Capricor Therapeutics, which received early funding from CureDuchenne, provided an update on their Deramiocel program in Duchenne after their recent FDA meeting. This meeting with the FDA was to address the concerns in the Complete Response Letter (CRL) that Capricor received from the FDA in July 2025, and align on a path forward toward consideration of approval for Deramiocel.
As a result of that meeting, Capricor reports that their HOPE-3 trial will be the “additional study” requested in the CRL. That trial is complete, and topline data is expected mid-Q4 2025.
The data from the HOPE-3 data can be submitted to the FDA under the current Biologics License Application (BLA) with the FDA, and will utilize the PUL 2.0 as the primary endpoint. Capricor also plans to suggest that the cardiac left ventricular ejection fraction (LVEF) be considered as a secondary endpoint, and that the label encompass both the cardiac and skeletal muscle function in Duchenne.
Link to press release: https://www.capricor.com/investors/news-events/press-releases/detail/326/capricor-therapeutics-provides-regulatory-update-on
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