Italfarmaco Licenses JCR’s Hunter Syndrome Therapy JR-141

Italfarmaco and JCR Pharmaceuticals have signed an exclusive agreement covering development and potential commercialization of JR-141, an experimental treatment for Hunter syndrome, in the United States, Europe and Latin America.

JR-141 is in a global Phase 3 clinical trial for Hunter syndrome, also known as mucopolysaccharidosis type II. The therapy is approved in Japan as Izcargo, where it was launched in 2021. JCR and Italfarmaco plan to seek approvals in the United States, European Union, United Kingdom and Brazil.

Under the agreement, Italfarmaco will commercialize and distribute JR-141 in the licensed territories if regulators authorize the product. JCR will continue to manufacture the treatment. JCR is entitled to upfront payments, milestone payments, royalties and revenue related to product supply; the companies did not disclose financial terms.

Hunter syndrome is a rare genetic disorder in which the body lacks or has a malfunctioning form of the enzyme iduronate-2-sulfatase. JR-141 combines that enzyme with an antibody directed at the human transferrin receptor.

The product was developed using JCR’s J-Brain Cargo technology, designed to transport the therapeutic enzyme across the blood-brain barrier. The approach is intended to address physical symptoms as well as neurological effects associated with the disease, including progressive cognitive decline.

The agreement expands an existing relationship between the companies. In December 2025, JCR granted Italfarmaco rights to commercialize givinostat, a treatment for Duchenne muscular dystrophy, in Japan. The companies also entered a strategic partnership focused on rare disease therapies.

The new agreement places responsibility for commercial development and distribution with Italfarmaco across the named territories while JCR retains manufacturing responsibility.

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