Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular Dystrophy
April 24 2024 - 8:00AM
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company
developing transformative cell and exosome-based therapeutics for
the treatment and prevention of rare diseases, today announced an
update from the Company’s recent Type-B Chemistry, Manufacturing
and Controls (“CMC”) 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 in
treating Duchenne muscular dystrophy (“DMD”).
The FDA has affirmed alignment with
Capricor on the following topics:
Pre-BLA Meeting and Rolling BLA
Submission
- The FDA advised Capricor to include
discussion for a pre-BLA meeting and rolling BLA schedule in the
upcoming Type-B meeting.
- Based on this feedback, Capricor
has already been granted a subsequent Type-B meeting to be held in
the second quarter of 2024 to discuss these topics, with the
results of those discussions to potentially lead to an accelerated
BLA filing.
- Capricor plans to share with FDA
its HOPE-2 open label extension (“OLE”) 3-year safety and efficacy
data which is expected to be available in the second quarter of
2024 as part of Capricor’s ISS and ISE strategy.
Establishment of Non-Clinical
Comparability
- The FDA agreed that comparability
between drug product manufactured at our two different facilities
(Los Angeles and San Diego) has been demonstrated using the
provided analytical comparability data.
- This allows for the use of CAP-1002
drug product manufactured at our San Diego manufacturing facility
upon potential product approval.
- Data from Cohort B of the HOPE-3
clinical trial will not be necessary for FDA approval of the
product.
"I am extremely pleased with our recent FDA
interactions as we continue to work collaboratively with the agency
to align on the most expeditious path towards registration of
CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D.,
Capricor’s chief executive officer. “Capricor has generated
extensive safety and efficacy data in multiple clinical trials and
we are very encouraged by the FDA’s agreement that we have
successfully demonstrated product comparability which allows for a
seamless transition to our San Diego manufacturing facility without
the need for additional clinical data. Contingent upon our upcoming
meeting, FDA is supportive of our plan to submit a rolling BLA,
which may expedite our path to potential approval. In addition,
establishment of non-clinical comparability allows us to be able to
conserve resources and focus on preparing our facility in San Diego
for potential launch. Furthermore, we continue to work diligently
with our partner, Nippon Shinyaku (U.S. subsidiary: NS Pharma,
Inc.) as we prepare for the potential launch of CAP-1002. Looking
ahead, later this quarter, we remain on track to report the 3-year
HOPE-2 OLE results as well as reporting the outcome of our next FDA
meeting.”
CAP-1002 for the treatment of DMD has received
Orphan Drug Designation and the regulatory pathway for CAP-1002 is
supported RMAT (Regenerative Medicine Advanced Therapy
Designation). In addition, if Capricor were to receive FDA
marketing approval for CAP-1002 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.
Virtual Investor Webcast and Conference
Call
Capricor management will host a virtual investor
webcast and conference call with slides on Monday, April 29, 2024,
at 8:30 a.m. ET. To participate in the conference call, please dial
888-886-7786 (domestic/toll-free) or 416-764-8658 (international)
and reference the conference ID: 34112256. Participants can use
guest dial-in numbers above to be answered by an operator or click
the Call me™ link for instant telephone access. To
participate via webcast, please click here to view the slides.
A replay of the webcast will be available following the conclusion
of the live broadcast and will be accessible on the Company’s
website.
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 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 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, 2023, as filed with the Securities and
Exchange Commission on March 11, 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
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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