This phase II trial tests how well ziftomenib works in treating patients with NPM1 mutated or KMT2A rearranged acute myeloid leukemia (AML) and are not eligible to receive standard therapy. AML is often due to genetic changes in the cancer cells, including mutations in the NPM1 gene and rearrangements involving the KMT2A gene. These mutations result in activation of the menin pathway. Menin is a type of protein in the body that helps to regulate some of the naturally occurring processes in the body, but can also be involved in some types of cancers. Ziftomenib blocks this menin pathway and may prevent the cancer cells from continuing to grow. Giving ziftomenib may kill more cancer cells in patients with NPM1 mutated or KMT2A rearranged AML that are not eligible to receive standard therapy.

开始日期2026-01-10

申办/合作机构

Ohio State University Comprehensive Cancer Center

[+1]

NCT07007312

/ Recruiting临床3期

Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with eligible genetic alterations. Ziftomenib is a type of therapy known to target the menin pathway in cancer cells.
This protocol has 2 separate studies that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) AML treatments in patients with certain genetic mutations who have not received any treatment for their AML. In the first study, the Nonintensive Therapy Study, older patients or those with serious medical problems will receive the SOC therapies venetoclax (ven) and azacitidine (aza), plus either ziftomenib or a placebo. In the second study, the Intensive Therapy Study, medically fit patients will receive (a) the SOC therapies cytarabine and daunorubicin, plus either ziftomenib or a placebo during a first treatment phase called induction, (b) cytarabine plus either ziftomenib or a placebo during a second treatment phase called consolidation, and (c) ziftomenib or a placebo during a third treatment phase called maintenance.
The physician will determine which study is the appropriate treatment for the patient, but neither the patient nor their physician will know whether the patient has been assigned to receive ziftomenib or a placebo. This design is called "double-blinded".

开始日期2025-09-26

申办/合作机构

Kura Oncology, Inc.

NCT06769490

/ Recruiting临床1期

Phase 1 Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia

The goal of this all-oral combination is to deliver safe and effective therapy for the largest portion of AML subtypes (NPM1mt, KMT2Ar, NUP98r
40-45%).

开始日期2025-07-10

申办/合作机构-

100 项与 Ziftomenib 相关的临床结果

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100 项与 Ziftomenib 相关的转化医学

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100 项与 Ziftomenib 相关的专利（医药）

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项与 Ziftomenib 相关的文献（医药）

2025-11-01·CURRENT OPINION IN ONCOLOGY

The role of menin inhibitors in acute myeloid leukemia

Review

作者: Marconi, Giovanni ; Isidori, Alessandro

Purpose of review:

Acute myeloid leukemia (AML) characterized by NPM1 mutations or KMT2A rearrangements depends on abnormal epigenetic programs mediated by menin, a critical scaffold protein for sustaining the expression of oncogenic HOX/MEIS1 genes. Therefore, menin inhibitors have become a promising class of AML treatments.

Recent findings:

Early-phase trials have shown that agents such as revumenib, ziftomenib, bleximenib, and enzomenib are active, particularly in relapsed/refractory disease, with 23–48% of patients achieving composite complete remission. However, the durability of single-agent treatment remains limited, and resistance mechanisms, such as MEN1 mutations or transcriptional reprogramming, have been identified. Consequently, combination approaches involving venetoclax, hypomethylating agents, or chemotherapy are being investigated to improve response depth and duration. Recent SAVE, KOMET-007, and cAMeLot-2 trials have demonstrated high overall response rates (68%-100%) and encouraging MRD-negative complete remissions when combining menin inhibitors with venetoclax-based regimens. This has been observed even in patients who have previously received venetoclax. Combinations with intensive chemotherapy (e.g., 7 + 3) in the frontline setting have also yielded high CRc rates (up to 94% in NPM1-mutated AML) without exacerbating toxicity.

Summary:

These findings justify the integration of menin inhibitors into the AML therapeutic landscape, and support ongoing randomized trials to confirm their benefit in both frontline and relapse or refractory settings.

2025-11-01·JOURNAL OF CLINICAL ONCOLOGY

Ziftomenib in Relapsed or Refractory NPM1 -Mutated AML

Article

作者: Balasubramanian, Suresh ; Schwarzer, Adrian ; Altman, Jessica K. ; Garcia, Olga Salamero ; Redner, Robert L. ; Papayannidis, Cristina ; Foran, James ; Heidel, Florian ; Fathi, Amir T. ; Madanat, Yazan F. ; Riches, Marcie ; Heidel, Florian H. ; Bergeron, Julie ; Pettit, Kristen ; de la Fuente Burguera, Adolfo ; Issa, Ghayas C. ; Corum, Daniel ; Rodríguez-Arbolí, Eduardo ; Deeren, Dries ; Zhang, Zijing ; del Castillo, Teresa Bernal ; Salamero Garcia, Olga ; Heiblig, Maël ; Ades, Lionel ; Venditti, Adriano ; Esteve Reyner, Jordi ; Soifer, Harris S. ; Walter, Roland B. ; Peterlin, Pierre ; Lanza, Francesco ; Ulrickson, Matthew ; Penedo, Agustín ; Wang, Eunice S. ; Giannini, Caterina ; Berthon, Céline ; Khawandanah, Mohamad ; Baer, Maria R. ; Elsawy, Mahmoud ; Erba, Harry ; Tabachri, Marilyn ; Roboz, Gail J. ; Pigneux, Arnaud ; Leoni, Mollie ; Shah, Mithun ; McCloskey, James ; Redner, Robert ; Shah, Mithun V. ; Mitra, Amitava ; Fedorov, Kateryna ; Montesinos, Pau ; Barabé, Frederic

PURPOSE:

Ziftomenib—a potent, highly selective, oral menin inhibitor—was well tolerated and demonstrated encouraging clinical activity as monotherapy for relapsed/refractory NPM1 -mutated ( NPM1 -m) and KMT2A -rearranged AML in the KOMET-001 phase I trial.

METHODS:

In the registration-enabling phase II part of KOMET-001, patients with relapsed/refractory NPM1 -m AML received ziftomenib 600 mg once daily. The primary end point was the rate of complete remission with full hematologic recovery (CR)/CR with partial hematologic recovery (CRh).

