更新于：2024-11-19

Nipocalimab

尼卡利单抗

更新于：2024-11-19

概要

概要

基本信息

药物类型

单克隆抗体

别名

anti-FcRn monoclonal antibody(Momenta Pharmaceuticals)、Immunoglobulin g1, anti-(human neonatal fc receptor) (human monoclonal m281 .gamma.-1 chain), disulfide with human monoclonal m281 .lambda.-chain, dimer、Nipocalimab (USAN)

+ [4]

靶点

FcRn

作用机制

FcRn拮抗剂(IgG受体FcRn大亚基p51拮抗剂)、免疫调节剂

治疗领域

肿瘤免疫系统疾病神经系统疾病+ [7]

在研适应症

重症肌无力胎儿/新生儿同种免疫性血小板减少症新生儿同种免疫性血小板减少症+ [10]

非在研适应症-

原研机构

Momenta Pharmaceuticals, Inc.

在研机构

强生（中国）投资有限公司Janssen Research & Development LLCJanssen-Cilag International NV

非在研机构

Momenta Pharmaceuticals, Inc.

最高研发阶段申请上市

首次获批日期-

最高研发阶段(中国)临床3期

特殊审评突破性疗法 (美国)、快速通道 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、孤儿药 (日本)、突破性疗法 (中国)

登录后查看时间轴

序列信息

Sequence Code 19395495L

来源: *****

Sequence Code 19395504H

来源: *****

关联

项与尼卡利单抗相关的临床试验

NCT04883619 / Not yet recruiting临床2期

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Nipocalimab in Adult Participants With Active Lupus Nephritis

The purpose of this study is to evaluate the efficacy of nipocalimab versus placebo in participants with active Lupus Nephritis (LN).

开始日期2026-01-15

申办/合作机构

Janssen Research & Development LLC

NCT06533098 / Not yet recruiting临床3期

Multicenter, Open-Label, Randomized Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

The purpose of this study is to assess the efficacy and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT).

开始日期2025-02-05

申办/合作机构

Janssen Research & Development LLC

NCT06449651 / Recruiting临床3期

Double-blind, Randomized, Placebo-controlled Study Evaluating the Safety and Efficacy of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) in At-risk Pregnancies

The purpose of this study is to evaluate the effectiveness of nipocalimab compared with placebo in reducing the risk of fetal and neonatal alloimmune thrombocytopenia (FNAIT).

开始日期2024-10-01

申办/合作机构

Janssen Research & Development LLC

100 项与尼卡利单抗相关的临床结果

登录后查看更多信息

100 项与尼卡利单抗相关的转化医学

登录后查看更多信息

100 项与尼卡利单抗相关的专利（医药）

登录后查看更多信息

项与尼卡利单抗相关的文献（医药）

2024-12-31·mAbs

Interactions of the anti-FcRn monoclonal antibody, rozanolixizumab, with Fcγ receptors and functional impact on immune cells in vitro

Article

作者: Craggs, Graham ; Bithell, Rosemary F. ; Rapecki, Stephen ; Humphreys, David P. ; Cutler, Rona M. ; Barnes, Nicholas M. ; West, Shauna M. ; Smith, Bryan J. ; Sutton, Emma J. ; Qureshi, Omar S. ; Shock, Anthony ; McCluskey, Gillian ; Maroof, Asher

Rozanolixizumab is a humanized anti-neonatal Fc receptor (FcRn) monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4) sub-class, currently in clinical development for the treatment of IgG autoantibody-driven diseases. This format is frequently used for therapeutic mAbs due to its intrinsic lower affinity for Fc gamma receptors (FcγR) and lack of C1q engagement. However, with growing evidence suggesting that no Fc-containing agent is truly "silent" in this respect, we explored the engagement of FcγRs and potential functional consequences with rozanolixizumab. In the study presented here, rozanolixizumab was shown to bind to FcγRs in both protein-protein and cell-based assays, and the kinetic data were broadly as expected based on published data for an IgG4 mAb. Rozanolixizumab was also able to mediate antibody bipolar bridging (ABB), a phenomenon that led to a reduction of labeled FcγRI from the surface of human macrophages in an FcRn-dependent manner. However, the presence of exogenous human IgG, even at low concentrations, was able to prevent both binding and ABB events. Furthermore, data from in vitro experiments using relevant human cell types that express both FcRn and FcγRI indicated no evidence for functional sequelae in relation to cellular activation events (e.g., intracellular signaling, cytokine production) upon either FcRn or FcγR binding of rozanolixizumab. These data raise important questions about whether therapeutic antagonistic mAbs like rozanolixizumab would necessarily engage FcγRs at doses typically administered to patients in the clinic, and hence challenge the relevance and interpretation of in vitro assays performed in the absence of competing IgG.

2024-08-08·NEW ENGLAND JOURNAL OF MEDICINE

Nipocalimab in Early-Onset Severe Hemolytic Disease of the Fetus and Newborn

Article

作者: Ling, Leona E ; Emery, Stephen P ; Markham, Kara ; Pandya, Pranav ; Ocón-Hernández, Olga ; Windrim, Rory ; Audibert, François ; Schwartz, Lisa B ; Bussel, James B ; Norton, Mary E ; Moise, Kenneth J ; Tjoa, May Lee ; Oepkes, Dick ; Streisand, James B ; Devlieger, Roland ; Mirza, Arpana ; Smith, Valerie ; Sachs, Ulrich J ; Leu, Jocelyn H ; Miller, Russell S ; Bein, Gregor ; Kilby, Mark D ; Verweij, E J T Joanne ; Saeed-Khawaja, Shumyla ; Lopriore, Enrico ; Smoleniec, John ; Silver, Robert M ; Tiblad, Eleonor ; Pereira, Leonardo ; Komatsu, Yosuke

BACKGROUND:

In early-onset severe hemolytic disease of the fetus and newborn (HDFN), transplacental transfer of maternal antierythrocyte IgG alloantibodies causes fetal anemia that leads to the use of high-risk intrauterine transfusions in order to avoid fetal hydrops and fetal death. Nipocalimab, an anti-neonatal Fc receptor blocker, inhibits transplacental IgG transfer and lowers maternal IgG levels.

METHODS:

In an international, open-label, single-group, phase 2 study, we assessed treatment with intravenous nipocalimab (30 or 45 mg per kilogram of body weight per week) administered from 14 to 35 weeks' gestation in participants with pregnancies at high risk for recurrent early-onset severe HDFN. The primary end point was live birth at 32 weeks' gestation or later without intrauterine transfusions as assessed against a historical benchmark (0%; clinically meaningful difference, 10%).

RESULTS:

Live birth at 32 weeks' gestation or later without intrauterine transfusions occurred in 7 of 13 pregnancies (54%; 95% confidence interval, 25 to 81) in the study. No cases of fetal hydrops occurred, and 6 participants (46%) did not receive any antenatal or neonatal transfusions. Six fetuses received an intrauterine transfusion: five fetuses at 24 weeks' gestation or later and one fetus before fetal loss at 22 weeks and 5 days' gestation. Live birth occurred in 12 pregnancies. The median gestational age at delivery was 36 weeks and 4 days. Of the 12 live-born infants, 1 received one exchange transfusion and one simple transfusion and 5 received only simple transfusions. Treatment-related decreases in the alloantibody titer and IgG level were observed in maternal samples and cord blood. No unusual maternal or pediatric infections were observed. Serious adverse events were consistent with HDFN, pregnancy, or prematurity.

