One of the objectives of our team is to inform you about new treatments. Here’s the latest news from a recent press released by Capricor which announces the clinical trial HOPE-2. Enjoy reading and thank you for sharing these articles within the DMD community.
CAP-1002 aim to maintain or improve cardiac and skeletal muscle function
Capricor’s lead candidate, CAP-1002, is a cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 is an allogeneic product, meaning that it is manufactured from donor heart tissue and then stored until needed for use. CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and alters the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.
Status by the FDA
CAP-1002 has been granted orphan drug designation by the FDA for the treatment of DMD.
About the HOPE study
Capricor’s previous clinical trial, the HOPE-Duchenne trial, evaluated the safety and efficacy of a single dose of CAP-1002 in boys and young men with heart disease related to Duchenne muscular dystrophy. It found CAP-1002 was safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function.
HOPE 2
Participants in the HOPE-2 trial will be randomized to receive either placebo or CAP-1002, delivered intravenously every three months for a total of 4 administrations. Participants will be followed for a yearlong period following randomization, and an open-label extension (OLE) study is planned. The trial evidence should suggest an appropriate risk/benefit profile of CAP-1002 in the medical indication. Capricor – HOPE-2
Where is the trial?
The medical center in Sacramento is the first site in the nation to begin enrolling and treating participants. Approximately 12 to 15 investigative sites are expected to participate in the trial.
About Capricor
Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy.
What is DMD?
Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,5000. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.
