| Stereochemistry | ABSOLUTE |
| Molecular Formula | C6H13NO3 |
| Molecular Weight | 147.1723 |
| Optical Activity | UNSPECIFIED |
| Defined Stereocenters | 3 / 3 |
| E/Z Centers | 0 |
| Charge | 0 |
SHOW SMILES / InChI
SMILES
OC[C@H]1CNC[C@@H](O)[C@@H]1O
InChI
InChIKey=QPYJXFZUIJOGNX-HSUXUTPPSA-N
InChI=1S/C6H13NO3/c8-3-4-1-7-2-5(9)6(4)10/h4-10H,1-3H2/t4-,5-,6-/m1/s1
| Molecular Formula | C6H13NO3 |
| Molecular Weight | 147.1723 |
| Charge | 0 |
| Count |
MOL RATIO
1 MOL RATIO (average) |
| Stereochemistry | ABSOLUTE |
| Additional Stereochemistry | No |
| Defined Stereocenters | 3 / 3 |
| E/Z Centers | 0 |
| Optical Activity | UNSPECIFIED |
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
Approval Year
Sample Use Guides
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
