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更新于：2025-06-18

Imatinib mesylate

甲磺酸伊马替尼

更新于：2025-06-18

概要

基本信息

药物类型

小分子化药

别名

imatinib、imatinib mesilate、α-(4-methyl-1-piperazinyl)-3'-((4-(3-pyridyl)-2-pyrimidinyl)amino)-p-toluidide

+ [32]

靶点

ABL x Bcr-Abl x PDGFRs x c-Kit

作用方式

抑制剂、拮抗剂

作用机制

ABL 抑制剂(Abl family tyrosine kinases inhibitors)、Bcr-Abl抑制剂(bcr-abl酪氨酸激酶抑制剂)、PDGFR拮抗剂(血小板衍生生长因子受体拮抗剂)

治疗领域

肿瘤免疫系统疾病消化系统疾病+ [5]

在研适应症

加速期费城染色体阳性慢性粒细胞白血病急变期费城染色体阳性慢性粒细胞白血病慢性期费城染色体阳性慢性粒细胞白血病+ [33]

非在研适应症

腹部肿瘤肢端雀斑恶性黑色素瘤急性嗜酸细胞白血病+ [176]

原研机构

Novartis Pharma AG

在研机构

Teva Pharmaceuticals Europe BV

The Children's Oncology Group Foundation, Inc.

National Cancer Institute

+ [14]

非在研机构

Novartis AG

Sanofi

Jade Biosciences, Inc.

+ [35]

权益机构

杭州畅溪制药有限公司

Vectura Group Ltd.

Novartis AG

+ [2]

最高研发阶段批准上市

首次获批日期

美国 (2001-05-10),

费城染色体阳性慢性粒细胞白血病

最高研发阶段(中国)批准上市

特殊审评加速批准 (美国)、孤儿药 (美国)、孤儿药 (日本)、优先审评 (中国)

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结构/序列

分子式C30H35N7O4S

InChIKeyYLMAHDNUQAMNNX-UHFFFAOYSA-N

CAS号220127-57-1

关联

656

项与甲磺酸伊马替尼相关的临床试验

ISRCTN16409847

/ RecruitingN/AIIT

Investigation of novel and established therapies in a human intravenous lipopolysaccharide model of sepsis

开始日期2025-08-06

申办/合作机构

Belfast Health & Social Care Trust

NCT06889168

/ Recruiting临床1期IIT

A Phase 1, Randomized, Double-blinded, Placebo Controlled, Trial Evaluating the Long-term Safety and Tolerability of Imatinib for the Treatment of Lymphangioleiomyomatosis [LAMP-2 Trial]

Lymphangioleiomyomatosis (LAM) is a rare cystic lung disease that appears to behave like a slowly growing cancer. Since clinical progression is very slow, new blood tests have been used to speed the time required to find safe and effective medications. A large National Institute of Health study called MILES showed that sirolimus (also known as Rapamycin) improved lung function in individuals with LAM. Since most individuals with LAM and impaired lung function are now on sirolimus, future studies may prove more difficult. Laboratory studies suggested that Imatinib mesylate (imatinib), an FDA-approved drug for leukemia, initiates LAM cell death. A pilot trial with imatinib titled "Imatinib Mesylate for the treatment of Lymphangioleiomyomatosis" - (LAMP-1) was funded by the Department of Defense in 2016, and documented (1) the safety of use of tyrosine kinase inhibitors in patients with LAM; (2) the safety of concurrent use of tyrosine kinase and mTOR inhibitors; and, (3) short term variability in vascular endothelial growth factor D (VEGF-D) - a LAM biomarker, as a response to therapies. Due to the short-term LAMP-1 trial, LAMP-2 will be a longer-term 6-month clinical study evaluating the safety and tolerability of imatinib in patients with LAM. Patients that participate in the trial will come in for 5 office visits and check-up phone calls every 2 weeks over the course of 6 months.

开始日期2025-06-01

申办/合作机构

Columbia University

[+1]

NCT06124157

/ Recruiting临床3期IIT

An International Pilot Study of Chemotherapy and Tyrosine Kinase Inhibitors With Blinatumomab in Patients With Newly-Diagnosed Philadelphia Chromosome-Positive or ABL-class Philadelphia Chromosome-Like B-cell Acute Lymphoblastic Leukemia

This phase III trial compares the effect of the combination of blinatumomab with dasatinib or imatinib and standard chemotherapy versus dasatinib or imatinib and standard chemotherapy for treating patients with Philadelphia chromosome positive (PH+) or ABL-class Philadelphia chromosome-like (Ph-Like) B-Cell acute lymphoblastic leukemia (B-ALL). Blinatumomab is a bispecific antibody that binds to two different proteins-one on the surface of cancer cells and one on the surface of cells in the immune system. An antibody is a protein made by the immune system to help fight infections and other harmful processes/cells/molecules. Blinatumomab may bind to the cancer cell and a T cell (which plays a key role in the immune system's fighting response) at the same time. Blinatumomab may strengthen the immune system's ability to fight cancer cells by activating the body's own immune cells to destroy the tumor. Dasatinib and imatinib are in a class of medications called tyrosine kinase inhibitors. They work by blocking the action of an abnormal protein that signals cancer cells to multiply, which may help keep cancer cells from growing. Giving blinatumomab and dasatinib or imatinib in combination with standard chemotherapy may work better in treating patients with PH+ or Ph-Like ABL-class B-ALL compared to dasatinib or imatinib and chemotherapy alone.

