Capricor to Present 24-Month HOPE-3 Data for Deramiocel in Duchenne at WMS 2026

Key Takeaways

• Capricor Therapeutics will present 24-month data from its Phase 3 HOPE-3 study and open-label extension (OLE) of deramiocel at the 2026 World Muscle Society (WMS) Congress, September 29 to October 3 in Hiroshima, Japan.
• The presentations will cover skeletal muscle and cardiac outcomes in patients with Duchenne muscular dystrophy (DMD) and build on data the company submitted to the FDA in a Biologics License Application (BLA) amendment in August.
• Capricor will give a late-breaking poster and an oral presentation. The FDA has extended the PDUFA target action date for deramiocel to November 22, 2026.

Capricor Therapeutics announced it will present new data from the Phase 3 HOPE-3 study and its open-label extension of deramiocel at this year's WMS Congress. The updated findings cover skeletal muscle and cardiac outcomes through 24 months in patients with DMD, a severe X-linked genetic disorder marked by progressive degeneration of skeletal, respiratory, and cardiac muscles.

The new data build on the 12-month HOPE-3 results and the 24-month OLE data that Capricor submitted to the FDA as part of a BLA amendment in August. The FDA accepted the amendment for review and extended the PDUFA target action date to November 22, 2026.

HOPE-3 is a randomized, double-blind, placebo-controlled Phase 3 trial that enrolled 106 patients with DMD. Participants received intravenous deramiocel or placebo every three months for 12 months, after which eligible patients could continue in the OLE. Of the 106 patients, 82 reached the 24-month time point: 40 originally assigned to deramiocel and 42 to placebo. The trial met its primary endpoint, with deramiocel slowing the decline in upper-limb function versus placebo, as measured by the Performance of the Upper Limb (PUL) 2.0 scale.

The late-breaking poster is titled "Deramiocel slows upper limb decline in the HOPE-3 OLE: cross-phase delayed-start analysis and 2-year comparison with natural history." The oral presentation, "HOPE-3, a phase 3 study of deramiocel, an allogeneic cell therapy, in advanced Duchenne muscular dystrophy: evidence to support both musculoskeletal and cardiac efficacy," is scheduled for October 3.