TLDR
- Capricor Therapeutics (CAPR) jumped after new Phase 3 data on deramiocel, its Duchenne muscular dystrophy treatment, was released at the World Muscle Society congress in Hiroshima.
- The data showed 82 of 106 HOPE-3 trial patients reached 24 months, with results now folded into the company’s FDA application.
- Deramiocel already met its primary endpoint at 12 months, slowing upper-limb deterioration by 54% versus placebo.
- An FDA advisory committee voted 9-3 in July against the drug’s cardiomyopathy benefit, though the upper-limb data drew more support.
- Capricor faces an FDA decision with a PDUFA date of November 22.
Capricor Therapeutics stock moved higher in late trading Tuesday after the company released longer-term data on deramiocel, its experimental Duchenne muscular dystrophy treatment. The news came via ePosters posted for the World Muscle Society’s 31st Annual Congress in Hiroshima, Japan.
Capricor Therapeutics, Inc., CAPR
The timing matters. Capricor is waiting on an FDA decision for deramiocel, with a target date of November 22. The new data isn’t just an academic update. It’s part of a major amendment the company filed to its Biologics License Application, meaning regulators are already looking at it.
The poster covers patients from the Phase 3 HOPE-3 trial and its open-label extension. Of the 106 patients originally randomized, 82 made it to the 24-month mark. That group split into 40 who started on deramiocel from the beginning and 42 who began on placebo.
What the Delayed-Start Design Shows
The trial used a delayed-start setup. Patients got either deramiocel or placebo for the first 12 months. After that, eligible patients could cross over into the open-label extension and start receiving deramiocel regardless of their original group.
This setup lets researchers compare early starters against late starters. It helps answer whether starting treatment sooner leads to a lasting edge, and whether patients who switched from placebo see any change in their trajectory afterward. The poster also stacks two-year results against natural-history data to give another point of comparison for how the disease normally progresses.
These longer-term numbers build on the original 12-month HOPE-3 results. That trial hit its main goal, slowing upper-limb function decline by 54% compared to placebo on the Performance of the Upper Limb 2.0 scale. The p-value came in at 0.03. Those findings were published in The Lancet back in July.
The FDA’s Mixed Signals So Far
The road to approval hasn’t been smooth. An FDA advisory committee voted 9-3 in July that the evidence didn’t support deramiocel’s effectiveness for treating cardiomyopathy in Duchenne patients specifically.
That vote was narrow in scope. It only addressed cardiomyopathy, not the drug’s overall risk-benefit picture, and committee members were more favorable when discussing the upper-limb data from HOPE-3. The FDA isn’t bound by the advisory vote either way.
Capricor is also using the WMS congress to showcase earlier-stage work. One poster set for Wednesday details the company’s StealthX exosome platform, engineered to deliver micro-dystrophin as a redosable Duchenne treatment. Another poster on Friday covers a similar delivery approach aimed at Pompe disease.
Both of these programs are still preclinical, meaning they’re years away from any regulatory decision. They give investors a look at Capricor’s pipeline beyond deramiocel.
There’s more scheduled this week too. Craig McDonald of UC Davis is set to give an oral presentation on October 3 covering HOPE-3 evidence for both skeletal-muscle and cardiac effects.
Capricor has said it will post presentation materials and posters to its website after each session wraps up. The company’s market cap sits around $498 million, and it currently generates no product revenue, relying instead on financing and partnerships like its Japan collaboration with Nippon Shinyaku.
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