An Interventional, Phase 3 Extension Study to Investigate Long-term Safety and Tolerability of Tolebrutinib in Participants With Relapsing Multiple Sclerosis, Primary Progressive Multiple Sclerosis, or Nonrelapsing Secondary Progressive Multiple Sclerosis

This is a Phase 3 extension, global, multicenter study to assess the long-term safety and tolerability of tolebrutinib in adult participants (aged ≥18 years) with RMS, PPMS, or NRSPMS who were previously enrolled in the Phase 2b LTS (LTS16004) or 1 of the 4 Phase 3 tolebrutinib pivotal trials (GEMINI 1 [EFC16033], GEMINI 2 [EFC16034], HERCULES [EFC16645], or PERSEUS [EFC16035]).
SUBSTUDY: ToleDYNAMIC substudy

开始日期2024-04-16

申办/合作机构

Sanofi

NCT05283915

/ Completed临床1期

An Open-label Phase 1, Pharmacokinetic and Tolerability Study of Tolebrutinib Given as a Single Dose in Adult Participants With Mild Hepatic Impairment, and in Matched Participants With Normal Hepatic Function

The purpose of this parallel group, Phase 1, open-label, 2-arm, single dose, multi-center study is to assess the effect of mild hepatic impairment on pharmacokinetics (PK), safety and tolerability of tolebrutinib compared with normal hepatic function, in male and female participants aged 18 to 79 years.

开始日期2022-03-18

申办/合作机构

Sanofi

NCT05282030

/ Completed临床1期

An Open-label, Multi-center Phase 1 Pharmacokinetic and Tolerability Study of Tolebrutinib Given as a Single Oral Dose in Participants With Renal Impairment and in Matched Participants With Normal Renal Function.

The purpose of this parallel group, Phase 1, open-label, 2-arm study is to assess the effect of severe (Part A) and moderate (Part B, conditional) renal impairment (RI) on pharmacokinetics (PK), safety and tolerability of tolebrutinib tablets compared with normal renal function, in male and female participants aged 18 to 79 years.

开始日期2022-03-10

申办/合作机构

Sanofi

100 项与 Tolebrutinib 相关的临床结果

登录后查看更多信息

100 项与 Tolebrutinib 相关的转化医学

登录后查看更多信息

100 项与 Tolebrutinib 相关的专利（医药）

登录后查看更多信息

405

项与 Tolebrutinib 相关的文献（医药）

2025-12-31·ANNALS OF MEDICINE

Clinical outcomes and therapeutic modalities in older Chinese patients with MCL: a multi-center real-world retrospective study

Article

作者: Wang, Shuye ; Zhang, Weilong ; Jing, Hongmei ; Liu, Hui ; Wang, Jing ; Li, Lihong ; Liu, Shuozi ; Li, Zhenling ; Sun, Xiuhua ; Wang, Jingwen ; He, Juan ; Cai, Qingqing ; Yang, Yuan ; Li, Chunyuan ; Li, Xiaoling ; Zhang, Wei ; Yang, Ping ; Chen, Yingtong ; Xiao, Xiubin

BACKGROUND AND AIMS:

Mantle cell lymphoma (MCL), an uncommon lymphoma subtype, is clinically characterized by its heterogenous behavior. Established prediction system including several clinical and biological parameters can help in determining the aggressiveness of MCL in younger patients. However, there are limited parameters on predicting the clinical outcome of older patients. The present study was performed to identify the prognostic factors and optimal treatment modalities in older Chinese MCL patients.

METHODS:

Patients (age ≥ 65 yrs) with MCL from 19 comprehensive hospitals in China were included. Clinical characteristics, therapeutic strategies, progression-free survival (PFS) and overall survival (OS) time of these patients were collected.

RESULTS:

Totally, 259 eligible patients were enrolled. The median age of patients was 69 years (range, 65-88). The median of PFS and OS were 29 months (95%CI: 26-37) and 76 months (95%CI: 61-96) months, respectively. Multivariate regression analysis determined that ECOG score ≥ 2, high MIPI score and absence of maintenance treatment were independently associated with poorer PFS of MCL patients; while ECOG score ≥ 2 and absence of maintenance treatment were independently correlated with a poorer OS. Patients with MCL who received BTKi-containing regimens or maintenance therapy showed significantly longer PFS and OS than those who did not receive these therapies. Maintenance treatment can improve the survival rate of older patients with MCL regardless of TP53 status.

CONCLUSIONS:

ECOG ≥ 2, high MIPI score, and absence of maintenance therapy were associated with poorer survival outcomes for older Chinese MCL patients. Maintenance therapy and BTKi-containing regimens have been shown to increase the survival rate of older Chinese MCL patients.

