Vafidemstat

DB16446InvestigationalSmall molecule

Vafidemstat is under investigation in clinical trial NCT03867253 (Testing the Safety and Preliminary Efficacy of the New Drug ORY-2001 in Mild to Moderate Alzheimer's Disease).

Mechanism: Vafidemstat is a small molecule irreversible inhibitor of histone demethylase LSD1 and also a dual inhibitor of LSD1/MAO-B.
Curator reviewed
Formula / weight: C₁₉H₂₀N₄O₂ · 336.395 g/mol (avg)
Code names: BCP29383 · CS-0058593 · HY-112623 · ORY-2001

Resolves to

Compound

UNIILZ82JLT4UPCAS1357362-02-7

Structure

InChIKeyXBBRLCXCBCZIOI-DLBZAZTESA-NSMILESNC1=NN=C(CN[C@@H]2C[C@H]2C2=CC=C(OCC3=CC=CC=C3)C=C2)O1

Targets

O60341KDM1AP27338MAOB

What you can answer from here — as of April 23, 2025

2Protein targetsEach mapped to UniProt, with action and pharmacological action

Listed above

2Clinical trialsPhase, status and sponsor resolved per trial

6+ReferencesStructured and connected to the statements they support

Which drugs share a target with Vafidemstat, and which of those have an active Phase 3 trial?

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