RESULTS:

From January 26, 2023, to May 13, 2024, 92 patients (median age, 69 years [range, 33-84]) were treated. The primary end point was met, with a CR/CRh rate of 22% (95% CI, 14 to 32; P = .0058); 61% were negative for measurable residual disease. Overall response rate was 33% (95% CI, 23 to 43), with a median duration of 4.6 months (95% CI, 2.8 to 7.4). Prespecified subgroup analyses showed comparable CR/CRh regardless of previous therapy, including venetoclax, or type of comutations. Median overall survival was 6.6 months (95% CI, 3.6 to 8.6). Common grade ≥3 treatment-emergent adverse events were febrile neutropenia (26%), anemia (20%), and thrombocytopenia (20%). Differentiation syndrome occurred in 25% of patients (15% grade 3; no grade 4-5) and was manageable with protocol-defined mitigation. Three patients (3%) discontinued treatment because of ziftomenib-related adverse events.

CONCLUSION:

Ziftomenib demonstrated significant clinical benefit and deep responses in patients with heavily pretreated, relapsed/refractory NPM1 -m AML. Ziftomenib was well tolerated with a safety profile consistent with previous studies, including manageable differentiation syndrome, lack of clinically significant QTc prolongation, and low rates of myelosuppression.

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.

215

项与 Ziftomenib 相关的新闻（医药）

2025-11-14

·三优生物医药

三优十周年｜血液肿瘤治疗革命—精准分层治疗新时代

作者：周予西陈小美美工：何国红罗真真排版：马超人体内的造血系统犹如一座精密的"生命工厂"，不断制造各类血细胞维持机体运转。而当这座工厂的"质量控制"系统出现故障，就会导致血液肿瘤的发生——源于造血细胞的恶性疾病，可以累及骨髓、血液及全身各个脏器和组织。临床常见的血液肿瘤主要包括各种类型的白血病、多发性骨髓瘤、淋巴瘤、骨髓增生异常综合征、骨髓增殖性肿瘤等，其发病率和死亡率均居前列，是严重危害人类健康的重大疾病。血液瘤中发病人数最多的是白血病、多发性骨髓瘤和淋巴瘤。白血病约占血液瘤的36%，多发性骨髓瘤约占13%，淋巴瘤约占51%，三种血液瘤全球发病率超过13/10万人（图1）。 ▲ 图1 血液肿瘤概览随着创新疗法的不断涌现，血液瘤药物市场持续扩容，其中大分子药物、CART等也成为治疗的重要方式。目前血液瘤大分子药物市场的“佼佼者”包括2024年全球销售额超百亿美元的CD38单抗，以及2025年业绩增长强劲的CART疗法和双抗药物，这些创新疗法是未来市场的重要增长点。 01 急性髓系白血病：精准分层的治疗新时代急性髓系白血病（AML）是成年人中最常见的急性白血病，其特征是骨髓中未成熟髓系细胞的异常增殖和分化阻滞，导致正常造血功能衰竭。该疾病具有高度的遗传异质性，其发病机制涉及多种遗传学改变，包括染色体易位、基因突变和表观遗传学异常。这些分子异常共同导致细胞增殖、分化、凋亡等基本生命活动的失调，最终引发白血病的发生。 01 AML的临床治疗对于能耐受强化疗的患者，以“7+3”方案（蒽环类药物+阿糖胞苷）为基石。针对FLT3突变阳性，则在此基础上联合Midostaurin已成为标准方案。对于不适合强化疗的患者，Venetoclax联合Azacitidine或Decitabine是广泛使用的新标准。在一线治疗无效或复发后，二线治疗的核心是靶向药物，例如针对FLT3突变可选用Gilteritinib，针对IDH2突变可使用Enasidenib。在免疫治疗领域，目前已上市的药物相对较少。Gemtuzumab Ozogamicin是一款靶向CD33的抗体药物偶联物（ADC），已在海外获批用于治疗CD33阳性的AML，并且可通过临床急需药品临时进口通道在中国申请使用。 ▲ 图2 急性髓系白血病靶向治疗药物及其作用机制的示意图 02 AML在研靶点和新兴治疗在AML靶点治疗领域，成熟靶点已展现扎实临床价值，新兴靶点与疗法则持续拓宽治疗边界。 ▷1）成熟靶点中，FLT3是核心之一，Gilteritinib已成为FLT3突变复发/难治AML的标准选择，新型FLT3/CHK1双靶点抑制剂TLX-83更瞄准耐药难题，为患者带来新希望； ▷2）CD33凭借ADC药物Gemtuzumab Ozogamicin在临床站稳脚跟； ▷3）针对NPM1突变或KMT2A重排的Menin抑制剂（如Ziftomenib）获FDA突破性疗法认定，在复发/难治患者中实现深度缓解，彰显精准治疗潜力。从全球靶点研究热度看，CD33以103项研究位居榜首，FLT3也有26项研究布局，足见其在研发领域的核心地位，这些成熟靶点的持续深耕与创新药物迭代，为AML患者构建了坚实的治疗防线。与此同时，新兴治疗手段正以多维度创新重塑AML治疗格局。 ▷1）细胞免疫领域，双靶点CAR-NK疗法通过同时靶向CD33和MSLN，结合基因编辑技术规避“自相残杀”，大幅提升疗效持久性，为细胞治疗开辟新路径； ▷2）双特异性抗体领域，针对CD33、CD123、FLT3及CLEC12A等靶点的T细胞衔接器正处于不同研究阶段，有望通过多靶点协同增强抗肿瘤活性。此外，从靶点研究分布可见，CD47、CD70等靶点也在快速推进，这些新兴方向不仅丰富了AML治疗的“武器库”，更有望突破现有治疗瓶颈，为患者带来更优的生存获益，凸显AML靶点治疗领域蓬勃的创新活力与广阔前景。 ▲ 图3 急性髓系白血病在研生物药物TOP20靶点进展情况（抗体类、偶联药物、细胞治疗） 02 多发性骨髓瘤：免疫治疗领跑下的突破多发性骨髓瘤（MM）是仅次于非霍奇金淋巴瘤的第二大常见血液系统恶性肿瘤，其特征是骨髓中浆细胞的恶性增殖，并伴有单克隆免疫球蛋白（M蛋白）的过度产生。该疾病具有高度异质性，可根据M蛋白类型分为IgG型、IgA型、IgD型等多种亚型。 01 多发性骨髓瘤的临床治疗在多发性骨髓瘤 (multiple myeloma, MM) 一线治疗中，适合自体干细胞移植 (ASCT) 的患者采用D-VRd方案；不适合移植的患者选用D-VMP方案或Isa-VRd方案 (Isatuximab+VRd) 。二线治疗针对CD38单抗耐药者，可选择BCMA靶向的CAR-T疗法 (如ide-cel、cilta-cel) 、ADC药物Belantamab mafodotin联合方案，或Pomalidomide为基础的方案 (如Lenalidomide耐药者可联用Elotuzumab、Dexamethasone) 。三线治疗聚焦“三重难治”患者，双特异性抗体 (如BCMA×CD3的Bictegravir、靶向GPRC5D的双抗) 、CAR-T疗法 (ide-cel、cilta-cel) 及核输出抑制剂Selinexor等创新疗法成为关键选择。 ▲ 图4 多发性骨髓瘤靶向治疗药物及其作用机制的示意图 02 MM在研靶点和新兴治疗在多发性骨髓瘤（MM）靶点治疗领域，BCMA以296项研究位居全球靶点热度榜首，已成为成熟且极具潜力的核心靶点。 ▷1）其中，BCMA/CD3双特异性抗体Bictegravir已获批用于三线治疗，其ADC药物Blenrep也在欧盟获批联合方案； ▷2）CD38靶点有93项研究布局，Daratumumab和Elotuzumab为患者带来显著获益，尤其是为CD38单抗难治患者提供了新选择； ▷3）GPRC5D靶点研究热度紧随其后，其特异性双抗Talquetamab展现出超70%的客观缓解率，成为靶向治疗新亮点； ▷4）最新突破的FcRH5靶点，其双特异性抗体Cevostamab在I期临床中斩获88%的客观缓解率，因在几乎全部骨髓瘤细胞上表达，有望克服BCMA耐药，已计划直接进入III期临床试验。这些靶点的蓬勃研究与药物获批，为 MM 患者构建了从一线到后线的精准治疗矩阵。与此同时，新兴治疗手段正通过多靶点创新与联合策略持续突破治疗瓶颈。 ▷1）例如，强生三特异性抗体JNJ-79635322可同时靶向BCMA、GPRC5D和CD3，I期临床推荐剂量组客观缓解率高达86%； ▷2）国内药企也在加速布局，武汉友芝友生物的双特异性抗体Y150（靶向CD38和CD3）已完成I期临床试验。这些多靶点抗体药物的研发，不仅丰富了MM治疗的“武器库”，更通过协同作用提升疗效、克服耐药，彰显出MM靶点治疗领域蓬勃的创新活力与广阔应用前景，为患者带来更多生存希望。 ▲ 图5 多发性骨髓瘤在研生物药物TOP20靶点进展情况（抗体类、偶联药物、细胞治疗） 03 淋巴瘤：异质性疾病的个体化治疗淋巴瘤作为一组高度异质性的血液系统恶性肿瘤，其治疗策略高度依赖精准的病理分型和分子特征。当前国际权威诊疗指南（包括NCCN、ESMO和CSCO指南）均强调基于疾病亚型、分期及分子标志物的个体化治疗。 01 淋巴瘤的临床治疗在淋巴瘤治疗中，一线治疗以R-CHOP免疫化疗方案为基石，针对存在MyD88 L265p或CD79B突变的弥漫大B细胞淋巴瘤患者，探索联合BTK抑制剂Zanubrutinib以提升疗效。