CONCLUSIONS:

Nipocalimab treatment delayed or prevented fetal anemia or intrauterine transfusions, as compared with the historical benchmark, in pregnancies at high risk for early-onset severe HDFN. (Funded by Janssen Research and Development; UNITY ClinicalTrials.gov number, NCT03842189.).

2024-08-01·CLINICAL DRUG INVESTIGATION

Pharmacokinetics and Pharmacodynamics of Nipocalimab, a Neonatal Fc Receptor Blocker, in Healthy Japanese Volunteers

Article

作者: Matsushima, Nobuko ; Harigae, Hideo ; Duffner, Jay ; Shibata, Sayori ; Schwartz, Lisa B ; Vermeulen, An ; Ling, Leona E ; Leu, Jocelyn H

BACKGROUND AND OBJECTIVES:

Nipocalimab is a high-affinity, fully human, effectorless immunoglobulin G1 monoclonal antibody targeting the neonatal Fc receptor and is currently under evaluation for the treatment of rare and prevalent immunoglobulin G autoantibody-mediated and alloantibody-mediated diseases. This phase I, randomized, double-blind, placebo-controlled, single-dose escalation study in healthy Japanese volunteers (N = 24) assessed the safety, pharmacokinetics, and effect on the serum immunoglobulin G level of single doses of nipocalimab.

METHODS:

Volunteers were grouped into three cohorts and received intravenous nipocalimab at 10, 30, or 60 mg/kg or placebo. To complement the study, genetic variation in the Fcγ receptor and transporter subunit of the neonatal Fc receptor was analyzed in Japanese and diverse populations.

RESULTS:

Nipocalimab was generally safe and well tolerated at all dose levels, with three (12.5% [3/24]) volunteers experiencing treatment-emergent adverse events across all nipocalimab doses. Mean serum immunoglobulin G levels decreased in a dose-dependent manner from baseline with nipocalimab treatment compared with placebo. Maximum serum nipocalimab concentrations demonstrated proportional increases with dose, while the area under the concentration-time curve was dose dependent and demonstrated non-linear increases with dose. Mean observed half-life was longer as the dose increased. Analysis of genetic variation in Fcγ receptor and transporter identified no unique Japanese variants or variants that alter amino acid sequences in or near the neonatal Fc receptor immunoglobulin G binding site targeted by nipocalimab.

CONCLUSIONS:

The comparable pharmacokinetic/pharmacodynamic profiles and highly conserved neonatal Fc receptor structure among diverse populations further support the clinical development of nipocalimab for the treatment of various immunoglobulin G autoantibody-mediated and alloantibody-mediated diseases across global sites and populations, including the Japanese population.

