更新于：2024-06-23

Decitabine

地西他滨

更新于：2024-06-23

概要

概要

基本信息

药物类型

小分子化药

别名

2'-deoxy-5-azacytidine、4-Amino-1-(2-deoxy-beta-D-erythro-pentofuranosyl)-1,3,5-triazin-2(1H)-one、4-amino-1-(2-deoxy-β-D-erythro-pentofuranosyl)-s-triazin-2(1H)-one

+ [19]

靶点

DNMT1

作用机制

DNMT1抑制剂(DNA（胞嘧啶-5）-甲基转移酶-1抑制剂)

治疗领域

肿瘤血液及淋巴系统疾病其他疾病+ [4]

在研适应症

难治性贫血难治性贫血伴原始细胞过多慢性粒单核细胞白血病+ [24]

非在研适应症

急性嗜碱性粒细胞白血病急性红细胞白血病急性白血病+ [72]

原研机构

Eisai Co., Ltd.

在研机构

The University of Texas MD Anderson Cancer Center

National Cancer Institute

Dana-Farber Cancer Institute, Inc.

+ [8]

非在研机构

Janssen Research & Development LLC

西安杨森制药有限公司Fred Hutchinson Cancer Center

+ [23]

最高研发阶段批准上市

首次获批日期

美国 (2006-05-02),

难治性贫血伴原始细胞过多难治性贫血慢性粒单核细胞白血病+ [2]

最高研发阶段(中国)临床3期

特殊审评孤儿药 (美国)、孤儿药 (欧盟)

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结构

分子式C8H12N4O4

InChIKeyXAUDJQYHKZQPEU-KVQBGUIXSA-N

CAS号2353-33-5

关联

430

项与地西他滨相关的临床试验

NCT06441097 / Not yet recruiting临床2期IIT

A Study to Evaluate the Efficacy and Safety of Genotype-guided Targeted Agents in Combination With POLA-RCHP VERSUS POLA-RCHP in Patients With Previously Untreated Diffuse Large B-cell Lymphoma

The purpose of this study is to compare the efficacy and safety of genotype-guided targeted agents in combination with polatuzumab vedotin plus rituximab, cyclophosphamide, doxorubicin, and prednisone (Pola RCHP-X) versus Pola RCHP in Chinese patients with previously untreated diffuse large B-cell lymphoma (DLBCL).

开始日期2024-12-31

申办/合作机构-

NCT03184935 / Suspended临床1/2期

Research for Human Umbilical Cord Mesenchymal Stem Cells in the Treatment of Myelodysplastic Syndrome (MDS)

The purposes of the study is to determine the safety and efficacy of human umbilical cord mesenchymal stem cells (hUC-MSC) in treating Myelodysplastic Syndrome patients.

开始日期2024-12-31

申办/合作机构

圣释（北京）生物工程有限公司

NCT06445959 / Not yet recruiting临床1/2期

Phase 1b/2 Study of Decitabine and Venetoclax in Combination With the Targeted Mutant IDH1 Inhibitor Olutasidenib

To find a recommended combination dose of decitabine and venetoclax that can be given in combination with olutasidenib to participants with AML.

开始日期2024-11-29

申办/合作机构

Rigel Pharmaceuticals, Inc.

100 项与地西他滨相关的临床结果

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100 项与地西他滨相关的转化医学

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100 项与地西他滨相关的专利（医药）

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7,921

项与地西他滨相关的文献（医药）

2024-04-01·Apoptosis : an international journal on programmed cell death

Synergistic cytotoxicity of decitabine and YM155 in leukemia cells through upregulation of SLC35F2 and suppression of MCL1 and survivin expression

Article

作者: Chiou, Jing-Ting ; Chang, Long-Sen

The hypomethylation agent decitabine (DAC), in combination with other apoptosis inducers, is considered a potential modality for cancer treatment. We investigated the mechanism underlying the combined cytotoxicity of DAC and YM155 in acute myeloid leukemia (AML) cells because of increasing evidence that YM155 induces apoptosis in cancer cells. Co-administration of DAC and YM155 resulted in synergistic cytotoxicity in AML U937 cells, which was characterized by the induction of apoptosis, NOXA-dependent degradation of MCL1 and survivin, and depolarization of mitochondria. Restoration of MCL1 or survivin expression attenuated DAC/YM155-induced U937 cell death. DAC initiated AKT and p38 MAPK phosphorylation in a Ca²⁺/ROS-dependent manner, thereby promoting autophagy-mediated degradation of β-TrCP mRNA, leading to increased Sp1 expression. DAC-induced Sp1 expression associated with Ten-eleven-translocation (TET) dioxygenases and p300 was used to upregulate the expression of SLC35F2. Simultaneously, the activation of p38 MAPK induced by DAC, promoted CREB-mediated NOXA expression, resulting in survivin and MCL1 degradation. The synergistic cytotoxicity of DAC and YM155 in U937 cells was dependent on elevated SLC35F2 expression. Additionally, YM155 facilitated DAC-induced degradation of MCL1 and survivin. A similar mechanism explained DAC/YM155-mediated cytotoxicity in AML HL-60 cells. Our data demonstrated that the synergistic cytotoxicity of DAC and YM155 in AML cell lines U937 and HL-60 is dependent on AKT- and p38 MAPK-mediated upregulation of SLC35F2 and p38 MAPK-mediated degradation of survivin and MCL1. This indicates that a treatment regimen that amalgamates YM155 and DAC may be beneficial for AML.

