Italy-based Italfarmaco S.p.A. and Japan-based JCR Pharmaceuticals Co., Ltd. (Tokyo Stock Exchange: 4552) have agreed an exclusive license covering pabinafusp alfa (IZCARGO; JR-141), a blood-brain barrier (BBB)-penetrating enzyme replacement therapy (ERT) for Hunter syndrome (mucopolysaccharidosis type II, or MPS II), granting Italfarmaco exclusive rights to develop and commercialize the asset in the United States, Europe, and Latin America.
Pabinafusp alfa is a recombinant fusion protein combining an antibody against the human transferrin receptor with iduronate-2-sulfatase, the enzyme deficient in MPS II. Developed using JCR's J-Brain Cargo blood-brain barrier technology, it is designed to cross the BBB through transferrin receptor-mediated transcytosis and address both somatic and neuronopathic manifestations of Hunter syndrome. Current enzyme replacement therapies do not address the central nervous system manifestations of the disease.
JR-141 is in a global Phase III trial (NCT04573023) conducted by JCR Pharmaceuticals, was approved in Japan in 2021 and approved in the United Arab Emirates in 2026, and the companies plan to seek approval from the US Food and Drug Administration, the European Medicines Agency, the UK Medicines and Healthcare products Regulatory Agency and the Brazilian Health Regulatory Agency.
Under the agreement, Italfarmaco will handle commercialization and distribution in the licensed territories following regulatory approvals, while JCR retains manufacturing responsibility. JCR will receive upfront, milestone, and royalty payments plus product supply revenues; specific figures were not disclosed.