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AstraZeneca: Selumetinib granted Orphan Drug Designation in the US for adjuvant treatment of differentiated thyroid cancer

AstraZeneca today announced that the US Food and Drug Administration
(FDA) has granted Orphan Drug Designation for the investigational MEK
1/2 inhibitor, selumetinib (AZD6244, ARRY-142886) for adjuvant
treatment of patients with stage III or IV differentiated thyroid
cancer (DTC).

DTC is diagnosed in approximately 60,000 people in the US each
year,1and radioactive iodine (RAI) is recommended for those with
known/suspected metastases at diagnosis and those at high risk of
recurrence.2A small proportion of patients do not benefit from
currently available treatment with RAI because they do not express
sufficient sodium/iodine symporter (NIS) which is important for RAI
uptake into thyroid cells.3,4Selumetinib is being tested for its
ability to increase expression of NIS with the potential to add a
treatment option for patients who do not respond well to RAI.

Sean Bohen Executive Vice President, Global Medicines Development and
Chief Medical Officer, at AstraZeneca, said: "Uptake of RAI is
crucial for patients with thyroid cancer where no other therapies
have proven beneficial. Selumetinib could significantly enhance
currently available treatment options for these patients. The Orphan
Drug Designation is an important achievement as we advance our
development plans for this potential treatment in differentiated
thyroid cancer."

The Orphan Drug Designation programme provides orphan status to drugs
and biologics, which are defined as those intended for the safe and
effective treatment, diagnosis or prevention of rare diseases or
disorders that affect fewer than 200,000 people in the US.5

Selumetinib inhibits the MEK pathway in cancer cells to prevent tumour
growth. It is being tested in the Phase III ASTRA trial in patients
with DTC who are at high risk of recurrence.6In a Phase II study of
selumetinib in patients with advanced thyroid cancer, clinically
meaningful increases in iodine uptake and retention were seen in
patients with thyroid cancer that was refractory to RAI.7

In addition to DTC, selumetinib is being tested in SELECT-1, a Phase
III trial of patients with KRAS-mutant advanced non-small cell lung
cancer (NSCLC) and in a Phase II registration trial of paediatric and
adolescent patients with neurofibromatosis Type 1 in collaboration
with the US National Cancer Institute.

- ENDS -


About thyroid cancer

Cancer of the thyroid gland is diagnosed in approximately 60,000
people in the US each year.1Nearly 95% of patients have
differentiated tumours with an associated five-year survival of over
90%.2DTC is usually treated by surgery and thyroxine hormone
replacement therapy, and radioactive iodine treatment (RAI) is
recommended for patients with known/suspected metastases at diagnosis
and for those at high risk of recurrence.2Up to 30% of patients
experience recurrence of DTC after initial treatment.2

Approximately 5-15% of patients with DTC do not respond to RAI.3Ten
year survival in patients who fail to take up radioactive iodine into
tumour cells is 10% compared to nearly 60% in those with normal
uptake.8Traditional chemotherapy has minimal efficacy in patients
with metastatic DTC.2

About selumetinib

Selumetinib is an oral, potent, selective inhibitor of MEK, part of
the mitogen-activated protein kinase (MAPK) pathway which is
frequently activated in cancer, including those with the KRAS
mutation, which is present in 20% of human cancers and 20-30% of
non-small cell lung cancer tumours.

MAPK activation also inhibits expression of thyroid hormone
biosynthesis genes, including the sodium/iodine symporter (NIS) which
facilitates iodine uptake into cells.7 Pre-clinical studies have
suggested that following MAPK inhibition, iodine uptake by thyroid
tumour cells is regained.7

AstraZeneca acquired exclusive worldwide rights to selumetinib from
Array BioPharma Inc. in 2003.

About the ASTRA trial

ASTRA is a Phase III randomised, double blind study which is comparing
the complete remission rate following a 5-week course of selumetinib
or placebo and single dose adjuvant radioactive iodine therapy in
patients with differentiated thyroid cancer.5

About AstraZeneca in Oncology
AstraZeneca has a deep-rooted heritage in Oncology and offers a
quickly growing portfolio of new medicines that has the potential to
transform patients' lives and the Company's future. With at least 6
new medicines to be launched between 2014 and 2020 and a broad
pipeline of small molecules and biologics in development, we are
committed to advance New Oncology as one of AstraZeneca's six Growth
Platforms focused on lung, ovarian, breast and blood cancers. In
addition to our core capabilities, we actively pursue innovative
partnerships and investments that accelerate the delivery of our
strategy, as illustrated by our investment in Acerta Pharma in

By harnessing the power of four scientific platforms --
immuno-oncology, the genetic drivers of cancer and resistance, DNA
damage response and antibody drug conjugates -- and by championing
the development of personalised combinations, AstraZeneca has the
vision to redefine cancer treatment and one day eliminate cancer as a
cause of death.

About AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business
that focuses on the discovery, development and commercialisation of
prescription medicines, primarily for the treatment of diseases in
three main therapy areas - respiratory, inflammation, autoimmune
disease (RIA), cardiovascular and metabolic disease (CVMD) and
oncology - as well as in infection and neuroscience. AstraZeneca
operates in over 100 countries and its innovative medicines are used
by millions of patients worldwide. For more information please visit:


Media Enquiries
Neil Burrows UK/Global +44 7842 350541
Vanessa Rhodes UK/Global +44 7880 400690
Karen Birmingham UK/Global +44 7818 524012
Jacob Lund Sweden +46 8 553 260 20
Abigail Bozarth US +1 302 885 2677
Investor Enquiries
Thomas Kudsk Larsen +44 7818 524185
Eugenia Litz RIA +44 7884 735627
Nick Stone CVMD +44 7717 618834
Henry Wheeler Craig Marks OncologyFinance +44 7788 354619+44 7881 615764
Christer Gruvris Consensus Forecasts +44 7827 836825
Lindsey Trickett Oncology, ING +1 240 543 7970
Mitch Chan Oncology +1 240 477 3771
Dial / Toll-Free +1 866 381 7277

Key: RIA - Respiratory, Inflammation and Autoimmunity, CVMD -
Cardiovascular and Metabolic Disease,

ING - Infection, Neuroscience and Gastrointestinal

1American Cancer Society. Key statistics for thyroid cancer. Available
Accessed May 2016.

2 National Comprehensive Cancer Network Clinical Practice Guidelines
in Oncology. Thyroid Cancer. Version 2.2015

3 Worden F. Treatment strategies for radioactive iodine-refractory
differentiated thyroid cancer.Ther Adv Med Oncol. 2014

4 Lakshmanan A
et al. Modulation of sodium iodide symporter in thyroid cancer. Horm
Cancer. ( 2014

5 US Food and Drug Administration. Developing Products for Rare
Diseases & Conditions
Accessed May 2016.

6 National Institutes of Health. Study Comparing Complete Remission
After Treatment With Selumetinib/Placebo in Patient With
Differentiated Thyroid Cancer. (ASTRA) Accessed May 2016

7 Ho AL et al. Selumetinib-Enhanced Radioiodine Uptake in Advanced
Thyroid Cancer N Engl J Med. 2013 February 14; 368(7): 623-632.

8 Durante C et al. Long-term outcome of 444 patients with distant
metastases from papillary and follicular thyroid carcinoma: benefits
and limits of radioiodine therapy. J Clin Endocrinol Metab. 2006


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