A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation

A PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN ADULTS AT HIGH RISK OF DELAYED GRAFT FUNCTION (DGF) FOLLOWING KIDNEY ALLOGRAFT TRANSPLANTATION

开始日期2025-09-01

申办/合作机构

Apellis Pharmaceuticals, Inc.

NCT07160179

/ Recruiting临床1/2期

Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy of ABBV-6628 in Subjects With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Age-related macular degeneration (AMD) is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. Geographic Atrophy (GA) is an advanced form of dry AMD. The purpose of this study is to assess the adverse events and how intravitreal ABBV-6628 moves through the body of adult participants with secondary to age-related macular degeneration
ABBV-6628 is an investigational monoclonal antibody fragment being developed for the treatment of geographic atrophy (GA) secondary to (AMD) age-related macular degeneration. Participants in the Stage 1 part will be placed in 1 of 4 groups, called treatment arms. Participants in Stage 2 will be placed into 1 of 2 groups. Each group receives different treatment. Adult participants aged 50 and older years with a diagnosis GA secondary to age-related macular degeneration will be enrolled. Around 66 participants will be enrolled in the study at approximately 27 sites across the US.
Participants in Stage 1 will be given ABBV-6628 as an intravitreal injection (injection into the jelly-like tissue that fills the eyeball injection) with dose escalation. Participants in Stage 2 will receive ABBV-6628 or SYFOVRE, an approved treatment for geographic atrophy, administered as per the FDA-approved label. The treatment duration is approximately 22 months and 3 months of follow-up.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular weekly visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

开始日期2025-08-13

申办/合作机构

AbbVie, Inc.

NCT06961370

/ Recruiting临床1期

A Phase I, Multi-center, Multi-part Study to Investigate Safety, Tolerability, PK, PD, and Immunogenicity of RO7669330 Intravitreal Injections in Participants With GA Secondary to AMD: Part 1A: Open-label, MAD; Part 1B: Randomized PD Pilot; Part 2: Masked, Randomized, Active-comparator-controlled

The main purpose of this study is to assess the ocular and systemic safety and tolerability of RO7669330 in GA secondary to AMD after multiple unilateral intravitreal (IVT) doses.

开始日期2025-07-16

申办/合作机构

Roche Holding AG

100 项与 Pegcetacoplan 相关的临床结果

登录后查看更多信息

100 项与 Pegcetacoplan 相关的转化医学

登录后查看更多信息

100 项与 Pegcetacoplan 相关的专利（医药）

登录后查看更多信息

202

项与 Pegcetacoplan 相关的文献（医药）

2025-09-01·RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

SILICONE OIL MICRODROPLETS FROM SYRINGES WITH INTRAVITREAL ANTI–VASCULAR ENDOTHELIAL GROWTH FACTOR AND COMPLEMENT INHIBITOR INJECTIONS

Article

作者: Wei, Libby ; Thompson, John T. ; Fan, Xiaoxuan ; Lee, Erica

Purpose::

To quantify the amount of silicone oil in intravitreal anti–vascular endothelial growth factor and complement inhibitor injections in prefilled and commercial syringes (silicone-lubricated and silicone-free).

Methods::

Patients deemed clinically appropriate to receive one of five intravitreal medications received the recommended dose. Syringes were overfilled such that 0.01-0.04 mL remained after injection for analysis. Aflibercept and ranibizumab were available in prefilled syringes. Pegcetacoplan, faricimab, and avacincaptad pegol were prepared with Becton Dickinson polycarbonate (BD) syringes. Faricimab was also prepared in Ocuject VitreJect (OV) silicone-free syringes. Samples were analyzed using spectral flow cytometry.

Results::

Silicone oil was found in all syringes. The lowest amounts of silicone oil were found in the Mili-Q water in OV syringes (4.02 ± 0.3 particles per microliter) and ranibizumab 0.5 mg prefilled syringes (8.33 ± 3.1). Intermediate levels of silicone oil were detected in aflibercept prefilled syringes (35.5 ± 21.8) and faricimab in OV syringes (74.6 ± 10.1), significantly greater than Mili-Q water in OV syringes and ranibizumab. Significantly higher levels of silicone oil were found with faricimab (324.9 ± 30), pegcetacoplan (378.1 ± 379.6), and avacincaptad pegol (113.5 ± 14) in BD syringes than the low or intermediate groups. The filter needles used to draw up medications from vials likely contributed to the amount of silicone oil detected in the samples.

Conclusion::

Commonly used BD polycarbonate syringes for intravitreal injections caused significantly higher levels of silicone oil to be injected into the eye than prefilled or silicone-free syringes. Repeated injections with more viscous intravitreal drugs increase the amount of silicone oil in the vitreous, leading to symptomatic floaters.

