Edgewise Therapeutics (EWTX) investor relations material

Clinical trial updates and data insights

• Recent data showed no link between ejection fraction changes and drug concentration, with strong improvements in patient function and key biomarkers, and a generally well-tolerated safety profile except for some atrial fibrillation cases.

• Protocol amendments included lowering the dose to 25 mg and implementing more rigorous patient screening to reduce high-risk atrial fibrillation cases, with robust patient recruitment since June and about 20 sites now open.

• Efficacy and safety updates for 25 mg dosing in Parts B and C are expected by year-end, with further Part D updates to follow.

• The focus is on demonstrating efficacy while minimizing safety concerns, aiming to differentiate from current therapies that require complex titration and frequent echoes.

Competitive positioning and market opportunity

• The drug aims to address limitations of current CMIs, such as the need for titration and echo monitoring, by offering a fixed-dose regimen with minimal ejection fraction impact.

• Eliminating the need for serial echoes could expand use beyond centers of excellence, addressing capacity constraints and improving access.

• Robust improvements in patient-reported outcomes (KCCQ) and NYHA class were observed, with efficacy comparable to or better than septal reduction therapy.

• The mechanism as a partial ATPase modulator allows higher target engagement without reducing ejection fraction, supporting both safety and efficacy.

Future development plans and regulatory strategy

• Plans include running phase 3 trials potentially head-to-head against beta blockers or similar to prior landmark studies, leveraging strong KCCQ improvements and NT-proBNP as key endpoints.

• For HFpEF, a phase 2A study is planned for 2026, focusing on NT-proBNP reduction as an early efficacy marker, with healthy volunteer data expected in the first half of 2026.

• In DMD, ongoing studies are evaluating stabilization in patients with prior gene therapy, with regulatory discussions on endpoints like SV95 and North Star underway.

• The company is well-funded with $560 million, supporting operations through 2028 and enabling a lean commercial launch strategy, especially in Becker muscular dystrophy.