Gilteritinib in Combination With Azacitidine and Venetoclax Compared to Induction Chemotherapy "7+3" in Combination With a FLT3-inhibitor in Fit, Newly Diagnosed, FLT3-ITD Mutated Adult AML Patients: a Randomized Trial of the EORTC Leukemia Group and GIMEMA.

This international, multicenter, randomized (1:1), open-label phase II/III trial will evaluate the efficacy and safety of gilteritinib combined with azacitidine and venetoclax (experimental arm) versus standard "7+3" induction plus a FLT3inhibitor (quizartinib or midostaurin) (control arm) in newly diagnosed FLT3-ITD mutated AML patients eligible for intensive chemotherapy.

开始日期2026-12-01

申办/合作机构

European Organisation for Research & Treatment of Cancer

[+2]

NCT07646821

/ Not yet recruiting临床1/2期

A Phase 1b/2 Study of Ropeginterferon Alfa-2b Added to Azacitidine in Newly Diagnosed MDS/AML (≥ 10% Blasts) With TP53 Mutations

To find the recommended dose of ropeginterferon alfa-2b in combination with azacitidine in patients with newly diagnosed MDS/AML with TP53 mutations. To learn if the recommended dose of ropeinterferon alfa-2b found in Dose Escalation in combination with azacitidine can help to control the disease.

开始日期2026-11-27

申办/合作机构

药华医药股份有限公司

NCT07623187

/ Not yet recruiting临床2/3期

A Two-part, Phase 2b/Phase 3 Double-blinded, Randomized Study of IPN60340 in Combination With Azacitidine and Venetoclax Versus Placebo in Combination With Azacitidine and Venetoclax in Participants With Newly Diagnosed Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy.

The purpose of this study is to find out how well the study drug IPN60340 works to treat participants with acute myeloid leukemia. Acute myeloid leukemia is a rare blood cancer that grows quickly. This study's main aim is to compare the percentage of participants who reach complete remission within the first 6 months of treatment between the 2 study arms (study drug and standard medicines compared to placebo and standard medicines).
In this study all participants will receive azacitidine and venetoclax plus either the study drug IPN60340 or placebo. Venetoclax will be given as a tablet by mouth once each day in 28-day cycles. Azacitidine will be given by injection under the skin (subcutaneously) or through the veins (intravenously) daily for the first 7 days of each 28-day cycle. IPN60340 or placebo (depending on which arm of the study the participant is assigned to) will be given through the veins (intravenously) on day 1 of each 28-day cycle.
There will be 4 periods in this study:
* A screening period (up to 28 days) to assess whether the participant can take part requiring at least 1 visit to the study center.
* A treatment period where all eligible participants will receive azacitidine and venetoclax plus either the study drug IPN60340 or placebo. The study requires 8 visits for the first month followed by 1 visit every month until unacceptable toxicity, disease progression, the start of new cancer treatment, or study closure, whichever is first.
* A safety follow-up period (at 28 days (±3 days) after the last dose of study medicine) to assess safety after participants have finished treatment.
* A long-term follow-up period where participants' health will be monitored using a telephone call or clinic visit every 12 weeks until the end of study.
Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), bone marrow aspirates (sampling of the liquid part of the bone marrow). Some participants will also undergo pregnancy testing. Participants in the Phase 3 portion of the study will also be asked to fill in questionnaires.
The time each participant will be in this study will vary based on how well the medicine works to treat the participant's AML. Azacitidine and venetoclax plus either IPN60340 or placebo will be provided to participants who tolerate it for as long as their disease does not progress. Participants may withdraw consent to participate at any time.

开始日期2026-11-01

申办/合作机构

Ipsen SA

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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7,243

项与阿扎胞苷相关的文献（医药）

2026-12-01·MOLECULAR BIOLOGY REPORTS

Dysregulated methylation of the human BOLL promoter hinders germ cell development from spermatogonia to spermatids

Article

作者: Ma, Hsiu-Yen ; Lin, Shih-Chieh ; Cheng, Yung-Hsuan ; Hou, Hsiu-Chiung ; Lu, Chun-Wun ; Lin, Yung-Ming ; Lin, Tsung-Yen ; Cheng, Yu-Sheng ; Chen, Hsing-Yi

PURPOSE:

BOLL, a highly conserved gene crucial for meiosis in spermatogenesis, is epigenetically regulated through DNA methylation in various species. This study aimed to determine whether BOLL promoter methylation contributes to its downregulation in azoospermic men with hypospermatogenesis (HS) and to explore its potential regulatory association with human spermiogenesis.

