Emixustat for Stargardt Disease

Our visual cycle modulation approach to Stargardt Disease: Emixustat hydrochloride


The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process. Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of A2E and related compounds. Since A2E is a byproduct of the visual cycle, modulation of the visual cycle may be an effective approach to reduce the accumulation of A2E. Because A2E has been implicated in the pathogenesis of STGD, it is hypothesized that a reduction of A2E accumulation may help to preserve the health and integrity of the retina and slow vision loss in STGD. In a preclinical mouse model of STGD, emixustat significantly reduced the levels of A2E.