Alexion, AstraZeneca Rare Disease data at the 2024 AANEM Annual Meeting and MGFA Scientific Session further demonstrate advancement in gMG care

Alexion, AstraZeneca Rare Disease, will present data from its leading generalised myasthenia gravis (gMG) portfolio at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and the Myasthenia Gravis Foundation of America (MGFA) Scientific Session taking place in Savannah, GA, 15 to 18 October 2024.

The company will present 11 abstracts, spanning clinical and real-world data, which add to the extensive body of evidence supporting the safety and efficacy of Ultomiris (ravulizumab) and Soliris (eculizumab) in treating anti-acetylcholine receptor (AChR) antibody-positive (Ab+) gMG, and offer new insights to inform clinical practice.

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: "We are pleased to present continued positive results for Ultomiris and Soliris in gMG, which showcase their potential to improve medical outcomes and the quality of life for patients. Alexion's data at the AANEM Annual Meeting and the MGFA Scientific Session will reinforce the benefits of sustained treatment, including reductions in the use of corticosteroids and the potential to achieve minimal symptom expression, supporting clinical decision-making and care."

Further evidence supporting Ultomiris and Soliris as steroid-sparing therapies

At the MGFA Scientific Session an analysis of global gMG registry data will demonstrate a reduced oral corticosteroid (OCS) burden in adults treated with Soliris or Ultomiris. Oral corticosteroid doses also continued to decrease from treatment initiation to last dose assessed in patients who transitioned from Soliris to Ultomiris. Following treatment, there was a greater number of patients who transitioned from a higher dose to a lower dose of daily OCS.

Two encore poster presentations at the AANEM Annual Meeting will further highlight changes in steroid usage patterns and outcomes following treatment initiation with Ultomiris or Soliris. Results from the open-label extension of the pivotal, global Phase III CHAMPION-MG trial evaluating the safety and efficacy of Ultomiris in adults with AChR Ab+ gMG will show decreased steroid use in Ultomiris-treated patients. Additionally, a retrospective cohort study from a United States (US) claims database will highlight that Ultomiris and Soliris facilitated significant steroid sparing within the first year of their initiation.

Real-world data underscoring the safety and efficacy of Ultomiris and Soliris in clinical practice

New real-world data from a retrospective medical record analysis will be presented at the MGFA Scientific Session, reporting outcomes among gMG patients in the US treated with Ultomiris, Soliris and Vyvgart. Although patient characteristics differed between the treatment groups, results will suggest that Ultomiris may provide greater symptom control than alternative therapies as measured by MG Activities of Daily Living (MG-ADL) scores.

At the AANEM Annual Meeting, two encore presentations of results from a global gMG registry will suggest Ultomiris and Soliris may improve activities of daily living and quality of life in adults with AChR Ab+ gMG. In an interim analysis, patients initiating Ultomiris showed improvements in MG-ADL, including achieving minimal symptom expression, and MG Foundation of America Clinical Class (MGFA-CC) scores. Further, sustained improvements were observed when transitioning from Soliris to Ultomiris. Additionally, an analysis of Myasthenia Gravis Quality of Life15-revised (MG-QOL15r) scores will show clinically meaningful improvement in patients who initiated treatment with Ultomiris and Soliris, with further improvements observed among patients transitioning from Soliris to Ultomiris.

Further, safety outcomes in pregnant patients treated with Soliris across all approved indications will be reported at the MGFA Scientific Session. This cumulative analysis from the Alexion pharmacovigilance safety database offers valuable insights for patients and for clinical decision-making.

Advancing understanding and care for the gMG community

At the MGFA Scientific Session, a poster presentation will offer insight into indirect and non-medical costs of gMG, as reported by US patients and caregivers via a web-based survey. Results will show high annual indirect and nonmedical costs for both patients and caregivers, including lost work and social productivity, contributing substantially to the total economic impact of gMG.

Another poster presentation will provide an overview of the ongoing pivotal, global Phase III PREVAIL trial evaluating the efficacy and safety of gefurulimab, an investigational C5 inhibitor optimised for weekly subcutaneous administration, in adults with AChR Ab+ gMG.

Alexion presentations during the 2024 AANEM Annual Meeting and MGFA Scientific Session

Lead Author  Abstract Title  Presentation Details 
Nicolle, M Concomitant AANEM Annual MeetingPoster Presentation 236
corticosteroid 16 October 202418:15-18:4517 October
use in 202414:45-15:15
ravulizumab
-treated adults
with anti
-acetylcholine
receptor antibody
-positive
generalized
myasthenia
gravis: final
results from the
phase 3 CHAMPION
-MG Open-label
Extension
(Encore)
Blackowicz, M Long-term AANEM Annual MeetingPoster Presentation 145
corticosteroid 16 October 202418:15-18:4517 October
treatments 202414:45-15:15
patterns and
steroid-sparing
effects of
approved
treatments for
generalized
myasthenia gravis
in the United
States (Encore)
Narayanaswami, Safety and AANEM Annual MeetingPoster Presentation 234
P effectiveness of 16 October 202418:15-18:4517 October
ravulizumab in 202414:45-15:15
generalized
myasthenia
gravis: evidence
from a global
registry (Encore)
Scheiner, C Quality of life AANEM Annual MeetingPoster Presentation 267
in generalized 16 October 202418:15-18:45 17 October
myasthenia 20249:30-10:00
gravis: results
from a global
registry of
eculizumab and
ravulizumab
treatment
(Encore)
Yee, K Patient AANEM Annual MeetingPoster Presentation
preferences for 31016 October 202418:15-18:45 17 October
generalized 202414:45-15:15
myasthenia gravis
treatment
profiles: results
of a web-based
survey (Encore)
Pandya, S Incidence and AANEM Annual MeetingPoster Presentation 240
outcome of 16 October 202418:15-18:4517 October
meningococcal 202414:45-15:15MGFA Scientific
infection with SessionPoster Presentation MG55 15 October
eculizumab or 202412:00-12:45
ravulizumab in
patients with gMG
or NMOSD: An
analysis of US
clinical practice
(Encore) 
Nowak, R Change in MGFA Scientific SessionPoster Presentation
concomitant MG8615 October 202412:00-12:45
immunosuppressive
therapies for
generalized
myasthenia gravis
in patients
receiving
complement C5
inhibitor
therapies: a
retrospective
analysis of
registry data
Scheiner, C Outcomes for MGFA Scientific SessionPoster Presentation
patients with MG8515 October 202412:00-12:45
generalized
myasthenia gravis
prescribed
ravulizumab,
eculizumab, or
efgartigimod
treatment:
interim analysis
of a
retrospective
medical record
analysis
(ELEVATE)
Narayanaswami, Safety outcomes MGFA Scientific SessionPoster Presentation
P in pregnant MG10115 October 202412:00-12:45
patients treated
with the
complement 5
inhibitor therapy
(C5IT)
eculizumab 
Howard, J The phase 3 MGFA Scientific SessionPoster Presentation
PREVAIL study MG9915 October 202412:00-12:45
assessing the
efficacy and
safety of
subcutaneous
gefurulimab in
adults with
generalized
myasthenia
gravis: trial in
progress 
Gwathmey, K Evaluation of the MGFA Scientific SessionPoster Presentation
indirect and MG9815 October 202412:00-12:45
nonmedical
impacts of
generalized
myasthenia gravis
on patients and
caregivers 

Notes

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 over 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com (http://www.astrazeneca.com/) and follow the Company on social media @AstraZeneca (https://www.linkedin.com/company/astrazeneca/mycompany/verification/).

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