This phase I/II trial tests the effect of pegcetacoplan in combination with oxaliplatin, irinotecan, leucovorin, and fluorouracil (mFOLFIRINOX) in treating patients with pancreatic ductal adenocarcinoma (PDAC) that has spread from where it first started (primary site) to other places in the body (metastatic). Pegcetacoplan works by targeting the immune complement process, a part of the immune system that defends against bacteria and may limit tumor progression and improve the immune system's response against tumor cells. Oxaliplatin is in a class of medications called platinum-containing antineoplastic agents. It damages the cell's deoxyribonucleic acid (DNA) and may kill tumor cells. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill tumor cells. Leucovorin is a drug used to lessen the toxic effects of substances that block the action of folic acid. Leucovorin is a form of folic acid. It is a type of chemoprotective agent and a type of chemosensitizing agent. Fluorouracil stops cells from making DNA and it may kill tumor cells. It is a type of antimetabolite. Giving pegcetacoplan in combination with mFOLFIRINOX may be safe, tolerable, and/or effecting in treating patients with metastatic PDAC. This trial also evaluates the effect of pegcetacoplan on the incidence of major thrombotic events and the resulting complications. Thrombosis is a common complication in patients with PDAC. Thrombosis occurs when blood clots block veins or arteries. Complications of thrombosis, such as stroke or heart attack, can be life-threatening. Giving pegcetacoplan may help prevent blood clots from forming and decrease the risk of major thrombotic events.

开始日期2025-12-01

申办/合作机构

Roswell Park Comprehensive Cancer Center

NCT07213960

/ Not yet recruiting临床2/3期

A Sequential Phase 2/3, Single-Arm, Open-Label Study in Adults Followed by a Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of APL2 in Adults and Adolescents With Focal Segmental Glomerulosclerosis

This is a sequential phase 2/3 study to evaluate the efficacy and safety of twice-weekly subcutaneous (SC) infusions of APL2 in patients diagnosed with FSGS. The initial phase 2 portion is a single-arm, open-label study in adults diagnosed with FSGS. Phase 2 will commence prior to randomizing for phase 3. The phase 3 portion of the study is a randomized, placebo-controlled, double-blinded, multicenter study in adults and adolescents diagnosed with FSGS.

开始日期2025-12-01

申办/合作机构

Apellis Pharmaceuticals, Inc.

NCT07214740

/ Completed临床3期

A Phase 3B, Single-Arm, Open-Label, Multicenter Study to Evaluate the Safety of Pegcetacoplan in a Prefilled Syringe in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

This is a phase 3, open-label study to evaluate the safety of pegcetacoplan in a prefilled syringe (PFS)

开始日期2025-10-24

申办/合作机构

Apellis Pharmaceuticals, Inc.

100 项与 Pegcetacoplan 相关的临床结果

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100 项与 Pegcetacoplan 相关的转化医学

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100 项与 Pegcetacoplan 相关的专利（医药）

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217

项与 Pegcetacoplan 相关的文献（医药）

2026-01-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

Real-World Experience With Intravitreal Pegcetacoplan for the Treatment of Geographic Atrophy in Age-Related Macular Degeneration

Article

作者: Le, Viet Hoan ; El-Mulki, Omar S ; Zhang, Yi ; Kastner, James ; Hiya, Farhan ; Beqiri, Sara ; Rosenfeld, Philip J ; Berni, Alessandro ; O'Brien, Robert C ; Liu, Jeremy ; Shen, Mengxi ; Gregori, Giovanni ; Herrera, Gissel ; Yehoshua, Zohar ; Nicola, Maura Di ; Wang, Ruikang K ; Cheng, Yuxuan ; Dubovy, Sander R ; Trivizki, Omer ; Kumar, Sivathanu

PURPOSE:

To report on the real-world experience of using intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) in age-related macular degeneration (AMD).

DESIGN:

Retrospective interventional case series.

METHODS:

Eyes with symptomatic GA secondary to AMD were treated with 15 mg of intravitreal pegcetacoplan and participated in an ongoing prospective swept-source optical coherence tomography angiography (SS-OCTA) imaging study. All eyes underwent SS-OCTA imaging before and during pegcetacoplan therapy to assess for GA lesion size, the presence of nonexudative macular neovascularization (MNV), and the onset of exudation. The growth rate of GA and best-corrected visual acuity (BCVA) were assessed for eyes followed for 1 year.

