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Updated: 14-Aug-26 14:53 ET
Capricor Therapeutics soars on Deramiocel update (CAPR)
- Capricor Therapeutics (CAPR) is soaring today, and while the biotech company focused on cell and exosome-based therapies for rare diseases reported Q2 results last night, investors are more focused on its Deramiocel update.
- For context, CAPR's 2024 BLA sought approval for Deramiocel for the treatment of cardiomyopathy in DMD. FDA declined to approve the application in 2025, and CAPR later resubmitted it with new data from HOPE-3, its Phase 3 study, while keeping the cardiomyopathy indication.
- However, HOPE-3 was designed around upper-limb function as its primary endpoint, with cardiac function as a secondary measure. Cardiomyopathy was still the question put to the FDA advisory committee on July 29, which voted 3 in favor and 9 against on whether the available evidence supported effectiveness for that indication, sending shares sharply lower.
- The big development last night is that Deramiocel remains under FDA review, and CAPR now plans to amend its application to focus on upper-limb function. The amendment will include 24-month HOPE-3 extension data and additional analyses of the existing results. FDA has indicated it is willing to review the amendment and extend the PDUFA date once it is submitted.
- This gives CAPR a potential approval path that is better aligned with HOPE-3's main result, where Deramiocel showed a statistically significant benefit. That said, FDA approval remains uncertain, and CAPR is slowing some commercial-readiness spending until the regulatory path becomes clearer.
