Capricor Moves Forward With DMD Cardiomyopathy Therapy

Food and Drug Administration (FDA) rejected Capricor Therapeutics is back in the regulatory spotlight with promising data for its investigational cell therapy, Deramiocel, after six months. Main reason behind the therapy treating Duchenne muscular dystrophy (DMD) cardiomyopathy demonstrated meaningful improvements in both upper-limb function and cardiac health in a pivotal Phase III trial, prompting Capricor to submit a renewed application to the FDA.

From Rejection to HOPE 3

In July 2025, the FDA issued a Complete Response Letter (CRL) for Capricor’s Biologics License Application (BLA) for Deramiocel, stating that the submitted data “fell short of the statutory requirement for substantial evidence of effectiveness.” The BLA had primarily relied on Phase II HOPE‑2 trial results, an open-label extension, and natural-history comparisons. The FDA’s decision came as a surprise to Capricor, given the positive interactions the company had previously experienced with regulators.

The FDA required a well-controlled Phase III trial after the CRL. The HOPE‑3 trial from Capricor followed FDA recommendations by using a randomized double-blind placebo-controlled design to study non-ambulatory and ambulatory DMD patients. The study used upper-limb function as its main outcome measure while left ventricular ejection fraction (LVEF) served as a vital secondary cardiac assessment to evaluate both skeletal and cardiac results.

HOPE‑3 Delivers Breakthrough Results

The December 3rd, 2025 HOPE-3 study was the first-ever Phase III trial in a largely non-ambulatory population with DMD to successfully meet its primary endpoint.

Primary endpoint (upper-limb function):

Dermiracel slowed skeletal muscle disease progression by 54% compared to placebo, measured via the PUL v2.0 scale.

Key secondary endpoints (cardiac function):

Dermiracel reduced cardiac decline by 91% versus placebo, assessed by MRI-based LVEF.
Craig McDonald, principal investigator at UC Davis Health, described the results as “extraordinary in Duchenne,” highlighting that HOPE‑3 is the first Phase III trial in a largely non-ambulatory population to successfully meet its primary endpoint. Capricor CEO Linda Marbán emphasized that the findings represent the culmination of 20 years of research and careful development of Deramiocel.

According to previous research, the therapy showed good tolerance in patients which shows safe and does not introduce any new adverse effects. The dual therapeutic effects on heart function and muscle strength make Deramiocel an innovative treatment option for patients with severe DMD.

Clinical Significance

The genetic disorder DMD causes fatal cardiomyopathy through progressive muscle degeneration that starts with muscle degeneration. The main focus of previous treatments has been to protect skeletal muscles from deterioration. The primary reason patients with this condition die is because of cardiomyopathy development.

The HOPE‑3 trial results suggest that Deramiocel could substantially improve quality of life. The 54% reduction in upper-limb deterioration enables non-ambulatory patients to perform daily activities independently, while heart function preservation leads to longer life expectancy and fewer complications from heart failure. The research findings represent a significant breakthrough in DMD treatment because existing therapies fail to stop both muscle and heart tissue deterioration.

Regulatory Path Forward

The HOPE‑3 results enable Capricor to submit its BLA application to the FDA for review after they address all concerns from the July CRL. The FDA previously granted Priority Review status to the application and new data should support regulatory approval. The resubmission process will include all trial outcomes together with safety information and results from previous studies including HOPE‑2 and its open-label extension.

The FDA approval of Deramiocel would establish a major achievement because it would become the first treatment to combat cardiomyopathy in patients with DMD. The approval of Deramiocel would confirm that donor-derived cell therapy works as a treatment for systemic and cardiac diseases, which could lead to new therapeutic options for additional medical conditions.

The path Capricor has taken has been full of difficulties. The CRL, together with internal FDA personnel changes in personnel and restricted agency-company communication, led to uncertainty which resulted in advisory committee meeting cancellations. The company continued to work on solving FDA issues while demonstrating Dermacel’s therapeutic value through the HOPE‑3 trial.

The trial design included non-ambulatory patients who typically get excluded from DMD research yet make up most of the patient base. The therapy Deramiocel shows dual effectiveness in treating both the functional and cardiac aspects of DMD, which brings new hope to patients and their families who live with advanced DMD.

Patient and Community Impact

If​‍​‌‍​‍‌​‍​‌‍​‍‌ approved, Deramiocel would be a game-changer for DMD, substantially. Besides the impressive feat of maintaining the patient’s mobility and independence, it is very likely that its enormous positive influence on the heart will significantly reduce the need for hospitals, delay heart failure, and prolong lifespan. This two-pronged blessing is very important, especially for families burdened with the challenges of DMD. We are very hopeful that the patients can sustain better functioning and living quality as time goes by.

Advocates have been vocal about how this therapy can meet the long-awaited demand that has been a problem for DMD with no solution. Given that the DMD condition progresses severely and leads to the loss of the patient’s ability to perform self-care in mid-to-late teens and then to heart disease that causes death in the twenties, a drug that can slow down both muscle and cardiac deterioration will have an overwhelmingly positive effect on the patient’s future.

Although​‍​‌‍​‍‌​‍​‌‍​‍‌ the results of HOPE‑3 are positive, the Food and Drug Administration approval is still not a definite decision. The FDA will consider a complete set of data that covers aspects such as safety, subgroup analyses, long-term follow-up, and manufacturing quality. Should there be any further inquiries, Capricor is prepared to answer them while also indicating that it is wholeheartedly dedicated to delivering a comprehensive ​‍​‌‍​‍‌​‍​‌‍​‍‌submission.

A green light from the FDA would pave the way for Deramiocel to be the first cell-engineered therapy targeting cardiomyopathy in DMD and serve as a proof of concept that cell therapy can be a viable clinical solution for complex, multi-system diseases. The availability of this drug would be a giant leap forward in patient care, making possible the dreams of patients and their families for improved function, health, and longevity.

The HOPE‑3 trial is a defining moment for Capricor and the DMD community. After the FDA rejection of the initial application and now the promising Phase III results, Deramiocel is on the right track for approval. By showing that the therapy can lead to better muscle function as well as cardiac health, it effectively tackles the problem areas most severely affected by DMD progression, which are the core issues the therapy indirectly addresses.

HOPE‑3 results bring new hope to patients, families, and clinicians. For Capricor, they affirm the company’s work through the years and highlight the transformative power of cell therapy in the treatment of Duchenne muscular dystrophy. As the company gets ready to submit its application to the FDA, the following months will determine the fate of whether this long-anticipated therapy will be available to patients who desperately need ​‍​‌‍​‍‌​‍​‌‍​‍‌it.