A Multi-Site Break Through Cancer Trial: Targeting Measurable Residual Disease in Patients With Acute Myeloid Leukemia: A Phase 1/2 Study of Tagraxofusp, Azacitidine, and Venetoclax

The purpose of this research study is to test the safety and efficacy of a new drug combination with three agents, azacitidine, venetoclax and tagraxofusp. Leftover (residual) leukemia disease that is not visible by eye can be increase the chance of disease recurrence. This research study is to determine if the combination therapy can safely help to control residual Acute Myeloid Leukemia (AML) and to prevent disease recurrence.
The names of the study drugs involved in this study are:
* Tagraxofusp (a type of CD123-directed cytotoxin)
* Azacitidine (a type of standard of care cytidine nucleoside analog)
* Venetoclax (a type of standard of care BCL-2 inhibitor)

开始日期2026-02-01

申办/合作机构

Dana-Farber Cancer Institute, Inc.

[+1]

NCT07148947

/ Not yet recruiting临床2期IIT

A Phase 2 Trial Investigating the Addition of Pacritinib to a Hypomethylating Agent as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant in Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms

This phase II trial tests if adding pacritinib to standard of care azacitidine or decitabine increases the number of patients able to proceed to hematopoietic stem cell transplantation (bridging) for patients with accelerated and blast phase myeloproliferative neoplasms. Pacritinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Azacitidine and decitabine are in a class of medications called hypomethylation agents. They work by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Adding pacritinib to standard of care azacitidine or decitabine may increase the number of patients able to proceed to hematopoietic stem cell transplantation for patients with accelerated and blast phase myeloproliferative neoplasms.

开始日期2026-02-01

申办/合作机构

University of Washington

[+1]

NCT07177079

/ Not yet recruiting临床1期IIT

High-dose Ascorbate (HDA) in Combination With Azacitidine and Venetoclax (Aza/Ven) in Newly Diagnosed Acute Myeloid Leukemia (AML)

This is a randomized, open-label, Phase I clinical study with expansion. It will assess the safety and efficacy of high-dose ascorbate administered concomitantly with azacitidine and venetoclax in newly diagnosed AML.

开始日期2025-12-31

申办/合作机构

University of Iowa

100 项与阿扎胞苷相关的临床结果

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100 项与阿扎胞苷相关的转化医学

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100 项与阿扎胞苷相关的专利（医药）

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8,333

项与阿扎胞苷相关的文献（医药）

2025-12-31·ANNALS OF MEDICINE

Significant prognostic improvement in elderly patients with acute myeloid leukemia treated with hypomethylating agents and venetoclax: outcomes from a retrospective real-world analysis

Article

作者: Wu, Yong ; Zheng, Jing ; Wu, Lanlan ; Chen, Yi ; Zhou, Jiayi ; Wu, Zhengjun ; Cai, Ruyu ; Wang, Zhengyang

OBJECTIVE:

This investigation aim to analyze the clinical features and therapeutic outcomes of elderly patients with acute myeloid leukemia (AML).

METHOD:

We conducted a retrospective analysis of the clinical and cytogenetic profiles of patients aged 60 years and older diagnosed with AML at the Fujian Medical University Union Hospital from 2016 to 2024.

RESULTS:

Our study cohort included 846 patients, with a median age of 67 years. The median duration of response (DOR) was 15.8 months, with 1-year, 3-year, and 5-year DOR rates of 55.3%, 23.4%, and 13.1%, respectively. The median overall survival (OS) was 21.8 months, with corresponding 1-year, 3-year, and 5-year OS rates of 58.7%, 41.2%, and 30.0%. Patients who received the azacitidine and venetoclax treatment protocol exhibited a complete remission (CR) rate of 57.1% and a minimal residual disease (MRD) - negative rate of 34.1%. These rates were comparable to the 59.2% CR rate and 39.4% MRD-negative rate observed in patients treated with intensive chemotherapy, and were superior to those with other low-intensity regimens. Compared to the treatment outcomes achieved at our center prior to 2016, when chemotherapy was the predominant treatment modality, which were characterized by a CR rate of 42.7%, a median OS of 9.2 months, and a mere 5-year OS rate of 13.5%, the remission rate and long-term survival of elderly patients with AML have been remarkable improved.

CONCLUSION:

The integration of hypomethylating agents and venetoclax into the treatment paradigm for elderly patients with AML has significantly enhanced survival rates.

2025-12-31·Hematology

Identification of t(X;1)(q28;q21) generating a novel GATAD2B::MTCP1 gene fusion in CMML and its persistence during progression to AML

Article

作者: Chen, Yu ; Zhu, Yi-yan ; Liu, Yi-zi ; Hou, Chun-xiao ; Zhang, Zhi-yu ; Chen, Su-ning ; Wang, Qian ; Zhang, Feng-hong

OBJECTIVE:

Hematological malignancies often involve chromosomal translocations and fusion genes that drive disease progression. While MTCP1 is well-known in T-cell prolymphocytic leukemia (T-PLL), its role in myeloid neoplasms is less understood. This report presents the first identification of the t(X;1)(q28;q21) translocation leading to the GATAD2B::MTCP1 fusion in acute myeloid leukemia (AML) transformed from chronic myelomonocytic leukemia (CMML).

METHODS:

The karyotypes were described according to the International System for Human Cytogenetic Nomenclature 2009. We performed targeted next-generation sequencing (NGS) on a panel of 172 genes commonly mutated in hematological malignancies (Supplemental Table 1), using an Illumina platform. RNA sequencing was conducted on total RNA extracted from bone marrow, also using the Illumina platform. The GATAD2B::MTCP1 fusion gene was confirmed by reverse transcription-polymerase chain reaction (RT-PCR) and Sanger sequencing, with specific primers for the fusion transcript (GATAD2B-F: CCTCTTTTTTTCGACGCC; MTCP1-R: ACTGAGCACAACACTTACGC).

