Capricor Therapeutics Reports Positive Phase 3 HOPE-3 Trial Results for Deramiocel in Duchenne Muscular Dystrophy, Planning FDA Resubmission
Capricor's Phase 3 HOPE-3 trial of Deramiocel showed a 54% slowing of upper limb disease progression versus placebo in Duchenne muscular dystrophy (DMD) patients.
The trial also demonstrated a 91% preservation of cardiac function (left ventricular ejection fraction) over 12 months, addressing a key mortality factor in DMD.
The HOPE-3 primary endpoint (Performance of Upper Limb score) and key secondary cardiac endpoint met statistical significance, with p-values of 0.03 and 0.04 respectively.
Deramiocel's safety and tolerability profile in this trial remained consistent with prior studies, showing no new safety signals.
Despite an earlier FDA Complete Response Letter rejecting approval, Capricor plans to resubmit their application incorporating the HOPE-3 data, which addresses prior regulatory concerns.
The trial was conducted across 20 U.S. sites with 106 boys and young men enrolled, average age 15 years, primarily non-ambulatory, receiving four infusions of 150 million cells every 3 months.
Successful results in a largely non-ambulatory DMD population make HOPE-3 the first Phase 3 study to achieve these milestones in this group.
Capricor intends to present detailed data at upcoming scientific conferences and pursue regulatory approval aggressively, generating renewed investor and community interest.