More than 35 presentations and sessions showcase Shire’s gene therapy pipeline and leading factor portfolio
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.1Therefore, 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
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
- 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.
- 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.
- 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
- 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
- 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
- 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
- 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
- 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.
- 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.
- 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.
- 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.
- 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.
For further information, please contact:
NOTES TO EDITORS
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.
Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:
- Shire’s products may not be a commercial success;
- increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire’s future revenues, financial condition and results of operations;
- Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services. Some of Shire’s products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire’s products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
- the manufacture of Shire’s products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to, among other things, significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
- certain of Shire’s therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
- Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
- the actions of certain customers could affect Shire’s ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire’s revenues, financial conditions or results of operations;
- Shire’s products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
- adverse outcomes in legal matters, tax audits and other disputes, including Shire’s ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the combined company’s revenues, financial condition or results of operations;
- inability to successfully compete for highly qualified personnel from other companies and organizations;
- failure to achieve the strategic objectives, including expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all with respect to Shire’s acquisition of NPS Pharmaceuticals Inc., Dyax Corp. or Baxalta Incorporated may adversely affect Shire’s financial condition and results of operations;
- Shire’s growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
- a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
- failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to Shire’s reputation, the withdrawal of the product and legal action against Shire;
- investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire’s activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
- Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire’s revenues, financial condition or results of operations;
- Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which may decrease its business flexibility and increase borrowing costs; and
a further list and description of risks, uncertainties and other matters can be found in Shire’s most recent Annual Report on Form 10-K and in Shire’s subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in “ITEM 1A: Risk Factors”, and in subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire’s website.
All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.