A Phase 1/2, Open-Label, Dose-Escalation, Dose-Expansion Study of DSP-5336 in Adult Patients with Acute Leukemia and Other Selected Hematologic Malignancies, with and Without Mixed Lineage Leukemia (MLL) Rearrangement or Nucleophosmin 1 (NPM1) Mutation

A Phase 1/2 dose escalation / dose expansion study of DSP 5336 in patients with relapsed or refractory AML.

开始日期2022-02-28

申办/合作机构

Sumitomo Pharma America, Inc.

100 项与 Enzomenib 相关的临床结果

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100 项与 Enzomenib 相关的转化医学

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100 项与 Enzomenib 相关的专利（医药）

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项与 Enzomenib 相关的文献（医药）

2023-09-01·Journal of cancer research and clinical oncology

Targeting the undruggable: menin inhibitors ante portas

Review

作者: Desole, Maximilian ; Sica, Simona ; Dempke, Wolfram C M ; Chiusolo, Patrizia ; Schmidt-Hieber, Martin

Acute myeloid leukaemias harbouring a rearrangement of the mixed lineage leukaemia gene (MLL) are aggressive haematopoietic malignancies that relapse early and have a poor prognosis (event-free survival less than 50%). Menin is a tumour suppressor, however, in MLL-rearranged leukaemias it functions as a co-factor which is mandatory for the leukaemic transformation by interaction with the N-terminal part of MLL, which is maintained in all MLL-fusion proteins. Inhibition of menin blocks leukaemogenesis and leads to differentiation and, in turn, to apoptosis of leukaemic blasts. Furthermore, nucleophosmin 1 (NPM1) binds to specific chromatin targets, which are co-occupied by MLL, and menin inhibition has been shown to trigger degradation of mNPM1 resulting in a rapid decrease in gene expression and activating histone modifications. Therefore, disruption of the menin-MLL axis blocks leukaemias driven by NPM1 mutations for which the expression of menin-MLL target genes (e.g., MEIS1, HOX etc.) is essential. To date at least six different menin-MLL inhibitors are undergoing clinical evaluation as first- and second-line monotherapy in acute leukaemias: DS-1594, BMF-219, JNJ-75276617, DSP-5336, revumenib, and ziftomenib, however, only for revumenib and ziftomenib early clinical data have been reported. In the revumenib phase I/II AUGMENT-101 trial (N = 68) with very heavily pretreated AML patients the ORR was 53% with a CR rate of 20%. The ORR in patients harbouring MLL rearrangement of mNPM1 was 59%. Patients who achieved a response had a mOS of 7 months. Similar results have been reported for ziftomenib in the phase I/II COMET-001 trial. ORR was 40% and CRc was 35% in AML patients with mNPM1. However, outcome was worse in AML patients with a MLL rearrangement (ORR 16.7%, CRc 11%). Differentiation syndrome was a notable adverse event. The clinical development of novel menin-MLL inhibitors is well in line with the currently ongoing paradigm shift towards targeted therapies seen in the AML treatment landscape. Moreover, the clinical assessment of combinations of these inhibitors with established therapy options in AML could be the fuel for an improved outcome of MLL/NPM1 patients.

2022-10-01·Clinical Lymphoma Myeloma & Leukemia

AML-391 Phase 1/2, Open-Label, Dose Escalation, Dose Expansion Study of Menin Inhibitor DSP-5336 in Adult Patients With Acute Leukemia With and Without Mixed-Lineage Leukemia (MLL)– Rearrangement or Nucleophosmin 1 (NPM1) Mutation

Article

作者: Daver, Naval ; Stoudemire, Jay ; Dobrowolska, Hanna ; Hitron, Matthew ; Affinito, John ; Eguchi, Ken ; Watanabe, Akinobu ; Cai, Hongliang

