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

NCT07250087

/ Recruiting临床1期IIT

Phase 1 Study of Asciminib Maintenance Therapy Following Allogeneic Stem Cell Transplant or Chimeric Antigen Receptor T Cell Therapy (CAR T) to Prevent Relapse in Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

The purpose of the study is to see if a study drug called asciminib is safe and okay for people to take after they've had treatment for a type of blood cancer called Philadelphia Chromosome
Positive Acute Lymphoblastic Leukemia (Ph+ B-ALL). We're looking at two groups of adults:
one group had an Allogeneic Stem Cell Transplant (alloHCT) cohort A, and the other group had chimeric antigen receptor T cell (CAR T) therapy, cohort B. We also want to figure out what the best dose of asciminib is to use moving forward in future studies.

开始日期2025-11-01

申办/合作机构

H. Lee Moffitt Cancer Center & Research Institute, Inc.

[+1]

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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328

项与盐酸阿思尼布相关的文献（医药）

2025-12-31·Hematology

Treatment of chronic myeloid leukemia chronic phase patients in third-line setting and beyond: recommendations from a Belgian expert panel in 2025

Review

作者: Salaroli, Adriano ; Vandenberghe, Peter ; Vertenoeil, Gaëlle ; Havelange, Violaine ; Mazure, Dominiek

INTRODUCTION:

Tyrosine kinase inhibitors (TKIs) have revolutionized the therapy of chronic myeloid leukemia (CML) in chronic phase, opening up the perspective of a normal life expectancy for most patients and of treatment-free remission for some of them. However, intolerance or resistance may necessitate treatment modifications.

METHODS:

A panel of five Belgian experts reviewed the treatment options in Belgium in 2025 for patients with chronic phase CML with resistance or intolerance to at least two lines of therapy.

DISCUSSION:

In contrast to the early years of TKI therapy, several options are available as third-line therapy or beyond for CML patients resistant or intolerant to at least two prior TKIs. These include the third generation TKI ponatinib, the allosteric BCR::ABL1 inhibitor asciminib, allogeneic hematopoietic stem cell transplantation, continuation of ongoing second-line TKI therapy with or without dose modification, or switching to alternative second generation TKIs. Careful consideration of the potency and safety profiles of these options, the clinical context, and treatment goal is needed. Ponatinib is currently reimbursed in Belgium for patients with CML showing resistance or intolerance to at least two prior TKIs, or with the BCR::ABL1 T315I mutation, regardless of the number of previous TKIs. Asciminib is currently reimbursed for patients with resistance or intolerance to at least two prior TKIs, excluding those with the BCR::ABL1 T315I mutation. This review provides guidance to weigh available safety and efficacy data in a setting of resistance or intolerance to second-line therapy with the goal of selecting the best available personalized treatment.

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 ; Sy, Sherwin K B ; Kawakita, Yasunori ; Yoon, Deokyong ; Wu, Shengyuan ; 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.

2025-12-01·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Exposure-response analysis of asciminib efficacy and safety in patients with chronic myelogenous leukemia in chronic phase

