预约演示

更新于：2026-10-05

Decitabine/Cedazuridine

地西他滨/西达尿苷

更新于：2026-10-05

概要

基本信息

药物类型

小分子化药

别名

(4R)-2'-Deoxy-2',2'-difluoro-3,4,5,6-tetrahydrouridine、Decitabine/cedazuridine、Decitabine/E7727

+ [6]

靶点

CDA x DNMT1

作用方式

抑制剂

作用机制

CDA抑制剂(胞嘧啶核苷脱氨酶抑制剂)、DNMT1抑制剂(DNA（胞嘧啶-5）-甲基转移酶-1抑制剂)、表观遗传学药物

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

急性髓性白血病慢性粒单核细胞白血病骨髓增生异常综合征+ [40]

非在研适应症

急性淋巴细胞白血病BCR/ABL阴性非典型性慢性髓系白血病造血干细胞移植+ [6]

原研机构

Astex Pharmaceuticals, Inc.

在研机构

Taiho Oncology, Inc.

Otsuka Australia Pharmaceutical Pty Ltd.

Otsuka Pharmaceutical Co., Ltd.

+ [8]

非在研机构

Forma Therapeutics, Inc.

Pfizer Inc.

权益机构

National Cancer Institute

The University of Texas MD Anderson Cancer Center

Astex Pharmaceuticals, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2020-07-07),

骨髓增生异常综合征

最高研发阶段(中国)临床1/2期

特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (澳大利亚)

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结构/序列

分子式C9H14F2N2O5

InChIKeyVUDZSIYXZUYWSC-DBRKOABJSA-N

CAS号1141397-80-9

查看全部结构式(2)

关联

项与地西他滨/西达尿苷相关的临床试验

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2026-11-27

申办/合作机构

National Cancer Institute

NCT06802523

/ Suspended临床1期IIT

A Phase I Study of Lintuzumab-Ac-225 in Combination With Venetoclax and ASTX-727 in Adults With Newly Diagnosed AML

This phase I trial tests the safety, side effects, and best dose of lintuzumab-Ac225 in combination with venetoclax and ASTX-727, and how well they work in treating patients with newly diagnosed acute myeloid leukemia (AML). Lintuzumab-Ac225 is a monoclonal antibody, called lintuzumab, linked to a radioactive agent called actinium Ac 225. Lintuzumab attaches to CD33 positive cancer cells in a targeted way and delivers actinium Ac 225 to kill them. 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. ASTX-727 is a combination of two drugs, 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. Giving lintuzumab-Ac225 in combination with venetoclax and ASTX-727 may be safe and tolerable in treating patients with newly diagnosed AML and may improve the chance of going into remission and staying in remission for a longer period of time.

开始日期2026-11-18

申办/合作机构

National Cancer Institute

NCT07472322

/ Not yet recruiting临床1/2期IIT

UW26001 Phase I/II Study on the Safety, Tolerability, and Preliminary Efficacy of ASTX727 (Decitabine/Cedazuridine) and Retifanlimab-dlwr in Patients With Advanced Merkel Cell Carcinoma Who Have Progressed on Anti-PD-(L)1 Inhibitor

The goal of this clinical trial is to learn if ASTX727 can be combined with retifanlimab to treat Merkel cell cancer. It will also learn about the safety of combining these drugs. The main questions it aims to answer are:
* Can the combination shrink cancer and lower the chance of the cancer growing or spreading?
* Is the combination better than standard of care for Merkel cell cancer?
Participants will:
* Take oral ASTX727 and retifanlimab through a vein in the arm for about 2 years.
* Visit the clinic once every 2 weeks for checkups and tests

开始日期2026-10-01

申办/合作机构

University of Wisconsin-Madison

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）

2026-06-04·NEW ENGLAND JOURNAL OF MEDICINE

All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia

Article

作者: Chan, Danna ; Roboz, Gail J. ; Gentile, Teresa C. ; Dijkstra, Margit ; Mannis, Gabriel N. ; Amin, Harshad V. ; Fathi, Amir T. ; Bernal del Castillo, Teresa ; Arnan, Montserrat ; Griffiths, Elizabeth A. ; Zeidan, Amer M. ; Montesinos, Pau ; Oganesian, Aram ; Liesveld, Jane L. ; DiNardo, Courtney D. ; Im, Annie P. ; McCloskey, James K. ; Rodríguez-Macías, Gabriela ; Keer, Harold N. ; Cerny, Jan ; Odenike, Olatoyosi ; Wan, Yubing ; Savona, Michael R.

BACKGROUND:

For patients with acute myeloid leukemia (AML) who are 75 years of age or older or who are ineligible for intensive induction chemotherapy, azacitidine or decitabine plus venetoclax is the standard of care, but parenteral administration imposes a burden on patients and providers. Oral decitabine-cedazuridine, approved in Europe for AML, has pharmacokinetic properties equivalent to those of intravenous decitabine but provides limited survival benefit as monotherapy.

METHODS:

In this phase 1-2, open-label, multicenter, nonrandomized trial, we assigned patients with newly diagnosed AML who were 75 years of age or older or who were ineligible for intensive chemotherapy to receive oral decitabine-cedazuridine plus oral venetoclax. To mitigate myelosuppression observed in phase 1, schedule adjustments were encouraged in phase 2b after bone marrow blast clearance. The primary end points were the venetoclax area under the curve from 0 to 24 hours and maximum observed concentration with or without decitabine-cedazuridine (measures of drug interaction) on days 5 and 15 of cycle 2 (phase 1-2a) and complete response (phase 2a-b).