2025-09-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

Visual Acuity and Quality of Life Outcomes With Pegcetacoplan Treatment: A Post Hoc Analysis From the OAKS and DERBY Trials

Article

作者: Jones, Daniel L ; Baumal, Caroline R ; Intorcia, Michele ; McKeown, Alex ; Sarda, Sujata P ; Sheidow, Tom ; Garg, Sunir ; Stevens, Warren ; Davis, Matthew ; Chiang, Allen ; Schmidt-Erfurth, Ursula M

OBJECTIVE:

To evaluate the effect of pegcetacoplan on visual function and patient quality of life (QoL) measures based on distance of geographic atrophy (GA) lesions from the center of the fovea.

DESIGN:

Post hoc analysis from OAKS and DERBY, two global, 24-month, multicenter, randomized, double-masked, sham-controlled phase 3 studies evaluating pegcetacoplan treatment for GA in age-related macular degeneration (AMD).

PARTICIPANTS:

888 study patients with GA for whom all data necessary for the post hoc analysis to be performed had been collected were included.

METHODS:

Best-corrected visual acuity (BCVA) and 25-item National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25) were assessed at baseline and every 4 and 6 months, respectively, to completion at month 24 in both pegcetacoplan-treated eyes and sham (observed) eyes. Eyes were stratified based on optical coherence tomography‒derived GA lesion location ≥250 µm (n = 192) or <250 µm (n = 696) from the foveal center.

MAIN OUTCOME MEASURES:

Change from baseline in BCVA and NEI VFQ-25 was evaluated over 24 months in GA lesions based on distance (≥250 or <250 µm) from the foveal center. Adjustment was conducted via propensity score weighting, where model specification and baseline covariate selection were done a priori based on clinical rationale.

RESULTS:

Pegcetacoplan-treated eyes with GA lesion margins ≥250 µm from the foveal center demonstrated directionally slower decline in visual acuity (mean +5.6 [SE 3.2] [P = .0785]) and QoL (mean +4.0 [SE 2.4] [P = .0905]) from baseline at 24 months compared with sham. The change in visual acuity (BCVA, mean -1.6 [SE 1.1] [P = .1522]) and QoL (NEI VFQ-25, -2.3 [1.1] [P = .0284]) in pegcetacoplan-treated eyes with GA lesion margins <250 µm from the foveal center were within the limits of variability compared with sham.

CONCLUSIONS:

Pegcetacoplan treatment demonstrated a potentially meaningful slower decline in visual acuity and QoL for patients with GA lesions ≥250 µm from the foveal center. The change in visual acuity and QoL seen with pegcetacoplan treatment for patients with GA lesions <250 µm from the foveal center were within the limits of variability.

2025-09-01·ADVANCES IN THERAPY

Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan

Article

作者: de Castro, Carlos ; Höchsmann, Britta ; Mulherin, Brian ; Wong, Raymond Siu Ming ; Kelly, Richard J ; de Latour, Regis Peffault ; Selvaratnam, Veena ; Griffin, Morag ; Patriquin, Christopher J ; Uchendu, Uchendu O ; Szamosi, Johan ; Surova, Elena ; Horneff, Regina ; Zhang, Yiwei ; Hillmen, Peter

INTRODUCTION:

Pegcetacoplan, the first C3 and C3b inhibitor for paroxysmal nocturnal hemoglobinuria (PNH), demonstrated efficacy and safety in C5 inhibitor-experienced and -naive patients in the phase 3 studies PEGASUS (NCT03500549) and PRINCE (NCT04085601), respectively. This integrated analysis of PEGASUS, PRINCE, and their subsequent open-label extension study (NCT03531255) evaluated pegcetacoplan long-term efficacy and safety.

METHODS:

Efficacy was assessed from pegcetacoplan initiation through 2.5 years (PRINCE) and 3 years (PEGASUS) by measuring hemoglobin, lactate dehydrogenase (LDH), absolute reticulocyte count (ARC), indirect bilirubin, and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores. Transfusion avoidance and safety were assessed during pegcetacoplan monotherapy. Of 133 patients in PRINCE and PEGASUS, 114 enrolled in the extension. Most patients (PRINCE, 81.1%; PEGASUS, 75.0%) were transfusion dependent at trial entry and baseline hemoglobin was below normal.

