Arimoclomol

Identification

Name
Arimoclomol
Accession Number
DB05025
Type
Small Molecule
Groups
Investigational
Description

Arimoclomol is an experimental drug compound developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California. The orally administered drug is intended to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, a neurodegenerative disease with no effective treatment.

Structure
Thumb
Synonyms
Not Available
External IDs
BRX-220 FREE BASE
Product Ingredients
IngredientUNIICASInChI Key
Arimoclomol citrateQ85FFY6179368860-21-3XSENLDLUMVYRET-BTQNPOSSSA-N
Arimoclomol maleate18D1V854HG289893-26-1OHUSJUJCPWMZKR-FEGZNKODSA-N
Categories
UNII
EUT3557RT5
CAS number
289893-25-0
Weight
Average: 313.78
Monoisotopic: 313.119319228
Chemical Formula
C14H20ClN3O3
InChI Key
SGEIEGAXKLMUIZ-CYBMUJFWSA-N
InChI
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
IUPAC Name
3-[chloro({[(2R)-2-hydroxy-3-(piperidin-1-yl)propoxy]imino})methyl]pyridin-1-ium-1-olate
SMILES
O[C@@H](CON=C(Cl)C1=C[N+]([O-])=CC=C1)CN1CCCCC1

Pharmacology

Indication

Investigated for use/treatment in amyotrophic lateral sclerosis (ALS), diabetes mellitus type 2, neurologic disorders, and neuropathy (diabetic).

Pharmacodynamics
Not Available
Mechanism of action

Arimoclomol is designed to stimulate a natural cellular repair pathway by activating compounds called “molecular chaperones.” Arimoclomol uses a unique 'molecular chaperone' co-induction mechanism. The small molecule drug candidate 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 activation of molecular chaperones could have therapeutic efficacy for a broad range of diseases.

TargetActionsOrganism
USuperoxide dismutase [Cu-Zn]Not AvailableHumans
Absorption
Not Available
Volume of distribution
Not Available
Protein binding
Not Available
Metabolism
Not Available
Route of elimination
Not Available
Half life
Not Available
Clearance
Not Available
Toxicity

R Upregulation of heat shock proteins rescues motoneurones from axotomy-induced cell death in neonatal rats. Exp Neurol

Affected organisms
Not Available
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
Not Available
Food Interactions
Not Available

References

General References
Not Available
External Links
PubChem Compound
208924
PubChem Substance
175426933
ChemSpider
181020
Wikipedia
Arimoclomol

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
2CompletedTreatmentAmyotrophic Lateral Sclerosis (ALS)1
2RecruitingTreatmentGaucher Disease, Type 1 / Gaucher Disease, Type 31
2RecruitingTreatmentInclusion Body Myositis (IBM)1
2WithdrawnTreatmentAmyotrophic Lateral Sclerosis (ALS)1
2, 3Active Not RecruitingTreatmentNiemann-Pick Disease, Type C1
2, 3CompletedTreatmentAcquired Amyotrophic Lateral Sclerosis / Amyotrophic Lateral Sclerosis (ALS) / Amyotrophic Lateral Sclerosis 11
2, 3CompletedTreatmentInclusion Body Myositis (IBM)1
3RecruitingTreatmentAmyotrophic Lateral Sclerosis (ALS)1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage forms
Not Available
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not Available
Predicted Properties
PropertyValueSource
Water Solubility0.237 mg/mLALOGPS
logP0.45ALOGPS
logP0.31ChemAxon
logS-3.1ALOGPS
pKa (Strongest Acidic)14ChemAxon
pKa (Strongest Basic)9.14ChemAxon
Physiological Charge1ChemAxon
Hydrogen Acceptor Count5ChemAxon
Hydrogen Donor Count1ChemAxon
Polar Surface Area70.52 Å2ChemAxon
Rotatable Bond Count6ChemAxon
Refractivity83.13 m3·mol-1ChemAxon
Polarizability32.93 Å3ChemAxon
Number of Rings2ChemAxon
Bioavailability1ChemAxon
Rule of FiveYesChemAxon
Ghose FilterYesChemAxon
Veber's RuleNoChemAxon
MDDR-like RuleNoChemAxon
Predicted ADMET features
PropertyValueProbability
Human Intestinal Absorption+0.81
Blood Brain Barrier+0.8583
Caco-2 permeable-0.5726
P-glycoprotein substrateSubstrate0.6062
P-glycoprotein inhibitor IInhibitor0.699
P-glycoprotein inhibitor IIInhibitor0.9221
Renal organic cation transporterInhibitor0.6469
CYP450 2C9 substrateNon-substrate0.7069
CYP450 2D6 substrateNon-substrate0.8045
CYP450 3A4 substrateNon-substrate0.5082
CYP450 1A2 substrateNon-inhibitor0.7652
CYP450 2C9 inhibitorNon-inhibitor0.8513
CYP450 2D6 inhibitorNon-inhibitor0.753
CYP450 2C19 inhibitorNon-inhibitor0.6055
CYP450 3A4 inhibitorNon-inhibitor0.844
CYP450 inhibitory promiscuityLow CYP Inhibitory Promiscuity0.7542
Ames testNon AMES toxic0.5
CarcinogenicityNon-carcinogens0.8385
BiodegradationNot ready biodegradable0.9964
Rat acute toxicity2.5777 LD50, mol/kg Not applicable
hERG inhibition (predictor I)Weak inhibitor0.6881
hERG inhibition (predictor II)Inhibitor0.7547
ADMET data is predicted using admetSAR, a free tool for evaluating chemical ADMET properties. (23092397)

Spectra

Mass Spec (NIST)
Not Available
Spectra
SpectrumSpectrum TypeSplash Key
Predicted GC-MS Spectrum - GC-MSPredicted GC-MSNot Available
Predicted MS/MS Spectrum - 10V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 10V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Negative (Annotated)Predicted LC-MS/MSNot Available

Taxonomy

Description
This compound belongs to the class of organic compounds known as pyridinium derivatives. These are compounds containing a pyridinium ring, which is the cationic form of pyridine.
Kingdom
Organic compounds
Super Class
Organoheterocyclic compounds
Class
Pyridines and derivatives
Sub Class
Pyridinium derivatives
Direct Parent
Pyridinium derivatives
Alternative Parents
Piperidines / Heteroaromatic compounds / Trialkylamines / Secondary alcohols / 1,2-aminoalcohols / Azacyclic compounds / Organopnictogen compounds / Organochlorides / Organic oxides / Hydrocarbon derivatives
Substituents
Piperidine / Pyridinium / Heteroaromatic compound / 1,2-aminoalcohol / Secondary alcohol / Tertiary amine / Tertiary aliphatic amine / Azacycle / Organic nitrogen compound / Hydrocarbon derivative
Molecular Framework
Aromatic heteromonocyclic compounds
External Descriptors
Not Available

Targets

Kind
Protein
Organism
Humans
Pharmacological action
Unknown
General Function
Zinc ion binding
Specific Function
Destroys radicals which are normally produced within the cells and which are toxic to biological systems.
Gene Name
SOD1
Uniprot ID
P00441
Uniprot Name
Superoxide dismutase [Cu-Zn]
Molecular Weight
15935.685 Da

Drug created on October 21, 2007 16:23 / Updated on March 11, 2019 19:38