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Shire Announces New HELP Study(TM) Data for Lanadelumab in Hereditary Angioedema (HAE) at 2018 ACAAI Annual Scientific Meeting


  • Data shows lanadelumab provides significant, clinically meaningful reduction of HAE attacks throughout the entire 26-week study duration [Oral Abstract #A152]
  • Interim results from open-label extension study further the understanding of safety and efficacy of continued treatment with lanadelumab in HAE [Poster #P168]

Dublin, Ireland. – 16 November 2018 Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, today announced additional data from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™, evaluating the efficacy and safety of subcutaneously administered lanadelumab in HAE, will be presented at the American College of Allergy, Asthma & Immunology (ACAAI) 2018 Annual Scientific Meeting. HAE is a rare, genetic and potentially life-threatening disorder that can result in recurrent attacks of oedema (swelling) in various parts of the body.

Data showed that patients treated with lanadelumab 300 mg every two weeks experienced significantly fewer HAE attacks, were less likely to have moderate or severe attacks or use rescue medication (primary and secondary endpoints), and were more likely to be HAE attack-free than those treated with placebo. These results were noted during the entire 26-week treatment period and, according to a post hoc sensitivity analysis, were greatest for patients during the 16-week steady state period (days 70-182).

Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire said, “For those living with this chronic and unpredictable disease, it is important that they can reduce the frequency of their HAE attacks. Additional analysis of the HELP Study continues to support the use of lanadelumab as a preventive subcutaneous treatment option for HAE in appropriate patients 12 years of age and older.”

Interim results from the HELP Study open-label extension found treatment with lanadelumab was generally well-tolerated and consistent with the previously observed safety profile. At the time of interim analysis, patients had been exposed to lanadelumab for a mean (SD) of 8.21 (2.17) months and continued to experience a reduction in HAE attacks.

“The data we have seen from the HELP open-label extension are very promising and we look forward to receiving the final results,” said Dr. Busch.

The full list of HELP and HELP open-label extension study abstracts being presented are as follows:

  • Lanadelumab 300mg Every 2 Weeks Effectively Prevented Hereditary Angioedema Attacks in the HELP Study
    • Oral Abstract #A152
    • Sunday 18 November; 1:00 pm
  • Lanadelumab Reduces HAE Attack Rate: Interim Findings from the HELP Open-Label Extension Study
    • Abstract #P168
    • Sunday 18 November; 12:10 pm
  • Lanadelumab Exposure During Steady State: Achievement of Effective Concentrations in Patients in the HELP Study
    • Abstract #P170
    • Sunday 18 November; 12:30 pm
  • Lanadelumab Safety and Immunogenicity: Results from the Phase 3 HELP Study
    • Abstract #P166
    • Sunday 18 November; 09:40 am
  • Lanadelumab Efficacy After Switching from Placebo: Results from the HELP and HELP Open-Label Extension Studies
    • Abstract #P171
    • Sunday 18 November; 12:40 pm
  • Subcutaneous Self-Administration of Lanadelumab for Prophylactic Treatment in Patients with Hereditary Angioedema (HAE)
    • Abstract #P352
    • Saturday 17 November; 11:40 am

About the HELP Study™
The HELP Study™ was a multicentre, randomised, double-blind, placebo-controlled parallel group trial that evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo over 26 weeks in 125 patients 12 years of age or older with HAE.

The primary endpoint of the HELP Study™ was the number of investigator-confirmed HAE attacks over the entire 26-week study duration. Lanadelumab demonstrated that subcutaneous injections every two or four weeks reduced the mean monthly number of attacks across all three lanadelumab treatment arms studied: 300 mg every two weeks, 300 mg every four weeks, and 150 mg of lanadelumab every four weeks.

No serious Treatment Emerging Adverse Events (TEAEs) or deaths were reported. The most common TEAE was HAE attack. The most commonly reported adverse events, excluding events related to HAE attacks, were injection site pain (42.9%), followed by upper respiratory infection (23.8%), headache (20.2%) and injection site erythema (9.5%). The majority of TEAEs were mild to moderate in severity. TEAEs resulted in discontinuation for 1 patient from the 300 mg every four weeks lanadelumab arm (ALT/AST elevation) and 2 from the placebo arm (tension headache, HAE attack).

About the HELP Study™ Extension
The long-term safety and efficacy of lanadelumab for the prevention of HAE attacks continues to be evaluated in a 132-week open-label extension study.

In the extension study, 212 adolescent and adult patients received at least one dose of lanadelumab. Of these, 109 patients are rollover participants from the HELP Study™ and 103 are new (non-rollover) participants who had a confirmed diagnosis of HAE and a historical baseline attack rate of >1 attack per 12 weeks.

About Lanadelumab
Lanadelumab is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity. Lanadelumab is produced in Chinese Hamster Ovary (CHO) cells by recombinant DNA technology. Lanadelumab is formulated for subcutaneous administration and has a half-life of approximately two weeks in patients with HAE.

Lanadelumab was approved under the brand name TAKHZYRO™ in the U.S. on 23 August 2018 and Canada on 19 September 2018 to prevent attacks of HAE in patients 12 and older, and additional regulatory submissions are ongoing worldwide. For full U.S. Prescribing Information, including the approved indication and important safety information, please visit https://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf. For full Canada Prescribing Information within Canadian Product Monograph, please visit https://www.shirecanada.com/-/media/shire/shireglobal/shirecanada/pdffiles/product%20information/takhzyro-pm-en.pdf.

About Hereditary Angioedema
HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide. The condition results in recurring attacks of oedema (swelling) in various parts of the body that can be debilitating and painful. Laryngeal attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation. Aside from the burden of the disease itself, patients can suffer anxiety, fatigue and depression between attacks.

Shire’s Commitment to Hereditary Angioedema
Shire is a dedicated, long-term partner to the HAE community with nearly a decade of experience supporting patients. We believe people living with HAE deserve a right-fit approach to treatment and are committed to serial innovation. Our existing portfolio of products includes a number of therapy options to help meet the needs of those living with the disease. Beyond our focus on developing novel treatments, we provide specialised services and support offerings tailored to the HAE community. Learn more at shire.com.

For further information please contact:

Investor Relations

Christoph Brackmannchristoph.brackmann@shire.com+41 41 288 41 29
Sun Kimsun.kim@shire.com+1 617 588 8175
Scott Burrowsscott.burrows@shire.com+41 41 288 4195
Media
Katie Joycekjoyce@shire.com+1 781 482 2779
Emily Buntingemily.bunting@shire.com+41 79 866 9703

NOTES TO EDITORS

About Shire

Shire is the global biotechnology leader serving patients with rare diseases and specialized conditions. We seek to push boundaries through discovering and delivering new possibilities for patient communities who often have few or no other champions. Relentlessly on the edge of what’s next, we are serial innovators with a diverse pipeline offering fresh thinking and new hope. Serving patients and partnering with healthcare communities in over 100 countries, we strive to be part of the entire patient journey to enable earlier diagnosis, raise standards of care, accelerate access to treatment, and support patients. Our diverse portfolio of therapeutic areas includes Immunology, Hematology, Genetic Diseases, Neuroscience, Internal Medicine and Ophthalmics.

Championing patients is our call to action - it brings the opportunity - and responsibility - to change people’s lives.

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 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 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 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;
  • the potential uncertainty among our employees, customers, suppliers, and other business partners resulting from the announcement by Takeda Pharmaceutical Company Limited on May 8, 2018 of a recommended offer for Shire under the UK Takeover Code; 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.

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