RESULTS:

Pegcetacoplan markedly and rapidly (within 4 weeks) improved all efficacy measures, which stabilized close to (hemoglobin, FACIT-Fatigue) or within (LDH, ARC, indirect bilirubin) normal range for up to 3 years; annual transfusion avoidance rates were 79.5-86.4% in PRINCE and 71.2-79.2% in PEGASUS. No new safety concerns were identified over 3 years. Serious adverse events were reported in 73 (55.3%) patients, deemed pegcetacoplan related in 6 (4.5%) patients. Most injection site reactions were mild, and their incidence decreased over time. Five (3.8%) deaths occurred (none deemed pegcetacoplan related); 37 (28.0%) patients experienced clinically significant and laboratory-confirmed breakthrough hemolysis; 4 thrombotic events occurred in 3 (2.3%) patients; no meningitis cases were reported.

CONCLUSION:

Pegcetacoplan efficacy and safety was sustained for up to 3 years in patients with PNH, with or without prior C5 inhibitor treatment, verifying the long-term favorable clinical profile of this proximal complement inhibitor.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT03500549 (PEGASUS), NCT04085601 (PRINCE), and NCT03531255 (307 OLE).

441

项与 Pegcetacoplan 相关的新闻（医药）

2025-08-20

·美迪西Medicilon

美迪西专访 | 瞄准百亿美元市场，领诺医药在补体抑制剂领域更进一步

2025年8月18日，领诺医药自主研发新一代补体抑制剂产品SLN12140获得国家药品监督管理局（CDE）通知，批准用于“补体参与介导的溶血性疾病包括但不限于阵发性睡眠性血红蛋白尿症PNH、补体介导的血栓性微血管病TMA、镰刀细胞病SCD等”的临床试验。这是该产品继2025年2月、2024年5月该产品分别获得CDE、FDA批准用于IgA肾病的临床试验之后，获得监管机构对第二类适应症的批准。作为领诺医药长期的合作伙伴，美迪西有幸参与了SLN12140的临床前研究。今天邀请领诺医药创始人&CEO韩照中博士带我们一起走进这款药物，了解创新成果背后的故事。韩照中博士领诺医药创始人&CEO 1991年毕业于武汉大学病毒学与分子生物学系，1997获军事医学科学院医学遗传学博士学位。2002年完成美国国家卫生研究院肿瘤研究所（NIH， NCI）新药研发博士后训练后，任职于能源部国家实验室生物部，从事抗体发现与工程改造研究。2004年任Vanderbilt University医学院肿瘤生物学助理教授，在联邦基金的支持下进行脑胶质瘤的基础与转化医学研究。2013年加入Alexion Pharmaceuticals公司，参与或主导补体靶向、针对严重罕见病的孤儿药研发。2017年底回国负责睿智化学（ChemPartner）抗体发现与工程改造平台。2019年8月于上海自由贸易区张江药谷注册成立领诺（上海）医药科技有限公司，开展基于补体靶向、穿越血脑屏障的大分子药物递送技术、罕见病孤儿药的创新药物研发。 Q1 美迪西：韩博您好，首先祝贺领诺医药的 SLN12140 近日获得 CDE 的临床试验批准，这无疑是一个重大的里程碑。能否和我们分享一下这个消息带来的感受？韩照中博士：这个消息确实让整个团队都非常振奋。首先要感谢团队的不懈努力，也要感谢投资人、合作方的大力支持。这个批准不仅是对我们转化能力和研发质量的认可，更重要的是，它为早日推进这款新一代补体抑制剂进入医药市场，为病患提供更安全、更有效、更方便用药的治疗手段提供了可能。我们团队都深受鼓舞，也更加坚定了加速推进这款创新产品临床转化的决心，希望能早日惠及更多患者。 Q2 美迪西：这款新一代补体抑制剂SLN12140获得在中国、美国、澳大利亚开展临床试验的许可，可以介绍一下这款药物的核心优势吗？