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.

NCT07212023

/ Not yet recruiting临床2期

A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of Revumenib in Combination With Intensive Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia With a KMT2A or NUP98 Gene Rearrangement (REVEAL-ND KMT2A)

The purpose of this study is to assess if adding revumenib to standard chemotherapy improves outcomes in participants with acute myeloid leukemia (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.

NCT07052994

/ Not yet recruiting临床1期IIT

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

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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）

2026-01-15·INTERNATIONAL JOURNAL OF CANCER

Targeting the Menin–KMT2A interaction in leukemia: Lessons learned and future directions

Review

作者: Florian Perner ; Jayant Y. Gadrey ; Michael W. M. Kühn ; Scott A. Armstrong

Abstract:

Chromosomal rearrangements involving the Mixed Lineage Leukemia gene ( MLL1 , KMT2A ) are defining a genetically distinct subset in about 10% of human acute leukemias. Translocations involving the KMT2A ‐locus at chromosome 11q23 are resulting in the formation of a chimeric oncogene, where the N‐terminal part of KMT2A is fused to a variety of translocation partners. The most frequently found fusion partners of KMT2A in acute leukemia are the C‐terminal parts of AFF1, MLLT3, MLLT1 and MLLT10. Unfortunately, the presence of an KMT2A‐rearrangements is associated with adverse outcomes in leukemia patients. Moreover, non‐rearranged KMT2A‐complexes have been demonstrated to be crucial for disease development and maintenance in NPM1‐mutated and NUP98‐rearranged leukemia, expanding the spectrum of genetic disease subtypes that are dependent on KMT2A . Recent advances in the development of targeted therapy strategies to disrupt the function of KMT2A‐complexes in leukemia have led to the establishment of Menin–KMT2A interaction inhibitors that effectively eradicate leukemia in preclinical model systems and show favorable tolerability and significant efficacy in early‐phase clinical trials. Indeed, one Menin inhibitor, Revumenib, was recently approved for the treatment of patients with relapsed or refractory KMT2A‐rearranged acute leukemia. However, single agent therapy can lead to resistance. In this Review article we summarize our current understanding about the biology of pathogenic KMT2A‐complex function in cancer, specifically leukemia, and give a systematic overview of lessons learned from recent clinical and preclinical studies using Menin inhibitors.

2025-12-01·ANNALS OF PHARMACOTHERAPY

Revumenib for Relapsed or Refractory Acute Leukemia With a KMT2A Translocation

Review

作者: Faro, Sarah Jane E. ; Lynch, Emilie J. ; Lindstrom, Alec M. ; Sethi, Nadia A. ; Seligson, Nathan D. ; Wang, Wendy Y.

Objective::

To review the pharmacology, efficacy, and safety of revumenib (Revuforj) for relapsed or refractory (r/r) acute leukemia with a lysine methyltransferase 2A ( KMT2A) gene rearrangement or translocation ( KMT2Ar ).

Data Sources::

A literature search was conducted using PubMed/MEDLINE, applicable published abstracts, and ongoing studies from ClinicalTrials.gov between January 1, 1981, and April 23, 2025. Keywords included Revuforj, revumenib, SNDX-5613, KMT2A , MLL1 , and menin.

Study Selection and Data Extraction::

All English-language studies involving revumenib for r/r acute leukemia with a KMT2Ar were included.

Data Synthesis::

Revumenib, a protein-protein inhibitor that interrupts the interaction between the KMT2A protein and the scaffold protein menin, was granted approval by the Food and Drug Administration (FDA) for r/r acute leukemia with KMT2Ar based on a phase 2 clinical trial in adult and pediatric patients (n = 57), which reported a complete remission or complete remission with partial hematologic recovery of 22.8%. Common grade 3/4 adverse reactions reported for revumenib include infectious (febrile neutropenia 33%; infection 29%; bacterial infection 20%) and hematologic events (differentiation syndrome 13%; hemorrhage 9%; thrombosis 5%). Grade 3/4 QT prolongation, the primary dose-limiting adverse effect, was present in 12% of patients. Differentiation syndrome, related to revumenib’s antileukemic effect, was observed in 29% of patients (grade 3/4: 13%; grade 5: <1%). We also include long-term follow-up for a total of 104 and 135 patients for efficacy and safety results, respectively.

