Capricor’s DMD Therapy Gets Lancet Backing Ahead Of August FDA Showdown – CEO Says Publication ‘Reinforce Our Confidence’ For Approval

The publication comes ahead of a crucial U.S. Food and Drug Administration advisory committee meeting and the agency’s final approval decision in August.

• Deramiocel met its primary endpoint and reduced the decline in upper-limb function by 54% compared with placebo.
• The HOPE-3  study also reported benefits related to cardiac function.
• CAPR stock has been placed under a trading halt under the exchange’s T1 code.

Capricor Therapeutics (CAPR) has received a significant boost after The Lancet published results from its much-discussed Phase 3 HOPE-3 trial of Deramiocel for Duchenne muscular dystrophy (DMD).

The publication comes ahead of a crucial U.S. Food and Drug Administration (FDA) advisory committee meeting and the agency’s final approval decision in August.

CAPR stock has been placed under a trading halt under the exchange’s T1 code, which implies the release of material news is pending.

HOPE-3 Results A Landmark Moment For Duchenne Community, Says Lead Author

The HOPE-3 study enrolled 106 patients in a randomized, double-blind, placebo-controlled trial. Deramiocel met its primary endpoint and reduced the decline in upper-limb function by 54% compared with placebo. The study also reported benefits related to cardiac function.

Capricor said the manuscript was reviewed by independent experts and was based on the company’s pre-specified Statistical Analysis Plan (SAP) version 3.0.

“The HOPE-3 results are a landmark moment for the Duchenne community, demonstrating a significant benefit on skeletal muscle function alongside compelling data shown in cardiac function,” said Craig McDonald, Distinguished Professor of Physical Medicine & Rehabilitation and Pediatrics at UC Davis Health, National PI of the HOPE-2 and HOPE-3 trials and lead author of the publication.

Lancet Publication Strengthens Argument For Deramiocel

Capricor CEO Linda Marbán said the Lancet publication strengthens confidence in Deramiocel’s clinically meaningful benefits and supports the company’s approval efforts ahead of the August 22 Prescription Drug User Fee Act (PDUFA) decision. It is the FDA’s target date for making a decision on a drug application, which allows the drug to be marketed in the U.S.

“This is the same body of evidence that forms the foundation of our BLA and will be discussed at our Advisory Committee meeting. The publication reinforces our confidence in the strength and durability of these results in advance of Deramiocel's PDUFA target action date of August 22,” said Linda Marbán, CEO of Capricor.  

FDA Reviewers Raise Concerns

This comes after the FDA questioned whether the trial provides convincing evidence that the therapy works. Agency reviewers said the study failed to meet its original pre-specified primary endpoint for upper-limb function and a key secondary endpoint measuring heart function, known as left ventricular ejection fraction (LVEF).

The main disagreement centers on which SAP should be used. FDA staff argued that Capricor changed how the trial data were analyzed, potentially affecting the reported results.

Retail Remains Extremely Bullish

Retail sentiment on Stocktwits remained ‘extremely bullish’ over the past 24 hours, amid ‘extremely high’ message volumes. CAPR was among the top trending tickers at the time of writing.

One user explained when the halt in the stock could be lifted.

View this Stocktwits post

The stock is down nearly 77% so far this year.

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