Capricor
Capricor refutes FDA’s DMD deramiocel rejection in public statement
Capricor Therapeutics has publicised a statement addressing the US Food and Drug Administration’s (FDA) decision to reject its Duchenne muscular dystrophy (DMD) drug deramiocel. Capricor Therapeutics has refuted claims made by the FDA in a complete response letter (CRL). rejecting its cell therapy deramiocel for use in patients with DMD-associated cardiomyopathy.
This comes after the US regulator released 89 complete response letters (CRL) in a bid to “promote transparency” within the organisation, of which Capricor’s was one.
The allogenic cardiosphere-based cell therapy was rejected by the FDA because the HOPE-2 (NCT05126758) and its open-label extension portion “failed to demonstrate efficacy for its prespecified primary … and secondary endpoints”.
However, Capricor rebuts this claim, noting that the primary endpoint data was analysed with a non-parametric test – meaning it needed transforming to reach a statistically significant p value of 0.014.
In the CRL, the FDA also stated that the study was designed to evaluate the efficacy of deramiocel on neuromuscular function rather than cardiomyopathy, resulting in HOPE-2 being “not an adequate and well-controlled study” for the intended indication.
Capricor also refutes this statement, claiming that despite the pivot away from a primary cardiac endpoint following FDA advice in early development meetings, the HOPE-2 trial still included a “detailed cardiac evaluation in DMD patients”.
The FDA’s claim of HOPE-2’s trial cardiac inadequacy will be unwelcome news for Capricor, which shifted deramiocel’s biologics licence application (BLA) focus to DMD-associated cardiomyopathy rather than its original focus of DMD. According to the biotech, this was due to encouragement from FDA reviewers during an informal cardiology meeting in August 2024.
In the CRL response letter, Capricor noted that the FDA’s call to reject deramiocel was “difficult to understand” and “unprecedented,” as the company was encouraged to file a BLA by the agency before its rejection.
