gintemetostat

Gintemetostat (KTX-1001) is a first-in-class, potent, and selective oral small molecule inhibitor of the MMSET (Multiple Myeloma SET domain)/NSD2 (Nuclear receptor binding SET domain protein 2) histone methyltransferase. Developed by K36 Therapeutics, it specifically targets the catalytic SET domain of MMSET, which is overexpressed in approximately 15-20% of multiple myeloma patients due to the t(4;14) chromosomal translocation. By inhibiting the methyltransferase activity, gintemetostat aims to reprogram myeloma cells, decrease oncogenic signaling, and reverse the high-risk epigenetic state associated with this translocation. It is currently being evaluated in Phase 1 clinical trials for relapsed and refractory multiple myeloma, both as a monotherapy and in combination with other standard-of-care agents such as proteasome inhibitors and IMiDs.

Other names

(S)-1-((R)-3-amino-1-(4-((6-amino-9H-purin-9-yl)methyl)-6-(2,5-difluoro-4-methoxyphenyl)pyridin-3-yl)piperidin-3-yl)-2,2-difluoroethan-1-olKTX-1001 D-tartrateKTX1001 D-tartrateKTX 1001 D-tartrateNSD2 inhibitor 1 D-tartrateNSD-2 inhibitor 1 D-tartrateNSD 2 inhibitor 1 D-tartrateKTX-1001 tartrate[4]