A Phase 1 Study of Oral Cedazuridine and Decitabine Combination (ASTX727, NSC# 820631) and Filgrastim as Maintenance Therapy Post-Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia

This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that does not respond to treatment (refractory) who have undergone allogenic hematopoietic stem cell transplantation. ASTX727 is a combination of cedazuridine and decitabine. 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. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Filgrastim stimulates the production of neutrophils (a type of white blood cell) which can help to prevent infection. Giving ATSX727 and filgrastim may be safe and tolerable in treating children with high risk, recurrent or refractory acute myeloid leukemia who have undergone allogenic hematopoietic stem cell transplantation.

开始日期2026-10-30

申办/合作机构

National Cancer Institute

NCT07437950

/ Not yet recruiting临床2期IIT

A Randomized Phase II Trial of ASTX727 With Standard Duration Versus Shorter Duration of Venetoclax in Genomically Heterogenous AML Among Adults Aged 60 or Older and Less Fit for Intensive Therapy: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial compares ASTX727 with standard duration versus shorter duration of venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML). ASTX727 is a combination of decitabine and cedazuridine. Decitabine is in a class of medications called hypomethylation agents. It works 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. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Shorter duration venetoclax may be as effective as standard duration venetoclax when given with ASTX727 for the treatment of newly diagnosed AML.

开始日期2026-10-14

申办/合作机构

National Cancer Institute

NCT07153497

/ Not yet recruiting临床2期IIT

A Randomized Phase 2 Trial of Olutasidenib-Based Therapies in Patients With Newly Diagnosed IDH1-Mutant Myeloid Malignancies: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment substudy tests the addition of olutasidenib to usual treatment in patients with higher-risk myelodysplastic syndrome (MDS) or patients with acute myeloid leukemia (AML) with a mutation in the IDH1 gene. Olutasidenib blocks the protein made by the mutated IDH1 gene. Blocking this protein may help keep cancer cells from growing. For patients with MDS, olutasidenib will be added to decitabine-cedazuridine (also called ASTX727). Decitabine is in a class of medications called hypomethylating agents and is the standard treatment for MDS. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. The cedazuridine makes it possible to take the decitabine by mouth. Adding olutasidenib to the usual treatment for MDS (ASTX727) may increase the likelihood of going into remission. For patients with AML, olutasidenib and ASTX727 will be combined with venetoclax, a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. Venetoclax may stop the growth of cancer cells by blocking BCL-2, a protein needed for cancer cell survival. Adding olutasidenib to the usual treatment for AML (ASTX727 and venetoclax) may increase the likelihood of going into remission. For low risk MDS, the substudy tests whether giving olutasidenib alone helps improve blood counts.

开始日期2026-05-27

申办/合作机构

National Cancer Institute

100 项与地西他滨/西达尿苷相关的临床结果

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100 项与地西他滨/西达尿苷相关的转化医学

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100 项与地西他滨/西达尿苷相关的专利（医药）

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项与地西他滨/西达尿苷相关的文献（医药）

2026-03-10·Blood Advances

Venetoclax/FluBu2 RIC transplant followed by all-oral venetoclax/decitabine maintenance for poor-risk MDS/AML

Article

作者: Stone, Richard M. ; Fiorilla, Jade ; DeAngelo, Daniel J. ; Auriemma, Elise ; Murdock, H. Moses ; Fell, Geoffrey ; Kelkar, Amar H. ; Panaro, Kevin M. ; Garcia, Jacqueline S. ; Lindsley, R. Coleman ; Antin, Joseph H. ; Cutler, Corey S. ; Ryan, Jeremy ; Soiffer, Robert J. ; Shapiro, Roman ; Gooptu, Mahasweta ; Lim, Felicia ; Brock, Jennifer ; Bosch-Vilaseca, Anna ; Bat-Erdene, Denbaa ; Ritz, Jerome ; Abel, Gregory A. ; Letai, Anthony ; Kim, Haesook T. ; Ho, Vincent T.

Abstract:

To improve the tolerability of posttransplant maintenance and outcomes despite poor-risk disease genetics, we conducted a phase 1 study of venetoclax (Ven) with FluBu2 (fludarabine/busulfan) reduced-intensity chemotherapy transplantation using tacrolimus/methotrexate graft-versus-host disease (GVHD) prophylaxis, followed by all-oral Ven/decitabine-cedazuridine (Ven/Dec-c) maintenance in patients with poor-risk MDS/AML (myelodysplastic syndrome/acute myeloid leukemia; N = 30). Overall, 58% had previous Ven exposure and 63% had TP53 mutation; 15/19 had TP53 multihit state. At a median of +55 days, prophylactic maintenance therapy with Ven (400 mg on days 1-14) and Dec-c (35 mg decitabine and 100 mg cedazuridine tablet on days 1, 3, and 5, or days 1, 2, and 3) was initiated for 8 cycles of 42 days each in 26 of 30 patients (87%; of the remaining patients, 3 relapsed early and 1 withdrew). On maintenance, grade 3/4 neutropenia (96%) occurred although infections were rare (n = 2). No dose-limiting toxicities occurred. The 6-month acute GVHD grade 2 to 4 rate was 13%. The 1-year moderate/severe chronic GVHD rate was 31%. At a median follow-up of 25.1 months (range, 15-33), median overall survival (OS) and progression-free survival (PFS) were not reached. On maintenance, 2-year OS was 77%, PFS was 62%, nonrelapse mortality was 0%, and cumulative incidence of relapse was 38%. Exploratory studies identified 96% of patients had pretransplant next-generation sequencing molecular residual disease (MRD) positivity, favorable survival in those with non-TP53 MRD positivity, and delayed conversion on maintenance in 11 of 18 (61%) with TP53-MRD positivity. Patient-reported outcomes assessed in the first 6 months of maintenance were stable except for emotional function, which significantly improved. This trial was registered at www.ClinicalTrials.gov as NCT03613532.

