Italfarmaco and JCR Pharmaceuticals: agreement on a new experimental treatment

Italfarmaco and JCR Pharmaceuticals have announced an exclusive licensing agreement for the development and commercialisation of JR-141 in the United States, Europe and Latin America, subject to regulatory approval. JR-141 is an enzyme replacement therapy capable of crossing the blood-brain barrier (BBB), currently undergoing Phase III trials (NCT04573023) as a treatment for Hunter syndrome (also known as mucopolysaccharidosis type II or MPS II). JCR and Italfarmaco intend to seek regulatory approval for JR-141 from the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the Brazilian Health Regulatory Agency (Anvisa). The molecule has already been approved and has been on the market in Japan since 2021 as pabinafusp alfa and was also approved in the United Arab Emirates (UAE) in 2026.

Following regulatory approvals, Italfarmaco will market and distribute the medicine in the authorised territories, whilst JCR will retain responsibility for the manufacture of JR-141 once marketing authorisations have been granted. JCR will receive upfront payments, milestone payments and royalties, in addition to revenue from the supply of the drug.

“This agreement,” explains a statement, “expands the relationship between JCR and Italfarmaco, which entered into an exclusive licence agreement in December 2025 for the commercialisation of givinostat in Japan, a treatment for Duchenne muscular dystrophy, as well as a strategic partnership agreement for therapies for rare diseases”.

JR-141 is a new-generation recombinant fusion protein consisting of an antibody against the human transferrin receptor and iduronate-2-sulfatase, an enzyme that is absent or does not function properly in individuals with Hunter syndrome. JR-141 was developed using J-Brain Cargo, JCR’s proprietary technology for penetrating the blood-brain barrier (BBB). J-Brain Cargo is designed to transport the therapeutic enzyme across the blood-brain barrier to reach the brain directly and address both the somatic and neuropathic symptoms of the disease, which can lead to progressive cognitive decline.

“We are delighted to enter into this strategic agreement with Italfarmaco and to collaborate with an ideal partner for global commercialisation, as we work towards our goal of making JR-141 available to people with Hunter syndrome worldwide – said Hiroyuki Sonoda, President and Chief Scientific Officer of JCR. Italfarmaco has expertise in the development and commercialisation of therapies on a global scale, and we look forward to working with them to make JR-141 available to patients outside Japan as soon as possible. “This agreement reinforces our commitment to our partnership with Italfarmaco, developing therapies for rare and genetic diseases for patients worldwide.”