2024-04-01·Drug metabolism and pharmacokinetics

Air–liquid interface culture and modified culture medium promote the differentiation of human induced pluripotent stem cells into intestinal epithelial cells

Article

作者: Minowa, Hanako ; Iwao, Takahiro ; Matsunaga, Tamihide ; Qiu, Shimeng ; Hashita, Tadahiro ; Shirai, Kotaro

An in vitro system that evaluates pharmacokinetics in the small intestine is crucial for the development of oral drugs. We produced human induced pluripotent stem cell-derived small intestinal epithelial cells (hiSIECs) with high drug metabolizing enzyme and drug transporter activities. However, the gene expression of our hiSIECs partially differed from that of the human small intestine, with low drug metabolizing enzyme activities. Therefore, we used air-liquid interface (ALI) culture and 5-aza-2'-deoxycytidine (5AZA)-free medium to generate hiSIECs (novel hiSIECs). Novel hiSIECs showed enhanced gene expression of drug metabolizing enzymes, such as cytochrome P450 (CYP)3A4, CYP2C9, CYP2C19, and carboxylesterase 2 that are highly expressed in the small intestine. In addition, the expression of genes involved in nutrient absorption-one of the major functions of the small intestine-also increased. The novel hiSIECs expressed ZO-1 and E-cadherin. Moreover, the novel hiSIECs exhibited a barrier function that allowed low lucifer yellow permeation. The novel hiSIECs showed high activities of CYP3A4, CYP2C9, and CYP2C19, which are abundantly expressed in the small intestine. In conclusion, the novel hiSIECs have great potential as an in vitro system to evaluate pharmacokinetics in the small intestine.

2024-04-01·Reproductive sciences (Thousand Oaks, Calif.)

Conservative Hypomethylation of Mesenchymal Stem Cells and Their Secretome Restored the Follicular Development in Cisplatin-Induced Premature Ovarian Failure Mice

Article

作者: Holah, Nanis Shawky ; El Barbary, Ahmed A ; Nabil Salama, Amira ; Hessien, Mohamed ; Badr, Eman Abd El-Fatah

Abstract:

Premature ovarian failure (POF) is one of the main causes of infertility in women under the age of 40 years. Recently, epigenetic reprogramming, particularly DNA hypomethylation, has emerged as a promising strategy to enhance the therapeutic potential of mesenchymal stem cells (MSCs). Thus, it is crucial to elucidate how far global hypomethylation of MSCs genome can maintain their pluripotency and viability and improve their therapeutic effect in chemotherapy-induced POF mice. Herein, the genomic DNA of bone marrow-derived MSCs (BM-MSCs) was hypomethylated by the DNA methyltransferase inhibitor (5-Aza-dC), and the degree of global hypomethylation was assessed by methylation-sensitive HepII/MspI restriction analysis. Next, mildly hypomethylated cells and their secretome were independently transplanted (or infused) in POF mice, established via cisplatin-mediated gonadotoxicity. We found that conservative global hypomethylation of BM-MSCs genome with low doses of 5-Aza-dC (≤0.5 μM) has maintained cell viability and MSCs-specific clusters of differentiation (CD). Engraftment of mildly hypomethylated cells in POF mice, or infusion of their secretome, improved the concentrations of estradiol (E2), follicle-stimulating hormone (FSH), and anti-Mullerian hormone (AMH). Furthermore, mice restored their normal body weight, ovarian size, and ovarian follicle count. This was associated with improved follicular development, where the populations of healthy primordial, primary, secondary, and tertiary follicles were significantly ameliorated, relative to mice transplanted with normally methylated cells. This observational study suggests that transplantation of mildly hypomethylated BM-MSCs cells and their secretome can restore the structural and functional integrity of the damaged ovaries in POF mice. Also, it presents conservative hypomethylation of BM-MSCs and their secretome as a promising alternative to MSCs transplantation.Graphical AbstractTransplantation of partially hypomethylated BM-MSCs improved the follicular count and integrity in the POF mouse model. Gonadotoxic drug (cisplatin) was used to establish the POF mouse model. In parallel, BM-MSCs were isolated, authenticated, and then incubated with the DNMTs inhibitor (5-Aza-dC). Partially hypomethylated cells and their secretome were independently transplanted into the POF mice, and both the follicular count, ovarian histology, and the serum levels of the fertility-related hormones (E2, AMH, and FSH) were assessed 1 week after transplantation or infusion. Hypomethylated BM-MSCs and their secretome increased the follicular count, increased the number of healthy follicles, and restricted apoptosis of the granulose cells. Also, the hormonal profile was improved compared to their corresponding level in mice transplanted with normally methylated cells.

