Business Wire

Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration with JCR Pharmaceuticals

Share

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

About JR-141

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.

Forward-Looking Statements

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.

Contact information

Takeda:
Japanese Media
Ryoko Matsumoto
ryoko.matsumoto@takeda.com
+81 (0) 3-3278-2037

Media outside Japan
Chris Stamm
chris.stamm@takeda.com
+1 (617) 347-7726

JCR Media:
Corporate Communications (PR/IR)
Takashi Kitamura
Yuri Koyama
ir-info@jcrpharm.co.jp
+81 (0) 797-32-1995

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

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

Total Telecom: Leveraging Technology Innovation for Building Energy Efficiency 5G Networks20.10.2021 09:00:00 CEST | Press release

Total Telecom attended Huawei’s Better World Summit (BWS) for Green ICT for Green development in Dubai in United Arab Emirates (UAE), found out that the ICT industry, which is a backbone of the modern-day global economy, must adopt green and energy-efficient practices and products and take the lead in correcting the course of the climate change crisis. Huawei is at the forefront to reduce our new carbon emissions and has taken several initiatives to bring down its own carbon emissions. "With our innovative products and solutions, we want to work with customers and industry partners to jointly define industry standards, and help operators better measure and manage their carbon reduction roadmaps," says Bob Cai, Chief Marketing Officer at Carrier Business Group, while opening the Summit. The company is not just taking measures to reduce its carbon emissions but is also pursuing innovations to make telecom networks more sustainable and energy-efficient. Cai further elaborated that the ICT

FourKites Expands its Market-Leading Ocean Visibility Platform with Industry-First Dynamic ETA ® for Ocean Shipping and New Tools to Manage Demurrage and Detention20.10.2021 08:30:00 CEST | Press release

FourKites®, the world’s leading real-time supply chain visibility platform, today introduced Dynamic ETA® for Ocean, a new AI-powered innovation as part of its Dynamic OceanSM offering. The new enhancement provides shippers, carriers and 3PLs with the market’s most accurate estimated times of arrival (ETAs) for 100% of their ocean shipments across all lanes worldwide. In addition, the company has rolled out new capabilities for monitoring and mitigating demurrage and detention risks. With these new enhancements, FourKites now provides the most advanced and robust solution on the market for real-time and predictive ocean visibility, exception management and cost controls. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211019006198/en/ FourKites Expands its Market-Leading Ocean Visibility Platform with Industry-First Dynamic ETA® for Ocean Shipping and New Tools to Manage Demurrage and Detention (Photo: Business Wire) These po

Evasc Neurovascular Announces the New eCLIPs Bifurcation Flow Diverter20.10.2021 07:00:00 CEST | Press release

Evasc Neurovascular is proud to announce the third generation of its eCLIPs™ device, the eCLIPs Bifurcation Flow Diverter. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211019006102/en/ The third generation of Evasc's eCLIPs™ device, the eCLIPs Bifurcation Flow Diverter. The new device is classified as a flow diverter, with a leaf density of 35%, which is 60% higher than the existing eCLIPs devices. As a result, the new eCLIPs Bifurcation Flow Diverter does not require coiling and provides for the same unique neck bridging delivery. The new eCLIPs Bifurcation Flow Diverter can cover up to a 6mm neck length. The eCLIPs Bifurcation Flow Diverter also simplifies deployment with greatly reduced delivery times. “We’re excited to see the latest innovation in Evasc Neurovascular’s eCLIPs devices, which will lead to better outcomes for patients and remarkable device delivery time—usually within 10 minutes,” said Leif Sorensen, Neur

Breaking the Silence in Osteoporosis20.10.2021 07:00:00 CEST | Press release

A new international survey of 1,008 women with osteoporosis from six countries, launched on World Osteoporosis Day, reveals that while more than two-thirds (67%) of women over the age of 55 mostly understand what osteoporosis is and the potential negative impact of future fractures, 19% felt poorly informed at the time of the diagnosis.1 This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211019005894/en/ The survey initiated by Theramex in cooperation with the International Osteoporosis Foundation (IOF), was conducted in Germany, France, Italy, Spain, Australia, and Brazil. It included 316 postmenopausal women over the age of 55 years and 315 premenopausal women (40-55 years) with osteoporosis (altogether 62% of the surveyed women). Of these women, osteoporosis was only diagnosed after a fracture in almost 60% of the cases. This survey highlights a gap in osteoporosis awareness, as one-fourth of the patients aged 55 years and ov

Rossum raises record $100 million Series A from General Catalyst to reinvent B2B document communication20.10.2021 06:00:00 CEST | Press release

Rossum, the artificial intelligence company that is re-inventing the way businesses send documents to each other, has raised $100m from one of the US’s best known funds, General Catalyst in what is Eastern Europe’s largest ever Series A. Previous investors, including LocalGlobe, Seedcamp, Miton and Elad Gil are participating, as Rossum looks to capitalise on surging corporate demand for automation in business processes. Having rapidly secured market leadership in document AI technology and become the go-to software for many of Europe’s biggest companies, Rossum is creating a universal document gateway designed to help the corporate world move faster and complete transactions in minutes not weeks. Rossum’s founders, three PhD students who dropped out of university to start the business, are automating the whole business communication stack with a view to making bureaucratic, costly and time-consuming document communication frictionless and paperless. Rossum has seen year-on-year growth

Deenova Keeps Trouncing its competitors in France’s Unit Dose Pharmacy Automation Market20.10.2021 06:00:00 CEST | Press release

Deenova announced today it continues its record-breaking market momentum started during Covid-19, by winning 2 mechatronic competitive public tenders this Summer for a total of 4 ACCED robotic medication solutions to be operated around the clock at Centre Hospitalier Sainte-Marie Rodez in Occitanie, and Hospital of St Die-des-Vosges near Strasburg. Loïc Bessin, Managing Director of Deenova in France stated: “When I joined Deenova 3 years ago, the company was a small unit dose specialty business only present in 2 of the Top 5 European markets, having had customers only in France and Italy. I am extremely proud that over the last year alone, we have grown to become the dominant force in the GDP Top 5 healthcare European mechatronics markets for unit-dose, by establishing close partnership with key customers.” Christophe Jaffuel, Deenova Chief Commercial Officer, stated: “After Deenova multi-million and multi-year recent entries into the two largest European GDP healthcare markets in Germ

Amedeo and AURA AERO Announce a Strategic Partnership Agreement and a Letter of Intent to Purchase 200 Electric Regional Aircraft (“ERA”)19.10.2021 19:21:00 CEST | Press release

AURA AERO has created a clean sheet design while integrating new technologies, as well as existing technologies such as fly by wire, and is driving digital efficiencies to build revolutionary momentum in creating an electrical aircraft. With its current prototype aircraft, INTEGRAL, AURA AERO is achieving design and production maturity at a fraction of the cost and will carry that to the commercially sized 19- seater, Electric Regional Aircraft named ‘ERA’. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211019006068/en/ “Amedeo are delighted to have signed an LOI for 200 Electric Regional Aircraft ‘ERA,’ that draws the commercial aviation industry closer to achieving its goal of net-zero by 2050. We are very pleased to be partnered with AURA AERO in leading such an important climate supporting initiative for the future. For Amedeo, sustainable aviation is more than aspirational and we are committed and focused on partnership