Edgewise Therapeutics: A Game Changer in HCM Treatment?

On Wednesday, April 2, 2025, at 8:30 am Eastern Time, Edgewise Therapeutics (NASDAQ: EWTX) will host a webcast event to unveil the top-line results from their Phase 2 CIRRUS-HCM 28-day trial of EDG-7500 in patients with hypertrophic cardiomyopathy (HCM). This event promises to be a pivotal moment for the biotech industry, as EDG-7500 has the potential to revolutionize the treatment of this debilitating disease. But is the hype justified, or is this another case of Wall Street optimism outpacing scientific reality?



Hypertrophic cardiomyopathy (HCM) is a genetic heart condition that affects approximately 1 in 500 people worldwide. It is characterized by the thickening of the heart muscle, which can lead to impaired cardiac performance and, in severe cases, sudden cardiac death. Current treatments for HCM focus on managing symptoms and may include medications like beta-blockers, calcium channel blockers, or disopyramide, as well as surgical interventions like septal myectomy or alcohol septal ablation. However, these treatments do not address the underlying cause of the disease and may have significant side effects or risks.

Enter EDG-7500, a novel oral, selective cardiac sarcomere modulator developed by Edgewise Therapeutics. Unlike existing treatments, EDG-7500 targets the underlying mechanism of HCM, specifically the increased acto-myosin head engagement that leads to impaired relaxation. By slowing early contraction velocity, EDG-7500 has the potential to reduce the hyperdynamic state of the heart and improve diastolic function.

The Phase 2 CIRRUS-HCM trial evaluated EDG-7500 in both obstructive and nonobstructive HCM patients over a 28-day period. The trial's primary objective was to measure the safety and tolerability of EDG-7500, as well as its effects on left ventricular outflow tract (LVOT) gradients, left ventricular ejection fraction (LVEF), biomarkers, and measures of feel and function.

The preliminary results from the Phase 2 CIRRUS-HCM trial are promising. In the single-dose arm of the trial, patients receiving 100 and 200 mg of EDG-7500 experienced a 67% mean reduction in resting LVOT pressure gradient (LVOT-G) and a 55% mean reduction in provokable (Valsalva) LVOT-G, respectively. Additionally, a 64% mean reduction in NT-proBNP, a key biomarker of heart failure, was observed in the 200 mg cohort. These results suggest that EDG-7500 has the potential to improve cardiac relaxation and filling, addressing the impaired diastolic function associated with HCM.

Furthermore, EDG-7500 has demonstrated tolerability and safety in both Phase 1 and Phase 2 trials, with no meaningful changes in LVEF or clinically significant adverse events reported. This suggests that EDG-7500 may offer a safer and more effective treatment option for patients with HCM, potentially improving patient outcomes and quality of life.

However, it is important to approach these results with a healthy dose of skepticism. While the preliminary data from the Phase 2 CIRRUS-HCM trial is encouraging, it is still too early to draw definitive conclusions about the efficacy and safety of EDG-7500. The ongoing 28-day trial will provide additional data on the drug's potential benefits for patients with HCM, and investors should closely monitor the results of this trial before making any investment decisions.

In conclusion, the upcoming webcast event by Edgewise Therapeutics has the potential to be a game-changer in the treatment of hypertrophic cardiomyopathy. EDG-7500's unique mechanism of action and promising preliminary results make it a compelling candidate for addressing the unmet needs of HCM patients. However, investors should remain cautious and wait for the full results of the Phase 2 CIRRUS-HCM trial before jumping to conclusions. As always, the devil is in the details, and only time will tell if EDG-7500 lives up to the hype.