Efficacy and Safety of Teplizumab in the Treatment of Japanese Pediatric and Adult Participants Aged 8 to 34 Years With Stage 2 Type 1 Diabetes: A Multicenter, Randomized, Open-label, Controlled Study.

This is a parallel, Phase 2, two-arm study to assess the efficacy and safety of 14-days intravenous (IV) infusion of teplizumab treatment.
Teplizumab has been approved by FDA to delay the onset of Stage 3 Type 1 Diabetes (T1D) in adults and pediatric patients aged 8 years and older with Stage 2 T1D. The dose regimen of teplizumab in this study is consistent with the regimen approved by US FDA.
Given prior clinical studies conducted in Western countries, this design is appropriate to assess the efficacy, safety and tolerability, pharmacokinetic, pharmacodynamic, and immunogenicity of a 14-day IV infusion regimen of teplizumab in Japanese Stage 2 T1D participants aged 8 to 34 years.

开始日期2025-07-25

申办/合作机构

Sanofi

NCT07088068

/ Not yet recruiting临床3期

A Randomized, Double-blind, 2-arm, Phase 3 Study to Investigate Efficacy and Safety of Teplizumab Compared With Placebo in Participants 1 to 25 Years of Age With Newly Diagnosed Stage 3 Type 1 Diabetes (T1D)

This is a multicenter, randomized, double-blind, parallel, placebo-controlled Phase 3, 2-arm study for treatment.
The purpose of this study is to measure change in glycemic control and prandial insulin independency over 52 weeks with teplizumab compared with placebo, both administered by intravenous (IV) infusion, in participants with recently diagnosed Stage 3 type 1 diabetes (T1D) aged 1 to 25 years, on standard insulin therapy.

开始日期2025-07-21

申办/合作机构

Sanofi

NCT06892002

/ RecruitingN/A

A Real-World Observational Study Characterizing Patients With Type 1 Diabetes Treated With Teplizumab

Type 1 diabetes mellitus (T1D) is a chronic autoimmune disease caused by the destruction of pancreatic β cells. T1D pathogenesis progresses through several stages: Stage 1 T1D includes the presence of β cell autoimmunity and thus presence of islet autoantibodies, without the presence of dysglycemia and symptoms. Stage 2 T1D includes the presence of islet autoantibodies and dysglycemia, also with no symptoms. Stage 3 T1D includes presence of islet autoantibodies, overt hyperglycemia, and symptoms; most patients with Stage 3 T1D meet standard diagnostic criteria for diabetes and require insulin treatment.
Teplizumab has been shown to delay progression to Stage 3 in participants at Stage 2 in a Phase 2 clinical trial, leading to subsequent approval in the United States of America (USA). Patients outside of the USA are able to receive the treatment through Pre-Registration Import Licenses and Managed Access Programs. The current study will collect data on the use of teplizumab in routine care, to better understand which patients received teplizumab and how these patients were managed after they received the treatment.

开始日期2025-02-11

申办/合作机构

Sanofi

100 项与 Teplizumab 相关的临床结果

登录后查看更多信息

100 项与 Teplizumab 相关的转化医学

登录后查看更多信息

100 项与 Teplizumab 相关的专利（医药）

登录后查看更多信息

612

项与 Teplizumab 相关的文献（医药）

2025-10-01·JOURNAL OF DIABETES AND ITS COMPLICATIONS

What type 1 diabetes endotype is most suitable for anti-CD3 antibodies prevention trials?

Review

作者: Paolo, Pozzilli ; Maria Aurora, Roma-Wilson

Type 1 diabetes (T1D) is a heterogeneous autoimmune disease with multiple endotypes, each demonstrating distinct clinical and immunological characteristics. Teplizumab, an anti-CD3 monoclonal antibody, has emerged as a promising immunomodulatory therapy capable of delaying the progression of T1D in individuals with stage 2 disease. However, variability in therapeutic response suggests that certain endotypes may derive greater benefit from treatment. This review evaluates the suitability of different T1D endotypes (T1DE) for teplizumab prevention trials, with a particular focus on early-onset T1DE1 and T1DE2. Clinical trials demonstrate that individuals under 15 years of age, who demonstrate the highest immune activity, marked by aggressive T-cell infiltration and rapid pancreatic β-cell destruction, experience the most significant delay in disease progression following teplizumab treatment, highlighting the importance of early intervention. Furthermore, shifting individuals from the rapidly progressing T1DE1 trajectory to the more gradual T1DE2 course may extend functional insulin production and improve long-term metabolic outcomes. This paper underscores the need for expanded endotype-specific prevention trials and optimised screening protocols to identify high-risk individuals at the earliest stage. Future research should explore teplizumab's efficacy in younger populations and refine predictive biomarkers to enhance personalised intervention strategies in T1D management.

2025-08-01·DIABETES OBESITY & METABOLISM

Teplizumab: Not all that glitters is gold

Letter

作者: Scaramuzza, Andrea E. ; Bonfanti, Riccardo ; Rabbone, Ivana

2025-08-01·Journal of Cachexia Sarcopenia and Muscle

Thymic Microenvironment Remodeling in Cancer Cachexia as a Determinant of Checkpoint Inhibitor Efficacy and Toxicity.

Article

作者: Peng, Lin-Jiao ; Yang, Qin-Tai ; Li, Xing ; Wu, Xiang-Yuan ; Shi, Zhao-Hui ; Pang, Xiu-Qing ; Huang, Run-Kai ; Xing, Yan-Fang ; Wei, Li ; Lv, Xiao-Tong

BACKGROUND:

The discovery of immune checkpoints links autoimmunity and cancer, with thymus atrophy reportedly causing autoimmune multiorgan inflammation. The impact of cancer cachexia on thymic involution and its clinical significance remains unclear. This study aimed to investigate this effect and its association with immune checkpoint inhibitor (ICI) treatment.

