Alexion, AstraZeneca Rare Disease, will present new data showing significant advances for the treatment of anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, 26 to 28 October 2022.

Data presented at the meeting will feature new insights from Alexion's complement portfolio illustrating the critical role of C5 inhibition in treating AQP4 Ab+ NMOSD and significantly reducing the risk of relapses, including findings on the first and only long-acting C5 complement inhibitor, Ultomiris (ravulizumab).

Marc Dunoyer, Chief Executive Officer, Alexion, said: "NMOSD relapses are unpredictable and often cause permanent disability, including blindness, paralysis and even premature death. Our data build on the established role of complement inhibition in managing NMOSD and reinforce our commitment to patient-centred innovation. Results from the CHAMPION-NMOSD trial highlight the potential for Ultomiris to substantially reduce the risk of relapse and ease treatment burden for the community."

Redefining NMOSD disease management

An oral presentation will showcase results of the CHAMPION-NMOSD trial, which evaluated the safety and efficacy of Ultomiris in adults with AQP4 Ab+ NMOSD, compared to the external placebo arm from the pivotal Soliris PREVENT clinical trial.

The data will show there were zero adjudicated on-trial relapses observed in patients with AQP4 Ab+ NMOSD with a median treatment duration of 73 weeks, representing a relapse risk reduction of 98.6% (p<0.0001) compared to the external placebo arm. The safety and tolerability of Ultomiris in the CHAMPION-NMOSD trial were consistent with previous clinical studies and other approved indications.

Improving understanding of the NMOSD patient experience

Two poster presentations detail findings from qualitative interviews with NMOSD patients. This work helps to inform the scientific community about the immediate and lasting impact of relapses, including ability to work and participate in daily activities, mobility and pain as well as reliance on caregiver support.

Alexion presentations during ECTRIMS 2022

Lead Abstract Presentation details
author title
Pittock, Efficacy and Oral Presentation O05127 October 202210:05
SJ safety of (CEST)
ravulizumab
in adults
with anti
-aquaporin-4
antibody
-positive
neuromyelitis
optica
spectrum
disorder:
outcomes from
the phase 3
CHAMPION
-NMOSD trial
Pittock, Efficacy Poster Presentation P01026 October 202216:30
SJ subgroup (CEST)ePoster Tour 528 October 202214:00
analyses from (CEST)
the phase 3
CHAMPION
-NMOSD trial
in adults
with anti
-aquaporin-4
antibody
-positive
neuromyelitis
optica
spectrum
disorder
Allen, K Sensitivity Poster Presentation P01226 October 202216:30
analysis (CEST)
using
propensity
score methods
for primary
efficacy
outcome in
the CHAMPION
-NMOSD trial
Palace, J Mortality Poster Presentation P01826 October 202216:30
estimates in (CEST)
patients with
anti
-aquaporin-4
autoantibody
positive
neuromyelitis
optica
spectrum
disorder
Kielhorn, Long-term Poster Presentation P01926 October 202216:30
A burden of (CEST)
relapse in
patients with
AQP4+ NMOSD:
simulation
study based
on network
meta-analysis
Osborne, B Activity Poster Presentation P41827 October 202217:00
impairment (CEST)
and support
needs in
patients with
neuromyelitis
optica
spectrum
disorder
Bernitsas, Understanding ePoster Presentation EP090126 October 2022
E the symptoms 8:00 (CEST)
of patients
with
neuromyelitis
optica
spectrum
disorder and
their impact
on patient's
lives: a
qualitative
interview
study 

Notes
 

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 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 (http://www.astrazeneca.com/) and follow the Company on Twitter @AstraZeneca (https://twitter.com/AstraZeneca).

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