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2020-01-20

AstraZeneca: Imfinzi and tremelimumab granted Orphan Drug Designation in the US for liver cancer

AstraZeneca's Imfinzi (durvalumab) and tremelimumab, an anti-CTLA4
antibody and potential new medicine, have both been granted Orphan
Drug Designation (ODD) in the US for the treatment of hepatocellular
carcinoma (HCC), the most common type of liver cancer.

The US Food and Drug Administration (FDA) grants ODD to medicines
intended for the treatment, diagnosis or prevention of rare diseases
or disorders that affect fewer than 200,000 people in the US.

Liver cancer is the third leading cause of cancer death worldwide and
for patients with unresectable or advanced disease, only 13% are
alive five years after diagnosis.[1-3]

José Baselga, Executive Vice President, Oncology R&D, said: "Many
patients with liver cancer are diagnosed and treated only after the
disease is advanced, and there is an urgent need for new effective
and tolerable treatments. We are eager to bring new potential options
to these patients and look forward to the results of our ongoing
Phase III HIMALAYA trial later this year."

The Phase III HIMALAYA trial is testing Imfinzi and the combination of
Imfinzi plus tremelimumab in patients with unresectable, advanced HCC
who have not been treated with prior systemic therapy and are not
eligible for locoregional therapy (treatment localised to the liver).
HIMALAYA is the first trial to test dual immune checkpoint blockade
in the 1st-line advanced HCC setting.

Imfinzi is not currently approved to treat HCC in any country, alone
or in combination with tremelimumab.

Hepatocellular carcinoma (HCC)

Liver cancer is the third leading cause of cancer death and the sixth
most commonly diagnosed cancer worldwide.[1] HCC represents about 80%
of all primary liver cancers.[4] Approximately 700,000 people were
diagnosed with HCC around the world in 2018, and an estimated 42,000
people were diagnosed in the US last year.[1,2] Between 80-90% of all
patients with HCC also have chronic liver disease, which is primarily
caused by infection with the hepatitis B or C viruses.[5,6] Chronic
liver disease is associated with inflammation that, over time,
results in immunosuppression and can lead to the development of
HCC.[7,8] The unique immune environment of liver cancer provides
clear rationale for researching medicines that harness the power of
the immune system to treat HCC.[9] A critical unmet need exists for
patients with HCC who face limited treatment options.[10] More than
half of patients are diagnosed at advanced stages of the disease,
often when symptoms first appear.[11,12]

HIMALAYA

HIMALAYA is a randomised, open-label, multicentre, global Phase III
trial of Imfinzi monotherapy and the combination of Imfinzi and
tremelimumab vs. the standard-of-care medicine sorafenib, a
multi-kinase inhibitor, in patients with unresectable, advanced HCC
who have not been treated with prior systemic therapy and are not
eligible for locoregional therapy. The trial is being conducted in
189 centres across 16 countries including in the US, Canada, Europe,
South America and Asia. The primary endpoint is overall survival and
key secondary endpoints include objective response rate and
progression-free survival.

Imfinzi

Imfinzi (durvalumab) is a human monoclonal antibody that binds to
PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80,
countering the tumour's immune-evading tactics and releasing the
inhibition of immune responses.

Imfinzi is approved in the curative-intent setting of unresectable,
Stage III non-small cell lung cancer (NSCLC) after chemoradiation
therapy in 54 countries, including the US, Japan, China and across
the EU, based on the Phase III PACIFIC trial. Imfinzi is also
approved for previously treated patients with advanced bladder cancer
in 11 countries, including the US.

Imfinzi is under Priority Review with FDA for the treatment of
patients with previously untreated extensive-stage small cell lung
cancer (SCLC) in combination with chemotherapy. A Prescription Drug
User Fee Act date is set for the first quarter of 2020.

As part of a broad development programme, Imfinzi is also being tested
as a monotherapy and in combination with tremelimumab, an anti-CTLA4
monoclonal antibody and potential new medicine, as a treatment for
patients with NSCLC, SCLC, bladder cancer, head and neck cancer,
liver cancer, biliary tract cancer, cervical cancer and other solid
tumours.

Tremelimumab

Tremelimumab is a human monoclonal antibody and potential new medicine
that targets the activity of cytotoxic T-lymphocyte-associated
protein 4 (CTLA-4). Tremelimumab blocks the activity of CTLA-4,
contributing to T cell activation, priming the immune response to
cancer and fostering cancer cell death. Tremelimumab is being tested
in a clinical trial programme in combination with Imfinzi in NSCLC,
SCLC, bladder cancer, head and neck cancer and liver cancer.

AstraZeneca's approach to Immuno-Oncology (IO)

Immuno-oncology (IO) is a therapeutic approach designed to stimulate
the body's immune system to attack tumours. The Company's IO
portfolio is anchored by immunotherapies that have been designed to
overcome anti-tumour immune suppression. AstraZeneca believes that
IO-based therapies offer the potential for life-changing cancer
treatments for the clear majority of patients.

