Lancet publishes Capricor's HOPE-3 data as FDA review continues

Capricor Therapeutics announced on July 29, 2026 that The Lancet has published results from its Phase 3 HOPE-3 trial of Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy. The trial was a randomized, double-blind, placebo-controlled study of 106 patients that met its primary endpoint, with Deramiocel slowing upper limb function decline by 54 percent versus placebo on the PUL 2.0 measure (p=0.03), alongside cardiac benefit described by the company as clinically meaningful.¹

The manuscript went through independent expert peer review, which the company said provides external validation of the trial's design, statistical methodology and findings.¹ The published results are based on the company's prespecified Statistical Analysis Plan version 3.0.¹

Separately, the 8-K discloses a change to the statistical model used for a key secondary endpoint. In connection with its FDA dialogue and the Lancet peer review, Capricor identified an update to the model used for left ventricular ejection fraction (LVEF) analysis; under the revised model, LVEF shows p=0.09 with a 1.8 percentage point treatment difference, compared with the previously reported p=0.04 and 2.4 percentage point difference.¹ In the prespecified cardiomyopathy subgroup, the result remains nominally significant at p=0.02 (2.8 percentage point difference), and the company said the primary efficacy endpoint and all other trial data are unchanged.¹

The filing also discloses an FDA Bioresearch Monitoring inspection. The FDA notified Capricor on July 4, 2026 of a BIMO inspection tied to the Deramiocel BLA, which ran from July 6 to July 20, 2026.¹ The FDA issued a Form 483 citing one observation on SOPs, documentation, vendor oversight and audit timeliness; Capricor said it does not believe this affects the integrity of the HOPE-3 data and is preparing a timely response.¹

The Deramiocel BLA remains under FDA review, with a PDUFA target action date of August 22, 2026.¹