Emixustat (hydrochloride)

Emixustat (ACU-4429) hydrochloride is an orally active RPE65 inhibitor with an IC₅₀ value of 4.4 nM. Emixustat hydrochloride is also a visual cycle modulator, capable of regulating visual cycle activity by inhibiting retinol isomerization, and holds potential for studying vision disorders such as age-related macular degeneration (AMD)^[1][2][3][4]. IC50 & Target:IC50: 4.4 nM (Retinal Pigment Epithelium 65-kDa protein (RPE65))^[4] In Vitro:Emixustat (0.1 nM - 10 μM, 1 h) concentration-dependently reduces the production of 11-cis-retinol, thereby inhibiting RPE65 activity^[4]. In Vivo:Emixustat (1-10 mg/kg, oral administration, single dose, or twice daily for 6 days; 1 mg/kg, i.p., measurement after 30-60 minutes) reduced cation influx and oxygen consumption in the retinas of brown Norway rats under dark adaptation conditions^[3].
Emixustat (0.03-3.0 mg/kg, intravenous injection, once daily for 5 days) inhibits neovascularization and protects the retina in the oxygen-induced retinopathy mouse model^[4].