Capricor Highlights Long-Term Efficacy of DMD Therapy Before FDA Application

Capricor Therapeutics has announced additional positive safety and efficacy data from a three-year evaluation of their Phase II HOPE-2 open-label extension (OLE) trial for deramiocel, a cell therapy aimed at treating Duchenne muscular dystrophy (DMD). The recent findings add to the growing body of evidence supporting the long-term benefits of deramiocel.

Last week, Capricor initiated the rolling submissions for a biologics license application (BLA) with the US Food and Drug Administration (FDA) for deramiocel as a treatment option for DMD cardiomyopathy. The company aims to complete this submission by the end of the year. This milestone is crucial as it reflects the potential for deramiocel to provide sustained cardiac and skeletal muscle benefits over a prolonged period, which is essential for patients living with this condition.

Capricor’s CEO, Linda Marbán, emphasized the significance of these findings for DMD patients, highlighting the consistent improvements in both cardiac and skeletal muscle function observed after three years of continuous deramiocel treatment. These results underscore the potential long-term efficacy of the therapy. She also mentioned that the company is working closely with the FDA to expedite the approval process for deramiocel. Once cardiac function in DMD patients is lost, it is typically irrecoverable, making timely treatment essential. Deramiocel is expected to be a lifelong therapy administered quarterly.

The HOPE-2 OLE study, identified as NCT03406780, involved 13 patients with DMD cardiomyopathy. Initially, in 2021, Capricor reported that the HOPE-2 trial achieved its primary endpoint by slowing muscle weakness, measured by changes in the mid-level dimension of the Performance of the Upper Limb (PUL v2.0). This success prompted the initiation of a Phase III pivotal study (NCT05126758), which is scheduled to complete in December 2026.

The latest Phase II data was presented at the 29th Annual Congress of the World Muscle Society held from October 8-12 in Prague. This new information builds on the long-term results disclosed in July, including subgroup analyses. Specifically, in a subgroup of eight patients with a left ventricular ejection fraction (LVEF) greater than 45%, deramiocel led to a median LVEF improvement of 3.1% at two years and 3% at three years, as evidenced by cardiac magnetic resonance imaging (cMRI). Comparatively, an external comparator group of untreated patients showed a median decline of 5% over two years. Additionally, the therapy resulted in an increase of 11.1ml/m² in end-systolic volume (ESV) at two years, again surpassing the external group.

In terms of muscle deterioration, deramiocel demonstrated its efficacy in slowing the process. Patients receiving the treatment showed a mean decline of 4 points on a 22-item PUL v2.0 scale over three years, while the external group experienced a 7.7 point decline.

Deramiocel is an allogeneic cell therapy derived from cardiosphere-derived stromal cells. Last month, Capricor entered into a $35 million agreement to license the European marketing rights to the therapy to Japanese company Nippon Shinyaku. This agreement builds on previous licensing deals for the US and Japanese markets. Capricor projects that milestone payments from these combined distribution agreements with Nippon could total around $1.5 billion.

In summary, the ongoing research and partnerships surrounding deramiocel highlight its potential to significantly impact the treatment of Duchenne muscular dystrophy, offering hope for improved long-term outcomes for patients suffering from this debilitating condition.