A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients

The goal of Phase 1a of this clinical research study is to find the highest tolerable dose of revumenib that can be given in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin to patients who have acute leukemia.
The goal of Phase 1b of this clinical research study is to learn if the dose of revumenib in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin found in Phase 1a can help to control the disease.

开始日期2025-12-01

申办/合作机构

The University of Texas MD Anderson Cancer Center

NCT07211958

/ Not yet recruiting临床3期

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Revumenib in Combination With Intensive Chemotherapy in Participants With Newly Diagnosed AML With an NPM1 Mutation

The purpose of this study is to assess if adding revumenib to standard chemotherapy improves outcomes in participants with AML with certain genetic mutations compared to chemotherapy alone. The study will also assess the safety of adding revumenib to chemotherapy.

开始日期2025-12-01

申办/合作机构

Syndax Pharmaceuticals, Inc.

NCT06652438

/ Recruiting临床3期IIT

Randomized Study to Assess Revumenib in Combination With Azacitidine + Venetoclax in Adult Patients With Newly Diagnosed NPM1-mutated or KMT2A-rearranged AML Ineligible for Intensive Chemotherapy

Treatment of patients with newly diagnosed AML who are not eligible for intensive chemotherapy has remained an area of high unmet medical need. The combination therapy with two medicines, azacitidine and venetoclax, is the usual plan of action. This has brought significant progress in the treatment, but it nevertheless is not curative and the disease does relapse over time.
Revumenib blocks a specific molecule called menin in the cell nucleus. Some types of AML are reliant on menin working properly. These are leukemia cells with a change in the DNA, i.e. a mutation in the NPM1 or KMT2A gene. Revumenib can prevent the production of these types of leukemia cells by disrupting the production of this menin.
The current study investigates whether adding revumenib to the combination therapy improves the prognosis for AML patients with a mutation in the NPM1 or KMT2A gene.
This is a randomized, double-blind, placebo-controlled clinical study where subjects will be treated until disease progression, or development of side effects or death. From the moment of inclusion of the last patient, there will be a 4-year observational follow-up study in order to register survival duration and follow-up visits.
Approximately 415 previously untreated patients with a mutation in the NPM1 or KMT2A gene and with newly diagnosed AML, who are not eligible for intensive chemotherapy. Patients must be ≥18 years of age.

开始日期2025-03-31

申办/合作机构

Hemato-Oncologie voor Volwassenen Nederland

100 项与瑞维美尼相关的临床结果

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100 项与瑞维美尼相关的转化医学

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100 项与瑞维美尼相关的专利（医药）

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项与瑞维美尼相关的文献（医药）

2025-10-01·LEUKEMIA RESEARCH

Menin inhibitors in KMT2A-rearranged leukemia: Mechanistic insights, clinical trial progress, and potential of combination therapies

Review

作者: Huang, Rui ; Yang, Xinyu

Leukemia driven by rearrangement of the Lysine Methyltransferase 2 A (KMT2A) gene, formerly known as mixed-lineage leukemia (MLL), is associated with poor prognosis due to the formation of oncogenic fusion proteins. Menin, a scaffold protein encoded by the MEN1 gene, plays a critical role in the pathogenesis of KMT2A-rearranged (KMT2A-r) leukemia. Targeting menin has emerged as a promising therapeutic strategy, leading to the development of several menin inhibitors, some of which have entered clinical trials. Notably, Revumenib has been approved for clinical use. However, resistance to menin inhibition is an increasing concern, necessitating alternative approaches. Combining menin inhibitors with other therapeutic strategies appears to enhance efficacy and mitigate resistance. This review summarizes recent advancements in menin inhibitor research for KMT2A-r leukemia, including mechanistic insights and clinical trial progress, while also exploring the potential of combination therapies. A deeper understanding of the mechanisms underlying menin inhibition and resistance is crucial for developing more effective treatments to improve patient outcomes.

2025-09-01·CRITICAL REVIEWS IN ONCOLOGY HEMATOLOGY

Menin inhibitors in KMT2A-rearranged and NPM1-mutated acute leukemia: A scoping review of safety and efficacy

Article

作者: Kalfah, Anas ; Dali, Sara Al ; Mohamed, Shehab F ; Al-Mashdali, Abdulrahman F

BACKGROUND:

Menin inhibitors represent a novel therapeutic approach targeting the Menin-KMT2A interaction in acute leukemias with KMT2A rearrangements or NPM1 mutations. This scoping review synthesizes current clinical evidence for emerging Menin inhibitors in development.

