Alexion data at AAN 2024 demonstrate how Ultomiris and Soliris can transform outcomes for rare neurological diseases

New findings from CHAMPION-NMOSD trial will reinforce the potential for Ultomiris to eliminate relapses and improve care in AQP4 Ab+ NMOSD Long-term data and real-world evidence in gMG will underscore vital role of Ultomiris and Soliris in treatment landscape and show sustained patient benefit Alexion, AstraZeneca Rare Disease, will present new clinical and real-world data from its leading rare neurology portfolio at the American Academy of Neurology (AAN) Annual Meeting in Denver, CO, 13 to 18 April 2024. The company will present 14 abstracts, including five oral presentations, across both generalised myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Presentations include new long-term results from the pivotal Phase III CHAMPION-MG and CHAMPION-NMOSD trials, as well as real-world data, adding to the robust evidence supporting the safety and efficacy of Ultomiris (ravulizumab) and Soliris (eculizumab) in gMG and NMOSD. Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “ Ultomiris and Soliris bring innovation and hope to the gMG and NMOSD communities, offering treatment options with the potential to transform care for these debilitating diseases. Our data at AAN will showcase outcomes in both clinical and real-world settings that clearly demonstrate the sustained benefit of Ultomiris and Soliris in these patient populations. We remain committed to advancing care and innovative solutions for people living with these rare neurological conditions.” Continued evidence supporting long-term efficacy and safety of Ultomiris in NMOSD and gMG Two oral presentations will detail new findings on the long-term safety and efficacy of Ultomiris in adults with the most common forms of NMOSD and gMG. Long-term results from the ongoing global, open-label CHAMPION-NMOSD trial will demonstrate the potential for Ultomiris to eliminate relapses in people living with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) NMOSD. Data will show there were zero adjudicated on-trial relapses observed in Ultomiris -treated patients with AQP4 Ab+ NMOSD, with a median treatment duration of 138 weeks. Further, the final analysis from the global CHAMPION-MG open-label extension will underscore the benefits of sustained treatment of Ultomiris in patients with anti-acetylcholine receptor (AChR) Ab+ gMG. Improvements in measures of functional activities and quality of life, including Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) total scores, were maintained in Ultomiris -treated patients for up to 164 weeks. Real-world data highlight benefit of C5 inhibitors in gMG clinical practice An oral presentation will report results from a retrospective US-based medical record analysis, which suggests earlier treatment initiation with C5 inhibitor therapy offers greater clinical benefit for patients with gMG. While MG-ADL scores improved for patients who initiated Soliris early or late, greater improvements were observed among those who started treatment within two years of their gMG diagnosis. Two poster presentations will discuss steroid usage patterns and outcomes when used to treat chronic gMG, including after treatment with C5 inhibitors. A retrospective observational cohort study evaluating Medicare claims will indicate high rates of comorbidities among patients with gMG, which may inform clinical practice when prescribing corticosteroids. Additional medical claims data will show statistically significant reductions in the daily use of corticosteroids after 12 months of treatment with C5 inhibitors, as well as reductions in gMG exacerbations, supporting the use of C5 inhibitors as steroid-sparing therapy. Findings from a gMG global registry will also be shared, including an encore oral presentation that will show patients who have been treated with Soliris in clinical practice experience decreased rates of myasthenic crisis, exacerbations and hospitalisations. Uncovering new insights in NMOSD therapeutic efficacy An oral presentation will highlight results from a study evaluating specific biomarkers as measures of biological response to treatment with Ultomiris and Soliris from the global Phase III PREVENT and CHAMPION-NMOSD trials. The data will show glial fibrillary acidic protein (GFAP) and neurofilament light chain (NfL) levels, biomarkers for astrocyte and neuronal injury, decreased over time with C5 inhibitor treatment and may be additional indicators of therapeutic efficacy when evaluating treatments for AQP4 Ab+ NMOSD. Alexion presentations during AAN 2024 Lead Author Abstract Title Presentation Details NMOSD Pittock, SJ Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive NMOSD: interim analysis from the ongoing phase 3 CHAMPION-NMOSD trial Oral Presentation Oral presentation 003 S32: Autoimmune Neurology: NMOSD/MOGAD 17 April 2024 13:24 MDT Wingerchuk, DM Evaluation of glial fibrillary acidic protein (GFAP) and neurofilament light chain (NfL) levels during eculizumab and ravulizumab treatments in aquaporin-4-positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD) Oral Presentation Oral presentation 004 S32: Autoimmune Neurology: NMOSD/MOGAD 17 April 2024 13:36 MDT Clardy, SL Indirect treatment comparison of ravulizumab versus approved treatment options for adults with aquaporin-4 immunoglobulin positive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD) Poster Presentation Poster presentation 005 P10: Autoimmune Neurology: NMOSD 17 