A Phase 2, Open-Label Study of Intravenous Pevonedistat in Combination With Oral Decitabine and Cedazuridine in Adult Patients With Higher-Risk Myelodysplastic Syndromes

The main aim of the study is to see if signs and symptoms of myelodysplastic syndromes disappear when treated with pevonedistat combined with decitabine and cedazuridine.
Participants will receive an infusion of pevonedistat 3 times during a 28-day cycle. They will also take decitabine and cedazuridine tablets once a day for the first 5 days of the same cycle. A minimum of 6 28-day cycles is recommended, but participants can stop treatment at any time.
A bone marrow biopsy, bone marrow aspirates, and blood samples will be collected during the study.
Participants will attend a follow-up visit 30 days after their last dose of pevonedistat. Once treatment has ended, participants will be followed up with either monthly clinic visits or will be contacted every 3 months.

Start Date01 Oct 2021

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

JPRN-jRCT2031210181

/ SuspendedPhase 2IIT

Phase II study of pevonedistat plus niraparib in patient with recurrent ovarian cancer

Start Date28 Sep 2021

Sponsor / Collaborator-

NCT04800627

/ TerminatedPhase 1/2IIT

A Phase I/II Trial of Pevonedistat in Combination With Pembrolizumab in Patients With dMMR/MSI-H Cancers

This phase I/II trial identifies the side effects and best dose of pevonedistat when given together with pembrolizumab in treating mismatch repair deficiency (dMMR)/high-frequency microsatellite instability (MSI-H) solid tumor that has spread to other places in the body (metastatic) or has spread to nearby tissue or lymph nodes (locally advanced) and cannot removed by surgery (unresectable). Pevonedistat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving pevonedistat and pembrolizumab may kill more tumor cells.

Start Date29 Mar 2021

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

100 Clinical Results associated with Pevonedistat

100 Translational Medicine associated with Pevonedistat

100 Patents (Medical) associated with Pevonedistat

730

Literatures (Medical) associated with Pevonedistat

31 Dec 2025·Hematology

Integrated transcriptome profiling and in vitro analysis reveals MLN4924’s role in inducing ferroptosis in acute myeloid leukemia

Article

Author: Zhao, Long ; Liu, Bei ; Guo, Yuancheng ; Jian, Jinli ; Tang, Xiao

OBJECTIVES:

While ferroptosis induction emerges as a therapeutic strategy for solid tumors, its role in acute myeloid leukemia (AML) remains unexplored. This study aimed to investigate the role of MLN4924 in modulating ferroptosis and its molecular targets in AML.

METHODS:

Transcriptome sequencing and bioinformatics analyses were performed to identify MLN4924 potential targets in ferroptosis. First, ferroptosis-related phenotypic assays were conducted, including assays of reactive oxygen species (ROS), glutathione (GSH), malondialdehyde (MDA), and Fe²⁺ levels. Second, cell viability assays were carried out with the combination of MLN4924 and ferroptosis inducers (Erastin, Sorafenib). Third, rescue experiments were used the ferroptosis inhibitor Ferrostatin-1 after MLN4924 treatment. In vivo efficacy was evaluated in NOD/SCID mice bearing AML xenografts treated with MLN4924, followed by tumor tissue analysis of GSH and Fe²⁺ levels, immunohistochemistry (IHC), and Western blotting for SLC7A11/GPX4 axis components.

RESULTS:

Transcriptome sequencing and bioinformatics analyses identified SLC7A11 and GPX4 as key MLN4924 target genes, both of which are glutathione-related proteins. MLN4924 significantly suppressed SLC7A11 and GPX4 expression, decreased GSH activity, and increased ROS, Fe²⁺, and MDA levels. Ferroptosis inducers (Erastin, Sorafenib) further enhanced the antileukemic activity of MLN4924, and ferroptosis inhibitor Ferrostatin-1 partially reversed this toxicity. In vivo, MLN4924 reduced tumor burden, accompanied by SLC7A11/GPX4 downregulation and Fe²⁺ accumulation in xenografts.

CONCLUSION:

This study provides the first evidence that MLN4924 triggers ferroptosis in AML by inhibiting the SLC7A11/GPX4 axis. These findings establish MLN4924 as a ferroptosis sensitizer through synergistic effects with ferroptosis inducers, supporting its therapeutic potential in AML.

