Ziftomenib

Ziftomenib
Clinical data
Other names	KO-539; KO539
Legal status
Legal status	Investigational;
Identifiers
	IUPAC name 4-Methyl-5-[[4-[[2-(methylamino)-6-(2,2,2-trifluoroethyl)thieno[2,3-d]pyrimidin-4-yl]amino]piperidin-1-yl]methyl]-1-[(2S)-2-(4-methylsulfonylpiperazin-1-yl)propyl]indole-2-carbonitrile;
CAS Number	2134675-36-6 ;
PubChem CID	138497449 ;
IUPHAR/BPS	11680 ;
DrugBank	DB17171 ;
ChemSpider	115009296 ;
UNII	4MOD1F4ENC ;
KEGG	D12419 ;
ChEMBL	ChEMBL5095038 ;
PDB ligand	K5O ( PDBe , RCSB PDB );
Chemical and physical data
Formula	C33H42F3N9O2S2
Molar mass	717.88 g·mol−1
3D model (JSmol)	Interactive image ;
	SMILES CC1=C(C=CC2=C1C=C(N2C[C@H](C)N3CCN(CC3)S(=O)(=O)C)C#N)CN4CCC(CC4)NC5=C6C=C(SC6=NC(=N5)NC)CC(F)(F)F;
	InChI InChI=1S/C33H42F3N9O2S2/c1-21(43-11-13-44(14-12-43)49(4,46)47)19-45-25(18-37)15-27-22(2)23(5-6-29(27)45)20-42-9-7-24(8-10-42)39-30-28-16-26(17-33(34,35)36)48-31(28)41-32(38-3)40-30/h5-6,15-16,21,24H,7-14,17,19-20H2,1-4H3,(H2,38,39,40,41)/t21-/m0/s1; Key:BGGALFIXXQOTPY-NRFANRHFSA-N;

Last updated June 07, 2025

Ziftomenib is an experimental drug for the treatment of cancer. It is being studied for use in patients with relapsed or refractory acute myeloid leukaemia.^[1]^[2]

Ziftomenib blocks the interaction between two proteins, menin (MEN1) and KMT2A (also known as mixed lineage leukemia protein, MLL).^[3]^[4] This results in an inhibition of the proliferation of leukemic cells.

Ziftomenib has been granted breakthrough therapy designation by the Food and Drug Administration.^[5]

References

↑ Wang ES, Issa GC, Erba HP, Altman JK, Montesinos P, DeBotton S, et al. (October 2024). "Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial". The Lancet. Oncology. 25 (10): 1310–1324. doi:10.1016/S1470-2045(24)00386-3. PMID 39362248.
↑ "Ziftomenib Meets CR/CRh End Point in R/R NPM1+ AML". onclive.com. February 6, 2025.
↑ "Ziftomenib". NCI Cancer Dictionary. National Cancer Institute.
↑ Rausch J, Dzama MM, Dolgikh N, Stiller HL, Bohl SR, Lahrmann C, et al. (October 2023). "Menin inhibitor ziftomenib (KO-539) synergizes with drugs targeting chromatin regulation or apoptosis and sensitizes acute myeloid leukemia with MLL rearrangement or NPM1 mutation to venetoclax". Haematologica. 108 (10): 2837–2843. doi:10.3324/haematol.2022.282160. PMC 10543165 . PMID 37102614.
↑ "FDA Grants Breakthrough Therapy Designation to Ziftomenib in NPM1-Mutant AML". onclive.com. April 22, 2024.

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[1] Wang ES, Issa GC, Erba HP, Altman JK, Montesinos P, DeBotton S, et al. (October 2024). "Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial". The Lancet. Oncology. 25 (10): 1310–1324. doi:10.1016/S1470-2045(24)00386-3. PMID 39362248.

[2] "Ziftomenib Meets CR/CRh End Point in R/R NPM1+ AML". onclive.com. February 6, 2025.

[3] "Ziftomenib". NCI Cancer Dictionary. National Cancer Institute.

[4] Rausch J, Dzama MM, Dolgikh N, Stiller HL, Bohl SR, Lahrmann C, et al. (October 2023). "Menin inhibitor ziftomenib (KO-539) synergizes with drugs targeting chromatin regulation or apoptosis and sensitizes acute myeloid leukemia with MLL rearrangement or NPM1 mutation to venetoclax". Haematologica. 108 (10): 2837–2843. doi:10.3324/haematol.2022.282160. PMC 10543165 . PMID 37102614.

[5] "FDA Grants Breakthrough Therapy Designation to Ziftomenib in NPM1-Mutant AML". onclive.com. April 22, 2024.

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