An independent Data and Safety Monitoring Board (DSMB) made the decision after its thorough safety and futility review and the recent announcement of positive interim trial results.
“We are pleased with the DSMB’s recommendation to continue the HOPE-2 clinical trial and supports our understanding of the safety of CAP-1002 in the DMD patient population, which is an important step toward establishing a potential new treatment to help these boys,” Linda Marbán, president and CEO of Capricor Therapeutics, the treatment’s developer, said in a press release.
The company is currently meeting with the U.S. Food and Drug Administration for guidance on CAP-1002’s clinical program. The investigational therapy has been granted rare pediatric disease, RMAT, and orphan drug designations.
“We look forward to sharing updates from the HOPE-2 trial in the coming months,” Marbán said.
Preclinical and clinical investigations have demonstrated that a single administration of CAP-1002 into the heart could improve cardiac and skeletal muscle function, and reduce tissue scarring in mice models of Duchenne and in patients.
Participants were assigned to receive CAP-1002 or a placebo through intravenous (in the vein) infusion every three months.
Despite the initial promising clinical data, Capricor decided to place the study on hold in December 2018 after a patient experienced a severe allergic reaction during blind testing.
An independent review on the cause of this adverse reaction suggested that the patient likely was allergic to something in the CAP-1002 formulation, the company stated in a press release. Based on this finding, the trial resumed the following month.
Preliminary data collected from 29 DMD patients showed that treatment with CAP-1002 induced significant improvements in lung function after three months, compared with those treated with a placebo. After six months, the patients also showed significant improvements in arm muscle function and grip strength.
Mild improvements in heart function, tip-to-tip pinch strength, and other lung function parameters were also reported.
CAP-1002 is a cell therapy that is comprised of cardiosphere-derived cells from donated tissue. But these engineered cells do not work as conventional stem cells. Instead of trying to restore dystrophin levels — the protein lacking in DMD — they release tiny vesicles containing factors that prevent inflammation and balance the immune response.