Patients who relapse after initial acute leukemia (AL) treatment have median survival <6 months for AML and <1 y for ALL, especially AML or ALL with MLL rearrangements (MLLr) or relapsed AML with NPM1 mutations. Translocations of the MLL gene (5% to 10% of adult patients) produce an aggressive subtype, with a median overall survival (OS) of approximately 9 months. The NPM1 mutation (NPM1m) occurs in 30%-35% of adult de novo AML and is associated with a favorable prognosis. However, 40%-50% of these patients have concurrent FLT3-internal tandem duplication mutations negating this favorability. DSP-5336, a novel oral small-molecule, was developed to prevent menin protein binding to MLL fusion proteins that drive leukemogenesis. Nonclinical DSP-5336 studies showed selective growth inhibition against human AL cell lines with MLLr or NPM1m. Evidence of antitumor activity and survival advantage was demonstrated in 3 AML xenograft models with MLLr and/or NPM1m. Treatment with DSP-5336 may reduce leukemic cells and benefit patients with relapsed or refractory (R/R) MLLr or NPM1m AML or patients with R/R MLLr ALL. An open-label, single-arm, phase 1/2 study (NCT04988555) will evaluate the safety and efficacy of DSP-5336 and determine the recommended phase 2 dose (RP2D) in adults aged ≥18 y with relapsed/refractory AML or ALL after at least one line of therapy. Key inclusion criteria are ECOG performance status 0-2 and adequate organ function. Patients will be dosed twice daily in 28-day cycles. In phase 1, 21-30 patients will be enrolled into multiple dose cohorts (1-6 patients per dose level), with dose escalation determined using a two-parameter Bayesian logistic regression model. The RP2D will be informed by the maximum biologic effect or maximum tolerated dose, whichever is lower. The phase 2 study will include two arms (R/R AML with MLLr and R/R AML with NPM1m). Patients will be treated at the RP2D to evaluate clinical activity and safety and monitored using Bayesian posterior probability to optimize enrollment with Bayesian stopping rules. Bayesian monitoring of responses begins after the first 10 enrolled patients are evaluable for efficacy. This study is recruiting in the United States and Japan.

项与 Enzomenib 相关的新闻（医药）

2024-12-09

·PRNewswire

Sumitomo Pharma America Presents New Data on Nuvisertib and Enzomenib at the 2024 American Society of Hematology Annual Meeting