2026-02-10·Blood Advances

Decitabine-cedazuridine in patients with MDS and TP53 mutations

Article

作者: Griffiths, Elizabeth A. ; Urrutia, Samuel ; Garcia-Manero, Guillermo ; Sano, Yuri ; Savona, Michael ; Bataller, Alex ; McCloskey, James ; Sasaki, Koji ; Keer, Harold N. ; Oganesian, Aram ; Montalban-Bravo, Guillermo ; Zeidan, Amer ; Kantarjian, Hagop ; Yee, Karen

Patients with myelodysplastic syndromes (MDS) harboring TP53 mutations have poor outcomes. The objective of this study is to evaluate patients with TP53 mutations treated in the phase 2/3 studies of decitabine-cedazuridine (DEC-C) in MDS. We divided patients into three groups: TP53wt, TP53single-hit, and TP53multi-hit. We then performed propensity matching of patients who were treated with DEC-C vs a cohort of patients treated with parenteral hypomethylating agents (HMA). 180 patients were analyzed of which 73 (40.5%) had TP53 mutations, 23 (12%) with TP53multi-hit. Patients with TP53multi-hit more frequently had complex cytogenetics (69.5%), and had fewer median number of co-mutations (2.5, IQR 2-3) compared to TP53single-hit(3, IQR 1-5), or TP53wt (4, IQR 3-6) (p=0.002). Patients with TP53multi-hit had a higher chance of lack of response with 39.1% vs 28.1% for TP53single-hit (p=0.2). Patients with TP53mult-hit lost response earlier at 8.2 months, vs 13.2 months for TP53single-hit, and 15.1 months for TP53wt (p=0.1). Median overall survival (mOS) was 11.5 months (95% CI: 8.6 - 19.1) for TP53mult-hit, 22.1 months (95% CI: 14.6 - 35.9) for TP53single-hit, and 31.7 months (95% CI: 19.5 - 51.1) for TP53wt (log-rank, p < 0.005). Propensity scores matched 47 TP53mutpatients treated with DEC-C and 47 TP53mut patients treated with single agent parenteral HMA. Median survival was 13.1 months (95% CI: 8.4 - 21.3) for DEC-C vs 8.0 months (95% CI: 5.2 - 13.0) for single agent parenteral HMA (log-rank, p=0.047). In patients with MDS harboring TP53mut, DEC-C may improve overall survival compared to parenteral HMA.

2026-02-01·Therapeutic Advances in Hematology

Real-world study of use patterns and clinical outcomes for patients with myelodysplastic syndrome initiating oral decitabine and cedazuridine or intravenous/subcutaneous hypomethylating agents

Article

作者: Salimi, Tehseen ; Zeidan, Amer M. ; Zhao, Ruizhi ; Tepsick, Jon G. ; Lan, Ziyu

Background::

Decitabine and cedazuridine (DEC-C) is the only oral hypomethylating agent (HMA) approved for myelodysplastic syndromes (MDS) in the United States. The other HMAs approved for MDS, decitabine and azacitidine, are administered intravenously (IV) or subcutaneously (SC).

Objectives::

This study examined real-world outcomes among MDS patients treated with oral DEC-C compared to IV/SC HMAs.

Design::

Adult patients diagnosed with MDS who received HMAs as their first treatment on or after July 1, 2020 through February 2024 in ConcertAI’s RWD360 ® electronic medical records dataset linked to open claims were included in the study.

Methods::

We used propensity score matching (PSM) to balance potential confounders. Kaplan–Meier survival analysis was utilized to compare real-world overall survival (rwOS), acute myeloid leukemia (AML)-free survival, and time to next treatment (rwTTNT) among patients treated with oral DEC-C and IV/SC HMAs.

Results::

A total of 2101 patients with MDS were included, with 405 treated with oral DEC-C and 1696 treated with IV/SC HMA. After PSM, there were 399 patients in each treatment group; demographic and clinical factors were well balanced. Patients treated with oral DEC-C had a similar median rwOS (22.7 months vs 19.5 months; p = 0.57) and AML-free survival (16.1 months vs 14.3 months; p = 0.10) compared with patients who received IV/SC HMA; however, there were nonsignificant trends in favor of oral DEC-C. The median rwTTNT was significantly longer for the oral DEC-C patients than for the IV/SC HMA patients (9.3 months vs 7.8 months, respectively; p = 0.02).

Conclusion::

This real-world study is the largest to date to examine clinical outcomes among MDS patients who initiated oral DEC-C compared to IV/SC HMAs. While study results indicate comparable rwOS and AML-free survival among patients treated with oral DEC-C or with IV/SC HMAs, patients treated with oral DEC-C had a significantly longer rwTTNT. These findings support the use of oral DEC-C as an alternative to parenteral HMA therapy.