METHODS:

We conducted pyrosequencing to assess methylation levels at the putative BOLL promoter on testicular samples from azoospermic men with HS versus normal spermatogenesis (NR) and evaluate correlations with spermatogenic severity and gene expression. In silico CpG island (CGI) prediction and luciferase reporter assays were used to confirm methylation-sensitive regulatory regions. Pathway enrichment analysis identified biological processes associated with BOLL. Functional assays using BOLL overexpression in HEK293T cells and GC2 cells, as well as demethylation using 5-Aza-2'-deoxycytidine (5-AZA) in GC2 cells, were conducted to explore downstream transcriptional effects.

RESULTS:

Eight CpG sites (CpGs) within the BOLL promoter CGI were significantly hypermethylated in HS samples, with seven exhibiting a significant inverse correlation with the spermatogenic score, and three with BOLL transcript levels. The region from - 1434 to + 180 was confirmed as a methylation-sensitive promoter. Gene ontology analysis indicated that spermatid development and differentiation were the top BOLL-associated pathways. BOLL overexpression by plasmid transfection in HEK293T cells and GC2 cells upregulated spermatid-specific genes (TNP2 and GAPDHS), while concurrently suppressing spermatogonia-associated markers (ID4 and PIWIL4). In GC2 cells, 5-AZA treatment enhanced Boll expression and induced downstream spermatid-specific genes, including Prm2, which was not responsive to BOLL overexpression alone.

CONCLUSION:

We identified specific BOLL promoter CpGs that are hypermethylated in HS and associated with reduced gene expression. These findings suggest that promoter methylation may contribute to BOLL silencing and impaired spermatid differentiation, supporting its regulatory role in human spermiogenesis.

2026-08-01·Blood neoplasia

Real-world pharmacokinetics of venetoclax in patients with acute myeloid leukemia in Japan

Article

作者: Mukai, Yuji ; Ishio, Takashi ; Izumiyama, Koh ; Morioka, Masanobu ; Teshima, Takanori ; Doki, Kosuke ; Suzuki, Toma ; Yoshimoto, Goichi ; Homma, Masato ; Moriyama, Mitsuru ; Chi, SungGi ; Harada, Shinpei ; Onozawa, Masahiro ; Kondo, Takeshi ; Mori, Akio ; Miyashita, Naoki ; Minami, Yosuke ; Kajikawa, Sayaka ; Matsukawa, Toshihiro ; Kobayashi, Mirei ; Kanaya, Minoru ; Yokoyama, Emi ; Saito, Makoto

Venetoclax (VEN) combined with azacitidine (AZA) or low-dose cytarabine (LDAC) has significantly improved outcomes for older patients with acute myeloid leukemia (AML). However, severe cytopenia often leads to treatment interruptions. Previous literature has reported that Asian patients tend to have higher plasma VEN concentrations and are more prone to severe neutropenia. We hypothesized that VEN pharmacokinetics (PK) influence these adverse events in the Japanese population. In a prospective observational study (UMIN000047371) involving 76 patients, we monitored VEN PK (trough, maximum plasma concentration, and area under the plasma concentration-time curve from 0 to 12 hours [AUC0-12]). Our analysis of an 81-sample PK data set revealed that samples taken 6 hours after VEN dose offered the best correlation to AUC0-12 (r = 0.945). Importantly, in patients who achieved composite complete remission, a positive correlation was found between the duration of grade 3 neutropenia and VEN AUC0-12 (r = 0.338; P = .028). We further investigated potential factors influencing VEN PK and neutropenia, specifically considering tumor burden and renal function. Samples from patients with pretreatment white blood cell (WBC) counts of <3000/μL and an estimated glomerular filtration rate (eGFR) of <60 mL/min exhibited significantly higher VEN AUC0-12 levels (P = .037) than other groups. These patients also demonstrated a significantly more extended period of grade 3 neutropenia (P = .037). These results suggest that VEN PK could be crucial for predicting neutrophil recovery after VEN-containing regimens in AML. Specifically, patients with low pretreatment WBC counts and low eGFR may represent a risk factor for higher VEN concentrations and, consequently, prolonged neutropenia.

