Dimebutic Acid

Details

Stereochemistry	ACHIRAL
Molecular Formula	C6H12O2
Molecular Weight	116.1583
Optical Activity	NONE
Defined Stereocenters	0 / 0
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

CCC(C)(C)C(O)=O

InChI

InChIKey=VUAXHMVRKOTJKP-UHFFFAOYSA-N

InChI=1S/C6H12O2/c1-4-6(2,3)5(7)8/h4H2,1-3H3,(H,7,8)

HIDE SMILES / InChI

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/23828223 | https://www.prnewswire.com/news-releases/hemaquest-pharmaceuticals-announces-first-patient-enrolled-in-phase-2-clinical-study-of-hqk-1001-in-patients-with-sickle-cell-disease-120655659.html | https://adisinsight.springer.com/drugs/800029658

2,2-DIMETHYLBUTYRIC ACID (HQK-1001) is an orally administered SCFAD (Short Chain Fatty Acid Derivative), which has shown an excellent safety profile and biologic effects on fetal hemoglobin induction and red blood cell production in the laboratory, relevant animal models, and in clinical trials carried out in healthy human subjects as well as patients with sickle cell disease and beta thalassemia. The compound has received Orphan Drug Designation in the United States and Europe for both sickle cell disease and beta thalassemia. HemaQuest Pharmaceuticals was developing HQK-1001 for the oral treatment of sickle cell anaemia and beta thalassaemia. HQK-1001 has been evaluated in phase II trials for beta thalassaemia and sickle cell anaemia.

Originator

Approval Year

PubMed

Title	Date	PubMed
Top-down systems biology integration of conditional prebiotic modulated transgenomic interactions in a humanized microbiome mouse model.	2008	18628745
Probiotic modulation of symbiotic gut microbial-host metabolic interactions in a humanized microbiome mouse model.	2008	18197175
Fetal globin gene inducers: novel agents and new potential.	2010 Aug	20712788
Biotechnological production and applications of statins.	2010 Jan	19820926
Beta-thalassemia.	2010 May 21	20492708

Patents

Sample Use Guides

In Vivo Use Guide

Hemoglobin SS or S/β(0) thalassemia: HQK-1001 was administered daily for 26 weeks at 30 mg/kg (n = 15), 40 mg/kg (n = 18) and 50 mg/kg (n = 19), either alone (n = 21) or with hydroxyurea (n = 31).

Route of Administration: Oral

Name

Type

Language

Dimebutic Acid

Official Name

English

NSC-16045

Code

English

NSC-741804

Code

English

2,2-DIMETHYLBUTANOIC ACID

Systematic Name

English

Butanoic acid, 2,2-dimethyl-

Systematic Name

English

Dimebutic acid [INN]

Common Name

English

2,2-DIMETHYLBUTYRIC ACID

Systematic Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

746120