Full Press Release Details
Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for CAP-1002
Registration Trial in Duchenne Muscular Dystrophy
on Track to Initiate in First Quarter of 2018
Host Conference Call and Webcast at 4:30 p.m. ET Today
Nov. 29, 2017 - Capricor Therapeutics (NASDAQ: CAPR) today announced that the U.S. Food and Drug Administration (FDA)
has cleared its Investigational New Drug (IND) application to conduct a new clinical trial of CAP-1002, its lead investigational
therapy, in boys and young men in advanced stages of Duchenne muscular dystrophy, a fatal genetic disorder for which there are
limited treatment options.
double-blind, placebo-controlled clinical trial will be called the HOPE-2 Trial and is designed to evaluate the safety and efficacy
of intravenous, repeat doses of CAP-1002 in boys and young men whose ability to walk has been seriously impaired by the loss of
muscle function that occurs as Duchenne muscular dystrophy progresses. The primary efficacy endpoint will be the relative change
in the mid-level dimension of the Performance of the Upper Limb test from baseline to Month 12. The HOPE-2 Trial is expected to
enroll approximately 84 patients and be conducted at 10-12 U.S. sites.
that if the primary endpoint is reached, the HOPE-2 Trial could serve as a registration trial, meaning that its results could
support the submission of a Biologics License Application (BLA) to obtain marketing approval of CAP-1002. Capricor expects to
initiate the HOPE-2 Trial in the first quarter of 2018.
to apply for the Regenerative Medicine Advanced Therapy (RMAT) Designation for CAP-1002 based on updated guidance recently issued
by the FDA. If granted, the RMAT Designation would be expected to facilitate CAP-1002's path to potential registration.
principal investigator for the HOPE-2 trial will be Craig M. McDonald, M.D., a distinguished thought leader in the clinical management
of neuromuscular diseases, including muscular dystrophies, and the development of novel outcome measures for Duchenne muscular
dystrophy clinical trials.
FDA's clearance of this IND upon its initial submission is a significant step forward in our development of CAP-1002,"
said Linda Marb n, Ph.D., president and chief executive officer. "While there are many clinical initiatives in Duchenne
muscular dystrophy, this is one of the very few to focus on non-ambulant patients. These boys and young men are looking to maintain
what function they have in their arms and hands and, based on our previous study, we think CAP-1002 may be able to do exactly
reported significant and sustained improvements in cardiac structure and function, as well as skeletal muscle function, following
a single dose of intracoronary CAP-1002. The HOPE-2 Trial will test the potential benefit of CAP-1002 as a repeated therapy delivered
intravenously, with the goal of providing long-term benefit in a format that is compatible with repeat dosing over time. Support
for intravenous infusion, a common mode of drug delivery, is provided by studies which have shown therapeutic benefit in an animal
model of Duchenne muscular dystrophy.
hold a conference call and slide presentation at 4:30 p.m. ET today to discuss this development. To join: please dial 1-866-652-5200
(domestic) or 1-412-317-6060 (international). Access to the webcast and a replay of the call may be found at http://capricor.com/news/events/.
was funded in part by the California Institute for Regenerative Medicine.
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
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
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.
Note Regarding Forward-Looking 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, 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 the prospectus included therein and prospectus supplements thereto, and in its Quarterly Report on Form 10-Q for
the quarter ended September 30, 2017, as filed with the Securities and Exchange Commission on November 14, 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.
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,
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