Takeda is poised to commercialize next generation Hunter's disease therapy through collaboration with JCR Pharmaceuticals

Takeda Pharmaceutical Company Limited | October 01, 2021

Takeda Pharmaceutical Company Limited and JCR Pharmaceuticals Co., Ltd. today announce a geographically focused exclusive collaboration and license agreement to commercialize JR-141, a recombinant fusion protein under development of a next-generation antibody against the human transferrin receptor and the enzyme iduronate- 2-sulfatase (IDS), used to treat Hunter's disease (also called mucopolysaccharidosis type II or MPS II). Hunter's disease is caused by the lack of IDS and manifests itself in different forms. Administered with J-Brain Cargo®, JCR's proprietary blood-brain barrier (BBB) ​​technology, the JR-141 was designed to deliver the therapeutic enzyme across the blood-brain barrier directly to the brain and somatic and address neuronopathic manifestations of the disease,

Under the exclusive collaboration and license agreement, Takeda will exclusively market the JR-141 outside of the United States, including Canada, Europe, and other regions (excluding Japan and certain other Asia Pacific countries). JCR will receive an upfront payment for the ex-US license and is eligible to receive additional development measures and commercial milestones as well as multi-tier royalties on potential product sales. Both companies will work together to provide this form of therapy to patients as soon as possible after the completion of the global Phase III program run by JCR.

Takeda has an option under a separate option agreement that allows Takeda to acquire an exclusive license to commercialize JR-141 in the United States upon completion of the Phase III program.

Takeda is committed to continuously improving the therapy it offers to treat Hunter's disease. JR-141 introduces a new way of delivering proteins by crossing the blood-brain barrier. This will overcome our current challenges in treating the underlying neuronopathic manifestations of Hunter's disease and help maintain or improve cognitive function in these patients, We will work closely with JCR to apply our expertise in enzyme replacement therapies with the hope of delivering this potentially transformative form of therapy to patients as soon as possible.

- Dan Curran, MD, Head, Rare Genetics & Hematology Therapeutic Area Unit at Takeda.

JR-141 met its primary endpoint in an open-label Phase II / III study in Japanand was able to show a significant reduction in heparan sulfate (HS) concentrations in the CSF. HS is a biomarker for assessing the effectiveness of the drug in reducing the disease-causing substrate in the central nervous system. This proof could be provided in all patients for whom measurements were available after 52 weeks of treatment. Somatic disease control was retained in patients switched from standard enzyme replacement therapy (ERT). The study also showed an improvement in somatic symptoms in participants who had not received standard ERT prior to the start of the study. In addition, an assessment of neurocognitive development in 21 of 25 patients at one year showed the maintenance or improvement of age-appropriate function.1

About JR-141
JR-141 is a recombinant fusion protein of an antibody against the human transferrin receptor and the enzyme iduronate-2-sulfatase. This enzyme is absent or defective in patients with Hunter's disease. It is expected to be effective against neuronopathic manifestations of the disease by crossing the blood-brain barrier (BBB) ​​through transferrin receptor-mediated transcytosis using J-Brain Cargo®, JCR's proprietary BBB technology. Uptake into the cells is mediated by the transferrin receptor and the mannose-6-phosphate receptor. JCR has driven development activities by allowing the company to maintain the necessary evidence from the molecular design phase through to the non-clinical and clinical study phases. In addition, JCR has confirmed that using J-Brain Cargo® technology, enzymes are absorbed into different brain tissues. A decrease in substrate accumulation could also be confirmed in an animal model of Hunter's disease. 2,3,4 In several clinical studies with JR-141, JCR was able to show a reduction in CSF heparan sulfate concentrations. This is a biomarker for assessing the effectiveness of drugs by reducing the disease-causing substrate in the central nervous system. This is done in accordance with the results obtained in non-clinical studies. JCR also produced clinical results that could show the positive effects of JR-141 on neurocognition. 5,6,7,8 JR-141 is approved by the Ministry of Health, Labor and Social Affairs and has been marketed under the brand name "IZCARGO® IV Infusion 10mg" since May 2021.

About Hunter's Disease
Hunter's disease is a severely debilitating, rare lysosomal disease caused by insufficient activity of the enzyme iduronate-2-sulfatase. This enzyme has the task of breaking down substances known as glycosaminoglycans (GAG) in the body. 9 The absence of this enzyme (GAG) can trigger a number of symptoms. 9,10 Around two in three people with Hunter's disease also experience progressive loss of cognitive function. 11 Hunter's disease affects 1 in 162,000 live births and almost exclusively male patients. 12th

About Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited is a global, values-based, leading group with a focus on research and development in the biopharmaceutical sector headquartered in Japan. His pursuit of life-changing treatments is shaped by his dedication to patients, people, and the planet. Takeda's research and development efforts focus on four therapeutic areas: Oncology, Rare Genetic Diseases and Hematology, Neuroscience and Gastroenterology (GI). We also invest parts of our research and development budget specifically in therapies with plasma derivatives and vaccines. We focus on developing highly innovative drugs that help improve people's quality of life. To this end, we are opening up new treatment options and using our improved, synergistic research area and our combined skills to create a robust pipeline based on different modalities. Our employees are committed to improving the quality of life for patients and work with our healthcare partners in around 80 countries and regions.

About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. is a global specialty pharmaceutical company that is redefining expectations and expanding opportunities for those with rare hereditary diseases around the world. We continue to build on our 46 year tradition in Japan as we expand our presence in the United States, Europe and Latin America. We improve the lives of patients by applying our scientific expertise and unique technologies to research, develop and deliver next generation therapies. Our approved products in Japan include therapies for the treatment of stunted growth, Fabry disease, acute graft versus host disease and renal anemia. Our test products, which are being developed around the world, aim, among other things, at the treatment of rare diseases, including MPS I (Hurler's disease, Hurler-Scheie and Scheie syndrome), MPS II (Hunter's disease) and Pompe disease. JCR strives to expand the possibilities for patients as the company accelerates medical advancement on a global basis. Our core values ​​- reliability, trust and perseverance - benefit all those involved, including employees, partners and patients. Together we can reach greater heights. For more information, see Our core values ​​- reliability, trust and perseverance - benefit all those involved, including employees, partners and patients. Together we can reach greater heights. For more information, see Our core values ​​- reliability, trust and perseverance - benefit all those involved, including employees, partners and patients. Together we reach greater heights.


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