The Company is pursuing a comprehensive clinical-trial programme that
includes Imfinzi as a monotherapy and in combination with
tremelimumab in multiple tumour types, stages of disease, and lines
of therapy, using the PD-L1 biomarker as a decision-making tool to
define the best potential treatment path for a patient. In addition,
the ability to combine the IO portfolio with radiation, chemotherapy,
small targeted molecules from across AstraZeneca's Oncology pipeline,
and from research partners, may provide new treatment options across
a broad range of tumours.

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 six
new medicines to be launched between 2014 and 2020, and a broad
pipeline of small molecules and biologics in development, the Company
is committed to advance oncology as a key growth driver for
AstraZeneca focused on lung, ovarian, breast and blood cancers. In
addition to AstraZeneca's main capabilities, the Company is actively
pursuing innovative partnerships and investments that accelerate the
delivery of our strategy, as illustrated by the investment in Acerta
Pharma in haematology.

By harnessing the power of four scientific platforms -
Immuno-Oncology, Tumour Drivers 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.

AstraZeneca

AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led
biopharmaceutical company that focuses on the discovery, development
and commercialisation of prescription medicines, primarily for the
treatment of diseases in three therapy areas - Oncology,
Cardiovascular, Renal and Metabolism, and Respiratory. AstraZeneca
operates in over 100 countries and its innovative medicines are used
by millions of patients worldwide. Please visit astrazeneca.com
(http://www.astrazeneca.com/) and follow the Company on Twitter
@AstraZeneca (https://twitter.com/AstraZeneca).

Media Relations
Gonzalo Viña +44 203 749 5916
Rob Skelding Oncology +44 203 749 5821
Rebecca Einhorn Oncology +1 301 518 4122
Matt Kent BioPharmaceuticals +44 203 749 5906
Angela Fiorin BioPharmaceuticals +44 1223 344 690
Jennifer Hursit Other +44 203 749 5762
Christina Sweden +46 8 552 53 106
Malmberg
Hägerstrand
Michele Meixell US +1 302 885 2677

Investor
Relations
Thomas Kudsk +44 203 749 5712
Larsen
Henry Wheeler Oncology +44 203 749 5797
Christer BioPharmaceuticals +44 203 749 5711
Gruvris (Cardiovascular,
Metabolism)
Nick Stone BioPharmaceuticals +44 203 749 5716
(Renal) Environmental,
Social and Governance
Josie Afolabi BioPharmaceuticals +44 203 749 5631
(Respiratory) Other
medicines
Craig Marks Finance Fixed income +44 7881 615 764
Jennifer Corporate access Retail +44 203 749 5824
Kretzmann investors
US toll-free +1 866 381 72 77

References

1. World Health Organization. IARC Globocan 2018 World Fact Sheet.
Available at
http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-shee....
Accessed January 2020.

2. National Cancer Institute. Cancer Stat Facts: Liver and
Intrahepatic Bile Duct Cancer. Available at:
https://seer.cancer.gov/statfacts/html/livibd.html. Accessed January
2020
(https://seer.cancer.gov/statfacts/html/livibd.html.%20Accessed%20January...).

3. Vogel A, et al. Hepatocellular carcinoma: ESMO Clinical Practice
Guidelines for diagnosis, treatment and follow-up. Annals of Oncology
29 (Supplement 4): iv238-iv255, 2018. doi:10.1093/annonc/mdy308.

4. ASCO Cancer.net. ASCO Answers Liver Cancer. Available at
https://www.cancer.net/sites/cancer.net/files/asco_answers_liver.pdf.
Accessed January 2020.

5. Dos Santos P, et al. Incidence of hepatocellular carcinoma in
patients with chronic liver disease due to hepatitis B or C and
coinfected with the human immunodeficiency virus: a retrospective
cohort study. World J Gastroenterol. 2018 February 7; 24(5): 613-622.
DOI: 10.3748/wjg.v24.i5.613.

6. Hiotis SP, et al. Hepatitis B vs. hepatitis C infection on viral
hepatitis-associated hepatocellular carcinoma. BMC Gastroenterol 12,
64 (2012) doi:10.1186/1471-230X-12-64

7. Del Campo JA., et al. Role of inflammatory response in liver
diseases: Therapeutic strategies. World journal of hepatology. 2018;
10(1), 1-7. doi:10.4254/wjh.v10.i1.1

8. Makarova-Rusher OV, et al. The yin and yang of evasion and immune
activation in HCC. J Hepatol. 2015; 62 (6): 1420-1429.

9. Han Y, et al. Human CD141CTLA-41Regulatory Dendritic Cells
Suppress T-Cell Re...

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