Capricor Therapeutics sees stock surge as FDA signals openness to extend review of DMD cell therapy

Capricor Therapeutics' CEO Linda Marbán told investors that the FDA is receptive to adding open label extension data to its Biologics License Application for deramiocel, its Duchenne muscular dystrophy cell therapy, as the Aug. 22 deadline approaches.

The company plans to file an amendment that incorporates 24 month data from the HOPE 3 study and additional analyses, aiming to narrow the indication to the primary endpoint.

The update came during the second quarter earnings call and sent the company's shares up about 68%, reflecting investor optimism despite a recent advisory committee vote that recommended against approval.

If the FDA accepts the amendment, the review timeline could be extended, giving Capricor more time to demonstrate efficacy and safety for the therapy.