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Takeda to Demonstrate Global Leadership in Hematology at ISTH 2019

48 presentations and sessions to showcase Takeda’s hematology gene therapy pipeline and leading factor portfolio

CAMBRIDGE, Mass. & OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”), the global biotechnology leader in rare diseases, will present research covering a broad range of rare bleeding disorders at the 27th Annual International Society on Thrombosis and Haemostasis Congress (ISTH), July 6-10, 2019 in Melbourne, Australia. Showcased in 10 oral presentations and 38 poster presentations, these data underscore Takeda’s pursuit of treatment innovation to achieve optimized and personalized patient care in hematology.

“At Takeda, we are proud of the hematology heritage Shire, Baxalta and Baxter built over 70 years and we plan on expanding on it through continued research and innovation, in pursuit of a world without bleeds,” said Dr. med. Wolfhard Erdlenbruch, Vice President Head of Global Medical Affairs Hematology, Takeda. “We look forward to presenting new data from PROPEL and important updates from our gene therapy and leading factor pipeline at ISTH 2019, showcasing our continued development and commitment in this area.”

Importance of personalized prophylaxis in haemophilia

At ISTH 2019, Takeda will unveil new data from the Phase IIIb/IV PROPEL study – a PROspective, randomized, multi-center study comparing the safety and efficacy of ADYNOVATE following PK-guided prophylaxis targeting two different Factor Eight (FVIII) trough Levels in subjects with severe hemophilia A.1 This data will build on results presented at the European Association of Haemophilia and Allied Disorders (EAHAD) 2019,2 to provide important insights into the role of personalized prophylaxis for enabling improved patient outcomes and bleed protection, in people living with hemophilia A.1

Advancing the Promise of Gene Therapy in Hemophilia

Takeda will also present 14 scientific updates regarding its gene therapy pipeline for both hemophilia A and B. The goal of gene therapy is to enable a hemophilia patient’s body to naturally produce a sufficient amount of the missing factor VIII or IX to alleviate bleeding episodes.3 Therefore, it may be possible to help convert a hemophilia patient’s bleeding phenotype from severe to mild or even normal in some cases.4 Takeda’s TAK-754 is our AAV FVIII gene therapy vector currently under investigation in a Phase I/II trial.

Notably, Takeda will showcase the following gene therapy data, in oral presentations:

Takeda will also present 33 other scientific data releases on the company’s recently acquired broad portfolio of treatments for bleeding disorders, in oral and poster presentations throughout ISTH. These will include:

6 Hematology Franchise Product Oral Presentations

38 Poster Presentations

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 Hemophilia A is more common than hemophilia B;5 hemophilia A affects about 150,000 people, whereas hemophilia B affects about 30,000 people worldwide.6

People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.7 Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.5,8

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 patients9 and a broad portfolio of 11 products across nine hemophilia indications10. Our experience as leaders in hematology means we are well prepared to meet today’s needs as we pursue future developments in the care of bleeding 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 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, Gastroenterology (GI), Neuroscience, and Rare Diseases. 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

References

1

Klamroth R, Windyga J, Radulescu V, et al., PK-guided rurioctocog alfa pegol prophylaxis in patients with severe hemophilia A targeting two FVIII trough levels: results from the phase 3 PROPEL Study. Presented at ISTH 2019 (International Society on Thrombosis and Haemostasis (ISTH) Biennial Congress. July 6-10, 2019. Abstract #A-1052-0038-01311.

2

Klamroth R, Windyga J, Radulescu V, et al., Results of a phase 3, randomized, multicenter study of RURIOCTOCOG ALFA PEGOL PK-guided prophylaxis targeting 2 FVIII trough levels in patients with severe Hemophilia A (propel study). Presented at European Association of Haematology and Allied Disorders (EAHAD) February 2019. Abstract #255.

3

National Institutes of Health: National Heart, Lung and Blood Institute. “Gene Therapy Helps Patients With Hemophilia B.” National Heart, Lung and Blood Institute website. https://www.nhlbi.nih.gov/news/enewsletter/professional/ProfHemophilia-article Last Accessed April 2019.

4

Nathwani AC, Tuddenham EG, Rangarajan S, et al., Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B. N Engl J Med. 2011;365(25):2357–2365. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3265081/ Last Accessed April 2019.

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

World Federation of Hemophilia. “About Bleeding Disorders: Treatment.” World Federation of Hemophilia website. https://www.wfh.org/en/page.aspx?pid=642 Last Accessed April 2019.

8

National Hemophilia Foundation. “Hemophilia A”. National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A. Last Accessed April 2019.

9

Shire Website. Standards of Care for Hemophilia. Website: https://www.shire.com/who-we-are/how-we-operate/policies-and-positions/standards-of-care-for-hemophilia Last Accessed April 2019.

10

Shire Website. Product List. Website: https://www.shire.com/products/product-list?t= Last Accessed June 2019

Contacts

Media Contacts:

Japanese Media

Kazumi Kobayashi

kazumi.kobayashi@takeda.com

+81 (0) 3-3278-2095

Media outside Japan

Tsuyoshi Tada

tsuyoshi.tada@takeda.com

+1 (617) 551-2933

Media outside Japan

Linda Calandra

linda.calandra1@takeda.com

+1 (617) 301-2092

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