Shire to Present Data at 2018 AAAAI/WAO Joint Congress Demonstrating Commitment to Ongoing Research and Innovation in Rare Immune Conditions

| Source: Shire Pharmaceuticals Group Shire Pharmaceuticals Group

Shire to Present Data at 2018 AAAAI/WAO Joint Congress Demonstrating Commitment to Ongoing Research and Innovation in
Rare Immune Conditions

  • Hereditary angioedema (HAE) and primary immunodeficiency (PI) data to be presented, including results from pivotal Phase 3 HELP(TM) study exploring investigational lanadelumab as a preventative treatment for HAE

CAMBRIDGE, Mass. - February 20, 2018 - Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, announced today that new immunology data will be presented at the 2018 American Academy of Allergy, Asthma & Immunology (AAAAI) and World Allergy Organization (WAO) Joint Congress, March 2-5 in Orlando, Fla.

The broad range of research being presented includes pivotal data on Shire's investigational hereditary angioedema (HAE) treatment lanadelumab, as well as new clinical data around FDA-approved HAE and primary immunodeficiency (PI) treatments. The company will also present new research on the burden of disease for people living with HAE.

"At Shire, we are focused on driving innovations that will make a meaningful difference in the lives of patients living with rare immune diseases," said Howard Mayer, Chief Medical Officer, Shire. "The depth and breadth of data being presented at this year's AAAAI/WAO Joint Congress clearly demonstrates the evolving needs of those living with HAE and PI and reinforces our commitment to developing treatments that help address the individual patient needs of these communities."

Hereditary Angioedema is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.[1],[2] The condition results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.1,[3],[4]

Primary Immunodeficiencies (PI) are a group of more than 300 disorders in which part of the body's immune system is missing or does not function properly.[5] It is estimated that as many as six million children and adults may be affected by PI worldwide.[6]

Key Shire data presentations at 2018 AAAAI/WAO Joint Congress include:

  • HAE Research (9:45-10:45 a.m. ET, Saturday, March 3, Convention Center, South Concourse, Level 1, South Hall A2)
    • CINRYZE® (C1 esterase inhibitor [human]) is Efficacious for Hereditary Angioedema Attack Prevention In Pediatric Patients: Final Phase 3 Efficacy And Safety Results (Poster Presentation 149)
    • Efficacy of Lanadelumab in Patients Switching from Long-Term Prophylaxis with C1-Inhibitor (C1-INH): Results from the Phase 3 HELP Study (Poster Presentation 150)
    • Consistent Lanadelumab Treatment Effect In Patients With Hereditary Angioedema Regardless Of Baseline Attack Frequency In The Phase 3 HELP Study (Poster Presentation 151)
    • Lanadelumab Markedly Improves Health-Related Quality of Life in Hereditary Angioedema Patients in the HELP Study (Poster Presentation 152)
    • Characterization Of Hereditary Angioedema Attacks Requiring Reinjection Of Icatibant: Findings From The Icatibant Outcome Survey (Poster Presentation 168)
    • Healthcare Resource Utilization Among a US Cohort Treated for Hereditary Angioedema, 2010-2015 (Poster Presentation 169)
    • Burden of Hereditary Angioedema: Findings From a US Patient Survey (Poster Presentation 180)
    • Long-term Safety Outcomes of Prekallikrein Deficiency: A Systematic Literature Review (Poster Presentation 184)
    • An Observational Study to Understand Patient and Physician Communication in Hereditary Angioedema (Poster Presentation 187)
  • PI Research  
    • Onboarding Experience of Patients With Primary Immunodeficiency Diseases Who Switched to Subcutaneous Human Immune Globulin 20% (Ig20Gly) From Intravenous or Subcutaneous Immune Globulin 10%. (Poster Presentation 79, 9:45-10:45 a.m. ET, Saturday, March 3, Convention Center, South Concourse, Level 1, South Hall A2) 
    • Infusion Parameters and Adverse Events in Patients With Primary Immunodeficiency Diseases Who Switched to Subcutaneous Human Immune Globulin 20% (Ig20Gly) From Intravenous or Subcutaneous Immune Globulin (Poster Presentation 845, 9:45-10:45 a.m. ET, Monday, March 5, Convention Center, South Concourse, Level 1, South Hall A2)
    • Onboarding Experience of Pediatric Patients With Primary Immunodeficiency Diseases With Subcutaneous Human Immune Globulin 20% (Ig20Gly). (Poster Presentation 846, 9:45-10:45 a.m. ET, Monday, March 5, Convention Center, South Concourse, Level 1, South Hall A2)

