CAP-1002 Shows Sustained Cardiac Benefit in Duchenne Muscular Dystrophy
核心洞察
Data from the HOPE-2 open-label extension study demonstrate that CAP-1002 continues to improve left ventricular ejection fraction (LVEF) in DMD patients over two years.
Patients treated with CAP-1002 exhibited statistically significant benefits in the Performance of the Upper Limb (PUL v2.0) scale compared to the decline observed in the placebo group.
CAP-1002 is currently being evaluated in the Phase 3 HOPE-3 trial, with interim analysis expected in the fourth quarter of 2023, potentially leading to a BLA submission.
New data from the open-label extension (OLE) of the Phase 2 HOPE-2 trial (NCT03406780) reveals that CAP-1002, a cell therapy developed by Capricor (搜索), continues to improve cardiac function in patients with Duchenne muscular dystrophy (搜索) (DMD) over a two-year period. The study highlights the potential of CAP-1002 to provide sustained benefits in both cardiac and skeletal muscle function.
Sustained Improvement in Cardiac Function
Following the double-blind, placebo-controlled phase, participants in the OLE received CAP-1002 at 150 million cells per infusion every three months for 24 months. The results indicated a continued improvement in left ventricular ejection fraction (LVEF), a key indicator of cardiac function, in treated patients. Notably, six out of nine patients showed improved heart function by the end of the HOPE-2 study, as measured by cardiac MRI.
Linda Marbán, PhD, CEO of Capricor (搜索), stated, "The results from this two-year open label study are tremendously impactful for DMD patients showing cardiac and skeletal functional benefits, which underscores the potential long-term benefits of CAP-1002 treatment in DMD." She also noted that the natural progression of DMD cardiomyopathy typically involves a steady decline in cardiac function, making the observed improvements particularly significant.
Benefits in Upper Limb Function
In addition to cardiac benefits, patients treated with CAP-1002 demonstrated statistically significant improvements in the Performance of the Upper Limb (PUL v2.0) scale after two years, compared to the original rate of decline observed in the placebo group during the first year of the study. There was an increasing correlation between PUL v2.0 and ejection fraction results (r = 0.75; P = .02), suggesting a link between cardiac and skeletal muscle function improvements.
Safety and Tolerability
The therapy was well-tolerated, with a safety profile consistent with previous observations. This favorable safety profile, combined with the observed functional benefits, positions CAP-1002 as a potential anchor therapy for DMD patients.
Ongoing Phase 3 Trial and Future Plans
CAP-1002 is also being evaluated in the Phase 3 HOPE-3 trial (NCT05126758), a multicenter, randomized, double-blind, placebo-controlled study involving both ambulatory and non-ambulatory boys and young men with DMD. The trial, expected to be completed in late June 2024, includes approximately 68 participants who will receive either CAP-1002 or placebo for 12 months. The primary endpoint is the mean change in full upper limb function as measured by PUL v2.0 scores, with changes in cardiac muscle function and structure as secondary outcomes.
Capricor (搜索) plans to discuss these results with the FDA, along with key features of the ongoing Phase 3 HOPE-3 trial, to outline a potential path towards submitting a Biologics License Application (BLA). Enrollment in HOPE-3 is expected to be completed in the second half of 2023, with an interim analysis reported in the fourth quarter of 2023.
Marbán emphasized the importance of early intervention, stating, "As the HOPE-2-OLE data highlights the disease modifying potential of CAP-1002, we believe it is imperative to start treatment as early as possible to prevent the irreversible loss of muscle."