1. - Global employees based across 80 countries and regions voted to support new partnerships with IntraHealth International, Jhpiego, Pathfinder International and the World Food Programme

    - New partnerships bring total Global CSR Program contributions to JPY 16.5 billion across 20 programs in 72 countries following launch in 2016

    - Global CSR Program partnerships help Takeda advance disease prevention measures, empower the health workforce and support health systems

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the addition of four partnerships to its Global Corporate Social Responsibility (CSR) Program, which makes long-term commitments to strengthen health systems and improve access to healthcare for all in developing

    - Global employees based across 80 countries and regions voted to support new partnerships with IntraHealth International, Jhpiego, Pathfinder International and the World Food Programme

    - New partnerships bring total Global CSR Program contributions to JPY 16.5 billion across 20 programs in 72 countries following launch in 2016

    - Global CSR Program partnerships help Takeda advance disease prevention measures, empower the health workforce and support health systems

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the addition of four partnerships to its Global Corporate Social Responsibility (CSR) Program, which makes long-term commitments to strengthen health systems and improve access to healthcare for all in developing and emerging countries. As part of an annual decision-making process, Takeda employees worldwide voted to add IntraHealth International, Jhpiego, Pathfinder International and the World Food Programme (WFP) as new partners for the Global CSR Program, which now supports 20 programs in 72 countries.

    Takeda's commitments to these new partners in FY2021 include:

    • JPY 953 million to IntraHealth International to work with 12 private schools in Mali, Senegal and Niger to increase the number of qualified, trained nurses who can serve rural communities facing a critical shortage of health workers in these countries. Over the next five years, the project will help private schools partner with the public sector, achieve the appropriate accreditations, enhance competency-based curricula, create effective learning approaches and graduate students from underserved areas—all as part of the effort to ensure that everyone everywhere has access to healthcare.
    • JPY 890 million to Jhpiego's iWIN project, to help accelerate progress in preventing maternal and newborn mortality and morbidity by mending the fragmented maternal and child health ecosystem in India via a holistic, woman-centered approach. Over the next five years, the project will catalyze adoption of an Indian-owned model to continuously track and utilize ground-breaking information on the health of pregnant women to create a sustainable, next generation approach to coordinated, patient-centered care before, during and post pregnancy.
    • JPY 1 billion to Pathfinder International to advance women's and girls' leadership in responding to the disproportionate impact of climate change and emergencies on women's health. Operating in South and Southeast Asia over four years, the project will position women and girls as change agents in places where gender inequalities and climate-related crises reduce their quality of health. The project will strengthen access to equitable health services, build community resilience to climate shocks and help women forge their own path to a healthier future.
    • JPY 1.3 billion to the World Food Programme's Project Boreas, over the next five years. The project will enhance the capacity of the United Nations Humanitarian Response Depot in Accra, Ghana, to more efficiently receive, store and transport temperature-sensitive health products in 17 countries in West Africa. The project will create a regional Logistics Knowledge Centre where supply chain professionals and national governments can receive training on best-in-class supply chain practices, ensuring that they are better equipped to face and manage health emergencies.

    "We at IntraHealth are thrilled to be partnering with Takeda to address the shortage of highly trained nurses in rural communities in Mali, Senegal and Niger. This partnership will revolutionize health worker training at private nursing schools, improve health care in the communities that need it most and attract more brilliant young students to the profession," said Polly Dunford, president and CEO of IntraHealth International.

    "Jhpiego is humbled and excited to have won the confidence of Takeda's employees to implement the Integrated Women in Health Network (iWIN) project in Madhya Pradesh, India. It is incredibly inspiring to be motivated by a shared belief: that all women deserve access to the high-quality pregnancy care that saves lives. Together, we will build a mother-focused, scalable maternal and newborn health system by raising quality and continuity of care and leveraging predictive analytics and innovative financing," said Leslie Mancuso, president and CEO of Jhpiego.

    "Pathfinder is honored to work with Takeda to advance the leadership of women and girls. This project is a living example of Takeda's commitment to better health for people and a brighter future for the world. We will elevate women's leadership in South and Southeast Asia for improved health and climate resilience," said Lois Quam, president and CEO of Pathfinder International.

    "The World Food Programme is honored to have been chosen by Takeda's employees as part of their Global CSR Program for a project that is so timely and important. The COVID-19 pandemic served as an important reminder of the need for countries to be prepared for, and equipped to respond to, health emergencies. With Takeda's help, we aim to provide critical support in 17 countries across West Africa – safeguarding vulnerable communities and building capacity for the future," said Tim Hunter, director of Private Partnerships and Fundraising at WFP.

    Since launching in 2016, Takeda's signature Global CSR Program has committed a total of 16.5 billion yen to 17 international and non-governmental organizations on 20 long-term projects that address more than 23 Sustainable Development Goal (SDG) Targets in 72 countries. Each project addresses strengthening health systems in unique ways and aligns with countries' national health needs. These four- to 10-year funding commitments reflect that sustainable impact takes time and there are no quick fixes to the complex and longstanding challenges that health systems face.

    "We are proud to partner with IntraHealth International, Jhpiego, Pathfinder International and the World Food Programme to increase the number of qualified health workers, transform supply chains, address the impact of climate change, conflict and emergencies on women's health, and leverage data and digital to transform maternal and newborn care," said Takako Ohyabu, Takeda chief global corporate affairs officer. "All our new partnerships address endemic challenges health systems face today while empowering communities to be ready for the future."

    Takeda's Global CSR Program partnerships have made notable progress since 2016:

    • Reached more than 5.84 million direct beneficiaries;
    • Trained more than 32,000 qualified health workers;
    • Provided more than 570,000+ community members with targeted health education;
    • Immunized millions of children against preventable diseases such as measles; and
    • Our partners are on track to reach 18.4 million direct beneficiaries by 2027, providing lifesaving care for vulnerable women and newborns, preventing stunting, strengthening holistic support for refugees, and so much more.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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.

    Important Notice

    For the purposes of this notice, "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 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 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 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 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.

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  2. Partnership leverages Takeda's leadership in rare disease, gastroenterology, and hepatology to advance maralixibat in a major market

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Takeda Pharmaceutical Company Limited ((TAK) announced that the companies have entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA). Maralixibat, an investigational, orally administered medication, is being evaluated globally in ALGS, PFIC, and BA.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210921005502/en/

    Under the terms of the agreement…

    Partnership leverages Takeda's leadership in rare disease, gastroenterology, and hepatology to advance maralixibat in a major market

    Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) and Takeda Pharmaceutical Company Limited ((TAK) announced that the companies have entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA). Maralixibat, an investigational, orally administered medication, is being evaluated globally in ALGS, PFIC, and BA.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210921005502/en/

    Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.

    "Takeda is a leading global biopharmaceutical company with extensive experience in development and commercialization of novel therapies to treat rare diseases as well as gastroenterology and hepatology, making them an ideal partner as we look to accelerate the delivery of maralixibat to children living with rare liver diseases in Japan," said Chris Peetz, president and chief executive officer of Mirum. "As we approach potential commercialization in the United States and complete the recent filing for Alagille syndrome in Europe, our goal is to partner with top companies outside of North America and Europe to ensure global reach for patients with these terrible diseases. We are excited for Takeda to engage in the development of maralixibat and collaborate in our effort to advance this potentially life-changing therapy."

    "There is a significant unmet medical need for a treatment to help patients with cholestatic diseases such as ALGS and PFIC in Japan and developing novel treatment for those patients suffering from rare liver diseases is a top priority for Takeda's global R&D strategy," said Dr. Naoyoshi Hirota, general manager of Takeda development center Japan. "This agreement reinforces Takeda's commitment to developing highly differentiated medicines to improve the health and quality of life of patients."

    Mirum has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome. The NDA is currently under priority review with a PDUFA, or FDA decision date, of September 29, 2021. Mirum also recently submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with ALGS.

    About Maralixibat

    Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated effects. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. Food and Drug Administration has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was shown to have a tolerable safety profile in the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Maralixibat has been studied extensively and its safety database represents the largest database for an ASBT inhibitor.

    Until maralixibat is approved and available for prescribing, the medication is available to patients with ALGS through Mirum's expanded access program. For more information, please visit ALGSEAP.com. For further information about maralixibat's ongoing studies in pediatric liver disease, please visit the study websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study for biliary atresia.

    About Mirum Pharmaceuticals, Inc.

    Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirum's lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat for the treatment of cholestatic pruritus in patients with ALGS in the U.S. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirum has submitted a marketing authorization application for maralixibat to the European Medicines Agency for the treatment of cholestatic liver disease for patients with ALGS. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

    To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

    Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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.

    Disclaimer

    The drug information contained herein is intended to disclose corporate information. Nothing contained in this document should be considered a solicitation, promotion, or indication for any prescription drug, including those currently under development.

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential development and commercialization by Takeda of maralixibat in Japan for various indications, Mirum and Takeda's receipt of revenue in connection with the license agreement with Takeda, as well as the regulatory approval pathway for maralixibat. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "will," "could," "would," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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  3. Approval based on Phase 1/2 trial results, which demonstrated clinically meaningful responses with a median duration of response (DoR) of approximately 1.5 years

    Next-generation sequencing (NGS) companion diagnostic test approved simultaneously to support identification of patients with EGFR Exon20 insertion mutations

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the U.S. Food and Drug Administration (FDA) has approved EXKIVITY (mobocertinib) for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations as detected by an FDA-approved test, whose disease has progressed on or after platinum-based…

    Approval based on Phase 1/2 trial results, which demonstrated clinically meaningful responses with a median duration of response (DoR) of approximately 1.5 years

    Next-generation sequencing (NGS) companion diagnostic test approved simultaneously to support identification of patients with EGFR Exon20 insertion mutations

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the U.S. Food and Drug Administration (FDA) has approved EXKIVITY (mobocertinib) for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. EXKIVITY, which was granted priority review and received Breakthrough Therapy Designation, Fast Track Designation and Orphan Drug Designation from the FDA, is the first and only approved oral therapy specifically designed to target EGFR Exon20 insertion mutations. This indication is approved under Accelerated Approval based on overall response rate (ORR) and DoR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

    "The approval of EXKIVITY introduces a new and effective treatment option for patients with EGFR Exon20 insertion+ NSCLC, fulfilling an urgent need for this difficult-to-treat cancer," said Teresa Bitetti, president, Global Oncology Business Unit, Takeda. "EXKIVITY is the first and only oral therapy specifically designed to target EGFR Exon20 insertions, and we are particularly encouraged by the duration of the responses observed with a median of approximately 1.5 years. This approval milestone reinforces our commitment to meeting the needs of underserved patient populations within the oncology community."

    The FDA simultaneously approved Thermo Fisher Scientific's Oncomine Dx Target Test as an NGS companion diagnostic for EXKIVITY to identify NSCLC patients with EGFR Exon20 insertions. NGS testing is critical for these patients, as it can enable more accurate diagnoses compared to polymerase chain reaction (PCR) testing, which detects less than 50% of EGFR Exon20 insertions.

    "EGFR Exon20 insertion+ NSCLC is an underserved cancer that we have been unable to target effectively with traditional EGFR TKIs," said Pasi A. Jänne, MD, PhD, Dana Farber Cancer Institute. "The approval of EXKIVITY (mobocertinib) marks another important step forward that provides physicians and their patients with a new targeted oral therapy specifically designed for this patient population that has shown clinically meaningful and sustained responses."

    "Patients with EGFR Exon20 insertion+ NSCLC have historically faced a unique set of challenges living with a very rare lung cancer that is not only underdiagnosed, but also lacking targeted treatment options that can improve response rates," said Marcia Horn, executive director, Exon 20 Group at ICAN, International Cancer Advocacy Network. "As a patient advocate working with EGFR Exon20 insertion+ NSCLC patients and their families every day for nearly five years, I am thrilled to witness continued progress in the fight against this devastating disease and am grateful for the patients, families, healthcare professionals and scientists across the globe who contributed to the approval of this promising targeted therapy."

    The FDA approval is based on results from the platinum-pretreated population in the Phase 1/2 trial of EXKIVITY, which consisted of 114 patients with EGFR Exon20 insertion+ NSCLC who received prior platinum-based therapy and were treated at the 160 mg dose. Results were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting from the Phase 1/2 trial and demonstrated a confirmed ORR of 28% per independent review committee (IRC) (35% per investigator) as well as a median DoR of 17.5 months per IRC, a median overall survival (OS) of 24 months and a median progression-free survival (PFS) of 7.3 months per IRC.

    The most common adverse reactions (>20%) were diarrhea, rash, nausea, stomatitis, vomiting, decreased appetite, paronychia, fatigue, dry skin, and musculoskeletal pain. The EXKIVITY Prescribing Information includes a boxed warning for QTc prolongation and Torsades de Pointes, and warnings and precautions for interstitial lung disease/pneumonitis, cardiac toxicity, and diarrhea.

    The FDA review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence (OCE), which provides a framework for concurrent submission and review of oncology products among international partners. We look forward to continuing our work with regulatory agencies across the globe to bring mobocertinib to patients.

    Delivering Takeda's Wave 1 Pipeline

    Takeda is positioned to deliver near-term growth through global brand expansions and its Wave 1 pipeline, which includes multiple best-in-class/first-in-class new molecular entities (NMEs) with potential for approvals through FY2024. Our Wave 2 pipeline contains approximately 30 NMEs and next-generation platforms that will support Takeda's sustainable growth through FY25 and beyond.

    About EXKIVITY (mobocertinib)

    EXKIVITY is a first-in-class, oral tyrosine kinase inhibitor (TKI) specifically designed to selectively target epidermal growth factor receptor (EGFR) Exon20 insertion mutations.

    EXKIVITY is approved in the U.S. for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.

    Results from the Phase 1/2 trial of mobocertinib have also been accepted for review by the Center for Drug Evaluation (CDE) in China for locally advanced or metastatic NSCLC patients with EGFR Exon20 insertion mutations who have been previously treated with at least one prior systemic chemotherapy.

    For more information about EXKIVITY, visit www.EXKIVITY.com. For the Prescribing Information, including the Boxed Warning, please visit https://takeda.info/Exkivity-Prescribing-Information.

    About EGFR Exon20 Insertion+ NSCLC

    Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 85% of the estimated 2.2 million new cases of lung cancer diagnosed each year worldwide, according to the World Health Organization.1,2 Patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ NSCLC make up approximately 1-2% of patients with NSCLC, and the disease is more common in Asian populations compared to Western populations.3-7 This disease carries a worse prognosis than other EGFR mutations, as EGFR TKIs – which do not specifically target EGFR Exon20 insertions – and chemotherapy provide limited benefit for these patients.

    Takeda is committed to continuing research and development to meet the needs of the lung cancer community through the discovery and delivery of transformative medicines.

    EXKIVITY IMPORTANT SAFETY INFORMATION

    QTc Interval Prolongation and Torsades de Pointes: EXKIVITY can cause life-threatening heart rate-corrected QT (QTc) prolongation, including Torsades de Pointes, which can be fatal, and requires monitoring of QTc and electrolytes at baseline and periodically during treatment. Increase monitoring frequency in patients with risk factors for QTc prolongation. Avoid use of concomitant drugs which are known to prolong the QTc interval and use of strong or moderate CYP3A inhibitors with EXKIVITY, which may further prolong the QTc. Withhold, reduce the dose, or permanently discontinue EXKIVITY based on the severity of QTc prolongation.

    Interstitial Lung Disease (ILD)/Pneumonitis: Monitor patients for new or worsening pulmonary symptoms indicative of ILD/pneumonitis. Immediately withhold EXKIVITY in patients with suspected ILD/pneumonitis and permanently discontinue EXKIVITY if ILD/pneumonitis is confirmed.

    Cardiac Toxicity: Monitor cardiac function, including left ventricular ejection fraction, at baseline and during treatment. Withhold, resume at reduced dose or permanently discontinue based on severity.

    Diarrhea: Diarrhea may lead to dehydration or electrolyte imbalance, with or without renal impairment. Monitor electrolytes and advise patients to start an antidiarrheal agent at first episode of diarrhea and to increase fluid and electrolyte intake. Withhold, reduce the dose, or permanently discontinue EXKIVITY based on the severity.

    Embryo-Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective non-hormonal contraception.

    Takeda's Commitment to Oncology

    Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it's with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

    1 Sung H. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries. https://pubmed.ncbi.nlm.nih.gov/33538338/. Accessed May 27, 2021

    2 American Cancer Society. What is Non-Small Cell Lung Cancer? https://www.cancer.org/cancer/non-small-cell-lung-cancer/about/what-is-non-small-cell-lung-cancer.html.

    3 Riess, Jonathan W. Diverse EGFR Exon 20 Insertions and Co-Occurring Molecular Alterations Identified by Comprehensive Genomic Profiling of NSCLC. https://www.jto.org/article/S1556-0864(18)30770-6/fulltext. Accessed April 7, 2020.

    4 Fang, Wenfeng. BMC Cancer. EGFR exon 20 insertion mutations and response to osimertinib in non-small-cell lung cancer. https://bmccancer.biomedcentral.com/articles/10.1186/s12885-019-5820-0. Accessed April 7, 2020.

    5 Kobayashi Y, Mitsudomi T. Not all epidermal growth factor receptor mutations in lung cancer are created equal: Perspectives for individualized treatment strategy. Cancer Sci. 2016;107(9):1179-1186. doi:10.1111/cas.12996

    6 Yatabe Y, Kerr KM, Utomo A, et al. EGFR mutation testing practices within the Asia Pacific region: results of a multicenter diagnostic survey. J Thorac Oncol. 2015;10(3):438-445. doi:10.1097/JTO.0000000000000422

    7 Kris MG, Johnson BE, Berry LD, et al. Using multiplexed assays of oncogenic drivers in lung cancers to select targeted drugs. JAMA. 2014;311(19):1998-2006. doi:10.1001/jama.2014.3741

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  4. Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the Phase 3 PANTHER (Pevonedistat-3001) study did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS). The trial evaluated whether the combination of pevonedistat plus azacitidine as first-line treatment for patients with higher-risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myeloid leukemia (AML) improved EFS versus azacitidine alone. An event in the trial is defined as death or transformation to AML in participants with higher-risk MDS or CMML, whichever occurs first, and death in participants with AML.

    "While we are disappointed with this outcome, we are continuing…

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the Phase 3 PANTHER (Pevonedistat-3001) study did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS). The trial evaluated whether the combination of pevonedistat plus azacitidine as first-line treatment for patients with higher-risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myeloid leukemia (AML) improved EFS versus azacitidine alone. An event in the trial is defined as death or transformation to AML in participants with higher-risk MDS or CMML, whichever occurs first, and death in participants with AML.

    "While we are disappointed with this outcome, we are continuing to gain a greater understanding of the full data set and hope that findings from this Phase 3 study will provide information to help guide research and development for potential treatment options for these underserved patient populations," said Chris Arendt, PhD, Head, Oncology Cell Therapy and Therapeutic Area Unit, Takeda. "We would like to thank the patients, families, advocacy organizations and investigators that participated in this trial, without whom this meaningful research would not have been possible. Takeda remains committed to conducting important research and transforming the lives of patients with cancer."

    Full data results will be submitted for presentation at an upcoming medical congress. Investigators have been informed of the outcome so they can discuss the potential impact with study participants. Takeda will work with investigators who will determine the most appropriate action for each individual patient enrolled in the study.

    About Pevonedistat

    Pevonedistat is a NEDD8-activating enzyme (NAE) inhibitor that leads to cancer cell death by disrupting protein homeostasis. The Phase 3 PANTHER study (Pevonedistat-3001) did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS) and the data results are currently being evaluated. The safety profile was consistent with previously reported data for this combination. Pevonedistat is an investigational drug for which safety and efficacy have not been established.

    Takeda's Commitment to Oncology

    Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it's with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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.

    Important Notice

    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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

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  5. The new Tukwila, WA center underscores the urgent patient need for plasma while supporting Takeda's sustainability goal to have zero carbon emissions by 2040

    BioLife Plasma Services, part of the global biopharmaceutical company Takeda Pharmaceutical Company Limited, today announced the opening of an all-electric plasma donation center in Tukwila, WA. The new center opening is the first in a sustainability initiative to build all-electric plasma donation centers in the United States (U.S.), contributing to the company's dedication to deliver better health for people and a brighter future for the world by addressing the urgent need for plasma as well as key environmental challenges.

    "It is vital we grow and transform our operations responsibly…

    The new Tukwila, WA center underscores the urgent patient need for plasma while supporting Takeda's sustainability goal to have zero carbon emissions by 2040

    BioLife Plasma Services, part of the global biopharmaceutical company Takeda Pharmaceutical Company Limited, today announced the opening of an all-electric plasma donation center in Tukwila, WA. The new center opening is the first in a sustainability initiative to build all-electric plasma donation centers in the United States (U.S.), contributing to the company's dedication to deliver better health for people and a brighter future for the world by addressing the urgent need for plasma as well as key environmental challenges.

    "It is vital we grow and transform our operations responsibly and sustainably with respect for the people and communities that we serve," said Sanjay Bhana, Head of U.S. BioLife Operations. "This first all-electric center represents our commitment and leadership in sustainability performance and outcomes, both from an environmental standpoint as well as ensuring a steady supply of medicine developed from plasma, a lifeline for thousands of people who are immune-compromised or live with a variety of other complex conditions. Leading by example, we are empowering our employees to go above and beyond to help conserve the world's natural resources."

    As the demand for medicine developed from plasma increases, BioLife is expanding to help meet the urgent need for plasma donations. The all-electric Tukwila center is part of BioLife's growing network of more than 150 state-of-the-art plasma donation centers in the U.S., recognized for their world-class donation safety standards. Plasma donations received at BioLife centers are used by Takeda to make established therapies that treat a range of rare and complex conditions, such as immunodeficiency disorders, for which there are often no alternative treatments.

    The commitment to build all-electric plasma donation centers is part of Takeda's dedication to making environmental stewardship and resource conservation central to its business operations and practices. This is one of several dedicated programs to continue to reduce the company's environmental footprint. BioLife Plasma Services is a key contributor to Takeda's pledge to have zero carbon emissions resulting from its own operations by 2040, zero waste to landfill from its major locations by 2030 and a 5% reduction in water use by 2025. More specifically, BioLife will be addressing key environmental concerns through efforts that involve on-site renewable technology, energy efficiency and waste reduction – including working to eliminate single-use plastics – to help meet Takeda's sustainability goals.

    Prospective donors can make online appointments to visit the Tukwila center (5951 S 180th St, Ste 101, Tukwila, WA 98188), which opens on Saturday, August 21. Individuals who choose to donate must pass a physical examination at their first visit and are screened at each visit to ensure they meet eligibility criteria. All donors are compensated for their time and commitment.

    To learn more about BioLife Plasma Services, the donation process and to schedule an appointment, please visit the BioLife website. Learn more about Takeda's commitment to environmental sustainability on the company's website.

    About BioLife Plasma Services

    BioLife Plasma Services is an industry leader in the collection of high-quality plasma that is processed into life-saving plasma-based therapies. Founded in 2002, BioLife has been in operation for 18 years. We operate more than 180 state-of-the-art plasma collection facilities across the United States and Europe. BioLife Plasma Services is part of Takeda Pharmaceutical Company Limited (NYSE:TAK), a global values-based, R&D-driven pharmaceutical company that produces and delivers plasma-based therapies among other specialty medicines. For more information, visit BioLifePlasma.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 healthcare in approximately 80 countries and regions. For more information, visit https://www.takeda.com.

    About Plasma

    Plasma is the clear, straw-colored liquid portion of blood that can be easily replaced by the body. Plasma makes up more than half of whole blood and consists primarily of water and proteins. During plasma donation, a donor's blood is collected into an automated device that separates the plasma from the other whole blood components, including red and white blood cells and platelets. While the plasma is collected, the other blood components are returned to the donor. Each donation procedure uses sterile and disposable collection materials. The body quickly replaces the plasma removed during the donation process, which allows healthy individuals to donate as often as twice in a seven-day period, with at least one day between donations.

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  6. The Phase 3 HELP Study™ Open-label Extension (OLE) showed preventative treatment with TAKHZYRO markedly reduced the frequency of hereditary angioedema (HAE) attacks by 87.4 percent overall compared to baseline over the course of approximately 2.5 years

    TAKHZYRO use demonstrated a safety profile consistent with the original pivotal study with prolonged attack-free periods

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the publication of the final results from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE) designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO® (lanadelumab) 300mg every two weeks for up to 2.5 years. The study results…

    The Phase 3 HELP Study™ Open-label Extension (OLE) showed preventative treatment with TAKHZYRO markedly reduced the frequency of hereditary angioedema (HAE) attacks by 87.4 percent overall compared to baseline over the course of approximately 2.5 years

    TAKHZYRO use demonstrated a safety profile consistent with the original pivotal study with prolonged attack-free periods

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the publication of the final results from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE) designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO® (lanadelumab) 300mg every two weeks for up to 2.5 years. The study results show that preventative treatment with TAKHZYRO markedly reduced the frequency of hereditary angioedema (HAE) attacks in patients 12 years of age and older who received treatment for a mean duration of almost 2.5 years (29.6 months; 8.2 standard deviation).1 The data were published online this month in the journal Allergy.

    Secondary endpoints of the study showed the mean (min;max) HAE attack rate observed in the study population (N=209) was reduced by 87.4 percent (-100; 852.8) overall versus baseline, and attacks requiring acute treatment (N=106) were reduced by 93.4 percent (-100; -52.8).1 Reductions were also shown (N=209) in the rate of moderate or severe attacks (84.3 percent). Patients treated with TAKHZYRO 300 mg every two weeks reduced the HAE disease burden by being attack-free for a mean (SD) of 97.7 percent (6.0 percent) of days during treatment, and the average duration of the attack-free period was 14.8 months. Nearly 7 out of 10 patients (68.9 percent) experienced an attack-free period of more than 12 months (n=209). Treatment-related treatment-emergent adverse events (TEAEs) were reported by 54.7 percent of patients (N=116), most commonly injection site reactions. There were no reports of serious treatment-related TEAEs.

    The validated Angioedema Quality of Life Questionnaire (AE-QoL) was among the patient-reported outcome tools used to evaluate patients' quality of life (QoL). Both rollovers and non-rollovers achieved the minimal clinically important difference for the AE-QoL total score: the mean (SD) change in total score from baseline was –10.2 (17.9) and –19.5 (21.3) for rollovers and non-rollovers, respectively. Most of the improvements in AE-QoL scores were observed during the early follow-up period (from day 0 to 56), before reaching a plateau, and scores were generally maintained during subsequent visits.1

    "Effective prevention backed by clinical evidence is critical for healthcare professionals who treat patients with HAE," said Aleena Banerji, M.D., Division of Rheumatology, Allergy and Immunology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School and principal investigator for the HELP Study. "The potential to be attack-free for periods of time can help to provide an additional sense of assurance for those living with this chronic and unpredictable disease."

    The original Phase 3 HELP Study was conducted in 125 patients aged 12 years and older over 26 weeks, making it one of the largest randomized, controlled prevention studies in HAE, with the longest active treatment duration, to date.2,3,4,5,6 The HELP Study OLE was designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO for up to 2.5 years. The complete results were based on data collected between May 2016 and October 2019 and included 109 rollover patients who were originally evaluated in the HELP Study, and 103 eligible non-rollover patients who did not participate in the initial study but had experienced at least one HAE attack in 12 weeks.1 The complete results from the HELP Study OLE showed that the safety profile of TAKHZYRO was consistent with the original findings from the HELP Study, with treatment-related TEAEs occurring in 54.7 percent of patients (n=116) and the most common being injection-site pain, upper respiratory tract infection, or headache.1

    "This study supports the use of TAKHZYRO as a long-term preventative treatment option for those 12 years of age and older living with HAE who are seeking a preventative treatment option that is proven to reduce HAE attacks," said Neil Inhaber, M.D., Vice President, Global Medical Heald, HAE and Transplant at Takeda. "Takeda has more than 10 years supporting people with this rare disease across the HAE portfolio and we are committed to providing these patients with effective treatment options that may help them experience periods of time without attacks. Our legacy and dedication to HAE hopefully empowers patients to confidently navigate their HAE journey."

    About the HELP Study™ Open-label Extension

    The HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE) is an evaluation of the long-term efficacy and safety of TAKHZYRO in hereditary angioedema (HAE) patients of at least 12 years of age and older. Two hundred and twelve patients received treatment with TAKHZYRO at the start of the OLE Study (109 rollover patients originally evaluated in the HELP Study and who continued into the OLE, and 103 eligible patients who did not participate in the HELP Study but who had experienced at least one attack in the last 12 weeks). Rollover patients received a dose of 300 mg TAKHZYRO on Day 0 and then every two weeks after their first attack. Non-rollover patients were treated with one 300 mg dose every two weeks, beginning on Day 0. The majority of patients (92.5%; N=196) completed at least 12 months in the study, and 81.6% (N=173) completed at least 30 months.1

    About Hereditary Angioedema

    Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of oedema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful.7,8,9 Attacks that obstruct the airways can cause asphyxiation and are potentially life threatening.9,10 HAE affects an estimated 1 in 50,000 people worldwide. It is often under recognized, under diagnosed and under treated.7,9,10

    Takeda in Hereditary Angioedema

    Hereditary Angioedema (HAE), like so many other rare diseases, is highly complex, and patients, their families and caregivers often undergo years of strain trying to understand their disease, get a definitive diagnosis and gain access to the medicines they need. At Takeda we are committed to be a champion for the patients we serve. Every individual living with HAE is unique and by listening and reacting to their needs, we translate the insights we gain into innovative solutions – from diagnosis to ongoing management. Advancing the science is crucial to the way we operate and we are bold in our mission to accelerate diagnosis and develop treatments that will make a difference to the lives of HAE patients, their support networks and those medical professionals who care for them.

    About TAKHZYRO® (lanadelumab) Injection

    TAKHZYRO is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein and is indicated for prophylaxis to prevent HAE attacks in patients 12 years and older. TAKHZYRO is formulated for subcutaneous administration and has a half-life of approximately two weeks.3 TAKHZYRO is intended for self-administration or administration by a caregiver. The patient or caregiver should be trained by a healthcare professional.7

    TAKHZYRO Safety Information for Europe

    Please consult the TAKHZYRO Summary Product Characteristics (SmPC) before prescribing.

    TAKHZYRO treatment should be initiated under the supervision of a physician experienced in the management of patients with hereditary angioedema (HAE). TAKHZYRO may be self-administered or administered by a caregiver only after training on SC injection technique by a healthcare professional.3

    Contraindication

    Hypersensitivity to the active substance or to any of the excipients.3

    Warnings and Precautions

    Traceability: In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.3

    Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, administration of TAKHZYRO must be stopped immediately and appropriate treatment must be initiated.3

    General: TAKHZYRO is not intended for treatment of acute HAE attacks. In case of a breakthrough HAE attack, individualized treatment should be initiated with an approved rescue medication. There are no available clinical data on the use of lanadelumab in HAE patients with normal C1-INH activity.3

    Interference with coagulation test: Lanadelumab can increase activated partial thromboplastin time (aPTT) due to an interaction of lanadelumab with the aPTT assay. The reagents used in the aPTT laboratory test initiate intrinsic coagulation through the activation of plasma kallikrein in the contact system. Inhibition of plasma kallikrein by lanadelumab can increase aPTT in this assay. None of the increases in aPTT in patients treated with TAKHZYRO were associated with abnormal bleeding adverse events. There were no differences in international normalised ratio (INR) between treatment groups.3

    Sodium content: This medicinal product contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially 'sodium-free'.3

    Interactions

    No dedicated drug-drug interaction studies have been conducted. Based on the characteristics of lanadelumab, no pharmacokinetic interactions with co-administered medicinal products is expected.3

    As expected, concomitant use of the rescue medication C1 esterase inhibitor results in an additive effect on lanadelumab-cHMWK response based on the mechanism of action (MOA) of lanadelumab and C1 esterase inhibitor.3

    Immunogenicity

    Treatment with lanadelumab has been associated with development of treatment emergent anti-drug antibodies (ADA) in 11.9% (10/84) of subjects. All antibody titres were low. The ADA response was transient in 20% (2/10) of ADA positive subjects. 2.4% (2/84) of lanadelumab-treated subjects tested positive for neutralizing antibodies.3

    The development of ADA including neutralising antibodies against TAKHZYRO did not appear to adversely affect the pharmacokinetic (PK) and pharmacodynamics (PD) profiles or clinical response.3

    Adverse Reactions

    The most commonly observed adverse reaction (52.4%) associated with TAKHZYRO was injection site reactions (ISR) including injection site pain, injection site erythema and injection site bruising. Of these ISRs, 97% were of mild intensity, 90% resolved within 1 day after onset with a median duration of 6 minutes.3

    Hypersensitivity reaction (mild and moderate pruritus, discomfort and tingling of tongue) was observed (1.2%)

    Very common

    (frequency ≥1/10):

    Injection site reactions*

    Common

    (≥1/100 to <1/10):

    Hypersensitivity**, dizziness, rash maculopapular, myalgia, alanine aminotransferase increased, aspartate aminotransferase increased.

    *Injection site reactions include: pain, erythema, bruising, discomfort, haematoma, haemorrhage, pruritus, swelling, induration, paraesthesia, reaction, warmth, oedema and rash.

    ** Hypersensitivity includes: pruritus, discomfort and tingling of tongue.

    For European Union Summary of Product Characteristics, please visit https://www.ema.europa.eu/en/documents/product-information/takhzyro-epar-product-information_en.pdf.

    For full U.S. Prescribing Information, including the approved indication and important safety information, please visit https://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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.

     

    Important Notice

    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.

