Kubota Vision Announces End-of-Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease
Kubota Vision announced today the completion of the phase 3 clinical trial investigating emixustat hydrochloride (emixustat) in patients with macular atrophy secondary to Stargardt disease.
The study was a multicenter, randomized, double-masked, and placebo-controlled phase 3 clinical study in which subjects were randomly assigned to emixustat 10 mg or placebo (2:1 ratio) once daily for 24 months. The target total number of subjects was 162; however, due to high interest in the study, a total of 194 subjects were enrolled in this study across 29 sites in 11 countries worldwide. The last patient has completed the study. 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. The database of this study will be locked in the third quarter 2022.
“Despite Stargardt being a rare disease, we were able to enroll more patients than originally planned and feel grateful that the patients trusted our drug with great expectation and continued taking the drug for 2 years during the pandemic," Ryo Kubota, MD, PhD, Chairman, President and CEO of Kubota Vision, said in a company news release. "We would like to sincerely thank all of the participants and their families for their cooperation and are very pleased that the long-term, large-scale clinical trial has been successfully completed. We are also looking forward to receiving the results of the trial in the coming months.”
The FDA granted orphan drug designation to emixustat for the treatment of Stargardt disease. (See January 5, 2017 press release titled “Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt Disease” and June 9, 2019 press release titled “Acucela Receives Orphan Designation from the EMA for Emixustat for the Treatment of Stargardt Disease”).