METHODS:

We systematically analyzed clinical trials, conference proceedings, and regulatory documents regarding Menin inhibitors in clinical development through December 2024. Primary outcomes included response rates, minimal residual disease (MRD) status, and safety profiles.

RESULTS:

Thirteen clinical trials investigating six Menin inhibitors (Revumenib, Ziftomenib, Bleximenib, BMF-219, DS-1594, and Enzomenib) were identified. Revumenib demonstrated consistent efficacy across five pivotal trials, achieving MRD-negative rates of 70-90 % in both KMT2A-rearranged and NPM1-mutated leukemias, leading to FDA approval in November 2024. Ziftomenib showed particular efficacy in NPM1-mutated cases but exhibited a higher incidence of differentiation syndrome (30 %) in KMT2A-rearranged patients. Bleximenib reported a 93 % overall response rate, pending MRD validation. Early resistance emergence, primarily through MEN1 mutations, was observed across trials, emerging as early as two treatment cycles.

CONCLUSIONS:

Menin inhibitors demonstrate promising clinical activity in molecularly defined leukemias, with Revumenib establishing proof-of-concept for this therapeutic approach. However, challenges remain, including resistance development, optimal timing of therapy initiation, and determination of effective combination strategies. Larger randomized trials with extended follow-up are needed to establish long-term efficacy and safety profiles. The rapid clinical development of multiple agents in this class suggests an expanding role for Menin inhibitors in leukemia treatment paradigms.

2025-08-28·BLOOD

Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT-101 study

Article

作者: Aldoss, Ibrahim ; Bagley, Rebecca G. ; Smith, Angela R. ; Amitai, Irina ; Heiblig, Maël ; DiPersio, John F. ; Yu, Li ; Dickens, David S. ; Mantzaris, Ioannis ; Arellano, Martha L. ; Traer, Elie ; Wolach, Ofir ; McMahon, Christine M. ; Raffoux, Emmanuel ; Mannis, Gabriel N. ; Stein, Eytan M. ; Döhner, Hartmut ; Papayannidis, Cristina ; Kovacsovics, Tibor ; Pigneux, Arnaud ; Stone, Richard M. ; de Botton, Stephane ; Kühn, Michael W. M. ; Grove, Carolyn S. ; Thirman, Michael J. ; Chudnovsky, Yakov ; Issa, Ghayas C. ; Žučenka, Andrius ; Blachly, James S. ; Schuh, Andre C. ; Perl, Alexander E. ; Bajel, Ashish ; Montesinos, Pau ; Harpel, John G. ; Greco, Corinna ; Shami, Paul J.

Abstract:

The prognosis for relapsed or refractory (R/R) nucleophosmin 1–mutated (NPM1m) acute myeloid leukemia (AML) is poor and represents an urgent unmet medical need. Revumenib, a potent, selective menin inhibitor, was recently approved for the treatment of R/R acute leukemia with a KMT2A translocation in patients aged ≥1 year based on results from the phase 1/2 AUGMENT-101 study. Here, we present results from patients with R/R NPM1m AML enrolled in the phase 2 portion of AUGMENT-101. Enrolled patients received revumenib with or without a strong CYP3A4 inhibitor every 12 hours in 28-day cycles. Primary end points were rate of complete remission (CR) or CR with partial hematologic recovery (CRh; CR + CRh), safety, and tolerability. Secondary end points included overall response rate (ORR) and duration of response. As of 18 September 2024, 84 patients received ≥1 dose of revumenib. Median age was 63 years; 1 patient was aged <18 years. The protocol-defined, efficacy-evaluable population for the primary analysis included 64 adult patients (≥3 previous lines of therapy, 35.9%; previous venetoclax, 75.0%). The CR + CRh rate was 23.4% (1-sided P = .0014); the ORR was 46.9%. Median duration of CR + CRh was 4.7 months. Of 30 responders, 5 (16.7%) proceeded to hematopoietic stem cell transplant (HSCT) and 3 resumed revumenib after HSCT. Treatment-related adverse events led to treatment discontinuation in 4 patients (4.8%). Revumenib demonstrated clinically meaningful responses in this heavily pretreated, older population with NPM1m AML, including remissions that enabled HSCT. The safety profile of revumenib was consistent with previously reported results. This trial was registered at www.clinicaltrials.gov as #NCT04065399.

