Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride

Summary

The purpose of this study is to determine if emixustat hydrochloride (ACU-4429) reduces the rate of progression of geographic atrophy compared to placebo in subjects with dry age-related macular degeneration.

Timeline

Start

2013-02

Primary completion

2016-04

Completion

2016-05

Outcome

Missed primary endpoint

paper Emixustat did not reduce the growth rate of GA in AMD. PMID 29716784 ↗

release “Acucela recently announced that the study primary endpoint was not met in a Phase IIb/III clinical trial of the compound in the U.S.” otsuka.co.jp ↗

Publications

• Yeong JL, Loveman E, Colquitt JL, Royle P, Waugh N, Lois N. Visual cycle modulators versus placebo or observation for the prevention and treatment of geographic atrophy due to age-related macular degeneration. Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD013154. doi: 10.1002/14651858.CD013154.pub2.
• Rosenfeld PJ, Dugel PU, Holz FG, Heier JS, Pearlman JA, Novack RL, Csaky KG, Koester JM, Gregory JK, Kubota R. Emixustat Hydrochloride for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Clinical Trial. Ophthalmology. 2018 Oct;125(10):1556-1567. doi: 10.1016/j.ophtha.2018.03.059. Epub 2018 Apr 30.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Emixustat	Small molecule	2.5 mg	Oral
Subject	Emixustat	Small molecule	5 mg	Oral
Subject	Emixustat	Small molecule	10 mg	Oral

Indications