项与地西他滨/西达尿苷相关的新闻（医药）

2026-01-26

·雪球

Syndax Pharmaceuticals：双产品商业化加速，前线拓展开启新篇章

$Syndax(SNDX)$摘要：SyndaxPharmaceuticals已成功转型为一家拥有双引擎驱动的商业化阶段肿瘤生物制药公司。其核心资产—首创口服menin抑制剂Revuforj(revumenib)和首创CSF-1R抑制剂Niktimvo(axatilimab)—均已获得FDA批准上市，并展现出成为各自领域“重磅炸弹”级药物的明确潜力。本文认为，Syndax正处于一个关键的市值拐点，其价值不仅体现在已获批的复发/难治性（R/R）适应症的快速商业化渗透，更在于其前瞻性且执行力强的临床开发管线，旨在将两款产品推向更广阔的一线（1L）治疗市场。核心结论如下：1.产品优势突出：Revuforj在KMT2Ar/NPM1m急性白血病中显示出深度且持久的疗效，安全性可控；Niktimvo为cGVHD治疗带来了全新的、针对纤维化核心机制的治疗选择，疗效显著且耐受性良好。2.商业化开局强劲：两款产品上市后收入增长曲线均超越同类药物早期表现，反映出卓越的产品竞争力和高效的商业执行能力。Niktimvo在上市首个完整季度即为公司贡献利润。3.管线价值深挖潜力巨大：通过多项精心设计的临床研究（如SAVE、与强化化疗联用、前线cGVHD试验），公司正系统性地将产品推向早期治疗线，市场总可触达空间（TAM）有望从目前的数十亿美元扩展至百亿美元级别。4.清晰的盈利路径：随着收入规模的快速增长和对Incyte的里程碑付款义务逐步减少，公司正向自给自足和盈利迈进。5.风险与机遇并存：面临的主要风险包括前线临床试验失败、市场竞争加剧及定价压力。然而，其首创药物的先发优势、强大的临床数据包及全面的商业化布局构成了坚实的护城河。基于对管线销售峰值的预估，我们预计Syndax两款核心产品的全球销售峰值有望达到50-70亿美元，其中Revuforj约占60-70%。当前市值尚未充分反映其前线适应症的潜力。一、公司概览：从临床开发到商业化的成功跨越SyndaxPharmaceuticals的战略聚焦于肿瘤与纤维化疾病领域未满足的医疗需求。公司发展历程体现了从生物科技公司向综合性生物制药企业的成功演变。2024年，随着Revuforj和Niktimvo相继获得FDA批准，Syndax正式进入商业化阶段，标志着其从一个纯粹的研发实体转型为拥有稳定现金流和明确增长路径的成熟企业。公司的核心竞争力在于其“精准靶向，深度开发”的策略。针对经过基因筛选的患者群体（如KMT2Ar/NPM1mAML），Revuforj提供了变革性的治疗选择；而针对疾病的核心病理机制（如cGVHD中的巨噬细胞驱动纤维化），Niktimvo开辟了全新的治疗路径。这种“患者细分”与“机制创新”的结合，使得产品在上市后能快速获得市场认可。二、核心产品管线深度分析：机制、数据与市场定位1.Revuforj(revumenib)：PPIbreaker重新定义Menin抑制剂的治疗标准白血病是一种发生在骨髓中的血液癌症，骨髓是正常血细胞生成的场所。未成熟的细胞会发生基因变化，从而形成急性白血病细胞（即原始细胞）。急性髓系白血病，又称AML。大约10%的急性白血病是由KMT2A基因的重排所导致的（即KMT2Ar）。据估计，超过95%的KMT2A型急性白血病患者存在KMT2A易位现象。NPM1突变型急性髓系白血病（NPM1mAML）是一种由NPM1基因突变所导致的白血病。这种突变是常见的遗传变异类型，在约30%的急性髓系白血病患者中被发现。与KMT2A重排型急性白血病类似，NPM1m急性髓系白血病高度依赖于特定发育基因的表达，而这些基因的表达会受到与menin-KMT2A交互作用的抑制剂的负面影响。作用机制：Revuforj是一种高选择性、强效的口服menin抑制剂。Menin蛋白是HOX基因表达的关键调控因子，而HOX基因的异常表达是KMT2A重排和NPM1突变急性白血病的核心致癌驱动因素。通过阻断menin-KMT2A复合物的形成，revumenib能特异性抑制白血病细胞的增殖并诱导分化。临床数据深度解读：注册试验与真实世界证据（RWE）的一致性：关键AUGMENT-101试验中，Revuforj在R/RKMT2ArAML患者中实现了高达63%的完全缓解率（CR/CRh）。来自Moffitt癌症中心的早期真实世界数据进一步验证了其在临床实践中的卓越疗效：在包含多线治疗、多种基因亚型（KMT2Ar,NPM1m,NUP98r）的17名患者中，总体缓解率（ORR）达77%，MRD阴性率达75%。