2025-06-01·Pharmacology Research & Perspectives

A Comparative Analysis of Cardiovascular Events Associated With Acalabrutinib Versus Ibrutinib in Chronic Lymphocytic Leukemia: Insights From a Global Federated Network.

Article

作者: Pettitt, Andrew R ; Majrashi, Abdulrahman ; Gue, Ying X ; Shantsila, Alena ; Smith, Catrin Tudur ; Lip, Gregory Y H ; Williams, Stella

Chronic lymphocytic leukemia (CLL) is the most common form of leukemia in adults, characterized by the accumulation of dysfunctional lymphocytes. Ibrutinib, a first-generation Bruton tyrosine kinase (BTK) inhibitor, has significantly improved CLL treatment but is associated with adverse cardiovascular events such as atrial fibrillation (AF) and hypertension (HTN). Second-generation BTK inhibitors (BTKi) such as acalabrutinib were developed to have greater selectivity for BTK to reduce off-target effects and improve safety. Comparative real-world data between ibrutinib and second-generation BTKi are limited. This study analyzed data from the TriNetX Global Collaborative Network to compare cardiovascular outcomes in CLL patients who received ibrutinib or acalabrutinib. The two groups were well-balanced using propensity score matching. The outcomes examined included new-onset AF, HTN, reported heart failure, ventricular arrhythmias, bleeding, and all-cause mortality. The incidence of AF/flutter was lower for acalabrutinib compared to ibrutinib [5.8% vs. 11.7%; HR 0.59 (95% CI 0.43-0.83); p = 0.002]. The incidence of HTN was also lower in the acalabrutinib cohort [15% vs. 26.3%; HR 0.65 (95% CI 0.53-0.81); p < 0.05]. The incidence of heart failure, ventricular arrhythmia, bleeding events, or all-cause mortality did not differ after 3 years of treatment with acalabrutinib or ibrutinib, respectively. Our findings indicate that acalabrutinib has a more favorable cardiovascular toxicity profile compared to ibrutinib; therefore, validating the ELEVATE-RR trial in a real-world setting.

2025-06-01·SEMINARS IN ARTHRITIS AND RHEUMATISM

Bruton's tyrosine kinase – A new target for immune mediated inflammatory diseases?

Review

作者: Isaacs, John D

Bruton's tyrosine kinase (BTK) is a cytoplasmic protein that plays a key role in signalling pathways downstream of diverse surface receptors in B-cells and myeloid cells. These include the B-cell receptor itself, Fc receptors including FcεRI, toll-like receptors and chemokine receptors. Congenital deficiency of BTK causes X-linked agammaglobulinaemia because of B-cell developmental block, and BTK inhibitors (BTKi) are approved for the treatment of certain B-cell malignancies. They have also been studied in a variety of autoimmune and inflammatory diseases. In rheumatic conditions, results have been disappointing in rheumatoid arthritis (RA) and systemic lupus erythematosus but with some evidence for efficacy in Sjogren's syndrome. Data are more positive in multiple sclerosis, as well as in the cutaneous disease chronic spontaneous urticaria and possibly pemphigus vulgaris. BTKi may also find a role in severe allergic disease such as food allergy. First generation BTKi had a safety profile that included cardiotoxicity, hypertension, haemorrhage and rash. Second generation inhibitors have a more acceptable safety profile although dose-limiting toxicity is still observed in some conditions, including RA. Pharmacokinetic factors aside, the variable efficacy in different diseases is not fully explained but is likely to reflect disease dependence on different pathways in B-cells and myeloid cells and their relative sensitivity to BTKi.