187

项与地西他滨相关的新闻（医药）

2024-06-20

·PRNewswire

Escend Announces Poster Presentation at the Annual Meeting of the European Hematology Association (EHS) from an Investigator-Initiated Phase I/II study evaluating ES-3000 in Myelodysplastic Syndrome (MDS)

MENLO PARK, Calif., June 20, 2024 /PRNewswire/ -- Escend Pharmaceuticals, Inc., a privately held oncology company, announced today a poster presentation with initial data from the Australasian Leukaemia and Lymphoma Group's (ALLG) investigator-initiated Phase I/II platform study (MYDAS-T MDS05 domain 1) evaluating ES-3000 alone and in combination with ASTX727 at European Hematology Association (EHS) in Madrid, Spain. The poster is entitled, 'Combining oral Wnt/β catenin/inflammasome pathway inhibitor, a bis-benzylisoquinoline alkaloid (ES-3000) with oral decitabine/cedazuridine (ASTX727)* as a treatment strategy for myelodysplasia'. The primary objectives of the initial phase of the trial are to assess the safety profile of ES-3000 and determine the recommended phase 2 dose and dosing schedule of ES-3000 in combination with fixed-dose decitabine/cedazuridine (ASTX727). The results indicate a response in 40% of evaluable patients who received ES-3000 as a single agent. The dose-limiting toxicities as per BION modeling allowed the study to dose escalate. A copy of the poster is available at Escend Pharmaceuticals' website About ES-3000 ES-3000 is a small molecule, oral investigational product that reduces leukemic stem cells by β-catenin and Calmodulin protein-dependent kinase II gamma (CamKIIγ). ES-3000 is being developed for the treatment of myeloid malignancies including acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndrome (MDS). About Escend Escend's development strategy is to select drug candidates with established clinical safety that have not yet achieved US marketing approval and develop them for oncology based on new mechanism of action details, where their effects on specific cellular pathways can be leveraged for the development of novel therapeutics. About Australasian Leukaemia and Lymphoma Group's (ALLG) The Australasian Leukaemia and Lymphoma Group (ALLG) is Australia's and New Zealand's only collaborative clinical trial organisation that sponsors local investigator-initiated clinical trials. Established in 1973, the ALLG's membership of over 1,300 blood cancer health professionals at 94 networked sites includes haematologists, clinician researchers, scientists and nurses treating leukaemia, lymphoma, myeloma, myelodysplastic syndromes and other haematological malignancies. The ALLG plans, designs, conducts, monitors and publishes investigator initiated clinical trials to create better treatments and better lives for patients with blood cancers. For further information about this press release, please contact Saira Bates ([email protected]) or visit *Astex Pharmaceuticals study drug SOURCE Escend Pharmaceuticals, Inc.

临床研究临床结果

2024-06-14

·GlobeNewswire-Health Care

Bio-Path Holdings Presents Data from Ongoing Phase 2 Combination Study of Prexigebersen for Treatment of Acute Myeloid Leukemia at European Hematology Association Congress

