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 a-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 worldwide, 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, company plan to start the clinical trial in Japan in the first half of fiscal 2024 (April to September). This collaboration highlights the commitment of MEDIPAL and JCR to pioneer treatments for ultrarare diseases.

By advancing therapies like JR-446, company 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.