Research Found Exosomes Increased
Exercise Capabilities and Muscle Activity in a Mouse
Model of Duchenne Muscular Dystrophy
During the Action Duchenne International Conference, Capricor
Therapeutics, Inc. (NASDAQ: 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. (NASDAQ: 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.
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 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.
For more information, please contact:
AJ Bergmann, Chief Financial Officer
+1-310-358-3200
abergmann@capricor.com
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