MEDIPAL and JCR Complete Clinical Trial Notification for JR-446 in Japan

HYOGO, Japan -- MEDIPAL HOLDINGS CORPORATION (TSE 7459, "MEDIPAL") and JCR Pharmaceuticals Co., Ltd. (TSE 4552, "JCR") have jointly announced the completion of the regulatory review by the Pharmaceuticals and Medical Devices Agency (PMDA) for their clinical trial notification concerning JR-446. This innovative treatment is aimed at mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome type B), an exceptionally rare lysosomal storage disorder characterized by severe central nervous system symptoms. Affecting between 500 and 1,000 individuals globally, MPS IIIB currently has no approved treatment options.

JR-446, a blood-brain barrier-penetrating α-N-acetylglucosaminidase, was developed using JCR's proprietary J-Brain Cargo® technology. The preclinical results have shown promise in addressing the devastating symptoms associated with MPS IIIB. In September 2023, MEDIPAL and JCR obtained a license to commercialize JR-446 internationally and entered into a partnership to develop and market the drug within Japan, specifically targeting MPS IIIB.

With the regulatory review completed, the companies plan to initiate the clinical trial in Japan within the first half of the fiscal year 2024, spanning from April to September. This collaboration underscores MEDIPAL and JCR's commitment to pioneering new treatments for ultra-rare diseases. By advancing therapeutic options such as JR-446, they aim to bring hope to affected patients and their families, while also enhancing their corporate value and contributing to societal well-being.

Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, arises from pathogenic mutations in the NAGLU gene, which encodes an enzyme responsible for the degradation of heparan sulfate within lysosomes. The accumulation of heparan sulfate in the brain’s central nervous system leads to rapid neurological decline. Symptoms include sleep disorders, loss of speech, and behavioral changes, all of which severely impact the quality of life for patients and their families.

MEDIPAL HOLDINGS CORPORATION operates as a holding company that administers and supports the activities of its subsidiary companies. It operates primarily in three business areas: Prescription Pharmaceutical Wholesale, Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale, and Animal Health Products and Food Processing Raw Materials Wholesale. MEDIPAL is also involved in business development for the entire MEDIPAL Group.

JCR Pharmaceuticals Co., Ltd. is a global specialty pharmaceuticals company focusing on treatments for rare and genetic diseases. With nearly half a century of expertise in Japan, JCR is expanding its reach to the US, Europe, and Latin America. The company develops innovative therapies for conditions such as growth disorders, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. Additionally, JCR is working on treatments for other rare diseases, including MPS I, MPS II, MPS IIIA, and B. The core values of reliability, confidence, and persistence drive JCR's mission to advance global medical progress.