397

项与 Tolebrutinib 相关的新闻（医药）

2025-05-22

·BioPharmaDive

Sanofi gains experimental Alzheimer’s drug through Vigil buyout

Sanofi has agreed to spend $470 million to take control of a young biotechnology company and its experimental drug for Alzheimers disease.The newly announced acquisition has Sanofi paying $8 for each share of Vigil Neuroscience it doesnt already own, reflecting a premium of nearly 250% to the price Vigil shares traded at Wednesday afternoon. The French pharmaceutical giant invested $40 million into Vigil last June, and in exchange got an exclusive right to be first in line for deal talks if Vigil chose to license out its platform of small molecule drugs that amplify a protein called TREM2.The most advanced of those drugs, code-named VG-3927, several months ago completed an early-stage study that enrolled both healthy volunteers and patients with Alzheimers disease. Vigils president and CEO, Ivana Magovevi-Liebisch, said in a statement that the acquisition should help fully unlock and accelerate the development of VG-3927.A second molecule from the platform, VGL101, is not being acquired by Sanofi.Instead, rights to that molecule will be returned to the original licensor, Amgen.Neuroscience is one of Sanofis core strategic research focuses, alongside cancer, rare diseases and the immune system. Yet, in terms of product sales, it pales in comparison to those other three areas. Sanofis portfolio lists just one neurology drug, the multiple sclerosis therapy Aubagio, which last year accounted for less than 1% of the 41 billion euros in net sales recorded by the companys biopharma unit.Sanofi is trying to grow this pool with a few experimental medicines that have progressed to the later stages of development. One, frexalimab, is what Sanofi touts as a pipeline-in-a-product asset, since the company believes it has the potential to treat a range of illnesses, from MS to lupus to diabetes. Another, riliprubart, is being evaluated against CIPD, a rare disorder that harms the peripheral nerves, leading to arm and leg weakness.A third, tolebrutinib, has been tested across different types of MS and is currently being considered for approval by Food and Drug Administration, with a decision deadline set for late September. Sanofi got ahold of tolebrutinib through its $3.7 billion purchase of Principia Biopharma in 2020.Notably, all of these medicines work by regulating the immune system, which is characteristic of Sanofis approach to neurology.The Vigil deal follows this approach, too, as TREM2 alerts the brains resident immune cells to threats like the toxic abeta plaques that are a hallmark of Alzheimers. When this protein is impaired or loses its function, it can increase the risk of nerve cell injury, inflammation and death.TREM2 represents a compelling target at the intersection of immune dysregulation and neurodegeneration, said Houman Ashrafian, Sanofis head of R&D, in that Wednesday statement.The acquisition also supports Sanofis strategy of leveraging our expertise in immunology to solve health problems with critical unmet need, Ashrafian added.The companies expect their deal to close sometime between July and the end of September, provided investors holding a majority of the outstanding shares of Vigil common stock approve it. Sanofi said Magovevi-Liebisch as well as Vigil backers Atlas Venture and Bruce Booth, a partner at the firm have signed voting and support agreements in favor of the deal.Together, they represent roughly 16.2% of Vigils total common shares outstanding.In addition to the upfront payment, Vigils shareholders will receive a so-called contingent value right that would entitle them to receive a deferred cash payment of $2 for each share they own once VG-3927 has its first commercial sale.“We believe this is a good outcome for shareholders in the current funding environment,“ wrote Myles Minter, an analyst at William Blair who covers Vigil, in a note to clients.Vigil debuted at $14 per share when it went public in January 2022, but has lost most of its market value since.In his own note, Stifel analyst Paul Matteis argued the timing of the deal is “interesting,“ given a TREM2-targeting drug from Alector and AbbVie recently failed in a mid-stage study. That setback “significantly diminished enthusiasm for the target,“ according to Matteis, so while there were reasons to think Vigil's drug could fare better, “it was going to be hard to have conviction in this until we saw [Phase 2] data.“ '

