LSD1-IN-43 is a highly selective, reversible, and orally active LSD1 inhibitor with blood-brain barrier permeability, showing an IC50 value of 0.8 μM. It exhibits lower inhibitory activity against LSD1 homologs MAO-A and MAO-B. LSD1-IN-43 significantly inhibits Aβ aggregation and enhances neuronal cell viability induced by Aβ. It is applicable in Alzheimer's disease (AD) research.

LSD1-IN-43 (10 μM) selectively and reversibly inhibits LSD1, with minimal impact on monoamine oxidase A (inhibition rate of 7%) and monoamine oxidase B (inhibition rate of 4.9%). At concentrations of 25-100 μM, LSD1-IN-43 demonstrates low toxicity and effectively reduces Aβ 1-42 production in HT22 cells. Additionally, at 10 μM, it significantly inhibits Aβ 1-42 aggregation with an inhibition rate of 66.19%, surpassing Curcumin's 49.98%.

LSD1-IN-43 (125-500 μM, 24-56 h) exhibits a dose-dependent lifespan extension and decreases paralysis incidence in the CL4176 transgenic nematode model. Additionally, LSD1-IN-43 (40-80 mg/kg, i.g.) can restore levels of the histone marker (H3K9me2) regulated by LSD1, improving learning, memory, and cognitive function deficits in Alzheimer's disease mice.