Pilot Study of Asciminib as a Maintenance Treatment Post Cellular Therapies in Adults With Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

This phase I trial tests the safety, side effects and best dose of asciminib as maintenance treatment for adults with Philadelphia chromosome positive acute lymphoblastic leukemia (ALL) who have undergone cellular therapies such as hematopoietic stem cell transplantation (HSCT) or chimeric antigen receptor (CAR) T cell therapy. Maintenance treatment is given to help keep cancer from coming back after it has disappeared following initial therapy. Asciminib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving asciminib may be safe and tolerable as maintenance treatment for adult patients with Philadelphia chromosome positive ALL who have undergone cellular therapies.

开始日期2026-05-01

申办/合作机构

City of Hope National Medical Center

[+1]

NCT07039760

/ Not yet recruiting早期临床1期IIT

An Early Phase 1 Study of Asciminib With or Without Sildenafil for Brain Tumors

Dissemination of medulloblastoma is an independent risk factor of poor prognosis. Dissemination of medulloblastoma at recurrence is nearly universally fatal. ABL1 and 2 have been recently found to mediate the dissemination of medulloblastoma. Genetically inactivating ABL1 and 2 resulted in decreased leptomeningeal medulloblastoma and improved overall survival (OS) in rodent models. Asciminib is an FDA approved for the treatment of chronic myeloid leukemia and is well tolerated, likely due to its specificity for ABL1 and ABL2. Asciminib is a P-glycoprotein (P-gp) substrate and thus may be susceptible to being pumped out of tumor cells and brain endothelial cells. It is unclear if asciminib can enter the central nervous system (CNS) and brain tumors in adequate concentration to have anti-tumor effects.

开始日期2025-12-01

申办/合作机构

Washington University School of Medicine

NCT06409936

/ Recruiting临床2期IIT

Asciminib as Single Agent or in Combination With Nilotinib in the 1st-line Treatment of BCR-ABL1+ Chronic Myeloid Leukemia: a Randomized GIMEMA-GELMC Phase II Study PEARL Study: PotEntial of Asciminib in the eaRly Treatment of CML

A phase 2, interventional, randomized unblinded study will be conducted in newly diagnosed CP CML patients, to investigate the efficacy and the safety of asciminib at a dose of 80 mg QD as single agent (arm A) or 40 mg BID in combination with nilotinib 300 mg BID (arm B).
All patients in both arm A and arm B will be treated for a minimum of 2 years (core phase). If they will have achieved a DMR (MR4), or if it will be in the interest of the patient, the treatment will be continued.
During the consolidation phase (2 years) asciminib will be continued at the same dose in both arms; in the combination arm the nilotinib dose will be reduced to 300 mg daily.
The patients maintaining a stable MR4 up to the end of the fourth year will discontinue the treatment (TFR phase). The rate of TFR at 5 year (1 year after discontinuation) will be evaluated.

开始日期2025-06-01

申办/合作机构

Gruppo Italiano Malattie EMatologiche dell'Adulto

100 项与盐酸阿思尼布相关的临床结果

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100 项与盐酸阿思尼布相关的转化医学

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100 项与盐酸阿思尼布相关的专利（医药）

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327

项与盐酸阿思尼布相关的文献（医药）

2025-12-01·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Population pharmacokinetic modeling of asciminib in support of exposure-response and ethnic sensitivity analyses in patients with chronic myeloid leukemia

Article

作者: Darstein, Christelle ; Kawakita, Yasunori ; Sy, Sherwin K B ; Wu, Shengyuan ; Yoon, Deokyong ; Grosch, Kai ; Yang, Yiqun ; Dasgupta, Kohinoor ; Kapoor, Shruti ; Hoch, Matthias

BACKGROUND:

The original population pharmacokinetics (popPK) model for asciminib in patients with chronic myeloid leukemia in chronic phase (CML-CP) was refined to address drug development needs in support of drug submission, namely, attainment of target drug exposure in specific patient populations, populating individual daily exposures for exposure-response analyses of key efficacy and safety endpoints, confirmation of comparability in exposure between 40 mg b.i.d. and 80 mg q.d., and assessment of ethnic insensitivity.

METHODS:

Participants from two organ dysfunction studies, patients with CML in blast and acute phases and acute lymphoblastic leukemia and patients from a phase III efficacy study in newly diagnosed Ph + CML-CP, and data from a dedicated phase II study in the Chinese patients previously treated with at least two prior tyrosine kinase inhibitors, and a phase IIIb study comparing two dose regimens of asciminib (40 mg b.i.d. and 80 mg q.d.) were included in the revised popPK model. Covariates evaluated were line of therapy, baseline renal and hepatic functions, Chinese or Japanese ethnicity.

RESULTS:

The apparent clearance and steady-state volume of distribution of asciminib were 6.84 L/h and 110 L, respectively, for a typical individual of 70 kg weight and 90 mL/min absolute glomerular filtration rate. Both the 40 mg b.i.d. and 80 mg q.d. resulted in a steady-state daily AUC of 12,600 ng.h/mL, and there was no difference between lines of therapy. Effects of renal or hepatic impairment on clearance were not clinically relevant. Chinese and Japanese exhibited similar PK as that of the global population.

CONCLUSIONS:

The 40 mg b.i.d. and 80 mg q.d. regimens are comparable in their daily exposure, supporting the use of the two dosing regimens in newly diagnosed and previously treated CML-CP patients. The PK of asciminib is insensitive to ethnic differences and no dose adjustment is required for severe renal and hepatic impaired patients.