并购免疫疗法临床结果临床2期

2025-05-20

·梅斯医学

NEJM：BTK 抑制剂 Tolebrutinib 首次证实可延缓非复发性继发进展型多发性硬化症残疾进展

多发性硬化症（MS）是一种中枢神经系统（CNS）的慢性炎症性疾病，其特征是免疫系统攻击保护神经纤维的髓鞘，导致神经损伤和功能障碍。随着疾病的进展，神经功能障碍可能会逐渐加重。目前，疾病修饰疗法（DMTs）主要针对外周淋巴细胞活性，有效减少了CNS的局灶性炎症和急性复发，但对与复发无关的残疾累积效果有限，且尚未有针对非复发性继发进展型多发性硬化症（SPMS）的批准治疗方法。近期，发表在NEJM期刊的一项题为“Tolebrutinib in Nonrelapsing Secondary Progressive Multiple Sclerosis”的研究，旨在评估Tolebrutinib是否能够降低非复发性SPMS患者疾病进展风险，并探索其在减少CNS神经炎症和神经退行性变化方面的潜力。这是一项全球多中心、随机、双盲、安慰剂对照的3期临床试验（HERCULES试验），在全球31个国家/地区的264个中心进行，共筛选了1438名患者，其中1131名患者被随机分配到Tolebrutinib组（754名，每日一次60 mg）或安慰剂组（377名），并根据年龄和地理区域进行分层。研究的主要终点是至少持续6个月的确认残疾进展，通过时间-事件分析进行评估。关键次要终点包括至少持续3个月的确认残疾进展、至少持续6个月的确认残疾改善（残疾改善定义为EDSS评分从基线下降≥1.0分）以及从第6个月到试验结束时大脑体积的百分比变化。结果显示，Tolebrutinib组和安慰剂组患者的人口统计学和疾病特征相似，平均年龄约为40岁，大多数为白人，基线时的平均EDSS评分为5.5左右，表明存在中度残疾。Tolebrutinib组中确认残疾进展持续至少6个月的参与者比例为22.6%，而安慰剂组为30.7%。与安慰剂组相比，Tolebrutinib组残疾进展的风险降低了31%（风险比为0.69；95%置信区间为0.55至0.88；P=0.003）。这一结果表明Tolebrutinib在延缓残疾进展方面具有显著效果。Tolebrutinib组与安慰剂组相比，在至少持续3个月的确认残疾进展方面也有改善（风险比为0.76；95%置信区间为0.61至0.94；P=0.01）。在MRI相关的病变进展方面，Tolebrutinib组每年新的或扩大的T2加权MRI病变的平均数量显著少于安慰剂组（相对风险为0.62；95%置信区间为0.43至0.90；P=0.01）。此外，Tolebrutinib组在九孔柱测试和定时25英尺行走测试中显示出了更好的功能保持趋势，尽管部分结果未达到统计学显著性。Tolebrutinib组中有81.5%的参与者报告了不良事件，略高于安慰剂组的78.1%。Tolebrutinib组中常见的不良事件包括COVID-19和尿路感染。严重的不良事件在Tolebrutinib组中发生率为15.0%，略高于安慰剂组的10.4%。特别注意的是，Tolebrutinib组中有4.0%的参与者出现了丙氨酸氨基转移酶（ALT）水平升高超过正常范围上限3倍的情况，而安慰剂组为1.6%。其中1名Tolebrutinib组的参与者因肝移植后的术后并发症死亡，被认为可能与Tolebrutinib相关。总之，在非复发性SPMS患者中，Tolebrutinib治疗相较于安慰剂显著降低了至少持续6个月的确认残疾进展风险，同时在减少新的或扩大的MRI病变数量方面也显示出优势。Tolebrutinib是首个在非复发性SPMS患者中显示出能够延缓残疾进展的BTK抑制剂，其独特的优势在于能够穿透血脑屏障，直接作用于CNS内的炎症细胞，这可能解释了其在减少残疾进展和MRI病变方面的效果。该研究结果为SPMS的治疗提供了一种新的潜在选择，有望改善患者的生活质量并减缓疾病进展。参考资料：[1]Robert J. Fox, M.D. Amit Bar-Or, M.D., Anthony Traboulsee, M.D.Tolebrutinib in Nonrelapsing Secondary Progressive Multiple Sclerosis.April 8, 2025 N Engl J Med 2025;392:1883-1892 DOI: 10.1056/NEJMoa2415988撰文 | 梅斯医学编辑 | 木白●中国痴呆发病率飙升300%！而久坐易痴呆？Science：缺氧才是大脑“常态”，它“助攻”痴呆发展，但运动可使缺氧负担减轻52%●热议！99年女医生晚上搞副业收破烂，挣的比在医院多！和送外卖比较时薪、和保姆比较日薪，当医生完败！真想搞副业挣钱，医生该干什么？● 为什么有人天生吃不胖？Nature子刊：关键在于父母受孕季节！这个时节出生的人棕色脂肪更多，BMI更低，且内脏脂肪更少版权说明：梅斯医学（MedSci）是国内领先的医学科研与学术服务平台，致力于医疗质量的改进，为临床实践提供智慧、精准的决策支持，让医生与患者受益。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。点击下方「阅读原文」立刻下载梅斯医学APP！