二线治疗以靶向药物为核心，BTK抑制剂应用广泛，还探索“外泌体负载si-BTK与异欧前胡素 (ISOIM)” 的新型联合递送系统，针对原发性渗出性淋巴瘤等罕见类型，PI3K/mTOR双靶点抑制剂（如BGT226/Dactolisib）展现新潜力。三线治疗依赖创新机制药物，如新型PI3Kδ/HDAC6双靶点抑制剂（先导化合物22E）、高选择性PI3Kδ抑制剂（TYM-3-98）等。 ▲ 图6 淋巴瘤信号传导途径和靶向药物 02 淋巴瘤在研靶点和新兴治疗在淋巴瘤靶点治疗领域：CD19、CD20、CD22等经典靶点的研发进入新阶段，其中CAR-T疗法雷尼基奥仑赛注射液(恒凯莱、HR001)在复发/难治性大B细胞瘤中显示持久缓解（3个月和6个月客观缓解率（ORR）分别是53.1%和45.7%），而CD20×CD3双特异性抗体通过T细胞介导的细胞毒作用开辟免疫治疗新路径，在中国市场，罗氏的Mosunetuzumab（商品名：皓罗华®）和Glofitamab均已获批用于治疗不同类型的淋巴瘤。与此同时，新兴靶点与治疗手段正加速突破治疗瓶颈。 ▷1）CD30 CAR-T疗法（如武汉波睿达生物的BRD-01）在复发/难治性霍奇金淋巴瘤的临床试验中已显示出显著疗效与良好安全性。同时，针对CD40/CD40L通路的新药研发也在积极推进，其中激动剂抗体（如Mitazalimab）旨在激活免疫以治疗实体瘤，而拮抗剂抗体（如Frexalimab）则通过抑制该通路在自身免疫病（如多发性硬化）领域展现出潜力。此外，以EB病毒特异性抗原（如gp350）为靶点的CAR-T等新型细胞疗法，也为治疗EBV相关淋巴瘤提供了新的研究方向； ▷2）双特异性抗体与ADC药物的联合策略成效显著，III期研究证实CD20×CD3双抗Mosunetuzumab与CD79b ADC药物Polatuzumab Vedotin联用可使患者中位无进展生存期延长近3倍； ▷3）新一代“装甲CAR-T”通过分泌IL-18等细胞因子改善肿瘤微环境，显著提升治疗持久性。这些多维度的创新探索，不仅丰富了淋巴瘤治疗的“武器库”，更通过协同作用与机制革新，为患者带来更多生存希望，彰显出淋巴瘤靶点治疗领域蓬勃的创新活力与广阔应用前景。 ▲ 图7 淋巴瘤在研生物药物TOP20靶点进展情况（抗体类、偶联药物、细胞治疗） 04 总结血液肿瘤治疗将在靶点创新与技术迭代中持续进阶。FLT3/CHK1双靶点抑制剂、FcRH5等新靶点研究将破解耐药困局；多特异性抗体、“装甲CAR-T”等创新疗法通过多机制协同，进一步提升疗效与安全性。随着精准靶向与免疫治疗的深度融合，血液肿瘤治疗的治愈边界将不断拓展，为抗体生物领域创新发展注入强劲动能。 Sanyou 10th Anniversary | Revolutionizing Hematologic Oncology Treatment — Ushering in the Era of Precision Stratified Therapy The hematopoietic system in the human body operates like a precise "life factory," continuously producing various blood cells to maintain bodily functions. When the "quality control" system of this factory malfunctions, hematologic malignancies can arise—malignant diseases originating from hematopoietic cells that can affect the bone marrow, blood, and various organs and tissues throughout the body. Clinically common hematologic malignancies primarily include various types of leukemia, multiple myeloma, lymphoma, myelodysplastic syndromes, and myeloproliferative neoplasms. Their incidence and mortality rates rank high, making them significant diseases that seriously endanger human health. Among hematologic tumors, the most prevalent in terms of case numbers are leukemia, multiple myeloma, and lymphoma. Leukemia accounts for approximately 36% of hematologic tumors, multiple myeloma for about 13%, and lymphoma for about 51%. The global combined incidence rate of these three hematologic tumors exceeds 13 per 100,000 people (Figure 1). ▲ Figure 1. Overview of Hematologic Malignancies With the continuous emergence of innovative therapies, the drug market for hematologic tumors continues to expand. Among these, macromolecular drugs and CAR-T have become important treatment modalities. Current "standout" products in the hematologic tumor macromolecular drug market include CD38 monoclonal antibodies with global sales exceeding $10 billion in 2024, as well as CAR-T therapies and bispecific antibodies showing strong performance growth in 2025. These innovative therapies represent key future growth drivers for the market. 01 Acute Myeloid Leukemia: A New Era of Precision Stratified Treatment Acute Myeloid Leukemia (AML) is the most common acute leukemia in adults, characterized by the abnormal proliferation and blocked differentiation of immature myeloid cells in the bone marrow, leading to failure of normal hematopoiesis. This disease exhibits high genetic heterogeneity, and its pathogenesis involves various genetic alterations, including chromosomal translocations, gene mutations, and epigenetic abnormalities. These molecular aberrations collectively disrupt fundamental cellular processes like proliferation, differentiation, and apoptosis, ultimately leading to the development of leukemia. 01 Clinical Management of AML For patients eligible for intensive chemotherapy, the "7+3" regimen (anthracycline + cytarabine) serves as the cornerstone. For those with FLT3 mutations, the addition of Midostaurin to this backbone has become the standard of care. For patients unsuitable for intensive chemotherapy, the combination of Venetoclax with Azacitidine or Decitabine is a widely used new standard. After first-line treatment failure or upon relapse, targeted agents form the core of second-line therapy. For instance, Gilteritinib can be chosen for FLT3 mutations, and Enasidenib can be used for IDH2 mutations. In the realm of immunotherapy, currently approved drugs are relatively few. Gemtuzumab Ozogamicin is an antibody-drug conjugate (ADC) targeting CD33, approved overseas for treating CD33-positive AML and accessible in China through the clinical urgent drug importation pathway. ▲ Figure 2. Schematic diagram of targeted therapeutic drugs and their mechanisms of action