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

Details

Stereochemistry ABSOLUTE
Molecular Formula C14H20ClN3O3.C6H8O7
Molecular Weight 505.903
Optical Activity UNSPECIFIED
Defined Stereocenters 1 / 1
E/Z Centers 1
Charge 0

SHOW SMILES / InChI
Structure of ARIMOCLOMOL CITRATE

SMILES

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

InChI

InChIKey=XSENLDLUMVYRET-BTQNPOSSSA-N
InChI=1S/C14H20ClN3O3.C6H8O7/c15-14(12-5-4-8-18(20)9-12)16-21-11-13(19)10-17-6-2-1-3-7-17;7-3(8)1-6(13,5(11)12)2-4(9)10/h4-5,8-9,13,19H,1-3,6-7,10-11H2;13H,1-2H2,(H,7,8)(H,9,10)(H,11,12)/t13-;/m1./s1

HIDE SMILES / InChI
Molecular Formula C14H20ClN3O3
Molecular Weight 313.78
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 1 / 1
E/Z Centers 1
Optical Activity UNSPECIFIED
Molecular Formula C6H8O7
Molecular Weight 192.1235
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

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 citrate 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.

CNS Activity

Originator

Approval Year

2024
711
Targets

Targets

Primary TargetPharmacologyConditionPotency
Activator
1447
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Phase III
665
Unknown

Approved Use

Unknown
Primary
Phase III
665
Unknown

Approved Use

Unknown
Primary
Phase III
665
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:
https://pubmed.ncbi.nlm.nih.gov/18551622/
unhealthy
Health Status: unhealthy
Sex: M+F
Food Status: UNKNOWN
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
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:
https://pubmed.ncbi.nlm.nih.gov/18551622/
25 mg 3 times / day multiple, oral
Studied dose
Dose: 25 mg, 3 times / day
Route: oral
Route: multiple
Dose: 25 mg, 3 times / day
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
unhealthy
Health Status: unhealthy
Sex: M+F
Food Status: UNKNOWN
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
Disc. AE: rash...
AEs leading to
discontinuation/dose reduction:
rash (1 pt)
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
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:
https://pubmed.ncbi.nlm.nih.gov/18551622/
unhealthy
Health Status: unhealthy
Sex: M+F
Food Status: UNKNOWN
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
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:
https://pubmed.ncbi.nlm.nih.gov/18551622/
unhealthy
Health Status: unhealthy
Sex: M+F
Food Status: UNKNOWN
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
rash 1 pt
Disc. AE
25 mg 3 times / day multiple, oral
Studied dose
Dose: 25 mg, 3 times / day
Route: oral
Route: multiple
Dose: 25 mg, 3 times / day
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
unhealthy
Health Status: unhealthy
Sex: M+F
Food Status: UNKNOWN
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
PubMed

PubMed

TitleDatePubMed
Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.
2016-09-07
Patents

Patents

20040006232
3
WO2016041561
3

Sample Use Guides

In Vivo Use Guide
PO (3 times daily). Doses:150-600 mg/day (based on weight)
Route of Administration: Oral
In Vitro Use Guide
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.
Substance Class Chemical
Created
by admin
on Mon Mar 31 23:42:20 GMT 2025
Edited
by admin
on Mon Mar 31 23:42:20 GMT 2025
Record UNII
Q85FFY6179
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ARIMOCLOMOL CITRATE
MI  
USAN  
Official Name English
ARIMOCLOMOL CITRATE [MI]
Preferred Name English
ARIMOCLOMOL CITRATE [USAN]
Common Name English
3-PYRIDINECARBOXIMIDOYL CHLORIDE, N-((2R)-2-HYDROXY-3-(1-PIPERIDINYL)PROPOXY)-, 1-OXIDE, 2-HYDROXY-1,2,3-PROPANETRICARBOXYLATE (1:1)
Systematic Name English
ORPHAZYME A/S
Brand Name English
N-[(2R)-2-Hydroxy-3-(1-piperidyl)propoxy]pyridine-3-carboximidoyl chloride, 1-oxide, 2-hydroxy-1,2,3-propanetricarboxylate
Systematic Name English
MIPLYFFA
Brand Name English
Classification Tree Code System Code
EU-Orphan Drug EU/3/16/1659
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
FDA ORPHAN DRUG 458814
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
Code System Code Type Description
DRUG BANK
DBSALT001870
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY
NCI_THESAURUS
C174877
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY
CAS
368860-21-3
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY
FDA UNII
Q85FFY6179
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY
SMS_ID
100000177291
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY
MERCK INDEX
m2045
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY Merck Index
PUBCHEM
72710735
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY
USAN
FG-165
Created by admin on Mon Mar 31 23:42:20 GMT 2025 , Edited by admin on Mon Mar 31 23:42:20 GMT 2025
PRIMARY
Related Record Type Details
Structure of ARIMOCLOMOL
PARENT -> SALT/SOLVATE
Related Record Type Details
Structure of ARIMOCLOMOL
ACTIVE MOIETY
NIH
NCATS
PRIVACY NOTICE
ACCESSIBILITY
DISCLAIMER
HHS VULNERABILITY DISCLOSURE
For language access assistance, contact the NCATS Public Information Officer
National Center for Advancing Translational Sciences (NCATS),
6701 Democracy Boulevard, Bethesda MD 20892-4874 • 301-594-8966