    References


    1 Banerji A, Bernstein JA, Johnston DT, et al; for HELP OLE Investigators. Long-term prevention of hereditary angioedema attacks with lanadelumab: the HELP OLE Study. Allergy. 2021 Jul 21. doi: 10.1111/all.15011. Epub ahead of print.

    2 Banerji A, Riedl MA, Bernstein JA, et al; for the HELP Investigators. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks: a randomized clinical trial. JAMA. 2018;320(20):2108-2121.

    3 TAKHZYRO (lanadelumab) European Summary of Product Characteristics.

    4 CINRYZE (C1 esterase inhibitor [human]) [prescribing information]. Lexington, MA: Shire ViroPharma Incorporated; 2018.

    5 HAEGARDA [prescribing information]. Kankakee, IL: CSL Behring LLC; 2017.

    6 Craig T, Zuraw B, Longhurst H, et al. Long-term outcomes with subcutaneous C1-inhibitor replacement therapy for prevention of hereditary angioedema attacks. J Allergy Clin Immunol Pract. 2019;7(6):1793-1802.

    7 Cicardi M, Bork K, Caballero T, et al; on behalf of HAWK (Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012; 67(2):147-157.

    8 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

    9 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.

    10 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.

    PromoMats job code: C-ANPROM/INT/TAKH/0019

    Date of preparation: July 2021

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  7.  − Takeda Intends to Challenge This Outcome through All Available Legal Means

    First Quarter FY2021 Reported IRFS-based Financial Results Will Be Updated to Reflect the Impact of the Decision with No Impact on Core and Underlying Financial Results

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the receipt of a decision by the Irish Tax Appeals Commission on July 30, 2021 (IST) to uphold the Irish Revenue Commissioners' position related to the treatment of a break fee received by Shire plc ("Shire") in October 2014 from AbbVie Inc. ("AbbVie"). Shire was acquired by Takeda in January 2019. Takeda intends to challenge this outcome through all available legal means including appealing the decision to the Irish courts…

     − Takeda Intends to Challenge This Outcome through All Available Legal Means

    First Quarter FY2021 Reported IRFS-based Financial Results Will Be Updated to Reflect the Impact of the Decision with No Impact on Core and Underlying Financial Results

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the receipt of a decision by the Irish Tax Appeals Commission on July 30, 2021 (IST) to uphold the Irish Revenue Commissioners' position related to the treatment of a break fee received by Shire plc ("Shire") in October 2014 from AbbVie Inc. ("AbbVie"). Shire was acquired by Takeda in January 2019. Takeda intends to challenge this outcome through all available legal means including appealing the decision to the Irish courts.

    On November 28, 2018, Shire received a tax assessment from the Irish Revenue Commissioners for 398 million EUR. This assessment sought to tax a 1,635 million USD break fee Shire received from AbbVie in connection with the terminated offer to acquire Shire made by AbbVie in 2014. Takeda appealed this assessment, and in late 2020 a hearing took place before the Irish Tax Appeals Commission.

    While Takeda is continuing to assess the substance of the decision, the company will record a provision for the case in the consolidated financial statements for the fiscal quarter, which ended on June 30, 2021 ("Q1 FY2021"), as a subsequent event that relates to a condition that existed as of June 30, 2021. The provision is currently estimated at approximately 63 billion JPY including interest accrued through June 30, 2021. In addition, to reflect the impact of the provision, Takeda will revise its "Summary of Financial Statements for the Three-month Period Ended June 30, 2021" (IFRS, Consolidated) and intends to re-file the revised information with the Tokyo Stock Exchange no later than August 6, 2021 (JST). Takeda will also revise other Q1 FY2021 financial materials to reflect the impact of the provision, and publish them on its website by August 6, 2021 (JST). There is no change to Takeda's Q1 FY2021 Core and Underlying financial results, as the tax charge resulting from the decision is a non-recurring item considered unrelated to Takeda's ongoing operations. To view the updated materials, once available, please visit: https://www.takeda.com/investors/financial-results/.

    At this time, Takeda is not revising its forecast for the full fiscal year ending March 31, 2022 ("Full Year FY2021"). Takeda will update its Full Year FY2021 consolidated financial forecasts (based on IFRS) at the appropriate timing by taking this decision as well as other factors into consideration. This decision will not impact the Full Year FY2021 outlook for Core or Underlying financial results.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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.

    Important Notice

    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.

    The product names appearing in this document are trademarks or registered trademarks owned by Takeda, or their respective owners.

    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.

    Financial information

    Takeda's financial statements are prepared in accordance with International Financial Reporting Standards ("IFRS").

    Certain Non-IFRS Financial Measures

    This press release and materials distributed in connection with this press release include certain IFRS financial measures not presented in accordance with International Financial Reporting Standards ("IFRS"), such as Underlying Revenue, Core Operating Profit, Underlying Core Operating Profit, Core Net Profit, Underlying Core EPS, Net Debt, EBITDA, Adjusted EBITDA and Free Cash Flow. Takeda's management evaluates results and makes operating and investment decisions using both IFRS and non-IFRS measures included in this press release. These non-IFRS measures exclude certain income, cost and cash flow items which are included in, or are calculated differently from, the most closely comparable measures presented in accordance with IFRS. By including these non-IFRS measures, management intends to provide investors with additional information to further analyze Takeda's performance, core results and underlying trends. Takeda's non-IFRS measures are not prepared in accordance with IFRS and such non-IFRS measures should be considered a supplement to, and not a substitute for, measures prepared in accordance with IFRS (which we sometimes refer to as "reported" measures). Investors are encouraged to review the reconciliation of non-IFRS financial measures to their most directly comparable IFRS measures.

    Further information on certain of Takeda's Non-IFRS measures is posted on Takeda's investor relations website at https://www.takeda.com/investors/financial-results/

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    • Delivered Year-over-Year Growth in Reported Revenue of +18.4% and Underlying Revenue of +3.8%, Driven by 14 Global Brands
    • Grew Reported Operating Profit of 248.6 Billion Yen ($2.2B) and Solid Underlying Core Operating Profit Margin of 30.5% While Increasing R&D Investments
    • Paid 242.9 Billion Yen Toward $2.2 Billion of Debt in Q1 and Company Expects to Pre-pay a Total of Approximately 450 Billion Yen (~$4.1B) of Debt in FY2021
    • Anticipated Inflection Year for the Pipeline with Five to Six Regulatory Submissions and Seven New Molecular Entities in Pivotal Studies by Fiscal Year-End
    • Confirms Full-year FY2021 Management Guidance and Forecast

    Takeda Pharmaceutical Company Limited (TYO:4502, NYSE:TAK) ("Takeda") today announced financial…

    • Delivered Year-over-Year Growth in Reported Revenue of +18.4% and Underlying Revenue of +3.8%, Driven by 14 Global Brands
    • Grew Reported Operating Profit of 248.6 Billion Yen ($2.2B) and Solid Underlying Core Operating Profit Margin of 30.5% While Increasing R&D Investments
    • Paid 242.9 Billion Yen Toward $2.2 Billion of Debt in Q1 and Company Expects to Pre-pay a Total of Approximately 450 Billion Yen (~$4.1B) of Debt in FY2021
    • Anticipated Inflection Year for the Pipeline with Five to Six Regulatory Submissions and Seven New Molecular Entities in Pivotal Studies by Fiscal Year-End
    • Confirms Full-year FY2021 Management Guidance and Forecast

    Takeda Pharmaceutical Company Limited (TYO:4502, NYSE:TAK) ("Takeda") today announced financial results for the first quarter of fiscal year 2021 (period ended June 30, 2021). Based on the solid first-quarter results, the Company also confirmed its fiscal year 2021 management guidance and forecast. Fiscal year 2021 remains a year of inflection with Takeda positioned for topline acceleration and continued pipeline progress, including critical regulatory submissions, potential approvals and additional new molecular entities (NMEs) advancing in pivotal studies.

    Costa Saroukos, Chief Financial Officer, commented:

    "With the Shire integration behind us and continued transformation over the last two years, we've pivoted towards accelerating topline growth and investing in R&D to fuel our highly innovative pipeline. Takeda's first-quarter results demonstrate the continued strength of our 14 global brands and, with the business momentum from this strong start to our fiscal year, we remain on track towards our full-year management guidance."

    "FY2021 is an inflection year for our pipeline as we ramp up strategic R&D investments and the pipeline starts to deliver. We anticipate having five to six Wave 1 pipeline regulatory submissions by the end of this fiscal year, with the potential for five approvals by end of H1 FY2022. The resilience of Takeda's business model is a testament to our unwavering commitment to serving patients, our people and the planet. We are focused on discovering and delivering life-transforming treatments to people around the world to create and maximize long-term value for society."

    FINANCIAL AND BUSINESS HIGHLIGHTS

    Results for Q1 FY2021 Ended June 30, 2021

    (billion yen, except percentages and per share amounts)

    REPORTED

    CORE

    (Non-IFRS)(a)

    UNDERLYING(b)

    (Non-IFRS)(a)

    Q1 FY2021

    vs. PRIOR YEAR

    Q1 FY2021

    vs. PRIOR YEAR

    Revenue

    949.6

    +18.4%

    816.6

    +1.8%

    +3.8%

    Operating Profit

    248.6

    +48.6%

    248.9(c)

    -11.4%

    -2.1%

    Margin

    26.2%

    +5.3pp

    30.5%

    -4.5pp

    30.5%

    Net Profit

    200.4

    +142.8%

    176.6

    -7.4%

     

    EPS (JPY)

    128 yen

    +141.9%

    113 yen

    -7.7%

    +3.9%

    Operating Cash Flow

    166.9

    +14.4%

     

     

     

    Free Cash Flow (Non-IFRS)(a)(d)

    129.9

    -11.2%

     

     

     

    (a) Further information on certain of Takeda's Non-IFRS measures is posted on Takeda's investor relations website at https://www.takeda.com/investors/financial-results/.

    (b) Underlying growth compares two periods (quarters or years) of financial results under a common basis and is used by management to assess the business. These financial results are calculated on a constant currency basis and excluding the impact of divestitures and other amounts that are unusual, non-recurring items or unrelated to our ongoing operations.

    (c) Core Operating Profit represents net profit adjusted to exclude income tax expenses, the share of profit or loss of investments accounted for using the equity method, finance expenses and income, other operating expenses and income, amortization and impairment losses on acquired intangible assets and other items unrelated to Takeda's core operations, such as non-recurring items, purchase accounting effects and transaction related costs.

    (d) Free Cash Flow represents cash flows from operating activities, excluding acquisition of plant, property and equipment, intangible assets and investments, and any other cash that is not available to Takeda's immediate or general business use, and including proceeds from sales of property, plant, sales and redemption of investments and businesses, net of cash and cash equivalents divested.

    https://www.takeda.com/investors/financial-results/

    Reported Revenue increased +18.4% to 949.6 billion yen ($8.6B); Underlying Core Revenue increased +3.8% vs. FY2020 Q1, driven by the 14 global brands

    • Takeda's 14 global brands, with an aggregate reported revenue of 335.6 billion yen ($3.0B), posted year-over-year underlying revenue growth of +6.8% despite quarterly phasing headwinds for TAKHZYRO and IG. Takeda's 5 key business areas with 708.0 billion yen ($6.4B) in reported revenue represented 87% of core revenues1.
      • GI with 210.5 billion yen ($1.9B) in reported revenue, with underlying revenue growth of +8% spearheaded by gut-selective ENTYVIO.
      • Rare Diseases with 155.5 billion yen ($1.4 B) in reported revenue declining -3% on an underlying basis, with HAE growth impacted by phasing, but remains in line with the current plan.
      • Plasma Derived Therapy (PDT) Immunology with 107.2 billion yen ($1.0B) in reported revenue declining -2% on an underlying basis, impacted by quarterly phasing of Immunoglobulin products, with full-year outlook unchanged.
      • Oncology with 121.4 billion yen ($1.1B) in reported revenue, with underlying revenue growth +9% driven by indication expansion across the portfolio.
      • Neuroscience with 113.4 billion yen ($1.0B) in reported revenue, with underlying revenue growth +3% driven by strong rebound of Vyvanse following impact of COVID-19 in the prior year.

    ------------------------

    1 Percentage of sales are based on Core revenue; adjusted to remove JPY 133.0B from sale of Japan diabetes portfolio recorded in revenue

    Reported Operating Profit increased 48.6% Underlying Core Operating Profit Margin was 30.5% for Q1

    • Reported operating profit increased 48.6% to 248.6 billion yen ($2.2B) compared to FY2020 Q1, driven by a gain on the sale of the diabetes portfolio in Japan, lower purchase price accounting expenses and lower integration costs. These items more than offset a decrease in other operating income due to a one-time gain recorded in FY2020 Q1.
    • Underlying core operating profit for the current period declined -2.1% reflecting an increase in R&D investment and is expected to recover to "mid-single digit" growth for the full year.
    • Continued progress in debt pre-payment with approximately 242.9 billion yen toward $2.2 billion paid in FY2021 Q1.

    Achieved several critical pipeline milestones to date in FY2021

    • Moderna's COVID-19 Vaccine, approved in Japan for adults in May and expanded age indication to adolescents 12+ years old in July, with distribution underway.
    • Mobocertinib (TAK-788) filing under review in US, China, and other countries.
    • Maribavir's (TAK-620) filing and acceptance in the US and EU, with FDA granting priority review.
    • Orexin (TAK-994) granted Breakthrough Therapy designation by the FDA for Narcolepsy Type 1.
    • TAK-999 granted Breakthrough Therapy designation by the FDA for AATD2 Liver Disease
    • Collaboration with Frazier Healthcare Partners to launch HilleVax, Inc., a biopharmaceutical company to develop and commercialize TAK-214, Takeda's norovirus vaccine candidate.

    ------------------------

    2 AATD = Alpha-1 antitrypsin deficiency

    Important recognition in Q1

    • Two of Takeda's facilities in Japan and Ireland were recognized by the International Society for Pharmaceutical Engineering (ISPE) with the 2021 Facility Of the Year Awards for the use of digital and innovative technologies to enhance manufacturing capabilities.

    FY2021 Guidance

    On track towards full-year FY2021 guidance (Unchanged from May 2021)

    (billion yen)

    FY2021

    CURRENT

    FORECAST

    Underlying

    Management Guidance

    Revenue

    3,370.0

    Mid-single-digit growth

    R&D Expenses

    -522.0

     

    Reported Operating Profit

    488.0

     

    Core Operating Profit

    930.0

    Mid-single-digit growth

    Reported EPS (Yen)

    160

     

    Core EPS (Yen)

    394

    Mid-single-digit growth

    Free Cash Flow

    600-700

     

    Annual Dividend per Share (Yen)

    180

     

     

    Key assumptions in FY2021 forecast

    Company guidance reflects management's expectations for continued business momentum across Takeda's five key business areas, underlying revenue growth of its 14 global brands, and accelerated realization of cost synergies, while continuing to invest in R&D.

    FY2021 guidance reflects the following key assumptions, including (1) Takeda expects at least one 505(b)2 competitor for subcutaneous VELCADE to launch in the U.S. around mid FY2021; (2) Takeda does not expect to restart sales of Natpara in the U.S. market in FY2021; and (3) FY2021 guidance does not include the impact of any potential further divestitures beyond what has already been disclosed by Takeda.

    To date, Takeda has not experienced a material effect on its financial results as a result of the global spread of the novel coronavirus infectious disease (COVID-19). Based on currently available information, Takeda believes that its financial results for FY2021 will not be materially affected by COVID-19 and, accordingly, Takeda's FY2021 forecast reflects this belief. However, the situation surrounding COVID-19 remains highly fluid, and future COVID-19-related developments in FY2021, including new or additional COVID-19 outbreaks and additional or extended lockdowns, shelter-in-place orders or other government action in major markets, could result in further or more serious disruptions to Takeda's business, such as slowdowns in demand for Takeda's products, supply chain related issues or significant delays in its clinical trial programs. These events, if they occur, could result in an additional impact on Takeda's business, results of operations or financial condition, as well as result in significant deviations from Takeda's FY2021 forecast.

    For more details on Takeda's Q1 FY2021 results and other financial information, please visit: https://www.takeda.com/investors/financial-results/

    More information on Takeda's Environmental, Social and Governance (ESG) approach and values-based corporate governance can be found in the 2021 Annual Integrated Report for FY2020, which ended March 31, 2021. This report can be accessed on Takeda's website at: https://air.takeda.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited (TYO: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 Genetic 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.

    Important Notice

    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.

    The product names appearing in this document are trademarks or registered trademarks owned by Takeda, or their respective owners.

    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.

    Financial information

    Takeda's financial statements are prepared in accordance with International Financial Reporting Standards ("IFRS"). Convenience translations of JPY figures into USD are included for reference and have been calculated at a rate of JPY/USD of 111.05.

    Certain Non-IFRS Financial Measures

    This press release and materials distributed in connection with this press release include certain IFRS financial measures not presented in accordance with International Financial Reporting Standards ("IFRS"), such as Underlying Revenue, Core Operating Profit, Underlying Core Operating Profit, Core Net Profit, Underlying Core EPS, Net Debt, EBITDA, Adjusted EBITDA and Free Cash Flow. Takeda's management evaluates results and makes operating and investment decisions using both IFRS and non-IFRS measures included in this press release. These non-IFRS measures exclude certain income, cost and cash flow items which are included in, or are calculated differently from, the most closely comparable measures presented in accordance with IFRS. By including these non-IFRS measures, management intends to provide investors with additional information to further analyze Takeda's performance, core results and underlying trends. Takeda's non-IFRS measures are not prepared in accordance with IFRS and such non-IFRS measures should be considered a supplement to, and not a substitute for, measures prepared in accordance with IFRS (which we sometimes refer to as "reported" measures). Investors are encouraged to review the reconciliation of non-IFRS financial measures to their most directly comparable IFRS measures.

    Further information on certain of Takeda's Non-IFRS measures is posted on Takeda's investor relations website at https://www.takeda.com/investors/financial-results/

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

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  8. Takeda Will Focus its Efforts on Dengue, Zika and Pandemic Vaccines

    Takeda Pharmaceutical Company Limited ((TAK) (Takeda) and Frazier Healthcare Partners (Frazier) today announced a collaboration to launch HilleVax, Inc. (HilleVax), a biopharmaceutical company to develop and commercialize Takeda's norovirus vaccine candidate. Takeda has granted a license to HilleVax for the exclusive development and commercialization rights to its norovirus vaccine candidate, HIL-214 (formerly TAK-214), worldwide outside of Japan, in exchange for upfront consideration, as well as future cash milestones and royalties on net sales. Takeda will retain commercialization rights in Japan and HilleVax will integrate certain Japan development activities into its global…

    Takeda Will Focus its Efforts on Dengue, Zika and Pandemic Vaccines

    Takeda Pharmaceutical Company Limited ((TAK) (Takeda) and Frazier Healthcare Partners (Frazier) today announced a collaboration to launch HilleVax, Inc. (HilleVax), a biopharmaceutical company to develop and commercialize Takeda's norovirus vaccine candidate. Takeda has granted a license to HilleVax for the exclusive development and commercialization rights to its norovirus vaccine candidate, HIL-214 (formerly TAK-214), worldwide outside of Japan, in exchange for upfront consideration, as well as future cash milestones and royalties on net sales. Takeda will retain commercialization rights in Japan and HilleVax will integrate certain Japan development activities into its global development. Takeda remains committed to vaccines, and this collaboration allows Takeda to focus its global resources on dengue, COVID-19, pandemic influenza and Zika, in addition to the vaccines it currently distributes in Japan.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210729005286/en/

    HIL-214, which is a virus-like particle (VLP) based vaccine candidate, completed a randomized, placebo-controlled Phase 2b field efficacy study in 4,712 adult subjects in which HIL-214 was well-tolerated and demonstrated clinical proof of concept in preventing moderate-to-severe cases of acute gastroenteritis from norovirus infection.1 To date, the candidate has been studied in nine human clinical trials with safety data from over 4,500 subjects and immunogenicity data from over 2,000 subjects.

    "Takeda and Frazier have a history of successfully partnering together, and we are confident in HilleVax's capabilities to progress HIL-214, the most advanced norovirus vaccine candidate in development with the potential to address the huge global burden of norovirus-associated acute gastroenteritis," said Rajeev Venkayya, M.D., President of the Global Vaccine Business Unit, Takeda. "This will allow Takeda to focus its efforts and resources on our dengue vaccine, which we have begun filing for licensure around the world, our pandemic programs, and our partnership with the US Government to develop a Zika vaccine."

    Norovirus is a common intestinal infection marked by diarrhea, vomiting, abdominal cramps, nausea and sometimes fever that may lead to clinically significant dehydration.2 Norovirus is recognized as the leading cause of acute gastroenteritis across the age spectrum.3 It is estimated that norovirus causes nearly 700 million cases of illness and more than 200,000 deaths worldwide per year with significant additional economic and social burden.3 No vaccines are currently approved for norovirus infection, and HIL-214 continues to be the most advanced norovirus vaccine candidate in human clinical trials.

    "Following our successful partnership to form Phathom Pharmaceuticals, we are extremely pleased to partner again with Takeda to form HilleVax," said Tachi Yamada, M.D., co-founder of HilleVax and Venture Partner with Frazier. "Norovirus causes significant morbidity and mortality as well as tremendous economic and social costs worldwide. We believe that HIL-214 represents an important opportunity to address this immense unmet need."

    Takeda's Commitment to Vaccines

    Vaccines prevent 2 to 3 million deaths each year and have transformed global public health. For more than 70 years, Takeda has supplied vaccines to protect the health of people in Japan. Today, Takeda's global vaccine business is applying innovation to tackle some of the world's most challenging infectious diseases, such as dengue, COVID-19, pandemic influenza and Zika. Takeda's team brings an outstanding track record and a wealth of knowledge in vaccine development and manufacturing to advance a pipeline of vaccines to address some of the world's most pressing public health needs. For more information, visit www.TakedaVaccines.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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 Frazier Healthcare Partners

    Founded in 1991, Frazier Healthcare Partners is a leading provider of growth and venture capital to healthcare companies. With nearly $4.8 billion total capital raised, Frazier has invested in over 200 companies, with investment types ranging from company creation and venture capital to buyouts of profitable lower-middle market companies. The firm's Growth Buyout team invests in healthcare and pharmaceutical services, medical products and related sectors. The Life Sciences team invests in therapeutics and related areas that are addressing unmet medical needs through innovation. Frazier has offices in Seattle, WA and Menlo Park, CA, and invests broadly across the US, Canada, and Europe. For more information about Frazier Healthcare Partners, visit the company's website at http://www.frazierhealthcare.com.

    About HilleVax

    HilleVax is a biopharmaceutical company focused on the development and commercialization of novel vaccine candidates. Its initial program, HIL-214, is a virus-like particle (VLP) based vaccine candidate in development for the prevention of moderate-to-severe acute gastroenteritis caused by norovirus infection. For more information about HilleVax, visit the company's website at http://www.HilleVax.com.

    Takeda Pharmaceutical Company Limited 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.

    References

    1 Sherwood J, et al. Vaccine 2020; 38(41):6442-6449

    2 https://www.cdc.gov/norovirus/index.html [accessed 2021 April 27].

    3 Hall AJ, et al. Expert Rev Vaccines 2016;15(8):949-951

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  9. – If Approved, Investigational TAK-994 May Provide a Future Treatment Option Targeting the Orexin Deficiency Underlying NT1

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to TAK-994,1 its Phase 2 investigational oral orexin agonist, which is designed to selectively target orexin 2 receptors. TAK-994 is currently being studied for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1),2 a chronic neurological disorder that alters the sleep-wake cycle.3 EDS is a hallmark symptom of NT1 and is characterized by a person's inability to stay awake and alert throughout the day, and falling…

    – If Approved, Investigational TAK-994 May Provide a Future Treatment Option Targeting the Orexin Deficiency Underlying NT1

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to TAK-994,1 its Phase 2 investigational oral orexin agonist, which is designed to selectively target orexin 2 receptors. TAK-994 is currently being studied for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1),2 a chronic neurological disorder that alters the sleep-wake cycle.3 EDS is a hallmark symptom of NT1 and is characterized by a person's inability to stay awake and alert throughout the day, and falling asleep unintentionally or at inappropriate times on a daily basis.3,4

    The FDA's Breakthrough Therapy Designation process is designed to expedite the development and review of a drug that is intended to treat a serious condition, for which preliminary clinical evidence exists indicating it may demonstrate a substantial improvement over available therapies on at least one clinically significant endpoint.5

    The TAK-994 BTD was based, in part, on early phase and preliminary clinical data that indicates Takeda's investigational oral orexin agonist may demonstrate substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients.1 Currently, TAK-994 is being studied in an ongoing Phase 2 (TAK-994-1501) study.2 Data from the completed Phase 2 study will be presented at a future scientific conference after the study's conclusion.

    "Individuals with narcolepsy type 1 suffer from excessive daytime sleepiness, which might mean routinely falling asleep at work or in school. If approved, investigational TAK-994 has the potential to transform the way we currently treat NT1 by addressing the underlying orexin deficiency central to the disease," said Sarah Sheikh, Head, Neuroscience Therapeutic Area Unit at Takeda. "Orexin plays a critical role in regulating a person's sleep wake cycle, and supports the body's natural wake promoting pathways in the brain."

    Investigational TAK-994, which was discovered at Takeda's laboratories in Shonan, Japan, part of the open innovation center at the Shonan Health Innovation Park, is the lead candidate in Takeda's pioneering multi-asset orexin-focused franchise. This designation marks a key milestone in Takeda's accelerated development efforts to bring this important potential therapy to patients with NT1.

    Takeda's Commitment to Neuroscience

    Takeda Neuroscience is driven by the unmet need of patients with neurologic and psychiatric diseases. Our mission is to bring innovative and potentially disease-modifying medicines to these individuals. Our dedication extends beyond our marketed products and research efforts. We are committed to raising awareness for these conditions, building strategic partnerships with both industry and advocacy, educating patients and physicians, and broadening access to therapies.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    References

    1. Data on File. Takeda Pharmaceuticals. 2021.
    2. A Study of TAK-994 in Participants With Narcolepsy With or Without Cataplexy (Narcolepsy Type 1 [NT1] or Narcolepsy Type 2 [NT2]). ClinicalTrials.gov. Published April 1, 2021. https://clinicaltrials.gov/ct2/show/NCT04096560
    3. Ruoff C, Rye D. The ICSD-3 and DSM-5 guidelines for diagnosing narcolepsy: clinical relevance and practicality. Curr Med Res Opin. 2016;32(10):1611-1622. doi:10.1080/03007995.2016.1208643
    4. Brown J, Makker H. An approach to excessive daytime sleepiness in adults. BMJ. 2020;(368):m1047.
    5. Breakthrough Therapy. FDA. Published online November 3, 2018. Accessed July 23, 2021. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy

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  10. Data Include Phase 3 Results Showing that the Primary Endpoint of Spontaneous, Treated Annualized Bleeding Rates During Prophylaxis with Recombinant von Willebrand Factor in Adults with von Willebrand Disease, was Met

    12 Abstracts Presented Across Takeda's Hematology Portfolio and Pipeline Support Takeda's Commitment to Improving Patient Care Today as well as Transforming Future Care

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the results of a phase 3 trial investigating the efficacy and safety of recombinant von Willebrand factor (rVWF) prophylaxis,1 one of the 12 abstracts being presented at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress 2021. Several of these abstracts explored…

    Data Include Phase 3 Results Showing that the Primary Endpoint of Spontaneous, Treated Annualized Bleeding Rates During Prophylaxis with Recombinant von Willebrand Factor in Adults with von Willebrand Disease, was Met

    12 Abstracts Presented Across Takeda's Hematology Portfolio and Pipeline Support Takeda's Commitment to Improving Patient Care Today as well as Transforming Future Care

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the results of a phase 3 trial investigating the efficacy and safety of recombinant von Willebrand factor (rVWF) prophylaxis,1 one of the 12 abstracts being presented at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress 2021. Several of these abstracts explored the use of prophylaxis as part of Takeda's commitment to improving outcomes for patients with rare bleeding disorders.

    The prospective, phase 3, open-label, international multicenter study included 23 patients with severe von Willebrand disease (VWD). The study period was 12 months and included two arms; the prior on-demand (OD) group included patients previously taking OD VWF and the switch group included patients previously taking prophylactic plasma-derived von Willebrand factor (pdVWF) treatment [LPB0128]. Results showed that prophylaxis with rVWF effectively reduced spontaneous, treated annualized bleeding rates (sABRs) in patients previously treated OD, and the same level of hemostatic control was maintained in patients who switched from prophylaxis with pdVWF. The sABR was reduced by 91.5% on study (while receiving prophylactic rVWF) compared with historical sABR in prior OD patients, and sABR was maintained on-study in switch patients (sABR on-study: historical ratio (95% CI): 0.09 (0.02, 0.35) in prior OD arm; 0.55 (0.09, 3.52) in switch arm). No new risks were identified, with no serious adverse events related to rVWF reported.1 The pharmacokinetics (PK) of VWF:ristocetin cofactor (VWF:RCo) and FVIII pharmacodynamics (PD) [PB0917] were also studied and presented at the congress.2

    "Stopping a bleed in VWD does not prevent subsequent bleeds, and the complications and unpredictable nature of these bleeds for many OD treated patients can impact their daily life," commented Dr. Flora Peyvandi, Director of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore, Policlinico, University of Milan, Italy. "These exciting phase 3 results are important in improving understanding about the potential benefit of prophylaxis with rVWF and what this may offer patients with VWD in reducing spontaneous bleeds in the future."

    A literature review being presented at ISTH 2021 also studied the rates of bleeding-related complications, quality of life and healthcare resource utilization in VWD [PB0924].3 Data gathered from a further retrospective cohort study investigating the characteristics and management of patients with VWD in UK general practice discussed the occurrence of bleeding events in patients with a mild disease status [PB0927].4

    Additional investigational data across Takeda's hematology portfolio assessed the efficacy and safety of PK-guided prophylaxis in patients with hemophilia A. Results from the real-world German AHEAD study, which aims to examine long-term effectiveness and safety of antihemophilic factor (recombinant), rAHF (ADVATE),5 help to provide an understanding of the bleeding rates in patients with hemophilia A receiving PK-guided prophylaxis versus standard prophylaxis [PB0509].6 PK-guided prophylaxis was further studied in another real-world analysis using data from the CHESS II database to assess the impact on clinical and health resource utilization outcomes [PB0554].7 Data from a post hoc analysis of the PROPEL phase 3 study on the effects of PK-guided prophylaxis targeting specific FVIII trough levels with rurioctocog alfa pegol (ADYNOVATE) were also presented at the congress [PB0542].8

    "At Takeda we are committed to helping make a difference to the lives of patients with rare hematological disorders, both today through our broad portfolio of treatments, and in the future by developing innovative solutions to address unmet needs," commented Wolfhard Erdlenbruch, MD, Head, Global Medical Affairs Hematology. "The data presented at ISTH 2021 highlights the potential for rVWF to address unmet needs in VWD, as well as helping us to understand the clinical and resource utilization impact when individualizing treatment through PK-guided prophylaxis in hemophilia."

    Also featured at the congress was the phase 3 study design exploring TAK-755, an investigational recombinant ADAMTS13 replacement therapy being studied for use as prophylactic and OD treatment for patients with severe congenital thrombotic thrombocytopenic purpura (cTTP) [PB0837],9 and an abstract which reviewed the clinical burden of TTP [PB0843].10 Improving awareness and understanding around this rare and life-threatening blood disorder associated with ADAMTS13 deficiency,9 together with the development of TAK-755, forms part of Takeda's commitment to invest in research that has the potential to transform the future care of even more patients affected by rare hematological disorders.

    ###

    About the Phase 3 Recombinant von Willebrand Factor Study

    In the prior OD group, subjects initiated rVWF prophylactic treatment with twice-weekly infusions of 50±10 IU/kg per infusion. In the switch group, subjects initiated rVWF prophylactic treatment based on matching (±10%) the weekly dose of their prior pdVWF prophylaxis regimen.

    23 enrolled patients received rVWF prophylaxis (prior OD arm: n=13; switch arm: n=10) and 18/23 (78.3%) patients had type 3 VWD. Over the 12-month study period, 11/13 (84.6%) prior OD patients and 7/10 (70.0%) switch patients had a treated, sABR of zero, whereas, historically, 13/13 prior OD and 1/10 switch patients had an sABR >2. Benefit-risk profile was maintained, with no newly identified risks. One adverse event, a headache of moderate severity, was considered possibly related to rVWF by the investigator and led to discontinuation of rVWF and study withdrawal.

    About von Willebrand Disease (VWD)

    VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population.11 VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting.11 Due to this defect or deficiency in VWF, blood is not able to clot effectively in people with VWD, resulting in prolonged bleeding.11 There is a broad spectrum of disease severity, however, severe bleeding can occur in anyone living with VWD.12

    About Hemophilia

    Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.13 Hemophilia A is more common than hemophilia B; in 2018, hemophilia A affected about 173,711 people, whereas hemophilia B affected about 34,289 people worldwide.14

    People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.15 Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.16

    About Takeda Hematology

    Following its recent acquisition of Shire, Takeda is a leader in hemophilia with the longest heritage and market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years driving innovation for patients17 and a broad portfolio of 11 products across multiple bleeding disorders.18 Our experience as a leader in hematology means we are well prepared to meet today's needs as we pursue future developments in the care of blood disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and full protection against bleeds, and more personalized patient care.

    About VEYVONDI/VONVENDI

    In Europe, VEYVONDI is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of hemorrhage and treatment/prevention of surgical bleeding. VEYVONDI should not be used in the treatment of hemophilia A.