临床结果临床3期

2025-05-20

·药明巨诺 JW Therapeutics

朱军教授、谢彦教授：《CSCO淋巴瘤诊疗指南2025》重磅更新，瑞基奥仑赛获推荐，引领R/R MCL治疗新方向

套细胞淋巴瘤（MCL）是非霍奇金淋巴瘤（NHL）的一种独特组织学亚型，其一线治疗后复发率较高，且治疗线数的增加与预后不良密切相关，有效的挽救治疗可以极大程度地延缓复发/难治性（R/R）MCL再次进展并改善患者预后1。近年来，随着双特异性抗体、新一代BTKi及嵌合抗原受体T（CAR-T）细胞治疗等创新疗法的涌现，MCL治疗格局正加速革新。在近期召开的中国临床肿瘤学会（CSCO）指南大会上，正式公布了《CSCO淋巴瘤诊疗指南2025》。新版指南将瑞基奥仑赛列为R/R MCL患者挽救治疗的II级推荐2，显示出其已进阶为R/R MCL标准治疗的重要组成。医脉通特邀北京大学肿瘤医院朱军教授、北京大学肿瘤医院谢彦教授结合本次指南更新，深入剖析CAR-T细胞治疗在MCL领域的应用前景。突破治疗困境，CAR-T细胞治疗开启R/R MCL治疗新篇章MCL因其兼具侵袭性淋巴瘤的快速进展性与惰性淋巴瘤的不可治愈性的特点3，在临床治疗中面临一定挑战。尽管现代治疗手段改善了MCL患者的生存，但R/R患者的预后仍不乐观。国际多中心回顾性研究显示，经BTKi治疗失败的患者中位总生存期（OS）仅2.9个月，其中接受后续治疗患者的中位OS可延长至5.8个月，而未接受后续治疗患者的中位OS仅为0.8个月4。这一结果同样在国内真实世界研究中得到验证，纳入的693例MCL患者在随访期间有409例（59.0%）患者出现疾病进展，而接受化疗、苯达莫司汀联合利妥昔单抗（BR）等后续治疗的360例R/R患者完全缓解（CR）率仅为7.2%-23.3%5。现有疗法存在一定局限性，如大剂量阿糖胞苷因毒性限制了适用人群6，R/R阶段仍缺乏有效治疗方案。因此积极探索新型治疗策略成为改善患者预后的关键。近年来，CAR-T细胞治疗的出现，为R/R MCL患者带来了新的治疗选择，该疗法是通过基因工程将自体T细胞进行改造，以表达CAR并对特定肿瘤抗原（如CD19）进行精准识别，进而特异性杀死肿瘤细胞，并产生记忆T细胞发挥长效抗肿瘤作用1,7。近年来，多项临床研究数据证实，CAR-T细胞治疗的应用显著改善了R/R MCL患者的临床结局。循证之光生辉，瑞基奥仑赛获CSCO指南R/R MCL II级推荐2024年8月，瑞基奥仑赛获得国家药品监督管理局（NMPA）批准，用于经过包括BTKi治疗在内的二线及以上系统性治疗的成人R/R MCL，成为当前国内唯一获批该适应症的CAR-T细胞治疗产品。在今年更新的《CSCO淋巴瘤诊疗指南2025》中，瑞基奥仑赛又首次被列为R/R MCL患者挽救治疗的II级推荐，为R/R MCL患者的治疗提供了权威指南的临床决策支持（图1）。图1 《CSCO淋巴瘤诊疗指南2025》对于R/R MCL的治疗推荐此次更新是基于JWCAR029-005研究8。截至2024年1月25日，这项开放标签、单臂、多中心II期临床研究纳入了70例R/R MCL患者，其中59例患者接受了CAR-T细胞输注。入组患者中位年龄59岁，既往均接受过BTKi治疗，其中70%为接受≥3线治疗的多重耐药患者。研究的主要终点为客观缓解率（ORR）。研究结果显示，对于预后较差的R/R MCL患者，瑞基奥仑赛可使其获得良好且持久的缓解。最佳ORR达81.36%，CR率为67.8%。中位无进展生存期（PFS）、中位缓解持续时间（DoR）和中位OS分别为13个月、18.1个月和19.5个月；在安全性方面，瑞基奥仑赛的不良事件（AEs）发生率低且可控，≥3级细胞因子释放综合征（CRS）及神经毒性（NT）发生率均仅为6.8%，且未发生药物相关性死亡事件。