Enrollment Continues to Progress for Phase 3 Stargardt Disease Trial

According to a story from Financial Buzz, the ophthalmology company Acucela Inc. has recently announced an update on the progress of its phase 3 clinical trial. This clinical trial is testing the experimental drug emixustat as a treatment for macular atrophy linked to Stargardt disease. The company says that it has achieved 65 percent of its patient enrollment goal so far for the study. 108 participants have enrolled so far and Acucela ultimately hopes for a total of 162 patients.

About Stargardt Disease

Stargardt disease is an inherited disease of the retina, the inner layer of the eye that is sensitive to light. The illness causes macular degeneration, which can begin at different times in the patient’s life. There are different types of Stargardt disease which vary depending on which gene is mutated; in type 1, it is caused by alternations to the ABCA4 gene. In the most severe cases, degeneration can begin in childhood or adolescence, and eventually lead to progressive vision loss. The center of the visual field is most heavily affected; symptoms include blurred vision, impaired color vision, wavy vision, blind spots, loss of depth perception, and poor night vision. Symptoms cannot be corrected with glasses. The pace of vision loss and the symptoms experienced vary from person to person. While there are some lifestyle changes that may slow the progression of Stargardt disease, there is currently no available treatment. Most patients will eventually become legally blind. To learn more about Stargardt disease, click here.

About Emixustat and the Clinical Trial

The trial will last for two years with subjects being randomized to take either a placebo or a 10 mg dose of emixustat once every day. The study includes a total of 30 trial sites in 11 different countries.

Emixustat has earned Orphan Drug designations from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Emixustat functions by interfering in the visual cycle, which is the manner in which vitamin A is processed in the eyes. It inhibits an enzyme involved in the process called RPE65. This slows the cycle and therefore slows the process of degeneration, as the cycle causes the production of toxic vitamin A derivatives in Stargardt disease patients. 

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