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Takeda Presents Rare Bleeding Disorders Research at Thrombosis & Hemostasis Summit of North America (THSNA) 2020

2020年10月27日 PM11:00
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CAMBRIDGE, Mass. & OSAKA, Japan

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”), today presented four abstracts at the Thrombosis and Hemostasis Summit of North America (THSNA), highlighting Takeda’s commitment to advancing personalized care in people living with bleeding disorders.

These studies may help provide further data to healthcare providers regarding treatment personalization in patients with hemophilia and von Willebrand’s disease (VWD).

“When living with a bleeding disorder that is complex, rare and lifelong, it is critically important to strive for personalized care, to help patients find an appropriate therapy option for them,” states Dr. Jonathan C. Roberts, MD, Associate Medical Director, Associate Research Director at the Bleeding & Clotting Disorders Institute, and Assistant Professor of Pediatrics, University of Illinois College of Medicine at Peoria, Peoria, Ill. “The opportunity for therapeutic individualization with factor products provides hope that bleeding disorder patients can live life with treatment plans that help to address their clinical needs.”

Takeda’s presence at THSNA 2020 includes the following poster presentations, which are intended for scientific discussion only:

Hemophilia A and B

  • A phase 3b, open-label, multicenter, continuation study of rurioctocog alfa pegol prophylaxis in previously treated patients with severe hemophilia A: clinical outcomes in patients with target joints by age group
    • This phase 3b Continuation study (NCT01945593) evaluated the clinical course and outcomes of patients with target joints across different age groups treated with extended half-life PEGylated recombinant FVIII, rurioctocog alfa pegol (TAK-660). 1
  • Implementing GOAL-Hēm, a Goal Attainment Scaling Instrument
    • Goal Attainment Scaling (GAS) is an individualized outcome measure for enabling patients and caregivers to identify and track goals. GOAL-Hēm, a hemophilia-specific goal menu categorized in 3 domains (hemophilia management, complications, and impact on life), was studied in patients to evaluate whether its use influenced time to complete goal-setting vs. traditional opened-ended GAS interviews, and to learn patient and caregiver implementation preferences. 2
  • Efficacy and Safety Results from a Phase 3, Randomized, Multicenter Study of Rurioctocog Alfa Pegol Pharmacokinetic-Guided Prophylaxis Targeting Two Factor VIII Trough Levels in Patients With Severe Hemophilia A (PROPEL Study): A Consideration for Personalized Prophylaxis
    • This phase 3, randomized, multicenter study investigated the clinical impact of pharmacokinetic (PK)-guided prophylaxis with extended half-life recombinant factor VIII (rFVIII) rurioctocog alfa pegol (TAK-660) targeting 1-3% or 8-12% FVIII troughs and the related increased FVIII exposure in previously treated patients with severe hemophilia A. 3

Von Willebrand Disease

  • Population Pharmacokinetics of Recombinant von Willebrand Factor in Patients with von Willebrand Disease or Severe Hemophilia A
    • This study aims to characterize the population pharmacokinetics (PK) of rVWF and predict effects such as VWF activity in patients with VWD or hemophilia A. This population PK model represents the first stage in development of a pharmacokinetic/pharmacodynamic model to describe VWF activity. Development of a population PK/PD model may help support personalized rVWF dosing strategies for patients with this heterogenous disease. 4

ADYNOVATE Professional Important Information10
ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Important Information
Indications and Limitation of Use

ADYNOVATE is a human antihemophilic factor indicated in children and adults with hemophilia A (congenital factor VIII deficiency) for:

  • On-demand treatment and control of bleeding episodes
  • Perioperative management
  • Routine prophylaxis to reduce the frequency of bleeding episodes

ADYNOVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS

Prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).

WARNINGS & PRECAUTIONS

Hypersensitivity Reactions

Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

Neutralizing Antibodies

Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.

ADVERSE REACTIONS

The most common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.

Prescribing Information: https://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf

VONVENDI Professional Important Information11
VONVENDI [von Willebrand factor (recombinant)] Important Information

Indications

VONVENDI [von Willebrand factor (recombinant)] is a recombinant von Willebrand factor (rVWF) indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for:

  • On-demand treatment and control of bleeding episodes
  • Perioperative management of bleeding

Detailed Important Risk Information

CONTRAINDICATIONS

Do not use in patients who have had life-threatening hypersensitivity reactions to VONVENDI or its components (tri-sodium citrate-dihydrate, glycine, mannitol, trehalosedihydrate, polysorbate 80, and hamster or mouse proteins).

WARNINGS AND PRECAUTIONS

Embolism and Thrombosis

Thromboembolic reactions, including disseminated intravascular coagulation, venous thrombosis, pulmonary embolism, myocardial infarction, and stroke, can occur, particularly in patients with known risk factors for thrombosis, including low ADAMTS13 levels. Monitor for early signs and symptoms of thrombosis such as pain, swelling, discoloration, dyspnea, cough, hemoptysis, and syncope, and institute prophylaxis measures against thromboembolism based on current recommendations.

In patients requiring frequent doses of VONVENDI in combination with recombinant factor VIII, monitor plasma levels for FVIII:C activity because sustained excessive factor VIII plasma levels can increase the risk of thromboembolic events.

