molecules of the month

“Compound 19” is a selective brain-penetrant inhibitor of the ceramide galactosyltransferase UGT8, intended for use in substrate reduction therapy for rare lysosomal storage disorders. The compound has good oral exposure in higher species (%F = 52 in dog) and is active in models looking at enzymatic activity using isotopically-labeled substrate. The molecule possesses a rare alkyl -OCF3 functional group, which appears to have improved potency vs. other small lipophilic groups.