Capricor Therapeutics Inc. (NASDAQ:CAPR) stock nose-dived on Thursday after the FDA Advisory Committee voted against its Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD).

The agency assigned a PDUFA target action date of August 22.

FDA Advisory Panel Questions Deramiocel Effectiveness

Reuters reported that the FDA advisory panel voted 9-3 that the company failed to provide sufficient evidence supporting Deramiocel’s effectiveness, echoing FDA staff concerns over post hoc changes to key analyses after the late-stage trial concluded.

In the FDA briefing document released on Monday, the staff noted that the submitted data from Study HOPE-3, considered together with the results from Study HOPE-2, does not provide substantial evidence of effectiveness for deramiocel in DMD.

"Overall, Study HOPE-3 demonstrates small and variable changes in measures of upper limb skeletal muscle function and in several cardiac imaging parameters that did not reach statistical significance and are, thus, difficult to interpret."

The results from Study HOPE-2 and HOPE-2-OLE did not demonstrate a statistically significant treatment difference between deramiocel and placebo on upper limb skeletal muscle decline or cardiac imaging findings (LVEF, LGE, and others) on cardiac MRI.

Company Highlights Peer-Reviewed HOPE-3 Findings

On Wednesday, The Lancet, a peer-reviewed journal in medicine, published results from Capricor Therapeutics’ pivotal Phase 3 HOPE-3 trial evaluating Deramiocel.

The company said that the manuscript underwent independent expert peer review, providing external validation of the trial’s design, statistical methodology and findings.

The results are based on the company’s prespecified Statistical Analysis Plan version 3.0 (SAP 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, 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.

"A 54 percent slowing of upper limb disease progression (p=0.03) is a substantial, meaningful effect in a population where functional decline is typically relentless and irreversible."

"HOPE-3 is the first Phase 3 trial to demonstrate a significant benefit on function in a largely non-ambulatory DMD population, and the concurrent benefits in several cardiac measures lend biological support to a consistent treatment effect across skeletal and cardiac muscle…"

CAPR Price Action: Capricor Therapeutics shares were down 52.36% at $3.13 at the time of publication on Thursday. The stock is trading at a new 52-week low, according to Benzinga Pro data.

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