这种“试验-现实”疗效的高度一致性，极大地增强了医生处方的信心。安全性成为差异化优势：与许多靶向药物不同，Revuforj表现出良好的耐受性。关键不良反应如QTc间期延长和分化综合征（DS）发生率较低、严重程度可控，且极少导致治疗中止。这支持了其长期给药，特别是在维持治疗和联合治疗方案中的应用。市场定位：作为首个且唯一获批的menin抑制剂，Revuforj在R/R市场已建立起领导地位。其目标不仅是巩固在R/RKMT2Ar和NPM1mAML患者中的使用，更是通过前线临床试验，将治疗窗口大幅前移，覆盖初诊患者和移植后维持治疗，从而将目标患者群体扩大数倍。2.Niktimvo(axatilimab)：首个靶向巨噬细胞的cGVHD治疗药物慢性移植物抗宿主病（GVHD）是一种由供体来源的造血细胞对受体组织产生的免疫反应，它是异基因造血干细胞移植（HSCT）的一种严重且可能危及生命的并发症，这种并发症可能会持续数年。GVHD的病理机制由慢性炎症和纤维化所驱动，这可能导致终末器官损伤，且该疾病通常会在多个器官系统中表现出来，皮肤和黏膜是常见的受累部位。作用机制：CSF-1/CSF-1R通路是慢性移植物抗宿主病（GVHD）中的关键通路，它调控单核细胞的增殖以及巨噬细胞的分化和活性。CSF-1R信号传导对于交替极化的巨噬细胞的发育和功能至关重要，这些细胞能够发挥促纤维化和炎症作用。在慢性GVHD中，单核细胞和巨噬细胞能够介导炎症和纤维化，导致组织损伤。Niktimvo是一种人源化单克隆抗体，通过靶向集落刺激因子1受体（CSF-1R），耗竭介导组织炎症和纤维化的单核细胞和巨噬细胞。这种机制直接针对慢性移植物抗宿主病（GVHD）和特发性肺纤维化（IPF）等疾病的病理核心，与传统免疫抑制剂的作用路径截然不同。临床数据深度解读：AGAVE-201试验的突破性成果：AGAVE-201是一项国际多中心、随机化、关键性II期临床研究。该研究纳入了241例经二线及二线以上治疗失败的难治性cGVHD患者。患者被随机分配至0.3mg、1mg和3mg剂量组，并接受每两周一次（0.3mg剂量组和1mg剂量组）或每四周一次（3mg剂量组）的静脉给药。结果显示：0.3mg组、1mg组、3mg组的的总体应答率分别为74%、67%和50%，其中分别有60%、69%和41%达到有临床意义的症状缓解（mLSS评分降低≥5分）；Niktimvo给药后显示出快速而持续的治疗效果，其中位应答时间小于2个月，中位缓解持续时间达到11.1个月；在器官特异性疗效中，胃肠道缓解率最高，上、下消化道症状缓解率分别为82%、89%，皮肤和眼部症状的缓解率也为26%-30%。长期耐受性数据坚实：长期随访（中位超2.8年）显示，安全性特征稳定，未出现新的累积性毒性。46个月的总生存（OS）率达74%，对于重度难治性cGVHD患者而言是一个卓越的结果。向一线治疗拓展的强劲信号：早期与ruxolitinib联用的数据显示，该组合作为潜在的类固醇减免方案耐受性良好。这为正在进行的、旨在评估Niktimvo+皮质类固醇vs.皮质类固醇单药一线治疗cGVHD的大型全球III期试验（AXemplify-357）奠定了基础。市场定位：Niktimvo已在≥3LcGVHD市场建立了“新标准疗法”的地位。其最大价值在于向1LcGVHD的拓展，若能成功，将直接进入一个患者基数更大、治疗周期更长的市场。同时，进军特发性肺纤维化（IPF）（一个患者群体远超cGVHD的领域）的临床计划，为其打开了另一个价值数十亿美元的想象空间。三、关键临床进展解读：通往一线治疗的路线图Syndax的临床开发策略具有高度的协同性和侵略性，旨在最大化每个产品的生命周期价值。1.Revuforj的前线组合策略：SAVE方案（ASTX727+BCL-2抑制剂+Menin抑制剂）：针对不适合强化化疗的老年/unfitAML患者。数据表明，这种全口服三联方案疗效惊人（CR/CRh81%），且安全性可控。这有望在未来成为一种新的标准治疗方案，极大提高患者的生活质量。与强化化疗（IC）联合：针对适合强化化疗的较年轻、fit的患者。早期数据显示，Revuforj的加入没有增加IC的预期外毒性，并带来了接近100%的响应率和极高的MRD阴性率。这直接支持了在III期REVEAL-ND研究中，将Revuforj+IC作为fit的NPM1mAML患者一线治疗方案进行验证。2.Niktimvo的管线拓展：一线cGVHD（AXemplify-357）：这是价值创造的关键试验。