Relevance to Patient Care and Clinical Practice in Comparison to Existing Drugs::

In the high-risk disease of r/r acute leukemia with KMT2Ar , given limited treatment options, revumenib appears to be a viable, novel treatment option demonstrating clinical efficacy and a manageable adverse effect profile that can be utilized as a bridge to stem cell transplant. Existing therapy options in this setting may include additional traditional chemotherapy, chimeric antigen receptor T-cell therapy (CAR-T), antibody-drug conjugates (eg, gemtuzumab, inotuzumab), bispecific T-cell engager (BiTE) therapies (eg, blinatumomab), DNA methyltransferase inhibitors (eg, azacitidine, decitabine), histone deacetylase inhibitors (eg, vorinostat, panobinostat), and BCL-2 inhibitors (venetoclax).

Conclusions::

Revumenib is an innovative targeted treatment with promising activity in r/r acute leukemia with KMT2Ar .

2025-11-03·Blood Cancer Discovery

Combination Strategies with Menin Inhibitors for Acute Leukemia

Review

作者: Kantarjian, Hagop M. ; Bataller, Alex ; Issa, Ghayas C. ; Stein, Eytan M. ; Cai, Sheng F.

Abstract:

Menin inhibitors are targeted therapies for the treatment of genetically defined subsets of acute leukemia. The menin inhibitor revumenib is approved for the treatment of relapsed or refractory leukemia with rearrangement of KMT2A. In addition, multiple menin inhibitors are currently in clinical development aimed at targeting other additional subsets, such as NPM1 mutations, which form up to 30% of acute myeloid leukemia. However, as observed with other targeted therapies for cancer, on-target resistance mutations emerged in advanced cases following monotherapy. Therefore, combination strategies incorporating menin inhibitors are needed to improve durability and depth of remission.

Significance::

Menin inhibitors have the potential to reshape treatment paradigms in acute leukemia. There are multiple promising preclinical combinations that require careful clinical trial design and implementation for a successful translation into the clinical setting.

