Capricor Therapeutics has announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study of CAP-1002 for the treatment of Duchenne. CAP-1002 is the company’s novel cell therapy.

The HOPE-2 is a double-blind, randomized, placebo-controlled trial in ambulatory or non-ambulatory patients with Duchenne. It is the first trial to use the Performance of Upper Limb measure (PUL) as a prespecified primary efficacy endpoint. 

Results indicated that patients treated with CAP-1002 continued to show positive benefits after 3 years of treatment, including reduction in decline of PUL 2.0 and stabilization of heart function when compared to an external comparator dataset of similar Duchenne patients. Additionally, CAP-1002 continues to have a consistently well-tolerated safety profile.

Capricor shared that at its recent Type-B meeting with the U.S. Food and Drug Administration (FDA), the company shared these results to support what it expects to see in the HOPE-3 pivotal study as Capricor continues to work towards potential Biologics License Application (BLA) approval and commercialization. The company plans to share details of this FDA meeting later this month, and has indicated that it expects to announce topline results from the Phase 3 HOPE-3 pivotal trial in the fourth quarter of 2024.

Capricor will be sharing results from the HOPE-2-OLE study in greater detail at PPMD’s 30th Annual Conference in Orlando, Florida, taking place June 27-29, 2024. Click here for more information on PPMD’s 30th Annual Conference and registration.

Read Capricor’s press release here.