    For full EU Summary of Product Characteristics, including approved indication(s) and important safety information, please visit here.

    In the US the product has been approved under the trade name VONVENDI® [von Willebrand factor (Recombinant)] and is indicated for use in adults (age 18 and older) diagnosed with VWD for on-demand treatment and control of bleeding episodes or perioperative management of bleeding.

    VEYVONDI SAFETY INFORMATION FOR EUROPE

    Please consult the VEYVONDI Summary Product Characteristics (SmPC) here before prescribing.

    Contraindications

    Hypersensitivity to the active substance or to any of the excipients listed in section 6.1 of the SmPC.

    Known allergic reaction to mouse or hamster proteins.

    Warnings and Precautions

    In actively bleeding patients it is recommended to co-administer a FVIII product with VEYVONDI as a first line treatment and depending on the FVIII activity levels (see 4.2 of the SmPC).

    Hypersensitivity reactions (including anaphylaxis) have occurred. Patients and/or their caregivers should be informed of the early signs of hypersensitivity reactions, which may include but are not limited to tachycardia, tightness of the chest, wheezing and/or acute respiratory distress, hypotension, generalised urticaria, pruritus, rhinoconjunctivitis, angioedema, lethargy, nausea, vomiting, paresthesia, restlessness, and may progress to anaphylactic shock.

    VEYVONDI contains trace amounts of mouse immunoglobulin G (MuIgG) and hamster proteins (less than or equal to 2 ng/IU VEYVONDI). VEYVONDI contains trace amounts of recombinant coagulation factor VIII.

    There is a risk of occurrence of thrombotic events, particularly in patients with known clinical or laboratory risk factors for thrombosis including low ADAMTS13 levels.

    In patients requiring frequent doses of VEYVONDI in combination with recombinant factor VIII, plasma levels for FVIII:C activity should be monitored to avoid sustained excessive FVIII:C plasma levels, which may increase the risk of thrombotic events. Any FVIII that would be administered along with VEYVONDI should be a pure FVIII product.

    Patients with VWD, especially Type 3, may develop neutralising antibodies (inhibitors) to von Willebrand factor. If the expected plasma levels of (VWF:RCo) are not attained, or if bleeding is not controlled with an appropriate dose, an appropriate assay should be performed to determine if a von Willebrand factor inhibitor is present.

    This medicinal product contains 5.2 mg sodium in each 650 IU vial or 10.4 mg sodium in each 1300 IU vial. This is equivalent to 2.2% of the WHO recommended maximum daily intake of 2 g sodium for an adult, assuming a body weight of 70 kg and a dose of 80 IU/kg body weight. This is to be taken into consideration by patients on a controlled sodium diet.

    Adverse Reactions

    During treatment with VEYVONDI, the following adverse reactions may occur: hypersensitivity or allergic reactions, thromboembolic events, inhibitor formation against VWF.

    The following adverse reactions, grouped by MedDRA SOC and frequency, were reported in clinical trials, post-authorisation safety studies, or post-marketing reporting: dizziness, vertigo, dysgeusia, tremor, tachycardia, deep venous thrombosis, hypertension, hot flush, vomiting, nausea, pruritus generalized, chest discomfort, infusion site paraesthesia, electrocardiogram T wave inversion, heart rate increased (common ≥ 1/100 to < 1/10), infusion-related reaction, anaphylactic reaction (frequency not known).

    Description of selected adverse reactions

    In clinical trials, one case of clinically asymptomatic deep vein thrombosis (DVT) was reported for a subject in the surgery study who had total hip replacement.

    In addition, one post-marketing case of DVT has been reported spontaneously for an elderly patient.

    For more information, please refer to the VEYVONDI Summary of Product Characteristics here.

    For US specific safety information, please refer to the VONVENDI US Prescribing Information here.

    E.U. Indication and Important Safety Information

    About ADVATE (human coagulation factor VIII (rDNA), octocog alfa)

    Please consult the ADVATE Summary of Product Characteristics (SmPC) before prescribing, particularly in relation to dosing and treatment monitoring (here).

    Contraindications

    Hypersensitivity to the active substance or to any of the excipients listed in the SmPC or to mouse or hamster proteins.

    Special warnings and precautions for use

    The product contains traces of mouse and hamster proteins. If symptoms of hypersensitivity occur, patients should be advised to discontinue use of the product immediately and contact their physician.

    Patients should be informed of the early signs of hypersensitivity reactions including hives, generalised urticaria, tightness of the chest, wheezing, hypotension and anaphylaxis.

    The formation of neutralising antibodies (inhibitors) to factor VIII is a known complication in the management of individuals with haemophilia A. These inhibitors are usually IgG immunoglobulins directed against the factor VIII procoagulant activity, which are quantified in Bethesda Units (BU) per mL of plasma using the modified assay.

    In general, all patients treated with coagulation factor VIII products should be carefully monitored for the development of inhibitors by appropriate clinical observations and laboratory tests. If the expected factor VIII activity plasma levels are not attained, or if bleeding is not controlled with an appropriate dose, testing for factor VIII inhibitor presence should be performed.

    This medicinal product contains 10mg sodium per vial, equivalent to 0.5% of the World Health Organisation recommended maximum daily intake of 2g sodium for an adult.

    Adverse Reactions

    Very common (≥1/10)

    Factor VIII inhibition in previously untreated patients (PUPs)

    Common (≥1/100 to <1/10)

    Headache, Pyrexia

    Uncommon (≥1/1000 to <1/100)

    Influenza, Laryngitis, Factor VIII inhibition in previously treated patients (PTPs), Lymphangitis, Dizziness, Dysgeusia, Memory impairment, Migraine, Syncope, Tremor, Eye inflammation, Palpitations, Haematoma, Hot flush, Pallor, Dyspnoea, Abdominal pain upper, Diarrhoea, Nausea, Vomiting, Hyperhidrosis, Pruritus, Rash, Urticaria, Chest discomfort, Chest pain, Chills, Feeling abnormal, Peripheral oedema, Vessel puncture site haematoma, Coagulation factor VIII level decreased, Haematocrit decreased, Laboratory test abnormal, Monocyte Count increased, Post procedural complication, Post procedural haemorrhage, Procedural site reaction

    Not known

    Anaphylactic reaction, Hypersensitivity, Fatigue, Injection site reaction, Malaise

     

    For the ADVATE US Prescribing Information, please refer here.

    About ADYNOVATE/ADYNOVI®▼ (Pegylated human coagulation factor VIII (rDNA), rurioctocog alfa pegol)

    Detailed Safety Information

    Please consult the ADYNOVI Summary of Product Characteristics (SmPC) before prescribing, particularly in relation to dosing and treatment monitoring (here)

    Contraindications

    Hypersensitivity to the active substance, to the parent molecule octocog alfa or to any of the excipients listed in the SmPC. Known allergic reaction to mouse or hamster protein

    Special warnings and precautions for use

    The medicinal product contains traces of mouse and hamster proteins. If symptoms of hypersensitivity occur, patients should be advised to discontinue use of the medicinal product immediately and contact their physician. Patients should be informed of the early signs of hypersensitivity reactions including hives, generalised urticaria, tightness of the chest, wheezing, hypotension, and anaphylaxis.

    The formation of neutralising antibodies (inhibitors) against factor VIII is a known complication in the management of individuals with haemophilia A. These inhibitors are usually IgG immunoglobulins directed against the factor VIII procoagulant activity, which are quantified in Bethesda Units (BU) per mL of plasma using the modified assay.

    In general, all patients treated with coagulation factor VIII should be carefully monitored for the development of inhibitors by appropriate clinical observations and laboratory tests. If the expected factor VIII activity plasma levels are not attained, or if bleeding is not controlled with an appropriate dose, testing for factor VIII inhibitor presence should be performed.

    This medicine contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially ‘sodiumfree'.

    Adverse Reactions

    Very common (≥1/10)

    Headache

    Common (≥1/100 to <1/10)

    Dizziness, Diarrhoea, Nausea, Rash

    Uncommon (≥1/1000 to <1/100)

    Factor VIII inhibition in previously-treated patients (PTPs), Hypersensitivity, Ocular hyperaemia, Flushing, Drug eruption, Eosinophil count increased, Infusion related reaction

    For the ADYNOVATE US Prescribing Information, please refer here.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    ###


    1 ISTH 2021. Abstract [LPB0128]. Phase 3 Trial Results: Prophylaxis With Recombinant Von Willebrand Factor in Patients With Severe von Willebrand Disease.

    2 ISTH 2021. Abstract [PB0917]. Pharmacokinetics/Pharmacodynamics (PK/PD) of Recombinant von Willebrand Factor (vonicog alfa) in Adult Patients with von Willebrand Disease (VWD) During Prophylactic Treatment.

    3 ISTH 2021. Abstract [PB0924]. Burden of Illness in Patients with von Willebrand Disease: A Systematic Review.

    4 ISTH 2021. Abstract [PB0927]. Characteristics and Treatment of Patients with von Willebrand Disease (VWD) in General Practice Settings in the United Kingdom.

    5 German Clinical Trials Register. DRKS-ID: DRKS00000556. Available at: https://www.drks.de/drks_web/navigate.do?navigationId=trial.HTML&TRIAL_ID=DRKS00000556. Last accessed: June 2021.

    6 ISTH 2021. Abstract [PB0509]. Does Pharmacokinetic-guided Prophylaxis with Antihemophilic Factor (Recombinant) Improve Bleeding Rates over Standard Prophylaxis? Real-world Data from the AHEAD German Study.

    7 ISTH 2021. Abstract [PB0554]. The Impact of PK-guided Prophylaxis on Clinical Outcomes and Resource Utilization in Hemophilia A Patients: Real-world Evidence From the CHESS II Study.

    8 ISTH 2021. Abstract [PB0542]. Looking Beyond Fixed-Dose Rurioctocog Alfa Pegol Prophylaxis: Post hoc Analysis of PK-guided Regimens From the PROPEL Phase 3 Study.

    9 ISTH 2021. Abstract [PB0837]. Design of a phase 3, randomized, controlled study of prophylactic and on-demand treatment with recombinant ADAMTS13 for patients with severe congenital thrombotic thrombocytopenic purpura.

    10 ISTH 2021. Abstract [PB0483]. The Clinical Burden of Congenital and Immune-mediated Thrombotic Thrombocytopenic Purpura: A Retrospective Cohort Analysis.

    11 National Hemophilia Foundation. Von Willebrand Disease. Available at:https://www.hemophilia.org/bleeding-disorders-a-z/types/von-willebrand-disease. Last accessed June 2021.

    12 Connell NT, et al. ASH ISTH NHF WFH 2021 Guidelines on the Management of von Willebrand Disease. Blood Adv (2021) 5 (1): 301–325.

    13 World Federation of Hemophilia. Introduction to Hemophilia: What is Hemophilia? Available at: https://bit.ly/356zUDQ. Last accessed June 2021.

    14 20 Anniversary Report on the Annual Global Survey 2018. World Federation of Hemophilia. Available at: https://bit.ly/3hGDUzU. Last accessed June 2021.

    15 World Federation of Hemophilia. Introduction to Hemophilia: Treatment. Available at: https://bit.ly/3b7Pfry. Last accessed June 2021.

    16 NHS. Haemophilia: Treatment. Available at: https://bit.ly/3oqhAwY. Last accessed June 2021.

    17 Takeda Website. Rare Diseases. Available at: https://bit.ly/3n9gvIN. Last accessed June 2021.

    18 Takeda Website. U.S. Product List. Available at: https://bit.ly/35a6HYF. Last accessed June 2021.

    Copyright 2021 Takeda Pharmaceutical Company Limited. All rights reserved. Takeda and the Takeda logo are registered trademarks of Takeda Pharmaceutical Company limited.

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  11. Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that the Ministry of Health, Labour and Welfare of Japan (MHLW) and Takeda Pharmaceutical Company Limited (NYSE:TAK) have agreed to purchase and distribute an additional 50 million doses of Moderna's COVID-19 vaccine and its updated variant booster vaccine candidate, if authorized, to begin delivery in 2022.

    This new supply agreement is in addition to the prior agreement for 50 million doses in 2021 resulting in a total of 100 million doses for Japan. Moderna is responsible for the manufacture and supply of Moderna's vaccine candidate, and Takeda, with the support of the MHLW and Moderna, is responsible for all import…

    Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that the Ministry of Health, Labour and Welfare of Japan (MHLW) and Takeda Pharmaceutical Company Limited (NYSE:TAK) have agreed to purchase and distribute an additional 50 million doses of Moderna's COVID-19 vaccine and its updated variant booster vaccine candidate, if authorized, to begin delivery in 2022.

    This new supply agreement is in addition to the prior agreement for 50 million doses in 2021 resulting in a total of 100 million doses for Japan. Moderna is responsible for the manufacture and supply of Moderna's vaccine candidate, and Takeda, with the support of the MHLW and Moderna, is responsible for all import, local regulatory, development and distribution activities in Japan for these additional 50 million doses beginning in 2022.

    "We thank the MHLW and Takeda for their support and for partnering with us to bring our mRNA COVID-19 vaccine to Japan," said Stéphane Bancel, Moderna's Chief Executive Officer. "We remain committed to making our vaccine available around the world as we seek to address the pandemic."

    About Moderna

    In 10 years since its inception, Moderna has transformed from a science research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale and at unprecedented speed. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use of one of the earliest and most-effective vaccines against the COVID-19 pandemic.

    Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Today, 24 development programs are underway across these therapeutic areas, with 15 programs having entered the clinic. Moderna has been named a top biopharmaceutical employer by Science for the past six years. To learn more, visit www.modernatx.com.

    Forward Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the Company's development of a vaccine to protect against the SARS-CoV-2 virus (mRNA-1273, also referred to as the Moderna COVID-19 Vaccine), as well as booster vaccine candidates; the Company's agreement to supply the vaccine to the government of Japan; the arrangements between the Company, Takeda and Japanese regulatory authorities for the importation of the vaccine; and the anticipated number of doses and timing for delivery of vaccine supply. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading "Risk Factors" in Moderna's most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date hereof.

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  12. Takeda Pharmaceutical Company Limited ("Takeda") (TYO:4502) (NYSE:TAK) today announced that it has filed its Corporate Governance Report with the Tokyo Stock Exchange ("TSE") in accordance with the regulations* for TSE listed companies. The Report can be accessed on Takeda's website at: https://www.takeda.com/who-we-are/corporate-governance/.

    This report is prepared based on the principles of the Corporate Governance Code as in effect as of June 1, 2018. Takeda will submit a revised Corporate Governance Report based on the Corporate Governance Code as in effect as of June 11, 2021, by the end of December 2021.

    Takeda's latest Corporate Governance Report can be found at the information disclosure site operated by the Japan Exchange Group which…

    Takeda Pharmaceutical Company Limited ("Takeda") (TYO:4502) (NYSE:TAK) today announced that it has filed its Corporate Governance Report with the Tokyo Stock Exchange ("TSE") in accordance with the regulations* for TSE listed companies. The Report can be accessed on Takeda's website at: https://www.takeda.com/who-we-are/corporate-governance/.

    This report is prepared based on the principles of the Corporate Governance Code as in effect as of June 1, 2018. Takeda will submit a revised Corporate Governance Report based on the Corporate Governance Code as in effect as of June 11, 2021, by the end of December 2021.

    Takeda's latest Corporate Governance Report can be found at the information disclosure site operated by the Japan Exchange Group which consists of the TSE and other exchanges in Japan at:

    https://www2.tse.or.jp/tseHpFront/CGK020010Action.do?Show=Show

    Takeda also furnished the English version of the Corporate Governance Report with the U.S. Securities and Exchange Commission (the "SEC"), available at www.sec.gov.

    As for the general information about the Corporate Governance Report, please refer to the Japan Exchange Group website at: https://www.jpx.co.jp/english/equities/listing/cg/01.html.

    Additionally, Takeda's corporate governance policy as well as overall Environmental, Social and Governance information is explained in detail in its 2021 Annual Integrated Report for the fiscal year ended March 31, 2021. This report can be accessed on Takeda's website at: https://air.takeda.com

    *TSE's Securities Listing Regulations [Rule 419].

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited (TYO: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.

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  13. Analysis of Safety and Efficacy for up to 2.5 years with TAKHYZRO is Consistent with Initial Period of Treatment, Building on Growing Body of Evidence on the Long-term Safety and Efficacy

    Final Patient Subgroup Analysis Suggests Reductions of HAE Attacks Across Range of Patient Demographics and Disease Characteristics with TAKHZYRO

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced results from two final analyses from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study Open-label Extension (OLE), which evaluated the long-term safety (primary endpoint) and efficacy of TAKHZYRO® (lanadelumab) 300 mg every two weeks for up to 2.5 years. In the first analysis, the mean (min, max) reduction in the attack…

    Analysis of Safety and Efficacy for up to 2.5 years with TAKHYZRO is Consistent with Initial Period of Treatment, Building on Growing Body of Evidence on the Long-term Safety and Efficacy

    Final Patient Subgroup Analysis Suggests Reductions of HAE Attacks Across Range of Patient Demographics and Disease Characteristics with TAKHZYRO

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced results from two final analyses from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study Open-label Extension (OLE), which evaluated the long-term safety (primary endpoint) and efficacy of TAKHZYRO® (lanadelumab) 300 mg every two weeks for up to 2.5 years. In the first analysis, the mean (min, max) reduction in the attack rate compared to baseline observed in the study population (N=212) was of 87.4 percent (-100; 852.8), and the median reduction was 97.7 percent and patients received treatment for a mean (standard deviation) duration of 29.6 (8.2) months.1 At steady state – day 70 to the end of the treatment period – attack rates were further reduced to a mean of 92.4 percent and a median reduction of 98.2 percent.2,3 An additional analysis further suggests TAKHZYRO was a well-tolerated treatment that prevented HAE attacks over an extended planned 132 week treatment period across specific HAE patient demographic and disease characteristic subgroups.3 These data are being presented at the 2021 European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress in Krakow, Poland and Madrid, Spain.

    "Hereditary angioedema is a lifelong condition and research shows that concerns about another attack can limit the way patients lead their lives," said Prof. Markus Magerl, M.D., Department of Dermatology, Venereology and Allergology, Charité – Universitätsmedizin in Berlin, Germany. "The efficacy of TAKHZYRO to prevent HAE attacks over the long term will be an important consideration for patients and physicians as they develop a treatment plan for patients that is focused on reducing the number of HAE attacks."

    The original Phase 3 HELP Study was conducted in 125 patients aged 12 years and older over 26 weeks, making it the largest randomized, controlled prevention study in HAE, with the longest active treatment duration, to date.4 The HELP Study OLE was designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO for up to 2.5 years. The complete results were based on data collected between May 2016 and October 2019 and included 109 rollover patients who were originally evaluated in the HELP Study, and 103 eligible non-rollover patients who did not participate in the initial study but had experienced at least one HAE attack within the previous 12 weeks.1

    Attack-free status during steady state of lanadelumab treatment in patients with hereditary angioedema: findings from the HELP open-label extension study (Electronic Poster Presentation: Abstract #342)

    Results from the HELP Study OLE found that TAKHZYRO sustained efficacy in the prevention of HAE attacks by reducing attack rates in a treatment period of up to 132 weeks. TAKHZYRO, which has a half-life of approximately 14 days, is expected to reach steady state at approximately 70 days. The HELP Study OLE analysis of attack-free status during the steady state period showed that the efficacy of TAKHZYRO 300 mg administered subcutaneously every two weeks in rollover patients was consistent with the original findings from the HELP Study.2

    The mean (min, max) reduction in the attack rate compared to baseline observed in the study population (N=212) was of 87.4 percent (-100; 852.8), and the median reduction was 97.7 percent (98.0 percent rollovers, 96.9 percent non-rollovers).1,2 At steady state, attack rates were further reduced to a mean of 92.4 percent (-100, 0.44) comprised of 92.7 percent rollovers (-100, -40.9) and 91.9 percent non-rollovers (-100, 0.44) and a median reduction of 98.2 percent (98.4 percent rollovers, 97.3 percent non-rollovers). During the first six months of treatment after day 70 during the steady-state period, 58.6 percent of patients (n=119) were attack-free, 54.7 percent rollovers and 62.9 percent non-rollovers. The maximum duration of attack-free period after day 70 ≥ 6 months was 83.7 percent and ≥ 12 months was 70 percent. The means of the average and maximum duration of attack-free period during steady state were 14.8 and 18.6 months, respectively, with 70.0 percent of patients (n=142) having a maximum duration of attack-free period greater than 12 months.2

    Long-term prevention of attacks with lanadelumab across subgroups of patients with hereditary angioedema (HAE): final results from the HELP open-label extension study (Oral Presentation: Abstract #392)

    In a further HELP Study OLE analysis, treatment with TAKHZYRO 300 mg every two weeks was well-tolerated and effectively reduced attack rates over an extended treatment period across different patient demographic and disease characteristics, including patient age, gender, race, HAE type, body mass index, history of long-term prophylaxis use, and baseline attack rate.3

    The safety profile of TAKHZYRO was comparable across all evaluated subgroups with treatment-related TEAEs occurring in 54.7 percent of patients (n=116) and the most common being injection-site pain.3

    "For more than a decade, we've listened to the HAE community to further understand the need for long-term, preventive targeted therapies and have committed our resources to developing treatment options," said Neil Inhaber, M.D., Vice President, Global Medical Head, HAE and Transplant at Takeda. "These analyses further assert the important role TAKHZYRO can play in the lives of people who live with HAE."

    About The HELP StudyOpen-label Extension

    The HELP (Hereditary Angioedema Long-term Prophylaxis) Study Open-label Extension (OLE) is an evaluation of the long-term efficacy and safety of TAKHZYRO in hereditary angioedema (HAE) patients of at least 12 years of age and older. Two hundred and twelve patients received treatment with TAKHZYRO at the start of the OLE Study (109 rollover patients originally evaluated in the HELP Study and who continued into the OLE, and 103 eligible patients who did not participate in the HELP Study but who had experienced at least one attack in the last 12 weeks). Rollover patients received a dose of 300 mg TAKHZYRO on Day 0 and then every two weeks after their first attack. Non-rollover patients were treated with one 300 mg dose every two weeks, beginning on Day 0. One hundred and ninety-six participants completed at least 12 months of treatment and 173 participants completed at least 30 months of treatment.1

    About Hereditary Angioedema

    Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of oedema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful.5,6,7 Attacks that obstruct the airways can cause asphyxiation and are potentially life threatening.7,8 HAE affects an estimated 1 in 50,000 people worldwide. It is often under recognized, under diagnosed and under treated.5,7,8

    Takeda in Hereditary Angioedema

    Hereditary Angioedema (HAE), like so many other rare diseases, is highly complex, and patients, their families and caregivers often undergo years of strain trying to understand their disease, get a definitive diagnosis and gain access to the medicines they need. At Takeda we are a committed champion for the patients we serve. Every individual living with HAE is unique and by listening and reacting to their needs, we translate the insights we gain into innovative solutions – from diagnosis to ongoing management. Advancing the science is crucial to the way we operate and we are bold in our mission to accelerate diagnosis and develop treatments that will make a difference to the lives of HAE patients, their support networks and those medical professionals who care for them.

    About TAKHZYRO® (lanadelumab)

    TAKHZYRO® (lanadelumab) is indicated for routine prevention of recurrent attacks HAE in patients aged 12 years and older. TAKHZYRO is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity. TAKHZYRO is produced in Chinese Hamster Ovary (CHO) cells by recombinant DNA technology.9

    TAKHZYRO is formulated for subcutaneous administration and has a half-life of approximately two weeks in patients with HAE. TAKHZYRO is intended for the self-administration or administration by a caregiver, only after training by a healthcare professional.9

    TAKHZYRO is not intended for treatment of acute HAE attacks.9

    Depending on regional marketing authorization, TAKHZYRO is available as a 300 mg dose in a vial or pre-filled syringe. Please consult local prescribing information for more information.

    TAKHZYRO Safety Information for Europe

    Please consult the TAKHZYRO Summary Product Characteristics (SmPC) before prescribing.

    TAKHZYRO treatment should be initiated under the supervision of a physician experienced in the management of patients with hereditary angioedema (HAE). TAKHZYRO may be self-administered or administered by a caregiver only after training on SC injection technique by a healthcare professional.9

    Contraindication

    Hypersensitivity to the active substance or to any of the excipients.9

    Warnings and Precautions

    Traceability: In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.9

    Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, administration of TAKHZYRO must be stopped immediately and appropriate treatment must be initiated.9

    General: TAKHZYRO is not intended for treatment of acute HAE attacks. In case of a breakthrough HAE attack, individualized treatment should be initiated with an approved rescue medication. There are no available clinical data on the use of lanadelumab in HAE patients with normal C1-INH activity.9

    Interference with coagulation test: Lanadelumab can increase activated partial thromboplastin time (aPTT) due to an interaction of lanadelumab with the aPTT assay. The reagents used in the aPTT laboratory test initiate intrinsic coagulation through the activation of plasma kallikrein in the contact system. Inhibition of plasma kallikrein by lanadelumab can increase aPTT in this assay. None of the increases in aPTT in patients treated with TAKHZYRO were associated with abnormal bleeding adverse events. There were no differences in international normalised ratio (INR) between treatment groups.9

    Sodium content: This medicinal product contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially 'sodium-free'.9

    Interactions

    No dedicated drug-drug interaction studies have been conducted. Based on the characteristics of lanadelumab, no pharmacokinetic interactions with co-administered medicinal products is expected.9

    As expected, concomitant use of the rescue medication C1 esterase inhibitor results in an additive effect on lanadelumab-cHMWK response based on the mechanism of action (MOA) of lanadelumab and C1 esterase inhibitor.9

    Immunogenicity

    Treatment with lanadelumab has been associated with development of treatment emergent anti-drug antibodies (ADA) in 11.9% (10/84) of subjects. All antibody titres were low. The ADA response was transient in 20% (2/10) of ADA positive subjects. 2.4% (2/84) of lanadelumab-treated subjects tested positive for neutralizing antibodies.9

    The development of ADA including neutralising antibodies against TAKHZYRO did not appear to adversely affect the pharmacokinetic (PK) and pharmacodynamics (PD) profiles or clinical response.9

    Adverse Reactions

    The most commonly observed adverse reaction (52.4%) associated with TAKHZYRO was injection site reactions (ISR) including injection site pain, injection site erythema and injection site bruising. Of these ISRs, 97% were of mild intensity, 90% resolved within 1 day after onset with a median duration of 6 minutes.9

    Hypersensitivity reaction (mild and moderate pruritus, discomfort and tingling of tongue) was observed (1.2%)

    Very common

    (frequency ≥1/10):

    Injection site reactions*

    Common

    (≥1/100 to <1/10):

    Hypersensitivity**, dizziness, rash maculopapular, myalgia, alanine aminotransferase increased, aspartate aminotransferase increased.

    *Injection site reactions include: pain, erythema, bruising, discomfort, haematoma, haemorrhage, pruritus, swelling, induration, paraesthesia, reaction, warmth, oedema and rash.

    ** Hypersensitivity includes: pruritus, discomfort and tingling of tongue.

    For European Union Summary of Product Characteristics, please visit https://www.ema.europa.eu/en/documents/product-information/takhzyro-epar-product-information_en.pdf.

    For full U.S. Prescribing Information, including the approved indication and important safety information, please visit https://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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/reports/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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.


    1 Banerji A, Hao J, Ming Y et al; Long-Term Efficacy and Safety of Lanadelumab: Final Results from the HELP Open-Label Extension Study. ACAAI 2020.

    2 Magerl M, Johnston D, et al. Attack-free status during steady state of lanadelumab treatment in patients with hereditary angioedema: findings from the HELP open-label extension study. EAACI 2021.

    3 Maurer M, Bernstein J, et al. Long-term prevention of attacks with lanadelumab across subgroups of patients with hereditary angioedema (HAE): final results from the HELP open-label extension study. EAACI 2021.

    4 Banerji A, Riedl MA, Bernstein JA, et al; for the HELP Investigators. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks: a randomized clinical trial. JAMA. 2018;320(20):2108-2121.

    5 Cicardi M, Bork K, Caballero T, et al; on behalf of HAWK (Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012; 67(2):147-157.

    6 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

    7 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.

    8 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.

    9 TAKHZYRO (lanadelumab) European Summary of Product Characteristics.

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  14. Northampton, MA

    --News Direct--

    Despite the unprecedented challenges of the past year, we continued to meet the needs of patients and deliver positive results both for shareholders and the communities we serve. Read our 2021 Annual Integrated Report to learn more: https://bit.ly/3haa9Yo

    View additional multimedia and more ESG storytelling from Takeda on 3blmedia.com

    View source version on newsdirect.com: https://newsdirect.com/news/timeless-values-life-changing-progress-832888865

    2021 News Direct Corp.

    Northampton, MA

    --News Direct--

    Despite the unprecedented challenges of the past year, we continued to meet the needs of patients and deliver positive results both for shareholders and the communities we serve. Read our 2021 Annual Integrated Report to learn more: https://bit.ly/3haa9Yo

    View additional multimedia and more ESG storytelling from Takeda on 3blmedia.com

    View source version on newsdirect.com: https://newsdirect.com/news/timeless-values-life-changing-progress-832888865

    2021 News Direct Corp.

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  15. Takeda Pharmaceutical Company Limited ("Takeda") (NYSE:TAK) today announced that it has filed its Annual Report on Form 20-F for the fiscal year ended March 31, 2021 (the "Annual Report") with the U.S. Securities and Exchange Commission (the "SEC"). Takeda filed the Annual Report with the SEC on June 29, 2021, and the Annual Report can be accessed on Takeda's website at: https://www.takeda.com/investors/sec-filings/.

    In addition, Takeda will also provide a hard copy of the Annual Report, which includes its most recent complete audited financial statements free of charge to any shareholder upon request. Please contact Takeda Investor Relations by e-mail at takeda.ir.contact@takeda.com.

    Additionally, Takeda also published today its 2021 Annual…

    Takeda Pharmaceutical Company Limited ("Takeda") (NYSE:TAK) today announced that it has filed its Annual Report on Form 20-F for the fiscal year ended March 31, 2021 (the "Annual Report") with the U.S. Securities and Exchange Commission (the "SEC"). Takeda filed the Annual Report with the SEC on June 29, 2021, and the Annual Report can be accessed on Takeda's website at: https://www.takeda.com/investors/sec-filings/.

    In addition, Takeda will also provide a hard copy of the Annual Report, which includes its most recent complete audited financial statements free of charge to any shareholder upon request. Please contact Takeda Investor Relations by e-mail at takeda.ir.contact@takeda.com.

    Additionally, Takeda also published today its 2021 Annual Integrated Report for the fiscal year ended March 31, 2021. It can be accessed on Takeda's website at: https://air.takeda.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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.

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  16. DUBAI, UAE, June 16, 2021 /PRNewswire/ -- Takeda Pharmaceuticals Company (TSE: 4502) (NYSE:TAK) ("Takeda"), a global, values-based, R&D-driven biopharmaceutical company, headquartered in Japan, is celebrating its 240th founding anniversary in June 2021, marking a significant milestone in bringing Better Health and a Brighter Future to people worldwide.

    The company was first founded by Chobei Takeda in 1781 in Osaka, Japan, with the Pharmaceutical Manufacturing Business starting in 1895 and the Takeda Pharmaceutical Company established in 1915. Today the company has a global presence in over 80 countries with 80 manufacturing and three research sites. Takeda's R&D efforts are currently focused on four therapeutic areas: oncology, rare diseases, neuroscience, and gastroenterology, with targeted R&D investments in plasma-derived therapies and vaccines.

    Dr. Mahender Nayak, Area Head, ICMEA (India, C.I.S., Middle East, Turkey, and Africa) said,

    "With roots as a family-startup, our incredible legacy is anchored by our values, and guided by a strong commitment to the highest ethical standards. Our growth will continue to be driven by decisions and actions based on Patient-Trust Reputation-Business, in that order. At both global and area levels, we will continue to deliver sustainable, profitable growth through ensuring patient access to our innovative treatments. As we begin our 240th year, Takeda seeks to deliver even greater value for our patients, people and the planet."

    He added, "Our focus here across ICMEA is on our innovative portfolio that will see multiple launches planned in 2021 and beyond. Through strategic partnerships, we aim to increase the number of patient lives positively impacted by various initiatives such as Takeda's global Access to Medicines initiatives.  Among recent examples is our work in supporting controlling mass disease outbreaks. Studies[1] suggest that our dengue vaccine candidate could help with outbreak prevention, reducing rates of hospitalization and protecting people from dengue regardless of their previous exposure throughout three years."

    Takeda-ism incorporates Integrity, Fairness, Honesty and Perseverance, with Integrity at the core. Takeda aims to achieve a revenue goal of JPY 1 trillion[2] by FY2030 for its Growth and Emerging Markets Business Unit which includes ICMEA in its remit. Takeda has also achieved carbon neutrality[3] in its value chain in FY 2019, delivered by a continued focus on internal energy conservation measures, procurement of green energy, and investment in renewable energy certificates and high-quality, verified carbon offsets, including more than 30 renewable energy and carbon offset projects across 12 countries. Takeda's ICMEA headquarters in Dubai have received silver level LEED certification.

    Globally, the company has almost 50,000 employees. The ICMEA Area comprises 38 countries with around 1,000 employees. The region has a gender mix of 58 percent women and 42 percent women with more than 54 nationalities. Takeda was named global Top Employer[4] for fourth consecutive year.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited (NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, 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. For more information, visit https://www.takeda.com.

    Important Notice

    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; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; 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/reports/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.