Article

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

342

项与盐酸阿思尼布相关的新闻（医药）

2025-12-13

·CCMTV血液频道

关爱相伴 “移”路相“髓”丨张晓辉教授：聚焦Ph+ALL移植全程管理——白消安预处理价值与TKI维持治疗新策略

前言费城染色体阳性急性淋巴细胞白血病（Ph+ALL）是急性淋巴细胞白血病中极具代表性的亚型，其核心致病驱动为BCR-ABL融合基因，这一特征不仅增强了疾病侵袭性，还让传统化疗面临较高的耐药困境，改善患者预后成为临床亟待突破的难题。酪氨酸激酶抑制剂（TKI）是Ph+ALL靶向治疗的核心药物，然而耐药问题始终是阻拦TKI治疗的最大障碍；造血干细胞移植则是Ph+ALL根治性治疗的关键手段，以白消安为基础的预处理方案经大量临床验证，疗效稳定且不可替代，为难治复发、微小残留病阳性患者带来根治希望。为深入解析Ph+ALL的治疗新方案与新思路，寻求TKI耐药处理，找寻最优移植预处理方案，CCMTV血液频道特邀北京大学人民医院张晓辉教授展开深度解读。 ▲专家精彩视频 Q1 在Ph+ALL患者移植前的预处理阶段,白消安联合方案是常用选择,这类方案与TKI协同应用时,在机制和疗效上有哪些优势?如何根据患者的疾病状态个体化调整预处理强度,以平衡疗效与安全性? 张晓辉教授急性髓系白血病和急性淋巴细胞白血病是白血病领域的两大主要类型，其中费城染色体（Ph）阳性急性淋巴细胞白血病是该领域中重点关注的重要亚型，其不仅发病率较高，还具备特征性的Ph阳性表型。在这类疾病的治疗中，靶向药物是核心治疗武器，既往临床多采用二代酪氨酸激酶抑制剂（TKI）进行联合治疗，但目前来看这类方案的耐药频率仍相对较高。泊那替尼等新型TKI联合方案的问世，在逆转BCR-ABL融合基因相关突变方面展现出良好疗效。临床研究证实，一旦出现BCR-ABL融合基因突变，往往会对患者远期预后造成不良影响，而新型TKI方案可有效改善这一困境[1-3]。在今年的美国血液学会（ASH）年会中，一项美国的研究证实阿昔替尼+泊那替尼+阿思尼布的三药联合方案能够改变BCR-ABL1寡聚体的构象动力学，为克服泊那替尼及阿思尼布耐药提供新的思路[6]。因此在“移植为先导、靶向治疗为联合”的整体治疗策略中，采用包括泊那替尼在内的靶向治疗方案，能够有效帮助患者在病程早期实现分子学完全缓解。在病程早期实现分子学完全缓解，有助于基于风险分层进一步优化造血干细胞移植、CAR-T细胞治疗等后续根治性治疗策略的选择。治疗全程需开展多维度的微小残留病（MRD）监测，包括流式细胞术（FCM）、聚合酶链反应（PCR）及二代测序（NGS）等。若某一监测维度提示MRD阳性，需通过动态监测追踪基因的演变过程，此时靶向药物治疗的优势会更为突出——只有在患者达到完全缓解（CR）且MRD转阴的双重目标后，后续的细胞治疗或造血干细胞移植才能真正实现疾病的深度缓解。在移植预处理阶段，白消安联合方案是经典且国际公认的预处理方案，该方案历经长期临床实践与大量研究验证，疗效稳定且不可替代[4]。临床数据证实，以白消安为基础的清髓性预处理方案，是保障移植疗效的核心前提，其在Ph+ALL移植中的治疗价值具有不可替代性[5]。在TKI治疗领域，一代、二代TKI已发挥重要作用，而新型药物的研发进一步丰富了治疗武器库。对于存在明确靶点的患者，这类药物还可延伸用于移植后的维持治疗，尤其是三代TKI的应用，有望帮助患者实现长期疾病缓解，这也是未来Ph+ALL治疗领域的重要突破方向。总体而言，Ph+急性淋巴细胞白血病的诊疗是急性白血病综合治疗的典范，通过分层诊断、综合治疗及个体化诊疗模式与方案的选择，未来有望实现该类疾病的彻底治愈。 Q2 移植后MRD监测是Ph+ALL患者长期管理的关键,TKI在MRD指导下的维持治疗中扮演着核心角色,ASH会议中相关研究对维持治疗的方案制定、剂量调整有何新的启示?TKI耐药后有哪些新的治疗方案? 