Edgewise Therapeutics (EWTX) 44th Annual J.P. Morgan Healthcare Conference summary

Key program updates and clinical progress

• Completed enrollment in pivotal Becker study for sevasemten, with data readout expected by year-end and strong patient retention in open-label extensions.

• Phase 2 and 3 studies in Becker muscular dystrophy show significant reduction in disease progression, with robust biomarker and functional endpoint data supporting efficacy.

• Over-enrollment in pivotal Becker study (175 patients) increases statistical power, and commercial infrastructure is being built in anticipation of positive results.

• Cardiovascular program EDG-7500 demonstrates strong efficacy and safety, with no reduction in ejection fraction, differentiating it from current therapies.

• IND filed for second-generation cardiovascular asset (15400), with healthy volunteer studies completed and HFpEF trials planned for later this year.

Strategic vision and pipeline progress

• Positioned as a global leader in muscle disease therapeutics, leveraging expertise in muscle biology and small molecule drug discovery.

• Rapidly advancing a portfolio targeting muscular dystrophies and serious cardiac conditions, with a focus on patient-centric development.

• Pipeline includes sevasemten for Becker and Duchenne muscular dystrophy, EDG-7500 for hypertrophic cardiomyopathy (HCM), and EDG-15400 for heart failure with preserved ejection fraction (HFPEF).

• Anticipates becoming a commercial company in 2027, with key data readouts and regulatory submissions planned for 2026 and 2027.

Clinical milestones and data highlights

• Positive topline data reported for sevasemten in Becker and Duchenne, and for EDG-7500 in HCM.

• Pivotal GRAND CANYON trial for sevasemten in Becker completed enrollment; primary endpoint powered at >98% to show NSAA difference at 18 months.

• Sevasemten demonstrated sustained stability in NSAA scores over three years, diverging positively from predicted declines.

• EDG-7500 showed rapid, multi-domain improvement in HCM clinical manifestations, with minimal changes in LVEF and strong patient-reported outcomes.

• Additional Duchenne muscular dystrophy data readout in Q2 to assess efficacy in more aggressive patient populations.