Shire Presentations: ASH 2016. Rare Hematologic and Specialty Conditions
November 4, 2016

Broad Range of Research to be Presented at ASH 2016 Demonstrates Shire’s Commitment to Addressing Unmet Needs for Patients with Rare Hematologic and Specialty Conditions

Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on serving individuals with rare diseases, announced today the depth and breadth of the data it will be presenting at the 58th American Society of Hematology (ASH) Annual Meeting, taking place December 3-6 in San Diego, California. The company’s robust research in hematology, oncology and genetic disease will be showcased in 4 oral presentations and 12 poster presentations.

“Shire’s extensive presence at ASH 2016 reinforces our commitment to discovering and delivering transformative treatments for patients with rare conditions,” said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. “We aim to continually lead the scientific dialogue for rare hematologic research and address some of the most urgent unmet needs for patients facing these challenging diseases.”


Data in hematology, the largest portfolio for the company, will focus on research that continues to build on the proven approach of direct factor replacement as the global standard of care for hemophilia. Data presentations will showcase the continued growth of the portfolio and new strategies to improve outcomes for people with hemophilia and other bleeding conditions. The company will also present research on several of its promising early- and late-stage pipeline programs:

Target Joint Status in Patients with Hemophilia A During 18 Consecutive Months of Prophylaxis with a Pegylated Full-Length Recombinant Factor VIII with Extended Half-Life Pub #2592. Session #322. Session Title: Disorders of Coagulation or Fibrinolysis. Poster # II. Sunday, Dec. 4, 2016 6 p.m. – 8 p.m. in Hall GH (San Diego Convention Center)

Modelling FVIII Levels for Prediction of Zero Spontaneous-Joint Bleeding in a Cohort of Severe Hemophilia A Subjects with Target Joints Initiated on Tertiary Prophylaxis Pub #2576. Session #322. Session Title: Disorders of Coagulation or Fibrinolysis. Poster # II. Sunday, Dec. 4, 2016 6 p.m. – 8 p.m. in Hall GH (San Diego Convention Center)

Pharmacodynamic Profile of a Recombinant ADAMTS13 (BAX930) in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome (USS)) Pub #135. Session #311. Session Title: Disorders of Platelet Number of Function: TTP and HUS. Saturday, Dec. 3, 2016 12 p.m. – 1:30 p.m. in Room 29 (San Diego Convention Center)

Appearance of High-Affinity Antibodies Precedes Clinical Diagnosis of FVIII Inhibitors – Preliminary Analysis from the Hemophilia Inhibitor PUP Study (HIPS) Pub #328. Session #322. Session Title: Disorders in Coagulation or Fibrinolysis: Hemophilia Inhibitors. Sunday, Dec. 4, 2016 9:30 a.m. – 11 a.m. in Room 33 (San Diego Convention Center)


As part of Shire’s focus on serving individuals with rare diseases, the company is committed to addressing unmet needs in areas of oncology where patients and providers have limited treatment options. Shire has pipeline assets and research targets for rare cancers including metastatic pancreatic cancer, colorectal cancer and targets in checkpoint inhibitors and allogeneic CAR-T, as well as a biologic treatment approved as part of a multi-agent chemotherapeutic regimen for the treatment of acute lymphocytic leukemia (ALL), ONCASPAR™ (pegaspargase), that is approved by the U.S. Food & Drug Administration (FDA) and all 27 EU member states. Data presented at ASH will include a focus on pegaspargase as a therapeutic option for ALL.


Shire is also dedicated to helping patients with inherited illnesses. Type I Gaucher disease is a rare, inherited metabolic condition, and the most common of a family of rare diseases known as lysosomal storage disorders (LSDs). It affects approximately 1 in 50,000 to 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. Every type I Gaucher patient is unique and will experience varying symptoms and degrees of disease severity, making type I Gaucher disease difficult to diagnose. To this end, Shire will be hosting an engaging, case-based presentation to raise disease awareness:

Product Theater: Julie M: A Diagnostic Journey. Monday, Dec. 5, 12:15 p.m. to 1:15 p.m.