RESULTS:

A total of 189 patients were enrolled (30 patients in phase 1, 58 patients in phase 2a, and 101 patients in phase 2b). No drug-drug interactions were observed between decitabine-cedazuridine and venetoclax. In the pivotal phase 2b, the percentage of patients with a complete response was 47% (95% confidence interval [CI], 36 to 57), the percentage with a complete response or complete response with incomplete hematologic recovery was 63% (95% CI, 53 to 73), and median overall survival was 15.5 months (95% CI, 7.6 to could not be estimated). Common adverse events of grade 3 or higher in phase 2b were anemia (in 30% of the patients), neutropenia (in 26%), and febrile neutropenia (in 25%). Mortality was 3% at 30 days and 10% at 60 days.

CONCLUSIONS:

Among patients with newly diagnosed AML who were ineligible for intensive chemotherapy, all-oral decitabine-cedazuridine plus venetoclax caused no drug interactions and resulted in a complete response in nearly half the patients, with myelosuppressive effects. (Funded by Taiho Oncology; ASCERTAIN-V ClinicalTrials.gov number, NCT04657081.).

2026-04-15·CANCER

A phase 1 study of ASTX727 plus talazoparib in patients with triple‐negative or hormone resistant/HER2‐negative metastatic breast cancer

Article

作者: Emily Douglas MD ; Erin Conder MS, CCRP ; Kathy D. Miller MD ; Sandra Althouse MS ; Kenneth P. Nephew PhD ; Feyruz V. Rassool PhD ; Ryan Burgos BS, MBA ; H. Josh Jang PhD ; Bryan P. Schneider MD ; Woonbok Chung PhD ; Alexandra Thomas MD ; Tarah Ballinger MD ; Yong Zang PhD ; Katherine Ansley MD ; Jean-Pierre Issa PhD

Abstract:

Background:

Poly(adenosine diphosphate ribose) polymerase (PARP) is recruited to DNA damage sites along with epigenetic factors such as DNA methyltransferase 1 (DNMT1). Inhibitors of DNMT modulate reactive oxygen species (ROS)‐cyclic adenosine monophosphate (cAMP)/Protein Kinase A signaling and induce a “BRCAness phenotype” that further sensitizes cells to PARPi. In preclinical studies, combined DNMTi + PARPi therapy was effective in both triple‐negative (TNBC) and hormone resistant (HRBC) models with intact BRCA.

Methods:

The authors conducted a phase 1 study combining the oral DNMTi ASTX727 with the PARPi talazoparib in patients with previously treated TNBC or HRBC. Patients with deleterious mutations of BRCA were excluded. A classical 3+3 design guided dose escalation/de‐escalation, and 28 days constituted each cycle. Serial peripheral blood mononuclear cells (PBMCs) were analyzed for changes in methylation using the Infinium Methylation EPIC BeadChip and LINE1 sequencing.

Results:

Thirty‐four evaluable patients were enrolled and treated in eight dose cohorts. Myelosuppression was common with grade >3 neutropenia in 42% and grade 3 anemia and thrombocytopenia in 13%. Dose‐limiting toxicity was limited to neutropenia. Efficacy was assessed in 29 patients. There were no objective responses, six patients had stable disease persisting for >4 months in three patients. LINE1 demethylation ranged from ∼2%–10% and immune‐specific CpGs (methylation in immune cells) changed 1%–5% at day 15. Methylation changes were not dose‐dependent.

Conclusions:

ASTX727 plus talazoparib produces significant myelosuppression without other adverse events. Modest methylation changes in PBMCs were detected. There were no objective responses, but some heavily pretreated patients had stable disease for >4 months despite the attenuated doses.

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.