    [1] https://www.takeda.com/newsroom/newsreleases/2021/potential-impact-of-takedas-dengue-vaccine-candidate-reinforced-by--long-term-safety-and-efficacy-results/

    [2] https://www.takeda.com/494098/siteassets/system/investors/investor--events/gem-ir-day-presentation.pdf

    [3] takeda.com/newsroom/newsreleases/2021/takeda-achieved-carbon-neutrality-in-2020/

    [4] https://www.takeda.com/newsroom/newsreleases/2021/takeda-named-global-top-employer-for-fourth-consecutive-year/#:~:text=Takeda%20Named%20Global%20Top%20Employer%20for%20Fourth%20Consecutive%20Year,-January%2024%2C%202021&text=OSAKA%2C%20Japan%20and%20CAMBRIDGE%2C%20Massachusetts,Employer%C2%AE%20certification%20for%202021

    Logo - https://mma.prnewswire.com/media/1534807/Takeda_Logo.jpg

     

     

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  17. DUBAI, UAE, June 9, 2021 /PRNewswire/ -- Takeda (TSE: 4502) (NYSE:TAK) has introduced the "In Their Shoes" platform, an award-winning, immersive simulation program to promote greater awareness and empathy for patients with Inflammatory bowel disease (IBD), a condition to describe Crohn's disease and ulcerative colitis, to a group of healthcare providers in the Middle East the first time. 

    The subscription-based program launched with a two-day simulation where healthcare providers and Takeda employees learned about the condition most effectively and profoundly: by "living with it."

    The program utilized a mobile application to guide participants through some of the everyday struggles patients face. An "IBD kit" of materials was used to participate…

    DUBAI, UAE, June 9, 2021 /PRNewswire/ -- Takeda (TSE: 4502) (NYSE:TAK) has introduced the "In Their Shoes" platform, an award-winning, immersive simulation program to promote greater awareness and empathy for patients with Inflammatory bowel disease (IBD), a condition to describe Crohn's disease and ulcerative colitis, to a group of healthcare providers in the Middle East the first time. 

    The subscription-based program launched with a two-day simulation where healthcare providers and Takeda employees learned about the condition most effectively and profoundly: by "living with it."

    The program utilized a mobile application to guide participants through some of the everyday struggles patients face. An "IBD kit" of materials was used to participate in "challenges" prompted by the app. These challenges were designed to simulate several physical and emotional aspects of the disease. Through role-play scenarios and interactions with actors playing nurses, and healthcare professionals, participants gained unparalleled insight into the impact that IBD can have across all aspects of someone's life, including professional and personal relationships. While a simulation can never fully replicate the pain and disruption associated with IBD, physical discomfort and regular interruptions incorporated into the program helped participants walk "in the shoes" of someone with IBD.

    Toby Shepard, Head of Medical Affairs for Takeda in ICMEA, said, "The platform has been highly successful in helping both employees and healthcare professionals gain more knowledge about IBD by 'becoming the patient' and experiencing what it's like to live with IBD. The program has been developed in consultation with immersive learning experts to help participants gain a deeper understanding of how IBD negatively affects people in their daily lives, inspiring new facets in their work in serving patients with IBD, whether it be in research and development, marketing, or patient outreach. The program seeks to support the wider healthcare community become true advocates with a deeper emotional connection to the disease, and ultimately improve how our healthcare partners and we support these patients."

    An evaluation study[1] conducted by the University of Westminster, London, UK, and published in Frontiers in Psychology demonstrated statistically significant increases in understanding, empathy, and connection to patients living with IBD, as well as a greater desire to help raise public awareness and improve access to patient support among the "In Their Shoes" participants.

    As part of the program, over 1,900 participants from more than 30 countries worldwide have had the opportunity to simulate an IBD patient's daily life via mobile app challenges, experiencing first-hand the emotional and physical burden of living with the disease. In early 2019, following the global success of In Their Shoes, Takeda expanded the program's scope with the launch of a new version simulating a patient's life with Crohn's disease living with a complex perianal fistula.

    1. Halton C, Cartwright T. Walking in a Patient's Shoes: An Evaluation Study of Immersive Learning Using a Digital Training Intervention. Front Psychol [Internet]. Available from: https://www.frontiersin.org/articles/10.3389/fpsyg.2018.02124/full.

    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 bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, 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. For more information, visit https://www.takeda.com.

    For more information:

    mohammed.alnasseri@fleishman.com 

    M: +971 507694646

    Cision View original content:http://www.prnewswire.com/news-releases/takeda-introduces-the-in-their-shoes-experience-to-the-middle-east-in-support-of-the-ibd-patient-community-301309252.html

    SOURCE Takeda Pharmaceuticals

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    • Building Upon Previous Data Showing More Than Twice as Many SOT Recipients Receiving Maribavir Achieved CMV Viremia Clearance Compared to Conventional Antiviral Therapies, Subgroup Analysis Showed Consistent Efficacy in SOT Recipients Receiving Investigational Drug Maribavir In Heart, Lung and Kidney Transplants1
    • With the U.S. Food and Drug Administration's (FDA) Recent Granting of Priority Review for Maribavir, Takeda Takes Another Step Towards Addressing Unmet Treatment Needs for Post-Transplant Recipients With CMV Infection

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today at the American Transplant Congress (ATC) 2021 Virtual Connect presented results from a new subgroup analysis of SOT recipients in the Phase 3 TAK-620-303…

    • Building Upon Previous Data Showing More Than Twice as Many SOT Recipients Receiving Maribavir Achieved CMV Viremia Clearance Compared to Conventional Antiviral Therapies, Subgroup Analysis Showed Consistent Efficacy in SOT Recipients Receiving Investigational Drug Maribavir In Heart, Lung and Kidney Transplants1
    • With the U.S. Food and Drug Administration's (FDA) Recent Granting of Priority Review for Maribavir, Takeda Takes Another Step Towards Addressing Unmet Treatment Needs for Post-Transplant Recipients With CMV Infection

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today at the American Transplant Congress (ATC) 2021 Virtual Connect presented results from a new subgroup analysis of SOT recipients in the Phase 3 TAK-620-303 (SOLSTICE) trial, for the investigational drug TAK-620 (maribavir). More than twice (55.6%, 79/142) as many SOT recipients with R/R CMV infection at baseline treated with maribavir achieved confirmed CMV viremia clearance at Study Week 8 (end of treatment phase) compared to those treated with conventional antiviral therapies (26.1%, 18/69) (investigator assigned treatment; IAT consists of one or a combination of ganciclovir, valganciclovir, foscarnet or cidofovir) (adjusted difference [95% CI]: 30.5% [17.3, 43.6]).1 The results presented showed consistent efficacy in SOT recipients receiving maribavir in heart, lung and kidney transplants.

    Key findings by transplant type included:1

    • More than 3 times as many lung transplant recipients with R/R CMV infection treated with maribavir achieved confirmed CMV viremia clearance as compared to those on conventional therapies (47.5% [19/40] vs 13.6% [3/22]; adjusted difference [95% CI]: 38.2% [16.89, 59.53]).
    • More than 1.5 times as many kidney transplant recipients with R/R CMV infection treated with maribavir achieved confirmed CMV viremia clearance as compared to those on conventional therapies (59.5% [44/74] vs 34.4% [11/32]; adjusted difference [95% CI]: 26.7% [7.48, 45.85]).
    • 42.9% (6/14) of heart transplant recipients with R/R CMV infection treated with maribavir achieved confirmed CMV viremia clearance as compared to 11.1% (1/9) of those on conventional therapies (adjusted difference [95% CI]: 30.7% [-1.72, 63.15]).

    "CMV infections are a common yet unpredictable threat for transplant teams, with an estimated incidence rate between 16-56% in solid organ transplant recipients," said Obi Umeh, MD, Vice President and Maribavir Global Program Leader, Takeda. "These results build on previously presented data on the potential of maribavir to treat CMV infection in some of these challenging patient populations."

    These findings reinforce the results from the overall trial population which showed the study met its primary endpoint, demonstrating that maribavir was superior to conventional antiviral therapies in CMV viremia clearance at Study Week 8. Specifically, 55.7% (131/235) of transplant recipients with R/R, CMV infection/disease treated with maribavir achieved confirmed CMV viremia clearance as compared to 23.9% (28/117) of those on conventional antiviral therapies (adjusted difference [95% CI]: 32.8%, [22.8, 42.7]; p<0.001).1*†‡

    Transplant recipients receiving maribavir exhibited lower incidence of treatment-related toxicities common with conventional antiviral therapies. Those receiving maribavir experienced lower rates of treatment-related neutropenia vs. valganciclovir/ganciclovir (1.7% [4/234] vs. 25% [14/56]) and acute kidney injury vs. foscarnet (1.7% [4/234] vs. 19.1% [9/47]). Incidence of any treatment-emergent adverse events (TEAEs) was 97.4% (228/234) for maribavir and 91.4% (106/116) for the conventional therapy group.1 The most common TEAEs in the maribavir group were dysgeusia (35.9%, 84/234), nausea (8.5%, 20/234) and vomiting (7.7%, 18/234).2 Incidence of TEAEs leading to study drug discontinuation was 13.2% (31/234) in the maribavir group and 31.9% (37/116) in the conventional therapy group.1 Two treatment-related serious TEAEs led to death (1 patient per treatment group).1

    About CMV

    CMV is a beta herpesvirus that commonly infects humans; serologic evidence of prior infection can be found in 40%-100% of various adult populations.3 CMV typically resides latent and asymptomatic in the body but may reactivate during periods of immunosuppression. Serious disease may occur in individuals with compromised immune systems, which includes patients who receive immunosuppressants associated with various types of transplants including hematopoietic cell transplant (HCT) or solid organ transplant (SOT).4,5 Out of the estimated 200,000 adult transplants per year, CMV is one of the most common viral infections experienced by transplant recipients, with an estimated incidence rate between 16-56% in SOT recipients and 30-70% in HCT recipients.5–10

    In transplant recipients, reactivation of CMV can lead to serious consequences including loss of the transplanted organ and, in extreme cases, can be fatal.11,12 Existing therapies to treat posttransplant CMV infections may demonstrate toxicities that require dose adjustments or may fail to adequately suppress viral replication.13–15 Additionally, existing therapies may require or prolong hospitalization due to administration.13,14

    About Maribavir

    Maribavir, an orally bioavailable anti-CMV compound, is the only antiviral agent presently in Phase 3 development for the treatment of post-transplant patients with CMV in SOT or HCT. Maribavir is an investigational treatment that has not been approved for use by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) or any other regulatory authorities. Maribavir is the only CMV antiviral drug that targets and inhibits the UL97 protein kinase and its natural substrates.16–19

    Maribavir has been granted Orphan Drug Designation by the European Commission as a treatment of CMV disease in patients with impaired cell mediated immunity and by the FDA for treatment of clinically significant CMV viremia and disease in at-risk patients. Orphan status is granted to certain investigational medicines intended for the treatment or prevention of a rare, life-threatening disease. The FDA has also granted maribavir Breakthrough Therapy Designation as a treatment for CMV infection and disease in transplant patients resistant or refractory to prior therapy. Breakthrough Therapy Designation expedites the development and review of investigational treatments for serious conditions with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy. Most recently, the FDA has granted priority review of maribavir for the treatment of post-transplant recipients with CMV infection in those R/R to prior anti-CMV treatment. These designations and NDA acceptance do not guarantee that the EMA or FDA will approve maribavir for the treatment of CMV infections in transplant patients, and the timing of any such approval is uncertain.

    About Takeda's SOLSTICE Trial

    The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing treatment with either maribavir or investigator assigned treatment, IAT, (conventional antiviral therapy) in hematopoietic cell transplant and solid organ transplant recipients with CMV infection refractory, with or without resistance, to one or a combination of the conventional antiviral therapies: ganciclovir, valganciclovir, foscarnet or cidofovir. Patients underwent a 2-week screening period, followed by randomization 2:1 to maribavir (n=235) (400 mg) or IAT (n=117) for an 8-week treatment period, plus 12 weeks of follow-up.

    The trial's primary endpoint was defined as the proportion of patients who achieved confirmed CMV viremia clearance (plasma CMV DNA <137 IU/mL in two consecutive tests ≥5 days apart at central laboratory) compared to IAT at the end of Study Week 8. The key secondary endpoint was defined as achievement of CMV viremia clearance and symptom control at end of Study Week 8, maintained through Study Week 16.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    *The difference in proportion of responders between treatment groups was obtained using Cochran-Mantel-Haenszel (CMH) weighted average across all strata and tested using stratum-adjusted CMH method, with transplant type and baseline plasma CMV DNA concentration as two stratification factors

    Refractory defined as documented failure to achieve >1 log10 decrease in CMV DNA level in whole blood or plasma after a 14 day or longer treatment period with IV ganciclovir/oral valganciclovir, IV foscarnet, or IV cidofovir

    Resistant defined as refractory CMV and documentation of >1 CMV genetic mutations associated with resistance to ganciclovir, valganciclovir, foscarnet, and/or cidofovir

    ________________________

    1. Avery R. Randomized Phase 3 Open-Label Study of Maribavir vs Investigator-Assigned Therapy for Refractory/Resistant Cytomegalovirus Infection in Transplant Recipients: Subgroup Analyses of Efficacy by Organ. In: American Transplant Congress (ATC) 2021 Virtual Connect. ; 2021.

    2. Duarte R. Maribavir Versus Investigator-Assigned Therapy for the Treatment of Transplant Recipients with Refractory/Resistant Cytomegalovirus Infection: Efficacy Data From a Randomized Phase 3 Open-Label Study. Abstract presented at the: 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT); 2021.

    3. Krech U. Complement-fixing antibodies against cytomegalovirus in different parts of the world. Bull WHO. 1973;49:103-106.

    4. de la Hoz R. Diagnosis and treatment approaches to CMV infections in adult patients. J Clin Virol. 2002;25:S1-S12.

    5. Azevedo L, Pierrotti L, Abdala E, et al. Cytomegalovirus infection in transplant recipients. Clinics. 2015;70(7):515-523. doi:10.6061/clinics/2015(07)09

    6. World Health Organization. International Report on Organ Donation and Transplantation Activities- Executive Summary 2018.; 2020. Accessed December 2, 2020. http://www.transplant-observatory.org/wp-content/uploads/2020/10/glorep2018-2.pdf

    7. World Health Organization. Haematopoietic Stem Cell Transplantation HSCtx. Accessed December 2, 2020. https://www.who.int/transplantation/hsctx/en/

    8. Razonable RR, Eid AJ. A Viral infections in transplant recipients. Minerva Med. 2009;100(6):23.

    9. Styczynski J. Who Is the Patient at Risk of CMV Recurrence: A Review of the Current Scientific Evidence with a Focus on Hematopoietic Cell Transplantation. Infect Ther. 2018;7:1-16.

    10. Cho S-Y, Lee D-G, Kim H-J. Cytomegalovirus Infections after Hematopoietic Stem Cell Transplantation: Current Status and Future Immunotherapy. Int J Mol Sci. 2019;20(2666):1-17.

    11. Fishman JA. Infection in Solid-Organ Transplant Recipients. N Engl J Med. Published online 2007:14.

    12. Kenyon M, Babic A, eds. The European Blood and Marrow Transplantation Textbook for Nurses. Springer International Publishing; 2018. doi:10.1007/978-3-319-50026-3

    13. Martín-Gandul C, Pérez-Romero P, González-Roncero FM, et al. Clinical impact of neutropenia related with the preemptive therapy of CMV infection in solid organ transplant recipients. J Infect. 2014;69(5):500-506. doi:10.1016/j.jinf.2014.07.001

    14. Chemaly RF, Chou S, Einsele H, et al. Definitions of Resistant and Refractory Cytomegalovirus Infection and Disease in Transplant Recipients for Use in Clinical Trials. Clin Infect Dis. 2019;68(8):1420-1426. doi:10.1093/cid/ciy696

    15. Beyer K. Outpatient Foscarnet Administration Incorporating Home Infusions Is Feasible Greatly Enhancing the Care of Hematopoietic Stem Cell Transplant Recipients. Biol Blood Marrow Transpl. 2017;23:S18-S391.

    16. Abdel-Magid AF. New Inhibitors of Cytomegalovirus DNA Polymerase. ACS Med Chem Lett. 2013;4(12):1129-1130. doi:10.1021/ml4004099

    17. Hamirally S, Kamil JP, Ndassa-Colday YM, et al. Viral Mimicry of Cdc2/Cyclin-Dependent Kinase 1 Mediates Disruption of Nuclear Lamina during Human Cytomegalovirus Nuclear Egress. Nelson JA, ed. PLoS Pathog. 2009;5(1):e1000275. doi:10.1371/journal.ppat.1000275

    18. Kotton CN, Kumar D, Caliendo AM, et al. The Third International Consensus Guidelines on the Management of Cytomegalovirus in Solid-organ Transplantation: Transplantation. 2018;102(6):900-931. doi:10.1097/TP.0000000000002191

    19. Krosky PM, Baek M-C, Coen DM. The Human Cytomegalovirus UL97 Protein Kinase, an Antiviral Drug Target, Is Required at the Stage of Nuclear Egress. J Virol. 2003;77(2):905-914. doi:10.1128/JVI.77.2.905-914.2003

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  18. Takeda's dengue vaccine candidate (TAK-003) prevented 83.6% of hospitalizations and 62.0% of dengue illness overall, with no identified important safety risks through three years following vaccination in ongoing pivotal Phase 3 TIDES trial

    Regulatory filings for TAK-003 progressing in European Union and in many dengue-endemic countries; filing in United States planned for later this year

    Dengue is the fastest-spreading mosquito-borne viral disease with an estimated 390 million cases and 500,000 hospitalizations per year globally, with limited options for prevention

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that its dengue vaccine candidate (TAK-003) demonstrated continued protection against dengue illness…

    Takeda's dengue vaccine candidate (TAK-003) prevented 83.6% of hospitalizations and 62.0% of dengue illness overall, with no identified important safety risks through three years following vaccination in ongoing pivotal Phase 3 TIDES trial

    Regulatory filings for TAK-003 progressing in European Union and in many dengue-endemic countries; filing in United States planned for later this year

    Dengue is the fastest-spreading mosquito-borne viral disease with an estimated 390 million cases and 500,000 hospitalizations per year globally, with limited options for prevention

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that its dengue vaccine candidate (TAK-003) demonstrated continued protection against dengue illness and hospitalization, regardless of an individual's previous dengue exposure, with no important safety risks identified through three years after vaccination in the ongoing pivotal Phase 3 Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial. TIDES enrolled more than 20,000 healthy children and adolescents ages four to 16 years in dengue-endemic countries in Latin America and Asia.

    "Dengue epidemics occur suddenly, and hospitals can become overwhelmed with severe disease cases and people seeking testing," said LakKumar Fernandoi, M.D., Center for Clinical Management of Dengue and Dengue Haemorrhagic Fever, Negombo General Hospital, Sri Lanka and a primary investigator of the TIDES trial. "Results from the long-term analysis of Takeda's dengue vaccine candidate suggest that it could help with outbreak prevention, reduce rates of hospitalization and protect people from dengue regardless of their previous exposure. Importantly, no important safety risks were identified."

    Safety and efficacy results from the 36-month follow-up exploratory analysis of TIDES were presented on May 22, 2021, at the 17th Conference of the International Society of Travel Medicine (CISTM). Through three years (36 months after the second dose), TAK-003 demonstrated overall vaccine efficacy (VE) of 62.0% (95% CI: 56.6% to 66.7%) against virologically-confirmed dengue (VCD), with 65.0% VE (95% CI: 58.9% to 70.1%) in seropositive individuals and 54.3% VE (95% CI: 41.9% to 64.1%) in seronegative individuals. TAK-003 also demonstrated 83.6% VE (95% CI: 76.8% to 88.4%) against hospitalized dengue, with 86.0% VE (95% CI: 78.4% to 91.0%) in seropositive individuals and 77.1% VE (95% CI: 58.6% to 87.3%) in seronegative individuals. Observations of varied VE by serotype remained consistent with previously reported results. No evidence of disease enhancement was observed. TAK-003 was generally well tolerated, and there were no important safety risks observed. The results reinforce the potential of TAK-003 to help protect those who are living in or traveling to dengue-endemic countries.

    "Our dengue vaccine candidate continued to provide protection against dengue throughout three years, and was especially robust in preventing hospitalization," said Derek Wallace, VP, Dengue Global Program Leader at Takeda. "These results reinforce my confidence that TAK-003 can help address the significant global burden of dengue."

    As previously reported, the TIDES trial met its primary endpoint of overall VE against VCD at 12-months follow-up (VE: 80.2%; 95% CI: 73.3% to 85.3%; p<0.001) and all secondary endpoints for which there were a sufficient number of dengue cases (measured at 18-months follow-up). The TIDES trial has been amended to include the evaluation of a booster dose to address the waning of overall VE observed over time (from 12 through 36 months after the second dose), largely driven by outpatient dengue. Takeda intends to publish results of the 36-month exploratory analysis in a peer-reviewed journal this year.

    TIDES safety and efficacy data through 36-months follow-up was included in regulatory submissions to the European Union and dengue-endemic countries and will be part of additional filings planned for 2021, including in the United States. Takeda will seek an indication for TAK-003 for the prevention of dengue disease in individuals four to 60 years of age, regardless of previous virus exposure, based on data in both adults and children. There remains a need for dengue vaccines that can be used in both dengue-naïve and dengue-exposed adults and children.

    About TAK-003

    Takeda's tetravalent dengue vaccine candidate (TAK-003) is based on a live-attenuated dengue serotype 2 virus, which provides the genetic "backbone" for all four vaccine viruses.1 Clinical Phase 2 data in children and adolescents showed that TAK-003 induced immune responses against all four dengue serotypes, in both seropositive and seronegative participants, which persisted through 48 months after vaccination, and the vaccine was found to be generally safe and well tolerated.2 The pivotal Phase 3 Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial met its primary endpoint of overall vaccine efficacy (VE) against virologically-confirmed dengue (VCD) at 12-months follow-up and all secondary endpoints at 18-months follow-up for which there were a sufficient number of dengue cases, including VE against hospitalized dengue and VE in baseline seropositive and baseline seronegative individuals.3,4 Efficacy varied by serotype. The results demonstrated TAK-003 was generally well tolerated, and there have been no important safety risks observed to date.

    About the Phase 3 TIDES (DEN-301) Trial

    The double-blind, randomized, placebo-controlled Phase 3 Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial is evaluating the safety and efficacy of two doses of TAK-003 in the prevention of laboratory-confirmed symptomatic dengue fever of any severity and due to any of the four dengue virus serotypes in children and adolescents.4 The TIDES trial is Takeda's largest interventional clinical trial to date and enrolled over 20,000 healthy children and adolescents ages four to 16 years living in dengue-endemic areas. Study participants were randomly assigned to receive either TAK-003 0.5 mL or placebo by subcutaneous injection on Day 1 and Day 90.4 The study is comprised of five parts. Part 1 and the primary endpoint analysis evaluated vaccine efficacy (VE) and safety through 12 months after the second dose.4 Part 2 continued for an additional six months to complete the assessment of the secondary endpoints of VE by serotype, baseline serostatus and disease severity, including VE against hospitalized dengue.4 Part 3 is evaluating VE and long-term safety by following participants for an additional two and a half to three years.5 Part 4 will evaluate efficacy and safety for 13 months following booster vaccination and Part 5 will evaluate long-term efficacy and safety for one year after completion of Part 4.5

    The trial is taking place at sites in dengue-endemic areas in Latin America (Brazil, Colombia, Panama, the Dominican Republic and Nicaragua) and Asia (Philippines, Thailand and Sri Lanka) where there are unmet needs in dengue prevention and where severe dengue is a leading cause of serious illness and death among children.4 Baseline blood samples were collected from all individuals participating in the trial to allow for evaluation of safety and efficacy based on serostatus. Takeda and an independent Data Monitoring Committee of experts are actively monitoring safety on an ongoing basis.

    About Dengue

    Dengue is the fastest spreading mosquito-borne viral disease and was one of the WHO's top 10 threats to global health in 2019.6,7 Dengue is mainly spread by Aedes aegypti mosquitoes and, to a lesser extent, Aedes albopictus mosquitoes. It is caused by any of four dengue virus serotypes, each of which can cause dengue fever or severe dengue. The prevalence of individual serotypes varies across different geographies, countries, regions, seasons and over time.8 Recovery from infection by one serotype provides lifelong immunity against only that serotype, and later exposure to any of the remaining serotypes is associated with an increased risk of severe disease.

    Dengue is pandemic prone, and outbreaks are observed in tropical and sub-tropical areas and have recently caused outbreaks in parts of the continental United States and Europe.9,10 Approximately half of the world now lives under the threat of dengue, which is estimated to cause 390 million infections and around 20,000 deaths globally each year.10,11 The dengue virus can infect people of all ages and is a leading cause of serious illness among children in some countries in Latin America and Asia.10

    Takeda's Commitment to Vaccines

    Vaccines prevent 2 to 3 million deaths each year and have transformed global public health.12 For the past 70 years, Takeda has supplied vaccines to protect the health of people in Japan. Today, Takeda's global vaccine business is applying innovation to tackle some of the world's most challenging infectious diseases, such as dengue, COVID-19, Zika and norovirus. Takeda's team brings an outstanding track record and a wealth of knowledge in vaccine development, manufacturing and global access to advance a pipeline of vaccines to address some of the world's most pressing public health needs. For more information, visit www.TakedaVaccines.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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/ reports/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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

    i Dr. LakKumar Fernando did not receive compensation for his statement.

    1 Huang CY-H, et al. Genetic and phenotypic characterization of manufacturing seeds for tetravalent dengue vaccine (DENVax). PLoS Negl Trop Dis. 2013;7:e2243.

    2Tricou, V, Sáez-Llorens X, et al. Safety and immunogenicity of a tetravalent dengue vaccine in children aged 2-17 years: a randomised, placebo-controlled, phase 2 trial. Lancet. 2020. doi:10.1016/S0140-6736(20)30556-0.

    3 Biswal S, et al. Efficacy of a tetravalent dengue vaccine in healthy children and adolescents. N Engl J Med. 2019; 2019;381:2009-2019.

    4 Biswal S, et al. Efficacy of a tetravalent dengue vaccine in healthy children aged 4-16 years: a randomized, placebo controlled, phase 3 trial. Lancet. 2020. 2020;395:1423-1433.

    5 ClinicalTrials.Gov. Efficacy, Safety and Immunogenicity of Takeda's Tetravalent Dengue Vaccine (TDV) in Healthy Children (TIDES). Retrieved March 2021.

    6 World Health Organization. Factsheet. Dengue and Severe Dengue. April 2019. Retrieved February 2021.

    7 World Health Organization. Ten threats to global health in 2019. 2019. Retrieved February 2021.

    8 Guzman MG, et al. Dengue: a continuing global threat. Nature Reviews Microbiology. 2010;8:S7-S16.

    9 Knowlton K, et al. Mosquito-Borne Dengue Fever Threat Spreading in the Americas. The Natural Resources Defense Council (NRDC). 2009. Retrieved February 2021.

    10 Chan E, et al. Using web search query data to monitor dengue epidemics: a new model for neglected tropical disease surveillance. PLoS Negl Trop Dis. 2011;5:e1206.

    11 Centers for Disease Control and Prevention. About Dengue: What You Need to Know. May 2019. Retrieved February 2021.

    12 UNICEF. Vaccination and Immunization Statistics. 2019. Retrieved February 2021.

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  19.  − If Approved, Maribavir Will Be the First and Only Treatment Indicated for Post-Transplant Cytomegalovirus (CMV) Infection in Those That Are Refractory, With or Without Resistance (R/R)

    NDA based on Phase 3 Trial of Maribavir Which Met Its Primary Endpoint of Superiority Compared to Conventional Antiviral Therapies in Transplant Recipients with R/R CMV Infection1

    The U.S. Food & Drug Administration Granted Maribavir Breakthrough Therapy Designation as a Treatment for CMV Infection in Transplant Patients Resistant or Refractory to Prior Therapy

    Maribavir is Takeda's Fourth New Molecular Entity Accepted for Regulatory Review in Six Months

    Takeda Pharmaceutical Company Limited Takeda Pharmaceutical Company Limited ((TAK) ("Takeda")

     − If Approved, Maribavir Will Be the First and Only Treatment Indicated for Post-Transplant Cytomegalovirus (CMV) Infection in Those That Are Refractory, With or Without Resistance (R/R)

    NDA based on Phase 3 Trial of Maribavir Which Met Its Primary Endpoint of Superiority Compared to Conventional Antiviral Therapies in Transplant Recipients with R/R CMV Infection1

    The U.S. Food & Drug Administration Granted Maribavir Breakthrough Therapy Designation as a Treatment for CMV Infection in Transplant Patients Resistant or Refractory to Prior Therapy

    Maribavir is Takeda's Fourth New Molecular Entity Accepted for Regulatory Review in Six Months

    Takeda Pharmaceutical Company Limited Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the U.S. Food & Drug Administration (FDA) has accepted a New Drug Application (NDA) for maribavir for the treatment of CMV infection in those that are refractory with or without resistance (R/R), in solid organ transplant (SOT) or hematopoietic cell transplant (HCT) recipients.

    This is an inflection year for Takeda's pipeline with up to six regulatory submissions and four potential approvals anticipated by the end of fiscal year 2021. The maribavir NDA acceptance is Takeda's fourth new molecular entity accepted for regulatory review in six months, following the FDA submissions of TAK-721 for the treatment of eosinophilic esophagitis, mobocertinib for the treatment of EGFR Exon20 insertion mutation positive metastatic non-small cell lung cancer, and the European Medicines Agency submission of the Company's dengue vaccine candidate (TAK-003), which is being investigated for the prevention of dengue due to any dengue virus serotype in individuals ages four to 60.

    "CMV is one of the most common viral infections experienced by transplant recipients, and current antiviral treatment options are limited, and physicians have to engage in a careful balance of viral clearance and side effect management that can impact patient care and transplant outcomes," said Obi Umeh, MD, Vice President and Maribavir Global Program Leader, Takeda. "If approved, maribavir has the potential to change the treatment landscape for post-transplant CMV, and the acceptance of this regulatory application is an important milestone on maribavir's path forward."

    The application is based on the pivotal Phase 3 TAK-620-303 (SOLSTICE) trial, results of which were presented at the 2021 Transplantation & Cellular Therapy (TCT) Meetings Digital Experience, with subgroup analysis presented during the Presidential Symposium 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT).

    "CMV infection puts transplant recipients at an increased risk of disease, such as pneumonia or gastrointestinal disease. It can also increase the risk of graft rejection, opportunistic co-infections, and in some cases, even death," said Michael Boeckh, M.D., Ph.D., Head, Infectious Disease Sciences Program at the Vaccine and Infectious Disease Division, Fred Hutch "The results of the SOLSTICE trial are promising and show that maribavir may help with post-transplant CMV viremia, including cases of drug-resistance for which there is an unmet need."

    Maribavir has been granted Orphan Drug Designation by the FDA for treatment of clinically significant CMV viremia and disease in at-risk patients. The FDA has also granted maribavir Breakthrough Therapy Designation as a treatment for CMV infection and disease in transplant patients resistant or refractory to prior therapy. These designations do not guarantee that the FDA will approve maribavir for the treatment of CMV infections in transplant patients, and the timing of any such approval is uncertain.

    About CMV

    CMV is a beta herpesvirus that commonly infects humans; serologic evidence of prior infection can be found in 40%-100% of various adult populations.2 CMV typically resides latent and asymptomatic in the body but may reactivate during periods of immunosuppression. Serious disease may occur in individuals with compromised immune systems, which includes patients who receive immunosuppressants associated with various types of transplants including hematopoietic cell transplant (HCT) or solid organ transplant (SOT).3,4 Out of the estimated 200,000 adult transplants per year, CMV is one of the most common viral infections experienced by transplant recipients, with an estimated incidence rate between 16-56% in SOT recipients and 30-70% in HCT recipients.4–9

    In transplant recipients, reactivation of CMV can lead to serious consequences including loss of the transplanted organ and, in extreme cases, can be fatal.10,11 Existing therapies to treat posttransplant CMV infections may demonstrate serious side effects that require dose adjustments or may fail to adequately suppress viral replication.12–14 Additionally, existing therapies may require or prolong hospitalization due to administration.12,13

    About Maribavir

    Maribavir, an orally bioavailable anti-CMV compound, is the only antiviral agent presently in Phase 3 development for the treatment of post-transplant patients with CMV in SOT or HCT. Maribavir is an investigational treatment that has not been approved for use by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) or any other regulatory authorities. Maribavir is the only CMV antiviral drug that targets and inhibits the UL97 protein kinase and its natural substrates.1,15–17

    Maribavir has been granted Orphan Drug Designation by the European Commission as a treatment of CMV disease in patients with impaired cell mediated immunity and by the FDA for treatment of clinically significant CMV viremia and disease in at-risk patients. Orphan status is granted to certain investigational medicines intended for the treatment or prevention of a rare, life-threatening disease. The FDA has also granted maribavir Breakthrough Therapy Designation as a treatment for CMV infection and disease in transplant patients resistant or refractory to prior therapy. Breakthrough Therapy Designation expedites the development and review of investigational treatments for serious conditions with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy. These designations do not guarantee that the EMA or FDA will approve maribavir for the treatment of CMV infections in transplant patients, and the timing of any such approval is uncertain.

