Acucela announced that on November 7, 2018, 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.
The study is a multicenter, randomized, double-masked, and placebo-controlled phase 3 clinical study in which subjects will be randomly assigned to emixustat 10 mg or placebo (2:1 ratio) once daily for 24 months. Approximately 160 subjects will be enrolled at 30 sites in 10 countries worldwide.
The primary objective of this study is to determine if emixustat reduces the rate of macular atrophy progression, in comparison to placebo, in subjects with Stargardt disease. Secondary objectives include assessing changes in visual function parameters such as BCVA (best-corrected visual acuity) letter score and reading speed.
“We are very excited to be proceeding with the phase 3 clinical study addressing Stargardt disease with emixustat,” Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela, said in the news release. “Stargardt is an unmet medical need with no known therapies to slow the progression of the disease. We are now focused on initiating our study to advance our clinical program, to help patients facing vision loss and blindness from this disease.”
The FDA granted orphan drug designation to emixustat for the treatment of Stargardt disease.