项与地西他滨/西达尿苷相关的新闻（医药）

2026-08-21

·息壤 I 创新药投研

mRNA癌症疫苗迎“登月时刻”，A股创新药板块涨停潮起

创新药行业要闻前瞻 2026年8月21日（周五） | 时段：8月20日07:00—8月21日07:00 ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 【今日核心提示】 8月20日，创新药赛道迎来“史诗级”催化——Moderna与默沙东联合开发的全球首款mRNA癌症疫苗在III期临床试验中达到主要终点，被业界称为肿瘤免疫治疗的“登月时刻”。受此提振，A股创新药板块掀起涨停潮，港股药企同步飙升。同日，NMPA批准两款国产创新药上市，恒瑞医药、中国生物制药相继发布亮眼中报，行业盈利拐点持续兑现。叠加美国扩大长期国债回购规模、美债收益率回落，利率敏感型创新药板块估值修复空间进一步打开。一、头版要闻：mRNA癌症疫苗III期成功，引爆全球行情 1. Moderna×默沙东：全球首款mRNA癌症疫苗III期达终点美东时间8月19日，Moderna（莫德纳）与默沙东（MSD）联合宣布，其个性化mRNA癌症疫苗intismeran autogene（原代号mRNA-4157/V940）联合PD-1抑制剂Keytruda（K药），在III期临床试验INTerpath-001中同时达到主要终点无复发生存期（RFS）和关键次要终点无远处转移生存期（DMFS），两项指标均取得统计学显著差异。该试验入组1137名已完成手术切除的高危IIB-IV期黑色素瘤患者，这是全球首个在III期临床中取得阳性结果的个性化新抗原mRNA癌症疗法，证实联合治疗方案疗效优于K药单药。资本市场反应剧烈。Moderna当日股价暴涨约177%，收报174.38美元/股，市值飙升至696亿美元，创上市以来最大单日涨幅；默沙东同步大涨12.6%，创2009年3月以来最大单日涨幅。纳斯达克生物科技指数大涨6.4%，创六年最大单日涨幅。Moderna CEO斯特凡·班赛尔表示，该产品最快可于2027年获批上市。这一突破验证了“测序肿瘤→AI筛选靶点→定制mRNA疫苗→激活T细胞→临床获益”的全新治疗路线可行性，意味着mRNA技术的应用正式从传染病疫苗跨越到肿瘤精准治疗领域。华创证券指出，该方案在黑色素瘤辅助治疗中实现了近50%的复发风险降低，且疗效可持续至少5年。来源：每经网、澎湃新闻、财联社、界面新闻（2026.08.20） 2. A股港股创新药板块掀起涨停潮受mRNA癌症疫苗III期成功消息驱动，8月20日A股创新药、疫苗、CRO等板块集体飙升。A股方面，三元基因开盘1分钟即30%封死涨停板；沃森生物、康希诺、百克生物、键凯科技、莱美药业、悦康药业、双鹭药业等20CM涨停；智飞生物、石药创新、康泰生物等涨超10%。港股方面，恒生创新药指数暴涨超8%，恒生生物科技指数涨6.94%。思路迪医药股份（01244.HK）收涨56.88%，盘中一度飙升近130%；云顶新耀（01952.HK）收涨41.43%，盘中最高涨幅近90%；康希诺生物（06185.HK）涨40.87%；中国生物制药（01177.HK）涨超14%；石药集团涨超15%。港股创新药ETF普遍涨超6%。创新药板块核心标的涨幅一览（8月20日）：标的代码涨幅备注 Moderna MRNA.US +177% 美股mRNA龙头，癌症疫苗III期成功默沙东 MRK.US +12.6% K药合作伙伴思路迪医药 01244.HK +56.88% 港股mRNA/肿瘤赛道云顶新耀 01952.HK +41.43% mRNA平台布局康希诺生物 688185.SH +20%（涨停） A股mRNA疫苗沃森生物 300142.SZ +20%（涨停）疫苗龙头百克生物 688276.SH +20%（涨停）疫苗标的中国生物制药 01177.HK +14.20% 创新药龙头来源：证券时报、财联社、网易财经（2026.08.20） ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 二、NMPA批准两款创新药上市，国产创新管线持续兑现 1. 三生国健：安弗利奇塔单抗（益赛那）获批，国内第2款IL-1β单抗 8月20日，国家药监局（NMPA）批准三生国健药业申报的安弗利奇塔单抗注射液（皮下注射）（商品名：益赛那）上市，适用于对非甾体类抗炎药和/或秋水仙碱禁忌、不耐受或缺乏疗效的，以及不适合反复使用类固醇激素的成人痛风性关节炎急性发作。该药（研发代号SSGJ-613）是三生国健自主研发、拥有自主知识产权的重组抗白介素1-β（IL-1β）人源化单克隆抗体。IL-1β是急性痛风发作的关键介质，SSGJ-613能特异性与IL-1β结合，阻断信号传导，抑制炎症效应。该药获批基于一项入组505例患者的多中心、随机、双盲双模拟、阳性对照的确证性III期研究，在缓解急性疼痛方面与复方倍他米松相当，在预防复发方面显著优于阳性对照药物。 SSGJ-613是国内第2款获批上市的IL-1β单抗（第1款为金赛药业的伏欣奇拜单抗，2025年6月获批）。国内IL-1β单抗领域将长期由这两款国产药主导，进口同机制药物卡那奴单抗尚未在中国申报。来源：NMPA官网、Insight数据库（2026.08.20） 2. 恒瑞医药：富马酸立康可泮胶囊（恒优达）获批，补体B因子抑制剂 8月20日，NMPA通过优先审评审批程序，批准恒瑞医药子公司成都盛迪医药申报的1类创新药富马酸立康可泮胶囊（商品名：恒优达）上市，用于治疗既往未接受过补体抑制剂治疗的阵发性睡眠性血红蛋白尿症（PNH）成人患者。该药是以补体活化系统旁路途径中B因子（CFB）为靶点的口服小分子抑制剂，通过靶向因子B抑制旁路途径，能同时控制血管内溶血和血管外溶血，弥补了C5补体抑制剂治疗的局限性，口服给药方式也提高了患者依从性。该药从报产到获批历时不到9个月。获批基于III期研究HRS-5965-301（76例患者，头对头依库珠单抗）。