01 Dec 2025·CANCER CHEMOTHERAPY AND PHARMACOLOGY

A phase I study of MLN4924 and belinostat in relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome

Article

Author: Harvey, R Donald ; Piekarz, Richard ; Bandyopadhyay, Dipankar ; Shafer, Danielle ; Wright, John ; Schaar, Dale ; Maher, Keri R ; Grant, Steven ; Rudek, Michelle A ; Deng, Xiaoyan

Abstract:

Purpose:

Relapsed and/or refractory acute myeloid leukemia and high-risk myelodysplastic syndrome continue to have a poor prognosis with limited treatment options despite advancements in rational combination and targeted therapies. Belinostat (an HDAC inhibitor) and Pevonedistat (a NEDD8 inhibitor) have each been independently studied in hematologic malignancies and have tolerable safety profiles with limited single-agent activity. Preclinical studies in AML cell lines and primary AML cells show the combination to be highly synergistic, particularly in high-risk phenotypes such as p53 mutant and FLT-3-ITD positive cells. Here, we present the safety, pharmacokinetics and pharmacodynamics of belinostat and pevonedistat in a dose escalation Phase I study in AML and High-Risk MDS.

Methods:

Eighteen patients (16 with AML, 2 with MDS) were treated at 5 dose levels (belinostat 800–1000 mg/m2, pevonedistat 20–50 mg/m2). Safety and tolerability were assessed according to protocol defined dose limiting toxicities (DLTs). Correlative pharmacokinetic and pharmacodynamic analyses were performed.

Results:

No dose limiting toxicities were noted. Most Grade 3 or 4 toxicities were hematologic in nature. The best response was stable disease in four patients, and complete remission in one patient who qualified as an exceptional responder. Pharmakokinetic studies revealed no association between drug exposure and best response. Pharmacodynamic RT-PCR studies demonstrated post-treatment increases in several proteins, including quantitative increases in the oxidative stress protein NQO1, ferroptosis protein SLC7A11, and GSR, linked to glutathione metabolism and oxidative stress, as did the anti-oxidants SRXN1 and TXNRD1.

Conclusions:

Patterns of post-treatment changes in correlative pharmacodynamic parameters may suggest possible mechanistic changes in the DNA damage response, oxidative damage, and ferroptosis pathways. The combination of pevonedistat plus belinosat is safe in an adult relapsed and/or refractory AML/High-Risk MDS population with modest but notable activity in this heavily treated, high risk population. Our findings also raise the possibility that certain extremely poor prognosis AML patients may respond to a regimen combining two targeted agents that have little or no activity when administered individually.

Trial registration:

ClinicalTrials.gov ID NCT03772925, first posted 12/12/2018; CTEP Identifier 10246.

01 Nov 2025·LIFE SCIENCES

MLN4924, neddylation inhibitor suppresses hypoxia induced retinal angiogenesis by targeting Human Antigen R signaling

Article

Author: NareshKumar, R N ; Majid, Nishath Fathima ; Mohan, Sruthi Priya ; Cheruvu, Sai Shreya ; Nirmal, Jayabalan ; Ranganathan, Sampathkumar ; Nagarajan, Hemavathy ; Ramasubramanyan, Sharada

PURPOSE:

Retinal hypoxia is a key pathological stimulus for neovascularization, leading to abnormal proliferation of blood vessels and vascular endothelial dysfunction leading to vision threatening conditions. The anti-angiogenic potential of MLN4924, a specific inhibitor of neddylation signaling has been evidenced in cancer cells, but remains abstract as therapy for ocular angiogenesis in normal retinal cells. The current work intended to delineate a novel molecular signaling cascade of combating retinal angiogenesis by inhibiting the neddylation-Human Antigen R (HuR) signaling pathway using MLN4924.

METHODS:

The effect of neddylation inhibition on hypoxia-induced HRMVEC was demonstrated by the real-time PCR, western blotting, immunofluorescence, and functional assays. In silico analysis was used to study the impact of the mutation of the RNA binding protein, HuR neddylation sites on its binding affinity towards VEGF mRNA. RNA immunoprecipitation and Actinomycin D experiments were performed to evidence the effect of neddylation inhibition on HuR binding and stabilization of VEGF mRNA in hypoxic HRMVECs.

