Capricor to Meet with FDA under its RMAT Designation to Discuss HOPE-2 Clinical Trial
November 12 2018 - 07:00AM
Focus will be on Milestones for Development
and Commercialization CAP-1002 Therapy for Duchenne Muscular
Dystrophy
Capricor Therapeutics (NASDAQ: CAPR) announced today that it will
meet with the U.S. Food and Drug Administration (FDA) in December
to discuss clinical trial design, surrogate or intermediate
endpoints and manufacturing processes for CAP-1002, Capricor’s
novel cell therapy. Currently, patients are being enrolled in the
HOPE-2 clinical trial, which is investigating CAP-1002 as a therapy
for the treatment of Duchenne muscular dystrophy.
The in-person meeting is part of the expedited
review process afforded to Capricor for its CAP-1002 product
candidate after being granted the Regenerative Medicine Advanced
Therapy (RMAT) designation by the FDA in February
2018. The FDA grants the RMAT designation to regenerative medicine
therapies intended to treat a serious condition and for which
preliminary clinical evidence indicates a potential to address
unmet medical needs for that condition.
“We are very pleased to have this opportunity to
discuss CAP-1002 and the HOPE-2 clinical trial with the FDA, and we
appreciate the key opinion leaders in the field of Duchenne
muscular dystrophy who will be joining us for the meeting,” said
Linda Marbán, Ph.D., Capricor’s chief executive officer. “HOPE-2 is
a randomized, placebo-controlled clinical trial testing repeat
intravenous doses of CAP-1002. The study is currently enrolling up
to 84 boys and young men at approximately 10-15 centers across the
U.S. The participants are in the advanced stages of Duchenne
muscular dystrophy and are typically not eligible for many of the
other clinical trials being conducted for Duchenne. We believe that
CAP-1002 could fill a currently unmet medical need for these
patients.”
Pre-clinical and clinical studies have shown
that CAP-1002 is generally safe, well-tolerated and demonstrated
significant and sustained signals of improvement in cardiac and
skeletal muscle function in patients with Duchenne muscular
dystrophy, a fatal genetic disease with limited treatment
options.
The RMAT designation allows eligible therapies
similar access to expedited development and review of a marketing
application that are available to drugs that receive breakthrough
therapy designation – including increased opportunities to meet
with the FDA, early interactions with the FDA to discuss any
potential surrogate or intermediate endpoints and the potential to
support accelerated approval of drugs receiving the RMAT
designation.
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 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 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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