关键数据：第18~24周血红蛋白达标率（Hb≥12g/dL）立康可泮组70.0% vs 对照组11.1%（P<0.0001）；Hb应答率（较基线升高≥2g/dL）95.0% vs 55.6%；第2周后脱离输血依赖率100% vs 86.1%。研究期间未发生主要不良血管事件（MAVEs），整体耐受性良好。来源：NMPA官网、恒瑞医药官方公告、美通社（2026.08.20） ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 三、中报季：创新药企盈利拐点持续兑现 1. 恒瑞医药：创新药收入占比首破63%，拟回购10-20亿元 8月19日晚，恒瑞医药（600276.SH）发布2026年半年报。上半年实现营业收入154.56亿元（同比-1.94%），其中产品销售收入139.48亿元（同比+1.87%）；归母净利润44.65亿元（同比+0.34%）。核心亮点在于创新药收入结构持续优化：上半年创新药销售收入88.09亿元（同比+16.38%），占药品销售收入的比重提升至63.16%，创历史新高。其中抗肿瘤创新药收入62.65亿元（同比+2.58%，占创新药71.11%），非肿瘤创新药收入25.45亿元（同比+73.97%），自免与代谢板块加速放量。研发投入46.05亿元（同比+18.96%），占营收29.8%。同日，恒瑞医药公告两则回购消息：一是此前10亿元回购方案已完成（实际回购1659.1万股，成交均价约60.51元/股）；二是拟继续以10-20亿元回购A股股份，用于员工持股计划，回购价格不超过81.78元/股。海外BD方面，自2023年起已完成13笔海外交易，潜在总交易价值约420亿美元，上半年确认对外许可收入14.22亿元。NewCo模式接连落地，Kailera和Braveheart先后登陆纳斯达克。来源：恒瑞医药半年报、上海证券报、第一财经（2026.08.19-20） 2. 中国生物制药：BD收入激增2100%，“双向循环”跑通 8月19日，中国生物制药（01177.HK）发布2026年半年报。上半年实现营业收入194.44亿元（同比+10.64%），税前利润73.39亿元（同比+23%），归母净利润34.35亿元（同比+1.36%）。其中创新药收入78.1亿元（同比+29.2%），对外授权收入9.8亿元（同比+2101.9%）。中国生物制药的“双向BD”模式跑通了闭环：既“引进来”——与GSK达成乙肝功能性治愈药物合作，拿下两款呼吸重磅产品国内权益；更“走出去”——将JAK/ROCK抑制剂罗伐昔替尼以15.3亿美元授权赛诺菲，COPD新药TQC3721以19亿美元授权阿斯利康。叠加旗下礼新医药向阿斯利康和默沙东授权的近40亿美元交易，累计与MNC达成4项对外授权，首付款合计超10亿美元，总交易规模逾70亿美元。来源：中国生物制药半年报、雪球、界面新闻（2026.08.19-20） 3. 百济神州：Q2产品收入17亿美元，净利暴增627% 百济神州（688235.SH/06160.HK）半年报显示，上半年营业总收入222.20亿元（同比+26.8%），归母净利润32.71亿元（同比+627.1%），扣非净利润29.18亿元（同比+1017.6%）。单二季度产品收入达17亿美元（同比+30%），连续两个季度收入破百亿元。核心产品泽布替尼（百悦泽）上半年全球销售额161.27亿元（同比+28.7%），占产品收入74.0%，其中美国市场113.90亿元（同比+27.2%）。基于上半年强劲表现，公司上调2026年营收预测至449-462亿元。来源：百济神州半年报、海报新闻（2026.08.05）创新药龙头中报核心数据对比：公司上半年营收同比增速创新药收入归母净利润同比增速恒瑞医药 154.56亿 -1.94% 88.09亿(+16.38%) 44.65亿 +0.34% 中国生物制药 194.44亿 +10.64% 78.1亿(+29.2%) 34.35亿 +1.36% 百济神州 222.20亿 +26.8% — 32.71亿 +627.1% 来源：各公司2026年半年报（2026.08） ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 四、BD交易与产业动态 1. 先声药业获长效RSV单抗大中华区独家商业化权益 8月20日，先声药业（2096.HK）宣布与瑞阳（山东）生物制药有限公司就国产1类生物新药库莱韦拜单抗注射液达成战略合作，获得该药在大中华区的独家商业化权益。库莱韦拜单抗是瑞阳生物自主研发的全人源长效RSV（呼吸道合胞病毒）单克隆抗体，靶向RSV融合蛋白（F蛋白），通过半衰期优化设计延长体内作用时间，旨在通过单次注射为婴幼儿提供覆盖整个RSV流行季的持续保护。该药上市申请已获CDE受理并纳入优先审评审批程序，若获批有望成为首款国产长效RSV预防单抗。来源：先声药业官网公告、证券时报（2026.08.20） 2. 2026上半年中国创新药BD交易总金额接近千亿美元据行业统计，2026年上半年中国创新药License-out出海总金额接近千亿美元，多家MNC持续加码中国创新资产。阿斯利康与石药集团达成185亿美元合作（迄今中国药企创新药对外授权最大单笔交易）；百时美施贵宝与恒瑞医药合作总额152亿美元；辉瑞与信达生物合作总额105亿美元；礼来上半年5笔交易总额达165.54亿美元。值得注意的是，2026年上半年大额BD交易形式更加多元，从单资产License-out转向依托中国药企技术平台的多资产联合研发合作。礼来是最积极利用中国药企技术平台开展研发合作的MNC，上半年与信达生物、英矽智能、海思科、和誉医药各达成一笔。来源：腾讯新闻、雪球（2026.08.20） ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 五、国际视野：FDA连批多款创新疗法 1. FDA批准Ultragenyx基因疗法Genglycos——首款GSD1a治疗药物 8月19日，FDA加速批准Ultragenyx Pharmaceuticals的基因疗法pariglasgene brecaparvovec-opnr（商品名：Genglycos），用于8岁及以上糖原贮积症Ia型（GSD1a）患者，作为营养管理的辅助治疗，降低每日玉米淀粉摄入量。