TARRYTOWN, N.Y., Aug. 26, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the primary and key secondary endpoints were met in the Phase 3 NIMBLE trial assessing investigational cemdisiran monotherapy in adults with generalized myasthenia gravis (gMG). Cemdisiran is an siRNA that reduces circulating levels of complement factor 5 (C5) and, as monotherapy in this trial, was associated with an average of 74% inhibition of complement activity.

纽约州塔里敦，2025年8月26日（环球新闻社）——再生元制药公司（纳斯达克股票代码：REGN）今天宣布，在评估成人全身性重症肌无力（gMG）患者使用西地昔兰单药治疗的3期NIMBLE试验中，主要终点和关键次要终点均已达成。西地昔兰是一种小干扰RNA（siRNA），可降低补体因子5（C5）的循环水平，在本试验中作为单药治疗，平均抑制了74%的补体活性。

The trial also assessed a combination of cemdisiran and pozelimab, a C5 antibody; this combination (“cemdi-poze”), which resulted in nearly 99% inhibition of complement activity, also met the primary and key secondary endpoints, though cemdisiran monotherapy was numerically better across these endpoints..

该试验还评估了cemdisiran与C5抗体pozelimab的组合；这个组合（“cemdi-poze”）导致了近99%的补体活性抑制，同样达到了主要和关键次要终点，尽管在这些终点上，cemdisiran单药治疗在数值上表现更好。

“Our pipeline approach to treating complement-mediated diseases allows us to tailor treatment to the underlying disease biology,” said L. Andres Sirulnik, M.D., Ph.D., Senior Vice President and Hematology Clinical Development Unit Head at Regeneron. “The results of the NIMBLE trial confirm that, in myasthenia gravis, robust efficacy can be achieved without complete complement blockade, whereas in other diseases such as paroxysmal nocturnal hemoglobinuria (PNH), complete inhibition is likely to be necessary.

“我们针对补体介导疾病的管线方法使我们能够根据潜在的疾病生物学特性定制治疗方案，”再生元公司高级副总裁兼血液学临床开发部门负责人L. Andres Sirulnik医学博士、哲学博士说道。“NIMBLE试验的结果证实，在重症肌无力中，即使不完全阻断补体也能实现显著的疗效，而在其他疾病如阵发性夜间血红蛋白尿症（PNH）中，完全抑制可能才是必要的。”

We have previously released data from the lead-in portion of our PNH Phase 3 trial, supporting the potential for the cemdi-poze combination to deliver best-in-class efficacy in PNH. We are also investigating systemic administration of both cemdisiran monotherapy and the cemdi-poze combination in our Phase 3 program for geographic atrophy secondary to age-related macular degeneration.”.

我们之前已经发布了我们PNH第三阶段试验引导部分的数据，支持cemdi-poze组合在PNH中提供最佳疗效的潜力。我们还在我们的年龄相关性黄斑变性继发地理萎缩的第三阶段项目中，研究cemdisiran单药治疗和cemdi-poze组合的全身给药。

“The NIMBLE trial results underscore the potential for cemdisiran to offer a best-in-class profile for those suffering with myasthenia gravis, providing for robust efficacy with a convenient quarterly subcutaneous administration,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron.

“NIMBLE 试验结果强调了 cemdisiran 有望为重症肌无力患者提供最佳的治疗效果，通过每季度一次的皮下注射实现强劲的疗效，”再生元公司董事会联合主席、总裁兼首席科学官 George D. Yancopoulos 医学博士表示。

“The potential for best-in-class efficacy with less than complete complement blockade with cemdisiran monotherapy may also provide for a more favorable safety profile. These exciting results highlight the transformative potential of our siRNA and genetic medicines pipeline to deliver paradigm-changing therapies for patients.”.

“Cemdisiran单药治疗可能在不完全补体阻断的情况下实现同类最佳疗效，同时还可能提供更有利的安全性。这些令人兴奋的结果突显了我们的siRNA和基因药物研发管线在为患者提供变革性疗法方面的巨大潜力。”

The NIMBLE trial evaluated adults with symptomatic gMG who have antibodies to the acetylcholine receptor (anti-AChR) and may be receiving standard of care immunosuppressants based on the investigator’s discretion. Patients were randomized to receive subcutaneous administrations of: cemdisiran (600 mg) every 12 weeks, cemdi-poze (cemdisiran 200 mg and pozelimab 200 mg) every 4 weeks, or placebo every 4 weeks.

