Eculizumab

Alexion, AstraZeneca Rare Disease, will present seven abstracts, including four oral presentations, from its leading rare neurology portfolio at the European Academy of Neurology (EAN) Annual Congress in Helsinki, Finland, 21 to 24 June 2025. Presentations will discuss real-world evidence related to the use of Ultomiris (ravulizumab) as a potential steroid-sparing therapy and build on the demonstrated safety and efficacy profiles of Ultomiris and Soliris (eculizumab) in adult patients with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalised myasthenia gravis (gMG). Christophe Hotermans, Head of Global Medical Affairs, Alexion, said: “At this year’s EAN, Alexion’s data will build upon our leadership in rare neurology. This includes real-world evidence which underscores the clinical benefit of sustained treatment with Ultomiris in adults with AChR-Ab+ gMG. Alexion data will reinforce the potential for Ultomiris to help achieve key treatment goals for individuals living with gMG, including symptom management and reduced steroid burden, and provide meaningful insights to help improve patient care.” New analyses further support steroid-sparing benefit of Ultomiris in gMG Data will be presented in two separate oral presentations evaluating the real-world impact of rapid and sustained C5 inhibition with ravulizumabon reducing the burden of oral corticosteroid (OCS) use in patients with AChR-Ab+ gMG. Clinical practice outcomes data from the global MG SPOTLIGHT registry will show that a reduction in concomitant immunosuppressive therapy and OCS burden was observed in adults with AChR-Ab+ gMG, following ravulizumabtreatment initiation, supporting its potential steroid-sparing role. Data from 44 patients analysed will show that 10/33 (30.3%) of patients receiving one or more concomitant immunosuppressive therapies at initiation discontinued at least one therapy following treatment with ravulizumab. In addition, after six months, the number of patients receiving ≤5 and ≤10 mg/day OCS increased from 11/26 (42.3%) and 16/26 (61.5%), respectively, to 16/26 (61.5%) and 20/26 (76.9%). Separately, observations from a retrospective analysis of medical records from adults with AChR-Ab+ gMG who initiated treatment with either ravulizumabor efgartigimod will show a potential trend toward greater reduction in OCS use and fewer event-related hospitalisations following treatment with ravulizumab. Data will show that among patients taking OCS at initiation, 17/19 (89.5%) ravulizumabtreated patients and 33/46 (71.7%) efgartigimod treated patients reduced/discontinued their OCS dose post-initiation. In addition, mean exacerbation-related hospitalisations per patient per month decreased from 0.17 to 0 among ravulizumabtreated patients and increased from 0.19 to 0.25 among efgartigimod treated patients. Real-world data reflects improved aspects of daily function in gMG patients treated with Ultomiris or Soliris An oral presentation of data from the global MG SPOTLIGHT registry will show that in adults with AChR-Ab+ gMG, treatment with ravulizumabor eculizumab resulted in statistically significant improvements in different aspects of the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores after treatment began (P<0.05). Patients on ravulizumabor eculizumab experienced complete or partial reductions in symptoms affecting vision (60.7% and 63.3%), speech (58.4% and 59.2%), breathing (33.7% and 26.5%) and mobility (51.7% and 49.0%). Similar improvements were seen when switching from eculizumab toravulizumab(67.5% (vision), 67.5% (speech), 45.0% (breathing) and 65.0% (mobility)). Advancing innovation in gMG and awareness of the impact of misdiagnosis in NMOSD An oral presentation will share results from Ad Scientiam’s decentralised research study, funded by Alexion, to evaluate the use of the app-based tool, ME&MG openTM for remote, daily tracking of gMG symptoms in adults with AChR-Ab+ gMG. These data will show that most data met quality criteria and that more than 65% of patients remained adherent after one year, highlighting the feasibility of digital biomarker collection for a broad gMG population. A poster presentation will share baseline characteristics of participants enrolled in the PREVAIL Phase III trial evaluating the safety and efficacy of gefurulimab, an investigational novel, dual-binding nanobody, optimised for weekly subcutaneous administration, in adult patients with AChR-Ab+ gMG. A virtual poster will provide an overview of the ongoing Phase III global study evaluating the safety and efficacy of gefurulimab for paediatric patients with AChR-Ab+ gMG. Another virtual poster will showcase findings from a European, cross-sectional survey of physicians and anti-aquaporin-4 (AQP4) antibody positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) patients. Results will demonstrate that patients initially misdiagnosed compared to those correctly diagnosed with AQP4-Ab+ NMOSD were more likely to experience physical disability (37.6% vs 24.5%, p=0.027), require caregiver involvement (61.0% vs 43.6%, p=0.006) and need help with a higher number of daily activities (mean [SD], 3.4 [2.8] vs 2.1 [1.8], p<0.001). Worsening of quality of life was also reported, emphasising the urgent need for increased understanding to facilitate reduction in time to diagnosis. Lead Author Abstract Title Presentation Details Lead Author gMG Lead Author Scheiner C, et al. Abstract Title Myasthenic Crises, Exacerbations, and Corticosteroid Use in US Patients Receiving Ravulizumab or Efgartigimod Presentation Details Oral Presentation Abstract ID: A-25-13152 21 June 2025 14:10 – 14:15 EEST Lead Author Saccà F, et al. Abstract Title Assessing Efficacy and Safety of Gefurulimab in Generalised Myasthenia Gravis: Baseline Characteristics From PREVAIL Presentation Details ePoster Presentation Abstract ID: A-25-13135 22 June 2025 13:09 – 13:12 EEST Lead Author Nowak R, et al. Abstract Title Concomitant Immunosuppressive Therapy Use With Ravulizumab: Analysis of a Generalised Myasthenia Gravis Global Registry Presentation Details Oral Presentation Abstract ID: A-25-13153 23 June 2025 14:10 – 14:15 EEST Lead Author Barnett-Tapia C, et al. Abstract Title Overcoming Challenges in Digital Biomarker Collection for Myasthenia Gravis: Insights from the ME&MGopen Decentralised Study* Presentation Details Oral Presentation Abstract ID: A-25-13186 23 June 2025 14:15 – 14:20 EEST Lead Author Juel V, et al. Abstract Title MG-ADL Subdomain Score Changes With Eculizumab or Ravulizumab: A Generalised Myasthenia Gravis Global Registry Analysis Presentation Details Oral Presentation Abstract ID: A-25-13154 23 June 2025 14:25 – 14:30 EEST Lead Author Dy S, et al. Abstract Title Phase 3 Study of Gefurulimab in Paediatric Participants With Generalised Myasthenia Gravis: Trial in Progress Presentation Details Virtual ePoster Presentation Available online on-demand Lead Author NMOSD Lead Author Kleman M, et al. Abstract Title A real-world survey assessing the extent and clinical impact of misdiagnosis for patients with AQP4-Ab+ NMOSD in Europe Presentation Details Virtual ePoster Presentation Available online on-demand *Ad Scientiam research study supported by Alexion 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 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. Corporate and financial