in Acute Myeloid Leukemia 02 Investigational Targets and Emerging Therapies in AML In the field of AML targeted therapy, established targets have demonstrated solid clinical value, while emerging targets and therapies continue to expand the treatment landscape. Among the mature targets: ▷1) FLT3 is a core one, with Gilteritinib established as a standard option for relapsed/refractory FLT3-mutated AML. The novel FLT3/CHK1 dual-target inhibitor TLX-83 specifically addresses the challenge of resistance, offering new hope for patients. ▷2) CD33 has secured its place in the clinic with the ADC drug Gemtuzumab ozogamicin. ▷3) Menin inhibitors (e.g., Ziftomenib) targeting NPM1 mutations or KMT2A rearrangements have received FDA Breakthrough Therapy designation, achieving deep responses in relapsed/refractory patients and highlighting the potential of precision medicine. From a global perspective of target research popularity, CD33 leads with 103 active studies, and FLT3 also has 26 research programs, underscoring their central role in the R&D landscape. The continued in-depth research and iterative innovation of drugs against these established targets are building a solid line of defense for AML patients. Concurrently, emerging therapeutic approaches are reshaping the AML treatment paradigm through multi-dimensional innovation. ▷1) In the cellular immunotherapy domain, dual-target CAR-NK therapies targeting both CD33 and MSLN, combined with gene-editing technology to avoid "fratricide," significantly enhance the durability of efficacy, opening new pathways for cell therapy. ▷2) In the bispecific antibody field, T-cell engagers targeting antigens like CD33, CD123, FLT3, and CLEC12A are in various stages of investigation, promising enhanced anti-tumor activity through multi-target synergy. Furthermore, the distribution of target research indicates that other targets like CD47 and CD70 are also advancing rapidly. These emerging directions not only enrich the AML treatment "arsenal" but also hold the potential to break through current therapeutic bottlenecks, offering patients improved survival benefits. This highlights the vibrant innovative activity and broad prospects within the AML targeted therapy field. ▲ Figure 3. Progress of TOP 20 Investigational Biological Drug Targets in Acute Myeloid Leukemia (Antibody-based, Conjugated Drugs, Cell Therapy) 02 Multiple Myeloma: Breakthroughs Led by Immunotherapy Multiple Myeloma (MM) is the second most common hematologic malignancy after non-Hodgkin lymphoma, characterized by the malignant proliferation of plasma cells in the bone marrow, accompanied by the overproduction of a monoclonal immunoglobulin (M protein). The disease is highly heterogeneous and can be classified into various subtypes such as IgG, IgA, and IgD types based on the M protein isotype. 01 Clinical Management of Multiple Myeloma In the first-line treatment of Multiple Myeloma (MM), patients eligible for autologous stem cell transplantation (ASCT) receive the D-VRd regimen (Daratumumab + Bortezomib + Lenalidomide + Dexamethasone). For transplant-ineligible patients, the D-VMP regimen (Daratumumab + Bortezomib + Melphalan + Prednisone) or the Isa-VRd regimen (Isatuximab + VRd) are chosen. Second-line therapy for patients refractory to CD38 monoclonal antibodies may include BCMA-targeted CAR-T therapies (e.g., Ide-cel, Cilta-cel), the ADC drug Belantamab mafodotin in combination regimens, or Pomalidomide-based regimens (e.g., combined with Elotuzumab and Dexamethasone for those refractory to Lenalidomide). Third-line therapy focuses on "triple-class refractory" patients, where innovative therapies such as bispecific antibodies (e.g., the BCMAxCD3 bispecific Bictegravir, bispecifics targeting GPRC5D), CAR-T therapies (Ide-cel, Cilta-cel), and the nuclear export inhibitor Selinexor become crucial options. ▲ Figure 4. Schematic diagram of targeted therapeutic drugs and their mechanisms of action in Multiple Myeloma 02 Investigational Targets and Emerging Therapies in MM In the field of Multiple Myeloma (MM) targeted therapy, BCMA ranks first globally in research popularity with 296 studies, establishing itself as a mature and highly promising core target. ▷1) Among these, the BCMA/CD3 bispecific antibody Bictegravir has been approved for third-line use, and its ADC counterpart Blenrep is also approved in the EU in combination regimens. ▷2) The CD38 target has 93 active studies; Daratumumab and Elotuzumab have provided significant patient benefit, particularly offering new options