Edgewise Therapeutics (EWTX) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Key clinical developments and data highlights

• Sevasemten advanced to Phase III for Becker muscular dystrophy, with positive data from ARCH and CANYON studies showing disease stabilization, functional improvements, and significant biomarker reductions; CANYON met its primary endpoint with a 28% decrease in CK and 77% decrease in TNNI2.

• GRAND CANYON pivotal study for Becker is fully enrolled, with results expected in Q4 2026; over-enrollment reflects strong patient demand and regulatory engagement is ongoing with FDA and EMA.

• Duchenne muscular dystrophy program identified optimal 5 mg and 10 mg doses, with LYNX and FOX studies guiding Phase 3 trial plans and key data readouts expected by H1 2025.

• EDG-7500 for hypertrophic cardiomyopathy showed robust reductions in cardiac gradients and NT-proBNP, with no significant ejection fraction changes, and is progressing through 28-day and 12-week CIRRUS studies with data expected in 2025.

• Sevasemten could become the first approved therapy for Becker muscular dystrophy, addressing a significant unmet need in a 12,000-patient market across the US, EU-5, and Japan.

Strategic and regulatory outlook

• Plans to seek accelerated approval for Sevasemten in Becker based on new FDA guidance and strong clinical data, with confirmatory GRAND CANYON study ongoing.

• Phase III studies for EDG-7500 in both obstructive and non-obstructive HCM targeted to start in 2026, following agency discussions in Q1 2026.

• Additional cardiovascular and cardiometabolic candidates are advancing, with new IND filings and candidate selection expected by year-end.

• Intellectual property for EDG-7500 extends into the late 2030s.

• Continued focus on expanding the pipeline in muscle and cardiometabolic diseases, leveraging deep expertise in muscle biology and drug discovery.

Financial position and operational milestones

• Raised $240 million in 2024, with ~$493 million in cash and a runway through 2027, supporting execution of key milestones and no debt as of September 2024.

• Multiple value-generating milestones anticipated in 2024 and 2025, including key clinical data readouts and regulatory feedback.

• 94 million shares outstanding, supporting continued investment in R&D and clinical programs.