CAPR Stock Rises After Deramiocel Cardiac Data Wins American Heart Association Award Ahead Of FDA Decision

The U.S. Food and Drug Administration is slated to decide on approving Deramiocel for the treatment of DMD by November 22.

• The award, granted on Saturday, recognized a presentation by lead author Jonathan Soslow of Vanderbilt University Medical Center.
• Last week, Craig M. McDonald, a lead investigator for Capricor’s trial, shared on LinkedIn that the 28 physician-scientists who participated in Capricor’s late-stage HOPE-3 trial had written to FDA leadership, urging the agency to approve the drug. 
• Capricor initially sought approval for cardiomyopathy associated with DMD for Deramiocel, but the company’s initial application was rejected citing a lack of substantial evidence of effectiveness.

Shares of Capricor Therapeutics (CAPR) rose about 8% on Monday after the company said that cardiac results from its HOPE-3 late-stage trial of Deramiocel in Duchenne muscular dystrophy (DMD) received the Outstanding Research Award in Pediatric Cardiology at the American Heart Association Scientific Sessions.

The award added momentum to the stock ahead of a pivotal regulatory decision on the company’s therapy. The U.S. Food and Drug Administration is slated to decide on approving Deramiocel for the treatment of DMD by November 22. DMD is a rare, inherited genetic disorder that causes progressive muscle degeneration and weakness that presents predominantly in young boys.

Fresh Award Adds To Optimism Around CAPR

The award, granted on Saturday, recognized a presentation of HOPE-3 cardiac results by lead author Jonathan Soslow of Vanderbilt University Medical Center. The data, drawn from the 106-patient trial, highlighted potential benefits of the therapy on heart function and scarring in a disease characterized by heart complications.

“Cardiomyopathy is the leading cause of death in patients with Duchenne muscular dystrophy. There is no therapy approved specifically to treat DMD cardiomyopathy; therefore, development of novel therapeutics that can slow or stop its progression is of the utmost importance,” said Soslow.

HOPE-3 trial randomized patients with Duchenne muscular dystrophy to receive Deramiocel or placebo for 12 months. The study previously met its primary endpoint of improved upper-limb function. Heart function, measured by left ventricular ejection fraction or the percentage of blood the heart pumps with each beat, improved by 1.8 percentage points versus placebo, though the difference did not reach statistical significance.

In a pre-specified group of patients who already had cardiomyopathy, the benefit was larger at 2.8 percentage points. Patients treated with Deramiocel also showed slower progression of heart scarring on MRI imaging.

Capricor’s Rocky Road Towards Approval

Capricor initially sought approval for cardiomyopathy associated with DMD for Deramiocel. The company’s first application was rejected in July 2025, citing a lack of substantial evidence of effectiveness. The company resubmitted the application earlier this year with 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.

Last week, Craig M. McDonald, a lead investigator for Capricor’s trial, shared on LinkedIn that the 28 physician-scientists who participated in Capricor’s late-stage HOPE-3 trial had written to FDA leadership, urging the agency to approve the drug. The letter stressed the stakes for patients with DMD who cannot walk. No approved therapy has shown efficacy in this population, the investigators noted. They also noted that all the physicians involved in the trial believe it would be “unethical” to proceed with another trial to prove efficacy, given existing data.

How Did CAPR Retail Traders React?

On Stocktwits, retail sentiment around CAPR stock stayed within the 'bullish' territory over the past 24 hours, while message volume stayed at 'high' levels.

Stocktwits users are now awaiting the FDA’s decision on the drug and the company’s third-quarter earnings slated for Tuesday.

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Another user highlighted the prestigious nature of the award granted to Capricor.

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CAPR stock has dropped 66% year-to-date. 

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