RESULTS:

The GATAD2B::MTCP1 fusion results from a breakpoint on 1q21 within GATAD2B exon 1 and Xq28 within MTCP1 exon 2. The patient with the GATAD2B::MTCP1 fusion exhibited disease progression from CMML to AML. Despite achieving initial remission with venetoclax-based therapy and allo-HSCT, the patient relapsed and died.

CONCLUSIONS:

We propose that the GATAD2B::MTCP1 fusion upregulates MTCP1 expression rather than generating a fusion protein, thereby contributing to transformation and relapse in AML. Further investigations are needed to elucidate the precise role of this fusion event in myeloid malignancies.

2025-12-31·Epigenetics

CpG island demethylation and recruitment of SP1 to the promoter region regulates human thymic stromal lymphopoietin expression

Article

作者: Surjit, Milan ; Ganti, Krishna Priya

Thymic Stromal Lymphopoietin (TSLP), an immunomodulatory cytokine, plays a pivotal role in the development and progression of atopic and allergic diseases. Atopy follows familial inheritance, and genome-wide studies have shown association of atopy with TSLP polymorphisms. Here, we analysed the conserved transcriptional regulatory elements in the human TSLP promoter, which revealed the presence of three CpG islands. Demethylation of the CpG island using 5-azacytidine or siRNA-mediated knockdown of DNA methyl transferases significantly upregulated TSLP expression. Sequence analysis revealed the presence of two overlapping SP1 transcription factor DNA-binding sites (DBSs), between -1494 and -1510 nucleotides on the human TSLP promoter. Further experiments showed that demethylation of the CpG island enables the binding of SP1 to its cognate DBS present on the TSLP promoter, resulting in its transcriptional activation. Moreover, retinoic acid-induced transcription of human TSLP was associated with CpG island demethylation and SP1 binding to the TSLP promoter. These findings unravel a distinct mechanism of transcriptional regulation of the human TSLP gene and suggest possible epigenetic regulation of TSLP expression in modulating atopic and allergic disease severity in different individuals.

