I'm Dr. Paul Bernstein from the Moran Eye Center of the University of Utah. I'm a specialist in inherited retina diseases. I'm here at the ASRS meeting in Montreal, and I presented work on the DRAGON study of tinlarebant for treatment of Stargardt disease. Tinlarebant is an investigational drug that is administered to patients with Stargardt disease in the hopes of slowing down the progression of the condition. The DRAGON study is a phase 3 study to see if we can slow down the progression of Stargardt disease. In the study, we had control arm that received placebo and another arm that received five milligrams of tinlarebant per day. The inclusion criteria included one mutation in ABCA4 and Stargardt disease and age between 12 and 20. These patients were followed for two years, and we found positive results in the study that showed that we could slow the progression of enlargement of atrophy in these patients by about 36%.

We also found that we could slow down the accumulation of autofluorescent compounds in the eye. This is a major mechanism of how tinlarebant works. It decreases the amount of RBP-4, the carry of vitamin A in the bloodstream, and that decreases the amount available to the eye and decreases the amount of toxic retinoids that form. Based on these results, we are very hopeful that the FDA will approve this medication for treatment of this previously untreatable disease that affects up to 50,000 people in the United States and causes significant blindness.