One out of 80 subjects treated with VONVENDI in clinical trials developed proximal deep vein thrombosis in perioperative period after total hip replacement surgery.

Hypersensitivity Reactions

Hypersensitivity reactions have occurred with VONVENDI. These reactions can include anaphylactic shock, generalized urticaria, angioedema, chest tightness, hypotension, shock, lethargy, nausea, vomiting, paresthesia, pruritus, restlessness, blurred vision, wheezing and/or acute respiratory distress. Discontinue VONVENDI if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Neutralizing Antibodies (Inhibitors)

Inhibitors to VWF and/or factor VIII can occur. If the expected plasma levels of VWF activity (VWF:RCo) are not attained, perform an appropriate assay to determine if anti-VWF or antifactory VIII inhibitors are present. Consider other therapeutic options and direct the patient to a physician with experience in the care of either VWD or hemophilia A.

In patients with high levels of inhibitors to VWF or factor VIII, VONVENDI therapy may not be effective and infusion of this protein may lead to severe hypersensitivity reactions. Since inhibitor antibodies can occur concomitantly with anaphylactic reactions, evaluate patients experiencing an anaphylactic reaction for the presence of inhibitors.

ADVERSE REACTIONS

In clinical trials, the most common adverse reactions observed in ≥2% of subjects (n=80) were generalized pruritus, vomiting, nausea, dizziness, and vertigo. One subject treated with VONVENDI in perioperative setting developed deep vein thrombosis after total hip replacement surgery.

Please see VONVENDI full Prescribing Information: https://www.shirecontent.com/PI/PDFs/VONVENDI_USA_ENG.pdf

About Hemophilia

Hemophilia is a challenging chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.5, 10, 11 Hemophilia A is more common than hemophilia B; hemophilia A affects about 158,225 people, whereas hemophilia B affects about 31,247 people worldwide.6 People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment. Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.5,6

About von Willebrand disease (VWD)

VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population. VWD is caused by a deficiency or dysfunction of von Willebrand factor (VWF), one of several types of proteins in the blood that are needed to facilitate proper blood clotting.7 Due to this defect or deficiency in VWF, blood is not able to clot effectively in people with VWD, which may result in heavy menstrual periods, easy bruising or frequent nose bleeds. Bleeding caused by VWD varies greatly among patients with this disease.8, 11

About Takeda Hematology

Takeda is a leader in hemophilia with the longest heritage and a market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years of experience driving innovation for patients and a broad portfolio of 11 products across multiple bleeding disorders.9 Our experience as leaders in hematology means we are well prepared to meet today’s needs as we pursue future developments in the treatment of bleeding disorders. Together with the hematology community, we are committed to raising expectations for the future, including earlier diagnosis, earlier and better protection against bleeds, and more personalized patient care.

About Takeda Pharmaceutical Company

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.

References

  1. Chowdary et al., (2019). A phase 3b, open-label, multicenter, Continuation study of rurioctocog alfa pegol prophylaxis in previously treated patients with severe hemophilia A: clinical outcomes in patients with target joints by age group. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #147.
  2. Stanley et al., (2019). Implementing GOAL-Hēm, a Goal Attainment Scaling Instrument. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #14.
  3. Radulescu, et al., (2019). Efficacy and Safety Results from a Phase 3, Randomized, Multicenter Study of Rurioctocog Alfa Pegol Pharmacokinetic-Guided Prophylaxis Targeting Two Factor VIII Trough Levels in Patients With Severe Hemophilia A (PROPEL Study): A Consideration for Personalized Prophylaxis. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #15.
  4. Bauer, et al., (2019). Population Pharmacokinetics of Recombinant von Willebrand Factor in Patients with von Willebrand Disease or Severe Hemophilia A. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #125.
  5. World Federation of Hemophilia. “What is hemophilia?” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646. Last Accessed April 2019.
  6. World Federation of Hemophilia. Report on the Annual Global Survey 2017. World Federation of Hemophilia website. http://www1.wfh.org/publications/files/pdf-1714.pdf Last Accessed April 2019.
  7. National Hemophilia Foundation. “Von Willebrand Disease.” National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease. Last accessed January 2020.
  8. National Hemophilia Foundation. “VWD Summit Highlights.” HEMAWARE website. https://hemaware.org/story/vwd-summit-highights . Last accessed January 2020.
  9. Takeda Website. Rare Diseases. Website: https://www.takeda.com/what-we-do/areas-of-focus/rare-diseases/ Last Accessed June 2020.
  10. ADYNOVATE Professional Important Information, Takeda. November 2017.
  11. VONVENDI Professional Important Information, Takeda. January 2019.

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. Forward-looking statements in this document are based on Takeda’s estimates and assumptions only as of the date hereof. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: 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 timing and impact of post-merger integration efforts with acquired companies; and the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s), any of which may cause Takeda’s actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takeda’s results, performance, achievements, or financial position, see “Item 3. Key Information—D. Risk Factors” 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. Future results, performance, achievements or financial position of Takeda could differ materially from those expressed in or implied by the forward-looking statements. Persons receiving this press release should not rely unduly on any forward-looking statements. Takeda undertakes no obligation 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 of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takeda’s future results.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201027005063/en/

CONTACT

Takeda

David Murdoch

david.murdoch@takeda.com

+1 781-421-1741

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