RESULTS:

From April 12, 2023, to November 11, 2024, 154 eyes were injected with pegcetacoplan, and 103 eyes had 1-year follow-up. At baseline, 11 eyes had been previously treated with anti-VEGF therapy, and 9 eyes were diagnosed with treatment-naïve nonexudative MNV by SS-OCTA. Each eye received an average of 10 ± 2 injections, with an average interval of 1.2 months between injections. For the 97 eyes with 1 year of follow-up and measurable GA, the square-root (sqrt) GA growth rate after pegcetacoplan treatment was 0.24 ± 0.15 mm/year. Of these 97 eyes, 63 eyes had prior annual visits before pegcetacoplan treatment was started. In these eyes, the annual sqrt GA growth rate was 0.33 ± 0.22 mm/year before pegcetacoplan and 0.21 ± 0.12 mm/year after pegcetacoplan, resulting in a 37% decrease in the growth rate (P < .001). There were no significant differences in GA growth rate between foveal and nonfoveal GA, either before or after the use of pegcetacoplan (all P ≥ .80). The mean BCVA declined from 63 ± 14 to 59 ± 15 letters over 1 year (P = .001), with no significant difference between foveal and nonfoveal GA (P = .36). Among the 29 eyes developing exudation during pegcetacoplan treatment, 19 (66%) had no evidence of any detectable MNV at baseline and during treatment on SS-OCTA.

CONCLUSIONS:

Eyes treated with pegcetacoplan after 1 year had a 37% reduction in GA growth rate compared with their prior annual growth rate. Pegcetacoplan slowed the growth for foveal and nonfoveal GA at a similar rate. Most of the pegcetacoplan-associated exudation was not due to detectable MNV.

2025-12-31·Hematology

Successful switch from pegcetacoplan to iptacopan after repeated severe breakthrough hemolysis events – case report

Article

作者: Füreder, Wolfgang ; Reinisch, Andreas

OBJECTIVES:

A subset of paroxysmal nocturnal hemoglobinuria (PNH) patients develops clinically relevant extravascular hemolysis when treated with complement C5 inhibitors. These patients may benefit proximal complement inhibitors such as pegcetacoplan, danicopan or iptacopan. No studies comparing these substances are available. Breakthrough hemolysis (BTH) - defined by exacerbation of intravascular hemolysis despite complement inhibition - can be severe especially in patients treated with proximal complement inhibitors.

METHODS:

We report on a PNH patient who had suffered repeated episodes of severe BTH with acute renal failure during 1 year of treatment with pegcetacoplan. The patient was switched to iptacopan and followed also for 1 year.

RESULTS:

After switching to iptacopan, no further BTH occurred for the duration of 12 months follow up. The patient maintained stable hemoglobin and reticulocyte counts as well as lactate dehydrogenase (LDH) levels within the normal range.

DISCUSSION:

Despite dose escalation of pegcetacoplan, BTH recurred in our patient. Therefore, an alternative therapy was warranted. During iptacopan therapy - chosen due to patient preference -no further BTH occurred. However, more data from a larger number of patients are needed.

CONCLUSION:

A switch to iptacopan may be an option for pegcetacoplan treated patients who experience repeated BTH in spite of dose escalation.

2025-12-05·INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE

Effect of Pegcetacoplan on Aqueous Humor Proteome in Geographic Atrophy: A Prospective Exploration

Article

作者: Wykoff, Charles C. ; van den Heuvel, Lambert ; Rabinovitch, David ; Cao, Jessica A. ; Smoor, Magda A. ; Vergroesen, Joëlle E. ; Zhou, Avery ; Lechanteur, Yara T. E. ; Trivizki, Omer ; van Gool, Alain J. ; Klaver, Caroline C. W. ; Gloerich, Jolein

Purpose:

Pegcetacoplan, a complement component 3 (C3) inhibitor, has recently received U.S. Food and Drug Administration approval for treating geographic atrophy (GA), an advanced stage of age-related macular degeneration (AMD). However, the limited treatment response prompts further investigations into its molecular effects.

Methods:

We prospectively collected aqueous humor (AH) samples from 11 patients with GA before and at 2 months during pegcetacoplan treatment. Liquid chromatography with tandem mass spectrometry (LC-MS/MS) was used to analyze the proteome, and global normalization was applied to account for differences in protein concentration. To assess global proteomic shifts over time, principal component analysis (PCA) was performed. The Friedman test was used to assess differences in protein intensities across time points while adjusting for multiple testing using Benjamini-Hochberg false discovery rate (FDR) correction.

Results:

A total of 283 proteins were analyzed. PCA revealed temporal changes in global protein expression profiles, with a significant shift between baseline and month 2 (P = 0.01). The levels of complement components C3 (P = 0.12) and C5 (P = 0.27) remained stable after initiation of treatment, but the levels of C1qB, C1RL, C2, C6, C7, C8, C9, factor D, factor H, and factor I increased significantly (all P < 0.05). Most non-complement proteins showed no significant changes; however, beta-2-glycoprotein 1 (FDR = 0.09), kininogen 1 (FDR < 0.05), and prothrombin (FDR < 0.05) increased significantly, and kallistatin (FDR < 0.05) and plasma serine protease inhibitor (FDR < 0.05) decreased.

