AFEGOSTAT TARTRATE

Investigational

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C6H13NO3.C4H6O6
Molecular Weight	297.2592
Optical Activity	UNSPECIFIED
Defined Stereocenters	5 / 5
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

OC[C@H]1CNC[C@@H](O)[C@@H]1O.O[C@H]([C@@H](O)C(O)=O)C(O)=O

InChI

InChIKey=ULBPPCHRAVUQMC-MUMXBIPUSA-N

InChI=1S/C6H13NO3.C4H6O6/c8-3-4-1-7-2-5(9)6(4)10;5-1(3(7)8)2(6)4(9)10/h4-10H,1-3H2;1-2,5-6H,(H,7,8)(H,9,10)/t4-,5-,6-;1-,2-/m11/s1

HIDE SMILES / InChI

Molecular Formula	C6H13NO3
Molecular Weight	147.1723
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	3 / 3
E/Z Centers	0
Optical Activity	UNSPECIFIED

Molecular Formula	C4H6O6
Molecular Weight	150.0868
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	2 / 2
E/Z Centers	0
Optical Activity	UNSPECIFIED

Description
Sources: https://adisinsight.springer.com/drugs/800023168 | http://xqdoc.imedao.com/165d869871d163a33fce5ca6.pdf | https://www.ncbi.nlm.nih.gov/pubmed/25037721

Amicus Therapeutics was investigating afegostat (isofagomine; Plicera; HGT-3410; AT-2101), an orally available pharmacological chaperone molecule, which binds to glucocerebrosidase (Gba) and stimulates proper folding and trafficking of the enzyme, for the potential treatment of Parkinsons disease(PD) and Gaucher disease. Afegostat specifically and reversibly binds GCase in the ER with highaffinity; this stabilizes the active form of the enzyme in the ER and increases trafficking of GCase to lysosomes. In 2006, Orphan Drug designations were granted in the U.S. and E.U. in 2006 and 2007, respectively. However, this research has been discontinued.

CNS Activity

Originator

Approval Year

PubMed

Title	Date	PubMed
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.	2011-02-24	21250698

Patents

Sample Use Guides

In Vivo Use Guide

For the first 2 weeks, afegostat tartrate was administered orally at a dose of 225 milligrams (mg) once daily (QD) for 7 consecutive days, followed by no study medication for 7 consecutive days. After 2 weeks, participants then took 225 mg afegostat tartrate QD for 3 consecutive days, followed by no study medication for 4 consecutive days. This 3-days-on/4-days-off treatment regimen was followed for 22 weeks.

Route of Administration: Oral

In Vitro Use Guide

Afegostat inhibits GCase with K(i) ~30 nM for wild-type and mutant enzymes (N370S and V394L)

Substance Class	Chemical Created by `admin` on `Mon Mar 31 18:40:18 GMT 2025` Edited by `admin` on `Mon Mar 31 18:40:18 GMT 2025`
Record UNII	285ZJJ9773
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

ISOFAGOMINE TARTRATE

Preferred Name

English

AFEGOSTAT TARTRATE

Official Name

English

PLICERA

Brand Name

English

AT2101

Code

English

AFEGOSTAT TARTRATE [MART.]

Common Name

English

(3R,4R,5R)-5-(Hydroxymethyl)piperidine-3,4-diol hydrogen (2R,3R)-2,3-dihydroxybutanedioate (salt)

Common Name

English

ISOFAGOMINE TARTRATE [MI]

Common Name

English

3,4-PIPERIDINEDIOL, 5-(HYDROXYMETHYL)-, (3R,4R,5R)-, (2R,3R)-2,3-DIHYDROXYBUTANEDIOATE (1:1)

Systematic Name

English

Afegostat tartrate [WHO-DD]

Common Name

English

AT-2101

Code

English

AFEGOSTAT TARTRATE [USAN]

Common Name

English

HGT-3410

Code

English

Classification Tree Code System Code

FDA ORPHAN DRUG

215605