Emixustat hydrochloride strongly inhibits 11-cis-retinol production with IC50 values of 232 ± 3 nM. IC50 value: 232 nM [1]Target: 11-cis-retinolEmixustat hydrochloride is a novel visual cycle modulator, in healthy volunteers. Oral Emixustat was safe and well tolerated when administered once daily for 14 days with minimal systemic adverse events reported. Evaluation of emixustat in subjects with geographic atrophy associated with dry age-related macular degeneration.[2]
[1]. Kiser PD, et al. Catalytic mechanism of a retinoid isomerase essential for vertebrate vision. Nat Chem Biol. 2015 Jun;11(6):409-15. [2]. Kubota R, et al. Phase 1, dose-ranging study of emixustat hydrochloride (ACU-4429), a novel visual cycle modulator, in healthy volunteers. Retina. 2014 Mar;34(3):603-9.