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2019-11-14

AstraZeneca: US FDA accepts regulatory submission for selumetinib in neurofibromatosis type 1 and grants Priority Review

AstraZeneca and MSD's selumetinib would become the first medicine
indicated for the treatment of paediatric patients with NF1 plexiform
neurofibromas if approved

AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck &
Co., Inc. inside the US and Canada) today announced that the US Food
and Drug Administration (FDA) has accepted a New Drug Application
(NDA) and granted Priority Review for selumetinib as a potential new
medicine for paediatric patients aged three years and older with
neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform
neurofibromas (PNs).

This is the first acceptance of a regulatory submission for an oral
monotherapy for the treatment of NF1, a rare and incurable genetic
condition. A Prescription Drug User Fee Act (PDUFA) date is set for
the second quarter of 2020.

The regulatory submission was based on positive results from the
National Cancer Institute (NCI) Cancer Therapy Evaluation Program
(CTEP)-sponsored SPRINT Phase II Stratum 1 trial. An Objective
Response Rate (ORR) was achieved in 66% of paediatric patients with
NF1 and symptomatic, inoperable PNs (n=33/50 patients) when treated
with selumetinib as a twice-daily oral monotherapy. ORR was defined
as the percentage of patients with a confirmed complete or partial
response of ? 20% tumour volume reduction.

Selumetinib, a MEK 1/2 inhibitor was granted US FDA Breakthrough
Therapy Designation
(https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2...)
in April 2019, Orphan Drug Designation in February 2018, EU Orphan
Designation
(https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2...)
in August 2018 and Swissmedic Orphan Drug Status in December 2018.
AstraZeneca and MSD are jointly developing and commercialising
selumetinib globally under a license agreement.

About SPRINT

SPRINT is a US NCI CTEP-sponsored Phase I/II trial. The Phase I trial
was designed to identify the optimal Phase II dosing regimen, and the
results were published in The New England Journal of Medicine
(https://www.nejm.org/doi/full/10.1056/NEJMoa1605943).[1]

About selumetinib
Selumetinib is a MEK 1/2 inhibitor. It is designed to inhibit the MEK
enzyme in the RAS/MAPK pathway, a cell-signalling pathway, associated
with cancer cell growth and proliferation in a number of different
tumour types.

About NF1

NF1 is an incurable genetic condition that affects one in every 3,000
to 4,000 individuals.[2,3] It is caused by a spontaneous or inherited
mutation in the NF1 gene and is associated with many symptoms,
including soft lumps on and under the skin (cutaneous neurofibromas),
skin pigmentation (so-called `cafe au lait' spots) and, in 30-50% of
patients, tumours develop on the nerve sheaths (plexiform
neurofibromas).[1] These plexiform neurofibromas can cause clinical
issues such as pain, motor dysfunction, airway dysfunction,
bowel/bladder dysfunction and disfigurement as well as having the
potential to transform into malignant peripheral nerve sheath tumours
(MPNST).[1]

People with NF1 may experience a number of complications such as
learning difficulties, visual impairment, twisting and curvature of
the spine, high blood pressure, and epilepsy. NF1 also increases a
person's risk of developing other cancers, including malignant brain
tumours, MPNST and leukaemia. Symptoms begin during early childhood,
with varying degrees of severity, and can reduce life expectancy by
up to 15 years.[4]

About the AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US,
known as MSD outside the United States and Canada, announced a global
strategic oncology collaboration to co-develop and co-commercialise
Lynparza, the world's first PARP inhibitor, and potential new
medicine selumetinib, a MEK inhibitor, for multiple cancer types.
Working together, the companies will develop Lynparza and selumetinib
in combination with other potential new medicines and as
monotherapies. Independently, the companies will develop Lynparza and
selumetinib in combination with their respective PD-L1 and PD-1
medicines.

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 six
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 Oncology as one of AstraZeneca's four 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, 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.

About AstraZeneca

AstraZeneca 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
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 (CV, +44 203 749 5711
Gruvris Metabolism)
Nick Stone BioPharmaceuticals +44 203 749 5716
(Renal), ESG
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. Dombi E, et al. Activity of Selumetinib in Neurofibromatosis Type
1-Related Plexiform Neurofibromas. N Engl J Med. 2016;375:2550-2560.

2. National Institute of Neurological Disorders and Stroke.
Neurofibromatosis Fact Sheet. "What is NF1?" Available at:
https://www.ninds.nih.gov/disorders/patient-caregiver-education/fact-she...
#3162_2 Accessed October 2019.

3. ASCO Cancer.Net. Neurofibromatosis Type 1. Available at
https://www.cancer.net/cancer-types/neurofibromatosis-type-1 Accessed
June 2019.

4. Evans DGR, et al. Reduced Life Expectancy Seen in Hereditary
Diseases Which Predispose to Early-Onset Tumors. Appl Clin Genet.
2013;6:53-61.

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