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 May 2022
Commission File
Number: 001-11960
AstraZeneca
PLC
1
Francis Crick Avenue
Cambridge
Biomedical Campus
Cambridge CB2
0AA
United
Kingdom
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AstraZeneca
PLC
INDEX
TO EXHIBITS
1.
Ultomiris NMOSD Ph. III trial met primary endpoint
5
May 2022 07:00 BST
Ultomiris met primary endpoint in
CHAMPION-NMOSD Phase III trial in adults with neuromyelitis optica
spectrum disorder
Zero adjudicated relapses observed among Ultomiris patients over a
median treatment duration of 73 weeks
Positive high-level results from the open-label
Phase III CHAMPION-NMOSD trial showed that Ultomiris (ravulizumab-cwvz) achieved a statistically
significant and clinically meaningful reduction in the risk of
relapse in adults with anti-aquaporin-4 (AQP4)
antibody-positive (Ab+) neuromyelitis optica spectrum disorder
(NMOSD) compared to the external placebo arm from the
pivotal Soliris PREVENT clinical trial.
Ultomiris, the first and only long-acting C5 complement
inhibitor, met the primary endpoint of time to first on-trial
relapse, as confirmed by an independent adjudication committee.
Notably, no relapse was observed in 58 patients over a median
treatment duration of 73 weeks.
NMOSD is a rare and devastating autoimmune disease
that affects the central nervous system (CNS), including the spine
and optic nerves.1-3 Most
people living with NMOSD often experience unpredictable relapses, a
new onset of neurologic symptoms or worsening of existing
neurologic symptoms, also referred to as attacks, which tend to be
severe and recurrent and may result in permanent
disability.4-6
Sean J. Pittock, MD, Director of Mayo Clinic's
Center for Multiple Sclerosis and Autoimmune Neurology and of
Mayo's Neuroimmunology Laboratory and lead primary investigator in
the CHAMPION-NMOSD trial, said: "Every NMOSD relapse can have
debilitating and irreversible consequences, so reducing relapses is
critical. Patients on Ultomiris remained relapse free over a median
treatment duration of 73 weeks in the trial."
Marc Dunoyer, Chief Executive Officer, Alexion,
said: "Soliris established the role of complement
inhibition in preventing relapses in NMOSD, and
with Ultomiris, we continue to innovate for patients with a
more convenient every eight-week dosing schedule. These trial
results show that Ultomiris may help patients move towards
eliminating relapses, which is an important advancement in the
treatment of NMOSD."
The safety and tolerability
of Ultomiris in the Champion-NMOSD trial were consistent
with previous clinical studies and other approved indications.
Fifty-six patients are continuing to receive treatment in a
long-term extension period, which is ongoing.
The data will be presented at a forthcoming
medical meeting and submitted to global health authorities as
rapidly as possible to bring forward Ultomiris to the NMOSD community.
Notes
NMOSD
NMOSD is a rare disease in which the immune system
is inappropriately activated to target healthy tissues and cells in
the CNS.1,2 Approximately
three-quarters of people with NMOSD are anti-AQP4 Ab+, meaning they
produce antibodies that bind to a specific protein, aquaporin-4
(AQP4).7 This
binding can inappropriately activate the complement
system, which is part of the immune system and is essential to the
body's defence against infection, to destroy cells in the optic
nerve, spinal cord and brain.1,8,9
It most commonly affects women and begins in
the mid-30s. Men and children may also develop NMOSD, but it is
even more rare.10,11 People
with NMOSD may experience vision problems, intense pain, loss of
bladder/bowel function, abnormal skin sensations (eg, tingling,
prickling or sensitivity to heat/cold) and impact on coordination
and/or movement.3-5,12,13 Most
people living with NMOSD experience unpredictable relapses, also
known as attacks. Each relapse can result in cumulative disability
including vision loss, paralysis and sometimes premature
death.4-6 NMOSD
is a distinct disease from other CNS diseases, including multiple
sclerosis. The journey to diagnosis can be long, with the disease
sometimes misdiagnosed.14-16
CHAMPION-NMOSD
CHAMPION-NMOSD is a global Phase III, open-label, multicentre trial
evaluating the safety and efficacy of Ultomiris in adults with NMOSD. The trial enrolled 58
patients across North America, Europe, Asia-Pacific and Japan.
Participants were required to have a confirmed NMOSD diagnosis with
a positive anti-AQP4 antibody test, at least one attack or relapse
in the twelve months prior to the screening visit, an Expanded
Disability Status Scale Score of 7 or less and body weight of at
least 40 kilograms at trial entry. Participants could stay on
stable supportive immunosuppressive therapy for the duration of the
trial.17
Due to the potential long-term functional impact
of NMOSD relapses, a direct placebo comparator arm was precluded
for ethical reasons. The active treatment was compared to an
external placebo arm from the pivotal Soliris PREVENT clinical trial.
Over a median treatment duration of 73 weeks, all
enrolled patients received a single weight-based loading dose
of Ultomiris on Day 1, followed by regular weight-based
maintenance dosing beginning on Day 15, every eight weeks. The
primary endpoint was time to first on-trial relapse, as confirmed
by an independent adjudication committee. The end of the primary
treatment period could have occurred either when all patients
completed or discontinued prior to the Week 26 visit and two or
more adjudicated relapses were observed, or when all patients
completed or discontinued prior to the Week 50 visit if fewer than
two adjudicated relapses were observed. In the trial, there
were zero adjudicated relapses so the end of the primary treatment
period occurred when the last enrolled participant completed the 50
week visit.
Patients
who completed the primary treatment period were eligible to
continue into a long-term extension period, which is
ongoing.
