| Stereochemistry | ABSOLUTE |
| Molecular Formula | C16H25NO2.ClH |
| Molecular Weight | 299.836 |
| Optical Activity | UNSPECIFIED |
| Defined Stereocenters | 1 / 1 |
| E/Z Centers | 0 |
| Charge | 0 |
SHOW SMILES / InChI
SMILES
Cl.NCC[C@@H](O)C1=CC=CC(OCC2CCCCC2)=C1
InChI
InChIKey=BPZWRYOUJMDQSY-PKLMIRHRSA-N
InChI=1S/C16H25NO2.ClH/c17-10-9-16(18)14-7-4-8-15(11-14)19-12-13-5-2-1-3-6-13;/h4,7-8,11,13,16,18H,1-3,5-6,9-10,12,17H2;1H/t16-;/m1./s1
| Molecular Formula | ClH |
| Molecular Weight | 36.461 |
| Charge | 0 |
| Count |
MOL RATIO
1 MOL RATIO (average) |
| Stereochemistry | ACHIRAL |
| Additional Stereochemistry | No |
| Defined Stereocenters | 0 / 0 |
| E/Z Centers | 0 |
| Optical Activity | NONE |
| Molecular Formula | C16H25NO2 |
| Molecular Weight | 263.3752 |
| Charge | 0 |
| Count |
MOL RATIO
1 MOL RATIO (average) |
| Stereochemistry | ABSOLUTE |
| Additional Stereochemistry | No |
| Defined Stereocenters | 1 / 1 |
| E/Z Centers | 0 |
| Optical Activity | UNSPECIFIED |
Emixustat (formerly ACU-4429) is a nonretinoid compound with a unique mode of action in the retinal pigment epithelium, where it modulates the biosynthesis of visual chromophore through its effect on retinal pigment epithelium protein 65 (RPE65). Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway RPE65. Acucela Inc., a Kubota Pharmaceutical company, is developing emixustat for the treatment of Stargardt disease and proliferative diabetic retinopathy.
Originator
Approval Year
Targets
| Primary Target | Pharmacology | Condition | Potency |
|---|---|---|---|
| 4.4 nM [IC50] |
PubMed
Patents
Sample Use Guides
Phase ii, randomized, placebo-controlled, study of emixustat hydrochloride in geographic atrophy associated with dry age-related macular degeneration: Subjects were randomly assigned to oral emixustat (2, 5, 7, or 10 mg once daily) or placebo (3:1 ratio) for 90 days.
Route of Administration:
Oral