254

项与瑞维美尼相关的新闻（医药）

2025-11-16

·无癌家园

“精准打击”癌细胞！癌症疫苗、ADC药物、CAR-T疗法十月肿瘤领域“黑科技”集中爆发！

关注无癌家园我们战癌到底对癌症患者而言，2025年10月无疑是一个充满希望的月份。在这个月，国家药监局批准了多款创新抗肿瘤药物上市，为晚期乳腺癌、肺癌患者提供了新的治疗选择。同时，国际顶级医学期刊相继发表多项重磅研究，北京大学肿瘤医院团队在尿路上皮癌治疗领域取得突破性进展，中国在CAR-T细胞治疗实体瘤方面也传出捷报。图源摄图网（已授权）抗体药物偶联物（ADC）：肿瘤治疗的“精准炸弹” 十月，抗体药物偶联物（ADC）领域迎来多项重大进展，这种精准靶向癌细胞的药物正在改变多种癌症的治疗格局。乳腺癌迎来新希望 2025年10月17日，国家药监局正式批准四川科伦博泰生物医药股份有限公司申报的注射用博度曲妥珠单抗（商品名：舒泰莱®）上市。图源NMPA官网该药物用于既往接受过一种或一种以上抗HER2药物治疗的不可切除或转移性HER2阳性成人乳腺癌患者，标志着中国自主创新ADC药物在肿瘤治疗领域取得重大进展。此次获批使博度曲妥珠单抗成为中国首个可以广泛覆盖二线及以上HER2阳性乳腺癌患者的国产HER2 ADC药物。这一突破性进展不仅丰富了国内HER2阳性乳腺癌的治疗选择，更彰显了中国企业在全球创新药物研发领域的实力与潜力，为晚期乳腺癌患者带来了新的希望。尿路上皮癌治疗突破瓶颈 2025年10月19日，由北京大学肿瘤医院郭军、盛锡楠教授团队主导的全球首个抗HER2抗体偶联药物用于晚期尿路上皮癌治疗的随机对照3期临床研究结果发布于国际医学期刊《新英格兰医学杂志》，并由郭军教授同步在2025年欧洲肿瘤内科大会（ESMO）主席论坛进行口头报告，引起国内外的广泛关注。这项名为RC48-016的研究评估了HER2-ADC维迪西妥单抗联合特瑞普利单抗疗法对比传统化疗用于既往未接受过系统治疗的晚期尿路上皮癌的疗效与安全性。研究结果显示，截至数据分析，中位随访时间为18.2个月，联合治疗组仍有37.4%受试者接受治疗，而化疗对照组为0。与化疗组相比，联合治疗组无进展生存期显著延长（13.1个月 vs 6.5个月）。更令人振奋的是，联合治疗组总生存期几乎翻倍（31.5个月 vs 16.9个月），死亡风险降低了46%；且客观有效率显著提高（76.1% vs 50.2%），治疗相关的严重不良反应发生率却更低（28.4% vs 40.5%）。新型ADC药物靶向结直肠癌 2025年11月5日，国际著名《Nature》杂志发表了一项关于新型氨基双膦酸盐类EGFR抗体药物偶联物的研究。研究人员将抗癌药物西妥昔单抗（Cetuximab）与两种新型氨基双膦酸盐类似物结合，开发出能同时直接抑制肿瘤细胞生长和激活Vδ2 T淋巴细胞的双功能ADC药物。临床前研究显示，该药物在结直肠癌模型中表现出显著抑制肿瘤生长能力，为EGFR阳性结直肠癌患者提供了新的治疗方向。免疫治疗：唤醒体内“抗癌卫士” 免疫治疗通过调动患者自身的免疫系统来攻击癌细胞，十月期间，这一领域也有多项突破。纳米铠甲CAR-T攻克实体瘤 2025年10月24日，上海大学附属孟超肿瘤医院联合多家机构公布了一项创新研究——研究人员给CAR-T细胞穿上了一层 “纳米铠甲” ，成功用于治疗恶性间皮瘤这一难治性实体瘤。研究团队将从羊驼身上获得的抗PD-1纳米抗体与靶向间皮素（MSLN）的CAR结合，造出“装甲化CAR-T细胞”，即NAC-T细胞。图源Advanced Science官网在首次开展的临床Ⅰ期试验中，11位经过标准治疗后失败的恶性间皮瘤患者接受了这种“装甲CAR-T”的治疗。结果显示，63.6%的患者肿瘤明显缩小，其中1位患者肿瘤完全消失，6位患者肿瘤大幅缩小，剩下4位患者病情稳定，病情控制率达到100%。患者的中位总生存期达到了25.6个月，远超目前二线治疗约11个月的水平。那位肿瘤完全消失的患者，已经健康无瘤生存了超过3年半（45.6个月），仍保持无瘤生存状态。目前无癌家园免疫临床试验急招胃癌、肝癌、卵巢癌、食管鳞癌、急性淋巴细胞白血病、非霍奇金淋巴瘤、间皮瘤、多发性骨髓瘤等癌种！除了上述提到的血液肿瘤外，实体瘤癌友在经济条件允许的情况下可以尝试CAR-T项目进行治疗。具体流程可将病理报告、治疗经历及出院小结等提交至无癌家园医学部400-626-9916进行初步评估！联合疗法应对罕见肿瘤 2025年10月31日，《Medine》杂志发表了一例SMARCA4缺陷未分化肿瘤（SMARCA4-UT）的成功治疗案例。这名60岁男性患者患有罕见的胸部SMARCA4-UT，基因组分析显示其肿瘤突变负荷高达15.95 muts/Mb。医疗团队采用免疫治疗（替雷利珠单抗）联合化疗和放疗的综合方案，使患者的总生存期超过33个月（截至2025年7月），为这类罕见肿瘤的治疗提供了新思路。小分子靶向药：精准打击癌细胞的“制导武器” 小分子靶向药物能精准抑制肿瘤生长关键蛋白，十月有多个此类药物获批准或有新研究进展。肺癌靶向治疗新选择 2025年10月24日，国家药监局批准我国申报的1类创新药马来酸美凡厄替尼片（商品名：迈瑞东）上市。该药适用于具有表皮生长因子受体（EGFR）外显子21（L858R）置换突变的局部晚期或转移性非小细胞肺癌成人患者的一线治疗，为特定肺癌人群提供了新的治疗选择。 RET抑制剂进军肺癌治疗 2025年10月23日，翰森制药宣布其自主研发的高选择性RET抑制剂HS-10365胶囊上市许可申请获国家药监局受理。该药用于治疗RET基因融合阳性的局部晚期或转移性非小细胞肺癌成人患者，是一种直接作用于胞内蛋白酪氨酸激酶的小分子抑制剂，通过与ATP竞争性结合，抑制酪氨酸激酶磷酸化，进而有效控制肿瘤生长。国际进展：FDA批准多款抗癌新药 10月份，美国FDA也批准了多款肿瘤治疗药物，为全球抗癌战线增添新武器。