1,317

项与阿扎胞苷相关的新闻（医药）

2025-10-03

·药明康德

致敬时代 | 五年生存率跃升6倍，多款新型抗癌药为这类患者点亮希望

编者按：2025年，药明康德迎来创立25周年的重要里程碑。值此契机，我们向所有与我们共同书写产业变革篇章的科学家、医药人和投资者致以衷心感谢与诚挚敬意，也特别推出“致敬时代”系列，回顾全球同仁如何借助科学与合作的力量，不断拓展治疗边界、改善患者命运。四分之一个世纪的坚守，只为加速每一款新药的诞生。下一个25年，我们将继续心怀感恩与敬畏，依托独特的CRDMO模式，与全球伙伴携手同行，共赴健康未来。急性髓系白血病（AML）是成年人中最常见的白血病类型之一，多发于60岁及以上的人群，但也可能累及年轻人和儿童。据流行病学统计，2021年全球新发AML病例数超14万例。AML病情发展迅猛，如果不及时接受治疗，患者可能在几周到几个月内就发展为重症并死亡。面对这一高危疾病，AML的治疗策略近年来取得显著进展。除传统化疗、放疗及造血干细胞移植外，靶向药物、免疫疗法、细胞治疗及抗体偶联药物（ADC）等新型治疗方式逐步应用于临床，推动疗效不断提升。数据显示，过去几十年间，AML患者的5年生存率已从1975年的5.4%提升至2017年的33.7%，增长近6倍。在推动AML治疗革新的进程中，药明康德依托25年的行业深耕，不仅见证了AML治疗药物从实验室走向临床转化应用的突破历程，同时凭借其独特的CRDMO业务模式，助力全球合作伙伴加速包括AML在内的多种疾病创新疗法的研发进程，为全球患者带来更多治愈希望。从“脓血”疑云到解密白血病细胞的200年之路 AML的认知经历了两个世纪的探索。19世纪初，医学文献中已零星记载了一类血液异常病例：患者常伴发热、虚弱、肝脾肿大，血液外观呈乳糜状或脓血样。当时虽未精准归类，但这些现象已提示存在一种独特血液疾病。 1845年，显微镜技术带来了关键突破。英国医生约翰·休斯·贝内特（John Hughes Bennett）报道了首例白血病病例：一名28岁男性患者脾肝显著肿大，血管内充斥乳白色脓液样物质。显微镜下，他观察到患者血液中布满无色球体（白细胞），与凝固的纤维蛋白丝交织，并由此提出“白血球增多症（leucocythemia）”的概念，首次明确该病为原发性全身性血液疾病，而非感染引发的继发表现。 ▲贝内特所绘制的白血病患者血液中的无色球体（图片来源：参考资料[7]）贝内特的研究迅速引发学界关注。同年，德国病理学家鲁道夫·菲尔绍（Rudolf Virchow）也报道了类似病例。他通过系统分析发现，患者体内白细胞异常增殖会抑制红细胞生成，进而指出：白细胞增多是疾病的本质特征，而非脓液形成所致。1847年，菲尔绍正式将这类疾病命名为“白血病（leukämie）”，并尝试将其分为脾源性和淋巴性两大类——虽然这一分类尚不精确，却为现代白血病分型奠定了基础。 19世纪下半叶，随着病例积累，白血病逐渐被确认为独立疾病实体，临床与病理学研究的深入，也为探索其本质创造了条件。 1870年代，德国医学家保罗·埃利希（Paul Ehrlich）改进了血细胞染色技术，使血细胞结构清晰可见，进而首次识别出“原始细胞”——即未成熟的血细胞前体（现代医学中的造血干细胞）。埃利希还提出根据病程快慢和细胞成熟度，将白血病分为急性、慢性两大类：急性白血病以未成熟原始细胞大量增殖、病情进展迅速为特征；慢性白血病则由相对成熟但功能异常的细胞增殖主导，进展缓慢。这一分类框架逐步演化为现代白血病四大亚型：AML、急性淋巴细胞白血病（ALL）、慢性髓系白血病（CML）和慢性淋巴细胞白血病（CLL）。从19世纪对“脓血”现象的误解，到埃利希时代对白血病细胞分化特征的精准界定，人类对AML的认识已从宏观深入微观。这些历史积累不仅奠定了现代血液学的基础，更为后续AML分子机制研究与靶向治疗的开发提供了不可或缺的科学前提。化疗筑基，靶向疗法为特殊患者群体破局数十年来，AML的治疗策略经历了从传统化疗到精准靶向治疗的演进。以阿糖胞苷联合蒽环类药物（如柔红霉素或去甲氧柔红霉素、伊达比星）的“7+3方案”为代表的强化诱导化疗，曾长期作为年轻、体健且携带良好风险特征AML患者的基础治疗方案，使部分患者获得长期缓解。然而，AML多发于老年人群，多数患者年龄超过65岁，常因合并症、高危遗传学异常及对强化化疗耐受差等因素，临床预后不佳。针对不适合强化化疗的患者，去甲基化药物如阿扎胞苷的应用显著改善了患者的生存结局。时至今日，传统化疗药物仍构成AML治疗的重要支撑，除“7+3”方案常用药物外，还包括克拉屈滨、氟达拉滨、米托蒽醌、依托泊苷、羟基脲、皮质类固醇（如泼尼松或地塞米松）、甲氨蝶呤、6-巯基嘌呤（6-MP）、地西他滨。随着脂质体递送系统的进步，复方制剂如Vyxeos（柔红霉素/阿糖胞苷脂质体）也进一步优化了传统AML治疗药物的治疗指数。图片来源：123RF 近年来，针对癌细胞特定分子组分的靶向药物迅速发展，显著改变了AML的治疗格局。靶向药物通过特异性抑制白血病细胞的异常信号通路或分子标志，不仅为复发/难治性AML提供了新选择，也逐渐进入一线治疗，为老年及不耐受化疗的脆弱患者群体带来了希望。从4000个化合物中筛出的希望，开启AML靶向治疗新篇在众多分子靶点中，FLT3基因突变尤为关键。FLT3是一种受体酪氨酸激酶，调控造血干细胞的增殖、分化与存活。其突变可促进白血病细胞无限增殖并抑制凋亡，特别是FLT3-ITD突变，与AML预后不良密切相关。随着机制研究的深入，FLT3被确立为AML治疗的重要靶点。 FLT3抑制剂的研发起步于约翰斯·霍普金斯大学唐纳德·斯莫尔（Donald Small）博士团队。上世纪90年代，斯莫尔博士克隆出了首个人类FLT3基因并揭示了其在AML中的致病作用。由于当时缺乏高通量筛选工具，研究团队利用96孔板手动筛选了4000余种化合物，最终发现CEP-701（lestaurtinib）为潜在FLT3抑制剂。随后，通过实验室和动物模型验证，并构建FLT3活性检测方法，研究人员进一步明确了其作用机制。临床研究显示，CEP-701可清除AML患者外周血中的白血病细胞，在部分患者体内甚至还能清除白血病细胞的发源地——骨髓中的白血病细胞。但这种疗效是暂时的，且存在药物与血浆蛋白结合过强等缺陷，最终限制了其应用。基于斯莫尔博士团队的发现以及其他研究成果，新一代的FLT3抑制剂逐步克服了这些局限性。数年后，研究者意外发现多激酶抑制剂Rydapt（midostaurin，米哚妥林）竟是一种强效FLT3抑制剂。该药物最初作为PKC抑制剂开发，在实体瘤模型中展现出抗增殖活性。体外实验表明，米哚妥林可在极低浓度下抑制FLT3-ITD阳性小鼠原B细胞株增殖，并通过诱导凋亡和阻滞细胞周期发挥作用。在动物模型中，其口服治疗显著改善了移植FLT3-ITD骨髓小鼠（模拟AML模型）的生存。由于该药物的安全剂量已在其他疾病患者中确立，研究人员迅速将其应用于AML临床试验。在早期单药研究中，米哚妥林虽能降低外周血原始细胞计数，但完全缓解率有限，引发了对其疗效的质疑。然而，联合治疗方案改变了这一局面：米哚妥林与标准化疗联合使用，显著提高了AML患者的治疗结局。最终，2017年，美国FDA批准米哚妥林联合标准阿糖胞苷+柔红霉素诱导治疗及阿糖胞苷巩固治疗，用于FLT3突变阳性的成人AML患者。图片来源：123RF 米哚妥林的成功标志着AML靶向治疗的开端，也推动了更多高效、选择性更强的FLT3抑制剂相继问世。其中，Xospata（gilteritinib，吉瑞替尼）于2018年获FDA批准，用于复发/难治性FLT3突变AML；2023年，Vanflyta（quizartinib）也获得FDA批准，可联合化疗用于新确诊FLT3-ITD阳性AML成人患者，并在巩固化疗后可作为维持治疗单药使用。