Encore Presentation Highlights Positive Results from Interim Analysis Demonstrating Significant Clinical Improvement and Tolerable Safety Profile for Prexigebersen Combination in High-Risk PatientsHOUSTON, June 14, 2024 (GLOBE NEWSWIRE) -- Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize® liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, presented interim results from the Company’s Phase 2 study of prexigebersen (BP1001) in combination with decitabine and venetoclax for the treatment of acute myeloid leukemia (AML) in a poster presentation at 2024 European Hematology Association (EHA) Congress, on June 14, 2024 in Madrid, Spain. Jorge Cortes, M.D., Director of the Georgia Cancer Center, presented data showing prexigebersen continues to be well-tolerated and has now demonstrated compelling efficacy results in two reporting cohorts including evaluable newly diagnosed AML patients and evaluable refractory/relapsed AML patients, both of which exceeded outcomes with frontline therapy. “It was a pleasure to present these compelling data to an audience of European oncologists who treat AML patients and understand the continued great need for new therapeutic options,” said Peter Nielsen, Chief Executive Officer of Bio-Path. “Given that our study is being conducted in the U.S., this encore presentation is an important step towards educating global oncology leaders on the benefits of prexigebersen and its potential to be another tool in their fight against AML.” Data Highlights In Cohort 1, 31 newly diagnosed patients were enrolled; 20 evaluable patients (9 male: 45%) with a median age of 75 years (range, 69 - 84), treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk (n=12, 2017 ELN guidelines) or secondary AML (sAML; n=7) evolved from myelodysplastic syndromes (n=4), chronic myelomonocytic leukemia (n=1) or treatment-related AML (n=2). Fifteen patients (75% of evaluable; 54% of enrolled) achieved complete remission (CR), CRh (CR with partial recovery of peripheral blood counts), or CRi (CR with incomplete hematologic recovery); two patients achieved partial remission (PR) and two patients achieved stable disease (SD). In Cohort 2, 38 relapsed/refractory patients were enrolled; 23 evaluable patients (13 male: 57%) with a median age of 63 years (range, 24 - 89), treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk (n=13) or sAML (n=5). Twelve patients (55% of evaluable; 32% of enrolled) achieved CR/CRi/CRh; one patient achieved PR, eight patients achieved SD and one patient had treatment failure. Among the evaluable patients of both cohorts, adverse events were consistent with those expected with decitabine and venetoclax and/or AML, including fatigue (72%), anemia (60%) and neutropenia (49%), while the most frequent severe adverse events were febrile neutropenia (26%) and sepsis (5%). Given these promising interim results, Bio-Path expects to continue enrollment of up to 98 and 54 evaluable patients for Cohorts 1 and 2, respectively. About Bio-Path Holdings, Inc. Bio-Path is a biotechnology company developing DNAbilize®, a novel technology that has yielded a pipeline of RNAi nanoparticle drugs that can be administered with a simple intravenous transfusion. Bio-Path’s lead product candidate, prexigebersen (BP1001, targeting the Grb2 protein), is in a Phase 2 study for blood cancers, and BP1001-A, a drug product modification of prexigebersen, is in a Phase 1/1b study for solid tumors. The Company’s second product, BP1002, which targets the Bcl-2 protein, is being evaluated for the treatment of blood cancers and solid tumors, including lymphoma and acute myeloid leukemia. In addition, an IND is expected to be filed for BP1003, a novel liposome-incorporated STAT3 antisense oligodeoxynucleotide developed by Bio-Path as a specific inhibitor of STAT3. For more information, please visit the Company's website at www.biopathholdings.com. Forward-Looking Statements This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws. These statements are based on management's current expectations and accordingly are subject to uncertainty and changes in circumstances. Any express or implied statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Any statements that are not historical facts contained in this release are forward-looking statements that involve risks and uncertainties, including Bio-Path’s ability to raise needed additional capital on a timely basis in order for it to continue its operations, have success in the clinical development of its technologies, the timing of enrollment and release of data in such clinical studies, the accuracy of such data, limited patient populations of early stage clinical studies and the possibility that results from later stage clinical trials with much larger patient populations may not be consistent with earlier stage clinical trials, its ability to obtain domestic and/or foreign regulatory approval for its drug candidates, the maintenance of intellectual property rights, that patents relating to existing or future patent applications will be issued or that any issued patents will provide meaningful protection of our drug candidates, the impact, risks and uncertainties related to global pandemics, including the COVID-19 pandemic, and actions taken by governmental authorities or others in connection therewith, and such other risks which are identified in Bio-Path's most recent Annual Report on Form 10-K, in any subsequent quarterly reports on Form 10-Q and in other reports that Bio-Path files with the Securities and Exchange Commission from time to time. These documents are available on request from Bio-Path Holdings or at www.sec.gov. Bio-Path disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Contact Information: Investors Will O’ConnorStern Investor Relations, Inc.212-362-1200will@sternir.com Doug Morris Investor Relations Bio-Path Holdings, Inc. 832-742-1369

