Capricor Therapeutics reported improvements in skeletal muscle function in nonambulatory patients with Duchenne muscular dystrophy (DMD) who were treated with the experimental drug CAP-1002, according to a press release by the company. 

These results were recorded from the HOPE-2 open-label extension study. According to the findings of this study, patients reported statistically significant improvements in their upper limb performance after being administered CAP-1002. 

“For patients with DMD, time is associated with loss of function,” Craig McDonald, national principal investigator for HOPE-2, said. “The progressive nature of the disease puts these patients on a slow, steady decline. Anything that we can do to delay the progression is an essential step in the right direction, allowing for patients to preserve key everyday activities requiring upper limb function.” 

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The researchers offered CAP-1002 to all 20 patients who participated in the HOPE-2 study. Among these patients, 13 enrolled in the extension study, and 12 completed the first year of follow-up. CAP-1002 was administered on a quarterly basis, and results were recorded upon analysis at the 1-year mark. 

“Once the patient group that chose to enter the open label extension study began receiving CAP-1002 quarterly injections, we saw DMD’s progression slowing, which started within the first three months and continued for the entire year,” Linda Marbán, PhD, chief executive officer of Capricor, said. “All of our findings to date suggest that CAP-1002 holds promise as a potential anchor therapy for non-ambulant DMD patients, a group for which few treatment options exist.” 

The results demonstrated that CAP-1002 when used over a period of time, can slow the muscle degenerative effects of DMD and may even be disease-modifying. The US Food and Drug Administration has granted CAP-1002 Regenerative Medicine Advanced Therapy and Orphan Drug designations. 

Because of the encouraging findings from the HOPE-2 trial, Capricor is currently conducting a phase 3 trial called HOPE-3. HOPE-3 is a randomized, double-blind study recruiting approximately 70 patients with DMD. 


Capricor Therapeutics announces statistically significant clinical benefits in skeletal muscle function in non-ambulant Duchenne muscular dystrophy patients treated with CAP-1002 in HOPE-2 open label extension study. News release. Capricor Therapeutics; June 27, 2022.