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.
Prnewswire | August 28, 2020
Fixx Pharmaceuticals Inc. is pleased to announce the launch of its biotechnology business operations in Las Vegas, Nevada and San Francisco, California. The company is focused on developing a cure for Hemophilia through the use of CRISPR gene editing. There are currently 20,000 people in the U.S. who suffer from hemophilia and 400,000 globally. "We are looking to commercialize a product as soon as possible," says Jesse John Francis Clark, founder of Fixx. "We are going to develop a product that is more cost-effective for payers. We're looking to keep it under $2 million per patient," he said, saying that the overall cost of care of hemophilia patients will decrease. "If we are successful in developing a commercialized product, we are looking at upwards of $40 billion in sales from the U.S. market alone. I think our investors will be happy with that," says Clark.
Invitae | September 07, 2021
Invitae , a leading medical genetics company, today announced it has entered into a definitive agreement to acquire Ciitizen, a patient-centric consumer health tech company. Backed by a16z, Section 32 and Verily, Ciitizen is working to build a global platform to help patients collect, organize, store and share their medical records digitally. The acquisition would enhance Invitae's platform by providing patients an easy-to-use, centralized hub for their genomic and clinical information, which together comprise a powerful dataset with the potential to drive research and improve healthcare decision making.
"Invitae is relentlessly pursuing a day when genetics is used routinely to improve healthcare for billions of people around the globe. We believe combining Ciitizen's state-of-the-art, transparent and patient-consented platform with our technologies and services will accelerate our evolution into a genome information company that informs healthcare throughout one's life. This would give us the ability to engage patients, create an innovative patient-centered data ecosystem and deliver better outcomes for everyone. Invitae views the acquisition of Ciitizen as an important part of its strategy to be the industry leader across the genetic testing, software and health information technology spaces," said Sean George, Ph.D., co-founder and CEO of Invitae.
"Ciitizen has already helped patients take ownership of their medical records to optimize therapy for themselves and contribute their data towards a variety of goals, including research on rare diseases," said Anil Sethi, CEO and founder of Ciitizen. "Patients leave a breadcrumb trail of records behind them, and we believe access to this real-world data is key to personalized therapeutics in the future. The combination of lifelong health history together with Invitae's world-class genetic and data services would enable a digital ecosystem of personalized services for each patient. Democratization of patient-consented data is expected to improve outcomes by providing access to third-parties for whom the patient has granted consent. Unlike many health data companies, we operate on the premise that it's the patient's data, not ours."
Invitae is working to aggregate results from the world's genetic tests into a single, easy-to-use service that makes genetic information accessible to all who can benefit from it - always with patient consent. Over the past five years, Invitae has completed 13 acquisitions that have expanded its testing menu, lowered the cost of its tests, improved its customer experience and enhanced its ability to attract partners. Invitae raised more than $1.4 billion since the beginning of 2021, in part to identify and acquire key assets that bring the company closer to delivering on its vision.
The addition of Ciitizen is expected to empower Invitae to more quickly extend its platform, adding a complementary patient service that streamlines the process of collecting and organizing health information from any source and uses machine learning to transform the unstructured (and largely untapped) health information to enable better care decisions, find clinical trials, and power research using real-world evidence. In addition, as the use of Invitae testing and the Ciitizen service expands, third-party access to consented genomic and other clinical data can be democratized to power a variety of applications that can enhance Invitae's business and growth strategy.
Invitae Corporation is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices.
Ciitizen is a patient-centric consumer health technology platform that enables patients to collect, store and share their medical records in a single location. Ciitizen developed proprietary machine learning and AI technologies to abstract critical information from unstructured health records to advance the understanding of diseases, match patients with potentially life-saving clinical trials, and enable better care decisions. Ciitizen also provides valuable big data analytics and data quality technologies to the network of Health Information Exchanges (HIEs) to produce research-ready, regulatory-grade data within minutes, instead of hours or days, and ensure HIEs can comply with the Information Blocking regulations.