RESULTS:

Functional angiogenic assays revealed that treatment with MLN4924 could suppress hypoxia-induced angiogenesis by reducing secretory VEGF levels without altering the barrier integrity of HRMVECs. In silico analysis revealed that mutation of two of the HuR neddylation sites decreased its binding affinity to 3'UTR region of VEGF mRNA. Indeed, HuR preferentially bound and stabilized VEGF mRNA upon hypoxia, which was significantly inhibited using MLN4924 in HRMVECs.

CONCLUSION:

Neddylation inhibition could suppress hypoxia-induced angiogenesis through HuR signaling without compromising the barrier integrity of HRMVECs.

News (Medical) associated with Pevonedistat

30 Jan 2023

·www.prnewswire.com

Acute Myeloid Leukemia Pipeline Experiences Momentum: DelveInsight Estimates a Diverse Pipeline Comprising 200+ Companies Working in the Domain

The incidence of AML has gradually increased around the globe. Males and older people had a higher possibility to develop AML. Developed countries tended to have higher age-standardized incidence rates and death rates than developing regions. Novel therapies for AML, including refinements of conventional cytotoxic chemotherapies, genetic and epigenetic targeted drugs, as well as immunotherapies, have significantly improved patient outcomes in recent years. LAS VEGAS, Jan. 30, 2023 /PRNewswire/ -- DelveInsight's ' Acute Myeloid Leukemia Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline acute myeloid leukemia therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the acute myeloid leukemia pipeline domain. Key Takeaways from the Acute Myeloid Leukemia Pipeline Report DelveInsight's acute myeloid leukemia pipeline report depicts a robust space with 200+ active players working to develop 260+ pipeline therapies for acute myeloid leukemia treatment. Key acute myeloid leukemia companies such as GlycoMimetics, CSPC ZhongQi Pharmaceutical Technology Co., Ltd., Takeda Oncology, Orca Bio, Gilead Sciences, Actinium Pharmaceuticals, Kronos Bio, Bristol-Myers Squibb, ImmunityBio, Bellicum Pharmaceuticals, Syros Pharmaceuticals, Armaceutica, Teva Pharmaceutical Industries, New Epsilon Innovation Limited, MediGene, TC Biopharm, PersonGen BioTherapeutics, Oncoceutics, Janssen Research & Development, LLC, Immune-Onc Therapeutics, Novartis, Jasper Therapeutics, Agastiya Biotech, Poseida Therapeutics, Molecular Partners, Allogene Therapeutics, and others are evaluating new acute myeloid leukemia drugs to improve the treatment landscape. Promising acute myeloid leukemia pipeline therapies in various stages of development include Uproleselan, SKLB1028, Pevonedistat, Orca T, Magrolimab, Iomab-B, Entospletinib, Nivolumab, ALT 803, BPX-501, TCB008, Tamibarotene, Pyronaridine, Trisenox (Arsenic Trioxide), NEI-01, MDG1011, OmnImmune, anti-FLT3 CAR-T, JNJ 63709178, IO 202, IDH 305, JSP 191, AB001, P-ckit-ALLO1, MP-0533, ALLO-316, ALL0-819, and others. In January 2023, Innate Pharma SA announced the publication in Nature Biotechnology of preclinical data showing the control of acute myeloid leukemia (AML) cells by a trifunctional NKp46-CD16a-NK cell engager (NKCE) targeting CD123. In January 2023, the FDA granted JBI-802 orphan drug designation for patients diagnosed with small cell lung cancer (SCLC) and acute myeloid leukemia (AML), according to a press release from Jubilant Therapeutics. In January 2022, Astex Pharmaceuticals, Inc. announced that the European Commission (EC) had granted orphan-drug designation (ODD) to the oral fixed-dose combination of decitabine and cedazuridine (ASTX727) for the treatment of Acute Myeloid Leukemia (AML). Request a sample and discover the recent advances in acute myeloid leukemia drug treatment @ Acute Myeloid Leukemia Pipeline Report The acute myeloid leukemia pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage acute myeloid leukemia drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the acute myeloid leukemia clinical trial landscape. Acute Myeloid Leukemia Overview Acute myeloid leukemia (AML) is the most common type of leukemia in adults, accounting for roughly 80% of all cases. Clonal expansion of immature "blast cells" in the peripheral blood and bone marrow results in ineffective erythropoiesis and bone marrow failure. There are various types of AML. Each affects a different type of blood cell. The acute