From www.shire.com February 23, 2017 News, Press Release

New England Journal of Medicine Publishes Phase 1b Results for Shire’s Investigational Treatment for Hereditary Angioedema, a Rare Genetic Disease

Cambridge, Mass. – February 23, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare diseases, today announced the publication of results from the Phase 1b study of lanadelumab (SHP643; formerly DX-2930) in the February 23, 2017 issue of the New England Journal of Medicine (NEJM).

Lanadelumab is a subcutaneously administered, human monoclonal antibody that specifically binds and inhibits plasma kallikrein, and it is being investigated for the prevention of angioedema attacks in patients with hereditary angioedema (HAE). HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.1 The condition results in recurrent, localized edema (swelling). The areas of the body most commonly affected are the extremities, gastrointestinal tract, and upper airways.2,3

“In this Phase 1b study, no serious adverse events or discontinuations due to adverse events were observed at all doses studied. Pre-specified efficacy analyses in patients with at least 2 attacks in the 3 months prior to enrolment demonstrated that from day 8 to day 50, the administration of two doses of lanadelumab (300 or 400 mg) 14 days apart, reduced the rate of attacks by 100% and 88% respectively, when compared with placebo. In addition, all subjects were attack-free in the 300 mg group and 82% were attack-free in the 400 mg group, compared to 27% in the placebo group,” said Dr. Aleena Banerji, Associate Professor, Massachusetts General Hospital, Boston.

“The overall results of this study are encouraging; it should be noted that while the duration of treatment was relatively short and only a small number of patients were investigated, the results supported further Phase 3 investigations, which are currently ongoing,” added Dr. Paula Busse, Associate Professor, Mount Sinai Hospital, New York.

“Despite improvements in the management of HAE in recent years, there is still a need for long-acting prophylactic treatment options. At Shire we are proud of our history in HAE and ongoing commitment to the clinical development of lanadelumab, an investigational prophylactic therapy for this rare genetic disease,” said Philip J. Vickers, Ph.D., Global Head of Research and Development at Shire.

The complete publication can be found at nejm.org.4 The main results were previously presented at the American College of Allergy, Asthma, and Immunology (ACAAI) Annual Scientific Meeting in 2015.

A pivotal Phase 3 trial evaluating the safety and efficacy of lanadelumab as a long-acting prophylactic treatment for HAE is currently underway.

With the clinical development of lanadelumab, Shire is building on its legacy in HAE and as the world leader in rare diseases.

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