183

项与尼卡利单抗相关的新闻（医药）

2024-11-16

·CPHI制药在线

默沙东以至高33亿美元引进礼新医药双抗；和誉医药匹米替尼治疗腱鞘巨细胞瘤全球关键Ⅲ期研究成功 | 制药在线一周药闻复盘

点击蓝字关注我们本周，热点不少，首先是审评审批方面，很值得关注的就是科笛集团痤疮新药CU-10201在中国获批上市以及诺华首创核药177Lu-PSMA-617在中国申报上市；其次是研发方面，多个药取得重要研发进展，值得一说的是，和誉医药匹米替尼治疗腱鞘巨细胞瘤全球关键Ⅲ期研究成功；其次是交易及投融资方面，金额较大的是默沙东以总交易额约32.88亿美元引进礼新医药PD-1/VEGF双抗；最后是其他方面，呋喹替尼被宣告全部无效。本期盘点包括审评审批、研发、交易及投融资和其他四大版块，统计时间为2024.11.11-11.15，包含31条信息。审评审批 NMPA 上市批准 1、11月11日，NMPA官网显示，科笛集团的盐酸米诺环素泡沫剂（CU-10201）获批上市，用于9岁及以上儿童和成人患者的非结节性中度至重度寻常痤疮炎症性病变的局部治疗。这是一款外用4%米诺环素泡沫剂，2020年，科笛集团全资附属公司科笛生物与Foamix公司签订授权协议，获得CU-10201在大中华区（包括中国大陆、中国台湾、中国香港及中国澳门地区）的权益。 2、11月11日，NMPA官网显示，再鼎医药的艾加莫德注射液（皮下注射）新适应症获批，用于治疗成人慢性炎性脱髓鞘性多发性神经根神经病（CIDP）。艾加莫德皮下注射是由人IgG1抗体的Fc片段艾加莫德α静脉输注，以及重组人透明质酸酶PH20（rHuPH20）共同配制。申请 3、11月13日，CDE官网显示，诺华的镥[177Lu] 特昔维匹肽注射液（177Lu-PSMA-617）申报上市，用于治疗前列腺特异性膜抗原（PSMA）阳性转移性去势抵抗性前列腺癌（mCRPC) 、已接受雄激素受体通路抑制和紫杉类化疗的成年患者。该药是一种静脉注射的放射配体疗法(RLT)，该疗法将一种靶向PSMA的小分子化合物(配体)与治疗性放射性核素(一种放射性粒子，本例中为镥-177)相结合。 4、11月13日，CDE官网显示，北海康成的注射用维拉苷酶β（CAN103）申报上市，用于治疗成人和儿童Ⅰ型和Ⅲ型戈谢病。CAN103是北海康成与药明生物在罕见病领域的首个合作项目，是中国首个针对戈谢病开发的酶替代疗法（ERT）。 5、11月14日，CDE官网显示，复星医药和Bial-Portela&Ca共同申报的5.1类新药奥吡卡朋胶囊申报上市。这是复星医药引进的治疗原发性帕金森病的新药Opicapone胶囊，为新一代的儿茶酚-氧位-甲基转移酶抑制剂（COMT）抑制剂。 6、11月15日，CDE官网显示，艾美疫苗旗下艾美坚持生物制药的3.3类预防用生物制品13价肺炎球菌多糖结合疫苗申报上市，主要用于6周龄至5岁婴幼儿和儿童，预防该疫苗包含的13种肺炎血清型引起的侵袭性疾病（包括菌血症性肺炎、脑膜炎、败血症和菌血症）。 7、11月16日，CDE官网显示，亚盛医药1类新药力胜克拉片申报上市，推测适应症为难治或复发性慢性淋巴细胞白血病（CLL）/小淋巴细胞淋巴瘤（SLL）。力胜克拉片（lisaftoclax，APG-2575片）为亚盛医药在研的一款Bcl-2选择性抑制剂。临床批准 8、11月12日，CDE官网显示，石药集团巨石生物1类新药SYS6026注射液获批临床，拟开发治疗人乳头瘤病毒（HPV）16/18型相关高级别鳞状上皮内病变（HSIL）患者。这是一款治疗性疫苗，靶向HPV16和18亚型早期蛋白抗原E6和E7蛋白。本次为该产品首次在中国获批IND。 9、11月13日，CDE官网显示，赛诺菲1类新药lunsekimig注射液获批临床，拟开发用于治疗成人高风险哮喘。该产品此前已经在中国获批IND，针对适应症为成人中重度哮喘。lunsekimig是一款将靶向IL-13和靶向TSLP的重链可变区（VHH）连接在一起的纳米抗体。 10、11月15日，CDE官网显示，阿斯利康1类新药AZD0486获批两项临床，分别拟用于治疗成人既往未经治疗的滤泡性淋巴瘤；至少接受2线或以上系统性治疗的成人复发难治性滤泡性淋巴瘤。AZD0486是阿斯利康在研的一款靶向CD19/CD3的双特异性T细胞衔接蛋白。申请 11、11月11日，CDE官网显示，和铂医药的HBM9378/SKB378申报临床，拟用于治疗慢性阻塞性肺疾病（COPD）。HBM9378/SKB378是和铂医药与科伦博泰共同开发的一款全人源抗体，双方共同享有其全球权益，靶向胸腺基质淋巴细胞生成素（TSLP），通过阻断TSLP与其受体之间的相互作用来抑制TSLP介导的信号通路。 12、11月11日，CDE官网显示，金赛药业的GenSci120注射液申报临床。GenSci120注射液是一款PD-1激动剂。全球范围内已有十几款PD-1激动剂类产品正在研发，但多数尚处于临床前阶段，进展较快的产品包括Anaptys Bio开发的激活性PD-1单克隆抗体rosnilimab，正在Ⅱ期临床研究中被探索用于治疗类风湿关节炎和溃疡性结肠炎。 13、11月13日，CDE官网显示，阿斯利康1类新药AZD0022申报临床。AZD0022是一款KRAS G12D抑制剂，拟开发治疗实体瘤。该产品目前在国际范围内处于Ⅰ期临床试验阶段。阿斯利康在2023年11月与祐森健恒达成授权合作，获得临床前阶段的靶向KRAS G12D突变的小分子候选药物UA022项目的全球独家授权，推测可能是本次申报临床的AZD0022。 14、11月14日，CDE官网显示，百济神州1类新药BG-60366片申报临床。BG-60366是一款新型EGFR嵌合式降解激活化合物（CDAC），该产品可广泛覆盖多种EGFR突变，破坏EGFR支架功能，产生持久的信号抑制作用。当用于前期治疗线时，非冗余机制有望防止发生耐药。优先审评 15、11月12日，CDE官网显示，强生的尼卡利单抗（nipocalimab）拟纳入优先审评，用于治疗自身抗体阳性（抗乙酰胆碱受体[AChR]阳性、抗肌肉特异性酪氨酸激酶[MuSK]阳性或抗低密度脂蛋白受体4 [LRP4]阳性）的全身型重症肌无力（gMG）成人患者和青少年患者（大于12岁）。该药是一款FcRn单抗，2020年8月，强生获得了这款药物。 