Coalition Duchenne Congratulates Capricor Therapeutics on Positive Six-Month Results from its Randomized CAP-1002 Phase I/II HOPE Clinical Trial in Duchenne

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Coalition Duchenne funded cardiosphere-derived cell studies in Duchenne carried out by Eduardo Marbán MD, the director of the Cedars-Sinai Heart Institute. This work was later licensed for commercial development by Capricor.

We are excited that our work with Dr. Eduardo Marbán is evolving and could become a treatment for Duchenne through Capricor’s stellar efforts.

Coalition Duchenne, a charity based in Newport Beach, California, committed to raising awareness for Duchenne muscular dystrophy, and funding for Duchenne research, congratulates Capricor Therapeutics on positive six-month results from its randomized CAP-1002 (cardiosphere-derived cells) Phase I/II HOPE clinical trial in Duchenne announced today.

Coalition Duchenne funded studies carried out by Eduardo Marbán MD, the director of the Cedars-Sinai Heart Institute, on cardiosphere-derived cells in Duchenne. This work was later licensed for commercial development by Capricor.

“We are excited that our work with Dr. Marbán is evolving and could become a treatment for Duchenne through Capricor’s stellar efforts. It is the cardiomyopathy associated with Duchenne that causes us to lose so many boys and young men with Duchenne. Their hearts fail them when they still have so many dreams to be fulfilled. We must strengthen those hearts,” said Catherine Jayasuriya, the founder and executive director of Coalition Duchenne.

The six-month results showed statistically-significant improvements in systolic thickening of the inferior wall of the heart and in the function of the middle and distal upper limb in patients treated with CAP-1002 as compared to control patients. In addition, differences observed in several other cardiac and skeletal muscle measures, including cardiac scar, were consistent with a treatment effect.

“These initial positive clinical results build upon a large body of preclinical data which illustrate CAP-1002’s potential to broadly improve the condition of those afflicted by Duchenne, as they show that cardiosphere-derived cells exert salutary effects on cardiac and skeletal muscle,” said Linda Marbán, PhD, Capricor’s president and chief executive officer.

John L. Jefferies, MD, Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at the University of Cincinnati and Director, Advanced Heart Failure and Cardiomyopathy, and Principal Investigator of the HOPE Trial, said, “In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial.”

Coalition Duchenne is hopeful that CAP-1002 will move forward rapidly and become a treatment for Duchenne. Such a development will realize a simple, but potentially game changing, lateral thought by a mother of a young man with Duchenne. Catherine’s 24-year-old son Dusty Brandom has the cardiomyopathy exhibited by all boys and young men with Duchenne. In November 2011, Catherine read an Economist article titled “Repairing Broken Hearts” featuring the research of Dr. Eduardo Marbán and others. She immediately thought about Dusty’s heart. Catherine’s quest, both as a mother and a leader in the Duchenne community, became to convince the researchers to apply stem cell technology to Duchenne. She wrote to all of the researchers mentioned in the article and pushed her simple thesis that Duchenne related cardiomyopathy would be a good candidate for therapy. Only Dr. Eduardo Marbán responded.

About Coalition Duchenne

Coalition Duchenne was founded in 2011 to raise global awareness for Duchenne muscular dystrophy, to fund research, and to find a cure for Duchenne. Coalition Duchenne is a 501c3 non-profit corporation. Its vision is to change the outcome for boys and young men with Duchenne, to rapidly move forward to a new reality of longer, fulfilled lives, by funding the best opportunities for research and creating awareness.

Coalition Duchenne has several research initiatives that are making advances in potential cardiac and pulmonary treatments for sufferers of Duchenne muscular dystrophy.

Through its Duchenne Without Borders initiative, Coalition Duchenne is helping medically underserved boys and young men with Duchenne worldwide. For example, Coalition Duchenne provides Ambu Bags to Duchenne families in rural Borneo to help maintain pulmonary function.

Catherine Jayasuriya, the founder and executive director of Coalition Duchenne, produced and directed the award winning documentary Dusty’s Trail: Summit of Borneo (2013) which has screened internationally and is included in the syllabus at teaching institutions worldwide.

For more information about Coalition Duchenne, visit http://www.coalitionduchenne.org.

About Duchenne muscular dystrophy

Duchenne muscular dystrophy is a progressive muscle wasting disease. It is the most common fatal genetic disease that affects children. Duchenne occurs in 1 in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that codes for the protein dystrophin. This is a vital protein that helps connect the muscle fiber to the cell membranes. Without dystrophin the muscle cells become unstable, are weakened and lose their functionality. Life expectancy ranges from the mid teenage years to age 30.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac progenitor cell. 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. CAP-1002 is currently being evaluated in the randomized, double-blind, placebo-controlled Phase II ALLSTAR Clinical Trial in adults who have suffered a large heart attack and in the Phase I/II HOPE Clinical Trial in boys and young men with Duchenne.

About Capricor Therapeutics

Capricor Therapeutics, Inc. is a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions. Capricor's lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of Duchenne muscular dystrophy, myocardial infarction (heart attack), and heart failure. Capricor is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. For more information, visit http://www.capricor.com.

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