METHODS:

Single-cell sequencing, immunofluorescence and flow cytometry analyses were conducted to explore changes in the thymus in orthotopic hepatocellular cancer (HCC) mice with cachexia. Patients with advanced and locally advanced cancers receiving anti-PD-1/L1 antibody treatment were followed up to investigate the relationship between the amount of serum autoantibodies and the efficacy of ICIs.

RESULTS:

Single-cell sequencing in cachexic HCC mice revealed thymic fibroblast maturity disorders characterized by elevated immature medullary fibroblasts, impaired antigen processing functions, reduced interaction with single-positive thymocytes and decreased expression of tissue-restricted antigen-related genes. The thymus of mice with cancer cachexia exhibited degradation of the thymic medulla and decreased expression of LtβR, Mmp9 and Ccl19 in thymus medullary fibroblasts (mFbs). Single-cell TCR sequencing showed that inflammatory-related V/J TCR genes were highly used in expanded thymocyte clonotypes in cachexic HCC mice, suggesting impaired T cell negative selection. Results from coculture and cell transfer assays suggest that cancer cachexic CD45⁺ erythroid progenitor cells (EPCs) induce the death of CD34⁺ progenitor cells and decrease the number of LtβR⁺, Mmp9⁺ and Ccl19⁺ mFbs in tumour-free mice. CD24⁺CD4⁺CD8^- single-positive thymocytes, typically eliminated in negative selection, did not decrease after the administration of anti-CD3 mAb. Serum autoantibodies were markedly produced in cachexic HCC mice, cachexic HCC mice administered with anti-PD1 and tumour-free mice that received cancer cachexic CD45⁺ EPCs. Autoantibodies against tumour-restricted antigens were found in patients with advanced and locally advanced cancer who received two cycles of ICI treatment. Univariate Cox regression analysis showed that patients with a low level of autoantibodies had a higher risk of disease progression (hazard ratio [HR]: 2.39, 95% CI [1.02-5.63], p = 0.046). Analysis of the receiver operating characteristic curve indicated that the number of autoantibodies against tumour tissues predicted treatment failure (area under the curve [AUC] 0.726, p = 0.021) and long-term duration of treatment response (AUC 0.697, p = 0.024). Patients with high levels of serum autoantibodies against tumours had favourable progression-free survival (HR, 0.389; 95% CI [0.158-0.960], p = 0.04).

CONCLUSIONS:

Cancer cachexia disrupts mFbs maturity, affecting T cell negative selection and expanding the TCR repertoire against tissue-restricted antigens. This might mediate the adverse and favourable effects of ICIs as anticancer treatments.

