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
“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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