    About Takeda's SOLSTICE Trial

    The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing treatment with either maribavir or investigator assigned treatment, IAT, (conventional antiviral therapy) in hematopoietic cell transplant and solid organ transplant recipients with CMV infection refractory, with or without resistance, to one or a combination of the conventional antiviral therapies: ganciclovir, valganciclovir, foscarnet or cidofovir. Patients underwent a 2-week screening period, followed by randomization 2:1 to maribavir (n=235) (400 mg) or IAT (n=117) for an 8-week treatment period, plus 12 weeks of follow-up.

    The trial's primary endpoint was defined as the proportion of patients who achieved confirmed CMV viremia clearance (plasma CMV DNA <137 IU/mL in two consecutive tests ≥5 days apart at central laboratory) compared to IAT at the end of Study Week 8. The key secondary endpoint was defined as achievement of CMV viremia clearance and symptom control at end of Study Week 8, maintained through Study Week 16.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    *The difference in proportion of responders between treatment groups was obtained using Cochran-Mantel-Haenszel (CMH) weighted average across all strata and tested using stratum-adjusted CMH method, with transplant type and baseline plasma CMV DNA concentration as two stratification factors

    Refractory defined as documented failure to achieve >1 log10 decrease in CMV DNA level in whole blood or plasma after a 14 day or longer treatment period with IV ganciclovir/oral valganciclovir, IV foscarnet, or IV cidofovir

    Resistant defined as refractory CMV and documentation of >1 CMV genetic mutations associated with resistance to ganciclovir, valganciclovir, foscarnet, and/or cidofovir

    References

    1.

     

    Marty F. A Phase 3 Randomized Study of Maribavir (MBV) Versus Investigator-Assigned Antiviral Therapy (IAT) for the Treatment (Tx) of Refractory/Resistant (R/R) Cytomegalovirus (CMV) Infection in Hematopoietic Cell Transplant (HCT) or Solid Organ Transplant (SOT) Recipients. In: The 2021 TCT Meetings Digital Experience. ; 2021.

    2.

    Krech U. Complement-fixing antibodies against cytomegalovirus in different parts of the world. Bull WHO. 1973;49:103-106.

    3.

    de la Hoz R. Diagnosis and treatment approaches to CMV infections in adult patients. Journal of Clinical Virology. 2002;25:S1-S12.

    4.

    Azevedo L, Pierrotti L, Abdala E, et al. Cytomegalovirus infection in transplant recipients. Clinics. 2015;70(7):515-523. doi:10.6061/clinics/2015(07)09

    5.

    World Health Organization. International Report on Organ Donation and Transplantation Activities- Executive Summary 2018.; 2020. Accessed December 2, 2020. http://www.transplant-observatory.org/wp-content/uploads/2020/10/glorep2018-2.pdf

    6.

    World Health Organization. Haematopoietic Stem Cell Transplantation HSCtx. Accessed December 2, 2020. https://www.who.int/transplantation/hsctx/en/

    7.

    Razonable RR, Eid AJ. A Viral infections in transplant recipients. MINERVA MEDICA. 2009;100(6):23.

    8.

    Styczynski J. Who Is the Patient at Risk of CMV Recurrence: A Review of the Current Scientific Evidence with a Focus on Hematopoietic Cell Transplantation. Infect Dis Ther. 2018;7:1-16.

    9.

    Cho S-Y, Lee D-G, Kim H-J. Cytomegalovirus Infections after Hematopoietic Stem Cell Transplantation: Current Status and Future Immunotherapy. Int J Mol Sci. 2019;20(2666):1-17.

    10.

    Fishman JA. Infection in Organ Transplantation. American Journal of Transplantation. 2017;17:856-879.

    11.

    Kenyon M, Babic A, eds. The European Blood and Marrow Transplantation Textbook for Nurses. Springer International Publishing; 2018. doi:10.1007/978-3-319-50026-3

    12.

    Martín-Gandul C, Pérez-Romero P, González-Roncero FM, et al. Clinical impact of neutropenia related with the preemptive therapy of CMV infection in solid organ transplant recipients. Journal of Infection. 2014;69(5):500-506. doi:10.1016/j.jinf.2014.07.001

    13.

    Chemaly RF, Chou S, Einsele H, et al. Definitions of Resistant and Refractory Cytomegalovirus Infection and Disease in Transplant Recipients for Use in Clinical Trials. Clinical Infectious Diseases. 2019;68(8):1420-1426. doi:10.1093/cid/ciy696

    14.

    Beyer K. Outpatient Foscarnet Administration Incorporating Home Infusions Is Feasible Greatly Enhancing the Care of Hematopoietic Stem Cell Transplant Recipients. Biol Blood Marrow Transplant. 2017;23:S18-S391.

    15.

    Hamirally S, Kamil JP, Ndassa-Colday YM, et al. Viral Mimicry of Cdc2/Cyclin-Dependent Kinase 1 Mediates Disruption of Nuclear Lamina during Human Cytomegalovirus Nuclear Egress. Nelson JA, ed. PLoS Pathog. 2009;5(1):e1000275. doi:10.1371/journal.ppat.1000275

    16.

    Krosky PM, Baek M-C, Coen DM. The Human Cytomegalovirus UL97 Protein Kinase, an Antiviral Drug Target, Is Required at the Stage of Nuclear Egress. JVI. 2003;77(2):905-914. doi:10.1128/JVI.77.2.905-914.2003

    17.

    Prichard MN. Function of human cytomegalovirus UL97 kinase in viral infection and its inhibition by maribavir: Human cytomegalovirus UL97 kinase. Rev Med Virol. 2009;19(4):215-229. doi:10.1002/rmv.615

     

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  20. The Ministry of Health, Labour and Welfare (MHLW) Grants Regulatory Approval of Moderna's COVID-19 Vaccine (COVID-19 Vaccine Moderna Intramuscular Injection) Following Positive Interim Results in Takeda's Phase 1/2 Immunogenicity and Safety Clinical Trial

    Interim Results Indicate Immune Response Consistent with Moderna's Pivotal Phase 3 COVE Trial Results

    Takeda Will Immediately Begin Distribution in Japan

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the Ministry of Health, Labour and Welfare (MHLW) granted special approval under article 14-3 of the Pharmaceuticals and Medical Devices Act for emergency use of Moderna's mRNA COVID-19 vaccine, TAK-919, now known as COVID-19 Vaccine Moderna Intramuscular…

    The Ministry of Health, Labour and Welfare (MHLW) Grants Regulatory Approval of Moderna's COVID-19 Vaccine (COVID-19 Vaccine Moderna Intramuscular Injection) Following Positive Interim Results in Takeda's Phase 1/2 Immunogenicity and Safety Clinical Trial

    Interim Results Indicate Immune Response Consistent with Moderna's Pivotal Phase 3 COVE Trial Results

    Takeda Will Immediately Begin Distribution in Japan

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the Ministry of Health, Labour and Welfare (MHLW) granted special approval under article 14-3 of the Pharmaceuticals and Medical Devices Act for emergency use of Moderna's mRNA COVID-19 vaccine, TAK-919, now known as COVID-19 Vaccine Moderna Intramuscular Injection, in Japan. The approval is based on positive clinical data from Takeda's Phase 1/2 immunogenicity and safety clinical trial of Moderna's COVID-19 vaccine in Japan, which showed an immune response consistent with results from Moderna's pivotal Phase 3 COVE trial conducted in the United States. Takeda plans to begin distribution in Japan immediately.

    "This is an important step in Takeda's support of Japan's pandemic response," said Rajeev Venkayya, president of the Global Vaccine Business Unit at Takeda. "Moderna's vaccine has demonstrated an excellent safety and effectiveness profile to date, and we are excited to make it available to the Japanese population."

    The approval is the result of a three-way agreement announced in October 2020 with Moderna and Government of Japan's Ministry of Health Labour and Welfare (MHLW) to distribute 50 million doses of TAK-919 in Japan in the first half of 2021. Takeda also entered into a collaboration with Novavax to develop, manufacture and commercialize Novavax' COVID-19 vaccine candidate (TAK-019) in Japan.

    Takeda's efforts to bring the COVID-19 Vaccine Moderna Intramuscular Injection and Novavax' vaccine candidates to Japan are supported by the MHLW and the Japan Agency for Medical Research and Development (AMED).

    TAK-919 Clinical Trial and Results

    Takeda is conducting a placebo-controlled Phase 1/2 study in Japan to evaluate the safety and immunogenicity of two vaccinations of TAK-919 given 28 days apart. Takeda enrolled 200 participants aged 20 years and older. Each participant was assigned to receive a placebo or a 0.5 ml dose of TAK-919 at both vaccinations. Participants will be followed for 12 months after the second vaccination.

    This interim analysis showed that binding antibody and neutralizing antibody titers were elevated at 28 days after the second dose in 100% of people vaccinated with two 0.5ml doses of TAK-919 given 28 days apart. The vaccine candidate was generally well-tolerated with no significant safety concerns reported. Takeda intends to publish additional data in a peer-reviewed journal.

    About Takeda's COVID-19 Efforts

    Takeda is taking a comprehensive approach to treat and prevent COVID-19 today, and future pandemics through multiple activities and partnerships including, but not limited to:

    • Hyperimmune globulin: Takeda co-founded the CoVIg-19 Plasma Alliance and joined forces with other leading plasma companies to evaluate a hyperimmune globulin medicine in a global clinical trial. While the data did not meet its endpoints, the program has contributed to the scientific understanding of antibody-based treatment to address the virus and highlighted the broader therapeutic value and importance of plasma to treat rare diseases.
    • Additional therapeutics: The company has assessed existing Takeda products for activity against the COVID-19 virus and co-founded the COVID R&D Alliance. In addition, Takeda has joined the Innovative Medicines Initiative (IMI) CARE consortium, the Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) partnership and the COVID RED project.
    • Vaccines: Takeda has partnered with the Government of Japan, Novavax and Moderna, to help accelerate the availability of COVID-19 vaccines. We are leveraging our extensive and well-established global manufacturing and supply capabilities and building upon our existing influenza pandemic preparedness efforts in Japan. Takeda also announced a mutual agreement with IDT Biologika GmbH (IDT) to utilize capacity at IDT for three months previously reserved for Takeda's dengue vaccine candidate to manufacture the single-shot COVID-19 vaccine developed by Janssen Pharmaceutical Companies of Johnson & Johnson. Takeda supports our partners and alliances in a shared goal to rapidly discover, develop and deliver effective treatments and vaccines for COVID-19 and ensure preparedness for future pandemics.

    Takeda's Commitment to Vaccines

    Vaccines prevent 2 to 3 million deaths each year and have transformed global public health. For the past 70 years, Takeda has supplied vaccines to protect the health of people in Japan. Today, Takeda's global vaccine business is applying innovation to tackle some of the world's most challenging infectious diseases, such as dengue, COVID-19, Zika and norovirus. Takeda's team brings an outstanding track record and a wealth of knowledge in vaccine development, manufacturing and global access to advance a pipeline of vaccines to address some of the world's most pressing public health needs. For more information, visit www.TakedaVaccines.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; 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/ reports/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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

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  21. – OPTIC Trial Evaluating Response-Based Dosing Regimens of ICLUSIG for the Treatment of Resistant / Intolerant Chronic-Phase CML Demonstrates Durable, Clinically Meaningful Depth of Response While Managing Arterial Occlusive Event Risk

    – Oral Presentation of the OPTIC Primary Analysis at the American Society of Clinical Oncology (ASCO) and European Hematology Association (EHA) Annual Meetings

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that primary analysis data from the Phase 2 OPTIC (Optimizing Ponatinib Treatment In CML) trial will be presented during an oral session at the virtual 57th American Society of Clinical Oncology (ASCO) Annual Meeting, and as an oral session at the virtual 26th European Hematology…

    – OPTIC Trial Evaluating Response-Based Dosing Regimens of ICLUSIG for the Treatment of Resistant / Intolerant Chronic-Phase CML Demonstrates Durable, Clinically Meaningful Depth of Response While Managing Arterial Occlusive Event Risk

    – Oral Presentation of the OPTIC Primary Analysis at the American Society of Clinical Oncology (ASCO) and European Hematology Association (EHA) Annual Meetings

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that primary analysis data from the Phase 2 OPTIC (Optimizing Ponatinib Treatment In CML) trial will be presented during an oral session at the virtual 57th American Society of Clinical Oncology (ASCO) Annual Meeting, and as an oral session at the virtual 26th European Hematology Association (EHA) Annual Meeting. The OPTIC trial is a randomized, open-label study prospectively evaluating response-based dosing regimens of ICLUSIG® (ponatinib) over a range of three starting doses (45-, 30-, or 15-mg) with the aim of optimizing its efficacy and safety in patients with chronic-phase chronic myeloid leukemia (CP-CML) who are resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy.

    The OPTIC trial, which evaluated treatment in patients with resistant disease, with and without mutations, met its primary endpoint. The study demonstrates that the optimal benefit-risk profile for ICLUSIG in patients with CP-CML is achieved with a daily starting dose of 45-mg and, upon achieving ≤1% BCR-ABL1IS, dose reduction to 15-mg. The results also suggest a clinically manageable safety and arterial occlusive event (AOE) profile for ICLUSIG.

    "The primary analysis of the OPTIC data reinforce that ICLUSIG is very valuable in the management of patients with resistant and intolerant chronic-phase CML. ICLUSIG should be considered following failure of two or more TKIs. This approach minimizes the need to use back-to-back second-generation TKIs, which is usually associated with low probability of response and poor outcomes," said Jorge Cortes, MD, Georgia Cancer Center at Augusta University, and an OPTIC trial principal investigator. "These findings further demonstrate that the optimal ICLUSIG benefit-risk profile can be achieved with a response-based dosing regimen, providing efficacy while reducing risk for arterial occlusive events."

    The first presentation of OPTIC data at the 56th ASCO and the 25th EHA virtual Annual Meetings in 2020 showcased revised benefit-risk outcomes of ICLUSIG from the interim analysis (cutoff date of July 2019), which evaluated 216 patients with a median follow-up of 21 months. The primary analysis evaluated 283 patients with a median follow-up time of 32 months, reinforcing the positive and prolonged duration of response in this resistant CP-CML patient population while maintaining a manageable safety profile at a longer follow-up period.

    "There is a misconception that chronic-phase CML is a ‘good cancer' due to the fact it can be well controlled, but for patients with resistant and intolerant disease, continued investigation and treatment options are critical. The primary analysis of OPTIC solidifies our understanding of how ICLUSIG can address gaps in care for these patients," said Christopher Arendt, Ph.D., Head, Oncology Therapeutic Area Unit, Takeda. "The OPTIC data, along with the recently updated U.S. FDA indication, demonstrate the benefit ICLUSIG can offer as a third-generation TKI."

    In December 2020, the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) for ICLUSIG based on the OPTIC data. The updated label includes an expanded indication for adult patients with CP-CML that is resistant or intolerant to at least two prior kinase inhibitors, as well as the optimized response-based dosing regimen.

    OPTIC Primary Analysis: A Dose-Optimization Study of Three Starting Doses of Ponatinib (PON).

    Key findings, to be presented by Dr. Jorge Cortes, include:

    • By the primary analysis (cutoff date of May 2020) with median follow-up time of 32 months, 100% of patients in the OPTIC trial were evaluable for the primary endpoint.
    • The maximum rates of ≤1% BCR-ABL1IS at 12 months were achieved in the 45-mg/day starting dose cohort (44.1%), and 73.3% of patients in this cohort maintained responses with the dose reduction to 15-mg/day. 30-mg/day and 15-mg/day cohorts also demonstrated benefit (29% and 23%, respectively), especially in patients with less-resistant disease and without the T315I mutation.
    • Positive survival outcomes were observed in all three arms, with an 89% 36-month overall survival (OS) probability anticipated for the 45-mg starting dose cohort and 73% progression-free survival (PFS) anticipated for the same cohort.
    • This indicates the dose-reduction strategy did not impact overall survival regardless of prior second-generation TKI resistance or the presence of BCR-ABL1 mutations.
    • Rates of arterial occlusive events (AOEs) observed at the time of primary analysis (6% overall and 9.6% in the 45-mg cohort) suggest a clinically manageable safety and AOE profile.
    • Safety data include:
      • Among all patients (N=283), the most common treatment emergent adverse events (TEAEs) Grade 3 or greater were thrombocytopenia (27%), neutropenia (17%) and anemia (7%).
      • Reported AOEs were 10%, 5% and 3% for the 45-, 30-, 15-mg/day starting dose cohorts, respectively. Grade 3 or greater AOEs were 5%, 5% and 3% for the 45-, 30-, 15-mg/day starting dose cohorts, respectively.
      • Reported serious AOEs were 4%, 4% and 3% for the 45-, 30-, 15-mg/day starting dose cohorts, respectively. There were 4 deaths related to AEs (2 sudden deaths and 2 pneumonia).

    Learn more about Takeda Oncology's presence at this year's ASCO and EHA annual meetings. Takeda will host a webcast for analysts and investors on Tuesday, June 8, at 6:30 p.m. ET to discuss these and other data being presented at ASCO, and to provide an update on the oncology pipeline. Please contact TakedaRandDEvents@fticonsulting.com for further details. Presentation slides and an archived replay of the webcast will be available at https://www.takeda.com/investors/reports/ir-events/.

    About the OPTIC Trial

    OPTIC (Optimizing Ponatinib Treatment In CML) is an ongoing randomized, dose-ranging trial designed to evaluate three starting doses of ICLUSIG (45-, 30-, and 15-mg) in patients with resistant chronic-phase chronic myeloid leukemia (CP-CML) or who had documented history of presence of T315I mutation after receiving any number of prior TKIs. Dose reduction at response occurred per study protocol. The trial is expected to inform the optimal use of ICLUSIG® (ponatinib) in these patients. 282 patients were enrolled at clinical sites around the world, with 94 patients receiving the 45-mg starting dose. The primary endpoint of the trial is achieving ≤1% BCR-ABL1IS at 12 months.

    OPTIC data showed that optimal benefit-risk with ICLUSIG can be obtained with a response-based dosing regimen, 45-mg/day to 15-mg/day upon achieving ≤1% BCR-ABL1IS in patients with CP-CML highly resistant to prior TKI therapies both with or without mutations. At 12 months, 44% (41/93) of patients who received the 45-mg starting dose achieved ≤1% BCR-ABL1IS. At a median follow up time of 32 months, the OPTIC study showed that, among patients receiving ICLUSIG 45- to 15-mg, 73% maintained their response. In these patients, 10% experienced an AOE of any Grade, 5% experienced Grade 3 or higher. The most common TEAEs occurring in > 15% of patients were thrombocytopenia, hypertension, headache, neutropenia, anemia, headache, lipase increased, alanine aminotransferase (ALT) increased, and arthralgia.

    About CML and Ph+ ALL

    CML – a rare malignancy – is one of four main types of leukemia; it is a result of a genetic mutation that takes place in early, immature versions of myeloid cells, which form red blood cells, platelets and most types of white blood cells. Subsequently, an abnormal gene called BCR-ABL1 forms, turning the damaged cell into a CML cell. CML typically progresses slowly, but it can change into a fast-growing acute leukemia that is hard to treat.

    Ph+ ALL is a rare form of ALL that affects approximately 25% of adult ALL patients in the U.S. and is characterized by the presence of an abnormal gene, known as the Philadelphia chromosome. In patients who are Philadelphia chromosome-positive (Ph+), an abnormal chromosome is formed when pieces of chromosomes 9 and 22 switch with each other. This forms a longer chromosome 9 and a shorter chromosome 22, which leads to the development of BCR-ABL1 and is associated with Ph+ ALL.

    About ICLUSIG® (ponatinib) tablets

    ICLUSIG is a kinase inhibitor targeting BCR-ABL1, an abnormal tyrosine kinase that is expressed in CML and Ph+ ALL. ICLUSIG is a targeted cancer medicine developed using a computational and structure-based drug-design platform, specifically designed to inhibit the activity of BCR-ABL1 and its mutations. ICLUSIG inhibits native BCR-ABL1, as well as all BCR-ABL1 treatment-resistant mutations, including the most resistant T315I mutation. ICLUSIG is the only approved TKI that demonstrates activity against the T315I gatekeeper mutation of BCR-ABL1. This mutation has been associated with resistance to all other approved TKIs. ICLUSIG received full approval from the FDA in November 2016. ICLUSIG is indicated for the treatment of adult patients with chronic-phase (CP) CML with resistance or intolerance to at least two prior kinase inhibitors, accelerated-phase (AP) or blast-phase (BP) CML or Ph+ ALL for whom no other kinase inhibitor is indicated, and T315I+ CML (CP, AP or BP) or T315I-positive Ph+ ALL. ICLUSIG is not indicated and is not recommended for the treatment of patients with newly diagnosed CP-CML.

    IMPORTANT SAFETY INFORMATION

     

    WARNING: ARTERIAL OCCLUSIVE EVENTS, VENOUS THROMBOEMBOLIC EVENTS, HEART FAILURE, and HEPATOTOXICITY

    See full prescribing information for complete boxed warning.

     

    • Arterial occlusive events (AOEs), including fatalities, have occurred in ICLUSIG-treated patients. AOEs included fatal myocardial infarction, stroke, stenosis of large arterial vessels of the brain, severe peripheral vascular disease, and the need for urgent revascularization procedures. Patients with and without cardiovascular risk factors, including patients age 50 years or younger, experienced these events. Monitor for evidence of AOEs. Interrupt or discontinue ICLUSIG based on severity. Consider benefit-risk to guide a decision to restart ICLUSIG.
    • Venous thromboembolic events (VTEs) have occurred in ICLUSIG-treated patients. Monitor for evidence of VTEs. Interrupt or discontinue ICLUSIG based on severity.
    • Heart failure, including fatalities, occurred in ICLUSIG-treated patients. Monitor for heart failure and manage patients as clinically indicated. Interrupt or discontinue ICLUSIG for new or worsening heart failure.
    • Hepatotoxicity, liver failure and death have occurred in ICLUSIG-treated patients. Monitor liver function tests. Interrupt or discontinue ICLUSIG based on severity.

    WARNINGS AND PRECAUTIONS

    Arterial Occlusive Events (AOEs): AOEs, including fatalities, have occurred in patients who received ICLUSIG in OPTIC and PACE. These included cardiovascular, cerebrovascular, and peripheral vascular events. The incidence of AOEs in OPTIC (45 mg→15 mg) was 13% of 94 patients; 5% experienced Grade 3 or 4. In PACE, the incidence of AOEs was 26% of 449 patients; 14% experienced Grade 3 or 4. Fatal AOEs occurred in 2.1% of patients in OPTIC, and in 2% of patients in PACE. Some patients in PACE experienced recurrent or multisite vascular occlusion. Patients with and without cardiovascular risk factors, including patients age 50 years or younger, experienced these events. The most common risk factors observed with these events in PACE were history of hypertension, hypercholesterolemia, and non-ischemic cardiac disease. In OPTIC and PACE, AOEs were more frequent with increasing age.

    In OPTIC, patients with uncontrolled hypertension or diabetes and patients with clinically significant, uncontrolled, or active cardiovascular disease were excluded. In PACE, patients with uncontrolled hypertriglyceridemia and patients with clinically significant or active cardiovascular disease within the 3 months prior to the first dose of ICLUSIG were excluded. Consider whether the benefits of ICLUSIG are expected to exceed the risks.

    Monitor for evidence of AOEs. Interrupt, then resume at the same or decreased dose or discontinue ICLUSIG based on recurrence/severity. Consider benefit-risk to guide a decision to restart ICLUSIG.

    Venous Thromboembolic Events (VTEs): Serious or severe VTEs have occurred in patients who received ICLUSIG. In PACE, VTEs occurred in 6% of 449 patients including serious or severe (Grade 3 or 4) VTEs in 5.8% of patients. VTEs included deep venous thrombosis, pulmonary embolism, superficial thrombophlebitis, retinal vein occlusion, and retinal vein thrombosis with vision loss. The incidence was higher in patients with Ph+ ALL (9% of 32 patients) and BP-CML (10% of 62 patients). One of 94 patients in OPTIC experienced a VTE (Grade 1 retinal vein occlusion). Monitor for evidence of VTEs. Interrupt, then resume at the same or decreased dose or discontinue ICLUSIG based on recurrence/severity.

    Heart Failure: Fatal, serious or severe heart failure events have occurred in patients who received ICLUSIG. In PACE, heart failure occurred in 9% of 449 patients; 7% experienced serious or severe (Grade 3 or higher). Heart failure occurred in 12% of 94 patients in OPTIC; 1.1% experienced serious or severe (Grade 3 or 4). In PACE, the most frequently reported heart failure events (≥2%) were congestive cardiac failure (3.1%), decreased ejection fraction (2.9%), and cardiac failure (2%). In OPTIC, the most frequently reported heart failure events (>1 patient each) were left ventricular hypertrophy (2.1%) and BNP increased (2.1%). Monitor patients for signs or symptoms consistent with heart failure and manage heart failure as clinically indicated. Interrupt, then resume at reduced dose or discontinue ICLUSIG for new or worsening heart failure.

    Hepatotoxicity: ICLUSIG can cause hepatotoxicity, including liver failure and death. Fulminant hepatic failure leading to death occurred in 3 patients, with hepatic failure occurring within 1 week of starting ICLUSIG in one of these patients. These fatal cases occurred in patients with BP-CML or Ph+ ALL. Hepatotoxicity occurred in 25% of 94 patients in OPTIC and 32% of 449 patients in PACE. Grade 3 or 4 hepatotoxicity occurred in OPTIC (6% of 94 patients) and PACE (13% of 449 patients). The most frequent hepatotoxic events were elevations of ALT, AST, GGT, bilirubin, and alkaline phosphatase. Monitor liver function tests at baseline, then at least monthly or as clinically indicated. Interrupt, then resume at a reduced dose or discontinue ICLUSIG based on recurrence/severity.

    Hypertension: Serious or severe hypertension, including hypertensive crisis, has occurred in patients who received ICLUSIG. Patients may require urgent clinical intervention for hypertension associated with confusion, headache, chest pain, or shortness of breath. Monitor blood pressure at baseline and as clinically indicated and manage hypertension as clinically indicated. Interrupt, dose reduce, or stop ICLUSIG if hypertension is not medically controlled. For significant worsening, labile or treatment-resistant hypertension, interrupt ICLUSIG and consider evaluating for renal artery stenosis.

    Pancreatitis: Serious or severe pancreatitis has occurred in patients who received ICLUSIG. Elevations of lipase and amylase also occurred. In the majority of cases that led to dose modification or treatment discontinuation, pancreatitis resolved within 2 weeks. Monitor serum lipase every 2 weeks for the first 2 months and then monthly thereafter or as clinically indicated. Consider additional serum lipase monitoring in patients with a history of pancreatitis or alcohol abuse. Interrupt, then resume at the same or reduced dose or discontinue ICLUSIG based on severity. Evaluate for pancreatitis when lipase elevation is accompanied by abdominal symptoms.

    Increased Toxicity in Newly Diagnosed Chronic Phase CML: In a prospective randomized clinical trial in the first line treatment of newly diagnosed patients with CP-CML, single agent ICLUSIG 45 mg once daily increased the risk of serious adverse reactions 2-fold compared to single agent imatinib 400 mg once daily. The median exposure to treatment was less than 6 months. The trial was halted for safety. Arterial and venous thrombosis and occlusions occurred at least twice as frequently in the ICLUSIG arm compared to the imatinib arm. Compared to imatinib-treated patients, ICLUSIG-treated patients exhibited a greater incidence of myelosuppression, pancreatitis, hepatotoxicity, cardiac failure, hypertension, and skin and subcutaneous tissue disorders. ICLUSIG is not indicated and is not recommended for the treatment of patients with newly diagnosed CP-CML.

    Neuropathy: Peripheral and cranial neuropathy occurred in patients in OPTIC and PACE. Some of these events in PACE were Grade 3 or 4. Monitor patients for symptoms of neuropathy, such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain or weakness. Interrupt, then resume at the same or reduced dose or discontinue ICLUSIG based on recurrence/severity.

    Ocular Toxicity: Serious or severe ocular toxicity leading to blindness or blurred vision have occurred in ICLUSIG-treated patients. The most frequent ocular toxicities occurring in OPTIC and PACE were dry eye, blurred vision, and eye pain. Retinal toxicities included age-related macular degeneration, macular edema, retinal vein occlusion, retinal hemorrhage, and vitreous floaters. Conduct comprehensive eye exams at baseline and periodically during treatment.

    Hemorrhage: Fatal and serious hemorrhage events have occurred in patients who received ICLUSIG. Fatal hemorrhages occurred in PACE and serious hemorrhages occurred in OPTIC and PACE. The incidence of serious bleeding events was higher in patients with AP-CML, BP-CML, and Ph+ ALL. Gastrointestinal hemorrhage and subdural hematoma were the most frequently reported serious hemorrhages. Events often occurred in patients with Grade 4 thrombocytopenia. Monitor for hemorrhage and manage patients as clinically indicated. Interrupt, then resume at the same or reduced dose or discontinue ICLUSIG based on recurrence/severity.

    Fluid Retention: Fatal and serious fluid retention events have occurred in patients who received ICLUSIG. In PACE, one instance of brain edema was fatal and serious events included pleural effusion, pericardial effusion, and angioedema. Monitor for fluid retention and manage patients as clinically indicated. Interrupt, then resume at the same or reduced dose or discontinue ICLUSIG based on recurrence/severity.

    Cardiac Arrhythmias: Cardiac arrhythmias, including ventricular and atrial arrhythmias, occurred in patients in OPTIC and PACE. For some patients, events were serious or severe (Grade 3 or 4) and led to hospitalization. Monitor for signs and symptoms suggestive of slow heart rate (fainting, dizziness) or rapid heart rate (chest pain, palpitations or dizziness) and manage patients as clinically indicated. Interrupt, then resume at the same or reduced dose or discontinue ICLUSIG based on recurrence/severity.

    Myelosuppression: Grade 3 or 4 events of neutropenia, thrombocytopenia, and anemia occurred in patients in OPTIC and PACE. The incidence of myelosuppression was greater in patients with AP-CML, BP-CML, and Ph+ ALL than in patients with CP-CML. Obtain complete blood counts every 2 weeks for the first 3 months and then monthly or as clinically indicated. If ANC less than 1 x 109/L or platelets less than 50 x 109/L, interrupt ICLUSIG until ANC at least 1.5 x 109/L and platelets at least 75 x 109/L, then resume at same or reduced dose.

    Tumor Lysis Syndrome (TLS): Serious TLS was reported in ICLUSIG-treated patients in OPTIC and PACE. Ensure adequate hydration and treat high uric acid levels prior to initiating ICLUSIG.

    Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS (also known as Posterior Reversible Encephalopathy Syndrome) has been reported in patients who received ICLUSIG. Along with neurological signs and symptoms, hypertension may be present. Diagnosis is made with supportive findings on magnetic resonance imaging (MRI) of the brain. Interrupt ICLUSIG until resolution. The safety of resumption of ICLUSIG in patients upon resolution of RPLS is unknown.

    Impaired Wound Healing and Gastrointestinal Perforation: Impaired wound healing occurred in patients receiving ICLUSIG. Withhold ICLUSIG for at least 1 week prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of ICLUSIG after resolution of wound healing complications has not been established. Gastrointestinal perforation or fistula occurred in patients receiving ICLUSIG. Permanently discontinue in patients with gastrointestinal perforation.

    Embryo-Fetal Toxicity: Based on its mechanism of action and findings from animal studies, ICLUSIG can cause fetal harm when administered to a pregnant woman. In animal reproduction studies, adverse developmental effects occurred at exposures lower than human exposures at the recommended human dose. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with ICLUSIG and for 3 weeks after the last dose.

    ADVERSE REACTIONS

    The most common (>20%) adverse reactions are rash and related conditions, arthralgia, abdominal pain, headache, constipation, dry skin, hypertension, fatigue, fluid retention and edema, pyrexia, nausea, pancreatitis/lipase elevation, hemorrhage, anemia, hepatic dysfunction and AOEs. The most common Grade 3 or 4 laboratory abnormalities (>20%) are platelet count decreased, neutrophil cell count decreased, and white blood cell decreased.

    To report SUSPECTED ADVERSE REACTIONS, contact Takeda Pharmaceutical Co. Ltd. At 1-844-817-6468 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

    DRUG INTERACTIONS

    Strong CYP3A Inhibitors: Avoid coadministration or reduce ICLUSIG dose if coadministration cannot be avoided.

    Strong CYP3A Inducers: Avoid coadministration.

    USE IN SPECIFIC POPULATIONS

    Females and Males of Reproductive Potential: Verify pregnancy status of females of reproductive potential prior to initiating ICLUSIG.

    Ponatinib may impair fertility in females, and it is not known if these effects are reversible.

    Lactation: Advise women not to breastfeed during treatment with ICLUSIG and for 6 days following last dose.

    For more information about ICLUSIG, visit www.ICLUSIG.com. For the Prescribing Information including the Boxed Warning for arterial occlusion, venous thromboembolism, heart failure, and hepatoxicity, please visit https://www.iclusig.com/pdf/ICLUSIG-Prescribing-Information.pdf. For more information about ongoing research, please visit www.clinicaltrials.gov.

