Edgewise reports positive results for muscular dystrophy drug

BOULDER, Colo. - Edgewise Therapeutics (NASDAQ:EWTX), a $1.52 billion market cap biotech company with a robust financial position and more cash than debt, announced Thursday that its experimental drug sevasemten showed sustained disease stabilization for up to three years in patients with Becker muscular dystrophy. According to InvestingPro data, the company maintains exceptional liquidity with a current ratio of 20.99x.

Data from the company’s MESA open label extension trial demonstrated that patients maintained stable North Star Ambulatory Assessment (NSAA) scores over extended treatment periods, contrasting with the functional declines typically observed in natural history studies of Becker muscular dystrophy. The stock, currently trading at $14.33, has faced significant pressure, declining over 51% in the past six months.

The company also reported encouraging results from Phase 2 trials of sevasemten in Duchenne muscular dystrophy, identifying a 10 mg dose for future Phase 3 studies based on consistent observations across multiple functional measures.

Edgewise recently completed a Type C meeting with the FDA, which provided guidance on the regulatory path for sevasemten in Becker muscular dystrophy. While the agency deemed earlier data insufficient for accelerated approval, it expressed support for the ongoing GRAND CANYON pivotal trial, which is expected to report topline results in the fourth quarter of 2026.

"We are well positioned to deliver the first ever therapy for individuals with Becker muscular dystrophy," said Joanne Donovan, Chief Medical Officer at Edgewise, in a press release statement.

The company plans to meet with the FDA in the fourth quarter of 2025 to discuss Phase 3 trial design for Duchenne muscular dystrophy, with plans to initiate the pivotal study in 2026.

Sevasemten is an oral fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies. The drug has received multiple regulatory designations, including FDA Orphan Drug Designation for both Becker and Duchenne muscular dystrophies. With the next earnings report due August 7, 2025, investors can access comprehensive analysis and additional insights through InvestingPro’s detailed research report, which includes expert analysis and key metrics among the 1,400+ covered US stocks.

In other recent news, Edgewise Therapeutics reported its first-quarter 2025 financial results, which aligned with analyst expectations. The company posted a net loss of $40.8 million, or $0.43 per share, matching the consensus estimate. Research and development expenses slightly increased to $36.8 million due to higher personnel costs and clinical development activities. Edgewise ended the quarter with a robust cash position of $436.4 million, further strengthened by a $200 million stock offering in April. The company anticipates reporting data from two Phase 2 trials of its Duchenne muscular dystrophy treatment, sevasemten, in the second quarter of 2025.

In other developments, Piper Sandler reiterated its Overweight rating on Edgewise Therapeutics, maintaining a $51.00 price target. The firm expressed optimism about the regulatory update for sevasemten in Becker muscular dystrophy, expected in the second quarter of 2025. Piper Sandler also highlighted the potential of EDG-7500 in treating hypertrophic cardiomyopathy. Meanwhile, RBC Capital maintained an Outperform rating with a $48.00 price target, expressing confidence in sevasemten’s effectiveness for BMD, despite skepticism about accelerated FDA approval. RBC Capital analysts noted mixed investor sentiment but projected a potential upside if the FDA opts for accelerated approval.

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