229

项与瑞维美尼相关的新闻（医药）

2025-09-23

·小药说药

FDA最有可能在2025年Q4批准上市的3款药物

十一小长假即将来临，随之而来的，还有2025年最后一个季度。时间真如白驹过隙，在2025年Q4，有哪些药物可能会获批上市？9月21日，Nature主编Kaniaw Dilzer在Nat Rev Drug Discov（IF=101.8）上撰写文章，预测了2025年Q4最有可能获FDA批准上市的3款药物。这3款药物分别是： Navepegritide（Ascendis Pharma），用于治疗软骨发育不全； Relacorilant（Corept Therapeutics），用于治疗库欣综合征； Ziftomenib（Kura Oncology/Kyowa Kirin），用于治疗NPM1突变急性髓系白血病。上述3款药物均处于优先评审的状态，PDUFA日期均在2025年11月~12月。 Tips：PDUFA日期是指FDA对新药进行审查的最后期限。 Navepegritide 软骨发育不全软骨发育不全一种软骨成骨障碍的遗传病，表现为四肢短小，躯干长，巨头；其主要病因是由于FGFR3基因激活突变，导致软骨内骨生长受抑制。图1软骨发育不全及潜在治疗手段现有治疗手段：BioMarin的Voxzogo（vosoritide），每日皮下注射的释放C型利钠肽（CNP）类似物。CNP通过拮抗FGFR3信号通路，促进软骨细胞增殖与骨生长。图2Voxzogo研发历程 Navepegritide Navepegritide可释放CNP，是一种前药，每周一次给药，相比Voxzogo（每日注射）更具便利性。图3 Navepegritide和vosoritide作用机制临床数据 Approach试验（关键性III期）： 100 μg/kg/周剂量下，年化生长速度（annualized growth velocity）为5.42 cm/年，安慰剂组为4.35 cm/年，差异显著。 COACH试验（II期）：与生长激素联用，年化生长速度超过平均身高儿童的第97百分位数，安全性良好。 Relacorilant 库欣综合征库欣综合征是由糖皮质激素分泌过多引发的疾病，常见症状包括腹部肥胖、满月脸、水牛背、高血压等。图4库欣综合征 Relacorilant Relacorilant是一种选择性糖皮质激素受体调节剂。主要特点是不结合孕激素、雌激素或盐皮质激素受体，因此避免脱靶效应（如肾上腺功能不全、低钾血症、QT延长）。临床数据 GRACE试验（III期）： 22周时，高血压患者收缩压平均下降7.9 mmHg，舒张压下降5.4 mmHg。高血糖患者血糖曲线下面积、HbA1c、空腹血糖均显著改善。 GRADIENT试验（III期）：主要终点（收缩压下降）未达统计学显著性，但仍提供支持性证据。 Ziftomenib NPM1突变AML NPM1突变约占成人AML的30%，部分患者会在一年内复发或难治。图5克隆性造血进展为AML的进程现有治疗：Venetoclax（Venclexta）广泛使用，但目前出现耐药。图6NPM1突变AML现有治疗方案决策树临床数据 KOMET-001试验（II期）：主要终点：完全缓解（CR）或完全缓解伴部分血液学恢复（CRh）。在92例可评估患者中，23%达到CR，其中67%达到微小残留病阴性（MRD-）。在venetoclax经治和未经治患者中均有效，这是其重要优势。需要注意的是，针对AML的治疗，2024年Syndax的revumenib（Revuforj）获批用于KMT2A重排的AML。但 Ziftomenib专注于NPM1突变，且在venetoclax耐药患者中仍有效，这可能是其差异化优势。总结这3个药物均获优先审评，意味着FDA认为它们可能提供显著优于现有疗法的优势。Ziftomenib主要靶向NPM1突变，relacorilant针对皮质醇受体，navepegritide靶向FGFR3通路。精准医学趋势日渐明显。至于是否能按时获批，还需综合考量其安全性、疗效。参考文献： Dilzer K. Upcoming FDA approval decisions in Q4 2025. Nat Rev Drug Discov. 2025 Sep 15. “小药说药知识文库”已落户知识星球，持续更新最新生物医药、免疫学、肿瘤学相关知识内容，加入可免费下载最新内容的精选PPT，加入方法在知识星球APP搜索“小药说药知识文库”或扫描下方二维码，小药邀请你到知识星球一起学习！公众号已建立“小药说药专业交流群”微信行业交流群以及读者交流群，扫描下方小编二维码加入，入行业群请主动告知姓名、工作单位和职务。

