Deramiocel (CAP-1002)

HOPE-3 randomized 106 participants. The reported baseline population included 90 non-ambulatory participants (84.9%). The publication reported a 4.55-percentage-point between-group difference in mean percentage change in total PUL2.0 score at 12 months, favouring deramiocel (95% CI 0.47 to 8.63; p=0.029). The key secondary LVEF endpoint did not reach statistical significance (p=0.0935). FDA concluded that the study did not meet its prespecified primary and secondary efficacy endpoints and identified additional statistical changes without an updated SAP. These assessments should not be treated as equivalent analyses. In the FDA safety presentation, headache occurred in 32 of 53 deramiocel recipients (60.4%) and 2 of 52 placebo recipients (3.8%); hypersensitivity reactions occurred in 22 of 53 (41.5%) and 8 of 52 (15.4%), respectively.