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 varied half-lives of 4.3 hours, 1.13 hours, and 13.6 hours in rats, mice, and dogs, respectively, following intravenous administration at 1 mg/kg. Its oral bioavailability (F%) at 3 mg/kg is 40%, 34%, and 52% in rats, mice, and dogs, respectively. Twice-daily oral doses of <3 mg/kg for three days achieve over 90% inhibition of 13C-Gal incorporation into GalCer and SFT, with estimated ED50s of <3 mg/kg for both targets. Additionally, UGT8-IN-1 exhibits extensive plasma protein and tissue binding, exceeding 99%.