Latest results in Phase 1/2 studies show promise for two investigational treatments for patients with relapsed/refractory blood and bone marrow cancers Nuvisertib (TP-3654), an investigational oral small molecule highly selective PIM1 kinase inhibitor, is being evaluated in a Phase 1/2 study in patients with relapsed/refractory myelofibrosis Enzomenib (DSP-5336), an investigational, oral small molecule designed to inhibit the menin and KMT2A protein interaction, is being evaluated in a Phase 1/2 study in patients with relapsed/refractory acute leukemia MARLBOROUGH, Mass., Dec. 9, 2024 /PRNewswire/ -- Sumitomo Pharma America, Inc. (SMPA) today presented new clinical data supporting further development of nuvisertib, an investigational small molecule being researched for the treatment of relapsed/refractory myelofibrosis (MF), and enzomenib, an investigational oral small molecule being researched for relapsed/refractory acute leukemia, at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition. Preliminary data presented from the ongoing Phase 1/2 study of nuvisertib monotherapy (N=74) in patients with relapsed/refractory MF show nuvisertib was well tolerated with no dose-limiting toxicities (DLTs). Preliminary data in evaluable patients showed clinical activity including spleen volume reduction (SVR25 of 22.2%), symptom reduction (TSS50 of 44.4%), and bone marrow fibrosis (47.8% patients) and hemoglobin (25% patients) and platelet count (27.6% patients) improvement. Additional data presented show nuvisertib treatment led to significant cytokine modulation (e.g. EN-RAGE, IL-18, MIP-1β, and adiponectin) over time correlating with spleen and symptom responses. The global study has been expanded to evaluate nuvisertib add-on to ruxolitinib, the first approved Janus-associated kinase (JAK) inhibitor, and in combination with momelotinib, a recently approved JAK inhibitor for MF patients with anemia, to assess safety and potential clinical activity. New preliminary clinical and translational data from the enzomenib Phase 1/2 study were also presented at the conference by Dr. Joshua Zeidner from the University of North Carolina. The safety population included 84 total patients with acute leukemia, most of whom (94%, 79/84) had acute myeloid leukemia (AML). The study population was diverse, with 47.6% non-white (40/84), and also heavily pre-treated, with a median of 3 prior regimens. In these patients, enzomenib was administered twice daily in continuous 28-day study cycles at dose levels from 40 mg BID up to 300 mg BID. Enzomenib was well tolerated with low overall rates of drug-related adverse events and no dose limiting toxicity (DLT) reported. Differentiation syndrome was reported in 10.7% of patients but did not result in patient deaths nor did it require permanent discontinuations of enzomenib. Also presented were clinical activity results from the dose optimization cohorts at the dose levels of 200 mg BID and 300 mg BID. The data included results from all patients with KMT2A rearrangement (KMT2Ar) or NPM1 mutation (NPM1m) who had received at least one dose of enzomenib and who had not received a menin inhibitor previously. In 23 patients with KMT2Ar, the Objective Response Rate (ORR) using ELN 2017 criteria was 65.2% (15/23) and the proportion to achieve CR+CRh was 30.4% (7/23). In the subset of patients with KMT2Ar to receive 300 mg BID (n = 15), the ORR was 73.3% (11/15) and CR+CR was 40% (6/15). In the 17 total patients with NPM1 mutation (NPM1m) who received 200 mg BID or 300 mg BID, the ORR was 58.8% (10/17) and the proportion to achieve CR+CRh was 47.1% (8/17). The 400 mg BID dose optimization cohort is ongoing, with 21 patients enrolled as of the clinical cut-off and data planned for presentation at a future conference. These encouraging clinical activity results combined with an excellent safety profile suggest that enzomenib may play an important role in the treatment of patients with relapsed/refractory acute leukemia with KMT2A rearrangement or NPM1 mutation. "These data signal growing momentum in our oncological pipeline and an important milestone in our development of new treatments for relapsed/refractory MF and acute leukemia," said Tsutomu Nakagawa, Ph.D., President and Chief Executive Officer of SMPA. "With these promising results in hand, we remain committed to advancing nuvisertib and enzomenib as we believe our investigational therapies may have the potential to improve the lives of patients living with these critical cancers who do not respond to currently available therapeutics." MF is a rare type of blood cancer that is characterized by the buildup of fibrous tissue in the bone marrow, which can affect the production of blood cells. This buildup is caused by a dysregulation in the JAK signaling pathway. MF is a serious and rare disease with 0.7 new cases per 100,000 people worldwide each year.4 Leukemia is a type of cancer that forms in blood-forming tissue, characterized by the uncontrolled growth of blood cells, usually white blood cells, in the bone marrow. Acute leukemia, a form of leukemia, requires immediate treatment as blood cells