2026-08-01·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Simultaneous analysis of various anticancer drugs by supercritical fluid chromatography-mass spectrometry. Part II: Method validation and comparison with liquid chromatography

Article

作者: Fleury-Souverain, Sandrine ; Rudaz, Serge ; Guillarme, Davy ; Bonnabry, Pascal ; Vallet, Virginie ; Nguyen, Nathalie

A complete SFC-MS/MS method validation enabling the simultaneous trace-level analysis of 27 conventional anticancer drugs (CADs) was performed. The selected drugs were the following: 5-fluorouracil, azacitidine, busulfan, carboplatin, cisplatin, cyclophosphamide, cytarabine, dacarbazine, daunorubicin, docetaxel, doxorubicin, epirubicin, etoposide, gemcitabine, idarubicin, ifosfamide, irinotecan, methotrexate, mitomycin, oxaliplatin, paclitaxel, pemetrexed, raltitrexed, topotecan, treosulfan, vinblastine and vincristine. Accuracy profiles were obtained for all CADs. Trueness ranged between 71.0% and 119.8%, while repeatability (1.3-17.5%) and intermediate precision (2.0-24.9%) were determined for all compounds, with limits of quantification comprised between 0.1 and 25 ng.mL^-1. These results were systematically compared with those obtained during the validation of a reference UHPLC-MS/MS method routinely used to analyze 25 of the 27 CADs contained in samples collected from work surfaces in a hospital pharmacy chemotherapy compounding unit. SFC-MS/MS method offered greater accuracy but was hampered by lower precision than the UHPLC-MS/MS method for most of the compounds. Furthermore, although the developed SFC-MS/MS method was characterized by a lower sensitivity, it allowed the quantification of compounds that were difficult to analyze by UHPLC-MS/MS, such as azacitidine and cisplatin, with analytical performance comparable to that obtained for the other CADs analyzed. This feature highlights the complementarity of the two chromatographic approaches in the context of CAD determination in a hospital environment.

1,884

项与阿扎胞苷相关的新闻（医药）

2026-06-18

·GBIHealth

填补全球空白！天广实倍捷欣获批原发性膜性肾病适应证

药械追踪 No.1 / 天广实CD20抗体倍捷欣在华获批pMN新适应证 2026年6月18日，北京天广实生物技术股份有限公司（以下简称“天广实”）宣布，其自主研发的创新型第三代CD20抗体药物倍捷欣（奥妥珠单抗β注射液）用于治疗原发性膜性肾病（pMN）的新药上市许可申请（NDA）已获国家药品监督管理局（NMPA）批准。中国是全球pMN发病率最高的国家之一，而pMN是终末期肾病主要的病因之一，需要肾移植或长期肾透析治疗。此前，全球范围内尚未批准任何一款针对pMN治疗的特效药物。倍捷欣是天广实自主研发的创新型第三代CD20抗体药物，此前已于2026年2月在中国获批用于治疗视神经脊髓炎谱系疾病（NMOSD）。其治疗pMN临床III期研究显示，倍捷欣单药治疗组的76周基于尿蛋白（UPCR）和肾小球滤过率（eGFR）评估的完全缓解率（CRR）显著优于环孢素组 (49.4% vs 3.9%，P＜0.0001)，且其104周CRR进一步提高至57.1%，总缓解率（ORR）为 85.7%。倍捷欣组中位至免疫缓解时间（1.2个月vs 3.0个月）和中位至肾脏缓解时间（3.8个月 vs 4.7个月）均显著优于环孢素组（P均<0.0001）。安全性方面，倍捷欣组第104周时肾脏治疗相关不良事件（TRAE）发生率为0.0%，而环孢素组的肾脏TRAE发生率高达30.3%。 ->点击文末阅读原文，解锁完整双语新闻 No.2 / 施维雅拓舒沃在华获批新适应证，联合阿扎胞苷一线治疗AML 2026年6月18日，施维雅旗下创新靶向药物拓舒沃（艾伏尼布片）获得国家药品监督管理局（NMPA）批准新增适应证，用于联合阿扎胞苷治疗携带易感异柠檬酸脱氢酶-1（IDH1）突变，因合并症无法使用强诱导化疗或者年龄≥75岁的新诊断急性髓系白血病（AML）患者。艾伏尼布是全球首个且目前中国唯一获批的IDH1抑制剂，此前已在中国获批用于治疗携带IDH1突变的复发性或难治性急性髓系白血病成人患者。此次一线适应证获批基于全球多中心、随机、双盲、安慰剂对照的III期AGILE研究，该研究共纳入146例不适合强化诱导化疗的新诊断IDH1突变急性髓系白血病患者。结果显示，艾伏尼布联合阿扎胞苷治疗组的mEFS可达22.9个月，mOS达29.3个月；47%的患者获得完全缓解（CR），其中88%的患者随访1年仍维持完全缓解。艾伏尼布联合阿扎胞苷整体安全性可控。 ->点击文末阅读原文，解锁完整双语新闻企业动态 No.1 /拜耳完成对Perfuse Therapeutics的收购 2026年6月17日，拜耳（德国证券交易所代码：BAYN）宣布已完成对Perfuse Therapeutics, Inc.的收购，拥有与PER-001玻璃体腔植入物相关的全部权益。该药物是一款同类首创（first-in-class）、缓释的小分子内皮素受体拮抗剂，目前处于治疗青光眼和糖尿病视网膜病变（DR）的Ⅱ期临床开发阶段。根据协议条款，拜耳支付了3亿美元的首付款。该交易的总潜在价值高达24.5亿美元，其中包括首付款以及未来基于成功标准的里程碑付款。 ->点击文末阅读原文，解锁完整双语新闻 No.2 /渤健拟10亿美元收购RayThera，扩充免疫学产品管线权益渤健（Nasdaq: BIIB）近日宣布与RayThera达成最终协议，同意以最高10亿美元的价格收购RayThera，其中包括一笔预付款以及主要取决于未来临床和监管里程碑达成情况的后续付款。 RayThera是一家专注于免疫学领域小分子疗法发现与开发的私有生物技术公司。该公司的产品组合涵盖多个抗炎分子，有潜力治疗免疫介导性疾病相关适应证。其领先候选药物预计将于2026年第三季度初进入Ⅰ期临床开发阶段。此项收购完成后，渤健将主导这些资产的开发、生产及全球商业化。 ->点击文末阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I ” 自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作 ▶ olivia.xu@generalbiologic.com 数据库 | 咨询服务 | 资讯追踪 ▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