这是FDA批准的首款GSD1a治疗药物，也是该疾病的首款基因疗法。 Genglycos采用一次性肝定向AAV8载体递送功能G6PC基因至肝细胞，恢复葡萄糖-6-磷酸酶活性。获批基于48周随机双盲安慰剂对照III期试验（GlucoGene研究，46名受试者），治疗组玉米淀粉摄入量较基线平均降低31%（vs安慰剂组，P<0.001）。该药获得罕见儿科疾病优先审评凭证、RMAT和快速通道认定。每例患者批发成本约270万美元。Ultragenyx盘后股价上涨约7.5%。来源：FDA官网、BioPharma Dive、Healio（2026.08.19-20） 2. FDA批准Regeneron的Pasatru——第二款FOP治疗药物 8月19日，FDA批准Regeneron Pharmaceuticals的Pasatru（garetosmab-grts），用于减少成人进行性骨化性纤维发育不良（FOP）患者新的异位骨化（HO）病灶和临床评估的急性发作。FOP是一种超罕见疾病，全球约900名患者，会导致肌肉和结缔组织逐渐变为骨骼。 Pasatru是全人源抗Activin A单克隆抗体，获批基于III期OPTIMA试验（63名成人患者）。10mg/kg组新HO病灶较安慰剂组减少90%（2个 vs 19个），3mg/kg组减少94%（1个 vs 19个）；急性发作减少88%。该药每月一次静脉输注（60分钟），年均每例患者定价约140万美元。将与Ipsen的Sohonos（2023年获批）展开市场竞争。来源：BioPharma Dive、AllSci（2026.08.19-20） 3. FDA批准Beqalzi和Inqovi——血液肿瘤新选择同日，FDA批准BeOne Medicines的Beqalzi（sonrotoclax），作为首个且唯一的BCL2抑制剂用于治疗复发/难治性套细胞淋巴瘤（MCL）成人患者，这些患者既往至少接受过两线系统性治疗包括BTK抑制剂。获批基于I/II期试验BGB-11417-201，总缓解率52%，中位缓解持续时间15.8个月。 FDA同时批准Taiho Oncology的Inqovi（口服地西他滨+西达尿苷）联合维奈托克，用于新诊断急性髓系白血病（AML）老年或不适合患者。该方案完全缓解率41.6%，中位至缓解时间约2个月，提供了全口服替代方案。来源：Pharmaceutical Executive（2026.08.19） ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 六、宏观环境与机构观点 1. 美国扩大长期国债回购，利率环境利好创新药估值美国财政部宣布扩大长期名义国债回购规模，将流动性支持回购操作规模至少增加一倍，单次操作最高规模从20亿美元提高至40亿美元。调整涵盖10-20年期和20-30年期债券，9月9日生效，持续至2026年11月4日。30年期美债收益率迅速下跌近10个基点至5.18%，从2007年以来最高水平大幅回落。创新药企多处于研发投入期，高度依赖外部融资，对利率极度敏感，美债收益率下行直接利好创新药板块估值修复。来源：证券时报、东方财富（2026.08.20） 2. 机构核心观点 ▎ 国金证券：底层逻辑从“讲故事”走向“验证”，盈利拐点和出海兑现是核心驱动力。2026上半年行业迎来大规模扭亏为盈的盈利拐点，产品销售与BD授权形成双轮驱动。 ▎ 招商证券：中报季业绩验证与创新药管线催化（WCLC、ESMO年会临近）共振，AI制药新催化带动CXO与上游景气延续，看好医药行情持续向上。 ▎ 中金公司：国内创新药已进入“并跑”甚至部分领域领跑的阶段，市场回归到关注商业化价值本身。 ▎ 开源证券：2026上半年创新药板块核心驱动力在于“盈利拐点落地”与“创新资产价值凸显”，建议围绕“商业化造血强劲+业绩确定性高”和“历史性扭亏为盈+对外授权合作”两条主线布局。来源：各券商研报（2026.08） ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 七、前瞻：下一阶段关注要点 ▎ 赛道价值重估——国内多数mRNA肿瘤疫苗管线仍处于临床前或I期临床，整体进度落后国外约3-5年。关注思路迪、云顶新耀、康希诺等mRNA平台公司BD合作预期和管线推进节奏。 ▎ 海外III期数据窗口——2026年下半年国内创新药首批海外III期临床数据将迎来集中读出，预计2027年起更多中国创新药企有望正式步入“赚美元”的价值兑现阶段。 ▎ 学术会议催化——WCLC（世界肺癌大会）与ESMO年会临近，创新药管线催化密集，关注ADC、双抗、KRAS、BTK等热门构型的临床数据读出。 ▎ 国内管线进展——恒瑞富马酸立康可泮胶囊经治PNH适应症及原发性IgA肾病适应症推进；三生国健SSGJ-613痛风间歇期适应症III期沟通。恒瑞瑞康曲妥珠单抗HER2阳性二线乳腺癌、达尔西利辅助治疗等新适应症已获批。 ▎ FDA审批节点——Ultragenyx另一基因疗法UX111（Sanfilippo综合征A型）FDA审批截止日9月19日；GTX-102（Angelman综合征）关键性试验数据预计9-10月读出，为重要催化剂。 ▎ 后K药时代——K药2028年专利到期压力下，默沙东布局mRNA癌症疫苗+皮下注射剂型Qlex应对。PD-1/VEGF双抗等新一代免疫药物开展头对头研究，关注竞争格局演变。 ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ ─ 【免责声明】本文仅基于公开信息整理，不构成任何投资建议。所引用信息均来源于公开渠道，包括但不限于NMPA官网、FDA官网、上市公司公告、证券时报、财联社、每经网、澎湃新闻、界面新闻等。文中涉及个股仅作信息示例，不构成实质性投资建议。股市有风险，投资需谨慎。 — 创新药行业要闻前瞻 | 每日更新 —

