Edgewise Therapeutics Announces 2025 Priorities Following Positive Phase 2 Results
Key Insights
Edgewise Therapeutics is prioritizing the completion of recruitment for the GRAND CANYON trial for Becker muscular dystrophy (search) in early 2025.
The company plans to seek FDA feedback on Phase 2 CANYON trial results for sevasemten in Becker muscular dystrophy (search) during the first half of 2025.
Initial positive results from the Phase 2 CIRRUS-HCM trial of EDG-7500 in obstructive hypertrophic cardiomyopathy (search) were reported, with more data expected in 2025.
Edgewise Therapeutics Inc. (Nasdaq: EWTX), a biopharmaceutical company focused on muscle disease, has announced its strategic priorities for 2025, building on the momentum gained from positive Phase 2 trial results in 2024. The company is advancing its pipeline of novel therapeutics for muscular dystrophies and cardiac conditions.
Muscular Dystrophy Program
Sevasemten, Edgewise's lead drug candidate, is an orally administered fast skeletal myosin (search) inhibitor designed to protect against contraction-induced muscle damage. It is currently in late-stage clinical trials for Becker and Duchenne muscular dystrophies. There are currently no approved therapies for individuals with Becker muscular dystrophy (search), a serious genetic, progressive neuromuscular disorder.
Key priorities for the sevasemten program in 2025 include:
- Completing recruitment for the GRAND CANYON pivotal placebo-controlled cohort in the first quarter.
- Seeking end-of-Phase 2 feedback from the FDA on CANYON results for sevasemten in Becker muscular dystrophy in the first half of the year.
- Reporting data from the Phase 2 LYNX and FOX trials in the first half of 2025 and outlining potential Phase 3 plans in individuals with Duchenne muscular dystrophy.
Positive topline data from the CANYON Phase 2 placebo-controlled trial in adults with Becker muscular dystrophy (search) has already been reported. Furthermore, substantial enrollment has been achieved in the GRAND CANYON global pivotal cohort of sevasemten in adults with Becker muscular dystrophy; data from GRAND CANYON, if positive, could support a marketing application.
Cardiovascular and Cardiometabolic Programs
EDG-7500, a novel oral, selective, cardiac sarcomere (search) modulator, is being developed for hypertrophic cardiomyopathy (search) (HCM) and other diseases of diastolic dysfunction. It is specifically designed to slow early contraction velocity and address impaired cardiac relaxation.
Priorities for the cardiovascular program in 2025 include:
- Reporting initial CIRRUS-HCM 28-day data in the first quarter.
- Releasing data from the 12-week CIRRUS-HCM trial in individuals with obstructive HCM and non-obstructive HCM by the second half of 2025.
- Filing an investigational new drug application for a second-generation heart failure candidate.
- Selecting a proprietary cardiometabolic drug candidate from preclinical proof of concept data.
Initial positive topline data of EDG-7500 has been reported from the single-dose arm of the Phase 2 CIRRUS-HCM trial in patients with obstructive HCM. Enrollment has commenced in the 28-day arms of CIRRUS-HCM in patients with obstructive and non-obstructive HCM.
Financial Position
Edgewise Therapeutics strengthened its balance sheet with net proceeds of approximately $232 million from a January 2024 public follow-on offering, supporting its muscular dystrophy and cardiovascular programs. According to CEO Kevin Koch, the company has great momentum heading into 2025.