2025-06-01·Clinical Lymphoma Myeloma & Leukemia

SOHO State of the Art Updates and Next Questions: Combination Therapy in Chronic Myeloid Leukemia in Chronic Phase

Review

作者: Costa, Alessandro ; Breccia, Massimo

Therapeutic strategies for chronic myeloid leukemia (CML) are rapidly evolving, with novel agents emerging to address the limitations of current treatments. Goals of CML management are disease control and achieve a deep and sustained molecular response for a possible successful treatment-free remission (TFR). However, a significant proportion of patients fail to reach adequate molecular response and require sequential therapies. A crucial aspect of treatment resistance lies in the persistence of leukemic stem cells (LSCs), which serve as a reservoir for disease recurrence. Increasing focus is placed on combination strategies to overcome the constraints of TKI monotherapy. Various strategies have been explored, starting with the combination of interferon (IFN) and TKIs. The investigation of alternative administration methods, dosing regimens, or extended treatment durations in clinical trials involving IFN represents potential avenues to address current conflicting results. Additionally, the combination of ATP-competitive TKIs with asciminib has shown encouraging preclinical and clinical results, with further data needed for a comprehensive safety profile. Recently, efforts to inhibit other signaling pathways have been explored but with contrasting results. Despite ongoing advancements, TKIs remain the cornerstone of both current and future combination therapies. Their integration with personalized approaches is crucial to overcome complex biological challenges and ensure long-term, effective and safe treatment for CML patients.

2025-06-01·American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting

Which Is the Best Tyrosine Kinase Inhibitor for Newly Diagnosed Chronic Myelogenous Leukemia?

Review

作者: Hughes, Timothy P. ; Osborn, Michael ; Shanmuganathan, Naranie

The choice of frontline therapy for a patient with chronic phase chronic myeloid leukemia (CP-CML) can have a profound effect on the long-term clinical outcome. Currently, five tyrosine kinase inhibitors (TKIs—imatinib, dasatinib, nilotinib, bosutinib, and asciminib) are available for frontline therapy, but no single TKI is optimal for all patients. EUTOS long-term survival (ELTS) risk score, comorbidities, and treatment-free remission (TFR) priority are the key determinants of frontline TKI selection. Higher ELTS score, low age and comorbidity score, and a high priority for achievement of TFR would all favor the frontline use of a more potent TKI than imatinib. However, no TKI has improved survival compared with imatinib. In children with CP-CML, imatinib, dasatinib, and nilotinib have similar long-term efficacy, with ease of administration and impact of toxicities on quality of life being key considerations. Recent adult trials of reduced-dose dasatinib frontline showed that efficacy may be equivalent to standard-dose dasatinib with a better tolerability and safety profile, but experience is limited in patients with high-risk ELTS scores. The ASC4FIRST trial has confirmed that tolerability and molecular response with asciminib are superior to those with both imatinib and the second-generation (2G)-TKIs. While the overall treatment failure rate was lower with asciminib, the rate of BCR::ABL1 mutations that emerged with asciminib appeared to be higher. The risk of emergent mutations appears to be highly associated with the presence of ASXL1 mutations in the CML cells at diagnosis, but more work is needed to understand the implications of this finding.

