Kubota Vision Announced End of Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease

Key Facts

(Tokyo 4596), announced today the completion of the phase 3 clinical trial investigating emixustat hydrochloride (“emixustat”) in patients with macular atrophy secondary to Stargardt disease.
“Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt Disease” > > Tweet this The study was a multi-center, 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 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.
It may also be referred to as Stargardt macular dystrophy or juvenile macular degeneration and affects approximately 1 in 8,000 - 10,000 individuals worldwide.*1 The most common form of the disease is caused by a genetic mutation of the ABCA4 gene leading to the accumulation of toxic vitamin A byproducts (primarily A2E) in the retina, which results in the gradual deterioration of photoreceptors and vision.