multiply rapidly leading to a sudden onset of symptoms.1 Approximately 30% of AML patients have NPM1 mutations2 and 5-10% of AML patients have KMT2A (MLL) rearrangements.3 "Patients and families living with incurable cancers of MF and AML are in need of new treatment options – and we are encouraged by the promising clinical activity for both nuvisertib and enzomenib," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "This additional preliminary data reinforces the strong potential for PIM1 inhibition in myelofibrosis, and for menin inhibitors in acute leukemia." About enzomenib (DSP-5336) Enzomenib (DSP-5336) is an investigational small molecule inhibitor of the menin and mixed-lineage leukemia (MLL) protein interaction. Menin is a scaffold nuclear protein which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.5,6 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations.5,7 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1, and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with MLL rearrangements and NPM1 mutation.8,9 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose escalation/dose expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in June 2024. The PMDA granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in September 2024. About nuvisertib (TP-3654) Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.10,11 Nuvisertib was observed to inhibit proliferation and induce apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2V617F mutation.10 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2V617F and MPLW515L murine models of myelofibrosis.11 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (NCT04176198). The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for nuvisertib for the indication of myelofibrosis in May 2022. The PMDA granted Orphan Drug Designation for nuvisertib for the indication of myelofibrosis in November 2024. About Sumitomo Pharma Sumitomo Pharma Co., Ltd. is a global pharmaceutical company based in Japan with key operations in the U.S. (Sumitomo Pharma America, Inc.), Canada (Sumitomo Pharma Canada, Inc.) and Europe (Sumitomo Pharma Switzerland GmbH) focused on addressing patient needs in oncology, urology, women's health, rare diseases, psychiatry & neurology, and cell & gene therapies. With several marketed products in the U.S., Canada, and Europe, a diverse pipeline of early- to late-stage assets, we aim to accelerate discovery, research, and development to bring novel therapies to patients sooner. For more information on SMPA, visit our website or follow us on LinkedIn. SUMITOMO PHARMA is a trademark of Sumitomo Pharma Co., Ltd., used under license. Sumitomo Pharma America, Inc. is a U.S. subsidiary of Sumitomo Pharma Co., Ltd. © 2024 Sumitomo Pharma America, Inc. All rights reserved. References Chennamadhavuni A, Lyengar V, Mukkamalla SKR, et al. Leukemia. [Updated 2023 Jan 17]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan-. Available from: . Brandi ML, Agarwal SK, Perrier ND, Lines KE, Valk GD, Thakker RV. Multiple Endocrine Neoplasia Type 1: Latest Insights. Endocr Rev. 2021;42(2):133-170. doi:10.1210/endrev/bnaa031 Borkin D, Klossowski S, Pollock J, et al. Complexity of Blocking Bivalent Protein-Protein Interactions: Development of a Highly Potent Inhibitor of the Menin-Mixed-Lineage Leukemia Interaction. J Med Chem. 2018;61(11):4832-4850. doi:10.1021/acs.jmedchem.8b00071. Hernández-Boluda J-C, Czerw T. Transplantation Algorithm for Myelofibrosis in 2022 and Beyond. Best Practice & Research Clinical Haematology. June 2022, 101369. doi: 10.1016/j.beha.2022.101369 Cierpicki T, Grembecka J. Challenges and opportunities in targeting the menin-MLL Interaction. Future Med Chem. 2014; 6(4):447-462. doi:10.4155/fmc.13.214. Matkar S, Thiel A, Hua X. Menin: a scaffold protein that controls gene expression and cell signaling. Trends Biochem Sci. 2013; 38(8):394-402. doi:10.1016/j.tibs.2013.05.005. Kuhn MWM, Song E, Feng Z, et al. Targeting chromatin regulators inhibits leukemogenic gene expression in NPM1 mutant leukemia. Cancer Discov. 2016;6(10):1166-1181. doi:10.1158/2159-8290.CD-16-0237. Eguchi K, Shimizu T, Kato D, et al. Preclinical Evaluation of a Novel Orally Bioavailable Menin-MLL Interaction Inhibitor, DSP-5336, for the Treatment of Acute Leukemia Patients with MLL-Rearrangement or NPM1 Mutation. Blood 2021; 138 (Supplement 1): 3339. Available at: . Daver N, Zeidner JF, Yuda J, et al. Phase 1/2 First-in-Human Study of the Menin-MLL Inhibitor DSP-5336 in Patients with Relapsed or Refractory Acute Leukemia. Blood 2023; 142 (Supplement 1): 2911. Available at: . Foulks JM, Carpenter KJ, Luo B, et al. A small-molecule inhibitor of PIM kinases as a potential treatment for urothelial carcinomas. Neoplasia. 2014;16(5):403-412. Dutta A., Nath D, Yang Y, et al. Genetic ablation of Pim1 or pharmacologic inhibition with TP-3654 ameliorates myelofibrosis in murine models. Leukemia. 2022; 36 (3): 746-759. doi: 10.1038/s41375-021-01464-2. For a copy of this release, visit Sumitomo Pharma America's website at . SOURCE Sumitomo Pharma America WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