开始日期2025-05-30

申办/合作机构

National Cancer Institute

100 项与甲磺酸伊马替尼相关的临床结果

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100 项与甲磺酸伊马替尼相关的转化医学

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100 项与甲磺酸伊马替尼相关的专利（医药）

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22,505

项与甲磺酸伊马替尼相关的文献（医药）

2025-12-31·ANNALS OF MEDICINE

Construction of a novel prognostic model based on lncRNAs-related to DNA damage repair for predicting the prognosis of clear cell renal cell carcinoma

Article

作者: Chen, Peng ; Li, Jian ; Tian, Renli

BACKGROUND:

CcRCC has the characteristics of high aggression, high metastasis, high mortality, wide tumour heterogeneity and variable clinical course. The purpose of this study was to explore the potential value of lncRNAs-related to DNA damage repair (DDR) in predicting the prognosis of ccRCC by construction and verification a novel prognostic model.

METHODS:

RNA-seq data and clinical data of ccRCC were downloaded from public databases. Subsequently, Pearson correlation analysis and differential expression analysis were performed to identify DElncRNAs-related to DDR. Then, through univariate Cox analysis and LASSO analysis, the DElncRNAs-related to DDR associated with prognosis were screened for the construction of novel risk score prognostic model. In addition, functional annotation, tumour mutation burden, immune correlation and drug sensitivity analyses were performed based on risk score to assess the characteristics of patients in different risk score groups.

RESULTS:

Based on univariate Cox analysis and LASSO analysis, four best DElncRNAs-related to DDR were selected. Subsequently, a novel risk score prognostic model based on these four DElncRNAs was constructed through LASSO. Multivariate Cox analysis showed that risk score and age were independent prognostic factors for ccRCC (p < 0.05). Functional enrichment analysis showed that DDR-related biological processes were mainly enriched in the high risk group. The highly mutated genes in the high and low risk groups were the same (VHL, PBRM1 and TTN), but they also had their own unique mutated genes. Pearson correlation analysis showed that the risk score was significantly (p < 0.05) positively correlated with the infiltration degree of CD8 T cells evaluated by six algorithms. In addition, it was found that the high and low risk groups had different sensitivities to the drugs Etoposide, Imatinib, Sorafenib, Bosutinib and Sunitinib.

CONCLUSION:

A novel prognostic model was constructed based on four DElncRNAs-related to DDR. The model has satisfactory accuracy in predicting survival of ccRCC patients.

2025-12-31·JOURNAL OF MEDICAL ECONOMICS

Non-optimal treatment with first tyrosine kinase inhibitor and associated economic burden in chronic myeloid leukemia

Article

作者: Guérin, Annie ; Jadhav, Kejal ; Romdhani, Hela ; Yang, Daisy ; Latremouille-Viau, Dominick ; Wei, David ; Kota, Vamsi

AIMS:

Although adenosine triphosphate (ATP)-competitive tyrosine kinase inhibitors (TKI) approved by the Food and Drug Administration have demonstrated effectiveness in treating chronic myeloid leukemia (CML), patients often experience intolerance or resistance, leading to non-optimal treatment (NOPT). This study assessed the treatment patterns, as well as NOPT and its associated economic burden, in newly diagnosed patients with CML receiving first TKI treatment.

METHODS:

This retrospective study identified adult patients with ≥2 CML diagnoses who initiated first TKI treatment (imatinib, dasatinib, nilotinib, or bosutinib) in 2012 or later from United States administrative health claims databases (01 January 2007-30 June 2022). Treatment sequence and time to treatment discontinuation/switch were assessed. NOPT, identified based on treatment modification/adherence criteria, and its associated incremental healthcare resource utilization and medical costs were evaluated.

RESULTS:

Nearly a quarter of patients experienced NOPT, as indicated by early treatment modifications or low treatment adherence. NOPT was associated with significant incremental healthcare resource utilization (80% more inpatient admissions; twice as many inpatient days; 30% more outpatient visits) and medical costs (adjusted mean cost difference = $13,551 per-patient-per-year).

LIMITATIONS:

Given the lack of information on reasons of treatment modification in health claims data, NOPT was identified based on indicators of management of intolerance and resistance to TKI.

CONCLUSION:

These findings highlight that unmet clinical needs and significant economic burden still persist in this population and that patients with CML may benefit from using therapeutic options with better efficacy and tolerability profiles as first treatment.

2025-12-01·Journal of Gastrointestinal Cancer

Neoadjuvant Imatinib in Recurrent/Metastatic Gastrointestinal Stromal Tumors: A Systematic Review and Meta-analysis of Proportions

Review

作者: Ntanasis-Stathopoulos, Ioannis ; Filippatos, Charalampos ; Zagouri, Flora ; Stavrou, Niki ; Gavriatopoulou, Maria ; Sergentanis, Theodoros N ; Memos, Nikolaos

Abstract:

Introduction:

Metastatic and recurrent gastrointestinal stromal tumors (GISTs) present challenging clinical management. Imatinib is the standard first-line therapy, improving survival and reducing tumor burden in the neoadjuvant use, facilitating surgical intervention. This systematic review and meta-analysis assessed the efficacy of neoadjuvant imatinib in metastatic/recurrent GISTs, highlighting its potential to enhance surgical outcomes and overall patient management.

