We continue today in our examination of companies advancing the development of therapeutics based on stem cells. We think Capricor Therapeutics (NASDAQ: CAPR) merits attention based on its clinical pipeline and collaborative, capital-efficient approach to drug development to treat heart disease and other serious medical conditions.
Capricor’s lead candidate, CAP-1002, is an “off-the-shelf” cardiac cell therapy that is currently in clinical development for the treatment of heart disease associated with Duchenne muscular dystrophy (DMD) as well as adult cardiology conditions. The science of Capricor’s technology to address heart disease was first demonstrated clinically in the randomized CADUCEUS clinical trial, (see below), which showed, following a single administration, significant reductions in the size of the scar that had resulted from a large heart attack, as well as significant increases in muscle mass at the affected area.
Source: Capricor Therapeutics Investor Overview, Feb 2017
The CADUCEUS trial
The 2012 Phase 1 trial of CArdiosphere-Derived aUtologous StemCElls to reverse ventricUlar dySfunction (CADUCEUS) was a proof-of-concept study that evaluated the safety and efficacy of Capricor’s investigational, autologous cardiosphere-derived cell (CDC) product, CAP-1001. CAP-1002, the current candidate, is similar but allogeneic (from a compatible donor) instead of autologous (from the patient’s own blood).
The study challenged long-standing convention that cardiac scarring is permanent and healthy heart muscle cannot be restored. CADUCEUS provided early evidence for therapeutic regeneration. The scars caused by the myocardial infarction shrank over the course of 12 months in patients treated with CAP-1001, and scientists observed a significant correlation between scar shrinkage and viability increases.
The trial was conducted at Cedars-Sinai and Johns Hopkins and was sponsored by Cedars-Sinai under the leadership of Dr. Eduardo Marbán, Director of the Cedars-Sinai Heart Institute and co-founder and Scientific Advisory Board Chairman of Capricor.
Pipeline and Janssen Biotech Collaboration
Capricor is pursuing several clinical paths in parallel, with top-line results from the Phase I/II HOPE (Halt cardiOmyopathy ProgrEssion) trial in Duchenne Muscular Dystrophy to be reported early this quarter. Heart disease is the leading cause of death in DMD patients.
The company has been collaborating on the manufacturing of CAP-1002 with Janssen Biotech, a unit of Johnson & Johnson (NYSE: JNJ), which holds an option to license CAP-1002 for certain cardiology indications. This option could potentially be worth $325 million plus royalites to Capricor if exercised.
Source: Capricor Therapeutics Investor Overview, Feb 2017
Exosomes
Capricor is also pursuing therapies based on exosomes from CDCs. Exosomes are nanosized particles with biologically active contents that are secreted by cells and serve to direct or redirect the activities of other cells and are emerging as an exciting class of potential therapeutic agents. Studies have shown that significant exosome activity in several disease models. Capricor plans to explore development of the exosome technology as a next generation regenerative medicine platform in a variety of cardiovascular and non-cardiovascular areas, with an investigational new drug submission planned for later this year.
Cash Position
Capricor ended 2016 with $16.2 million in cash, equivalents and marketable securities, which the company believes will fund operations into the fourth quarter of this year. Capricor management has had a successful record in securing non-dilutive capital, having received over $30 million in competitive grant and loan awards. Investors should look for continued news out of the clinic this year in addition to a decision by Janssen Biotech on its license option. It may not take much in the way of good news for the current $61 million market cap to seem like a bargain.
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