San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) said The Lancet has published results from its Phase 3 HOPE-3 trial, in which Deramiocel slowed upper limb function decline by 54 percent versus placebo in Duchenne muscular dystrophy (PUL 2.0, p=0.03).
The randomized, double-blind, placebo-controlled trial enrolled 106 participants and met its primary endpoint. Capricor also reports a clinically meaningful cardiac benefit. The results rest on the company's prespecified Statistical Analysis Plan version 3.0 (SAP 3.0).
FDA review of the Deramiocel BLA continues toward an August 22, 2026 action date
The Biologics License Application for Deramiocel remains under active FDA review, with a PDUFA target action date of August 22, 2026. According to CEO Linda Marbán, the HOPE-3 data form the foundation of the BLA and will be discussed at an Advisory Committee meeting. She said Capricor has continued to work with the FDA throughout the review.
Capricor aims to bring Deramiocel to market as the first approved cell therapy for Duchenne. Approval could also qualify the company for a Priority Review Voucher, given the FDA's Rare Pediatric Disease Designation.
Peer review at The Lancet adds external validation of trial design and statistics
The manuscript underwent independent expert peer review, which Capricor says provides external validation of the trial's design, statistical methodology and findings. The paper is titled "Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3)."
Craig McDonald, M.D., of UC Davis Health, National PI of the HOPE-2 and HOPE-3 trials and lead author, said HOPE-3 is the first Phase 3 trial to show a significant functional benefit in a largely non-ambulatory DMD population. He added that concurrent benefits in several cardiac measures support a consistent treatment effect across skeletal and cardiac muscle.
Regulatory designations and open litigation frame the commercial path
Deramiocel (CAP-1002) holds Orphan Drug Designation from both the U.S. FDA and the European Medicines Agency. It also holds Regenerative Medicine Advanced Therapy designation in the U.S. and Advanced Therapy Medicinal Product designation in Europe.
Capricor's forward-looking statements reference litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc. The company cites its ability to commercialize Deramiocel independent of its existing distribution agreement. DMD affects approximately 15,000 people in the United States.