Cell therapy biotech Capricor has best day in a month on updated data

Shares of small-cap biotech Capricor Therapeutics jumped 10.5% on Wednesday, posting their best day in more than a month. The market was reacting to updated clinical trial data for its lead candidate for treatment of Duchenne muscular dystrophy, a rare disease.

Capricor rose 10.5% on the Nasdaq on Wednesday to $9.50 per share. This was their best day in more than a month, Stocktwits notes.

At the World Muscle Society Congress in Japan, the company presented updated data on its experimental Duchenne muscular dystrophy therapy, deramiocel. The rare genetic disease, which primarily affects boys, causes progressive muscle weakness and premature death. The only gene therapy currently available for the disease is Sarepta Therapeutics’ Elevidys, but its use has been severely restricted.

The deramiocel data is currently being presented at the congress in a poster that is circulating widely on X, Stocktwits writes. The image was posted, among others, by Eduardo Marbán, whose scientific research underpins Capricor’s work. He is also the husband of CEO Linda Marbán. The poster contains two-year follow-up data for patients treated with deramiocel. The study evaluates their upper-limb function.

The company is scheduled to deliver an oral presentation on deramiocel on Saturday. The U.S. Food and Drug Administration is due to decide whether to approve the therapy on November 22.

This is not Capricor’s first attempt to secure FDA approval for deramiocel. In July 2025, the FDA rejected the initial application, citing a lack of substantial evidence of effectiveness. Early this year, Capricor resubmitted its application, but an FDA advisory committee voted against the therapy in late July. The FDA was due to issue its final decision on August 22, but the company provided updated two-year patient follow-up data and asked the regulator to consider a refined proposed indication.

Capricor’s first two applications focused on deramiocel’s efficacy in treating cardiomyopathy, or changes in the heart muscle, while the updated application focuses on preserving upper-limb function. The FDA classified the submission as a major amendment and extended its review deadline to November 22.

Against this backdrop, two Wall Street firms, Piper Sandler and B. Riley Securities, recommended buying Capricor in early September while hiking their target prices: Piper Sandler to $25 from $2 per share, and B. Riley Securities to $21 from $5 per share.

Wall Street overall remains cautious on the stock, however. According to MarketWatch data, it has six “hold” ratings versus four “buy” calls. The average target price is $33.56 per share, implying more than 253% upside from the last close.