不杀死癌细胞也能治病的抗癌药在AML研究的另一条路径上，科学家的目光逐渐转向了癌细胞代谢。2009年，基因测序揭示了一个意外的发现：异柠檬酸脱氢酶（IDH）基因在部分AML患者中发生突变。最初，这一结果让研究者颇感困惑，毕竟，IDH是糖代谢和三羧酸循环中的关键酶，长期以来并未被视为典型的癌基因。然而，深入研究揭示了谜底。IDH突变改变了细胞代谢的走向：原本应生成α-酮戊二酸（a-KG）的反应被打断，反而触发了其“逆向反应”，产生异常代谢物2-羟基戊二酸（2-HG）。2-HG的大量积累抑制了a-KG依赖性酶的活性，导致DNA甲基化紊乱和基因表达失调，由此使癌基因被激活、抑癌基因沉默。更为重要的是，这一代谢异常阻碍了造血细胞的正常分化，迫使其停留在“幼稚”的状态，从而推动白血病的发生与进展。研究者同时发现，在星形细胞瘤、胶质母细胞瘤等多种肿瘤中也存在IDH1/2突变，凸显了其在肿瘤发生中的普遍性作用，高水平的2-HG更是与AML的不良预后相关。由此，一个全新的治疗设想浮现：如果能够抑制突变IDH的功能，让分化阻滞解除，使白血病细胞重新分化为成熟血细胞，疾病或许就能被逆转。这一理念不同于“直接杀死癌细胞”的传统模式，而是采用了“诱导分化”的全新思路。带着这一希望，医药界纷纷针对IDH靶点开展研发工作。经过多年探索，IDH2抑制剂Idhifa（enasidenib）率先问世，并于2017年获得FDA批准，用于治疗IDH2突变的复发/难治性AML。随后，IDH1抑制剂Tibsovo（ivosidenib，艾伏尼布）在2018年被FDA批准用于IDH1突变的复发/难治性AML；2019年，其适应症扩展至一线治疗——适用于≥75岁或因合并症无法耐受强化化疗的AML患者。2022年，FDA进一步批准艾伏尼布联合阿扎胞苷用于高龄（≥75岁）或其他不适合强化治疗的新确诊AML患者，显著拓宽了受益人群。同年，Rezlidhia（olutasidenib）也获FDA批准用于治疗IDH1突变的复发/难治性AML，为患者提供了更多选择。与依赖强烈细胞毒性的传统化疗不同，IDH抑制剂的作用机理别具一格：它们并不是直接摧毁癌细胞，而是通过解除分化阻滞，让“迷途”的白血病细胞回归正常发育轨迹，重新成为成熟的血细胞。这种“以分化代替杀伤”的新理念，为更多AML患者带来了与癌症长期共存甚至逆转疾病的希望。多靶点突破，AML治疗百花齐放随着靶向治疗的不断进展，AML治疗领域中多个关键靶点逐渐被开发，CD33、BCL-2、Hedgehog信号通路以及menin等靶点相继迎来突破性药物，持续推动AML治疗模式的革新。在CD33靶向药物方面，目前已获批的药物包括Mylotarg（gemtuzumab ozogamicin），其于2017年获美国FDA批准，用于新确诊的CD33阳性AML成人患者，以及2岁及以上的复发/难治性CD33阳性AML儿童和成人患者。 BCL-2抑制剂中，已有Venclexta（venetoclax，维奈克拉）获批上市。该药物于2018年获美国FDA加速批准，与阿扎胞苷/地西他滨或低剂量阿糖胞苷联合，用于≥75岁或因合并症无法接受强化化疗的AML患者一线治疗。在Hedgehog信号通路抑制剂方面，Daurismo（glasdegib）于2018年获FDA批准，可联合低剂量阿糖胞苷用于≥75岁或因合并症不适合强化化疗的新确诊AML成人患者，进一步拓宽了化疗不耐受人群的治疗选择。图片来源：123RF Menin抑制剂及DNA去甲基化药物组合也取得重要进展：2024年11月，美国FDA批准menin抑制剂Revuforj（revumenib）上市，用于治疗成人及≥1岁儿童中携带KMT2A易位的复发/难治性急性白血病患者；2023年，欧盟委员会批准Inaqovi（地西他滨 + cedazuridine），用于新确诊且不适合标准诱导化疗的AML成人患者，目前美国FDA已受理该药物与维奈克拉联合治疗同类患者的申请，预计将于2026年2月做出决定。在生物制品领域，已有多款药物用于AML辅助治疗：重组人粒细胞集落刺激因子Neutrogin（lenograstim）、Neupogen（filgrastim）在日本获批，用于缩短AML患者化疗后中性粒细胞恢复时间及发热持续时间；氨肽酶N（APN）抑制剂Bestatin（ubenimex，乌苯美司）上世纪80年代在日本获批用于AML维持治疗；硫鸟嘌呤（tabloid/tioguanine）早在上世纪60年代就获美国FDA批准，用于AML缓解诱导及巩固治疗；Ceplene（histamine dihydrochloride）在2008年获EMA批准，与IL-2联合用于首次缓解期AML成人患者的维持治疗。一体化CRDMO为AML患者带来曙光过去25年间，靶向疗法、免疫疗法和细胞疗法等创新治疗模式的不断涌现，持续重塑着血液癌症的治疗格局，让越来越多患者在延长生命的同时获得更好的生活质量。而这些疗法突破的背后，离不开科研人员对医学前沿的执着探索，以及产业伙伴间高效协同的支撑。长期以来，药明康德始终凭借“一体化、端到端”的CRDMO平台，持续赋能合作伙伴在血液癌症新药研发中不断突破，加速创新成果落地，惠及全球患者。在AML治疗领域，过去25年间，全球监管机构已先后批准了数十款AML创新疗法，为长期面临治疗困境的AML患者带来了新的治疗希望。作为创新的赋能者，药明康德很高兴能为其中多款疗法提供赋能，助力合作伙伴将这些创新疗法带到全球患者身边。在25年发展历程中，药明康德始终致力于支持全球合作伙伴从药物研究发现（R）、开发（D）到商业化生产（M）各个阶段的需求，助力突破性疗法加速研发进程。以血液肿瘤的小分子药物为例，药明康德化学服务平台能支持各种化学药物的分子形式及类别，满足从药物发现到商业化生产各个阶段、各个规模的各种物料需求。药明康德生物学业务平台作为综合性的早期发现和转化生物学赋能平台，能为包括血液肿瘤在内的多个疾病领域的新药研发提供从早期发现到临床研究阶段的生物学解决方案。药明康德测试业务平台可为包括血液肿瘤在内多个疾病领域的药物提供全生命周期的一体化研发测试服务及全方位的临床研究服务，助力合作伙伴的药物成功申报IND、NDA以及通过核查上市。随着对血液肿瘤发病机制理解的持续深化和治疗技术的不断创新，全球血液肿瘤药物研发正迈向更加精准、高效的新阶段。展望未来，药明康德将继续秉承“让天下没有难做的药，难治的病”的愿景，助力合作伙伴加速更多好药新药问世。