UGT8-IN-1 is a brain-permeable and orally-active inhibitor that specifically targets the ceramide galactosyltransferase enzyme (UGT8). By inhibiting UGT8, this compound has the potential to be employed in research pertaining to lysosomal storage disorders.

UGT8-IN-1 (compound 19) exhibits the T1/2 of 4.3 h, 1.13 h and 13.6 h by iv (1 mg/kg) administration in rat, mouse and dog, respectively. UGT8-IN-1 (compound 19) shows F% of 40%, 34% and 52% by oral (3 mg/kg) administration in rat, mouse and dog, respectively[1]. UGT8-IN-1 (compound 19, orally twice a day for three days) shows ≥90% inhibition of incorporation of 13 C-Gal into GalCer and SFT, at all three doses tested, with the estimated ED 50 s of <3 mg/kg for inhibition of both SFT and GalCer. UGT8-IN-1 (compound 19) has very high plasma protein and tissue binding (>99%)[1].