The company is slated to orally present long-term trial results of deramiocel at the World Muscle Society congress in Hiroshima on October 3.

  • The FDA is slated to decide on approval of Deramiocel by November 22. 
  • Capricor submitted the additional 24-month clinical data analyses to the FDA and sought a reevaluation of its application in August. 
  • Earlier this month, B. Riley upgraded the stock to Buy from Neutral with a $21 target, up from $5, in anticipation of the data being presented at the conference.

Shares of Capricor Therapeutics (CAPR) rose 10.5% on Wednesday, clocking its best day in over a month, as investors focused on the two-year HOPE-3 follow-up data on deramiocel, the company’s intravenous cell therapy for Duchenne muscular dystrophy, now circulating as a poster at the World Muscle Society congress in Hiroshima.

The company is slated to present the long-term trial results orally at the conference on October 3; it recently submitted the results to the FDA to support its application.

The poster for the presentation is already circulating on X. CEO Linda Marbán has called the follow-up one of the most extensive clinical datasets evaluating upper-limb function in Duchenne.

CAPR's Efforts At FDA Approval

Deramiocel is an allogeneic cell therapy given by IV infusion every three months. The company’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 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.

What The Two-Year Analysis Shows

HOPE-3 randomized 106 patients. Eighty-two reached 24 months in the open-label extension: 40 who started on deramiocel and 42 who started on placebo, then switched.

In the delayed-start group, estimated decline on the Performance of the Upper Limb 2.0 (PUL 2.0) scale slowed from 2.05 points in the placebo year to 0.49 points after crossover, a 76% reduction. Patients treated from the start declined 0.95 points in year one and 0.89 in year two. Both groups lost less function than an external natural-history model predicted. Investigators said the pattern pointed to a treatment-linked slowing of upper-limb decline in later-stage disease.

The shorter-term 12-month HOPE-3 readout, published in The Lancet, had shown a 54% slowing of upper-limb decline versus placebo. An oral HOPE-3 review is set for the last day of the conference.

Analyst Weighs In

Earlier this month, B. Riley upgraded the stock to 'Buy' from 'Neutral' with a $21 target, up from $5, in anticipation of the data being presented at the conference. Analyst Madison El-Saadi said the share setup is favorable into the November 22 FDA action date. Riley said it saw 50% upside in a positive scenario at the conference and expects the shares to hold near current levels in a negative scenario. Riley's price target applies a 40% approval probability to modeled deramiocel peak sales.

How Did Retail Traders React?

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

A Stocktwits user said that they now expect approval ahead of the FDA’s target date.

View this Stocktwits post

CAPR stock has fallen 67% year-to-date.   

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