At the Gordon Research Conference on Extracellular Vesicles in
Newry, Maine, Capricor Therapeutics (NASDAQ: CAPR) presented
research findings on the mechanism of action and the
immunomodulatory capacities of CAP-2003, the company’s
investigational therapy comprised of proprietary extracellular
vesicles, including exosomes, which are derived from
cardiosphere-derived cells (CDC-EVs). Capricor is developing
CAP-2003 as a therapeutic platform for treating diseases involving
inflammation and fibrosis.
The Gordon Research Conference on Extracellular
Vesicles is focused on cutting-edge research on the biogenesis,
molecular composition, functions, physio-pathological roles and
potential clinical applications of extracellular vesicles. Gordon
Research Conferences are a group of prestigious international
scientific conferences that are at the forefront of research in the
biological, chemical and physical sciences and their related
technologies.
“The pre-clinical studies presented at the
Gordon Research Conference further elucidate Capricor’s
progress in developing this exciting new class of therapeutics, the
exosomes which comprise our investigational therapy, CAP-2003,”
said Linda Marbán, Ph.D., Capricor chief executive officer. “The
studies further demonstrate that exosomes may be the active
pharmaceutical ingredient (API) in CAP-1002, our cell therapy
product, because these extracellular vesicles serve as
cellular-messengers, altering function and physiology to balance
inflammation so that cellular repair can be facilitated.”
In the first study, Capricor compared CAP-2003
with exosomes made from mesenchymal stem cells (MSCs). The research
found that CAP-2003 contains a unique cocktail of microRNAs,
non-coding RNAs and proteins that drive greater immunomodulation,
suggesting that CAP-2003 may be a more efficacious product
candidate in diseases of inflammation and fibrosis.
This study also showed that CAP-2003 polarizes
macrophages – a type of white blood cells involved in removing
cellular debris and in tissue repair – to elicit anti-inflammatory
and pro-regenerative responses. In addition, the study found that
CAP-2003 has an immunomodulatory effect on T-cells, modifying the
immune response of this critical component of the immune system.
Researchers concluded that this study supports the use of CAP-2003
for the potential treatment of inflammatory indications.
An abstract from the second study reported that
in a mouse model of Duchenne muscular dystrophy, CAP-2003 produced
improvements that are similar to those attributed to CAP-1002,
Capricor’s cell therapy product. The findings suggest that the
exosomes that make up CAP-2003 mediate the CDCs’ mechanism of
action. Capricor researchers also tested in vitro uptake of CDC-EVs
by different cell types and found that CDC-EVs are taken up by
immune cells with little uptake by fibroblasts and epithelial
cells. This finding sheds further light on the mechanism of action
of CDC-EVs in modulating inflammation and stimulating tissue
repair.
Capricor is now conducting HOPE-2, a Phase II,
randomized, double-blind, placebo-controlled study of repeat doses
of CAP-1002 delivered intravenously. The study is enrolled in
approximately 84 boys and young men in advanced stages of Duchenne
muscular dystrophy. For more information on HOPE-2, please visit
www.HOPE2Trial.com.
The posters presented at the Gordon Conference
will be 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 is comprised 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 CAP-2003
CAP-2003 is being developed as a next-generation
therapeutic platform in regenerative medicine. CAP-2003 is
comprised of nano-sized extracellular vesicles, including exosomes
and microvesicles, derived from human CDCs, which exert
anti-inflammatory, pro-angiogenic, anti-apoptotic, and
anti-fibrotic effects. CAP-2003 contains several characteristic
lipids, proteins, and RNA molecules (e.g., microRNAs). They act as
messengers to regulate the functions of neighboring cells.
Pre-clinical research has shown that exogenously-administered
extracellular vesicles can direct or, in some cases, re-direct
cellular activity, supporting their therapeutic potential. Their
size, ease of crossing cell membranes and ability to communicate in
native cellular language make them an exciting class of potential
therapeutic agents.
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, 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 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.
For more information, please contact:
AJ Bergmann, Chief Financial Officer
+1-310-358-3200
abergmann@capricor.com
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