STORY RECORD
FDA blasts Capricor's Duchenne data integrity
FDA briefing documents released Monday for the July 29 advisory committee meeting state Capricor's deramiocel failed its Phase 3 HOPE-3 trial on primary and secondary endpoints at 12 months.
More damaging: the FDA says Capricor made unauthorized post-hoc changes to the statistical analysis plan after the double-blind phase ended, generating multiple late SAP versions and never submitting the final one for FDA agreement.
STAT News reports CEO Linda Marban stands by the efficacy claims. CAPR stock fell roughly 65% in pre-market trading.
Why It Matters
This isn't just another PhIII failure. The FDA is accusing Capricor of moving the goalposts after the game was over — rewriting endpoint definitions, analytical methods, and data imputation after unblinding, then filing without FDA knowledge.
If the advisory committee agrees, this destroys trust not just in deramiocel but in the company's entire development program. The stock collapse suggests the market had priced in approval as a high probability; now the PDUFA date of August 22 faces a much steeper hill.
The Facts
6FDA review of Capricor's BLA for deramiocel states that Study HOPE-3 did not meet its pre-specified primary or secondary efficacy endpoints, showing no statistically significant difference between deramiocel and placebo at 12 months.
multiple independent sources · confidence 0.85
After completion of the randomized double-blind portion of HOPE-3 and during the open-label extension HOPE-3-OLE, Capricor made changes to the pre-specified statistical analysis plan, generating at least two additional versions, with modifications to primary and key secondary endpoint definitions, analytical methods, and data imputation strategy.
multiple independent sources · confidence 0.85
The final version of the statistical analysis plan (SAP v.3.0, dated November 24, 2025) was not submitted to the FDA for review prior to BLA submission and was not discussed or agreed upon by the agency.
multiple independent sources · confidence 0.8
The FDA Cellular, Tissue, and Gene Therapies Advisory Committee will meet on July 29, 2026 to discuss BLA 125842 for deramiocel for cardiomyopathy in Duchenne muscular dystrophy.
official source direct · confidence 1
STAT News reported on July 27, 2026 that FDA questions the efficacy of Capricor's Duchenne drug, with comments from CEO Linda Marban. Capricor had claimed in December 2025 that deramiocel met both primary and secondary endpoints.
multiple independent sources · confidence 0.85
$CAPR stock fell approximately 65% in pre-market trading on July 27, 2026 from a Friday close of $19.70, trading near $6.83 on heavy volume.
single source · confidence 0.7