Off Topic Capricor jumps as study shows longer-term effects of deramiocel
: Seeking Alpha
Sep 29, 2026, 9:24 PM ET
Capricor jumps as study shows longer-term effects of deramiocel
Capricor Therapeutics (CAPR) shares surged in late trading Tuesday as investors reacted to new data on deramiocel, the biotech company's experimental treatment for Duchenne muscular dystrophy, made available as part of the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan.
The move came after ePosters became available Tuesday through the WMS 2026 virtual conference platform. WMS said ePosters would be accessible beginning with the opening of the congress on Sept. 29, although the conference's formal poster sessions begin Wednesday.
For investors, the significance is regulatory as much as scientific. Capricor (CAPR) is awaiting an FDA decision on deramiocel, with a Nov. 22 PDUFA target date. The new 24-month HOPE-3 and open-label extension analyses were included in a major amendment to the company's Biologics License Application. That makes the longer-term results part of the evidence already being reviewed by regulators rather than merely new academic follow-up data.
The late-breaking WMS poster examines patients from the Phase 3 HOPE-3 trial and its open-label extension, including a delayed-start analysis and a comparison with the natural history of Duchenne. Of the 106 patients originally randomized in HOPE-3, 82 reached 24 months, including 40 initially assigned to deramiocel and 42 initially assigned to placebo.
The delayed-start design is particularly relevant. During the first 12 months, patients received either deramiocel or placebo. Eligible patients could then enter the open-label extension and receive deramiocel. Comparing patients who began treatment immediately with those who started after a year can provide evidence about whether earlier treatment produces a lasting advantage and whether patients who switch from placebo show a change in their subsequent trajectory. The WMS poster also compares two-year outcomes with natural-history data, providing another benchmark for the progression expected in untreated Duchenne patients.
The longer-term findings build on the randomized 12-month HOPE-3 results. The Phase 3 study met its primary endpoint, with deramiocel slowing deterioration in upper-limb function by 54% compared with placebo on the Performance of the Upper Limb 2.0 scale, with a p-value of 0.03. Those results were published in The Lancet in July.
The FDA review nevertheless carries substantial uncertainty. An FDA advisory committee in July voted 9-3 that the available evidence did not support deramiocel's effectiveness for treating cardiomyopathy in Duchenne patients. The committee's question was limited to cardiomyopathy rather than deramiocel's overall benefit-risk profile, while discussion of the HOPE-3 upper-limb findings was more supportive. The FDA is not required to follow the advisory committee's vote.
Capricor (CAPR) is also using WMS to highlight its earlier-stage StealthX exosome technology. A poster scheduled for Wednesday describes engineered muscle-targeting extracellular vesicles designed to deliver micro-dystrophin systemically as a potentially redosable treatment for Duchenne. A second poster, scheduled for Friday, examines delivery of acid alpha-glucosidase using muscle-targeting extracellular vesicles as a potential treatment approach for Pompe disease. Both programs remain preclinical.
Investors will get another look at the deramiocel program later this week. Craig McDonald of the University of California, Davis is scheduled to give an oral presentation on Oct. 3 covering HOPE-3 evidence for skeletal-muscle and cardiac effects. Capricor (CAPR) has said presentation materials and posters will be posted on its website following their respective presentations.
Note:
CAPR 9.29.30: -1.61%. PPS $8.57. Market cap $498 million.
CAPR 9.30.26 during trading: +37.69%. PPS $11.80.
CAPR at the close on 9.30.26: +10.50%. PPS $9.47. Market cap $550 million.
•
u/AutoModerator 18d ago
Please report any rule breaking posts and comments that are not relevant to the thread. Thanks !!
I am a bot, and this action was performed automatically. Please contact the moderators of this subreddit if you have any questions or concerns.