Gildeuretinol Acetate

Details

Stereochemistry	ACHIRAL
Molecular Formula	C22H32O2
Molecular Weight	331.5068
Optical Activity	NONE
Defined Stereocenters	0 / 0
E/Z Centers	4
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

[2H]C([2H])([2H])C(=C/COC(C)=O)\C=C\C=C(C)\C=C\C1=C(C)CCCC1(C)C

InChI

InChIKey=QGNJRVVDBSJHIZ-QJZZLBIKSA-N

InChI=1S/C22H32O2/c1-17(9-7-10-18(2)14-16-24-20(4)23)12-13-21-19(3)11-8-15-22(21,5)6/h7,9-10,12-14H,8,11,15-16H2,1-6H3/b10-7+,13-12+,17-9+,18-14+/i2D3

HIDE SMILES / InChI

Description
Sources: http://grantome.com/grant/NIH/R44-EY021988-02A1 | http://www.alkeuspharma.com/stargardt.html | https://www.ncbi.nlm.nih.gov/pubmed/28285324

Alkeus Pharma's lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to prevent the formation of these toxic vitamin A dimers in the eye. ALK-001 is a chemically-modified vitamin A, in which 3 hydrogen atoms have been replaced by 3 deuterium atoms at carbon number 20. Replacing the retina's vitamin A with ALK-001 slows the formation of toxic vitamin A dimers. ALK-001 is in phase II clinical trials for the treatment of Stargardt's disease. The compound was co-developed by Alkeus Pharmaceuticals and Columbia University.

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Vitamin A dimerization 1 Target ID: Vitamin A dimerization Sources: https://www.ncbi.nlm.nih.gov/pubmed/28285324 \| http://www.alkeuspharma.com/preclinical.html	Inhibitor 8297

Conditions

Condition	Modality	Targets	Highest Phase	Product
Stargardt disease 1 Sources: http://www.alkeuspharma.com/trials.html	Primary		Phase II 1132	Unknown Approved Use Unknown

PubMed

Title	Date	PubMed
Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization.	2015 Jul 7	26106163
Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies?	2016	26427432
Novel therapeutics for Stargardt disease.	2017 Jun	28285324

Sample Use Guides

In Vivo Use Guide

Mice: C20-D3-retinyl acetate (ALK-001) was blended with olive oil, and added to a grain-based diet at 15,000 IU/kg diet otherwise deficient in vitamin A.

Route of Administration: Oral

In Vitro Use Guide

Unknown

Name

Type

Language

Gildeuretinol Acetate

Official Name

English

ALK-001

Code

English

VITAMIN A ACETATE C20-D3

Common Name

English

C20-D3-RETINYL ACETATE

Common Name

English

(2E,4E,6E,8E)-7-methyl-3-(methyl-d3)-9-(2,6,6-trimethylcyclohex-1-en-1-yl)nona-2,4,6,8-tetraen-1-yl acetate

Systematic Name

English

Retin-20,20,20-d3-ol, acetate

Systematic Name

English

Retin-20,20,20-d3-ol, 15-acetate

Systematic Name

English

GILDEURETINOL ACETATE [USAN]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

317210