上述结果证实，瑞基奥仑赛治疗R/R MCL患者具有良好的疗效以及安全性，这一循证证据不仅支持其被纳入《CSCO淋巴瘤诊疗指南2025》中R/R MCL的II级推荐，更为挽救治疗方案的选择提供了重要依据。专家点评谢彦教授在《CSCO淋巴瘤诊疗指南2025》中，瑞基奥仑赛基于JWCAR029-005研究结果被纳入推荐，这标志着其在淋巴瘤治疗领域的重要地位得到进一步认可。在研究中，70%的入组患者接受过≥3线的治疗，结果显示，瑞基奥仑赛可使近七成预后较差的R/R MCL患者达到CR，为此类难治性患者提供了实现深度缓解的机会。在安全性上，≥3级CRS及≥3级NT的发生率低，这表明瑞基奥仑赛或可成为R/R MCL患者兼顾疗效与安全性的更好选择。相比弥漫大B细胞淋巴瘤（DLBCL）患者，MCL患者的细胞表面CD19的表达更高，因此在使用靶向CD19的CAR-T细胞治疗时，CRS和NT的发生概率可能高于DLBCL患者9；并且MCL多发于老年，而老年患者由于耐受性较差，更容易出现AEs10，因此对于老年患者的治疗选择应更加谨慎。相关临床研究显示，相较于接受CD28共刺激结构域的CAR-T细胞治疗的患者，接受4-1BB共刺激结构域的CAR-T细胞治疗的患者CRS和NT的发生率更低11，而JWCAR029-005研究结果也进一步证明了4-1BB CAR-T细胞治疗具有良好的安全性。此外，对于存在TP53突变/缺失、CDKN2A缺失、侵袭性变型、Ki-67％＞50％等高危因素的患者，既往研究显示，CAR-T细胞治疗亦展现了良好的疗效3。综上所述，瑞基奥仑赛凭借其良好的疗效与安全性，成为预后不良的R/R MCL患者的良好治疗方案，在MCL治疗领域具有重要的临床价值与应用前景。朱军教授MCL作为具有独特生物学特征的B细胞NHL，其治疗面临高复发率与耐药性等挑战。CHOICE研究对中国1261例MCL患者的真实世界治疗模式和预后情况进行了回顾性队列分析，结果显示48%的MCL患者在一线治疗（包括免疫化疗、BTKi方案等）后发生复发或难治，进入到R/R MCL挽救治疗阶段12。这一数据不仅暴露了目前治疗方案的局限性，也揭示了MCL患者在靶向治疗耐药后的治疗空白。在此背景下，《CSCO淋巴瘤诊疗指南2025》依据国内外研究结果，将CAR-T细胞治疗纳入推荐，为临床决策提供了与时俱进的指南指引。CAR-T细胞治疗的出现为R/R MCL患者带来了新的生机。JWCAR029-005研究表明R/R MCL患者接受瑞基奥仑赛治疗后可实现深度、长期的缓解，且安全性良好。其纳入指南推荐，不仅为临床医生提供了明确的治疗依据，还将推动MCL治疗向规范化、精准化方向发展。除CAR-T细胞单药治疗方案外，目前专家也正探究CAR-T细胞治疗与其他药物的联合治疗策略，以期实现更大的突破。如TARMAC研究在R/R MCL患者中探索了单采前给予BTKi治疗并持续至CAR-T细胞回输后6个月的联合治疗模式，结果显示，联合治疗后CR率为80%，12个月OS率和PFS率分别为100%和75%13，提示CAR-T细胞与BTKi的联合治疗模式具有良好疗效。未来随着对CAR-T细胞治疗更深入的研究，我们有望进一步提高MCL患者的生存获益，进而改写MCL的治疗格局。朱军教授北京大学肿瘤医院大内科主任、淋巴瘤科主任中国人体健康科技促进会理事长中国临床肿瘤学会（CSCO）理事会副理事长中国临床肿瘤学会监事会副监事长北京市希思科临床肿瘤研究基金会副理事长中国临床肿瘤学会淋巴瘤专家委员会前任主任委员北京抗癌协会副理事长北京癌症康复会会长中华医学会肿瘤分会副主任委员谢彦教授主任医师、医学博士现就职于北京大学肿瘤医院淋巴瘤科北京大学肿瘤医院内科教研室副主任北京医师协会血液内科专科医师分会委员擅长淋巴瘤诊治、造血干细胞移植，具有丰富的临床研究管理经验参考文献：1.王晓晖,等. 肿瘤药学,2024,14(01):1-8+143.2.中国临床肿瘤学会(CSCO)淋巴瘤诊疗指南 2025.3.