272

项与盐酸阿思尼布相关的新闻（医药）

2025-06-30

·氨基观察

自免新靶点，不是一个好选择了？

氨基观察-创新药组原创出品作者 | 武月仅次于肿瘤的自免市场，正迎来一场无声的“靶点焦虑”。过去几年，最显著的进展发生在靶向TNF-α以外的TNF超家族成员的药物中，目前靶向OX40、TL1A等靶点的药物均推进到III期临床阶段。FIC的诱惑固然巨大，但背后的风险同样不可忽略。尤其是，随着成熟靶点逐步突破，给新靶点的突围设定了更高的标准，包括OX40在内几款大热的自免新靶点，也并未带来超预期的结果。曾发现OX40、TSLP等靶点的免疫学家刘勇军，如今也对“追新”持谨慎态度，面对下一代的自免药物开发，他的建议是聚焦成熟靶点的迭代创新，新机制的靶点仍然存在不确定性。这与大药企的选择也不谋而合。大药企缺药，在未来5年，专利悬崖之后市场缺口比较大，但是在未来5~8年里能补上缺口的产品，往往并非是FIC，而是在经过验证的一些致病机制、通路和靶点的基础上，再去做下一代的更好的产品。在颠覆性突破稀缺的时代，是时候要重新审视这场豪赌了吗？/ 01 /靶点很热，道路阻长过去25年，自免疾病治疗领域经历了从广谱抗炎到靶向精准调控的深刻变革。以TNF抑制剂为代表的细胞因子信号调节药物，奠定了免疫治疗创新的基石。近年来，随着白细胞介素（IL）家族抑制剂、JAK/TYK2激酶抑制剂及新靶点的崛起，行业更是加速向前。根据发布在Nature上的“Trends in the drug target landscape for autoimmune diseases”文章统计，自2020年以来，已有4种FIC的自免药物获批上市，其中3款为IL-13、IL-31Rα和IL-36R抑制剂，以及治疗银屑病的首个选择性TYK2抑制剂deucravacitinib。皮肤病领域进展不断的同时，首个TSLP靶向药物tezepelumab获批用于治疗严重哮喘，度普利尤单抗也成功突围慢性阻塞性肺病（COPD）。在研管线方面，最显著的进展发生在靶向TNF-α以外的TNF超家族成员的药物中。2020年，还只有CD40/CD40等少数几个新靶点有III期临床，到了2024年，靶向OX40、TL1A等靶点的药物均推进到III期临床阶段。只是，随着成熟靶点逐步突破，给新靶点的突围设定了更高的标准，包括OX40在内几款大热的自免新靶点，也并未带来超预期的结果。3月8日，安进和协和麒麟共同宣布抗OX40单抗Rocatinlimab，治疗特应性皮炎（AD）的两项III期研究达到主要终点，有望成为全球获批上市的首个OX40单抗。只不过，与去年9月公开的首个III期临床（HORIZON）结果相似，最新的临床结果中，疗效也未能撼动IL-4R及IL-13单抗的领先地位。以HORIZON结果为例，Rocatinlimab和安慰剂组的EASI-75（湿疹面积和严重程度指数评分）响应率分别为32.8%和13.7%，Rocatinlimab和安慰剂组的vIGA-AD 0/1（经验证的研究者总体评估0为清除、1为几乎清除）的响应率为19.3%和6.6%。在市场看来，这样的数据不及预期。Rocatinlimab的vIGA-AD 0/1响应率不仅明显低于其在2021年公布的2期临床数据（30.8%），也低于赛诺菲同类抗体 Amlitelimab二期临床的数据（37%~44.4%）。临床II期到III期数据有所下滑是常见现象，但它仍要面对疗效不及度普利尤单抗的问题。作为非头对头对比，度普利尤单抗的三期临床大概响应率是36%-38%，EASI-75缓解率达到50%左右。尽管近年来中至重度特应性皮炎的生物制剂疗法进展颇多，但目前看，包括OX40L、IL-22R、IL-31R、TSLP和IL-22等靶点的新药暂未显示出更佳疗效。被再生元寄予厚望的IL-33单抗Itepekimab也于5月底带来了一个坏消息：两项COPD三期研究中，一项成功一项失败。此前，赛诺菲和再生元希望将Itepekimab打造成度普利尤的继任者，因为前者更能惠及更广泛的COPD患者群体（非2型炎症患者，约占重度COPD患者的60-70%），曾预测前者的销售额将达35亿美元。然而，最新的临床结果，却让Itepekimab的前景变得扑朔迷离。Truist Securities分析师甚至直言，如果不进行进一步的测试，Itepekimab不太可能获得批准。这些挫折背后，既可能是机制复杂性被低估，或许是因为疾病异质性，也可能二者兼而有之。唯一确定的是，靶点很热，但道路阻长。/ 02 /迭代创新明星靶点的光环，正被残酷的现实稀释。尤其是，当“追新”成本飙升，药企开始挖掘迭代创新的机会与价值。新一代自免大分子产品虽然诞生不少商业神话，但是这个领域仍存在着大量未被满足的临床需求。