参考资料： [1] Acute myelogenous leukemia，Retrieved september 9, 2025 from https://www.mayoclinic.org/diseases-conditions/acute-myelogenous-leukemia/symptoms-causes/syc-20369109 [2] Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version，Retrieved september 9, 2025 from https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq [3] Acute myeloid leukaemia，Retrieved september 9, 2025 from https://www.nhsinform.scot/illnesses-and-conditions/cancer/cancer-types-in-adults/acute-myeloid-leukaemia [4] Acute Myeloid Leukemia (AML)，Retrieved september 9, 2025 from https://my.clevelandclinic.org/health/diseases/6212-acute-myeloid-leukemia-aml [5] Key Statistics for Acute Myeloid Leukemia (AML). Retrieved september 9, 2025 from https://www.cancer.org/cancer/types/acute-myeloid-leukemia/about/key-statistics.html#:~:text=About%2022%2C010%20people%20will%20be,is%20about%20%C2%BD%20of%201%25. [6] Thomas X. First contributors in the history of leukemia. World J Hematol 2013; 2(3): 62-70 [DOI: 10.5315/wjh.v2.i3.62] [7] Kampen, Kim R. “The discovery and early understanding of leukemia.” Leukemia research vol. 36,1 (2012): 6-13. doi:10.1016/j.leukres.2011.09.028 [8] Strebhardt, K., Ullrich, A. Paul Ehrlich's magic bullet concept: 100 years of progress. Nat Rev Cancer 8, 473–480 (2008). https://doi.org/10.1038/nrc2394 [9] The History of Leukemia. Retrieved september 9, 2025 from https://bloodcancerunited.org/resources/blog/history-leukemia#early-leukemia-discoveries [10] Blum S, Bacher U, Pabst T. The Evolving Treatment Landscape of AML. healthbook TIMES Onco Hema. 2022;11(1):32-41. doi:10.36000//hbT.OH.2022.11.065 [11] Targeted Therapy Drugs for Acute Myeloid Leukemia (AML)，Retrieved september 9, 2025 from https://www.cancer.org/cancer/types/acute-myeloid-leukemia/treating/targeted-therapy.html [12] FLT3 Inhibitors. Retrieved september 9, 2025 from https://www.hopkinsmedicine.org/news/articles/2018/05/flt3-inhibitors#:~:text=Pediatric%20Oncology%20Director%20and%20leukemia,laborious%2C%20time%2Dconsuming%20process. [13] Stone, Richard M et al. “Midostaurin: its odyssey from discovery to approval for treating acute myeloid leukemia and advanced systemic mastocytosis.” Blood advances vol. 2,4 (2018): 444-453. doi:10.1182/bloodadvances.2017011080 [14] Acharya, Baku et al. “FLT3 inhibitors for acute myeloid leukemia: successes, defeats, and emerging paradigms.” RSC medicinal chemistry vol. 13,7 798-816. 23 May. 2022, doi:10.1039/d2md00067a [15] Daver, N., Schlenk, R.F., Russell, N.H. et al. Targeting FLT3 mutations in AML: review of current knowledge and evidence. Leukemia 33, 299–312 (2019). https://doi.org/10.1038/s41375-018-0357-9 [16] Chowdhury, Rasheduzzaman et al. “The oncometabolite 2-hydroxyglutarate inhibits histone lysine demethylases.” EMBO reports vol. 12,5 (2011): 463-9. doi:10.1038/embor.2011.43 [17] Sun, X.-J.; Chen, Z.; Chen, S.-J. Mutations in DNA Methyltransferases and Demethylases. In Encyclopedia of Cancer, 3rd ed.; Boffetta, P., Hainaut, P., Eds.; Academic Press: Cambridge, MA, USA, 2019; pp. 528–537. [18] Mardis, Elaine R et al. “Recurring mutations found by sequencing an acute myeloid leukemia genome.” The New England journal of medicine vol. 361,11 (2009): 1058-66. doi:10.1056/NEJMoa0903840 [19] Issa, Ghayas C, and Courtney D DiNardo. “Acute myeloid leukemia with IDH1 and IDH2 mutations: 2021 treatment algorithm.” Blood cancer journal vol. 11,6 107. 3 Jun. 2021, doi:10.1038/s41408-021-00497-1 [20] Yun, Zhangjun, et al. "Global, Regional and National Burden of Leukemia, 1990-2021: Analysis of Data from the Global Burden of Disease Study 2021." Blood 144 (2024): 7907. 免责声明：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

