The FDA requested the full data from the phase 3 HOPE-3 study as part of its review of the biologics license application for deramiocel, which Capricor plans to submit in February 2026.
In a new company update, Capricor Therapeutics announced that the FDA has formally requested the complete clinical study report and supporting data from the phase 3 HOPE-3 trial (NCT05126758) of deramiocel, as the agency continues to review of the company’s biologics license application (BLA). The investigational cell therapy, which remains under review, is aiming to become the first cell-based product for patients with Duchenne
Capricor stated in its company update that preparation of the HOPE-3 clinical study report is already underway and that the requested materials are expected to be submitted to the FDA in February 2026. The company noted that it anticipates that this submission will support continued review of the BLA and may lead to the assignment of a new PDUFA action date.
According to the company, the agency’s request follows its review of
“We are actively engaging with the FDA in order to facilitate an efficient review of the HOPE-3 data that directly address the issues raised in the CRL we received in July 2025. We were pleased that the FDA requested the HOPE-3 clinical study report, as this is an expected and appropriate next step following their initial review of the topline data,” Linda Marbán, PhD, chief executive officer at Capricor, said in a statement.1 “Our near-term priority is to address the FDA’s request and continue working collaboratively so that patients with late-stage DMD, who currently have very limited treatment options, may gain access to Deramiocel as soon as possible.”
HOPE-3 is a randomized, double-blind, placebo-controlled, phase 3 trial testing deramiocel in boys and young men living with DMD across 20 sites in the United States. In the trial, participants received intravenous deramiocel at 150 million cells per infusion or placebo every 3 months for 12 months. Overall, the mean age of participants was approximately 15 years, and all were on a stable corticosteroid regimen during the trial. Baseline demographics were well balanced between treatment arms; approximately 90% were receiving cardiac medications at baseline, and over 75% of patients had a clinical diagnosis of cardiomyopathy.
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In our latest Peer Exchange program, John F. Brandsema, MD; Oscar Mayer, MD; David Weber, MD; Carol Wittlieb-Weber, MD, discussed the pathophysiology and disease progression of Duchenne muscular dystrophy before exploring the evolving treatment landscape, with a specific focus on newer therapeutic agents.
Topline data from HOPE-3 demonstrated that treatment with deramiocel significantly delayed disease progression compared with placebo on Performance of Upper Limb total score (PUL v2.0), showing a 54% slowing of progression and meeting the primary end point in the intent-to-treat population (ITT) at 12 months (n = 105; P = .029). For the key secondary end point, left ventricular ejection fraction, deramiocel treatment was associated with a 91% slowing of decline based on centrally reviewed cardiac MRI evaluations in the ITT population across 12 months (n = 83; P = .041). Notably, the company observed that the safety and tolerability of deramiocel were consistent with prior studies in participants.3
“The HOPE-3 results demonstrated statistically significant and clinically meaningful improvements in both skeletal muscle and cardiac function—key drivers of disease progression and long-term outcomes in Duchenne,” Marbán, said in a statement.1 “These findings build on more than a decade of consistent clinical evidence and reinforce our confidence in Deramiocel’s potential.”
In the company’s issued CRL, the agency stated that it was unable to review the BLA because it did not meet the requirements for substantial evidence of efficacy.2 In addition, the FDA noted outstanding items in the Chemistry, Manufacturing, and Controls section of the application, most of which the company stated it believed had been addressed in prior communications with the FDA. For context, the original BLA submission for deramiocel was based on data from the
HOPE-2 enrolled 26 patients with DMD cardiomyopathy, 8 who were randomized to deramiocel, 12 to placebo, and 6 who were excluded due to screening failure. All told, deramiocel-treated patients showed a statistically significant 36.2% improvement over placebo in 12-month mid-level elbow PUL1.2 scores (mean difference, 2.6 points; 95% CI, 12.7-59.7; P = .014). Treatment with the cell therapy led to significant reductions in creatine kinase (CK-MB) as a proportion of total CK over 12 months, indicating less cardiac muscle damage, with a 29.1% difference vs placebo (95% CI, 4.0-54.2; P = .025).4
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