更新于：2024-09-20

Venetoclax

维奈克拉

更新于：2024-09-20

概要

概要

基本信息

药物类型

小分子化药

别名

4-{4-[(4'-chloro-5,5-dimethyl[3,4,5,6-tetrahydro[1,1'-biphenyl]]-2-yl)methyl]piperazin-1-yl}-N-(3-nitro-4-{[(oxan-4-yl)methyl]amino}benzene-1-sulfonyl)-2-[(1H-pyrrolo[2,3-b]pyridin-5-yl)oxy]benzamide、VENCLYXTO、Venetoclax (JAN/USAN/INN)

+ [14]

靶点

Bcl-2

作用机制

Bcl-2抑制剂(细胞凋亡调控因子Bcl-2抑制剂)

治疗领域

癌症免疫系统疾病血液及淋巴系统疾病+ [7]

在研适应症

难治性慢性淋巴细胞白血病复发性慢性淋巴细胞白血病小淋巴细胞淋巴瘤+ [108]

非在研适应症

侵袭性非霍奇金淋巴瘤复发性B细胞淋巴瘤难治性B细胞淋巴瘤+ [27]

原研机构

Genentech, Inc.

AbbVie, Inc.

在研机构

Genentech, Inc.

AbbVie, Inc.AbbVie Deutschland GmbH & Co. KG

+ [23]

非在研机构

Janssen Research & Development LLCOhio State University Comprehensive Cancer Center

最高研发阶段批准上市

首次获批日期

美国 (2016-04-11),

慢性淋巴细胞白血病

最高研发阶段(中国)批准上市

特殊审评突破性疗法 (美国)、加速批准 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、附条件批准 (中国)、特殊审批 (中国)、孤儿药 (澳大利亚)

登录后查看首次获批时间轴

结构

分子式C45H50ClN7O7S

InChIKeyLQBVNQSMGBZMKD-UHFFFAOYSA-N

CAS号1257044-40-8

关联

673

项与维奈克拉相关的临床试验

NCT06522386 / Not yet recruiting临床2期

GATE1: A Multicenter Phase II Study of Pirtobrutinib, Rituximab and Venetoclax Combination Therapy for Patients With Previously Untreated Mantle Cell Lymphoma

Primary Objectives:
To estimate the percent of participants who achieve a best response of complete response by the end of the PRV combination therapy in the induction therapy phase in patients with previously untreated MCL.

开始日期2025-12-01

申办/合作机构

M.D. Anderson International España SA

NCT06454409 / Not yet recruiting临床1期IIT

A Phase 1b Study of the Multi-Kinase Inhibitor Regorafenib in Combination With the BCL-2 Inhibitor Venetoclax Plus Azacitidine in Patients With Relapsed/Refractory Acute Myeloid Leukemia

This phase Ib trial tests the safety, side effects, best dose and effectiveness of regorafenib in combination with venetoclax and azacitidine in treating patients with acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Regorafenib is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply. This helps to slow or stop the spread of cancer cells. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking BCL-2, a protein needed for cancer cell survival. Azacitidine is in a class of medications called demethylation agents. It works by helping the bone marrow to produce normal blood cells and by killing abnormal cells. Giving regorafenib in combination with venetoclax and azacitidine may be safe, tolerable and/or effective in treating patients with relapsed or refractory AML.

开始日期2025-03-20

申办/合作机构

City of Hope National Medical Center

[+1]

NCT06317649 / Recruiting临床2期IIT

A Randomized Phase II Study of Venetoclax and HMA-Based Therapies for the Treatment of Older and Unfit Adults With Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia (AML): A MyeloMATCH Treatment Trial

This phase II MyeloMATCH treatment trial compares the usual treatment of azacitidine and venetoclax to the combination treatment of azacitidine, venetoclax and gilteritinib in treating older and unfit patients with acute myeloid leukemia and FLT3 mutations. Azacitidine is a drug that is absorbed into DNA and leads to the activation of cancer suppressor genes, which are genes that help control cell growth. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Gilteritinib is in a class of medications called kinase inhibitors. It works by blocking the action of a certain naturally occurring substance that may be needed to help cancer cells multiply. This study may help doctors find out if these different approaches are better than the usual approaches. To decide if they are better, the study doctors are looking to see if the study drugs lead to a higher percentage of patients achieving a deeper remission compared to the usual approach.

开始日期2025-03-03

申办/合作机构

National Cancer Institute

100 项与维奈克拉相关的临床结果

登录后查看更多信息

100 项与维奈克拉相关的转化医学

登录后查看更多信息

100 项与维奈克拉相关的专利（医药）

登录后查看更多信息

2,834

项与维奈克拉相关的文献（医药）

2024-12-31·Hematology (Amsterdam, Netherlands)

Reduced duration and dosage of venetoclax is efficient in newly diagnosed patients with acute myeloid leukemia

Article

作者: Yan, Qiong ; Cui, Jingying ; Yuan, Guolin ; Chen, Xuexing ; Li, Chunfang

OBJECTIVES:

The combination of Venetoclax (VEN) and Azacitidine (AZA) increases survival outcomes and yields excellent responses in patients with acute myeloid leukemia (AML). However, dose reduction (or discontinuation) is commonly encountered due to therapy-related toxicity. Thus, this study aimed to investigate the efficiency and safety of a lower dosage of venetoclax for the treatment of AML.