如果成功，Niktimvo将从“最后的选择”变为“早期的支柱”，显著扩大市场渗透率和治疗时长。IPF（MAXPIRe试验）：基于CSF-1R在肺纤维化中的核心作用，该拓展具有坚实的科学基础。IPF全球市场庞大，现有疗法仅能减缓疾病进展，Niktimvo有望提供一种全新的、针对疾病根源的治疗选择。四、商业化表现与销售峰值预测分析当前商业化表现：Revuforj：上市后收入增长迅猛，超越了过去其他AML新药上市初期的增长速度。公司预计到2025年底将治疗1000名KMT2Ar患者。NPM1m适应症的获批带来了显著的需求拐点。强大的医保覆盖和NCCN指南的纳入为其扫清了支付障碍。Niktimvo：表现更为亮眼，上市8个月内销售额即按年化计算接近2亿美元，并在上市后第一个完整季度为Syndax贡献了利润。这得益于其与Revuforj在血液肿瘤领域的商业协同效应，以及cGVHD领域对创新疗法的高度渴望。销售峰值（PeakSales）预测模型：我们对Syndax两款核心产品的全球销售峰值进行分情景预估。核心假设基于：目标患者人群流行病学数据、当前及未来预期治疗线中的渗透率、定价假设（参考美国及全球主要市场）、治疗时长以及竞争格局。预测总结与情景分析：基本情景（概率最高）：假设前线关键III期试验（如REVEAL-ND,AXemplify-357）取得阳性结果，但面临一定竞争。在此情景下，全球销售峰值约为50亿美元（Revuforj~35亿，Niktimvo~15亿）。乐观情景：假设所有前线试验大获成功，迅速改变临床实践，且在IPF等新适应症中取得突破。竞争产品进展缓慢。在此情景下，销售峰值可能冲击70亿美元或更高。保守情景：假设前线试验数据不及预期或竞争加剧，导致渗透率低于预期。在此情景下，销售峰值可能主要依赖当前R/R市场，约为25-30亿美元。五、研发战略与未来增长动力Syndax的研发战略可概括为“纵向深入”与“横向拓展”。1.纵向深入（LifecycleManagement）：全力推进Revuforj和Niktimvo在已获批疾病领域内向更前线治疗阶段的拓展。这是价值增长最确定、最高效的路径。公司设计的临床试验（如SAVE、与IC联合、AXemplify-357）直接针对当前标准治疗的痛点，成功可能性较高。2.横向拓展（NewIndications）：基于核心机制的科学合理性，探索新的疾病领域。Niktimvo在IPF的开发是典型代表。此外，menin抑制剂在其他依赖HOX通路的恶性肿瘤（如某些实体瘤）中的潜力也值得未来探索。3.外部合作与引进：公司与Incyte就Niktimvo在cGVHD和IPF领域的合作，分担了开发成本和风险。未来，公司可利用商业化产生的现金流，选择性引进早期或临床阶段资产，进一步丰富管线。公司的财务资源能够支持其雄心勃勃的研发计划。截至2025年第三季度，公司拥有充足的现金储备，且随着产品收入的快速增长，对资本市场的依赖度将逐渐降低，走向自我造血。六、投资亮点与2026里程碑投资亮点：1.明确的双重增长引擎：两款已上市产品均具有“重磅炸弹”资质，且各自拥有清晰且潜力巨大的临床拓展路径，风险分散。2.强大的先发优势和临床数据护城河：作为首创药物，建立了深厚的专利壁垒和医生认知。持续的积极临床数据（包括RWE）不断强化其市场领导地位。3.高效的商业化执行力已验证：两款产品上市后快速放量，表明公司拥有出色的市场准入、医学事务和销售团队。4.向盈利拐点迈进：Niktimvo已实现季度盈利，Revuforj收入增长迅猛，公司整体迈向现金流平衡和可持续盈利的路径清晰。5.估值具备吸引力：相对于其管线蕴含的峰值销售潜力，尤其是尚未被充分定价的前线适应症价值，当前市值存在上行空间。2026里程碑：1.将于2026年下半年公布关于axatilimab在特发性肺纤维化（IPF）患者中的总体疗效数据。2.加快revumenib在关键的1线试验中的全球患者招募工作。3.启动revumenib在病情稳定的KMT2A阳性患者中的1L试验七、结论SyndaxPharmaceuticals已经完成了从生物科技公司到商业化生物制药企业的关键一跃。凭借Revuforj和Niktimvo这两款机制独特、疗效卓越的首创药物，公司不仅在当前市场站稳脚跟，更通过极具远见和执行力的临床开发计划，描绘了未来五年乃至更长期的持续增长蓝图。我们相信，Syndax的核心投资逻辑在于其“近期有业绩，远期有故事”的特质。近期的业绩由两款上市产品的快速商业化提供坚实支撑；远期的故事则由其在前线AML和cGVHD，乃至IPF领域的巨大市场拓展潜力所书写。