韩照中博士：SLN12140 是我们自主研发的新一代补体抑制剂产品，它的优势是多方面的。靶点选择上选用了生物学机制得到完美临床前、临床验证的补体信号通路，首选适应症选择上选用了临床、监管和市场转化路径更加清晰的PNH、IgA肾病，因此有效避开了很多新药研发中的风险。同时，在经过临床验证的生物学通路上，我们选用了更加容易成药的靶点，分子设计上考虑补体相关疾病的用药特点，因此与现有的补体产品不同，SLN12140的产品设计体现出了显著的差异化优势。从目前收集到的一期临床试验结果来看，它展现出了良好的安全性、药物代谢特征和靶点抑制效果。而且，高浓度、皮下给药、长给药周期的药物特征，让它具备了成为Best-In-Class 的潜力。作为同机制、同靶点的全球第一款皮下给药大分子治疗产品，这一特性也让它在市场竞争中拥有较大的潜力。 Q3 美迪西：在本次CDE的审批中，批准了SLN12140用于PNH等溶血性疾病的适应症，这些疾病给患者带来了极大的痛苦。能否具体介绍一下这些疾病的情况以及 SLN12140 在治疗这些疾病上的潜力？韩照中博士：PNH 是一种极端罕见、严重、高致死率、高致残率的溶血性疾病，和补体的异常活化高度相关。由于其临床试验周期短、临床终点明确，成为了补体抑制剂产品的试金石，尤其是对于需要体现和标准治疗差异化优势的新一代产品而言。像 Apellis 的 Pegcetacoplan（2021年5月获得FDA批准）和诺华的 Iptacopan（2023年12月获得FDA批准），都是在 PNH 适应症上首先获批的代表性产品。而和补体异常相关的TMA，包括非典型性尿毒症（aHUS）、干细胞移植术后微血管病（HSCT-TMA）等，也是近年一些补体产品的获批适应症。镰刀细胞病 SCD 在欧美地区相对高发，临床需求和市场空间巨大，且和补体关联性较强，目前尚没有相关产品获批上市。SLN12140 在这些适应症上的获批，不仅扩大SLN12140的适应症拓展空间，更是为患者尽快带来新的治疗希望创造了可能性，我们期待它能在临床试验中展现出优异的疗效。 Q4 美迪西：领诺医药自 2019 年成立以来，在创新药物研发方面取得了不少成果，除了 SLN12140，还有穿越血脑屏障的重组酶替代治疗项目 LIN-2003 等。领诺医药未来的研发重心和发展方向是怎样的呢？韩照中博士：领诺医药一直秉承 “领引创新，诺言践行” 的宗旨，我们自主开发了独有的血液半衰期延长（长效）、穿越血脑屏障等专利技术。目前，补体项目SLN12140已启动I期临床试验，穿越血脑屏障的重组酶替代治疗项目LIN-2003获得FDA孤儿药资格认定，并已启动IND申报准备工作。未来，我们会继续聚焦于治疗免疫性疾病的补体靶向抗体和治疗 CNS 相关疾病的重组蛋白类药物等管线产品, 稳步推进管线的临床试验和市场准入工作，争取能在更多国家和地区获批上市，为全球患者服务。非常感谢韩博士的分享，让我们对领诺医药以及 SLN12140 有了更深入的了解。美迪西作为领诺医药的合作伙伴，我们为领诺取得的成绩感到由衷的高兴。期待在未来的合作中，双方能携手取得更多突破性成果，为医药行业的发展和患者的健康贡献更多力量。关于领诺医药领诺医药成立于2019年8月，苏州、上海两地运营，在上海自由贸易试验区张江药谷拥有近1000平米的研发实验室。公司获得多家产业机构和私募基金的股权投资。公司研发团队覆盖从分子发现到早期临床开发的全流程，秉承“领引创新，诺言践行”的宗旨，自主开发了独有的血液半衰期延长（长效）、穿越血脑屏障等专利技术。领诺医药以自有平台技术为基础，致力于能更好满足临床需求的创新药物研发，开发针对新靶点、新机制、新适应症的First-in-class（FIC）产品，以及平台技术赋能、针对成熟靶点的Best-in-class（BIC）产品。目前，领诺医药的管线产品包括治疗免疫性疾病的补体靶向抗体和治疗CNS相关疾病的重组蛋白类药物。其中，补体项目LIN-2102（SLN12140）已启动I期临床试验，穿越血脑屏障的重组酶替代治疗项目LIN-2003获得FDA孤儿药资格认定，并已启动IND申报准备工作。此外，公司还有多个管线产品处于临床前概念验证阶段。领诺医药将持续加大研发投入，通过技术和产品授权、合作开发等渠道，加速产品在国内、外市场的临床转化，为病患者提供更安全、有效、方便的治疗选择。往期推荐美迪西专访 | 美济医药黄慧瑜博士：口服紫杉醇药物研发的破局者美迪西专访 | 新药研发智在必行：听任峰博士畅谈AI制药的无畏前行美迪西专访 | 祥根生物原晨光博士畅谈抗真菌药物市场趋势及创新美迪西专访陈涛博士 | 南芯医疗：做活体生物药领域一往无前的弄潮儿美迪西专访| 周文波博士：3年3个IND获批，宇耀生物有何优势？关于美迪西美迪西（股票代码：688202.SH）成立于2004年，总部位于上海，致力于为全球制药企业、研究机构及科研工作者提供全方位的临床前新药研究服务。美迪西的一站式综合服务以强有力的项目管理和更高效、高性价比的研发服务助力客户加速新药研发进程，服务涵盖医药临床前新药研究的全过程，包括药物发现、药学研究及临床前研究。至2025年6月底，美迪西已为全球超2000家客户提供药物研发服务，参与研发完成的新药及仿制药项目已有580+件IND获批临床，与国内外优质客户共同成长。美迪西将继续立足全球视野，聚力中国创新，为人类健康贡献力量！

