HALOFUGINONE HYDROBROMIDE

Investigational

Details

Stereochemistry	RACEMIC
Molecular Formula	C16H17BrClN3O3.BrH
Molecular Weight	495.593
Optical Activity	( + / - )
Defined Stereocenters	2 / 2
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

Br.O[C@H]1CCCN[C@@H]1CC(=O)CN2C=NC3=C(C=C(Cl)C(Br)=C3)C2=O

InChI

InChIKey=SJUWEPZBTXEUMU-LIOBNPLQSA-N

InChI=1S/C16H17BrClN3O3.BrH/c17-11-6-13-10(5-12(11)18)16(24)21(8-20-13)7-9(22)4-14-15(23)2-1-3-19-14;/h5-6,8,14-15,19,23H,1-4,7H2;1H/t14-,15+;/m1./s1

HIDE SMILES / InChI

Molecular Formula	C16H17BrClN3O3
Molecular Weight	414.681
Charge	0
Count

Stereochemistry	RACEMIC
Additional Stereochemistry	No
Defined Stereocenters	2 / 2
E/Z Centers	0
Optical Activity	UNSPECIFIED

Molecular Formula	BrH
Molecular Weight	80.912
Charge	0
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/24703880
Curator's Comment: description was created based on several sources, including http://musculardystrophynews.com/2016/01/12/grunenthal-and-akashi- therapeutics-inc-announce-joint-drug-development-program-for-ht-100-in-the-treatment-of-patients-with-duchenne-muscular-dystrophy-dmd/

Halofuginone is a low molecular weight quinazolinone alkaloid, and a potent inhibitor of collagen alpha1(I) and matrix metalloproteinase 2 (MMP-2) gene expression. Halofuginone also effectively suppresses tumor progression and metastasis in mice. Halofuginone is a potent inhibitor of collagen a1(I) and matrix metalloproteinase 2 (MMP-2) gene expression. Halofuginone also suppresses extracellular matrix deposition and cell proliferation. Also was shown that halofuginone increased apoptosis in α smooth muscle actin- and prolyl 4-hydroxylase β-expressing cells in mdx diaphragm and in myofibroblasts, the major source of extracellular matrix. The profound antitumoral effect of halofuginone is attributed to its combined inhibition of tumour-stromal support, vascularization, invasiveness, and cell proliferation. HT-100 (delayed-release halofuginone), currently in clinical phase 1b/2a in five U.S. hospitals, is a small molecule drug candidate taken orally for the treatment of Duchenne muscular dystrophy (DMD) patients primarily through its ability to reduce fibrosis and inflammation and promote muscle fiber regeneration. The medicine candidate has been granted orphan drug designation in the U.S. and the EU — meaning it has been commercially undeveloped due to its limited profitability — and fast-track designation in the U.S. — an FDA process that aims to facilitate the development and patients’ reach to novel therapies for unmet medical needs.

Originator

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Matrix metalloproteinase-2 13 Target ID: CHEMBL333 Sources: https://www.ncbi.nlm.nih.gov/pubmed/11099465	Inhibitor 8504
Collagen alpha-1(I) chain 11 Target ID: P02452\|\|\|Q15176\|\|\|Q9UML6 Gene ID: 1277.0 Gene Symbol: COL1A1 Target Organism: Homo sapiens (Human) Sources: https://www.ncbi.nlm.nih.gov/pubmed/11099465	Inhibitor 8504

Conditions

Condition	Modality	Targets	Highest Phase	Product
Duchenne muscular dystrophy 18 Sources: https://www.ncbi.nlm.nih.gov/pubmed/24703880	Primary		Phase II 1147	Unknown Approved Use Unknown

C_max

Value	Dose	Analyte	Population
0.54 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	0.5 mg 1 times / day multiple, oral dose: 0.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
3.09 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	3.5 mg 1 times / day multiple, oral dose: 3.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
1.7 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	2 mg 1 times / day multiple, oral dose: 2 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
0.42 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	0.5 mg 2 times / day multiple, oral dose: 0.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED

AUC

Value	Dose	Analyte	Population
12.58 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	0.5 mg 1 times / day multiple, oral dose: 0.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
85.66 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	3.5 mg 1 times / day multiple, oral dose: 3.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
67.97 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	2 mg 1 times / day multiple, oral dose: 2 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
15.2 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	0.5 mg 2 times / day multiple, oral dose: 0.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED

T_1/2

Value	Dose	Analyte	Population
17.2 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	0.5 mg 1 times / day multiple, oral dose: 0.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
30.8 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	3.5 mg 1 times / day multiple, oral dose: 3.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
37 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	2 mg 1 times / day multiple, oral dose: 2 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED
25 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/16815702	0.5 mg 2 times / day multiple, oral dose: 0.5 mg route of administration: Oral experiment type: MULTIPLE co-administered:	HALOFUGINONE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED

Sourcing

Vendor/Aggregator	ID	URL
1PlusChem LLC	1P0039TA	https://www.1pchem.com/search?query=1P0039TA
A2B Chem	AB51886	http://a2bchem.com/prod/AB51886
Alfa Chemistry	17395-31-2	http://www.alfa-chemistry.com/search.aspx?q=17395-31-2
Ambeed	A162412	http://www.ambeed.com/search/Search.html?keyword=A162412
Angene	AGN-PC-0SJRAP	http://www.angenechemical.com/productshow/AGN-PC-0SJRAP.html
AstaTech	42217	http://astatechinc.com/CPSResult.php?CRNO=42217
AstaTech	44055	http://astatechinc.com/CPSResult.php?CRNO=44055
BLD Pharm	BD132622	http://www.bldpharm.com/search/Search.html?keyword=BD132622
Cayman Chemical	13370	http://www.caymanchem.com/app/template/Product.vm/catalog/13370
eMolecules	300127039	https://orderbb.emolecules.com/search/#?query=300127039
eMolecules	44463241	https://orderbb.emolecules.com/search/#?query=44463241
eMolecules	49264256	https://orderbb.emolecules.com/search/#?query=49264256
eMolecules	49853752	https://orderbb.emolecules.com/search/#?query=49853752
eMolecules	56972371	https://orderbb.emolecules.com/search/#?query=56972371
Hairui	HR141501	http://www.hairuichem.com/en/product/search.do?q=HR141501
Hairui	HR142335	http://www.hairuichem.com/en/product/search.do?q=HR142335
KeyOrganics	AS-35193	http://www.keyorganicsinc.com/bionet/catalogsearch/result?q=AS-35193
mcule	MCULE-6593934512	https://mcule.com/MCULE-6593934512/
MolPort	MolPort-008-266-629	https://www.molport.com/shop/molecule-link/MolPort-008-266-629
MolPort	MolPort-044-561-164	https://www.molport.com/shop/molecule-link/MolPort-044-561-164
MuseChem	R005528	https://www.musechem.com/product/R005528.html
MuseChem	R051332	https://www.musechem.com/product/R051332.html
Selleck Chemicals	S8144	http://www.selleckchem.com/search.html?searchDTO.searchParam=S8144
Tetrahedron	TS09180	http://www.thsci.com/search.do?q=TS09180
Toronto Research Chemicals	H102500	https://www.trc-canada.com/product-detail/?CatNum=H102500
Wonderchem	WD04XKX	http://www.wonder-chem.com/search.html?text=WD04XKX
Wonderchem	WD06IPV	http://www.wonder-chem.com/search.html?text=WD06IPV

