Edgewise Therapeutics faced dual setbacks this week as the company disclosed regulatory challenges for its muscular dystrophy program and safety concerns in its heart disease trial, leading to significant stock declines and analyst downgrades.
FDA Finds Muscular Dystrophy Data Insufficient for Accelerated Approval
The FDA determined that Edgewise's mid-stage study data was insufficient for accelerated approval of its experimental drug targeting Becker and Duchenne muscular dystrophies. These rare genetic disorders affect muscles, causing progressive muscle wasting over time. However, the agency provided a pathway forward by validating the measure of physical abilities in Becker muscular dystrophy as a valid primary endpoint for traditional approval.
The company reported that its experimental drug showed sustained disease stabilization for up to three years in the mid-stage study. Edgewise plans to meet with the FDA in the fourth quarter of 2025 to discuss late-stage study design for the traditional approval pathway.
Safety Concerns Emerge in Heart Disease Trial
Separately, Edgewise disclosed safety issues in a mid-stage clinical trial for EDG-7500, its investigational heart disease drug targeting hypertrophic cardiomyopathy. This condition involves thickening of the heart muscle, which can block blood flow and cause heart failure.
Two trial participants developed dangerous cases of atrial fibrillation, and one participant discontinued the study due to dizziness. While atrial fibrillation has been observed with other cardiac myosin inhibitors, the company had positioned EDG-7500 as having superior safety compared to existing treatments.
The company maintains that EDG-7500 can reduce left ventricular outflow tract gradients and improve diastolic relaxation without impairing ejection fraction, with these benefits remaining evident in the study data.
Analyst Reactions and Market Impact
Scotiabank downgraded Edgewise from Sector Outperform to Sector Perform, slashing the price target from $50 to $14. Analyst Louise Chen stated that the safety issues call into question the drug's overall safety story, which was its key differentiator from other drugs on the market. The firm warned that these concerns could make it harder for the drug to secure a position in the increasingly competitive heart medicine market.
J.P.Morgan took a more measured approach, noting that the FDA's findings were in line with expectations. The firm stated, "We continue to remain positive on EWTX but for the most part still see the cardiovascular assets driving the most value for the stock in the mid-term."
Stock Performance
Shares of Edgewise Therapeutics fell as much as 18.4% to $12.66 following the heart disease trial disclosure, and dropped an additional 9.7% to a near two-month low of $12.15 after the muscular dystrophy regulatory update. Including session moves, the stock is down 51.6% year-to-date.
The dual setbacks highlight the challenges facing Edgewise as it attempts to differentiate its cardiac myosin inhibitor in a competitive market while advancing its muscular dystrophy program through traditional regulatory pathways.