NIMBLE 试验评估了具有乙酰胆碱受体抗体（抗 AChR）且可能根据研究者判断接受标准免疫抑制治疗的症状性 gMG 成人患者。患者被随机分配接受以下皮下注射治疗：每 12 周一次 cemdisiran（600 毫克）、每 4 周一次 cemdi-poze（cemdisiran 200 毫克和 pozelimab 200 毫克），或每 4 周一次安慰剂。

The primary endpoint assessed total score changes from baseline to week 24 in the Myasthenia Gravis Activities of Daily Living (MG-ADL) total score, a patient-reported questionnaire that measures daily functions impacted by gMG, such as talking, eating, breathing, vision and mobility. The key secondary endpoint assessed total score changes from baseline in the Quantitative Myasthenia Gravis (QMG) total score, a physician-administered assessment evaluating vision, speaking/swallowing, breathing and limb function..

主要终点评估了从基线到第24周重症肌无力日常生活活动（MG-ADL）总分的总分变化，MG-ADL是一份患者报告的问卷，用于衡量受gMG影响的日常功能，如说话、进食、呼吸、视力和行动能力。关键次要终点评估了定量重症肌无力（QMG）总分从基线开始的总分变化，QMG是由医生进行的评估，用于评价视力、说话/吞咽、呼吸和肢体功能。

Historical clinical trial data report that currently approved C5 inhibitor therapies have shown a placebo-adjusted treatment difference in MG-ADL total scores ranging from -1.6 to -2.1 at 12 to 26 weeks.

历史临床试验数据显示，目前获批的C5抑制剂疗法在12至26周内，MG-ADL总分相较于安慰剂组的治疗差异范围为-1.6到-2.1。

Both cemdisiran and cemdi-poze demonstrated improvements in activities of daily functioning at week 24, with cemdisiran showing numerically better results across all gMG-specific outcomes. In the MG-ADL and QMG, greater reductions in total scores indicate greater improvement in disease symptoms and better treatment effect..

在第24周，cemdisiran和cemdi-poze均显示出日常活动功能的改善，其中cemdisiran在所有特定于gMG的结果上表现出数值上更好的结果。在MG-ADL和QMG中，总分的更大减少表明疾病症状的更大改善和治疗效果更好。

Means and relative risks were adjusted per the amount immunosuppressant treatment and the Myasthenia Gravis Foundation of America Clinical Classification determined at screening. Means were also adjusted for baseline MG-ADL or QMG score.

根据免疫抑制剂治疗量和美国重症肌无力协会临床分类（在筛查时确定）对均值和相对风险进行了调整。均值还根据基线MG-ADL或QMG评分进行了调整。

There were no meningococcal infections in any patient. There were no treatment discontinuations due to adverse events through week 24 in the cemdisiran arm.

任何患者均未发生脑膜炎球菌感染。在第24周之前，cemdisiran组没有因不良事件导致的治疗中断。

Across all arms, treatment-emergent adverse events (TEAEs) occurred in 69% of patients treated with cemdisiran, 81% with cemdi-poze, and 77% with placebo. Serious TEAEs occurred in 3% of patients treated with cemdisiran, 9% with cemdi-poze and 14% with placebo. The most common TEAEs observed in ≥5% of patients receiving cemdisiran, cemdi-poze or placebo were: worsening of MG (1%, 5%, 17%), upper respiratory tract infection (12%, 8%, 11%), urinary tract infection (5%, 6%, 3%), nasopharyngitis (5%, 3%, 4%), headache (5%, 11%, 10%), rash (5%, 3%, 1%), injection site reaction (4%, 8%, 1%), diarrhea (3%, 14%, 7%), arthralgia (1%, 6%, 1%), pain in extremity (1%, 5%, 1%), cough (1%, 5%, 1%), and pruritus (0%, 5%, 0%).