PubMed

Title	Date	PubMed
Novel therapy in the treatment of scleroderma.	2001 Jan	11116279
Effect of halofuginone on the development of tight skin (TSK) syndrome.	2002 Jul	12482197
Hepatitis C and liver fibrosis.	2003 Jan	12655347
Halofuginone to treat fibrosis in chronic graft-versus-host disease and scleroderma.	2003 Jul	12869955
Prevalence and control of bovine cryptosporidiosis in German dairy herds.	2003 Mar 25	12623207
A medical compound derived from herbs.	2003 Mar 28	12663900
The production and characterisation of an antibody to detect the coccidiostat toltrazuril and its metabolite ponazuril.	2003 May	12790197
Development and validation of a method for the confirmation of halofuginone in chicken liver and eggs using electrospray tandem mass spectrometry.	2003 May 5	12668068
Examination of antimicrobial activity of selected non-antibiotic drugs.	2004 Dec	15909927
Residue analysis for halofuginone in sturgeon muscle by immunoaffinity cleanup and liquid chromatography.	2005 Nov-Dec	16526445
Involvement of the tyrosine phosphatase early gene of liver regeneration (PRL-1) in cell cycle and in liver regeneration and fibrosis effect of halofuginone.	2006 Jun	16508789
Halofuginone induces matrix metalloproteinases in rat hepatic stellate cells via activation of p38 and NFkappaB.	2006 Jun 2	16489207
Evolving concepts of liver fibrogenesis provide new diagnostic and therapeutic options.	2007 Jul 30	17663771
Involvement of ERK signaling in halofuginone-driven inhibition of fibroblast ability to contract collagen lattices.	2007 Nov 14	17706637
The liver pharmacological and xenobiotic gene response repertoire.	2008	18364709
The preventive effect of halofuginone on posterior glottic stenosis in a rabbit model.	2008 Jul	18585568
The effect of halofuginone, a specific inhibitor of collagen type 1 synthesis, in the prevention of pancreatic fibrosis in an experimental model of severe hyperstimulation and obstruction pancreatitis.	2008 Jul	18570924
Type I collagen expression contributes to angiogenesis and the development of deeply invasive cutaneous melanoma.	2008 Mar 1	17957794
Prevention of muscle fibrosis and improvement in muscle performance in the mdx mouse by halofuginone.	2008 Nov	18672370
Effectiveness of topically applied halofuginone in management of subglottic stenosis in rats.	2009 May	19393418

Patents

Sample Use Guides

In Vivo Use Guide

Unknown

Route of Administration: Oral

In Vitro Use Guide

Small molecule halofuginone (HF) (with a median inhibitory concentration (IC50) of 3.6 ± 0.4 nM) selectively inhibits mouse and human TH17 differentiation by activating a cytoprotective signaling pathway, the amino acid starvation response (AAR). Inhibition of TH17 differentiation by HF is rescued by the addition of excess amino acids and is mimicked by AAR activation after selective amino acid depletion. HF also induces the AAR in vivo and protects mice from TH17-associated experimental autoimmune encephalomyelitis

Substance Class	Chemical Created by `admin` on `Mon Mar 31 18:03:07 GMT 2025` Edited by `admin` on `Mon Mar 31 18:03:07 GMT 2025`
Record UNII	PTC2969MV1
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

STENOROL

Preferred Name

English

HALOFUGINONE HYDROBROMIDE

Official Name

English

4(3H)-QUINAZOLINONE, 7-BROMO-6-CHLORO-3-(3-(3-HYDROXY-2-PIPERIDINYL)-2-OXOPROPYL)-, HBR, TRANS-(±)-

Common Name

English

HALOFUGINONE HYDROBROMIDE [MART.]

Common Name

English

HALOFUGINONE HYDROBROMIDE [MI]

Common Name

English

NSC-713205

Code

English

(±)-TRANS-7-BROMO-6-CHLORO-3-(3-(3-HYDROXY-2-PIPERIDYL)-ACETONYL)-4(3H)-QUINAZOLINONE MONOHBR

Common Name

English

4(3H)-QUINAZOLINONE, 7-BROMO-6-CHLORO-3-(3-(3-HYDROXY-2-PIPERIDINYL)-2-OXOPROPYL)-, HYDROBROMIDE, TRANS-(±)-

Common Name

English

HALOFUGINONE HBR

Common Name

English

RU-19110

Code

English

(±)-TRANS-7-BROMO-6-CHLORO-3-(3-(3-HYDROXY-2-PIPERIDYL)-ACETONYL)-4(3H)-QUINAZOLINONE MONOHYDROBROMIDE

Systematic Name

English

HALOFUGINONE HYDROBROMIDE [USAN]

Common Name

English

HALOFUGINONE HYDROBROMIDE [GREEN BOOK]

Common Name

English

Classification Tree Code System Code

CFR

21 CFR 556.308