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Details

Stereochemistry ABSOLUTE
Molecular Formula C14H20ClN3O3.C4H4O4
Molecular Weight 429.852
Optical Activity UNSPECIFIED
Defined Stereocenters 1 / 1
E/Z Centers 2
Charge 0

SHOW SMILES / InChI
Structure of ARIMOCLOMOL MALEATE

SMILES

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

InChI

InChIKey=OHUSJUJCPWMZKR-GARNONDBSA-N
InChI=1S/C14H20ClN3O3.C4H4O4/c15-14(12-5-4-8-18(20)9-12)16-21-11-13(19)10-17-6-2-1-3-7-17;5-3(6)1-2-4(7)8/h4-5,8-9,13,19H,1-3,6-7,10-11H2;1-2H,(H,5,6)(H,7,8)/b16-14-;2-1-/t13-;/m1./s1

HIDE SMILES / InChI
Molecular Formula C4H4O4
Molecular Weight 116.0722
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 1
Optical Activity NONE
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

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.

CNS Activity

Originator

Approval Year

Unknown
139594
Targets

Targets

Primary TargetPharmacologyConditionPotency
Activator
1430
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Phase III
656
Unknown

Approved Use

Unknown
Primary
Phase III
656
Unknown

Approved Use

Unknown
Primary
Phase III
656
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
n = 20
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 20
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 (unknown)
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
n = 19
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 19
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
n = 20
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 20
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
n = 20
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 20
Sources:
https://pubmed.ncbi.nlm.nih.gov/18551622/
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:
https://pubmed.ncbi.nlm.nih.gov/18551622/
unhealthy
n = 19
Health Status: unhealthy
Condition: amyotrophic lateral sclerosis
Sex: M+F
Food Status: UNKNOWN
Population Size: 19
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 Sep 7
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 Sat Dec 16 08:31:00 GMT 2023
Edited
by admin
on Sat Dec 16 08:31:00 GMT 2023
Record UNII
18D1V854HG
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ARIMOCLOMOL MALEATE
MI  
Common Name English
BRX-220
Code English
3-PYRIDINECARBOXIMIDOYL CHLORIDE, N-((2R)-2-HYDROXY-3-(1-PIPERIDINYL)PROPOXY)-, 1-OXIDE, (2Z)-2-BUTENEDIOATE (1:1)
Systematic Name English
ARIMOCLOMOL MALEATE [MI]
Common Name English
3-PYRIDINECARBOXIMIDOYL CHLORIDE, N-((2R)-2-HYDROXY-3-(1-PIPERIDINYL)PROPOXY)-, 1-OXIDE, (2Z)-2-BUTENEDIOATE (1:1) (SALT)
Systematic Name English
Code System Code Type Description
SMS_ID
100000177258
Created by admin on Sat Dec 16 08:31:01 GMT 2023 , Edited by admin on Sat Dec 16 08:31:01 GMT 2023
PRIMARY
EPA CompTox
DTXSID30861525
Created by admin on Sat Dec 16 08:31:01 GMT 2023 , Edited by admin on Sat Dec 16 08:31:01 GMT 2023
PRIMARY
MERCK INDEX
m2045
Created by admin on Sat Dec 16 08:31:01 GMT 2023 , Edited by admin on Sat Dec 16 08:31:01 GMT 2023
PRIMARY Merck Index
DRUG BANK
DBSALT001869
Created by admin on Sat Dec 16 08:31:01 GMT 2023 , Edited by admin on Sat Dec 16 08:31:01 GMT 2023
PRIMARY
FDA UNII
18D1V854HG
Created by admin on Sat Dec 16 08:31:01 GMT 2023 , Edited by admin on Sat Dec 16 08:31:01 GMT 2023
PRIMARY
PUBCHEM
72941795
Created by admin on Sat Dec 16 08:31:01 GMT 2023 , Edited by admin on Sat Dec 16 08:31:01 GMT 2023
PRIMARY
CAS
289893-26-1
Created by admin on Sat Dec 16 08:31:01 GMT 2023 , Edited by admin on Sat Dec 16 08:31:01 GMT 2023
PRIMARY
Related Record Type Details
Structure of ARIMOCLOMOL
ACTIVE MOIETY
NIH
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