afegostat-hcl

Description: Afegostat HCl (Isofagomine HCl, AT2101), designed as a pharmacological chaperone, is an investigational drug for the treatment ofcertain forms of Gaucher's disease. Isofagomine (IFG) is an acidbeta-glucosidase (GCase) active site inhibitor that acts as apharmacological chaperone. FG inhibits GCase with K(i) ca.30 nM forwild-type and mutant enzymes (N370S and V394L). Administration of IFG(30 mg/kg/day) to the mice homozygous for GCase mutations (V394L, D409H, or D409V) led to increased GCase activity in visceral tissues and brainextracts.

References: RichterF, et al. A GCase chaperone improves motor function in a mouse model ofsynucleinopathy. Neurotherapeutics. 2014 Oct; 11(4):840-56; Front S, Almeida S, Zoete V, Charollais-Thoenig J, Gallienne E, Marmy C, PilloudV, Marti R, Wood T, Martin OR, Demotz S. 4-epi-Isofagomine derivativesas pharmacological chaperones for the treatment of lysosomal diseaseslinked to beta-galactosidase mutations: Improved synthesis and biologicalinvestigations. Bioorg Med Chem. 2018 Nov 1; 26(20):5462-5469.

Related CAS #: 161302-93-8 (HCl) 1084896-48-9 (acetate) 957230-65-8 (tartrate) 169105-89-9 (free base) 1084896-50-3 (TFA)