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

Clinical and real-world data will reinforce the long-term safety and efficacy profiles of Ultomiris and Soliris and highlight the potential for sustained treatment to minimise steroid use 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 corticosteroid use in ravulizumab-treated adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: final results from the phase 3 CHAMPION-MG Open-label Extension ( Encore ) AANEM Annual Meeting Poster Presentation 236 16 October 2024 18:15-18:45 17 October 2024 14:45-15:15 Blackowicz, M Long-term corticosteroid treatments patterns and steroid-sparing effects of approved treatments for generalized myasthenia gravis in the United States ( Encore) AANEM Annual Meeting Poster Presentation 145 16 October 2024 18:15-18:45 17 October 2024 14:45-15:15 Narayanaswami, P Safety and effectiveness of ravulizumab in generalized myasthenia gravis: evidence from a global registry ( Encore ) AANEM Annual Meeting Poster Presentation 234 16 October 2024 18:15-18:45 17 October 2024 14:45-15:15 Scheiner, C Quality of life in generalized myasthenia gravis: results from a global registry of eculizumab and ravulizumab treatment ( Encore ) AANEM Annual Meeting Poster Presentation 267 16 October 2024 18:15-18:45 17 October 2024 9:30-10:00 Yee, K Patient preferences for generalized myasthenia gravis treatment profiles: results of a web-based survey ( Encore ) AANEM Annual Meeting Poster Presentation 310 16 October 2024 18:15-18:45 17 October 2024 14:45-15:15 Pandya, S Incidence and outcome of meningococcal infection with eculizumab or ravulizumab in patients with gMG or NMOSD: An analysis of US clinical practice ( Encore ) AANEM Annual Meeting Poster Presentation 240 16 October 2024 18:15-18:45 17 October 2024 14:45-15:15 MGFA Scientific Session Poster Presentation MG55 15 October 2024 12:00-12:45 Nowak, R Change in concomitant immunosuppressive therapies for generalized myasthenia gravis in patients receiving complement C5 inhibitor therapies: a retrospective analysis of registry data MGFA Scientific Session Poster Presentation MG86 15 October 2024 12:00-12:45 Scheiner, C Outcomes for patients with generalized myasthenia gravis prescribed ravulizumab, eculizumab, or efgartigimod treatment: interim analysis of a retrospective medical record analysis (ELEVATE) MGFA Scientific Session Poster Presentation MG85 15 October 2024 12:00-12:45 Narayanaswami, P Safety outcomes in pregnant patients treated with the complement 5 inhibitor therapy (C5IT) eculizumab MGFA Scientific Session Poster Presentation MG101 15 October 2024 12:00-12:45 Howard, J The phase 3 PREVAIL study assessing the efficacy and safety of subcutaneous gefurulimab in adults with generalized myasthenia gravis: trial in progress MGFA Scientific Session Poster Presentation MG99 15 October 2024 12:00-12:45 Gwathmey, K Evaluation of the indirect and nonmedical impacts of generalized myasthenia gravis on patients and caregivers MGFA Scientific Session Poster Presentation MG98 15 October 2024 12:00-12:45 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. 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