Capricor Therapeutics (CAPR) Stock Plummets 70% Following FDA Concerns

Key Takeaways

Table of Contents

Shares of Capricor Therapeutics plummeted up to 70% following critical FDA staff commentary on deramiocel
Federal regulators highlighted that the company modified outcome measurements post-trial completion
FDA staff expressed doubts about whether study participants genuinely suffered from DMD-associated heart disease
Questions emerged regarding whether adequate amounts of the therapy successfully reached cardiac tissue
A scheduled advisory committee review is set for Wednesday, July 29

Shares of Capricor Therapeutics (CAPR) experienced a devastating 70% collapse on Monday following the release of FDA briefing materials in advance of the company’s scheduled July 29 advisory panel review.

Capricor Therapeutics, Inc., CAPR

The briefing documents from FDA staff members highlighted significant concerns regarding efficacy data for deramiocel, the company’s experimental cell-based treatment designed to address heart disease in male Duchenne muscular dystrophy (DMD) patients.

Trading activity showed the stock had declined approximately 40% during early market hours before accelerating losses throughout the remainder of the session.

The upcoming Wednesday session of the Cellular, Tissue, and Gene Therapies Advisory Committee will evaluate whether data from the critical HOPE-3 clinical trial demonstrates substantial proof of deramiocel’s therapeutic effectiveness.

A primary issue identified by the FDA involves Capricor’s decision to modify its methodology for evaluating the study’s primary endpoint after trial completion.

The biotech firm transitioned from measuring raw scores on a 42-point upper extremity functionality assessment to presenting findings as percentage-based changes. Federal reviewers determined this modification lacked proper scientific rationale.

“FDA does not consider the conversion of raw change to percent change and then back to raw change to have been scientifically justified, as it adds complexity and reduces accuracy,” the agency’s staff wrote.

Cardiac Assessment Methods Modified Mid-Stream

Changes weren’t limited to skeletal muscle evaluations. The methodology for assessing cardiac performance underwent revision as well. The initial protocol called for monitoring changes in left ventricular ejection fraction, but Capricor subsequently opted for a ranking-based patient assessment approach.

Federal reviewers emphasized that available data provided insufficient proof that therapeutically meaningful quantities of the intravenously administered treatment successfully reached cardiac tissue.

Doubts About Patient Population Selection

FDA staff also raised questions about whether trial participants genuinely exhibited DMD-associated cardiomyopathy. Reviewers pointed out that enrolled patients demonstrated average heart-pumping capabilities within normal parameters at baseline.

Deramiocel represents an experimental allogeneic cellular therapy manufactured from cardiac tissue obtained from deceased donors.

The company’s initial biologics license application relied on findings from the preceding phase 2 HOPE-2 investigation, which failed to demonstrate meaningful improvements in either skeletal muscle or cardiac performance. That submission resulted in a Complete Response Letter citing insufficient effectiveness evidence and an unfavorable risk-benefit profile.

Currently, no FDA-sanctioned treatments exist specifically targeting cardiomyopathy associated with Duchenne muscular dystrophy.

Available DMD therapeutics, including Sarepta Therapeutics’ (SRPT) gene therapy Elevidys and various exon-skipping medications, address the fundamental genetic disorder itself rather than the progressive cardiac complications that emerge as the disease advances.

The advisory committee evaluation is scheduled for Wednesday, July 29.

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