Capricor Therapeutics (CAPR) Study result summary

Regulatory and indication

• FDA extended the PDUFA date from Aug. 22 to Nov. 22, 2026, after classifying the BLA amendment as major; the BLA remains under active review.

• The refined indication seeks treatment of upper-limb impairment in patients with DMD ages 9 and older.

• The amendment added HOPE-3 12-month results, robustness analyses, 24-month OLE muscle and cardiac data, and integrated HOPE-2/HOPE-3 efficacy analyses.

• Deramiocel remains investigational and is not approved for commercial use.

HOPE-3 phase 3 results

• HOPE-3 met its primary PUL 2.0 endpoint, with 4.55% better-preserved arm function versus placebo at 12 months (p=0.029).

• A pre-specified sensitivity analysis found 1.1 points less PUL 2.0 decline versus placebo at 12 months (p=0.050).

• More than 600 alternative analyses favored Deramiocel; the least favorable estimate was 0.64 points, and 95% of distinct values ranged from 0.74 to 1.17.

• Pooled HOPE-2/HOPE-3 results showed a 1.29-point PUL 2.0 benefit at Month 12 (p=0.0087; 95% CI 0.33–2.25); all eight sensitivity analyses had estimates of 1.11–1.31 points with confidence intervals excluding zero.

HOPE-3 open-label extension

• Of 106 randomized patients, 98 entered the OLE and 82 completed two years; all received Deramiocel in Year 2 after the randomized first year.

• Delayed-start patients had a 1.56-point difference in PUL 2.0 decline between their placebo year and Deramiocel year (95% CI 0.47–2.64), a 76% lower decline rate on treatment.

• Early-start patients’ Year 2 versus Year 1 difference was 0.05 points (95% CI −1.03 to 1.14); the Month 24 analyses were exploratory and not powered for comparisons.

• At Month 24, observed declines were 3.42 points for early-start and 3.76 for delayed-start patients, versus natural-history predictions of 5.88 and 5.35, respectively.