EMIXUSTAT

Investigational

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

Structure Search

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

HIDE SMILES / InChI

Molecular Formula	C16H25NO2
Molecular Weight	263.3752
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	1 / 1
E/Z Centers	0
Optical Activity	UNSPECIFIED

Description
Sources: https://www.acucela.com/pipeline/index.html | https://www.ncbi.nlm.nih.gov/pubmed/25970164

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
Retinoid isomerohydrolase 3 Target ID: Q16518 Gene ID: 6121.0 Gene Symbol: RPE65 Target Organism: Homo sapiens (Human) Sources: https://www.ncbi.nlm.nih.gov/pubmed/25970164	Inhibitor 8504		4.4 nM [IC50]

PubMed

Title	Date	PubMed
Stargardt macular dystrophy and evolving therapies.	2018-10	30129371
Pharmacotherapy of retinal disease with visual cycle modulators.	2018-04	29542350

Patents

Sample Use Guides

In Vivo Use Guide

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

Substance Class	Chemical Created by `admin` on `Mon Mar 31 20:51:02 GMT 2025` Edited by `admin` on `Mon Mar 31 20:51:02 GMT 2025`
Record UNII	02DZ1HBF0M
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

emixustat [INN]

Preferred Name

English

EMIXUSTAT

Official Name

English

ACU-4429

Code

English

(1R)-3-Amino-1-[3-(cyclohexylmethoxy)phenyl]propan-1-ol

Systematic Name

English

EMIXUSTAT [USAN]

Common Name

English

(R)-(+)-3-AMINO-1-(3-(CYCLOHEXYLMETHOXY)PHENYL)PROPAN-1-OL

Systematic Name

English

BENZENEMETHANOL, .ALPHA.-(2-AMINOETHYL)-3-(CYCLOHEXYLMETHOXY)-, (.ALPHA.R)-

Common Name

English

Emixustat [WHO-DD]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

548816