for patients refractory to CD38 monoclonal antibodies. ▷3) Research on the GPRC5D target follows closely in popularity, with its specific bispecific antibody Talquetamab demonstrating an objective response rate exceeding 70%, emerging as a new highlight in targeted therapy. ▷4) The recently targeted FcRH5 antigen, with its bispecific antibody Cevostamab achieving an 88% objective response rate in Phase I trials, shows potential to overcome BCMA resistance due to its expression on nearly all myeloma cells. Plans are underway to proceed directly to Phase III clinical trials. The vigorous research and drug approvals surrounding these targets are constructing a precision treatment matrix for MM patients from first to later lines of therapy. Simultaneously, emerging therapeutic strategies are continuously pushing the boundaries of treatment through multi-target innovation and combination approaches. ▷1) For example, Johnson & Johnson's trispecific antibody JNJ-79635322, targeting BCMA, GPRC5D, and CD3 simultaneously, achieved a high 86% objective response rate at the recommended Phase II dose in Phase I studies. ▷2) Domestic pharmaceutical companies are also accelerating their pipelines; for instance, Wuhan YZY Biopharma's bispecific antibody Y150 (targeting CD38 and CD3) has completed Phase I clinical trials. The development of these multi-target antibody drugs not only enriches the MM treatment "arsenal" but also enhances efficacy and overcomes resistance through synergistic effects, demonstrating the dynamic innovation and broad application prospects in the MM targeted therapy landscape, bringing more hope for survival to patients. ▲ Figure 5. Progress of TOP 20 Investigational Biological Drug Targets in Multiple Myeloma (Antibody-based, Conjugated Drugs, Cell Therapy) 03 Lymphoma: Personalized Therapy for a Heterogeneous Disease Lymphoma represents a group of highly heterogeneous hematologic malignancies whose treatment strategies heavily rely on precise pathological classification and molecular profiling. Current authoritative international treatment guidelines (including NCCN, ESMO, and CSCO) all emphasize individualized treatment based on disease subtype, stage, and molecular markers. 01 Clinical Management of Lymphoma In lymphoma treatment, first-line therapy is cornerstoneed by the R-CHOP immunochemotherapy regimen. For patients with diffuse large B-cell lymphoma harboring MyD88 L265p or CD79B mutations, exploration of combining the BTK inhibitor Zanubrutinib is underway to improve efficacy. Second-line therapy centers on targeted agents. BTK inhibitors are widely used, and novel approaches like a "novel delivery system loading si-BTK and Isoimperatorin (ISOIM)" are being explored. For rare types like primary effusion lymphoma, PI3K/mTOR dual-target inhibitors (e.g., BGT226/Dactolisib) show new potential. Third-line treatment relies on drugs with innovative mechanisms, such as novel PI3Kδ/HDAC6 dual-target inhibitors (lead compound 22E) and highly selective PI3Kδ inhibitors (TYM-3-98). ▲ Figure 6. Lymphoma Signal Transduction Pathways and Targeted Drugs 02 Investigational Targets and Emerging Therapies in Lymphoma In the field of lymphoma targeted therapy, research on classic targets like CD19, CD20, and CD22 has entered a new stage. The CAR-T therapy Reni Orelen Syringe Solution (恒凯莱®, HR001) has demonstrated durable responses in relapsed/refractory large B-cell lymphoma (with 3-month and 6-month objective response rates (ORR) of 53.1% and 45.7%, respectively). Meanwhile, CD20×CD3 bispecific antibodies are pioneering new paths in immunotherapy by mediating T cell-dependent cytotoxicity. In the Chinese market, Roche's Mosunetuzumab (trade name: 皓罗华®) and Glofitamab have been approved for treating different types of lymphoma. Concurrently, emerging targets and therapeutic modalities are rapidly advancing to overcome treatment bottlenecks. ▷1) CD30 CAR-T therapy (e.g., Wuhan Borui Biotechnology's BRD-01) has shown significant efficacy and a favorable safety profile in clinical trials for relapsed/refractory Hodgkin Lymphoma. Development of drugs targeting the CD40/CD40L pathway is also progressing actively. Agonist antibodies (e.g., Mitazalimab) aim to activate the immune system against solid tumors, while antagonist antibodies (e.g., Frexalimab) show potential in autoimmune diseases like multiple sclerosis by inhibiting this pathway. Furthermore, novel cell therapies targeting Epstein-Barr virus (EBV) specific antigens (e.g., gp350) provide new research directions for treating