Capricor Therapeutics (CAPR) has announced that the U.S. Food and Drug Administration (FDA) has scheduled an advisory committee meeting on July 29, 2026, to review the company’s Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD). This follows the release of five-year data from Capricor’s HOPE-2 Open-Label Extension (OLE) study, which demonstrated durable benefits in skeletal and cardiac function for DMD patients.
Deramiocel, a novel therapy targeting DMD, has shown significant clinical improvements in long-term outcomes. Key findings from the five-year HOPE-2 OLE trial include:
- Attenuated year-over-year decline in Performance of the Upper Limb (PUL v2.0) to approximately 1 point.
- Stable left ventricular ejection fraction (LVEF), with cardiac MRI data showing no decline over five years, compared to the modelled 3.2% annual decrease observed in external cardiac comparators.
- A favorable safety profile with no new safety signals reported.
The HOPE-2 OLE study extended follow-up of patients who participated in the Phase 2 HOPE-2 trial and completed 12 months of therapy. The trial evaluated 20 intravenous doses of Deramiocel administered over five years, confirming sustained efficacy and safety. Additional positive results from the Phase 3 HOPE-3 trial, including statistical significance on primary and key secondary endpoints (e.g., PUL v2.0, LVEF), further support the therapy’s potential.
Capricor’s BLA for Deramiocel remains on track for its projected PDUFA target action date of August 22, 2026. The company highlighted that the advisory committee meeting will provide critical input as it advances toward regulatory approval. “We are encouraged by the opportunity to bring Deramiocel before the Advisory Committee and engage directly with the FDA,” stated CEO Linda Marbán.
The HOPE-2 OLE findings were presented at the 2026 Parent Project Muscular Dystrophy (PPMD) Annual Conference in Orlando, Florida, underscoring the therapy’s growing clinical relevance. Capricor’s stock, which closed at $26.44 on June 26, 2026, has fluctuated between $4.30 and $40.37 over the past year. Pre-market trading on Friday saw shares rise 2.50% to $27.10.
HOPE-2 OLE Study and Five-Year Key Findings
The HOPE-2 OLE Study is a Phase 2, multi-centre, open-label extension trial that provided deramiocel (CAP-1002) to DMD subjects who were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial explored the safety and efficacy of 20 intravenous administrations of Deramiocel, each 3 months apart, over approximately 60 months. According to the company, in the five-year HOPE-2 OLE study, Deramiocel demonstrated
- Durable skeletal and cardiac muscle benefits over five years.
- PUL 2.0 year-over-year decline attenuated to approximately 1 point.
- Cardiac function, as measured by left ventricular ejection fraction (LVEF) on cardiac MRI, remained stable over the full five-year period, in contrast to the modelled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator.
- A favourable safety profile with no new safety signals.
The new five-year HOPE-2 OLE data, alongside HOPE-3 Phase 3 trial data, were presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference in Orlando, Florida.
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