Capricor Therapeutics (CAPR) investor relations material

Regulatory and Clinical Update

• FDA held a Type A meeting and agreed to review HOPE-3 data as part of the ongoing BLA for DMD therapy, with both skeletal muscle and cardiac endpoints considered for potential dual labeling.

• The current BLA remains open, no new submission is required, and all CMC issues from the CRL have been addressed.

• FDA maintains regulatory flexibility, especially regarding the cardiac indication, and expressed commitment to flexibility in reviewing HOPE-3 data.

• Submission of HOPE-3 data as a response to the CRL is planned, with updates to be provided as available.

• The regulatory strategy aims for a label covering both cardiac and skeletal muscle function in DMD.

HOPE-3 Pivotal Trial Status and Design

• HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled trial that enrolled 105 ambulatory and non-ambulatory boys with DMD.

• All participants have completed 12 months of follow-up, with database lock and top-line data release expected before the end of the quarter or mid-Q4 2025.

• About 70% of participants had cardiomyopathy at baseline, supporting robust evaluation of both skeletal and cardiac outcomes.

• The trial is powered to detect statistical significance in both primary (skeletal) and key secondary (cardiac) endpoints.

• Participants received four doses over 12 months, with endpoints focused on muscle function.

Endpoints and Statistical Considerations

• The primary endpoint remains the Performance of the Upper Limb (PUL) 2.0, with cardiac function (LVEF) as a key secondary endpoint.

• FDA was unwilling to change the primary endpoint but may exercise flexibility for cardiac labeling even if the primary is not met.

• The statistical analysis plan is finalized and will be submitted imminently.

• Both endpoints are expected to achieve statistical significance based on powering and prior data.

• HOPE-3 data will be submitted as the additional study requested in the CRL.