张晓辉教授在Ph阳性急性淋巴细胞白血病的治疗中，临床面临的一大瓶颈是BCR-ABL融合基因的相关突变，如T315I突变等。尽管当前新型药物研发进展迅速，但仍缺乏可有效控制或清除这类突变细胞、帮助患者实现完全缓解的高效分子靶向药物。因此，移植前实现分子学完全缓解，是奠定患者未来治愈的核心前提。造血干细胞移植在该领域的核心价值，是为难治复发、MRD阳性的Ph+ALL患者搭建治疗桥梁。若能在移植前通过TKI治疗，实现Ph阳性白血病细胞及融合基因阳性细胞的完全清除，不仅能为后续移植创造良好条件、提升移植疗效，还能优化移植后的维持治疗效果。关于移植后维持治疗的用药选择，目前临床主要依据靶点检测结果及MRD监测数据，尤其是分子水平MRD状态来制定方案。基于动态监测结果，可将治疗分为复发后挽救治疗、干预性治疗及预防性治疗三类，而这三类治疗的实施，均离不开新型分子靶向药物与移植后动态MRD监测的协同应用，二者结合是实现患者长期生存的关键。本次ASH会议中的研究提示，第三代TKI治疗后MRD动态变化与突变克隆进化相关，持续MRD阳性提示耐药风险，需调整维持方案[8]；在维持治疗方案制定与联合策略优化方面，分子靶向药物，特别是第三代TKI药物，未来可通过创新治疗组合与模式，进一步提升患者的完全缓解率。例如，本次ASH会议中一项研究报道，与单药治疗相比，联合用药策略在一定程度上显示出更优疗效，其中泊那替尼联合维奈克拉的双靶点方案在延缓疾病进展和改善无事件生存期方面展现出积极信号[7]。泊那替尼的上市为Ph+ALL治疗带来重要契机，该药物的应用为克服TKI耐药提供了重要手段，有望帮助更多患者获得深度缓解并实现长期生存，从而显著改善疾病的长期预后。在临床上则追求的是在总生存期（OS）基础上，进一步延长无病生存期（DFS）。以MRD监测为保障、以便捷的靶向药物为治疗根基，使Ph+ALL的整体治疗水平必将实现长足进步。专家介绍 Expert presentation 张晓辉教授北京大学人民医院血液科、北京大学血液病研究所副所长主任医师、二级教授、博士生导师/博士后合作导师国家血液系统疾病临床医学研究中心副主任兼办公室主任中华医学会血液学分会委员会候任主任委员兼造血干细胞应用学组组长中国免疫学会血液免疫分会委员会副主任委员北京癌症防治学会血液工作委员会主任委员兼理事长课题负责人主持：国家科技重大专项、国家重点研发计划、国家自然科学基金重点项目等44项一作/通讯发表SCI论著152篇牵头撰写中国移植/出凝血指南/共识49项受邀在国际高级别血液病大会发言（ASH、EHA）45次授权国家发明专利15项获“国之名医·卓越建树”称号以第一完成人获：华夏医学科技奖一等奖中华医学科技奖二等奖以共同完成人获：国家科技进步二等奖（2项）参考文献： [1] Tachibana, Takayoshi et al. “The impacts of BCR-ABL1 mutations in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia who underwent allogeneic hematopoietic cell transplantation.” Annals of hematology vol. 99,10 (2020): 2393-2404. doi:10.1007/s00277-020-04212-1 [2] Garcia-Gutierrez, Valentin et al. “Ponatinib vs. asciminib in post-second-generation tyrosine kinase inhibitor therapy for chronic-phase chronic myeloid leukemia: a matching-adjusted indirect comparison.” Frontiers in oncology vol. 14 1455378. 