Capricor publishes advisory committee presentation on deramiocel clinical data in Duchenne muscular dystrophy
CAPR•Program summary and safety history
The program summary cited more than 200 DMD patients across trials, with generally favorable tolerability and no serious safety issues highlighted in earlier studies.
Protocol and statistical revisions tied to FDA feedback
The materials highlighted protocol and statistical analysis plan revisions tied to FDA feedback. SAP 3.0 was finalized before database lock and unblinding.
Advisory committee presentation details Phase 3 HOPE-3 results
Capricor outlined Phase 3 HOPE-3 data for deramiocel in Duchenne muscular dystrophy, citing a statistically significant PUL 2.0 total score benefit.
- The primary endpoint showed a 4.55% treatment difference on percent change, with p=0.029 (MMRM).
- A sensitivity analysis on absolute change was 1.1, with p=0.05.
- The cardiomyopathy subgroup with evaluable MRI LVEF (n=64) showed a 2.9% LVEF difference, with p=0.035 (ranked ANCOVA) and p=0.026 (Wilcoxon).