仍以AD为例，度普利尤单抗的吸金力度之大，让后来者们蠢蠢欲动。一来，这一市场患者规模庞大；二来，由于致病机制尚未完全清晰，因此无法治愈，患者需要长期用药。最重要的是，现有药物普遍都有较大的副作用，或是依从性不高，如度普利尤单抗需要每两周给药一次，许多AD患者会经历“针头疲劳”，尤其是儿科患者经常害怕针头。这使得，市场对特应性皮炎的治疗存在极大的临床未满足需求。强生的一则调研数据显示：约有50-70%适用于最新疗法的银屑病/IBD患者并没有在使用那些最新生物制剂，其中约30%的调查样本给出的理由是注射给药方式问题。另外，约75%正在使用注射类药物的愿意切换到口服药物，当然，前提是安全性和疗效类似。目前看，迭代创新正沿三条路径突进。首先是延长半衰期，这是决定生物制剂给药周期的核心。LS和YTE突变是半衰期延长方面最常见的技术平台，其中YTE技术已经在多个自免项目中得到验证，半衰期均可以达到60天以上。以Apogee为例，其利用YTE突变优化抗体结构，使药物半衰期和给药周期大幅度延长，提高患者的用药依从性。其核心管线主打的都是半衰期延长，且围绕自免领域的热门靶点做了多重布局，进展最快的APG777，意在挑战甚至超越度普利尤单抗，此前公司一期临床数据显示APG777的人类半衰期约为77天，这能够支持其每年给药的潜力。另外，GSK的下一代IL-5抗体Depemokimab已经提交上市申请，作为超长效生物制剂，其给药周期则达到了6个月。根据EvaluatePharma预测，其2023年销售额将突破10亿美元。其次，给药方式也影响着药物的依从性，从静脉注射迭代到皮下注射，迭代到注射笔，目前口服药物则成为新的趋势。3月10日，强生和Protagonist公布了IL-23R口服环肽抑制剂JNJ-2113，治疗溃疡性结肠炎2b期临床数据，研究达到临床缓解主要终点，有望改变溃疡性结肠炎的治疗格局。艾伯维则在乌帕替尼缓释片基础上，新推出了口服液剂型。由于缓释片需要整片吞咽，这对于吞咽困难的儿科患者来说挑战很大，而口服液剂型的更新，无疑给这部分有用药需求的患者，带来了依从性的提升。这或许也会成为其新的卖点。最后则是多靶点协同破局，如同时靶向IL-17和TNF，或开发双激酶抑制剂（JAK1, 2 or 3 with TYK2, SYK or TEC）进一步扩大治疗选择。辉瑞甚至在自免领域已经卷到了三抗，其开发的IL4/IL-13/IL-33三抗药物PF-07264660，已经启动了特应性皮炎的二期临床。而自去年以来，在自免领域按下加速键的TCE，也早已从双抗卷到了三抗。/ 03 /大药企越来越务实事实上，这种迭代浪潮不仅席卷自免领域，肿瘤、CNS同步正在进行；也不只是大分子，小分子领域这种迭代创新，也在如火如荼地展开。本质上，这是因为大药企在变得越来越务实，随着规模越来越大，迫使它们过度聚焦于临床后期项目，然后由于预算平衡压力，过早终止早期项目或降低其优先级。这种战略选择也体现在它们的BD偏好中，进而影响着整个行业的风向。以诺华为例，去年10月，其新型慢性粒细胞白血病治疗药物Scemblix获得FDA批准上市。此前，在III期临床试验中，其表示Scemblix数据优于其第一代重磅药物伊马替尼，而且也优于诺华、BMS和辉瑞的第二代药物。诺华在慢性粒细胞白血病领域已有数十年的积淀，明星产品有第一代酪氨酸激酶抑制剂伊马替尼和第二代药物厄洛替尼。诺华肿瘤学全球主管Jeff Legos在接受采访时曾表示：许多慢性粒细胞白血病患者仍没有完全从TKI抑制剂中获益，这种疾病尚有很多未被满足的临床需求。在他看来，尽管从伊马替尼到厄洛替尼时代都有创新，但不幸的是，大约50%的患者未能达到他们的主要分子反应治疗目标。由于安全性和耐受性方面的问题，大约25%的患者实际上在第一年就更换了TKI。因此，在明确的临床需求下，大药企都变得比较实际，比较注重“当下”。在专利悬崖危机面前，大药企普遍缺药，但是在未来几年里能补上缺口的产品，往往并非FIC，而是在经过验证的一些致病机制、通路和靶点的基础上，迭代而来的下一代产品。事实上，从近两年以ASCO为代表的国际顶尖大会的情况也能看出来，并未出现太多惊艳全场的新研究或新数据。无论自免还是肿瘤，大药企更偏向改进经过验证的靶点、通路，通过下一代疗法来寻求突破。某种程度上，这也是现实的桎梏。此前，免疫治疗和ADC等“革命性”药物，把肿瘤治疗水平抬到了一个新高度，而在当前制药工业基础研究的水平之上，很难有新的颠覆性疗法能够突破天花板。那些现存的、被寄予厚望的新技术们，或多或少又因为各种现实的原因，还没迎来自己的高光时刻。当然，平静的水面下也藏有暗涌，孕育着变局。寻找新靶点这条路风险更高，回报更大，总有人愿意去做从零到一的事情。PS：欢迎扫描下方二维码，添加氨基君微信号交流。