中国抗癌协会血液肿瘤专业委员会,等. 中华血液学杂志, 2022, 43(7) : 529-536.4.Martin P. et al. 2016 Mar 24;127(12):1559-63.5.Yang P, et al. Cancer Med. 2023;00:1-13.6.Romaguera J, et al. J Clin Oncol. 2005 Oct 1;23(28):7013-23.7.Davila Ml，et al,. Int J Hematol. 2014 Apr;99(4):361-71.8.Yuqin Song, et al. Blood 2023; 142 (Supplement 1): 3024.9.Mark P Hamilton, et al. Blood Adv.2024 Jun 25;8(12):3314-3326.10.Rozental A, et al. Leuk Lymphoma. 2023;64(9):1514-1526.11.Cappell, et al. Nature reviews. Clinical oncology vol. 18,11 (2021): 715-727.12.Zeng D, et al. Int J Cancer. 2023 Sep 1;153(5):1016-1025.13.Minson A, et al. Blood. 2024;143(8):673-684.免责声明本文转载自医脉通血液科，版权归原作者或原平台所有，转载此文旨在传递行业资讯与学术分享。文章内容仅供读者参考，不可替代专业医疗建议，具体诊疗请遵循医疗机构或医师指导。关于瑞基奥仑赛注射液（商品名：倍诺达®）- 滑动查看更多介绍 -瑞基奥仑赛注射液（简称relma-cel，其血液肿瘤适应症的商品名：倍诺达®）是药明巨诺在巨诺医疗（百时美施贵宝旗下的公司）的CAR-T细胞工艺平台的基础上，自主开发的一款靶向CD19的自体CAR-T细胞免疫治疗产品。作为药明巨诺的首款产品，瑞基奥仑赛注射液已被中国国家药品监督管理局批准三项适应症，包括治疗经过二线或以上系统性治疗后成人患者的复发或难治性大B细胞淋巴瘤（r/r LBCL）、治疗经过二线或以上系统性治疗的成人难治性或24个月内复发的滤泡性淋巴瘤（r/r FL），以及治疗经过包括布鲁顿酪氨酸激酶抑制剂(BTKi)治疗在内的二线以及系统性治疗的成人复发或难治性套细胞淋巴瘤(r/r MCL)，成为中国首个获批为1类生物制品的CAR-T产品。倍诺达®是一款获得“重大新药创制”专项、新药上市申请优先审评资格及突破性治疗药物认定三项殊荣的CAR-T细胞免疫治疗产品。关于药明巨诺- 滑动查看更多介绍 -药明巨诺（股份代码：2126）是一家独立的、创新型的生物科技公司，专注于研发、生产及商业化细胞免疫治疗产品，并致力于以创新为先导，成为细胞免疫治疗引领者。创建于2016年，药明巨诺已成功打造了国际领先的细胞免疫治疗的综合性产品开发平台，以及涵盖血液肿瘤、实体肿瘤和自身免疫性疾病的细胞免疫治疗产品管线。药明巨诺致力于以突破性、高质量的细胞免疫治疗产品给中国乃至全球患者带来治愈的希望，引领中国细胞免疫治疗产业的健康规范发展。欲了解更多详情，请访问：www.jwtherapeutics.com前瞻性声明- 滑动查看更多介绍 -本发布所包含的前瞻性声明是基于管理层现有的期望和信心，会存在一定的不确定性或风险从而导致实际结果在实质上与所描述的有所区别。显著的风险和不确定性，可能包括了以下涉及的内容，以及在公司提交给香港交易所（HKEx）的报告中予以更为全面的描述。除非另有注明，公司提供截至发布日期当日的信息，并且明确无义务更新相关事项及其所含内容或提供任何解释。具体内容，详见公司官网：www.jwtherapeutics.com/cn/forward-looking-statements/