Methods:

A systematic search was conducted in PubMed, Embase and Scopus (end-of-search: February 13, 2025) for records on neoadjuvant imatinib therapy in recurrent/metastatic GISTs. Pooled proportions and 95% confidence intervals were calculated with common-effect and random-effects models. Subgroup and meta-regression analysis were performed, addressing heterogeneity and examining any potential association between the factors that varied and the outcomes reported. The present meta-analysis was performed following the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) guidelines.

Results:

The search identified 957 articles, and 14 were analyzed. The meta-analysis of proportions indicated that 2-year and 5-year PFS were 76% (95% CI 58–88%) and 43% (95% CI 17–74%), respectively, while 2-year and 5-year OS were 84% (95% CI 78–89%) and 60% (95% CI 51–68%), respectively. The pooled R0 resection rate was 82% (95% CI 64–92%), associated positively with that of radiological partial response (PR) (β = 3.92, p < 0.001). Further meta-regression analysis yielded no significant association with preoperative imatinib duration.

Conclusion:

The present meta-analysis of trials and studies on metastatic or recurrent GISTs highlights key insights into post-surgery patient outcomes following neoadjuvant treatment with imatinib. Pooled effect estimates revealed promising 2-year and 5-year PFS rates of 76% and 43%, respectively, and 2-year and 5-year OS rates of 84% and 60%, respectively. Furthermore, the high pooled R0 resection rate of 82% emphasizes a substantial surgical efficacy in this population, while it was significantly correlated with successful R0 resections in patients with favorable outcomes.

