Capricor's Deramiocel Shows Promise in Phase III Trial for Duchenne Muscular Dystrophy Cardiomyopathy

Capricor Therapeutics has announced positive results from its pivotal Phase III HOPE-3 trial for deramiocel, a cell therapy targeting Duchenne muscular dystrophy (DMD) cardiomyopathy. The treatment demonstrated significant benefits in both upper-limb function and cardiac function, potentially addressing a critical unmet need in DMD patient care.

Pivotal Trial Results

The HOPE-3 trial, which focused on a largely non-ambulatory DMD population, met both its primary and key secondary endpoints. Deramiocel showed a 54% slowing of skeletal muscle disease progression compared to placebo, an effect that principal investigator Dr. Craig McDonald of UC Davis Health described as "extraordinary in Duchenne." Additionally, the treatment slowed the decline in cardiac function by 91%, as measured by left ventricular ejection fraction.

"This call this morning represents the culmination of 20 years of work," said Capricor CEO Linda Marbán during a conference call with analysts. "We are now proud to be able to present today the data ... that shows that the [HOPE-3 trial] has shown statistically significant improvement in both skeletal and cardiomyopathy."

Regulatory Implications

The positive results come six months after the FDA issued a Complete Response Letter (CRL) to Capricor, citing a "lack of substantial evidence of effectiveness" for deramiocel. The company plans to use the HOPE-3 data to address the clinical issues raised in the CRL, consistent with prior FDA guidance that these results should be sufficient to support regulatory approval.

"We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 [open label extension] studies, position us to address the clinical issues in the Complete Response Letter received earlier this year," Marbán stated.

Industry Context and Challenges

The development of deramiocel has been marked by regulatory challenges and communication issues with the FDA. In June, a previously scheduled advisory committee meeting for the therapy was unexpectedly canceled, catching Capricor off guard. The company also found itself at the center of controversy surrounding personnel changes at the FDA's Center for Biologics Evaluation and Research.

Despite these setbacks, Capricor remains focused on the potential impact of deramiocel on DMD patients. Marbán emphasized the importance of addressing cardiomyopathy in DMD, noting that it is typically the aspect of the disease that leads to mortality. "Duchenne takes typically between 25 and 30 years to take the life of a boy or a young man," she explained. "We hope to be able to attenuate the course of this disease with deramiocel moving forward."

As Capricor prepares to resubmit its application to the FDA, the positive HOPE-3 results represent a significant step forward in the treatment of DMD cardiomyopathy, offering new hope to patients and families affected by this devastating condition.