Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration with JCR Pharmaceuticals
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) and JCR Pharmaceuticals Co., Ltd. (TSE:4552) (“JCR”) announced today a geographically-focused exclusive collaboration and license agreement to commercialize JR-141 (INN: pabinafusp alfa), an investigational, next-generation recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase (IDS) enzyme for the treatment of Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II). Hunter syndrome is caused by a deficiency of IDS and manifests in different forms. JR-141, applied with J-Brain Cargo®, JCR’s proprietary blood-brain barrier (BBB) technology, is engineered to transport the therapeutic enzyme across the BBB to directly reach the brain and address both the somatic and neuronopathic manifestations of the disease, which can lead to progressive cognitive decline.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210930005353/en/
Under the terms of the exclusive collaboration and license agreement, Takeda will exclusively commercialize 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 such ex-U.S. license, and is eligible to receive additional development and commercial milestones as well as tiered royalties on potential sales. The two companies will collaborate to bring this therapy to patients as quickly as possible upon completion of the global Phase 3 program, which will be conducted by JCR.
Takeda receives an option under a separate option agreement, which allows Takeda to acquire an exclusive license to commercialize JR-141 in the U.S. upon completion of the Phase 3 program.
“Takeda is committed to continuously improving the way Hunter syndrome is treated. JR-141 introduces a new way to deliver proteins across the blood-brain barrier, overcoming our current challenges to treat the underlying neuronopathic manifestations of Hunter syndrome and help maintain or improve cognitive function in these patients,” said Dan Curran, M.D., Head, Rare Genetics & Hematology Therapeutic Area Unit at Takeda. “We will work closely with JCR to apply our expertise in enzyme replacement therapies with the hope of bringing this potentially transformative therapy to patients as quickly as possible.”
“JCR is pleased to have reached an agreement with Takeda who is well placed to achieve our common goal of maximizing the impact of JR-141,” said Shin Ashida, President, Chairman of JCR. “Our mission is to provide transformative treatment options as soon as possible to patients with lysosomal storage disorders (LSDs) with central nervous system symptoms, such as Hunter syndrome. JR-141 is the first-ever approved biopharmaceutical in Japan that penetrates the blood-brain barrier. I expect that we will be able to achieve this mission through our partnership with Takeda to deliver a new treatment option to Hunter patients around the world as swiftly as possible.”
JR-141 met its primary endpoint in an open-label Phase 2/3 clinical trial in Japan demonstrating significant reductions in heparan sulfate (HS) in the cerebrospinal fluid, a biomarker for assessing the drug’s effectiveness in reducing disease-causing substrate in the central nervous system, in all subjects for whom measurements were available after 52 weeks of treatment. Somatic disease control was maintained in patients who switched from standard enzyme replacement therapy (ERT). The study also demonstrated an improvement in somatic symptoms in participants who had not previously received standard ERT prior to the start of the trial. Additionally, a neurocognitive development assessment demonstrated maintenance or improvement of age-equivalent function in 21 of the 25 patients at one year. There were no reports of serious treatment-related adverse events in the trial.1
JR-141 is a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in subjects with Hunter syndrome. It is expected to be effective against the neuronopathic manifestations of the disease by crossing the BBB through transferrin receptor mediated transcytosis using J-Brain Cargo®, JCR’s proprietary BBB technology. Uptake into cells is mediated through the transferrin receptor and mannose-6-phosphate receptor. JCR has advanced development activities by establishing the necessary evidence from the molecular design stage to the nonclinical and clinical trial phases. In non-clinical trials, JCR has confirmed both high affinity binding of JR-141 to transferrin receptors, and passage across the BBB into neuronal cells as evidenced by electron microscopy.
In addition, JCR has confirmed that using J-Brain Cargo® technology, enzymes are taken up into various brain tissues. A decrease in substrate accumulation has also been confirmed in an animal model of Hunter syndrome.2,3,4 In several clinical trials with JR-141, JCR obtained evidence of reduction of heparan sulfate concentrations in the CSF, a biomarker for assessing the drug’s effectiveness in reducing disease-causing substrate in the central nervous system, consistent with the results obtained from non-clinical studies. JCR also obtained clinical results that demonstrate positive effects of JR-141 on neurocognition.5,6,7,8
JR-141 was approved by the Ministry of Health, Labour and Welfare and marketed since May 2021 under the brand name “IZCARGO® I.V. Infusion 10mg.”