299

项与 Teplizumab 相关的新闻（医药）

2025-07-08

·国际糖尿病idiabetes

1型糖尿病迈入新时代：从ADA 2025看早期筛查、精准诊断与干预进展

点击“蓝字”关注我们以下内容仅供医疗卫生专业人士阅读参考编者按：第85届美国糖尿病协会科学会议（ADA）在1型糖尿病（T1D）领域掀起了理念上的重大革新。大会强调，从传统的“被动诊断”到如今的“主动筛查”，防控模式发生了深刻转变，推动了早期诊断、精准干预策略的全面实施。本期特邀中南大学湘雅二医院周智广教授，全面梳理大会中T1D领域的关键研究进展，为您深入解析发展趋势与临床应用前景。T1D防控破局从“被动诊断”到“主动筛查”无论是2025版《ADA糖尿病诊疗标准》[1]还是《中国1型糖尿病高危人群筛查、监测与管理专家共识（2024版）》[2]均强调T1D早期筛查的重要性。然而，T1D的早期筛查仍面临许多挑战，包括缺乏足够的公众认知、筛查工具的完善以及高危人群筛查覆盖不全面等。在此背景下，本届ADA大会在T1D领域发布了一系列创新性的进展，尤其是将“主动筛查”作为核心策略，以期推动早期筛查的普及与实施。在本次大会上，一项针对性文献综述（TLR）（摘要号：1509-P）[3]指出，相关自身免疫性疾病（AADs）与T1D之间存在双向关联，且AADs可能增加T1D的风险。研究提示，在AAD患者中早期检测T1D风险并及时干预，可能有助于延缓疾病进展。在筛查方法方面，一项ADA主席精选摘要（摘要号：71-OR）[4]，聚焦遗传学特征对T1D风险的预测。研究表明，基于不同种族的SNPs和HLA等位基因构建的遗传风险评分（meta-GRS）在各人群中表现良好且相似。将非HLA遗传风险变异（种群特异性或元分析）纳入其中，预计能进一步提升T1D风险的遗传预测能力。多项研究展示了机器学习模型在早期预测T1D中的潜力。研究（摘要号：2058-LB）[5]和（摘要号：1119-P）[6]表明，机器学习模型能够比现有方法提前12-24个月以更高的识别率检测到无症状患者。基于机器学习的早期识别使得及时干预成为可能，减少了糖尿病酮症酸中毒（DKA）的发生，提升了患者的生活质量，并减轻了治疗领域的压力。此外，一项回顾性队列分析（2051-LB）[7]表明，通过理赔记录可识别早期T1D个体，且通过分析人口统计信息、频繁的消化系统诊断和常规内分泌检查，可以提示T1D的风险。另一项针对哥伦布市（乔治亚州）项目的研究调查（摘要号：2074-LB）[8]发现，要实现T1D筛查的广泛应用，必须付出系统性努力，尤其是识别并解决筛查过程中存在的障碍，如缺乏意识和可获得性问题。打破误诊“魔咒”精准诊断T1D，拯救“错失的黄金治疗期”根据抗体筛查和糖代谢监测结果，T1D的诊断可分为3期：1期为免疫紊乱期，2期为血糖异常期，3期为临床症状期。对无症状1、2期的早期识别和精准诊断对于防止病情进展为临床T1D至关重要[2]。本届ADA大会发布了多项重要研究成果，强调了早期诊断的重要性，重点讨论了避免漏诊、误诊的关键策略，并提出了优化早期诊断方法的建议。一项来自意大利的糖尿病管理分析（AIDA）研究（摘要号：2118-LB）[9]指出，T1D在意大利儿童人群中造成了沉重的健康和社会经济负担，随着发病率和患病率的增加，以及相关共病和直接医疗费用的增长，T1D对社会经济产生了巨大的影响。研究强调了早期诊断的重要性以及合理配置专门医疗资源的迫切需求。目前临床中T1D的漏诊和误诊问题依然严重。根据大会发布的两项基于美国管理医疗人群的研究（摘要号：1080-P、1327-P）[10-11]，约40%的T1D成年患者曾被误诊为2型糖尿病（T2D）。研究发现，具有T2D既往诊断的成年人，T1D诊断的平均延迟时间为3.6年，且未进行抗体检测的患者诊断延迟更长；较高的BMI可能是导致误诊为T2D的原因之一。这些发现突显了改进诊断方法的紧迫性，确保T1D患者能得到及时、准确的管理。不仅成人患者，青少年群体也面临类似的误诊风险。根据本次大会的另一项研究（摘要号：1195-P）[12]，四分之一的青少年患者被误诊为其他类型的糖尿病，其中超过1/6被误诊为T2D。T1D的延迟诊断与并发症密切相关，糖尿病的错误分类可能延误治疗并影响护理效果。该研究强调了及时、准确诊断T1D的重要性，尤其是通过检测自身抗体来明确诊断，以减少误诊风险、避免治疗延误，从而改善患者预后。此外，持续葡萄糖监测（CGM）作为一种新兴的诊断工具，在识别2期T1D方面显示出潜力。相关研究（摘要号：2106-LB）[13]表明，CGM可用于识别2期T1D患者，结合静脉血糖检测结果，为免疫药物应用提供依据。随着技术的进步，未来CGM可能不再仅作为辅助手段，而是有望取代血浆葡萄糖检测和HbA1c检测，成为识别2期T1D患者并指导免疫疗法的重要手段。替利珠单抗延缓发病，T1D防治的未来已来T1D的早期发病显著增加了致死率和并发症风险，而早期免疫干预则被视为阻止T1D发生和发展的“制胜法宝”。随着治疗方法的不断进步，早期干预已不再是梦想，而是成为了可能。在这一背景下，本届ADA大会进一步推动了精准干预理念，特别是在2期T1D患者的管理中，强调了疾病修饰疗法（即免疫病因治疗）在延缓疾病进程中的关键作用。本次ADA主席精选的另一项研究（摘要号：145-OR）[14]探讨了T1D诊断年龄与致命性心血管和肾脏事件的关系。结果发现，T1D的早期发病显著增加了严重并发症和死亡的风险。这一结果支持了延迟青少年T1D发病可能减少未来并发症风险的观点。换言之，对亚临床期T1D，特别是2期患者，进行早期干预，具有至关重要的意义。替利珠单抗（Teplizumab）是首个且目前唯一被美国食品药品监督管理局（FDA）批准用于延缓T1D发病的疾病修饰疗法。该药已于2024年6月进入我国，获批先行先试临床急需进口药品，落地海南博鳌乐城先行区，并于近日在博鳌乐城先行区开出了亚洲首批处方[15]。其适用于延缓8岁（含）以上儿童和成人T1D 2期患者向临床3期进展。TrialNet-10研究（TN-10）是替利珠单抗获批的关键循证之一。本次大会再次公布了该研究的扩展研究结果（摘要号：840-P）[16]。该扩展研究是一项多中心、单臂、开放标签的研究，旨在评估参与过TN-10试验接受治疗或安慰剂的2期T1D患者，在发展为3期T1D后接受替利珠单抗治疗的效果。共6名参与者接受了替利珠单抗治疗，平均随访时间为1.4（0.3）年。结果显示，在随访期间，没有参与者出现低血糖、DKA或特殊关注不良事件（AESI）。研究表明，替利珠单抗在3期T1D患者中使用时是安全且耐受良好的。接受过1次替利珠单抗治疗的2期T1D患者，在进入3期T1D后再次接受治疗时未出现新的安全信号。另一项药物警戒研究（摘要号：835-P）[17]，纳入了自2023年以来报告的200例替利珠单抗治疗病例，表明替利珠单抗的药物警戒数据总体呈现出较为良好的安全性。大多数报告的副作用包括皮疹和恶心，这与其作为单克隆抗体的作用机制以及输注相关反应一致。此外，大会还公布了一项关于T1D患儿照护者对抗体筛查和替利珠单抗使用认知与看法的研究（摘要号：711-P）[18]。结果显示，大多数照护者愿意为高风险儿童使用替利珠单抗。该研究强调了提高公众意识和确保及时获取该治疗的重要性。总结在2025年ADA大会上，T1D领域迎来了一系列引人注目的研究成果，标志着防控模式的深刻转变。从过去的“被动诊断、对症治疗”到如今的“主动筛查、早期诊断、精准干预”的实施，为T1D防控开辟了新篇章。大会强调了对T1D高危人群的早期筛查与干预的重要性，尤其是在2期T1D患者的治疗中，替利珠单抗（Teplizumab）作为首个被FDA批准用于延缓T1D发病的疾病修饰疗法，其在延缓3期T1D发病方面展现出了突破性意义。大会公布的多项研究数据进一步巩固了该药物在T1D早期干预中的可行性和长远价值。随着筛查技术、诊断方法和干预策略的逐步成熟，T1D的防控正迈向更加精准、个性化的未来。