申请上市

2025-12-11

·觅健

注射用地西他滨2025报销范围

注射用地西他滨2025报销范围2025年，注射用地西他滨已正式纳入国家医保乙类目录，报销比例40%~70%，具体数值由参保类型与地区政策共同决定：城镇职工医保：60%~70%城乡居民医保：40%~50%医惠保/大病保险：在珠海等地，若诊断符合“骨髓增生异常综合征（MDS）”且药品使用适应症与病种匹配，可再获自费项目补偿，进一步降低自付额。价格基准：国产每支约200元，进口约400元；若选用集采中选品种，医保支付线更低，患者实际自付可压缩至每支60~120元区间。注射用地西他滨注意事项血液学毒性：中性粒细胞减少症与血小板减少症发生率极高，每个给药周期前必须复查全血+血小板计数；若出现Ⅲ度以上骨髓抑制，下一周期剂量需下调或延迟。早期预防：建议同步备用G-CSF与抗微生物药物，降低感染性休克风险。分化综合征：表现为发热、肺浸润、外周水肿，死亡率可达5%；一旦疑诊，立即静脉给予大剂量甲强龙1g/d×3d，并暂停地西他滨直至症状完全缓解。生殖毒性：育龄妇女：治疗期间及停药后至少6个月内需采用双重避孕；建议治疗前完成卵母细胞冷冻保存咨询。男性患者：治疗期及结束后3个月内需避孕，并推荐精子冷冻以防不可逆不育。肝肾功能：CrCl<30ml/min或Child-Pugh B/C者无安全数据，必须减量25%并密集监测；出现胆红素>2×ULN或ALT>5×ULN时应立即停药。新增迟发不良反应：上市后发现间质性肺病、心肌病（射血分数下降）、Sweet综合征，若出现不明原因干咳、气促或LVEF下降>10%，需永久停药并启动相应专科治疗。注射用地西他滨耐药了再吃什么地西他滨耐药机制以DNA甲基化酶再激活与p53通路突变为主，后续选择需基于基因组测序结果分层：TP53突变阳性优先进入临床试验：CD47单抗（Magrolimab）联合阿扎胞苷，ORR 60%以上。靶向口服方案：APR-246（eprenetapopt）联合阿扎胞苷，可逆转p53功能，突变型TP53患者CR率提升至35%。TET2/ASXL1突变阳性口服ASTX727（地西他滨/cedazuridine复方）：生物等效于静脉地西他滨，可绕过胞苷脱氨酶耐药，剂量35mg×5d，每28d重复，Ⅱ期试验显示总缓解率42%。多基因复合突变/高危核型联合免疫化疗：维奈克拉（Venetoclax）+阿扎胞苷：BCL-2抑制增强凋亡，对既往去甲基化药物失败患者ORR 54%，中位OS 10.1个月。FLAG-Ida（氟达拉滨+阿糖胞苷+G-CSF+伊达比星）作为桥接移植的挽救方案，CR率45%~50%。移植候选者立即启动HLA配型；若骨髓原始细胞<10%，可先行低剂量阿糖胞苷+维奈克拉降肿瘤负荷，2~4疗程后进仓，移植后1年DFS约55%。非移植、体能差口服环孢素A+沙利度胺调节免疫，支持治疗为主；或进入CD123-ADC（Tagraxofusp）同情用药计划，BPDCN样表达者仍可能获益。核心策略：耐药后必须重做骨髓NGS与染色体核型，避免单药重复去甲基化治疗；临床试验>靶向联合>挽救化疗是延长生存的三级路径。