METHODS:

This observational study analyzed the characteristics and outcomes of newly diagnosed AML patients who received 100 mg VEN combined with AZA for 14 days at our institution.

RESULTS:

A total of 36 patients were enrolled, and the median age at diagnosis was 64 years. After a median follow-up of 15 (range 4-29) months, the median overall survival (OS) and progression-free survival (PFS) for the whole cohort were 17 (4-29) months and 12 (1-28) months, respectively. Meanwhile, the overall response rate (ORR) was 69.4%, and the CRc rate was 66.7% in the whole cohort. Subgroup analysis revealed that NPM1 mutations and FAB-M5 subtype were associated with higher response rates, whereas the adverse ELN risk group was predictive of an inferior response. Moreover, ASXL1, NPM1, and IDH1/2 mutations negatively impacted PFS.

DISCUSSION:

Our study optimized the administration of venetoclax plus azacytidine for the treatment of AML patients. Response rates were favorable, with median survival in agreement with the findings of earlier reports, offering valuable insights for optimizing VEN-based regimens.

CONCLUSION:

In summary, the VEN combination regimen is effective for the treatment of newly diagnosed AML patients in the real world despite VEN dose reductions .

2024-12-31·Hematology (Amsterdam, Netherlands)

Advancing the understanding of venetoclax in t(11;14)-positive multiple myeloma: a comprehensive review of clinical evidence and future prospects

Review

作者: AlZahrani, Abdullah ; Alrajhi, Abdullah M ; Tailor, Imran K ; Alsuhebany, Nada

Venetoclax is a selective inhibitor of the anti-apoptotic protein B-cell lymphoma 2 (BCL2), as a targeted therapy for multiple myeloma (MM) patients. It was initially approved by the United States Food and Drug Administration for the treatment of chronic lymphocytic leukemia in April 2016 and later for acute myeloid leukemia in October 2020. However, venetoclax is used as an off-label in a subset group of relapsed and refractory multiple myeloma (RRMM) patients with the presence of translocation t(11;14). Preclinical and clinical studies have highlighted the potential of venetoclax in the management of MM patients, with a specific focus on t(11;14) as a predictive biomarker for initiating venetoclax-based treatment. Later, several studies in RRMM patients that used venetoclax in combination with dexamethasone or/and proteasome inhibitors have shown promising results, in which management guidelines have included venetoclax as one of the options to treat MM patients. Hence, this review focuses on the use of venetoclax in RRMM, clinical efficacy, safety, dosing strategies, and predictive biomarkers for initiating venetoclax. Additionally, we discuss ongoing studies that are investigating different combination of venetoclax regimens in MM patients.

2024-12-31·Hematology (Amsterdam, Netherlands)

Case report: Venetoclax plus Azacitidine in treatment of acute undifferentiated leukemia

Review

作者: Chen, Lin ; Mi, Ruihua ; Wei, Xudong ; Wang, Lin ; Cui, Yu ; Li, Dongbei

OBJECTIVES:

Acute undifferentiated leukemia (AUL) is a clinical rare leukemia with an overall poor prognosis. Currently, there are no well-established treatment guidelines for AUL, further exploration of optimal treatment options is now required.

METHODS:

We report an AUL patient who was complicated by a NRAS mutation and del5q was admitted to our hospital and we present the clinical features. In addition, we conducted a literature review.

RESULTS:

The "VA" scheme combines agents Venetoclax and Azacitidine that have synergistic therapeutic effect with a tolerable safety profile. There is no previous report of the "VA" scheme employed in AUL treatment. An AUL patient who was complicated by a NRAS mutation and del5q was admitted to our hospital. The "VA" scheme was administrated, and complete remission (CR) was achieved at the end of the first cycle. The patient then underwent HLA-identical sibling allogeneic hematopoietic stem cell transplantation.

DISCUSSION:

The "VA" scheme has been extensively used in AML treatment, but its application in AUL treatment has not yet been reported. This study is the first to report an AUL patient treated with the "VA" scheme and achieved CR. Our result preliminarily suggested the effectiveness and safety of the "VA" scheme in AUL treatment, but validation is required in more clinical samples. The "VA" scheme provides a new treatment option for AUL patients and deserves further clinical promotion.

1,051

项与维奈克拉相关的新闻（医药）

6 小时之前

·PRNewswire

Mantle Cell Lymphoma Clinical Trial Pipeline Insights Featuring 20+ Companies | DelveInsight

