Alexion, AstraZeneca Rare Disease, will present new clinical and real-world data from its leading rare neurology portfolio at the American Academy of Neurology (AAN) Annual Meeting in Denver, CO, 13 to 18 April 2024. The company will present 14 abstracts, including five oral presentations, across both generalised myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD).

Presentations include new long-term results from the pivotal Phase III CHAMPION-MG and CHAMPION-NMOSD trials, as well as real-world data, adding to the robust evidence supporting the safety and efficacy of Ultomiris (ravulizumab) and Soliris (eculizumab) in gMG and NMOSD.

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: "Ultomiris and Soliris bring innovation and hope to the gMG and NMOSD communities, offering treatment options with the potential to transform care for these debilitating diseases. Our data at AAN will showcase outcomes in both clinical and real-world settings that clearly demonstrate the sustained benefit of Ultomiris and Soliris in these patient populations. We remain committed to advancing care and innovative solutions for people living with these rare neurological conditions." 

Continued evidence supporting long-term efficacy and safety of Ultomiris in NMOSD and gMG

Two oral presentations will detail new findings on the long-term safety and efficacy of Ultomiris in adults with the most common forms of NMOSD and gMG.

Long-term results from the ongoing global, open-label CHAMPION-NMOSD trial will demonstrate the potential for Ultomiris to eliminate relapses in people living with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) NMOSD. Data will show there were zero adjudicated on-trial relapses observed in Ultomiris-treated patients with AQP4 Ab+ NMOSD, with a median treatment duration of 138 weeks.

Further, the final analysis from the global CHAMPION-MG open-label extension will underscore the benefits of sustained treatment of Ultomiris in patients with anti-acetylcholine receptor (AChR) Ab+ gMG. Improvements in measures of functional activities and quality of life, including Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) total scores, were maintained in Ultomiris-treated patients for up to 164 weeks.

Real-world data highlight benefit of C5 inhibitors in gMG clinical practice

An oral presentation will report results from a retrospective US-based medical record analysis, which suggests earlier treatment initiation with C5 inhibitor therapy offers greater clinical benefit for patients with gMG. While MG-ADL scores improved for patients who initiated Soliris early or late, greater improvements were observed among those who started treatment within two years of their gMG diagnosis.

Two poster presentations will discuss steroid usage patterns and outcomes when used to treat chronic gMG, including after treatment with C5 inhibitors. A retrospective observational cohort study evaluating Medicare claims will indicate high rates of comorbidities among patients with gMG, which may inform clinical practice when prescribing corticosteroids. Additional medical claims data will show statistically significant reductions in the daily use of corticosteroids after 12 months of treatment with C5 inhibitors, as well as reductions in gMG exacerbations, supporting the use of C5 inhibitors as steroid-sparing therapy.

Findings from a gMG global registry will also be shared, including an encore oral presentation that will show patients who have been treated with Soliris in clinical practice experience decreased rates of myasthenic crisis, exacerbations and hospitalisations.

Uncovering new insights in NMOSD therapeutic efficacy

An oral presentation will highlight results from a study evaluating specific biomarkers as measures of biological response to treatment with Ultomiris and Soliris from the global Phase III PREVENT and CHAMPION-NMOSD trials. The data will show glial fibrillary acidic protein (GFAP) and neurofilament light chain (NfL) levels, biomarkers for astrocyte and neuronal injury, decreased over time with C5 inhibitor treatment and may be additional indicators of therapeutic efficacy when evaluating treatments for AQP4 Ab+ NMOSD.  

