EYE-003 is an orally active RPE65 inhibitor with an IC50 value of 102 nM. It regulates the visual cycle in mice by decreasing the synthesis of 11-cis-Retinal (11cRAL) and increasing all-trans-retinyl esters (atREs). EYE-003 also inhibits dark-adapted electroretinogram (ERG) b-wave amplitudes and provides protection against retinal degeneration in Abca4⁻/⁻Rdh8⁻/⁻ mice by dose-dependently reducing retinal autofluorescent spots and preserving outer nuclear layer (ONL) thickness. EYE-003 is applicable for researching retinal disorders related to the visual cycle, such as Stargardt disease type 1 (STGD1).

EYE-003 (0.1-10 μM) inhibits the activity of RPE65 in bovine retinal pigment epithelium (RPE) microsomes, with an IC₅₀ of 102 nM.

EYE-003, administered at 10 mg/kg via intraperitoneal injection 30 minutes before photobleaching and followed by 2 hours of dark adaptation, reduces the synthesis of 11-cis-retinal (11cRAL) and increases all-trans-retinyl esters (atREs) in BALB/cJ mice. EYE-003, given under the same conditions and measured between 0.5 to 4 hours post-administration, restores 11cRAL and atREs levels to control group levels 4 hours after administration. When administered orally, EYE-003 significantly inhibits dark-adapted electroretinogram (ERG) a-wave and b-wave amplitudes in BALB/cJ mice. A single dose of EYE-003, whether by intraperitoneal injection or oral gavage 30 minutes before light exposure, reduces retinal autofluorescent spots and uniform lipofuscin autofluorescence in Abca4 ⁻/⁻ Rdh8 ⁻/⁻ mice and preserves the thickness of the outer nuclear layer (ONL).