在所有组中，接受 cemdisiran 治疗的患者中有 69% 发生了治疗中出现的不良事件 (TEAE)，cemdi-poze 组为 81%，安慰剂组为 77%。严重 TEAE 在接受 cemdisiran 治疗的患者中发生率为 3%，cemdi-poze 组为 9%，安慰剂组为 14%。在接受 cemdisiran、cemdi-poze 或安慰剂治疗的患者中，最常见的（≥5% 的患者）TEAE 包括：重症肌无力 (MG) 加重（1%、5%、17%）、上呼吸道感染（12%、8%、11%）、尿路感染（5%、6%、3%）、鼻咽炎（5%、3%、4%）、头痛（5%、11%、10%）、皮疹（5%、3%、1%）、注射部位反应（4%、8%、1%）、腹泻（3%、14%、7%）、关节痛（1%、6%、1%）、四肢疼痛（1%、5%、1%）、咳嗽（1%、5%、1%）和瘙痒（0%、5%、0%）。

There were no deaths during the 24-week placebo-controlled portion of the trial. During the extension period, one death due to pneumonia occurred in the cemdisiran arm, and one death due to septic shock occurred in the combination arm; both deaths occurred in patients who were on concomitant immuno-suppressive therapies..

在试验的24周安慰剂对照阶段没有发生死亡。在延长期期间，cemdisiran组有一例因肺炎导致的死亡，联合用药组有一例因脓毒性休克导致的死亡；这两例死亡均发生在同时接受免疫抑制治疗的患者中。

Detailed results from the NIMBLE trial will be presented at an upcoming medical meeting. The U.S. regulatory application for cemdisiran is planned for the first quarter of 2026, pending discussions with the FDA.

NIMBLE 试验的详细结果将在即将召开的医学会议上公布。Cemdisiran 的美国监管申请计划于 2026 年第一季度进行，具体时间待与 FDA 讨论后确定。

The potential use of cemdisiran and/or pozelimab for the treatment of gMG is investigational and has not been approved by any regulatory authority. In the U.S., pozelimab monotherapy is approved as Veopoz

Cemdisiran 和/或 pozelimab 用于治疗 gMG 的潜在用途尚在研究中，且尚未获得任何监管机构的批准。在美国，pozelimab 单药疗法已作为 Veopoz 获得批准。

(pozelimab-bbfg) for adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy, which includes a Boxed Warning for life-threatening and fatal meningococcal infections.

(pozelimab-bbfg) 适用于1岁及以上患有CHAPLE病（也称为CD55缺陷性蛋白丢失性肠病）的成人和儿童患者，该药物附有针对危及生命和致命性脑膜炎球菌感染的黑框警告。

About Myasthenia Gravis (MG)

关于重症肌无力 (MG)

MG is a rare and chronic autoimmune disease where abnormal antibodies activate the complement system including C5, disrupting communication between nerves and muscles that results in debilitating and potentially life-threatening muscle weakness. In the U.S., the disease impacts approximately 85,000 people.

MG是一种罕见的慢性自身免疫性疾病，异常抗体激活包括C5在内的补体系统，破坏神经与肌肉之间的通信，导致严重且可能危及生命的肌肉无力。在美国，该疾病影响约85,000人。

Initial manifestations are usually ocular, but approximately 85% of MG patients experience additional advancements to the disease manifestations, which is known as generalized myasthenia gravis (gMG). For these patients, the disease affects muscles throughout the body, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing and mobility.

初始症状通常是眼部症状，但大约85%的重症肌无力（MG）患者会经历疾病表现的进一步发展，这被称为全身型重症肌无力（gMG）。对于这些患者，疾病会影响全身的肌肉，导致极度疲劳，并出现面部表情、说话、吞咽和行动方面的困难。

Treatment-related challenges – which include frequent hospital visits, inconsistent symptom control, and lack of durable treatment effects – can further affect quality of life and long-term disease management..

治疗相关的挑战——包括频繁的医院就诊、症状控制不稳定以及缺乏持久的治疗效果——会进一步影响生活质量和长期疾病管理。

About the Complement Factor 5 (C5)

关于补体因子5（C5）

Clinical Program

临床项目

NIMBLE is a randomized, double-blind, placebo-controlled trial evaluating cemdisiran and cemdi-poze in patients with gMG who have antibodies for AChR. The primary endpoint assessed changes in the MG-ADL total score from baseline to week 24. The MG-ADL scale is an eight-question patient-reported tool that measures how gMG affects aspects of daily life and provides a total score ranging from 0 to 24.

