Capricor's Duchenne Cell Therapy Achieves Breakthrough in Phase 3 Trial, Setting Stage for FDA Approval

Capricor Therapeutics has made a significant leap forward in the treatment of Duchenne muscular dystrophy (DMD) with its cell therapy, deramiocel, achieving remarkable success in a pivotal Phase 3 trial. The results have not only revitalized the company's prospects but also ignited hope for patients suffering from this debilitating genetic disorder.

HOPE-3 Trial Results Exceed Expectations

The HOPE-3 trial, involving 105 boys and young men with DMD, demonstrated that deramiocel substantially slowed the progression of the disease. Patients receiving the therapy every three months for a year showed a 54% slower deterioration in upper limb function compared to those on placebo. This outcome met the study's primary endpoint with statistical significance.

Moreover, the trial revealed a striking 91% reduction in the decline of left ventricular ejection fraction, a crucial measure of heart function, in 83 patients. This key secondary endpoint further underscores the potential of deramiocel to address both skeletal and cardiac complications of DMD.

Linda Marbán, Ph.D., CEO of Capricor, emphasized the therapy's potential, stating, "We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the complete response letter received earlier this year."

Market Response and Regulatory Outlook

The announcement of these positive results triggered a dramatic market response, with Capricor's stock price soaring by approximately 300%. Shares that closed at $6.36 on Tuesday reached a high of $40.37 before settling around $25.56 by late morning on December 3.

This turnaround comes after a challenging period for Capricor, which faced an FDA rejection of deramiocel in July 2025. The company now plans to submit a response to the FDA's complete response letter (CRL), incorporating the latest HOPE-3 data. Capricor expressed confidence that these results should be sufficient to support regulatory approval, citing prior alignment with the FDA on the path forward.

Implications for DMD Treatment

If approved, deramiocel would become the first drug available to treat Duchenne cardiomyopathy, the leading cause of death in DMD patients. The therapy, derived from donor cardiac cells called cardiosphere-derived cells, aims to reprogram macrophages to combat inflammation.

Kristen Kluska, an analyst at Cantor Fitzgerald, noted that the results far surpassed investor and analyst expectations. "We expect many physicians will elect to use this therapy, particularly given a strong safety profile," Kluska wrote, adding that prior discussions with doctors indicated intrigue but a need for more data – a need that today's update fulfills.

As Capricor prepares its response to the FDA, the DMD community watches with renewed hope. The potential approval of deramiocel could mark a significant milestone in the treatment of this devastating disease, offering patients and their families a new avenue for managing both skeletal and cardiac symptoms of Duchenne muscular dystrophy.