16、11月12日，CDE官网显示，诺华的阿曲生坦（atrasentan）拟纳入优先审评，用于降低有疾病进展风险的原发性免疫球蛋白A肾病（IgAN）成人患者的蛋白尿。该药是一款ETA拮抗剂，2023年，诺华获得了atrasentan，同年，atrasentan治疗IgA肾病的Ⅲ期ALIGN研究在36周期中分析取得积极结果，达到了主要疗效终点，能够显著降低患者蛋白尿。 17、11月12日，CDE官网显示，荣昌生物的注射用泰它西普拟纳入优先审评，该产品与常规治疗药物联合，用于治疗成人全身型重症肌无力（gMG）患者。泰它西普是一款双靶抗体融合蛋白，可同时靶向BLyS和APRIL，直击致病性抗体产生的源头——B细胞及浆细胞，从而减少致病性抗体的产生，发挥治疗作用。突破性疗法 18、11月14日，CDE官网显示，宝济药业1类新药注射用KJ103拟纳入突破性疗法，用于高度致敏的肾移植患者进行脱敏治疗，有效清除预存HLA抗体预防超急性排斥反应。KJ103是一种新型低免疫原性IgG降解酶，来源于非人致病性的马链球菌亚种。 19、11月15日，CDE官网显示，信达的IBI354拟纳入突破性疗法，适应症为铂耐药卵巢癌。IBI354是信达基于创新ADC linker-payload NT3技术平台，自主研发的抗HER2单克隆抗体-喜树碱衍生物偶联物。 FDA 上市申请 20、11月13日，FDA官网显示，阿斯利康和第一三共联合开发的Trop2靶向抗体偶联药物（ADC）datopotamab deruxtecan（Dato-DXd）递交生物制品许可申请（BLA），寻求该疗法获加速批准，用以治疗携带表皮生长因子受体（EGFR）突变肿瘤的局部晚期或转移性非小细胞肺癌（NSCLC）成年患者，这些患者之前曾接受过全身性治疗（包括EGFR靶向治疗）。 21、11月15日，FDA官网显示，赛诺菲和再生元的度普利尤单抗（商品名：Dupixent）递交补充生物制品许可申请（sBLA），用于治疗慢性自发性荨麻疹（CSU），PDUFA预定审批日期为2025年4月18日。目前，美国仅奥马珠单抗一款生物制剂获批用于治疗CSU。此外，度普利尤单抗已在日本获批用于治疗CSU。研发临床状态 22、11月11日，药物临床试验登记与信息公示平台显示，加科思登记了一项评估JAB-23E73用于KRAS基因改变的晚期实体瘤的安全性、耐受性、药代动力学以及初步抗肿瘤活性的多中心、开放性Ⅰ/Ⅱa期研究。这是关于该药的首个临床试验。JAB-23E73可同时抑制活性及非活性状态的KRAS，对HRAS、NRAS无明显抑制。 23、11月13日，药物临床试验登记与信息公示平台显示，石药登记了一项评估SYSA1801联合卡培他滨对比奥沙利铂联合卡培他滨（CAPOX方案）一线治疗CLDN18.2阳性、PD-L1 CPS<5的不可切除的局部晚期或转移性胃或胃食管结合部腺癌的有效性和安全性的随机、多中心、Ⅰb/Ⅲ期研究。SYSA1801是石药自主研发的新型抗CLDN18.2的ADC药物。 24、11月14日，药物临床试验登记与信息公示平台显示，宜联生物启动了一项评估注射用YL201对比注射用盐酸托泊替康在复发性小细胞肺癌（SCLC）患者中的有效性和安全性的多中心、随机对照、开放标签的Ⅲ期研究。YL201是宜联生物自主研发的靶向B7-H3的ADC药物。临床数据 25、11月12日，和誉医药宣布，匹米替尼治疗腱鞘巨细胞瘤（TGCT）的Ⅲ期MANEUVER研究在第25周客观缓解率（ORR）方面取得了统计学意义上的显著改善，达到54.0%，而安慰剂为3.2%。该药是一款高选择性、高活性CSF-1R小分子抑制剂，已获中美欧三地突破性治疗药物和优先药物认定，以及美国快速通道认定。2023年12月，和誉医药与默克签署授权合约。 26、11月14日，GSK宣布，一项评估BCMA ADC药物belantamab mafodotin（Blenrep）联合硼替佐米和地塞米松（BorDex）头对头对比达雷妥尤单抗联合BorDex二线治疗复发或难治性多发性骨髓瘤（RRMM）的Ⅲ期DREAMM-7研究的中期分析取得积极结果，到达了总生存期（OS）的关键次要终点。交易及投融资 27、11月12日，Apollo Therapeutics宣布，已与东阳光药就APL-18881的开发达成独家许可协议。APL-18881是东阳光药开发的一种靶向FGF21和GLP-1受体的双特异性融合蛋白。根据协议，东阳光药将保留APL-18881在中国的开发、生产和商业化权利，并授予Apollo Therapeutics该药物在全球其他地区开发、生产和商业化所有当前和未来适应症的权利。东阳光药将获得预付款1200万美元和高达9.26亿美元的开发、监管和商业里程碑付款。 28、11月13日，普米斯生物宣布，与BioNTech达成股权收购协议。根据协议，BioNTech将以8亿美元预付款收购普米斯100%已发行股本（可根据惯例对收购价格进行调整），支付方式主要为现金和部分美国存托股份（ADS）。此外，BioNTech将在普米斯达到双方约定的里程碑条件时，额外支付最高1.5亿美元里程碑付款。 29、11月14日，默沙东宣布。与礼新医药达成协议，获得礼新医药在研PD-1/VEGF双特异性抗体LM-299的全球开发、生产和商业化独家许可权利。根据协议，礼新医药已授权默沙东LM-299的全球开发、生产和商业化独家许可。礼新医药将获得5.88亿美元的首付款，还将获得最高27亿美元的里程碑付款。其他 30、11月11日，国家知识产权局针对保护呋喹替尼的晶型Ⅰ的专利CN201580047368.6发出无效宣告请求审查决定书（决定号581990），宣告专利权全部无效。无效宣告请求人为个人（刘奇）。该专利首次经历无效挑战就被全部无效成功。呋喹替尼是由和记黄埔的一种高选择性的血管内皮生长因子受体（VEGFR）口服抑制，2018年4月在中国首次获批上市。 31、11月15日，NMPA批准首个优化创新药临床试验审评审批试点项目。CDE按照试点工作方案的要求，完成了拟由北京大学肿瘤医院开展的用于治疗晚期实体瘤的注射用MK-6204（SKB535）临床试验申请审评审批，为首个按照试点工作方案批准的临床试验申请，审评审批用时21日。通过CDE官网查询，这是由科伦博泰申报的1类生物制品新药。【智药研习社线上研习会报名】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