白血病新药上市 2025年10月，FDA批准了Revuforj用于治疗复发/难治性NPM1突变急性髓系白血病。此次批准基于AUGMENT-101临床试验，该研究显示接受治疗的患者完全缓解或伴血小板减少的完全缓解率达到23.1%，中位缓解持续时间4.5个月。在研究开始时依赖红细胞或血小板输注的46例患者中，8例（17%）在治疗后56天内实现了输血独立。这些结果表明，Revuforj有望成为NPM1突变驱动的复发或难治性急性髓系白血病（AML）患者的一种有效治疗选择。多发性骨髓瘤迎新方案 FDA还批准了Blenrep与Velcade和地塞米松联合用于复发多发性骨髓瘤。此次批准基于III期DREAMM-7试验的数据，该试验表明 BVd 疗法显著改善了患者的预后。对于接受过两线或两线以上既往治疗的患者，与基于达雷妥尤单抗的方案相比，BVd 疗法使死亡风险降低了 51%，并将中位无进展生存期延长至 31.3 个月，是后者的三倍。该联合疗法总体耐受性良好，其安全性与每种单药的已知作用一致。这项批准为已经尝试过多种疗法的多发性骨髓瘤患者提供了一种新的治疗选择，既能提高生存率，又能更好地控制病情。小细胞肺癌联合疗法 FDA还批准了Zepzelca（lurbinectedin）与 Tecentriq 联合使用。（atezolizumab）用于治疗接受 Tecentriq、卡铂和依托泊苷一线诱导治疗后病情未进展的广泛期小细胞肺癌（ES-SCLC）成人患者。在 IMforte 临床试验中，483 名患者被随机分配接受 Zepzelca 联合 Tecentriq 治疗或单独使用 Tecentriq 治疗。联合治疗组的中位总生存期从 Tecentriq 单药治疗组的 10.6 个月提高至 13.2 个月。中位无进展生存期也从 2.1 个月延长至 5.4 个月。Zepzelca 是一种烷化剂，通过破坏癌细胞 DNA 并阻止其分裂来杀死癌细胞。高危皮肤鳞状细胞癌辅助治疗 FDA批准Libtayo（cemiplimab-rwlc）用于手术和放疗后复发风险高的成人皮肤鳞状细胞癌的辅助治疗。此次批准基于C-POST试验，该试验纳入了415名复发风险高的患者。接受Libtayo治疗的患者中位无病生存期尚未达到，而安慰剂组为49.4个月，这凸显了该药物预防复发的能力。机不可失！多款抗癌新药临床招募进行中众所周知，肿瘤患者的抗癌过程是与时间的赛跑，国际肿瘤新药从在国外上市到国内投入临床使用的“时间差”，直接影响治愈率和生存率，成为很多患者熬不过去的坎儿。通常情况下这些上市的抗癌新药大多远在美国，价格昂贵，被称为“昂贵的国外专利药”。中国只有少数经济条件雄厚的患者能够前往美国接受治疗，而多数癌友们只能望药兴叹，白白错过最佳的药物治疗时机。据国家癌症中心数据显示，中国以22%的人口占全球癌症新发人数的21.79%以上，对最新抗癌药的需求量巨大。尽管国外新药审批上市正在提速，上市周期长，数量少是现实，与临床需求之间仍存在巨大缺口，国内患者“新药难求”的困境一直存在。近几年，随着政府的重视，我国加快了抗癌药入市和研发的步伐，国内抗癌新药的研发速度也呈现井喷状态。好消息是，目前国外上市的以及中国自主研发的多款靶向及免疫的抗肿瘤新药，开始招募国内患者了！这意味着，国内的患者也终于有机会免费用上这些效果更好，堪称天价的抗癌新疗法！想申请的患者可以联系无癌家园医学部（400-626-9916）进行初步评估，我们也将及时为大家提供招募信息。未来展望随着十月这些创新疗法陆续获批和研究突破，肿瘤治疗领域又向前迈出了坚实的一步。从精准靶向的ADC药物到改造免疫细胞的CAR-T技术，从克服耐药的小分子抑制剂到联合多种机制的免疫疗法，癌症正逐渐从“不治之症”转变为可控可治的慢性病。本文为无癌家园原创，转载需授权参考文献 1.https://www.ccfdie.org/cn/gdxw/webinfo/2025/10/1759927998978157.htm 2.https://www.nature.com/articles/s41598-025-22669-w 3.https://www.nejm.org/doi/abs/10.1056/NEJMoa2511648 4.https://advanced.onlinelibrary.wiley.com/doi/10.1002/advs.202508754 5.https://journals.lww.com/md-journal/fulltext/2025/10310/effective_management_of_smarca4_deficient.153.aspx 6.https://www.curetoday.com/view/covering-every-fda-oncology-approval-from-october-2025 特别提醒需要特别提醒病友们的是，临床试验并不适合极晚期的患者，比如进展期的脑转移患者，体力评分不佳或卧床不起，肝肾功能低下和贫血较严重者通常无法入组，需要提升免疫，调整身体状态后再进行评估，可以通过无癌家园营养免疫的相关专家寻求改善晚期状态的方案。此外，正在接受正规治疗的早期患者也要多关注新药进展，一旦病情进展，可以寻求新药的临床试验。无癌家园抗癌资讯｜新技术｜新药研发｜权威专家热门临床试验相关文章 Review past ▶▶▶ 1.【实体瘤细胞疗法招募】急招宫颈癌患者！实体瘤“杀手锏”TILS细胞治疗临床试验强势来袭！ 2.【TCR-T疗法免费招募】TC-N201注射液终于启动临床啦！现正急招滑膜肉瘤等NY-ESO-1阳性实体瘤患者！ 3.【肺癌新药招募】@EGFR 20ins非小细胞肺癌患者注意啦！新一代靶向药PLB1004终于启动临床啦！ 4. 肺癌治疗耐药难题破解！TILs疗法联合PD-1仍能让转移性肺癌患者完全缓解！ 5.【CAR-T-19招募】RC19D2注射液终于启动临床啦！急招B细胞急性淋巴细胞白血病患者！分享、收藏、点赞、在看安排一下？

