Ziftomenib

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

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

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]