The poster presentations are intended for scientific discussion only.

Shire will also be sponsoring a booth 311 in the Orange County Convention Center, as well as several events listed below that focus on HAE and PI.

Events Sponsored by Shire:

  • Hereditary Angioedema
    • Patient Perspectives on Hereditary Angioedema (HAE): Personalized Management Through Shared Decision Making

Friday, March 2 from 7:30-9:30 a.m. ET
Hyatt, Convention Level, Regency PQ

  • Expert Perspectives on Evolving Approaches in HAE Patient Management

Friday, March 2 from 6:30-8:30 p.m. ET
Hyatt, Convention Level, Windermere Y

  • Primary Immunodeficiency
    • The Impact of Shared Decision-Making on Patients with PIDD

Thursday, March 1 from 6:30-8:30 p.m. ET
Hyatt, Convention Level, Regency PQ

  • Patient Perspectives on Primary Immunodeficiency (PI): Shared Decision-Making for Personalized PI Management

Saturday, March 3 from 8:30-10:30 p.m. ET
Hyatt, Convention Level, Regency PQ

INDICATION AND IMPORTANT SAFETY INFORMATION FOR CINRYZE® (C1 esterase inhibitor [human])

INDICATION
CINRYZE® (C1 esterase inhibitor [human]) is indicated for routine prophylaxis against
angioedema attacks in adolescent and adult patients with Hereditary Angioedema (HAE).

IMPORTANT SAFETY INFORMATION
CINRYZE is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis to the product.

Hypersensitivity Reactions: Severe hypersensitivity reactions may occur during or after administration of CINRYZE. Consider treatment methods carefully, because hypersensitivity reactions and HAE attacks may have similar symptoms. In case of hypersensitivity, discontinue CINRYZE infusion and institute appropriate treatment. Have epinephrine immediately available for treatment of an acute severe hypersensitivity reaction.

Thromboembolic Events: Serious arterial and venous thromboembolic (TE) events have been reported at the recommended dose of C1 Esterase Inhibitor (Human) products, including CINRYZE, following administration in patients with HAE. Risk factors may include presence of an indwelling venous catheter/access device, prior history of thrombosis, underlying atherosclerosis, use of oral contraceptives, certain androgens, morbid obesity, and immobility. Benefits of CINRYZE for routine prophylaxis of HAE attacks should be weighed against the risks of TE events in patients with underlying risk factors. Monitor patients with known risk factors for TE events during and after CINRYZE administration.

Transmissible Infectious Agents: Because CINRYZE is made from human blood, it may carry a risk of transmitting infectious agents e.g. viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. ALL infections thought by a physician possibly to have been transmitted by CINRYZE should be reported to Shire Medical Information at 1-800-828-2088.

Adverse Reactions: The only serious adverse reaction observed in clinical studies of CINRYZE was cerebrovascular accident. The most common adverse reactions observed were headache, nausea, rash, and vomiting. Post-marketing adverse reactions include local infusion site reactions and hypersensitivity. Post-marketing thromboembolic events have been reported, including catheter-related and deep venous thrombosis, transient ischemic attack, and stroke.

For additional safety information, click for Prescribing Information and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR FIRAZYR® (icatibant injection)

INDICATION
FIRAZYR® (icatibant injection) is a medicine used to treat acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older.

IMPORTANT SAFETY INFORMATION
Laryngeal attacks can become life threatening. If you have an HAE attack of the throat (laryngeal attack), inject FIRAZYR and then go to the nearest hospital emergency room right away.