临床结果临床2期寡核苷酸

2024-06-14

·药融圈

抗肿瘤百亿市值Biotech，被低估的潜在收购对象:新品有望今年获批

▲8月15-16日 NDC2024生物医药创新者峰会扫码立即报名注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。 2024年3月26日，Syndax Pharmaceuticals宣布美国FDA接受其在研管线Revumenib递交的NDA并授予优先审评资格，用于治疗携带KMT2A重排（KMT2Ar）的复发/难治性急性白血病患者。如果进展顺利的话，Revumenib有望于2024年在美国获批。从药融云数据库获悉，Syndax Pharmaceuticals（以下简称Syndax）成立于2005年，是一家临床阶段的生物制药公司，致力于开发创新的癌症疗法管线。药融云数据库：融资信息 2023年底，Syndax宣布完成此前宣布的12432431股普通股的承销公开发行，其中包括行使全部承销商购买1621621股额外股票的选择权。在扣除承销折扣和佣金以及预计发行费用之前，Syndax从此次发行中获得的总收益约为2.3亿美元。此次增发无疑又为公司的融资和业务合作提供了更多机会，公司目前合作伙伴有Incyte、Vitae Pharmaceuticals（艾伯维的子公司）、UCB和拜耳。截止2024年5月23日，Syndax总市值17.09亿美元（124亿人民币）。截止2024年6月13日，收盘，每股17.40美元。公司市值研发管线 Syndax目前与合作伙伴进行公司管线的研发，主要包括以下三条研发管线，各管线具体进展情况如下： 01 Revumenib Revumenib（SNDX-5613）是一种新型口服Menin-KMT2A抑制剂。KMT2A重排的白血病发生在婴儿、儿童和成人患者中，NPM1突变是急性髓系白血病中最常见的基因改变，KMT2A重排和NPM1突变合计占急性髓系白血病（AML）病例的40%。在KMT2A重排或NPM1突变的急性白血病中，menin蛋白与KMT2A的相互作用对于驱动白血病促进基因的表达至关重要。临床前研究表明，靶向menin和KMT2A之间的相互作用可能是治疗携带这些突变的癌症的有效策略。目前正在开发Revumenib用于治疗KMT2Ar（KMT2A重排，也称为混合谱系白血病重排或MLLR）急性白血病（包括急性淋巴细胞白血病和急性髓系白血病），以及NPM1突变（NPM1m）急性髓系白血病（AML）。 Revumenib目前正在多项临床试验中进行评估，包括用于治疗复发/难治性（R/R）急性白血病的关键性AUGMENT-101试验，各项临床试验具体进展情况如下：（1）AUGMENT-101-2A是Revumenib单药治疗复发/难治性KMT2A基因重排急性淋巴细胞白血病（ALL）的试验，这是一项临床试验1/2期开放标签、剂量递增和剂量扩展队列研究。（2）AUGMENT-101-2B是Revumenib单药治疗复发/难治性KMT2A基因突变急性髓系白血病的试验。（3）AUGMENT-101-2C是Revumenib单药治疗复发/难治性NPM1m（核磷蛋白1突变）的急性髓系白血病的试验。2024年3月，公司宣布完成针对R/RNPM1mAML患者的AUGMENT-101关键试验队列的入组，顶线数据预计将于2024年第四季度公布，并可能支持2025年上半年Revumenib在R/R NPM1m AML中的sNDA（补充新药申请）。药融云数据库：药品信息此外，Revumenib治疗NPM1m和KMT2Ar急性白血病的多项临床试验1期联合研究正在进行，这些试验目前正在扩大，以验证推荐的临床试验2期剂量，预计2024年下半年将获得更多数据。这些试验包括：（1）AUGMENT-102试验是Revumenib联合化疗治疗KMT2Ar或NPM1m急性白血病患者的临床试验1期研究。（2）BEAT-AML主要评估Revumenib与维奈克拉（VEN）和阿扎胞苷（AZA）联合治疗一线AML患者的情况，该试验是白血病和淋巴瘤协会的Beat AML临床试验的一部分。（VEN+AZA：VEN是一种口服的B细胞淋巴瘤2选择性抑制剂，目前多项研究及临床试验证实VEN联合去甲基化药物或低剂量阿糖胞苷在AML患者中可获得较高的治疗反应和无病生存率，2020年10月FDA批准VEN与AZA、地西他滨或LDAC联合用于≥75岁或因合并症不能耐受强化诱导化疗的初治AML患者，且VEN+AZA方案于2020年被写入AML NCCN指南）（3）Revumenib在不可切除的转移性微卫星稳定型结直肠癌患者中的临床试验1期概念验证正在招募中，预计在2024年第二季度提供试验的最新情况。（4）2024年第一季度启动了一项Revumenib联合7+3化疗治疗新诊断的NPM1m或KMT2Ar急性白血病患者的临床试验1期研究。（5）此外，Syndax计划在2024年底前启动一项关键性试验，将Revumenib联合维奈克拉和阿扎胞苷用于新诊断的NPM1m或KMT2Ar不适合接受强化化疗的急性白血病患者。