Data Demonstrate Dose-Dependent Pharmacologic Activity of Emixustat in Patients with Geographic Atrophy Associated with Dry Age-Related Macular Degeneration
SEATTLE–(BUSINESS WIRE)–Acucela Inc. (TOKYO: 4589), a clinical-stage biotechnology company that specializes in discovering and developing novel drug candidates to potentially treat and slow the progression of sight-threatening ophthalmic diseases, today announced the publication of results from a Phase 2a clinical trial of emixustat hydrochloride (emixustat) in patients with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD) in the June online edition of RETINA: The Journal of Retinal and Vitreous Diseases. The lead author on the paper is Pravin U. Dugel, MD, Managing Partner, Retinal Consultants of Arizona and Clinical Professor, USC Eye Institute, Keck School of Medicine, University of Southern California. He was also a principal investigator of the emixustat study.

“We are pleased about the publication of the Phase 2a study data”

Emixustat, the first internally developed compound by Acucela, is orally dosed, targets the visual cycle, and is in development for the potential treatment of GA associated with dry AMD for which there is currently no U.S. Food and Drug Administration (FDA)-approved therapy available.
“We are pleased about the publication of the Phase 2a study data,” Dr. Dugel said. “The data, as detailed in this month’s online edition ofRETINA, showed that emixustat achieved dose-dependent pharmacologic activity in the retina of patients with geographic atrophy.”
The published article is titled “Phase II, Randomized, Placebo-Controlled, 90-Day Study of Emixustat HCL in Geographic Atrophy Associated with Dry Age-Related Macular Degeneration” and can be accessed at:,_RANDOMIZED,_PLACEBO_CONTROLLED,_90_DAY.17.aspx. The study was designed to assess the safety, tolerability, and pharmacodynamics of emixustat in subjects diagnosed with GA. Seventy-two subjects were randomized to receive one of four emixustat doses (2, 5, 7, or 10 mg once daily) or placebo for a 90-day treatment period. Biologic activity of emixustat in the retina was measured by electroretinography (ERG). Safety evaluations included analysis of adverse events and ophthalmic examinations. The ERG data showed a dose-dependent pharmacologic effect of emixustat, consistent with the proposed mechanism of action……..
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Source: Business Wire