The most common side effects of FIRAZYR include:

  • redness, bruising, swelling, warmth, burning, itching, irritation, hives, numbness, pressure, or pain at the injection site
  • fever
  • too much of an enzyme called transaminase in your blood
  • dizziness
  • nausea
  • headache
  • rash

These are not all of the possible side effects of FIRAZYR. Tell your healthcare provider if you have any side effect that bothers you or that does not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Tell your healthcare provider if you have any other medical conditions, if you are breastfeeding or plan to breastfeed, or if you are pregnant or planning to become pregnant. FIRAZYR has not been evaluated in pregnant or nursing women. You and your healthcare provider will decide if FIRAZYR is right for you.

If your symptoms continue or come back, you may repeat your FIRAZYR injection at least 6 hours apart. Do not use more than 3 doses of FIRAZYR in a 24-hour period.

Tiredness, drowsiness, and dizziness have been reported following the use of FIRAZYR. If this occurs, do not drive a car, use machinery, or do anything that needs you to be alert.

For additional safety information, click for Prescribing Information and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR CUVITRU [Immune Globulin Subcutaneous (Human), 20% Solution] 


What is CUVITRU?

  • CUVITRU is a ready-to-use, liquid medicine that contains immunoglobulin G (IgG) antibodies, which protect the body against infection.
  • CUVITRU is indicated for the treatment of primary humoral immunodeficiency (PI) in adult and pediatric patients two years of age and older.
  • CUVITRU is made from human plasma that is donated by healthy people. CUVITRU contains antibodies collected from these healthy people that replace the missing antibodies in PI patients.
  • CUVITRU is given under the skin (subcutaneously).
  • Most of the time infusions under the skin are given at home by self infusion or by caregivers. Only use CUVITRU by yourself after you have been instructed by your healthcare provider.

Important Risk Information

What is the most important information that I should know about CUVITRU?

CUVITRU can cause the following serious reactions:

  • Severe allergic reactions causing difficulty in breathing or skin rashes
  • Decreased kidney function or kidney failure
  • Blood clots in the heart, brain, lungs, or elsewhere in the body
  • Severe headache, drowsiness, fever, painful eye movements, or nausea and vomiting
  • Dark colored urine, swelling, fatigue, or difficulty breathing

Who should not use CUVITRU?

Do not use CUVITRU if you:

  • Are allergic to immune globulin or other blood products.
  • Have selective (or severe) immunoglobulin A (IgA) deficiency with antibodies to IgA.

CUVITRU can cause serious side effects.  Call your healthcare professional or go to the emergency department right away if you get:

  • Hives, swelling in the mouth or throat, itching, trouble breathing, wheezing, fainting or dizziness. These could be signs of a serious allergic reaction.
  • Bad headache with nausea, vomiting, stiff neck, fever, and sensitivity to light. These could be signs of irritation of the lining around your brain.
  • Reduced urination, sudden weight gain, or swelling in your legs. These could be signs of a kidney problem.
  • Pain, swelling, warmth, redness, or a lump in your legs or arms. These could be signs of a blood clot.
  • Brown or red urine, fast heart rate, yellow skin or eyes. These could be signs of a liver or blood problem.
  • Chest pain or trouble breathing, or blue lips or extremities. These could be signs of a serious heart or lung problem.
  • Fever over 100ºF. This could be sign of an infection.

What are the possible or reasonably likely side effects of CUVITRU?

The following one or more possible side effects may occur at the site of infusion: mild or moderate pain, redness, and itching. These generally go away within a few hours, and are less likely after the first few infusions.

The most common side effects that may occur are: headache, nausea, fatigue, diarrhea, and vomiting.

These are not all the possible side effects. Talk to your healthcare professional about any side effects that bother you or that don't go away.

