Vafidemstat
Experimental enzyme inhibitor
From Wikipedia, the free encyclopedia
Vafidemstat (INN; 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]
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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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| Formula | C19H20N4O2 |
| Molar mass | 336.395 g·mol−1 |
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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), and is being researched for the treatment of cancer.[5][6][7] Vafidemstat contains the chemical structure of (1S,2R)-tranylcypromine within its own structure.[8]