Capricor Announces New Pre-Clinical Study Finds Repeat Doses of CAP-1002 Lead to Enhanced Exercise Capacity in Duchenne Muscular Dystrophy Disease Model

LOS ANGELES, April 19, 2018 -- Capricor Therapeutics (NASDAQ:CAPR) today announced that  researchers found that repeat dosing of the company’s proprietary cellular therapy yields an increase in exercise performance in a disease model of Duchenne muscular dystrophy, the mdx mouse. The company has previously presented data showing that single doses lead to significant improvement in treadmill run times.

In an abstract presentation at the 11^th Annual Neuromuscular Translational Research Conference in Cambridge, England, researchers reported that while the therapy, CAP-1002, is recognized by the immune system, its low immunogenic profile and immunomodulatory capabilities allowed it to be administered multiple times without significant safety issues. They also reported that repeat dosing was shown to sustain the effect of the therapeutic. 

“These important findings were the foundation for the design of the HOPE-2 clinical trial we will be initiating shortly,” said Linda Marbán, Ph.D., Capricor president and chief executive officer. “The research found improvements in absolute force in soleus (leg) and in the diaphragm of a mouse model of Duchenne muscular dystrophy, the mdx mouse. Additionally, the researchers observed decreased fibrosis in skeletal and cardiac muscles after three administrations of CAP-1002.”

A previous clinical trial, the HOPE-Duchenne trial, found a single intracoronary dose of CAP-1002 was generally safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function in boys and young men in advanced stages of Duchenne muscular dystrophy.

The HOPE-2 clinical trial will test the safety and efficacy of repeat doses of CAP-1002 in boys and young men with Duchenne muscular dystrophy, a devastating and fatal genetic disorder with limited treatment options and no cure. Up to 84 boys and young men with Duchenne muscular dystrophy will be enrolled in the Phase 2, randomized, double-blind, placebo-controlled trial. HOPE-2 will test repeat doses of CAP-1002 in participants with advanced stages of Duchenne muscular dystrophy.

“CAP-1002 is one of the very few clinical initiatives to focus on helping boys and young men that are in later stages of the disease process and are unable or close to being unable to ambulate any longer,” said Dr. Marbán. “We are eager to get the HOPE-2 trial underway as we believe it may potentially be a registration trial and because we have seen the potential for improvements in muscle function in both pre-clinical studies and in our earlier HOPE-Duchenne clinical trial.”

Capricor has been granted the RMAT and orphan disease designations by the U.S. Food and Drug Administration (FDA). These designations will enable the company to work closely with the FDA in finalizing the regulatory approval pathway for CAP-1002 and to receive expedited FDA reviews.

“CAP-1002 also has the potential to work synergistically with gene and other therapies for Duchenne muscular dystrophy,” she said. “While these other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects. CAP-1002 may be able to work with these therapies because its primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”

For more information about the HOPE-2 trial, please visit www.HOPE2Trial.com.

The poster presented in Cambridge is available on the Events & Presentations section of Capricor's website.

About Duchenne Muscular Dystrophy

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. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

About CAP-1002

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 stimulate 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 Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ:CAPR) 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. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. The HOPE-Duchenne trial was funded in part by the California Institute for Regenerative Medicine. For more information, please visit www.capricor.com.

Keep up with Capricor on social media: www.facebook.com/capricortherapeutics, www.instagram.com/capricortherapeutics/ and https://twitter.com/capricor.

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. 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.

For more information, please contact:

AJ Bergmann, Chief Financial Officer
+1-310-358-3200
[email protected]