Italfarmaco S.p.A. and JCR Pharmaceuticals have entered into an exclusive license agreement to develop and commercialise JR-141 (pabinafusp alfa) in the US, Europe and Latin America, subject to regulatory approval. JR-141 is a Blood-Brain Barrier (BBB)-penetrating enzyme replacement therapy currently being evaluated in a Phase 3 trial for Hunter syndrome.

Under the agreement, Italfarmaco will seek regulatory approvals from the US FDA, European Medicines Agency (EMA), UK MHRA and Brazil’s ANVISA. Following approvals, Italfarmaco will commercialise and distribute JR-141 in the licensed territories, while JCR will retain responsibility for manufacturing. JCR will receive upfront, milestone and royalty payments, along with product supply revenues.

JR-141 is a recombinant fusion protein combining an antibody targeting the human transferrin receptor with iduronate-2-sulfatase, the enzyme deficient in people with Hunter syndrome. JCR developed the therapy using its proprietary J-Brain Cargo technology, designed to enable therapeutic enzymes to cross the BBB and reach the Central Nervous System (CNS).

Hunter syndrome is an X-linked lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase, resulting in the accumulation of glycosaminoglycans and a range of systemic and neurological symptoms. Current enzyme replacement therapies generally do not address CNS-related manifestations of the disease.

JR-141 is already approved and marketed in Japan as Izcargo (pabinafusp alfa) and was also approved in the UAE in 2026. The therapy is currently being evaluated in a Phase 3 study (NCT04573023) for potential use in additional markets.

The agreement builds on the companies’ existing relationship established in 2025 for the commercialization of givinostat in Japan for Duchenne muscular dystrophy, alongside a strategic partnership covering rare disease therapies.