FDA Disputes Capricor Deramiocel Phase 3 Results, Shares Plunge 65%

The FDA’s review staff concluded that Capricor Therapeutics’ (CAPR) lead candidate deramiocel did not meet its pre-specified primary and secondary efficacy endpoints in the registrational HOPE-3 Phase 3 trial, according to briefing documents published July 27, 2026. The documents were released two days before a July 29 advisory committee meeting to review the BLA for Duchenne muscular dystrophy cardiomyopathy.

CAPR shares fell more than 65% to $6.53 in intraday trading on July 27, according to BioSpace. The stock had closed at $19.70 on July 25, the prior trading day. The crash wiped out roughly $700 million in market capitalization.

What the FDA said

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee is scheduled to meet July 29 to review BLA 125842. In briefing documents posted ahead of the meeting, agency reviewers wrote that HOPE-3 “did not meet its pre-specified primary and secondary efficacy endpoints, showing no statistically significant difference between deramiocel and placebo at 12 months.”

Capricor reported in December 2025 that deramiocel hit both the primary and secondary endpoints in HOPE-3. The FDA’s disagreement centers on post-hoc changes to the statistical analysis plan. According to the briefing documents, “changes were made to the pre-specified statistical analysis plan (SAP), generating at least two additional versions” after the randomized, double-blind portion of the trial was completed and during the open-label extension period. The modifications included changes to the primary and key secondary endpoint definitions, analytical methods, and data imputation strategy.

The FDA also raised concerns about the integrity of blinding in HOPE-3. The briefing documents noted a “distinctive adverse event profile observed between treatment groups,” with hypersensitivity reactions occurring in 42% of deramiocel-treated patients versus 15% of placebo-treated patients. That imbalance, reviewers wrote, “raises the possibility that treatment assignment could be inferred even under formal blinding conditions.”

Capricor’s response

CEO Linda Marban told BioSpace the company “takes great issue with how FDA analyzed the data using [SAP version 1.1],” which she described as “an unsigned, incomplete internal draft that became obsolete with the addition of cohort B.” Marban said the FDA received that draft only when it requested all draft SAPs during an information request following the BLA resubmission.

Marban also said Capricor attempted to schedule a meeting with the FDA to discuss the analysis but the agency is “refusing to meet with us.”

Capricor issued a statement defending the HOPE-3 results, saying the trial “demonstrate[s] a statistically significant benefit on the primary endpoint [upper limb performance], with supportive benefits in cardiac function.”

Regulatory history

This is the second FDA review cycle for deramiocel. The agency issued a Complete Response Letter in July 2025 after canceling a planned AdComm without notifying the company. Capricor resubmitted the BLA, and the FDA rescinded the CRL before scheduling the July 29 meeting. That unusual reversal was the basis for our earlier Capricor deramiocel AdComm preview, which flagged the meeting as a binary catalyst.

Deramiocel (CAP-1002) is an allogeneic cardiosphere-derived cell therapy with immunomodulatory and anti-fibrotic properties. It targets the cardiac complications of DMD, the leading cause of death in Duchenne patients. If approved, it would be the first cell therapy for DMD cardiomyopathy.

What to watch next

Update July 31, 2026 (Polygon): The CTGTAC voted 9-3 against deramiocel on July 29, siding with the FDA staff concerns outlined in these briefing documents. See our full coverage: FDA Panel Votes 9-3 Against Capricor Deramiocel for DMD. The August 22 PDUFA now leans toward rejection. CAPR closed at $3.85 on July 31 (Polygon), down 80.6% from the $19.83 article reference price. Market cap has fallen below $100 million.

The July 29 AdComm vote. The FDA’s briefing documents suggest agency staff will recommend against approval, but the committee of outside experts could disagree based on the company’s analysis and patient testimony. A negative vote would make the August 22 PDUFA date a likely rejection. A positive or split vote could preserve a narrow path given the unmet need in DMD cardiomyopathy.

FDA briefing documents (PDF) | BioSpace coverage | FDA AdComm meeting announcement