抗体药物偶联物细胞疗法免疫疗法临床3期临床结果

2025-11-14

·Pharmaceutical Technology

Kura and Kyowa Kirin secure FDA go-ahead for targeted leukaemia therapy

Kura Oncology and Kyowa Kirin’s Komzifti has gained FDA approval in adult patients with relapsed or refractory acute myeloid leukaemia (r/r AML) with a NPM1 mutation, joining Syndax’s Revuforj as the second menin inhibitor on the AML market. Image credit: Ekahardiwito via Shutterstock.com. Kyowa Kirin’s bet on Kura Oncology appears to have paid off, as the companies have secured approval for their jointly developed drug, Komzifti (ziftomenib). Following the US Food and Drug Administration’s (FDA) call to greenlight the drug, Komzifti will now be available to adult patients with relapsed or refractory acute myeloid leukaemia (r/r AML). The targeted therapy is specifically indicated for patients with a susceptible Nucleophosmin 1 (NPM1) mutation. Komzifti obtained the approval based on the results of the Phase III KOMET-001 study (NCT04067336), which saw the drug trigger complete remission (CR) plus CR with partial haematological recovery (CRh) in 21.4% of patients. This effect was sustained over a median duration of five months. As Komzifti enters the market, it becomes the first once-daily oral menin inhibitor to gain FDA approval in NPM1-mutated r/r AML – an indication with limited treatment options that is commonly associated with poor patient prognoses. It is currently estimated that just under one-third of AML cases present a NPM1 founder mutation. Within this patient population, around 70% of patients will relapse after first-line treatment within three years while 20% do not respond to first-line therapies at all. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Following Komzifti’s approval in this indication, Kura Oncology’s CEO and president Troy Wilson noted that the therapy could become the menin inhibitor of choice within its approved indication due to its “best-in-class profile”. Wilson adds that Kura and Kyowa Kirin are “fully prepared” to launch Komzifti. The pair will also turn their attention to exploring Komzfti’s efficacy in the front-line and combination settings, which could further bolster the drug’s positioning in the AML market. Outside of this indication, Kura is also looking into Komzifti’s potential in treating solid tumours, with the drug having entered the Phase Ia/Ib KOMET-015 trial for gastrointestinal stromal tumours (GIST) in April 2025. Vying for menin inhibitor glory Kyowa Kirin joined forces with Kura in November 2024 to assist with the global development and commercialisation of the drug – setting the company back up to $1.53bn. While Kura will take responsibility for the marketing and manufacturing of Komzifti in the US, Kyowa Kirin will deal with these tasks outside of the US. However, Komzifti will not join the market alone, as less than one month ago, Syndax Pharmaceuticals’ Revuforj (revumenib) got the FDA go-ahead in NPM1-mutated r/r AML. While these drugs exhibited similar efficacy in their respective studies, Revuforj did trigger CR plus CRh in 23% – sitting marginally above Komzifti’s performance in the KOMET-001 study. However, Komzifti does hold a dosing edge over its rival, as it is administered once daily, rather than the twice-daily regimen indicated for Revuforj. Despite this, analysts at GlobalData forecast that Revforj will come out on top sales-wise, becoming a blockbuster seller for Syndax. In 2031, the drug is estimated to pull in $1.1bn for the Massachusetts-based pharma. Meanwhile, analysts predict that Komzifti is set to make $793m for Kura and Kyowa Kirin’s therapy in the same year. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

