Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA
The company requested priority review for the BLA.
Capricor Therapeutics has completed submission of a rolling biologics license application (BLA) for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, to the FDA.1
Notably, the company has requested priority review for the BLA. In addition, the submission makes Capricor eligible to receive a $10 million milestone payment from partner Nippon Shinyaku under a United States Commercialization and Distribution Agreement between the companies.
“The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD,” Linda Marbán, PhD, the chief executive officer of Capricor, said in a statement.1 “This BLA is the culmination of a body of work that has been focused on bringing this potentially transformational therapy to those patients in need. We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. We look forward to working with the FDA throughout the review process to support this potential approval.”
According to the company’s press release announcing the news, the BLA is supported by data from the HOPE-2 clinical trial (NCT03406780) and HOPE-2’s open-label extension (OLE) study (HOPE-2-OLE; NCT04428476). The results will be compared to an FDA funded natural history dataset.
In June 2023, Capricor announced 24-month data from HOPE-2-OLE showing improvements in left ventricular ejection fraction (LVEF).2 Among 9 patients who reached 24 months of follow-up in the OLE study, 6 patients showed improvement in LVEF as measured by cardiac magnetic resonance imaging in comparison to when they were assessed at the conclusion of HOPE-2. In addition to the LVEF results, the company also reported that at 24 months of follow-up patients demonstrated statistically significant benefit (P = .021) on the Performance of the Upper Limb (PUL version 2.0) scale in comparison to the rate of decline seen in the placebo group in HOPE-2 at 1 year of follow-up.
“The results from this 2-year open label study are tremendously impactful for DMD patients showing cardiac and skeletal functional benefits, which underscores the potential long-term benefits of CAP-1002 treatment in DMD,” Marbán said in a statement at the time.2 “Importantly, the natural history of DMD cardiomyopathy suggests a steady decline in cardiac function as measured by ejection fraction, however, in HOPE-2-OLE, we observed improvements in heart function in 6 of 9 patients. Furthermore, as the HOPE-2-OLE data highlights the disease modifying potential of CAP-1002, we believe it is imperative to start treatment as early as possible to prevent the irreversible loss of muscle. Taken together with the favorable safety/tolerability profile, these data position CAP-1002 as a potential anchor therapy for DMD patients.”
Capricor previously received feedback from the FDA in September 2023 in a Type-B meeting on the design of the separate HOPE-3 clinical trial (NCT05126758) evaluating CAP-1002.3 At the time, Capricor noted that this meeting put the company in alignment with the FDA and helped to establish a roadmap to a BLA for Deramiocel. According to the clinicaltrials.gov page for HOPE-3, the study’s primary end point is the mean change from baseline in the full Performance of the Upper Limb (PUL version 2.0), measured at 12 months posttreatment. The company stated that based on the meeting, the trial’s primary end points will remain the same.
Deramiocel has previously been granted orphan drug designation (ODD), rare pediatric disease designation, and regenerative medicine advanced therapy designation by the FDA.1 It has also been granted ODD and advanced therapy medicinal product designation by the European Medicines Agency
