MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Dose First Patient in Global Phase I/II Trial of JR-446 for Mucopolysaccharidosis Type IIIB
- The study aims to evaluate the safety, tolerability, and preliminary efficacy of JR-446 for the treatment of people with MPS IIIB, for which there is no approved therapy -
MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd.(TSE 4552, JCR) announced today that the first participant has been dosed in the global Phase I/II trial of JR-446 for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B; “MPS IIIB”). JR-446 is a blood-brain barrier-penetrating α-N-acetylglucosaminidase developed by JCR using its proprietary J-Brain Cargo® technology. MEDIPAL holds commercialization rights for JR-446 outside Japan.
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MPS IIIB is a lysosomal storage disorder that causes severe central nervous system impairment. This ultra-rare disease is estimated to affect approximately 500 to 1,000 people worldwide,1 and there is currently no approved treatment.
