Arimoclomol

Arimoclomol

File:Arimoclomol.svg
Systematic (IUPAC) name
N-{[(2R)-2-hydroxy-3-piperidin-1-ylpropyl]oxy}pyridine-3-carboximidoyl chloride 1-oxide
Clinical data
Routes of administration	Oral
Legal status
Legal status	Investigational
Identifiers
CAS Number	289893-25-0
ATC code	none
PubChem	CID 208924
ChemSpider	21106260
Chemical data
Formula	C14H20ClN3O3
Molar mass	313.78 g/mol[[]]

Arimoclomol (INN, originally codenamed BRX-220) is an experimental drug developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California. The orally administered drug is intended to treat amyotrophic lateral sclerosis (ALS).

Mechanism of action

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.

Arimoclomol activates the heat shock response. It is believed to act at Hsp70.

History

Arimoclomol has been shown to extend life in an animal model of ALS and was well tolerated in healthy human volunteers in a Phase I study. CytRx is currently conducting a Phase II clinical trial.

Arimoclomol was discovered by Hungarian researchers, as a drug candidate to treat insulin resistance and diabetic complications such as retinopathy, neuropathy and nephropathy. Later, the compound, along with other small molecules, was screened for further development by Hungarian firm Biorex, which was sold to CytRx Corporation, who developed it toward a different direction from 2003.