MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) announced today the initiation of the Phase I/II clinical trial for JR-446 in Japan, with the first participant dosed. JR-446 is a proprietary α-N-acetylglucosaminidase designed to cross the blood-brain barrier (BBB) and is being developed for the treatment of mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B or MPS IIIB).
Mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B, affects an estimated 500 to 1,000 individuals worldwide, causing severe central nervous system (CNS) symptoms. Despite the urgent need for treatment, there are currently no approved therapies for this condition. JR-446, developed with JCR’s proprietary J-Brain Cargo® technology, has shown promising non-clinical results in addressing MPS IIIB’s challenging symptoms.
The Phase I/II clinical trial is an open-label, single-arm, multi-center study enrolling individuals under 18 years of age with MPS IIIB. The trial aims to evaluate the safety, tolerability, and preliminary efficacy of JR-446, while determining the optimal dosage through multiple doses. For additional trial details, visit the Clinical Research Submission and Disclosure System (JR-446-101, jRCT2071240043).
Dr. Motomichi Kosuga, Medical Director at the National Center for Child Health and Development, and the study’s medical expert, expressed optimism, saying, “We are pleased to offer a long-awaited treatment for MPS IIIB, a condition for which there are no approved therapies. MPS IIIB primarily affects the CNS and leads to severe neurological symptoms. This trial represents a hopeful step toward addressing these challenges and improving the quality of life for patients and their families.”
In September 2023, MEDIPAL and JCR entered into a licensing agreement where MEDIPAL will commercialize JR-446 outside of Japan and support JCR in its clinical development in Japan, including distribution, disease awareness, and trial advancement. This collaboration underscores the commitment of both companies to advancing treatments for ultra-rare diseases, aiming to provide hope to patients and enhance corporate value while contributing to a healthier society.
About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)
MPS IIIB is an autosomal recessive disease caused by mutations in the NAGLU gene, which encodes an enzyme responsible for breaking down heparan sulfate. The accumulation of this substance in the brain leads to rapid neurological decline, with symptoms including sleep disorders, loss of speech, and behavioral changes, severely impacting the lives of affected individuals and their families.
About the J-Brain Cargo® Platform Technology
JCR’s J-Brain Cargo® platform is a proprietary blood-brain barrier-penetrating technology designed to deliver biotherapeutics to the central nervous system. The first drug developed with this technology, IZCARGO® (pabinafusp alfa), was approved in Japan for treating a lysosomal storage disorder.
About MEDIPAL HOLDINGS CORPORATION
MEDIPAL is a holding company managing companies in the prescription pharmaceutical wholesale, cosmetics, OTC pharmaceuticals, animal health, and food-related businesses. It provides business development services for the MEDIPAL Group.
About JCR Pharmaceuticals Co., Ltd.
JCR is a global pharmaceutical company focused on advancing treatments for rare and genetic diseases. With nearly 50 years of experience in Japan, JCR is expanding into the U.S., Europe, and Latin America. The company develops innovative therapies for diseases like MPS II, Fabry disease, and renal anemia. JCR’s commitment to reliability, confidence, and persistence drives its mission to advance global medical progress.