About Hunter Syndrome
Hunter syndrome is a severely debilitating, rare lysosomal disease caused by a deficiency of iduronate-2-sulfatase, an enzyme that is needed to break down substances in the body called glycosaminoglycans (GAGs).9 Without this enzyme, GAGs can build up, causing a range of disease-related signs and symptoms.9,10 Roughly two of every three patients with Hunter syndrome are also affected with progressive cognitive decline.11 Hunter syndrome affects 1 in 162,000 total live births, and almost exclusively males.12
About Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is redefining expectations and expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 46-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives 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 growth disorder, Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II (Hunter syndrome), Pompe disease, and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. Together we soar. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/.
Important Notice From Takeda
For the purposes of this notice, “press release” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.
The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.
This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/sec-filings/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results.
JCR Pharmaceuticals Cautionary Statement Regarding Forward-Looking Statements
This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions.
This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue.
Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.
1. Okuyama T, Eto Y, Sakai N, et al. A phase 2/3 trial of pabinafusp alfa, IDS fused with anti-human transferrin receptor antibody, targeting neurodegeneration in MPS-II. Molecular Therapy. 2021;29(2):671-679.
2. Sonoda, et al. A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II. Mol Ther. 2018; 26(5): 1366-74.
3. Morimoto, et al. Clearance of heparin sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice. Mol. Ther. 2021; https://doi.org/10.1016/j.ymthe.2021.01.027.
4. Yamamoto et al. Nonclinical Safety evaluation of pabinafusp alfa, an anti-human transferrin receptor antibody and iduronate-2-sulfatase fusion protein, for the treatment of neuronopathic mucopolysaccharidosis type II. Mol Genet Metab Rep. 2021; https://doi.org/10.1016/j.ymgmr.2021.100758.
5. Okuyama, et al. Iduronate-2-sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2020; 27(2): 456-464.
6. Okuyama, et al. A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II. Mol Ther. 2020; 29(2): 671-679.
7. Giugliani, et al. Iduronate-2-sulfatase fused with anti-human transferrin receptor antibody, pabinafusp alfa, for treatment of neuronopathic and non-neuronopathic mucopolysaccharidosis II: Report of a phase 2 trial in Brazil. Mol Ther. 2021; https://doi.org/10.1016/j.ymthe.2021.03.019.
8. Giugliani, et al. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II; an Integrated Analysis of Preclinical and Clinical Data. Preprints 2021; 2021090192.
9. Wraith JE, et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008; 167(3):267-77.
10. Martin R. Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome). PEDIATRICS. Volume 121, Number 2, February 2008.
11. Young I. A clinical and genetic study of Hunter's syndrome. 2 Differences between the mild and severe forms.
12. Meikle PJ, et al. Prevalence of Lysosomal Storage Disorders. JAMA. 1999; 281(3):249-54.
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
+81 (0) 3-3278-2037
Media outside Japan
+1 (617) 347-7726
Corporate Communications (PR/IR)
+81 (0) 797-32-1995
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Janssen Receives Positive CHMP Opinion for RYBREVANT® (amivantamab) for the Treatment of Patients with Advanced Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations After Failure of Platinum-Based Therapy15.10.2021 19:30:00 EEST | Press release
FOR EU TRADE AND MEDICAL MEDIA ONLY. NOT TO BE DISTRIBUTED TO UK AND BENELUX BASED MEDIA The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product amivantamab, intended for the treatment of adult patients with advanced NSCLC with activating epidermal growth factor receptor (EGFR) exon 20 insertion mutations, after failure of platinum-based therapy.3 If approved, amivantamab will be the first treatment in the European Union specifically targeting EGFR exon 20 insertion mutations for advanced NSCLC.1 “This group of patients often face a poor prognosis as there are currently no targeted therapy options for this specific mutation nor does it typically respond to existing therapies used to treat more common EGFR mutations,” 1,4,5 commented Catherine Taylor, M.D., Vice President, Medical Affairs
Leading Researchers Present 24-Plex Digital PCR Assay and Novel Multiplex Applications Uniquely Enabled by Stilla’s 6-Color naica® system15.10.2021 16:08:00 EEST | Press release
Stilla Technologies, the multiplex digital PCR company, joined researchers from SAGA Diagnostics in presenting a 24-plex digital PCR assay generated on Stilla’s six-color naica® system. To mark the platform’s global commercial launch at the American Society of Human Genetics (ASHG) 2021 Virtual Meeting, Stilla’s Cofounder and Chief Technology Officer Rémi Dangla, PhD, and researchers from SAGA Diagnostics, Fred Hutchinson Cancer Research Center, and University of Athens highlighted the new data during a discussion on how highly sensitive, multiplex digital PCR technology is transforming complex genomic data into actionable insights across a breadth of research and clinical applications. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211015005140/en/ Stilla’s six-color naica® system, the industry’s first six-color digital PCR system, answers market needs for higher performance and multiplexing technology. Learn more here: www
