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Drugs Fut 2014, 39(9): 615
ISSN 0377-8282
Copyright 2014 Clarivate Analytics
CCC: 0377-8282
DOI: 10.1358/dof.2014.039.09.2207181
Emixustat hydrochloride
Kefauver, S.C.
Age-related macular degeneration (AMD) is one of the leading causes of vision loss worldwide, especially in elderly populations, which are expected to nearly triple in the next few decades. As recent as 10 years ago there were no treatment options for either the early or advanced stages of wet (neovascular, exudative) and dry (geographic atrophy, non-exudative) AMD, but development has since advanced rapidly on a number of fronts. Vascular endothelial growth factor (VEGF) antagonists have been largely successful in the prevention or delay of blindness due to wet AMD. While anti-VEGF therapy has greatly improved treatment of wet AMD, there is still a large unmet therapeutic need concerning dry AMD. Considerable progress has been made in improving our understanding of the epidemiology and pathogenesis of AMD, but the intermediate and advanced forms of dry AMD still have no approved therapeutic treatments. Emixustat hydrochloride (ACU-4429), a novel modulator of the visual cycle, has shown promise for the treatment of dry AMD by suppressing the production and accumulation of toxic retinoid by-products, and thus preserving the overall health of ageing ocular tissues. It has shown considerable promise, with favorable safety and efficacy profiles in preclinical and phase I studies, and is currently in phase II/III studies.

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