Drug Type Small molecule drug |
Synonyms KO-381, KO-382, KO-539 + [3] |
Target |
Action inhibitors |
Mechanism MLL1 inhibitors(lysine methyltransferase 2A inhibitors), menin inhibitors(Menin inhibitors), Protein interaction domain and motifs inhibitors |
Therapeutic Areas |
Inactive Indication- |
Originator Organization |
Active 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) |
Molecular FormulaC33H42F3N9O2S2 |
InChIKeyBGGALFIXXQOTPY-NRFANRHFSA-N |
CAS Registry2134675-36-6 |
| Indication | Country/Location | Organization | Date |
|---|---|---|---|
| Acute myeloid leukemia with mutated NPM1 | United States | 13 Nov 2025 |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Acute Lymphoblastic Leukemia | Phase 2 | United States | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | Belgium | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | Canada | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | France | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | Germany | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | Italy | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | Poland | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | Spain | 12 Sep 2019 | |
| Acute Lymphoblastic Leukemia | Phase 2 | United Kingdom | 12 Sep 2019 | |
| Mixed phenotype acute leukemia | Phase 2 | United States | 12 Sep 2019 |
Phase 1/2 | Acute myeloid leukemia with mutated NPM1 NPM1-mutated | 92 | Ziftomenib 600 mg | unklleezdr(batouqzwqw) = hrqwhnjpdl fzbequwbow (nhwcssezdn, 14 - 32) View more | Positive | 01 Nov 2025 | |
Phase 1/2 | Acute myeloid leukemia with mutated NPM1 NPM1 Mutation | 112 | (Phase 1b/2) | iyyeszahkj(gwuozuzmfc) = edyphkhoct ytrqszzoom (oapypxfnmm, 17 - 34) View more | Positive | 30 May 2025 | |
(Phase 2) | iyyeszahkj(gwuozuzmfc) = xrpcbrpske ytrqszzoom (oapypxfnmm, 15 - 33) | ||||||
Phase 1/2 | Acute myeloid leukemia with mutated NPM1 NPM1-mutant | 112 | jfnpyioukz(gxqlwjodps) = yasjyplllz aiyjrragrb (iqtafifybq, 17 - 34) View more | 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 ) View more | Positive | 14 May 2025 | ||
Phase 1/2 | Acute Myeloid Leukemia NPM1 Mutation | - | 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 View more | Positive | 07 Feb 2025 | ||
Phase 1 | Acute myeloid leukemia with mutated NPM1 | Acute Leukemia with a KMT2A Translocation First line NPM1 Mutation | KMT2A Rearrangement | 54 | vpnjnsuybh(vppkxdtbid) = bfteepsttx mugfijpbcs (fupewmhyjc ) View more | Positive | 09 Dec 2024 | ||
vpnjnsuybh(vppkxdtbid) = mebnjboscu mugfijpbcs (fupewmhyjc ) View more | |||||||
Not Applicable | - | - | lkcjvpsgwb(enuurlyqvo) = rbejvnutod afozxmxons (gcrajwwwrt ) View more | - | 08 Dec 2024 | ||
lkcjvpsgwb(enuurlyqvo) = cxsioldhpe afozxmxons (gcrajwwwrt ) View more | |||||||
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 ) View more | Positive | 01 Oct 2024 | |
Phase 1 | Acute myeloid leukaemia with 11q23 abnormality | Acute myeloid leukemia with mutated NPM1 First line KMT2A Rearrangement | NPM1 Mutation | 20 | ziftomenib+SOC | 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+SOC (ziftomenib and 7+3) |





