Capricor Reports 24-Month HOPE-3 Data for Deramiocel

Capricor Therapeutics released 24-month open-label extension data from its HOPE-3 study for deramiocel in Duchenne muscular dystrophy, showing a 76% reduction in upper limb decline for patients switching from placebo.

The findings provide long-term efficacy metrics as the biotechnology company moves through the regulatory review process for its lead candidate.

"When the young men who had been declining on placebo started Deramiocel, their rate of upper limb loss dropped by roughly 75% in their first year of treatment," said Craig McDonald, principal investigator of the HOPE-3 trial at UC Davis Health, in a statement regarding the release.

Detailed HOPE-3 Clinical Scores and Patient Trajectories

According to an Investing.com report, patients who started deramiocel after spending one year on placebo experienced a slowdown in disease progression. The Performance of Upper Limb 2.0 total score showed a least-squares mean change of -2.05 during the placebo year, which improved to a change of -0.49 after switching to active treatment.

For patients who received deramiocel continuously across both years of the study, functional scores demonstrated sustained stability. The cohort recorded a least-squares mean change of -0.95 in the first year followed by -0.89 in the second year.

Regulatory Timelines and FDA Review

The 24-month dataset has been submitted to the U.S. Food and Drug Administration as part of an amendment to the deramiocel Biologics License Application. The regulatory agency has set a Prescription Drug User Fee Act target action date of November 22, 2026.

Duchenne muscular dystrophy is a severe genetic disorder characterized by progressive muscle degeneration and weakness, primarily affecting young males. The condition leads to a gradual loss of upper and lower limb function, making therapies that preserve arm and hand mobility important for maintaining patient independence.

Capricor's ongoing evaluation of deramiocel represents a milestone for the company as it seeks commercial approval for the cell-based therapy in an indication with unmet medical needs.