1,428

项与甲磺酸伊马替尼相关的新闻（医药）

2025-06-17

·今日头条

挺过TACE只是序章，肝癌TACE术后三大防线+创新疗法，防复发/转移

TACE（经肝动脉化疗栓塞术）作为中晚期肝癌的核心治疗手段，通过精准栓塞肿瘤供血动脉并释放局部化疗药物，为无数患者筑起生命防线。但临床数据显示，术后1年仍有40%-60%的复发率——这不是治疗的终点，而是防复发"攻坚战"的起点！每一位挺过TACE的患者都该知道：科学预防能改写命运，让复发阴影不再成为"必然"。那么，肝癌TACE术后如何预防复发、争取更长的生存期呢？下面全球肿瘤医生网小编就为大家简单介绍一下，建议先收藏以备不时之需（（注：以下内容仅供就医参考，具体治疗/复查方案需遵医嘱）。肝癌TACE术后复发率居高不下的原因肝癌TACE术后复发率居高不下的根源与下列因素脱不开关系： 1、TACE对直径>5cm肿瘤或多发转移灶控制有限，残留癌细胞易复发。 2、肝癌细胞易通过门静脉微转移，TACE难以完全清除微小病灶。 3、90%肝癌患者合并肝硬化，肝组织慢性损伤易诱发新病灶。肝癌患者必看：复发的高危人群及复发征象高危人群预警存在下列情况的肝癌患者，在TACE术后应遵医嘱定期复查，并采取相应的治疗，谨防肝癌复发/转移。 1、肿瘤直径 > 5cm、多发结节、门静脉癌栓； 2、术后甲胎蛋白（AFP）未降至正常（<20ng/mL）； 3、乙肝 / 丙肝病毒活跃（HBV-DNA>10³ IU/mL）。防复发的 “红线警示” 若肝癌TACE术后，出现以下症状，可能提示复发，建议立即就医：右上腹持续性疼痛、不明原因消瘦（1 个月 > 5kg）、出现黄疸、出现黑便等。肝癌TACE术后防复发是场“系统战”：三重防线筑牢生存希望如今的肝癌防复发已进入"精准时代"，从过去"经验性预防"到现在"个体化干预"的跨越，正让越来越多患者突破"5年生存魔咒"！这需要医学监测、规范治疗与生活管理的黄金三角配合——就像为肝脏装上"复发预警系统"+"免疫防护盾"+"健康调节器"，每一个环节都在为长生存加码。精准监测：早发现复发的“雷达系统” 1、影像学检查：常用检查包括腹部超声、增强MRI/CT（灵敏度高于超声）、PET-CT（常规影像难以鉴别病灶性质时应用）等。 2、肿瘤标志物检测：动态监测AFP、异常凝血酶原（PIVKA-II），若持续升高需警惕复发（如AFP>400ng/mL且排除活动性肝炎）。 3、液体活检：通过ctDNA检测术后外周血中的肿瘤基因突变，比影像学早3-6个月发现复发迹象，灵敏度达92%。主动干预：从“被动等待”到“主动出击” 1、靶向+免疫联合：根据CSCO指南，高危患者可术后使用阿替利珠单抗+贝伐珠单抗（证据等级1A），降低复发风险38%。 2、酪氨酸激酶抑制剂：索拉非尼、仑伐替尼术后辅助治疗，适用于合并血管侵犯或高复发风险者。 3、抗病毒治疗：乙肝患者需终身服用恩替卡韦/替诺福韦，确保HBV-DNA持续阴性（降低复发风险50%）。 4、放疗辅助：门静脉癌栓患者可考虑立体定向放疗（SBRT），延长中位生存期至14个月。 5、 TACE再干预：术后发现微小残留灶，可再次行TACE或联合微波消融。生活方式：构筑防复发的“免疫城墙” 1、睡眠与压力管理：保证充足的睡眠，避免熬夜。研究显示，熬夜可致皮质醇升高，从而可能增加肿瘤生长的风险。 2、饮食调理：严格戒酒（酒精可诱发肝损伤）、避免黄曲霉毒素（霉变坚果/谷物）、限制亚硝酸盐（腌制食品）。可摄入有助于保肝的食物（如西蓝花、深海鱼等），还可适当补充益生菌、益生元等，以调节肠道菌群，改善肝内免疫微环境，增强机体抗癌免疫力。三大前沿技术：肝癌TACE术后防复发新武器 SCG101-TCR-T疗法：暴击乙肝病毒相关肝细胞癌，完成抗癌和抗病毒双杀 SCG101是一款乙肝抗原特异性TCR-T细胞疗法，具有抗病毒和抗肿瘤的双重疗效，已获中国国家药品监督管理局（NMPA）、美国食品药品监督管理局（FDA）等批准，用于乙肝病毒相关肝细胞癌的治疗。2023年国际细胞与基因治疗大会（ISCT）上，公布了SCG101突破性临床数据，结果显示如下： 1、 HBV感染改善：在回输SCG101后，乙肝表面抗原阳性肝细胞实现100%清除！ HBsAg（HBV血清学标志）从治疗前的557.96 IU/mL，骤降至1.3 IU/mL （治疗第7天）、 0.08 IU/mL （治疗第28天）。 ▼回输SCG101前后，HBsAg的变化 ▲图源“CISION”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 2、肿瘤显著缩小：患者在治疗第28天，达到部分缓解（PR），肿瘤靶病灶较基线时缩小66% ；在治疗第4个月时，肿瘤靶病灶进一步缩小74.5% ；更为惊喜的是，另一处病灶完全消失。截至数据统计时止，肿瘤已超过6.9个月未进展！ ▼患者回输SCG101前后的影像学变化 ▲图源“CISION”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 C-CAR031 CAR-T细胞疗法精准打击肝细胞癌，客观缓解率高达75.0%！ C-CAR031是一种针对肝细胞癌（HCC）的新型GPC3 CAR-T细胞疗法，2024年ASCO大会上，公布了I期临床研究（NCT05155189）的惊艳数据。结果显示：全部剂量水平患者的客观缓解率（ORR）达56.5%、疾病控制率高达91.3%！这也意味着，超90%的肝细胞癌患者在接受C-CAR031 CAR-T细胞治疗后，肝内和肝外的肿瘤病灶均出现缩小表现。肿瘤中位缩小率达到42.2% （范围：-28.1%~94.4%）。 ▲图源“ApexOnco”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除癌症疫苗癌症疫苗通过向人体输入肿瘤抗原，激活免疫系统对癌细胞的识别和攻击能力，回输到体内后，可引导T细胞精准识别和攻击癌细胞，提升机体的抗肿瘤免疫能力，最终达到延长肝癌患者的无复发生存期，提高患者的生活质量的目的。在2024年美国临床肿瘤学会（ASCO）上，公布了我国自主研发一款基于树突状细胞(DC)的mRNA疫苗首次人体试验（NCT03674073）的振奋数据。疫苗接种组和对照组患者的中位随访时间为48.4个月和38.8个月。结果显示：疫苗接种组与对照组相比， 1年复发率分别为18.2% vs 33.3%，2年复发率分别36.4% vs 51.4%。 ▲截图源自“ASCO” 小编寄语随着医学技术的不断进步，免疫细胞治疗和癌症疫苗有望成为肝癌 TACE 术后复发预防的重要手段。未来，通过多学科协作，将这些前沿疗法与传统治疗方法相结合，定制个性化的预防方案，或许能为肝癌患者带来更长的生存期和更高的生活质量。参考资料 [1]Liang P,et al.A first-in-human study to evaluate a personalized neoantigen-based mRNA-loaded dendritic cell vaccine, in combination with ablation, in patients with hepatocellular carcinoma[J]. 2024. https://ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.e14513 [2]https://www.oncologypipeline.com/apexonco/asco-2024-astrazenecas-gpc3-secret-sauce 本文为全球肿瘤医生网原创，未经授权严禁转载

