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
Lead Author |
Abstract Title |
Presentation Details |
|---|---|---|
Lead Author gMG |
||
Lead Author Gwathmey, K. |
Abstract Title Efficacy and Safety of Subcutaneous Self-Administered Gefurulimab in Generalized Myasthenia Gravis: Primary Results From the Phase 3, Randomized, Double-Blind, Placebo-Controlled PREVAIL Study |
Presentation Details Oral Presentation 006 Abstract #1879 20 April 2026 02:00 PM CT |
Lead Author Juel, V. |
Abstract Title MG-ADL Subdomain Score Changes With Eculizumab or Ravulizumab: An Analysis of the Global MG SPOTLIGHT Registry (Encore) |
Presentation Details Poster Presentation Abstract #1774 20 April 2026 08:00 - 09:00 AM CT |
Lead Author Berling, E. |
Abstract Title Validation of ME&MGTM Digital Biomarkers in Monitoring Generalized Myasthenia Gravis Symptoms: The DOMYA Study* |
Presentation Details Poster Presentation Abstract #2859 21 April 2026 05:00 - 06:00 PM CT |
Lead Author Barnett-Tapia, C. |
Abstract Title ME&MGTM Digital Biomarkers Correlate with Equivalent MG-ADL and MG-QoL15r Sub-items Scores* |
Presentation Details Poster Presentation Abstract #2851 21 April 2026 05:00 - 06:00 PM CT |
Lead Author Yungher, B. |
Abstract Title The Phase 4 OCTAGON Study Investigating Oral Corticosteroid Tapering in Adult Patients With Generalized Myasthenia Gravis (gMG) Treated With Ravulizumab: Trial in Progress |
Presentation Details Poster Presentation Abstract #2146 21 April 2026 05:00 - 06:00 PM CT |
Lead Author Nowak, R. |
Abstract Title Concomitant Immunosuppressive Therapy Use With Ravulizumab: Analysis of the Global MG SPOTLIGHT Registry (Encore) |
Presentation Details Poster Presentation Abstract #1761 21 April 2026 05:00 - 06:00 PM CT |
Lead Author Shugars, C. |
Abstract Title Patient Satisfaction With Ravulizumab Treatment for Generalized Myasthenia Gravis (gMG) in the United States (US) |
Presentation Details Poster Presentation Abstract #1868 22 April 2026 11:45 AM - 12:45 PM CT |
Lead Author Snook, R. |
Abstract Title Impact on Healthcare Resource Utilization in Early Initiators of Ravulizumab or Efgartigimod for Treatment of Generalized Myasthenia Gravis in the USA (Encore) |
Presentation Details Poster Presentation Abstract #1694 22 April 2026 11:45 AM - 12:45 PM CT |
Lead Author Mahler, J. |
Abstract Title Real-World Biologics in Generalized Myasthenia Gravis: Evidence for Reduced Hospitalization Rates (Encore) |
Presentation Details Poster Presentation Abstract #3312 22 April 2026 11:45 AM - 12:45 PM CT |
Lead Author NF1 |
||
Lead Author Lyons, G. |
Abstract Title Effect of Selumetinib Treatment on Long-Term Prescription Pain Medication Utilization in Adults: A Retrospective Study of a US Claims Database |
Presentation Details Oral Presentation 006 Abstract #4173 20 April 2026 02:00 PM CT |
Lead Author Swampillai, A. |
Abstract Title Adult Patients' Experiences with Selumetinib Treatment Versus Placebo for Neurofibromatosis Type 1-Plexiform Neurofibroma Associated Symptoms and Their Impacts: A Qualitative Sub-Study of a Phase 3, Placebo-Controlled Trial (KOMET) |
Presentation Details Oral Presentation 007 Abstract #3807 20 April 2026 02:12 PM CT |
Lead Author NMOSD |
||
Lead Author Pittock, S. |
Abstract Title Gene Expression Signatures Associated With Prior Rituximab Treatment in Patients With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) in the CHAMPION-NMOSD Trial |
Presentation Details Oral Presentation 007 Abstract #2164 19 April 2026 02:12 PM CT |
Lead Author Sotirchos, E. |
Abstract Title Real-World Clinical Outcomes With Eculizumab and Ravulizumab in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Results From the Global NMO SPOTLIGHT Registry (Encore) |
Presentation Details Oral Presentation 009 Abstract #3238 19 April 2026 02:36 PM CT |
Lead Author Wruble, M. |
Abstract Title The Predictive Value of Billing Codes and Treatment Data to Identify Relapse Hospitalizations among Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) (Encore) |
Presentation Details Poster Presentation Abstract #4999 19 April 2026 08:00 - 09:00 AM CT |
Lead Author Sato, W. |
Abstract Title A real-world study of the effectiveness and safety of ravulizumab in AQP4-Ab+ NMOSD patients with suboptimal response to satralizumab in Japan - interim analysis (Encore) |
Presentation Details Poster Presentation Abstract #3426 19 April 2026 05:00 - 06:00 PM CT |
Lead Author Bernitsas, E. |
Abstract Title Financial and Mental Health Burden on Caregivers of Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD) in the United States |
Presentation Details Poster Presentation Abstract #2135 19 April 2026 05:00 - 06:00 PM CT |
Lead Author Okuda, D. |
Abstract Title Clinical and Radiological Outcomes in People with Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder Following Ravulizumab Treatment (AMAZE) |
Presentation Details Poster Presentation Abstract #4722 20 April 2026 08:00 - 09:00 AM CT |
Lead Author Conway, D. |
Abstract Title Latent Class Analysis (LCA) of Treatment Preferences Among Adults With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) in the United States |
Presentation Details Poster Presentation Abstract #2092 20 April 2026 05:00 - 06:00 PM CT |
Lead Author Bhattacharyya, S. |
Abstract Title Quantifying the Lifetime Health and Societal Benefits From Earlier Diagnosis and Access to Approved Targeted Immunotherapies in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) in the United States |
Presentation Details Poster Presentation Abstract #2080 21 April 2026 08:00 - 09:00 AM CT |
Lead Author Pittock, S. |
Abstract Title Long-term Efficacy and Safety of Ravulizumab in Anti-aquaporin-4 Antibody-positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Final Analysis of the Phase 3 CHAMPION-NMOSD Trial (Encore) |
Presentation Details Poster Presentation Abstract #1923 21 April 2026 08:00 - 09:00 AM CT |
*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/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.
