Edgewise Therapeutics (EWTX) investor relations material

Key program updates and milestones

• Approaching phase III readout for sevasemten in Becker, aiming for the first approved therapy for this high unmet need population.

• CIRRUS Part D data in hypertrophic cardiomyopathy (HCM) to include both obstructive and non-obstructive cohorts, with optimized dosing strategies.

• Second cardiac program, EDG-15400, set for phase I in the second half of the year, with healthy volunteer data expected in Q2.

• Phase III trials for HCM targeted to initiate in Q4, with parallel or single-study designs under consideration.

• Commercial infrastructure being established ahead of anticipated 2027 approval and 2028 launch for sevasemten.

Clinical trial design and regulatory strategy

• Screening criteria and cardiac monitoring in CIRRUS Part D were enhanced to minimize AFib risk, including core lab echo review and extended look-back periods.

• No observed AFib burden in over 2,000 screening measures, supporting a favorable safety profile.

• FDA feedback suggests less intensive echo monitoring may be required if LVEF and plasma concentration remain decoupled.

• End of phase II meeting with FDA will follow CIRRUS Part D data disclosure, with phase III design details to be shared in anticipation of trial initiation.

Competitive positioning and market opportunity

• 7500 offers potential advantages over CMIs, including no black box warning, no need for intensive echo monitoring, and dose optimization based on feel and function.

• In non-obstructive HCM, 7500 demonstrated superior KCCQ improvements and allows for broader community physician adoption.

• Registration strategy may pursue both obstructive and non-obstructive HCM, leveraging strong efficacy and safety data.

• High patient retention in open-label studies signals strong commercial potential and patient benefit.