Alexion data presented at 2025 AANEM Annual Meeting and MGFA Scientific Session underscores pioneering C5 inhibition leadership in gMG

New data on gefurulimab, a novel dual-binding nanobody, from the PREVAIL Phase III trial will highlight its potential as an effective, self-administered treatment option 

Robust real-world evidence will reinforce the clinical benefits of Ultomiris and Soliris, including reduced steroid burden

Alexion, AstraZeneca Rare Disease, will deliver 18 presentations, including four oral presentations, from its leading rare neurology portfolio at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and the Myasthenia Gravis Foundation of America (MGFA) Scientific Session in San Francisco, California, 29 October to 1 November 2025.

Presentations will feature topline data from the global PREVAIL Phase III trial evaluating gefurulimab in adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalised myasthenia gravis (gMG), as well as robust real-world data and clinical insights that further reinforce the safety profiles and efficacy of Ultomiris (ravulizumab) and Soliris (eculizumab).  

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “At this year’s MGFA Scientific Session, we are proud to reinforce our pioneering leadership in C5 inhibition with topline data from the PREVAIL Phase III trial supporting the potential for gefurulimab to deliver early and sustained disease control to patients with gMG. Alongside this, we will share real-world and clinical evidence further establishing Ultomiris as a differentiated therapeutic option and underscoring the impact of our portfolio in addressing critical unmet needs in gMG treatment, such as reducing steroid burden.”

New data on gefurulimab will highlight its potential to provide early and sustained disease control
An oral presentation at the MGFA Scientific Session will share topline results from the global PREVAIL Phase III trial evaluating gefurulimab, a dual-binding nanobody optimised for subcutaneous self-administration, in adult patients with AChR-Ab+ gMG. The trial met its primary and all secondary endpoints, with gefurulimab demonstrating a statistically significant and clinically meaningful improvement from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26 compared to placebo.

Two additional posters at the MGFA Scientific Session will provide further insights into the delivery of gefurulimab via prefilled syringe (PFS) and auto injector (AI).  Results from a Phase I parallel-group study in healthy adults will show that pharmacokinetic exposure following a single subcutaneous dose of gefurulimab administered via autoinjector (AI) was comparable to administration via prefilled syringe (PFS). Separately, results from a human factors validation study will demonstrate that gefurulimab AI and PFS user interfaces can be used safely and effectively with positive user experiences.

Evidence further substantiates the steroid-sparing role of Ultomiris and Soliris
Several presentations at the MGFA Scientific Session will explore the real-world impact of corticosteroid use on patients with gMG. An encore oral presentation will highlight a retrospective study of patients with gMG showing that corticosteroid-related toxicity was higher in patients with ≥3 months’ continuous use of corticosteroids versus those with <3 months’ continuous use, or no use at all. Furthermore, a retrospective study of patients with gMG will demonstrate a significant increase in healthcare resource utilisation in patients with high corticosteroid toxicity.

Data will also be presented at the AANEM Annual Meeting on the reduced corticosteroid burden following treatment with Ultomiris or Soliris. An encore poster presentation will highlight data from the global MG SPOTLIGHT Registry showing reduced concomitant immunosuppressive therapies and oral corticosteroids burden in patients with AChR-Ab+ gMG treated with Ultomiris in routine clinical practice. Additionally, an encore poster presentation of a retrospective analysis of a United States (US) claims database will demonstrate greater reduction in oral corticosteroid use after 12 months in patients with gMG receiving treatment with Ultomiris or Soliris compared with a neonatal Fc receptor (FcRn) inhibitor.

Studies spotlight the effectiveness of meningococcal infection risk mitigation strategies for treatment with Ultomiris and Soliris
Two oral presentations at the MGFA Scientific Session will reinforce the effectiveness of Alexion’s meningococcal infection risk mitigation strategies. An analysis of the Alexion safety database will suggest that US Neisseria meningitidis-related risk mitigation strategies are effective in patients receiving Ultomiris or Soliris. Separately, a study of antibiotic prophylaxis (AB-PPx) risk mitigation practices across pivotal trials of Alexion’s C5 inhibitors in adults will demonstrate AB-PPx durations varied at initiation of treatment with Ultomiris or Soliris, with the majority of patients receiving it for either 1-30 days (US) or >1y (ex-US). Penicillin-class AB-PPx was most utilised, regardless of geography.  

Real-world evidence underscores HCP confidence in Ultomiris and its value across the gMG treatment paradigm
At the MGFA Scientific Session, a poster presentation of a retrospective subanalysis of US medical records will show that patients treated with Ultomiris within 2 years of gMG diagnosis trended toward greater improvements in MG-ADL scores, minimal manifestation and MG exacerbation-associated hospitalizations than those receiving efgartigimod, a FcRn inhibitor. A separate poster presentation will highlight real-world evidence showing that patients with gMG who initiated treatment with Ultomiris had a statistically significantly greater reduction in healthcare resource utilisation than patients who started on efgartigimod.

Two additional poster presentations will provide real-world insights on the drivers of healthcare provider-initiated switches to Ultomiris in gMG, revealing that transitions were driven by unmet needs and provider confidence in Ultomiris, or limitations of intermittent therapy and inability to meet expectations for improved, sustained disease control.

Alexion presentations during the 2025 AANEM Annual Meeting and MGFA Scientific Session

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.

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