    Takeda's Commitment to Oncology

    Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it's with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

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  22. Mobocertinib, an Oral Targeted Therapy, Demonstrated a Median Overall Survival of 24 Months, and Responses were Observed across Diverse EGFR Exon20 Insertion Variants

    Results Included in Mobocertinib's New Drug Application (NDA), which was Recently Granted Priority Review by the U.S. Food and Drug Administration (FDA)

    Data to be Featured at ASCO Annual Meeting Reinforce Mobocertinib's Potential in a Patient Population with No Approved Targeted Therapies

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced updated data from the Phase 1/2 trial of mobocertinib (TAK-788) orally administered in patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small…

    Mobocertinib, an Oral Targeted Therapy, Demonstrated a Median Overall Survival of 24 Months, and Responses were Observed across Diverse EGFR Exon20 Insertion Variants

    Results Included in Mobocertinib's New Drug Application (NDA), which was Recently Granted Priority Review by the U.S. Food and Drug Administration (FDA)

    Data to be Featured at ASCO Annual Meeting Reinforce Mobocertinib's Potential in a Patient Population with No Approved Targeted Therapies

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced updated data from the Phase 1/2 trial of mobocertinib (TAK-788) orally administered in patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC) who received prior platinum-based chemotherapy. The results showed mobocertinib continued to demonstrate clinically meaningful benefit after over a year of follow up and will be presented at the virtual 57th American Society of Clinical Oncology (ASCO) Annual Meeting on June 4.

    "Patients with EGFR Exon20 insertion+ mNSCLC have no proven targeted therapy options," said Suresh S. Ramalingam, MD, Deputy Director of Winship Cancer Institute of Emory University. "The updated results from the Phase 1/2 study of mobocertinib demonstrate an encouraging objective response rate, duration of response and overall survival in patients who have received prior platinum-based chemotherapy."

    The analysis from the Phase 1/2 trial included patients with EGFR Exon20 insertion+ mNSCLC who received prior platinum-based chemotherapy. All patients were treated at the 160 mg once daily oral dose. Building on the findings presented in January at the International Association for the Study of Lung Cancer (IASLC) 2020 World Conference on Lung Cancer (WCLC), results showed a median overall survival (OS) of 24 months with a median follow up of 14 months, and responses were observed across diverse EGFR Exon20 insertion variants. Other key data points remained consistent with previously reported data, including a confirmed objective response rate (ORR) of 28%, a median duration of response (DoR) of 17.5 months and a disease control rate (DCR) of 78% per independent review committee (IRC).

    The safety profile observed was manageable and consistent with previous findings. The most common treatment-related adverse events (TRAEs; ≥ 20%) in platinum-pretreated patients from the updated data were diarrhea (91%), rash (45%), paronychia (38%), decreased appetite (35%), nausea (34%), dry skin (31%) and vomiting (30%). The only Grade ≥3 TRAE (≥5%) was diarrhea (21%). AEs leading to discontinuation in >2% were diarrhea (4%) and nausea (4%).

    "We are excited to add this promising overall survival data to the body of evidence demonstrating mobocertinib's potential as an effective oral treatment option for platinum-pretreated patients with EGFR Exon20 insertion+ mNSCLC," said Christopher Arendt, PhD, Head, Oncology Therapeutic Area Unit, Takeda. "Mobocertinib is currently undergoing priority review with the U.S. FDA, and we look forward to continuing conversations with regulatory agencies around the world to introduce mobocertinib as a new treatment option for these patients."

    The FDA previously granted mobocertinib Breakthrough Therapy Designation in April 2020 and priority review for the New Drug Application (NDA) in April 2021. If approved, mobocertinib will be the first oral therapy available that is specifically designed to selectively target EGFR Exon20 insertion mutations.

    Takeda has established an Expanded Access Program (EAP) for patients who may be eligible to receive access to mobocertinib while this investigational therapy is under review by regulatory authorities. Additional information, including the specific conditions to qualify for Takeda's EAP, is available here.

    Learn more about Takeda Oncology's presence at this year's ASCO Annual Meeting. Takeda will host a webcast for analysts and investors on Tuesday, June 8, at 6:30 p.m. ET to discuss these and other data being presented at ASCO and to provide an update on the oncology pipeline. Please contact TakedaRandDEvents@fticonsulting.com for further details. Presentation slides and an archived replay of the webcast will be available at https://www.takeda.com/investors/reports/ir-events/.

    About Mobocertinib (TAK-788)

    Mobocertinib is an investigational, first-in-class, oral tyrosine kinase inhibitor (TKI) specifically designed to selectively target epidermal growth factor receptor (EGFR) Exon20 insertion mutations. In 2019, the U.S. FDA granted mobocertinib Orphan Drug Designation for the treatment of lung cancer with HER2 mutations or EGFR mutations including Exon20 insertion mutations. In April 2020, mobocertinib received Breakthrough Therapy Designation from the FDA for patients with EGFR Exon20 insertion+ metastatic non-small cell lung cancer (mNSCLC) whose disease has progressed on or after platinum-based chemotherapy. In October 2020, mobocertinib was designated as a Breakthrough Therapy in China by the Center for Drug Evaluation (CDE) for locally advanced or metastatic NSCLC patients with EGFR Exon20 insertion mutations who have been previously treated with at least one prior systemic chemotherapy.

    About the Phase 1/2 Trial

    The Phase 1/2 trial evaluated the safety, pharmacokinetics and anti-tumor activity of oral mobocertinib in patients with non-small cell lung cancer (NSCLC). The trial is comprised of a Phase 1 dose-escalation, which evaluated mobocertinib as a monotherapy and in combination with chemotherapy, several expansion cohorts and an extension cohort in patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC).

    The platinum-pretreated population efficacy analysis investigated 114 patients with EGFR Exon20 insertion+ mNSCLC who received prior platinum-based therapy in the Phase 1/2 trial and were treated with mobocertinib at the 160 mg once daily dose.

    About EGFR Exon20 Insertion+ mNSCLC

    Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 85% of the estimated 1.8 million new cases of lung cancer diagnosed each year worldwide, according to the World Health Organization.1,2 Patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC) make up approximately 1-2% of patients with NSCLC, and the disease is more common in Asian populations compared to Western populations.3-7 This disease carries a worse prognosis than other EGFR mutations because there are currently no FDA-approved therapies that target EGFR Exon20 insertions, and current EGFR TKIs and chemotherapy provide limited benefit for these patients.

    Takeda is committed to continuing research and development in EGFR Exon20 insertion+ mNSCLC with the hope of introducing a targeted treatment option for the approximately 30,000 patients diagnosed with the disease worldwide each year, including 3,000 in the U.S. alone.3,4

    Takeda's Commitment to Oncology

    Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it's with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

    ###

    1 World Health Organization. Latest Global Cancer Data. https://www.who.int/cancer/PRGlobocanFinal.pdf. Accessed May 11, 2019.

    2 American Cancer Society. What is Non-Small Cell Lung Cancer? https://www.cancer.org/cancer/non-small-cell-lung-cancer/about/what-is-non-small-cell-lung-cancer.html.

    3 Riess, Jonathan W. Diverse EGFR Exon 20 Insertions and Co-Occurring Molecular Alterations Identified by Comprehensive Genomic Profiling of NSCLC. https://www.jto.org/article/S1556-0864(18)30770-6/fulltext. Accessed April 7, 2020.

    4 Fang, Wenfeng. BMC Cancer. EGFR exon 20 insertion mutations and response to osimertinib in non-small-cell lung cancer. https://bmccancer.biomedcentral.com/articles/10.1186/s12885-019-5820-0. Accessed April 7, 2020.

    5 Kobayashi Y, Mitsudomi T. Not all epidermal growth factor receptor mutations in lung cancer are created equal: Perspectives for individualized treatment strategy. Cancer Sci. 2016;107(9):1179-1186. doi:10.1111/cas.12996

    6 Yatabe Y, Kerr KM, Utomo A, et al. EGFR mutation testing practices within the Asia Pacific region: results of a multicenter diagnostic survey. J Thorac Oncol. 2015;10(3):438-445. doi:10.1097/JTO.0000000000000422

    7 Kris MG, Johnson BE, Berry LD, et al. Using multiplexed assays of oncogenic drivers in lung cancers to select targeted drugs. JAMA. 2014;311(19):1998-2006. doi:10.1001/jama.2014.3741

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    • Delivered FY2020 Management Guidance with Results Driven by +16% Underlying Revenue Growth of 14 Global Brands (e.g., ENTYVIO ® +26.2% YOY, TAKHZYRO® +30.0% YOY, Immunoglobulin + 15.7% YOY)
    • Accelerated Cost Synergies and Achieved $2.3 Billion Target One Year Ahead of Plan
    • Reported Net Profit Growth of Approximately +750% YOY, Reflecting Gains on Non-core Asset Sales and Lower Acquisition-related Expenses
    • Robust Operating Cash Flow and Divestiture Proceeds Resulting in Free Cash Flow of JPY 1,237.8 Billion, Driving Deleveraging to Net Debt / Adjusted EBITDA of 3.2x
    • FY2021 Anticipated to be an Inflection Year for Wave 1 Pipeline with Five to Six Wave 1 NMEs Submitted and Under Regulatory Review by the FDA with the Potential for Four
    • Delivered FY2020 Management Guidance with Results Driven by +16% Underlying Revenue Growth of 14 Global Brands (e.g., ENTYVIO ® +26.2% YOY, TAKHZYRO® +30.0% YOY, Immunoglobulin + 15.7% YOY)
    • Accelerated Cost Synergies and Achieved $2.3 Billion Target One Year Ahead of Plan
    • Reported Net Profit Growth of Approximately +750% YOY, Reflecting Gains on Non-core Asset Sales and Lower Acquisition-related Expenses
    • Robust Operating Cash Flow and Divestiture Proceeds Resulting in Free Cash Flow of JPY 1,237.8 Billion, Driving Deleveraging to Net Debt / Adjusted EBITDA of 3.2x
    • FY2021 Anticipated to be an Inflection Year for Wave 1 Pipeline with Five to Six Wave 1 NMEs Submitted and Under Regulatory Review by the FDA with the Potential for Four Approvals

    Takeda Pharmaceutical Company Limited (TYO:4502) (NYSE:TAK) ("Takeda") today announced financial results for fiscal year 2020 (period ended March 31, 2021).

    TAKEDA PRESIDENT AND CHIEF EXECUTIVE OFFICER CHRISTOPHE WEBER commented:

    "Over the course of FY2020, Takeda remained resilient as we operated in new ways through the COVID-19 pandemic. This is a testament to the dedication of our employees, and Takeda's unwavering commitment to serving patients, our people, and the planet to bring better health for people and a brighter future for the world. We maintained business continuity, ensured patient access to our medicines and safeguarded the health and well-being of our employees while helping to address the pandemic.

    "This focus enabled us to deliver on our full-year management guidance, with underlying revenue growth driven by our 14 global brands, and the acceleration of cost synergies contributed to strong margins and a robust cashflow. Furthermore, we continued to experience growth momentum in our pipeline, including receiving 12 approvals across Takeda's key markets.

    "With FY2021 anticipated to serve as a critical inflection year, we remain focused on leveraging our expected topline growth to ramp up our R&D investment and further fuel our transformative pipeline. We are well-positioned to reach our goal of mid-single-digit revenue growth over the next decade amounting to JPY5 trillion ($47 billion) by FY2030.1

    "As we celebrate Takeda's 240th anniversary next month, I am extremely proud of our progress and confident in our outlook for the future. We will advance on our growth trajectory, maximize value creation for all of our stakeholders, and continue to position the company for long-term success."

    FINANCIAL AND BUSINESS HIGHLIGHTS

    Results for FY2020 Ended March 31, 2021

    (Billion yen, except

    percentages and per

    share amounts)

    REPORTED

    CORE

    (Non-IFRS)(a)

    UNDERLYING (b)

    (Non-IFRS) (a)

    FY2020

    vs. PRIOR

    YEAR

    FY2020

    vs. PRIOR

    YEAR

     

    Revenue

    3,197.8

    -2.8%

    3,197.8

    -2.8%

    +2.2%

    Operating Profit

    509.3

    +407.2%

    967.9(c)

    +0.6%

    +13.0%

    Margin

    15.9%

    +12.9pp

    30.3%

    +1.0pp

    30.2%

    Net Profit

    376.0

    +749.9%

    655.5

    +8.9%

     

    EPS (JPY)

    241

    +747.3%

    420

    +8.5%

    +24.6%

    Operating Cash Flow

    1,010.9

    +50.9%

     

     

     

    Free Cash Flow

    (Non-IFRS) (a) (d)

    1,237.8

    +27.9%

     

     

     

    (a) Further information on certain of Takeda's Non-IFRS measures is posted on Takeda's investor relations website at https://www.takeda.com/investors/financial-results/

    (b) Underlying growth compares two periods (quarters or years) of financial results under a common basis and is used by management to assess the business. These financial results are calculated on a constant currency basis and excluding the impact of divestitures and other amounts that are unusual, non-recurring items or unrelated to our ongoing operations.

    (c) Core Operating Profit represents net profit adjusted to exclude income tax expenses, the share of profit or loss of investments accounted for using the equity method, finance expenses and income, other operating expenses and income, amortization and impairment losses on acquired intangible assets and other items unrelated to Takeda's core operations, such as purchase accounting effects and transaction related costs.

    (d) Free Cash Flow represents cash flows from operating activities, excluding acquisition of plant, property and equipment, intangible assets and investments, and any other cash that is not available to Takeda's immediate or general business use, and including proceeds from sales of plant, property and equipment, as further adjusted to exclude the acquisition of intangible assets and the acquisition of investments, and to include the proceeds from sales of property, plant, sales and redemption of investments and businesses, net of cash and cash equivalents divested.

    FY2020 RESULTS DEMONSTRATE TAKEDA'S RESILIENT PORTFOLIO

    • Reported revenueat JPY 3,197.8 billion (~$28.9B)2, declined by 2.8% impacted primarily by foreign exchange and divestitures. Underlying revenue growth in FY2020 was +2.2% driven by the growth of Takeda's 14 global brands, up 16% year-on-year.
    • Takeda delivered reported operating profit of JPY 509.3 billion (~$4.6B)2, which grew 407.2% with gains from non-core asset sales and acquisition-related expenses. Core operating profit, which adjusts for purchase price accounting ("PPA") and non-recurring items (including gains on sales of assets), increased year-on-year to JPY 967.9 billion (~$8.8B)2. The core operating profit margin was 30.3%. Underlying core operating profit, which further adjusts for the impact of foreign exchange and divestitures, grew 13% year-on-year. The underlying core operating profit margin was 30.2%.
    • Takeda's reported net profit was JPY 376 billion, a 749.9% increase compared with the same period in the prior year.
    • Operating cash flow increased by 50.9% to JPY 1,010.9 billion. There was an increase in other financial liabilities of JPY 175.5 billion primarily attributable to an increase of deposits restricted to certain vaccines operations.
    • Free cash flow, which adjusts out deposits restricted to certain vaccine operations and reflects capital expenditures and proceeds from asset sales, was JPY 1,237.8 billion (~$11.2B)2. This represented an increase of 27.9% versus the prior year, comfortably covering the full year dividend, debt repayment and interest. Robust cash flow enabled further de-leveraging in Q4.

    The overall impact of the global spread of COVID-19 on Takeda's consolidated financial results for the twelve-month period ended March 31, 2021, was not material, with several offsetting factors. There were adverse effects due to COVID-19 observed in certain therapeutic areas, especially in Neuroscience when stay-at-home restrictions reduced patient visits to medical care providers. This trend has fluctuated throughout the twelve-month period. These adverse effects have been partially offset by benefits from prescribing trends during the pandemic, such as an expansion of certain products with a more convenient administration profile. Regarding operating expenses, voluntary suspension of certain business activities such as business travel and events in response to COVID-19 led to lower spending. As a result of these factors, the impact on Takeda's profit was immaterial.

    For the latest Takeda communications regarding COVID-19, please click here to visit the COVID-19 Information Center on Takeda's website.

    COMMERCIAL UPDATES ACROSS FIVE KEY BUSINESS AREAS

    Takeda's five key business areas — Gastroenterology, Rare Diseases, Plasma-Derived Therapies, Oncology and Neuroscience — with JPY 2,623.7 billion of reported revenue representing approximately 82% of total FY2020 revenues – delivered year-on-year underlying revenue growth of 4.7%. Takeda's 14 global brands, with reported revenue of JPY 1,215.3 billion (~$11.0B) 2 in aggregate, delivered a 16% increase in FY2020 underlying revenue growth compared to a year before.

    Gastroenterology

    Gastroenterology with JPY 777.8 billion in reported revenue represented 24% of sales, with underlying revenue growth of 14%. This was spearheaded by continued exceptional growth through expanded first line share of gut selective ENTYVIO in the U.S., EU and Japan.

    Rare Diseases

    Rare Diseases with JPY 591.7 billion in reported revenue represented 19% of sales, with underlying revenue declining 2% driven by a decline in rare hematology that was in line with expectations. The hereditary angioedema (HAE) portfolio saw 10% underlying revenue growth, driven by continued excellent performance from TAKHZYRO, which continues to expand the hereditary angioedema prophylaxis market, as well as launching into additional geographies. Rare Metabolic increased 2% on an underlying basis but excluding NATPARA® the portfolio saw 8% underlying growth.

    PDT Immunology

    PDT Immunology with JPY 420.4 billion in reported revenue represented 13% of sales, with underlying revenue growth of 10% driven by strong Gammagard-Liquid demand in the U.S. and subcutaneous IG worldwide. Albumin underlying revenue declined 13% in FY2020, mostly due to H2 sales impact caused by temporary interruption in submitting batches of Albumin Glass for release in China, and in some part due to phasing and supply dynamics in China in FY2019. COVID-19 has impacted plasma collections industry-wide, but operational excellence, implementation of digital initiatives and ongoing center expansion helped limit plasma collection volume decline to only -11%.

    Oncology

    Oncology with JPY 416.5 billion in reported revenue represented 13% of sales, with underlying revenue growth of 1% driven by NINLARO®, ADCETRIS® and ALUNBRIG®. Takeda's strong oncology portfolio continues to expand indications as growth brands offset the decline of older products in the portfolio.

    Neuroscience

    Neuroscience with JPY 417.3 billion in reported revenue represented 13% of sales, declining 2% on an underlying basis. The portfolio experienced a slowdown in momentum attributable to COVID-19 stay-at-home restrictions that reduced patient visits and diagnoses. A recovery of prescribing trends has been noted, but new patient starts are not yet back to pre-COVID levels.

    Global Brand Highlights

    Business Area

    Product

    Reported Product

    Revenue


    JPY (billion)

    Year-over-Year Underlying

    Revenue Growth

    Gastroenterology

    ENTYVIO

    429.3

    +26.2%

    Rare Diseases

    TAKHZYRO

    86.7

    +30.0%

    PDT Immunology

    Immunoglobulin

    334.9

    +15.7%

    Oncology

    NINLARO

    87.4

    +15.9%

    Oncology

    ALUNBRIG

    8.8

    +24.4%

    COST SAVINGS AND DIVESTITURES

    Synergy deliverables and operational efficiencies supported margin performance, as Takeda delivered an underlying core operating profit margin of 30.2%. Takeda is also deleveraging rapidly, with a net debt/adjusted EBITDA ratio of 3.2x at the end of Q4, down from 3.8x in March 2020. Net debt has decreased by JPY 1,668.5 billion in two years since March 31, 2019 (the Shire Acquisition closed on January 8, 2019), and Takeda is on course to meet its medium-term deleveraging goal of 2x (low-twos) within FY2021-FY2023.

    Takeda exceeded its $10B non-core asset divestiture target and has announced 12 deals since January 2019 to date for a total aggregate value of up to ~$12.9 billion3, most recently including:

    • The completion of the previously announced sale of a portfolio of four type 2 diabetes products to Teijin Pharma Limited for a total value of JPY 133.0 billion.4 (Press Release)
    • The completion of the previously announced sale of a portfolio of approximately 130 select over the counter and prescription products, and two manufacturing sites to Orifarm for a total value of up to $670 million USD. (Press Release)
    • The completion of the previously announced sale of Takeda Consumer Healthcare Company Limited to Oscar A-Co KK, a company controlled by funds managed by The Blackstone Group Inc. and its affiliates, for a total value of JPY 227.7 billion.5 (Press Release)

    Takeda has also exceeded its original $700 million target for incremental cash from sales of real estate and marketable securities in FY2020, receiving a total of ~$1.4B.

    PIPELINE UPDATE: FY2021 – ANTICIPATED TO BE AN INFLECTION YEAR WITH FIVE TO SIX WAVE 1 NMES SUBMITTED AND UNDER REGULATORY REVIEW BY THE FDA WITH THE POTENTIAL FOR FOUR APPROVALS

    Takeda's world-class R&D engine is fueled by leveraging internal research capabilities and actively engaging with innovative ecosystems around the world. This efficient R&D model has enabled us to focus on more targeted patient populations where there is potential for greater therapeutic benefit, smaller and less costly development programs, and faster tracks to registration with enhanced patent protection and marketing rights. We plan to increase our R&D investment to 522 billion JPY to further advance our 40+ prioritized NMEs and new partnerships, as well as building additional capabilities in oncology, clinical trial initiation and data and digital sciences. In FY2020, we obtained 12 global and regional brand approvals with the U.S., EU, China and Japan, demonstrating our drive to develop best-in-class therapies for patients with high unmet needs around the world. FY2021 is expected to be an inflection year for Takeda as we anticipate up to six regulatory submissions by year-end FY2021, with the potential for four approvals.

    Takeda's pipeline portfolio has the potential to contribute significantly to its growth over the next decade, with recent highlights including:

    • TAK-003:Takeda's tetravalent dengue vaccine candidate, has completed its first regulatory submissions with possible approval in the EU and some endemic countries in FY2021. (Press release)
    • Mobocertinib (TAK-788): A potential new oral standard of care for adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC), has completed its New Drug Application (NDA) submission with a potential approval in FY2021. (Press release)
    • Maribavir (TAK-620): A robust primary analysis from the Phase 3 trial showed significantly more patients achieved cytomegalovirus (CMV) clearance versus conventional therapies in transplant recipients with CMV infection. Met key secondary endpoints, maintaining superior CMV viremia clearance, on track for NDA submission. Takeda continues to investigate Maribavir in the ongoing 302 Phase 3 study for First-Line Treatment of CMV in Hematopoietic Cell Transplant Recipients. (Press release)
    • Soticlestat (TAK-935): Takeda recently re-acquired global rights from Ovid Therapeutics to develop and commercialize this potential first-in-class therapy, with a novel mechanism of action for the treatment of developmental and epileptic encephalopathies. We intend to initiate Phase 3 studies of soticlestat in children and young adults with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in FY2021. If successful, it has the potential to bring new treatment options that provide greater seizure control, tolerability and function to DS and LGS patients around the world. (An Inflection Year for Our Wave 1 Pipeline)
    • Orexin (TAK-994): Potentially the first therapy to treat orexin deficiency, Takeda plans to move this asset into registrational trials, first in narcolepsy type 1 (NT1) — a rare, underdiagnosed and undertreated condition caused by an orexin deficiency which disrupts the sleep awake cycles, with narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH) to follow as potential additional indications. (An Inflection Year for Our Wave 1 Pipeline)
    • TAK-186: A conditionally active T-cell engager, first in its class to enter the clinic with recent phase 1/2 study initiation in EGFR-expressing solid tumors. TAK-186 is one of the latest additions to our pipeline through the acquisition of Maverick Therapeutics. In addition to TAK-186, Takeda also obtained Maverick's T-cell engager COBRA™ platform and TAK-280 which is expected to enter the clinic in the second half of FY2021 for the treatment of patients with B7H3-expressing solid tumors. (Press release)

    KEY CORPORATE INITIATIVES

    Several recent examples of Takeda's corporate achievements in FY2020 demonstrate Takeda's progress toward its purpose of "better health for people, brighter future for the world":

    Patients:

    • Launched R&D Center for Health Equity and Patient Affairs to identify and address health inequities.
    • Developed the Health Outcomes Observatory (H20) project, which brings together diverse public and private partners to amplify the patient voice in Europe.
    • Awarded the 2021 Facility of the Year Awards (FOYA) by the International Society for Pharmaceutical Engineering (ISPE) in two categories. Takeda's new solid pharmaceutical packaging building in Hikari, Japan, was recognized with the 2021 "Process Intelligence and Innovation" category award and the end-to-end high potent drug facility in Grange Castle, Ireland, was selected as "Facility Integration" category winner.
    • Earned an industry-leading position within the 2021 Access to Medicine (AtM) Index where the company ranked sixth overall and led the pharmaceutical industry in Governance of Access.

       

    People:

    • Launched our first Global DE&I Council, led by members of the Takeda Executive Team, to further embed DE&I into our culture.
    • Achieved global Top Employer® certification for fourth consecutive year and was named as a Top Employer in four regions and 38 countries.
    • Preparing for post-pandemic ways of working with new hybrid working models that foster a flexible working culture, aligned to local business needs, that optimize employee engagement.

    Planet:

    • Achieved carbon neutrality in the value chain for fiscal year 2019.
    • Named to Corporate Knights Global 100 Most Sustainable Corporations in the World (Global 100) for the sixth consecutive year.

    Governance:

    • Takeda recently announced candidates for its Board of Directors that will be proposed at the 145th Ordinary General Meeting of Shareholders to be held on June 29, 2021. As part of its commitment to exercising strong corporate governance practices, Takeda decided that all members of the Audit and Supervisory Committee will be external directors (as defined under the rules of the Tokyo Stock Exchange) to further enhance the independence of the Committee. This change will promote the long-term interests of shareholders and all its stakeholders as well as strengthening its Board of Directors and management accountability. (Press release)

    COVID-19 UPDATE

    Guided by its values, Takeda's response to COVID-19 has focused on protecting the health and safety of employees, striving to ensure its medicines are available to patients who rely on them and playing a part to reduce transmission and support the communities where its employees live and work.

    While the results of our CoVIg-19 Plasma Alliance clinical trial were not favorable, the effort strengthened relationships within and outside the industry, enabled a renewed perspective toward pragmatic regulation based on scientific evidence and need, and provided a well-defined, legally compliant framework for future collaborative opportunities to address urgent public health needs. Takeda has also undertaken several efforts to help the world respond to COVID-19, and our most recent accomplishments include:

    • Takeda is making two COVID-19 vaccines available in Japan, by manufacturing Novavax' recombinant vaccine candidate and distributing Moderna's mRNA vaccine candidate, with the support of the Ministry of Health, Labour and Welfare and the Japan Agency for Medical Research and Development (AMED). Pending regulatory approval, Takeda intends to start distributing TAK-919 (Moderna) in the first half of 2021 and aims to start distributing TAK-019 (Novavax) in late 2021 or early 2022. (Press Release)
    • Takeda and IDT Biologika GmbH (IDT) have a mutual agreement to support manufacturing of Johnson & Johnson's COVID-19 vaccine for three months utilizing capacity previously reserved for Takeda's dengue vaccine candidate. (Press Release)

       

    FY2021 GUIDANCE: Growth Momentum Expected to Continue

    (Billion Yen, Except Percentages)

    FY2020

    RESULTS

    FY2021

    FORECAST

    Underlying

    Management Guidance

    Revenue

    3,197.8

    3,370.0

    Mid-single-digit growth

    Reported Operating Profit

    509.3

    488.0

     

    Core Operating Profit

    967.9

    930.0

    Mid-single-digit growth

    Core Operating Profit Margin

    30.3%

     

    ~30% margin

    Reported Net Profit

    376.0

    250.0

     

    Reported EPS (Yen)

    241

    160

     

    Core EPS (Yen)

    420

    394

    Mid-single-digit growth

    Free Cash Flow

    1,237.8

    600.0 - 700.0

     

    Annual Dividend per Share (Yen)

    180

    180

    Takeda has solid growth momentum heading into FY2021 and expects underlying revenue growth to accelerate to "mid-single-digit" driven by continued momentum of Takeda's 14 global brands.

    Reported revenue is forecast to be 3,370 billion JPY, a year-on-year increase of 172.2 billion JPY or +5.4% from FY2020, with underlying revenue momentum and a one-time gain from the sale of diabetes portfolio in Japan fully offsetting impacts from divestitures completed in FY2020.

    Underlying Core Operating Profit and Underlying Core EPS are expected to also grow at "mid-single-digit", reflecting revenue growth and continued cost efficiencies, whilst also incorporating a significant increase in R&D expenses to support Takeda's innovative pipeline.

    Reported Operating Profit is expected to be 488 billion JPY, a decrease of 21.3 billion JPY, or -4.2%, impacted by a significant increase in R&D expenses as well as lower one-time gains on asset sales. Core Operating Profit is expected to decrease by 37.9 billion JPY, or -3.9%, to 930 billion JPY, reflecting a significant increase in R&D expenses. Reported net profit for the year is expected to be 250 billion JPY, a decrease of 126 billion JPY, or -33.5%, reflecting the impacts on reported operating profit as well as an expected increase in the effective tax rate.

    Key Assumptions in FY2021 Forecast

    Company guidance reflects management's expectations for continued business momentum across Takeda's five key business areas, underlying revenue growth of its 14 global brands, and accelerated realization of cost synergies.

    FY2021 guidance also reflects the following key assumptions: (i) The gain on sale of a diabetes portfolio in Japan is booked as revenue (JPY 133 billion), and adjusted out of Core Operating Profit for FY2021; (ii) Takeda expects at least one 505(b)2 competitor for subcutaneous VELCADE® to launch in the U.S. around mid FY2021; (iii) Takeda does not expect to restart sales of NATPARA in the U.S. market in FY2021; (iv) FY2021 guidance does not include the impact of any potential further divestitures beyond what has already been disclosed by Takeda.

    Based on currently available information, Takeda believes its financial results for FY2021 will not be materially affected by COVID-19 and, accordingly, Takeda's FY2021 forecast reflects this belief. However, the situation surrounding COVID-19 remains highly fluid, and future COVID-19-related developments in FY2021, including new or additional COVID-19 outbreaks and additional or extended lockdowns, shelter-in-place orders or other government action in major markets, could result in further or more serious disruptions to Takeda's business, such as slowdowns in demand for Takeda's products, supply chain related issues or significant delays in its clinical trial programs. These events, if they occur, could result in additional impacts on Takeda's business, results of operations or financial condition, as well as resulting in significant deviations from Takeda's FY2021 forecast.

    For more details on Takeda's FY2020 results and other financial information, please visit: https://www.takeda.com/investors/financial-results/

    Further Information

    Takeda will share details regarding its commercial strategies in oncology and finance at its upcoming Oncology Strategic Update Call in June 2021 and Finance Strategy Day in June/July 2021, respectively (dates to be confirmed). Additionally, Takeda will share details regarding COVID-19 efforts, the current state of the business, and the short- and long-term outlook of the Company at the Annual General Meeting of Shareholders​ on June 29, 2021.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited (TYO: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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    Certain Non-IFRS Financial Measures

    This press release and materials distributed in connection with this press release include certain IFRS financial measures not presented in accordance with International Financial Reporting Standards ("IFRS"), such as Underlying Revenue, Core Operating Profit, Underlying Core Operating Profit, Core Net Profit, Underlying Core EPS, Net Debt, EBITDA, Adjusted EBITDA and Free Cash Flow. Takeda's management evaluates results and makes operating and investment decisions using both IFRS and non-IFRS measures included in this press release. These non-IFRS measures exclude certain income, cost and cash flow items which are included in, or are calculated differently from, the most closely comparable measures presented in accordance with IFRS. By including these non-IFRS measures, management intends to provide investors with additional information to further analyze Takeda's performance, core results and underlying trends. Takeda's non-IFRS measures are not prepared in accordance with IFRS and such non-IFRS measures should be considered a supplement to, and not a substitute for, measures prepared in accordance with IFRS (which we sometimes refer to as "reported" measures). Investors are encouraged to review the reconciliation of non-IFRS financial measures to their most directly comparable IFRS measures.

    Further information on certain of Takeda's Non-IFRS measures is posted on Takeda's investor relations website at https://www.takeda.com/investors/reports/quarterly-announcements/

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

    Financial information

    Takeda's financial statements are prepared in accordance with International Financial Reporting Standards ("IFRS"). The revenue of Shire plc ("Shire"), which was historically, presented by Shire in accordance with accounting principles generally accepted in the United States ("U.S. GAAP"), has been conformed to IFRS, without material difference. Convenience translations of JPY figures into USD are included for reference and have been calculated at a rate of JPY/USD of 110.6. During FY2019, Takeda completed the purchase price allocation for the assets acquired and the liabilities assumed as part of the Shire acquisition. Accordingly, PL statements for FY2019 Q3 were retrospectively adjusted.


    1 Includes incremental revenue not adjusted for Probability of Technical Success (PTS) and is not a "forecast" or "target" figure. PTS applies to the probability that a given clinical trial/study will be successful based on pre-defined endpoints, feasibility and other factors and regulatory bodies will grant approval. Actual future net sales achieved by our commercialized products and pipelines will be different, perhaps materially so, as there is a range of possible outcomes from clinical development, driven by a number of variables, including safety, efficacy and product labelling. If a product is approved, the effect of commercial factors including the patient population, the competitive environment, pricing and reimbursement is also uncertain.

    2 USD included for reference, calculated at JPY/USD of 110.6

    3 Includes transactions yet to close and the full value of milestones and other contingent payments not guaranteed to be made.

    4 The sales price includes the price of product inventory. The sales price will be adjusted based on the value of the product inventory on the date of the completion of the asset transfer.

    5 Enterprise value. Actual sales price will be determined after adjustment for items including net debt and working capital of TCHC and Takeda Healthcare Products Company Limited ("THP").

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    • Both Awards, Issued by the International Society for Pharmaceutical Engineering (ISPE), Underscore Takeda's Leadership in Digital and Innovative Technologies
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    • Both Awards, Issued by the International Society for Pharmaceutical Engineering (ISPE), Underscore Takeda's Leadership in Digital and Innovative Technologies
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    • End-to-End High Potent Drug Facility in Grange Castle, Ireland, Is Recognized as Category Winner for Facility Integration

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that it was awarded by the International Society for Pharmaceutical Engineering (ISPE) for the 2021 Facility Of the Year Awards (FOYA) in two categories. Takeda's new solid pharmaceutical packaging building in Hikari, Japan, was recognized with the 2021 "Process Intelligence and Innovation" category award. Additionally, the end-to-end high potent drug facility in Grange Castle, Ireland, was selected as "Facility Integration" category winner.