Conclusions:

This exploratory study suggests that pegcetacoplan modulates the AH proteome in GA. Although no changes in the target protein C3 were detected following treatment, significant changes in proteins tightly connected to complement, inflammation, and coagulation were observed. These findings underscore the need for further investigation into the biological and clinical relevance of the observed molecular shifts.

481

项与 Pegcetacoplan 相关的新闻（医药）

2025-12-16

·药品圈

FDA发布81份BE指南，包含多个国内有参比无指南复杂品种

*识林“BE指南数据库”截图，仅作示意用。 12月4日，FDA共发布了81份产品特定指南（PSG）草案，包括44份新指南和37份修订指南。54份PSG针对尚无获批ANDA的产品，其中包含19种复杂产品；28份PSG针对复杂产品，其中7份为新指南，21份为修订指南； Lachman的仿制药专家Pollock指出值得关注的PSG涵盖银屑病首创外用疗法、脂溢性皮炎治疗、营养不良性和交界性大疱性表皮松解症相关创面治疗及其他适应症，具体包括： 1.新PSG：治疗脂溢性皮炎：罗氟司特外用泡沫剂（RLD：ZORYVE，NDA 217242）；治疗罕见皮肤病（营养不良性和交界性大疱性表皮松解症）：Birch Triterpenes外用凝胶（RLD：FILSUVEZ，NDA 215064）。 2.修订PSG：银屑病非甾体辅助治疗：Tapinarof外用乳膏（RLD：VTAMA，NDA 217806），新增基于表征的BE方法；多种缓释（modified-release）药物：GDUFA新资助研究表明体外乙醇诱导的剂量倾泻研究（alcohol dose dumping studies）不再必需，故在PSG中移除此类研究，包括：Carbinoxamine Maleate口服缓释混悬液（RLD：KARBINAL ER，NDA 022556），磷酸卡维地洛口服缓释胶囊（RLD：COREG CR，NDA 022012），盐酸地尔硫卓口服缓释胶囊（RLD：CARDIZEM CD，NDA 020062），泊沙康唑口服迟释混悬剂（RLD：NOXAFIL POWDERMIX KIT，NDA 214770），曲司氯铵口服缓释胶囊（RLD：SANCTURA XR，NDA 022103）；复杂肽类药物：明确体外先天免疫反应试验建议，并规定仿制重组肽类药物的简化申报路径，包括：鲑降钙素（RLD：CALCIMAR，NDA 017769），胰高血糖素（RLD：BAQSIMI，GVOKE，NDA 210134，212097），Dasiglucagon Hydrochloride（RLD：ZEGALOGUE，NDA 214231），Pegcetacoplan（RLD：EMPAVELI，SYFOVRE，NDA 215014，217171），替尔泊肽（RLD：MOUNJARO，NDA 215866），特立帕肽（RLD：FORTEO，NDA 021318），利拉鲁肽（RLD：VICTOZA，SAXENDA，NDA 022341，206321），司美格鲁肽（RLD：WEGOVY，NDA 215256），伏索利肽（RLD：VOXZOGO，NDA 214938）。基于识林知识平台的“BE指南数据库”和“国内参比制剂数据库”进行简要调研后，编者将中美欧WHO的指南发布和国内参比发布情况列表如下，供仿制药企业立项调研时参考。如有差错，请读者指正（会员可至识林资讯页面下载excel版本）。 ● 表示查到对应指南或品规。 ✱ 表示查到对应分子，但品规不同。 N/A表示未查到对应指南或品规，或未查到官方中文通用名。

