease “looks solid,” but investors were hoping for data in non-obstructive cardiomyopathy that showed EDG-7500 “could meaningfully improve beyond” Myqorzo. The cases of atrial fibrillation mean it is an “open question” whether more might emerge in larger studies, he wrote.

And given the amount of time before a Phase 3 trial might read out in 2028 or 2029, “we don't think these data will meaningfully change peoples' views” on EDG-7500 or Edgewise, Condulis added.