孤儿药

2025-08-18

·PMLiVE

FDA approves Apellis' Empaveli for two rare kidney diseases

The US Food and Drug Administration (FDA) has approved Apellis Pharmaceuticals’ Sobi-partnered Empaveli (pegcetacoplan) to treat two rare kidney diseases. The targeted C3 therapy has been authorised to reduce excess protein in the urine (proteinuria) in patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). C3G and primary IC-MPGN are estimated to affect 5,000 people in the US. Excessive deposits of the C3 protein are a key marker of disease activity in both conditions and can lead to kidney inflammation, damage and failure, with approximately 50% of patients experiencing kidney failure within five to ten years of diagnosis. Empaveli is designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, and is already approved in the US to treat the rare blood disorder paroxysmal nocturnal haemoglobinuria. The FDA’s latest decision was supported by positive results from the late-stage VALIANT trial, in which Empaveli reduced proteinuria by 68% compared to placebo. Empaveli was also associated with stabilisation of kidney function, as measured by estimated glomerular filtration rate, as well as substantial clearance of C3 deposits, assessed by C3 staining. These benefits were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence. Apellis’ co-founder and chief executive officer, Cedric Francois, said: “Empaveli has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. “In the largest pivotal study of these diseases, Empaveli demonstrated its potential to preserve kidney function by controlling all three key markers of disease.” Apellis has exclusive commercialisation rights for systemic pegcetacoplan in the US, while Sobi holds these everywhere else. Apellis also has global commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. Commenting on the latest approval of the drug, lead principal investigator for the VALIANT study, Carla Nester, said: “With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. “Given the urgent need, particularly in children, the approval of Empaveli marks a pivotal moment in the treatment of rare kidney diseases.”

