Capricor’s Positive 5-Year Data For DMD Treatment Comes With A Regulatory Catch – CAPR Stock Slides 11%

The U.S. Food and Drug Administration will hold an advisory committee meeting to review the biologics license application for Capricor’s DMD therapy on July 29

• Capricor Hope-2 extension study showed Deramiocel continued to slow disease progression over five years.
• The therapy also maintained stable cardiac function throughout the study.
• The company said that the biologics license application for Deramiocel remains on track for the Prescription Drug User Fee Act target action date of Aug. 22, 2026.

Capricor Therapeutics (CAPR) was in the spotlight after the biotech company reported positive five-year data for its Duchenne muscular dystrophy cell therapy, Deramiocel, and confirmed that the U.S. Food and Drug Administration will hold an advisory committee meeting to review its biologics license application on July 29.

The company added that the BLA remains on track for the Prescription Drug User Fee Act (PDUFA) target action date of Aug. 22, 2026. A PDUFA date is the deadline by which the FDA decides whether to approve a drug for marketing in the United States.

In March, the FDA lifted a previously issued Complete Response Letter and resumed review of its BLA seeking full approval of the drug.

CAPR stock was down 11% at the time of writing.

Deramiocel Study Shows Favorable Safety Profile Over Five Years

Capricor Therapeutics’ trial data showed that among patients who remained in the Hope-2 extension study, Deramiocel continued to slow disease progression over five years, with patients showing less decline in upper limb function than expected.

The therapy also maintained stable cardiac function throughout the study and continued to demonstrate a favorable safety profile, with no new safety concerns reported after more than 800 intravenous infusions.

The long-term results from the ongoing Hope-2 open-label extension study will be presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference alongside previously reported Phase 3 HOPE-3 data.

Retail’s Take On CAPR

Retail sentiment for CAPR on Stocktwits shifted to ‘extremely bullish’ from ‘bullish’ a day earlier, amid ‘high’ message volumes.

Users debated whether the advisory committee meeting could put the brakes on CAPR’s BLA.

One user expects approval ahead of the PDUFA date.

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However, another user said that an advisory committee meeting is “not good at this stage.”

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The stock has shed around 4% of its value so far this year.

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