2026-06-17

·骑着毛驴赶火车

药政日报 | 2026年6月17日

药政日报 | 2026年6月17日每日药政资讯，尽在掌握国内药政动态1. 施维雅拓舒沃®（艾伏尼布）AML一线适应症在华获批发布时间：2026-06-156月10日，国家药品监督管理局正式批准拓舒沃®（艾伏尼布片）新增适应症，用于联合阿扎胞苷治疗携带易感异柠檬酸脱氢酶-1（IDH1）突变、因合并症无法使用强诱导化疗或者年龄≥75岁的新诊断急性髓系白血病（AML）患者。此前，拓舒沃已在中国获批用于治疗携带IDH1突变的复发性或难治性AML成人患者。此次一线适应症获批标志着该联合疗法正式跨入AML全程管理新阶段，填补了国内新诊断、不适合强化疗患者无特异性靶向药的临床空白。此次获批基于全球多中心、随机、双盲、安慰剂对照的Ⅲ期AGILE研究数据。研究显示，艾伏尼布联合阿扎胞苷治疗组中位无事件生存期显著延长，中位总生存期提升至近30个月。该药已于2025年底纳入国家医保目录。2. 默沙东安福畅®（克莱罗韦单抗）在华获批发布时间：2026-06-11 国家药监局通过优先审评审批程序批准默沙东申报的克莱罗韦单抗注射液（商品名：安福畅）上市，用于即将进入或出生在第一个呼吸道合胞病毒（RSV）流行季的新生儿和婴儿预防RSV所致的下呼吸道感染。安福畅是国内首款、现阶段唯一固定剂量长效RSV单抗，所有符合适应症的新生儿、婴儿统一采用105mg剂量单次肌肉注射，无需测量体重，大幅简化临床接种流程。该药基于CLEVER全球关键性Ⅲ期临床研究，可将RSV相关住院率降低84.3%，单次注射提供长达6个月防护。这是继赛诺菲/阿斯利康尼塞韦单抗（乐唯初）后，国内第二款获批的RSV预防性单抗。3. 泽璟制药盐酸吉卡昔替尼片斑秃适应症获批发布时间：2026-06-15 泽璟制药公告，公司自主研发的盐酸吉卡昔替尼片（曾用名：盐酸杰克替尼）用于治疗成人重度斑秃的上市申请获得NMPA批准。这是该药继中、高危骨髓纤维化适应症后第二个获批的适应症，也是首个获批的治疗免疫炎症性疾病的临床适应症。本次批准基于Ⅲ期临床试验数据：治疗24周后，脱发严重程度工具量表（SALT）评分≤20分（头发覆盖面积大于80%）的受试者比例，吉卡昔替尼片50mg Bid组为34.5%，安慰剂组为3.5%，差异具有统计学意义。4. 两款原料药同日通过CDE审评发布时间：2026-06-16~17 天宇股份（300702）6月17日公告，公司美阿沙坦钾原料药近日通过CDE审评，适用于治疗成人原发性高血压。目前国内已有10家厂家获得CDE审评通过，公司尚需完成GMP符合性检查后方可上市销售。同和药业（300636）6月16日公告，公司罗沙司他原料药（登记号Y20240001574）通过CDE审批，审评结果为"A"（已批准在上市制剂中使用）。罗沙司他主要用于治疗慢性肾脏病（CKD）引起的贫血，2025年中国大陆医院采购金额约25.93亿元。5. CDE发布第107批参比制剂目录征求意见发布时间：2026-06-12 CDE组织遴选了第一百零七批化学仿制药参比制剂目录，现予以公示征求意见。公示期间，社会各界如对公示内容有不同意见，可通过仿制药质量和疗效一致性评价专栏提出。6. NMPA发布第106批仿制药参比制剂目录发布时间：2026-06-15 经国家药监局仿制药质量和疗效一致性评价专家委员会审核确定，现发布仿制药参比制剂目录（第一百零六批）。行业动态1. 康宁杰瑞恩尼妥®（KN026）全国首批商业发货发布时间：2026-06-16来源：康宁杰瑞公告 6月16日，康宁杰瑞自主研发、与石药集团合作开发的恩尼妥®（安尼妥单抗注射液，研发代号KN026）全国首批商业发货仪式在苏州工业园区举行，标志着中国首个国产自研HER2双特异性抗体药物正式投入临床应用。恩尼妥®于2026年5月28日获NMPA优先审评审批程序批准上市，用于联合化疗治疗既往至少接受过一种含曲妥珠单抗治疗方案的局部晚期或转移性HER2阳性成人胃或胃食管结合部腺癌患者。该药物基于Fc异二聚体平台技术（CRIB）开发，可同时精准结合HER2受体两个非重叠表位，全面阻断下游肿瘤信号通路。其关键性Ⅲ期研究显示，恩尼妥联合化疗可将晚期胃癌患者中位生存期提升至20.9个月。