Philip J. Ferrone, MD, Vitreoretinal Consultants of New York, discusses results from the TEASE-2 clinical trial of gildeuretinol in patients with Stargardt disease.
Stargardt disease is a genetic eye disorder that causes progressive vision loss. It affects the macula, an area of the retina responsible for sharp, central vision. Vision loss is due to abnormal accumulation of lipofuscin in the cells within the macula. Patients with Stargardt disease also have problems with night vision, and some have problems with color vision. It is most commonly caused by genetic changes in the ABCA4 gene. There are currently no available treatments for this condition.
Gildeuretinol is an investigational compound designed to reduce the dimerization of vitamin A without modulating the visual cycle. TEASE-2 was a randomized, double-masked, placebo-controlled study in 79 patients with moderate Stargardt disease. Results from the TEASE-2 clinical trial (NCT02402660) of gildeuretinol for the treatment of moderate Stargardt disease were recently presented at the 2025 American Academy of Ophthalmology (AAO) meeting.
Of those treated with gildeuretinol, there was a 28% reduction in progression of disease. However, due to the heterogeneity of the patient population enrolled, the p-value was not statistically significant. As Dr. Ferrone notes, these results still show great promise in the potential of this treatment in Stargardt disease. Additionally, the safety profile of gildeuretinol is favorable with limited side effects and adverse events.
Next steps include further analysis of the TEASE-2 population and ongoing TEASE-3 and TEASE-4 studies hope to bring data to the U.S. Food and Drug Administration.
To learn more about Stargardt disease and other rare eye conditions, visit https://checkrare.com/diseases/ophthalmology-eye-diseases/