The investigational Duchenne muscular dystrophy (DMD) therapy, CAP-1002, continues to be beneficial and well tolerated in the long term, according to results from the open-label extension study of the phase 2 HOPE-2 trial (HOPE-2-OLE). 

These results will be presented at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place from March 19 to 22, 2023 in Dallas, TX.

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“CAP-1002 demonstrates novel, clinically meaningful, and cumulative preservation of upper limb function by potentially modifying the underlying disease,” the authors of the study wrote. 

During HOPE-2-OLE, 13 patients from the HOPE-2 trial (6 who received CAP-1002 and 7 who received placebo) were treated with CAP-1002 for 18 months after a gap phase of 12 months, which allowed the evaluation of patients both on and off treatment.

The results showed significant differences between patients participating in HOPE-2-OLE and those given a placebo during HOPE-2 in terms of upper limb performance, reinforcing the results obtained in HOPE-2. 

Moreover, the progression of the disease slowed significantly among patients given a placebo during HOPE-2 once CAP-1002 was initiated in the extension phase. This led to a decrease in the between-group difference observed in the original HOPE-2 trial.

“CAP-1002 slows upper limb functional decline in DMD by modifying disease,” the researchers concluded.

DMD is a rare genetic disease and a type of muscular dystrophy caused by a mutation in the gene coding for the essential muscle protein, dystrophin. It is characterized by muscle wasting and degeneration. 

CAP-1002 is an investigational therapy being developed by Capricor Therapeutics based on cardiosphere-derived cells that can modify the activity of the immune system to drive cellular repair and regeneration. The experimental treatment has been granted Orphan Drug designation for DMD.

Reference

Marban L, McDonald CM, Hendrix S, et al. Late breaking: CAP-1002, an allogeneic cell therapy demonstrates disease modification in later-stage DMD patients: 18-month results from the HOPE-2-open label extens. Poster presented at: 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX.

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