By: IPP Bureau
Last updated : October 31, 2025 5:30 pm
New Phase III data on gefurulimab highlight potential as a self-administered treatment option; real-world evidence reinforces clinical benefits of Ultomiris and Soliris
Alexion, AstraZeneca Rare Disease, announced that it will present 18 scientific abstracts, including four oral presentations, at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and the Myasthenia Gravis Foundation of America (MGFA) Scientific Session, taking place from 29 October to 1 November 2025 in San Francisco. These presentations will underscore Alexion’s pioneering leadership in C5 inhibition and highlight the company’s continued innovation in improving outcomes for patients with generalised myasthenia gravis (gMG).
The data include topline results from the global Phase III PREVAIL trial evaluating gefurlimab, a novel dual-binding nanobody optimized for subcutaneous self-administration, in adults with anti-acetylcholine receptor (AChR) antibody-positive gMG. The study met its primary and all secondary endpoints, demonstrating statistically significant and clinically meaningful improvements in Myasthenia Gravis Activities of Daily Living (MG-ADL) total scores at week 26 compared to placebo.
Christophe Hotermans, Senior Vice President and Head of Global Medical Affairs at Alexion, said: “At this year’s MGFA Scientific Session, we are proud to reinforce our leadership in C5 inhibition with compelling data from the PREVAIL Phase III trial. These results support the potential of gefurulimab to provide early and sustained disease control for patients with gMG. Alongside this, real-world and clinical data on Ultomiris further highlight our commitment to addressing key unmet needs in gMG management, including reducing steroid burden and improving long-term outcomes.”
Additional data on gefurulimab will explore its delivery via prefilled syringe (PFS) and autoinjector (AI). A Phase I study in healthy adults demonstrated comparable pharmacokinetic exposure between AI and PFS administration, while a human factors validation study confirmed that both devices can be used safely and effectively, with positive user experiences.
Alexion will also present extensive real-world evidence reinforcing the steroid-sparing benefits of Ultomiris (ravulizumab) and Soliris (eculizumab). Retrospective studies will show that prolonged corticosteroid use is associated with higher toxicity and increased healthcare resource utilization in patients with gMG. Data from the global MG SPOTLIGHT Registry will further demonstrate that treatment with Ultomiris significantly reduces reliance on oral corticosteroids and other immunosuppressive therapies in routine clinical practice.
At the AANEM Annual Meeting, an encore poster presentation based on a U.S. claims database analysis will reveal that patients treated with Ultomiris or Soliris achieved greater reductions in oral corticosteroid use after 12 months compared with those receiving a neonatal Fc receptor (FcRn) inhibitor.
Two oral presentations will highlight the effectiveness of Alexion’s meningococcal infection risk mitigation strategies for patients receiving Ultomiris or Soliris. Analyses of the Alexion safety database and antibiotic prophylaxis practices across pivotal trials demonstrate that these strategies are highly effective globally, with penicillin-class antibiotics most commonly utilized.
Real-world studies will also highlight healthcare provider confidence in Ultomiris and its growing role across the gMG treatment landscape. Patients who initiated Ultomiris within two years of diagnosis experienced greater improvements in MG-ADL scores, minimal manifestation rates, and fewer hospitalizations related to MG exacerbations compared with those treated with FcRn inhibitors. Additional analyses reveal that provider-initiated transitions to Ultomiris are driven by unmet clinical needs, sustained disease control, and strong physician confidence in its therapeutic value.
Through this broad portfolio of studies, Alexion continues to advance its legacy of innovation and leadership in rare neurology, reaffirming its commitment to transforming the treatment landscape for people living with gMG.