Blueprint
FORM 6-K
SECURITIES
AND EXCHANGE COMMISSION
Washington,
D.C. 20549
Report
of Foreign Issuer
Pursuant
to Rule 13a-16 or 15d-16 of
the
Securities Exchange Act of 1934
For the
month of April
2019
Commission
File Number: 001-11960
AstraZeneca PLC
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Selumetinib gets
Breakthrough Therapy Designation
1 April 2019 07:00 BST
Selumetinib granted US Breakthrough Therapy
Designation in neurofibromatosis type 1
Designation based on Phase II SPRINT trial in
paediatric patients with NF1 plexiform neurofibromas
Selumetinib is a MEK 1/2 Inhibitor being co-developed by
AstraZeneca and MSD
AstraZeneca and MSD, Inc., Kenilworth, NJ, US (MSD: known as Merck
& Co., Inc. inside the US and Canada) today announced that the
US Food and Drug Administration (FDA) has granted Breakthrough
Therapy Designation (BTD) for the MEK 1/2 inhibitor and potential
new medicine selumetinib.
This designation is for the treatment of paediatric
patients aged three years and older with
neurofibromatosis type 1 (NF1) symptomatic and/or progressive,
inoperable plexiform neurofibromas (PN), a rare, incurable genetic
condition.
José Baselga, Executive Vice President, Research and
Development, Oncology, said: "Selumetinib shows promise in the
treatment of NF1-related plexiform neurofibromas, a rare and
debilitating disease with no approved medications to date. The
Breakthrough Therapy Designation acknowledges the significant unmet
need of these patients and the potential benefit of selumetinib in
this setting."
Roy Baynes, Senior Vice President and Head of Global Clinical
Development, Chief Medical Officer, at MSD Research Laboratories,
said: "This new designation validates our ongoing development of
selumetinib. As a result of this, selumetinib has the
potential to receive expedited regulatory review and we hope to
bring this medicine to patients as soon as possible."
The BTD is based on Phase II data from the SPRINT trial, testing
selumetinib as an oral monotherapy in paediatric patients, aged
three years or older with inoperable NF1-related
PN. The results of the
trial were presented by
the National Cancer Institute (NCI) at the 2018 American Society of
Clinical Oncology Annual Meeting.
This is the ninth BTD that AstraZeneca has received from the FDA
since 2014. BTD is designed to expedite the development and
regulatory review of medicines that are intended to treat a serious
condition and that have shown encouraging early clinical results,
which may demonstrate substantial improvement on a
clinically-significant endpoint over available
medicines.
Selumetinib was granted Orphan Drug Designation for the treatment
of NF1 by the US FDA in February 2018 and the European Medicines
Agency in August 2018.
Selumetinib is a MEK 1/2 inhibitor and potential new medicine
licensed by AstraZeneca from Array BioPharma Inc. in 2003.
AstraZeneca and MSD entered a co-development and
co-commercialisation agreement for selumetinib in
2017.
The NF1 gene provides instructions for making a
protein called neurofibromin, which negatively regulates the
RAS/MAPK pathway, helping to control cell growth, differentiation
and survival. Mutations in the NF1gene may result in dysregulations in
RAS/RAF/MEK/ERK signalling, which can cause cells to grow, divide
and copy themselves in an uncontrolled manner, and may result in
tumour growth. Selumetinib inhibits the MEK enzyme in this pathway,
potentially leading to inhibition of tumour
growth.
Selumetinib is being assessed as a monotherapy and in combination
with other treatments in ongoing trials.
About SPRINT
The SPRINT trial is a US Cancer Therapy Evaluation Program (CTEP)
NCI-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.1
About NF1
Neurofibromatosis type 1 (NF1) is an incurable genetic condition
that affects one in 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
20-50% of patients, tumours develop on the nerve sheaths (plexiform
neurofibromas). 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).
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 (olaparib), 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 New Oncology as one of AstraZeneca's five
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 &
Metabolism and Respiratory. AstraZeneca operates in over 100
countries and its innovative medicines are used by millions of
patients worldwide. For more information, please
visit astrazeneca.com and
follow us on Twitter @AstraZeneca.
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Adrian Kemp
Company Secretary
AstraZeneca PLC
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 NHS Choices. Neurofibromatosis Type 1. Available
at https://www.nhs.uk/conditions/neurofibromatosis-type-1/.
Accessed March 2019.
3 Johnson KJ, et al. Development of an International Internet-based
Neurofibromatosis Type 1 Patient Registry. Contemporary Clinical
Trials.
2013;34:305-311.
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
SIGNATURES
Pursuant
to the requirements of the Securities Exchange Act of 1934, the
Registrant has duly caused this report to be signed on its behalf
by the undersigned, thereunto duly authorized.
Date:
01 April
2019
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By: /s/
Adrian Kemp
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Name:
Adrian Kemp
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Title:
Company Secretary
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