CSCO会议

2025-06-16

·药事纵横

下一个口服阿尔兹海默症治疗药物破局者花落谁家？

阿尔茨海默病（AD）是一种慢性进行性神经退行性疾病，表现为记忆力及其他认知功能的进行性减退，其中部分病人进展为痴呆。近年来，以Aβ假说、Tau假说、神经炎症假说为代表的多种假说的发展，对APOE4等遗传风险因素的深入研究，以及新兴靶点的不断涌现，推动着全球药企加入AD研发行列。口服药物由于服药的便利性，也成为近年来阿尔茨海默病（AD）药物研发的热点。2024年7月29日，Alpha Cognition公司宣布，其治疗阿尔茨海默病的口服缓释药物Zunveyl（benzgalantamine）获得美国FDA批准上市，用于治疗轻度至中度阿尔茨海默病。该药物具有潜在更优的胃肠道安全性，有望提高患者的用药依从性，因此被认为能为阿尔茨海默病患者的认知和整体功能提供长期益处。这也是近十年来FDA批准的第二种阿尔茨海默病口服疗法（上一款为2014年获批的美金刚/多奈哌齐复方制剂）。下一款口服药物花落谁家，以下盘点几款口服AD药物的研发进展。ALZ-801药物名称：Valiltramiprosate（ALZ-801）研发公司：Alzheon阶段：3期临床ALZ-801是Alzheon旗下一款在研口服小分子药物，为高牛磺酸前体药物。可抑制β淀粉样蛋白(Aβ)错误折叠，稳定单体，阻断聚集，从而抑制可溶性神经毒性淀粉样蛋白寡聚体的形成，ALZ-801于2017年获得美国FDA的快速通道资格。此前公布的2期临床数据显示，52周时，与基线相较，ALZ-801造成病患血浆P-tau181蛋白41%显著下降。与对照组相较，神经影像学分析亦显示药物可能具有对脑结构的神经保护功用。此外，在第3、6个月与一年时，患者的记忆测试皆高于基线值，这与所观察到的生物标志物水平与大脑影响结果相符。在超过2000位阿尔茨海默病患者中，ALZ-801展现良好的安全性，与之前试验结果一致，没有观察到血管源性脑水肿的情形。2025年4月10日，Alzheon公布了ALZ-801治疗早期阿尔茨海默病（AD）患者的关键性III期APOLLOE4研究的结果。结果显示，治疗第78周，在总人群中，该研究未达到主要终点（11%获益，p=0.607）。在轻度认知障碍（MCI）阶段AD亚组中，患者的ADAS-Cog13评分显著下降（52%获益，p=0.0041），并且CDR-SB评分和痴呆残疾评估（DAD）评分的降低具有临床意义。Alzheon计划未来在携带一个或不携带APOE4基因拷贝的AD患者中开展治疗性研究和预防性研究。药物名称：masitinib研发公司：AB Science阶段：3期临床Masitinib是一种口服酪氨酸激酶抑制剂，通过抑制肥大细胞和小胶质细胞/巨噬细胞活性，重塑神经元微环境，将神经免疫系统从神经毒性状态转换为神经保护状态，在神经退行性疾病中表现出神经保护作用。肥大细胞和小胶质细胞/巨噬细胞是存在于中枢神经系统中的一类先天性免疫细胞，越来越多的证据表明它们与神经退行性疾病如阿尔茨海默病、进行性多发性硬化和肌萎缩侧索硬化（ALS）的病理生理学有关。Masitinib一方面通过特异性抑制c-Kit、Lyn、和Fyn蛋白激酶的活性，来抑制肥大细胞的激活。另一方面，它也可通过抑制1型巨噬细胞集落刺激因子受体（MCSFR-1）来靶向小胶质细胞/巨噬细胞。在一项针对轻中度AD患者的关键性3期临床试验中，与安慰剂相较，masitinib能够显著减缓患者的认知恶化情形。药物名称：simufilam研发公司：Cassava Sciences阶段：3期临床Simufilam是一款口服小分子化合物， Simufilam的靶点细丝蛋白A（filamin A，FLNA）是在大脑中高度表达的一种支架蛋白。在患者大脑中，FLNA的构象发生改变，会让β淀粉样蛋白片段（Aβ42）能够通过与受体结合，引发过度磷酸化的tau蛋白的产生，以及炎性细胞因子的释放。Simufilam通过与FLNA结合，恢复其正常构象和功能，阻止Aβ42的信号传导，因此具有通过靶向一个靶点，同时减少神经变性和神经炎症的潜力。在2期临床试验中simufilam取得了积极结果，不仅表现出良好的安全性和耐受性，在探索性疗效终点方面，47%接受治疗的患者在评估认知衰退的ADAS-Cog评分上，在接受治疗1年后与基线相比，改善4.7点。然而，2024年11月25日，Cassava Sciences（Cassava）宣布其在研的阿尔茨海默病（AD）新药——simufilam的III期ReThink-ALZ研究未达主要终点、次要终点及探索性生物标志物终点。相比于安慰剂组，试验组患者的ADAS-COG12和ADCS-ADL量表评分并无明显改善趋势。其中，经ADCS-ADL量表所得检测结果显示，试验组的情况甚至还不如安慰剂组。但好的消息是，尽管没有显示出期待的疗效，但simufilam的安全性数据良好，试验组不良事件发生率与安慰剂组类似。药物名称：VG-3927研发公司：Vigil Neuroscience阶段：2期临床在研究神经退行性疾病的过程中发现，小胶质细胞的功能障碍会导致中枢神经系统中斑块的聚集及慢性炎症。而通过激活TREM2，能够增强小胶质细胞的吞噬和修复能力，从而提供神经保护。由于VG-3927不与sTREM2结合，与抗体TREM2激动剂相比，该疗法可最大限度地提高受体活化和小胶质细胞功能，这可能会增加其进入AD病灶的机会。此外，VG-3927没有Fc结构域，不会增加免疫系统中与淀粉样蛋白相关成像异常（ARIA）风险。VG-3927 的1 期临床试验结果喜人，在所有队列中安全性和耐受性良好，所有相关不良事件严重程度为轻度或中度，可自行消退，无需停药，也未报告严重的 AE；高度脑渗透性，有良好且可预测的 PK 曲线，支持每日一次给药；在脑脊液（CSF）中实现高达约 50% 的sTREM2 的稳健和剂量依赖性降低，显示出强烈的 PK/PD 关系、持续的靶标参与和 TREM2 激动剂活性。5月22日，国际医药行业巨头赛诺菲宣布与Vigil Neuroscience达成收购协议，计划以每股8美元现金的价格收购Vigil Neuroscience的所有已发行普通股，交易总价值约为4.7亿美元。本次收购的核心在于VG-3927。阿尔茨海默病患者众多，全球阿尔茨海默病药物市场规模预计将从 2023 年的 36.9 亿美元增长到 2030 年的52.1 亿美元，如此庞大的市场让各大药企争相进入该研发领域。然而，AD药物的研发向来九死一生，本文盘点了部分在研的AD口服药物，究竟谁能在这场竞赛中脱颖而出，我们拭目以待。参考文献：1.https://www.alphacognition.com/investors/news/alpha-cognitions-oral-therapy-zunveyl-receives-fda-approval-to-treat-alzheimers-disease2.https://synapse.zhihuiya.com/blog/药事纵横投稿须知：稿费已上调，欢迎投稿