临床结果上市批准临床3期

2025-11-13

·FiercePharma

Kyowa's bet on Kura pays off as partners' oral med for AML subset wins FDA approval

While specific treatment options for the roughly one-third of AML patients with NPM1 mutations have historically been limited, Syndax Pharmaceuticals broke new ground in late October when the FDA cleared its drug Revuforj as the first menin inhibitor in the indication. Kyowa Kirin’s big bet on Kura Oncology has paid off in short order, delivering an FDA approval for a medicine to treat a subset of patients with acute myeloid leukemia.On Thursday, the FDA signed off on Kura’s menin inhibitor ziftomenib as a new treatment for adults with relapsed or refractory acute myeloid leukemia (AML) who have a susceptible nucleophosmin 1 (NPM1) mutation. To qualify for the treatment, which will be marketed under the brand name Komzifti, patients must not be a good fit for any alternative treatments, the FDA said in a Nov. 13 approval announcement.While specific treatment options for the roughly one-third of AML patients with NPM1 mutations have historically been limited, Syndax Pharmaceuticals broke new ground in late October when the FDA cleared its drug Revuforj as the first menin inhibitor in the indication. Syndax’s drug was originally approved last November to treat a genetic type of leukemia called lysine methyltransferase 2A (KMT2A).The two meds, both members of the same class, will now likely compete directly over the indication.NPM1 mutations rank among the most common founder mutations in AML, Kura and Kyowa Kirin said in a joint press release Thursday. Roughly 20% of patients with NPM1-m AML do not respond to frontline therapy, and of those who do, some 70% will relapse within three years, according to the partners. The FDA granted its green light after reviewing data from the partners’ pivotal KOMET-001 trial, in which 21.4% patients on Komzifti achieved complete remission (CR) or CR with partial hematologic recovery (CRh), Kura and Kyowa Kirin said in their release. The median duration of remission among those who achieved CR or CRh clocked in at 5 months, the partners said. Further, 88% of patients who hit either CR or CRh did so within half a year of starting treatment with Komzifti, Kura and Kyowa Kirin added.Komzifti will carry a boxed warning for differentiation syndrome—a potentially severe reaction to certain leukemia treatments—though patients and doctors applying more scrutiny to which drugs they prescribe alongside Komzifti can ease those potential risks, according to the companies’ approval announcement. The drug also bears precautions around QTc interval prolongation and embryo-fetal toxicity.Syndax’s Revuforj label bears similar warnings and precautions, with its boxed warnings discussing risks of differentiation syndrome, QTc prolongation and the fast heart rhythm condition Torsades de Pointes.The safety profile of Kura and Kyowa's drug "overall looks a bit better" than that for Revuforj, analysts at Mizuho Securities concluded, adding that a differentiation syndrome boxed warning for the menin inhibitor class comes as "[n]o real surprise." “Komzifti combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options,” Troy Wilson, Ph.D., CEO of Kura, said in a statement. “These features highlight Komzifti’s potential to serve as the menin inhibitor of choice in its approved indication.”Kyowa Kirin hitched its wagon to Kura and ziftomenib just shy of a year ago, paying $330 million upfront in November 2024—and pledging up to $1.1 billion in potential milestones—to split development and commercialization on the asset.Under the deal, Kura is in charge of development, regulatory and commercial strategy for Komzifti in the U.S., as well as manufacturing, while Kyowa Kirin is holding down R&D and marketing duties outside the U.S. In their press release, the partners said that following the approval, they will now “jointly perform certain commercialization activities in accordance with a co-created U.S. territory commercialization plan.”Kura’s stock was trading up around 1% on Thursday morning. Editor's note: This story was updated to clarify information on Komzifti's label, and to include analyst commentary.

