LOS ANGELES, Jan. 24, 2019 (GLOBE NEWSWIRE) -- Neurology®, an international peer-reviewed journal, on
Wednesday published the results of the HOPE-Duchenne clinical trial that evaluated the safety and efficacy of a single
intracoronary dose of Capricor Therapeutics’ (NASDAQ: CAPR) novel cell therapy candidate, CAP-1002, in boys and young
men in advanced stages of Duchenne muscular dystrophy.
The Phase I/II, randomized, controlled, open-label trial found that CAP-1002 demonstrated improvement in cardiac
muscle function and reduction in cardiac scarring that were statistically-significant and sustained improvement of skeletal muscle
functions in patients with Duchenne muscular dystrophy, a fatal genetic disease with limited treatment options. The HOPE-Duchenne
trial also found no serious safety issues, according to the study published in the January 23, 2019, online issue of Neurology, the medical
journal of the American Academy of Neurology.
“CAP-1002 is the only therapy so far that has shown reduction in myocardium scar and improvement of localized
cardiac function in late stage Duchenne associated cardiomyopathy. Interestingly, it also showed improvement in upper limb
strength,” said Michael Taylor, M.D., Ph.D., the study’s corresponding author and Director of Cardiac MR research at Children's
Healthcare of Atlanta. “These findings suggest that CAP-1002 could be an option for older patients who have scarring in the heart
as a result of the underlying disease. As many of these patients are in later stages of the disease, the improvement in upper limb
strength is important in order to maintain independence.”
The HOPE-Duchenne clinical trial enrolled 25 patients and was conducted at three centers in the U.S. All
participants had significant cardiac scarring and approximately two-thirds were wheelchair-dependent at the time they began the
trial. During the 12-month course of the trial, all patients received the standard-of-care for Duchenne muscular dystrophy,
including oral steroids, and 13 also received one dose of intracoronary CAP-1002 (75 million cells) upon randomization.
Using the Performance of the Upper Limb (PUL) test, which is a validated functional assessment of upper limb
activities of daily living (ADL), the researchers found sustained improvement in eight of the nine CAP-1002 treated patients in the
mid-distal PUL, which was manifested by improvements in the patients’ abilities to use their arms and hands. The control patients
had no improvement in the mid-distal PUL tests.
Researchers also reported that magnetic resonance imaging (MRI) found that the participants who received
CAP-1002 had significant myocardial scar reduction and improvement in heart inferior wall systolic thickening compared to the usual
care group. Progressive cardiac scarring eventually impairs the heart’s pumping ability and is the leading cause of death in
Duchenne muscular dystrophy. Usual care patients had no relevant changes in cardiac scar or function over the course of the trial,
while treated patients had less scar in their hearts at both the 6 month and 12 month follow-up after treatment, which suggests a
beneficial effect of CAP-1002.
“Capricor’s pre-clinical studies and the HOPE-Duchenne trial provide evidence that CAP-1002 can help preserve
skeletal and cardiac muscle function in patients in the advanced stages of Duchenne muscular dystrophy,” said Linda Marbán, Ph.D.,
Capricor CEO. “Most importantly, we have shown that there is an opportunity for stabilization or even improvement of disease in
patients with later stage Duchenne muscular dystrophy, which represents a large proportion of those living with the disease.”
Capricor is investigating CAP-1002 in the HOPE-2 clinical trial, a Phase II, randomized, placebo-controlled clinical trial testing repeat
intravenous doses of CAP-1002. Earlier this week, Capricor announced positive outcomes from its Comprehensive Multidisciplinary
meeting with the FDA regarding that trial.
The HOPE-Duchenne trial was funded by Capricor and in part by the California Institute for Regenerative Medicine
(CIRM).
A more detailed presentation on the data is available on Capricor’s website at http://capricor.com/news/events/.
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 type of progenitor cell that has been
shown in pre-clinical and clinical studies to exert potent immuno-modulatory activity, and is being investigated for its potential
to modify 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 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. For more
information, 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 and in its Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, as filed with the
Securities and Exchange Commission on November 14, 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.
For more information, please contact: AJ Bergmann, Chief Financial Officer +1-310-358-3200 abergmann@capricor.com