孤儿药临床结果ASH会议快速通道

2024-11-14

·生物制药小编

连续两次“疗效显著，股价大跌”？

近日（11月12日），Syndax公司宣布了核心管线menin抑制剂revumenib的临床数据，意外的是，即便revumenib达到了临床终点，但是公司股价却截止12日收盘大跌了约25.6%，实际上这已经不是Syndax第一次出现这种“疗效显著，但是股价大跌”的情况了，在去年10月份时，看似积极的消息同样引发了股价下跌。缩水的临床数据 revumenib主要有两个适应症，伴有NPM1突变的急性髓系白血病（AML）和KMT2A重排急性白血病患者。此次遭到诟病的是NPM1突变AML的临床数据，而早先出现问题的则是KMT2A重排急性白血病的临床数据：在2022年年底时，该公司发布了revumenib的I期临床AUGMENT-101的第一阶段临床数据。当时公布的数据显示KMT2A重排急性白血病患者的完全缓解率(CR/CRh)为27%。完全缓解的中位持续时间为9.1个月。然而到了2023年10月份的时候，这个数据出现了显著缩水，合并的KMT2Ar AML和所有队列的CR/CRh率为23%，比早期读数下降了4个百分点。中位持续时间也从原来的9.1个月缩减到了6.4个月，显然比I期试验短。在这个问题出现后，Syndax的CEO Michael Metzger当时对此的解释是，“6.4个月”这个数据还有变化的空间。而且现阶段的“6.4个月”还包括大约一半仍在接受治疗和处于缓解期的患者，现在妄下定论还为时过早，有几名患者仍在继续治疗，他预计这一时间会延长。虽然如此，但是这一数据似乎已经足够让Syndax推动上市进程了，今年12月26日就是FDA的PDUFA日，届时revumenib将迎来FDA的最终裁决。从管线机制来看，menin抑制剂主要是针对menin与KMT2A形成的复合物异常导致的癌变。在细胞核中，menin作为一种支架蛋白，会与KMT2A等蛋白相互作用，参与调节基因转录。而在某些白血病中，如上述两种适应症中，menin与KMT2A的相互作用变得异常，这会导致白血病相关基因（如HOX、MEIS1等）的异常表达，进而引发白血病。然而，不可否认的是使用menin抑制剂可能存在耐药情况，这种临床耐药性是通过获得menin突变导致的，而这一突变会阻止抑制剂结合，并可导致临床复发。早先发表的论文就表明，患者进入第二个治疗周期后，可能会出现抵抗性的情况。这一隐忧同样被带到了NPM1突变AML中，此次NPM1突变AML中的临床数据不及预期，未来可能无法战胜对手，下一阶段的临床读数会不会更差？或许输给了竞争对手而这次发布的NPM1突变AML的临床数据则主要输在了和竞争对手的对比上。竞争对手Kura Oncology同样开发了一款Menin抑制剂ziftomenib，在单药针对NPM1突变AML的KOMET-001临床中，600mg剂量的ziftomenib有35%（7/20）的患者达到了完全缓解，而相比之下，revumenib 23%（15/64）的CR+sCR率就很有压力了。尽管样本量很大，未来在这一适应症上，会不会出现KMT2A重排适应症中的数据衰退现象值得担忧。毕竟，市场对这次数据的普遍的预期是27%以上。类比同赛道的其他竞争对手，住友制药的enzomenib在13例同适应症的小规模临床中这一数值也是23%左右。另外，menin抑制剂这一赛道还有，第一三共的DS-1594，和黄医药的HMPL-506，烨辉医药的BN104，强生的JNJ-75276617等竞争对手，未来的竞争压力并不容乐观。总结总的来说，这次公布的数据对于刚刚与Royalty Pharma做交易的Syndax并不是一个好消息，与Royalty Pharma的交易意味着Royalty Pharma会获得另外一款产品核心管线Niktimvo的版税分成。而此次公布的临床数据又让另外一款核心管线revumenib的未来收益预期有所下调。参考来源： Wang ES, et al. Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial.Lancet Oncol . 2024 Oct;25(10):1310-1324. doi: 10.1016/S1470-2045(24)00386-3. https://ir.syndax.com/news-releases/news-release-details/syndax-reports-third-quarter-2024-financial-results-and-provides https://ir.syndax.com/static-files/0c985b19-5533-41c6-b38f-86ff9e57259d

