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

Key program updates

• Completed enrollment for the pivotal Becker muscular dystrophy study; data readout expected by year-end 2025, with the program powered to detect a 1.7-point North Star change and NDA filing possible upon positive results.

• Duchenne program continues with ongoing data collection; additional readout planned for Q2 2025.

• Cardiovascular asset EDG-7500 showed robust efficacy and safety, with no reduction in ejection fraction, and rapid, multi-domain improvement in HCM clinical manifestations.

• IND filed for second cardiovascular asset (15400); healthy volunteer studies underway, with HFpEF trial initiation planned for H2 2025.

• Pipeline includes sevasemten for Becker and Duchenne muscular dystrophy, EDG-7500 for HCM, and EDG-15400 for HFpEF.

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.

• 89% of EDG-7500 patients achieved normal gradient; 56% normalized NT-proBNP; 89% improved KCCQ by >10 points.

• Phase 3 trial design for EDG-7500 may leverage feel/function endpoints, with regulatory discussions ongoing.

Market opportunity and commercial readiness

• Becker muscular dystrophy represents a $5B market with 6,000 U.S. and 12,000 global patients; commercial infrastructure and activities underway.

• HCM market opportunity estimated at $10B, with EDG-7500 targeting both obstructive and nonobstructive forms.

• Data supports expansion into community cardiology, potentially unlocking a $10B market.

• Commercial activities include education, payor engagement, and field force planning.