MEDIPAL and JCR Pharmaceuticals Announce Regulatory Review Completion for Phase I/II Trial of JR-446 in Treating Ultra-Rare Sanfilippo Syndrome Type B

03 July 2024 | Wednesday | News

PMDA Approval Paves Way for Clinical Trial of Promising Blood-Brain Barrier-Penetrating Therapy, JR-446, Targeting MPS IIIB in Japan by FY2024
Image Credit : BioPharma APAC Creative Studio

Image Credit : BioPharma APAC Creative Studio

MEDIPAL HOLDINGS CORPORATION  and JCR Pharmaceuticals Co., Ltd.  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.


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.


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