a8630j
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
Indicate
by check mark whether the registrant files or will file annual
reports under cover of Form 20-F or Form 40-F.
Form
20-F X Form 40-F __
Indicate
by check mark if the registrant is submitting the Form 6-K in paper
as permitted by Regulation S-T Rule 101(b)(1):
Indicate
by check mark if the registrant is submitting the Form 6-K in paper
as permitted by Regulation S-T Rule 101(b)(7): ______
Indicate
by check mark whether the registrant by furnishing the information
contained in this Form is also thereby furnishing the information
to the Commission pursuant to Rule 12g3-2(b) under the Securities
Exchange Act of 1934.
Yes __
No X
If
“Yes” is marked, indicate below the file number
assigned to the Registrant in connection with Rule 12g3-2(b):
82-_____________
AstraZeneca PLC
INDEX
TO EXHIBITS
1.
ALXN1840
Wilson Phase III met primary endpoint
26 August 2021 07:00 BST
ALXN1840 FoCus Phase III trial in Wilson disease met primary
endpoint demonstrating improvement in copper mobilisation from
tissues
ALXN1840 demonstrated approximately three times greater copper
mobilisation from tissues than standard-of-care
treatments
Positive high-level results from the FoCus Phase III trial in
Wilson disease showed ALXN1840 met the primary endpoint with a
statistically significant improvement in daily mean copper
mobilisation from tissues, demonstrating superiority compared with
standard-of-care (SoC) treatments.
The primary endpoint gauged the daily mean Area Under the Effect
Curve (AUEC) for directly measured non-ceruloplasmin-bound copper
(dNCC) over 48 weeks. This novel measure assesses the daily mean
copper mobilised from tissues, reflecting the underlying burden of
the copper accumulation.
Wilson disease is a rare and progressive genetic condition in which
the body's pathway for removing excess copper is
compromised.1 Damage
from toxic copper build-up in tissues and organs leads to liver
disease, psychiatric and/or neurological
symptoms.1
ALXN1840, a potential new once-daily, oral medicine, demonstrated
approximately three times greater copper mobilisation than
SoC. The trial enrolled 214 patients, including
treatment-naive participants and those who have been on SoC therapy
for an average of ten or more years. Additional analyses, including
individual patient-reported outcomes and clinician-reported
functional assessments, are ongoing and will be presented at an
upcoming medical meeting.
Marc Dunoyer, Chief Executive Officer, Alexion, said: "Where
existing treatments remove copper from the blood, these 48-week
Phase III results demonstrate ALXN1840's significant impact in
mobilising copper from tissues. As we advance this first innovation
in Wilson disease treatment in more than 30 years, we will continue
to follow these patients long term to further assess clinical
impact on disease symptoms. We look forward to learning more about
how we can evolve the treatment of this progressive and devastating
disease."
Dr Michael Schilsky, Medical Director of Adult Liver Transplant at
Yale-New Haven Transplantation Center, Yale University, New Haven,
Connecticut, US and principal investigator of the FoCus Phase III
trial, said: "It is encouraging to see the effect of ALXN1840 on
both treatment-naive patients and those who have been on SoC for an
average of ten or more years. The Phase III results provide
evidence that tissue-bound copper remains built-up in the organs
even in patients who have been on SoC therapy for many years, and
the potential for ALXN1840 to provide a new approach to mobilise
and safely sequester copper from tissues."
ALXN1840 was generally well-tolerated with most reported adverse
events considered mild to moderate, and no neurological worsening
upon initiation of treatment was observed. In the ALXN1840
treatment group, the most frequently reported adverse event was a
reversible increase in transaminase levels.
Alexion is working closely with health authorities worldwide and
intends to submit these data for review in the coming
months.
Wilson disease
Wilson disease is a rare and progressive genetic condition in which
the body's pathway for removing excess copper is
compromised.1 It
affects one in 30,000 live births in the US.1 Over
time this results in the build-up of toxic copper levels in the
liver, brain, and other organs, leading to damage that greatly
impacts a patient's life.1 Patients
can develop a wide range of symptoms, including liver disease
and/or psychiatric or neurological symptoms, such as personality
changes, tremors and difficulty walking, swallowing or
talking.1 In
some cases, the damage and loss of function may be
irreversible.1, 2,
3
FoCus
FoCus is a pivotal, Phase III, randomised, rater-blinded,
multi-centre clinical trial designed to evaluate the efficacy and
safety of ALXN1840 versus SoC in patients with Wilson disease aged
12 years and older. The primary endpoint assessed copper
mobilisation, defined as daily mean AUEC for directly measured
dNCC over 48 weeks. In the trial, 214 patients were enrolled
in one of two cohorts on a 3:1 basis
(treatment-experienced:treatment-naive). Each cohort was then
randomised 2:1 (ALXN1840:SoC). The first cohort enrolled 161
patients who received SoC (chelation therapy with penicillamine or
trientine, zinc therapy or a combination of both chelation and zinc
therapy) for more than 28 days and the second cohort enrolled 53
patients who were treatment naive or had received SoC for 28 days
or less.
Patients who completed the primary 48-week treatment period of the
trial were offered the opportunity to participate in an up to
60-month extension period to evaluate the long-term safety and
efficacy of ALXN1840.
ALXN1840
ALXN1840 is a potential new once-daily, oral medicine in
development for the treatment of Wilson disease. It is designed to
be the first targeted de-coppering therapy that selectively and
tightly binds to, and removes, copper from the body's tissues and
blood. ALXN1840 has been granted Orphan Drug Designation in the US
and EU for Wilson disease.
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. Patil, M., et al. (2013) J Clin Exp
Hepatol, 3,
321-336.
2. Roberts,
E.A., Schilsky, M.L. American Association for the Study of Liver D.
(2008). Diagnosis and treatment of Wilson disease: An
update. Hepatology,
47(6), 2089-2111.
3. European Association for the Study of the Liver. (2012). EASL
clinical practice guidelines: Wilson's
disease. J Hepatol, 56(3), 671-685.
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:
26 August 2021
|
By: /s/
Adrian Kemp
|
|
Name:
Adrian Kemp
|
|
Title:
Company Secretary
|