Mantle cell lymphoma is a rare and aggressive type of non-Hodgkin lymphoma originating from B-cells in the "mantle zone" of the lymph nodes. The development of novel targeted therapies and immunotherapies, such as Bruton's tyrosine kinase (BTK) inhibitors, is improving treatment outcomes and driving market growth for Mantle cell lymphoma. LAS VEGAS, Sept. 19, 2024 /PRNewswire/ -- DelveInsight's ' Mantle Cell Lymphoma Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline mantle cell lymphoma therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the mantle cell lymphoma pipeline domain. Key Takeaways from the Mantle Cell Lymphoma Pipeline Report DelveInsight's mantle cell lymphoma pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for mantle cell lymphoma treatment. Key mantle cell lymphoma companies such as AbbVie, Adicet Bio, InnoCare Pharma Inc., Guangzhou Lupeng Pharmaceutical Company LTD., BeiGene, Nurix, LegoChem Biosciences, Xynomic Pharmaceuticals, Merck & Co, Incyte Corporation, TG Therapeutics, Janssen/Pharmacyclics, Eternity Bioscience, Traws Pharm, Advenchen Laboratories, and others are evaluating new mantle cell lymphoma drugs to improve the treatment landscape. Promising mantle cell lymphoma pipeline therapies such as Venetoclax, ADI-001, Orelabrutinib, LP-168, BGB-11417, NX 2127, LCB 71, NX 5948, Abexinostat, Pembrolizumab, Parsaclisib, Umbralisib, Ibrutinib, Edralbrutinib, Narazaciclib, Lucitanib, and others are under different phases of mantle cell lymphoma clinical trials. In May 2024, AstraZeneca had announced positive interim results from the Phase III ECHO clinical trial, which evaluated Bruton's tyrosine kinase (BTK) inhibitor Calquence (acalabrutinib) in combination with standard chemoimmunotherapy for treating mantle cell lymphoma (MCL). In March 2024, BeiGene, Ltd. announced the presentation of emerging oncology pipeline data at the American Association for Cancer Research (AACR) Annual Meeting. Additional highlights include preclinical characterization data for an investigational BTK degrader, BGB-16673, currently in clinical development for B-cell malignancies, including mantle cell lymphoma In October 2023, Ascentage Pharma announced that it has entered into a clinical collaboration with AstraZeneca Investment. The two companies will jointly conduct a registrational Phase III study of the Bcl-2 inhibitor, APG-2575 (lisaftoclax), in combination with AstraZeneca's Bruton's tyrosine kinase (BTK) inhibitor, CALQUENCE® (acalabrutinib), in treatment-naive patients with first-line chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). In January 2023, the FDA granted an orphan drug designation to LP-284, a novel small-molecule, next-generation acylfulvene, for the treatment of mantle cell lymphoma (MCL), a rare and aggressive form of B-cell non-Hodgkin lymphoma (NHL), according to an announcement from Lantern Pharma. Request a sample and discover the recent advances in mantle cell lymphoma treatment drugs @ Mantle Cell Lymphoma Pipeline Report The mantle cell lymphoma pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage mantle cell lymphoma drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the mantle cell lymphoma clinical trial landscape. Mantle Cell Lymphoma Overview Mantle cell lymphoma (MCL) is a rare subtype of B-cell non-Hodgkin lymphomas (NHLs) characterized by the (11,14) translocation, leading to overexpression of the cyclin D1 (CCND1) gene. Its various morphological variants can make diagnosis challenging, though some cases are straightforward. Many individuals with MCL are asymptomatic in the early stages, but may later seek medical attention due to persistent, typically painless swelling of lymph nodes, especially in the neck and throat region (e.g., Waldeyer's ring). MCL is marked by a reciprocal chromosomal translocation t(11;14)(q13;q32), which places the cyclin D1 gene next to the immunoglobulin heavy chain gene, causing continuous cyclin D1 expression. This overexpression contributes to tumor cell proliferation through cell cycle dysregulation, chromosomal instability, and epigenetic changes. Rarely, MCL cases without this translocation may involve CCND2 or CCND3 translocations. Hypothetical molecular subtypes, based on the cell of origin, correlate with clinical phenotypes: classical or aggressive MCL arises from naive B cells lacking or having limited iGVH mutations and expressing SOX11, a neural transcription factor inhibiting terminal B cell differentiation. The indolent variant originates from antigen-experienced B cells with IGVH somatic hypermutations, typically SOX11-negative and genetically stable. Diagnosis of MCL involves detailed patient history, thorough clinical evaluation, and specialized tests, including biopsy of an affected lymph node or bone marrow. These tests confirm the NHL type and subtype, assess disease extent, and determine appropriate treatments. Treatment for MCL is multifaceted and tailored to each patient, often including chemotherapy, immunotherapy drugs like rituximab, targeted therapies such as ibrutinib, and stem cell transplants, particularly for aggressive disease or younger patients. Radiation therapy may target specific lymphoma areas. Participation in clinical trials can offer new treatment options. Treatment plans are developed collaboratively by healthcare professionals and patients, considering disease stage, overall health, and personal preferences. Regular monitoring and follow-up care are essential to track treatment response and manage side effects effectively. Find out more about mantle cell lymphoma treatment drugs @ Drugs for Mantle Cell Lymphoma Treatment A snapshot of the Mantle Cell Lymphoma Pipeline Drugs mentioned in the report: Learn more about the emerging mantle cell lymphoma pipeline therapies @ Mantle Cell Lymphoma Clinical Trials Mantle Cell Lymphoma Therapeutics Assessment The mantle cell lymphoma pipeline report proffers an integral view of the mantle cell lymphoma emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Mantle Cell Lymphoma Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Apoptosis stimulants, Proto-oncogene protein c-bcl-2 inhibitors, Proteasome inhibitors, Immunologic cytotoxicity, T lymphocyte replacements, Agammaglobulinemia tyrosine kinase inhibitors Key Mantle Cell Lymphoma Companies: AbbVie, Adicet Bio, InnoCare Pharma Inc., Guangzhou Lupeng Pharmaceutical Company LTD., BeiGene, Nurix, LegoChem Biosciences, Xynomic Pharmaceuticals, Merck & Co, Incyte Corporation, TG Therapeutics, Janssen/Pharmacyclics, Eternity Bioscience, Traws Pharm, Advenchen Laboratories, and others Key Mantle Cell Lymphoma Pipeline Therapies: Venetoclax, ADI-001, Orelabrutinib, LP-168, BGB-11417, NX 2127, LCB 71, NX 5948, Abexinostat, Pembrolizumab, Parsaclisib, Umbralisib, Ibrutinib, Edralbrutinib, Narazaciclib, Lucitanib, and others Dive deep into rich insights for new drugs for mantle cell lymphoma treatment, visit @ Mantle Cell Lymphoma Drugs Table of Contents For further information on the mantle cell lymphoma pipeline therapeutics, reach out @ Mantle Cell Lymphoma Treatment Drugs Related Reports B-Cell Lymphoma Epidemiology B-Cell Lymphoma Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the B-cell lymphoma epidemiology trends. B-Cell Lymphoma Market B-Cell Lymphoma Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key B-cell lymphoma companies, including Vincerx Pharma, Ubix Therapeutics, Biomea Fusion Inc, Shanghai Hengrui Pharmaceutical, BeiGene, Hanmi Pharmaceutical, among others. Diffuse Large B-Cell Lymphoma Pipeline Diffuse Large B-Cell Lymphoma Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DLBCL companies, including Genmab, Roche, Acerta Pharma, Autolus, Xynomic Pharmaceuticals, Genentech, Celgene, AbbVie, Mabion, among others. Diffuse Large B-Cell Lymphoma Market Diffuse Large B-Cell Lymphoma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key B-cell lymphoma companies, including AbbVie, Genmab, Merck, Roche, Xencor and Janssen, Denovo Biopharma, Calithera Biosciences, IMV, Biogen, Autolus Therapeutics, Allogene Therapeutics, Novartis, Miltenyi Biomedicine, Regeneron Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期免疫疗法孤儿药