临床3期疫苗上市批准优先审批放射疗法

2024-11-15

·凯莱英药闻

IgG降低超过77%！强生披露潜在“同类最佳”FcRn单抗最新2期研究积极数据

欢迎关注凯莱英药闻 2024年11月14日，强生在在美国风湿病学会（ACR）上，披露了在研药物nipocalimab在中度至重度干燥综合征 (SjD) 成人患者中的2期DAHLIAS 研究最新分析结果，显示接受nipocalimab的SjD患者的疾病活动性关键指标得到改善，IgG显著降低超过77%。 Nipocalimab 是一款潜在同类最佳的靶向FcRn的抗体疗法；亦有望成为该靶点首个用于治疗SjD的药物。关于最新研究数据 DAHLIAS是一项II期多中心、随机、安慰剂对照双盲研究，研究对象是抗Ro60和/或抗Ro52 IgG抗体血清阳性的中度至重度活动性原发性SjD成人患者，按照1:1:1的比例被随机分配到接受每2周一次静脉注nipocalimab（5或15 mg/kg）或安慰剂，直至第22 周，并接受方案允许的背景标准治疗。安全性评估持续到第30周。结果显示：在 24 周时，接受nipocalimab治疗的患者ClinESSDAIa 评分显著改善，达到主要终点。此外，关键次要终点包括包括多器官评估（DALc）、医生评估（PhGAd）和临床试验终点综合工具（STARe、CRESSf）；重要的SjD症状呈改善趋势，包括口干、眼干和阴道干涩。具体来看，每两周接受 15 mg/kg 治疗的患者 IgG（包括自身抗体）水平显著降低；抗 Ro 和抗 La 自身抗体基线水平最高的受试者的 ClinESSDAI 改善通常最大，这与nipocalimab诱导的 IgG 和总 IgG 自身抗体大幅减少有关。此外，在第 24 周，与安慰剂组 (32.7% vs. 16%) 相比，高剂量nipocalimab组 (15 mg/kg) 患者客观唾液流量有所改善 (即至少比基线增加 50%)。关于Nipocalimab Nipocalimab旨在以高亲和力结合来阻断 FcRn 并降低循环免疫球蛋白 G (IgG) 抗体的水平，而不会影响其他免疫功能。 2020 年8 月，强生以65 亿美元收购生物技术公司Momenta，获得3期药物nipocalimab，加强自身免疫病管线的布局。目前，该药物获FDA和EMA授予胎儿和新生儿溶血性疾病(hddn)和温热性自身免疫性溶血性贫血(wAIHA)、全身型重症肌无力（gMG）、胎儿新生儿同种免疫性血小板减少症(FNAIT)快速通道指定；获FDA和EMA授予wAIHA、hddn、慢性炎性脱髓鞘性多发性神经根神经病（CIDP）和FNAIT孤儿药认定；获FDA授予hddn突破性疗法；获EMA授予hddn孤儿药认定。关于干燥综合征干燥综合征是最常见的自身抗体驱动疾病之一，目前尚无任何疗法获批用于治疗该疾病的系统疗法。临床上多隐匿起病，常有唾液腺、泪腺功能受损，血清学特征为自身抗体的大量产生和高免疫球蛋白血症。超过 50% 的干燥综合征患者患有中至重度疾病，疾病负担可能与类风湿性关节炎或系统性红斑狼疮一样高，通常导致生活质量和免疫功能紊乱以及死亡风险增加。据统计，全球约 400 万人患病，女性发病率是男性的 9 倍。关于FcRn药物 Part.1 靶点介绍 FcRn 又称Brambell受体，是一种由Fcgrt 基因编码的非典型的Fcγ 受体（FcγRs）。FcγRs 对免疫球蛋白（IgG）的识别代表了一种重要的免疫调节方式，即能够不IgG 上保守的Fc 结构域相结合，再以IgG免疫复合物（IgG IC）的形式递送独特的抗原决定簇。这些抗原决定簇被装载到I 类和II 类MHC 分子上，随后刺激相关CD8+和CD4+ T 细胞。IgG主要由脾脏以及淋巴结中的浆细胞合成和分泌，常以单体形式存在，是血清中最主要的抗体成分。在大多数体液免疫过程中，无论是针对病毒还是细胞病原体的保护作用也都涉及IgG 所介导的效应子功能。IgG能够清除侵入人体的外源异物，FcRn则在人体内用于维持IgG水平，增长IgG抗体半衰期，减缓IgG在溶酶体中的降解速度。研究表明，FcRn 广泛表达于各个组织的细胞中，其中包括与血管壁对齐的内皮细胞，肠上皮细胞、气道上皮细胞、胎盘合胞体滋养细胞、肝细胞、内皮细胞、髓样细胞以及支气管导管细胞等；此外，在造血细胞如B 细胞、巨噬细胞以及树突状细胞中也収现了高水平表达的FcRn，理论上可导致七八十种自身免疫性疾病。由FcRn 介导的抗原递呈过程不能影响各个部位的稳态免疫激活，是内源抗肿瘤活性及肿瘤免疫监视系统中的重要组成部分，该过程已被广泛认可的CD8+ T 细胞能够抗肿瘤方面的重要性相一致，因此FcRn 介导的交叉递呈也能够启动肿瘤保护的机制。 Part.2 交易情况据不完全统计，围绕FcRn开展相关交易近9笔，除强生收购Momenta的nipocalimab外，其它交易主要围绕巴托利单抗、efgartigimod（艾加莫德）以及tifalibep（替法利贝普）等药物开展。 Part.3 在研药物的进展据不完全统计，目前全球在研的FcRn药物约十余种，以单克隆抗体为主；已获批的适应症主要包括重症肌无力、慢性炎症性脱髓鞘性多发性神经病、原发免疫性血小板减少症等。 1、艾加莫德艾加莫德（Efgartigimod）是一款针对抗体介导自身免疫疾病的、高度靶向IgG 的新型治疗药物，是全球首款FcRn 抑制剂。Efgartigimod 于2021 年12月获FDA批准上市，用于治疗乙酰胆碱受体（AChR）抗体阳性的成人gMG。2024年6月，FDA批准了艾加莫德皮下注射（VYVGART Hytrulo）用于治疗CIDP。再鼎医药于2021年1月从Argenx 引进艾加莫德，拥有在大中华区（包括中国大陆、香港、澳门和台湾地区）的独家开发和商业化权利。2023年7月，NMPA批准了艾加莫德α注射液的生物制品上市许可申请，用于与常规治疗药物联合，治疗AChR抗体阳性的成人gMG患者；同月，该药物皮下注射剂型的生物制品上市许可申请（BLA）获NMPA受理，用于治疗成人gMG。2024年11月，NMPA已经批准了艾加莫德皮下注射制剂用于治疗CIDP成人患者的上市申请，这是国内首个且目前唯一获批CIDP适应证的药物。除此以外，日本厚生劳动省（MHLW）于2024年3月批准艾加莫德静脉输注剂型用于治疗原发免疫性血小板减少症（ITP）成人患者。同月，公司宣布，基于对2期临床研究RHO的数据分析，将计划继续推进艾加莫德在SjD成人患者中的3期临床研究。 gMG：在关键性III 期ADAPT临床研究中，以1:1的比例随机分配接受efgartigimod或安慰剂。结果显示：研究达到主要研究终点：在乙酰胆碱受体抗体阳性（AChR Ab+）的gMG 患者中，根据重症肌无力日常生活活动（MG-ADL）评分，与安慰剂组相比，efgartigimod 治疗组有更高比例的患者为应答者（67.7%vs 29.7%；p<0.0001）。