April 2024 11:45 – 12:45 MDT Levy, M Validation process of NMOSDCopilot TM , a software as a medical device (SaMD) for patients living with neuromyelitis optica spectrum disorder* Poster Presentation Poster presentation 001 P10: Autoimmune Neurology: NMOSD 17 April 2024 11:45 – 12:45 MDT gMG Vu, T Long-term efficacy and safety of ravulizumab, a long-acting terminal complement inhibitor, in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: final results from the Phase 3 CHAMPION MG open-label extension Oral Presentation Oral presentation 010 S15: Autoimmune Neuromuscular Diseases: New Observations and Therapeutic Approaches 15 April 2024 14:48 MDT Muppidi, S Effectiveness of eculizumab treatment by time from diagnosis in patients with generalized myasthenia gravis: a retrospective electronic medical record analysis Oral Presentation Oral presentation 006 S15: Autoimmune Neuromuscular Diseases: New Observations and Therapeutic Approaches 15 April 2024 14:00 MDT Tandan, R Rates of myasthenic crisis, exacerbation and healthcare resource utilization in eculizumab treated patients with generalized myasthenia gravis in a global registry Oral Presentation Oral presentation 001 S38: Autoimmune Neurology: Peripheral Autoimmunity, Paraneoplastic Disease, Checkpoint Inhibitors, and Neurosarcoidosis 17 April 2024 15:30 MDT McEneny, A Phase 1 study of gefurulimab pharmacokinetics (PK) and safety following delivery via autoinjector in healthy adults Poster Presentation Poster presentation 004 P10: Neuromuscular and Clinical Neurophysiology (EMG): Myasthenia Gravis 3 15 April 2024 11:45 – 12:45 MDT Lee, J Patterns of steroid use and outcomes in us patients with generalized myasthenia gravis (gMG) receiving C5 inhibitor therapy (C5IT) Poster Presentation Poster presentation 011 P10: Neuromuscular and Clinical Neurophysiology (EMG): Myasthenia Gravis 3 17 April 2024 11:45 – 12:45 MDT Juel, V Improvement in myasthenia gravis activities of daily living subdomain scores in patients treated with eculizumab: results from a generalized myasthenia gravis registry study Poster Presentation Poster presentation 008 P4: Neuromuscular and Clinical Neurophysiology (EMG): Myasthenia Gravis 1 15 April 2024 11:45 – 12:45 MDT Sabatella, G Effectiveness and safety of transitioning to ravulizumab from eculizumab in patients with generalized myasthenia gravis: evidence from a global registry Poster Presentation Poster presentation 002 S15: Autoimmune Neuromuscular Diseases: New Observations and Therapeutic Approaches 17 April 2024 11:45 – 12:45 MDT Blackowicz, M Comorbidity burden and steroid use in generalized myasthenia gravis: a retrospective analysis of Medicare fee-for-service claims Poster Presentation Poster presentation 015 P4: Neuromuscular and Clinical Neurophysiology (EMG): Myasthenia Gravis 1 15 April 2024 11:45 – 12:45 MDT Barnett Tapia, C First cross-sectional analysis from the ME&MG open study: a decentralized study on app-based myasthenia gravis* Poster Presentation Poster presentation 005 P4: Neuromuscular and Clinical Neurophysiology (EMG): Myasthenia Gravis 1 15 April 2024 11:45 – 12:45 MDT Berling, E ME&MG, novel digital device for patients with generalized myasthenia gravis: a first step towards validation* Poster Presentation Poster presentation 016 P1: Neuromuscular and Clinical Neurophysiology (EMG): New Tools in Neuromuscular Disease: Diagnosis and Assessment 14 April 2024 8:00 – 9:00 MDT *Ad Scientiam research study supported by Alexion Notes Ultomiris (ravulizumab) Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks in adult patients, following a loading dose. Ultomiris is approved in the US, EU, Japan and other countries for the treatment of certain adults with generalised myasthenia gravis (gMG). Ultomiris is also approved in the US, EU, Japan and other countries for the treatment of certain adults with paroxysmal nocturnal haemoglobinuria (PNH) and for certain children with PNH in the US and EU. Additionally, Ultomiris is approved in the US, EU, Japan and other countries for certain adults and children with atypical haemolytic uraemic syndrome to inhibit complement-mediated thrombotic microangiopathy (aHUS). Further, Ultomiris is approved in the US, EU and Japan for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD). As part of a broad development programme, Ultomiris is being assessed for the treatment of additional haematology and neurology indications. Soliris (eculizumab) Soliris (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells. Soliris is administered intravenously every two weeks, following an introductory dosing period. Soliris is approved in the US, EU, Japan, China and other countries for the treatment of patients with PNH and aHUS. Additionally, Soliris is approved in Japan and the EU for the treatment of certain adult and paediatric patients with gMG, and in the US, China and other countries for certain adults with gMG. Further, Soliris is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with NMOSD. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related haemolytic uraemic syndrome. Alexion Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for more than 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in 70 countries. AstraZeneca AstraZeneca (LSE/STO/Nasdaq: 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 operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca . Contacts For details on how to contact the Investor Relations Team, please click here . 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