Adnkronos (Adnkronos) – Italfarmaco Spa and JCR Pharmaceuticals today announced an exclusive licensing agreement for the development and commercialization of JR-141 in the United States, Europe, and Latin America (the “Licensed Territories”), subject to regulatory approval. JR-141, according to a statement, is a blood-brain barrier (BBB)-crossing enzyme replacement therapy currently in Phase 3 (NCT04573023) evaluation as a treatment for Hunter syndrome (also known as mucopolysaccharidosis type II or MPS II).
JCR and Italfarmaco intend to obtain regulatory approval for JR-141 from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the UK's Medicines and Healthcare Products Regulatory Agency (MHARA), and the Brazilian Health Regulatory Agency (ANVISA). The molecule is already approved and marketed in Japan since 2021 as pabinafusp alfa.
Following regulatory approvals from the FDA, EMA, MHRA, and ANVISA, Italfarmaco will market and distribute the drug in the authorized territories, while JCR will retain responsibility for the production of JR-141 upon approval. JCR will receive upfront payments, milestone payments, and royalties, in addition to revenues associated with the supply of the drug.
JR-141 – the note details – is a next-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 correctly in individuals affected by Hunter syndrome.
JR-141 was developed using J-Brain Cargo, JCR's proprietary BBB penetration technology. J-Brain Cargo is designed to deliver the therapeutic enzyme through 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 licensing agreement in December 2025 for the commercialization of Givinostat in Japan, a treatment for Duchenne muscular dystrophy, as well as a strategic partnership agreement for rare disease therapies.
“We are pleased to enter into this strategic agreement with Italfarmaco,” said Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR, “and to collaborate with an ideal global commercialization partner as we work toward our goal of making JR-141 available to people with Hunter syndrome worldwide. Italfarmaco has expertise in developing and commercializing therapies globally, and we look forward to collaborating with them to make JR-141 available to patients outside of Japan as soon as possible. This agreement strengthens our commitment to our partnership with Italfarmaco, developing therapies for rare and genetic diseases for patients worldwide.”
"This collaboration," said Francesco Di Marco, Chief Executive Officer of Italfarmaco Group, "marks an important milestone in the relationship between Italfarmaco and JCR Pharmaceuticals and reflects a shared commitment to developing innovative therapies for people affected by rare and genetic diseases. By combining JCR's pioneering expertise in blood-brain barrier technologies and Italfarmaco's global expertise in the development and commercialization of therapies for rare diseases, we are creating a solid foundation to accelerate innovation for the benefit of the patient communities who need it most." "Building on the success of the collaboration between Italfarmaco and JCR in Duchenne muscular dystrophy, this agreement further strengthens our partnership and reinforces our shared ambition to be a leading company in the field of rare and genetic diseases," said Antonio Nardi, Vice President and Head of Business & Portfolio Development of Italfarmaco. "Together with our partner, we will continue our commitment to making innovative treatments available to the patients and families who need them most."
The upfront payment under this agreement, the statement concludes, has been included in JCR's consolidated earnings guidance for the fiscal year ending March 31, 2027. JCR Pharmaceuticals has developed a proprietary technology capable of crossing the blood-brain barrier (BBB), J-Brain Cargo, to deliver biopharmaceuticals into the central nervous system (CNS). The first drug developed based on this technology is pabinafusp alfa, approved in Japan for the treatment of Hunter syndrome, a lysosomal storage disease. With J-Brain Cargo, JCR is committed to addressing the unsolved clinical challenges of these diseases by delivering the enzyme both into the body and the brain.
Hunter syndrome (mucopolysaccharidosis type II or MPS II) is an X-linked recessive lysosomal storage disease caused by a deficiency of iduronate-2-sulfatase, an enzyme that breaks down complex carbohydrates called glycosaminoglycans (GAGs, also known as mucopolysaccharides) in the body. Hunter syndrome, which affects approximately 2.000–3.000 individuals worldwide (according to research by JCR), causes a wide range of somatic and neurological symptoms. The current standard of care for Hunter syndrome is enzyme replacement therapy, which does not address symptoms related to the central nervous system impairment in this lysosomal disorder.
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