San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) disputed the post-hoc analyses in the FDA's briefing materials, two days before an advisory committee meets on July 29 to discuss its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD).
Capricor says FDA analyses rely on an obsolete draft analysis plan
The company said the FDA's post-hoc analyses use statistical analysis plan (SAP) version 1.1. Capricor described that version as an unsigned, incomplete internal draft that became obsolete when cohort B was added. It also said the draft lacked content the FDA had specifically requested.
Capricor said its results are governed by SAP version 3.0, which was finalized before unblinding.
Phase 3 HOPE-3 data show a statistically significant primary endpoint benefit, company says
According to Capricor, the Phase 3 HOPE-3 trial demonstrated a statistically significant benefit on the primary endpoint, PUL 2.0, with supportive benefits in cardiac function. CEO Linda Marbán said the company has been responsive to every FDA request throughout the review. She described Deramiocel as a meaningful option for boys and young men with Duchenne who face a significant unmet medical need.
Capricor posts portion of meeting slides to its investor website
Capricor said it has posted a portion of its slides ahead of the meeting to frame the discussion for stakeholders. The materials will be available shortly in the Investors section of the company's website.
Deramiocel has not been approved for commercial use in any indication. The advisory committee's discussion forms part of the FDA's ongoing review of the application.