临床3期临床结果快速通道临床2期上市批准

2025-06-16

·上海和誉生物医药科技有限公司

和誉医药依帕戈替尼完成治疗肝细胞癌的注册性临床试验首例患者给药

2025年6月16日，和誉医药（港交所代码：02256）宣布，其自主研发的高选择性小分子FGFR4抑制剂依帕戈替尼已于近日完成治疗肝细胞癌的注册性临床的首例患者给药。依帕戈替尼于今年5月获国家药品监督管理局药品审评中心（CDE）突破性疗法认定。依帕戈替尼也是首个采用靶向分子生物标志物精准治疗肝细胞癌的药物。绝大多数接受目前标准疗法（包括免疫检查点抑制剂 (ICI) 和多靶点激酶抑制剂 (mTKI)）治疗的晚期 HCC 患者在一年内都会出现疾病进展。此外，约有 30% 的 HCC 患者表现出 FGF19 过表达，这是一种与更具侵袭性的肿瘤生物学特性和更差的预后相关的生物标记物。此项注册性临床研究是一项多中心、随机、双盲、安慰剂对照的临床研究，旨在评估依帕戈替尼联合最佳支持性治疗（BSC）与安慰剂联合BSC在经免疫检查点抑制剂(ICI)和多靶点酪氨酸激酶抑制剂 (mTKI) 治疗的FGF19过表达的晚期或不可切除的肝细胞(HCC)患者中的有效性和安全性。符合入组标准的晚期肝癌患者将以2：1的比例随机接受依帕戈替尼联合最佳支持治疗和安慰剂联合最佳支持治疗。关于依帕戈替尼(Irpagratinib/ABSK-011)依帕戈替尼（ABSK-011）是一种高选择性小分子FGFR4抑制剂，旨在靶向FGF19信号通路的过表达。研究表明，全球约30%的HCC患者存在FGF19过表达。开发针对该信号通路的靶向疗法代表了治疗HCC的一种新颖的创新方法。当前，全球尚无FGFR4抑制剂获批上市，根据弗若斯特沙利文的分析，依帕戈替尼凭借其在竞争格局中的领先地位，有望成为首个治疗FGF19过表达aHCC患者的突破性药物。除单药之外，和誉医药同时在探索联合抗PD-L1抗体阿替利珠单抗（由F. Hoffmann-La Roche Ltd.及罗氏（中国）投资有限公司制造）的II期试验。在2024年ESMO-GI大会上，和誉医药发布了联合用药治疗aHCC患者的最新临床试验数据，其中，依帕戈替尼220mg BID联用阿替利珠单抗队列在既往接受过 ICI 治疗的 FGF19+ HCC 患者中实现了 50% 的客观缓解率 (ORR)。Abbisko Therapeutics Completes First Patient Dosing in Registrational Study of Irpagratinib for HCCJune 16, 2025 — Abbisko Therapeutics (HKEX Code: 02256) today announced that it has completed first patient dosing in a registrational study of irpagratinib, a self-developed and highly-selective small molecule FGFR4 inhibitor, for the treatment of Hepatocellular Carcinoma (HCC). In May, irpagratinib received Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). It is the first therapeutic agent to utilize molecularly defined biomarkers for precision-targeted treatment of HCC.The vast majority of patients with advanced HCC treated with current standard-of-care therapies—including immune checkpoint inhibitors (ICIs) and multi-targeted kinase inhibitors (mTKIs)— experience disease progression within one year. Additionally, approximately 30% of HCC patients exhibit FGF19 overexpression, a biomarker associated with more aggressive tumor biology and poorer prognosis. The registrational study of irpagratinib (ABSK-011-205) is a multi-center, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the efficacy and safety of irpagratinib in combination with Best Supportive Care (BSC) versus placebo in combination with BSC, in patients with advanced or unresectable HCC who exhibit FGF19 overexpression and have previously been treated with ICIs and mTKIs. Eligible patients will be randomized in a 2:1 ratio to receive irpagratinib or placebo.About Irpagratinib (ABSK-011)Irpagratinib is a highly-selective FGFR4 small molecule inhibitor designed to target overexpression of the FGF19 signaling pathway. Several epidemiological studies indicate that approximately 30% of HCC patients worldwide exhibit FGF19 overexpression. Development of targeted therapies against FGFR4 represent an innovative and novel approach to the treatment of HCC.To date, no FGFR4 inhibitor has been granted regulatory approval globally. According to Frost & Sullivan, irpagratinib is expected to become the first breakthrough treatment for the treatment of HCC patients with FGF19 overexpression.In addition to monotherapy, Abbisko Therapeutics is exploring irpagratinib in combination with atezolizumab, an anti-PD-L1 antibody manufactured by F. Hoffmann-La Roche and Roche (China), in a Phase II study. At the previous 2024 ESMO GI Congress, Abbisko presented clinical data demonstrating 220mg irpagratinib BID in combination with atezolizumab achieved a 50% objective response rate (ORR) in FGF19+ HCC patients who had previously received immune checkpoint inhibition therapy.关于和誉和誉医药（香港联交所代码：02256）成立于2016年，是一家立足中国，着眼全球的创新药研发公司。公司的创始人和管理团队拥有多年顶尖跨国药企的研发和管理经验，并参与了多个临床及上市新药的研发。和誉医药专注于肿瘤新药研发，以小分子肿瘤精准治疗和小分子肿瘤免疫治疗药物为核心，着眼病患及医药市场的需求，秉承国际新药开发的理念和标准，致力于开发新颖及高潜力药物靶点的潜在first-in-class或best-in-class创新药物，用于改善中国及全球病人的生活质量。自成立以来，和誉医药已经建立了丰富的创新产品管线，涵盖肿瘤精准治疗领域以及肿瘤免疫治疗领域。更多信息，欢迎访问 www.abbisko.com。