临床结果上市批准

2025-08-17

·研发客

80亿美元补体市场，打响常见病争夺战

// • 肾病和眼科是补体药物适应症拓展的热门方向，但国内开发眼科疾病的补体药物却不多； • 在研补体药物的开发思路，正在从补体系统整体完全阻断，向途径或组织特异性阻断转变； • 开发思路转变，着眼点之一是降低安全风险； • 在研补体药物的多靶点联合也是方向之一。全球补体药物市场规模正不断扩张，2024年比2023年增长22%，达到82.66亿美元。治疗地图样萎缩（GA）的眼科药物Syfovre贡献了最主要的增长份额，销量达6.12亿美元，同比增长123%。另一个GA药物Izervay销量也达到了3.96亿美元。这两款药物印证了补体药物扩展到常见病用药领域的商业价值。还不止于此。最畅销的补体药物仍是阿斯利康的两个C5抗体Soliris和Ultomiris，销售额占总额的79%，但从适应症拓展层面来看，Ultomiris作为长效迭代产品，正从阵发性睡眠性血红蛋白尿症（PNH）、重症肌无力等罕见病，向肾病等常见病拓展。当下正值补体药物开发“跨圈”之时：从罕见病到常见病，探索纳米抗体、小核酸药物等不同的分子类型，以及MASP-3、备解素P因子等更精准的创新靶点。两大常见病战场：肾病和眼科虽然阿斯利康的两款C5单抗销量不俗，但诺华的Fabhalta（伊普可泮）、Apellis开发的Empaveli已开启追赶模式，2024年销量分别达到1.29亿美元和1.33亿美元。更重要的是，后两款药物均已获批了肾病适应症。伊普可泮是首个获批肾病治疗的补体药物，2024年8月获得FDA批准治疗IgA肾病，隔年3月又获批治疗C3肾小球病（C3G）。此外，伊普可泮还在开展免疫复合物膜增生性肾小球肾炎（IC-MPGN）和狼疮性肾炎等临床研究。 Empaveli是C3抑制剂，于2025年7月获得FDA批准治疗C3G或原发性IC-MPGN。中国的在研补体药物大多都以IgA肾病作为开发适应症。其中恒瑞医药的HRS-5965已经启动了治疗原发性IgA肾病的III期临床研究，在国内同类产品研发中进展最快。其他管线均在I期和II期临床阶段，产品疗效有待更多数据验证。（见下图）可见，肾病已然成为国内补体药物临床开发的必争之地。另一个补体药物聚焦的常见病领域，是眼科。治疗年龄相关黄斑变性（AMD）及地图样萎缩已有两款药物获批，分别是Apellis的C3环肽抑制剂Syfovre（pegcetacoplan）、安斯泰来的Izervay（avacincaptad pegol）。如开头所述，两款药物的销售额验证了补体在眼科的商业价值。然而，国内开发眼科疾病的补体药物却不多。信达生物的依莫芙普（IBI302）已推进至III期临床，这是一款VEGFR/人补体受体1双靶向的融合蛋白，临床II期数据也在AMD治疗中非劣于阿柏西普。此外，领诺医药开发的LIN-2006是靶向VEGF/补体的双特异性抗体，处于临床前阶段。在国内至全球，肾病和AMD都存在巨大的临床需求。据WHO数据，2040年慢性肾脏病将上升为全球第五大死亡原因；根据摩熵咨询的流行病学数据，2050年我国AMD患者将增加至五千万例。放眼未来，补体药向大病市场扩展的前景如何？领诺医药创始人韩照中博士表示，除了目前获批的治疗严重罕见病、IgA肾病、AMD以外，补体异常活化和出血再灌注损伤（IRI）病理机制相关的术后器官保护、器官移植以及心梗、脑梗、脑水肿等疾病，都是潜在的大市场。目前已有企业开启了此类研究。6月，阿斯利康公布了Ultomiris治疗造血干细胞移植出现的血栓性微血管病的III期研究早期数据，总生存率达到了87.2%；Ultomiris还正在开展针对心脏手术相关急性肾损伤的III期临床试验。Apellis的官网显示，将启动Empaveli治疗肾脏移植物功能延迟恢复的确证性临床研究。长效制剂：新趋势 2024年，阿斯利康的长效C5补体抑制剂Ultomiris销售额相比2023年增长了近10亿美元；而一代C5产品Soliris相比同期下降了5.6亿美元。且2024年Ultomiris销量首次反超Soliris，补体长效制剂的商业化价值正在兑现。 Soliris通常需要一周或两周给药一次，Ultomiris通过对抗体Fc段进行修饰，半衰期延长至普通C5抑制剂的3倍以上，实现每八周一次的长效给药。