应答者被定义为在MG-ADL 评分上至少有2 分的改善，且连续4 周或更长时间。此外，efgartigimod 治疗组有40%的患者实现了微小状态表达（定义为MG-ADL 评分为0[无症状]或1），而安慰剂组实现这一目标的患者比例仅为11.1%。在AChR-Ab+应答者中，84.1%的患者在治疗的头2 周内MGADL评分有临床意义的改善。安全性方面，efgartigimod 治疗组与安慰剂相当（AEs：77% vs 84%），最常见的不良反应是头痛（29% vs 28%）、鼻咽炎（12% vs 18%）。此外，3期ADAPT-SC研究显示，在成年gMG患者中，与静脉输注相比，皮下注射剂型在第29天时总IgG的降低具有非劣效性。在第29天，与基线水平相比，艾加莫德α皮下注射剂型平均总IgG减少66.4%，而静脉输注剂型为62.2%。支持皮下注射剂的上市申请。 CIDP：ADHERE研究是迄今最大规模的治疗CIDP的临床研究；其中，69%（221/322）接受艾加莫德皮下注射治疗的患者，无论此前是否接受过治疗都确认出现临床改善，包括行动、功能和力量的改善。研究达到了主要研究终点（p<0.0001），研究证实，即与安慰剂相比，艾加莫德皮下注射可降低复发风险61%（HR：0.39 95% CI：0.25；0.61）。安全性与此前艾加莫德静脉输注临床研究和实际应用中的安全性特征基本一致。对中国患者的亚组分析显示，与安慰剂相比，艾加莫德皮下注射的复发率降低了69%。此外，在该研究的开放标签部分接受治疗的中国患者中，有78%表现出临床改善的证据（ECI）。 ITP：全球3期临床研究ADVANCE-IV结果已发表于2023年9月出版的《柳叶刀》，研究达到了主要终点，即与安慰剂相比，接受艾加莫德治疗的慢性ITP患者获得血小板计数持续应答的比例更高。研究显示艾加莫德治疗慢性ITP患者和持续性ITP患者起效快速，以及在国际工作组（IWG）评分中有51%缓解率（IWG评分是由ITP国际工作组制定的原发免疫性血小板减少症评估工具，与临床治疗高度相关）。在不同类型的患者中均观察到了主要终点应答者，无论患者年龄、疾病严重程度、确诊时间、既往ITP治疗或背景用药情况。在这项为期24周的研究中，艾加莫德耐受性良好，观察到的安全性和耐受性与此前的临床研究一致。 2、Rozanolixizumab Rozanolixizumab是优时比（UCB）开发的一种皮下注射的人源化单克隆抗体，以高亲和力特异性结合FcRn；通过阻断FcRn 和免疫球蛋白 G (IgG) 的相互作用，加速抗体的分解代谢并降低致病性 IgG 自身抗体的浓度。2023年6月，FDA批准Rystiggo（rozanolixizumab）的上市申请，用于治疗gMG成人患者。 2023年5月，公司发布了一项随机、双盲、安慰剂平行对照、适应性两阶段设计的3期MycarinG试验数据，将300例患者随机接受rozanolixizumab治疗7 mg/kg或10 mg/kg或安慰剂，按每周一次皮下注射，治疗期为6周，随访观察期为8周。主要终点指标为MG-ADL，次要终点指标为MGC、QMG、患者自主报告的结局（PRO）及MG-ADL；安全性终点为导致治疗中断的不良事件（TEAEs）。结果显示：（1）在治疗后的第43天，高、低剂量组均显现MG-ADL的大幅改善，相比基线变化值为–3.40与-3.37，安慰剂组为-0.78，且药物治疗组显著优于安慰剂组；（2）对MuSK抗体阳性gMG患者的亚组分析显示，高剂量组改善幅度为–4.16，低剂量组为-7.28，安慰剂组为2.28；（3）在治疗后的第43天，高、低剂量组在MGC、QMG分值及PRO的改善均显著优于安慰剂组；症状改善最早于治疗后的第8天显现，贯穿整个治疗期，直至第99天又恢复至基线水平。（4）MuSK抗体阳性gMG患者亚组显示，无论接受的是高剂量或低剂量药物，全部都获得MG-ADL明显改善（改善幅度≥2分），而安慰剂组仅14%（1/7）的患者达到这一改善幅度；以经治后达到最轻症状的患者比率看，高剂量组为28%，低剂量组为26%，安慰剂组为3%。（5）总IgG水平的大幅下降出现在治疗后的第8天，然后第99天恢复至基线水平。在安全性上，任何TEAEs或药物相关TEAEs的发生率均很低，且与安慰剂组相近；高剂量组因TEAEs导致治疗中断的发生率略高，症状包括腹泻、上腹痛、呕吐、口腔疱疹、转移性鳞状细胞癌、瘙痒症和深静脉血栓形成。研究期间未出现死亡、严重超敏反应及自杀倾向的患者，患者对药物皮下注射的耐受性良好。 3、巴托利单抗巴托利单抗（HBM9161）是一款抗FcRn单抗，可阻断FcRn-IgG相互结合，加速体内IgG（包括致病性IgG）的清除，有望为包括重症肌无力在内的致病性IgG介导的自身免疫性疾病带来新一代疗法。2022年10月，石药集团与和铂医药达成授权协议，取得巴托利单抗在大中华区的开发、生产和商业化权利。 2024年3月，公司公布了一项Ⅲ期临床研究结果，在这项随机、双盲、安慰剂对照平行研究中，共纳入132例全身型重症肌无力成人患者。这些患者随机分配至巴托利单抗治疗组及安慰剂组，进行以6周为一个周期的皮下注射治疗，以患者重症肌无力日常生活量表（MG-ADL）得分较基线的变化情况，评估巴托利单抗治疗对疾病症状的改善效果。数据显示，在治疗开始的第2周，巴托利单抗治疗组患者MG-ADL评分改善率曲线就较对照组产生了明显的分离，提示巴托利单抗治疗能够快速起效，改善症状；在第43天，结束了第一个治疗周期后，巴托利单抗治疗组的ADL评分持续改善率（ADL评分较基线改善3分且连续持续4周的比例）达58.2%（对照组为31.1%）；在第一个治疗周期中，巴托利单抗治疗组患者达到最轻微症状表现（MSE，定义为ADL评分为0或1）的比例为25.4%，远高于对照组4.7%。除此以外，在MG定量评分、MG复合评分、15项重症肌无力生活质量评分分析中，巴托利单抗治疗组与对照组也呈现了相似的趋势；在安全性上，巴托利单抗治疗组与对照组治疗后出现的不良事件（TEAEs）发生率相近，总体耐受性及安全性数据良好。 4、Orilanolimab Orilanolimab (SYNT001)是一种FcRn单抗，有望能改善多种由IgG介导的罕见病的治疗。该药物最初由Syntimmune公司开发，后由Alexion收购；2020年12月，阿斯利康拟斥资约390亿美元，收购Alexion公司；该药物正式纳入阿斯利康管线。目前，orilanolimab正在评估用于治疗温热自身免疫性溶血性贫血（WAIHA）患者和寻常型PV或PF患者。参考资料 1、各公司官网 2、中信建投、西南证券感谢关注、转发，转载授权、加行业交流群，请加管理员微信号“hxsjjf1618”。 “在看”点一下

