Alexion, AstraZeneca Rare Disease, will unveil a major slate of 20 presentations—including five oral sessions—at the American Academy of Neurology Annual Meeting in Chicago, Illinois, from 18–22 April 2026.

 

All by spotlighting new advances across generalized myasthenia gravis (gMG), neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN), and neuromyelitis optica spectrum disorder (NMOSD).

 

The programme features headline data from pivotal Phase III trials, including PREVAIL in gMG, KOMET in NF1-PN, and CHAMPION-NMOSD, alongside real-world evidence supporting established therapies.

 

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs at 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.”

 

A key oral presentation from the global PREVAIL Phase III study evaluates gefurulimab as a once-weekly, self-administered subcutaneous therapy in adults with AChR antibody-positive gMG.

 

Results show a statistically significant and clinically meaningful improvement in Myasthenia Gravis Composite scores at Week 26 versus placebo, reinforcing the potential of at-home complement inhibition in a disease with significant treatment burden.

 

New data from the KOMET Phase III programme highlights meaningful patient-reported improvements in adults with NF1-PN treated with Koselugo (selumetinib).

 

Patients reported sustained reductions in pain, tingling, tumour burden symptoms, and improvements in sleep, fatigue, and mobility. A separate US claims analysis also shows long-term reductions in prescription pain medication use over three years among treated patients

 

Multiple presentations from the CHAMPION-NMOSD programme and real-world registries deepen understanding of disease biology and treatment impact in AQP4-Ab+ NMOSD.

 

Key findings include transcriptomic insights, long-term efficacy data, and real-world outcomes supporting C5 inhibition therapies such as Ultomiris (ravulizumab).

 

Additional studies explore relapse prediction, caregiver burden, and the societal value of earlier diagnosis and treatment access.

 

Across the meeting, Alexion will present data spanning digital biomarkers, corticosteroid tapering strategies, healthcare utilisation, and patient satisfaction in gMG, alongside registry and outcomes research across NMOSD and NF1-PN.

 

The breadth of presentations underscores a coordinated push across rare neurological diseases—linking pipeline innovation with real-world evidence to support long-term disease management.