突破性疗法申请上市临床结果临床2期临床1期

100 项与甲磺酸伊马替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D01441	甲磺酸伊马替尼	L01XE01	DB00619

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
加速期费城染色体阳性慢性粒细胞白血病	美国	Shorla Pharma Ltd.	2024-11-22
急变期费城染色体阳性慢性粒细胞白血病	美国	Shorla Pharma Ltd.	2024-11-22
慢性期费城染色体阳性慢性粒细胞白血病	美国	Shorla Pharma Ltd.	2024-11-22
转移性胃肠道间质瘤	欧盟	Accord Healthcare SLU	2013-06-30
转移性胃肠道间质瘤	冰岛	Accord Healthcare SLU	2013-06-30
转移性胃肠道间质瘤	列支敦士登	Accord Healthcare SLU	2013-06-30
转移性胃肠道间质瘤	挪威	Accord Healthcare SLU	2013-06-30
骨髓增生性疾病	欧盟	Teva Pharmaceuticals Europe BV	2013-01-07
骨髓增生性疾病	冰岛	Teva Pharmaceuticals Europe BV	2013-01-07
骨髓增生性疾病	列支敦士登	Teva Pharmaceuticals Europe BV	2013-01-07
骨髓增生性疾病	挪威	Teva Pharmaceuticals Europe BV	2013-01-07
Ph样急性淋巴细胞白血病	欧盟	Teva Pharmaceuticals Europe BV	2013-01-07
Ph样急性淋巴细胞白血病	冰岛	Teva Pharmaceuticals Europe BV	2013-01-07
Ph样急性淋巴细胞白血病	列支敦士登	Teva Pharmaceuticals Europe BV	2013-01-07
Ph样急性淋巴细胞白血病	挪威	Teva Pharmaceuticals Europe BV	2013-01-07
费城染色体阳性白血病	欧盟	Teva Pharmaceuticals Europe BV	2013-01-07
费城染色体阳性白血病	冰岛	Teva Pharmaceuticals Europe BV	2013-01-07
费城染色体阳性白血病	列支敦士登	Teva Pharmaceuticals Europe BV	2013-01-07
费城染色体阳性白血病	挪威	Teva Pharmaceuticals Europe BV	2013-01-07
急性淋巴细胞白血病	美国	Novartis Pharmaceuticals Corp.	2006-10-19

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
混合表型急性白血病	临床3期	美国	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	澳大利亚	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	奥地利	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	比利时	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	加拿大	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	智利	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	捷克	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	芬兰	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	法国	The Children's Oncology Group Foundation, Inc.	2017-08-08
混合表型急性白血病	临床3期	德国	The Children's Oncology Group Foundation, Inc.	2017-08-08

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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