2. 常山药业艾本那肽收到CDE二次发补通知发布时间：2026-06-12 常山药业在投资者互动平台确认，其核心创新药艾本那肽注射液（现通用名：阿贝那肽）在6月8日完成首轮发补、五大专业审评全部办结后，再次收到CDE补充资料通知。公司表示正在准备补充资料，无法预计完成审评时间。艾本那肽是一款GLP-1受体激动剂，用于2型糖尿病治疗，2024年4月提交上市申请。此次二次发补属于专业技术类资料补充，而非单纯文书整改。受此影响，该产品大概率将错过2026年医保谈判窗口。3. 三款仿制药同日获批上市发布时间：2026-06-17 千金药业子公司湖南千金湘江药业获得盐酸氨溴索口服溶液（100ml:0.3g，化药4类，按甲类非处方药管理）和非布司他片（20mg、40mg，化药4类）《药品注册证书》。华润双鹤子公司获得三款药品注册证书：利格列汀二甲双胍片(Ⅱ)（2型糖尿病，视同通过一致性评价）、黄体酮注射液(Ⅱ)、氢溴酸伏硫西汀片。4. 励晶太平洋创新药Senstend™获NMPA上市批准发布时间：2026-06-17 励晶太平洋(00575)宣布旗下创新疗法Senstend™的新药申请正式获得NMPA上市批准，并附带为期四年数据保护期。此次获批触发商业合作伙伴江苏万邦医药（复星医药控股子公司）向励晶太平洋支付500万美元（约3900万港元）里程碑款项，预计2026年下半年首次商业销售时将再支付200万美元。国际药政动态1. FDA批准拜耳Ambelvist™ 成为美国最低剂量大环钆造影剂发布时间：2026-06-15（美国时间）拜耳宣布，美国FDA已批准Ambelvist™（gadoquatrane）用于成人及儿科患者（包括足月新生儿）的对比增强磁共振成像（MRI）检查，以检测和显示中枢神经系统及非中枢神经系统体区血管异常病变。 Ambelvist是一种新一代大环钆基造影剂（mGBCA），采用新型四聚体结构，具有高弛豫率。推荐剂量为0.01 mmol/kg体重（相当于0.04 mmol Gd/kg），与传统0.1 mmol Gd/kg的大环钆造影剂相比，钆含量减少60%；与0.05 mmol Gd/kg的gadopiclenol相比，钆含量减少20%。该药是目前美国市场上剂量最低的大环类钆造影剂。此次批准基于全球Ⅲ期QUANTI系列研究数据，证明在较低钆剂量下仍能有效增强病灶可视化效果。2. FDA批准赛诺菲Tzield® 用于3期1型糖尿病发布时间：2026-06-12（美国时间）美国FDA通过加速批准程序，批准赛诺菲Tzield®（替利珠单抗，teplizumab-mzwv）的补充生物制剂许可申请（sBLA），用于延缓近期被诊断为3期1型糖尿病（T1D）的8至17岁儿科患者内源性胰岛素分泌功能的衰退。 Tzield成为全球首个针对新诊断3期T1D患者的疾病修饰疗法。此前，该药已于2022年获批用于延缓2期T1D进展为3期疾病，2024年4月进一步扩大至1岁及以上儿童。此次加速批准基于Ⅲ期PROTECT研究数据：治疗组患者平均C肽水平下降速度较安慰剂组显著减缓（最小二乘差异：0.13 pmol/mL，p<0.001）。替利珠单抗是一种抗CD3单克隆抗体，通过与效应T细胞表面的CD3结合，抑制T细胞对胰岛β细胞的攻击。3. Gilead lenacapavir每周一次口服HIV预防疗法获FDA受理发布时间：2026-06-16（美国时间） Gilead Sciences宣布，FDA已受理其lenacapavir（商品名：Yeztugo）作为每周一次口服剂型用于HIV暴露前预防（PrEP）的补充新药申请（sNDA）。PDUFA目标日期为2027年2月2日。 Lenacapavir是一款新一代衣壳抑制剂，通过阻断HIV病毒衣壳的组装和分解来发挥作用。如果获批，将为HIV预防领域提供新的长效选择。4. EMA发布多项监管更新发布时间：2026-06-15~17 6月15日，EMA管理委员会批准多项议程，包括支持欧盟埃博拉疫情应对、2025年年度活动报告评估等。6月16-18日，孤儿药委员会（COMP）、兽药委员会（CVMP）会议召开。6月17日，先进疗法委员会（CAT）会议、临床试验信息系统（CTIS）信息日活动举行。

