KYOTO, Japan, Jan. 25, 2022 /PRNewswire/ -- *NS Pharma
Note: CAP-1002 is an investigational therapy and not approved in
the US.
This press release is being provided for a US audience as a
reference. The original news release was issued by Nippon Shinyaku
Co., Ltd., the parent company of NS Pharma on January 25, 2022 (Japan standard time). Please click here
(https://www.nippon-shinyaku.co.jp/english/index.php). The text
contains content related to U.S. unapproved drugs and unapproved
indications.
Nippon Shinyaku Co., Ltd. (Headquarters: Kyoto, Japan, President: Toru Nakai) announced today that Nippon Shinyaku
and Capricor Therapeutics (Headquarters: California, USA, CEO: Linda Marbán, NASDAQ: CAPR) have entered into an
exclusive distribution agreement for CAP-1002 for the treatment* of
Duchenne muscular dystrophy (DMD) in the territory of US.
DMD is a progressive muscular dystrophy caused by a deficiency
of the dystrophin protein leading to weakness of skeletal, cardiac,
and pulmonary muscles. DMD has a variety of genotypic variants, and
therapeutic agents have been developed for patients with DMD caused
by specific genetic mutations, thus contributing to the fulfillment
of unmet medical needs. However, treatment for DMD patients due to
the other type of genetic mutations and treatment for relatively
older, non-ambulant DMD patients is still unsatisfactory, and
effective therapies are required to be developed.
CAP-1002 is comprised of human allogeneic cardiosphere-derived
cells. Exosomes (extracellular vesicles) secreted by CAP-1002 are
thought to be the mechanism of action of CAP-1002 and have been
shown to reduce oxidative stress, inflammation, fibrosis, and
increase myocyte generation, as well as improve motor and cardiac
function. CAP-1002 is expected to be effective in a wide range of
DMD patients, regardless of the type of genetic mutation, and a
Phase II clinical trial (HOPE-2 study) suggested improved upper
limb function and improved cardiac function in non-ambulatory DMD
patients. CAP-1002 has received orphan drug designation and RMAT
designation from the U.S. Food and Drug Administration (FDA) and
Capricor is currently ready to initiate a Phase III clinical trial
(HOPE-3 study) at several US health institutions. After the BLA
approval in US, CAP-1002 will be marketed by our wholly owned
subsidiary NS Pharma, Inc. (New Jersey,
USA, President: Tsugio
Tanaka).
Nippon Shinyaku is focusing on the field of intractable, rare
disorders, and have commercialized our in-house developed DMD
treatment, Viltepso® (an antisense exon skipping agent)
in the US. Executing this agreement with Capricor, Nippon Shinyaku
aims to contribute further to the treatment for patients suffering
from DMD.
About exosomes
Exosomes (extracellular vesicles) are
vesicles of about 30 to 150 nanometers in diameter surrounded by a
lipid-doublet membrane that are secreted by cells. Exosomes were
previously considered relating to the excretion of intracellular
waste products, but in recent years, it has been discovered and
well known that exosomes contain proteins and nucleic acids and act
as important mediators of intercellular communication. Research and
development of clinical applications, such as therapeutic,
diagnostic, and DDS, using this function has been actively
pursued.
About Orphan Drug Designation
The Orphan Drug
Designation is provided for orphan drugs and biologics intended for
the safe and effective treatment, diagnosis or prevention of rare
diseases/disorders that affect fewer than 200,000 people in the US,
or that affect more than 200,000 persons but are not expected to
recover the costs of developing and marketing a treatment drug.
With orphan drug designation, the FDA grants a seven-year market
exclusivity and provides tax incentives to the orphan drugs and
biologics.
About RMAT designation
RMAT (Regenerative Medicine
Advanced Therapy) is an FDA-designated system for advanced
regenerative medicine therapies targeted at developing serious
diseases that have shown certain effect in clinical trials based on
the 21st Century Treatment Act (21st Century Cures Act) in US.
Companies with RMAT designations are given the opportunity for
priority review and accelerated approval for the product.
About Nippon Shinyaku
Our mission is to help people
lead healthier and happier lives. Through creating unique medicines
that will bring hope to patients and families struggling with
illness, we aim to be an organization trusted by the community.
Please visit our website
(https://www.nippon-shinyaku.co.jp/english/) for products or
detailed information.
About Capricor Therapeutics
Capricor Therapeutics,
Inc. (NASDAQ: CAPR) is a biotechnology company focused on
developing transformative cell and exosome-based therapeutics and
vaccines for treating and preventing a broad spectrum of diseases.
Capricor is also developing its exosome technology as a
next-generation therapeutic platform for the delivery of nucleic
acids, including mRNA to treat or prevent a variety of diseases.
For more information, visit https://www.capricor.com.
Contact
Corporate Communications Dept., Nippon
Shinyaku Co., Ltd.
FAX: +81-75-321-9128
About VILTEPSO® (viltolarsen) injection
Indication
VILTEPSO is indicated for the treatment of
Duchenne muscular dystrophy (DMD) in
patients who have a confirmed mutation of the DMD gene that is
amenable to exon 53
skipping. This indication is approved under accelerated approval
based on an increase
in dystrophin production in skeletal muscle observed in patients
treated with VILTEPSO.
Continued approval for this indication may be contingent upon
verification and
description of clinical benefit in a confirmatory trial.
Important Safety Information
In clinical studies, no patients experienced kidney toxicity during
treatment with
VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be
possible. Your
doctor may monitor the health of your kidneys before starting and
during treatment with
VILTEPSO.
Common side effects include upper respiratory tract infection,
injection site reaction,
cough, and fever.
For more information about VILTEPSO, see full Prescribing
Information.
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SOURCE NS Pharma