20 Nov. 2024, doi:10.3389/fonc.2024.1455378 [3] Jabbour E, Kantarjian HM, Aldoss I, et al. Ponatinib vs Imatinib in Frontline Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia: A Randomized Clinical Trial. JAMA. 2024 Jun 4;331(21):1814-1823. [4] 中华医学会血液学分会造血干细胞应用学组.成人急性白血病自体造血干细胞移植中国专家共识（2024年版）[J].中华血液学杂志,2024,45(7):637-644.DOI:10.3760/cma.j.cn121090-20240611-00218. [5] Ahn J,et al.Comparative analysis of reduced toxicity conditioning regimens between fludarabine plus melphalan and fludarabine plus busulfex in adult patients with acute lymphoblastic leukemia.Bone Marrow Transplant . 2024 Jul 12. doi: 10.1038/s41409-024-02363-7. [6] Valentina Nardi et al. Novel BCR::ABL1 fusion alongside T315I reveals mechanism for dual ponatinib/asciminib resistance and proves sensitive to addition of axitinib. ASH 2025 abs25-9652 [7] Adam Laznicka et al. Ponatinib-based combination therapies including asciminib and venetoclax were effective on patient-derived xenograft models of chronic myeloid leukemia in blast Phase. ASH 2025 abs25-11051 [8] Xiaoshuai Zhang et al. Dynamic mutational evolution and transcriptomic remodeling on 3rd-generation TKI therapy in TKI-resistant patients with chronic myeloid leukemia. ASH 2025 abs25-13046 本平台致力于为医疗卫生专业人士提供丰富的医学信息。请注意，本平台发布的内容不能替代专业的医疗建议，也不应被用作诊疗指导。如果将这些信息用于非医学信息了解的目的，本平台不承担相关责任。此外，本平台对发布的内容并不代表赞同其描述和观点。如有涉及版权问题，请权利人与我们联系，我们将尽快处理。 — END — CCMTV临床频道注册会员数据突破210万+，平均每日更新视频及资讯内容300余条，目前拥有医学视频数量10万+，累积时长达185万+分钟，致力于打造贯穿医学教育全过程的专业服务平台。CCMTV药械营销推广覆盖开发期（上市前）、导入期（上市）、成长期、成熟期、衰退期。通过CCMTV朱雀AI大模型，实现权威精准触达和智能反馈分析，助力药械推广进入数字化闭环营销时代。

