Alexion, AstraZeneca Rare Disease, presented data from its generalized 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, showcasing the potential of Ultomiris (ravulizumab) and Soliris (eculizumab) in treating anti-acetylcholine receptor (AChR) antibody-positive (Ab+) gMG.
The company presented 11 abstracts, encompassing clinical and real-world data, reinforcing the safety and efficacy of Ultomiris and Soliris and providing new insights to inform clinical practice. Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, stated that the data reinforces the benefits of sustained treatment, including reductions in corticosteroid use and the potential to achieve minimal symptom expression, supporting clinical decision-making and care.
Steroid-Sparing Effects of Ultomiris and Soliris
An analysis of global gMG registry data presented at the MGFA Scientific Session demonstrated a reduced oral corticosteroid (OCS) burden in adults treated with Soliris or Ultomiris. Oral corticosteroid doses continued to decrease from treatment initiation to the last dose assessed in patients who transitioned from Soliris to Ultomiris. Following treatment, a greater number of patients transitioned from a higher dose to a lower dose of daily OCS.
Two encore poster presentations at the AANEM Annual Meeting further highlighted changes in steroid usage patterns and outcomes following treatment initiation with Ultomiris or Soliris. Results from the open-label extension of the pivotal Phase III CHAMPION-MG trial evaluating the safety and efficacy of Ultomiris in adults with AChR Ab+ gMG showed decreased steroid use in Ultomiris-treated patients. A retrospective cohort study from a US claims database also highlighted that Ultomiris and Soliris facilitated significant steroid sparing within the first year of their initiation.
Real-World Evidence of Efficacy and Safety
New real-world data from a retrospective medical record analysis presented at the MGFA Scientific Session reported outcomes among gMG patients in the US treated with Ultomiris, Soliris, and Vyvgart. Although patient characteristics differed between the treatment groups, results suggested that Ultomiris may provide greater symptom control than alternative therapies, as measured by MG Activities of Daily Living (MG-ADL) scores.
Two encore presentations of results from a global gMG registry at the AANEM Annual Meeting suggested Ultomiris and Soliris may improve activities of daily living and quality of life in adults with AChR Ab+ gMG. An interim analysis showed patients initiating Ultomiris experienced improvements in MG-ADL, including achieving minimal symptom expression, and MG Foundation of America Clinical Class (MGFA-CC) scores. Sustained improvements were observed when transitioning from Soliris to Ultomiris. An analysis of Myasthenia Gravis Quality of Life15-revised (MG-QOL15r) scores showed clinically meaningful improvement in patients who initiated treatment with Ultomiris and Soliris, with further improvements observed among patients transitioning from Soliris to Ultomiris.
Safety outcomes in pregnant patients treated with Soliris across all approved indications were reported at the MGFA Scientific Session. This cumulative analysis from the Alexion pharmacovigilance safety database offers insights for patients and clinical decision-making.
Additional Insights into gMG Care
A poster presentation at the MGFA Scientific Session offered insight into indirect and non-medical costs of gMG, as reported by US patients and caregivers via a web-based survey. Results showed 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 provided an overview of the ongoing Phase III PREVAIL trial evaluating the efficacy and safety of gefurulimab, an investigational C5 inhibitor optimized for weekly subcutaneous administration, in adults with AChR Ab+ gMG.
