Drug intelligence / Profile preview

vafidemstat

Development stage
Phase 2
Lead developer
Oryzon Genomics
Modality
Small Molecules
Administration
Oral
01

Overview

Vafidemstat is an oral, CNS-optimized small molecule that acts as a covalent inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1; KDM1A) and also inhibits monoamine oxidase B (MAO-B). It is being developed primarily for central nervous system disorders. Vafidemstat reduces cognitive impairment, neuroinflammation, and has neuroprotective effects. In preclinical models, it restores memory function and reduces aggressive behavior in models of accelerated aging and Alzheimer's disease. Clinical trials have shown positive results in reducing aggression and agitation in psychiatric disorders such as borderline personality disorder (BPD) and Alzheimer's disease, as well as anti-inflammatory activity in multiple sclerosis. The drug is under development by Oryzon for several indications including aggression, Alzheimer's disease, BPD, multiple sclerosis, COVID-19 acute respiratory disease, and schizophrenia[1][3][6][7].

Brand names
vafidemstat
Other names
ORY2001ORY-2001ORY 2001
02

Targets

KDM1A (LSD1)MAOB (Monoamine oxidase B)

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