Shire's Investigational Treatment Lanadelumab Reduces Hereditary Angioedema Monthly Attack Rate by 87% Versus Placebo in Phase 3 26-week Pivotal Trial

Lexington, Massachusetts (ots/PRNewswire) -

- Significant reduction in monthly Hereditary Angioedema (HAE) attack
rate with infrequent subcutaneous administration
- Data to serve as basis for U.S. BLA filing expected in Q4 2017 to
Q1 2018
- Shire to host Investor Call today at 10 a.m. ET; details below

Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare
diseases, announces positive topline Phase 3 results for the HELP(TM)
study, a global, multi-center, randomized, double-blind
placebo-controlled parallel group trial that evaluated the efficacy
and safety of subcutaneously administered lanadelumab versus placebo
over 26 weeks in patients 12 years of age or older with Hereditary
Angioedema (HAE). Lanadelumab is an investigational treatment being
evaluated for the prevention of angioedema attacks in patients with
HAE, a rare genetic disease characterized by recurrent swelling of
extremities, gastrointestinal tract, and upper airways.

This study met its primary endpoint and all secondary endpoints
with highly statistically significant and clinically meaningful
results for all three lanadelumab treatment arms compared to placebo.
The 300 mg dose administered once every two weeks resulted in a
statistically significant reduction in mean HAE attack frequency of
87% compared to placebo (p <0.001). Results were consistent
regardless of baseline attack rate. Notably for each of the three
lanadelumab regimens studied, whether administered biweekly or
monthly, a significantly higher proportion of patients-compared to
placebo-were attack free throughout the entire 26 week study period.

This study was representative of the full HAE disease spectrum.
Overall, 52% of patients experienced three or more attacks per month
at baseline, 65% of patients reported a history of laryngeal attacks
and 56% were on long-term prophylaxis (LTP). Ninety percent of
patients completed the study. Ninety-six percent of those who
completed the study chose to roll-over into the ongoing long-term
safety study (HELP(TM) Study Extension).

"In the U.S., available treatment options include either
injections for acute attacks or short-acting intravenous infusions
administered twice a week," said Aleena Banerji, M.D., Massachusetts
General Hospital, Boston, MA and clinical trial investigator. "If
approved, lanadelumab may offer patients a long-acting treatment
option that significantly reduces HAE attacks when administered
subcutaneously as infrequently as every four weeks."

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
recurrent, localized edema (swelling). The areas of the body most
commonly affected are the extremities, gastrointestinal tract, and
upper airways. The swelling can be debilitating and painful,
potentially impacting both work and education for people living with
HAE. Swelling of the throat can be life-threatening due to
asphyxiation.

"The possibility of a new way to address the underlying cause of
HAE to prevent attacks could transform how we treat the disease in
the future," said Professor Marcus Maurer, M.D., Charité
-Universitätsmedizin Berlin, Germany and clinical trial investigator.
"Patients with HAE want to live independently and without fear of an
angioedema attack."

Lanadelumab was generally well tolerated over the 26-week
treatment period. No treatment-related serious adverse events or
deaths were reported. The most common adverse event was injection
site pain (29.3% placebo vs. 42.9 % combined lanadelumab arms).

"We are extremely encouraged by these topline Phase 3 results,"
said Flemming Ornskov, M.D., M.P.H., Shire Chief Executive Officer,
"We have nearly a decade of experience and a strong portfolio and
pipeline in HAE and believe these data demonstrate high potential for
transforming the way patients living with this condition are
treated."

Shire plans to submit a biologics license application (BLA) for
evaluation by the U.S. Food and Drug Administration (FDA) by late
2017 or early 2018. Lanadelumab has received both Orphan Drug
Designation and Breakthrough Therapy Designation from the FDA and
Orphan Drug Designation from the European Medicines Agency (EMA).

About the HELP(TM) Study

The global, multicenter, randomized, parallel group, double-blind,
placebo-controlled, Phase 3 trial is the largest prevention study
conducted to date treating 125 patients 12 years of age or older with
type I/II HAE. Patients were randomized into four arms to receive
repeated subcutaneous administrations of lanadelumab 300 mg every two
weeks, 300 mg every four weeks, 150 mg every four weeks or placebo in
a 2:1 ratio. The volume of drug administered at each injection in the
clinical trial was 2 mL, administered subcutaneously as two 1 mL
separate injections in the upper arm to maintain the study blind.

The primary efficacy endpoint of the study was the number of
investigator-confirmed angioedema attacks observed in each
lanadelumab treatment arm versus placebo arm during the 26 week
treatment period.

Shire's Commitment to Hereditary Angioedema (HAE)

Shire is a dedicated, long-term partner to the HAE community with
nearly a decade of clinical and real-world experience supporting
patients. We believe each patient deserves a right-fit approach to
treatment, and our existing portfolio of products currently includes
three distinct therapy options. We are committed to serial innovation
and rely on our expertise to help fulfill unmet treatment needs for
patients with HAE. Beyond our focus on developing novel treatments,
we provide specialized services and support offerings that help meet
the needs of the HAE community. Learn more at shire.com.

About Lanadelumab

Lanadelumab is an investigational fully human monoclonal antibody
that specifically binds and inhibits plasma kallikrein and is being
developed as a treatment for the prevention of angioedema attacks in
patients with HAE. Lanadelumab is formulated for subcutaneous
administration with a half-life of approximately 14 days in patients
with HAE.

Live conference call for investors:

Shire's Management Team will host a conference call for investors
and analysts today, May 18, 2017 at 10 a.m., EDT.

The details of the conference call are as follows:

UK dial in: 0808 237 0030 or 020 3139 4830

US dial in: 1 866 928 7517 or 1 718 873 9077

International Access

Numbers: Click here (https://urldefense.proofpoint.com/v2/url?u=http-3A__events.arkadin.com_ev_docs_NE-5FFEL-5FEvents-5FInternational-5FAccess-5FList.pdf&d=DwMGaQ&c=5oszCido4egZ9x-32Pvn-g&r=79xCfmfp9fAsefgXbE_UNRPKJBr8J6aolJr26D5QtB8GSn75WkQ-vbSFke8lGxw2&m=I6X2Bfazj664lDpFWYUvDdLfkMUoqB4w2QdQHLykHBU&s=ATalWeCslTIbnC5kafvFCXKPrTu_nNNT9T0t1Z4UsNY&e=)

Password/Conf ID: 93795050#

Live Webcast: Click here (http://investors.shire.com/presentations-and-webcasts)

Replay:

A replay of the presentation will be available for two weeks by
phone and by webcast for three months. Replay information can be
found on the Investor Relations section of Shire's website at
http://investors.shire.com/.

NOTES TO EDITORS

Stephen Williams, Deputy Company Secretary is responsible for
arranging the release of this announcement.

Inside Information

This announcement contains inside information.

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

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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; 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 "ITEM 1A: 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.

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acting on our behalf are expressly qualified in their entirety by
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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.

ots Originaltext: Shire Pharmaceuticals Group Plc
Im Internet recherchierbar: http://www.presseportal.de

Contact:
please contact:

Media Relations

Elizabeth Kalina
ekalina@shire.com
+1-781-482-2713
Katie Joyce
kjoyce@shire.com
+1-781-482-2779

Investor Relations

Ian Karp
ikarp@shire.com
+1-781-482-9018
Robert Coates
rcoates@shire.com
+44-1256-894874

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