Trials beginning for Emixustat Treatment of Stargardt Disease

Acucela has announced that the first patient has enrolled in a study to evaluate emixustat hydrochloride (“emixustat”) in subjects with Stargardt disease. Approximately 30 subjects will be enrolled at 4 to 6 clinical sites in the United States, with subjects taking oral doses of the drug once daily in the evening for one month.
Emixustat modulates the visual cycle by significantly reducing accumulation in the retina of the retinal related toxic by-product A2E, believed to damage retinal cells. The drug has been in Phase 1/2 trials since 2008 for treatment of dry macular degeneration, and now a recent orphan drug designation by the FDA has opened the door for Stargardt disease to be included.
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