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  • Acucela, Inc.

    A new drug for treating geographic atrophy (GA) in dry AMD patients failed to slow the rate of lesion growth in a phase 2b/3 trial.

    Top-line results for emixustat hydrochloride also showed that BCVA did not improve in treated patients compared with placebo after 24 months of follow-up. Incidence of adverse effects appeared to be equal between the two groups.

    Developed by Acucela, emixustat hydrochloride is an orally administered small molecule that inhibits RPE65, an enzyme crucial to the visual cycle, the chemical pathway in the retina central to the initiation of visual perception. 

    Previously, the drug showed promise for treating GA after it was discovered to reduce oxidative stress by slowing the metabolic rate of photoreceptors and accumulation of vitamin A-based toxins.

    “We are carefully reviewing the data in geographic atrophy before we decide on our next steps with emixustat in this indication. We will continue to advance our in-licensed projects as well as our in-house research," stated Ryo Kubota, MD, PhD, and Chairman, president and CEO of Acucela.