2025-11-13

·PRNewswire

Taiho Oncology Announces 14 Presentations, Including Six Oral Presentations, at the 67th American Society of Hematology Annual Meeting and Exposition

Six oral presentations will detail new data on the outcomes of all-oral regimens containing azacitidine and cedazuridine or decitabine and cedazuridine in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia or acute myeloid leukemia PRINCETON, N.J., Nov. 13, 2025 /PRNewswire/ -- Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, announced the presentation of new data from 14 studies across company-sponsored and company-funded externally led studies at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, to be held Dec. 6-9, 2025, in Orlando, Florida. Taiho Oncology will share findings from clinical trials in two oral presentations and evidence from real-world studies in two online abstracts. The oral presentations will highlight results from the Phase 2 part of ASTX030-01, a randomized, open-label, crossover trial comparing oral combination of azacitidine and cedazuridine to subcutaneous azacitidine and the Phase 2 portion of the Phase 1/2 trial ASTX727-03 of low-dose versus standard-dose oral decitabine and cedazuridine in patients with lower-risk myelodysplastic syndromes (MDS). The two online posters will present real-world data on U.S. adults with acute myeloid leukemia (AML), describing treatment patterns and outcomes with hypomethylating agent (HMA) plus venetoclax, and evaluating the time burden of initiating parenteral HMA-based therapy in routine practice. Additionally, 10 presentations – including four oral presentations – led by independent investigators will feature data from Taiho-funded investigator-initiated or collaborative studies. These presentations will highlight: Results from a UK multicenter Phase 2 randomized trial comparing ASTX727 with hydroxycarbamide or best supportive care in advanced MDS/ myeloproliferative neoplasm (MPN). Data from a Phase 2 study evaluating the all-oral combination of ASTX727, venetoclax and revumenib in newly diagnosed AML patients with NPM1m, KMT2Ar or NUP98r AML mutations. Results of a Phase 1/2 clinical trial of the combination of oral decitabine/cedazuridine with the venetoclax in patients with untreated high-risk MDS and chronic myeloid leukemia (CMML). Safety and efficacy results from a Phase 1/2 trial evaluating an all-oral combination of ASTX727, venetoclax and gilteritinib in both relapsed refractory and newly diagnosed FLT3-mutated AML, high-risk MDS or MPN. "As the leader of oral HMA development, we're excited that these data will be featured in six oral presentations at the ASH Annual Meeting," said Harold Keer, MD, PhD, Taiho Oncology Chief Medical Officer. "These data highlight our increasing understanding of novel oral regimens in hematology. We are invested in developing treatments that have the potential to improve flexibility and lower the treatment burden for patients, and collaborating with leading institutions, evidenced by our continued support of independent clinical research to advance cancer care. We look forward to participating in the essential scientific exchange that contributes to progress in hematology at the ASH Annual Meeting." Data being presented by Taiho Oncology and its partners at the 2025 ASH Annual Meeting and Exposition include: Company-Sponsored Studies Oral Presentations Title: A phase 2 dose confirmation trial of oral ASTX030, a combination of oral azacitidine with cedazuridine among patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia Abstract Number: 491 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML Session Date: Dec . 7, 2025 Session Time: 9:30 a.m. to 11a.m. Presentation Time: 10:15 to 10:30 a.m. Location: OCCC - Valencia Room W415D Presenter: Guillermo Garcia-Manero, MD Title: Low-dose oral decitabine and cedazuridine among patients with low-risk myelodysplastic syndromes Abstract Number: 790 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Lower risk Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms Session Date: Dec . 8, 2025 Session Time: 10:30 a.m. to 12 p.m. Presentation Time: 11:15 to 11:30 a.m. Location: OCCC - Valencia Room W415A Presenter: Guillermo Garcia-Manero, MD Online Abstracts Title: Time burden of treatment with parenteral HMAs in combination with venetoclax among patients with newly diagnosed AML Abstract Number: 6990 Lead Author: Amer Zeidan, MD Title: Real-World Characteristics, Treatment Modifications, and Outcomes for AML Patients Receiving HMAs + Venetoclax (1L) Abstract Number: 8176 Lead Author: Amer Zeidan, MD Company-Funded Externally Led Studies Oral Presentations Title: Phase II Study of the All-Oral Combination of Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax (SAVE) in Newly Diagnosed AML Abstract Number: 47 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens Session Date: Dec . 6, 2025 Session Time: 9:30 to 11 a.m. Presentation Time: 10:30 to 10:45 a.m. Location: OCCC - Valencia Room W415A Presenter: Wei-Ying Jen, BM BCh, FRCPath Title: Oral decitabine/cedazuridine in combination with venetoclax in treatment-naïve high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia: Updates of a Phase 1/2 clinical trial Abstract Number: 237 Session Name: 637 . Myelodysplastic Syndromes : Clinical and Epidemiological: Treatment Advances in Higher Risk Myelodysplastic Syndromes Session Date: Dec . 6, 2025 Session Time: 2 to 3:30 p.m. Presentation Time: 2:30 to 2:45 p.m. Location: OCCC - Chapin Theater W415BC Presenter: Alex Bataller, MD, PhD Title: ASTX727 delivers superior response rates and associated survival benefit versus hydroxycarbamide/best supportive care in CMML and other MDS/MPN overlap syndromes: First results from the Phase 2 UK multicenter randomized ammo trial Abstract Number: 488 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML Session Date: Dec. 7, 2025 Session Time: 9:30 to 11 a.m. Presentation Time: 10:45 to 11 a.m. Location: OCCC - Valencia Room W415D Presenter: Daniel H. Wiseman, MB, ChB, PhD Title: Phase I/II Study of Decitabine/Cedazuridine (ASXT727), Venetoclax, and Gilteritinib for Patients with FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Abstract Number: 997 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens Session Date: Dec . 8, 2025 Session Time: 4:30 to 6 p.m. Presentation Time: 4:30 to 4:45 p.m. Location: OCCC - Chapin Theater W320 Presenter: Roberta S. Azevedo, MD Poster Presentations Title: Translational Investigation of Tolinapant (ASTX660) in Acute Myeloid Leukemia Using Integrated Clinical, Bioinformatic, and Pharmacological Approaches Abstract Number: 2552 Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I Session Date: Dec. 6, 2025 Session Time: 5:30 to 7:30 p.m. Location: OCCC - West Halls B3-B4 Title: A Randomized Phase II Trial of ASTX727 and Venetoclax With or Without Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy (MM1OA-S03) Abstract Number: 3466 Session Name: 617 . Acute Myeloid Leukemias: Commercially Available Therapies: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: Trial in progress: A Phase I Study Evaluating the Safety of cirtuvivint (CIRT) as Monotherapy and in Combination with ASTX727 in Patients with MDS and AML Abstract Number: 3431 Session Name: 616 . Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: Trial in Progress - A Randomized Study of ASTX727 With or Without Ladademstat in Accelerated/Blast-Phase Myeloproliferative Neoplasms Abstract Number: 3827 Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: A Comparison of Overall Survival and Quality of Life in MDS Patients Treated with Azacitidine vs Decitabine: A Propensity-Matched Registry Study Abstract Number: 3862 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: A Multiarm Phase 1b Study of Personalized Oral Maintenance Therapy with Decitabine/Cedazuridine (ASTX727) Plus Physician's Choice of Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib in Acute Myeloid Leukemia Abstract Number: 5228 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster III Session Date: Dec. 8, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 About Taiho Oncology, Inc. The mission of Taiho Oncology, Inc. is to improve the lives of patients with cancer, their families and their caregivers. The company specializes in the development and commercialization of orally administered anti-cancer agents for various tumor types. Taiho Oncology has a robust pipeline of small-molecule clinical candidates targeting solid-tumor and hematological malignancies, with additional candidates in pre-clinical development. Taiho Oncology is a subsidiary of Taiho Pharmaceutical Co., Ltd. which is part of Otsuka Holdings Co., Ltd. Taiho Oncology is headquartered in Princeton, New Jersey and oversees its parent company's European and Canadian operations, which are located in Baar, Switzerland and Oakville, Ontario, Canada. For more information, visit , and follow us on LinkedIn and X. Taiho Oncology and the Taiho Oncology logo are registered trademarks of Taiho Pharmaceutical Co., Ltd. Taiho Oncology Contact: Leigh Labrie (609) 664-9878 [email protected] SOURCE Taiho Oncology 21% more press release views with Request a Demo

