CAPR Stock Rises As Clinical Trial Investigators Write To FDA Urging Approval For Deramiocel

• Craig M. McDonald, a lead investigator for Capricor’s trial, shared on LinkedIn that 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, dated Oct. 25, highlighted trial results and stressed the stakes for patients with Duchenne muscular dystrophy who cannot walk. 
• No approved therapy has shown efficacy in this population, the investigators noted.

Shares of Capricor Therapeutics (CAPR) climbed roughly 14% on Tuesday as a public letter from clinical investigators added momentum ahead of a pivotal regulatory decision on the company’s therapy Deramiocel.

The FDA is slated to decide on approving the drug for the treatment of Duchenne muscular dystrophy by November 22. Duchenne muscular dystrophy (DMD) is a rare, inherited genetic disorder that causes progressive muscle degeneration and weakness that presents predominantly in young boys.

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CAPR stock has gained 29% this week, after clocking 8% gains in the week ended Oct. 30.

Experts Write To FDA Seeking Approval

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, dated Oct. 25, highlighted trial results and stressed the stakes for patients with Duchenne muscular dystrophy who cannot walk. No approved therapy has shown efficacy in this population, the investigators noted.

They pointed to preserved upper-limb function in the trial, or the ability to self-feed, bring a hand to the mouth, or operate a powered wheelchair or communication device, as meaningful improvement. The group also referenced the FDA’s Rare Disease Framework Initiative, which encourages flexibility and consistency when reviewing therapies for small patient populations, and 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.

Capricor’s Rocky Road Towards Approval

Capricor initially sought approval for cardiomyopathy associated with DMD. 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 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 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.

A Stocktwits user pinned the stock’s rally on Tuesday to renewed optimism, while another pinned it to shorts covering ahead of a binary event.

Another user said that it is very hard to imagine a rejection after all the physicians involved in the study backed it.

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

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