Research Found Exosomes Increased Exercise Capabilities and Muscle Activity in a Mouse Model of Duchenne Muscular Dystrophy
LOS ANGELES, Nov. 08, 2018 (GLOBE NEWSWIRE) -- During the Action Duchenne International Conference, Capricor Therapeutics, Inc. (CAPR) will present a corporate update on its Duchenne muscular dystrophy program and a poster reporting on a pre-clinical study of its exosome-based therapy.
The Action Duchenne International Conference will bring together approximately 600 families and others involved in the Duchenne muscular dystrophy community in Birmingham, UK from November 9-11.
Capricor Chief Financial Officer A.J. Bergmann will provide the company’s corporate update during the 9-11 a.m. GMT conference session on Sunday, November 11. Action Duchenne plans to stream the presentation on its Facebook page.
The poster, entitled “Extracellular Vesicles from Cardiosphere-Derived Cells Show Immunomodulatory Properties In Vitro and Improve Muscle Activity in a Mouse Model of Duchenne Muscular Dystrophy,” will be on display throughout the conference in the Viscount (Monarch) room of the Hilton Birmingham Metropole and will be presented by Ann-Sophie Walravens, Ph.D., Capricor’s analytical development manager.
The poster will report on a pre-clinical study that found extracellular vesicles, or exosomes, secreted by cardiosphere derived cells (CDCs), the active component in Capricor’s novel cell therapy, CAP-1002, were effective in increasing exercise capabilities and skeletal muscle activity in a mouse model of Duchenne muscular dystrophy. Exosomes are nano-sized, membrane-enclosed vesicles, which are secreted by cells and contain bioactive molecules, including proteins, RNAs and microRNAs. They serve as messengers to regulate cellular functions.
The poster will also report that the exosomes secreted by the CDCs reduced muscle fibrosis, which causes a loss of muscle function in Duchenne patients, and the proliferation of activated T cells, which help govern the body’s immune response, in the Duchenne mouse model. The poster is available at the Events & Presentations section of Capricor’s website.
Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. Pre-clinical and clinical studies have already shown CAP-1002 to be strongly immunomodulatory, meaning it regulates the immune system to allow healthy muscle to form, and anti-fibrotic, so that it reduces the scarring that occurs in Duchenne muscular dystrophy.
Capricor is conducting HOPE-2, a Phase II, randomized, double-blind, placebo-controlled clinical trial that is testing the safety and efficacy of repeat doses of CAP-1002 in approximately 84 boys and young men in advanced stages of Duchenne muscular dystrophy.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (CAPR) is a clinical-stage biotechnology company developing biological therapies for Duchenne muscular dystrophy (DMD) and other rare diseases. Capricor's lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of DMD. Capricor is also exploring the potential of CAP-2003, a cell-free, extracellular vesicle-based candidate, to treat a variety of disorders. For more information, visit www.capricor.com.
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.
About the Action Duchenne International Conference
Action Duchenne is the UK's first charity dedicated solely to improving the lives of all affected by Duchenne muscular dystrophy. The Action Duchenne International Conference provides young adults living with Duchenne and their families an opportunity to find out about cutting edge technology, research and health and well-being. For more information, please visit https://www.actionduchenne.org/Event/action-duchenne-international-conference
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2017 as filed with the Securities and Exchange Commission on March 22, 2018, in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015, together with the prospectus included therein and prospectus supplements thereto and in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2018, as filed with the Securities and Exchange Commission on August 13, 2018. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
CAP-1002 is an Investigational New Drug and is not approved for any indications. CAP-2003 has not yet been approved for clinical investigation.