Capricor Therapeutics has reported new results from the HOPE-3 study evaluating Deramiocel in people with Duchenne muscular dystrophy (DMD). The 24-month open-label extension data focused on Upper Limb Function and showed that patients who started Deramiocel after one year on placebo experienced a significantly slower rate of upper limb decline during their first year of treatment.
The new findings were presented at the 2026 World Muscle Society Congress. The HOPE-3 study is a Phase 3 clinical trial evaluating Deramiocel in patients with Duchenne muscular dystrophy. A total of 106 patients were initially enrolled in the randomized, placebo-controlled part of the study. Patients who completed this period could continue receiving Deramiocel in an open-label extension. Learn More: What is Deramiocel?
Upper Limb Decline Slowed After Starting Deramiocel
One of the most important findings came from patients who received placebo during the first year and then switched to Deramiocel.
During their first year on placebo, these patients experienced an average 2.05-point decline in their PUL 2.0 total score. During the following year, after starting Deramiocel, their average decline slowed to 0.49 points.
According to Capricor, this represents a 76% reduction in the rate of upper limb decline compared with the patients’ own previous year on placebo.
Upper limb function is particularly important for people with Duchenne because it is closely connected to independence and everyday activities.
Patients Receiving Deramiocel From the Beginning
The study also followed patients who received Deramiocel from the beginning of the trial.
In this group, the average PUL 2.0 score declined by 0.95 points during the first year and 0.89 points during the second year. The similar results across the two years suggest that the slower decline continued during the second year of treatment.
Comparison With Natural History
At 24 months, both groups showed less decline in upper limb function than predicted by natural history models.
Patients who received placebo for the first year and Deramiocel for the second year had an observed change of −3.76 points, compared with a predicted natural-history decline of −5.35 points.
Patients who received Deramiocel for both years had an observed change of −3.42 points, compared with a predicted decline of −5.88 points.
However, Capricor noted that these natural-history comparisons were descriptive only and were not based on patient matching or statistical testing.
What These Results Mean
The 24-month results provide additional evidence that Deramiocel may slow the loss of upper limb function in people with Duchenne muscular dystrophy. The most notable finding was the 76% reduction in the rate of decline among patients who switched from placebo to Deramiocel.
At the same time, the open-label extension was not designed or powered for formal statistical comparisons at 24 months. Therefore, the results should be interpreted within the limitations of the study design.
Capricor has submitted these data as part of a major amendment to its Deramiocel Biologics License Application (BLA). The FDA’s current PDUFA target action date for the application is November 22, 2026.
Discover More:Â Duchenne Clinical Trial Locations Map