申请上市

2025-12-15

·FierceBiotech

Aviceda geographic atrophy hopeful fails to top Astellas\' med in phase 2

Despite the fail, Aviceda still plans to test its lead asset in two sham-controlled phase 3 trials in 2026.\n Aviceda Therapeutics has hit a snag in its goal to deliver a new treatment for geographic atrophy (GA), an advanced form of dry age-related macular degeneration. The biotech’s lead asset, AVD-104, failed to beat Astellas’ Izervay (avacincaptad pegol) at slowing the rate of retina lesion growth, missing the primary endpoint of a phase 2 trial.The trial enrolled 300 patients with GA and treated them with monthly eye injections of either Izervay or AVD-104 at low or high doses.\"The study did not meet its primary endpoint, which was designed to assess non-inferiority,\" a company spokesperson told Fierce Biotech. \"No statistical differences in GA lesion growth rates were observed across the three study arms.\"AVD-104 led to no serious adverse events, Aviceda reported in a Dec. 15 release, with the most common side effect being eye floaters. AVD-104 is a nanoparticle adorned with polysialic acid, designed to bind to receptors on the surfaces of overactive immune cells like macrophages that cause eye inflammation in GA.Though it failed to top Izervay, AVD-104 did reduce the growth rate of GA lesions by 31% compared to previously published sham-treatment rates from Izervay’s phase 3 trial, according to Aviceda. The company described this finding as “clinically meaningful.” In addition, the company highlighted that patients given AVD-104 were able to read an average of 0.6 more letters in a visual acuity test after 12 months of treatment.“These results reinforce our belief that AVD-104 can provide meaningful functional vision benefit while reducing lesion progression, and has the potential to become a differentiated therapy addressing a significant unmet medical need for patients living with GA,” Aviceda CEO Jeffrey Nau, Ph.D., said in the release. Despite the failure, which Aviceda attributed to “imbalances in key baseline lesion characteristics across treatment arms,” the company still plans to test its lead asset in two sham-controlled phase 3 trials in 2026, according to the release. The Massachusetts-based biotech closed a $207.5 million series C in January to support its phase 3 vision.Aviceda was also testing the nanoparticle in a phase 2 trial for diabetic macular edema, but halted the study early due to “insufficient study drug supply and study funding constraints,” according to the clinicaltrials.gov database.GA has been a tough indication for pharma to crack. Roche blockbuster hopeful lampalizumab flopped in a critical phase 3 trial in 2017, with the Swiss pharma subsequently ditching the asset. Novartis scrapped its own GA candidate, acquired from Gyroscope Therapeutics, in September 2023 because of weak data.And in September 2024, Alkeus Pharmaceuticals reported that its oral candidate failed to reduce GA lesion growth in a phase 3 trial, with the company nevertheless declaring the results “clinically meaningful.”Apellis\' Syfovre (pegcetacoplan) was the first GA drug to break through, securing FDA approval in February 2023. However, the med was flagged by the agency for rare cases of severe eye inflammation that can lead to blindness just months later. Astellas picked up Izervay through the $5.9 billion acquisition of Iveric Bio in March 2023, garnering an approval of its own that August.Neither Izervay nor Syfovre is able to restore vision, and injections of both come with the risk of side effects like inflammation, bleeding and blurred sight.Outside of therapeutics, Science Corp. announced in October that its retinal implant was able to give GA patients some vision back in a clinical trial.Editor\'s note: This story was updated at 4:00 p.m. ET with a statement from Aviceda.