15 小时之前

·PRNewswire

Ryvu Therapeutics Announces Dosing of the First Patient in the REMARK Phase II Study of RVU120 for the Treatment of Anemia in Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS)

Ryvu Therapeutics has announced the dosing of the first patient in the REMARK Phase II study of RVU120. The primary goal of the REMARK study is to evaluate the safety and efficacy of RVU120 in patients with lower-risk myelodysplastic syndromes (MDS). The study is conducted as an investigator-initiated study through the European Myelodysplastic Neoplasms Cooperative Group (EMSCO) network with Prof. Uwe Platzbecker, a globally renowned expert in the field of LR-MDS, as the Coordinating Principal Investigator. The REMARK study has already received approval from the Competent Authorities and Ethics Committees of Germany, Spain, Poland, Italy, and France under the European Union Clinical Trial Regulation (EU-CTR) 2023-509947-29. It will cover up to 25 clinical sites globally. The planned overall enrollment is up to approximately 40 patients. KRAKOW, Poland, Sept. 19, 2024 /PRNewswire/ -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, announced today that the first patient has been dosed in the REMARK study, a Phase II clinical trial investigating RVU120 as a monotherapy for the treatment of patients with LR-MDS. "We are glad to announce the initiation of the REMARK study for RVU120, which can potentially help patients with lower-risk MDS. This study builds upon the promising results from our Phase Ib study in patients with AML and high-risk MDS, where we observed hematologic improvement in several patients, including cases of transfusion independence. The objective is to further assess the safety and efficacy of RVU120 in patients with lower-risk MDS, underpinned by robust preclinical and mechanistic evidence. We believe this represents a significant advancement toward our objective of developing effective treatments for hematological diseases and offering therapeutic options for patients. I am delighted that we can count on the support of the EMSCO network and Prof Uwe Platzbecker in this endeavor." - said Hendrik Nogai, M.D., Chief Medical Officer of Ryvu Therapeutics. REMARK is an open-label, multicenter Phase II study of RVU120, a novel small-molecule cyclin-dependent kinase (CDK) 8/19 inhibitor. The study aims to treat anemia in patients with lower-risk myelodysplastic syndromes (MDS). In REMARK, RVU120 is being explored as a single agent in patients with LR-MDS who have exhausted available treatment options. The REMARK study is being conducted as an investigator-initiated study through the EMSCO network with Prof. Uwe Platzbecker, a globally renowned expert in the field of LR-MDS, as the Coordinating Principal Investigator. "I am proud that we could start the REMARK study in line with our ambitious plans. RVU120 has shown promising hematologic improvement in patients with off-hematologic impaired bone marrow function. I am optimistic that this clinical evidence will translate into a positive outcome of the REMARK study. RVU120 has important features that should be considered a potential new treatment option for patients with LR-MDS. It may aid in achieving our ultimate aim to alleviate the need for red blood cell transfusions in these patients." - said Uwe Platzbecker, M.D., Director of the Clinic and Poliklinik for Hematology, Cell Therapy and Hemostaseology at the Leipzig University Hospital. REMARK is being initiated based on the clinical safety and efficacy data gathered so far, as well as strong preclinical and mechanistic rationale. MDS pathogenesis is influenced by gene expression alterations that hinder the maturation of hematopoietic cells. RVU120 triggers erythroid gene expression programs orchestrated by STAT5 and GATA1 in aberrant stem cells from MDS patients. Importantly, RVU120's activity does not lead to significant toxicity in the hematopoietic system. As a result, RVU120 emerges as a promising drug candidate for treating transfusion-dependent MDS patients. In REMARK, patients will receive RVU120 for at least 8 complete cycles (24 weeks). The primary goal is to achieve hematologic improvement in the form of an erythroid response (HI-E), with secondary goals including independence from RBC transfusions, improvement in hemoglobin levels, quality of life, disease progression, and analysis of specific gene mutations. REMARK represents the third of four planned RVU120 Phase II clinical studies scheduled for launch in 2024. Ryvu has already started patient treatment in the RIVER-52 and RIVER-81 studies in AML, and the fourth Phase II trial is the planned to be initiated shortly POTAMI-61 study, evaluating both monotherapy and combination therapy for the treatment of patients with myelofibrosis (MF). About Ryvu Therapeutics Ryvu Therapeutics is a clinical-stage drug discovery and development company focused on novel small-molecule therapies that address emerging targets in oncology. Internally discovered pipeline candidates at Ryvu use diverse therapeutic mechanisms driven by emerging knowledge of cancer biology, including small molecules directed at kinases, synthetic lethality, and immuno-oncology targets. Ryvu's most advanced program is RVU120, a selective CDK8/CDK19 kinase inhibitor with the potential to treat hematological malignancies and solid tumors. RVU120 is currently in Phase II development (i) as a monotherapy for the treatment of patients with relapsed/refractory acute myeloid leukemia (r/r AML) and high-risk myelodysplastic syndromes (HR-MDS) – the RIVER-52 study, (ii) in combination with venetoclax for the treatment of patients with r/r AML – the RIVER-81 study, and (iii) a monotherapy for the treatment of patients with lower risk myelodysplastic syndromes (LR-MDS) – the REMARK study. SEL24 (MEN1703) is a dual PIM/FLT3 kinase inhibitor licensed to the Menarini Group that is expected to start a Phase II study in diffuse large B-cell lymphoma (DLBCL) in Q4 2024. RVU305, a potentially best-in-class PRMT5 inhibitor aiming to treat multiple solid tumors, is currently undergoing IND/CTA-enabling studies. Ryvu Therapeutics has signed 11 partnering and licensing deals with global companies, including BioNTech and Exelixis. The Company was founded in 2007 and is headquartered in Kraków, Poland. Ryvu is listed on the Warsaw Stock Exchange and is a component of the mWIG40 index. For more information, please visit About EMSCO The European Myelodysplastic Neoplasms Cooperative Group (EMSCO) serves as a platform to foster academic clinical research, cooperation and education in the field of myelodysplastic syndromes (MDS). EMSCO is administered by GWT-TUD GmbH and was founded in 2013 upon an initiative of the European Leukemia Network (ELN). Originally founded to facilitate European MDS-specific trials, EMSCO soon also became active in organizing indication-specific conferences and in serving as a platform for education and outreach in MDS. SOURCE Ryvu Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期引进/卖出