孤儿药临床2期突破性疗法临床3期临床结果

2024-11-14

·PRNewswire

Nipocalimab demonstrates significant clinical improvement in disease activity and IgG reduction in Phase 2 Sjögren's disease study

Adults with moderately-to-severely active Sjögren's disease who received investigational FcRn blocker nipocalimab had improvements in disease activity scores at 24 weeks with accompanying significant reductions in IgG and autoantibody levels Nipocalimab was granted U.S. FDA Breakthrough Therapy Designation for the treatment of adults living with moderate-to-severe Sjögren's disease based on results from the Phase 2 DAHLIAS study WASHINGTON, Nov. 14, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced results from additional analyses of the Phase 2 DAHLIAS study highlighting improvement in key measures of disease activity and significant IgG reduction by over 77% following treatment with investigational nipocalimab in adult patients with moderate-to-severe Sjögren's disease (SjD). These data were included in a plenary session presentation (Abstract #2527) and two posters (Abstracts #1427 and #2294) and are among the Company's 43 oral and poster presentations at the American College of Rheumatology (ACR) Convergence 2024. Patients receiving nipocalimab, an investigational FcRn blocker, showed a significant improvement in the ClinESSDAIa score at 24 weeks, achieving the primary endpoint. Additionally, key secondary endpoints were met, indicating reduced disease activity both systemically and across multiple organ systems, as well as improvements in physician assessments and composite SjD assessment tools. Results also showed a significant reduction in IgG including autoantibody levels among patients receiving 15 mg/kg every two weeks, providing further evidence of nipocalimab's mechanism of action through interaction with the FcRn. Moreover, improvements in ClinESSDAI were generally greatest in the participants with the highest baseline levels of anti-Ro and anti-La autoantibodies, associated with substantial nipocalimab-induced reductions in IgG and total IgG autoantibodies. "These data highlight the relevance of autoantibodies in SjD pathogenesis. The observed reduction in IgG and pivotal autoantibodies, particularly the anti-Ro antibodies, in association with improvement in systemic disease activity and saliva production, represent an exciting advance in our understanding of the disease and how it may be treated effectively. I am also encouraged by the observed trend in many patient-reported measures as they are most important to patients. I look forward to future research to confirm these observations," said Ghaith Noaiseh, M.D., Associate Professor, Allergy, Clinical Immunology, and Rheumatology, The University of Kansas Medical Center.b "People living with SjD need targeted treatment options that can help address the underlying causes and alleviate the potentially serious health consequences of the disease." Many people living with SjD experience symptoms that interfere with daily activities and quality of life, including chronic and severe mucosal dryness.1,2 Extraglandular manifestations – more systemic symptoms of SjD – are also common and may impact multiple organ systems, including joints, lungs, kidneys, and nervous system.3 These patients with high activity in more than one organ or disease area have an increased mortality risk of up to five-fold.4 In the Phase 2 study, patients reported a decrease in symptoms, with numerical improvements compared with placebo in the symptom categories most important to them, including mouth dryness, eye dryness, vaginal dryness, fatigue and joint pain. Additionally, an improvement in objective salivary flow (i.e., at least 50% increase from baseline) was observed in more than twice as many patients in the high dose nipocalimab group (15 mg/kg) compared to the placebo group (32.7% vs. 16%) at Week 24. "No advanced therapies have been approved for SjD to date. A clear need exists for new immunoselective treatments with demonstrated safety profiles that can provide sustained relief from the heavy burden of the overall disease for patients living with SjD," said Federico Zazzetti, Director, Rheumatology, Global Medical Affairs Lead, Johnson & Johnson Innovative Medicine. "Johnson & Johnson is committed to continued research to help address this unmet need, and the data presented at ACR demonstrate the potential of nipocalimab in a disease where patients have very few options." Editor's Notes: ABOUT SJÖGREN'S DISEASE Sjögren's disease (SjD) is one of the most prevalent autoantibody-driven diseases for which no therapies are currently approved that treat the underlying and systemic nature of the disease.4 It is a chronic autoimmune disease that is estimated to impact approximately four million people worldwide and is nine times more common in women than men.5,6 SjD is characterized by autoantibody production, chronic inflammation, and lymphocytic infiltration of exocrine glands. Most patients are affected by mucosal dryness (eyes, mouth, vagina), joint pain and fatigue.4 More than 50% of SjD patients have a moderate to severe form of the condition, and disease burden can be as high as that of rheumatoid arthritis or systemic lupus erythematosus and is often associated with impaired quality of life and functional capacity, and increased mortality risk.3,5,7 ABOUT DAHLIAS DAHLIAS (NCT04969812) is a Phase 2 multicenter, randomized, placebo-controlled double-blind study to evaluate the effects of nipocalimab in participants with primary SjD. DAHLIAS is a Phase 2 dose-ranging study of adults with moderately-to-severely active primary SjD who were seropositive for anti-Ro60 and/or anti-Ro52 IgG antibodies. 163 adults aged 18-75 were randomized 1:1:1 to receive intravenous nipocalimab at 5 or 15 mg/kg or placebo every two weeks through Week 22 and received protocol-permitted background standard of care. Safety assessments were conducted through Week 30. The primary endpoint was change in baseline in the ClinESSDAI Score at Week 24. Select secondary endpoints included: Multiple organ system assessments: European League Against Rheumatism Sjögren's Syndrome Disease Activity Index (ESSDAI) is a systemic diseases activity index designed to measure disease activity in patients with primary SjD. It is based on 12 domains including: constitutional, lymphadenopathy, glandular, articular, cutaneous, respiratory, renal, muscular, peripheral nervous system, central nervous system, biological, and hematological. Disease Activity Level (DAL) response is a reduction from baseline in disease activity level by at least one level in at least one ClinESSDAI domain (e.g., articular, hematological, cutaneous, constitutional). Physician assessment: The Physician Global Assessment of Disease Severity (PhGA) is recorded by the investigator, independent of study participants' assessment, on a scale with responses ranging from 0 ("No SjD activity") to 100 ("Extremely active SjD"). Composite tools for clinical trial endpoints: Sjögren's Tool for Assessing Response (STAR) is a composite responder index that includes all main SjD features, including systemic disease activity, patient-reported symptoms, tear gland item, salivary gland item and serology, in a single tool. Composite of Relevant Endpoints for Sjogren's Syndrome (CRESS), a composite endpoint tool consisting of five complementary items: systemic disease activity, patient-reported symptoms, tear gland item, salivary gland item and serology, for use in trials of primary SjD. Patient reported outcomes: European League Against Rheumatism Sjögren's Syndrome Patient-Reported Index (ESSPRI) is a patient-reported assessment of the severity of dryness, fatigue, and pain associated with primary SjD, in which patients report their symptom severity over the last two weeks on a numeric rating scale (NRS), ranging from 0 "No symptoms (dryness, fatigue or pain)" to 10 "maximal imaginable (dryness, fatigue, pain)". Sjögren's Symptoms tool is a patient-reported assessment of the worst severity of their ocular, oral, and vaginal dryness and joint pain over the past 7 days on a 0 to 10 NRS, from 0 "No [specific symptom]" to 10, "Severe [specific symptom]". ABOUT NIPOCALIMAB Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Prevalent Rheumatology.8,9,10,11,12,13,14,15,16 Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.17,18 The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:   U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021 and fetal neonatal alloimmune thrombocytopenia (FNAIT) in March 2024 U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023 U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for SjD in November 2024 EU EMA Orphan medicinal product designation for HDFN in October 2019 ABOUT JOHNSON & JOHNSON At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at or at . Follow us at @JanssenUS and @JNJInnovMed. Janssen Research & Development, LLC, Janssen Biotech, Inc. and Janssen Global Services, LLC are Johnson & Johnson companies. Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of nipocalimab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments. 1 Sjogren's Disease Foundation. Understanding Sjogrens – Treatment. Available at: . Last accessed: November 2024. 2 McCoy SS, Woodham M, Bunya VY, Saldanha IJ, Akpek EK, Makara MA, Baer AN. A comprehensive overview of living with Sjögren's: results of a National Sjögren's Foundation survey. Clin Rheumatol. 2022 Jul;41(7):2071-2078. doi: 10.1007/s10067-022-06119-w. Epub 2022 Mar 8. PMID: 35257256; PMCID: PMC9610846. 3 Carsons SE, Patel BC. Sjogren Syndrome. [Updated 2023 Jul 31]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: 4 Huang H, Xie W, Geng Y, Fan Y, Zhang Z. Mortality in patients with primary Sjögren's syndrome: a systematic review and meta-analysis. Rheumatology (Oxford). 2021 Sep 1;60(9):4029-4038. doi: 10.1093/rheumatology/keab364. PMID: 33878179. 5 Nat Rev Rheumatol 20, 158–169 (2024). 6 Beydon, M., McCoy, S., Nguyen, Y. et al. Epidemiology of Sjögren syndrome. 7 Hackett KL, et al. Arthritis Care Res (Hoboken). 2012;64(11):1760-1764. 8 ClinicalTrials.gov Identifier: NCT04968912. Available at: . Last accessed: November 2024. 9 ClinicalTrials.gov Identifier: NCT04951622. Available at: . Last accessed: November 2024. 10 ClinicalTrials.gov Identifier: NCT05327114. Available at: . Last accessed: November 2024. 11 ClinicalTrials.gov Identifier: NCT04119050. Available at: . Last accessed: November 2024. 12 ClinicalTrials.gov Identifier: NCT05379634. Available at: . Last accessed: November 2024. 13 ClinicalTrials.gov Identifier: NCT05912517. Available at: . Last accessed: November 2024. 14 ClinicalTrials.gov Identifier: NCT06028438. Available at: . Last accessed: November 2024. 15 ClinicalTrials.gov Identifier: NCT04882878. Available at: . Last accessed: November 2024. 16 ClinicalTrials.gov. NCT03842189. Available at: . Last accessed: November 2024. 17 Lobato G, Soncini CS. Relationship between obstetric history and Rh(D) alloimmunization severity. Arch Gynecol Obstet. 2008 Mar;277(3):245-8. DOI: 10.1007/s00404-007-0446-x. Last accessed: November 2024. 18 Roy S, Nanovskaya T, Patrikeeva S, et al. M281, an anti-FcRn antibody, inhibits IgG transfer in a human ex vivo placental perfusion model. Am J Obstet Gynecol. 2019;220(5):498 e491-498 e499. SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期快速通道孤儿药临床结果突破性疗法

100 项与尼卡利单抗相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11666	-	-	DB16257

研发状态

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
重症肌无力	申请上市	美国	强生（中国）投资有限公司	2024-08-29
胎儿/新生儿同种免疫性血小板减少症	临床3期	比利时	Janssen Research & Development LLC	2024-10-01
胎儿/新生儿同种免疫性血小板减少症	临床3期	巴西	Janssen Research & Development LLC	2024-10-01
胎儿/新生儿同种免疫性血小板减少症	临床3期	法国	Janssen Research & Development LLC	2024-10-01
胎儿/新生儿同种免疫性血小板减少症	临床3期	意大利	Janssen Research & Development LLC	2024-10-01
胎儿/新生儿同种免疫性血小板减少症	临床3期	挪威	Janssen Research & Development LLC	2024-10-01
胎儿/新生儿同种免疫性血小板减少症	临床3期	斯洛伐克	Janssen Research & Development LLC	2024-10-01
胎儿/新生儿同种免疫性血小板减少症	临床3期	西班牙	Janssen Research & Development LLC	2024-10-01
胎儿/新生儿同种免疫性血小板减少症	临床3期	瑞典	Janssen Research & Development LLC	2024-10-01
新生儿同种免疫性血小板减少症	临床3期	比利时	Janssen Research & Development LLC	2024-10-01

