Emixustat

Research use only, not for human use.

Emixustat (ACU-4429) is a first in class small-molecule inhibitor of the visual cycle isomerase (RPE65) with IC50 of 4.4 nM.

Emixustat (ACU-4429) is a first in class small-molecule inhibitor of the visual cycle isomerase (RPE65) with IC50 of 4.4 nM; reduces the production of visual chromophore (11-cis retinal) in wild-type mice following a single oral dose (ED50 = 0.18 mg/kg).Other Indication Phase 3 Clinical

Emixustat potently inhibits isomerase activity in vitro (IC50=4.4 nM). Treatment of emixustat shows a concentration dependent reduction of 11-cis-ROL production . Emixustat strongly inhibits 11-cis-retinol production with an IC50 value of 232±3 nM.

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. 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 auto fluorescence 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).

ACU-4429

Others

Other Targets

Other Targets

Other Indications

Other Disease

1141777-14-1

263.3752

C16H25NO2

>98% (HPLC)

DMSO: ≥ 43 mg/mL

C1CCC(CC1)COC2=CC=CC(=C2)C(CCN)O

Benzenemethanol, α-(2-aminoethyl)-3-(cyclohexylmethoxy)-, (αR)-

(-20℃)

With Ice Pack

≥ 2 years