Capricor plans BLA amendment as FDA extends review

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Capricor Therapeutics announced plans to amend its Biologics License Application for Deramiocel after an FDA advisory committee voted 3 to 9 against the cardiomyopathy question, with the PDUFA target action date set for August 22. CEO Linda Marbán said the company will submit an amendment including 24-month open-label extension data from the HOPE-3 study and additional analyses, and the FDA has indicated willingness to review it and extend the action date upon receipt. The company also disclosed a statistical model change that shifted the LVEF endpoint from a 2.4 percentage point treatment difference with a p-value of 0.04 to a 1.8 percentage point difference with a p-value of 0.09, while the primary skeletal function endpoint remained statistically significant. Capricor reported cash and marketable securities of approximately $237.9 million as of June 30, 2026, a net loss of $40.7 million or $0.70 per share for the second quarter, and said all non-Deramiocel pipeline work is on hold pending regulatory clarity.

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Biotech & Genomic Medicine · 1 stocks
Capricor Therapeutics Inc
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FDA advisory committee voted against cardiomyopathy question and FDA extended review, delaying approval.

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