2026-08-14

·Briinsight

单药狂收7亿美金！又一抗癌神药斩获三期临床，医药巨头炸场“最难啃”肺癌市场！

抗癌界又现“世纪大对决”！在全球最致命的癌症——肺癌领域，有一个让无数医生和患者谈之色变“硬骨头”：EGFR exon 20插入突变。长期以来，这个领域的患者用药选择极其匮乏，生存希望渺茫。然而就在今天，医药界迎来了一场震撼级的破局！针对这一难治靶点，日本抗癌先锋大鹏药品（Taiho Pharmaceutical）与美国新锐Cullinan Therapeutics强强联手，正式官宣其重磅小分子抑制剂在三期临床中击穿终点！不仅敲响了进军一线治疗的大门，更直接拉开了与全球医药巨头强生（J&J）的正面硬刚。 7亿美金交易额、1亿美元通关尾款、三家跨国药企厮杀…… 这一场关于“肺癌最难靶点”的生死较量，正在彻底改写癌症治疗的版图！一、重磅官宣：击穿三期临床，抗癌神药即将斩获“一线治疗” 在抗癌药物的研发长跑中，能够在一线治疗（即患者确诊后接受的首次治疗）中取得突破，永远是衡量一款药物临床价值的“金标准”。大鹏药品与 Cullinan Therapeutics 近日联合宣布，其研发的小分子 EGFR 抑制剂 Zipalertinib 联合铂类化疗，在一项针对之前未经治疗、局部晚期或转移性非小细胞肺癌（NSCLC，且携带 EGFR exon 20 插入突变）的 285 名患者的三期临床试验中，取得了具有里程碑意义的胜利！根据中期分析数据，接受 Zipalertinib 联合化疗组的患者，在无进展生存期（PFS）上显著优于单纯化疗组，达到了该研究的主要有效性终点。独立数据监察委员会在审查了其“可控”的安全性和卓越的疗效后，直接建议提前对该研究进行双盲解密！这意味着，Zipalertinib 已经拿到了冲刺一线治疗上市许可的关键通关文牒！治疗方案对比（三期临床设计）：二、1亿美元临门一脚！Cullinan 与大鹏的巨额“对赌” 这项喜讯不仅对肺癌患者来说是天大的利好，对研发方 Cullinan 而言，更是一张通往百亿美元市值的“超级支票”。早在 2022 年，Cullinan 与大鹏药品达成深度合作协议，将 Zipalertinib 的商业化权利进行深度绑定。根据协议条款：二线治疗批准奖金：当 Zipalertinib 获得 FDA 批准用于二线治疗（化疗失败后的后续治疗）时，Cullinan 将直接斩获 3000 万美元的里程碑付款；一线治疗通关大奖：一旦该药成功拿下 FDA 的一线治疗批件，Cullinan 将迎来 1 亿美元（约合 7.2 亿元人民币）的巨额现金奖励！利润平分：双方还将以 50:50 的比例，平分该药物未来在美国市场的税前销售利润。目前，FDA 已于今年 4 月正式受理了 Zipalertinib 用于化疗后进展患者的上市申请，并预计在明年 2 月 27 日之前给出最终答复。一旦二线与一线相继“敲钟”，大鹏药品负责操盘销售（其旗下已有 Inqovi、Lonsurf 和 Lytgobi 三款斩获 FDA 批件的成熟肿瘤药），这组合拳无疑将直接撼动现有的市场格局。三、三强争霸：强生、阿斯利康、大鹏药品角逐“最难靶点” 在 Zipalertinib 异军突起之前，EGFR exon 20 插入突变肺癌市场几乎是强生（Johnson & Johnson）的“独角戏”。强生旗下的双特异性抗体 Rybrevant 自 2021 年获批二线、2024 年获批一线以来，一路狂飙。在 2026 年上半年，Rybrevant 及其联合用药的销售额猛增 70%，达到了创纪录的 5.46 亿美元。然而，没有哪一片蓝海会永远静止。这个曾经被武田制药（Takeda）因临床失败而遗憾撤退的战场，正迎来前所未有的三国杀局面：从强生的一独秀，到阿斯利康重金下注迪哲医药的 Zegfrovy，再到如今大鹏与 Cullinan 的 Zipalertinib 三期大战告捷——短短几年内，针对该肺癌细分市场的竞争已从“无药可用”陡然演变成“三强争霸”！结语：癌症不再是终点，科技正在重塑生命 Zipalertinib 的成功，绝不仅是一家药企的商业胜利，更是全球靶向精密医疗对复杂癌症的又一次伟大跨越。当越来越多的创新药物突破临床瓶颈，当药企巨头们在竞争中不断推高疗效的天花板，最终受益的，永远是每一个在黑暗中苦苦等待希望的肺癌患者与其家庭。这场围绕 EGFR exon 20 靶点的争夺战才刚刚拉开序幕。究竟谁能在未来的全球抗癌战场上问鼎王者？我们拭目以待！互动话题抗癌靶向药百花齐放，你最关注哪一类癌症治疗的突破？你或身边的朋友是否也曾面临过“罕见靶点无药可用”的困境？对于各大药企巨头发力的这些创新抗癌药，你最看好哪一家？欢迎在评论区留下你的看法，让我们一起为医学的每一次进步点赞！ #肺癌靶向药 #抗癌新药突破 #医药前沿 #大鹏药品 #强生 #阿斯利康 #健康科普 #医学重磅NEWS 欢迎在评论区留下你的看法，我们一起见证医药史上的疯狂一年！高质量观点，我们会精选置顶。我们将持续输出：洞察行业变局，预判未来先机。我们持续深耕医疗健康赛道，为您提供：深度解读：穿透表象，还原商业底层逻辑。标的拆解：拆解并购核心，寻找估值锚点。趋势研判：捕捉投融资风向，预警行业巨震。把复杂问题，讲清楚。比毒品还疯！美剧都不敢这么演：还没上市的神药，黑市已经抢疯了！突发！美国国防部胜诉未果反被打脸，药明康德绝地翻盘！华盛顿这场裁决切中了谁的命脉？首发爆款！13亿美元砸下，神药巨头罕见病赛道“下暴雨” 打破无药可救的死局！FDA紧急批准重磅“黑科技”：用改造病毒精准杀灭癌细胞 👇 访问网站：https://www.briinsight.com 了解更多👇 👇 商务合作与咨询，敬请扫描二维码 👇 生物医药前沿洞察｜职业成长加速器聚焦创新药、制剂技术、CDMO、AI药物研发等领域的最新突破；专注生物医药研发、注册申报、临床进展与市场趋势解析；汇聚业内声音、人才资讯与实战经验，打造最具价值的行业交流平台。 👇 星标 Briinsight，轻松不迷路👇 Tips：亲爱的朋友，由于微信改版，有时我们会“走散”。为了不错过每一篇深度好文，记得点击 “Briinsight” 名片，进入主页并将我们设为星标 ⭐️。这样，我们的每一次推送都会准时和你相遇。

