Capricor Therapeutics announced positive topline results from the pivotal Phase 3 HOPE-3 trial evaluating Deramiocel for the treatment of Duchenne muscular dystrophy (DMD).
The study met the primary endpoint (PUL v2.0) and key secondary cardiac endpoint (LVEF) with statistical significance.
Results demonstrated clinically meaningful skeletal and cardiac benefits, supporting Deramiocel as a potential first-in-class therapy for Duchenne cardiomyopathy.
Positive Topline Results
Study achieved statistical significance in primary and key secondary endpoints, showing meaningful skeletal and cardiac benefits.
Safety and Tolerability
Deramiocel maintained a favorable safety profile consistent with past experience.
Response to FDA
Company plans to submit response to the Complete Response Letter using HOPE-3 data after FDA alignment.
- HOPE-3 demonstrated significant improvements in both skeletal and cardiac function in DMD patients.
- The study results support Deramiocel as a potential therapeutic option for Duchenne cardiomyopathy.
- Positive findings pave the way for regulatory approval and addressing clinical issues highlighted in the Complete Response Letter.
The HOPE-3 trial results highlight the efficacy and safety of Deramiocel in treating Duchenne muscular dystrophy, signaling a potential breakthrough in addressing this condition. Capricor Therapeutics is poised to advance with confidence towards regulatory approval and addressing clinical concerns.