参考文献：（上下滑动查看更多）[1] American Diabetes Association Professional Practice Committee. 2. Diagnosis and Classification of Diabetes: Standards of Care in Diabetes-2025. Diabetes Care. 2025 Jan 1;48(1 Suppl 1): S27-S49. doi: 10.2337/dc25-S002. PMID: 39651986; PMCID: PMC11635041.[2] 中华医学会糖尿病学分会,中华医学会儿科学分会,中华医学会内分泌学分会,等.中国1型糖尿病高危人群筛查,监测与管理专家共识(2024版)[J].中华糖尿病杂志, 2024, 16(11):1183-1199.DOI:10.3760/cma.j.cn115791-20241001-00592.[3] LAURA WILSON, MARIAM HANNA, NISHU GAIND, et al. Association between Autoimmune Diseases and Development of Autoimmune Type 1 Diabetes (T1D)—A Targeted Literature Review (TLR). 2025 ADA：1509-P.[4] DOMINIKA A. MICHALEK, WEI-MIN CHEN, SUNA ONENGUT-GUMUSCU, et al. HLA-Focused Meta-Genetic Risk Score (GRS) Performance in Populations of Diverse Ancestry with Type 1 Diabetes. 2025 ADA：71-OR.[5] FELIX LAM, VINAYAK TIWARI, GIULIANO MION, et al. Identification of Earlier Stage Autoimmune Type 1 Diabetes Using Machine Learning Algorithms. 2025 ADA：2058-LB.[6] DAVID HOOD, KETAN WALIA, SUKRITI PODDAR, et al. Early T1D Prediction with ML—Enhancing Screening and Reducing DKA Risk. 2025 ADA：1119-P. [7] DANIEL EINOR, OLEKSANDR BUIKO, KENI C.S. LEE, et al. Predictive Modeling for Presymptomatic Type 1 Diabetes Detection Using Open Claims Data. 2025 ADA：2051-LB.[8] STEVEN B. LEICHTER. The Columbus, GA Project—A Model for Population Screening for Type 1 Diabetes in a Metropolitan Area. 2025 ADA：2074-LB.[9] RAFFAELLA BUZZETTI, RICCARDO CANDIDO, VALENTINO CHERUBINI, et al. Administrative Italian Diabetes Analysis (AIDA) on Epidemiology and Economic Burden of Type 1 Diabetes in Pediatric Patients. 2025 ADA：2118-LB. [10] DIANA BRIXNER, TARIKU J. BEYENE, MALVIKA VENKATARAMAN,et al. Diagnostic Delay in People with Type 1 Diabetes and a Prior Type 2 Diabetes Diagnosis—A U.S. Managed Care Population. 2025 ADA：1080-P.[11] DIANA BRIXNER, TARIKU J. BEYENE, MALVIKA VENKATARAMAN, et al. Body Mass Index in People with Type 1 Diabetes with and without Prior Type 2 Diabetes Diagnosis—A U.S. Managed Care Population. 2025 ADA：1327-P. [12] GABRIEL ELLISON, RICARDO MEDINA PE?ARANDA, LAUREN FIGG, et al. Disparities in Type 1 Diabetes Diagnosis—Autoantibodies Are Critical. 2025 ADA：1195-P.[13] ALESSANDRA AYERS, CINDY HO, JULIA K. MADER, et al. The Use of Continuous Glucose Monitoring to Diagnose Stage 2 Type 1 Diabetes. 2025 ADA：2106-LB.[14] ARAZ RAWSHANI, BJORN ELIASSON, DARREN K. MCGUIRE,et al. Association of Age at Type 1 Diabetes Diagnosis with Fatal Cardiovascular and Kidney Events. 2025 ADA：145-OR. [15] 1型糖尿病新药在乐城开出亚洲首批处方，有望延缓疾病进展. https://mp.weixin.qq.com/s/p3BrU1mCokLCGhUmrDYZwA.[16]KEVAN C. HEROLD, JENNIFER L. SHERR, MICHAEL J. HALLER, et al. TN-10 Extension Study—Teplizumab following Stage 3 Type 1 Diabetes Diagnosis in Participants from the TN-10 Trial. 2025 ADA：840-P. [17] ARUNIMA SHARMA, DAMLA N. COSTA, JUAN P. SCARANO, et al. Assessing the Adverse Drug Reactions for TZIELD (Teplizumab) in Type 1 DM Patients—A Pharmacovigilance Study. 2025 ADA：835-P.[18] RACHEL L. LONGENDYKE, JODY GRUNDMAN, PALLAVI DWIVEDI, et al. Assessing Caregivers’ Knowledge and Opinions on T1D Antibody Screening and Teplizumab Infusions. 2025 ADA：711-P.声明：本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。4M编号：MAT-CN-2511859 1.0 2025.7最新《国际糖尿病》读者专属微信交流群建好了，快快加入吧！扫描左边《国际糖尿病》小助手二维码（微信号：guojitnb），回复“国际糖尿病读者”，ta会尽快拉您入群滴！（来源：《国际糖尿病》编辑部）版权声明版权属《国际糖尿病》所有。欢迎个人转发分享。其他任何媒体、网站未经授权，禁止转载。

