Novel Clinical Study Collaboration Announced Designed to Improve Clinical Care for Hemophilia Patients through Innovative Personalized Treatment

Novel Clinical Study Collaboration Announced Designed to Improve Clinical Care for Hemophilia Patients through Innovative Personalized Treatment

December 14, 2017

Cambridge, MA – December 14, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotech leader in rare diseases, RCSI (Royal College of Surgeons Ireland ), and Science Foundation Ireland (SFI), in collaboration with the Irish Haemophilia Society, today jointly announced the initiation of a novel clinical study called “The Irish Personalised Approach to the Treatment of Haemophilia (iPATH).” Globally, approximately 187,000 people have been diagnosed with hemophilia, an inherited bleeding disorder.

“Today, in developed countries, most patients receive prophylactic treatment, which is recognized as the standard of care. For those patients on prophylaxis, treatment should be optimized by combining innovation with personalization,” said Peter Turecek, Senior Director, Global Medical Affairs, Shire. “Shire is committed to driving innovation and advancing standards of care in hemophilia. Through the iPATH study, we hope to uncover new solutions that build on and maximise the role of factor therapy and further personalize care for hemophilia patients.”

Instead of focusing on standardized treatments, the iPATH study seeks to take the next step in treatment and will investigate new personalized treatment approaches by tailoring care based on the needs of individual patients. Patients in Ireland with hemophilia are registered at a single National Coagulation Centre where data on the use of factor concentrates and bleeding rates have already been collected. This provides a unique opportunity to conduct a hemophilia study aimed at better understanding the underlying biology of hemophilia with the goal of developing a personalized approach to hemophilia care that can then potentially be extended to the global hemophilia community. This clinical study approach also has the potential to be adapted for other diseases.

“Over the past 30 years, the principal focus of physicians engaged in caring for patients with hemophilia has been to ensure that our treatments are effective and have a good safety profile,” said study lead Professor James O’Donnell, Director of the Irish Centre for Vascular Biology, RCSI, and a Consultant Hematologist at the National Coagulation Center in St James’ Hospital, Dublin. “Hoping to enhance quality of life for people with hemophilia in the future, we need to begin developing innovative treatment strategies that can be tailored specifically according to the needs of each individual patient. To achieve this objective, we first need to understand the biological mechanisms that underpin the marked differences in bleeding risks and long-term complications that exist between individual patients with hemophilia. By understanding these mechanisms, the iPATH study could potentially pave the way for the introduction of personalized medicine for patients with hemophilia.”

Commenting on the announcement, Director General of Science Foundation Ireland and Chief Scientific Adviser to the Government of Ireland, Professor Mark Ferguson, said, “The iPATH study combines the best expertise to improve treatment options and outcomes for patients with hemophilia worldwide. Science Foundation Ireland is committed to supporting collaborations such as these, which can significantly improve health care services.”

Brian O’Mahony, Chief Executive of the Irish Haemophilia Society said, “The future of hemophilia care should be based on a personalized approach to treatment. We anticipate that this exciting, innovative and collaborative research program may provide us with answers to potentially optimize future treatments for individuals with hemophilia in Ireland and to hopefully further apply this research to hemophilia patients globally.”

Director of Research and Innovation at RCSI, Professor Ray Stallings, welcomed the announcement saying, “RCSI is delighted to announce this new collaboration with SFI and Shire. At RCSI we have a strong focus on translating scientific research into new diagnostics and treatments for the benefit of patients and healthcare systems. This study exemplifies how collaboration between academia and industry can help improve the health of people with hemophilia through high-quality scientific research.”

The iPATH study will take place over a four-year period. The study is supported by an SFI Strategic Partnership initiative and involves scientific researchers in RCSI, Trinity College Dublin (TCD) and Shire, based on its strong and established presence in Ireland. The iPATH partnership also includes clinical researchers based in St James’s Hospital Dublin, Our Lady’s Children’s Hospital Crumlin, Cork University Hospital and University Hospital Galway.

For further information please contact:

Jaren Madden (Herron)
Head, R&D Communications
+617 588 8137

Freeha Rafiq
Communications Lead, UK and Ireland
+44 (0)7500 953471

Jane Butler
Senior Communications Officer
+353 (0)1 402 8610 / +353 (0) 87 7531877

Louise Loughran
Head of Communications
+353 (0)1 402 2242


About Shire

Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.

We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people’s lives with medicines that have a meaningful impact on patients and all who support them on their journey.

About RCSI

RCSI is ranked among the top 250 (top 2%) of universities worldwide in the Times Higher Education World University Rankings (2018) and its research is ranked first in Ireland for citations. It is an international not-for-profit health sciences institution, with its headquarters in Dublin, focused on education and research to drive improvements in human health worldwide. RCSI is a signatory of the Athena SWAN Charter.

About Science Foundation Ireland

Science Foundation Ireland funds oriented basic and applied research in the areas of science, technology, engineering, and mathematics (STEM) which promotes and assists the development and competitiveness of industry, enterprise and employment in Ireland. The Foundation also promotes and supports the study of and engagement with STEM and promotes an awareness and understanding of the value of STEM to society and, in particular, to the growth of the economy. See

Science Foundation Ireland has launched the #BelieveInScience campaign to promote the potential that science and discovery offer Ireland, today and in tomorrow’s world. The #BelieveInScience campaign will see Science Foundation Ireland work in partnership with the Irish research community to share a mutual passion for science with the public; to promote an understanding of the ability of science, technology, engineering and maths (STEM) to create positive change in the world and to drive a sustainable economy in Ireland.