US-based biotechnology firm Capricor Therapeutics has reported positive top-line six-month results from the Phase I/II HOPE clinical trial of CAP-1002 to treat duchenne muscular dystrophy (DMD).
CAP-1002 is made up of a type of cardiac progenitor cell known as allogeneic cardiosphere-derived cells (CDCs).
The results from the randomised, single-dose, three-centre HOPE trial showed statistically significant improvement as compared to standard care controls and CAP-1002 was found to be well-tolerated.
The trial is being conducted to assess the safety and exploratory efficacy of CAP-1002 in 25 DMD patients aged 12 or above with advanced cardiac disease.
Capricor president and chief executive officer said: "These initial positive clinical results build upon a large body of preclinical data that illustrate CAP-1002's potential to broadly improve the condition of those afflicted by DMD, as they show that cardiosphere-derived cells exert salutary effects on cardiac and skeletal muscle.
"We have submitted an FDA meeting request to discuss these results, as well as next steps in our development of CAP-1002 for duchenne muscular dystrophy, which includes our plan to begin a clinical trial of intravenously administered CAP-1002 in the latter half of this year."
The trial evaluated all cardiac parameters using MRI, while the skeletal muscle performance was assessed with a validated test of upper limb function and the performance of the upper limb (PUL) test.
Capricor plans to submit the CAP-1002 results to the US Food and Drug Administration (FDA) to obtain breakthrough therapy or regenerative medicine advanced technology (RMAT) designations.
It is expected that the top-line, one-year results from the HOPE Phase I/II trial will be reported in the fourth quarter of this year.