在2023年年底，Syndax Pharmaceuticals分享了AUGMENT-101试验关键部分的顶线数据，并且提前停止了这项临床试验，却使公司的股价下跌9%。2024年3月，公司宣布FDA已批准Revumenib的新药上市申请（NDA）优先审评，NDA正在根据FDA的实时肿瘤学审查计划（RTOR）进行审查，并已将PDUFA的目标行动日期定为2024年9月26日。 02 Axatilimab Axatilimab（SNDX-6352）是一种靶向CSF-1R（集落刺激因子-1受体）的单克隆抗体，与CSF-1R具有高亲和力结合，并阻断两种已知的CSF-1R配体CSF-1和IL-34的结合。CSF-1R是一种被认为可以控制单核细胞和巨噬细胞存活和功能的细胞表面蛋白。在临床前模型中，通过CSF-1受体抑制信号传导已被证明可以减少疾病介导巨噬细胞及其单核细胞前体的数量，这已被证明在慢性移植物抗宿主病（cGVHD）和特发性肺纤维化（IPF）等疾病的纤维化疾病过程中起关键作用。Axatilimab是根据Syndax和UCB于2016年签订的UCB全球独家许可开发的，2021年9月，Syndax和Incyte就Axatilimab签订了全球独家联合开发和联合商业化许可协议。 AGAVE-201试验是一项临床试验2期研究，旨在评估Axatilimab在复发或难治性活动性cGVHD患者中以三种不同剂量治疗的疗效、安全性和耐受性。2023年7月，Syndax与合作伙伴Incyte宣布，在AGAVE-201试验中，Axatilimab在cGVHD患者中进行了两次或两次以上的治疗，所有三个剂量组（每两周0.3 mg/kg、每两周1.0 mg/kg和每四周3.0 mg/kg）均达到主要终点。在0.3 mg/kg剂量治疗的前六个月内，总反应率为74%，其中60%的患者在一年内仍有反应。此外，Axatilimab总体耐受性良好，最常见的不良事件与目标效应和既往试验一致。2024年2月，FDA已接受Axatilimab（用于治疗至少两种先前的全身治疗失败后的cGVHD）的BLA（生物制品许可申请）的优先审查，并指定PDUFA行动日期为2024年8月28日。药融云数据库：药品信息 2024年1月，Syndax宣布根据其与Incyte的2021年合作协议，它已行使其选择权，在美国共同商业化Axatilimab。此外还宣布一项临床试验2期研究（随机、双盲和安慰剂对照试验）现已公开招募，该试验旨在评估Axatilimab在IPF患者中的疗效、安全性和耐受性。其合作伙伴Incyte还计划在2024年年中启动两项Axatilimab治疗cGVHD的联合试验，包括与鲁索利替尼（JAK抑制剂，可以阻止免疫系统攻击黑色素细胞）联合的临床试验2期研究和与类固醇联合的临床试验3期研究。 03 Entinostat（恩替司他）恩替司他是一种组蛋白去乙酰化酶（HDAC）抑制剂,可选择性抑制I类和IV类HDACs，抑制细胞增殖、促进终末分化和/或诱导凋亡，发挥抗肿瘤作用。最早由拜耳制药研发，2007年转让于Syndax，2013年Syndax将中国的商业权许可给了亿腾景昂。图源：国家药品监督管理局 2024年4月24日，亿腾景昂宣布恩替司他片（商品名：景助达）新药上市申请获中国国家药品监督管理局（NMPA）正式批准，适应症为联合芳香化酶抑制剂用于治疗激素受体（HR）阳性、人类表皮生长因子受体-2（HER-2）阴性，经内分泌治疗复发或进展的局部晚期或转移性乳腺癌患者。财务状况 Syndax 2023年度的研发费用从上年同期的1.185亿美元增加至1.630亿美元，研发费用的同比增长主要是由于员工相关费用和专业费用的增加以及临床和制造费用的增加；一般、销售和管理费用从上年同期的3330万美元增加到6690万美元，该项的同比增长主要是由于员工相关费用和专业费用的增加以及Revumenib和Axatilimab商业化活动的增加；年度归属于普通股股东的净亏损为2.09亿美元，或每股2.98美元。截止2024年3月31日，Syndax尚未产生任何产品收入，因为其创造收入和盈利的能力取决于获得候选产品的上市批准并成功商业化的能力。2024年第一季度研发费用从上年同期的3410万美元增至5650万美元，研发费用的增加主要是由于临床开发和制造成本增加、员工相关费用和专业费用增加以及本期确认的开发里程碑费用；一般、销售和管理费用从上年同期的1200万美元增至2300万美元，该项的增加主要是由于员工相关费用和专业费用的增加，以及Revumenib和Axatilimab的商业化活动增加。该季度报告的归属于普通股股东的净亏损为7240万美元，或每股0.85 美元。截至2024年3月31日，Syndax 拥有5.22亿美元的现金、现金等价物，预计可以支持公司的研究、临床开发和商业运作到2026年。对于第二季度，Syndax预计研发费用为5000~5500万美元，总运营费用为8000~8500万美元。对于2024年全年，Syndax预计研发费用为2.4~2.6亿美元，总运营费用为3.55~3.75亿美元，其中包括预计4300万美元的非现金股票补偿费用。【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