Boston Metal Wins S&P Global Platts Global Metals Award15.10.2021 16:04:00 EEST | Press release
Boston Metal won the New Technology for the Metals and Mining Industry award at the 2021 S&P Global Platts Global Metals Awards. Boston Metal is commercializing ground-breaking technology to decarbonize primary steelmaking. The company’s technology combines patented innovations with best practices from the aluminum and steel industries to deliver a revolutionary solution to the carbon emissions challenge facing the steel value chain. Powered by renewable electricity, molten oxide electrolysis converts iron ore into liquid metal and oxygen. The modular, scalable platform produces no CO2 emissions, is highly energy efficient, and works with a wide range of iron ore grades. “We congratulate all the winners and finalists for persevering through unique challenges and continuing to drive performance while embracing change,” said Saugata Saha, president of S&P Global Platts. Dave Ernsberger, Global Head of Pricing and Market Insight, S&P Global Platts, said: “Not surprisingly, but certainly e
The Ministry of Justice makes public the evidence supporting of the Republic of Kazakhstan’s case against Anatolie and Gabriel Stati15.10.2021 14:46:00 EEST | Press release
The Ministry of Justice has launched a new website to spotlight evidence gathered by the Republic of Kazakhstan regarding the fraud committed by Anatolie and Gabriel Stati: www.kzarbitration.com. The purpose of the website is to present the facts of the ongoing and complex legal case and provide easy access to the corresponding evidence. This public repository will increase transparency around the case and dispel the disinformation and lies perpetrated by these individuals against the Republic of Kazakhstan and its people. The evidence - in the form of court rulings, expert opinions, and other publicly available documents - confirms that the Statis have been engaged in a complex and multi-faceted fraud since at least 2006. The website explains how Anatolie and Gabriel Stati defrauded international investors of their money, falsified financial statements, fraudulently obtained audit opinions and pursued an international arbitration against the Republic of Kazakhstan to recoup from the S
TP Awards Schlumberger Sakarya Offshore Gas Field Phase-1 Contract15.10.2021 14:00:00 EEST | Press release
Schlumberger announced today a significant contract award by Turkish Petroleum (TP) for the engineering, procurement, construction and installation (EPCI) of end-to-end production solutions for the Sakarya gas field, Turkey’s largest gas reserve. The contract is awarded to Schlumberger and Subsea 7, as part of a consortium. The integrated project scope will cover subsurface solutions to onshore production, including well completions, subsea production systems (SPS), subsea umbilicals, risers, flowlines (SURF), and an early production facility (EPF). Schlumberger will deliver the well completions scope and the design, construction, and commissioning of the early production facility capable of handling up to 350 MMscfd of gas. The SPS and SURF scope will be delivered by OneSubsea®, the subsea technologies, production, and processing systems division of Schlumberger, and Subsea 7. “Schlumberger is uniquely positioned to integrate solutions from the subsurface to the processing facility, a
Pony.ai to Start Driverless Tests on Public Roads in Beijing15.10.2021 10:00:00 EEST | Press release
Silicon Valley-based leading autonomous driving company Pony.ai, today announced that it has been authorized to conduct driverless tests on public roads in China’s capital city, Beijing. The company is one of the two autonomous software companies to receive this important Beijing permit. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211015005137/en/ Driverless tests on public roads in Beijing (Photo: Business Wire) Prior to receiving this driverless test permit in Beijing, Pony.ai received a permit to conduct tests on certain sections of Beijing highways in July 2021. The company was among the first group to obtain this permit. In Beijing, Pony.ai is now authorized to conduct driverless testing in an area of around 20 square kilometers in a pilot zone for autonomous driving vehicles. The area covers major subway stations, residential areas, and tech parks, which allows Pony.ai to test its autonomous technology in the most c
Announcing the 2021 .eu Web Awards Winners!15.10.2021 10:00:00 EEST | Press release
Last night the 2021 .eu Web Awards ended in epic fashion at the Teatro Antico in Taormina, Italy. Hosted by EURid, the .eu Web Awards are a competition for the best .eu, .ею and .ευ websites. After last year’s virtual only event, we were pleased to once again celebrate the finalists and winners in person. View the hybrid event here. Hosted by Sally Bundock, esteemed journalist for BBC world news, the gala night brought us sincere joy reflected by the winners, combined with amazing performances by featured artists like THePETEBOX, Women Orchestra, MM Dance Company, Compagnia Joculares and the GAP’s Orchestra. The magical event ended with fireworks. “Being able to see real faces and raw emotion conveyed by the guests again made the night unforgettable” noted Giovanni Seppia, EURid’s External Relations Manager. “This is what the Web Awards is all about, bringing together people from various industries and backgrounds and honouring them for having chosen a .eu for their online presence.” P
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.Visit our pressroom