Italfarmaco, JCR Sign JR-141 Licensing Deal for Hunter Syndrome

Italfarmaco and JCR Pharmaceuticals have entered into an exclusive licensing agreement covering the development and potential commercialization of JR-141 (pabinafusp alfa) in the United States, Europe, and Latin America, subject to regulatory approval. JR-141 is an investigational enzyme replacement therapy designed to cross the blood-brain barrier (BBB) and is being evaluated for the treatment of Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II).

Under the agreement, Italfarmaco will be responsible for the commercialization and distribution of JR-141 in the licensed territories following potential approvals from the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), U.K. Medicines and Healthcare products Regulatory Agency (MHRA), and Brazil’s National Health Surveillance Agency (ANVISA). JCR will retain manufacturing responsibilities and will receive upfront, milestone, royalty, and product supply-related payments under the agreement.

JR-141 Enters Global Phase III Development

JR-141 is currently being evaluated in the global Phase III study (NCT04573023) in patients with Hunter syndrome. The program is intended to further evaluate the therapy’s safety and efficacy, including its potential to address neurological manifestations of MPS II that remain inadequately treated by conventional enzyme replacement therapies.

JR-141 has already received regulatory approval in Japan, where it is marketed under the brand name IZCARGO™, and was also approved in the United Arab Emirates in 2026. IZCARGO™ is the first approved therapy developed using JCR’s proprietary platform. The new licensing agreement is intended to support development and potential regulatory submissions in additional major international markets.

The agreement builds on an existing relationship between Italfarmaco and JCR established in 2025 for the commercialization of givinostat in Japan for Duchenne muscular dystrophy. The companies are expanding this collaboration into another rare disease therapy.

Hunter Syndrome and the Unmet CNS Need

Hunter syndrome is a rare X-linked recessive lysosomal storage disorder caused by deficiency of the enzyme iduronate-2-sulfatase (IDS). The enzyme normally participates in the breakdown of glycosaminoglycans. Its deficiency results in progressive accumulation of these substances in tissues and organs and can lead to both somatic and neurological manifestations.

Current treatment for Hunter syndrome includes enzyme replacement therapy. However, conventional enzyme replacement therapies have limited ability to cross the blood-brain barrier, leaving an important unmet need for patients with neuronopathic disease, in which central nervous system manifestations can contribute substantially to disease burden.

According to the companies, approximately 2,000 to 3,000 people worldwide are affected by Hunter syndrome.

JR-141 Uses J-Brain Cargo Technology

JR-141 is a recombinant fusion protein consisting of iduronate-2-sulfatase linked to an antibody targeting the human transferrin receptor. It incorporates JCR’s proprietary J-Brain Cargo® technology, which is designed to facilitate transport of therapeutic proteins across the blood-brain barrier through transferrin receptor-mediated transcytosis.

The approach is intended to allow enzyme replacement therapy to reach both peripheral tissues and the central nervous system. This distinguishes JR-141 from conventional enzyme replacement therapies that have limited BBB penetration.

Preclinical studies reported by JCR demonstrated BBB penetration, neuronal uptake, and reductions in substrate accumulation in the central nervous system and peripheral organs. Clinical studies have also reported reductions in cerebrospinal fluid heparan sulfate, a biomarker associated with CNS disease, together with reported effects on CNS manifestations.

 Safety Profile from Approved Use

JR-141 is approved for the treatment of Hunter syndrome in Japan and the United Arab Emirates. The product is contraindicated in patients with a history of anaphylactic shock to its components. Serious anaphylaxis, shock, and infusion-associated reactions may occur, requiring monitoring during and after administration and availability of appropriate emergency measures.

Reported infusion-associated reactions include headache, chills, syncope, fatigue, dizziness, pyrexia, rash, erythema, and urticaria. Management may involve slowing or temporarily interrupting the infusion and providing supportive treatment. Premedication may be considered for subsequent infusions when appropriate. Pyrexia and urticaria were among the most commonly reported adverse reactions with IZCARGO™.

Safety information, prescribing information, indications, contraindications, and adverse reaction profiles may differ between jurisdictions.

What the Licensing Agreement Means

The development program is particularly focused on the potential of BBB-penetrating enzyme replacement therapy to address both peripheral and neurological manifestations of Hunter syndrome. The ongoing Phase III program will provide additional clinical evidence needed to support potential regulatory submissions outside the markets where JR-141 is already approved.

For patients with neuronopathic MPS II, the ability to deliver enzyme replacement therapy to the central nervous system represents an important area of therapeutic development. The ongoing international development of JR-141 will determine whether this approach can translate into clinically meaningful benefits across the broader Hunter syndrome population.

Reference

Italfarmaco and JCR Pharmaceuticals Enter into Exclusive License Agreement for the Development and Potential Commercialization of Novel Investigational Therapy for Hunter Syndrome in the United States, Europe, and Latin America, Italfarmaco, 02 October 2026