NCT00476190 (CTgov)	临床2期	成人急性淋巴细胞白血病	112	Radiation Therapy+PEG-Asparaginase+Etoposide+doxorubicin+Hydrocortisone Sodium Succinate+6-MP+Methotrexate+Cytarabine+Methylprednisone+Cyclophosphamide+Imatinib+Dexamethasone+E. coli Asparaginase+Vincristine (Pre-amendment Cohort, 2500 IU/m2 q2 Weeks)	廠蓋顧淵廠窪齋築顧繭 = 夢憲鬱齋顧憲鏇廠築鏇窪窪憲鏇鏇範繭餘顧鬱 (積醖觸顧膚膚遞淵衊製, 願獵壓蓋獵鏇鹽餘廠憲 ~ 願獵衊衊繭網觸鏇衊鑰) 更多	-	2025-06-12
				Radiation Therapy+PEG-Asparaginase+Etoposide+Doxorubicin+Hydrocortisone Sodium Succinate+6-MP+Methotrexate+Cytarabine+Methylprednisone+Cyclophosphamide+Imatinib+Dexamethasone+E. coli Asparaginase+Vincristine (Post-amendment Cohort, 2000 IU/m2 q3 Weeks)	廠蓋顧淵廠窪齋築顧繭 = 鏇糧憲衊夢齋襯鏇鑰廠窪窪憲鏇鏇範繭餘顧鬱 (積醖觸顧膚膚遞淵衊製, 壓簾憲淵製選鬱糧窪簾 ~ 繭繭淵製齋醖範築顧築) 更多
IRIS trial (ASCO2025)	N/A	慢性粒细胞性白血病 BCR::ABL1 fusion gene	-	Imatinib	衊糧餘淵鹹觸製夢獵窪(淵獵憲襯鹹夢觸窪膚願) = 廠淵齋構繭鹹蓋遞壓繭願製積願壓夢艱淵鬱醖 (簾窪鬱醖製鏇襯築構製 ) 更多	积极	2025-05-30
The Max Foundation (ASCO2025)	N/A	胃肠道间质瘤辅助 KIT-positive	721	Metastatic/unresectable GIST patients receiving imatinib or imatinib+sunitinib	範選衊襯糧鑰蓋窪構鬱(願簾鹹醖憲觸廠鹽窪衊) = 鬱獵繭壓醖鬱鏇積襯壓窪願襯構艱夢鬱繭願遞 (夢窪鹹夢選觸鬱鬱廠顧, 28.4% ~ 44.2) 更多	积极	2025-05-30
				Adjuvant treatment GIST patients receiving imatinib	範選衊襯糧鑰蓋窪構鬱(願簾鹹醖憲觸廠鹽窪衊) = 繭窪壓艱簾觸範壓夢願窪願襯構艱夢鬱繭願遞 (夢窪鹹夢選觸鬱鬱廠顧, 48.6% ~ 69.5) 更多
ASCO2025	N/A	慢性期慢性髓性白血病	100	Imatinib	窪壓鬱鹹選憲衊構糧鑰(築窪齋膚遞醖獵鬱醖願) = 範淵鹹願壓簾夢遞憲鑰積憲鑰膚餘夢鬱願簾簾 (築糧鑰構選構簾憲夢壓 ) 更多	-	2025-05-30
ASCO2025	N/A	转移性胃肠道间质瘤	234	Imatinib only	製築繭簾壓襯醖繭壓積(膚襯衊積壓衊鬱糧夢憲) = 夢齋糧範鹽繭醖顧簾獵鬱繭鏇窪製醖衊餘廠簾 (鑰壓遞願艱齋願範鹽餘 )	积极	2025-05-30
				imatinib (IM and surgery before IM)	製築繭簾壓襯醖繭壓積(膚襯衊積壓衊鬱糧夢憲) = 遞網窪窪鬱願網顧觸鑰鬱繭鏇窪製醖衊餘廠簾 (鑰壓遞願艱齋願範鹽餘 )
ASCO2025	N/A	慢性期慢性髓性白血病一线 BCR-ABL1	118	Imatinib	遞獵範襯廠積壓範願簾(網膚顧憲顧範願艱觸衊) = 製淵糧遞鏇網艱觸憲襯構獵選鏇壓選願鹹簾衊 (壓鹽淵餘構壓壓簾衊壓 ) 更多	积极	2025-05-30
NCT01991379 (GIST Trial 13-162, ASCO2025)	临床2期	胃肠道间质瘤 ETV1 \| KIT	42	Imatinib 400mg daily + Binimetinib 30mg twice daily	簾糧膚製鑰獵製窪觸鏇(鬱積鏇窪艱簾鑰範壓鬱) = 構顧製廠繭鹽簾憲襯蓋鑰憲鑰選製積窪鬱糧衊 (鬱糧壓選鏇衊鬱衊窪構, 52.9% ~ 82.4%) 更多	积极	2025-05-30
AACR2025	N/A	慢性期慢性髓性白血病	60	imatinib (BMI <18.5)	範壓襯製淵餘憲願積廠(鹹蓋壓積鏇構鏇齋齋憲) = 憲夢壓網膚積獵襯鏇繭製鑰糧壓繭網憲廠簾餘 (襯醖齋醖壓齋觸艱網鏇 ) 更多	-	2025-04-29
NCT02365441 (ALT-GIST, Pubmed \| Br J Cancer)	临床2期	胃肠道间质瘤一线	-	Imatinib	鏇憲窪餘餘壓獵窪構糧(鬱鏇鬱鹽顧構繭觸顧蓋) = No Arm A patients stopped protocol therapy due to unacceptable toxicity, with 12 (30.0%) stopping in Arm B 襯夢齋選廠遞夢衊積夢 (艱繭廠艱衊鏇鬱築顧鏇 ) 更多	不佳	2025-03-25
				Imatinib + Regorafenib
NCT03578367 (ASC4MORE, Pubmed \| J Hematol Oncol)	临床2期	慢性期慢性髓性白血病 \| 慢性期费城染色体阳性慢性粒细胞白血病追加	84	Asciminib 40 mg QD add-on to Imatinib 400 mg QD	鹽構鏇淵製範鏇衊構鏇(遞鑰廠積願範構蓋遞鑰) = 糧鹹艱選範構鏇願窪鬱艱鹹醖襯選簾蓋夢鏇鏇 (憲艱顧鏇淵壓襯獵遞餘 ) 更多	积极	2024-12-18
				Asciminib 60 mg QD add-on to Imatinib 400 mg QD	鹽構鏇淵製範鏇衊構鏇(遞鑰廠積願範構蓋遞鑰) = 鏇鹽選淵窪憲構網願範艱鹹醖襯選簾蓋夢鏇鏇 (憲艱顧鏇淵壓襯獵遞餘 ) 更多