2026-06-17

·PRNewswire

Ligufalimab (CD47)-Based Combination Achieves Deep Responses and Survival Benefit in Frontline AML: Phase II Results Presented in Oral Session at EHA 2026

HONG KONG, June 17, 2026 /PRNewswire/ -- Akeso, Inc. (9926.HK) ("Akeso" or the "Company") today announced that compelling results from its randomized, double-blind, placebo-controlled Phase II trial (AK117-206) of ligufalimab (AK117) were presented as an oral presentation at the 2026 European Hematology Association (EHA) Congress. Ligufalimab is Akeso's proprietary next-generation humanized IgG4 anti-CD47 monoclonal antibody. The study evaluated ligufalimab in combination with azacitidine (AZA) and venetoclax (VEN) in patients with treatment-naïve acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. Ligufalimab-based combination therapy demonstrates a significant trend toward survival benefit At a median follow-up of 10 months in the ligufalimab group and 8.8 months in the control group, median event-free survival (EFS) was 9.1 months in the ligufalimab group versus 6.9 months in the control group (hazard ratio [HR] = 0.46). The 6-month EFS rate was 67.8% versus 55.5% in the control group, and the 9-month EFS rate was 53.2% versus 14.1%. Median overall survival (mOS) was not reached in the ligufalimab group versus 8.3 months in the control group (HR = 0.46). The 6-month OS rate was 83.3% versus 73.2%, and the 9-month OS rate was 78.7% versus 43.1% in the control group. Ligufalimab-based combination therapy yields deep tumor responses The objective response rate (ORR) was 80.0% in the ligufalimab group versus 66.7% in the control group, and the composite complete response (CRc) rate was 56.7% versus 53.3%; the proportion of patients achieving CRc with minimal residual disease (MRD) negativity was higher in the ligufalimab group than in the control group (46.7% vs. 36.7%). The median duration of CRc was 10.4 months in the ligufalimab group, which was markedly superior to 5.6 months in the control group. Favorable Safety Profile With No New Safety Signals Observed The incidence of overall treatment-emergent adverse events (TEAEs) and serious adverse events was comparable between treatment arms. The most common TEAEs were generally consistent with those expected in the context of AML and AZA+VEN therapy. Anemia occurred in 46.7% of patients in the ligufalimab arm versus 50.0% in the control arm. Notably, ligufalimab has already received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of AML. Akeso is advancing its ligufalimab clinical development programs at a globally competitive pace across both hematologic malignancies and solid tumors. Ligufalimab is also the first anti-CD47 monoclonal antibody worldwide to enter a registrational Phase III clinical trial in solid tumors. About Akeso Akeso (HKEX: 9926.HK) is a leading biopharmaceutical company committed to the research, development, manufacturing and commercialization of the world's first or best-in-class innovative biological medicines. Founded in 2012, Akeso has built a comprehensive R&D innovation ecosystem anchored by its proprietary Tetrabody antibody technology platform, AI-powered drug R&D platform, Dual-Shield ADC technology platform, Dual-Lock T-cell engager (TCE) technology platform, Tissue-Smart siRNA/mRNA technology platform, and cell therapy technology platforms. Backed by world-class GMP manufacturing facilities and a highly efficient, integrated commercialization system, Akeso has developed into a globally competitive biopharmaceutical enterprise. Leveraging its fully integrated, multi-functional platform, the company maintains a robust pipeline of more than 50 innovative assets targeting cancer, autoimmune diseases, inflammation, metabolic disorders, and other major therapeutic areas. Of these, 27 candidates have advanced into clinical trials—including 15 bispecific or multispecific antibodies and bispecific ADCs—and 8 innovative drugs have reached commercial stage. Through efficient and groundbreaking R&D, Akeso integrates premier global resources to develop transformative medicines, deliver high-quality, affordable therapeutic antibodies to patients worldwide, and generate sustained commercial and societal value as it strives to become a global leader in biopharmaceutical innovation. Forward-Looking Statements This announcement by Akeso, Inc. (9926.HK, "Akeso") contains "forward-looking statements". These statements reflect the current beliefs and expectations of Akeso's management and are subject to significant risks and uncertainties. These statements are not intended to form the basis of any investment decision or any decision to purchase securities of Akeso. There can be no assurance that the drug candidate(s) indicated in this announcement or Akeso's other pipeline candidates will obtain the required regulatory approvals or achieve commercial success. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in P.R.China, the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; Akeso's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the Akeso's patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. Akeso does not undertake any obligation to publicly revise these forward-looking statements to reflect events or circumstances after the date hereof, except as required by law. SOURCE Akeso, Inc. 21% more press release views with Request a Demo

