ACALABRUTINIB ORPHAN DRUG DESIGNATION RECOMMENDED

Acalabrutinib RecommendEd for Orphan Drug Designation IN EUROPE for THREE
indications

AstraZeneca and Acerta Pharma BV, a company in which AstraZeneca has a majority
equity investment, today announced that the European Medicines Agency (EMA)
Committee for Orphan Medicinal Products (COMP) adopted three positive opinions
recommending acalabrutinib (ACP-196) for designation as an orphan medicinal
product. The three positive opinions are for the treatment of chronic
lymphocytic leukaemia (CLL) / small lymphocytic lymphoma (SLL), mantle cell
lymphoma (MCL) and lymphoplasmacytic lymphoma (Waldenström's macroglobulinaemia,
MG).

Sean Bohen, Executive Vice-President of Global Medicines Development and Chief
Medical Officer at AstraZeneca, said: "Today's three positive opinions
recommending acalabrutinib for designation as an orphan medicinal product are
important milestones. They reinforce the strategic rationale for our investment
in Acerta, demonstrating clear progress in developing a potential best-in-class
medicine that could transform treatment for patients across a range of blood
cancers. The positive opinions underscore the continued need for the development
of new therapies in these serious and life-threatening conditions and support
our commitment to bring new medicines to patients as quickly as possible."

CLL is a slow-growing blood and bone marrow cancer that accounts for
approximately one in four cases of leukaemia.i,ii Most CLL patients experience
disease progression despite initial response to therapy and may require
additional treatment. iii SLL is a clinically similar disease localized to the
lymph nodes.iv

MCL is an aggressive non-Hodgkin's lymphoma (NHL) typically associated with very
poor outcomes.v MCL represents around 5% of all NHLs.vi The name comes from the
fact that the tumour cells originate in the mantle zone of the lymph node. vi

WG is a rare, slow-growing cancer predominantly affecting older individuals,
with a mean age of 60 at diagnosis.vii, viii and median survival from five to
nearly eleven years. vii, viii

The COMP adopts an opinion on Orphan Drug Designation, after which the opinion
is submitted to the European Commission (EC) for endorsement. Orphan Drug
Designation is a status assigned to a medicine intended for use in rare
diseases.ix To be granted orphan status by the EC, a medicine must be intended
for the treatment, prevention or diagnosis of a disease that is life threatening
and has a prevalence of up to five in 10,000 in the European Union.
Additionally, the intended medicine must aim to provide significant benefit to
those affected by the condition. Orphan status provides companies with
development and market exclusivity incentives for designated compounds and
medicines.
In addition to ongoing Phase II/III trials in CLL, MCL and WG, acalabrutinib is
currently being tested in Phase I/II trials in monotherapy as well as in
combination with immunotherapy or chemotherapies in a range of other blood
cancers and solid tumours.

i Chronic Lymphocytic Leukemia. Leukemia & Lymphoma Society Website.
http://www.lls.org/leukemia/chronic-lymphocytic-leukemia/  Accessed February 19,
2016.

ii What are the key statistics for chronic lymphocytic leukemia? American Cancer
Society Website. http://www.cancer.org/cancer/leukemia
-chroniclymphocyticcll/detailedguide/leukemia-chronic-lymphocytic-key-statistics
. Accessed February 19, 2015.

iii Veliz M, Pinilla-Ibarz J. Treatment of relapsed or refractory chronic
lymphocytic leukemia. Cancer Control. 2012; 19(1):37-53.

iv Chronic lymphocytic leukemia/Small lymphocytic lymphoma, National Cancer
Institute Website. http://www.cancer.gov/publications/dictionaries/cancer
-terms?cdrid=641290 Accessed February 19, 2016.

v Campo E and Rule S. Mantle cell lymphoma: evolving management strategies.
Blood. 2015 Jan 1;125(1):48-55.

vi Mantle Cell Lymphoma, Lymphoma.org Website.
http://www.lymphoma.org/site/pp.asp?c=bkLTKaOQLmK8E&b=6300157 Accessed February
19, 2016.

vi Lymphoplasmacytic lymphoma. National Cancer Institute. Surveillance,
Epidemiology, and End Results program.
http://seer.cancer.gov/seertools/hemelymph/51f6cf57e3e27c3994bd5363/ Accessed
February 19, 2016.

vii Dimopoulos MA, Kastritis E, Ghobrial IM. Waldenström's macroglobulinemia: a
clinical perspective in the era of novel therapeutics. Ann Oncol. 2016
Feb;27(2):233-40.

viii Oza and Rajkumar. Waldenstrom macroglobulinemia: prognosis and management.
Blood Cancer Journal (2015) 5, e394; doi:10.1038/bcj.2015.28

ix European Medicines Agency web site. "Orphan Designation."
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_con
t 
ent_000029.jsp&mid=WC0b01ac05800240ce. Accessed February 17, 2016.



About Acalabrutinib

Acalabrutinib is a highly selective, irreversible, second generation BTK
inhibitor, with approximately 1,000 patients treated to date in clinical studies
across the entire development programme. More than 600 patients have been
treated with acalabrutinib monotherapy. Phase I/II data showing a favourable
safety profile and efficacy in relapsed/refractory chronic lymphocytic leukaemia
patients was presented at the American Society of Haematology Annual Meeting &
Exposition in December 2015, with simultaneous publication in the New England
Journal of Medicine.

Potentially registrational studies in haematological malignancies are ongoing.
In addition, a head-to-head study versus ibrutinib in high risk chronic
lymphocytic leukaemia patients is currently ongoing.

Acalabrutinib is also currently being tested in multiple Phase I/II studies in
solid tumours, as monotherapy or in combination with immune checkpoint
inhibitors or other standard of care regimens.



About Acerta Pharma

Acerta is a leader in the field of covalent binding technology and is applying
this technology to create novel selective therapies intended for the treatment
of cancer and autoimmune diseases. Acerta's lead molecule, acalabrutinib (ACP
-196), is a selective and potent inhibitor of BTK. The company has operations in
Oss, the Netherlands and multiple US sites. The US headquarters is in Redwood
City, CA. AstraZeneca is the parent company of Acerta BV.



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 haematology.

By harnessing the power of four scientific platforms -- immuno-oncology, the
genetic drivers of cancer and resistance, DNA damage repair 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: www.astrazeneca.com

CONTACTS



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

ING - Infection, Neuroscience and Gastrointestinal

25 February 2016

-ENDS-