EBV-associated lymphomas. ▷2) Combination strategies employing bispecific antibodies and ADCs have shown marked success. A Phase III study confirmed that combining the CD20×CD3 bispecific antibody Mosunetuzumab with the CD79b ADC drug Polatuzumab Vedotin nearly tripled the median progression-free survival of patients. ▷3) Next-generation "Armored CAR-T" cells, designed to secrete cytokines like IL-18 to modify the tumor microenvironment, significantly enhance the durability of responses. These multi-dimensional innovative explorations not only enrich the lymphoma treatment "arsenal" but also, through synergistic effects and mechanistic innovations, offer patients greater hope for survival, highlighting the vigorous innovative activity and broad application prospects in the lymphoma targeted therapy field. ▲ Figure 7. Progress of TOP 20 Investigational Biological Drug Targets in Lymphoma (Antibody-based, Conjugated Drugs, Cell Therapy) 04 Summary The treatment of hematologic malignancies will continue to advance through target innovation and technological iteration. Novel targets like FLT3/CHK1 dual inhibitors and FcRH5 aim to 破解 overcome resistance challenges. Innovative therapies such as multispecific antibodies and "Armored CAR-T" further enhance efficacy and safety through multi-mechanistic synergy. With the deepening integration of precision targeting and immunotherapy, the boundaries of curability for hematologic tumors are continually expanding, injecting powerful momentum into the innovative development of the antibody and biologics field. ▶ References: (Sant M, Allemani C, Tereanu C, et al. Incidence of hematologic malignancies in Europe by morphologic subtype: results of the HAEMACARE project[J]. Blood, The Journal of the American Society of Hematology, 2010, 116(19): 37243734.) (YE Xiangjun, LU Xingguo. Interpretation of the 5th edition of the WHO classification of haematolymphoid tumours on MDS and AML[J]. Journal of Diagnostics Concepts & Practice, 2023, 22(05): 421428.) (5th edition of the WHO Classification of Haematolymphoid Tumours.) (Zhonghua Xue Ye Xue Za Zhi [Chinese Journal of Hematology], 2023,44(9) : 705712.) (Haubner S, Subklewe M, Sadelain M. Honing CAR T cells to tackle acute myeloid leukemia[J]. Blood, 2025, 145(11): 11131125) (Song F, Lin S, Xu T, et al. Targeted therapy in acute myeloid leukemia: resistance and overcoming strategy[J]. Drug Resistance Updates, 2025: 101286) (Lentzsch S, Ehrlich L, Roodman G. Pathophysiology of multiple myeloma bone disease. Hematol Oncol Clin North Am. 2007;21(6):10351049, viii. DOI: 1016/j.hoc.2007.08.009.) (Pinto V et al. Multiple Myeloma: Available Therapies and Causes of Drug Resistance. Cancers. 2020; 12(2):407.) (Chinese Medical Doctor Association Hematology Branch, Chinese Society of Hematology. Chinese Guidelines for the Diagnosis and Management of Multiple Myeloma (2024 Revision) [J]. Zhonghua Nei Ke Za Zhi, 2024, 63(12): 11861195. DOI: 10.3760/cma.j.cn1121382024092800616.) (Nishida, H. Rapid Progress in Immunotherapies for Multiple Myeloma: An Updated Comprehensive Review. Cancers 2021, 13, 2712.) (Lewis W D, Lilly S, Jones K L. Lymphoma: diagnosis and treatment[J]. American family physician, 2020, 101(1): 3441.) (Shankland K R, Armitage J O, Hancock B W. Nonhodgkin lymphoma[J]. The Lancet, 2012, 380(9844): 848857.) (Wang L, Qin W, Huo Y J, et al. Advances in targeted therapy for malignant lymphoma[J]. Signal transduction and targeted therapy, 2020, 5(1): 15.) (Yung L, Linch D. Hodgkin's lymphoma[J]. The lancet, 2003, 361(9361): 943951.) (Connors J M, Cozen W, Steidl C, et al. Hodgkin lymphoma[J]. Nature Reviews Disease Primers, 2020, 6(1): 61.) (Hennessy B T, Hanrahan E O, Daly P A. NonHodgkin lymphoma: an update[J]. The lancet oncology, 2004, 5(6): 341353.) (DXY Database) 关于三优生物三优生物是一家以“让天下没有难做的创新生物药”为使命，以超万亿分子库和智能科技驱动的生物医药高科技企业。公司致力于打造全球顶尖的原创新药创新工场。公司以智能超万亿分子库（AI-STAL）为核心；以干湿结合、国际领先的创新生物药智能化及一体化研发平台为依托；以多样化的业务模式推动全球创新药物的研发及产业化。公司总部位于中国上海，在亚洲、北美洲、欧洲等多地建立了业务中心，形成了全球化的业务网络，现有投产及布局的研发及GMP场地20000多平方米。公司已与全球2000多家药企、生技公司等建立了良好的合作关系，已赋能1200多个新药研发项目；已完成50多个合作研发项目，其中10多个协同研发项目已推至IND及临床研发阶段。公司已申请130多项发明专利，其中30多项发明专利已获得授权，并获得了国家级高新技术企业、上海市专精特新、ISO9001、ISO27001等10余项资质及体系认证。 1 推荐阅读三优十周年｜抗体药免疫原性全方位解析三优十周年｜肿瘤免疫2025及2018双诺奖解析三优十周年｜TCE药物-重塑免疫治疗版图的新力量三优十周年｜神经系统疾病治疗进展三优十周年｜AI-STAL 智能超万亿分子库发展历程三优十周年｜打造全球顶尖的原创新药创新工场三优十周年｜抗体治疗50年风雨江湖三优十周年｜针对宠物疾病的单克隆抗体药物三优十周年｜CAR-T细胞治疗现状挑战与未来三优十周年｜服务篇-体外药效筛选解决方案三优十周年｜AI-STAL篇-Anticalin靶向蛋白库三优十周年｜靶点篇-自身免疫疾病靶点介绍创新前沿｜三优生物类器官产学研联盟启航中和抗体“硬核出击”，斩断CHIKV病毒感染链三优十周年｜服务篇-靶点至PCC药物研发三优十周年｜靶点篇-六大恶性肿瘤靶点介绍

