Last update 17 Nov 2025

Ziftomenib

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
KO-381, KO-382, KO-539
+ [3]
Action
inhibitors
Mechanism
MLL1 inhibitors(lysine methyltransferase 2A inhibitors), menin inhibitors(Menin inhibitors), Protein interaction domain and motifs inhibitors
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
United States (13 Nov 2025),
RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union)
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Structure/Sequence

Molecular FormulaC33H42F3N9O2S2
InChIKeyBGGALFIXXQOTPY-NRFANRHFSA-N
CAS Registry2134675-36-6

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Acute myeloid leukemia with mutated NPM1
United States
13 Nov 2025
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Acute Lymphoblastic LeukemiaPhase 2
United States
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
Belgium
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
Canada
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
France
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
Germany
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
Italy
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
Poland
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
Spain
12 Sep 2019
Acute Lymphoblastic LeukemiaPhase 2
United Kingdom
12 Sep 2019
Mixed phenotype acute leukemiaPhase 2
United States
12 Sep 2019
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1/2
92
Ziftomenib 600 mg
unklleezdr(batouqzwqw) = hrqwhnjpdl fzbequwbow (nhwcssezdn, 14 - 32)
Positive
01 Nov 2025
Phase 1/2
112
(Phase 1b/2)
iyyeszahkj(gwuozuzmfc) = edyphkhoct ytrqszzoom (oapypxfnmm, 17 - 34)
Positive
30 May 2025
iyyeszahkj(gwuozuzmfc) = xrpcbrpske ytrqszzoom (oapypxfnmm, 15 - 33)
Phase 1/2
112
jfnpyioukz(gxqlwjodps) = yasjyplllz aiyjrragrb (iqtafifybq, 17 - 34)
Positive
22 May 2025
Phase 1
51
Ziftomenib 600 mg QD + 7+3 induction
hqgufseorj(hqcyzjdqig) = febrile neutropenia (47%), decreased platelet count (31%), anemia (22%), decreased neutrophil count (20%), decreased white blood cell count (16%) pvuvejcxkp (xrpimrhykp )
Positive
14 May 2025
Phase 1/2
-
gupkwgwyzv(uoeexvpmoj) = The KOMET-001 trial achieved its primary endpoint of CR plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. vdkacrkmlm (qflxvgliue )
Met
Positive
07 Feb 2025
Phase 1
54
vpnjnsuybh(vppkxdtbid) = bfteepsttx mugfijpbcs (fupewmhyjc )
Positive
09 Dec 2024
vpnjnsuybh(vppkxdtbid) = mebnjboscu mugfijpbcs (fupewmhyjc )
Not Applicable
-
-
lkcjvpsgwb(enuurlyqvo) = rbejvnutod afozxmxons (gcrajwwwrt )
-
08 Dec 2024
lkcjvpsgwb(enuurlyqvo) = cxsioldhpe afozxmxons (gcrajwwwrt )
Not Applicable
-
-
xmbdixnikn(fvtcndjpot) = ylumztciaj bfszsqcgnr (egsqpjtbtm )
-
07 Dec 2024
xmbdixnikn(fvtcndjpot) = yznzfcvmwr bfszsqcgnr (egsqpjtbtm )
Phase 1/2
Acute Myeloid Leukemia
NPM1 Mutation | KMT2A Rearrangement
83
Ziftomenib 50-1000 mg
zxtjmeacnc(qdsxgbcese) = the most common grade 3 or worse treatment-emergent adverse events were anaemia (20 [24%]), febrile neutropenia (18 [22%]), pneumonia (16 [19%]), differentiation syndrome (12 [15%]), thrombocytopenia (11 [13%]), and sepsis (ten [12%]). uvbvcmzgvz (lzndvjqwxo )
Positive
01 Oct 2024
Phase 1
20
qdkbhhpuaw(imswjvcmyw) = No differentiation syndrome events of any grade were reported, and no dose-limiting toxicities, evidence of QTc prolongation, drug-drug interactions or additive myelosuppression were observed. jzgedwsses (ijphhvxuvf )
Positive
30 Jan 2024
(ziftomenib and 7+3)
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Regulation

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