诊断试剂

2025-06-17

·研发客

100亿欧元在手，赛诺菲要怎么花？

// •接连完成几笔有针对性的收购和BD交易来延续Dupixent的辉煌战绩；•在MNC们纷纷砍掉罕见病管线的当口，逆流而上收购罕见病公司、补充自免罕见病产品；•对原有旧管线进行整理之后，今年又两度出手CNS领域。在宣布95亿美元收购Blueprint消息的同时，赛诺菲CEO Paul Hudson表示，公司仍然保留相当大的进一步收购能力。这句话并非空穴来风。同一天，赛诺菲与Nurix就一款STAT6降解剂达成授权交易，包括1500万美元的独家许可权费用和最高4.65亿美元的开发、商业化等里程碑付款。今年，赛诺菲频繁出手，表面来看，原因非常简单。一个月前，公司将消费者健康业务欧彼乐正式归于D&R麾下，作为其“瘦身”计划的一部分，50%欧彼乐的控股权换到了100亿欧元的净现金收益，也让它在近期的收购和BD交易中表现得更加慷慨。然而，有别于辉瑞的挥金如土、礼来的以守为攻，赛诺菲的“买买买”更接近一种庖丁解牛的姿势，这家曾经靠并购扩张起来的法国公司，精确地知道自己要什么。当然，这不代表它的每一次出手都是正确的。那么，接下来的钱，它会怎么花？延续Dupixent的战绩目前，赛诺菲拥有多条自免核心管线，包括白介素因子、TSLP、TL1A等靶点。其中，OX40单抗amlitelimab一直被视为Dupixent的接班者。不过，针对哮喘的临床研究结果差强人意。据赛诺菲最近公布的TIDE-Asthma Ⅱ期研究的初步结果显示，在中重度哮喘患者中，amlitelimab在缓解病情加重、改善肺功能和减轻症状方面取得了具有临床意义的效果，但是最高剂量组在第48周的年化严重哮喘急性发作率（AAER）未达到预期目标。尽管amlitelimab在治疗特应性皮炎方面已经取得了一些积极成果，但速度上或将落后于安进的rocatinlimab。rocatinlimab是全球首个完成Ⅲ期临床的OX40单抗。还有另一款OX40单抗为创响生物所有。去年，IMG-007在特应性皮炎和斑秃的Ⅱa期临床均取得积极的结果，展现出BIC潜力。同年，创响生物在纳斯达克完成了反向收购。拓展阅读猛虎嗅蔷薇：岸迈创响等多桩交易背后的资本推手为了巩固江湖地位，赛诺菲这两年接连完成了几笔有针对性的收购和BD交易。首先瞄准的是蛋白降解药物。2019年，赛诺菲与Nurix达成合作，支付5500万美元首付款，利用Nurix专有的DEL-AI药物发现平台开发特定的靶向蛋白降解药物。第二年，赛诺菲又为该合作追加2200万美元。今年4月，赛诺菲支付1500万美元，得到Nurix一款之前无法成药的自身免疫性疾病转录因子降解剂的独家授权。这两家公司最近一笔合作，即本文开头提到的本月初达成的STAT6降解剂授权交易。站在TCE的风口上，赛诺菲以6亿美元首付款拿到了Dren Bio旗下一款髓系细胞衔接器双抗DR-0201。这是一款潜在首创靶向CD20双抗，在临床前和早期临床研究中显示出强大的B细胞耗竭作用。深层B细胞耗竭能重置适应性免疫系统，有望在难治性B细胞介导的自身免疫性疾病如狼疮等发挥潜力。目前，DR-0201正在开展干燥综合征、多发性肌炎等适应症Ⅰ期临床试验。去年，赛诺菲又以1.25亿美元的首付款押注TL1A（肿瘤坏死因子样配体1A）赛道，与专注AI驱动药物开发的Earendil Labs达成合作，拿到Integrin α4β7/TL1A双抗HXN-1002与TL1A/IL-23双抗HXN-1003的全球独家权益。此外，赛诺菲在白介素赛道上继续发力，与Synthekine达成合作，开发用于治疗炎症性疾病的IL-10受体激动剂并将其商业化。另以7亿美元收购Belharra Therapeutics，利用其非共价化学蛋白质组学平台筛选及验证赛诺菲免疫学相关的药物靶点。实际上，CEO Paul Hudson上任之后的一系列重组计划，也在为Dupixent之后的接棒者做准备。