上市批准临床结果临床研究

100 项与瑞维美尼相关的药物交易

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适应症	国家/地区	公司	日期
NPM1突变急性髓系白血病	美国	Syndax Pharmaceuticals, Inc.	2025-10-24
KMT2A易位的急性白血病	美国	Syndax Pharmaceuticals, Inc.	2024-11-15

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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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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2期	NPM1突变急性髓系白血病	84	Revumenib 160 mg	鏇醖膚膚衊獵餘醖繭構(網艱衊艱獵齋鹽齋醖製) = 餘壓鏇製鏇鑰醖網壓襯網膚襯構範醖壓構齋廠 (觸窪繭構蓋糧網鬱餘艱, 36.5% ~ 59.7) 更多	积极	2025-05-14
NCT04065399 (AUGMENT-101, EHA2025)	临床1期	急性白血病 NUP98r	34	Revumenib	簾糧廠鹽鏇窪構齋製遞(艱鏇顧壓淵艱網鏇築壓) = 鏇壓蓋繭糧鏇觸蓋艱範選顧獵選構遞獵鹹願積 (範獵構獵鹹鹹襯願鹹築 ) 更多	积极	2025-05-14
NCT04065399 (AUGMENT-101, EHA2025)	临床2期	急性白血病	116	Revumenib 163 mg	鏇願淵夢簾簾遞獵廠簾(壓選製範憲衊齋繭遞蓋) = 齋憲蓋淵選選築願艱選網鹽繭鬱壓遞壓衊網憲 (鑰蓋憲壓繭衊憲製鑰繭, 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	襯膚廠餘範壓簾淵衊廠(蓋鏇積構遞艱糧憲齋觸) = 願餘繭衊獵窪網顧築獵餘壓淵鹹淵鬱餘餘繭餘 (選憲夢醖夢積網網衊膚, 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	壓齋範鬱鬱選窪鑰築範(顧範簾餘範窪醖鬱鏇繭) = 製衊願艱範範鏇夢憲膚遞衊齋襯願廠構選鑰蓋 (願範夢齋夢願鬱鹹鬱膚, 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)	網憲憲糧窪築壓襯艱積(糧鑰遞襯觸範鬱選壓憲) = 獵觸選製醖選艱願廠製衊選淵鑰窪構鹹窪鏇艱 (遞顧鏇簾範築淵衊構憲 ) 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	窪餘廠構選鑰膚憲鑰醖(憲餘繭鬱築醖築廠窪糧) = 鹹鹽廠壓鏇鏇醖廠齋顧積顧廠窪製鬱顧觸製壓 (簾醖顧遞鹹構鏇範網獵, 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)	積夢積衊網願觸醖簾餘(襯築淵鏇淵獵壓鹽願襯) = 淵遞醖鑰壓鬱窪製衊衊壓築鑰餘鹽觸淵構鏇鹽 (鑰鹹範餘衊繭鏇觸窪築, 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	鬱範築衊衊築顧鹹鬱夢(構齋膚襯窪鹽構夢築鑰) = 鹽餘夢艱築網艱淵製顧糧憲範廠鑰願夢顧鹽鹽 (鹽製構範鬱廠餘餘蓋願, 12.7 ~ 35.8) 更多	积极	2024-05-14
EHA2024	临床1/2期	急性髓性白血病 KMT2A-r \| NPM1m	55	Revumenib	顧廠觸鹹鏇願廠襯觸窪(衊獵簾構糧鬱構鹽糧醖) = 選餘廠鹽齋衊糧襯範製齋窪餘鹽壓積鏇築醖廠 (窪遞製網積選構艱積繭 ) 更多	积极	2024-05-14