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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

Friday, November 4, 2016 Research News J E 4
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LEXINGTON, Massachusetts, November 4, 2016 /PRNewswire/ --

16 presentations to highlight innovation in hematology, oncology and genetic diseases

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."

HEMATOLOGY

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)

ONCOLOGY

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.

GENETIC DISEASE

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.  

Important Information for ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Indications ADYNOVATE, [Antihemophilic Factor (Recombinant), PEGylated], is a human antihemophilic factor indicated in adolescent and adult patients (12 years and older) with hemophilia A (congenital factor VIII deficiency) for:

  • On-demand treatment and control of bleeding episodes
  • Routine prophylaxis to reduce the frequency of bleeding episodes

ADYNOVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION 

CONTRAINDICATIONS ADYNOVATE is contraindicated in patients who have had prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).

WARNINGS & PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

Neutralizing Antibodies Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.

ADVERSE REACTIONS Common adverse reactions (?1% of subjects) reported in the clinical studies were headache and nausea.

For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf    

Important Information for ADVATE [Antihemophilic Factor (Recombinant)] Indications ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:

  • Control and prevention of bleeding episodes
  • Perioperative management
  • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes

ADVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION 

CONTRAINDICATIONS ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.

WARNINGS & PRECAUTIONS Hypersensitivity Reactions Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting. Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Neutralizing Antibodies Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.

ADVERSE REACTIONS Serious adverse reactions seen with ADVATE are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII. The most common adverse reactions observed in clinical trials (frequency ?5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.

For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADVATE_USA_ENG.pdf    

Important Information for ONCASPAR(pegaspargase)

Indications 

ONCASPAR is an asparagine specific enzyme indicated as a component of a multi-agent chemotherapeutic regimen for treatment of patients with:

  • First line acute lymphoblastic leukemia
  • Acute lymphoblastic leukemia and hypersensitivity to asparaginase

DETAILED IMPORTANT RISK INFORMATION 

CONTRAINDICATIONS 

ONCASPAR is contraindicated in patients who have:

  • History of serious allergic reactions to ONCASPAR
  • History of serious thrombosis with prior L-asparaginase therapy
  • History of pancreatitis with prior L-asparaginase therapy
  • History of serious hemorrhagic events with prior L-asparaginase therapy

WARNINGS & PRECAUTIONS Anaphylaxis and Serious Reactions Anaphylaxis and serious allergic reactions can occur therefore, patients should be observed for one hour after administration. Discontinue ONCASPAR in patients with serious allergic reactions.

Thrombosis 

Serious thrombotic events, including sagittal sinus thrombosis can occur Discontinue ONCASPAR in patients with serious thrombotic events.

Pancreatitis  

Pancreatitis can occur. Evaluate patients with abdominal pain for evidence of pancreatitis. Discontinue ONCASPAR in patients with pancreatitis.

Glucose Intolerance 

Glucose intolerance can occur. In some cases, glucose intolerance is irreversible. Monitor serum glucose.

Coagulopathy 

Increased prothrombin time, increased partial thromboplastin time, and hypofibrinogenemia can occur. Monitor coagulation parameters at baseline and periodically during and after treatment.

Hepatotoxicity and Abnormal Liver Function 

Hepatotoxicity and abnormal liver function can occur. Perform appropriate monitoring.

ADVERSE REACTIONS 

Most common adverse reactions (?2%) are allergic reactions (including anaphylaxis),

hyperglycemia, pancreatitis, central nervous system (CNS) thrombosis, coagulopathy, hyperbilirubinemia, and elevated transaminases.

Hyperlipidemia (hypercholesterolemia and hypertriglyceridemia) has been reported in patients exposed to ONCASPAR.

For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ONCASPAR_USA_ENG.pdf  

Important Information for VPRIV® (velaglucerase alfa for injection) 

Indications 

VPRIV is a hydrolytic lysosomal glucocerebroside-specifi enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

DETAILED IMPORTANT RISK INFORMATION CONTRAINDICATIONS 

None

WARNINGS & PRECAUTIONS 

Hypersensitivity Reactions  

Hypersensitivity reactions, including anaphylaxis, have occurred with VPRIV. The most commonly observed symptoms of hypersensitivity reactions were headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia/body temperature increased. Additional hypersensitivity reactions of chest discomfort, dyspnea, and pruritus have been reported with VPRIV. As with any intravenous protein product, hypersensitivity reactions are possible, therefore appropriate medical support including personnel adequately trained in cardiopulmonary resuscitative measures and access to emergency measures should be readily available when VPRIV is administered. If anaphylactic or other acute reactions occur, immediately discontinue the infusion of VPRIV and initiate appropriate medical treatment. Discontinue VPRIV if hypersensitivity symptoms occur.

ADVERSE REACTIONS 

Most common adverse reactions during clinical studies in ?10% of patients were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated PTT, fatigue/asthenia, and pyrexia.

For Full Prescribing Information, visit http://pi.shirecontent.com/PI/PDFs/Vpriv_USA_ENG.pdf  

NOTES TO EDITORS 

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

Forward-Looking Statements 

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 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 with respect to Shire's acquisition of NPS Pharmaceuticals, Inc., Dyax Corp. ("Dyax") or Baxalta Inc. ("Baxalta") 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 the 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;
  • difficulties in integrating Dyax or Baxalta into Shire may lead to the combined company not being able to realize the expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all; and

other risks and uncertainties detailed from time to time in Shire's filings with the Securities and Exchange Commission, including those risks outlined in "ITEM 1A: Risk Factors" in Shire's Quarterly Report on Form 10-Q for the quarter ended June 30, 2016.

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.

References

  1. American Society of Hematology. "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". December 2016. Available at: https://ash.confex.com/ash/2016/webprogram/Paper96416.html
  2. American Society of Hematology. "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" December 2016. Available at: https://ash.confex.com/ash/2016/webprogram/Paper91571.html
  3. American Society of Hematology. Pharmacodynamic Profile of a Recombinant ADAMTS13 (BAX930) in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome (USS)) December 2016. Available at: https://ash.confex.com/ash/2016/webprogram/Paper92519.html
  4. American Society of Hematology. Appearance of High-Affinity Antibodies Precedes Clinical Diagnosis of FVIII Inhibitors - Preliminary Analysis from the Hemophilia Inhibitor PUP Study (HIPS) December 2016. Available at: https://ash.confex.com/ash/2016/webprogram/Paper95566.html
  5. American Society of Hematology. "Randomized Study of Pegasparagase (SS-PEG) and Calaspargase Pegol (SC-PEG) in Pediatric Patients with Newly Diagnosed Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma: Results of DFCI ALL Consortium Protocol 11-001". December 2016. Available at: https://ash.confex.com/ash/2016/webprogram/Paper94488.html
  6. National Library of Medicine. National Institutes of Health. Gaucher disease.http://www.nlm.nih.gov/medlineplus/ency/article/000564.htm. Accessed October 30, 2016
  7. Guggenbuhl P, GrosboisB, ChalèsG. Gaucher disease. Joint Bone Spine. 2008;75(2):116-124

For further information, please contact: Investor Relations Sarah Elton-Farr -seltonfarr@shire.com -+44-(0)1256-894157 Ian Karp - ikarp@shire.com - +1-781-482-9018 Robert Coates - rcoates@shire.com +44-(0)1256-894874 Media Gwen Fisher -gfisher@shire.com - +1-484-595-9836 Molly Poarch - molly.poarch@shire.com - +1-312-965-3413

SOURCE Shire plc

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