Capricor Therapeutics Reports Long-Term Efficacy of Deramiocel for Duchenne Muscular Dystrophy

Capricor Therapeutics, a biotechnology firm focused on developing cell and exosome-based treatments for rare diseases, has announced promising long-term data from its HOPE-2 open label extension clinical trial. This study highlights the effectiveness of their leading product, deramiocel, in slowing the progression of Duchenne muscular dystrophy (DMD) and preserving upper limb function. The data was shared at the 2025 Muscular Dystrophy Association Clinical and Scientific Conference held in Dallas, Texas.

The trial demonstrated that patients receiving deramiocel over a span of three years experienced a reduced decline in their Performance of the Upper Limb (PUL 2.0) total score, averaging a decrease of 3.46 points. This is notably lower compared to a 7.19-point decline observed in a matched external comparator group, translating to a 52% decrease in disease progression. This significant finding underscores the potential of deramiocel for long-term therapeutic benefits.

The study also revealed that the treatment effect of deramiocel improved over time. Patients showed a yearly decline in the PUL 2.0 score by 1.8 points in the first year, 1.2 points in the second year, and only 1.1 points in the third year. Additionally, even during a one-year treatment gap, those initially treated with deramiocel showed a slower decline rate of 2.8 points per year, compared to the 3.7 points per year decline in untreated patients.

Deramiocel has displayed a favorable safety profile, with no new safety concerns arising and a continued positive benefit-risk balance. This adds to its attractiveness as a potential treatment option for DMD.

Duchenne muscular dystrophy is a severe genetic disorder characterized by the progressive weakening and chronic inflammation of skeletal, cardiac, and respiratory muscles, often leading to mortality around the age of 30. The disorder affects approximately 15,000 to 20,000 individuals in the United States. DMD results from deficient production of functional dystrophin, a protein crucial for maintaining muscle integrity. This deficiency causes significant muscle cell damage, leading to scar tissue formation and muscle cell death. The resulting cardiomyopathy is the primary cause of mortality in DMD patients, and current treatment options remain limited.

Deramiocel, also known as CAP-1002, consists of allogeneic cardiosphere-derived cells (CDCs). These cells have demonstrated potent immunomodulatory and antifibrotic effects in both preclinical and clinical studies, helping to preserve cardiac and skeletal muscle function in conditions like DMD. CDCs function by releasing exosomes, which modify macrophages to promote healing over inflammation. With over 100 scientific publications and more than 200 human subjects treated in various clinical trials, CDCs have shown significant promise.

Capricor Therapeutics has made strides in advancing their treatment for DMD. The company has recently submitted a Biologics License Application to the U.S. Food and Drug Administration (FDA) for the use of deramiocel in treating cardiomyopathy associated with DMD. The FDA has set a target action date of August 31, 2025, under the Prescription Drug User Fee Act.

Capricor remains committed to redefining treatment paradigms for rare diseases through its innovative therapies. Aside from deramiocel, the company is leveraging its exosome technology platform, StealthX™, to develop preclinical programs in vaccinology and targeted delivery of various therapeutic agents. The company's mission is to continue breaking new ground in treatment options for those afflicted by rare and challenging diseases.