19 小时之前

·药渡Daily

亚盛医药的成功上岸早在意料之中

亚盛医药的成功上岸早在意料之中。 2024年上半年，亚盛医药实现营业收入8.24亿元，同比增长477%；首次实现扭亏为盈，净利润达1.63亿元。抛开奥雷巴替尼出海带来的巨额收入不说，单就在国内的商业化成绩就已开始放量。2024年上半年，奥雷巴替尼的销售收入较2023年下半年及上半年分別增加120%及5%；进入国家医保定点医院数量较2023年底增加79%。7月又在澳门获批，适应症为治疗任何酪氨酸激酶抑制剂（TKI）耐药、并伴有T315I突变的慢性髓细胞白血病（CML）慢性期（-CP）和加速期（-AP）的成年患者，以及治疗对一代和二代TKI耐药及/或不耐受的CML-CP成年患者。奥雷巴替尼的最新进展还包括被纳入2024美国国家综合癌症网络（NCCN）慢性髓细胞白血病（CML）治疗指南、被纳入2024版《CSCO恶性血液病诊疗指南》，作为涉及慢性髓系白血病(CML)、费城染色体阳性急性淋巴细胞白血病（Ph+ ALL）领域的治疗的指南。临床研究也取得诸多进展，2024年5月，获国家药监局临床试验许可开展奥雷巴替尼针对系统性全身治疗失败的琥珀酸脱氢酶（SDH）缺陷型胃肠间质瘤（GIST）患者的注册III期临床研究POLARIS-3；2024年2月，获得美国FDA的许可开阵奥雷巴替尼针对过往接受过治疗的慢性髓细胞白血病慢性期（CML-CP，伴有及并无伴有T315I突变）患者的注册III期临床试验POLARIS-2。另外在EHA 2024、第60届ASCO年会、美国癌症研究协会（AACR）年会公布的最新临床数据都显示了奥雷巴替尼依旧强悍。奥雷巴替尼的商业化前景从来不是需要担忧的选项，但出海的预期却大大出人意料。 2024年6月，亚盛医药与武田订立独家选择权协议，授予武田制药独家选择权以就奥雷巴替尼订立独家许可协议。根据协议，武田拥有全球范围内（除中国、香港特别行政区、澳门特别行政区及台湾等地区以外）开发以及商业化奥雷巴替尼的独家权利。亚盛医药可收取1亿美元的选择权付款，并将有资格获得最高约12亿美元的选择权行使费和额外的潜在里程碑付款，以及年销售额两位数百分比的销售分成。此外，亚盛医药还向武田制药成功发行及配发合共2430.73万股股份，总额为7500万美元，武田由此成为亚盛医药的二股东。 MNC巨头的站台，无疑为亚盛医药巩固血液瘤龙头地位提供了有力支持，以至于其股价在三个月时间内就实现了翻倍。那么武田为何不惜下血本也要夺得奥雷巴替尼的海外权益？事实上，武田很早就拥有全球首款第三代BCR-ABL抑制剂，或许这个化学名对大部分中国人来说比较陌生，但如果换成“格列卫”，就耳熟能详了。电影《我不是药神》把治疗慢性粒细胞白血病（慢粒）的格列卫（一种BCR-ABL抑制剂）带入大众认知范围，伊马替尼为一代格列卫。伊马替尼疗效显著，但获得性耐药一直是慢粒白血病治疗的主要挑战。BCR-ABL激酶区突变是获得性耐药的重要机制之一，其中T315I突变是常见的耐药突变类型之一，在耐药慢粒白血病中的发生率为25%左右。因此二代达沙替尼、尼洛替尼和博舒替尼登场，但很快也耐药了，直到2012年12月，武田的三代格列卫普纳替尼在美国上市才缓解了耐药的局面。不过令人惋惜的是，一年以后，因“危及生命的血栓和血管重度狭窄”风险，美国FDA要求武田暂停这款药的销售和推广，普纳替尼也成了首个被要求撤市的小分子激酶抑制剂类抗肿瘤药。不过，由于临床需求太大，FDA最终没有让这款药物彻底退市，而是要求企业在说明书上以黑粗框警告风险。更让武田焦虑的是，2021年美国FDA批准诺华的阿思尼布用于慢性髓系白血病（Ph+CML-CP）的三线治疗，阿思尼布也是一款三代格列卫。阿思尼布在2023年销售额达4亿美元，入选2023年20大畅销新药，并且，今年6月1日，一项关于阿思尼布用于慢性髓系白血病一线治疗的三期临床达到主要终点，销售额可能还会再翻一番。武田的普纳替尼存在心脑血管不良事件等安全短板，而且专利在2026-2027年到期，选择和竞争对手亚盛医药合作，成了武田的最优选择。