myeloid leukemia symptoms you experience are determined by the type of blood cell that is affected. The common acute myeloid leukemia symptoms include tiredness, weakness, pale skin, irregular heartbeat, dizziness, and others. Many tests are used for acute myeloid leukemia diagnosis. Doctors will also perform tests to determine the subtype of AML. Chemotherapy is the primary treatment for most AML types, sometimes in conjunction with a targeted therapy drug. A stem cell transplant could follow this. Other drugs (aside from standard chemotherapy drugs) may be used to treat people with acute promyelocytic leukemia (APL). Find out more about drugs for acute myeloid leukemia @ New Acute Myeloid Leukemia Drugs A snapshot of the Acute Myeloid Leukemia Pipeline Drugs mentioned in the report: Learn more about the emerging acute myeloid leukemia pipeline therapies @ Acute Myeloid Leukemia Clinical Trials Acute Myeloid Leukemia Therapeutics Assessment The acute myeloid leukemia pipeline report proffers an integral view of acute myeloid leukemia emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Acute Myeloid Leukemia 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, Intravenous, Subcutaneous Therapeutics Assessment By Molecule Type: Small molecule, Cell therapy, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: Cell replacements, E-selectin inhibitors, Interleukin-15 receptor agonists, Immunologic cytotoxicity, T lymphocyte replacements, Bcr-abl tyrosine kinase inhibitors, Epidermal growth factor receptor antagonists, Fms-like tyrosine kinase 3 inhibitors, Lyn protein-tyrosine kinase inhibitors, NEDD 8 activating enzyme inhibitors Key Acute Myeloid Leukemia Companies: GlycoMimetics, CSPC ZhongQi Pharmaceutical Technology Co., Ltd., Takeda Oncology, Orca Bio, Gilead Sciences, Actinium Pharmaceuticals, Kronos Bio, Bristol-Myers Squibb, ImmunityBio, Bellicum Pharmaceuticals, Syros Pharmaceuticals, Armaceutica, Teva Pharmaceutical Industries, New Epsilon Innovation Limited, MediGene, TC Biopharm, PersonGen BioTherapeutics, Oncoceutics, Janssen Research & Development, LLC, Immune-Onc Therapeutics, Novartis, Jasper Therapeutics, Agastiya Biotech, Poseida Therapeutics, Molecular Partners, Allogene Therapeutics, and others. Key Acute Myeloid Leukemia Pipeline Therapies: Uproleselan, SKLB1028, Pevonedistat, Orca T, Magrolimab, Iomab-B, Entospletinib, Nivolumab, ALT 803, BPX-501, TCB008, Tamibarotene, Pyronaridine, Trisenox (Arsenic Trioxide), NEI-01, MDG1011, OmnImmune, anti-FLT3 CAR-T, JNJ 63709178, IO 202, IDH 305, JSP 191, AB001, P-ckit-ALLO1, MP-0533, ALLO-316, ALL0-819, and others. Dive deep into rich insights for new drugs for acute myeloid leukemia treatment; visit @ Acute Myeloid Leukemia Medications Table of Contents For further information on the acute myeloid leukemia pipeline therapeutics, reach out @ Acute Myeloid Leukemia Drug Treatment Related Reports Acute Myeloid Leukemia Market Acute Myeloid Leukemia Market Insight, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key acute myeloid leukemia companies, including Takeda Oncology, Immunity Bio, Teva Pharmaceuticals, among others. Relapsed/Refractory Acute Myeloid Leukemia Epidemiology Relapsed/Refractory Acute Myeloid Leukemia Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical, and forecasted relapsed/refractory acute myeloid leukemia epidemiology in the 7MM. Acute Lymphocytic Leukemia Pipeline Acute Lymphocytic Leukemia Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acute lymphocytic leukemia companies, including AbbVie, Novartis, Jazz Pharmaceutical, among others. Chronic Lymphocytic Leukemia Pipeline Chronic Lymphocytic Leukemia Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key chronic lymphocytic leukemia companies, including Celgene, Loxo Oncology, Octapharma, among others. Chronic Myeloid Leukemia Pipeline Chronic Myeloid Leukemia Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key chronic myeloid leukemia companies, including Kartos Therapeutics, Novartis, Incyte Corporation, among others. 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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 . Connect with us on LinkedIn |Facebook|Twitter Additionally, get in touch with our business executive to explore @ Healthcare Due Diligence Services Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

