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| Clinical data | |
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| 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] |
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| CAS Number | |
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| ChEMBL | |
| Chemical and physical data | |
| Formula | C19H20N4O2 |
| Molar mass | 336.395 g·mol−1 |
| 3D model (JSmol) | |
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Vafidemstat (INNTooltip International Nonproprietary Name; developmental code nameORY-2001) is a dualinhibitor of theenzymeslysine-specific demethylase 1 (LSD1; KDM1A) andmonoamine oxidase B (MAO-B) which is under development for the treatment of a variety ofmedical conditions, includingaggression,Alzheimer's disease,borderline personality disorder,multiple sclerosis,acute respiratory disease inCOVID-19 infection, andschizophrenia.[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 takenby mouth.[1]
As of October 2024, vafidemstat is inphase 2clinical 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 forautism,dementia,Huntington's disease,Parkinson's disease, andtelomeric 22q13 monosomy syndrome.[1] It is being developed by Oryzon.[1][3]
Other LSD1 inhibitors that are under development for medical use includebomedemstat (IMG-7289),iadademstat (ORY-1001),phenelzine (Nardil),pulrodemstat (CC-90011),seclidemstat (SP-2577), andtranylcypromine (Parnate).[2][4] Another drug,zavondemstat (QC8222, TACH101), is a pan-inhibitor oflysine-specific demethylase 4 (LSD4; KDM4).[5][6][7] Vafidemstat contains thechemical structure of (1S,2R)-tranylcypromine within its own structure.[8]
-stat- or enzyme inhibitors -stat [...] -demstat lysine-specific histone demethylase inhibitors (a) bomedemstat (122), iadademstat (119), pulrodemstat (124), seclidemstat (118), vafidemstat (119), zavondemstat (128)