MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) announced that the two companies have signed an exclusive global licensing deal and a co-development and commercialization partnership in Japan for JR-479, an investigational therapy for GM2 gangliosidosis, an ultra-rare lysosomal storage disorder.

GM2 gangliosidoses are a group of rare, inherited disorders caused by genetic mutations that prevent the normal breakdown of GM2 gangliosides in lysosomes, leading to their buildup, especially in neurons. Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis depending on the specific mutation. GM2 gangliosidosis affects an estimated one in 300,000 people worldwide and is considered an ultra-rare, life-threatening disorder for which no approved treatments currently exist.

JR-479 is a novel drug candidate developed with JCR’s proprietary J-Brain Cargo^® platform, clinically validated through IZCARGO^® for I.V. infusion 10mg, which gained approval in Japan in 2021 for the treatment of a lysosomal storage disorder. In preclinical studies,¹ JR-479 was delivered throughout the body, including to the central nervous system, reducing disease-related substrates and extending survival in animal models.

Under the global licensing deal, MEDIPAL will have exclusive worldwide rights (excluding Japan) to develop, manufacture, and commercialize JR-479, with sub-licensing rights. In Japan, through the co-development partnership, MEDIPAL will handle clinical trial logistics, support disease awareness, and assist with operations, while JCR leads commercialization and records domestic revenue. JCR will also receive an upfront payment, and royalties on overseas sales.

This agreement adds JR-479 to the strategic collaboration between MEDIPAL and JCR, launched in October 2022, to develop treatments for ultra-rare diseases. It follows JR-471 for fucosidosis² and JR-446 for mucopolysaccharidosis type IIIB,³ making JR-479 the third pipeline in the partnership.

Both companies have already factored the financial impact of this agreement into their consolidated forecasts for the fiscal year ending March 31, 2026.