Edgewise Therapeutics (EWTX) Status Update summary

Company background and community engagement

• Focused on muscle biology and developing therapies for muscular dystrophies since 2017.

• Developed strong patient engagement through interviews, focus groups, and educational resources.

• Supported community initiatives like BEAD and contributed to standards of care.

Scientific approach and trial design

• Sevasemten targets contraction-induced muscle injury, especially in type II muscle fibers.

• Biomarkers CK and TNNI2 are used to measure muscle damage and therapy effect.

• North Star Ambulatory Assessment is used to track functional changes.

Canyon phase 2 trial results

• Largest interventional Becker study to date, with 69 enrolled and 4% discontinuation.

• Achieved primary endpoint: 28% reduction in CK and 77% reduction in TNNI2 with sevasemten.

• North Star scores remained stable in treated group, while placebo declined as expected.

• Functional tests mostly favored sevasemten; safety profile remained consistent with no new signals.