Stauprimide is a non-broad spectrum inhibitor that binds to the MYC transcription factor NME2 and blocks its nuclear localization in ESCs, which causes down-regulation of MYC transcription.

Stauprimide (10 μM; 6 hours) inhibits MYC transcription in most cell lines tested (EC50: 30 nM-8 μM), reducing MYC levels by 15% to over 90%. At 5 μM for 3 hours, it suppresses MYC transcription by decreasing NME2 nuclear translocation, and at 2-8 μM for 24-72 hours, it down-regulates MYC, inhibiting cell proliferation in vitro (IC50: 780 nM in RXF 393 cells). Stauprimide (4-10 μM; 6 hours) exhibits various EC50s and degrees of MYC mRNA down-regulation across different cell lines [1].

Stauprimide (p.o.; 50 mg/kg; once daily; 30 days, 55 days) blocks tumor growth, decreases MYC protein levels in xenograft mouse with RXF 393 or CAKI-1 cells. It also inhibits MYC transcription in the RXF 393 tumor [1].