$Aviceda geographic atrophy hopeful fails to top Astellas\' med in phase 2$

临床3期并购临床2期上市批准临床结果

2025-12-13

·药明康德

礼来公布三靶点减重疗法积极3期结果；赛诺菲血友病siRNA疗法在中国获批上市…… | TIDES周报

近期，全球多肽和寡核苷酸（TIDES）领域迎来系列进展。礼来（Eli Lilly and Company）公布了其潜在“first-in-class”三靶点减重多肽疗法retatrutide的积极3期临床试验结果，患者治疗68周后，平均减重最高达28.7%。赛诺菲（Sanofi）宣布其用于治疗血友病的小干扰RNA（siRNA）疗法芬妥司兰钠注射液（商品名：赛菲因）已正式获得中国国家药品监督管理局（NMPA）批准上市。Enterome公司基于肿瘤肽的免疫疗法EO2463联用利妥昔单抗一线治疗低肿瘤负荷滤泡性淋巴瘤患者，在一项2期临床试验中的客观缓解率（ORR）达100%。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。 Retatrutide：公布3期临床试验数据礼来宣布，其TRIUMPH-4临床3期试验取得积极的主要研究结果。该试验评估其在研、每周一次、潜在“first-in-class”葡萄糖依赖性促胰岛素多肽（GIP）、GLP-1和胰高血糖素（glucagon）受体靶向三重激动剂retatrutide，在患有肥胖或超重且合并膝骨关节炎、但无糖尿病的成年人中，作为健康饮食与运动的辅助治疗方案时的安全性和有效性。在共同主要终点中，基于有效性估计目标评估，retatrutide可平均降低体重最多达28.7%（71.2磅），基于WOMAC疼痛评分平均降低疼痛最多达4.5分（75.8%），并显著改善身体功能指标。在试验结束时，超过八分之一接受retatrutide治疗的患者完全无膝部疼痛。在额外的次要终点方面，retatrutide降低了已知心血管风险标志物。Retatrutide的不良事件类型与其他肠促胰素类药物临床试验中观察到的情况一致。礼来预计将在2026年完成另外七项评估retatrutide用于肥胖和2型糖尿病治疗的3期临床试验。 ▲TRIUMPH-4临床3期试验有效性估计目标结果摘要（图片来源：参考资料[5]）芬妥司兰钠注射液：在中国获批上市赛诺菲宣布，其创新非因子疗法芬妥司兰钠注射液已正式获得NMPA批准上市，适用于患有以下疾病的12岁及以上儿童和成人患者的常规预防治疗，以防止出血或降低出血发作的频率：存在或不存在凝血因子VIII抑制物的重型A型血友病（先天性凝血因子VIII缺乏，FVIII<1%）或存在或不存在凝血因子IX抑制物的重型B型血友病（先天性凝血因子IX缺乏，FIX<1%）。通过降低抗凝血酶（AT），芬妥司兰钠注射液可促使凝血酶生成增加，从而恢复血友病患者的凝血功能。新闻稿指出，芬妥司兰钠注射液是血友病领域首个通过降低AT水平实现治疗的药物，每年仅需最少六次皮下注射，可通过预充注射笔或西林瓶与注射器进行给药，从而为患者提供持续稳定的保护。此次获批主要基于3期临床研究ATLAS的数据。研究结果表明，与按需治疗相比，接受芬妥司兰钠注射液治疗的患者，无论是否伴有抑制物，均显示出显著疗效：合并抑制物患者平均年化出血率（ABR）降低73%，无抑制物患者ABR降低71%。芬妥司兰钠注射液的不良反应包括血栓事件、急性及复发性胆囊疾病（部分患者可能行胆囊切除）以及肝毒性。最常见的不良反应（发生率超过10%）为病毒感染、鼻咽炎和细菌感染。 EO2463：公布2期联合治疗试验的中期数据 Enterome公司公布了其基于肿瘤肽的免疫疗法EO2463与利妥昔单抗联用治疗低肿瘤负荷滤泡性淋巴瘤的积极2期临床结果。EO2463由4种模拟B细胞肿瘤相关抗原CD20、CD22、CD37和CD268（BAFF受体）的多肽以及辅助性的UCP2肽组成，能够激活抗肿瘤免疫反应，清除癌变B细胞。此次公布的结果显示，在队列3中，EO2463联合利妥昔单抗一线治疗初治的低肿瘤负荷滤泡性淋巴瘤患者，客观缓解率为100%（6/6），5名患者为完全缓解（CR），1名为部分缓解（PR）。达到客观缓解的中位时间为17周，到CR的中位时间为18周。此外，在EO2463单药治疗的初治滤泡性淋巴瘤患者（队列2）中，ORR为52.6%。 Zeleciment rostudirsen：公布1/2期临床试验数据 Dyne Therapeutics公司宣布，其在研疗法zeleciment rostudirsen在1/2期DELIVER试验的注册性扩展队列（REC）中，用于适合外显子51跳跃治疗的杜氏肌营养不良症（DMD）患者时取得了积极顶线结果。Zeleciment rostudirsen（z-rostudirsen，也称为DYNE-251）由磷酸二氨基甲酰吗啉寡核苷酸（PMO）与靶向转铁蛋白受体1（TfR1）的抗体片段（Fab）偶联而成。TfR1在肌肉组织中高度表达。该疗法旨在实现靶向肌肉组织递送，促进核内外显子跳跃，从而使肌肉细胞生成截短但功能正常的抗肌萎缩蛋白，目标是停止或逆转疾病进展。该疗法此前已被FDA授予快速通道资格、孤儿药资格、罕见儿科疾病认定，以及突破性疗法认定。Dyne表示其预计在2026年第二季度向美国FDA递交加速批准的上市申请目前进展良好。分析显示，REC达到试验主要终点，显示在六个月时，与基线相比，zeleciment rostudirsen组患者肌肉含量调整后的抗肌萎缩蛋白（dystrophin）表达提高到正常水平的5.46%（p<0.0001），且具有统计学显著性。此结果重现了此前在注册剂量观察到的抗肌萎缩蛋白相较基线增加7倍的变化。此外，在REC的顶线结果中，所有六项预定的功能性终点相对安慰剂均观察到改善。其中两项指标——起身时间（TTR）速度和10米行走/跑步（10MWR）速度——在六个月时相对安慰剂均有改善，且p<0.05。值得注意的是，以用力肺活量预测百分比（FVC%p）衡量接受治疗患者的肺功能时发现（在DMD中肺功能丧失是主要死亡原因），zeleciment rostudirsen组患者的肺功能在六个月时得到维持，而安慰剂组出现下降。该疗法持续展现良好的安全性和耐受性。 WVE-007：公布1期临床试验数据 Wave Life Sciences公司宣布，其正在开展的首次人体试验INLIGHT中，最低治疗剂量队列已取得积极中期结果，该试验主要评估在研疗法WVE-007用于肥胖治疗的潜力。