临床3期免疫疗法上市批准临床结果引进/卖出

2025-06-28

·医脉通

CAH 2025白血病及PNH会场 | 聚焦诊疗革新：靶向突破与全程管理并进

2025年6月20日至22日，中国医师协会第十九届血液科医师年会暨2025年中国血液病大会圆满落幕。本文聚焦白血病及阵发性睡眠性血红蛋白尿症（PNH）会场精华，将会议内容整理如下。在中国工程院、北京大学人民医院黄晓军教授、华中科技大学同济医学院附属协和医院胡豫教授致辞后，学术分享正式开始。CML篇靶向突破与全程管理在张晓辉教授、纪春岩教授、范磊教授、陈苏宁教授、杜新教授、朱焕玲教授的主持下，江倩教授、黄健教授、刘兵城教授、朱雨教授、段明辉教授、黎纬明教授围绕慢性髓系白血病（CML）的中国诊疗探索、一线诊疗前沿进展、治疗药物、停药策略以及全程管理等热点议题进行了专题分享。江倩教授和黄建教授指出，酪氨酸激酶抑制剂（TKI）是CML治疗的核心，但传统TKI存在脱靶效应高、深度分子学缓解（DMR）率低、耐受性差、生活质量差等未满足需求。近年来，创新药物的问世进一步推动了CML治疗目标的发展，功能性治愈（TFR）成为大多数CML患者的治疗目标。朱雨教授介绍道，CML的进程由BCR-ABL1激酶依赖途径和非激酶依赖途径共同驱动。传统TKI无法阻断非激酶依赖途径，易引起脱靶效应。而特异性靶向ABL1肉豆蔻酰口袋（STAMP）药物阿思尼布可双重阻断两种致病途径，特异性高，脱靶效应低，疗效和耐受性更优。刘兵城教授深入解读ASC4FIRST研究指出，阿思尼布一线治疗96周DMR率翻倍，可帮助CML患者更早、更快实现TFR。段明辉教授表示“每个CML患者都值得TFR机会”，传统TKI整体停药成功率不足25%，而阿思尼布治疗融合基因下降更快、DMR率更高、依从性更优，有望突破TFR瓶颈。面对CML管理中面临的依从性低、检测不规范、患者心理问题等挑战，黎纬明教授分享了CML慢病化管理“湖北模式”，该模式依托“慢粒学院”数字平台，构建慢病管理体系，为全球CML管理提供可复制的中国方案。左右滑动查看更多在张苏江教授和杜欣教授的主持下，朱尊民教授、罗建民教授、付丹晖教授、许娜教授、姜中兴教授、张龑莉教授、翁建宇教授、曾庆曙教授、王一教授、杨云帆教授、文钦教授共同探讨了“CML治疗方案的变革”。对话专家认为，新型靶向药物的可及将进一步推动CML治疗目标从追求长期生存转向实现DMR和TFR。期待在创新机制药物阿思尼布更快、更深、更持久分子学缓解，更好耐受性的加持下，CML患者的生活质量得到显著改善，更多患者实现功能性治愈。左右滑动查看更多PNH篇中外共话PNH诊疗新进展与新模式在张连生教授的主持下，石红霞教授、施均教授、王顺清教授、佟红艳教授、王伟教授和洪梅教授共话“中国PNH诊疗目标的变革”。对话专家认为，中国临床医生需提升对PNH的疾病认知和早期筛查重视度。C5补体抑制剂未满足临床需求，新型补体抑制剂（如补体B因子抑制剂）将推动PNH治疗目标从“缓解症状”到“改善血管内溶血”再到“完全缓解”不断升级。在付蓉教授的主持下，Austin Gladston Kulasekararaj教授深度剖析“PNH前沿诊疗发展趋势”。Austin教授深度解读补体B因子抑制剂APPLUSE等临床研究数据，强调PNH治疗目标不断升级，并分享了英国PNH国家服务中心采用集中化管理模式，剖析未来药物研发趋势。在金阿荣教授的主持下，付蓉教授分享了“从指南更新看中国PNH诊疗的发展趋势”。付蓉教授认为中国PNH诊疗呈现诊断敏准化、治疗规范化、管理慢病化的发展趋势；指南确立补体抑制剂的一线地位；PNH诊疗倡导多学科协作，制定随访和监测模式，加强患者教育。在张凤奎教授的主持下，何广胜教授分享了“PNH的疗效评估与全程管理规范化”。何广胜教授认为新型补体抑制剂（如补体B因子抑制剂）可实现PNH完全缓解，推动治疗目标升级；末端补体抑制剂疗效不佳时，需分析残留血管内溶血（IVH）、血管外溶血（EVH）、突破性溶血（BTH）等原因，转换使用补体B因子抑制剂是有效策略。在张凤奎教授的主持下，韩冰教授分享了“PNH克隆筛查及补体抑制剂治疗监测中国专家共识”。韩冰教授认为早期筛查十分必要，明确5类需筛查人群：不明原因贫血/血栓、骨髓衰竭、抗人球蛋白试验阴性的溶血性贫血等，且治疗监测需动态评估疗效指标。审批码FAP0049221-104344，有效期为2025-06-27至2026-06-26，资料过期，视同作废撰写：Vera、Vitalis审校：Vera、Vitalis排版：Atai执行：Atai本平台旨在为医疗卫生专业人士传递更多医学信息。本平台发布的内容，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。如该等信息被用于了解医学信息以外的目的，本平台不承担相关责任。本平台对发布的内容，并不代表同意其描述和观点。若涉及版权问题，烦请权利人与我们联系，我们将尽快处理。