优先审批临床2期上市批准临床结果

2025-09-22

·Business Wire

U.S. Food and Drug Administration Authorizes CytoCell KMT2A Breakapart FISH Probe Kit PDx as a New Companion Diagnostic for KMT2A rearranged Acute Leukaemia

OXFORD, England--(BUSINESS WIRE)--OGT, a leading global provider of genomic diagnostic and research solutions, announced today that the U.S. Food and Drug Administration (FDA) has granted their De Novo Classification Request for the CytoCell® KMT2A Breakapart FISH Probe Kit PDx as a companion diagnostic (CDx) for Syndax’s first-in-class menin inhibitor, REVUFORJ® (revumenib). Revuforj is FDA approved for the treatment of relapsed or refractory (R/R) acute leukaemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and paediatric patients one year and older. We are optimistic that the authorization of OGT's CDx will help this underserved patient group benefit from developments in precision oncology OGT’s CytoCell KMT2A Breakapart FISH Probe Kit PDx detects clinically relevant rearrangements that occur in patients with acute leukaemia, providing a robust, accessible, rapid turnaround test for KMT2Ar detection that will maximize the ability for clinicians to quickly identify patients who may be eligible for treatment with Revuforj. It is estimated that more than 95% of patients with KMT2Ar acute leukaemia have a KMT2A translocation, a type of rearrangement that occurs when part of one chromosome breaks and fuses to a different chromosome. “We are delighted that our CytoCell KMT2A Breakapart FISH Probe Kit PDx has received marketing authorization as a CDx for Revuforj.” said Dr. Leila Luheshi, VP of Pharma Partnering at OGT. “The development and subsequent authorization of this new CDx is an important demonstration of the skill and commitment of our clinical scientists and regulatory specialists to deliver safe and effective diagnostics for patients with one of the most devastating forms of leukaemia.” Steve Chatters, OGT’s EVP of Regulatory and Medical Affairs noted “Our strong foundation in haematology diagnostics has been a key factor in our success bringing the KMT2Ar CDx to market as a Class II device, as opposed to the more common Class III classification for companion diagnostics. We have decades of experience developing regulated FISH products, gained from our prior development of IVDR-certified and FDA-cleared FISH probes. Combined with our extensive customer partnerships in haematology labs in the USA, we have refined insights into the application of these products and are able to access a wealth of real-world data across a multitude of clinical and analytical factors, which is invaluable for product development. This is a core pillar of our success." The emergence of precision therapies, such as the menin inhibitor Revuforj (an oral inhibitor of the menin–KMT2A interaction), offers new avenues for potentially improving outcomes for patients with these challenging acute leukaemia cases. “Accurately identifying acute leukaemia patients with KMT2Ar is a key factor in selecting appropriate therapeutic options for a group of patients who have traditionally had a very poor prognosis” said Adrian Smith, CEO of OGT. “We are optimistic that the authorization of OGT's CDx will help this underserved patient group benefit from developments in precision oncology.” *Note to editors: About KMT2Ar Acute Leukaemia The KMT2A (lysine methyltransferase 2A) gene at 11q23.3 is commonly rearranged in acute leukaemias, especially in infant leukaemia. KMT2A rearrangements can be detected in approximately 70%-80% of infants with acute lymphoblastic leukaemia (ALL) and in 5-10% of paediatric and adult ALLs1,2. They can also be found in more than 50% of infants with acute myeloid leukaemia (AML) and is also seen in 10% of adolescents and 3-10% of adults with AML. KMT2A rearrangements are also seen in mixed-phenotype acute leukaemia, a rare type of acute leukaemia more common in infants and children than adults2. To date, more than 90 partners have been identified with the most common partner genes being AFF1 (4q21), MLLT3 (9p22) and MLLT1 (19p13.3)2. References 1. Van der Burg M, Poulsen TS, Hunger SP, et al., Split-signal FISH for detection of chromosome aberrations in acute lymphoblastic leukemia. Leukemia 2004;18(5):895-908 2. WHO Classification of Tumours Editorial Board. Haematolymphoid tumours [Internet; beta version ahead of print]. Lyon (France): International Agency for Research on Cancer; 2022 [cited 2025 June 26]. (WHO classification of tumours series, 5th ed.; vol. 11). Available from: https://tumourclassification-iarc-who-int.libproxy1.nus.edu.sg/chapters/63. REVUFORJ® is a registered trademark of Syndax Pharmaceuticals, Inc. About OGT’s CytoCell KMT2A Breakapart FISH Probe Kit PDx (CDA-LPH013) The KMT2A Breakapart FISH Probe Kit PDx is a fluorescence in situ hybridization (FISH) test used to detect rearrangement of the KMT2A region on chromosome 11 at location 11q23.3 in 3:1 methanol/glacial acetic acid fixed bone marrow specimens from patients with acute leukaemia with KMT2A rearrangement. The assay is indicated for detecting the presence of rearrangements involving the KMT2A region as a companion diagnostic to aid in identifying those patients for whom treatment with REVUFORJ® (revumenib) is indicated in accordance with the approved therapeutic product labeling. The KMT2A Breakapart FISH Probe Kit PDx is not intended for monitoring of residual disease. Reporting and interpretation of FISH results should be performed by a qualified pathologist or cytogeneticist. For In Vitro Diagnostic Use. Caution: Federal law restricts this device to sale by or on the order of a physician. (Rx Only) For full limitations and other information, see the device package insert or go to https://www.ogt.com/kmt2a-pdx. About OGT OGT, a Sysmex Group company, is a leading global provider of clinical and diagnostic genomic solutions that are created for scientists by scientists - including CytoCell®, CytoSure® and SureSeq™ ranges of FISH, microarray and NGS products. The company is dedicated to creating products that enable researchers and clinical decision makers to reach the right care decisions for each patient, every time. OGT strives to unlock the future of genetic clinical care with a commitment to working in partnership with its customers - not only by sharing its expertise of 30 years at the forefront of genetic endeavour, but also by working closely with scientists to understand their unique challenges, and to customise its approach to meet their exact needs. With OGT’s precision medicine partnerships, we strive to accelerate clinical trial assay and companion diagnostic development programmes to help our partners make the right decisions every time. Dedicated to improving clinical care, OGT believes that through partnership—together—we’ll achieve more. CytoSure®, SureSeq™ and myProbes®: For Research Use Only, not for use in diagnostic procedures. CytoCell®: Some products may not be available in your region. For more information on the Company, please visit our website at www.ogt.com. About Sysmex Corporation Sysmex Corporation, headquartered in Kobe, Japan, is a global leader in in vitro diagnostics. Since its foundation in 1968, Sysmex has focused on diagnostics as the core of its business, and today, it supports the health of people in over 190 countries and regions worldwide. Sysmex continues to innovate in diagnostics, and to collaboratively create unique values in the areas of personalized medicine and novel treatments, under its long-term vision of "Together for a better healthcare journey." Through its unique technology, solutions, and co-creation with various partners, Sysmex delivers new value and addresses the universal desire of people to live longer and healthier lives. The company employs more than 10,000 employees worldwide. Sysmex Corporation is listed in the Prime Market on the Tokyo Stock Exchange. For more information about Sysmex Corporation and its affiliate companies, please visit www.sysmex.co.jp/en/.