NIMBLE是一项随机、双盲、安慰剂对照试验，评估了cemdisiran和cemdi-poze在具有AChR抗体的gMG患者中的疗效。主要终点评估了从基线到第24周MG-ADL总分的变化。MG-ADL量表是一个包含八个问题的患者报告工具，用于衡量gMG如何影响日常生活的各个方面，并提供从0到24的总分。

The key secondary endpoint was the change from baseline in QMG total score at week 24. The QMG is a physician-administered 13-item standardized assessment evaluating muscle function that provides a total score ranging from 0 to 39. In both MG-ADL and QMG, a higher total score indicates greater disease severity, and a larger reduction in total score indicates greater improvement in disease symptoms and better treatment effect..

关键的次要终点是第24周时QMG总分从基线的变化。QMG是由医生进行的包含13个项目的标准化评估，用于评估肌肉功能，总分范围为0到39。在MG-ADL和QMG中，总分越高表示疾病越严重，而总分减少越多则表示疾病症状改善越大且治疗效果更好。

Cemdisiran and pozelimab are also being evaluated in separate Phase 3 trials as both monotherapy and combination therapy for additional complement-mediated disorders, including PNH and geographic atrophy secondary to age-related macular degeneration. For more information, visit the Regeneron clinical trials website, or contact clinicaltrials@regeneron.com or +1 844-734-6643..

Cemdisiran和pozelimab也正在单独的III期临床试验中作为单一疗法和联合疗法进行评估，用于治疗其他补体介导的疾病，包括阵发性睡眠性血红蛋白尿症（PNH）和与年龄相关性黄斑变性继发的地理萎缩。欲了解更多信息，请访问Regeneron临床试验网站，或联系clinicaltrials@regeneron.com或+1 844-734-6643。

Cemdisiran as a monotherapy and in combination with pozelimab was being developed under an initial agreement with Alnylam Pharmaceuticals, Inc. In June 2024, Regeneron and Alnylam entered into an amended and restated C5 License Agreement, which granted Regeneron a worldwide license to cemdisiran as a monotherapy in addition to a license to cemdisiran in combination with C5 antibodies.

Cemdisiran作为单药疗法并与pozelimab联合使用时，最初是根据与Alnylam Pharmaceuticals, Inc.的协议进行开发的。2024年6月，Regeneron和Alnylam签署了一份修订和重述的C5许可协议，该协议授予Regeneron在全球范围内将cemdisiran作为单药疗法的许可，同时也包括其与C5抗体联合使用的许可。

Regeneron is solely responsible for development, manufacturing, and commercialization of cemdisiran as a monotherapy and in combination with C5 antibodies. For cemdisiran as a monotherapy, Alnylam is entitled to receive certain regulatory milestone payments as well as tiered, double-digit royalties (up to 15%) on calendar-year net sales.

再生元全权负责cemdisiran作为单药疗法及与C5抗体联合使用的开发、生产和商业化。对于cemdisiran作为单药疗法，Alnylam有权获得某些监管里程碑付款，以及按年净销售额计算的分层两位数版税（最高可达15%）。

If cemdisiran is used as part of a combination product, Alnylam is entitled to receive a flat, low double-digit royalty on calendar-year net sales as well as commercial milestones of up to $325.0 million..

如果Cemdisiran作为组合产品的一部分使用，Alnylam有权获得固定的、较低的两位数比例的年度净销售额特许权使用费，以及高达3.25亿美元的商业里程碑付款。

About Regeneron

关于再生元

Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories.

Regeneron（纳斯达克代码：REGN）是一家领先的生物技术公司，致力于为患有严重疾病的患者发明、开发和商业化改变生命的药物。公司由医生科学家创立并领导，我们独特的能力在于能够反复且持续地将科学转化为医学成果，目前已推出众多获批的治疗方法，同时还有多个在研产品候选药物，其中大部分都是在我们实验室自主研发的。

Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases..

我们的药物和研发管线旨在帮助患有眼疾、过敏性和炎症性疾病、癌症、心血管和代谢疾病、神经系统疾病、血液病、传染病和罕见病的患者。

Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as

再生元利用我们的专有技术，如某些技术，推动科学发现的边界并加速药物开发。

VelociSuite

速度套件

, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center

，该公司生产优化的全人源抗体和新型双特异性抗体。我们正在利用再生元遗传学中心的数据驱动洞察力，塑造医学的下一个前沿领域。

and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.

并开创基因医学平台，使我们能够识别创新靶点和互补方法，以潜在治疗或治愈疾病。