In This Article:
Shares of Edgewise Therapeutics EWTX were up more than 18% on Monday after it announced positive results from the mid-stage CANYON study on its lead pipeline drug, sevasemten, in patients with a rare genetic disorder called Becker Muscular Dystrophy (“BMD”).
The study met its primary endpoint of change from baseline in creatine kinase (CK), a biomarker associated with skeletal muscle damage. Data from the study showed a significant reduction in CK levels in the sevasemten-treated group, with an average decrease of 28% compared to placebo between six and 12 months. The drug was also well-tolerated by study participants.
Treatment with sevasemten showed a trend toward improvement in the key secondary endpoint of the North Star Ambulatory Assessment, a scale commonly used to rate motor function. Patients who received the drug also showed a significant reduction in plasma levels of fast skeletal muscle troponin I (TNNI2), a target-specific biomarker for fast skeletal muscle damage. Per EWTX, the sevasemten-treated group included patients with more advanced disease compared to those in the placebo group.
Edgewise remains on schedule to complete recruitment for a pivotal cohort of the CANYON study, called GRAND CANYON, which is expected to be achieved in the first quarter of 2025. If data from this cohort is also positive, management intends to start discussions with the FDA and EMA for seeking approval for sevasemten in the BMD indication.
EWTX Stock Performance
Investors were impressed with the CANYON study results and management’s plans for sevasemten, which sent the stock price soaring. Some investors believe that the drug has the potential to become the first approved therapy for BMD.
EWTX’s shares have skyrocketed nearly 200% year to date against the industry’s 11.4% decline.
Image Source: Zacks Investment Research
More on EWTX’s Sevasemten
Other than BMD, Edgewise is also evaluating sevasemten in other muscular disorders. The drug is also being evaluated in separate mid-stage studies for Duchenne muscular dystrophy (DMD), Limb-Girdle muscular dystrophy (LGMD) and McArdle Disease.
Sevasemten has achieved significant regulatory milestones from the FDA. These include the orphan drug and fast-track designations in both BMD and DMD indications and a rare pediatric disease designation for DMD.
Other Candidates in EWTX’s Pipeline
Apart from sevasemten, Edgewise is also evaluating another candidate, EDG-7500, in a mid-stage study for patients with obstructive hypertrophic cardiomyopathy (HCM).
In September, management reported positive top-line data for EDG-7500 from an early-stage study in healthy subjects and Part A (single-dose arm) of a mid-stage study in patients with HCM. While the early-stage data showed that treatment with the drug was well-tolerated with no changes in left ventricle ejection fraction (an important predictor of mortality), the mid-stage data showed gradient relief without reductions in left ventricular outflow tract (part of the heart that directs blood from the left ventricle to the aorta).