今年一季度，Dupixent卖出了34.8亿欧元，实现20.3%的同比增长，占2025Q1总收入的1/3。尽管在适应症上还有不小的拓展空间，但是，要成为真正意义上的免疫巨头，只有一款产品是远远不够的。至少，艾伯维和强生在免疫领域的基本功同样扎实。Dupixent的核心专利将在2031年到期，赛诺菲需要更多种子选手。罕见病的路好走吗？2011年斥资200亿美元收购了健赞之后，赛诺菲就有了罕见病的基因。而在MNC们纷纷砍掉罕见病管线的当口，以95亿美元收购Blueprint的决策更有些逆流而上的味道。这笔交易可能成为今年欧洲制药企业最大金额收购案。从Blueprint手中，赛诺菲获得已上市产品Ayvakit，用于治疗系统性肥大细胞增多症（一种罕见免疫性疾病），以及下一代高选择性KIT D816V 抑制剂elenestinib。此外还有一款野生型KIT 抑制剂BLU-808，针对慢性荨麻疹/过敏性鼻炎等自免适应症。赛诺菲的几款已上市罕见病产品中，用于治疗血友病A的Altuviiio表现不俗，2025Q1实现2.51亿欧元的销售额，同比增长100%。CEO Paul Hudson认为，随着产品的继续放量，Altuviiio将有可能成为全年的下一个重磅炸弹。今年，另一款血友病药物Qfitlia获FDA批准，成为首个获批用于预防或减少成人和12岁以上儿童血友病患者出血频率的疗法，并且该疗法已在中国提交上市申请。此外，BTK抑制剂rilzabrutinib上市在即，用于治疗持续性或慢性免疫性血小板减少症（ITP）。这款产品是2020年赛诺菲以37亿美元收购Principia Biopharma而获得。去年，赛诺菲又以22亿美元收购Inhibrx，获得了一款罕见病在研产品INBRX-101，用于治疗α-1抗胰蛋白酶缺乏症（AATD），现已进入临床Ⅱ期。此番收购Blueprint的举动可以看出，赛诺菲在罕见病领域的布局将继续围绕自免适应症。再次进攻CNS2022年开始，赛诺菲着手调整神经系统领域的产品和管线，将治疗中枢神经系统疾病、疼痛和血管疾病的15款产品组合卖给了Neuraxpharma，又将精神治疗、抗焦虑、抗癫痫和抗精神病的11种药物出售给Pharmanovia。不过，这并不表示赛诺菲从CNS领域离场。相反，今年5月，赛诺菲花4.7亿美元收购了纳斯达克上市公司Vigil Neuroscience，旨在进攻阿尔茨海默病。旗下一款口服小分子TREM2激动剂VG-3927，已完成了该适应症的Ⅰ期临床试验，并取得积极结果。预计在今年三季度开展Ⅱ期临床。上周，Vigil另一款TREM2激动剂iluzanebart启动Ⅱ期临床，治疗成人脑白质病伴轴索球样变和色素胶质细胞（ALSP）。此外，今年早些时候，赛诺菲与Alloy达成合作，利用其AntiClastic反义平台开发CNS基因药物，并意图实现跨越血脑屏障。下一笔钱，花在哪里？看似低调的赛诺菲一直在并购交易的路上，搭建自免领域“护城河”应当是其最重要的目标。那么，未来这家公司可能在哪些领域投资呢？从近两年的几笔投资来看，疫苗领域没有涉及。2025Q1，赛诺菲疫苗领域销售额为13.26亿欧元，同比增长11.4%。RSV疫苗Beyfortus是主要功臣。目前，赛诺菲有几款mRNA疫苗在研产品，包括流感、痤疮、衣原体感染等。而CNS素来是最容易摔跟头的领域，想要拔得头筹恐怕还需要投入更多的机会成本。那么，曾经在糖尿病治疗创下辉煌的赛诺菲，会不会重新杀回代谢领域？2023年，赛诺菲以29亿美元收购了Provention Bio，其核心资产Tzield（用于治疗延迟1型糖尿病的发作）。这笔收购似乎并没有带来太好的回报，Tzield在2025Q1卖出1100万欧元，同比增长10%，表现不理想。如果要在短时间内重回巅峰，恐怕还要依靠新的交易来实现。编辑 | 姚嘉yao.jia@PharmaDJ.com总第2473期访问研发客网站，深度报道和每日新闻抢鲜看www.PharmaDJ.com