临床研究诊断试剂

2025-09-22

·医学新视点

权威报告：30多年来癌症死亡率下降34%！但有这些习惯的人，更容易得癌症

日前，美国癌症研究协会（AACR）发布了2025年抗癌进展报告（AACR Cancer Progress Report 2025）。这一长达282页的综合性报告回顾了过去一年在抗癌路上获得的众多进展。其中值得一提的是，近年来癌症死亡率显著下降。除早期筛查和生活方式改变外，创新癌症疗法的持续开发也是癌症患者预后改善的主要原因。本文将与读者分享这一报告的精彩内容。点击文末“阅读原文”即可访问AACR网站查看报告原文。癌症死亡率下降分析显示，1991—2023年，总体癌症死亡率下降34%，相当于避免超过450万例癌症死亡。这一下降主要得益于公共卫生措施，尤其是预防、筛查与疫苗接种。统计显示，1975—2020年，控烟、筛查项目及疫苗接种共计避免约594万例癌症死亡；同时，部分癌种治疗的持续进展也为降低癌症死亡率作出了重要贡献。图片来源：参考资料[1] 报告同时指出，约40%的癌症与可改变的危险因素相关，包括烟草使用、超重/肥胖、体力活动不足、过度紫外线暴露、饮酒、环境暴露以及致癌性病原体感染。尽管近年吸烟率显著下降，但儿童与成人肥胖及饮酒上升仍构成重要公共卫生隐患；如不及时干预，过去50年取得的抗癌进展可能因此受损。 ▲≥30岁成年人癌症病例中可归因于所选因素的百分比（图片来源：参考资料[1]） ▲与肥胖、运动不足相关的癌症种类（图片来源：参考资料[1]）另一方面，随着早期检测研究持续进展，美国FDA相继批准多项基于人工智能的设备和软件，用于更早、更准确地检测癌症。微创筛查也在加速推进，包括用于结直肠癌筛查的血液与粪便检测，以及支持居家自取样的宫颈癌筛查装置。同时，影像学创新与对癌症风险相关遗传学的更深入理解，共同提升了早期检测的精准性与可及性。这些技术的进展共同提升了早期检测的精准性与可及性，并推动了多种癌症死亡率的显著下降。 ▲1975—2020年因筛查与去除癌前病变而避免的癌症死亡占总数的比例（图片来源：参考资料[1]）此外，尽管整体抗癌取得显著成效，但不同癌种的抗癌进展不均：五年总生存率方面，胰腺癌为13%、多形性胶质母细胞瘤为6%，而乳腺癌达92%、前列腺癌达98%。这仍是学术界与产业界需要共同应对的关键挑战。 ▲不同时期部分癌症的5年总生存率比较（图片来源：参考资料[1]）抗癌疗法监管里程碑进展自2024年7月1日至2025年6月30日，FDA共批准20款用于多种癌症类型的全新疗法和1款用于肺癌治疗的新装置，并将8款已批准疗法的适应症拓展至新的癌种。值得一提的是，在1990至2022年间，在全球范围内共有568款创新抗癌疗法上市。 ▲1990—2022年期间不同国家和地区的上市创新抗癌疗法数量（图片来源：参考资料[1]）免疫治疗近年来取得多项突破。例如，“first-in-class”工程化T细胞受体（TCR）T细胞疗法Tecelra（afami-cel）于去年8月获批，成为首个用于治疗实体瘤的工程化T细胞疗法，也是十余年来滑膜肉瘤领域首个有效治疗选择。靶向胃癌细胞中创新靶点Claudin18.2的治疗性抗体Vyloy（zolbetuximab）亦于去年11月获批，惠及胃癌或胃食管结合部（GEJ）腺癌患者。自2011年首份AACR抗癌进展报告发布以来，FDA批准的免疫检查点抑制剂（ICI）已由仅用于黑色素瘤的1种，增长至15种，现用于治疗20余种癌症类型。 ▲美国FDA在2024年7月1日至2025年6月30日期间所批准的抗癌疗法（图片来源：参考资料[1]）除免疫疗法外，靶向治疗同样持续推进。