Italfarmaco licenses JCR’s investigational Hunter syndrome therapy for US, Europe and Latin America
Italfarmaco S.p.A. and JCR Pharmaceuticals Co., Ltd. announced an exclusive license agreement to develop and commercialize JR-141 in the United States, Europe, and Latin America upon regulatory approval.
JR-141 is a blood-brain barrier (BBB) penetrating enzyme replacement therapy currently in Phase III (NCT04573023) evaluation as a treatment of Hunter syndrome (also known as mucopolysaccharidosis type II or MPS II).
Approved and launched in Japan in 2021 as Izcargo (INN: pabinafusp alfa), JCR and Italfarmaco seek to obtain regulatory approval for JR-141 from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), and the Brazilian Health Regulatory Agency (ANVISA).
Following regulatory approvals by the FDA, EMA, MHRA and ANVISA, Italfarmaco will commercialize and distribute the product in the licensed territories, while JCR will retain responsibility for manufacturing JR-141 upon marketing authorizations. JCR will receive upfront, milestone and royalty payments, in addition to revenues associated with product supply.
JR-141 is a next-generation recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in individuals with Hunter syndrome. JR-141 was developed using J-Brain Cargo, JCR’s proprietary BBB-penetration technology. J-Brain Cargo is engineered to transport the therapeutic enzyme across the BBB to directly reach the brain and address both the somatic and neuronopathic symptoms of the disease, which can lead to progressive cognitive decline.
This agreement expands the relationship between JCR and Italfarmaco, which entered into an exclusive license agreement in December 2025 for the commercialization of givinostat in Japan, a treatment for Duchenne muscular dystrophy, together with a strategic partnership agreement for rare disease therapies.
“We are pleased to enter into this strategic agreement with Italfarmaco and collaborate with an ideal global commercialization partner as we work toward our goal of delivering JR-141 to people living with Hunter syndrome around the world. Italfarmaco has expertise in developing and commercializing therapies worldwide, and we look forward to working with Italfarmaco to make JR141 available to patients outside Japan as soon as possible. This agreement extends our commitment to the partnership with Italfarmaco, developing therapies for rare and genetic diseases for patients around the globe.” – Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR Pharmaceuticals.
“This agreement marks an important milestone in the relationship between Italfarmaco and JCR Pharmaceuticals and reflects a shared commitment to advancing innovative therapies for people living with rare and genetic diseases. By bringing together JCR’s pioneering expertise in blood-brain barrier technologies and Italfarmaco’s global capabilities in rare disease development and commercialization, we are creating a strong foundation to accelerate innovation for underserved patient communities.” – Francesco Di Marco, Chief Executive Officer of Italfarmaco Group.
“Building on the success of our existing collaboration in Duchenne muscular dystrophy, this agreement further strengthens our partnership and reinforces our shared ambition to be a leading force in the rare and genetic disease field. Alongside our trusted partner, we look forward to advancing our mission of delivering transformative treatments to the patients and families who need them most.” – Antonio Nardi, Vice President and Head of Business & Portfolio Development of Italfarmaco.
