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 August
2021
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.
Update on Ultomiris
Phase III ALS trial
20 August 2021 07:05 BST
Update on CHAMPION-ALS Phase III trial
of Ultomiris in amyotrophic lateral
sclerosis
Alexion is discontinuing CHAMPION-ALS, the global Phase III
clinical trial of Ultomiris (ravulizumab)
in adults with amyotrophic lateral sclerosis
(ALS).
This decision is based on the recommendation of the Independent
Data Monitoring Committee (IDMC), following their review of data
from a pre-specified interim analysis. The IDMC recommended that
the trial be discontinued due to lack of efficacy. No new safety
findings were observed and the data were consistent with the
established safety profile of Ultomiris.
ALS is a rare, fatal neurodegenerative disease that affects motor
neurons (a type of nerve cell that controls voluntary movements) in
the brain and spinal cord.
Gianluca Pirozzi, MD, PhD, Senior Vice President, Head of
Development and Safety, Alexion, said: "We are disappointed by
this outcome and what it means for patients with this devastating
disease. We would like to thank the entire ALS community as well as
investigators and healthcare professionals who dedicated their time
and expertise to this trial. We continue to be confident in the
potential of targeting C5 for complement-driven diseases and remain
fully committed to our efforts to serve the rare disease
community."
Patients who enrolled in the trial will discontinue study
medication and complete any necessary follow-up evaluations. Data
from the trial will be provided to inform ongoing
research.
ALS
ALS is a neurological disorder characterised by progressive
degeneration of nerve cells (motor neurons) in the brain and the
spinal cord that control muscles throughout the body. When the
nerve cells die, the brain can no longer initiate and control
muscle movement, which results in severe disability, paralysis and
eventually death. ALS is a relentlessly progressive disorder.
People with ALS may lose the ability to speak, eat, move and
breathe. The rate of progression between individuals is variable
and the natural history generally reflects progressive worsening
over time until death occurs. The average life expectancy from
symptom onset is between two and five years. Currently approved
medications may slow disease progression, but ALS management is
mostly supportive, palliative and symptom based.
CHAMPION-ALS
The CHAMPION-ALS Phase III trial was a randomised, double-blind,
placebo-controlled multicentre global trial designed to evaluate
the efficacy and safety of Ultomiris across a broad ALS population. The trial
enrolled 382 adults with sporadic or familial ALS who had disease
onset (in the form of first motor symptoms) within the prior 36
months, demonstrated a slow vital capacity (SVC) of at least 65
percent predicted, and were not dependent on respiratory support.
The primary endpoint was change in ALS functional rating
scale-revised (ALSFRS-R) score. Trial participants were randomised
on a 2:1 basis to receive Ultomiris or placebo every eight weeks following an
initial loading dose. The trial was conducted at approximately 90
clinical trial sites across North America, Europe and
Asia.
Ultomiris
Ultomiris (ravulizumab),
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 or, for paediatric
patients less than 20 kg, every four weeks, following a loading
dose. Ultomiris is
approved in the United States (US) for the treatment of adults and
children (one month of age and older) with paroxysmal
nocturnal haemoglobinuria (PNH), as well as in the European Union
(EU) and Japan as a treatment for adults with PNH. It is also
approved in the US for atypical haemolytic uraemic syndrome
(aHUS) to inhibit complement-mediated thrombotic microangiopathy in
adult and paediatric (one month of age and older) patients, in the
EU for the treatment of adults and children with a body weight of
at least 10 kg with aHUS, as well as in Japan for adults and
children with aHUS.
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.
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:
20 August
2021
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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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