ImmunotherapyOrphan DrugASCO

14 Sep 2021

·www.biospace.com

Best Known for Therapeutics, Takeda Sees Growing Value in Vaccines

Tokyo-based Takeda Pharmaceutical announced that the company plans to grow its vaccines business. CEO Christophe Weber discussed this today in an interview at Reuters Events’ Pharma Japan 2021 conference. Takeda distributes Moderna’s COVID-19 vaccine in Japan but the company is better known for its cancer and gastrointestinal therapeutics than its vaccines. Vaccine production, “when established,” Weber said, “has a very long life. There is no generic of vaccines, for example. So, it’s a different type of lifecycle, but it can be a very good business if you bring innovation.” In May, the company presented long-term safety and efficacy data from its ongoing pivotal Phase III Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial for its TAK-003, a vaccine against dengue. The data has also been included in a submission to the European Medicines Agency (EMA). The data showed the vaccine provided 83.6% protection against hospitalization and 62% overall efficacy against dengue. Dengue is the fastest spreading mosquito-borne viral disease. It affects about 390 million people worldwide, with 500,000 hospitalizations. In addition to the Moderna COVID-19 vaccine, the company has a license to manufacture Novavax’s COVID-19 vaccine, which is still being evaluated in clinical trials. The Japanese government had entered a contract to acquire another 50 million Moderna doses to be delivered in 2022, as well as 150 million Novavax doses. The Novavax vaccine, TAK-019, is known outside Japan as NVX-CoV2373. As part of an earlier announcement, Takeda is developing manufacturing capabilities for TAK-109 in Japan with a goal of being distributed in early 2022. The licensing deal allows Takeda to manufacture the vaccine antigen, and Novavax is supplying the Matrix-M adjuvant to Takeda that is used in the vaccine. Takeda will handle Japanese clinical trial and regulatory submission to the Pharmaceuticals and Medical Devices Agency (PMDA) and distribute TAK-019 in Japan if it is approved by Japan’s Ministry of Health, Labour and Welfare (MHLW). In July 2021, Takeda and Frazier Healthcare Partners partnered to launch HilleVax, a biopharma company designed to develop and commercialize Takeda’s norovirus vaccine candidate. HilleVax received a license from Takeda for exclusive development and commercialization rights to the vaccine candidate, HIL-214, worldwide outside Japan, in exchange for an upfront fee, future cash milestones and royalties on net sales. Takeda will keep commercialization rights in Japan and HilleVax, will integrate some of the Japan development activities into its global development plan. Takeda also has vaccine programs for pandemic influenza and Zika. “Takeda and Frazier have a history of successfully partnering together, and we are confident in HilleVax’s capabilities to progress HIL-214, the most advanced norovirus vaccine candidate in development with the potential to address the huge global burden of norovirus-associated acute gastroenteritis,” said Rajeev Venkayya, President of the Global Vaccine Business Unit, Takeda. “This will allow Takeda to focus its efforts and resources on our dengue vaccine, which we have begun filing for licensure around the world, our pandemic programs, and our partnership with the U.S. Government to develop a Zika vaccine.” Weber was quick to note that the company has 40 clinical-stage compounds, with more than 10 in late-stage development. There was some concern when its Pevonedistat for blood cancer had poor clinical trial data. Weber said, “We don’t rely on one product in our pipeline, or two, when we have 40 in clinical stage.”

VaccineLicense out/inCollaborate

14 Sep 2021

·www.biospace.com

Best Known for Therapeutics, Takeda Sees Growing Value in Vaccines

Takeda announced that the company plans to grow its vaccines business. CEO Christophe Weber discussed this today in an interview at Reuters Events’ Pharma Japan 2021 conference. Tokyo-based Takeda Pharmaceutical announced that the company plans to grow its vaccines business. CEO Christophe Weber discussed this today in an interview at Reuters Events’ Pharma Japan 2021 conference. Takeda distributes Moderna’s COVID-19 vaccine in Japan but the company is better known for its cancer and gastrointestinal therapeutics than its vaccines. Vaccine production, “when established,” Weber said, “has a very long life. There is no generic of vaccines, for example. So, it’s a different type of lifecycle, but it can be a very good business if you bring innovation.” In May, the company presented long-term safety and efficacy data from its ongoing pivotal Phase III Tetravalent Immunization against Dengue Efficacy Study (TIDES) trial for its TAK-003, a vaccine against dengue. The data has also been included in a submission to the European Medicines Agency (EMA). The data showed the vaccine provided 83.6% protection against hospitalization and 62% overall efficacy against dengue. Dengue is the fastest spreading mosquito-borne viral disease. It affects about 390 million people worldwide, with 500,000 hospitalizations. In addition to the Moderna COVID-19 vaccine, the company has a license to manufacture Novavax’s COVID-19 vaccine, which is still being evaluated in clinical trials. The Japanese government had entered a contract to acquire another 50 million Moderna doses to be delivered in 2022, as well as 150 million Novavax doses. The Novavax vaccine, TAK-019, is known outside Japan as NVX-CoV2373. As part of an earlier announcement, Takeda is developing manufacturing capabilities for TAK-109 in Japan with a goal of being distributed in early 2022. The licensing deal allows Takeda to manufacture the vaccine antigen, and Novavax is supplying the Matrix-M adjuvant to Takeda that is used in the vaccine. Takeda will handle Japanese clinical trial and regulatory submission to the Pharmaceuticals and Medical Devices Agency (PMDA) and distribute TAK-019 in Japan if it is approved by Japan’s Ministry of Health, Labour and Welfare (MHLW). In July 2021, Takeda and Frazier Healthcare Partners partnered to launch HilleVax, a biopharma company designed to develop and commercialize Takeda’s norovirus vaccine candidate. HilleVax received a license from Takeda for exclusive development and commercialization rights to the vaccine candidate, HIL-214, worldwide outside Japan, in exchange for an upfront fee, future cash milestones and royalties on net sales. Takeda will keep commercialization rights in Japan and HilleVax, will integrate some of the Japan development activities into its global development plan. Takeda also has vaccine programs for pandemic influenza and Zika. “Takeda and Frazier have a history of successfully partnering together, and we are confident in HilleVax’s capabilities to progress HIL-214, the most advanced norovirus vaccine candidate in development with the potential to address the huge global burden of norovirus-associated acute gastroenteritis,” said Rajeev Venkayya, President of the Global Vaccine Business Unit, Takeda. “This will allow Takeda to focus its efforts and resources on our dengue vaccine, which we have begun filing for licensure around the world, our pandemic programs, and our partnership with the U.S. Government to develop a Zika vaccine.” Weber was quick to note that the company has 40 clinical-stage compounds, with more than 10 in late-stage development. There was some concern when its Pevonedistat for blood cancer had poor clinical trial data. Weber said, “We don’t rely on one product in our pipeline, or two, when we have 40 in clinical stage.”

VaccinePhase 3License out/inClinical Result

100 Deals associated with Pevonedistat

External Link

KEGG	Wiki	ATC	Drug Bank
D10413	Pevonedistat	-	DB11759

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Acute Myeloid Leukemia	Phase 3	United States	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	China	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	Japan	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	Australia	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	Belgium	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	Brazil	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	Canada	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	Czechia	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	France	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017
Acute Myeloid Leukemia	Phase 3	Germany	Takeda Pharmaceutical Co., Ltd.	28 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04175912 (EA2187, ASCO2025)	Phase 2	Intrahepatic Cholangiocarcinoma First line gemcitabine-based therapy	40	Pevonedistat monotherapy	yulxvsqbwk(cpexwexgje) = One patient on Arm B achieved stable disease lasting ≥24 weeks, corresponding to a CBR of 5.9% (95% CI: 0.1%-28.7%) vhztqokrhh (mnfgyiuvpx ) View more	Negative	30 May 2025
				Pevonedistat + Carboplatin + Paclitaxel
PB2774 (EHA2025)	Phase 1/2	Myelodysplastic Syndromes	770	Rigosertib	ennhyosxiq(stczofxpwn) = lkxyknyuqg jmsksidyho (zeswhhooln, 0.012 - 0.039) View more	Positive	14 May 2025
NCT04266795 (Pubmed \| Leuk Lymphoma)	Phase 2	Acute Myeloid Leukemia	-	Pevonedistat + Azacitidine + Venetoclax	hquhrkwzhf(ztflnzovje) = ksvzwrpuib zsnsuvywbq (jznumtnnpm ) View more	Negative	28 Nov 2024
				Azacitidine + Venetoclax	hquhrkwzhf(ztflnzovje) = sdnuharorh zsnsuvywbq (jznumtnnpm ) View more
NCT03319537 (CTgov)	Phase 1/2	Mesothelioma, Malignant \| Malignant Mesothelioma of Peritoneum \| Malignant Pleural Mesothelioma	9	pevonedistat (Pevonedistat)	ouspvcyngy = fcjsqyubjo rifedleoew (nuzlhdqfqw, tvmjolhtmh - qseczuusey) View more	-	23 May 2024
				cisplatin+pevonedistat+pemetrexed (Pevonedistat in Combination With Pemetrexed and Cisplatin)	vuhdqifljw(rvfanzcbwe) = zvgyvcbnhr ypjvjqusuq (qioxvtsalt, bzfglbxbvk - cnpgfupnfn) View more
NCT04090736 (PETHEMA PEVOLAM, EHA2024)	Phase 3	Acute Myeloid Leukemia	310	Pevonedistat (PEVO) + Azacitidine (AZA)	mcybkoxqzv(bxknijqqhm) = yhaenkccha owgohjkimi (pogexvmmqk ) View more	Negative	14 May 2024
				Azacitidine (AZA)	mcybkoxqzv(bxknijqqhm) = sifdenlxrb owgohjkimi (pogexvmmqk ) View more
NCT04172844 (Pubmed) Manual	Phase 1	Acute Myeloid Leukemia Second line MCL1	16	azacitidinet 1venetoclaxpevonedistat	fkwihtoqza(umkwzsnwjj) = mgnbqhlnwq dvmgfuergc (lvqmzthiys ) View more	Positive	04 Apr 2024
NCT04266795 (pevenaza, AACR2024) Manual	Phase 2	Acute Myeloid Leukemia First line FLT3 \| IDH2 Mutation \| ASXL1 \| ... View more	120	Venetoclax anAzacitidinene	lnjuriwyqa(czwegxdhqg) = hlskonozza uwednlcnoo (apvqfijrot ) View more	Positive	22 Mar 2024
				Venetoclax, AAzacitidine anPevonedistatat	sqyctdismx(ozdbxuyrnp) = yvvjfeatqc zgqatsekvq (kztymbfaka )
NCT03330821 (ASH2023)	Phase 1/2	Acute Myeloid Leukemia	28	Pevonedistat	xlyxckctgc(bblfwpxfeq) = rfivoumwpi lezjipttpv (dxbihnrsjk ) View more	Positive	11 Dec 2023
NCT03238248 (CTgov)	Phase 2	Myeloproliferative Disorders \| Myelodysplastic Syndromes	71	Bone Marrow Biopsy & Aspirate+Pevonedistat Infusion+Azacitidine Subcutaneous Injection or Intravenous Infusion	hhtsoypwts(shiidhlswn) = cnavorbhrv wjcfrovtvo (cwwvnwwclo, tztyxtozjj - cqnbimgtua) View more	-	21 Nov 2023
NCT02782468 (CTgov)	Phase 1	Acute Myeloid Leukemia \| Myelodysplastic Syndromes	23	Pevonedistat (Single Agent Arm: Pevonedistat 25 mg/m^2)	jxmtxswcpc = qanzhrxwwx wbeyhhnmvq (nzhtmajwzw, rcvyzoshbf - ufhvjxncdx) View more	-	03 Nov 2023
				Pevonedistat (Single Agent Arm: Pevonedistat 44 mg/m^2)	jxmtxswcpc = vrsuenxpse wbeyhhnmvq (nzhtmajwzw, mptucuoxfc - iukqdhvgtl) View more

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