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


NCT04968912 (DAHLIAS, ACR2024) 人工标引	临床2期	干燥综合征	163	Placebo	顧觸膚顧觸夢積積顧齋(憲網鬱構獵網襯艱夢獵) = 窪糧範鑰簾獵範鑰鏇鹹簾憲遞夢淵繭築鹹觸製 (築鹽構憲選築齋艱簾膚, -4.74 ~ -2.75) 达到更多	积极	2024-11-18
				nipocalimab 5 mg/kg	顧觸膚顧觸夢積積顧齋(憲網鬱構獵網襯艱夢獵) = 蓋繭憲鑰鹽積壓艱觸憲簾憲遞夢淵繭築鹹觸製 (築鹽構憲選築齋艱簾膚, -5.10 ~ -3.07) 达到更多
NCT04968912 (DAHLIAS, ACR2024) 人工标引	临床2期	干燥综合征	163	nipocalimab 5 mg/kg	淵廠簾淵遞觸窪糧淵鑰(範製齋醖壓醖廠鹽壓網) = 齋鹹鹽鹹夢衊顧糧夢糧蓋範選顧選觸壓積構蓋 (艱鏇鏇鏇鬱獵艱艱鬱積 )	积极	2024-11-18
				nipocalimab 15 mg/kg	淵廠簾淵遞觸窪糧淵鑰(範製齋醖壓醖廠鹽壓網) = 獵齋構築鏇憲廠鑰鏇繭蓋範選顧選觸壓積構蓋 (艱鏇鏇鏇鬱獵艱艱鬱積, -66， ~ 46)
NCT04968912 (DAHLIAS, ACR2024) 人工标引	临床2期	干燥综合征	163	Nipocalimab 5 mg/kg	構齋鏇遞顧糧網鹽鏇獵(鏇壓糧願觸觸繭願夢壓) = 網齋繭襯糧鑰築齋鹽壓艱積鏇鬱製憲衊衊廠願 (淵膚衊選遞襯網獵憲艱 ) 更多	积极	2024-11-17
				Nipocalimab 15 mg/kg	構齋鏇遞顧糧網鹽鏇獵(鏇壓糧願觸觸繭願夢壓) = 淵觸鹹選簾鑰網鬱築選艱積鏇鬱製憲衊衊廠願 (淵膚衊選遞襯網獵憲艱 ) 更多
NCT05265273 (Vibrance-MG, Corporate Publications) 人工标引	临床2/3期	重症肌无力	7	Nipocalimab plus standard of care (SOC)	膚鏇遞鹹積餘衊壓憲遞(築廠餘積憲蓋鬱艱簾衊) = 鬱憲獵選餘鹹壓鬱壓壓繭糧構蓋積夢襯遞壓鹽 (顧廠淵蓋選網鹹構廠鬱 ) 达到更多	积极	2024-10-15
NCT03842189 (UNITY, Pubmed \| N Engl J Med)	临床2期	胎儿成红细胞增多症 maternal antierythrocyte IgG alloantibodies	13	Nipocalimab 30 mg/kg	醖餘醖醖夢鑰糧壓顧齋(鹽齋蓋鹽淵築糧窪鹹餘) = consistent with HDFN, pregnancy, or prematurity 築餘鬱衊鬱艱夢範鬱鬱 (艱鑰襯獵餘鏇鬱餘網獵 )	积极	2024-08-08
NCT04951622 (Vivacity-MG3, GlobeNewswire) 人工标引	临床3期	重症肌无力 anti-AChR+ \| anti-MuSK+ \| anti-LRP4+	153	nipocalimab plus SOC	網淵鬱築鏇築齋膚餘網(餘艱鏇獵齋簾夢糧鹽夢) = 壓廠餘範夢鏇顧膚壓鑰範網廠窪襯網襯顧廠醖 (憲顧壓夢繭範餘範簾夢 ) 达到更多	积极	2024-06-28
				Placebo plus SOC	網淵鬱築鏇築齋膚餘網(餘艱鏇獵齋簾夢糧鹽夢) = 夢範願網艱齋膚積醖衊範網廠窪襯網襯顧廠醖 (憲顧壓夢繭範餘範簾夢 ) 达到更多
NCT04968912 (DAHLIAS, EULAR2024) 人工标引	临床2期	干燥综合征	163	NIPOCALIMAB 5 mg/kg	淵艱願積憲積餘憲繭觸(繭鬱構願夢遞壓觸膚鬱) = 繭觸廠鬱鏇觸淵蓋糧襯網網醖壓築鑰構願簾鏇 (觸觸衊醖壓齋範積選鹹, -5.10 ~ -3.07) 更多	积极	2024-06-12
				NIPOCALIMAB 15 mg/kg	淵艱願積憲積餘憲繭觸(繭鬱構願夢遞壓觸膚鬱) = 範廠選觸願夢夢夢網範網網醖壓築鑰構願簾鏇 (觸觸衊醖壓齋範積選鹹, -7.43 ~ -5.36) 更多
NCT04968912 (DAHLIAS , Corporate Publications) 人工标引	临床2期	干燥综合征	-	Nipocalimab	製繭糧構範觸鬱範醖遞(蓋積築糧糧積淵鬱願積) = with a statistically significant reduction in clinESSDAIb score from baseline at week 24 compared with placebo (PBO) 簾願網鑰憲繭艱餘衊蓋 (淵艱廠鬱襯願網遞鹽獵 ) 达到	积极	2024-02-05
				placebo
NCT04951622 (VIVACITY, Corporate Publications) 人工标引	临床3期	重症肌无力	-	Nipocalimab	餘衊衊願齋積鑰夢顧齋(鏇壓構齋襯鏇齋簾鬱觸) = achieving statistically significant reduction in MG-ADLa score from baseline over weeks 22 to 24 compared with placebo (PBO) 鬱積蓋衊餘願鏇夢獵窪 (網鏇鹹選鏇鏇夢餘範夢 ) 达到	积极	2024-02-05
				placebo
EUCTR2018-002247-28-BE \| NCT03772587 (###DELETE, Pubmed \| Neurology)	临床2期	重症肌无力 antiacetylcholine receptor autoantibodies \| antimuscle-specific tyrosine kinase autoantibodies	-	Nipocalimab 5 mg/kg Q4W	選獵糧構積築範願齋簾(窪夢醖憲憲鏇蓋範窪獵) = A statistically significant dose response was observed for change from baseline in MG-ADL at day 57 鑰鏇壓築製鑰遞網艱壓 (鹹艱蓋獵築構窪繭壓鬱 )	积极	2024-01-23
				Nipocalimab 30 mg/kg Q4W