免疫疗法细胞疗法抗体药物偶联物临床研究

2025-10-01

·GlobeNewswire-Health Care

ORYZON Strengthens Patent Portfolio for iadademstat with European Grant Decision Covering Combinations with PD1/PD-L1 Inhibitors

MADRID and CAMBRIDGE, Mass., Oct. 01, 2025 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and European leader in epigenetics, today announced that the European Patent Office (EPO) has issued a Decision to Grant for its patent application EP20712594.9, entitled “Combinations of iadademstat for cancer therapy”. The allowed claims protect the use of iadademstat in combination with PD1 or PD-L1 inhibitors for the treatment of cancer, including small cell lung cancer (SCLC). Once formally granted, the patent will remain in force until at least 2040, excluding potential patent term extensions. Corresponding patents have already been allowed or granted in Australia and Russia, with additional applications pending in the United States, Japan, China, and other territories. “We are delighted to have secured this grant, which strengthens protection for iadademstat in combination with immune checkpoint inhibitors such as atezolizumab and durvalumab, a therapeutic approach we are actively pursuing in clinical trials,” said Neus Virgili, Oryzon’s Chief IP Officer. Iadademstat is currently being investigated in combination with PD-L1 inhibitors (atezolizumab or durvalumab) in first line, extensive-disease SCLC patients in a Phase I/II trial conducted and sponsored by the U.S. National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) with Oryzon. More than 30 sites across the U.S. participate in the trial, including leading institutions such as Memorial Sloan Kettering Cancer Center, Johns Hopkins, City of Hope, Yale University and the University of Chicago, among others. About OryzonFounded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company and the European leader in epigenetics, with a strong focus on personalized medicine in CNS disorders and oncology. Oryzon’s team is composed of highly qualified professionals from the pharma industry located in Barcelona, Boston, and San Diego. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors, vafidemstat in CNS (Phase III-ready) and iadademstat in oncology (Phase II). The company has other pipeline assets directed against other epigenetic targets like HDAC-6 where a clinical candidate, ORY-4001, has been nominated for its possible development in CMT and ALS. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. For more information, visit www.oryzon.com About IadademstatIadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L AML patients (ALICE trial) (see Salamero et al., ASH 2022 oral presentation & The Lancet Haematology, 2024, 11(7):e487-e498). Iadademstat is currently being evaluated in combination with gilteritinib in the ongoing Phase Ib FRIDA trial in patients with relapsed/refractory AML with FLT3 mutations, and in combination with azacitidine and venetoclax in 1L AML in an investigator-initiated study led by OHSU and in a trial sponsored by the U.S. National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) signed between Oryzon and the NCI to collaborate on further clinical development of iadademstat in different types of hematologic and solid cancers. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). Iadademstat is in a Phase I/II randomized trial in 1L ED-SCLC in combination with ICI sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center. Oryzon is further expanding the clinical development of iadademstat in oncology through additional CRADA and investigator-initiated studies. In addition, Oryzon is expanding iadademstat’s clinical development into non-oncological hematology indications, with trials in sickle cell disease (CTA approved) and essential thrombocythemia (trial in preparation). Iadademstat has orphan drug designation for SCLC in the US and for AML in the US and EU. FORWARD-LOOKING STATEMENTSThis communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives, and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees, or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events, or otherwise. This document does not constitute an offer or invitation to purchase or subscribe shares in accordance with the provisions of Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017, and/or the restated text of the Securities Market Law, approved by Law 6/2023 of 17 March, and its implementing regulations. Nothing in this document constitutes investment advice. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration.. SpainOryzonIR & Media, Europe & USPatricia Cobo/Mario CorderaEmili TorrellSandya von der WeidAtreviaChief BD OfficerLifeSci Advisors, LLC+34 91 564 07 25 +34 673 33 97 65+34 93 515 1313+41 78 680 05 38pcobo@atrevia.com mcordera@atrevia.cometorrell@oryzon.comsvonderweid@lifesciadvisors.com

临床2期孤儿药临床1期临床结果

2025-09-30

·news.abbvie.com

AbbVie Submits Biologics License Application (BLA) to U.S. FDA for Pivekimab sunirine (PVEK) - an Investigational Antibody-Drug Conjugate (ADC) to Treat Rare Cancer with Limited Treatment Options

Oncology R&D News – BLA based on data from the global Phase 1/2 CADENZA trial NORTH CHICAGO, Ill., Sept. 30, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced submission of a new Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of investigational Pivekimab sunirine (PVEK) for treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The submission is based on data from the Phase 1/2 CADENZA trial, a global study evaluating the safety and efficacy of PVEK in BPDCN. BPDCN is a rare and aggressive blood cancer that has features of both leukemia and lymphoma. Patients typically present with skin lesions and the disease often spreads to the bone marrow, central nervous system and the lymph nodes. First-line treatments are typically intensive chemotherapy and often followed by stem cell transplant. The need for additional and innovative treatment is high for both newly diagnosed patients and for those whose prior treatments have resulted in relapsed or refractory disease. PVEK is a CD123-targeting Antibody-Drug Conjugate (ADC) in clinical development for hematological malignancies (blood cancers), including BPDCN and acute myeloid leukemia (AML). ADCs are designed to deliver potent cancer cell death-inducing agents called 'payloads' directly to the cancer cells expressing a specific protein. CD123 (IL-3Rα) is a protein overexpressed in BPDCN, making it an ideal target for therapy. "Meaningful innovations in cancer research and treatment are happening every day. It is important that these innovations reach patients who desperately need them, including those with rare cancers who have limited options," said Roopal Thakkar, executive vice president, research and development and chief scientific officer, AbbVie. "We look forward to next steps in the regulatory process for our latest Antibody-Drug Conjugate (ADC), our first ADC in blood cancer, and how it may advance treatment for those living with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)." About the CADENZA Trial CADENZA is a Phase 1/2 multicenter, open-label study designed to determine the maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D), and recommended dosing schedule for PVEK monotherapy and to assess the safety, tolerability, PK, immunogenicity, and antileukemia activity of PVEK when administered to subjects with CD123+ hematologic malignancies (including subjects with BPDCN, AML, and others). About Pivekimab Sunirine (PVEK) PVEK is a CD123-targeting ADC in clinical development for hematological malignancies, including blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML). PVEK is currently being evaluated as monotherapy for patients with BPDCN and in combination with Vidaza® (azacitidine) and Venclexta® (venetoclax) for patients with untreated and relapsed/refractory AML. In October 2020, the FDA granted PVEK Breakthrough Therapy designation in relapsed/refractory BPDCN. About AbbVie in Oncology AbbVie is committed to elevating standards of care and bringing transformative therapies to patients worldwide living with difficult-to-treat cancers. We are advancing a dynamic pipeline of investigational therapies across a range of cancer types in both blood cancers and solid tumors. We are focusing on creating targeted medicines that either impede the reproduction of cancer cells or enable their elimination. We achieve this through various, targeted treatment modalities and biology interventions, including small molecule therapeutics, antibody-drug conjugates (ADCs), immuno-oncology-based therapeutics, multispecific antibody and novel CAR-T platforms. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potential breakthrough medicines. Today, our expansive oncology portfolio comprises approved and investigational treatments for a wide range of blood cancers and solid tumors. We are evaluating more than 35 investigational medicines in multiple clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit http://www.abbvie.com/oncology. About AbbVie AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas including immunology, oncology, neuroscience and eye care – and products and services in our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on LinkedIn, Facebook, Instagram, X (formerly Twitter) and YouTube. Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, changes to laws and regulations applicable to our industry, the impact of global macroeconomic factors, such as economic downturns or uncertainty, international conflict, trade disputes and tariffs, and other uncertainties and risks associated with global business operations. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2024 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its Quarterly Reports on Form 10-Q and in other documents that AbbVie subsequently files with the Securities and Exchange Commission that update, supplement or supersede such information. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. U.S. Media: Ilke Limoncu Ilke.limoncu@abbvie.com Investors: Liz Shea liz.shea@abbvie.com SOURCE AbbVie