今年5月，组合疗法Avmapki Fakzynja Co-pack（avutometinib与defactinib）获批上市，用于治疗复发性低级别浆液性卵巢癌（LGSOC）成年患者，适用于既往接受过系统性治疗且肿瘤携带KRAS突变的人群，该方案此前已获FDA突破性疗法认定。Voranigo（vorasidenib）则于去年8月获批，成为首个在脑肿瘤中抑制IDH1与IDH2突变蛋白的靶向疗法，惠及2级星形细胞瘤或少突胶质细胞瘤患者。同时，双特异性抗体Ziihera（zanidatamab）亦于去年11月获加速批准，为胆道癌患者带来新的治疗可能。 ▲2011年与2025年，FDA批准抗癌免疫疗法、双特异性T细胞接合器以及抗体偶联药物的数量（图片来源：参考资料[1]）此外，创新治疗模式的推进同样显著。根据报告，FDA在2025年已批准9款双特异性T细胞接合器和13款抗体偶联药物（ADC），其中11款ADC针对独特靶点，进一步丰富了个体化治疗版图。综上，过去一年在FDA批准与适应症拓展的推动下，免疫与靶向等个体化疗法加速落地，为多类难治性癌症提供更精准、更有效的治疗选项。血液肿瘤领域的进展在本次报告中，AACR特别提及血液肿瘤领域的抗癌进展。血液肿瘤起源于造血组织（如骨髓），主要包括白血病、淋巴瘤和多发性骨髓瘤。2025年全球预计新发近140万例、死亡约74.5万例。在研究与技术创新的推动下，血液肿瘤死亡率显著下降，更多患者生存期延长、生活质量提升。1991—2023年间，慢性髓系白血病（CML）、非霍奇金淋巴瘤（NHL）以及多发性骨髓瘤的死亡率分别下降71%、43%和31%。 ▲2015—2025年，FDA批准针对白血病的创新靶向疗法（图片来源：参考资料[1]）血液肿瘤死亡率下降的主要原因之一，在于对其分子机理的深入研究与理解。这也促成了早期靶向疗法之一Gleevec（imatinib，伊马替尼）的问世并获批用于CML治疗。该药物的批准不仅改变了CML的临床照护，也引领了肿瘤精准医学的革命。近期，首个靶向menin的疗法Revuforj（revumenib）获批，为急性髓系白血病（AML）患者提供了新的治疗选择。2015—2025年间，FDA已批准25款用于血液肿瘤的创新靶向疗法和21款创新免疫疗法。 ▲部分再获FDA批准应用于其他癌症种类的抗癌疗法（图片来源：参考资料[1]）同时，源自白血病与淋巴瘤领域的多种疗法正被评估，并在部分情况下用于实体瘤；相关发现也逐步应用于自身免疫性疾病和罕见遗传性疾病，进一步推动跨疾病领域的转化与应用。结语 2025年AACR抗癌进展报告显示，危险因素预防与早期筛查的加强，以及免疫疗法与靶向疗法等创新疗法的相继获批，正持续推动癌症死亡率下降；尤其在血液肿瘤领域，精准医学驱动的突破显著，并带动疗法跨疾病应用。尽管挑战仍在，但在跨学科协作与持续科技创新的推动下，患者照护有望迎来更具革命性的改变，获得更长久、更高质量的生存。参考资料： [1] AACR Cancer Progress Report 2025: Contents. Retrieved September 18, 2025 from https://cancerprogressreport-aacr-org.libproxy1.nus.edu.sg/progress/cpr25-contents/ 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，传递医学新知