奥雷巴替尼目前已成为三代格列卫中事实上的Best-in-class，因其对普纳替尼、阿思尼布耐药或者无效的慢粒白血病患者有效。临床数据显示，奥雷巴替尼对比普纳替尼用于慢粒白血病慢性期的MMR率（主要分子学缓解率，是评估慢粒白血病患者治疗反应的重要指标）分别为53%和40%，对比用于慢粒白血病加速期的MMR率分别为40%和22%。从全球临床进展来看，奥雷巴替尼2024年2月获得FDA批准开展针对慢粒白血病慢性期的全球III期注册性临床，预计2026年完成注册临床并在美国申报上市，刚好与专利到期的普纳替尼实现无缝衔接。这是一个双赢的局面，武田获得续血的大秘宝，亚盛医药则成功获得巨额资金并将借此机会在美国申请高价发行股票上市。在现金流充裕之后，亚盛医药的脚步可能要更快了。根据半年报显示，截至上半年结束，亚盛医药的现金及银行结余为11亿元，7月，收到武田支付的独家选择权协议项下知识产权收入及选择权付款相关的1亿美元，后续很快还会有大额里程碑付款进账，如今的亚盛医药可谓兵强马壮，粮草充足。后续的研发进度可能将加快脚步。 Lisaftoclax（APG-2575）是一款由亚盛医药自主研发的、全球领先的Bcl-2选择性抑制剂，它通过阻断Bcl-2来恢复癌细胞的正常凋亡过程，已在中国、美国、澳洲及欧洲开展21项针对多种血液恶性肿瘤和实体瘤的Ib/II期临床研究，并获得了FDA授予的五项孤儿药资格认定。作为全球第二个进入全球注册临床试验的Bcl-2选择性抑制剂，Lisaftoclax相较于维奈克拉具备更优的有效性和安全性，展现出Best-in-class潜力。据临床验证，Lisaftoclax在治疗维奈克拉（唯一上市药物）无效的BTK耐药患者中显示出有效性，并且与BTKi联合使用时，对于TN CLL/SLL患者达到100%的ORR，对于r/r CLL/SLL患者也实现了98%的ORR。在安全性方面，Lisaftoclax中性粒细胞减少症和血小板减少症的发生率分别为30.3 %和13%，相较于维奈克拉显著降低（45%和21%），同时临床肿瘤溶解综合征的风险也较低。此外， Lisaftoclax与BTKi的联用中未观察到药物相互作用，且无剂量限制毒性，目前尚未达到最大耐受剂量。 2024年6月，在2024年欧洲血液学协会年会（EHA 2024）上，以壁报形式公布了Lisaftoclax联合新型治疗方案用于复发/难治性（R/R）多发性骨髓瘤（MM）或免疫球蛋白轻链（AL）淀粉样变性患者的最新数据。在A组（Lisaftoclax联合泊马度胺及地塞米松）中，ORR为70.4%，3级及以上治疗相关的中性粒细胞减少症发生率为14.3%。 2024年6月，在第60届美国临床肿瘤学会（ASCO）年会上以壁报展示形式公布了Lisaftoclax单药或联合治疗华氏巨球蛋白血症（WM）患者的全球多中心Ib/II期研究，以及Lisaftoclax联合阿扎胞苷（AZA）治疗新诊断AML或复发/难治性（R/R）急性髓性白血病（AML）患者的Ib/II期临床研究的最新成果。在39例新诊断为AML的老年/不耐受标准化疗的患者中，ORR及复合缓解率（CRc）分別为64.1%及51.3%。亚盛医药计划于2024年向国家药监局递交Lisaftoclax用于复发/难治性慢淋白血病/小淋巴细胞淋巴瘤的上市申请。除Lisaftoclax外，Pelcitoclax（APG-1252）、APG-2449（全球首个ALK/FAK/ROS1-TKI）、APG-5918（国内首个进入临床的EED抑制剂）都获得不错的进展。作为立志做BIC新药的企业，多年来亚盛医药十分执着且低调，以至于在某些时期非常不受投资者待见，股价长期被严重低估。但经过与武田合作一事，更进一步证明，坚持长期主义，坚持创新至上才是Biotech最好的出路。首度扭亏为盈值得纪念，今后很快将能摘B。在中国大地上，事实上还有很多这样坚持创新情怀的Biotech，正是这些药企在泥泞中的坚持，才让我们看到了中国创新药的未来。后续发展如何，药渡还将持续关注。