2025-12-11

·药携信息

中国原创第三代TKI奥雷巴替尼，超爆的数据，照亮CML患者新希望

在癌症治疗领域，慢性髓系白血病（CML）曾被视为“绝症”，患者中位生存期仅3-6年。但自2001年第一代酪氨酸激酶抑制剂（TKI）伊马替尼问世以来，CML治疗发生了革命性变化，如今多数慢性期患者（CML-CP）寿命已接近正常人。然而，治疗耐药和不耐受问题仍困扰约20%-30%的患者，尤其是携带T315I突变的“顽固”病例。奥雷巴替尼（Olverembatinib，商品名：耐立克®），作为中国原创第三代BCR-ABL1抑制剂，正以其强大效能和良好耐受性，成为这一领域的“新星”。本文基于2025年12月最新数据，对其研发历程、最新临床进展、市场潜力及竞争格局等方面进行更新解读。研发历程：从实验室到临床突破的“中国速度” 奥雷巴替尼由亚盛医药（Ascentage Pharma）开发，源于对CML耐药机制的深入洞察。CML的“元凶”是费城染色体导致的BCR-ABL1融合蛋白，该蛋白异常激活下游信号通路，促进癌细胞增殖。第一代TKI如伊马替尼虽有效，但无法应对T315I“门卫”突变——这一突变如同一道“铁闸”，阻挡大多数TKI进入活性位点，导致约20%的患者耐药。 2006年，广州健康院引进丁克研究员团队，并于2008年开始布局基于酪氨酸激酶抑制剂的治疗恶性肿瘤的新药开发领域。2013年，经实验室初步验证，确定D824为抗白血病候选药物，并将相关专利转让给广州顺健生物医药科技公司进行产业化开发。2016年，D824（HQP1351，奥雷巴替尼）获得1.1类新药临床批件，后由广州顺健的母公司江苏亚盛推进临床试验。2021年11月24日奥雷巴替尼获得上市批准，历时13年。奥雷巴替尼这一小分子口服药通过广泛的供体-受体氢键网络，精准抑制野生型及多种突变BCR-ABL1（包括T315I），IC50低至0.5 nM。体外实验显示，它能诱导CML细胞周期阻滞和凋亡；在小鼠异种移植模型中，奥雷巴替尼显著抑制肿瘤生长，甚至逆转T315I突变模型。临床开发起步于2016年10月的I期“3+3”剂量递增试验（SJ-0002，NCT03289874），招募101名TKI耐药CML患者。剂量从1mg/隔日一次（QOD）递增至60mg，推荐II期剂量（RP2D）定为40mg QOD。该药药代动力学支持隔日给药，半衰期长，生物利用度高。2021年11月，基于II期注册性试验（NCT03896269）数据，奥雷巴替尼获中国国家药品监督管理局（NMPA）批准，用于成人TKI耐药CML-CP或加速期CML-AP（CML-AP）伴T315I突变患者。这是中国首个本土第三代BCR-ABL1抑制剂，标志着我国在精准肿瘤药领域的重大突破。2021年7月，与信达生物达成战略合作，在中国共同开发和推广。 2023年11月，其适应症扩展至对一线/二线TKI耐药或不耐受的CML-CP患者，进一步拓宽应用。2024年6月14日，亚盛医药与武田制药签署独家期权协议，总价值13亿美元（包括1亿美元期权费及潜在里程碑付款），武田获全球（除中国大陆、港澳台及俄罗斯外）开发权。这不仅验证了奥雷巴替尼的全球潜力，还为后续国际注册铺路。目前，美国FDA已批准其III期注册性试验POLARIS-2（NCT06423911），针对CML-CP患者；2025年12月5日，FDA和EMA批准POLARIS-1 III期在初治Ph+ ALL患者中开展。2025年1月24日，亚盛医药在美国纳斯达克上市（股票代码：AAPG），进一步提升全球影响力。最新临床情况与结果解读：ASH 2025数据闪耀，全球III期加速奥雷巴替尼的临床开发正加速全球化。截至2025年12月，其多项III期试验（如POLARIS-1/2/3）已获FDA/EMA批准，覆盖CML、费城染色体阳性急性淋巴细胞白血病（Ph+ ALL）和琥珀酸脱氢酶缺陷型胃肠道间质瘤（SDH缺陷GIST）。2025年美国血液学会（ASH）年会（12月7-10日）成为其“高光时刻”，多篇口头/海报报告揭示了最新数据，包括POLARIS-1首发数据集。在TKI耐药/不耐受CML-CP患者中的注册II期试验（4年随访）：随机对照设计，对比奥雷巴替尼 vs. 最佳支持治疗（BAT）。中位事件自由生存期（EFS）达21.2个月（vs. BAT的2.9个月，P<0.001），无T315I突变亚组EFS为11.9个月（vs. 3.1个月，P=0.0159）。主要细胞遗传学反应（MCyR）率超80%，主要分子反应（MMR）率达60%。安全性佳，血管闭塞事件（AOE）发生率仅7%（远低于ponatinib的25%-30%），3级以上不良事件多为可控的血液毒性。解读：这些长程数据证实奥雷巴替尼在重度预处理患者中的持久效能，尤其对ponatinib/asciminib耐药者有效，填补了“后三线”治疗空白。