Alexion presentations during AAN 2024

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Lead Abstract Title  Presentation Details 
Author 
NMOSD
Pittock, SJ Efficacy and Oral Presentation Oral presentation 003S32:
safety of Autoimmune Neurology: NMOSD/MOGAD17 April
ravulizumabin 202413:24 MDT
adults with anti
-aquaporin-4
antibody
-positiveNMOSD:
interim analysis
from the ongoing
phase 3 CHAMPION
-NMOSD trial
Wingerchuk, Evaluation of Oral Presentation Oral presentation 004S32:
DM glial fibrillary Autoimmune Neurology: NMOSD/MOGAD17 April
acidic protein 202413:36 MDT
(GFAP) and
neurofilament
light chain (NfL)
levels during
eculizumab and
ravulizumab
treatments in
aquaporin-4
-positive (AQP4+)
neuromyelitis
optica spectrum
disorder (NMOSD)
Clardy, SL Indirect Poster Presentation Poster presentation
treatment 005P10: Autoimmune Neurology: NMOSD17 April
comparison of 202411:45 - 12:45 MDT 
ravulizumab
versus approved
treatment options
for adults with
aquaporin-4
immunoglobulin
positive(AQP4
-IgG+)
neuromyelitis
optica spectrum
disorder (NMOSD)
Levy, M Validation Poster Presentation Poster presentation
process of 001P10: Autoimmune Neurology: NMOSD17 April
NMOSDCopilot[TM], 202411:45 - 12:45 MDT
a software as a
medical device
(SaMD) for
patients living
with
neuromyelitis
optica spectrum
disorder*
gMG
Vu, T Long-term Oral Presentation Oral presentation 010S15:
efficacy and Autoimmune Neuromuscular Diseases: New
safety of Observations and Therapeutic Approaches15
ravulizumab, a April 202414:48 MDT
long-acting
terminal
complement
inhibitor, in
adults with anti
-acetylcholine
receptor antibody
-positive
generalized
myasthenia
gravis: final
results from the
Phase 3 CHAMPION
MG open-label
extension
Muppidi, S Effectiveness of Oral PresentationOral presentation 006S15:
eculizumab Autoimmune Neuromuscular Diseases: New
treatment by time Observations and Therapeutic Approaches15
from diagnosis in April 2024 14:00 MDT
patients with
generalized
myasthenia
gravis: a
retrospective
electronic
medical record
analysis
Tandan, R Rates of Oral Presentation Oral presentation 001S38:
myasthenic Autoimmune Neurology: Peripheral Autoimmunity,
crisis, Paraneoplastic Disease, Checkpoint Inhibitors,
exacerbation and and Neurosarcoidosis17 April 202415:30 MDT
healthcare
resource
utilization in
eculizumab
treated patients
with generalized
myasthenia gravis
in a global
registry
McEneny, A Phase 1 study of Poster Presentation Poster presentation
gefurulimab 004P10: Neuromuscular and Clinical
pharmacokinetics Neurophysiology (EMG): Myasthenia Gravis 315
(PK) and safety April 202411:45 - 12:45 MDT
following
delivery via
autoinjector in
healthy adults
Lee, J Patterns of Poster Presentation Poster presentation
steroid use and 011P10: Neuromuscular and Clinical
outcomes in us Neurophysiology (EMG): Myasthenia Gravis 317
patients with April 202411:45 - 12:45 MDT
generalized
myasthenia gravis
(gMG) receiving
C5 inhibitor
therapy (C5IT)
Juel, V Improvement in Poster Presentation Poster presentation 008
myasthenia gravis P4: Neuromuscular and Clinical Neurophysiology
activities of (EMG): Myasthenia Gravis 115 April 202411:45 -
daily living 12:45 MDT
subdomain scores
in patients
treated with
eculizumab:
results from a
generalized
myasthenia gravis
registry study
Sabatella, Effectiveness and Poster Presentation Poster presentation 002
G safety of S15: Autoimmune Neuromuscular Diseases: New
transitioning to Observations and Therapeutic Approaches17
ravulizumab from April 202411:45 - 12:45 MDT
eculizumab in
patients with
generalized
myasthenia
gravis: evidence
from a global
registry
Blackowicz, Comorbidity Poster Presentation Poster presentation 015P4:
M burden and Neuromuscular and Clinical Neurophysiology
steroid use in (EMG): Myasthenia Gravis 115 April 202411:45 -
generalized 12:45 MDT
myasthenia
gravis: a
retrospective
analysis of
Medicare fee-for
-service claims
Barnett First cross Poster Presentation Poster presentation 005P4:
Tapia, C -sectional Neuromuscular and Clinical Neurophysiology
analysis from the (EMG): Myasthenia Gravis 115 April 202411:45 -
ME&MG open study: 12:45 MDT
a decentralized
study on app
-based myasthenia
gravis*
Berling, E ME&MG, novel Poster Presentation Poster presentation 016P1:
digital device Neuromuscular and Clinical Neurophysiology
for patients with (EMG): New Tools in Neuromuscular Disease:
generalized Diagnosis and Assessment14 April 20248:00 -
myasthenia 9:00 MDT
gravis: a first
step towards
validation*

*Ad Scientiam research study supported by Alexion

Notes

Ultomiris (ravulizumab)

Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides 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, EU, Japan and other countries for the treatment of certain adults with generalised myasthenia gravis (gMG).

Ultomiris is also approved in the US, EU, Japan and other countries for the treatment of certain adults with paroxysmal nocturnal haemoglobinuria (PNH) and for certain children with PNH in the US and EU.

Additionally, Ultomiris is approved in the US, EU, Japan and other countries for certain adults and children with atypical haemolytic uraemic syndrome to inhibit complement-mediated thrombotic microangiopathy (aHUS).

Further, Ultomiris is approved in the US, EU and Japan for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

As part of a broad development programme, Ultomiris is being assessed for the treatment of additional haematology and neurology indications.

Soliris (eculizumab)

Soliris[ ](eculizumab) is a first-in-class C5 complement inhibitor. 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 terminal complement cascade over-responds, leading the body to attack its own healthy cells. Soliris is administered intravenously every two weeks, following an introductory dosing period.

Soliris is approved in the US, EU, Japan, China and other countries for the treatment of patients with PNH and aHUS. 

Additionally, Soliris is approved in Japan and the EU for the treatment of certain adult and paediatric patients with gMG, and in the US, China and other countries for certain adults with gMG.  

Further, Soliris is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with NMOSD. 

Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related haemolytic uraemic syndrome. 

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 more than 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 70 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-us.com/)and follow the Company on social media @AstraZeneca (https://www.linkedin.com/company/astrazeneca/).

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