抗体药物偶联物突破性疗法免疫疗法

100 项与阿扎胞苷相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D03021	阿扎胞苷	L01BC07	DB00928

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
幼年型骨髓单核细胞白血病	美国	Bristol Myers Squibb Co.	2022-05-20
骨髓增生性疾病	欧盟	betapharm Arzneimittel GmbH	2020-03-24
骨髓增生性疾病	冰岛	betapharm Arzneimittel GmbH	2020-03-24
骨髓增生性疾病	列支敦士登	betapharm Arzneimittel GmbH	2020-03-24
骨髓增生性疾病	挪威	betapharm Arzneimittel GmbH	2020-03-24
费城染色体阳性慢性粒细胞白血病	中国	Bristol-Myers Squibb Pharma EEIG	2017-04-24
高危骨髓增生异常综合征	澳大利亚	Celgene Pty Ltd.	2009-11-30
急性髓性白血病	欧盟	Bristol-Myers Squibb Pharma EEIG	2008-12-17
急性髓性白血病	冰岛	Bristol-Myers Squibb Pharma EEIG	2008-12-17
急性髓性白血病	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2008-12-17
急性髓性白血病	挪威	Bristol-Myers Squibb Pharma EEIG	2008-12-17
慢性粒单核细胞白血病	欧盟	Bristol-Myers Squibb Pharma EEIG	2008-12-17
慢性粒单核细胞白血病	冰岛	Bristol-Myers Squibb Pharma EEIG	2008-12-17
慢性粒单核细胞白血病	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2008-12-17
慢性粒单核细胞白血病	挪威	Bristol-Myers Squibb Pharma EEIG	2008-12-17
骨髓增生异常综合征	美国	Bristol Myers Squibb Co.	2004-05-19

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
免疫母细胞性淋巴结病	临床3期	日本	Celgene Corp.	2018-11-06
血小板减少症	临床3期	美国	Celgene Corp.	2013-04-26
血小板减少症	临床3期	澳大利亚	Celgene Corp.	2013-04-26
血小板减少症	临床3期	比利时	Celgene Corp.	2013-04-26
血小板减少症	临床3期	巴西	Celgene Corp.	2013-04-26
血小板减少症	临床3期	加拿大	Celgene Corp.	2013-04-26
血小板减少症	临床3期	捷克	Celgene Corp.	2013-04-26
血小板减少症	临床3期	丹麦	Celgene Corp.	2013-04-26
血小板减少症	临床3期	芬兰	Celgene Corp.	2013-04-26
血小板减少症	临床3期	法国	Celgene Corp.	2013-04-26

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临床结果

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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


