Vafidemstat

Vafidemstat

Clinical data
Other names	ORY-2001; ORY2001
Routes of administration	Oral[1][2]
Drug class	Lysine-specific demethylase 1 (LSD1) inhibitor; Monoamine oxidase B (MAO-B) inhibitor[1]
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.^[1][3][2] It is or was also being developed for several other indications, but no recent development has been reported for these uses.^[1] The drug is taken by mouth.^[1]

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.^[1] Conversely, no recent development has been reported for autism, dementia, Huntington's disease, Parkinson's disease, and telomeric 22q13 monosomy syndrome.^[1] It is being developed by Oryzon.^[1][3]

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).^[2][4] Another drug, zavondemstat (QC8222, TACH101), is a pan-inhibitor of lysine-specific demethylase 4 (LSD4; KDM4).^[5][6][7] Vafidemstat contains the chemical structure of (1S,2R)-tranylcypromine within its own structure.^[8]