紧接着，阿斯利康又开发了第三代C5产品gefurulimab，分子形式是双结合纳米抗体，每周一次皮下注射。近期，阿斯利康宣布gefurulimab在治疗重症肌无力的III期临床研究中达到主要终点。领诺医药的LIN-2102同样是皮下注射给药的纳米抗体，但靶向的是旁路路径靶点。纳米抗体的优势是既可保证较长给药周期，皮下注射便于用药，依从性更好，且在实体组织内有较好药物渗透性。LIN-2102正在进行I期临床试验，且在准备II期试验的启动计划。小核酸药物则以更加长效的策略，开始介入补体领域。国内企业中，圣因生物、炫景生物、瑞博生物、舶望制药等国内头部siRNA开发公司，均开发了补体药物。圣因生物创始人、CEO王为民认为，相比小分子和抗体药物，小核酸药物在长效性方面展示出显著优势，不仅如此，siRNA药物的作用机制更上游，通过靶向疾病相关基因的mRNA直接调控基因表达，从源头抑制致病蛋白的合成，还可能降低其他药物治疗中常见的耐受性或耐药性风险。以圣因生物开发的靶向补体C3的siRNA候选药物SGB-9768为例，临床试验显示其可实现每3个月或6个月给药一次的频率，同时表现出良好的安全性和耐受性。利用非常成熟的肝靶向递送系统，加上经过临床验证的C3、C5等补体靶点，开发肾病等常见病，从而避免过度的同质化竞争，是目前大多数小核酸企业入局补体领域的主要策略。更精准的靶点路径自2007年首个补体药物获批至今约20年，补体药物经历了早期的C3、C5，到CFB、CFD、MASP-2等新靶点的进阶。 C3、C5属于补体末端的共同通路，靶向C3、C5的产品从最初的单克隆抗体，目前已经拓展到已经批准上市的环肽、核酸适配体，以及在临床试验阶段的ASO、siRNA等小核酸药物。“这些药物完全阻断补体活化，个别药物也获批用于严重、罕见疾病的临床治疗，但补体的完全阻断也带来了安全风险担忧，尤其对于安全要求更高的大病、慢病治疗而言。”韩照中说。因此，这些获批的补体药物普遍带有黑框警告且要求接种疫苗。 “未来补体靶点的开发会更加精准。”恒瑞医药相关研发人员告诉研发客，不完全阻断的支路靶点是一个方向，在抑制补体减弱致病的基础上，最大限度保留补体对机体的保护性，降低安全风险，且更具疾病特异性。在研补体药物的开发思路，正在从补体系统整体完全阻断，向途径或组织特异性阻断转变。临床开发的产品多以旁路途径、凝集素途径为主。旁路途径中，诺华开发的CFB靶向药物伊普可泮，阿斯利康的补体因子D（CFD）药物Voydeya已经获批上市。国内中，恒瑞医药开发的补体因子B（CFB）抑制剂HRS-5965已经启动了III期临床研究。恒瑞医药表示，CFB能够避开了自身抗体依赖的经典途径抑制可能带来的感染风险，靶向更为精准，又能够保留抗感染免疫功能，实现疗效与安全性的平衡。凝集素途径的研发中，MASP-2是一个新兴靶点。Omeros公司开发MASP-2单抗narsoplimab已经在治疗移植相关性血栓性微血管病的III期临床研究中达到研究终点，商业化在即。康诺亚的CM338则是国内最先进入临床的MASP-2抗体。更加创新的靶点也在被不断挖掘。例如Omeros公司开发的靶向MASP-3的OMS906。NovelMed公司开发了一款靶向旁路途径备解素P因子的单抗NM5072。多靶点的联合也是方向之一。例如旁路途径与凝集素途径的联合，同时阻断两个路径，可以进一步提高产品疗效，还可以减少单一途径长期阻断所致的代偿性激活。阿斯利康将口服CFD抑制剂Voydeya与C5补体抑制剂联合，III期临床数据显示，能够改善PNH伴血管外溶血患者的长期疗效与安全性。再生元也在开展C5补体siRNA疗法cemdisiran与C5补体抑制剂pozelimab联合治疗PNH的临床研究。此外，在肿瘤治疗中，补体与免疫疗法的联合使用也能进一步增加免疫效应，提高抗肿瘤效果。虽然补体赛道相对小众且机制较为复杂，但近年跨国药企相继入局、新适应症不断获批、新靶点不断被验证，均在传递积极的信号——补体领域值得更多期待。编辑 | 姚嘉 yao.jia@PharmaDJ.com 总第2542期访问研发客网站，深度报道和每日新闻抢鲜看 www.PharmaDJ.com

