Capricor Therapeutics (NASDAQ:CAPR) shares tumble 67% after FDA raises concerns over Deramiocel data

CAPR shares were last changing hands at $6.44, dropping 67.3% from the previous session, as of 10:57 Eastern. Trading volume reached 12.7 million shares, with an intraday low of $5.32. The selloff followed a sharply critical FDA briefing document on deramiocel. FDA reviewers raised questions on efficacy, post-hoc analyses, and the patient group included in the study. Attention is now fixed on Wednesday’s upcoming advisory vote, which is expected to drive short-term share moves. Reuters

HOPE-3 enrolled 106 patients and followed them for twelve months, administering four infusions at three-month intervals. The predefined upper-limb difference was 0.66 points, yielding a p-value of 0.24. The predefined LVEF difference was minus 0.041 percentage points, with a p-value of 0.97. Both outcomes failed to reach conventional statistical significance. As a result, the FDA states that the original primary and secondary efficacy endpoints were not achieved. U.S. Food and Drug Administration

Capricor based its findings on later analyses that used alternative endpoint definitions and underlying assumptions. The company’s submitted PUL analysis reported a 4.55 percentage-point benefit, with p=0.029. A ranked LVEF analysis indicated an 11.65-point difference, nominal p=0.041. The FDA notes these methods were altered after the blinded study ended. Reviewers state that how missing data for two placebo participants were handled had a significant impact on the outcomes. Capricor claims the FDA did not offer feedback before it finalized its statistical plan in November. U.S. Food and Drug Administration

The FDA notes the average starting LVEF in the HOPE-3 group was 57%. Only five participants, corresponding to 4.7%, began with LVEF under 45%. As a result, reviewers raise doubts about whether most of the group had confirmed DMD cardiomyopathy. This point is significant since the proposed indication specifically addresses this heart condition. The inconsistency is central to the discussion of whether HOPE-3 backs the requested claim. U.S. Food and Drug Administration

The panel is scheduled to convene July 29, running from 9:30 a.m. to 4:50 p.m. Eastern. Its formal agenda centers on evaluating whether HOPE-3 delivers substantial evidence of effectiveness. The indication under review is cardiomyopathy tied to Duchenne muscular dystrophy. The committee’s recommendation is advisory only, with the FDA retaining ultimate decision-making power. Monday’s 67% drop signals the significance investors assign to this vote. U.S. Food and Drug Administration

Capricor’s most recent corporate update still lists August 22, 2026, as the PDUFA target date. The FDA resumed its review and categorized the submission as a Class 2 resubmission. The initial application received a Complete Response Letter due to inadequate efficacy data. While a negative panel vote would elevate the chance of rejection, it would not directly decide approval. The FDA can disagree with its advisers, so the ultimate outcome is uncertain. Capricor Therapeutics, Inc.

The HOPE-3 trial recorded hypersensitivity reactions in 41.5% of patients given deramiocel, compared to 15.4% in the placebo group, prompting the FDA to question the risk of functional unblinding. Six serious adverse events were observed, with five occurring in the placebo arm and one in the deramiocel arm. Among these, a placebo patient experienced life-threatening anaphylaxis, which may be associated with shared excipients. The FDA considers the benefit-risk ratio unfavorable if efficacy is not demonstrated. U.S. Food and Drug Administration

Capricor reported $278.6 million in cash and securities as of March 31, with no revenue in the first quarter and a net loss of $33.9 million. The company said available funds should last through the fourth quarter of 2027. Its latest market value at $6.44 per share came to about $370 million, around $91 million higher than total cash and securities at the end of March. This figure does not include second-quarter expenditures or liabilities totaling $47.6 million. SEC

Capricor reports its San Diego site has undergone and completed FDA inspection, with Form 483 observations resolved. The FDA notes in a briefing that products from Los Angeles and San Diego are comparable. The main dispute now surrounds clinical efficacy, not manufacturing-site equivalence. This marks a positive development. Full approval still depends on resolving all manufacturing concerns. SEC