ASCO会议

2025-06-22

·药明康德

3个月一次，降低发作率95%的单抗疗法；使超80%肿瘤患者获完全缓解的靶向疗法…… | 一周盘点

本期看点1. 潜在“first-in-class”突变型钙网蛋白（mutCALR）靶向单克隆抗体早期临床数据积极，超80%骨髓增殖性肿瘤（MPN）患者达完全缓解（CR）。2. 血浆激肽释放酶单抗抑制剂navenibart用于治疗遗传性血管性水肿，在一项早期临床试验中使患者每月的发作率大幅降低，平均降幅达92%，降幅中位数达97%。INCA033989：公布首个临床研究数据Incyte近日公布其创新疗法INCA033989的首个临床研究数据，该药物为一款潜在“first-in-class”、mutCALR靶向单克隆抗体，用于治疗携带此突变的骨髓增殖性肿瘤患者。数据显示，在接受INCA033989治疗的高危原发性血小板增多症（ET）的患者中，各剂量组均实现了快速且持久的血小板计数正常化，高剂量组（>400 mg）患者应答更为显著。其中，86%的患者达到完全或部分血液学缓解，82%达CR；89%（34/38）的可评估患者mutCALR等位基因频率（VAF）降低，且21%（8/38）患者在仅接受3个治疗周期后即实现超过50%的VAF下降。单细胞DNA测序探索性研究进一步显示，INCA033989能够选择性靶向并减少携带mutCALR的造血干祖细胞（CD34阳性）及髓红系细胞，同时促进正常（野生型CALR）细胞的恢复，支持健康造血功能的重建。骨髓活检结果亦验证了这一机制，mutCALR阳性巨核细胞减少、阴性巨核细胞增加。安全性方面，INCA033989在24至2500 mg的全部剂量范围内耐受性良好，无剂量限制性毒性（DLT），仅1例患者因治疗伴发不良事件（TEAEs）停药，另有1例发生剂量调整。最常见的TEAEs为疲劳（26.5%）和上呼吸道感染（20.4%），均为1-2级。Navenibart（STAR-0215）：公布1b/2期临床试验长期开放标签试验的数据Astria Therapeutics公司公布了其血浆激肽释放酶单抗抑制剂navenibart治疗遗传性血管性水肿的1b/2期临床试验长期开放标签试验的数据。此次公布的结果显示，患者每月的发作率大幅降低，平均降幅达92%，降幅中位数达97%。频率为每3个月给药一次组别患者的平均每月发作率降低了95%，而每6个月给药一次组别患者的平均降幅为86%。安全性方面，navenibart总体耐受性良好，无严重的治疗伴发不良事件，无停药现象。ELVN-001：公布1期临床试验的新数据Enliven Therapeutics公司公布其小分子激酶抑制剂ELVN-001治疗慢性粒细胞白血病（CML）患者的1期临床试验的新数据。ELVN-001是一种强效、高选择性、潜在“best-in-class”的小分子激酶抑制剂，专门针对CML患者的致癌驱动因子BCR-ABL融合蛋白。ELVN-001还具有针对耐药性最强的BCR-ABL1耐药突变体T315I和其他已知耐药突变体的活性。截至2025年4月28日的数据，在53例可评估的患者中，47%（25/53）在24周时达到主要分子学缓解（MMR），其中最后一线治疗时对酪氨酸激酶抑制剂（TKI）耐药的患者中有41%（14/34）在24周时处于MMR，曾接受asciminib或ponatinib治疗的患者中也有35%（12/34）在24周时处于MMR。所有达到或维持MMR的患者在数据截止时仍保持缓解。在该临床试验中所纳入的患者群体此前接受过大量治疗的情况下，ELVN-001与已获批的针对BCR-ABL融合蛋白的TKI在1期试验中所观察到的MMR相比，结果依然更好。安全性方面，ELVN-001在所有剂量水平上继续表现出良好的安全性和耐受性。BMF-500：公布1期临床试验的初步数据Biomea Fusion公司公布了其共价FLT3抑制剂BMF-500治疗复发/难治性急性白血病的初步临床数据。BMF-500是一种新型、口服可利用的、高效、具选择性的FLT3共价小分子抑制剂。此前的研究表明，BMF-500潜在的脱靶风险较小，对活化的FLT3突变（包括FLT3-ITD和各种酪氨酸激酶结构域突变）具有皮摩尔级的亲和力。在携带FLT3-ITD突变的急性髓系白血病（AML）小鼠模型中，BMF-500使小鼠的肿瘤完全消退，且不需要持续暴露即可维持疗效。BMF-500在治疗FLT3突变的复发/难治性急性白血病患者中表现出令人鼓舞的初步疗效。在可评估的11例FLT3突变患者中，9人出现骨髓原始细胞下降，5人降幅超过50%。同时，在FLT3野生型患者中也观察到疾病控制效果。所有接受治疗的FLT3突变患者（n=18）的中位总生存期（mOS）为3.8个月（无CYP3A4抑制剂）和3.5个月（有CYP3A4抑制剂）。该数据优于历史对照数据，接受过gilteritinib和venetoclax治疗失败后的复发/难治性FLT3突变AML患者的历史mOS仅为2.1个月。BMF-500在各个剂量水平上总体耐受良好，未报告剂量限制性毒性、QT间期延长或因治疗相关不良事件导致的停药。Misetionamide（GP-2250）：公布1期临床试验的中期数据Panavance Therapeutics公司公布了其小分子疗法misetionamide（GP-2250）联用吉西他滨治疗晚期不可切除或转移性胰腺导管腺癌患者1期临床试验的中期数据。Misetionamide具有独特的作用机制，可抑制两种致癌转录因子c-MYC和NFκB。此次公布的结果显示，与单独使用吉西他滨的历史结果相比，接受misetionamide联用吉西他滨治疗患者的确认的缓解率、疾病稳定率和无进展生存期更优，约42%的患者达到部分缓解或疾病稳定。此外，misetionamide联用吉西他滨的安全性良好，与单独使用吉西他滨的预期毒性相比，没有明显的新毒性或加剧的毒性。参考资料（可上下滑动查看）[1] Enliven Therapeutics Announces Updated Positive Data from Phase 1 Clinical Trial of ELVN-001 in CML at EHA 2025 Congress. Retrieved June 20, 2025, from https://www.prnewswire.com/news-releases/enliven-therapeutics-announces-updated-positive-data-from-phase-1-clinical-trial-of-elvn-001-in-cml-at-eha-2025-congress-302481050.html[2] Beam Therapeutics Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Supporting Differentiated Profile in Sickle Cell Disease (SCD) at European Hematology Association (EHA) 2025 Congress. Retrieved June 20, 2025, from https://www.globenewswire.com/news-release/2025/06/13/3098931/0/en/Beam-Therapeutics-Announces-New-Data-from-BEACON-Phase-1-2-Clinical-Trial-of-BEAM-101-Supporting-Differentiated-Profile-in-Sickle-Cell-Disease-SCD-at-European-Hematology-Associatio.html[3] U.S. Food & Drug Administration (FDA) Approves Investigational New Drug (IND) Application for Myrio's Lead Product (PHOX2B PC-CAR T) for the Treatment of Neuroblastoma. Retrieved June 20, 2025, from U.S. Food & Drug Administration (FDA) Approves Investigational New Drug (IND) Application for Myrio's Lead Product (PHOX2B PC-CAR T) for the Treatment of Neuroblastoma[4] Intellia Therapeutics Announces Positive Three-Year Data from Phase 1 Trial of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema (HAE) at the European Academy of Allergy and Clinical Immunology Congress. Retrieved June 20, 2025, from https://www.globenewswire.com/news-release/2025/06/15/3099489/0/en/Intellia-Therapeutics-Announces-Positive-Three-Year-Data-from-Phase-1-Trial-of-Lonvoguran-Ziclumeran-lonvo-z-in-Patients-with-Hereditary-Angioedema-HAE-at-the-European-Academy-of-A.html[5] Vial Initiating Phase 1 Healthy Volunteer Trial of VIAL-TL1A-HLE, a Novel Subcutaneous Half-Life Extended Anti-TL1A Antibody for the Treatment of IBD and Other Inflammatory and Fibrotic Diseases. Retrieved June 20, 2025, from https://vial.com/updates/pr/vial-initiating-phase-1-healthy-volunteer-trial-of-vial-tl1a-hle/?