临床3期核酸药物临床2期申请上市

100 项与 Pegcetacoplan 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11613	Pegcetacoplan	B03XA	-

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
C3肾小球病	美国	Apellis Pharmaceuticals, Inc.	2024-10-07
膜增生性肾小球肾炎	美国	Apellis Pharmaceuticals, Inc.	2024-10-07
地图样萎缩	美国	Apellis Pharmaceuticals, Inc.	2023-02-17
阵发性睡眠性血红蛋白尿症	美国	Apellis Pharmaceuticals, Inc.	2021-05-14

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
移植物功能延迟恢复	临床3期	-	Apellis Pharmaceuticals, Inc.	2025-09-01
终末期肾脏病	临床3期	-	Apellis Pharmaceuticals, Inc.	2025-09-01
冷凝集素病	临床3期	美国	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	日本	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	奥地利	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	比利时	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	加拿大	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	芬兰	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	格鲁吉亚	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	德国	Swedish Orphan Biovitrum AB	2022-10-20

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05067127 (VALIANT \| APL2-C3G-310, CTgov)	临床3期	膜增生性肾小球肾炎 \| C3肾小球病 \| 肾小球肾炎慢性补体成分 3 肾小球病	124	Pegcetacoplan (Randomized Controlled Period: Pegcetacoplan)	觸顧衊鹽壓衊簾鹹築鹽(襯壓選鹹繭鏇衊鹽醖觸) = 壓鑰顧醖顧壓製憲鬱構餘衊鹽鬱鹹獵襯廠選製 (憲鏇蓋壓廠襯衊憲製鬱, 衊構築鹽簾窪願衊鹹醖 ~ 餘鑰廠獵繭鏇繭醖齋積) 更多	-	2025-08-06
				placebo+pegcetacoplan (Randomized Controlled Period: Placebo)	觸顧衊鹽壓衊簾鹹築鹽(襯壓選鹹繭鏇衊鹽醖觸) = 範淵網構鬱構顧願鬱糧餘衊鹽鬱鹹獵襯廠選製 (憲鏇蓋壓廠襯衊憲製鬱, 網蓋繭膚衊顧鹽糧簾蓋 ~ 範積廠繭憲淵顧範簾觸) 更多
NCT04770545 (OAKS, DERBY, and GALE, Pubmed \| Am J Ophthalmol)	临床3期	年龄相关性黄斑变性	790	Pegcetacoplan monthly	鑰簾簾築艱齋膚繭蓋壓(遞願衊顧繭顧糧積糧蓋) = 33 (4.5%) eyes with exudative AMD 廠壓鏇鹽鏇鑰窪齋艱範 (廠鹽襯鹽蓋製獵積襯夢 ) 更多	积极	2025-08-01
NCT05067127 (APL2-C3G-310 \| VALIANT, FDA_CDER \| PipelineReview) 人工标引	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	124	Pegcetacoplan	窪範顧範夢窪構築鑰蓋(願壓廠憲淵醖齋鑰窪顧) = 觸網觸艱膚衊餘願淵顧選鏇簾膚醖願選餘獵餘 (網壓製構衊艱餘構襯夢, 0.25，0.43)	积极	2025-07-28
				Placebo	窪範顧範夢窪構築鑰蓋(願壓廠憲淵醖齋鑰窪顧) = 壓製獵蓋鬱築鹹範膚鏇選鏇簾膚醖願選餘獵餘 (網壓製構衊艱餘構襯夢, 0.91，1.16)
PF675 (EHA2025)	N/A	C5 inhibitors	26	Pegcetacoplan	繭膚獵蓋鏇鹹網網鏇積(構鑰製衊餘窪憲製壓鬱) = 鏇衊壓範衊獵蓋網齋築鏇築鑰獵憲鬱夢窪艱遞 (鬱鏇繭襯簾糧顧鑰膚壓 ) 更多	-	2025-05-14
NCT04085601 (PRINCE, EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症一线	46	PEGCetacoplan	齋製壓製積壓願醖鏇願(築壓鬱廠鑰襯範簾憲範) = 構繭選顧鏇範鑰壓衊顧鏇糧壓遞壓簾繭衊夢襯 (窪積選範醖鬱淵餘衊艱 )	积极	2025-05-14
NCT05776472 (COMPLETE, EHA2025)	N/A	阵发性睡眠性血红蛋白尿症	70	Pegcetacoplan	選襯夢淵觸膚繭膚鏇構(遞襯窪遞蓋積艱膚積簾) = 遞憲觸願鑰壓製鏇鑰鏇鏇網觸觸願餘齋網獵鏇 (選網艱衊醖遞鬱襯夢餘, 0.8 ~ 3.3) 更多	-	2025-05-14
PS1665 (EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症	-	PEGcetacoplan (PNH patients with <4 RBCs)	顧齋築鹹壓簾夢蓋觸糧(蓋憲築襯積餘襯構製構) = Initial reductions in mean ARC were sustained 鹽顧膚衊製範繭積衊築 (簾獵淵憲觸齋顧鹹構艱 ) 更多	积极	2025-05-14
				PEGcetacoplan (PNH patients with ≥4 RBCs)
NCT03500549 (Pubmed \| Eur J Haematol)	临床3期	阵发性睡眠性血红蛋白尿症 complement 5 inhibitors (C5i; eculizumab, ravulizumab)	-	Pegcetacoplan	製構範齋憲壓選鏇鑰廠(壓獵鑰齋蓋鬱膚膚選簾) = 製顧構醖選壓遞構鹽鑰夢醖觸觸夢獵蓋鹹繭襯 (廠膚餘糧願製選齋願範, -90.02 ~ 21.21)	积极	2025-04-25
NCT04579666 (CTgov)	临床2期	肌萎缩侧索硬化	249	Pegcetacoplan (RTP: Pegcetacoplan)	齋築淵獵鏇齋膚鏇餘壓(遞鑰蓋製餘窪構觸鑰蓋) = 築鬱夢範鬱鬱餘醖鹹積襯餘廠膚網製蓋鏇鬱選 (夢積積憲齋繭艱艱艱壓, 4.71) 更多	-	2025-04-24
				placebo+pegcetacoplan (RTP: Placebo)	齋築淵獵鏇齋膚鏇餘壓(遞鑰蓋製餘窪構觸鑰蓋) = 鹽膚願壓鬱鑰蓋選獵簾襯餘廠膚網製蓋鏇鬱選 (夢積積憲齋繭艱艱艱壓, 6.89) 更多
NCT03453619 (CTgov)	临床2期	特发性膜性肾病 \| 膜增生性肾小球肾炎 \| C3肾小球病 ... 更多	21	pegcetacoplan (Part A: IgAN Subjects)	衊窪襯淵網顧壓製構醖(簾簾淵糧積繭獵簾顧鹹) = 夢範壓窪繭糧壓廠獵壓襯壓觸齋憲遞衊醖網願 (餘艱鬱鹽遞壓膚壓獵範, 鬱觸蓋窪遞觸壓鹽鹽積 ~ 願顧築鹹衊齋憲憲製艱) 更多	-	2025-02-13
				pegcetacoplan (Part A: LN Subjects)	衊窪襯淵網顧壓製構醖(簾簾淵糧積繭獵簾顧鹹) = 願製築鑰築窪淵餘艱襯襯壓觸齋憲遞衊醖網願 (餘艱鬱鹽遞壓膚壓獵範, 願遞觸淵願廠壓築鑰膚 ~ 獵憲鏇廠夢窪齋鬱膚餘) 更多