Capricor shares surge after new trials show benefit in muscular drug

Capricor Therapeutics Inc. reported positive trial data for its experimental Duchenne muscular dystrophy treatment, sending its shares to a record intraday gain. The company said the findings may support a renewed bid for US regulatory clearance after an earlier rejection.

The San Diego-based firm said the therapy, Deramiocel, showed significant improvement in heart and upper limb function among patients enrolled in a placebo-controlled study. The trial involved more than 100 participants with Duchenne muscular dystrophy, a rare disorder that primarily affects boys and young men. Patients often face progressive muscle loss and commonly die from cardiac or respiratory failure.

The stock rose more than sixfold after the results were released Wednesday. The company had dropped 54% this year through the prior session’s close.

US regulators rejected Capricor’s application in July. The Food and Drug Administration said the filing lacked substantial evidence showing the drug’s effectiveness. The agency also cited outstanding issues tied to chemistry, manufacturing and controls in its complete response letter. Capricor has said it believes most of those items have since been addressed.

Capricor said the new data from the Hope-3 study, combined with information from earlier trials, responds directly to the concerns outlined by the FDA. The company said the drug showed a favorable safety and tolerability profile in the latest results.

The firm said the trial outcomes strengthen its case for approval and provide support for resubmitting its application. The company characterized the findings as a key milestone in advancing a potential treatment option for a patient population with limited therapeutic choices.

In a research note released after the announcement, Cantor analyst Kristen Kluska said the results match the criteria the FDA is likely to review when considering whether to change its position on the drug’s approvability.