数据于ASH 2025口头报告，强调其优于当前最佳疗法的疾病控制。二线CML-CP治疗（ChiCTR2200061655，2025年1月13日数据截点）：单臂、多中心研究，招募144名患者（包括105名无T315I突变）。完整细胞遗传学反应（CCyR）率71.8%，MMR率43.6%；二代TKI一线失败亚组CCyR达76.7%。随治疗周期延长，反应加深：21周期时MMR升至60%。无新安全信号。解读：这暗示奥雷巴替尼可前移至二线，甚至挑战一线地位，支持其在更广人群中的应用。ASH 2025更新显示，分子响应持续加深，早期线潜力明显。新诊断Ph+ ALL（POLARIS-1 III期，Part A）：奥雷巴替尼+低强度化疗，3周期诱导后，最佳MRD阴性率66%，MRD阴性完全缓解（CR）率64.2%；高危IKZF1plus亚组分子反应率90%。安全性良好，低强度方案减少了化疗毒性。解读：Ph+ ALL预后差（5年生存<50%），传统TKI+化疗MRD阴性率仅30%-40%。奥雷巴替尼的“深应答”潜力，或重塑一线方案，FDA/EMA已批准全球III期。首发数据于ASH 2025发布，MRD阴性率超60%为最佳记录。 SDH缺陷GIST（Ib期，NCT03594422）：26名TKI失败患者，中位PFS 25.7个月，客观缓解率（ORR）23.1%，临床获益率（CBR）84.6%。转化研究揭示其调控脂质代谢的新机制（如抑制脂质摄取，下调MCL-1）。解读：SDH缺陷GIST罕见且无标准治疗，传统TKI（如伊马替尼）无效。奥雷巴替尼的耐久PFS提供基准，III期POLARIS-3（NCT06640361）即将启动。无2025年新数据，但Ib期结果稳定。其他试验包括：NCT07178912（奥雷巴替尼+blinatumomab在Ph+ ALL），II期招募中；NCT06423911（CML-CP），招募中。总体而言，这些2025年数据强调奥雷巴替尼的“广谱覆盖”（pan-BCR-ABL1突变，包括对ponatinib/asciminib耐药）和低AOE风险，患者依从性高。未来，联合BCL-2抑制剂（如lisaftoclax）或探索AML/ALL扩展，将进一步放大其价值。市场潜力：销售强劲增长，全球“蓝海”待开拓奥雷巴替尼已纳入中国国家医保目录（NRDL），覆盖所有适应症，显著提升可及性。截至2024年6月，已进入670家医院。2024全年，亚盛医药总收入达1.343亿美元（增长342%），其中奥雷巴替尼中国销售增长52%。2025上半年，奥雷巴替尼销售达3030万美元（增长93%）。市场预测显示，全球奥雷巴替尼市场2024年规模5.6亿美元，至2033年将达17.4亿美元，复合年增长率（CAGR）14.1%。驱动因素包括：精准医疗浪潮（T315I检测普及）、早期线扩展潜力及多适应症布局（CML占70%，Ph+ ALL/GIST占30%）。武田期权协议注入7.5亿美元股权+潜在12亿美元里程碑，加速全球商业化。北美/欧洲市场潜力巨大：CML TKI市场超100亿美元，但耐药患者占比15%-20%，奥雷巴替尼的“后线首选”定位或抢占ponatinib份额。亚洲（尤其是中国）增长最快，得益于临床试验扩张和医保支持。挑战在于监管审批（如FDA BLA）和定价，但其“中国原创+全球验证”标签，将助力出口。纳斯达克上市进一步提升融资和市场信心，预计2025全年销售持续强劲。同类产品及靶点竞争：第三代TKI的“优胜劣汰” BCR-ABL1靶点竞争激烈，分三代：第一代伊马替尼（Glivec，诺华）奠基但耐药率高；第二代如达沙替尼（Dasatinib，BMS）、尼洛替尼（Nilotinib，诺华）、博舒替尼（Bosutinib，Pfizer）和氟马替尼（Flumatinib，中国汉莎）效能提升，但对T315I无效；第三代ponatinib（Iclusig，武田）覆盖T315I但AOE高（黑框警告）；asciminib（Scemblix，诺华）为变构抑制剂，创新但价格高昂、覆盖不全（部分突变无效）。奥雷巴替尼在2025年更新中，显示出克服ponatinib和asciminib耐药的能力，安全性优越。欧洲白血病网（ELN）2025指南推荐其用于ponatinib/asciminib失败患者。其他新兴TKI如vamotinib（PF-114）在开发中，但奥雷巴替尼的多激酶靶点（FLT3、KIT等）扩展至GIST/AML，竞争壁垒高。结语：点亮更多生命之光奥雷巴替尼的崛起，不仅是中国创新药的骄傲，更是CML患者重获希望的曙光。从实验室“铁闸”到临床“长效堡垒”，它以科学严谨和人文关怀，挑战耐药难题。展望未来，随着全球III期数据落地和商业化提速，奥雷巴替尼有望惠及更多患者，推动精准肿瘤治疗新时代。癌症无国界，创新无止境——让我们共同期待这一“中国方案”照亮全球蓝天。关注我们，下期聊更多前沿。健康，从觉醒开始！点赞、转发吧~