[6] Lyell Immunopharma Announces Positive New Clinical Data Demonstrating High Rates of Durable Complete Responses from the Phase 1/2 Trial of LYL314 for the Treatment of Aggressive Large B-cell Lymphoma. Retrieved June 20, 2025, from https://www.globenewswire.com/news-release/2025/06/17/3100483/0/en/Lyell-Immunopharma-Announces-Positive-New-Clinical-Data-Demonstrating-High-Rates-of-Durable-Complete-Responses-from-the-Phase-1-2-Trial-of-LYL314-for-the-Treatment-of-Aggressive-La.html[7] Azitra Reports Promising Safety Data from Phase 1b Trial in Netherton Syndrome. Retrieved June 20, 2025, from https://www.prnewswire.com/news-releases/azitra-reports-promising-safety-data-from-phase-1b-trial-in-netherton-syndrome-302483136.html[8] 882-P - Eloralintide, a Selective, Long-Acting Amylin Receptor Agonist for Obesity—Phase 1 Proof of Concept. Retrieved June 16, 2025 from https://eppro02.ativ.me/src/EventPilot/php/express/web/planner.php?id=ADA25[9] Positive Late-Breaking Data for Incyte’s First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025. Retrieved June 16, 2025 from https://investor.incyte.com/news-releases/news-release-details/positive-late-breaking-data-incytes-first-class-mutcalr-targeted[10] Oncoinvent Announces Positive Final Data from Phase 1/2a Trial of Radspherin® in Patients with Colorectal Peritoneal Metastases. Retrieved June 16, 2025 from https://www.prnewswire.com/news-releases/oncoinvent-announces-positive-final-data-from-phase-12a-trial-of-radspherin-in-patients-with-colorectal-peritoneal-metastases-302484870.html[11] Zealand Pharma announces positive topline results from 28-week Phase 1b trial with GLP-1/GLP-2 receptor dual agonist dapiglutide. Retrieved June 16, 2025 from https://www.globenewswire.com/news-release/2025/06/18/3101731/0/en/Zealand-Pharma-announces-positive-topline-results-from-28-week-Phase-1b-trial-with-GLP-1-GLP-2-receptor-dual-agonist-dapiglutide.html[12] Spyre Therapeutics Announces Positive Interim Phase 1 Results for Two Next-Generation TL1A Antibody Programs, and Provides Clinical Development Updates Expected to Deliver 9 Phase 2 Readouts. Retrieved June 20, 2025 from https://www.prnewswire.com/news-releases/spyre-therapeutics-announces-positive-interim-phase-1-results-for-two-next-generation-tl1a-antibody-programs-and-provides-clinical-development-updates-expected-to-deliver-9-phase-2-readouts-302483628.html[13] Centessa Pharmaceuticals Announces Clearance of Investigational New Drug Application (IND) for ORX142, a Novel Orexin Receptor 2 (OX2R) Agonist; Clinical Data in Acutely Sleep-Deprived Healthy Volunteers Planned for this Year. Retrieved June 20, 2025 from https://investors.centessa.com/news-releases/news-release-details/centessa-pharmaceuticals-announces-clearance-investigational-new[14] Eolo Pharma Publishes First-in-Human Study in Nature Metabolism on Novel Obesity Drug Activating Energy-Burning Pathway. Retrieved June 20, 2025 from https://www.globenewswire.com/de/news-release/2025/06/16/3099684/0/en/Eolo-Pharma-Publishes-First-in-Human-Study-in-Nature-Metabolism-on-Novel-Obesity-Drug-Activating-Energy-Burning-Pathway.html[15] Debiopharm’s ADC Research Gains Momentum With Launch of First-in-human Trial Assessing Debio 1562M in Acute Myeloid Leukemia Patients. Retrieved June 20, 2025 from https://www.businesswire.com/news/home/20250616081884/en/Debiopharms-ADC-Research-Gains-Momentum-With-Launch-of-First-in-human-Trial-Assessing-Debio-1562M-in-Acute-Myeloid-Leukemia-Patients[16] Biomea Fusion Presents Updated Preliminary Clinical Data for Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory Acute Leukemia at EHA 2025. Retrieved June 20, 2025 from https://investors.biomeafusion.com/news-releases/news-release-details/biomea-fusion-presents-updated-preliminary-clinical-data#:~:text=The%20presentation%20by%20Dr.%20Farhad%20Ravandi%20of%20The,inhibitor%2C%20in%20heavily%20pretreated%20patients%20with%20R%2FR%20AL.[17] Panavance Therapeutics Announces Promising Misetionamide (GP-2250) Phase 1 Clinical Trial Interim Findings in Pancreatic Cancer at BIO 2025. Retrieved June 20, 2025, from https://panavance.com/press/panavance-therapeutics-announces-promising-misetionamide-gp-2250-phase-1-clinical-trial-interim-findings-in-pancreatic-cancer-at-bio-2025/[18] Astria Therapeutics Announces Positive Initial Results from the ALPHA-SOLAR Long-Term Open-Label Trial of Navenibart in Hereditary Angioedema Patients at the European Academy of Allergy and Clinical Immunology Annual Congress. Retrieved June 20, 2025, from https://ir.astriatx.com/news-releases/news-release-details/astria-therapeutics-announces-positive-initial-results-alpha[19] Johnson & Johnson's dual-targeting CAR T-cell therapy shows encouraging first results in large B-cell lymphoma. Retrieved June 20, 2025, from https://www.prnewswire.com/news-releases/johnson--johnsons-dual-targeting-car-t-cell-therapy-shows-encouraging-first-results-in-large-b-cell-lymphoma-302480701.html[20] Galapagos Presented New Data at ICML 2025 From Cohort 3 of ATALANTA-1 in Relapsed/Refractory Indolent NHL Patients, Demonstrating High Complete Response and MRD Negativity Rates With CAR-T Candidate GLPG5101. Retrieved June 20, 2025, from https://www.globenewswire.com/news-release/2025/06/18/3101896/0/en/Galapagos-Presented-New-Data-at-ICML-2025-From-Cohort-3-of-ATALANTA-1-in-Relapsed-Refractory-Indolent-NHL-Patients-Demonstrating-High-Complete-Response-and-MRD-Negativity-Rates-Wit.html免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新2