AACR会议疫苗

100 项与瑞维美尼相关的药物交易

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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
KMT2A易位的急性白血病	美国	Syndax Pharmaceuticals, Inc.	2024-11-15

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
急性髓性白血病	临床3期	-	Syndax Pharmaceuticals, Inc.	2025-12-01
结直肠癌	临床2期	美国	Syndax Pharmaceuticals, Inc.	2023-04-04
急性淋巴细胞白血病	临床2期	美国	Syndax Pharmaceuticals, Inc.	2019-11-05
急性淋巴细胞白血病	临床2期	澳大利亚	Syndax Pharmaceuticals, Inc.	2019-11-05
急性淋巴细胞白血病	临床2期	加拿大	Syndax Pharmaceuticals, Inc.	2019-11-05
急性淋巴细胞白血病	临床2期	法国	Syndax Pharmaceuticals, Inc.	2019-11-05
急性淋巴细胞白血病	临床2期	德国	Syndax Pharmaceuticals, Inc.	2019-11-05
急性淋巴细胞白血病	临床2期	以色列	Syndax Pharmaceuticals, Inc.	2019-11-05
急性淋巴细胞白血病	临床2期	意大利	Syndax Pharmaceuticals, Inc.	2019-11-05
急性淋巴细胞白血病	临床2期	立陶宛	Syndax Pharmaceuticals, Inc.	2019-11-05