2026-08-13

·FierceBiotech

Cullinan tees up J&J fight—and $100M payday—as Taiho-partnered drug hits phase 3 cancer goal

Taiho Pharmaceutical and Cullinan Therapeutics have scored a second phase 3 victory for their small molecule EGFR inhibitor, teeing the partners up to challenge Johnson & Johnson for a lung cancer niche. The study evaluated the combination of zipalertinib and platinum-based chemotherapy in the first-line treatment of adults with previously untreated, locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations. Investigators randomized 285 patients to receive the combination or chemotherapy alone. Progression-free survival (PFS) was significantly longer on the combination than on chemotherapy alone, hitting the study’s primary efficacy endpoint at a planned interim analysis. The partners have yet to share the PFS data but called the improvement “clinically meaningful.” Based on the data, which the partners said showed a “manageable” safety profile, the independent data monitoring committee recommended unblinding the study. Investigators will continue to monitor safety and efficacy. Taiho and Cullinan plan to seek approval for the zipalertinib-chemotherapy combination in the first-line setting. In April, the FDA accepted zipalertinib for review in NSCLC patients with EGFR exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy. The FDA is set to approve or reject zipalertinib in that setting by Feb. 27 next year. Under the terms of its 2022 deal with Taiho, Cullinan is in line to receive $30 million when zipalertinib is approved for second-line use and $100 million when it secures a first-line nod from the FDA. Taiho and Cullinan will split pretax profits from U.S. sales equally. Responsibility for driving those sales will fall on Taiho, which already markets three cancer drugs—Inqovi, Lonsurf and Lytgobi—that won FDA approval this decade. Zipalertinib will enter an NSCLC niche already served by Johnson & Johnson’s Rybrevant, a bispecific that received FDA approval for second-line use in 2021 and a first-line nod in 2024. Sales of Rybrevant and Lazcluze, a drug used in combination with the bispecific in some settings, grew (PDF) 70% to $546 million in the first half of 2026. J&J has grown sales amid a lack of competition for the EGFR exon 20 NSCLC market. Takeda withdrew its challenger, Exkivity, in 2023 over a failed trial, leaving J&J without a rival until the FDA approved Dizal Pharmaceutical’s Zegfrovy in 2025. Competition for the market is intensifying. AstraZeneca, which created Dizal in a spinout in 2017, paid $600 million upfront for exclusive global rights to Zegfrovy last month. If zipalertinib wins FDA approval, Taiho will be locked in a three-way fight with AstraZeneca and J&J for the NSCLC niche.

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
D11848	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