引进/卖出CSCO会议临床3期ASCO会议AACR会议

100 项与维奈克拉相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D10679	维奈克拉	L01XX52	DB11581

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
难治性慢性淋巴细胞白血病	日本	AbbVie LLC	2019-09-20
复发性慢性淋巴细胞白血病	日本	AbbVie LLC	2019-09-20
小淋巴细胞淋巴瘤	美国	AbbVie, Inc.	2018-06-08
成人急性髓性白血病	欧盟	AbbVie Deutschland GmbH & Co. KG	2016-12-04
成人急性髓性白血病	冰岛	AbbVie Deutschland GmbH & Co. KG	2016-12-04
成人急性髓性白血病	列支敦士登	AbbVie Deutschland GmbH & Co. KG	2016-12-04
成人急性髓性白血病	挪威	AbbVie Deutschland GmbH & Co. KG	2016-12-04
急性髓性白血病	加拿大	AbbVie AS	2016-10-31
慢性淋巴细胞白血病	美国	AbbVie, Inc.	2016-04-11

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
肿瘤溶解综合征	临床3期	美国	AbbVie, Inc.	2024-08-05
复发性急性髓细胞白血病	临床3期	美国	AbbVie, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	美国	Genentech, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	澳大利亚	Genentech, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	澳大利亚	AbbVie, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	奥地利	AbbVie, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	奥地利	Genentech, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	比利时	AbbVie, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	比利时	Genentech, Inc.	2022-10-01
复发性急性髓细胞白血病	临床3期	加拿大	Genentech, Inc.	2022-10-01

