LOS ANGELES, July 27, 2017 /PRNewswire/ -- Capricor
Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company
developing biological therapies for Duchenne muscular dystrophy
(DMD) and other rare diseases, today announced that it has received
official minutes of the meeting held recently between the U.S. Food
and Drug Administration (FDA) and Capricor to discuss the
development of intravenous CAP-1002 (allogeneic
cardiosphere-derived cells) for the treatment of Duchenne muscular
dystrophy (DMD).
The minutes indicate:
- the FDA's willingness to accept Capricor's proposal to use the
Performance of the Upper Limb (PUL), an outcomes instrument that
was specifically designed to assess upper limb function in ambulant
and non-ambulant patients with DMD, as the basis for the primary
efficacy endpoint for clinical studies intended to provide
substantial evidence of effectiveness of CAP-1002 in support of a
Biologics License Application (BLA); and,
- the sufficiency of the existing nonclinical safety and efficacy
database to support submission of an Investigational New Drug
application (IND) to clinically evaluate repeat intravenous
administration of CAP-1002.
Capricor has reported positive six-month results from the
ongoing randomized Phase I/II HOPE-Duchenne clinical trial of
CAP-1002) in 25 boys and young men with DMD, in which patients
treated with CAP-1002 demonstrated statistically-significant
improvement compared to usual care control in certain measures of
upper limb function as assessed by the PUL, as well as in certain
cardiac functional measures. CAP-1002 was generally safe and
well-tolerated over the initial six-month follow-up period.
"The FDA's response to our proposed clinical development plan
supports our near-term objective of submitting an IND for
intravenous CAP-1002 as well as provides us with clarity on a path
to potential product registration," said Linda Marbán, Ph.D.,
president and chief executive officer of Capricor. "We look forward
to commencing a randomized, double-blind, placebo-controlled Phase
II clinical trial of intravenous, repeat-dose CAP-1002 in boys and
young men with DMD in the second half of 2017, subject to
regulatory approval."
"The cells in CAP-1002 release exosomes that are
immunomodulatory and exert anti-inflammatory, anti-fibrotic, and
anti-apoptotic effects. By ameliorating the myocyte damage induced
by dystrophin mutations, our product has been demonstrated to
preserve and improve the structure and function of dystrophic
skeletal muscle. Its differentiated mechanism of action supports
its potential to be a standalone therapy as well as an adjunct to
dystrophin-modulating agents," added Dr. Marbán.
About CAP-1002
CAP-1002 consists of allogeneic cardiosphere-derived cells, or
CDCs, a type of progenitor cell that has been shown to exert potent
immuno-modulatory activity. 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 Duchenne Muscular Dystrophy
DMD is a genetic disorder characterized by progressive muscle
degeneration and weakness. It is caused by an abnormality in the
dystrophin complex, a structural element that plays a critical role
in muscle fiber integrity, which leads to chronic muscle damage.
Patients with DMD typically die in their twenties, most commonly
due to heart disease. The incidence of DMD is estimated to be one
in every 3,600 live male births, and DMD is believed to afflict
approximately 15,000 to 20,000 boys and young men in the U.S.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage
biotechnology company developing first-in-class biological
therapies. Capricor's lead candidate, CAP-1002, is a cell-based
candidate currently in clinical development for the treatment of
Duchenne muscular dystrophy. Capricor is also 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.
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; the timing of regulatory approvals; 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, 2016 as
filed with the Securities and Exchange Commission on March 16, 2017, in its Registration Statement on
Form S-3, as filed with the Securities and Exchange Commission on
September 28, 2015, together with
prospectus supplements thereto, and in its Quarterly Report on Form
10-Q for the quarter ended March 31,
2017, as filed with the Securities and Exchange Commission
on May 15, 2017. 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. Capricor's exosomes technology, including
CAP-2003, has not yet been approved for clinical
investigation.
For more information, please contact:
AJ Bergmann, Vice President of Finance
+1-310-358-3200
abergmann@capricor.com
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SOURCE Capricor Therapeutics, Inc.