并购临床2期临床结果临床3期

2025-05-22

·PRNewswire

Laureate Announced for Hamm International Prize for Diabetes Research

Honoree has made outstanding contributions toward understanding the pathogenesis and prevention of Type 1 diabetes OKLAHOMA CITY, May 22, 2025 /PRNewswire/ -- The prestigious Harold Hamm International Prize for Biomedical Research in Diabetes, an honor bestowed by OU Health Harold Hamm Diabetes Center at the University of Oklahoma Health Sciences, will be awarded this fall to Professor Dr. Anette-Gabriele Ziegler, Director of the Institute of Diabetes Research, Helmholtz Munich, German Research Center for Environmental Health. She is also Chair in Diabetes and Gestational Diabetes at Technische Universität München, School of Medicine. She is being honored for her research to understand the pathogenesis of Type 1 diabetes and efforts toward its prediction, early diagnosis and prevention. Continue Reading Professor Dr. Anette-Gabriele Ziegler, director of the Institute of Diabetes Research at the Helmholtz Munich, German Research Center for Environmental Health, has been selected as the 2025 recipient of the prestigious OU Health Herald Hamm Diabetes Center's Harold Hamm International Prize for Biomedical Research in Diabetes. The Hamm Prize recognizes and encourages lasting advances in research related to Type 1 or Type 2 diabetes. It is awarded to an individual who has either demonstrated lifelong contributions to the field or realized a singular advance, especially one that promotes curative potential. The honor includes a $250,000 award, the largest of its kind in the world. It was established to recognize and promote lasting achievements in diabetes research focused on progress toward a cure. "The award is an incredible motivation to look ahead and continue on the path toward understanding the pathogenesis of Type 1 diabetes and developing preventive therapies – with the ultimate goal of finding a cure," Ziegler said. The prize, established in 2012, is named for Harold Hamm, chairman and chief executive officer of Oklahoma-based Continental Resources Inc., who provided an endowment to fund the prize in perpetuity. The Hamm Prize laureate is selected by a rotating jury of national and international leaders in the diabetes research community and is awarded every two years. "This prize honors work that changes the course of science – and Dr. Ziegler's research does exactly that. Her leadership in uncovering how Type 1 diabetes begins and how we might stop it before it starts is the kind of bold, life-changing progress this prize was meant to recognize. Her work fuels our hope – and brings us all one step closer to the cure," Hamm said. Ziegler's research has been at the forefront of translating important research questions into longitudinal studies, including more than 20 clinical trials, as well as implementing findings into public health. Her achievements include: Launched BabyDiab, the first birth cohort to study when and how Type 1 diabetes begins. Launched Fr1da, a public screening program to detect early signs of Type 1 diabetes, now a model for similar global efforts. Initiated and serves as speaker of GPPAD (Global Platform for the Prevention of Autoimmune Diabetes), a European platform to translate knowledge about disease pathogenesis into trials seeking to prevent Type 1 diabetes. More than 2,000 infants have been enrolled in GPPAD's clinical trials and screened for genetic risk of Type 1 diabetes. Conducted research that led to a landmark clinical trial resulting in Food and Drug Administration approval for teplizumab, the first immunotherapy drug shown to delay the onset of Type 1 diabetes in at-risk individuals. In addition, Ziegler has made the following research discoveries: Autoimmunity to insulin is a key starting point for Type 1 diabetes in children, and Type 1 diabetes susceptibility genes predispose for autoimmunity. Islet autoimmunity (when the immune system mistakenly attacks and destroys insulin-producing cells in the pancreas) starts early in life, with a peak incidence around age 1 to 2. Children with two or more islet autoantibodies almost always develop diabetes. Respiratory viral infections in early life, including COVID-19, raise the risk of islet autoimmunity in genetically at-risk children. Early changes in blood sugar and immune activity appear to precede the onset of autoimmunity to insulin. A physician who conducts research, Ziegler said caring for patients has informed her career as a scientist. "My mentor always used to say, 'A researcher is the best doctor,'" she said. "I'm not sure if the reverse is true, but my daily work with people living with diabetes and my experience as a physician have certainly shaped my research. They have especially influenced my strong focus on translational research, clinical trials, and investigating the origins of the disease in humans." Ziegler will receive the Hamm Prize during a gala in October. Jed Friedman, Ph.D., director of Harold Hamm Diabetes Center, said Ziegler's distinguished research career has significantly advanced the aim of preventing Type 1 diabetes. "Professor Dr. Ziegler's visionary leadership and unwavering commitment have not only advanced scientific understanding but also paved the way for novel interventions that improve patient outcomes by delaying the onset of Type 1 diabetes," Friedman said. "Her work exemplifies the integration of rigorous research with compassionate clinical application, embodying the highest ideals of medical science. We are privileged to honor [her] for her unparalleled contributions to diabetes research and her enduring impact on public health." About the project For more information about the Harold Hamm International Prize for Biomedical Research in Diabetes, visit the prize website here. About the University of Oklahoma Founded in 1890, the University of Oklahoma is a public research university with campuses in Norman, Oklahoma City and Tulsa. As the state's flagship university, OU serves the educational, cultural, economic and health care needs of the state, region and nation. In Oklahoma City, OU Health Sciences is one of the nation's few academic health centers with seven health profession colleges located on the same campus. OU Health Sciences serves approximately 4,000 students in more than 70 undergraduate and graduate degree programs spanning Oklahoma City and Tulsa and is the leading research institution in Oklahoma. For more information about OU Health Sciences, visit . SOURCE University of Oklahoma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床研究免疫疗法

100 项与 Teplizumab 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D09013	Teplizumab	-	DB06606

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
1型糖尿病	美国	Provention Bio, Inc.	2022-11-17

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
葡萄糖耐受不良	临床2期	美国	National Institute of Diabetes & Digestive & Kidney Diseases	2010-08-01
葡萄糖耐受不良	临床2期	加拿大	National Institute of Diabetes & Digestive & Kidney Diseases	2010-08-01
葡萄糖耐受不良	临床2期	德国	National Institute of Diabetes & Digestive & Kidney Diseases	2010-08-01
慢性大斑块银屑病	临床2期	美国	MacroGenics, Inc.	2009-12-01
慢性大斑块银屑病	临床2期	美国	Eli Lilly & Co.	2009-12-01
2型糖尿病	临床阶段不明	美国	Sanofi	2024-09-27