2025-12-09

·BioSpace

Terns Climbs 10% As ‘Unprecedented’ Leukemia Data Beats Novartis’ Scemblix

iStock, NWM TERN-701 more than doubled the response rate of Novartis’ rival approved therapy in an early-stage trial, sending the biotech’s shares flying. Terns Pharmaceuticals shares soared after chronic myeloid leukemia therapy TERN-701 doubled the response rate of Novartis’ approved STAMP inhibitor Scemblix in an early-stage trial. “Unprecedented remains the only suitable adjective to describe the compound’s clinical profile,” William Blair analysts wrote Tuesday morning. Terns’ shares climbed 10% to $44.45 as the markets opened Tuesday—but pre-market trading saw the stock rise as high as 55% over the weekend in anticipation of Monday’s readout. The results for TERN-701, revealed at the American Society of Hematology (ASH) annual meeting on Monday afternoon , showed a double whammy of improvement over Novartis’ offering. The Phase I CARDINAL trial featured patients with previously treated CML, with the readout including 38 efficacy-evaluable participants. Terns’ allosteric BCR/ABL1 inhibitor achieved an overall major molecular response (MMR) rate of 75% at week 24, while 64% of patients achieved the MMR. The data also supported daily dosing and had no food effect. “It is rare for an investigational agent to demonstrate unequivocal improvement in both clinical efficacy and safety and concurrently provide patients with better convenience of daily dosing with no food effect,” analysts at William Blair wrote in a note Tuesday. “We believe TERN-701 is on track to challenge Scemblix’s dominance and disrupt the treatment paradigm of CML.” For comparison, Scemblix and another investigational CML asset, Enliven’s ELVN-001, had response achievement rates in the range of 24%-32% in their own trials, the firm wrote. The William Blair analysts wondered, therefore, will the “differentiated data turn into a Big Pharma bidding war?” TERN-701 also “demonstrated the ability to salvage those who had previously been treated with Scemblix,” and relapsed, according to William Blair. An analysis of a small group of 10 patients who had previously received Scemblix showed a 43% MMR after treatment with Terns’ drug. Analysts at BMO Capital Markets, in their own note on Monday, said the 75% MMR rate is impressive, but key to TERN-701’s commercial success is the ability to maintain that response. The CARDINAL data showed just that, with patients switching to the drug showing stronger responses than they had previously achieved on other treatments. BMO projected TERN-701 could bring in peak sales of $3.4 billion in the long term. Patients with CML typically take a drug for several years before switching to another to kick-start a response again. With TERN-701 exhibiting such strong response rates, BMO is confident in the revised peak sales target. “CML often exhibits treatment switches, and active treatment commonly utilizes long, multi-year durations for therapy in between switches. The readout represents a comeback story for Terns, which was previously focused on metabolic diseases, including assets in obesity and metabolic dysfunction-associated steatohepatitis (MASH). The biotech changed focus to TERN-701 earlier this year, citing an oversaturated market in obesity. Earnings Terns Seeks Partners for Metabolic Assets Amid Oversaturated Obesity Market Terns, once a rising star in obesity and the MASH space, will refocus on cancer and partner out a handful of obesity assets. August 6, 2025 · 2 min read · Annalee Armstrong As for next steps, Terns will release more data from the CARDINAL trial next year and meet with regulators to develop a registrational trial in front-line and second-line CML. Executives told investors during a call Monday afternoon that the ASH abstract had galvanized patients and enrollment had “substantially improved.” “With enrollment timelines likely becoming more impactful in 2026, we see this expedited timeline as a meaningful positive that could benefit development timelines in the coming year,” BMO wrote. MMR typically translates well from Phase I to Phase III, according to William Blair’s analysts. “We are optimistic that the best-in-disease results will likely be replicated in the pivotal setting,” the firm said of the future clinical programs for TERN-701. Terns immediately used the positive data to raise some cash , offering $400 million worth of shares in a public offering on Tuesday. The cash will be used to fund development of TERN-701, including launch preparations.

临床结果临床1期ASH会议

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
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
慢性期费城染色体阳性慢性粒细胞白血病	澳大利亚	Novartis Pharmaceuticals Australia Pty Ltd.	2022-07-15
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