临床结果临床2期临床3期孤儿药免疫疗法

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

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03573024 (CTgov)	临床2期	急性髓性白血病	36	Venetoclax+Azacitidine	夢製壓鹹遞網艱遞選夢 = 構衊夢壓淵淵觸範觸餘膚構糧築蓋醖構簾鹹鑰 (選製憲鬱網憲選襯遞糧, 遞網鑰糧夢廠構襯構鬱 ~ 鑰築願範淵觸鬱網醖製) 更多	-	2026-06-11
NCT03146871 (CTgov)	临床2期	慢性粒单核细胞白血病 \| 难治性骨髓增生异常综合征 \| 伴有骨髓发育不良相关变化的急性髓系白血病	7	Pharmacological Study+Decitabine+Recombinant EphB4-HSA Fusion Protein+Azacitidine	選衊蓋廠膚夢淵窪艱範 = 餘壓觸鏇範鏇夢淵鬱壓醖構醖築鏇蓋膚襯廠壓 (衊鹹齋獵鹽獵夢膚餘鏇, 鹽獵鏇觸鬱淵襯製餘繭 ~ 憲壓築齋襯鹹鏇壓廠觸) 更多	-	2026-06-08
ASCO2026	N/A	急性髓性白血病诱导	154	3+7 regimen	襯艱鬱選艱鏇網壓膚鑰(積鬱獵蓋窪願齋餘築觸) = 憲鹹積糧鏇鹹觸顧夢淵獵製壓鑰選鬱糧艱襯築 (衊積築遞淵製艱蓋夢齋 ) 更多	积极	2026-05-29
ASCO2026	N/A	急性髓性白血病诱导	154	azacitidine + venetoclax (AZA + VEN)	襯艱鬱選艱鏇網壓膚鑰(積鬱獵蓋窪願齋餘築觸) = 夢淵顧選構襯艱鏇選顧獵製壓鑰選鬱糧艱襯築 (衊積築遞淵製艱蓋夢齋 ) 更多	积极	2026-05-29
NCT03471260 (ASCO2026)	临床1/2期	IDH1突变急性髓性白血病一线 IDH1-mutated	40	Azacitidine+Venetoclax+Ivosidenib	膚選觸醖觸膚艱壓膚鏇(製餘鹽餘衊簾壓壓繭範) = 範製齋齋鹽願窪襯簾醖鬱鑰壓膚鹽顧獵糧憲範 (構壓範網範鹹遞壓壓築 ) 更多	积极	2026-05-29
ASCO2026	N/A	-	280	7+3	顧壓齋網範積顧製蓋齋(鏇積醖積夢鬱膚衊鹹鑰) = 鬱觸壓鹽選積淵構獵鹹艱獵鹽壓鹹構餘鹹鹹壓 (憲鬱簾淵齋襯築憲齋憲 ) 更多	积极	2026-05-29
ASCO2026	N/A	-	280	azacitidine plus venetoclax	顧壓齋網範積顧製蓋齋(鏇積醖積夢鬱膚衊鹹鑰) = 簾製選鏇鏇顧膚蓋製鏇艱獵鹽壓鹹構餘鹹鹹壓 (憲鬱簾淵齋襯築憲齋憲 ) 更多	积极	2026-05-29
