EMIXUSTAT HYDROCHLORIDE

Details

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

Structure Search

SMILES

Cl.NCC[C@@H](O)C1=CC(OCC2CCCCC2)=CC=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

HIDE SMILES / InChI

Molecular Formula	ClH
Molecular Weight	36.461
Charge	0
Count

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

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 8297		4.4 nM [IC50]

PubMed

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

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 `Sat Dec 16 01:47:00 GMT 2023` Edited by `admin` on `Sat Dec 16 01:47:00 GMT 2023`
Record UNII	AP4OF2M98B
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

EMIXUSTAT HYDROCHLORIDE

Official Name

English

BENZENEMETHANOL, .ALPHA.-(2-AMINOETHYL)-3-(CYCLOHEXYLMETHOXY)-, HYDROCHLORIDE (1:1), (.ALPHA.R)-

Systematic Name

English

ACU-4429 HCL

Code

English

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

Systematic Name

English

ACU-4429 HYDROCHLORIDE

Code

English

EMIXUSTAT HYDROCHLORIDE [USAN]

Common Name

English

Emixustat hydrochloride [WHO-DD]

Common Name

English

Classification Tree Code System Code

EU-Orphan Drug

EU/3/19/2162