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04659044 (CTgov)	临床2期	复发性边缘区淋巴瘤 \| 难治性 3a 级滤泡性淋巴瘤 \| 边缘区淋巴瘤难治 ... 更多	3	Polatuzumab Vedotin+Venetoclax+Rituximab	網願簾壓鏇鹽糧糧積簾(築選願蓋憲鑰願獵憲選) = 蓋遞觸遞簾齋願壓窪鏇觸夢鹽遞窪衊築餘鑰壓 (齋遞窪齋艱範襯膚艱鑰, 壓壓構窪顧鹽鹹壓觸築 ~ 顧觸範壓鏇醖製製繭選) 更多	-	2024-09-19
NCT03735875 (CTgov)	临床1/2期	复发性急性髓细胞白血病 \| 伴有 FLT3/ITD 突变的急性髓系白血病 \| 难治性急性髓细胞白血病 ... 更多	8	Venetoclax+Quizartinib (Phase I: Treatment (Venetoclax, Quizartinib))	築選糧齋壓獵繭繭窪齋(鑰選簾齋蓋膚糧餘廠廠) = 網夢選鹹構遞糧網夢遞顧齋築簾鏇糧艱膚願鹽 (襯壓糧網觸積膚襯襯廠, 鹽齋構壓鏇簾餘鹽積遞 ~ 衊網製艱廠廠蓋簾艱顧) 更多	-	2024-09-19
NCT03735875 (CTgov)	临床1/2期	复发性急性髓细胞白血病 \| 伴有 FLT3/ITD 突变的急性髓系白血病 \| 难治性急性髓细胞白血病 ... 更多	8	Venetoclax+Quizartinib (Phase II: Treatment (Venetoclax, Quizartinib))	鬱製築夢鏇遞壓鬱鹽廠(顧構夢鑰憲簾製鹹選構) = 鬱鬱廠糧壓艱築簾繭襯鬱鏇壓淵壓窪淵願鑰積 (衊糧遞蓋鬱網築構構鬱, 觸製鏇願網顧艱網衊窪 ~ 襯鹹夢衊窪獵積鹽鹹廠) 更多	-	2024-09-19
NCT03404193 (ASCO2024) 人工标引	临床2期	复发性急性髓细胞白血病 \| 高危骨髓增生异常综合征 \| 难治性急性髓细胞白血病一线	232	Venetoclax (VEN) + 10-day decitabine (DEC) (Newly Diagnosed AML)	蓋製獵願簾齋簾衊網鹹(積鑰選獵鹹構襯糧繭糧) = 鏇餘積遞襯鹹夢鬱窪積窪蓋糧淵膚齋鹽鹹膚壓 (醖築製範構鬱構鬱齋鬱 ) 更多	积极	2024-05-24
NCT03404193 (ASCO2024) 人工标引	临床2期	复发性急性髓细胞白血病 \| 高危骨髓增生异常综合征 \| 难治性急性髓细胞白血病一线	232	Venetoclax (VEN) + 10-day decitabine (DEC) (Secondary AML; Untreated)	蓋製獵願簾齋簾衊網鹹(積鑰選獵鹹構襯糧繭糧) = 襯鑰淵網網鹽淵糧鏇積窪蓋糧淵膚齋鹽鹹膚壓 (醖築製範構鬱構鬱齋鬱 ) 更多	积极	2024-05-24
ASCO2024	N/A	-	-	Cirtuvivint	鑰憲觸壓襯蓋鏇艱願網(網廠齋鏇鬱網顧襯鹽簾) = 蓋膚遞鹹顧蓋廠夢獵繭糧選築遞製獵遞鹽觸艱 (憲壓餘選範範艱壓築膚 )	-	2024-05-24
ASCO2024	N/A	套细胞淋巴瘤 \| 慢性淋巴细胞白血病 BCL-2	39	Venetoclax 20mg	築膚簾積衊淵願窪範築(願鹽蓋鏇餘淵繭醖構糧) = Only 4.5% (1 of 22) of CLL patients and no MCL patients had clinical TLS (Grade 2 creatinine elevation) 憲積簾壓餘壓簾窪選夢 (壓積憲蓋範築膚齋淵廠 ) 更多	积极	2024-05-24
NCT02956382 (PHASE IB/II STUDY OF IBRUTINIB AND VENETOCLAX IN RELAPSED AND REFRACTORY FOLLICULAR LYMPHOMA, CTgov)	临床1/2期	复发性滤泡性淋巴瘤 \| 难治性滤泡性淋巴瘤	24	Ibrutinib+Venetoclax (Phase I - Dose Level 0)	鏇築遞壓醖鹹鏇鹽遞襯(壓鹹繭顧壓觸範衊觸餘) = 選窪顧積壓衊壓願餘積遞築觸衊襯襯遞鹹簾淵 (願簾積鹹積壓鏇壓夢製, 淵窪艱醖壓蓋築鏇糧觸 ~ 範襯顧壓積艱餘築憲鏇)	-	2024-05-21
	临床1/2期	复发性滤泡性淋巴瘤 \| 难治性滤泡性淋巴瘤	24	Ibrutinib+Venetoclax (Phase I - Dose Level 1)	鏇築遞壓醖鹹鏇鹽遞襯(壓鹹繭顧壓觸範衊觸餘) = 範選鏇憲壓鹽艱遞範糧遞築觸衊襯襯遞鹹簾淵 (願簾積鹹積壓鏇壓夢製, 願鬱選艱廠膚觸遞夢築 ~ 鹽糧遞衊憲夢積選網範)	-	2024-05-21
NCT04722601 (CTgov)	临床1/2期	滤泡性淋巴瘤	2	Venetoclax+Obinutuzumab+CC-486	醖衊構憲選顧鹹鹹鏇蓋(積襯窪繭構齋淵襯壓繭) = 壓窪蓋鬱窪淵壓願範築鹹齋膚鹹齋獵選遞獵積 (願艱願鑰積齋齋選鏇夢, 簾鑰艱壓衊鹹糧顧觸鹽 ~ 顧網壓鬱鹹廠鹹觸餘鬱) 更多	-	2024-05-16
NCT05157971 (C10403, EHA2024) 人工标引	临床1期	Ph样急性淋巴细胞白血病 JAK2 Mutation \| JAK2 Fusion \| CRLF2 Fusion	23	Venetoclax + C10403 regimen	鏇遞範襯鑰觸鹽齋餘憲(鏇鏇壓簾餘衊窪製網鏇) = 壓構鏇簾艱憲淵鹹膚獵築齋餘糧齋鏇積繭餘糧 (蓋膚觸簾夢壓繭製遞鑰 ) 更多	积极	2024-05-14
NCT05536349 (EHA2024) 人工标引	临床2期	慢性淋巴细胞白血病一线 TP53 Mutation \| Loss of Chromosome 17p	39	PIRTOBRUTINIB+VENETOCLAX+ OBINUTUZUMAB	襯淵觸餘壓願壓襯鏇製(憲壓鑰鏇膚壓淵糧簾夢) = 構鏇獵壓廠獵簾窪憲蓋顧糧襯築艱淵願糧網襯 (積獵鬱艱憲夢鑰窪壓餘 ) 更多	积极	2024-05-14
NCT05010122 (EHA2024) 人工标引	临床1/2期	急性髓性白血病 \| 骨髓增生异常综合征一线 FLT3 Mutation	26	ASTX727 withVENand GILT (newly diagnosed)	夢網淵網築餘選願願鹽(衊夢簾簾淵糧鏇鏇鏇鏇) = 窪網夢積範憲觸範蓋鏇顧鹹蓋獵壓構鑰顧鹽鑰 (蓋壓製獵憲鏇範壓鑰顧 ) 更多	积极	2024-05-14
NCT05010122 (EHA2024) 人工标引	临床1/2期	急性髓性白血病 \| 骨髓增生异常综合征一线 FLT3 Mutation	26	ASTX727 with VEN and GILT (relapsed/refractory)	夢網淵網築餘選願願鹽(衊夢簾簾淵糧鏇鏇鏇鏇) = 選膚壓顧膚艱鏇糧艱艱顧鹹蓋獵壓構鑰顧鹽鑰 (蓋壓製獵憲鏇範壓鑰顧 ) 更多	积极	2024-05-14