CSCO会议免疫疗法细胞疗法临床结果孤儿药

100 项与 Tolebrutinib 相关的药物交易

登录后查看更多信息

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
继发进展型多发性硬化	申请上市	澳大利亚	Sanofi-Aventis Australia Pty Ltd.	2025-02-25
重症肌无力	临床3期	美国	Sanofi	2021-12-03
重症肌无力	临床3期	中国	Sanofi	2021-12-03
重症肌无力	临床3期	日本	Sanofi	2021-12-03
重症肌无力	临床3期	巴西	Sanofi-Aventis Recherche et Développement SA	2021-12-03
重症肌无力	临床3期	巴西	赛诺菲（中国）投资有限公司	2021-12-03
重症肌无力	临床3期	加拿大	Sanofi	2021-12-03
重症肌无力	临床3期	德国	Sanofi-Aventis Recherche et Développement SA	2021-12-03
重症肌无力	临床3期	德国	赛诺菲（中国）投资有限公司	2021-12-03
重症肌无力	临床3期	匈牙利	Sanofi	2021-12-03

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


NCT04411641 (HERCULES, Pubmed \| N Engl J Med)	临床3期	继发进展型多发性硬化	1,131	Tolebrutinib 60 mg once daily	鹽遞觸艱齋願襯範鏇繭(艱繭築鑰襯構膚鹹蓋範) = 襯衊顧製鏇簾醖獵願鏇襯齋壓廠構遞艱築艱願 (衊壓衊蓋鏇齋顧鹹獵鹽 )	积极	2025-05-15
				Placebo	鹽遞觸艱齋願襯範鏇繭(艱繭築鑰襯構膚鹹蓋範) = 餘遞鬱鑰夢鏇鏇獵襯築襯齋壓廠構遞艱築艱願 (衊壓衊蓋鏇齋顧鹹獵鹽 )
NCT04410978 \| NCT04410991 (GEMINI 1, Pubmed \| N Engl J Med) 人工标引	临床3期	复发性多发性硬化	-	Tolebrutinib 60 mg once dailyTolebrutinib 60 mg once daily	膚構壓觸顧糧顧壓蓋觸(艱淵願膚顧淵蓋蓋膚選) = 願餘願鬱壓襯淵衊願觸範觸遞鏇製築遞餘膚醖 (範衊遞製鏇簾蓋餘齋膚 ) 更多	不佳	2025-04-08
				Teriflunomide 14 mg once daily	膚構壓觸顧糧顧壓蓋觸(艱淵願膚顧淵蓋蓋膚選) = 範構窪糧鹹構蓋觸簾蓋範觸遞鏇製築遞餘膚醖 (範衊遞製鏇簾蓋餘齋膚 ) 更多
ASH2024	N/A	慢性淋巴细胞白血病 \| 小淋巴细胞淋巴瘤	-	BTKi	選製夢淵選願願蓋鑰願(糧鏇顧憲淵窪醖衊築願) = 願襯選糧繭鹹顧觸餘齋鏇憲窪遞網糧製簾築獵 (願簾鏇蓋餘糧壓遞襯繭 )	-	2024-12-09
				Anticoagulant/Antiplatelet	選製夢淵選願願蓋鑰願(糧鏇顧憲淵窪醖衊築願) = 鬱繭網糧廠繭觸鏇夢選鏇憲窪遞網糧製簾築獵 (願簾鏇蓋餘糧壓遞襯繭 )
ASH2024	N/A	小淋巴细胞淋巴瘤	-	BTKi (Ibrutinib)	糧築網顧積蓋積齋鏇願(窪蓋糧膚鏇繭觸糧製艱) = A quarter of all pts experienced a non-fatal bleed, resulting in <15% of the pts discontinuing and >50% pts interrupting their BTKi tx 觸廠齋窪顧廠鬱積繭憲 (廠繭範艱鬱觸蓋顧製網 )	-	2024-12-09
				Prescription Anticoagulants or Antiplatelets
NCT04411641 (HERCULES, NEWS) 人工标引	临床3期	继发进展型多发性硬化	-	Tolebrutinib	鏇鏇範鹹築膚夢顧簾築(製願獵鑰糧網窪築築淵): HR = 0.69 (95% CI, 0.55 ~ 0.88), P-Value = 0.0026 达到更多	积极	2024-09-02
				placebo
PINC AI Healthcare Database (ASCO2024)	N/A	慢性淋巴细胞白血病	2,091	Ibrutinib	憲網範鹹鬱製遞願顧鑰(齋簾築糧餘獵構網醖鏇) = 遞選糧壓鹹鬱窪鬱醖鏇廠鬱鬱壓遞襯膚鑰網膚 (構蓋獵齋構蓋衊廠襯構 )	积极	2024-05-24
				Acalabrutinib	憲網範鹹鬱製遞願顧鑰(齋簾築糧餘獵構網醖鏇) = 壓鏇構鏇淵鬱衊鹽網鑰廠鬱鬱壓遞襯膚鑰網膚 (構蓋獵齋構蓋衊廠襯構 )
NCT04411641 (HERCULES, ECTRIMS2023)	临床3期	继发进展型多发性硬化	-	Tolebrutinib 60 mg	繭構觸夢鏇繭膚糧獵鑰(觸範鏇壓構築糧淵憲築) = 糧遞顧願遞淵夢襯獵繭鏇繭製衊鏇窪鑰窪簾獵 (繭願鹽齋網顧淵獵艱選, 14.4) 更多	-	2023-09-30
NCT03996291 (Phase 2b tolebrutinib trial, ECTRIMS2023)	临床2期	复发性多发性硬化	125	5/60 mg	獵衊願鏇選衊遞醖築憲(選蓋鬱製遞鏇觸網醖積) = 糧窪艱鏇築醖遞窪艱製獵餘繭鹹顧廠構繭鑰壓 (淵願積製積選鹹願觸夢 ) 更多	-	2023-09-30
				15/60 mg	獵衊願鏇選衊遞醖築憲(選蓋鬱製遞鏇觸網醖積) = 鹹餘觸鹹範鏇鑰鹹鹹網獵餘繭鹹顧廠構繭鑰壓 (淵願積製積選鹹願觸夢 ) 更多
NCT04410978 \| NCT04410991 (GEMINI 1 and 2, EAN2023)	临床3期	复发性多发性硬化	-	Tolebrutinib 60 mg	衊齋構構膚積獵糧壓鬱(鏇選鹽糧窪窪繭齋觸衊) = 齋觸範憲遞範簾醖築構壓膚築艱齋簾築夢憲鹽 (醖構鬱範繭鑰糧醖繭襯 )	积极	2023-06-28
				Teriflunomide 14 mg	衊齋構構膚積獵糧壓鬱(鏇選鹽糧窪窪繭齋觸衊) = 顧餘觸選鑰觸襯製淵製壓膚築艱齋簾築夢憲鹽 (醖構鬱範繭鑰糧醖繭襯 )