Capricor's Deramiocel Therapy Shows Promise in Treating DMD

Capricor's Deramiocel Therapy Shows Promise in Treating DMD

Igor Petrovich Kuznetsov
2 min read

Capricor Therapeutics' Three-Year Results for Deramiocel Cell Therapy Show Promise in Treating Duchenne Muscular Dystrophy

Capricor Therapeutics has recently released encouraging three-year findings regarding its deramiocel cell therapy for the treatment of Duchenne muscular dystrophy (DMD). The study revealed a 47% deceleration of disease advancement in skeletal muscle and a notable enhancement in cardiac function, especially among patients with initially higher ejection fractions. Despite all participants experiencing adverse events, predominantly of a mild nature, no severe cases were documented. These results advocate for early intervention to potentially impede the progression of cardiomyopathy, a predominant cause of mortality in DMD patients. Capricor is set to engage in discussions with the FDA to move forward with its biologics license application (BLA) in Q3 2024. Furthermore, a Phase III pivotal investigation is presently ongoing, with anticipated release of topline data in Q4 2024. Notably, there are currently no approved treatments specifically targeted at DMD cardiomyopathy, underscoring the urgent necessity for efficacious therapies in this domain.

Key Takeaways

  • Capricor Therapeutics to engage with the FDA for deramiocel BLA in Q3 2024.
  • Deramiocel demonstrates a 47% slowdown in DMD disease progression in skeletal muscle.
  • A three-year study indicates enhanced cardiac function, particularly in individuals with higher ejection fractions.
  • No severe adverse events reported, aligning with previous deramiocel investigations.
  • Capricor aims to file BLA based on sustained advantages and preceding trial outcomes.


Capricor Therapeutics' progress in DMD treatment has the potential to reshape the pharmaceutical landscape, exerting an influence on companies such as Sarepta and Pfizer. The financial markets are likely to react positively, enhancing Capricor's stock value and attracting investments. The FDA's endorsement could establish a precedent for early intervention in DMD, potentially transforming treatment protocols universally. In the short term, Capricor's market value and investor confidence are expected to soar. In the long term, wider adoption of deramiocel might redefine DMD management, significantly ameliorating patient results and reducing healthcare expenses linked to DMD cardiomyopathy.

Did You Know?

  • Deramiocel Cell Therapy:
    • Deramiocel cell therapy presents a pioneering approach where cells are derived from the patient's own body and then manipulated to bolster their therapeutic impacts. In the context of Capricor Therapeutics' investigation, these cells are utilized to address Duchenne muscular dystrophy (DMD), a genetic ailment distinguished by ongoing muscle degeneration and debility.
  • Duchenne Muscular Dystrophy (DMD):
    • DMD constitutes a severe form of muscular dystrophy predominantly affecting males. It is triggered by mutations in the gene responsible for dystrophin, a crucial protein for muscle cell durability and functionality. The disease leads to progressive muscle debilitation, impacting both skeletal muscles and the heart (cardiomyopathy), which can be perilous.
  • Biologics License Application (BLA):
    • A Biologics License Application (BLA) is a regulatory submission presented to the U.S. Food and Drug Administration (FDA) to secure authorization to market a biological product. Unlike a New Drug Application (NDA) for small molecule drugs, a BLA is requisite for biological products, typically intricate and derived from living sources. Capricor Therapeutics is preparing to discuss its deramiocel cell therapy findings with the FDA to potentially advance its BLA for approval.

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