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.

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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).

Based on the above results, EWTX started the part B portion of the mid-stage study on EDG-7500 in patients with obstructive HCM. Initial data from this study is expected in the first quarter of 2025.

EWTX’s Zacks Rank

Edgewise currently carries a Zacks Rank #3 (Hold).

Edgewise Therapeutics, Inc. Price

Edgewise Therapeutics, Inc. price | Edgewise Therapeutics, Inc. Quote

Our Key Picks Among Biotech Stocks

Some better-ranked stocks from the sector are Castle Biosciences CSTL, CytomX Therapeutics CTMX and Spero Therapeutics SPRO, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Bottom-line estimates for Castle Biosciences have improved from a loss of 58 cents per share to earnings of 34 cents for 2024 in the past 60 days. During the same timeframe, loss per share estimates for 2025 have narrowed from $2.13 to $1.84. Year to date, shares of Castle Biosciences have surged 33.6%.

CSTL’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 172.72%.

In the past 60 days, estimates for CytomX Therapeutics’ 2024 loss per share have narrowed from 29 cents to 5 cents. Estimates for 2025 loss per share have narrowed from 56 cents to 35 cents during the same timeframe. Year to date, CTMX stock has lost 25.8%.

CytomX’s earnings beat estimates in two of the trailing four quarters and missed the mark in the other two, delivering an average surprise of 115.70%.

In the past 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.29. Estimates for 2025 loss per share have narrowed from $1.54 to 79 cents during the same timeframe. Year to date, Spero’s shares have lost 27.2%.

SPRO’s earnings beat estimates in two of the trailing four quarters and missed the mark in the other two, delivering an average surprise of 94.42%.

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