Details
| Stereochemistry | ABSOLUTE |
| Molecular Formula | C16H25NO2 |
| Molecular Weight | 263.3752 |
| Optical Activity | UNSPECIFIED |
| Defined Stereocenters | 1 / 1 |
| E/Z Centers | 0 |
| Charge | 0 |
SHOW SMILES / InChI
SMILES
NCC[C@@H](O)C1=CC=CC(OCC2CCCCC2)=C1
InChI
InChIKey=WJIGGYYSZBWCGC-MRXNPFEDSA-N
InChI=1S/C16H25NO2/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/t16-/m1/s1
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 |
|---|---|---|---|
Target ID: Q16518 Gene ID: 6121.0 Gene Symbol: RPE65 Target Organism: Homo sapiens (Human) Sources: https://www.ncbi.nlm.nih.gov/pubmed/25970164 |
4.4 nM [IC50] |
Sample Use Guides
In Vivo Use Guide
Sources: https://www.ncbi.nlm.nih.gov/pubmed/25932553
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
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FDA ORPHAN DRUG |
548816
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1141777-14-1
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C171669
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02DZ1HBF0M
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Emixustat
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100000177199
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ACTIVE MOIETY
SALT/SOLVATE (PARENT)