Ultomiris
Ultomiris (ravulizumab-cwvz),
the first and only long-acting C5 complement inhibitor, offers
immediate, complete and sustained complement inhibition. The
medication works by inhibiting the C5 protein in the terminal
complement cascade, a part of the body's immune system. When
activated in an uncontrolled manner, the complement cascade
over-responds, leading the body to attack its own healthy
cells. Ultomiris is administered intravenously every eight
weeks in adult patients, following a loading
dose.
Ultomiris is approved in the US for the treatment of
certain adults with generalised myasthenia
gravis.
Ultomiris is also approved in the US, EU and Japan for
the treatment of certain adults and children with paroxysmal
nocturnal haemoglobinuria.
Additionally, Ultomiris is approved in the US, EU and Japan for
certain adults and children with atypical haemolytic uraemic
syndrome to inhibit complement-mediated thrombotic
microangiopathy.
As part of a broad development
programme, Ultomiris is being assessed for the treatment of
additional haematology and neurology
indications.
Alexion
Alexion,
AstraZeneca Rare Disease, is the group within AstraZeneca focused
on rare diseases, created following the 2021 acquisition of Alexion
Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30
years, Alexion is focused on serving patients and families affected
by rare diseases and devastating conditions through the discovery,
development and commercialisation of life-changing medicines.
Alexion focuses its research efforts on novel molecules and targets
in the complement cascade and its development efforts on
haematology, nephrology, neurology, metabolic disorders, cardiology
and ophthalmology. Headquartered in Boston, Massachusetts, Alexion
has offices around the globe and serves patients in more than 50
countries.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global,
science-led biopharmaceutical company that focuses on the
discovery, development, and commercialisation of prescription
medicines in Oncology, Rare Diseases, and BioPharmaceuticals,
including Cardiovascular, Renal & Metabolism, and Respiratory
& Immunology. Based in Cambridge, UK, AstraZeneca operates in
over 100 countries and its innovative medicines are used by
millions of patients worldwide. Please
visit astrazeneca.com and
follow the Company on Twitter @AstraZeneca.
Contacts
For details on how to contact the Investor
Relations Team, please click here.
For Media contacts, click here.
References
1.
Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker
BG. The spectrum of neuromyelitis optica. Lancet Neurol.
2007;6(9):805-815.
2.
Wingerchuk DM. Diagnosis and treatment of neuromyelitis
optica. Neurologist. 2007;13(1):2-11.
3.
Hamid SHM, Whittam D, Mutch K et al. What proportion of
AQP4-IgG-negative NMO spectrum disorder patients are MOG-IgG
positive? A cross sectional study of 132 patients. J Neurol.
2017;264(10):2088-2094.
4.
Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr
Treat Options Neurol. 2008;10(1):55-66.
5. Kitley
J, Leite MI, Nakashima I, et al. Prognostic factors and
disease course in aquaporin-4 antibody-positive patients with
neuromyelitis optica spectrum disorder from the United Kingdom and
Japan. Brain. 2012;135(6):1834-1849.
6.
Jarius S, Ruprecht K, Wildemann B, et al. Contrasting disease
patterns in seropositive and seronegative neuromyelitis optica: a
multicentre study of 175 patients. J Neuroinflamm.
2012;9:14.
7.
Wingerchuk DM, Hogancamp WF, O'Brien PC, Weinshenker BG. The
clinical course of neuromyelitis optica (Devic's syndrome).
Neurology. 1999;53(5):1107-1114.
8.
Papadopoulos MC, Bennett JL, Verkman AS. Treatment of neuromyelitis
optica: state-of-the-art and emerging therapies. Nat Rev Neurol.
2014;10(9):493.
9.
Cossburn, M., et al. (2012). The Prevalence of Neuromyelitis Optica
in South East Wales." Eur J Neurol., 19(4): 655-659.
10.
Takata K, Matsuzaki T, Tajika Y. Aquaporins: water channel proteins
of the cell membrane. Prog Histochem Cytochem.
2004;39(1):1-83.
11.
Mori M, Kuwabara S, Paul F. Worldwide prevalence of neuromyelitis
optica spectrum disorders. J Neurol Neurosurg Psychiatry. 2018
Jun;89(6):555-556. doi: 10.1136/jnnp-2017-317566. Epub 2018 Feb 7.
PMID: 29436488.
12. Quek
AML, Mckeon A, Lennon VA et al. Effects of age and sex on
aquaporin-4 autoimmunity. Arch Neurol 2012 and
69:1039-43.
13.
Tüzün E, Kürtüncü M, Türkoğlu R, et al. Enhanced complement
consumption in neuromyelitis optica and Behcet's disease patients.
J Neuroimmunol. 2011;233(1-2):211-215.
14. Kuroda
H, Fujihara K, Takano R, et al. Increase of complement
fragment C5a in cerebrospinal fluid during exacerbation of
neuromyelitis optica. J Neuroimmunol.
2013;254(1-2):178-182.
15.
Jarius, S., Wildemann, B. (2013). The History of Neuromyelitis
Optica. J Neuroinflammation 10, 797.
16. Mealy,
M. A., et al. (2019). Assessment of Patients with
Neuromyelitis Optica Spectrum Disorder Using the EQ-5D.
International journal of MS care, 21(3), 129-134.
17.
ClinicalTrials.gov. An Efficacy and Safety Study of Ravulizumab in
Adult Participants With NMOSD. NCT Identifier: NCT04201262.
Available online. Accessed April 2022.
Dr.
Pittock has provided consulting services to Alexion.
Adrian Kemp
Company Secretary
AstraZeneca PLC
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:
05 May 2022
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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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