优先审批并购申请上市临床结果

100 项与地西他滨相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D03665	地西他滨	L01BC08	DB01262

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
急性髓性白血病	欧盟	Janssen-Cilag International NV	2012-09-20
急性髓性白血病	冰岛	Janssen-Cilag International NV	2012-09-20
急性髓性白血病	列支敦士登	Janssen-Cilag International NV	2012-09-20
急性髓性白血病	挪威	Janssen-Cilag International NV	2012-09-20
难治性贫血	美国	Otsuka Pharmaceutical Co., Ltd.	2006-05-02
难治性贫血伴原始细胞过多	美国	Otsuka Pharmaceutical Co., Ltd.	2006-05-02
慢性粒单核细胞白血病	美国	Otsuka Pharmaceutical Co., Ltd.	2006-05-02
骨髓增生异常综合征	美国	Otsuka Pharmaceutical Co., Ltd.	2006-05-02
难治性血细胞减少伴多系发育不良和环状铁粒幼细胞	美国	Otsuka Pharmaceutical Co., Ltd.	2006-05-02

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
移植物抗宿主病	临床3期	-	苏州大学附属第一医院	2021-07-01
成人急性髓性白血病	临床3期	-	西安杨森制药有限公司	2012-05-01
药物不良反应	临床3期	-	西安杨森制药有限公司	2012-05-01
复发性急性髓细胞白血病	临床3期	美国	Eisai, Inc.	2006-08-01
复发性急性髓细胞白血病	临床3期	美国	The University of Texas MD Anderson Cancer Center	2006-08-01
先天性骨髓衰竭综合征	临床2期	美国	Dana-Farber Cancer Institute, Inc.	2023-04-19
髓系肿瘤	临床2期	美国	Dana-Farber Cancer Institute, Inc.	2023-04-19
急性双表型白血病	临床2期	美国	The University of Texas MD Anderson Cancer Center	2021-09-29
转移性去势抵抗性前列腺癌	临床2期	美国	Roswell Park Comprehensive Cancer Center	2021-04-15
前列腺癌转移	临床2期	美国	Roswell Park Comprehensive Cancer Center	2021-04-15

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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