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


NCT04270942 (CTgov)	临床2期	1型糖尿病	6	Teplizumab	醖鹹築鏇鹹遞蓋繭鑰膚 = 夢遞蓋製醖襯構艱衊鹹窪壓簾鹽膚製鬱製簾蓋 (蓋壓積衊衊窪衊繭顧鹹, 網製鬱顧襯範蓋艱遞構 ~ 憲鹹窪齋膚鹹願遞積築) 更多	-	2025-02-12
NCT01030861 (Pubmed \| J Clin Invest)	临床2期	1型糖尿病	-	Teplizumab	衊願淵衊齋窪範鑰獵網(壓構簾襯衊餘獵鏇憲獵) = reduced with teplizumab treatment 憲餘窪鑰衊膚範網夢蓋 (獵糧鹹積憲糧遞醖鹹蓋 ) 更多	积极	2024-08-13
NCT03875729 (PROTECT, ADA2024) 人工标引	临床3期	1型糖尿病	275	Teplizumab	餘廠餘淵積構製觸遞繭(襯鑰窪遞蓋襯獵積襯醖) = 繭淵築選築夢齋膚觸蓋願淵鬱鏇淵夢築鏇膚窪 (襯繭糧獵網鹽窪憲襯蓋, -2.27 ~ -1.87) 更多	积极	2024-06-20
				Placebo	餘廠餘淵積構製觸遞繭(襯鑰窪遞蓋襯獵積襯醖) = 夢網積壓獵蓋鏇餘鏇鏇願淵鬱鏇淵夢築鏇膚窪 (襯繭糧獵網鹽窪憲襯蓋, -1.94 ~ -1.67) 更多
ADA2024	N/A	1型糖尿病	-	Teplizumab	窪願積鬱壓鏇淵壓淵齋(構壓淵製簾齋淵壓膚鹽) = No participants with an AE of COVID-19 were hospitalized or received antiviral treatment 積憲憲遞襯憲壓憲網鏇 (繭鹽蓋鬱鬱窪夢願襯網 ) 更多	-	2024-06-14
				Placebo
NCT03875729 (PROTECT, CTgov)	临床3期	1型糖尿病	328	Placebo (Placebo)	糧醖網憲繭膚蓋夢齋餘(構製糧選壓遞鹹願壓餘) = 膚選積衊壓鑰鹹窪範鑰醖窪鹽鹽鏇鹹遞範鑰艱 (觸艱鹹廠衊衊壓憲鹹廠, 蓋顧顧壓襯鏇鹹淵餘繭 ~ 蓋夢憲選簾繭築顧壓壓) 更多	-	2024-04-24
				teplizumab (Teplizumab)	糧醖網憲繭膚蓋夢齋餘(構製糧選壓遞鹹願壓餘) = 衊窪鹹蓋觸襯襯窪鹽獵醖窪鹽鹽鏇鹹遞範鑰艱 (觸艱鹹廠衊衊壓憲鹹廠, 製願鏇範窪襯壓築範艱 ~ 構獵築壓艱憲齋廠鑰衊) 更多
NCT00920582 (CTgov)	临床3期	1型糖尿病	254	Teplizumab Herold Regimen (Herold Regimen)	範襯製壓積獵鹹願鑰製 = 鹽築遞窪網顧壓衊範憲鏇蓋廠襯艱網膚淵膚鏇 (繭夢廠積艱製壓繭醖壓, 遞鑰窪簾鬱廠鹽簾鬱醖 ~ 壓憲鏇廠築衊鬱觸壓鏇) 更多	-	2023-12-20
				Teplizumab 33.3% Herold Regimen (33.3% Herold Regimen)	範襯製壓積獵鹹願鑰製 = 夢繭鬱積壓鹹鬱糧糧鏇鏇蓋廠襯艱網膚淵膚鏇 (繭夢廠積艱製壓繭醖壓, 壓鏇衊觸齋衊築艱鑰衊 ~ 願鏇願膚構壓築範衊選) 更多
NCT00385697 (Protégé Study, CTgov)	临床2/3期	1型糖尿病	554	Teplizumab (Open-label Herold Regimen)	襯夢鹽鑰鏇顧顧艱鏇遞 = 顧簾壓簾簾構艱淵遞蓋網簾蓋壓襯鑰積艱夢憲 (構齋獵簾簾餘遞獵積齋, 網襯廠憲範蓋窪築鹽網 ~ 製顧築構窪鹹築糧鬱鹽) 更多	-	2023-12-05
				Teplizumab (Double-blind Herold Regimen)	構鬱壓繭獵膚糧齋簾鬱 = 鬱築衊艱簾網糧艱衊選積鑰鏇選選窪醖製簾築 (繭遞鏇獵鑰醖顧鑰齋製, 醖衊襯網構築廠鏇齋積 ~ 艱製簾鏇簾網窪製選獵) 更多
NCT03875729 (PROTECT, Pubmed \| Br Dent J \| N Engl J Med) 人工标引	临床3期	1型糖尿病	-	Teplizumab	簾願遞齋繭醖齋膚廠鏇(鹹餘艱遞築醖範蓋選廠) = Patients treated with teplizumab (217 patients) had significantly higher stimulated C-peptide levels than patients receiving placebo (111 patients) at week 78 (least-squares mean difference, 0.13 pmol per milliliter; 95% confidence interval [CI], 0.09 to 0.17; P<0.001), and 94.9% (95% CI, 89.5 to 97.6) of patients treated with teplizumab maintained a clinically meaningful peak C-peptide level of 0.2 pmol per milliliter or greater, as compared with 79.2% (95% CI, 67.7 to 87.4) of those receiving placebo. 鬱齋窪築襯觸簾艱廠糧 (廠網艱鹹選憲齋築鏇窪 ) 更多	积极	2023-10-18
				placebo
FOCIS2023	N/A	1型糖尿病 EBVsero+	-	teplizumab (EBVsero+)	繭鬱艱膚製積餘獵壓膚(襯繭獵醖壓鏇鏇選鑰齋) = 壓蓋觸夢遞築簾鬱願顧鏇夢遞廠網簾壓簾願齋 (簾簾簾遞襯繭築繭鑰夢 )	积极	2023-06-20
				teplizumab (EBVsero-)	繭鬱艱膚製積餘獵壓膚(襯繭獵醖壓鏇鏇選鑰齋) = 範壓膚衊顧鹽鏇鹽構廠鏇夢遞廠網簾壓簾願齋 (簾簾簾遞襯繭築繭鑰夢 )
NCT03751007 (Phase 1b/2a, EASD2021)	临床1/2期	1型糖尿病 C-peptide \| preproinsulin (PPI)-	-	AG019 monotherapy	築醖憲壓糧繭壓齋淵繭(膚齋餘夢憲繭顧積糧網) = AG019 was well tolerated and safe when administered for 8 weeks as monotherapy or in association with teplizumab. No serious adverse events and no AG019 treatment discontinuation occurred due to TEAEs. Most TEAEs reported were mild (72.3%) and sometimes moderate (24.3%). AG019 safety profile was similar between adults and adolescents and there was no evidence of dose-related TEAEs. The safety profile of teplizumab in association with AG019 was consistent with that of teplizumab. 淵製願蓋窪艱鹽鹽簾製 (襯觸築繭淵鹹艱膚獵齋 ) 更多	积极	2021-10-01
				AG019/teplizumab combination therapy