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分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04065399 (AUGMENT-101, EHA2025)	临床1期	急性白血病 NUP98r	34	Revumenib	觸醖淵餘鬱顧製艱繭簾(鑰鹽廠鑰餘糧夢鏇衊蓋) = 壓積衊窪繭鑰選壓艱鹹糧製憲壓繭選淵衊製構 (膚構蓋齋齋鑰壓夢憲蓋 ) 更多	积极	2025-05-14
NCT04065399 (AUGMENT-101, EHA2025)	临床2期	急性白血病	116	Revumenib 163 mg	鹽艱壓壓築糧憲襯範廠(鏇窪夢廠鏇餘築觸遞選) = 選窪鹽繭糧鬱簾範簾醖蓋構鹽製鑰醖製築選壓 (憲廠簾遞窪廠願積壓齋, 54 ~ 73) 更多	积极	2025-05-14
NCT04065399 (AUGMENT-101, Company_Website \| Pubmed) 人工标引	临床2期	NPM1突变急性髓系白血病 NPM1 Mutation	84	Revumenib	鏇廠鏇鹹積繭淵窪願衊(觸築淵鹹壓夢鹽淵觸製) = 糧膚繭衊構襯構醖蓋衊艱積蓋餘觸蓋糧願繭夢 (膚醖網膚願獵觸鬱廠觸, 14 ~ 36) 更多	积极	2025-05-07
NCT04065399 (AUGMENT-101, ASH2024 \| Company_Website) 人工标引	临床2期	急性白血病 KMT2A Rearrangement	116	Revumenib 163 mg	獵夢鬱襯簾積簾鹽顧繭(廠遞網糧醖齋醖衊糧觸) = 膚願憲築襯積積壓廠糧築醖餘蓋憲願壓襯願糧 (鬱鹽鬱蓋淵範淵餘襯廠, 15 ~ 32) 更多	积极	2024-12-07
NCT05360160 (ASH2024) 人工标引	临床1/2期	急性髓性白血病 NPM1 Mutation \| KMT2A Rearrangement \| NUP98 Rearrangement	26	Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax (SAVE)	繭構觸憲選鏇範構夢鹽(餘鹽簾顧顧願糧廠顧鹽) = 鹹鬱鏇艱選醖築鹹廠齋選淵鬱壓獵顧壓醖鹽膚 (繭製簾繭夢餘築蓋齋醖 ) 更多	积极	2024-12-07
NCT04065399 (AUGMENT-101 \| SNDX-5613-0700, FDA_CDER) 人工标引	临床1/2期	KMT2A易位的急性白血病 11q23 partial tandem duplication	104	REVUFORJ	製願襯鹹鹽鑰繭齋鏇衊(鹽鑰餘餘艱憲觸顧鹹網) = 壓鹹網顧築網糧顧築鑰觸積餘積製網鬱願夢膚 (糧獵膚鹹醖糧廠積鏇醖, 13.8 ~ 30.3) 更多	积极	2024-11-15
NCT04065399 (AUGMENT-101, Pubmed \| J Clin Oncol) 人工标引	临床1/2期	伴有 11q23 异常的急性髓系白血病	94	Revumenib 163 mg (95 mg/m2 if weight <40 kg)	鑰獵膚蓋鑰鏇醖廠範憲(鹹淵衊網蓋構築範遞鹽) = 蓋繭窪窪壓糧糧構蓋膚選淵選顧鹽選鑰鑰夢艱 (餘糧夢鬱構窪繭觸淵憲, 12.7 ~ 35.8) 更多	积极	2024-08-09
NCT04065399 (AUGMENT-101, EHA 12024 \| EHA 22024) 人工标引	临床2期	伴有 11q23 异常的急性髓系白血病一线 KMT2A Rearrangement	94	Revumenib 163 mg	壓鹽鏇鏇築鹽範鑰鏇鏇(鏇齋願齋觸齋蓋艱鏇築) = 製廠觸積壓築廠夢淵憲窪鹽製顧獵糧蓋廠憲夢 (壓鏇顧獵繭蓋衊獵憲簾, 12.7 ~ 35.8) 更多	积极	2024-05-14
NCT05326516 (EHA2024)	临床1期	白血病	27	Revumenib + FLA DL1	憲觸選遞製齋願網淵廠(憲顧壓選鹽範壓網壓築) = occurred in >50% of pts 願憲糧鹹製範構襯範糧 (廠遞憲願齋淵鏇選選餘 ) 更多	积极	2024-05-14
NCT05326516 (EHA2024)	临床1期	白血病	27	Revumenib + FLA DL2		积极	2024-05-14
NCT04065399 (AUGMENT-101, PRNewswire) 人工标引	临床2期	急性白血病 KMT2A Rearrangement	57	Revumenib	積選遞廠網窪廠鏇夢淵(廠鑰願襯願膚夢獵積襯) = 衊範製壓構蓋壓顧顧遞廠艱獵壓網遞鑰網壓獵 (構願窪構選製積艱艱積, 5.0 ~ 53.8) 更多	积极	2024-04-08