免疫疗法细胞疗法抗体药物偶联物

2025-11-14

·BioPharmaDive

Lundbeck sparks bidding war with Alkermes; Pfizer closes Metsera deal

Today, a brief rundown of news from Pfizer and Alkermes, as well as updates from Kura Oncology, Gilead Sciences and Zealand Pharma that you may have missed.Pfizer said Thursday it has closed its $10 billion deal to acquire GLP-1 drug developer Metsera, less than a week after the target company agreed to the final offer made by the big pharma. The Federal Trade Commission had already signed off on the acquisition first announced Sept. 22, amid a bidding war sparked by counteroffers from Wegovy manufacturer Novo Nordisk. With the acquisition complete, Pfizer can advance our shared goal of further developing Metseras five experimental metabolic drugs for obesity, Pfizer CEO Albert Bourla said in a statement. Jonathan GardnerAs the Metsera saga winds down, another bidding war is just beginning. Weeks after Avadel Pharmaceuticals agreed to sell to fellow Dublin-based drugmaker Alkermes, it has now received an unsolicited offer from Lundbeck. The Alkermes deal could hand Avadel investors up to $20 per share, while the offer from Lundbeck goes as high as $23 per share. In a statement, Avadel said terms of the Alkermes deal allow it to still talk and negotiate with Lundbeck. But, so far, Avadels board has not determined whether this new proposal is superior, nor has it changed its recommendation in support of the Alkermes acquisition. Alkermes, meanwhile, said its own board is weighing options with the help of company advisors.Avadelsells a daytime sleepiness medicine that analysts from Jefferies expect to eventually generate north of $550 million in annual sales. Jacob BellIn other Alkermes news, the company announced on Wednesday positive results from a mid-stage clinical trial that evaluated different doses of an experimental medicine in people with Type 2 narcolepsy. The trial used two key measures: a daytime sleepiness questionnaire and a test that puts patients in a dark, quiet and calm room and tasks them with staying awake. According to Alkermes, participants taking a high dose of its medicine, alixorexton, did significantly better on both tests compared to their placebo-treated counterparts. Those on a middle dose did substantially better on only the latter measure. The companys share price dipped about 7% on the news. Paul Matteis, an analyst at the investment firm Stifel, wrote in a note to clients that the results were messier than some investors had hoped to see. Even so, Matteis and his team still see alixorexton as a viable product commercially. Jacob BellThe Food and Drug Administration on Thursday approved an oral medicine Kura Oncology and Kyowa Kirin have been developing for leukemia. Called Komzifti and previously known as ziftomenib, the drug has been cleared for people with treatment-resistant acute myeloid leukemia expressing a mutation called NPM1 and who don't have other good treatment options. The clearance makes Komzifti the second so-called menin inhibitor recently approved for these malignancies, following Syndax Pharmaceuticals' Revuforj. But Komzifti doesn't have a safety warning cautioning of the risk of an abnormal heart rhythm, a “major winning factor“ that may separate it from Syndax's drug, wrote Jefferies analyst Roger Song. Kura and Kyowa Kirin share rights to Komzifti through a 2024 deal. Ben FidlerGilead Sciences said a single-tablet combination of two of its HIV drugs succeeded in the first of two Phase 3 trials. Study recruits were taking between two and 11 pills per day at the start of the trial, Artistry-1, before they were randomized to either continue on or switch to a one-tablet form of Gileads bictegravir and lenacapavir. Gilead said Thursday that its drug proved non-inferior to the multitablet regimens in those enrollees, all of whom are adults whose HIV-1 had been suppressed to very low levels. Gilead has a second Phase 3 study underway, with results expected by the end of the year. Both trials would form the basis of a future regulatory filing. Delilah AlvaradoZealand Pharma said in an earnings report Thursday that it will stop developing an experimental weight-loss drug called dapiglutide. The company initially planned to advance the drug, which targets the gut hormones GLP-1 and GLP-2, into Phase 2b testing following positive Phase 1 results in June. But Zealand now intends to focus on programs with the greatest potential for clinical differentiation and long-term value creation, it said. Two of those prospects, petrelintide and survodutide, are either in or nearing late-stage testing. Delilah Alvarado '

上市批准并购临床3期临床2期临床结果

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 Ziftomenib 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	Kura Oncology, Inc.	2025-11-13

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适应症	最高研发状态	国家/地区	公司	日期
急性淋巴细胞白血病	临床2期	美国	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	比利时	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	加拿大	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	法国	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	德国	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	意大利	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	波兰	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	西班牙	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	英国	Kura Oncology, Inc.	2019-09-12
混合表型急性白血病	临床2期	美国	Kura Oncology, Inc.	2019-09-12

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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


