molecules of the month

“Compound 19”

brain pen. UGT8 galactosyltransferase inh.

excellent oral PK/PD in brain model

from 30k cmpd cellular MTS and optimization

J. Med. Chem., Jun. 26, 2020

Sanofi R&D, Waltham, MA

Structure of compound 19
1 min read

“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.

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