Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular Dystrophy with an Aim to Expedite BLA Pathway
11 June 2024 - 11:25PM
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company
developing transformative cell and exosome-based therapeutics for
the treatment of rare diseases, today announced the successful
completion of a Type-B meeting with the U.S. Food and Drug
Administration (FDA) on next steps for the Biologics License
Application (BLA) submission with its lead asset, CAP-1002 for the
treatment of Duchenne muscular dystrophy (DMD). Additionally,
Deramiocel has been selected as the proposed International
Nonproprietary Name (INN) for CAP-1002 by the World Health
Organization.
“The last year has been transformative for
Capricor as we have moved rapidly towards potential approval for
CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D.,
Capricor’s chief executive officer. “The most recent meeting had
several very important outcomes; the first of which was that the
FDA has agreed to a pre-BLA meeting based on their review of our
clinical data from HOPE-2, HOPE-2 open label extension (OLE) and
available data from HOPE-3. This key agreement will allow us to
begin our BLA filing with a rolling submission planned to initiate
in the third quarter of 2024.”
The rolling submission process allows for the
submission of pre-agreed components of the BLA to be submitted as
they are completed, which can streamline the regulatory review
process and de-risk the BLA. Capricor plans to further outline the
submission schedule with the FDA in the coming months.
Dr. Marbán continued, “We continue to make
significant progress on this program as we are awaiting final data
in the fourth quarter of 2024 from our already enrolled HOPE-3
pivotal trial and prepare for potential commercialization,
including completion of our commercial manufacturing facility and
actively preparing for our pre-BLA meeting and rolling BLA
submission. Furthermore, we are looking forward to our presentation
of additional 3-year data from our HOPE-2 OLE study at the upcoming
PPMD conference which we believe will add further evidence of the
efficacy and safety of CAP-1002.”
Deramiocel (CAP-1002) for the treatment of DMD
has received Orphan Drug Designation and the regulatory pathway for
this drug is supported by RMAT (Regenerative Medicine Advanced
Therapy Designation). In addition, if Capricor were to receive FDA
marketing approval for Deramiocel for the treatment of DMD,
Capricor would be eligible to receive a Priority Review Voucher
(PRV) based on its previous receipt of a rare pediatric disease
designation. Capricor retains full rights to the PRV, if
received.
About Duchenne Muscular
Dystrophy
Duchenne muscular dystrophy (DMD) is a
devastating genetic disorder characterized by progressive weakness
and chronic inflammation of the skeletal, heart and respiratory
muscles with mortality at a median age of approximately 30 years.
It is estimated that DMD occurs in approximately one in every 3,500
male births and that the patient population is estimated to be
approximately 15,000-20,000 in the United States. DMD
pathophysiology is driven by the impaired production of functional
dystrophin, which normally functions as a structural protein in
muscle. The reduction of functional dystrophin in muscle cells
leads to significant cell damage and ultimately causes muscle cell
death and fibrotic replacement. Treatment options are limited and
there is no cure.
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, Deramiocel (CAP-1002), an allogeneic
cardiac-derived cell therapy. Extensive preclinical and clinical
studies have shown Deramiocel to demonstrate immunomodulatory,
antifibrotic, and regenerative actions specifically tailored for
dystrophinopathies and heart disease. Deramiocel 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 in preclinical development 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;
dates for regulatory meetings; statements about our financial
outlook; 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 revenue streams and
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, 2023, as filed with the Securities and
Exchange Commission on March 11, 2024, and in our Quarterly Report
on Form 10-Q for the quarter ended March 31, 2024, as filed with
the Securities and Exchange Commission on May 14, 2024. 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 an agreement for
the exclusive commercialization and distribution of Deramiocel
(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. Deramiocel (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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