NCT04067336 (KOMET-001, Pubmed \| J Clin Oncol)	临床1/2期	NPM1突变急性髓系白血病 NPM1-mutated	92	Ziftomenib 600 mg	積廠遞蓋膚衊築膚選鏇(築夢簾衊醖選餘齋鏇夢) = 積範膚廠遞繭願齋夢襯衊餘艱衊鏇憲繭繭夢鬱 (顧鬱網夢構網積餘襯夢, 14 ~ 32) 更多	积极	2025-11-01
NCT04067336 (KOMET-001, ASCO2025) 人工标引	临床1/2期	NPM1突变急性髓系白血病 NPM1 Mutation	112	Ziftomenib 600 mg QDZiftomenib 600 mg QD (Phase 1b/2)	糧願構鏇遞餘糧鑰膚艱(範膚鹽齋齋艱淵醖膚觸) = 範壓鹹衊積鏇鹽鹽範構夢積餘壓觸齋憲齋衊膚 (淵築觸鹽齋糧網膚憲製, 17 ~ 34) 更多	积极	2025-05-30
				Ziftomenib 600 mg QDZiftomenib 600 mg QD (Phase 2)	糧願構鏇遞餘糧鑰膚艱(範膚鹽齋齋艱淵醖膚觸) = 積膚窪憲繭鏇夢艱窪鹹夢積餘壓觸齋憲齋衊膚 (淵築觸鹽齋糧網膚憲製, 15 ~ 33)
NCT04067336 (KOMET-001, EHA2025)	临床1/2期	NPM1突变急性髓系白血病 NPM1-mutant	112	Ziftomenib 600 mg QD	簾襯餘窪糧顧顧築糧鹽(網網壓鬱蓋鏇壓蓋鹹構) = 築衊廠鏇範鬱醖糧餘衊構範構積夢積製觸淵鏇 (顧淵網獵餘襯網範淵廠, 17 ~ 34) 更多	积极	2025-05-22
NCT05735184 (KOMET-007, EHA2025)	临床1期	伴有 11q23 异常的急性髓系白血病 \| NPM1突变急性髓系白血病 NPM1-m \| KMT2A-r	51	Ziftomenib 600 mg QD + 7+3 induction	構膚選餘窪襯襯遞齋醖(鏇簾簾鬱憲構艱構鑰廠) = febrile neutropenia (47%), decreased platelet count (31%), anemia (22%), decreased neutrophil count (20%), decreased white blood cell count (16%) 顧齋糧憲網選糧鹽簾構 (鏇顧網遞鬱糧糧衊簾醖 ) 更多	积极	2025-05-14
NCT04067336 (KOMET-001, Company_Website) 人工标引	临床1/2期	急性髓性白血病 NPM1 Mutation	-	Ziftomenib	壓淵鹹夢廠膚蓋觸醖構(鹹製憲醖壓憲選壓壓憲) = The KOMET-001 trial achieved its primary endpoint of CR plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. 襯顧鑰艱壓夢夢積簾觸 (衊窪築觸齋網醖壓膚製 ) 达到更多	积极	2025-02-07
KOMET-007 (GlobeNewswire) 人工标引	临床1期	NPM1突变急性髓系白血病 \| KMT2A易位的急性白血病一线 NPM1 Mutation \| KMT2A Rearrangement	54	ziftomenib +venetoclax/azacitidine	選壓蓋鏇範窪積繭顧糧(製鏇糧簾選壓窪觸廠窪) = 範繭蓋繭廠積選憲顧淵糧獵糧憲範淵顧選鹹積 (鏇簾鏇繭顧廠齋繭網襯 ) 更多	积极	2024-12-09
				Ziftomenib+cytarabinecytarabine/daunorubicindaunorubicin	選壓蓋鏇範窪積繭顧糧(製鏇糧簾選壓窪觸廠窪) = 襯鏇獵糧鬱範壓壓鬱餘糧獵糧憲範淵顧選鹹積 (鏇簾鏇繭顧廠齋繭網襯 ) 更多
ASH2024	N/A	-	-	Ziftomenib 200 mg	憲選繭廠糧顧繭壓網窪(夢繭膚壓衊積網網選製) = 範積製蓋鏇簾餘製糧願醖醖築壓蓋鹹蓋壓醖願 (繭選顧製憲壓窪觸襯艱 ) 更多	-	2024-12-08
				Ziftomenib 400 mg	憲選繭廠糧顧繭壓網窪(夢繭膚壓衊積網網選製) = 鏇襯鏇齋鹽糧顧鹽鏇範醖醖築壓蓋鹹蓋壓醖願 (繭選顧製憲壓窪觸襯艱 ) 更多
ASH2024	N/A	-	-	Ziftomenib 200 mg	壓鹽築膚鹽窪範壓鏇願(繭網簾衊鏇壓鬱夢憲製) = 繭壓鏇鏇製遞夢廠襯糧鬱鑰襯衊選壓積鹽選繭 (鬱窪範製糧鹽窪選餘願 )	-	2024-12-07
				Ziftomenib 400 mg	壓鹽築膚鹽窪範壓鏇願(繭網簾衊鏇壓鬱夢憲製) = 鹽淵願襯壓窪憲齋衊網鬱鑰襯衊選壓積鹽選繭 (鬱窪範製糧鹽窪選餘願 )
NCT04067336 (KOMET-001, Pubmed \| Lancet Oncol) 人工标引	临床1/2期	急性髓性白血病 NPM1 Mutation \| KMT2A Rearrangement	83	Ziftomenib 50-1000 mg	繭構淵衊範淵選鏇淵襯(淵醖積範廠衊蓋艱壓繭) = the most common grade 3 or worse treatment-emergent adverse events were anaemia (20 [24%]), febrile neutropenia (18 [22%]), pneumonia (16 [19%]), differentiation syndrome (12 [15%]), thrombocytopenia (11 [13%]), and sepsis (ten [12%]). 願鹹淵願鑰製膚夢襯構 (廠窪築艱積積蓋艱廠構 ) 更多	积极	2024-10-01
NCT05735184 (KOMET-007, Corporate Publications) 人工标引	临床1期	伴有 11q23 异常的急性髓系白血病 \| NPM1突变急性髓系白血病一线 KMT2A Rearrangement \| NPM1 Mutation	20	ziftomenib+SOC	鑰糧製衊鬱選窪願鏇繭(糧觸鹹構廠壓觸蓋衊簾) = No differentiation syndrome events of any grade were reported, and no dose-limiting toxicities, evidence of QTc prolongation, drug-drug interactions or additive myelosuppression were observed. 簾襯願顧獵鹽膚廠壓繭 (鹹繭鹹襯夢製繭獵製製 )	积极	2024-01-30
				ziftomenib+SOC (ziftomenib and 7+3)