CAPR Stock Rises Nearly 5% As Capricor Presents Heart Data For Deramiocel Ahead Of November FDA Decision

• Chief executive Linda Marbán said that the company would pursue a heart indication for Deramiocel later.
• Capricor is currently seeking approval for Deramiocel, highlighting improvement in upper limb function.
• FDA is slated to decide on Deramiocel by Nov. 22.

Shares of Capricor Therapeutics rose 5% on Wednesday after the company presented clinical data from the trials of its experimental cell therapy Deramiocel for Duchenne muscular dystrophy, including stabilization of heart function in boys who already had heart damage, in addition to previously announced improvements in limb function.

Duchenne muscular dystrophy is a severe, progressive genetic disorder that causes muscle degeneration and weakness, starting with the limbs and eventually impacting the heart and breathing muscles. Capricor is now awaiting a U.S. Food and Drug Administration verdict on approving the drug.

Deramiocel Heart Findings

Craig McDonald, the lead investigator of the late-stage HOPE-3 trial at UC Davis, said on Wednesday on a Parent Project Muscular Dystrophy webinar that 64 of 106 boys already had weakened hearts. In that group, he said treatment produced “essentially complete stabilization” over a year, a gap of about 2.8 percentage points in the share of blood the heart pumps out, against the decline expected on placebo.

In the follow-up year, when patients who had been on placebo could switch to the drug, he said the gap was nearly 2 points at 24 months.

Nathaniel Hogan, a company statistician, added a combined look at the mid-stage HOPE-2 trial as well as HOPE-3: in boys who already had heart damage, the difference was 3.1 points at 12 months. Chief executive Linda Marbán said that the company would pursue a heart indication for Deramiocel later. The company is currently seeking approval, highlighting the drug’s impact on upper limb function in DMD patients.

CAPR Puts Forth Defense The FDA Did Not Request

Hogan also said the company re-ran the HOPE-3 results more than 600 ways, changing the math, which patients were counted, and how missing data were handled. In most of those versions, patients on deramiocel still kept at least one extra point of arm and hand function versus placebo. None of the re-runs wiped the benefit out.

Over a year, patients on the drug across HOPE-2 and HOPE-3 lost less than one point of arm function, he added. Patients on placebo lost about two. The gap was 1.29 points.

Marbán said the FDA had not asked for this extra work. Capricor did it on its own and added it to the updated application now under review.

Capricor had already announced that boys who switched from placebo to deramiocel in the follow-up year slowed their loss of arm function by 76% versus their own prior year. Patients treated from the start declined at a similar pace in both years, and both groups lost less function than a natural-history model predicted, the company said.

CAPR's Past Efforts At FDA Approval

Capricor’s first application seeking approval for the therapy was rejected in July 2025, citing a lack of substantial evidence of effectiveness. The company resubmitted the application earlier this year with late-stage HOPE-3 trial data, and the agency said it will decide on the application by August 22. However, an advisory committee met at the end of July and voted 9–3 that the evidence did not support effectiveness for cardiomyopathy.

Capricor then submitted additional 24-month clinical data analyses from the HOPE-3 trial and asked FDA to consider a refined label built on the trial’s primary endpoint—preservation of upper-limb function—rather than cardiomyopathy. The agency treated that package as a major amendment and moved the decision to Nov. 22.

How Did Retail Traders React?

On Stocktwits, retail sentiment around CAPR stock fell from extremely bullish to bullish territory over the past 24 hours, while message volume fell from extremely high to high levels.

A Stocktwits user cheered the data shared on Wednesday as unbelievable and raised their hopes for approval.

Another user said they are “super bullish” on the stock and on approval for the cell therapy.

CAPR stock has fallen 72% year-to-date. 

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