临床结果临床2期

100 项与盐酸阿思尼布相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11403 D11404	-	-	DB12597

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
慢性期费城染色体阳性慢性粒细胞白血病	美国	Novartis Pharmaceuticals Corp.	2024-10-29
T315I 突变的慢性期费城染色体阳性慢性粒细胞白血病	美国	Novartis Pharmaceuticals Corp.	2024-10-29
慢性粒细胞性白血病	日本	Novartis Pharmaceuticals KK	2022-03-28
费城染色体阳性慢性粒细胞白血病	美国	Novartis Pharmaceuticals Corp.	2021-10-29

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
费城染色体阳性急性淋巴细胞白血病	临床3期	美国	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	日本	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	阿根廷	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	奥地利	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	巴西	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	保加利亚	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	加拿大	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	捷克	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	丹麦	Novartis Pharma AG	2022-08-30
费城染色体阳性急性淋巴细胞白血病	临床3期	法国	Novartis Pharma AG	2022-08-30

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临床结果

适应症

分期

评价

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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


NCT05384587 (ASC2ESCALATE, ASCO2025)	临床2期	慢性期慢性髓性白血病 BCR::ABL1	101	Asciminib 80 mg once daily	餘淵繭繭襯鏇襯製鏇夢(膚構觸獵觸鹹鏇憲繭構) = AEs led to dose adjustment/interruption in 27 pts (26.7%). AEs led to discontinuation in 4 pts; 1 of these AEs occurred >30 d after last ASC dose. No arterial-occlusive events or on-Tx deaths occurred. 觸齋艱範鏇蓋糧積繭窪 (範糧憲選鬱齋網蓋鏇鹹 )	积极	2025-05-30
NCT03595917 (Phase I study, ASCO2025)	临床1期	ABL1 T315I \| p190 \| p210 ... 更多	15	Asciminib 80 mg/d	獵淵顧艱鹹築積築餘簾(醖淵齋膚構選範廠觸積) = 鹽憲選遞選鹽鹹醖廠膚觸簾繭繭願衊願鹽鹹餘 (獵鏇遞網餘繭觸繭獵夢 ) 更多	积极	2025-05-30
				Dasatinib 140 mg/d	鹽積淵觸獵夢範鹽窪窪(齋壓鹹鏇願觸簾襯廠鏇) = 製憲鑰壓遞夢鹹遞觸膚積襯衊鬱衊範艱醖遞糧 (襯製夢鏇遞遞蓋鑰膚餘 )
NCT04925479 (ASC4Kids, ASCO2025)	临床1/2期	慢性期慢性髓性白血病 T315I mutation	19	Pediatric Formulation of Asciminib 1.3 mg/kg BID	顧遞構夢願淵獵構廠醖(鏇簾觸廠鏇簾憲窪膚選) = 18 pts experienced adverse events (AEs; any grade); 2 had Grade ≥3 AEs 衊構範築壓願鹽鏇鑰襯 (淵膚簾鹽膚餘簾餘觸醖 ) 更多	积极	2025-05-30
NCT05456191 (ASC4START, ASCO2025)	临床3期	慢性期慢性髓性白血病	-	Asciminib 80 mg	鏇鏇範繭鏇獵積廠構憲(衊鹹獵蓋鏇築積醖顧積): cause-specific hazard ratio = 0.45 (95% CI, 0.25 ~ 0.81) 更多	积极	2025-05-30
				Nilotinib 300 mg
NCT05456191 (ASC4START, EHA2025)	临床3期	慢性期慢性髓性白血病	568	ASC 80 mg once daily	積糧繭範襯艱選選遞選(網鏇齋夢簾選製簾築鹹): cause-specific hazard ratio = 0.45 (95% CI, 0.25 ~ 0.81) 更多	积极	2025-05-22
				NIL 300 mg twice daily
NCT04971226 (ASC4FIRST, EHA2025)	临床3期	费城染色体阳性慢性粒细胞白血病	405	Asciminib	願齋蓋顧糧蓋夢鏇壓憲(夢壓繭糧蓋願選糧顧願) = 壓膚選蓋衊餘鬱顧範範願壓網獵齋鑰範範願鏇 (襯夢製廠鏇憲觸蓋鬱範 ) 更多	积极	2025-05-14
				Investigator-Selected Tyrosine Kinase Inhibitors	願齋蓋顧糧蓋夢鏇壓憲(夢壓繭糧蓋願選糧顧願) = 選範齋壓鹽鏇壓選網網願壓網獵齋鑰範範願鏇 (襯夢製廠鏇憲觸蓋鬱範 ) 更多
NCT05143840 (HJKC3, EHA2025)	临床2期	慢性粒细胞性白血病	50	Asciminib 40 mg	齋膚鏇鏇齋鬱衊鑰窪鏇(築築淵網製觸蓋鏇繭願) = 網觸鏇遞觸願願鹹壓襯鑰鹽壓鹹憲壓顧繭憲齋 (壓衊艱淵窪淵餘廠衊選 ) 更多	积极	2025-05-14
				Asciminib 80 mg	鹹觸壓廠鏇鏇築鏇廠範(構餘窪醖蓋廠廠蓋網鹹) = 鏇壓壓築鏇顧選餘餘製獵夢鹽鹹餘膚壓選廠築 (鏇夢積範壓構簾範鹹獵 ) 更多
PS1594 (EHA2025)	N/A	慢性粒细胞性白血病 T315I mutation	69	Asciminib 200 mg BID	構網膚願艱淵鑰網遞網(廠蓋顧窪衊獵窪繭選鏇) = 襯獵積範範艱鹹網蓋鹽餘積餘齋鏇積選顧蓋夢 (膚淵選願構願淵製鬱繭 ) 更多	-	2025-05-14
				Asciminib 40 mg BID	遞淵積鏇繭鑰壓夢憲膚(積範觸願淵齋壓憲製選) = 製顧廠遞齋鹹築繭鏇積糧願壓鹽鹹蓋襯鹽糧網 (願鏇積鏇鹽壓壓醖觸窪 ) 更多
PB2718 (EHA2025)	N/A	慢性期慢性髓性白血病 BCR-ABL1 fusion gene	200	Non-prior ponatinib-treated (Non-PPT) CML patients	鏇糧鹹窪糧顧憲繭積觸(鹽鹹鏇窪製範構窪糧鬱) = 淵構願襯範窪範壓壓獵餘遞鏇夢憲製鑰鏇淵淵 (願憲顧觸選遞淵鏇鏇鹹 ) 更多	积极	2025-05-14
				Prior ponatinib-treated (PPT) CML patients	鏇糧鹹窪糧顧憲繭積觸(鹽鹹鏇窪製範構窪糧鬱) = 齋鑰壓鏇鬱醖構鏇製顧餘遞鏇夢憲製鑰鏇淵淵 (願憲顧觸選遞淵鏇鏇鹹 ) 更多
PF594 (EHA2025)	N/A	慢性期慢性髓性白血病 BCR::ABL1 \| t315i	36	ASCIMINIB	鏇網淵構觸築鑰築鬱蓋(壓獵鬱鑰製選齋襯壓構) = 壓醖膚糧餘鹽餘衊餘夢鑰廠襯壓顧糧衊簾廠糧 (蓋窪壓鑰鏇餘築獵壓夢 ) 更多	积极	2025-05-14
				Placebo	積鏇夢膚簾積遞觸膚壓(壓製積願壓襯壓壓鑰遞) = 夢蓋顧衊鹹糧積艱製積鑰蓋製顧鹹構鑰範積壓 (窪夢蓋艱願膚簾構獵遞 )