U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ABSOLUTE
Molecular Formula C14H20ClN3O3
Molecular Weight 313.78
Optical Activity UNSPECIFIED
Defined Stereocenters 1 / 1
E/Z Centers 1
Charge 0

SHOW SMILES / InChI
Structure of ARIMOCLOMOL

SMILES

O[C@@H](CON=C(Cl)C1=CC=C[N+]([O-])=C1)CN2CCCCC2

InChI

InChIKey=SGEIEGAXKLMUIZ-CYBMUJFWSA-N
InChI=1S/C14H20ClN3O3/c15-14(12-5-4-8-18(20)9-12)16-21-11-13(19)10-17-6-2-1-3-7-17/h4-5,8-9,13,19H,1-3,6-7,10-11H2/t13-/m1/s1

HIDE SMILES / InChI

Description
Curator's Comment: description was created based on several sources, including https://www.ncbi.nlm.nih.gov/pubmed/27605553 | https://clinicaltrials.gov/ct2/show/NCT00706147 | https://clinicaltrials.gov/ct2/show/NCT00706147 | https://www.ncbi.nlm.nih.gov/pubmed/27273088

Arimoclomol (INN; originally codenamed BRX-345, which is a citrate salt formulation of BRX-220) is an experimental drug developed by a biopharmaceutical company CytRx Corporation. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS. The European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment for Niemann-Pick type C in 2014 and 2015 respectively. Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Unknown

Approved Use

Unknown
Primary
Unknown

Approved Use

Unknown
Primary
Unknown

Approved Use

Unknown
Doses

Doses

DosePopulationAdverse events​
100 mg 3 times / day multiple, oral
Highest studied dose
Dose: 100 mg, 3 times / day
Route: oral
Route: multiple
Dose: 100 mg, 3 times / day
Sources:
unhealthy
n = 20
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 20
Sources:
Disc. AE: pulmonary embolism, increased lower extremity weakness...
AEs leading to
discontinuation/dose reduction:
pulmonary embolism (1 pt)
increased lower extremity weakness (1 pt)
Sources:
25 mg 3 times / day multiple, oral (unknown)
Studied dose
Dose: 25 mg, 3 times / day
Route: oral
Route: multiple
Dose: 25 mg, 3 times / day
Sources:
unhealthy
n = 19
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 19
Sources:
Disc. AE: rash...
AEs leading to
discontinuation/dose reduction:
rash (1 pt)
Sources:
AEs

AEs

AESignificanceDosePopulation
increased lower extremity weakness 1 pt
Disc. AE
100 mg 3 times / day multiple, oral
Highest studied dose
Dose: 100 mg, 3 times / day
Route: oral
Route: multiple
Dose: 100 mg, 3 times / day
Sources:
unhealthy
n = 20
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 20
Sources:
pulmonary embolism 1 pt
Disc. AE
100 mg 3 times / day multiple, oral
Highest studied dose
Dose: 100 mg, 3 times / day
Route: oral
Route: multiple
Dose: 100 mg, 3 times / day
Sources:
unhealthy
n = 20
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 20
Sources:
rash 1 pt
Disc. AE
25 mg 3 times / day multiple, oral (unknown)
Studied dose
Dose: 25 mg, 3 times / day
Route: oral
Route: multiple
Dose: 25 mg, 3 times / day
Sources:
unhealthy
n = 19
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 19
Sources:
PubMed

PubMed

TitleDatePubMed
Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.
2016 Sep 7
Patents

Sample Use Guides

PO (3 times daily). Doses:150-600 mg/day (based on weight)
Route of Administration: Oral
Human SH-SY5Y cells were used for activity evaluation. For quantitative analysis, of cell viability by fluorescence microscopy, cells were harvested at the indicated time point after single or co-application of celastrol and Arimoclomol (50 or 250 mkM). An equal volume of cell suspension was mixed with 0.4 % trypan blue (T10282; Life Technologies) and incubated for 2 min. The cell suspension was loaded into a disposable Countess® cell counting chamber slide, and the percent of cells stained by trypan blue was quantified using a Countess® automated cell counter.
Name Type Language
ARIMOCLOMOL
INN   MI  
INN   USAN  
Official Name English
BRX-220 FREE BASE
Code English
N-[(2R)-2-Hydroxy-3-(1-piperidyl)propoxy]pyridine-3-carboximidoyl chloride, 1-oxide
Systematic Name English
ARIMOCLOMOL [USAN]
Common Name English
3-PYRIDINECARBOXIMIDOYL CHLORIDE, N-((2R)-2-HYDROXY-3-(1-PIPERIDINYL)PROPOXY), 1-OXIDE
Systematic Name English
ARIMOCLOMOL [MI]
Common Name English
Arimoclomol [WHO-DD]
Common Name English
arimoclomol [INN]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 611317
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
EU-Orphan Drug EU/3/06/406
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
FDA ORPHAN DRUG 202705
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
NCI_THESAURUS C2080
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
Code System Code Type Description
SMS_ID
100000172946
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
PUBCHEM
208924
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
DRUG BANK
DB05025
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
CAS
289893-25-0
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
EPA CompTox
DTXSID5057701
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
USAN
FG-164
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
ChEMBL
CHEMBL2107726
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
INN
8300
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
WIKIPEDIA
ARIMOCLOMOL
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
CAS
289893-23-8
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
NON-SPECIFIC STEREOCHEMISTRY
NCI_THESAURUS
C79744
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
FDA UNII
EUT3557RT5
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
MESH
C486387
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY
MERCK INDEX
m2045
Created by admin on Fri Dec 15 16:15:30 GMT 2023 , Edited by admin on Fri Dec 15 16:15:30 GMT 2023
PRIMARY Merck Index