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 July 2025
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. Gefurulimab nanobody met Phase
III endpoints
24 July
2025
Gefurulimab dual-binding nanobody demonstrated statistically
significant and clinically meaningful improvement in functional
activities of daily living in adults with generalised myasthenia
gravis in PREVAIL Phase III trial
Once-weekly self-administered subcutaneous C5 inhibitor showed
statistically significant and clinically meaningful reduction in
disease severity at
week 26
Positive high-level results from a global, randomised,
double-blind, placebo-controlled Phase
III trial in adults with anti-acetylcholine receptor (AChR)
antibody-positive (Ab+) generalised myasthenia gravis (gMG) showed
that gefurulimab met its primary and all secondary endpoints. Data
demonstrated a statistically significant and clinically meaningful
improvement from baseline in Myasthenia Gravis Activities
of Daily Living (MG-ADL) total score at week 26 compared
to placebo.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular
disease that leads to a loss of muscle function and severe
weakness.1 Those
living with gMG may initially experience slurred speech, double
vision, droopy eyelids and weakness, with symptoms becoming more
severe as the disease progresses, including extreme fatigue,
difficulty swallowing, choking and respiratory
failure.2,3
Kelly Gwathmey, MD, Associate Professor of Neurology, Chief of
Neuromuscular Division, Virginia Commonwealth University, Richmond,
VA, Vice Chair of the MGFA Medical & Scientific Advisory
Council and principal investigator in the trial,
said: "Rapidly fluctuating symptoms and the unpredictable
disability associated with gMG can affect nearly every aspect
of a patient's life, making early intervention and sustained
disease control a critical treatment goal. A once-weekly,
self-administered C5 treatment option would offer patients greater
convenience and independence in managing their condition,
empowering them to have more control over their
therapy."
Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare
Disease, said: "Building
on Alexion's pioneering leadership in gMG, these positive results
from the PREVAIL Phase III trial demonstrate the potential for
gefurulimab to offer rapid and sustained disease control for this
patient community. These data, reflecting patient participation
across 20 countries, reinforce the
established safety profile and efficacy of C5 inhibition and show
the potential for gefurulimab as a first line biologic, with the
convenience of a self-administered option."
Gefurulimab was well-tolerated, and the safety profile was
consistent with previous trials of C5 inhibitors in gMG with no new
safety signals observed. These data will be presented at a
forthcoming medical meeting and shared with global regulatory
authorities.
Notes
gMG
gMG is a rare autoimmune disorder characterised by loss of muscle
function and severe muscle weakness.1
Eighty-five percent of people with gMG are AChR antibody-positive
meaning they produce specific antibodies (anti-AChR) that bind to
signal receptors at the neuromuscular junction (NMJ), the
connection point between nerve cells and the muscles they
control.4 This
binding activates the complement system, causing the immune system
to attack the NMJ, leading to inflammation and a breakdown in
communication between the brain and the muscles.5
gMG can occur at any age, but it most commonly begins for women
before the age of 40 and for men after the age of
60.6 Initial
symptoms may include slurred speech, double vision, droopy eyelids
and lack of balance; these can often lead to more severe symptoms
as the disease progresses such as, impaired swallowing, choking,
extreme fatigue and respiratory failure.2,3
Gefurulimab
Gefurulimab, an investigational complement C5 inhibitor, is a novel
dual-binding nanobody optimised for subcutaneous
self-administration in development as a treatment for AChR-Ab+ gMG.
The investigational medication works by binding to 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. Gefurulimab's concurrent binding to serum albumin provides
an extended half-life, enabling once-weekly dosing. Gefurulimab has
been granted Orphan Drug Designation in the US for the treatment of
myasthenia gravis.
PREVAIL (ALXN1720-MG-301)
PREVAIL (ALXN1720-MG-301) is a global, Phase III, randomised,
double-blind, placebo-controlled, parallel, multicentre study
evaluating the safety and efficacy of gefurulimab in adults with
generalised myasthenia gravis (gMG). The trial enrolled 260
patients from 20 countries across North America, Europe, Asia and
the Pacific region. Participants were required to have a confirmed
myasthenia gravis diagnosis at least three months prior to the
screening visit with a positive serological test for autoantibodies
against AChR and Myasthenia Gravis Foundation of America Clinical
Classification Class II to IV at screening.7
Patients were randomised 1:1 to receive gefurulimab or placebo for
a total of 26 weeks in the randomised
controlled treatment period. Patients received a single
weight-based loading dose on Day 1, followed by regular
weight-based maintenance dosing beginning on Day 8 and once every
week thereafter. The primary endpoint of the change from baseline
in the Myasthenia Gravis Activities of Daily Living (MG-ADL) total
score, a patient-reported scale that assesses patients' abilities
to perform daily activities, was assessed at week 26 along with
multiple secondary endpoints evaluating improvement in
disease-related measures.7
Patients who completed the randomised controlled treatment period
were eligible to continue into an open-label extension period
evaluating the safety and efficacy of gefurulimab, which is
ongoing.7
Alexion
Alexion, AstraZeneca Rare Disease, is focused on serving patients
and families affected by rare diseases and devastating conditions
through the discovery, development and delivery of life-changing
medicines. A pioneering leader in rare disease for more than three
decades, Alexion was the first to translate the complex biology of
the complement system into transformative medicines, and today it
continues to build a diversified pipeline across disease areas with
significant unmet need, using an array of innovative modalities. As
part of AstraZeneca, Alexion is continually expanding its global
geographic footprint to serve more rare disease patients around the
world. It is headquartered in Boston, US.
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's innovative
medicines are sold in more than 125 countries and used by millions
of patients worldwide. Please visit astrazeneca.com and
follow the Company on social media @AstraZeneca.
Contacts
For details on how to contact the Investor Relations Team, please
click here.
For Media contacts, click here.
References
1. Jung-Plath W, et al.
Assessment of myasthenia gravis patients' quality of
life. The Journal of Neurological
and Neurosurgical Nursing.
2023;12(2):74-83.
2. Catalin J, et al. Clinical
presentation of myasthenia gravis. Thymus. 2019.
3. Farid ZR, et al. Factors affecting
generalization of ocular myasthenia
gravis. Sriwijaya
Journal of Ophthalmology.
2020;3(2):48-54.
4. Lazaridis K, et al. Myasthenia
gravis: autoantibody specificities and their role in MG
management. Front
Neurol. 2020;11:596981.
5. Huang YF, et al. Visualization
and characterization of complement activation in acetylcholine
receptor antibody seropositive myasthenia
gravis. Muscle
Nerve.
2024.
6.
Cavanagh N,
et al. Exploring the impairments and allied health professional
utilization in people with myasthenia gravis:
a cross-sectional study. J Clin
Neurosci.
2023;114:9-16.
7. ClinicalTrials.gov.
Safety and efficacy of ALXN1720 in adults with generalized
myasthenia gravis. NCT Identifier: NCT05556096.
Available here.
Accessed July 2025.
Matthew Bowden
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.
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AstraZeneca
PLC
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Date: 24 July 2025
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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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