Capricor Therapeutics Announces Upcoming Type-B Meeting with the FDA to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy
February 27 2024 - 8:30AM
– Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company
developing transformative cell and exosome-based therapeutics for
the treatment and prevention of rare diseases, announced today that
the Company has been granted an in-person Type-B meeting with the
U.S. Food & Drug Administration (FDA) scheduled for late March.
In the upcoming meeting with the FDA, the Company intends to
discuss its proposed chemistry, manufacturing and controls (CMC)
plans for commercial launch, if approved which may enable
opportunities to expedite the pathway to a Biologics License
Application (BLA) submission. The Company has previously been
granted Regenerative Medicine Advanced Therapy (RMAT) and orphan
drug designations for the use of CAP-1002 in Duchenne muscular
dystrophy (DMD) and these designations enable sponsors to work
closely with the FDA and receive its guidance on potential
expedited pathways for approval.
“At this time, with our pivotal Phase 3, HOPE-3
trial fully enrolled in Cohort A and top-line data expected in the
fourth quarter of this year, we believe that discussing our CMC
plans for potential commercialization with the FDA will allow us
the opportunity to bring CAP-1002 to patients in the most
expeditious manner possible for patients in need,” said Linda
Marbán, Ph.D., Capricor’s chief executive officer. “At Capricor, we
remain focused on getting to approval as quickly as possible and we
look forward to providing more details after this meeting.”
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a
biotechnology company dedicated to advancing transformative cell
and exosome-based therapeutics to redefine the treatment landscape
for rare diseases. At the forefront of our innovation is our lead
product candidate, CAP-1002 — an allogeneic cardiac-derived cell
therapy. Extensive preclinical and clinical studies have shown
CAP-1002 to demonstrate immunomodulatory, antifibrotic, and
regenerative actions specifically tailored for dystrophinopathies
and heart disease. CAP-1002 is currently advancing through Phase 3
clinical development for the treatment of Duchenne muscular
dystrophy (DMD). Capricor is also harnessing the power of our
exosome technology, using our proprietary StealthX™ platform which
is focused on the areas of vaccinology, targeted delivery of
oligonucleotides, proteins and small molecule therapeutics to
potentially treat and prevent a diverse array of diseases. At
Capricor, we stand committed to pushing the boundaries of
possibility and forging a path toward transformative treatments for
those in need. For more information, visit capricor.com, and
follow Capricor
on Facebook, Instagram and Twitter.
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; manufacturing capabilities; the
ability to achieve product milestones and to receive milestone
payments from commercial partners; 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 and revenue
projections; 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, 2022, as filed with the Securities and
Exchange Commission on March 17, 2023 and in our Quarterly Report
on Form 10-Q for the quarter ended September 30, 2023, as filed
with the Securities and Exchange Commission on November 14, 2023.
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.
Capricor has entered into
a partnership for the exclusive commercialization and
distribution of CAP-1002 for DMD in the United States and Japan
with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma,
Inc.), subject to regulatory approval. CAP-1002 is an
Investigational New Drug and is not approved for any indications.
None of Capricor’s exosome-based candidates have been approved for
clinical investigation.
For more information, please
contact:
Capricor Company Contact:AJ
Bergmann, Chief Financial Officerabergmann@capricor.com
858.727.1755
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