Key Takeaways:
Capricor Therapeutics defended its Duchenne therapy Deramiocel ahead of a July 29 FDA panel, after the stock plunged 67%.
"Our results are governed by the final analysis plan, SAP version 3.0, which was finalized prior to unblinding," Linda Marbán, chief executive officer of Capricor, said in a statement.
FDA reviewers said the Phase 3 Hope-3 trial failed to meet its pre-specified primary and secondary efficacy endpoints, finding no statistically significant difference between Deramiocel and placebo after 12 months. The reviewers also raised concerns over changes made to the statistical analysis plan, noting the final SAP was not submitted for review before the company's Biologics License Application. Capricor countered that the FDA's briefing materials rely on SAP version 1.1, an unsigned incomplete internal draft that became obsolete with the addition of cohort B.
The advisory committee's recommendation will feed into the FDA's Aug. 22 PDUFA decision, which determines whether Capricor can commercialize its only late-stage asset. The company has also signed a 171,000-square-foot headquarters lease in San Diego that is contingent on FDA approval by Dec. 31, 2026.
The Cellular, Tissue, and Gene Therapies Advisory Committee will review Capricor's BLA for Deramiocel, an allogeneic cardiac-derived cell therapy for cardiomyopathy in patients with Duchenne muscular dystrophy. There are no FDA-approved treatments specifically for the heart disease that develops as the condition worsens.
Capricor said the Phase 3 Hope-3 results demonstrate a statistically significant benefit on the primary endpoint measuring upper-limb function, with supportive benefits in cardiac function. The company posted slides on its website to frame the discussion ahead of the meeting.
Deramiocel has received Orphan Drug Designation from both the FDA and European Medicines Agency, as well as Regenerative Medicine Advanced Therapy designation in the US and Advanced Therapy Medicinal Product designation in Europe. The company said it has enough funds for operations through the fourth quarter of 2027.
CAPR shares traded near their lowest levels since December 2025 after the 67% single-day decline, the stock's biggest ever. The stock was on track for its first weekly gain in five weeks ahead of the panel meeting.
The advisory committee vote will set the tone for the Aug. 22 FDA decision and determine whether Capricor can transition from a clinical-stage developer to a commercial enterprise. Investors will watch the July 29 meeting for the panel's assessment of Deramiocel's efficacy and the statistical analysis dispute that has become central to the review.
This article is for informational purposes only and does not constitute investment advice.