临床2期ASH会议临床结果临床1期细胞疗法

100 项与地西他滨/西达尿苷相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	地西他滨/西达尿苷	L01XY	DB15694

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	Otsuka Pharmaceutical Netherlands B.V.	2023-09-15
急性髓性白血病	冰岛	Otsuka Pharmaceutical Netherlands B.V.	2023-09-15
急性髓性白血病	列支敦士登	Otsuka Pharmaceutical Netherlands B.V.	2023-09-15
急性髓性白血病	挪威	Otsuka Pharmaceutical Netherlands B.V.	2023-09-15
慢性粒单核细胞白血病	澳大利亚	Otsuka Australia Pharmaceutical Pty Ltd.	2020-10-29
骨髓增生异常综合征	美国	Taiho Oncology, Inc.	2020-07-07

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
难治性贫血	临床3期	美国	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	奥地利	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	加拿大	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	捷克	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	法国	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	德国	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	匈牙利	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	意大利	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	西班牙	Astex Pharmaceuticals, Inc.	2018-02-15
难治性贫血	临床3期	英国	Astex Pharmaceuticals, Inc.	2018-02-15

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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


NCT05360160 (SAVE, ASH2025)	临床2期	急性髓性白血病 NPM1 mutation \| KMT2A rearrangement	17	Decitabine/cedazuridine + venetoclax + revumenib	艱醖蓋糧製簾願齋製製(選艱艱鏇築齋廠壓鹽艱) = QTc prolongation occurred in 8 (47%) pts; 3 were grade 2 (18%), the rest were grade 1 (29%) 鹽壓製衊觸築構觸簾願 (夢醖齋窪獵範顧艱膚獵 ) 更多	积极	2025-12-06
NCT04655755 (ASH2025)	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一线	69	Oral decitabine/cedazuridine + venetoclax	構鬱積膚鹹膚簾憲糧醖(夢願襯窪簾壓淵壓繭鹽) = Patients with ASXL1mut had a median OS not reached (vs 18 months in patients with ASXL1wt, P=0.008) 築製鹹遞壓觸鹹積觸餘 (襯築鑰構簾膚獵淵築壓 ) 更多	积极	2025-12-06
NCT06661915 (ASH2025)	临床2期	骨髓增生性疾病	50	ASTX727 + iadademstat	構廠蓋襯壓願夢齋選蓋(製夢範積鹹構構廠餘糧) = 網範淵積蓋獵衊積顧構憲壓淵製淵糧鹹遞鹹蓋 (窪糧構餘範鬱糧膚顧糧 )	积极	2025-12-06
				ASTX727	構廠蓋襯壓願夢齋選蓋(製夢範積鹹構構廠餘糧) = 簾鑰獵襯鏇鏇艱衊糧齋憲壓淵製淵糧鹹遞鹹蓋 (窪糧構餘範鬱糧膚顧糧 )
NCT06802146 (ASH2025)	临床1期	意义不明的克隆性血细胞减少症	138	DEC/CED	糧築鑰鬱鹹憲範淵構窪(網選積積鬱網築醖廠範) = 鏇鹽糧繭膚選淵糧糧願餘製遞衊簾淵蓋願餘築 (膚範襯觸築範壓窪鏇選 ) 更多	积极	2025-12-06
NCT05010772 (ASH2025)	临床1期	急性髓性白血病维持	35	ASTX727 35/100 mg PO	窪糧遞遞鏇築鹹觸積選(構願製製壓糧夢鹽憲醖) = 簾選夢齋鏇醖醖襯遞衊願鬱繭壓鹹築網選鹹選 (鏇願襯鏇餘顧觸鏇製獵 ) 更多	积极	2025-12-06
				ASTX727 35/100 mg PO + venetoclax 400 mg (azole adjusted) PO	窪糧遞遞鏇築鹹觸積選(構願製製壓糧夢鹽憲醖) = 衊選觸壓顧蓋廠齋夢觸願鬱繭壓鹹築網選鹹選 (鏇願襯鏇餘顧觸鏇製獵 ) 更多
ISRCTN30808508 (AMMO, ASH2025)	临床2期	骨髓增生性疾病	77	ASTX727	遞夢構顧憲網艱蓋廠壓(選鬱艱鹹鏇夢膚憲衊願) = 衊襯築顧鏇襯膚廠餘餘鹹顧壓製願遞糧淵淵衊 (鏇顧範淵積憲簾廠餘壓, 39.3 ~ 66.3) 更多	积极	2025-12-06
				Hydroxycarbamide/Best Supportive Care	遞夢構顧憲網艱蓋廠壓(選鬱艱鹹鏇夢膚憲衊願) = 餘壓夢膚蓋壓鬱選鬱壓鹹顧壓製願遞糧淵淵衊 (鏇顧範淵積憲簾廠餘壓, 14.9 ~ 49.4) 更多
NCT04340843 (ETCTN/NCI 10330, CTgov)	临床2期	软骨肉瘤 \| 中枢神经系统肿瘤 \| 脑转移瘤	19	ASTX727+Belinostat (Treatment (Belinostat, ASTX727))	膚壓構顧構淵餘製艱襯 = 淵憲艱網夢膚鹽廠獵鹽願鏇繭簾觸願襯構觸遞 (淵襯積衊鑰鹽繭淵鹹鏇, 憲繭蓋選選鑰鹽網築憲 ~ 觸糧網淵範艱構網構襯) 更多	-	2025-09-16
				SGI-110+Belinostat (Treatment (Belinostat, SGI-110))	繭糧築簾夢積鏇觸獵範 = 糧顧夢淵醖餘網窪淵窪衊網製鏇膚壓築觸鑰壓 (鏇範鹹網憲製選製鑰觸, 艱選鬱鹹齋網遞餘範艱 ~ 鑰蓋顧簾築繭鏇構鏇襯) 更多
NCT04657081 (ASCO2025) 人工标引	临床2期	急性髓性白血病一线	101	decitabine-cedazuridine (DEC-C)+venetoclax	糧膚獵膚壓範繭鹽願襯(艱顧醖憲衊鏇顧選醖蓋) = 鹽窪鑰範襯選壓壓觸襯憲淵齋鹽窪糧觸廠壓夢 (鑰壓窪齋製壓餘窪齋廠, 36.5 ~ 56.7) 更多	积极	2025-05-23
NCT04657081 (S135, EHA2025)	临床1/2期	急性髓性白血病	189	DECITABINE-CEDAZURIDINE (DEC-C) (DEC-C + VEN)	觸顧範鏇範鬱繭壓齋鏇(範製製鏇醖顧醖鑰蓋窪) = 蓋顧願艱襯醖範膚繭簾齋壓積蓋糧餘鏇積構積 (衊繭鏇顧壓顧鬱構蓋構, 36.5 ~ 56.7) 更多	积极	2025-05-14
NCT05010772 (4277, ASH2024)	临床1期	急性髓性白血病 complex cytogenetics \| antecedent MDS/MPN \| therapy-related AML ... 更多	31	ASTX727 alone	構鏇構憲積遞淵夢繭範(夢積憲築蓋網簾夢願願) = 鹹醖憲鹽襯鏇襯選膚鹹鏇艱糧憲鏇鹽遞蓋壓觸 (簾網築襯糧簾鹽範窪顧 ) 更多	积极	2024-12-09
				ASTX727 plus venetoclax	構鏇構憲積遞淵夢繭範(夢積憲築蓋網簾夢願願) = 獵遞夢簾簾顧壓窪願齋鏇艱糧憲鏇鹽遞蓋壓觸 (簾網築襯糧簾鹽範窪顧 ) 更多