MEDIPAL and JCR Announce Completion of Clinical Trial Notification Process in Japan for Phase I/II Study of JR-446 for Mucopolysaccharidosis Type IIIB
July 02 2024 - 7:30AM
Business Wire
MEDIPAL HOLDINGS CORPORATION (TSE 7459 “MEDIPAL”) and JCR
Pharmaceuticals Co., Ltd. (TSE 4552 “JCR”) today announced the
completion of the regulatory review by the Pharmaceuticals and
Medical Devices Agency (PMDA) for the clinical trial notification
for the phase I/II study of JR-446, a blood-brain
barrier-penetrating α-N-acetylglucosaminidase, for the treatment of
mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome type
B), a devastating and ultra-rare lysosomal storage disorder.
MPS IIIB affects an estimated 500 to 1,000 individuals
worldwide1, causing severe central nervous system symptoms. Despite
the dire need, there are currently no approved treatments available
for this condition. JR-446, developed using JCR’s proprietary
J-Brain Cargo® technology, has shown promising preclinical results
in addressing the symptoms of this challenging disorder.
In September 2023, MEDIPAL and JCR secured a license to
commercialize JR-446 overseas and partnered to develop and
commercialize it in Japan, targeting MPS IIIB2. With the regulatory
review now complete, we plan to start the clinical trial in Japan
in the first half of FY2024 (April to September).
This collaboration highlights the commitment of MEDIPAL and JCR
to pioneer treatments for ultra- rare diseases. By advancing
therapies like JR-446, we aim to bring hope to patients and their
families, while enhancing corporate value and contributing to a
society where everyone can live with physical and mental
well-being.
About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome
Type B) Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome
type B, is an autosomal recessive disease caused by pathogenic
mutations in the NAGLU gene, encoding a lysosomal enzyme involved
in the degradation of heparan sulfate. With the accumulation of
heparan sulfate in the central nervous system in the brain,
individuals with this condition present rapid neurological decline,
including sleep disorders, loss of speech, and behavioral changes,
which may significantly affect the quality of life of patients and
their families.
About MEDIPAL HOLDINGS CORPORATION MEDIPAL is a holding
company which controls, administers and supports the operating
activities of companies in which it holds shares in the
Prescription Pharmaceutical Wholesale Business; the Cosmetics,
Daily Necessities and OTC Pharmaceutical Wholesale Business; and
the Animal Health Products and Food Processing Raw Materials
Wholesale and Related Business, and conducts business development
for the MEDIPAL Group.
For more information, visit
https://www.medipal.co.jp/english/
About JCR Pharmaceuticals Co., Ltd.
JCR is a global specialty pharmaceuticals company dedicated to
advancing treatments for rare and genetic diseases. With nearly 50
years of expertise in Japan, we are expanding to the US, Europe,
and Latin America. Our innovative therapies address conditions like
growth disorder, MPS II, Fabry disease, acute graft-versus-host
disease, and renal anemia. We are also developing treatments for
rare diseases like MPS I, MPS II, MPS IIIA and B, and more. Our
core values of reliability, confidence, and persistence drive our
mission to enhance global medical progress.
For more information, visit
https://www.jcrpharm.co.jp/en/site/en/.
References
1 Based on data from the Ministry of Health, Labour and
Welfare’s public research and JCR’s own investigations.
2 Reference: Press release on the licensing agreement for JR-446
between MEDIPAL and JCR (September 28, 2023).
This news release is intended to provide the
latest information on two companies businesses to shareholders and
other investors, and members of the press. The information on
pharmaceutical products (including those under development) in this
document is not intended for advertising, providing medical advice
or other such purposes.
The English version of this document is a
translation of the Japanese original and is provided for
information purposes only. While reasonable efforts have been made
to provide an accurate translation, no liability is assumed by
MEDIPAL and JCR for any errors, omissions, or ambiguities in the
translation. In the event of any inconsistency or conflict between
the English version and the Japanese original, the Japanese
original shall prevail.
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MEDIPAL HOLDINGS CORPORATION Public Relations Department TEL:
(+81)-3-3517-5171 JCR Pharmaceuticals Co., Ltd. Corporate
Communications TEL: (+81)-797-32-1995