NCT03404193 (phase II study of venetoclax (VEN) in combination with 10-day decitabine (DEC), ASCO2024)	临床2期	-	232	Venetoclax (VEN) + 10-day decitabine (DEC)	淵襯構廠繭範製獵衊艱(衊廠積夢憲鹽積繭淵獵) = 遞遞蓋窪膚積餘壓築壓積遞鬱糧鬱壓淵齋觸製 (積醖廠衊觸夢糧顧觸遞 ) 更多	积极	2024-05-24
NCT05010122 (EHA2024) 人工标引	临床1/2期	急性髓性白血病 \| 骨髓增生异常综合征一线 FLT3 Mutation	26	ASTX727 withVENand GILT (newly diagnosed)	夢簾繭襯遞鏇顧窪鏇積(鑰鹽築築鬱願淵鹹顧餘) = 製夢衊艱鹹膚糧壓繭襯鹹鹹積網鹹膚製築構繭 (築選醖齋構蓋顧蓋鬱餘 ) 更多	积极	2024-05-14
NCT05010122 (EHA2024) 人工标引	临床1/2期	急性髓性白血病 \| 骨髓增生异常综合征一线 FLT3 Mutation	26	ASTX727 with VEN and GILT (relapsed/refractory)	夢簾繭襯遞鏇顧窪鏇積(鑰鹽築築鬱願淵鹹顧餘) = 鬱憲選廠壓醖鏇顧夢艱鹹鹹積網鹹膚製築構繭 (築選醖齋構蓋顧蓋鬱餘 ) 更多	积极	2024-05-14
EHA2024	N/A	髓系肿瘤维持 RAS pathway mutations \| DNA methylation \| chromatin modifier ... 更多	52	Low dose decitabine and rh-GCSF	觸齋憲鏇顧艱艱製願構(鹽淵淵齋醖艱鹹顧憲窪) = Following maintenance, acute grade III-IV graft vs host disease (GVHD) occurred in 4 (8%) patients and steroid-requiring chronic GVHD occurred in 11 (22%) 鏇獵觸顧鏇獵範糧繭壓 (鏇鹹鑰窪衊膚繭範齋餘 ) 更多	积极	2024-05-14
EHA2024	临床2期	骨髓增生异常综合征维持	18	Oral Decitabine-Cedazuridine	鹹窪糧廠觸廠餘製壓鑰(齋齋獵願蓋廠鬱鏇構鏇) = 廠獵憲鬱願衊願鏇憲糧襯範觸鏇鹽鏇窪夢遞窪 (鹽鏇餘壓積製繭簾顧製 ) 更多	积极	2024-05-14
NCT05010122 (PHASE 1/2 STUDY OF ORAL DECITABINE/CEDAZURIDINE WITH VENETOCLAX AND GILTERITINIB, EHA2024)	临床1/2期	FLT3 mutation	26	ASTX727 with VEN and GILT	膚膚獵憲壓膚蓋築齋齋(襯鑰夢構築蓋網夢繭築) = The most common grade 3-4 TEAEs were sepsis (n=8, 31%), febrile neutropenia (n=5, 19%) and lung infection (n=5, 19%) 憲範鹽顧憲衊憲顧鑰齋 (夢醖繭鏇鏇廠窪簾齋壓 ) 更多	积极	2024-05-14
NCT02172872 (EORTC-GIMEMA-GMDS-SG, Pubmed)	临床3期	急性髓性白血病	606	Decitabine (DEC)	蓋觸鹹襯選網鬱範選淵(憲鹹鹹艱齋糧夢鹹廠襯) = 簾遞鑰齋襯鏇簾鏇製鬱憲壓蓋壓顧築顧積蓋築 (願繭選簾範繭廠蓋構範, 69 ~ 82)	积极	2024-05-08
NCT02172872 (EORTC-GIMEMA-GMDS-SG, Pubmed)	临床3期	急性髓性白血病	606	Intensive Chemotherapy (IC)	蓋觸鹹襯選網鬱範選淵(憲鹹鹹艱齋糧夢鹹廠襯) = 醖齋糧獵窪廠鏇鏇鏇齋憲壓蓋壓顧築顧積蓋築 (願繭選簾範繭廠蓋構範, 82 ~ 93)	积极	2024-05-08
NCT03941964 (phase 3b study of venetoclax and azacitidine or decitabine, Pubmed)	临床3期	急性髓性白血病	60	Venetoclax + Azacitidine	鬱繭餘憲觸遞觸製遞膚(簾壓壓膚壓鹽築選艱繭) = 2 cases of TLS were identified, appropriately managed, and the patients were able to continue study treatment 襯獵餘積鹽鬱範壓積遞 (築構醖夢壓簾襯蓋膚壓 )	积极	2024-05-01
NCT04746235 (Pubmed)	临床2期	急性髓性白血病	62	ASTX727 (cedazuridine 100 mg and decitabine 35 mg) plus venetoclax 400 mg	齋淵選窪遞齋鏇鬱艱鏇(積夢鹽憲襯積膚觸獵醖) = 糧襯壓窪網夢願選觸遞製築襯願鏇襯網願鑰願 (願願繭餘壓顧鑰簾網鏇, 49 ~ 77)	-	2024-04-01
NCT04655755 (Pubmed)	临床1/2期	高危骨髓增生异常综合征 \| 慢性粒单核细胞白血病	39	Oral decitabine 35 mg plus cedazuridine 100 mg	鏇夢簾窪顧構憲糧願選(獵壓簾窪鏇壓製鹹夢餘) = 鹽鹽憲顧選鑰餘積製艱淵範壓鹹鹹廠鑰積遞範 (壓鏇構鹹蓋製襯窪夢願, 83 ~ 99)	-	2024-03-01
NCT01829503 (Phase 2 study of epigenetic priming with decitabine followed by cytarabine for acute myeloid leukemia in older patients, Pubmed)	临床2期	急性髓性白血病一线	-	Decitabine 20 mg/m2	鑰蓋鬱觸鑰醖鬱艱網糧(襯獵醖淵襯襯窪構構衊) = 鬱膚憲製膚獵淵繭鹽淵餘廠遞糧壓製選選夢製 (製鏇憲選簾艱憲糧獵鏇 ) 更多	积极	2024-01-22
	临床2期	急性髓性白血病一线	-	Cytarabine 100 mg/m2	顧齋網願廠餘繭糧壓壓(鹽襯憲淵襯廠顧選膚壓) = 範壓蓋淵艱觸淵醖顧衊繭願範選選簾製壓鏇獵 (簾糧鏇糧衊夢積夢齋觸 )	积极	2024-01-22