For additional safety information, including Warning regarding blood clots, click Prescribing Information for information for patients, and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

About the Immunology Franchise
Shire's immunology franchise has a strong legacy in developing therapies for people living with hereditary angioedema and primary immunodeficiency. Our broad portfolio includes commercial products and investigational therapies. We are committed to serial innovation and rely on our expertise to help fulfill unmet treatment needs for patients living with these diseases. Beyond our focus on developing novel treatments, we provide specialized services and support offerings that help meet the needs of our patients.  

About Hereditary Angioedema
Hereditary Angioedema is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.1,2 The condition results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.1,3,4 The swelling can be debilitating and painful, resulting in people with HAE taking an average of 20 days off work/education per year due to HAE. Attacks that obstruct the airways (asphyxiation) are potentially life-threatening.1,3,4,[7]

About Primary Immunodeficiency
Primary Immunodeficiencies (PI) are a group of more than 300 disorders in which part of the body's immune system is missing or does not function properly. Normally, the immune system protects the body from pathogenic microorganisms like bacteria, viruses, and fungi, which can cause infectious diseases. When any part of a person's immune system is absent or dysfunctional, these individuals are susceptible to infections, and it may take longer to recover from infections. When a defect in the immune system is inherited and genetically determined, it is called primary immune deficiency.5 It is estimated that as many as six million children and adults may be affected by PI worldwide.6

For further information please contact:

Media Relations    
Elizabeth Kalina ekalina@shire.com +1 781 482 2713
Gwen Fisher gfisher@shire.com +1 781 482 9649


Investor Relations    
Christoph Brackmann christoph.brackmann@shire.com  +41 79 543 3259
Robert Coates rcoates@shire.com  +44 203 549 0874
Sun Kim sun.kim@shire.com +1 617 588 8175

NOTES TO EDITORS

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.

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, projected revenues, 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 depends on third parties to supply certain inputs and services critical to its operations including certain inputs, services and ingredients critical to its manufacturing processes. 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;
  • the nature of producing plasma-based therapies 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;
  • failure to comply with laws and regulations governing the sales and marketing of its products could materially impact Shire's revenues and profitability;
  • Shire's products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
  • Shire's patented products are subject to significant 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 Shire's revenues, financial condition or results of operations;
  • Shire may fail to obtain, maintain, enforce or defend the intellectual property rights required to conduct its business;
  • Shire faces intense competition for highly qualified personnel from other companies and organizations;
  • failure to successfully execute or attain strategic objectives from Shire's acquisitions and growth strategy may adversely affect the 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, could have negative consequences for Shire's business and increase the risk of non-payment by Shire's customers;
  • changes in foreign currency exchange rates and interest rates could have a material adverse effect on Shire's operating results and liquidity;
  • Shire is subject to evolving and complex tax laws, which may result in additional liabilities that may adversely affect the Shire's financial condition or results of operations;
  • if a marketed product fails to work effectively or causes adverse side effects, this could result in damage to Shire's reputation, the withdrawal of the product and legal action against Shire;
  • 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 faces risks relating to the expected exit of the United Kingdom from the European Union;
  • Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which has increased its borrowing costs and may decrease its business flexibility;
  • Shire's ongoing strategic review of its Neuroscience franchise may distract management and employees and may not lead to improved operating performance or financial results; there can be no guarantee that, once completed, Shire's strategic review will result in any additional strategic changes beyond those that have already been announced; 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 "ITEM1A: Risk Factors", and in Shire's 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.




[1] Cicardi M, Bork K, Caballero T, et al, on behalf of HAWK (Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012; 67(2):147-157.

[2] Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.

[3] Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

[4] Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.

[5] Blaese RM, Bonilla FA, Stiehm ER, Younger ME, eds. Patient & Family Handbook for Primary Immunodeficiency Diseases. 5th ed. Towson, MD: Immune Deficiency Foundation; 2015.

[6] Bousfiha AA et al. Primary immunodeficiency diseases worldwide: more common than generally thought. J Clin Immunol. 2013 Jan;33(1):1-7.

[7] Aygören-Pürsün et al. Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe. Orphanet Journal of Rare Diseases. 2014, 9:99