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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	繭窪鑰鏇鹽壓廠夢窪繭(構顧觸積鏇構網鏇鬱繭) = 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. 選憲蓋鬱憲簾醖選範艱 (糧窪壓窪鑰壓憲餘窪範 )	积极	2025-05-30
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	構夢網獵鹹憲遞艱鹽齋(築艱蓋齋鬱顧遞憲廠齋) = 18 pts experienced adverse events (AEs; any grade); 2 had Grade ≥3 AEs 憲網製願構鏇鏇願構顧 (窪繭齋積醖膚簾鑰鹽遞 ) 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	齋範觸構衊醖範網積夢(夢鹽醖夢衊艱衊憲壓構): cause-specific hazard ratio = 0.45 (95% CI, 0.25 ~ 0.81) 更多	积极	2025-05-30
				Nilotinib 300 mg
NCT03595917 (Phase I study, ASCO2025)	临床1期	急性白血病 ABL1 T315I \| p190 \| p210 ... 更多	15	Asciminib 80 mg/d	鹹淵選齋糧艱淵積廠糧(餘窪鹹遞遞襯選簾廠糧) = 觸壓鬱艱醖廠膚獵廠鏇餘衊壓鏇構構構遞襯糧 (選廠獵獵夢獵積選壓顧 ) 更多	积极	2025-05-30
				Dasatinib 140 mg/d	鹽簾餘鹽鬱範襯願壓選(範製衊獵鹹鹹膚鹹範壓) = 憲壓壓觸鹽醖窪醖襯醖鏇夢顧簾壓鹹膚襯鹹衊 (選醖繭願衊憲網觸淵壓 )
NCT05456191 (ASC4START, EHA2025)	临床3期	慢性期慢性髓性白血病	568	ASC 80 mg once daily	憲膚鑰選糧網淵願衊鹽(蓋製醖衊築範夢廠範簾): cause-specific hazard ratio = 0.45 (95% CI, 0.25 ~ 0.81) 更多	积极	2025-05-22
				NIL 300 mg twice daily
EHA2025	N/A	慢性期慢性髓性白血病 BCR::ABL1 \| t315i	36	ASCIMINIB	觸蓋積築壓糧觸齋鏇繭(夢淵觸獵鏇窪鑰衊壓齋) = 餘艱鹽遞窪齋構網鹽壓壓鹹壓艱夢積艱製積鑰 (窪觸鬱積夢繭廠觸範醖 ) 更多	积极	2025-05-14
				Placebo	齋範簾鹹艱窪網範願齋(範網齋醖繭網獵積鑰壓) = 窪製醖觸襯膚鬱夢窪製廠廠簾壓衊窪鏇繭選壓 (繭壓廠遞選壓鹽願鏇鏇 )
NCT05384587 (ASC2ESCALATE, EHA2025)	临床2期	慢性期慢性髓性白血病 BCR::ABL1	101	ASC 80 mg once daily	淵範製襯鑰簾膚餘選獵(獵範製衊選構鑰齋艱壓) = 壓願範窪艱窪獵範窪構觸淵範窪範獵蓋積範網 (壓廠鹹鹽廠鏇糧餘積壓 ) 更多	积极	2025-05-14
PB2728 (EHA2025)	N/A	慢性粒细胞性白血病二线	15	Asciminib	餘壓繭積鹹鑰齋鑰觸選(獵餘鹽鑰網淵艱艱衊糧) = 網壓壓艱蓋鏇衊齋膚鹹衊繭鹽觸膚鬱鑰簾艱鑰 (淵膚網蓋觸鏇鬱積網齋, 29 ~ 80) 更多	积极	2025-05-14
PS1594 (EHA2025)	N/A	慢性粒细胞性白血病 T315I mutation	69	Asciminib 200 mg BID	淵築齋顧構繭範齋鑰餘(窪窪蓋壓網鏇蓋糧鑰餘) = 鏇窪鹹積醖糧窪願襯鹽鏇顧遞蓋觸獵鑰衊鬱壓 (壓壓齋選淵築衊遞膚窪 ) 更多	-	2025-05-14
				Asciminib 40 mg BID	範積鑰鏇鹹鏇窪獵壓鬱(膚製艱鏇鑰糧觸壓壓鏇) = 築繭遞鏇鬱齋積鏇餘構鑰鹽餘積構淵積構膚衊 (構簾餘糧鹹積廠鑰鬱憲 ) 更多
NCT05143840 (HJKC3, EHA2025)	临床2期	慢性粒细胞性白血病	50	Asciminib 40 mg	築鹹膚窪壓襯憲遞醖製(糧窪鑰觸網淵構廠艱夢) = 衊積壓窪齋鹽顧築鏇窪鏇觸願糧鹽餘鬱構鬱蓋 (蓋廠餘膚餘餘構鏇艱醖 ) 更多	积极	2025-05-14
				Asciminib 80 mg	艱齋簾鬱鹹觸餘鑰壓壓(鬱餘衊遞襯遞壓醖夢鹹) = 顧淵鏇遞願遞淵遞衊憲鏇網醖鹹鹽襯鬱網壓夢 (鹽選壓壓積願顧鬱醖鬱 ) 更多