SODIUM 2,2-DIMETHYLBUTYRATE

Investigational

Details

Stereochemistry	ACHIRAL
Molecular Formula	C6H11O2.Na
Molecular Weight	138.1401
Optical Activity	NONE
Defined Stereocenters	0 / 0
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure of SODIUM 2,2-DIMETHYLBUTYRATE

Structure Search

SMILES

[Na+].CCC(C)(C)C([O-])=O

InChI

InChIKey=OOPXYEYPPHJHSE-UHFFFAOYSA-M

InChI=1S/C6H12O2.Na/c1-4-6(2,3)5(7)8;/h4H2,1-3H3,(H,7,8);/q;+1/p-1

HIDE SMILES / InChI

Molecular Formula	C6H11O2
Molecular Weight	115.1503
Charge	-1
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Molecular Formula	Na
Molecular Weight	22.98976928
Charge	1
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

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
Fetal globin gene inducers: novel agents and new potential.	2010-08	20712788
Beta-thalassemia.	2010-05-21	20492708
Biotechnological production and applications of statins.	2010-01	19820926
Liquid chromatography-mass spectrometric assay for quantitation of the short-chain fatty acid, 2,2-dimethylbutyrate (NSC 741804), in rat plasma.	2008-02-01	18155649
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
A top-down systems biology view of microbiome-mammalian metabolic interactions in a mouse model.	2007	17515922
Novel short chain fatty acids restore chloride secretion in cystic fibrosis.	2006-03-31	16472777
New polynuclear manganese clusters from the use of the hydrophobic carboxylate ligand 2,2-dimethylbutyrate.	2005-06-27	15962962
Simvastatin.	2003-08	12909768
[Chemical components of essential oils from the herb of Ligularia virgaurea].	2003-07	15139106
Differential effects of short-chain fatty acids on head and neck squamous carcinoma cells.	2002-04	12150517

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

Substance Class	Chemical Created by `admin` on `Mon Mar 31 18:29:50 GMT 2025` Edited by `admin` on `Mon Mar 31 18:29:50 GMT 2025`
Record UNII	XKM99SPB5I
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

HQK-1001

Preferred Name

English

SODIUM 2,2-DIMETHYLBUTYRATE

Systematic Name

English

Dimebutate sodium

Common Name

English

SODIUM 2,2-DIMETHYLBUTANOATE

Systematic Name

English

2,2-DIMETHYLBUTYRIC ACID, SODIUM SALT

Common Name

English

BUTANOIC ACID, 2,2-DIMETHYL-, SODIUM SALT

Common Name

English

SDMB

Common Name

English

SODIUM 2,2 DIMETHYLBUTYRATE

Systematic Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

265108