Capricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data on Upper Limb Function in Duchenne Muscular Dystrophy at 2026 World Muscle Society Congress
Key Highlights
- ➤Placebo starters cut upper-limb decline 76% after beginning Deramiocel treatment
- ➤24-month decline reached −3.42 points after continuous Deramiocel treatment
- ➤Deramiocel BLA major amendment includes HOPE-3 extension data
- ➤FDA PDUFA target action date is November 22, 2026
Expert Statements
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“Upper limb function is critical to quality of life for young men with DMD because it is directly tied to their independence”
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“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.”
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“Just as important, patients who were on Deramiocel both years declined at a similar rate in year 1 and year 2, which suggests that the slower decline was triggered by starting treatment, not the passage of time.”
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“Being able to follow the same young man before and after he starts treatment is a powerful way to see a treatment effect, and what we saw was consistent with the randomized, placebo-controlled period.”
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“As a clinician seeing these patients, I have had very little to offer them that could meaningfully slow the progression of upper limb functional loss once they were non-ambulant.”
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“These data suggest there could be a way to treat this aspect of the disease.”
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“Their arms are how they feed themselves, control their wheelchairs and use their phones, and how they stay independent.”
Craig McDonald, MD and Principal Investigator of the HOPE-3 trial at UC Davis Health
“Time is muscle, and slowing that loss means more time doing the things that matter most to them.”
Linda Marbán, Ph.D. and CEO of Capricor
“These results add to a growing body of evidence for Deramiocel”
Linda Marbán, Ph.D. and CEO of Capricor
“What we saw in the HOPE-3 open-label extension aligns with our previous clinical studies, which together show the same pattern across multiple years of treatment.”
Linda Marbán, Ph.D. and CEO of Capricor
“The FDA has received these data as part of the recent major amendment to the Deramiocel Biologics License Application, along with extensive sensitivity and robustness analyses we produced to support the results of the HOPE-3 randomized clinical trial.”
Linda Marbán, Ph.D. and CEO of Capricor
“We will continue to work with the FDA through the remainder of the review, ahead of the November 22, 2026, PDUFA target action date.”
-24-Month Crossover Analysis: Patients Starting Deramiocel After a Year on Placebo Slowed Upper Limb Decline by 76% Compared with Their Own Prior Year-
-At 24 Months, Both Groups Showed Slower Decline in Upper Limb Function Than Natural History Predicts-
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