    "I am honored that Takeda receives two awards for the production and packaging of small molecule solid dosage form products," said Thomas Wozniewski, global manufacturing & supply officer of Takeda. "In 2018, the Los Angeles plasma facility received two FOYA awards, now, two of our sites in Japan and in Europe got awarded in the categories ‘Process intelligence and innovation' as well as ‘Facility integration'. This illustrates that Takeda is constantly investing into state of the art facilities applying best in class process as well as digital standards. Both projects in Hikari and in Grange Castle also demonstrate that significant technology improvements for small molecule modalities are still achievable, both in a cost as well as in a time efficient manner."

    Takeda's Hikari plant is located in the south of Japan in the Yamaguchi prefecture. One of Takeda`s largest plants, it features advanced production systems for active pharmaceutical ingredients (API), drug formulation, biological products, and others offering a stable supply of high-quality pharmaceutical products throughout the world. The project at the Hikari site is a four-story building designed to elevate pharmaceutical packaging operations to a new industrial standard. The facility features highly automated end-to-end packaging equipment, including "end of line" case packers, automated guided vehicles (AGVs) and robots to feed the automated storage and retrieval system (ASRS). Additionally, the site developed and introduced an automatic line clearance system (ALC) with 360° cameras and laser sensors utilizing artificial intelligence (AI) to help significantly increase efficiency in the pharmaceutical packaging process. Takeda's Hikari plant exemplifies how novel application of commercially available and custom developed process manufacturing tools leads to superior results and advanced process understanding.

    The Grange Castle site in Ireland is located in the Dublin area, and it includes three manufacturing facilities. The production facility which now got awarded by the ISPE is a standalone, high containment, cutting-edge production facility dedicated to manufacturing Takeda's treatment for multiple myeloma. The application of good design practices and superior conceptual planning led to the excellent integration of facility and process. The innovative design as an ‘all-in-one' facility incorporates the entire end-to-end production process from active pharmaceutical ingredients to drug product and packaging under one roof. This significantly simplifies the supply chain for one of Takeda´s global oncology products to ensure unconstrained availability to patients worldwide.

    "The design teams have implemented best-in-class digital technologies to guarantee the facilities use the latest developments in paperless automation, robotics and augmented reality. This strategic use of digital and automated systems has led to a state-of-the-art facility design that positions Takeda as a frontrunner in our industry," added Gunter Baumgartner, head of Global Engineering at Takeda.

    An official ceremony of all winning projects and awards is planned at this year's ISPE Annual Meeting & Expo in Boston, MA, in November.

    About the ISPE Facility of the Year Awards Program

    Established in 2004, the Facility of the Year Awards (FOYA) recognizes state-of-the-art projects utilizing new, innovative technologies to improve the quality of products, reduce the cost of producing high-quality medicines, and demonstrate advances in project delivery. The FOYA program provides a platform for the pharmaceutical science and manufacturing industry to showcase its accomplishments in facility design, construction, and operations, while sharing the development of new applications of technology and cutting-edge approaches. For more information, visit https://ispe.org/facility-year-awards.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

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  23. Prescription Drug User Fee Act (PDUFA) Target Action Date Set for October 26, 2021

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the U.S. Food and Drug Administration (FDA) has granted priority review for the company's New Drug Application (NDA) for mobocertinib (TAK-788) for the treatment of adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC), as detected by an FDA-approved test, who have received prior platinum-based chemotherapy. Mobocertinib is the first oral therapy specifically designed to selectively target EGFR Exon20 insertion mutations.

    "Patients with EGFR Exon20 insertion+ mNSCLC face considerable…

    Prescription Drug User Fee Act (PDUFA) Target Action Date Set for October 26, 2021

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the U.S. Food and Drug Administration (FDA) has granted priority review for the company's New Drug Application (NDA) for mobocertinib (TAK-788) for the treatment of adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC), as detected by an FDA-approved test, who have received prior platinum-based chemotherapy. Mobocertinib is the first oral therapy specifically designed to selectively target EGFR Exon20 insertion mutations.

    "Patients with EGFR Exon20 insertion+ mNSCLC face considerable challenges, as current treatment options provide limited benefit, resulting in poor survival outcomes," said Christopher Arendt, head, Oncology Therapeutic Area Unit, Takeda. "We are excited to be one step closer to offering mobocertinib as an effective oral therapy for NSCLC patients with EGFR Exon20 insertions that have received prior platinum-based chemotherapy and look forward to continuing conversations with regulatory agencies in the U.S. and around the globe."

    The NDA for mobocertinib is primarily based on results from the Phase 1/2 trial, which is evaluating the safety and efficacy of oral mobocertinib in patients with mNSCLC. The application was submitted under the FDA's accelerated approval program. The review is being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence (OCE), which provides a framework for concurrent submission and review of oncology products among international partners.

    Takeda has established an Expanded Access Program (EAP) (NCT04535557) for patients in the U.S. who may be eligible to receive access to mobocertinib during the review of the NDA. Additional information about Takeda's EAP is available here.

    About Mobocertinib (TAK-788)

    Mobocertinib is an investigational, first-in-class, oral tyrosine kinase inhibitor (TKI) specifically designed to selectively target epidermal growth factor receptor (EGFR) Exon20 insertion mutations. In 2019, the U.S. FDA granted mobocertinib Orphan Drug Designation for the treatment of lung cancer with HER2 mutations or EGFR mutations including Exon20 insertion mutations. In April 2020, mobocertinib received Breakthrough Therapy Designation from the FDA for patients with EGFR Exon20 insertion+ metastatic non-small cell lung cancer (mNSCLC) whose disease has progressed on or after platinum-based chemotherapy. In October 2020, mobocertinib was designated as a Breakthrough Therapy in China by the Center for Drug Evaluation (CDE) for locally advanced or metastatic NSCLC patients with EGFR Exon20 insertion mutations who have been previously treated with at least one prior systemic chemotherapy.

    About the Phase 1/2 Trial

    The Phase 1/2 trial aims to evaluate the safety, pharmacokinetics and anti-tumor activity of oral mobocertinib in patients with non-small cell lung cancer (NSCLC). The trial is comprised of a Phase 1 dose-escalation, evaluating mobocertinib as a monotherapy and in combination with chemotherapy, several expansion cohorts and an extension cohort in patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC).

    The platinum-pretreated population efficacy analysis investigated 114 patients with EGFR Exon20 insertion+ mNSCLC who received prior platinum-based therapy in the Phase 1/2 trial and were treated with mobocertinib at the 160 mg once daily dose.

    About EGFR Exon20 Insertion+ mNSCLC

    Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 85% of the estimated 1.8 million new cases of lung cancer diagnosed each year worldwide, according to the World Health Organization.1,2 Patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC) make up approximately 1-2% of patients with NSCLC, and the disease is more common in Asian populations compared to Western populations.3-7 This disease carries a worse prognosis than other EGFR mutations because there are currently no FDA-approved therapies that target EGFR Exon20 insertions, and current EGFR TKIs and chemotherapy provide limited benefit for these patients.

    Takeda is committed to continuing research and development in EGFR Exon20 insertion+ mNSCLC with the hope of introducing a targeted treatment option for the approximately 30,000 patients diagnosed with the disease worldwide each year, including 3,000 in the U.S. alone.3,4

    Takeda's Commitment to Oncology

    Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it's with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

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  24. CAMBRIDGE, Mass., ROSTOCK, Germany and BERLIN, April 14, 2021 (GLOBE NEWSWIRE) -- Centogene N.V. (NASDAQ:CNTG), a commercial-stage company focused on rare diseases that transforms real-world clinical and genetic data into actionable information for patients, physicians, and pharmaceutical companies, announced today that it has extended its partnership with Takeda Pharmaceutical Company Limited (NYSE:TAK) to diagnose patients with certain genetic disorders.

    As part of the agreement, which has been extended until March 2022, CENTOGENE will continue providing access to genetic testing to patients around the world.

    Andrin Oswald, M.D., CEO of CENTOGENE, said, "We are pleased to be extending our partnership with Takeda, which will allow us to…

    CAMBRIDGE, Mass., ROSTOCK, Germany and BERLIN, April 14, 2021 (GLOBE NEWSWIRE) -- Centogene N.V. (NASDAQ:CNTG), a commercial-stage company focused on rare diseases that transforms real-world clinical and genetic data into actionable information for patients, physicians, and pharmaceutical companies, announced today that it has extended its partnership with Takeda Pharmaceutical Company Limited (NYSE:TAK) to diagnose patients with certain genetic disorders.

    As part of the agreement, which has been extended until March 2022, CENTOGENE will continue providing access to genetic testing to patients around the world.

    Andrin Oswald, M.D., CEO of CENTOGENE, said, "We are pleased to be extending our partnership with Takeda, which will allow us to continue diagnosing and connecting rare disease patients globally. There is still a lot of work to be done, but with each step, we are closer to bringing patients the life-saving medical solutions they need."

    In January 2015, CENTOGENE originally entered into an agreement with Shire Pharmaceuticals, which is now a subsidiary of Takeda Pharmaceutical Company Limited, to provide diagnostic testing capability to enhance early diagnosis of patients suffering from genetic rare diseases.

    About CENTOGENE

    CENTOGENE engages in diagnosis and research around rare diseases transforming real-world clinical and genetic data into actionable information for patients, physicians, and pharmaceutical companies. Our goal is to bring rationality to treatment decisions and to accelerate the development of new orphan drugs by using our extensive rare disease knowledge, including epidemiological and clinical data, as well as innovative biomarkers. CENTOGENE has developed a global proprietary rare disease platform based on our real-world data repository with over 3.6 billion weighted data points from approximately 595,000 patients representing over 120 different countries as of September 30, 2020.



    The Company's platform includes epidemiologic, phenotypic, and genetic data that reflects a global population, and also a biobank of these patients' blood samples. CENTOGENE believes this represents the only platform that comprehensively analyzes multi-level data to improve the understanding of rare hereditary diseases, which can aid in the identification of patients and improve our pharmaceutical partners' ability to bring orphan drugs to the market. As of September 30, 2020, the Company collaborated with over 40 pharmaceutical partners covering over 45 different rare diseases.

    Important Notice and Disclaimer



    This press release contains statements that constitute "forward-looking statements" as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Company's opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include discussion of our strategies, financing plans, growth opportunities, and market growth. In some cases, you can identify such forward-looking statements by terminology such as "anticipate," "intend," "believe," "estimate," "plan," "seek," "project" or "expect," "may," "will," "would," "could," or "should," the negative of these terms or similar expressions. Forward-looking statements are based on management's current beliefs and assumptions and on information currently available to the Company. However, these forward-looking statements are not a guarantee of our performance, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks, uncertainties, and other variable circumstances, such as negative worldwide economic conditions and ongoing instability and volatility in the worldwide financial markets, the effects of the COVID-19 pandemic on our business and results of operations, possible changes in current and proposed legislation, regulations and govern-mental policies, pressures from increasing competition and consolidation in our industry, the expense and uncertainty of regulatory approval, including from the U.S. Food and Drug Administration, our reliance on third parties and collaboration partners, including our ability to manage growth and enter into new client relationships, our dependency on the rare disease industry, our ability to manage international expansion, our reliance on key personnel, our reliance on intellectual property protection, fluctuations of our operating results due to the effect of exchange rates, or other factors. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. Many of these risks are outside of the Company's control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law.



    For further information, please refer to the Risk Factors section in our Annual Report for the year ended December 31, 2019, on Form 20-F filed with the SEC on April 23, 2020, Form 6-K containing our financial results for the three months ended March 31, 2020, furnished to the SEC on June 15, 2020, Form 6-K containing our financial results for the three and nine months ended September 30, 2020, furnished to the SEC on December 16, 2020, and other current reports and documents furnished to or filed with the U.S. Securities and Exchange Commission (SEC). You may get these documents by visiting EDGAR on the SEC website at www.sec.gov.



    Media Contact:
    
    CENTOGENE
    Ben Legg
    Corporate Communications
    press@centogene.com
    
    FTI Consulting
    Bridie Lawlor O'Boyle
    +1.917.929.5684
    bridie.lawlor@fticonsulting.com

    Primary Logo

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  25. Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that it will host a conference call on April 6, 2021 to provide updates on select New Molecular Entities (NMEs) in its Wave 1 pipeline portfolio. With several NME regulatory filings expected by year-end FY2021, the company will outline plans for organic and sustainable revenue growth over the next several years.

    Date
    April 6, 2021

    Time
    8:00 a.m. – 10:30 a.m. ET / 9:00 p.m. – 11:30 p.m. JT

    Agenda
    The agenda for Takeda's call is as follows:

    TIME (ET)

    TIME (JT)

    Session/Speaker

    8:00 a.m. – 8:05 a.m.

    9:00 p.m. – 9:05 p.m.

    Introduction
    Christophe Weber, president and CEO

    8:05 a.m. – 8:10 a.m.

    9:05 p.m. – 9:10 p.m.

    Delivering an Innovative Pipeline…

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that it will host a conference call on April 6, 2021 to provide updates on select New Molecular Entities (NMEs) in its Wave 1 pipeline portfolio. With several NME regulatory filings expected by year-end FY2021, the company will outline plans for organic and sustainable revenue growth over the next several years.

    Date

    April 6, 2021

    Time

    8:00 a.m. – 10:30 a.m. ET / 9:00 p.m. – 11:30 p.m. JT

    Agenda

    The agenda for Takeda's call is as follows:

    TIME (ET)

    TIME (JT)

    Session/Speaker

    8:00 a.m. – 8:05 a.m.

    9:00 p.m. – 9:05 p.m.

    Introduction

    Christophe Weber, president and CEO

    8:05 a.m. – 8:10 a.m.

    9:05 p.m. – 9:10 p.m.

    Delivering an Innovative Pipeline to Our Patients: Spotlight on Select Wave 1 Programs

    Andy Plump, president of Research and Development

    8:10 a.m. – 8:35 a.m.

    9:10 p.m. – 9:35 p.m.

    Maribavir (TAK-620): Potential Game Changer in the Treatment for Post-Transplant Cytomegalovirus (CMV) Infection

    Obi Umeh, global program lead, Rare Genetic and Hematology Therapeutic Area Unit

    Claus Jepsen, head of Global Product and Launch Strategy, Rare Genetic and Hematology Therapeutic Area Unit 

    8:35 a.m. – 8:40a.m.

    9:35 p.m. – 9:40p.m.

    Break

    8:40 a.m. – 9:00 a.m.

    9:40 p.m. – 10:00 p.m.

    Soticlestat (TAK-935): Novel MoA for Treatment of Dravet Syndrome and Lennox-Gastaut Syndrome

    Sarah Sheikh, head of Neuroscience Therapeutic Area Unit 

    Erika Gill, head of Global Product and Launch Strategy, Neuroscience Therapeutic Area 

    9:00 a.m. – 9:35 a.m.

    10:00 p.m. – 10:35 p.m.

    Orexin Franchise Strategy Update: First Potential Medicine to Treat the Underlying Disease in Patients with Narcolepsy Type 1

    Elena Koundourakis, head of Orexin Franchise Development, Neuroscience Therapeutic Area 

    Erika Gill, head of Global Product and Launch Strategy, Neuroscience Therapeutic Area 

    9:35 a.m. – 9:40 a.m.

    10:35 p.m. – 10:40 p.m.

    Delivering an Innovative Pipeline to Our Patients: Spotlight on Select Wave 1 Programs

    Ramona Sequeira, president of U.S. Business Unit & Global Portfolio Commercialization 

    9:40 a.m. – 10:30 a.m.

    10:40 p.m. – 11:30 p.m.

    Panel Q&A Session

    To access the live webcast, including presentation slides, visit our web site at www.takeda.com/investors/ir-events. An archived copy of the presentation slides will be available following the conclusion of the event.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

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  26. Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the completion of its previously-announced sale of a portfolio of select products to Orifarm Group ("Orifarm") for a total value of up to $670 million USD. The portfolio includes approximately 130 over-the-counter (OTC) and prescription pharmaceutical products sold in Europe, and two manufacturing sites located in Denmark and Poland. This divestment agreement was first announced in April 2020.

    The divested portfolio includes OTC products and food supplements, plus select products within the Cardiovascular, Anti-inflammatory, Respiratory, and Endocrinology therapeutic areas, primarily sold in Denmark, Norway, Belgium, Poland, Finland, Sweden, the Baltics and Austria. The…

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced the completion of its previously-announced sale of a portfolio of select products to Orifarm Group ("Orifarm") for a total value of up to $670 million USD. The portfolio includes approximately 130 over-the-counter (OTC) and prescription pharmaceutical products sold in Europe, and two manufacturing sites located in Denmark and Poland. This divestment agreement was first announced in April 2020.

    The divested portfolio includes OTC products and food supplements, plus select products within the Cardiovascular, Anti-inflammatory, Respiratory, and Endocrinology therapeutic areas, primarily sold in Denmark, Norway, Belgium, Poland, Finland, Sweden, the Baltics and Austria. The portfolio generated FY2020 net sales of approximately $240 million USD, driven by sales of cough/cold and vitamin OTC brands, along with prescription products such as Warfarub and Levaxin, and other strong regional brands. The products, while addressing key patient needs in these countries, are outside of the business areas Takeda has chosen as core to its global long-term growth.

    As previously announced, Takeda and Orifarm have also entered into manufacturing and supply agreements under which Takeda will continue to manufacture select products on behalf of Orifarm. In addition, approximately 600 employees from the manufacturing sites, sales and marketing professionals and others supporting the divested portfolio and manufacturing sites have transitioned to Orifarm.

    Takeda remains focused on executing its long-term growth strategy to optimize our business mix around our key business areas, and simplifying our operations to better serve patients by delivering innovative treatments in these areas.

    The Company intends to use the proceeds from the sale to reduce its debt and accelerate deleveraging towards its target of 2x net debt/adjusted EBITDA within Fiscal Years 2021–2023.

    Takeda has exceeded its $10 billion non-core asset divestiture target and has announced 12 deals since January 2019 to date for a total aggregate value of up to approximately $12.9 billion.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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.

    For more information, visit https://www.takeda.com.

    Important Notice

    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; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; 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/reports/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.

    View Full Article Hide Full Article
  27. Takeda Pharmaceutical Company Limited (TYO:4502) (NYSE:TAK) ("Takeda") today announced the completion of its previously-announced sale of Takeda Consumer Healthcare Company Limited ("TCHC") to Oscar A-Co KK, a company controlled by funds managed by The Blackstone Group Inc. and its affiliates (collectively "Blackstone") for a total value of JPY 242.0 billion1. This divestment agreement was first announced in August 2020.

    Following the transfer of shares, TCHC will be excluded from the scope of consolidation of Takeda2, and operates as Alinamin Pharmaceutical Co., Ltd ("Alinamin Pharmaceuticals").

    The divested portfolio included a variety of over-the-counter ("OTC") medicines and health products that generated total revenues of over JPY 60.0…

    Takeda Pharmaceutical Company Limited (TYO:4502) (NYSE:TAK) ("Takeda") today announced the completion of its previously-announced sale of Takeda Consumer Healthcare Company Limited ("TCHC") to Oscar A-Co KK, a company controlled by funds managed by The Blackstone Group Inc. and its affiliates (collectively "Blackstone") for a total value of JPY 242.0 billion1. This divestment agreement was first announced in August 2020.

    Following the transfer of shares, TCHC will be excluded from the scope of consolidation of Takeda2, and operates as Alinamin Pharmaceutical Co., Ltd ("Alinamin Pharmaceuticals").

    The divested portfolio included a variety of over-the-counter ("OTC") medicines and health products that generated total revenues of over JPY 60.0 billion in fiscal year 2019. TCHC's strong regional brands included Alinamin, its top selling product and Japan's first vitamin B1 preparation, and Benza, a cold remedy. Takeda is confident that under Blackstone, Alinamin Pharmaceuticals will be well-positioned to continue growing and developing its product offerings in the years to come to address the evolving needs of consumers.

    Takeda intends to use the proceeds from the sale to reduce its debt and accelerate deleveraging towards its target of 2x net debt/adjusted EBITDA within FY2021 – FY2023.

    Takeda has sustained momentum in its divestiture strategy and has exceeded its $10 billion non-core asset divestiture target. Takeda has announced 12 deals since January 2019, for a total aggregate value of up to approximately $12.9 billion.

    The sales price is currently anticipated to be approximately JPY 230.0 billion, subject to certain adjustments, including net debt and working capital of TCHC and Takeda Healthcare Products Company Limited as of March 31, 2021. With the completion of the transfer of shares, a pre-tax gain of approximately JPY 140.0 billion on the sale of shares of a subsidiary will be recognized, and Takeda anticipates Reported Net Profit attributable to owners of the Company to increase by approximately the same amount in the fiscal year ending March 31, 2021 (FY2020). Since the gain on the sale of shares of the subsidiary relates to the divestiture of a non-core business, there will be no impact on Core Operating Profit or Core Net Profit.

    1 Enterprise value. Actual sales price will be determined after adjustment for items including net debt and working capital of TCHC and Takeda Healthcare Products Company Limited ("THP").

    2 As a result of the share transfer, a wholly owned subsidiary of TCHC, THP, will also be excluded from the scope of consolidation of Takeda.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited (TYO: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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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.

    View Full Article Hide Full Article
  28. European Medicines Agency to Conduct First-Ever Parallel Assessment of a Medicinal Product, Takeda's Dengue Vaccine Candidate (TAK-003), for use in the EU; Countries Outside of the EU through the EU-M4all (Previously Article 58) Procedure

    Takeda Intends to Submit Regulatory Filings in Argentina, Brazil, Colombia, Indonesia, Malaysia, Mexico, Singapore, Sri Lanka and Thailand During 2021

    TAK-003 is Being Studied for the Prevention of Dengue Due to any Dengue Virus Serotype in Individuals Ages Four to 60

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the European Medicines Agency (EMA) has accepted the Company's filing packages for its dengue vaccine candidate (TAK-003) which is being investigated for the…

    European Medicines Agency to Conduct First-Ever Parallel Assessment of a Medicinal Product, Takeda's Dengue Vaccine Candidate (TAK-003), for use in the EU; Countries Outside of the EU through the EU-M4all (Previously Article 58) Procedure

    Takeda Intends to Submit Regulatory Filings in Argentina, Brazil, Colombia, Indonesia, Malaysia, Mexico, Singapore, Sri Lanka and Thailand During 2021

    TAK-003 is Being Studied for the Prevention of Dengue Due to any Dengue Virus Serotype in Individuals Ages Four to 60

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced that the European Medicines Agency (EMA) has accepted the Company's filing packages for its dengue vaccine candidate (TAK-003) which is being investigated for the prevention of dengue due to any dengue virus serotype in individuals ages four to 60. Takeda intends to submit regulatory filings in Argentina, Brazil, Colombia, Indonesia, Malaysia, Mexico, Singapore, Sri Lanka and Thailand during 2021.

    "Submission of regulatory filings for our dengue vaccine candidate, TAK-003, marks an important development for people who are living in or traveling to communities burdened by the threat of dengue," said Derek Wallace, VP, Dengue Global Program Leader at Takeda. "Dengue outbreaks, which result in half a million hospitalizations globally each year, can overwhelm communities and governments because of the broad impact on the health care system. With limited options to prevent the disease, there is a pressing need for widely available dengue vaccines. Takeda is committed to working with regulatory authorities and recommending bodies to support evaluation of our submissions and achieve access for TAK-003."

    Takeda is participating in the EMA's first-ever parallel assessment of a medicinal product for use in the European Union (EU), and through the EU-M4all (previously Article 58) procedure for countries outside of the EU. Along with the scientific opinion issued by the Committee for Medicinal Products for Human Use (CHMP), national regulators in countries participating in the EU-M4all procedure will conduct their own assessments to determine if national marketing authorizations for TAK-003 are granted. Takeda is also seeking approval of TAK-003 in dengue-endemic countries that are not participating in the EU-M4all procedure.

    Regulatory submissions for TAK-003 include long-term safety and efficacy data through 36 months from the ongoing pivotal Phase 3 Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial. Takeda intends to present and publish details of the 36-month data at a scientific meeting and in a peer-reviewed journal this year.

    Takeda also intends to submit regulatory filings in the United States, followed by additional countries in Asia and Latin America.

    EU-M4all1

    EU-M4all (or EU-Medicines for all) is a procedure designed to facilitate patient access to essential medicines or vaccines intended to prevent or treat diseases of major public health interest. Through the EU-M4all procedure (previously known as the Article 58 procedure), the EMA, in cooperation with the World Health Organization (WHO), can provide scientific opinion on medicines and vaccines for public health priority diseases that are intended for markets outside of the EU.

    About TAK-003

    Takeda's tetravalent dengue vaccine candidate (TAK-003) is based on a live-attenuated dengue serotype 2 virus, which provides the genetic "backbone" for all four vaccine viruses.2 Clinical Phase 2 data in children and adolescents showed that TAK-003 induced immune responses against all four dengue serotypes, in both seropositive and seronegative participants, which persisted through 48 months after vaccination, and the vaccine was found to be generally safe and well tolerated.3 The pivotal Phase 3 TIDES trial met its primary endpoint of overall vaccine efficacy (VE) against virologically confirmed dengue (VCD) at 12 months follow-up and all secondary endpoints at 18 months follow-up for which there were a sufficient number of dengue cases, including VE against hospitalized dengue and VE in baseline seropositive and baseline seronegative individuals.4,5 Efficacy varied by serotype. The results demonstrated TAK-003 was generally well tolerated, and there have been no important safety risks observed to date.

    About the Phase 3 TIDES (DEN-301) Trial

    The double-blind, randomized, placebo-controlled Phase 3 TIDES trial is evaluating the safety and efficacy of two doses of TAK-003 in the prevention of laboratory-confirmed symptomatic dengue fever of any severity and due to any of the four dengue virus serotypes in children and adolescents.4 The TIDES trial is Takeda's largest interventional clinical trial to date and enrolled over 20,000 healthy children and adolescents ages four to 16 years living in dengue-endemic areas. Study participants were randomly assigned to receive either TAK-003 0.5 mL or placebo by subcutaneous injection on Day 1 and Day 90.4 The study is comprised of five parts. Part 1 and the primary endpoint analysis evaluated vaccine efficacy (VE) and safety through 15 months after the first dose (12 months after the second dose).4 Part 2 continued for an additional six months to complete the assessment of the secondary endpoints of VE by serotype, baseline serostatus and disease severity, including VE against hospitalized dengue.4 Part 3 is evaluating VE and long-term safety by following participants for an additional two and a half to three years.6 Part 4 will evaluate safety for 13 months following booster vaccination and Part 5 will evaluate long-term safety for one year after completion of Part 4.6

    The trial is taking place at sites in dengue-endemic areas in Latin America (Brazil, Colombia, Panama, the Dominican Republic and Nicaragua) and Asia (Philippines, Thailand and Sri Lanka) where there are unmet needs in dengue prevention and where severe dengue is a leading cause of serious illness and death among children.4 Baseline blood samples were collected from all individuals participating in the trial to allow for evaluation of safety and efficacy based on serostatus. Takeda and an independent Data Monitoring Committee of experts are actively monitoring safety on an ongoing basis.

    About Dengue

    Dengue is the fastest spreading mosquito-borne viral disease and was one of the WHO's top 10 threats to global health in 2019.7,8 Dengue is mainly spread by Aedes aegypti mosquitoes and, to a lesser extent, Aedes albopictus mosquitoes. It is caused by any of four dengue virus serotypes, each of which can cause dengue fever or severe dengue. The prevalence of individual serotypes varies across different geographies, countries, regions, seasons and over time.9 Recovery from infection by one serotype provides lifelong immunity against only that serotype, and later exposure to any of the remaining serotypes is associated with an increased risk of severe disease.

    Dengue is pandemic prone, and outbreaks are observed in tropical and sub-tropical areas and have recently caused outbreaks in parts of the continental United States and Europe.10,11 Approximately half of the world now lives under the threat of dengue, which is estimated to cause 390 million infections and around 20,000 deaths globally each year.10,12 The dengue virus can infect people of all ages and is a leading cause of serious illness among children in some countries in Latin America and Asia.10

    Takeda's Commitment to Vaccines

    Vaccines prevent 2 to 3 million deaths each year and have transformed global public health.13 For the past 70 years, Takeda has supplied vaccines to protect the health of people in Japan. Today, Takeda's global vaccine business is applying innovation to tackle some of the world's most challenging infectious diseases, such as dengue, COVID-19, Zika and norovirus. Takeda's team brings an outstanding track record and a wealth of knowledge in vaccine development, manufacturing and global access to advance a pipeline of vaccines to address some of the world's most pressing public health needs. For more information, visit www.TakedaVaccines.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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/ reports/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.

    Medical information

    This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

    ###


    1The European Medicines Agency. Medicines for use outside the EU — EU-M4all. July 2020. Retrieved March 2021.

    2 Huang CY-H, et al. Genetic and phenotypic characterization of manufacturing seeds for tetravalent dengue vaccine (DENVax). PLoS Negl Trop Dis. 2013;7:e2243.

    3Tricou, V, Sáez-Llorens X, et al. Safety and immunogenicity of a tetravalent dengue vaccine in children aged 2-17 years: a randomised, placebo-controlled, phase 2 trial. Lancet. 2020. doi:10.1016/S0140-6736(20)30556-0.

    4 Biswal S, et al. Efficacy of a tetravalent dengue vaccine in healthy children and adolescents. N Engl J Med. 2019; 2019;381:2009-2019.

    5 Biswal S, et al. Efficacy of a tetravalent dengue vaccine in healthy children aged 4-16 years: a randomized, placebo controlled, phase 3 trial. Lancet. 2020. 2020;395:1423-1433.

    6 ClinicalTrials.Gov. Efficacy, Safety and Immunogenicity of Takeda's Tetravalent Dengue Vaccine (TDV) in Healthy Children (TIDES). Retrieved March 2021.

    7 World Health Organization. Factsheet. Dengue and Severe Dengue. April 2019. Retrieved February 2021.

    8 World Health Organization. Ten threats to global health in 2019. 2019. Retrieved February 2021.

    9 Guzman MG, et al. Dengue: a continuing global threat. Nature Reviews Microbiology. 2010;8:S7-S16.

    10 Knowlton K, et al. Mosquito-Borne Dengue Fever Threat Spreading in the Americas. The Natural Resources Defense Council (NRDC). 2009. Retrieved February 2021.

    11 Chan E, et al. Using web search query data to monitor dengue epidemics: a new model for neglected tropical disease surveillance. PLoS Negl Trop Dis. 2011;5:e1206.

    12 Centers for Disease Control and Prevention. About Dengue: What You Need to Know. May 2019. Retrieved February 2021.

    13 UNICEF. Vaccination and Immunization Statistics. 2019. Retrieved February 2021.

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    • EVOTEC LEVERAGES ITS PROPRIETARY SMALL MOLECULE RNA TARGETING PLATFORM AGAINST MULTIPLE RNA TARGETS ACROSS TAKEDA'S KEY INDICATIONS
    • EVOTEC RECEIVES RESEARCH FUNDING AND IS ELIGIBLE FOR SUCCESS-BASED MILESTONES AND TIERED ROYALTIES

    HAMBURG, GERMANY / ACCESSWIRE / March 22, 2021 / Evotec SE ((Frankfurt Stock Exchange: EVT, MDAX/TecDAX, OTC:EVOTF) today announced that the Company has entered into a multi-RNA target alliance with Takeda Pharmaceutical Company Limited ("Takeda") with the goal to discover and develop RNA targeting small molecule therapeutics for highly attractive targets that are difficult to address via more conventional approaches.

    Evotec and Takeda will jointly identify and develop small molecules targeting a range of RNA targets…

    • EVOTEC LEVERAGES ITS PROPRIETARY SMALL MOLECULE RNA TARGETING PLATFORM AGAINST MULTIPLE RNA TARGETS ACROSS TAKEDA'S KEY INDICATIONS
    • EVOTEC RECEIVES RESEARCH FUNDING AND IS ELIGIBLE FOR SUCCESS-BASED MILESTONES AND TIERED ROYALTIES

    HAMBURG, GERMANY / ACCESSWIRE / March 22, 2021 / Evotec SE ((Frankfurt Stock Exchange: EVT, MDAX/TecDAX, OTC:EVOTF) today announced that the Company has entered into a multi-RNA target alliance with Takeda Pharmaceutical Company Limited ("Takeda") with the goal to discover and develop RNA targeting small molecule therapeutics for highly attractive targets that are difficult to address via more conventional approaches.

    Evotec and Takeda will jointly identify and develop small molecules targeting a range of RNA targets aligned with Takeda's research and development areas. The collaboration will leverage Evotec's extensive RNA targeting platform to optimally identify promising RNA sequences to target with small molecule ligands that can be developed into potentially first-in-class therapeutics.

    Under the terms of the agreement, Evotec will receive significant research funding and will be eligible to receive discovery, pre-clinical, clinical, commercial and sales milestone payments of up to US$ 160 m per programme. Additionally, Evotec is entitled to tiered royalties on net sales of any products resulting from the collaboration.

    Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "Many highly validated targets have proven to be intractable via conventional protein targeting approaches. For this reason, Evotec has been pioneering RNA targeting strategies and approaches for quite some time. We are very excited about the opportunity to collaborate with Takeda in this field as both companies share the vision to jointly develop small molecule therapeutics against high value RNA targets that will deliver long awaited therapeutics."