WVE-007是一款长效GalNAc修饰siRNA，可靶向遗传学验证靶点INHBE的mRNA，旨在通过抑制INHBE表达减少脂肪累积，同时维持肌肉质量。INHBE基因主要在肝脏中表达，其编码的Activin E蛋白参与脂肪代谢调控，因此INHBE已成为改善肥胖治疗的高潜力靶点。本次中期分析结果显示，在三个月时，相较基线，单次240 mg皮下注射WVE-007可使内脏脂肪减少9.4%（p=0.02），全身脂肪减少4.5%（3.5磅，p=0.07），瘦体重增加3.2%（4.0磅，p=0.01），而安慰剂组则未见具有统计学意义的变化。试验结果还显示，受试者血清中的Activin E持续且显著受到抑制，有望带来身体组成的持续改善、进一步减脂并维持肌肉量。整体来看，该药物安全性良好，仅观察到轻度治疗相关不良事件，实验室检测指标（包括血脂和肝功能）未见具有临床意义的异常变化。这一结果为WVE-007有望实现每年给药一至两次的给药方案提供了重要依据。圣因生物完成超1.1亿美元B轮融资聚焦RNAi疗法开发的临床阶段生物技术公司圣因生物宣布已完成超1.1亿美元B轮融资，募集资金将用于依托公司RNAi药物研发平台——包括LEAD（Ligand and Enhancer Assisted Delivery，新型配体与增效基团协同递送）肝外递送平台，推动临床阶段管线进入注册研究，并加速多个治疗领域在研管线的开发。该B轮融资由一家知名产业机构领投，一家国际主权基金、中国生物制药集团、君联资本、维梧资本、Invus、SymBiosis、苏创投、真脉投资、清池资本跟投，并获礼来公司战略投资。同时，现有股东高瓴创投、启明创投、险峰淇云、泰福资本、元禾控股、北极光创投等继续支持。公开资料显示，圣因生物依托GalNAc肝脏递送平台和LEAD肝外递送平台，致力于解决肥胖症、心血管代谢疾病和免疫介导性疾病领域等重大未满足的临床需求。LEAD平台能够高效地将siRNA特异性递送至脂肪、肌肉、免疫细胞及中枢神经系统等肝外组织，实现对致病基因高效、安全且持久的沉默，并大幅降低给药频率。圣因生物已建立快速增长的在研产品管线，目前已有4款临床阶段候选药物（1项临床2期，3项临床1期）及多个临床前管项目。参考资料： [1] Wave Life Sciences Announces Positive Interim Data from Phase 1 INLIGHT Trial of WVE-007 (INHBE) for Obesity; Single Dose Resulted in Improvement in Body Composition With Fat Loss Similar to GLP-1 at Three Months Without Muscle Loss. Retrieved December 8, 2025 from https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-announces-positive-interim-data-phase-1 [2] Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD). Retrieved December 8, 2025 from https://www.globenewswire.com/news-release/2025/12/08/3201363/0/en/Dyne-Therapeutics-Announces-Positive-Topline-Results-from-Phase-1-2-DELIVER-Trial-of-Z-Rostudirsen-in-Duchenne-Muscular-Dystrophy-DMD.html [3] The New England Journal of Medicine Publishes Positive Phase 3 VALIANT Results of pegcetacoplan for C3G and Primary IC-MPGN. Retrieved December 4, 2025 from https://www.prnewswire.com/news-releases/the-new-england-journal-of-medicine-publishes-positive-phase-3-valiant-results-of-pegcetacoplan-for-c3g-and-primary-ic-mpgn-302632767.html [4] 圣因生物完成超1.1亿美元B轮融资. Retrieved December 12, 2025, from https://mp.weixin.qq.com/s/E-o4xUaWa9N2x0u9V683lg [5] Lilly's triple agonist, retatrutide, delivered weight loss of up to an average of 71.2 lbs along with substantial relief from osteoarthritis pain in first successful Phase 3 trial. Retrieved December 11, 2025 from https://investor.lilly.com/news-releases/news-release-details/lillys-triple-agonist-retatrutide-delivered-weight-loss-average [6] Syneron Bio（元思生肽）完成A轮及A+轮近亿美元融资. Retrieved December 12, 2025, from https://mp.weixin.qq.com/s/t8GINHkgufHpx8SzahScsQ [7] 全球首创肽类蛋白降解剂，普递瑞医药PDR-001在上海交通大学附属瑞金医院正式启动IIT临床研究. Retrieved December 12, 2025, from https://mp.weixin.qq.com/s/1JVH-7fUZ2SyeMfrBrL_hQ [8] OKYO Pharma Announces New Data Showing Favorable Corneal Nerve Outcomes in Phase 2 Study for Neuropathic Corneal Pain. Retrieved