Capricor Stock Plunges Pre-Market After US FDA Declines Approval For Muscular Disorder Therapy

Capricor Therapeutics (CAPR) on Friday said the U.S. Food and Drug Administration (FDA) has refused to approve its lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy.

CAPR shares were trading 39% lower in the pre-market session.

The FDA told Capricor in a letter that it is unable to approve its application for Deramiocel in its current form as it does not meet the requirement for substantial evidence of effectiveness and expressed the need for additional clinical data.

Capricor said the health regulator also referenced certain outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section of the application.

The company said it plans to engage further with the FDA to determine the next appropriate steps.

Capricor CEO Linda Marbán expressed surprise at the FDA’s decision, calling it “unexpected.”  

“We have followed their guidance throughout the process. Prior to the CRL (complete response letter), the review had advanced without major issues, including a successful pre-licensure inspection and completion of the mid-cycle review,” she said.

The CEO added that the company plans to submit data from a late-stage trial of 104 patients to provide additional evidence of effectiveness. Results from the study are expected in the third quarter of 2025.

“We believe these data, if positive, along with our existing long-term clinical results showing cardiac stabilization, preservation of skeletal muscle function, and a consistent safety profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD,” she said.

Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. The disease affects approximately 15,000 individuals in the United States and primarily impacts boys.

Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD.

DMD therapies have been under increased scrutiny since last month after the FDA announced it is investigating the death of two pediatric patients with Duchenne Muscular Dystrophy (DMD) following treatment with Sarepta Therapeutics’ (SRPT) FDA-approved gene therapy Elevidys.

FDA is investigating the risk of acute liver failure with serious outcomes, including hospitalization and death, following Elevidys, and is evaluating the need for further regulatory action, the agency said.

On Stocktwits, retail sentiment around Capricor remained unmoved within ‘bearish’ territory over the past 24 hours while message volume rose from ‘low’ to ‘normal’ levels.

CAPR stock is down by 17% this year but up by over 148% over the past 12 months.

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