| Description | Emixustat, is an inhibitor of the visual cycle isomerase with an IC50 value of 4.4 nM in vitro. a novel visual cycle modulator. |
| In vitro | Emixustat strongly inhibits 11-cis-retinol production with an IC50 value of 232±3 nM.Emixustat potently inhibits isomerase activity in vitro (IC50=4.4 nM). Treatment of emixustat shows a concentration dependent reduction of 11-cis-ROL production . |
| In vivo | Pre-treatment with a single dose of emixustat (0.3 mg/kg) provids a 50%protective effect against light-induced photoreceptor cell loss, while higher doses (1-3 mg/kg) are nearly 100%effective.?In Abca4-/- mice, chronic (3 month) emixustat treatment markedly reduces lipofuscin autofluorescence and reduces A2E levels by 60%( ED50=0.47 mg/kg).?In the retinopathy of prematurity rodent model, treatment with emixustat during the period of ischemia and reperfusion injury produces a 30%reduction in retinal neovascularization (ED50=0.46mg/kg).Emixustat reduces the production of visual chromophore (11-cis retinal) in wild-type mice following a single oral dose (ED50=0.18mg/kg). In albino mice, emixustat is shown to be effective in preventing photoreceptor cell death caused by intense light exposure. |
| Target activity | Visual cycle isomerase:4.4 nM |
| Synonyms | ACU-4429 |
| molecular weight | 263.38 |
| Molecular formula | C16H25NO2 |
| CAS | 1141777-14-1 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
| Solubility | DMSO: 43 mg/mL (163.26 mM) |
| References | 1. Bavik C, et al. Visual Cycle Modulation as an Approach toward Preservation of Retinal Integrity. PLoS One. 2015 May 13;10(5):e0124940. 2. Kiser PD, et al. Catalytic mechanism of a retinoid isomerase essential for vertebrate vision. Nat Chem Biol. 2015 Jun;11(6):409-15. |