Eculizumab

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. 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. For Media contacts, click here. Corporate and financial

权顺福（Soonwook Kwon）|徐俊宇（Junwoo Seo）|郑妍鹤（Yeon Hak Chung）|赵朱熙（Juhee Cho）|姜丹蓓（Danbee Kang）|闵柱洪（Ju-Hong Min） 韩国仁川仁荷大学医学院仁荷大学医院神经科 摘要 背景 视神经脊髓炎谱系疾病（NMOSD）可发生在老年人中，但传统免疫抑制疗法（ISTs）在该人群中的疗效和安全性仍不明确。 方法 我们利用韩国国家健康保险服务数据库（2011-2022年数据）进行了回顾性模拟试验。共构建了636个模拟试验，纳入了1020名开始接受IST治疗的患者和1965名未接受治疗的对照组患者（通过倾向得分匹配进行分组）。主要结局指标为复发率，安全性结局指标包括严重感染和其他不良事件。年龄分层分析以60岁为分界点进行预指定。 结果 接受IST治疗的患者年复发率低于未接受治疗的患者，但调整后的风险比（aHR）未达到统计学显著性（aHR 0.57，95% CI 0.28–1.19）。年龄分层分析显示，60岁以下患者的复发率有所下降，但60岁以上患者的复发率未见显著变化。随访期间，两组患者的严重感染总体风险相似（aHR 1.11，95% CI 0.84–1.46）。在开始IST治疗后的前6个月内，尤其是60岁以上患者的严重感染风险增加（aHR 5.80，95% CI 2.05–16.40）。 结论 现有证据不足以确定ISTs在老年NMOSD患者中的疗效是否降低。然而，IST的使用与早期严重感染风险增加相关，尤其是在60岁以上患者中，这表明对老年患者的治疗决策需要更加谨慎。 引言 视神经脊髓炎谱系疾病（NMOSD）是一种慢性炎症性脱髓鞘疾病，与水通道蛋白-4抗体相关，其特征是女性患者占多数，发病年龄中位数为30至45岁[1]。因此，大多数患者在年轻时就开始接受免疫抑制疗法（ISTs），可能需要长期治疗（可能持续数十年）[2]。然而，约29%的NMOSD患者在60岁或以上发病（晚发性NMOSD）[3]，2.3%的患者在70岁或以上发病（非常晚发性NMOSD）[4][5]。 与多发性硬化症和髓鞘少突胶质细胞糖蛋白抗体相关疾病不同，NMOSD以严重的复发和有限的残疾恢复为特征[1]。这些复发通常伴随着显著的功能障碍，因此制定预防疾病复发的策略至关重要。因此，早期开始IST治疗并考虑长期维持治疗对于降低复发风险和改善长期预后非常重要[6][7]。这种方法同样适用于晚发性NMOSD患者，后者常伴有广泛的脊髓受累和较差的临床预后[8]，因此也应在老年NMOSD患者中得到应用。 尽管ISTs在年轻患者中的疗效和安全性已得到较好验证，但在老年患者中的数据仍不明确。尽管一项近期未经控制的回顾性研究表明ISTs可能有助于降低复发率且安全性可接受[9]，但传统ISTs在老年NMOSD患者中的适用性仍不明确。最近，一些生物制剂（如inebilizumab、eculizumab、ravulizumab和satralizumab）已被批准用于NMOSD治疗，并在临床研究中显示出良好的疗效[9][10][11][12]。然而，由于成本和监管限制，韩国仍主要使用硫唑嘌呤（AZA）、霉酚酸酯（MMF）和利妥昔单抗（RTX）来治疗NMOSD。 因此，本研究根据年龄评估了ISTs的疗效和安全性，重点关注老年NMOSD患者。 数据来源 本研究使用韩国国家健康保险服务（K-NHIS）数据库（2011年1月1日至2022年12月31日的数据）进行。K-NHIS是韩国唯一的公共卫生服务提供者，该数据库包含个体层面的社会人口统计信息以及所有住院、门诊和急诊机构的诊断和医疗记录（如药物处方和医疗程序），数据丢失率较低[10]。 基线特征 接受IST治疗组和未接受IST治疗组的平均年龄分别为46.6±14.3岁和46.7±14.5岁，男性占比分别为19.8%和18.5%。两组在年龄、性别、居住地区、医疗保险类型和合并症方面均具有较好平衡性（表1）。总体而言，16.2%的研究对象年龄在60岁以上，56名患者（1.9%）年龄在18岁以下（其中非IST治疗组40名，IST治疗组16名）。从NMOSD诊断到开始IST治疗的 median 时间... 讨论 本研究评估了传统ISTs在NMOSD患者中的疗效和安全性，并探讨了这些效果随年龄的变化情况。虽然接受IST治疗的患者年复发率较低，但调整后的风险比未达到统计学显著性。在安全性方面，老年患者的严重感染风险较高，尤其是在开始IST治疗后的前6个月内，60岁以上患者的风险增加了5.80倍。 结论 我们的研究不足以得出ISTs在老年NMOSD患者中的疗效低于年轻患者的结论。相反，严重感染的风险明显增加，尤其是在治疗开始后的前6个月内，这一风险在60岁以上患者中更为显著。这些发现强调了在治疗初期密切监测感染的重要性，以便及时发现和处理感染问题。 作者贡献声明 权顺福（Soonwook Kwon）：撰写初稿、数据可视化、研究设计。 徐俊宇（Junwoo Seo）：撰写初稿、研究设计、数据分析、数据管理。 郑妍鹤（Yeon Hak Chung）：撰写初稿、研究设计、数据管理。 赵朱熙（Juhee Cho）：撰写与编辑、研究设计、数据管理。 姜丹蓓（Danbee Kang）：撰写与编辑、方法学设计、研究设计、数据分析、概念构建。 闵柱洪（Ju-Hong Min）：撰写与编辑、监督工作、研究设计、资金申请。 伦理审批和参与同意 本研究遵循《赫尔辛基宣言》进行，并获得了三星医疗中心（IRB编号：SMC-2023-05-135）的批准。由于本研究为回顾性研究且使用去标识化数据，因此无需患者签署知情同意书。 资助 本研究得到了韩国卫生福利部资助的“以患者为中心的临床研究协调中心（PACEN）”（项目编号：HC23C0249，资助对象：闵柱洪）以及三星医疗中心“未来医学2030项目”（项目编号：SMO126004，资助对象：闵柱洪）的支持。 利益冲突声明 闵柱洪（JH Min）获得了韩国国家研究基金会（MIST和KHIDI）和三星医疗中心研究与发展基金的支持。她曾为拜耳医疗保健、默克、Biogen Idec、赛诺菲、UCB、三星Bioepis、三菱田边、Celltrion、罗氏、詹森、阿斯利康和Neurophet Inc.等公司提供咨询并收取费用。权顺福（Soonwook Kwon）、徐俊宇（Junwoo Seo）、郑妍鹤（Yeon Hak Chung）、赵朱熙（Juhee Cho）和姜丹蓓（Danbee Kang）均无需要声明的利益冲突。 致谢 数据分析使用了韩国国家健康保险服务（NHIS）的大数据平台（NHIS-2023-1-763）的数据，使用前已获得该机构的审查和访问许可。