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New data from PREVAIL Phase III trial will add to the clinical evidence supporting investigational self-administered C5 inhibitor in adults with gMG
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Qualitative sub-study of KOMET Phase III trial and real-world evidence will further demonstrate impact of Koselugo on symptoms in adults with NF1-PN, building on established efficacy profile across age groups
10 April 2026--Alexion, AstraZeneca Rare Disease, will deliver 20 presentations, including five oral presentations across generalised myasthenia gravis (gMG), neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN) and neuromyelitis optica spectrum disorder (NMOSD) at the American Academy of Neurology (AAN) Annual Meeting in Chicago, Illinois, 18 to 22 April 2026.
Key presentations will include new results from the PREVAIL Phase III trial evaluating novel dual-binding nanobody gefurulimab in adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) gMG as well as a new sub-study from the KOMET Phase III trial evaluating Koselugo (selumetinib) in adults with NF1 who have symptomatic, inoperable PN. An oral presentation will share new transcriptomics data from the CHAMPION-NMOSD Phase III trial, and additional presentations will highlight real-world evidence, including radiological outcomes, on the approved use of Ultomiris (ravulizumab) in NMOSD.
Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “At AAN 2026, we will have our broadest set of neurology data to date, with presentations showcasing our rare disease portfolio and pipeline innovation across gMG, NMOSD and NF1-PN. New data from the PREVAIL Phase III trial will reinforce C5 inhibition in gMG, with investigational once-weekly self-administered gefurulimab showing rapid, sustained improvements through 26 weeks and new insights from the KOMET Phase III trial will highlight Koselugo’s impact in adults with NF1-PN. These findings reflect Alexion’s determination to advance innovations that make a meaningful impact for patients.”
New data highlights gefurulimab potential as an effective, self-administered treatment option for gMG
An oral presentation from the global PREVAIL Phase III trial will share new results evaluating gefurulimab as a self-administered, once-weekly subcutaneous treatment option for adults with gMG. New data will show statistically significant and clinically meaningful improvement in the secondary endpoint of change in Myasthenia Gravis Composite at Week 26 compared to placebo (treatment difference, -3.1 [0.69]; P<0.0001), expanding on data previously presented at the 2025 Myasthenia Gravis Foundation of America (MGFA) Scientific Session during the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting.
Oral presentations spotlight meaningful symptom improvement with Koselugo, including reductions in pain in adult NF1-PN
A qualitative sub-study conducted with participants from KOMET, the largest and only placebo-controlled global Phase III trial in adults with NF1-PN, will show that patients on Koselugo reported noticeable decreases in pain, tingling, PN size and improvement in sleep, fatigue and body movements that were sustained or further improved over the course of the trial. Finally, an oral presentation of retrospective US claims data over three years will demonstrate lasting reductions in prescription pain medication utilisation among adults with NF1-PN taking Koselugo.
Alexion presentations during the 2026 AAN Annual Meeting
*Ad Scientiam research study supported by Alexion
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/NYSE: 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.