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
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.
UConn | November 20, 2020
Consistent assembling has just begun to override customary group handling in the domain of tablet creation. The following region due for an assembling update? Nanoparticle drug conveyance frameworks, one startup thinks—and it's currently prepared to pitch its foundation to drugmakers.
The University of Connecticut as of late authorized its ceaseless nanoparticle fabricating innovation to DIANT Pharma, a startup helped to establish by the stage's designers Antonio Costa, Ph.D., and Diane Burgess, Ph.D.
The high-throughput framework can possibly spare tidy up room space, bring down the quantity of preparing steps and diminish the quantity of bunches created every year. Since the stage is persistent, it should empower makers to hop in the event that anything turns out badly, redirecting or eliminating just a little bit of the item instead of rejecting a whole group, Costa, CEO at DIANT and partner research teacher at UConn, said in a meeting.
Biognosys | November 10, 2021
Biognosys, a leader in proteomics solutions for drug discovery and development, announces the launch of its next-generation blood biomarker discovery solution, which will be offered as a contract research service. The service is designed to help researchers decipher the complexity of the blood proteome and unlock unbiased discoveries for pharma and diagnostics development and precision medicine.
The launch will feature a series of events and resources that detail the science and technology behind the new solution, as well as its applications
Launch webinar on November 9, entitled: “Plasma Proteomics: The Next Frontier of Biomarker Discovery in the Precision Medicine Era.”
Presentation and exhibition at the Society for Immunotherapy of Cancer (SITC) Annual Meeting on November 12-14 in Washington, D.C.
The new service offers unprecedented depth, quantitative precision, and throughput for discovery research. The solution will search the complete plasma proteome to quantify up to 3,000 of the most relevant proteins and measure tens of thousands of peptide-level datapoints to provide a deep understanding of key disease pathways.
The solution can be applied across all biological species and to any other biofluid, such as cerebrospinal fluid or urine. In addition to the biofluid biomarker discovery service, an improved tissue biomarker discovery service is available, offering an industry-leading
depth of 10,000 proteins.
Both solutions are based on Biognosys’ proprietary, patented Hyper Reaction Monitoring (HRM™) technology and its optimized Liquid Chromatography - Mass Spectrometry (LC-MS) workflow. The clinical transferability of this technology allows quick transition from early stage discovery to specific, targeted panels that can be applied in a regulated setting.
Kanna Palaniappan, PhD, Staff Scientist at Freenome, a company that develops blood tests for early cancer detection, powered by a multi-omics platform, testifies: “We have used Biognosys’ services for a number of biomarker discovery studies focused on improving cancer detection, taking advantage of their constantly improving platform, and their ability to run large-scale studies while maintaining high data quality. We have had a valuable and productive relationship and we look forward to future collaborations.”
“We are thrilled to offer customers our innovative, next-generation biomarker discovery solution and enable them to gain biological insights beyond what pre-defined panels can provide. By diving deep into the plasma proteome with an unbiased view, we profile the proteins that are of highest relevance to our customer’s disease areas of focus. This is what we call true discovery.”
Kristina Beeler, PhD, Chief Business Officer of Biognosys
Lukas Reiter, PhD, Chief Technology Officer of Biognosys states: “Mass spectrometry-based proteomics is the ideal tool to explore the blood proteome in an unbiased manner. Our continuous R&D improvements have enabled us to reach an unprecedented depth to quantify the very low abundant protein ranges that are of highest biological significance, while ensuring high scalability for clinical research.”
Biognosys is a leader in next-generation proteomics, dedicated to transforming life science by inventing and developing cutting-edge proteomics technology and solutions and making them widely available for pharmaceutical and biotechnology researchers and proteomics experts. The Company offers a versatile portfolio of proprietary proteomics services, software, and kits that provide a multi-dimensional view of protein expression, function, and structure in all biological species and sample types. Biognosys’ unique, patented technologies utilize high-resolution mass spectrometry to quantify thousands of proteins across thousands of samples with industry-leading precision, depth, and throughput. Through advanced data analytics, Biognosys translates data into actionable insights for R&D and clinical research.