Kubota Vision, a subsidiary of Kubota Pharmaceutical Holdings, has recently finished enrollment in the third phase of their emixustat hydrochloride (emixustat) trial. Emixustat hydrochloride is a treatment for Stargardt disease that leads to macular atrophy.

About Stargardt Disease

Stargardt disease is an inherited disorder that affects the eyes. It impacts the retina and results in vision loss. This loss typically occurs in childhood or adolescence, but some do not notice changes in vision until they are adults. The major symptom is loss of vision in both eyes, but other symptoms include spots in the center of vision, light sensitivity, loss of some peripheral vision, color blindness, and a delay in adjustment when moving from light to dark. The most common cause of these symptoms is a mutation in the ABCA4 gene, which results in issues in the clearing of vitamin A photoreceptors. The cones and rods of the eyes then die.

An examination of the retina is used for a diagnosis, along with color testing, visual field testing, Fundus photo, ERG, and OCT. There is no treatment for this disease; doctors recommend glasses and healthy lifestyle choices.

About the Study

This study spans 29 sites and 11 countries and will be double-masked, randomized, and placebo-controlled. Participants will receive 10 mg of emixustat or a placebo daily for 24 months. 194 patients were enrolled, exceeding the original expectation of 162.

The primary endpoint is the reduction of macular degeneration, while secondary endpoints include reading speed and changes in visual function parameters. If successful, this medication will offer a viable treatment for Stargardt disease patients. This is important as there is currently no treatment for this condition.

The FDA and EMA have already granted emixustat the Orphan Drug designation, giving hope to researchers and patients. Hopefully this trial will result in success.

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