    "Takeda recognizes targeting RNA with small molecules as a promising new modality that has tremendous potential for much needed medicines for patients through modulating historically undruggable targets", said Dr Larry Hamann, Head, Drug Discovery Sciences, Takeda. "We are excited to be working with Evotec and their impressive capabilities."

    About Evotec's RNA platform
    The structure-based recognition of RNA tertiary structures by RNA-targeted small molecules ("rSM") provides an alternative to sequence-based approaches, such as antisense oligonucleotides ("ASOs"). rSM approaches enable novel therapeutic potential by allowing to target highly conserved parts of RNA, creating pathways in cases where the encoded protein cannot be targeted conventionally, and unlocking the largely unexplored field of non-coding RNAs, which can also be disease drivers.

    Evotec's proprietary RNA targeting platform is specifically designed to

    1. identify RNA tertiary structural elements where rSM are able to bind with sufficient selectivity and affinity,
    2. discover and develop suitable rSM binders that potentially deliver orally available drugs, and
    3. identify and deliver proof-of-target engagement for disease-relevant RNA structures, allowing biologically active rSM binders.

    Evotec's cutting-edge RNA small molecule platform builds on the well-established drug discovery routes within Evotec and combines them with novel, highly innovative technologies such as third generation sequencing, sequencing-based structure elucidation of RNA molecules, which constitute a first-class expertise in this area.

    ABOUT TAKEDA PHARMACEUTICAL COMPANY LIMITED
    Takeda Pharmaceutical Company Limited (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 Genetic 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. For more information, visit www.takeda.com.

    ABOUT EVOTEC SE
    Evotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,500 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and women's health. On this basis, Evotec has built a broad and deep pipeline of more than 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to www.evotec.com and follow us on Twitter @Evotec.

    FORWARD-LOOKING STATEMENTS
    Information set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

    Media Contact Evotec SE:
    Gabriele Hansen, SVP Head of Global Corporate Communications & Marketing, Phone: +49.(0)40.56081-255, gabriele.hansen@evotec.com

    IR Contact Evotec SE:
    Volker Braun, SVP Head of Global Investor Relations & ESG, Phone: +49.(0)40.56081-775, volker.braun@evotec.com

    SOURCE: Evotec AG



    View source version on accesswire.com:
    https://www.accesswire.com/636729/Evotec-and-Takeda-Enter-Strategic-RNA-Targeting-Drug-Discovery-and-Development-Alliance

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  29. First-line indication offers Canadians with advanced lung cancer new treatment options

    TORONTO, March 17, 2021 /CNW/ - Takeda Canada Inc. is pleased to announce that Health Canada has issued ALUNBRIG® (brigatinib tablets) marketing authorization without conditions as monotherapy for the first-line treatment of adult patients with anaplastic lymphoma kinase positive (ALK+) locally advanced (not amenable to curative therapy) or metastatic non-small cell lung cancer (NSCLC).1 The approval is based on results from the phase 3 ALTA-1L trial which evaluated 275 patients and showed that once-daily ALUNBRIG® was superior to crizotinib on measures of efficacy and tolerability making it a promising first-line treatment option.2

    First-line indication offers Canadians with advanced lung cancer new treatment options

    TORONTO, March 17, 2021 /CNW/ - Takeda Canada Inc. is pleased to announce that Health Canada has issued ALUNBRIG® (brigatinib tablets) marketing authorization without conditions as monotherapy for the first-line treatment of adult patients with anaplastic lymphoma kinase positive (ALK+) locally advanced (not amenable to curative therapy) or metastatic non-small cell lung cancer (NSCLC).1 The approval is based on results from the phase 3 ALTA-1L trial which evaluated 275 patients and showed that once-daily ALUNBRIG® was superior to crizotinib on measures of efficacy and tolerability making it a promising first-line treatment option.2

    "This is important news for Canadians living with ALK+ locally advanced or metastatic non-small cell lung cancer. New treatment options are essential to support patients on their journey. Having access to clinically evaluated therapies provides choices to stay ahead of cancer," said Dr. Geoffrey Liu, Senior Scientist, Princess Margaret Cancer Care Centre, University Health Network. "This new indication will be crucial to improving the health of Canadians impacted by ALK+ locally advanced or metastatic NSCLC. I look forward to sharing this positive announcement with patients."

    Non-small cell lung cancer is the most common form of lung cancer, accounting for approximately 80 to 85 per cent of lung cancers diagnosed in Canada each year.There are many different subtypes of NSCLC that start in the different types of cells and tissues of the lung. ALK+ is a subset of NSCLC, often striking people at a younger age, many of whom have never smoked.4 Studies indicate that chromosomal rearrangements in the ALK gene is a key driver in this subset of NSCLC patients. Between three to five per cent of patients diagnosed with locally advanced or metastatic NSCLC test positive for the ALK gene rearrangement.5

    "The approval of ALUNBRIG® is a significant step forward for Canadians battling advanced forms of lung cancer, who now have a new treatment option available in their fight against a devastating disease," says David Fyshe, Oncology Country Head, Takeda Canada. "We are proud of ALUNBRIG® clinical results demonstrating first-line treatment benefits. This new oncology indication directly supports Takeda's on-going commitments to uncover solutions for people living with cancer."  

    In 2018, ALUNBRIG® (brigatinib) was issued marketing authorization with conditions, pending the results of trials, as a monotherapy for the treatment of adult patients with ALK+ metastatic NSCLC who have progressed on or who were intolerant to an ALK inhibitor (crizotinib).

    About the Phase 3 ALTA 1L Trial

    The safety and efficacy of brigatinib was evaluated in a, Phase 3 open-label, multicenter trial (ALTA 1L) in 275 adult patients with advanced ALK+ NSCLC who had not previously received an ALK-targeted therapy with a documented ALK rearrangement based on a validated ALK testing. Patients could have up to 1 prior regimen of systemic anticancer therapy in the locally advanced or metastatic setting and had an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2. The major efficacy outcome measure was progression-free survival as evaluated by a Blinded Independent Review Committee (BIRC).

    About ALUNBRIG® (brigatinib)

    ALUNBRIG® is a targeted medicine that blocks the action of the altered ALK gene to help shrink or slow cancer growth. ALUNBRIG® is administered orally, beginning with seven days of 90 mg once daily, followed by 180 mg once daily thereafter. ALUNBRIG® is now indicated as a monotherapy for the first-line treatment of adult patients with ALK+ locally advanced (not amenable to curative therapy) or metastatic NSCLC and as a monotherapy for the treatment of adult patients with ALK+ metastatic NSCLC who have progressed on or who were intolerant to an ALK inhibitor (crizotinib).1

    ALUNBRIG® and the ALUNBRIG Logo® are registered trademarks of ARIAD Pharmaceuticals, Inc.

    About Takeda Canada Inc.

    Takeda Canada Inc. is the Canadian marketing and sales organization of Takeda Pharmaceutical Company Limited, headquartered in Japan. Takeda Pharmaceutical Company Limited ((TAK) is a global, values-based, R&D-driven biopharmaceutical leader committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Rare Diseases and Neuroscience. 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. 

    Additional information about Takeda Canada is available at www.takeda.com/en-ca

    References

    1 ALUNBRIG® Product Monograph. Date of Revision March 2, 2021. Available at https://www.takeda.com/4a4cc3/siteassets/en-ca/home/what-we-do/our-medicines/product-monographs/alunbrig/alunbrig-pm-en.pdf. Accessed March 2021.

    2 Camidge DR, Kim HR, Ahn MJ, Yang JCH, Han JY, Hochmair MJ, Lee KH, Delmonte A, García Campelo MR, Kim DW, Griesinger F, Felip E, Califano R, Spira A, Gettinger SN, Tiseo M, Lin HM, Gupta N, Hanley MJ, Ni Q, Zhang P, Popat S. Brigatinib Versus Crizotinib in Advanced ALK Inhibitor-Naive ALK-Positive Non-Small Cell Lung Cancer: Second Interim Analysis of the Phase III ALTA-1L Trial. J Clin Oncol. 2020 Nov 1;38(31):3592-3603. doi: 10.1200/JCO.20.00505. Epub 2020 Aug 11. PMID: 32780660; PMCID: PMC7605398. Accessed March 2021.

    3 Canadian Cancer Society. Malignant tumours of the lung. Available at http://www.cancer.ca/en/cancer-information/cancer-type/lung/lung-cancer/cancerous-tumours/?region=on. Accessed February 2021.

    4 Gridelli, et al. ALK inhibitors in the treatment of advanced NSCLC. Cancer Treat Rev 2014;40(2): 300–306.

    5 Gainor JF, Varghese AM, Ou SH, et al. ALK rearrangements are mutually exclusive with mutations in EGFR or KRAS: an analysis of 1,683 patients with non-small cell lung cancer. Clin Cancer Res. 2013; 19(15):4273-81.

    SOURCE Takeda Canada Inc.

    Cision View original content to download multimedia: http://www.newswire.ca/en/releases/archive/March2021/17/c7635.html

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  30. More Than Three Times as Many Transplant Recipients With Confirmed Resistant Cytomegalovirus (CMV) Infection at Baseline Receiving the Investigational Drug Maribavir Achieved CMV Viremia Clearance Compared to Conventional Antiviral Therapies, Building on Previously Presented Results Supporting the Efficacy of Maribavir

    Transplant Recipients Receiving Maribavir Experienced Lower Rates of Treatment-Related Neutropenia and Acute Kidney Injury Compared to Conventional Antiviral Therapies

    Takeda Continues to Investigate Maribavir for the First-Line Treatment of CMV in Hematopoietic Cell Transplant Recipients in an Ongoing Phase 3 Clinical Trial

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today during the Presidential Symposium…

    More Than Three Times as Many Transplant Recipients With Confirmed Resistant Cytomegalovirus (CMV) Infection at Baseline Receiving the Investigational Drug Maribavir Achieved CMV Viremia Clearance Compared to Conventional Antiviral Therapies, Building on Previously Presented Results Supporting the Efficacy of Maribavir

    Transplant Recipients Receiving Maribavir Experienced Lower Rates of Treatment-Related Neutropenia and Acute Kidney Injury Compared to Conventional Antiviral Therapies

    Takeda Continues to Investigate Maribavir for the First-Line Treatment of CMV in Hematopoietic Cell Transplant Recipients in an Ongoing Phase 3 Clinical Trial

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today during the Presidential Symposium at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) announced the results from a subgroup analysis of the Phase 3 TAK-620-303 (SOLSTICE) trial, for the investigational drug TAK-620 (maribavir), which supported the efficacy results from the overall randomized population. More than three times as many (62.8%; 76/121) transplant recipients with confirmed genotypic resistant CMV infection at baseline treated with maribavir achieved confirmed CMV viremia clearance at Study Week 8 (end of treatment phase) compared to those treated with conventional antiviral therapies (20.3%, 14/69) (investigator assigned treatment; IAT consists of one or a combination of ganciclovir, valganciclovir, foscarnet or cidofovir) (adjusted difference [95% CI]: 44.1% [31.3, 56.9]).1

    Findings from the overall trial population showed the study met its primary endpoint, demonstrating that maribavir was superior to conventional antiviral therapies in CMV viremia clearance at Study Week 8. Specifically, 55.7% (131/235) of transplant recipients with refractory, with or without resistance (R/R), CMV infection/disease treated with maribavir achieved confirmed CMV viremia clearance as compared to 23.9% (28/117) of those on conventional antiviral therapies (adjusted difference [95% CI]: 32.8%, [22.8, 42.7]; p<0.001).1*†‡

    "Transplant recipients with CMV infections resistant to conventional antiviral therapies are some of the hardest to treat. Current treatment options are limited, and hematologist-oncologists have to engage in a careful balance of viral clearance and side effect management," said Dr. Rafael Duarte, Hospital Universitario Puerta de Hierro, Madrid. "We believe these data are important as they build on previously presented results supporting the potential of maribavir, which, if approved, could transform the management of CMV in these patients."

    Transplant recipients receiving maribavir exhibited lower incidence of treatment-related toxicities common with conventional antiviral therapies. Those receiving maribavir experienced lower rates of treatment-related neutropenia vs. valganciclovir/ganciclovir (1.7% [4/234] vs. 25% [14/56]) and acute kidney injury vs. foscarnet (1.7% [4/234] vs. 19.1% [9/47]). Incidence of any treatment-emergent adverse events (TEAEs) was 97.4% (228/234) for maribavir and 91.4% (106/116) for the conventional therapy group.1 The most common TEAEs in the maribavir group were dysgeusia (35.9%, 84/234), nausea (8.5%, 20/234) and vomiting (7.7%, 18/234).2 Incidence of TEAEs leading to study drug discontinuation was 13.2% (31/234) in the maribavir group and 31.9% (37/116) in the conventional therapy group.1 Two treatment-related serious TEAEs led to death (1 patient per treatment group).1

    "Current CMV management is associated with difficult tradeoffs, including management of toxicities and viremia clearance," said Obi Umeh, MD, Vice President and Maribavir Global Program Leader, Takeda. "As we continue our research of maribavir, an oral antiviral compound, across patient populations, we are committed to addressing this unmet need so physicians potentially have an additional treatment option."

    About CMV

    CMV is a beta herpesvirus that commonly infects humans; serologic evidence of prior infection can be found in 40%-100% of various adult populations.3 CMV typically resides latent and asymptomatic in the body but may reactivate during periods of immunosuppression. Serious disease may occur in individuals with compromised immune systems, which includes patients who receive immunosuppressants associated with various types of transplants including hematopoietic cell transplant (HCT) or solid organ transplant (SOT).4,5 Out of the estimated 200,000 adult transplants per year, CMV is one of the most common viral infections experienced by transplant recipients, with an estimated incidence rate between 16-56% in SOT recipients and 30-70% in HCT recipients.5–10

    In transplant recipients, reactivation of CMV can lead to serious consequences including loss of the transplanted organ and, in extreme cases, can be fatal.11,12 Existing therapies to treat posttransplant CMV infections may demonstrate toxicities that require dose adjustments or may fail to adequately suppress viral replication.13–15 Additionally, existing therapies may require or prolong hospitalization due to administration.13-14

    About Maribavir

    Maribavir, an orally bioavailable anti-CMV compound, is the only antiviral agent presently in Phase 3 development for the treatment of post-transplant patients with CMV in SOT or HCT. Maribavir is an investigational treatment that has not been approved for use by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) or any other regulatory authorities. Maribavir is the only CMV antiviral drug that targets and inhibits the UL97 protein kinase and its natural substrates.16-19

    Maribavir has been granted Orphan Drug Designation by the European Commission as a treatment of CMV disease in patients with impaired cell mediated immunity and by the FDA for treatment of clinically significant CMV viremia and disease in at-risk patients. Orphan status is granted to certain investigational medicines intended for the treatment or prevention of a rare, life-threatening disease. The FDA has also granted maribavir Breakthrough Therapy Designation as a treatment for CMV infection and disease in transplant patients resistant or refractory to prior therapy. Breakthrough Therapy Designation expedites the development and review of investigational treatments for serious conditions with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy. These designations do not guarantee that the EMA or FDA will approve maribavir for the treatment of CMV infections in transplant patients, and the timing of any such approval is uncertain.

    About Takeda's SOLSTICE Trial

    The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing treatment with either maribavir or investigator assigned treatment, IAT, (conventional antiviral therapy) in hematopoietic cell transplant and solid organ transplant recipients with CMV infection refractory, with or without resistance, to one or a combination of the conventional antiviral therapies: ganciclovir, valganciclovir, foscarnet or cidofovir. Patients underwent a 2-week screening period, followed by randomization 2:1 to maribavir (n=235) (400 mg) or IAT (n=117) for an 8-week treatment period, plus 12 weeks of follow-up.

    The trial's primary endpoint was defined as the proportion of patients who achieved confirmed CMV viremia clearance (plasma CMV DNA <137 IU/mL in two consecutive tests ≥5 days apart at central laboratory) compared to IAT at the end of Study Week 8. The key secondary endpoint was defined as achievement of CMV viremia clearance and symptom control at end of Study Week 8, maintained through Study Week 16.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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/reports/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.

    *The difference in proportion of responders between treatment groups was obtained using Cochran-Mantel-Haenszel (CMH) weighted average across all strata and tested using stratum-adjusted CMH method, with transplant type and baseline plasma CMV DNA concentration as two stratification factors

    Refractory defined as documented failure to achieve >1 log10 decrease in CMV DNA level in whole blood or plasma after a 14 day or longer treatment period with IV ganciclovir/oral valganciclovir, IV foscarnet, or IV cidofovir

    Resistant defined as refractory CMV and documentation of >1 CMV genetic mutations associated with resistance to ganciclovir, valganciclovir, foscarnet, and/or cidofovir

    _________________________

    1. Duarte R. Maribavir Versus Investigator-Assigned Therapy for the Treatment of Transplant Recipients with Refractory/Resistant Cytomegalovirus Infection: Efficacy Data From a Randomized Phase 3 Open-Label Study. In: 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), 2021. Abstract.
    2. Duarte R. Maribavir Versus Investigator-Assigned Therapy for the Treatment of Transplant Recipients with Refractory/Resistant Cytomegalovirus Infection: Efficacy Data From a Randomized Phase 3 Open-Label Study. In: 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), 2021. Oral Presentation.
    3. Krech U. Complement-fixing antibodies against cytomegalovirus in different parts of the world. Bull WHO. 1973;49:103-106.
    4. de la Hoz R. Diagnosis and treatment approaches to CMV infections in adult patients. J Clin Virol. 2002;25:S1-S12.
    5. Azevedo L, Pierrotti L, Abdala E, et al. Cytomegalovirus infection in transplant recipients. Clinics. 2015;70(7):515-523. doi:10.6061/clinics/2015(07)09.
    6. World Health Organization. International Report on Organ Donation and Transplantation Activities- Executive Summary 2018.; 2020. Accessed December 2, 2020. http://www.transplant-observatory.org/wp-content/uploads/2020/10/glorep2018-2.pdf.
    7. World Health Organization. Haematopoietic Stem Cell Transplantation HSCtx. Accessed December 2, 2020. https://www.who.int/transplantation/hsctx/en/.
    8. Razonable RR, Eid AJ. A Viral infections in transplant recipients. Minerva Med. 2009;100(6):23.
    9. Styczynski J. Who Is the Patient at Risk of CMV Recurrence: A Review of the Current Scientific Evidence with a Focus on Hematopoietic Cell Transplantation. Infect Ther. 2018;7:1-16.
    10. Cho S-Y, Lee D-G, Kim H-J. Cytomegalovirus Infections after Hematopoietic Stem Cell Transplantation: Current Status and Future Immunotherapy. Int J Mol Sci. 2019;20(2666):1-17.
    11. Fishman JA. Infection in Organ Transplantation. Am J Transplant. 2017;17:856-879.
    12. Kenyon M, Babic A, eds. The European Blood and Marrow Transplantation Textbook for Nurses. Springer International Publishing; 2018. doi:10.1007/978-3-319-50026-3.
    13. Martín-Gandul C, Pérez-Romero P, González-Roncero FM, et al. Clinical impact of neutropenia related with the preemptive therapy of CMV infection in solid organ transplant recipients. J Infect. 2014;69(5):500-506. doi:10.1016/j.jinf.2014.07.001.
    14. Chemaly RF, Chou S, Einsele H, et al. Definitions of Resistant and Refractory Cytomegalovirus Infection and Disease in Transplant Recipients for Use in Clinical Trials. Clin Infect Dis. 2019;68(8):1420-1426. doi:10.1093/cid/ciy696.
    15. Beyer K. Outpatient Foscarnet Administration Incorporating Home Infusions Is Feasible Greatly Enhancing the Care of Hematopoietic Stem Cell Transplant Recipients. Biol Blood Marrow Transpl. 2017;23:S18-S391.
    16. Abdel-Magid AF. New Inhibitors of Cytomegalovirus DNA Polymerase. ACS Med Chem Lett. 2013;4(12):1129-1130. doi:10.1021/ml4004099.
    17. Hamirally S, Kamil JP, Ndassa-Colday YM, et al. Viral Mimicry of Cdc2/Cyclin-Dependent Kinase 1 Mediates Disruption of Nuclear Lamina during Human Cytomegalovirus Nuclear Egress. Nelson JA, ed. PLoS Pathog. 2009;5(1):e1000275. doi:10.1371/journal.ppat.1000275.
    18. Kotton CN, Kumar D, Caliendo AM, et al. The Third International Consensus Guidelines on the Management of Cytomegalovirus in Solid-organ Transplantation: Transplantation. 2018;102(6):900-931. doi:10.1097/TP.0000000000002191.
    19. Krosky PM, Baek M-C, Coen DM. The Human Cytomegalovirus UL97 Protein Kinase, an Antiviral Drug Target, Is Required at the Stage of Nuclear Egress. JVI. 2003;77(2):905-914. doi:10.1128/JVI.77.2.905-914.2003.

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  31. Takeda will make manufacturing capacity available at IDT's facilities in Germany

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced a mutual agreement with IDT Biologika GmbH ("IDT"), a contract development and manufacturing organization, to utilize capacity at IDT previously reserved for Takeda's dengue vaccine candidate (TAK-003) to manufacture the single-shot COVID-19 vaccine developed by the Janssen Pharmaceutical Companies of Johnson & Johnson. At the end of a three-month period, the capacity will be returned to Takeda to resume critical manufacturing for the planned launch of its dengue vaccine, subject to regulatory approvals.

    "We are pleased to work with IDT to support Janssen's efforts to make its COVID-19…

    Takeda will make manufacturing capacity available at IDT's facilities in Germany

    Takeda Pharmaceutical Company Limited ((TAK) ("Takeda") today announced a mutual agreement with IDT Biologika GmbH ("IDT"), a contract development and manufacturing organization, to utilize capacity at IDT previously reserved for Takeda's dengue vaccine candidate (TAK-003) to manufacture the single-shot COVID-19 vaccine developed by the Janssen Pharmaceutical Companies of Johnson & Johnson. At the end of a three-month period, the capacity will be returned to Takeda to resume critical manufacturing for the planned launch of its dengue vaccine, subject to regulatory approvals.

    "We are pleased to work with IDT to support Janssen's efforts to make its COVID-19 vaccine available and accessible to as much of the world as possible," said Rajeev Venkayya, President, Global Vaccine Business Unit at Takeda. "We also recognize the massive unmet need for a dengue vaccine and will work closely with IDT to mitigate the impact on the supply of TAK-003."

    "I am grateful to our longstanding customer Takeda for their flexibility, allowing us to help provide much-needed COVID-19 vaccines to the world," said Jürgen Betzing, IDT Biologika's CEO. "It has become abundantly clear over the past months that the challenges posed by the pandemic can only be solved by cooperation and commitment. I believe this short-term arrangement between three industry organizations demonstrates our sector's willingness and ability to contribute to creatively solving this crisis. With our production for Janssen and AstraZeneca plus the development of our own COVID-19 vaccine in cooperation with the German Center for Vaccine Research, DZIF, our company can make a major contribution to the fight against COVID-19."

    With this agreement, Takeda is now supporting global access to three different COVID-19 vaccines. Takeda previously announced its commitment to providing rapid and sustained access to COVID-19 vaccines in Japan through partnerships with Novavax and Moderna. Takeda will receive a manufacturing technology transfer from Novavax and will be responsible for the development and commercialization based on manufacturing capacity of over 250 million doses. The company will also import and distribute 50 million doses of Moderna's mRNA COVID-19 vaccine as part of a joint partnership with Moderna and the Government of Japan's Ministry of Health Labour and Welfare (MHLW).

    Takeda is well positioned to meet its commitments to support the public health needs associated with both COVID-19 and dengue. Takeda is working with regulatory authorities and recommending bodies, as appropriate, to bring its dengue vaccine candidate to people who can potentially benefit from it and are living in or traveling to communities burdened by the threat of dengue. More than half the world's population is at risk of dengue, with that number expected to increase over the next several decades due to population growth, globalization and urbanization.

    About Takeda's COVID-19 Efforts

    Takeda is taking a comprehensive approach to treat and prevent COVID-19 today, and future pandemics through multiple activities and partnerships including, but not limited to:

    • Hyperimmune globulin: Takeda co-founded the CoVIg-19 Plasma Alliance and joined forces with other leading plasma companies to develop and manufacture a hyperimmune globulin medicine which is currently being evaluated in a global clinical trial. The Alliance is also participating in The Fight Is In Us coalition and related convalescent plasma donation campaign.
    • Additional therapeutics: The company is assessing existing Takeda products for activity against the COVID-19 virus, and co-founded the COVID R&D Alliance. In addition, Takeda has joined the IMI Care Alliance and the Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) partnership.
    • Vaccines: Takeda has partnered with the Government of Japan, Novavax and Moderna, to help accelerate the availability of a COVID-19 vaccine. We are leveraging our extensive and well-established global manufacturing and supply capabilities and building upon our existing influenza pandemic preparedness efforts in Japan. Takeda supports our partners and alliances in a shared goal to rapidly discover, develop and deliver effective treatments and vaccines for COVID-19 and ensure preparedness for future pandemics.

    Takeda's Commitment to Vaccines

    Vaccines prevent 2 to 3 million deaths each year and have transformed global public health. For the past 70 years, Takeda has supplied vaccines to protect the health of people in Japan. Today, Takeda's global vaccine business is applying innovation to tackle some of the world's most challenging infectious diseases, such as dengue, COVID-19, Zika and norovirus. Takeda's team brings an outstanding track record and a wealth of knowledge in vaccine development, manufacturing and global access to advance a pipeline of vaccines to address some of the world's most pressing public health needs. For more information, visit www.TakedaVaccines.com.

    About Takeda Pharmaceutical Company Limited

    Takeda Pharmaceutical Company Limited ((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 Genetic 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. For more information, visit https://www.takeda.com.

    Important Notice

    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

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  32. Lanadelumab is an investigational monoclonal antibody for the preventive treatment of hereditary angioedema (HAE) in patients 12 years and older1

    Takeda is committed to bringing important therapies to patients with high unmet medical needs around the world

    Takeda Pharmaceutical Company Limited (NYSE:TAK) ("Takeda") today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).

    HAE is a rare genetic disorder that results in recurrent attacks of oedema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands…

    Lanadelumab is an investigational monoclonal antibody for the preventive treatment of hereditary angioedema (HAE) in patients 12 years and older1

    Takeda is committed to bringing important therapies to patients with high unmet medical needs around the world

    Takeda Pharmaceutical Company Limited (NYSE:TAK) ("Takeda") today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).

    HAE is a rare genetic disorder that results in recurrent attacks of oedema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.2,3,4 HAE affects an estimated 1 in 50,000 people worldwide.5 In Japan, it is estimated that between 2,000 and 3,000 people are living with HAE, but only approximately 450 have been diagnosed due to low awareness of the disorder in the country.6

    "HAE is unpredictable, debilitating and potentially life-threatening, and recognition of the disease remains low in Japan, meaning there are significant challenges relating to diagnosis and access to effective therapies," said Naoyoshi Hirota, General Manager, Takeda Development Center, Japan. "Lanadelumab is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity, with a proven efficacy and safety profile as a preventive treatment for HAE attacks. Takeda is committed to bringing important therapies such as lanadelumab to patients with high unmet medical needs around the world. Subject to approval, we are looking forward to providing lanadelumab as a new treatment option for patients in Japan living with HAE."

    Lanadelumab, under the tradename TAKHZYRO®, received its first approval for the prevention of HAE attacks in patients 12 years and older in 2018 and is now available in more than 20 countries with additional regulatory submissions ongoing worldwide.7

    The submission of the New Drug Application in Japan is primarily based on results of the global Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ and the Phase 3 HELP Study Open-label Extension (OLE), in addition to interim results of a Phase 3 study evaluating the efficacy and safety of lanadelumab in Japanese subjects.1,8 Combined, these studies have demonstrated the efficacy and safety profile of lanadelumab as a preventive treatment for HAE attacks. If approved, lanadelumab will be available to patients in Japan as a pre-filled syringe presentation.

    In the randomized, double-blind, placebo-controlled HELP study, which included 125 people with HAE, lanadelumab reduced the mean number of monthly HAE attacks by 87% relative to placebo when administered at 300 mg every two weeks and 73% relative to placebo when administered at 300 mg every four weeks (adjusted P<0.001).1 A prespecified, exploratory analysis showed that over the entire 26-week study (days 0-182), 44% (n=12/27) of patients taking lanadelumab 300 mg every two weeks were attack-free vs. 2% (n=1/41) of patients taking placebo.1 A post-hoc sensitivity analysis showed that 77% (n=20/26) of the patients receiving lanadelumab 300 mg every two weeks were attack-free during a steady-state (day 70-182) vs. 3% of patients on placebo (n=1/37).1 The most commonly reported treatment-emergent adverse events (excluding HAE attacks) in patients treated with lanadelumab (n=84) during the entire treatment period were injection site pain (42.9%), viral upper respiratory tract infection (23.8%), headache (20.2%), injection site erythema (9.5%), injection site bruising (7.1%), and dizziness (6.0%).1 Most treatment-emergent adverse events (98.5%) were mild to moderate in severity.1 The HELP Study is the largest randomized, controlled clinical prevention study conducted to date in HAE.

    The regulatory submission in Japan will be evaluated by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Following its review, the PMDA will issue a report to the Ministry of Health, Labour and Welfare (MHLW) of Japan for a final decision.

    Takeda in Hereditary Angioedema

    Hereditary Angioedema (HAE), like so many other rare diseases, is highly complex, and patients, their families and caregivers often undergo years of strain trying to understand their disease, get a definitive diagnosis and gain access to the medicines they need. At Takeda we are a committed champion for the patients we serve. Every individual living with HAE is unique and by listening and reacting to their needs, we translate the insights we gain into innovative solutions – from diagnosis to ongoing management. Advancing the science is crucial to the way we operate and we are unafraid to push at the boundaries of success in our mission to accelerate diagnosis and develop transformative and sustainable treatments that will make a difference to the lives of HAE patients, their support networks and those medical professionals who care for them.

    About Lanadelumab

    Lanadelumab is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity and is indicated for prophylaxis to prevent HAE attacks in patients 12 years and older. Lanadelumab is formulated for subcutaneous administration and has a half-life of approximately two weeks.7 Lanadelumab is intended for self-administration or administration by a caregiver. The patient or caregiver should be trained by a healthcare professional.7

    TAKHZYRO® (lanadelumab) Safety Information for Europe

    Please consult the TAKHZYRO Summary Product Characteristics (SmPC) before prescribing.

    TAKHZYRO treatment should be initiated under the supervision of a physician experienced in the management of patients with hereditary angioedema (HAE). TAKHZYRO may be self-administered or administered by a caregiver only after training on SC injection technique by a healthcare professional.7

    Contraindication

    Hypersensitivity to the active substance or to any of the excipients.7

    Warnings and Precautions

    Traceability: In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.7

    Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, administration of TAKHZYRO must be stopped immediately and appropriate treatment must be initiated.7

    General: TAKHZYRO is not intended for treatment of acute HAE attacks. In case of a breakthrough HAE attack, individualized treatment should be initiated with an approved rescue medication. There are no available clinical data on the use of lanadelumab in HAE patients with normal C1-INH activity.7

    Interference with coagulation test: Lanadelumab can increase activated partial thromboplastin time (aPTT) due to an interaction of lanadelumab with the aPTT assay. The reagents used in the aPTT laboratory test initiate intrinsic coagulation through the activation of plasma kallikrein in the contact system. Inhibition of plasma kallikrein by lanadelumab can increase aPTT in this assay. None of the increases in aPTT in patients treated with TAKHZYRO were associated with abnormal bleeding adverse events. There were no differences in international normalised ratio (INR) between treatment groups.7

    Sodium content: This medicinal product contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially 'sodium-free'.7

    Interactions

    No dedicated drug-drug interaction studies have been conducted. Based on the characteristics of lanadelumab, no pharmacokinetic interactions with co-administered medicinal products is expected.7

    As expected, concomitant use of the rescue medication C1 esterase inhibitor results in an additive effect on lanadelumab-cHMWK response based on the mechanism of action (MOA) of lanadelumab and C1 esterase inhibitor.7

    Immunogenicity

    Treatment with lanadelumab has been associated with development of treatment emergent anti-drug antibodies (ADA) in 11.9% (10/84) of subjects. All antibody titres were low. The ADA response was transient in 20% (2/10) of ADA positive subjects. 2.4% (2/84) of lanadelumab-treated subjects tested positive for neutralizing antibodies.7

    The development of ADA including neutralising antibodies against TAKHZYRO did not appear to adversely affect the pharmacokinetic (PK) and pharmacodynamics (PD) profiles or clinical response.7

    Adverse Reactions

    The most commonly observed adverse reaction (52.4%) associated with TAKHZYRO was injection site reactions (ISR) including injection site pain, injection site erythema and injection site bruising. Of these ISRs, 97% were of mild intensity, 90% resolved within 1 day after onset with a median duration of 6 minutes.7

    Hypersensitivity reaction (mild and moderate pruritus, discomfort and tingling of tongue) was observed (1.2%)

    Very common

    (frequency ≥1/10):

    Injection site reactions*

    Common

    (≥1/100 to <1/10):

    Hypersensitivity**, dizziness, rash maculo-papular, myalgia, alanine aminotransferase increased, aspartate aminotransferase increased.

    *Injection site reactions include: pain, erythema, bruising, discomfort, haematoma, haemorrhage, pruritus, swelling, induration, paraesthesia, reaction, warmth, oedema and rash.

    ** Hypersensitivity includes: pruritus, discomfort and tingling of tongue.

    For full U.S. Prescribing Information, including the approved indication and important safety information, please visit https://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf.