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Azacytidine

Phase 2

Leukemia | Small molecule | Oncology |Bayer AG|Last Updated: Jan 14, 2020

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment16

FDA Designations

No designations recorded

Clinical trial landscape

Azacytidine · 1 trial · 1 indication

Phase 2 1
NCT02196857Sorafenib Plus 5-Azacitidine Initial Therapy of Patients With Acute Myeloid Leukemia (AML) and High Risk Myelodysplastic Syndrome (MS) With FLT3-ITD MutationLeukemia
COMPLETED16 Analytics
PHASE2COMPLETED
Sorafenib Plus 5-Azacitidine Initial Therapy of Patients With Acute Myeloid Leukemia (AML) and High Risk Myelodysplastic Syndrome (MS) With FLT3-ITD Mutation
LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Participants With a Response CR + PR + CRi
After 3, 28 day cycles

Criteria for response per the International Working Group for myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Complete Response (CR) was defined as \</= 5% blasts in the bone marrow (BM) with peripheral blood (PB) demonstrating greater thatn 1x10\^9/L platelets with no detectable extramedullary disease. CR with incomplete recovery of PB counts (CRi) is the above criteria but neutrophil or platelet counts less than the stated values. Partial Response (PR) required all of the hematologic values for a CR but with a reduction of at least 50% in the percentage of blasts to 5% to 25% in the bone marrow aspirate.

Secondary Endpoints

Toxicity Profile of Azacytidine and Sorafenib
After 3, 28 day cycles
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Azacytidine + SorafenibEXPERIMENTALAzacitidine 75 mg/m2 administered subcutaneously (SQ) or intravenously (IV) daily for 7 days per 28 day cycle. Sorafenib administered orally at a dose of 400 mg twice daily every day continuously.

Interventions

NameTypeDescription
AzacytidineDRUG75 mg/m2 administered subcutaneously (SQ) or intravenously (IV) on Days 1 - 7 for a 28 day cycle.
SorafenibDRUG400 mg by mouth twice daily about 12 hours apart, every day for a 28 day cycle.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Patients with untreated AML (\> or equal to 20% blasts in bone marrow and/or peripheral blood) or high risk MDS (\> or equal to 10% blasts in bone marrow). A. Patients with AML and history of MDS who have received prior therapy with a hypomethylating agent (including azacytid...

Countries:United States
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Competitive Landscape -Leukemia 327 trials

Top 20 of 63 competitors

CompanyTickerTrialsLead PhaseDrugs
Amgen Inc.AMGN8PHASE3Blinatumomab, Low-intensity chemotherapy regimen
Eli Lilly and CompanyLLY9PHASE3Pirtobrutinib, Idelalisib, Bendamustine, Rituximab
BeOne Medicines Ltd. Sponsored ADRONC13PHASE3Sonrotoclax, Zanubrutinib, Venetoclax, Obinutuzumab
AbbVie, Inc.ABBV24PHASE3Venetoclax
AstraZeneca PLCAZN18PHASE3Acalabrutinib, Rituximab, Chlorambucil
Merck & Co., Inc.MRK7PHASE3Nemtabrutinib, Fludarabine, Cyclophosphamide, Bendamustine, Rituximab
Johnson & JohnsonJNJ12PHASE3Ibrutinib, Venetoclax, Chlorambucil, Obinutuzumab
Novartis AG Sponsored ADRNVS21PHASE3Imatinib, Nilotinib, Bosutinib, Dasatinib, Asciminib
Kura Oncology, Inc.KURA5PHASE3Ziftomenib, Venetoclax, Azacitidine, Daunorubicin, Cytarabine
Nurix Therapeutics, Inc.NRIX5PHASE3NX-5948, Pirtobrutinib
Syndax Pharmaceuticals IncSNDX4PHASE3Revumenib, Intensive Chemotherapy Regimen
Takeda Pharmaceutical Co. Ltd. Sponsored ADRTAK1PHASE3Ponatinib, Imatinib, Vincristine, Dexamethasone, Cytarabine
SELLAS Life Sciences Group, Inc.SLS1PHASE3Galinpepimut-S, Azacitidine, Venetoclax, Decitabine, Cytarabine
Grifols, S.A. Sponsored ADR Class BGRFS1PHASE3Xembify
Bristol-Myers Squibb CompanyBMY7PHASE2Azacitidine
Pfizer Inc.PFE6PHASE2Gemtuzumab ozogamicine- Cytarabine- Gilteritinib
Ascentage Pharma Group International Unsponsored ADRAAPG11PHASE3Olverembatinib
Tango Therapeutics, Inc.TNGX7PHASE3AG-120, Azacitidine
Incyte CorporationINCY6PHASE2Ruxolitinib
Actinium Pharmaceuticals, Inc. (Delaware)ATNM2PHASE3Iomab-B, Conventional Care
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Frequently asked questions about Azacytidine

What is Azacytidine used for in leukemia?

Azacytidine is being studied for the treatment of leukemia, specifically in combination with sorafenib as initial therapy for patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MS) who have the FLT3-ITD mutation. It is an investigational small molecule in Phase 2 clinical development.

Who makes Azacytidine?

Azacytidine is being developed by Bayer AG, a company traded under the ticker BAYRY. The drug is currently in Phase 2 clinical development for the treatment of leukemia.

What phase is Azacytidine in?

Azacytidine is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. One Phase 2 clinical trial has been completed, and the drug is not yet available as a standard treatment.

What clinical trials is Azacytidine in?

Azacytidine has been studied in one completed Phase 2 clinical trial, NCT02196857. This trial evaluated sorafenib plus 5-azacitidine as initial therapy for patients with acute myeloid leukemia and high-risk myelodysplastic syndrome with the FLT3-ITD mutation. The trial enrolled 16 participants in the United States.

Is Azacytidine the same as 5-azacitidine?

Yes, Azacytidine is also known as 5-azacitidine. In clinical trial NCT02196857, the drug is referred to as 5-azacitidine, and it is being studied in combination with sorafenib for the treatment of leukemia.