NCT04372433 (Pubmed \| Blood Neoplasia)	临床1期	急性髓性白血病 \| 慢性粒单核细胞白血病	18	IO-202 + azacitidine	觸簾淵遞獵積醖襯夢積(網憲範範簾壓夢鹽鹹衊) = 餘襯積廠齋範繭顧觸糧淵繭遞廠顧構鏇膚鹹齋 (衊膚衊構憲顧積觸糧簾 ) 更多	积极	2025-11-01
NCT04102020 (VIALE-M, CTgov)	临床3期	急性髓性白血病	112	Venetoclax+Azacitidine (Part 1: Venetoclax 400 mg + Azacitidine 20 mg/m^2)	觸顧膚醖鏇願獵簾鑰夢 = 構窪願衊築獵顧簾廠齋齋齋鹹鹽壓餘餘艱襯範 (積構艱願築鏇範積簾鏇, 淵醖壓遞淵選遞壓願憲 ~ 衊鹽膚築網餘選夢蓋觸) 更多	-	2025-08-20
				Venetoclax+Azacitidine (Part 1: Venetoclax 400 mg + Azacitidine 36 mg/m^2)	觸顧膚醖鏇願獵簾鑰夢 = 艱鬱網築鏇醖糧鹹網鏇齋齋鹹鹽壓餘餘艱襯範 (積構艱願築鏇範積簾鏇, 願廠積選艱獵選鏇衊遞 ~ 築餘鹹鏇遞顧簾糧齋齋) 更多
NCT03013998 (Pubmed \| J Clin Oncol)	临床1期	难治性急性髓细胞白血病 NPM1m \| KMT2Ar	43	Azacitidine, Venetoclax, and Revumenib	齋構淵衊餘膚餘淵鏇醖(壓艱夢製糧醖遞蓋網蓋) = present in 8 (19%) patients 醖廠網廠艱廠鬱積鑰衊 (鹹構網製顧積範餘醖鹹 ) 更多	积极	2025-08-10
NCT02269280 (PF629, CTgov)	临床2期	慢性粒单核细胞白血病 \| 白血病 \| 骨髓增生异常综合征	185	AZA+Azacitidine (Azacitidine (AZA) - Days 1 - 3)	鏇鬱觸獵襯醖衊膚艱襯(構壓築網襯繭鏇範積鹹) = 網選壓壓鹽願網獵遞蓋積艱蓋壓壓願遞餘蓋膚 (蓋夢鹹製襯鏇觸網簾遞, 願醖鹹積願壓鹹鹽遞選 ~ 鑰鬱簾積憲鬱觸蓋鬱網) 更多	-	2025-08-07
				Azacitidine (AZA) Days 1 - 5 (Azacitidine (AZA) - Days 1 - 5)	鏇鬱觸獵襯醖衊膚艱襯(構壓築網襯繭鏇範積鹹) = 淵鹹壓廠築艱簾鬱廠積積艱蓋壓壓願遞餘蓋膚 (蓋夢鹹製襯鏇觸網簾遞, 觸繭醖鹹積齋築遞顧願 ~ 構壓簾鑰選蓋壓齋觸鬱) 更多
NCT05209152 (MDS-537, CTgov)	临床1期	高危骨髓增生异常综合征 \| 慢性粒单核细胞白血病	7	AMG 176 (Part 1A Dose Exploration: AMG 176 Dose Level 1)	觸遞製壓憲獵淵築齋蓋 = 鹽餘製醖簾積簾醖夢淵齋網蓋顧鹹積淵窪夢鏇 (窪繭繭鬱襯獵築齋廠艱, 壓醖觸淵顧繭餘醖壓憲 ~ 製鹹遞鏇鏇鑰糧選壓獵) 更多	-	2025-07-14
				AMG 176 (Part 1A Dose Exploration: AMG 176 Dose Level 1 Then Dose Level 2)	觸遞製壓憲獵淵築齋蓋 = 鑰範憲衊餘網築蓋鬱簾齋網蓋顧鹹積淵窪夢鏇 (窪繭繭鬱襯獵築齋廠艱, 構廠齋窪網觸觸衊壓顧 ~ 醖築窪糧繭膚憲鹹衊夢) 更多
Pubmed \| Ther Adv Hematol	临床2期	复发性弥漫性大B细胞淋巴瘤	15	Azacitidine + R-GDP	壓鑰遞廠鬱鹽鹽齋齋網(衊構壓鬱鏇夢簾積壓襯) = 糧齋觸憲鹽襯衊淵願蓋築選壓壓鑰壓製鏇廠鏇 (網構壓艱醖願廠鏇衊齋 ) 更多	积极	2025-07-01
EHA2025 人工标引	N/A	外周T细胞淋巴瘤	14	Linperlisib + Azacitidine	餘鏇網衊選蓋窪襯選醖(夢鏇製鬱憲醖壓餘鑰繭) = 壓選鏇窪觸築憲顧簾糧醖憲鏇遞廠構簾網構鬱 (廠鹽鏇構壓廠網築鹹衊 ) 更多	积极	2025-06-12
NCT03850574 (APTIVATE \| TUSCANY, GlobeNewswire) 人工标引	临床1/2期	急性髓性白血病一线 TP53 \| CKB \| FLT3	10	TUS(40mg)+VEN+AZA	餘願範遞蓋鑰獵憲壓鏇(糧夢築糧選餘蓋積鑰夢) = 餘衊顧鹽淵壓齋糧製齋獵鹹醖鬱齋願蓋構糧遞 (齋選簾築齋構鑰艱願繭 )	积极	2025-06-12
				TUS(80mg)+VEN+AZA	餘願範遞蓋鑰獵憲壓鏇(糧夢築糧選餘蓋積鑰夢) = 網積鏇糧製鏇構艱艱構獵鹹醖鬱齋願蓋構糧遞 (齋選簾築齋構鑰艱願繭 )
NCT00629343 (CTgov)	临床1期	恶性间皮瘤 \| 软组织肉瘤	27	Temozolomide+Azacitidine	鏇襯築遞窪衊膚窪襯憲 = 築醖膚顧遞鏇鹽醖壓鑰網壓鑰蓋構壓鑰鏇膚鏇 (鏇淵糧鏇積網繭蓋廠積, 餘鬱製鏇憲鏇廠製遞鹽 ~ 膚膚繭膚窪鬱遞築製願) 更多	-	2025-06-05
NCT02775903 (FUSION, Pubmed \| Ther Adv Hematol)	临床2期	急性髓性白血病 \| 高危骨髓增生异常综合征 SF3B1 \| SRSF2 \| U2AF1 ... 更多	-	Azacitidine + Durvalumab	夢窪糧製願襯膚餘淵範(製衊鏇餘餘糧鏇構醖壓) = 襯顧構憲齋築夢壓蓋鬱齋鹽鹽壓積顧鑰獵鏇壓 (遞遞網齋鏇簾範鏇艱鏇 ) 更多	不佳	2025-06-01
				Azacitidine	夢窪糧製願襯膚餘淵範(製衊鏇餘餘糧鏇構醖壓) = 廠夢窪憲衊衊艱夢鏇餘齋鹽鹽壓積顧鑰獵鏇壓 (遞遞網齋鏇簾範鏇艱鏇 ) 更多