Date: 2017-01-26
Type of information: Recruitment of the first patient
phase: 2
Announcement: recruitment of the first patient
Company: Acucela (USA - MA)
Product: emixustat
Action
mechanism: enzyme inhibitor/RPE65 inhibitor. Emixustat hydrochloride (emixustat) is a once-daily, 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. Emixustat has been shown to play a critical role in slowing the progression of multiple retinal degenerative diseases in animal models, including GA secondary to AMD.
Disease: macular atrophy secondary to Stargardt disease
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Country: USA
Trial
details: This multicenter, randomized, masked phase 2a study is evaluating the pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. (NCT03033108)
Latest
news: * On January 26, 2017, Acucela announced that the first patient has enrolled in a study to evaluate Acucela’s leading drug candidate, emixustat hydrochloride (“emixustat”) in subjects with macular atrophy secondary to Stargardt disease. This multicenter, randomized, masked phase 2a study is designed to evaluate the pharmacodynamics, safety and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt disease. Approximately 30 subjects will be enrolled at 4 to 6 clinical sites in the United States. Subjects will be randomly assigned to one of three treatment arms in a 1:1:1 ratio. Treatment arms include: emixustat 2.5 mg, emixustat 5 mg, and emixustat 10 mg. Subjects will orally take study drug once daily in the evening for one month.