临床1期临床结果

2024-11-13

·医药观澜

住友制药两款血液肿瘤新药亮相ASH口头报告

▎药明康德内容团队报道 2024年美国血液学会（ASH）年会即将于当地时间12月7日~10日在美国圣地亚哥举行。住友制药（Sumitomo Pharma）两款血液肿瘤新药将于本次ASH年会上以口头报告形式公布最新临床研究结果，包括：enzomenib （DSP-5336），一款Menin-KMT2A相互作用抑制剂，针对复发或难治性急性白血病患者；以及nuvisertib （TP-3654），一款高选择性PIM1激酶抑制剂，针对复发/难治性骨髓纤维化患者。 DSP-5336治疗复发或难治性急性白血病患者的1/2期首次人体临床研究结果积极 DSP-5336是住友制药在研的一款Menin-KMT2A相互作用抑制剂，正在开展针对复发或难治性急性白血病患者的1/2期临床研究，该产品还曾获FDA授予用于急性髓性白血病的孤儿药资格。基于Menin-KMT2A相互作用开发的Menin抑制剂被认为是一种治疗白血病的有前景的新疗法，尤其是对那些携带KMT2A基因重排或NPM1基因突变的AML患者。研究人员对以KMT2Ar、NPM1m和其他HOXA9/MEIS1驱动的白血病亚群为特征的R/R急性白血病成人患者进行了一项enzomenib单药治疗1/2期研究。截至2024年6月24日，共有81名患者入组。在1期部分，DSP-5336剂量从40mg BID增加到300mg BID (n=81)，未观察到剂量限制性毒性（DLT）。结果显示，在22例KMT2Ar患者（20例AML，2例ALL）中，根据ELN 2017（CR+CRi+MLFS）评估的客观缓解率（ORR）为59.1% ，22.7%的患者达到完全缓解（CR）或带部分血液学恢复的完全缓解（CRh）。在13例NPM1m AML患者中，ORR为53.8%，23.1%患者达到CR+CRh。根据研究人员得出的结论，在81例R/R急性白血病患者中，DSP-5336耐受良好，无DLT。未观察到与治疗相关的停药或死亡，药代动力学（PK）研究未发现与强CYP3A4抑制剂唑类药物有显著的药物相互作用。在140mg至300mg BID的治疗范围内，在R/R KMT2Ar和NPM1m急性白血病患者中观察到有希望的单药活性。 TP-3654在复发/难治性骨髓纤维化患者中显示出持久的临床反应和持续的血液学改善 PIM1在包括骨髓纤维化（MF）在内的血液恶性肿瘤中往往表达上调。在临床前模型中，PIM1敲除（PIM1 knockout，KO）被证明可以在不影响血小板（PLT）计数的情况下阻止MF进展，而泛PIM KO会导致PLT计数下降。TP-3654是一种口服的高选择性PIM1激酶抑制剂，该产品单药和联合JAK抑制剂在小鼠骨髓纤维化模型中显示白细胞和中性粒细胞计数正常化，并减少脾脏大小和骨髓纤维化的作用。FDA曾于2022年授予该产品治疗骨髓纤维化的孤儿药资格。本次这项全球1/2期研究评估了该产品单药治疗MF患者的安全性和有效性。截至2024年6月10日，65名患者参加了从480mg QD到720mg BID的5个剂量水平的研究。根据研究人员得出的结论，TP-3654单药治疗无DLT，耐受性良好。TP-3654在复发/难治性MF患者中的初步数据显示，其临床活性包括SVR25、与细胞因子减少相关的症状改善、BM纤维化减少以及血红蛋白（Hgb）和血小板计数（PLT）反应。目前的单药治疗和临床前数据支持TP-3654联合JAK抑制剂进行后续开发，目前相关的研究正在进行中。参考资料： [1] Sumitomo Pharma America to Present New Investigational Data at the 2024 American Society of Hematology Annual Meeting. Retrieved Nov 6, 2024, from https://news.us.sumitomo-pharma.com/2024-11-06-Sumitomo-Pharma-America-to-Present-New-Investigational-Data-at-the-2024-American-Society-of-Hematology-Annual-Meeting [2]ASH官网. 本文来自药明康德内容团队，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。转载授权及其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。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。

临床结果临床1期孤儿药

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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	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	日本	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	比利时	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	加拿大	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	法国	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	意大利	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	新加坡	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	韩国	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	西班牙	Sumitomo Pharma America, Inc.	2022-02-28
急性淋巴细胞白血病	临床2期	中国台湾	Sumitomo Pharma America, Inc.	2022-02-28