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More than 35 presentations and sessions showcase Shire’s gene therapy pipeline and leading factor portfolio: Shire to present new data at ISTH 2017 to advance the standard of care in haemophilia

Zug, Switzerland (June 23, 2017) – Shire plc (LSE: SHP, NASDAQ: SHPG), a leading biotechnology company focused on serving individuals with rare diseases, will present research covering a broad range of rare bleeding disorders at the 26th Biennial International Society on Thrombosis and Haemostasis Congress (ISTH), July 8-13, 2017 in Berlin, Germany. Showcased in eight oral presentations, 29 poster presentations and three symposia, these data underscore Shire’s pursuit of treatment innovation rooted in safety, efficacy, and individualized care for hemophilia patients.

“As the global leader in rare hematology with over 70 years of expertise, Shire is proud to showcase the depth of our hemophilia portfolio and the latest data reinforcing the proven safety and efficacy of our products,” said Bruce Ewenstein, M.D., Ph.D., VP Clinical Development for Hematology at Shire. “In addition, we look forward to presenting new research in gene therapy and a novel patient-reported outcome measure that may help patients individualize their hemophilia treatment goals.”

Advancing the Promise of Gene Therapy in Hemophilia

Throughout ISTH, Shire will present 11 scientific updates regarding its gene therapy pipeline, which shows promise for both hemophilia A and B populations. 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.1 Therefore, it may be possible to help convert a hemophilia patient’s bleeding phenotype from severe to mild or even normal in some cases.2 Notably, Shire will showcase the following new data presentations on the development of SHP654 for the treatment of hemophilia A.

• Development of SHP654, a highly efficient AAV8-based BDD-FVIII gene therapy vector for treatment of hemophilia A. Session Title: Gene Therapy for Hemophilia: Clinical. Oral # OC 13.6 .10th July, 17:45-19:00 CEST; Hall B3
• Integration site analysis in mice demonstrates excellent biosafety profile of a recombinant (r) FVIII adeno-associated virus (AAV8) gene therapy product Session Title: Poster Session. Poster #PB 1094. 11th July, 12:00-13:15 CEST, Exhibition Hall 4.24

In addition, Shire will give an oral presentation on its gene therapy technology for hemophilia B:

• SHP648: A high performing next generation FIX gene therapy vector based on AAV8 Session Title: Gene Therapy for Hemophilia: Preclinical. Oral# OC 62.2. 12th July, 09:30-10:45 CEST, Helsinki 15

Paving a Frontier in Patient-Centered Care in Hemophilia

Shire will present new data on its novel patient-reported outcome measure, Goal Attainment Scale for Hemophilia (GAS-Hēm). GAS-Hēm is intended to support advanced hemophilia care by supplementing standard clinical outcome measures with those that are patient-centered, customizable, and sensitive to change within individual patients and across patient populations.6 Patient goals are identified and agreed upon together and can span various goal areas, such as managing hemophilia complications and impact on life. Results of a 12-week study of 42 patients aged 5-65 years of age with hemophilia A or B on prophylaxis treatment suggest that GAS-Hēm is successfully uncovering outcome measures not captured by currently available tools.6

• Responsiveness and construct validity of Goal Attainment Scaling for Hemophilia (GAS-Hēm): a novel, personalised, patient-reported outcome for haemophilia, Session Title: Poster Session. Poster #PB 203. 10th July, 12:00-13:15 CEST, Exhibition Hall 2.2.6

About Hemophilia

Hemophilia is a challenging chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.7 Hemophilia A is more common than hemophilia B;7 hemophilia A affects about 150,000 people, whereas hemophilia B affects about 30,000 people worldwide.8

People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.9 Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.7,10

Inhibitors are a serious complication in patients with hemophilia.11 They form when the body’s immune system attacks the proteins in factor therapy, causing it to be ineffective.11 Immune tolerance induction and bypassing agents are essential therapies for this patient population that help either train the body to tolerate the factor concentrate or bypass the inhibitor to help the body form a clot and stop bleeding.9,11,12

References

1. 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 – Accessed May 31, 2017.
2. Amit et al. “Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B.” N Engl J Med. 2011 Dec 22; 365(25): 2357–2365. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3265081 Accessed May 31, 2017.
3. Falkner et al. “Development of SHP654 a highly efficient AAV8-based BDD-FVIII gene therapy vector for treatment of hemophilia A.” International Society on Thrombosis and Haemostasis Congress. Berlin, Germany July 8-13, 2017. Available at: http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)2475-0379
4. Hoellriegl et al. “Integration site analysis in mice demonstrates excellent biosafety profile of a recombinant ® FVIII adeno-associated virus (AAV8) gene therapy product.” International Society on Thrombosis and Haemostasis Congress. Berlin, Germany July 8-13, 2017. Available at: http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)2475-0379
5. Horling et al. “SHP648: A high performing next generation FIX gene therapy vector based on AAV8.” International Society on Thrombosis and Haemostasis Congress. Berlin, Germany July 8-13, 2017. Available at: http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)2475-0379
6. Recht et al. “Responsiveness and construct validity of Goal Attainment Scaling for Hemophilia (GAS-Hēm): a novel, personalised, patient-reported outcome for haemophilia.” International Society on Thrombosis and Haemostasis Congress. Berlin, Germany July 8-13, 2017. Available at: http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)2475-0379
7. World Federation of Hemophilia. “What is hemophilia?” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646 . Accessed January 12, 2017
8. World Federation of Hemophilia. Report on the Annual Global Survey 2015. World Federation of Hemophilia website. http://www1.wfh.org/publication/files/pdf-1669.pdf . Accessed January 12, 2017.
9. World Federation of Hemophilia. “About Bleeding Disorders: Treatment.” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=642 . Accessed January 12, 2017.
10. National Hemophilia Foundation. “Hemophilia A”. National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A . Accessed January 12, 2017.
11. World Federation of Hemophilia. “What are inhibitors?” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=651 . Accessed June 12, 2017.
12. Hemophilia Federation of America. “Treatment & Eradication of Inhibitors.” Hemophilia Federation of America website. http://www.hemophiliafed.org/bleeding-disorders/inhibitors/treatment-eradication-inhibitors/ . Accessed June 12, 2017.

About Shire

Shire is the leading global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. We strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; and a growing franchise in Oncology.

Our employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.

• http://www.shire.com

Source: Shire, 23.06.2017 (tB).