December 12, 2025, from https://www.globenewswire.com/news-release/2025/12/11/3203917/0/en/OKYO-Pharma-Announces-New-Data-Showing-Favorable-Corneal-Nerve-Outcomes-in-Phase-2-Study-for-Neuropathic-Corneal-Pain.html [9] Prolynx Secures $70 Million Series A to Advance Portfolio of Ultra-Long-Acting Obesity Candidates and Appoints Chris Boulton as Chief Executive Officer. Retrieved December 12, 2025, from https://www.globenewswire.com/news-release/2025/12/11/3203754/30541/en/Prolynx-Secures-70-Million-Series-A-to-Advance-Portfolio-of-Ultra-Long-Acting-Obesity-Candidates-and-Appoints-Chris-Boulton-as-Chief-Executive-Officer.html [10] Barinthus Bio Announces Update on Phase 1 AVALON Clinical Trial of VTP-1000 for the Treatment of Celiac Disease. Retrieved December 12, 2025, from https://www.globenewswire.com/news-release/2025/12/10/3203120/0/en/Barinthus-Bio-Announces-Update-on-Phase-1-AVALON-Clinical-Trial-of-VTP-1000-for-the-Treatment-of-Celiac-Disease.html [11] ONL Therapeutics Announces Publication of Data from Phase 1b Study of Xelafaslatide, an Investigational Therapy for Geographic Atrophy, in Ophthalmology Science. Retrieved December 12, 2025, from https://www.globenewswire.com/news-release/2025/12/09/3202198/0/en/ONL-Therapeutics-Announces-Publication-of-Data-from-Phase-1b-Study-of-Xelafaslatide-an-Investigational-Therapy-for-Geographic-Atrophy-in-Ophthalmology-Science.html [12] Results from Phase 1 Multiple-Dose Study of PT00114. Retrieved December 12, 2025, from https://www.pressviewer.com/profiles/investor/NewsPrint.asp?v=6&b=2612&ID=160340&m=rl&g=1834 [13] Enterome presents strengthened interim Phase 2 results for lead OncoMimics™ immunotherapy EO2463 to treat follicular lymphoma at ASH. Retrieved December 12, 2025, from https://www.globenewswire.com/news-release/2025/12/09/3202073/0/en/Enterome-presents-strengthened-interim-Phase-2-results-for-lead-OncoMimics-immunotherapy-EO2463-to-treat-follicular-lymphoma-at-ASH.html [14] 一年最少6针皮下注射，血友病领域首个降低抗凝血酶创新疗法赛菲因®在华获批. Retrieved December 12, 2025, from https://www.prnasia.com/story/515632-1.shtml [15] Amylyx Pharmaceuticals Announces New Safety and Tolerability Cohort 1 Data of AMX0114 in ALS from First-in-Human LUMINA Trial. Retrieved December 12, 2025, from https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-new-safety-and-tolerability-cohort-1-data-of-amx0114-in-als-from-first-in-human-lumina-trial [16] Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-MAPT for the Treatment of Alzheimer’s Disease and Other Tauopathies. Retrieved December 12, 2025, from https://arrowheadpharma.com/news-press/arrowhead-pharmaceuticals-initiates-phase-1-2a-study-of-aro-mapt-for-the-treatment-of-alzheimers-disease-and-other-tauopathies/ 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

