Arimoclomol

Arimoclomol
Clinical data
Routes of; administration	Oral
ATC code	N07XX17 (WHO) ;
Legal status
Legal status	Investigational;
Identifiers
	IUPAC name N-{[(2R)-2-hydroxy-3-piperidin-1-ylpropyl]oxy}pyridine-3-carboximidoyl chloride 1-oxide;
CAS Number	289893-25-0 ;
PubChem CID	208924;
ChemSpider	21106260 ;
UNII	EUT3557RT5;
KEGG	D11374;
ChEMBL	ChEMBL2107726 ;
CompTox Dashboard (EPA)	DTXSID5057701 ;
Chemical and physical data
Formula	C14H20ClN3O3
Molar mass	313.78 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES O[C@H](CN1CCCCC1)CO\N=C(\Cl)c2ccc[n+]([O-])c2;
	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/b16-14+/t13-/m1/s1 ; Key:SGEIEGAXKLMUIZ-ZPTIMJQQSA-N ;
	(what is this?) (verify)

Arimoclomol (INN; originally codenamed BRX-345, which is a citrate salt formulation of BRX-220) is an experimental drug developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS.^[1] 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.^[2]^[3]

^ "CytRx Sells Molecular Chaperone Assets to Orphazyme in Deal Worth $120M | GEN Genetic Engineering & Biotechnology News - Biotech from Bench to Business | GEN". GEN. 17 May 2011.
^ "European Medicines Agency - - EU/3/14/1376". www.ema.europa.eu. Archived from the original on 2017-07-28. Retrieved 2022-02-15.
^ "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov.

[1] "CytRx Sells Molecular Chaperone Assets to Orphazyme in Deal Worth $120M | GEN Genetic Engineering & Biotechnology News - Biotech from Bench to Business | GEN". GEN. 17 May 2011.

[2] "European Medicines Agency - - EU/3/14/1376". www.ema.europa.eu. Archived from the original on 2017-07-28. Retrieved 2022-02-15.

[3] "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov.

[1]

[2]

[3]

Clinical data

Routes of administration	Oral
ATC code	N07XX17 (WHO)
Legal status
Legal status	Investigational
Identifiers
IUPAC name N-{[(2R)-2-hydroxy-3-piperidin-1-ylpropyl]oxy}pyridine-3-carboximidoyl chloride 1-oxide
CAS Number	289893-25-0^Y
PubChem CID	208924
ChemSpider	21106260^Y
UNII	EUT3557RT5
KEGG	D11374
ChEMBL	ChEMBL2107726^N
CompTox Dashboard (EPA)	DTXSID5057701
Chemical and physical data
Formula	C₁₄H₂₀ClN₃O₃
Molar mass	313.78 g·mol⁻¹
3D model (JSmol)	Interactive image
SMILES O[C@H](CN1CCCCC1)CO\N=C(\Cl)c2ccc[n+]([O-])c2
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/b16-14+/t13-/m1/s1^Y Key:SGEIEGAXKLMUIZ-ZPTIMJQQSA-N^Y
^NY (what is this?) (verify)