NCT04657081 (ASCERTAIN-V, Pubmed \| N Engl J Med)	临床1/2期	急性髓性白血病	189	Oral Decitabine-Cedazuridine + Oral Venetoclax	壓鏇鬱鏇廠網築淵選廠(構網襯繭襯範糧齋鏇鑰) = 構築餘遞醖醖糧衊艱壓願遞鏇淵簾壓鹹願壓遞 (衊鬱壓願醖壓簾築顧淵, 36 ~ 57) 更多	积极	2026-06-04
ASCO2026	N/A	急性髓性白血病一线	466	Decitabine/Cedazuridine + Venetoclax	廠簾觸築選襯衊窪鹽鑰(鏇願鹽蓋製願鏇餘窪衊) = 顧顧鬱齋淵齋繭艱鏇網憲鹽網醖繭鹹鹹構糧糧 (鹽壓廠構齋鑰範鹽網顧 ) 更多	积极	2026-05-29
				Azacitidine + Venetoclax	廠簾觸築選襯衊窪鹽鑰(鏇願鹽蓋製願鏇餘窪衊) = 廠鬱網簾齋築選構窪構憲鹽網醖繭鹹鹹構糧糧 (鹽壓廠構齋鑰範鹽網顧 ) 更多
EHA2026	临床2期	急性髓性白血病	68	ASTX727+Venetoclax (de novo AML)	糧壓選艱積範齋繭鹹鏇(齋鑰構衊鹹壓壓簾鏇壓) = 製觸遞衊襯襯獵繭遞築範鹽築選製繭蓋淵膚憲 (廠齋襯襯壓簾鏇憲艱襯 ) 更多	积极	2026-05-12
				ASTX727+Venetoclax (secondary AML (sAML))	糧壓選艱積範齋繭鹹鏇(齋鑰構衊鹹壓壓簾鏇壓) = 鏇製繭構鬱蓋膚製獵構範鹽築選製繭蓋淵膚憲 (廠齋襯襯壓簾鏇憲艱襯 ) 更多
NCT05360160 (EHA2026)	临床1/2期	难治性急性髓细胞白血病 KMT2A rearrangement \| NPM1 mutation \| NUP98 rearrangement	42	Revumenib+ASTX727+Venetoclax	夢醖膚醖壓蓋餘積鹽襯(鹹積簾鹽範蓋顧顧簾膚) = The most common all-grade adverse events were nausea (60%), vomiting (52%), QTc prolongation (50%), and hypokalemia (45%), mostly grade 1-2. Grade ≥3 events were most frequently febrile neutropenia (35%), thrombocytopenia (22%), lung infection (20%), and respiratory failure (15%). QTc prolongation grade ≥3 occurred in 2 pts (5%). Differentiation syndrome occurred in 4 pts (10%) [grade ≥3, in 5%], all resolved with steroids. 齋簾夢艱醖築網鬱簾範 (築範鑰艱餘糧憲窪淵鹽 ) 更多	积极	2026-05-12
				Revumenib+ASTX727+Venetoclax (KMT2A rearrangement)
NIBIT-ML1 (AACR2026)	临床2期	转移性黑色素瘤	42	ASTX727 plus I+N	憲壓憲衊築膚鑰糧網積(製淵構觸顧憲鏇鑰獵製) = 淵齋顧積積鬱遞餘積觸鹽齋鬱淵鹽範窪糧窪廠 (簾襯鹹鏇蓋窪窪遞築觸, 5.0 ~ 13.8) 更多	积极	2026-04-21
				I+N	憲壓憲衊築膚鑰糧網積(製淵構觸顧憲鏇鑰獵製) = 顧顧製築糧齋蓋齋鹽襯鹽齋鬱淵鹽範窪糧窪廠 (簾襯鹹鏇蓋窪窪遞築觸, 5.0 ~ 6.6) 更多
NCT06802146 (ASH2025)	临床1期	意义不明的克隆性血细胞减少症	138	DEC/CED	觸積簾鹹觸鏇壓艱鏇淵(衊繭遞壓膚網蓋夢艱鬱) = 廠憲壓遞範繭顧廠憲鬱選築醖淵鹽鏇顧獵壓範 (壓蓋構齋艱選醖餘願糧 ) 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	廠繭鑰積顧繭襯簾齋夢(觸艱積襯願壓淵鏇窪製) = 衊餘鏇願願簾糧鏇鹽獵網餘鹹範選積膚網壓鏇 (範積簾鬱獵鬱窪壓窪觸 ) 更多	积极	2025-12-06
				ASTX727 35/100 mg PO + venetoclax 400 mg (azole adjusted) PO	廠繭鑰積顧繭襯簾齋夢(觸艱積襯願壓淵鏇窪製) = 遞選鑰蓋願鹽廠獵顧鑰網餘鹹範選積膚網壓鏇 (範積簾鬱獵鬱窪壓窪觸 ) 更多
ISRCTN30808508 (AMMO, ASH2025)	临床2期	骨髓增生性疾病	77	ASTX727	築衊艱襯鑰積襯簾憲蓋(顧壓繭窪觸觸憲鏇淵壓) = 餘窪構觸醖構醖鑰衊選簾鹹膚顧衊鬱壓鏇鬱構 (築廠壓遞製範襯襯繭顧, 39.3 ~ 66.3) 更多	积极	2025-12-06
				Hydroxycarbamide/Best Supportive Care	築衊艱襯鑰積襯簾憲蓋(顧壓繭窪觸觸憲鏇淵壓) = 顧廠選鬱淵膚夢蓋鑰淵簾鹹膚顧衊鬱壓鏇鬱構 (築廠壓遞製範襯襯繭顧, 14.9 ~ 49.4) 更多
NCT06661915 (ASH2025)	临床2期	骨髓增生性疾病	50	ASTX727 + iadademstat	膚積觸膚鏇繭憲衊壓鏇(衊壓製壓醖廠選鬱蓋醖) = 齋簾衊製蓋製蓋選繭醖窪艱積簾膚觸鹽憲製襯 (醖膚鹹淵鹹窪窪範蓋構 )	积极	2025-12-06
				ASTX727	膚積觸膚鏇繭憲衊壓鏇(衊壓製壓醖廠選鬱蓋醖) = 構蓋網膚鏇廠艱糧襯範窪艱積簾膚觸鹽憲製襯 (醖膚鹹淵鹹窪窪範蓋構 )
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	窪願夢鬱糧衊憲簾膚壓(鬱構積窪壓選獵選壓餘) = Patients with ASXL1mut had a median OS not reached (vs 18 months in patients with ASXL1wt, P=0.008) 簾餘壓構窪遞築糧鹹夢 (夢範遞鏇鬱夢願壓顧廠 ) 更多	积极	2025-12-06