CSRM617 hydrochloride is a selective small-molecule inhibitor of the transcription factor ONECUT2 (OC2, a master regulator of androgen receptor), with a Kd of 7.43 µM in SPR assays, directly binding to the OC2-HOX domain. CSRM617 hydrochloride induces apoptosis through the appearance of cleaved Caspase-3 and PARP and is well tolerated in the mouse model of prostate cancer [1].

CSRM617 hydrochloride, within a concentration range of 0.01-100 μM applied over 48 hours, effectively inhibits the growth of various prostate cancer (PC) cell lines, including PC-3, 22RV1, LNCaP, and C4-2 cells. Furthermore, at concentrations of 10-20 μM over the same duration, it triggers concentration-dependent apoptosis in 22Rv1 cells, evidenced by cell death. Notably, a higher concentration of 20 μM administered for 72 hours leads to apoptosis in 22Rv1 cells, marked by the presence of cleaved Caspase-3 and PARP.

CSRM617, administered orally at a dosage of 50 mg/kg daily for 20 days, significantly inhibits tumor growth in SCID mice with 22Rv1 xenografts. This regimen was observed to notably impede the development and proliferation of diffuse metastases in the tested animal model.