Capricor Plunges on FDA Rejection of DMD Cell Remedy
The FDA has turned down Capricor Therapeutics’ investigational cell remedy deramiocel for the therapy of cardiomyopathy related to Duchenne muscular dystrophy, pointing to a scarcity of supporting proof for the candidate.
Capricor was down 40% earlier than the opening bell on Friday, buying and selling at $6.79 per share, a big drop from its $11.40 on the earlier market shut.
In its full response letter (CRL), the FDA flagged Capricor’s knowledge bundle for deramiocel, saying it fell in need of the “statutory requirement for substantial proof of effectiveness,” in response to the biotech’s information launch on Friday.
Capricor and deramiocel have been on the middle of a current inside FDA conflict. Nicole Verdun, the previous prime regulator of cell and gene therapies on the FDA’s Heart for Biologics Analysis and Analysis (CBER), had scheduled an advisory committee assembly to debate deramiocel, as per reporting from STAT Information final month. CBER head Vinay Prasad unilaterally canceled that assembly, in response to a person acquainted with the matter, STAT mentioned. Verdun and her deputy Rachael Anatol have been each pushed out of company.
The FDA’s choice got here down early, as deramiocel’s PDUFA date was set for Aug. 30, after being granted precedence overview in March. The regulator has requested extra knowledge for deramiocel and has given Capricor the chance to request a gathering to debate a means ahead for the cell remedy.
The CRL additionally cited sure deficiencies concerning deramiocel’s Chemistry, Manufacturing, and Controls. Capricor claimed in its press launch that it has already addressed these considerations in earlier communications, however the FDA was not in a position to overview these submissions “as a result of timing of the CRL issuance.”
In a ready assertion on Friday, Capricor CEO Linda Marbán mentioned the corporate is “shocked” by the rejection. “We’ve adopted their steering all through the method,” she mentioned, noting that other than the CRL, the overview of deramiocel “had superior with out main points, together with a pre-licensure inspection and completion of the mid-cycle overview.”
Capricor plans to resubmit its software for deramiocel, backed by upcoming extra findings from the Part III HOPE-3 trial. Information from this trial are anticipated within the third quarter.
Deramiocel consists of allogeneic cardiosphere-derived cells (CDC)—a uncommon subset of cells within the coronary heart which were proven to cut back fibrosis and modulate the immune response—which produces therapeutic exercise in macrophages, differing from their traditional inflammatory habits. In accordance with Capricor, CDC cells may help protect cardiac and skeletal muscle.
Capricor is proposing to make use of deramiocel to deal with cardiomyopathy in Duchenne muscular dystrophy (DMD). The biotech’s software was backed by knowledge from the Part II HOPE-2 research, which in 2020 confirmed vital enhancements in upper-limb power and efficiency in sufferers handled with the cell remedy.
Open-label extension knowledge revealed in June 2024 demonstrated that deramiocel may preserve these upper-limb past 3 years of therapy. Right now level, the cell remedy additionally stabilized left ventricular ejection fraction, indicating it might protect coronary heart operate in DMD sufferers.
Friday’s rejection delivers one other blow to the embattled DMD house. Final month, the FDA rejected one other experimental remedy: Edgewise Therapeutics’ skeletal myosin blocker sevasemten. As within the case of Capricor, the regulator discovered Edgewise’s knowledge bundle “inadequate.”
A number of days prior, Sarepta Therapeutics reported {that a} second affected person taking its accredited DMD remedy Elevidys had died. The deaths—the primary one was documented in March—have been linked to acute liver failure, a recognized aspect impact of adeno-associated virus-based gene therapies.