ASCO2026	N/A	急性髓性白血病	55	Azacitidine	網顧壓鑰艱鹹積網網鹽(獵構鹹構鏇鏇憲襯膚顧) = 齋餘廠鹽鏇積遞廠窪範餘餘艱簾膚夢窪廠蓋築 (廠願獵醖膚餘鑰窪製齋 ) 更多	积极	2026-05-29
ASCO2026	N/A	急性髓性白血病	55	Combinations of HMAs±LDAC	積膚夢醖齋選顧簾築獵(鬱簾蓋網範顧築憲願獵) = 顧範糧築鏇觸選襯顧網獵膚餘鹹觸壓網繭鹹積 (蓋廠衊餘糧獵遞範鏇範, 17 ~ 35) 更多	积极	2026-05-29
NCT02900560 (AACR2026)	临床2期	铂耐药上皮性卵巢癌	34	PembrolizumabAzacitidineab + PembrolizumabAzacitidine	衊選鹹餘壓製餘選壓範(構鏇遞製醖簾構繭襯淵) = 醖蓋衊廠獵獵鏇壓壓窪鏇鹽網廠憲獵鑰窪觸壓 (憲膚餘鑰觸餘憲鏇獵獵 ) 更多	积极	2026-04-21
NCT03466294 (CTgov)	临床2期	急性髓性白血病	42	Venetoclax+Azacitidine	膚積獵憲觸構憲蓋積淵(積憲簾膚壓鑰構餘構夢) = 繭積蓋衊壓蓋蓋構鬱鬱廠鹹築積窪糧淵築鬱糧 (餘築襯糧積艱鏇齋願膚, 構積獵蓋窪齋鑰壓壓築 ~ 鑰鹽繭蓋觸窪遞繭膚夢) 更多	-	2026-04-07
NCT02494258 (CTgov)	临床2期	血液肿瘤	5	CC-486 (CC-486 200 mg QD x 14 Days)	繭廠鏇鑰憲選獵繭鏇簾 = 膚襯鹹餘鑰餘廠網築蓋淵衊觸選夢鬱蓋壓醖鹽 (鏇衊觸窪廠網願窪選顧, 壓衊顧鑰顧願觸鬱鏇鹹 ~ 願觸積醖繭遞夢糧衊鏇) 更多	-	2026-04-02
NCT02494258 (CTgov)	临床2期	血液肿瘤	5	CC-486 (CC-486 600 mg QD x 7 Days)	繭廠鏇鑰憲選獵繭鏇簾 = 積憲餘夢醖願鹹憲鹹襯淵衊觸選夢鬱蓋壓醖鹽 (鏇衊觸窪廠網願窪選顧, 廠積憲鹹鑰膚製製廠鏇 ~ 範夢鑰鬱衊淵鏇鏇簾積) 更多	-	2026-04-02
NCT03264404 (Pubmed \| Oncologist)	临床2期	转移性胰腺导管腺癌二线	36	Azacitidine plus Pembrolizumab (locally advanced or metastatic pancreatic ductal adenocarcinoma)	簾艱製鹽網鹹遞顧醖淵(淵醖餘鹹鏇構蓋膚願簾) = Grade 1-2 diarrhea most common AEs 餘夢襯獵積築製餘選鑰 (糧醖衊遞蓋築鹹膚鏇範 ) 更多	不佳	2026-03-09