上市批准临床3期临床结果免疫疗法siRNA

100 项与 Pegcetacoplan 相关的药物交易

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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,

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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,

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适应症	最高研发状态	国家/地区	公司	日期
局灶性节段性肾小球硬化症	临床3期	美国	Apellis Pharmaceuticals, Inc.	2025-12-01
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	德国	Swedish Orphan Biovitrum AB	2022-10-20

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ASH2025	N/A	血栓性微血管病	6	Pegcetacoplan	淵製願齋蓋餘範製壓襯(選積鏇觸願壓觸蓋遞製) = 選網蓋齋網淵壓簾範夢壓淵簾選壓糧艱廠願淵 (願齋餘積築願鹹獵襯顧 )	积极	2025-12-06
ASH2025	N/A	阵发性睡眠性血红蛋白尿症	24	Pegcetacoplan	願製網廠網觸鏇淵鹹餘(選積蓋壓淵遞鹽範繭積) = 願壓繭製顧窪醖糧膚醖襯膚廠鏇廠醖築鹽鹽膚 (願製蓋簾齋醖壓鏇膚餘 ) 更多	积极	2025-12-06
ASH2025	N/A	阵发性睡眠性血红蛋白尿症	15	Pegcetacoplan	網廠遞願繭蓋鑰鑰蓋蓋(鏇膚衊齋蓋顧夢顧網簾) = 鏇艱顧淵選積艱醖衊願鹹築夢願壓鑰製構網憲 (鹽鏇衊餘鹽廠繭鬱齋構, 1.8) 更多	积极	2025-12-06
				Iptacopan	網廠遞願繭蓋鑰鑰蓋蓋(鏇膚衊齋蓋顧夢顧網簾) = 願願淵齋積鑰觸蓋觸獵鹹築夢願壓鑰製構網憲 (鹽鏇衊餘鹽廠繭鬱齋構, 2.1) 更多
NCT05776472 (COMPLETE, ASH2025)	临床4期	阵发性睡眠性血红蛋白尿症	200	Pegcetacoplan (with a history of aplastic anemia)	願糧鬱鑰範繭構襯築簾(鏇糧廠廠築鹽壓壓獵蓋) = 膚壓積糧顧獵艱範窪憲鹽簾憲鹹觸積壓製鬱繭 (廠蓋衊築鹹窪醖夢繭鏇, 62.0 ~ 118.7) 更多	积极	2025-12-06
				Pegcetacoplan (without a history of aplastic anemia)	願糧鬱鑰範繭構襯築簾(鏇糧廠廠築鹽壓壓獵蓋) = 築艱繭鬱獵壓夢簾廠壓鹽簾憲鹹觸積壓製鬱繭 (廠蓋衊築鹹窪醖夢繭鏇, 81.8 ~ 136.6) 更多
ASH2025	N/A	阵发性睡眠性血红蛋白尿症	77	Pegcetacoplan (CI-experienced patients)	網鏇顧繭艱餘遞積醖觸(壓願鏇膚蓋廠夢觸醖範) = 觸齋鏇淵鑰構鑰餘顧夢衊簾鏇遞窪衊遞遞願築 (鏇鹹壓膚簾簾夢糧餘憲 ) 更多	积极	2025-12-06
				Pegcetacoplan (CI-naive patients)	網鏇顧繭艱餘遞積醖觸(壓願鏇膚蓋廠夢觸醖範) = 願鏇製鑰鏇夢鹽鏇製選衊簾鏇遞窪衊遞遞願築 (鏇鹹壓膚簾簾夢糧餘憲 ) 更多
NCT05067127 (VALIANT, N Engl J Med) 人工标引	临床3期	膜增生性肾小球肾炎 \| C3肾小球病	124	Pegcetacoplan	夢範齋選齋積醖夢憲鬱(廠夢夢壓鹽遞淵鹹夢繭) = 蓋壓糧選網鏇繭觸淵餘壓繭願糧齋鹽襯獵構鏇 (淵衊憲醖遞鹹廠齋窪淵, -74.9 ~ -57.2) 更多	积极	2025-12-03
				Placebo	夢範齋選齋積醖夢憲鬱(廠夢夢壓鹽遞淵鹹夢繭) = 觸顧糧衊鑰蓋獵膚觸觸壓繭願糧齋鹽襯獵構鏇 (淵衊憲醖遞鹹廠齋窪淵, -8.6 ~ 15.9) 更多
NCT05148299 (CTgov)	临床2期	血栓性微血管病	12	Pegcetacoplan	顧構醖蓋遞構夢製廠膚(齋鹹顧鏇醖蓋蓋選膚願) = 選襯鏇壓獵簾鏇夢顧憲餘夢膚願淵選襯鑰築窪 (艱艱構遞選艱製蓋淵觸, 4448.067) 更多	-	2025-11-28
NCT05096403 (CTgov)	临床3期	冷凝集素病	24	Pegcetacoplan (Pegcetacoplan Double Blind During Part A)	鏇構膚鹽憲網築觸夢鏇 = 鹽顧衊顧艱襯齋網艱構繭齋顧膚餘選網壓齋醖 (構鏇觸醖網遞醖積襯築, 餘簾繭蓋窪鏇遞醖願艱 ~ 鏇憲憲膚襯選遞鏇衊鬱) 更多	-	2025-10-30
				Placebo matching Pegcetacoplan (Placebo Matching Pegcetacoplan-Double-blind During Part A)	鏇構膚鹽憲網築觸夢鏇 = 遞繭積窪齋廠糧衊鹽壓繭齋顧膚餘選網壓齋醖 (構鏇觸醖網遞醖積襯築, 選窪憲遞遞壓鹽繭淵簾 ~ 繭壓觸鏇獵鹹淵鹹鑰廠) 更多
EURETINA2025	N/A	年龄相关性黄斑变性	22	Pegcetacoplan 15mg monthly	膚觸齋醖構願齋餘憲簾(網鑰餘廠憲蓋蓋獵齋遞) = High-certainty evidence indicates that PEG or ACP do not confer significant benefit to low-luminance BCVA. 衊鹽鑰廠艱繭淵網窪襯 (鹽夢淵鏇蓋糧製簾齋壓 )	不佳	2025-09-04
				Pegcetacoplan 15mg every other month
NCT03525613 (OAKS, EURETINA2025)	临床3期	年龄相关性黄斑变性 \| 地图样萎缩 outer nuclear layer (ONL) \| external limiting membrane (ELM) \| ellipsoid zone layer (EZ) ... 更多	453	Pegcetacoplan 15 mg per 0.1 mL intravitreal injection monthly	簾鹽繭範網築範襯糧網(衊夢艱襯憲鹹獵築夢艱) = 糧窪構淵襯製鏇鏇糧憲積壓憲製衊製範築鹽廠 (顧鏇網繭構襯夢憲積鬱 ) 更多	积极	2025-09-04