Acucela announced that the company has achieved over 65% subject enrollment in its ongoing phase 3 clinical trial investigating emixustat hydrochloride in patients 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 are randomly assigned to emixustat 10 mg or placebo (2:1 ratio) once daily for 24 months. For a rare disease like Stargardt, enrolling subjects into a clinical study can be challenging. A total of 108 patients have been enrolled in this study as of January 31, 2020, and the target total of 162 subjects are expected to be fully enrolled across 30 sites in 11 countries worldwide during 2020.
The FDA and European Medicines Agency (EMA) granted orphan drug designation to emixustat for the treatment of Stargardt disease.
This projection has been included in the financial results of FY2019 announced on February 13, 2020 in Japan.