Capricor Therapeutics (CAPR) Study result summary

Regulatory and development update

• FDA extended the PDUFA date to Nov. 22, 2026, to review OLE data and additional evidence; the proposed indication was refined to upper-limb impairment in patients older than 9.

• A major BLA amendment included OLE results and analyses addressing Advisory Committee questions; upper-limb function remained the focus, with further FDA review of cardiac data hoped for.

HOPE-3 efficacy and robustness

• HOPE-3 met its primary PUL 2.0 endpoint: deramiocel showed 4.55% less decline than placebo at 12 months; a pre-specified sensitivity analysis found a 1.1-point difference, nominally significant at 0.05.

• More than 600 alternative analyses showed a stable treatment effect; reported analyses showed benefit and none approached zero.

• Pooled HOPE-2/HOPE-3 results showed a 1.29-point PUL 2.0 difference at 12 months (P=0.0087); eight sensitivity analyses estimated 1.1–1.3 points of benefit, each with P<0.02.

Two-year open-label extension

• Of 106 randomized HOPE-3 patients, 98 entered the OLE (49 per original group) and 82 completed two years.

• Delayed-start patients declined 2.05 PUL 2.0 points on placebo in Year 1 and 0.49 points on deramiocel in Year 2: a 1.56-point difference (95% CI 0.47–2.64) and 76% lower decline rate.

• Early-start patients declined 0.95 points in Year 1 and 0.89 points in Year 2 on deramiocel; the difference was 0.05 points (95% CI −1.03 to 1.14).

• At Month 24, observed declines were 3.76 points after 12 months and 3.42 points after 24 months on deramiocel, versus natural-history predictions of 5.35 and 5.88; comparisons were descriptive and unadjusted.

• Month 24 OLE analyses were not powered for comparisons and had no alpha allocated; negative PUL 2.0 total-score changes indicate decline.