Vafidemstat

Vafidemstat
Clinical data
Other names	ORY-2001; ORY2001
Routes of; administration	Oral
Drug class	Lysine-specific demethylase 1 (LSD1) inhibitor; Monoamine oxidase B (MAO-B) inhibitor
Identifiers
	IUPAC name 5-[[[(1R,2S)-2-(4-phenylmethoxyphenyl)cyclopropyl]amino]methyl]-1,3,4-oxadiazol-2-amine;
CAS Number	1357362-02-7;
PubChem CID	66714983;
DrugBank	DB16446;
ChemSpider	68019508;
UNII	LZ82JLT4UP;
ChEMBL	ChEMBL4802155;
Chemical and physical data
Formula	C19H20N4O2
Molar mass	336.395 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C1[C@H]([C@@H]1NCC2=NN=C(O2)N)C3=CC=C(C=C3)OCC4=CC=CC=C4;
	InChI InChI=1S/C19H20N4O2/c20-19-23-22-18(25-19)11-21-17-10-16(17)14-6-8-15(9-7-14)24-12-13-4-2-1-3-5-13/h1-9,16-17,21H,10-12H2,(H2,20,23)/t16-,17+/m0/s1; Key:XBBRLCXCBCZIOI-DLBZAZTESA-N;

Vafidemstat (INNTooltip International Nonproprietary Name; developmental code name ORY-2001) is a dual inhibitor of the enzymes lysine-specific demethylase 1 (LSD1; KDM1A) and monoamine oxidase B (MAO-B) which is under development for the treatment of a variety of medical conditions, including aggression, Alzheimer's disease, borderline personality disorder, multiple sclerosis, acute respiratory disease in COVID-19 infection, and schizophrenia. It is or was also being developed for several other indications, but no recent development has been reported for these uses. The drug is taken by mouth.

As of October 2024, vafidemstat is in phase 2 clinical trials for aggression, Alzheimer's disease, borderline personality disorder, multiple sclerosis, COVID-19 acute respiratory disease, and schizophrenia. Conversely, no recent development has been reported for autism, dementia, Huntington's disease, Parkinson's disease, and telomeric 22q13 monosomy syndrome. It is being developed by Oryzon.

Other LSD1 inhibitors that are under development for medical use include bomedemstat (IMG-7289), iadademstat (ORY-1001), phenelzine (Nardil), pulrodemstat (CC-90011), seclidemstat (SP-2577), and tranylcypromine (Parnate). Another drug, zavondemstat (QC8222, TACH101), is a pan-inhibitor of lysine-specific demethylase 4 (LSD4; KDM4), and is being researched for the treatment of cancer. Vafidemstat contains the chemical structure of (1S,2R)-tranylcypromine within its own structure.