Today at the 2024 meeting of the American Society of Retina Specialists in Stockholm, Michael Gorin, MD, PhD, presented late-breaking data on the TEASE-3 trial of oral gildeuretinol acetate (ALK-001; Alkeus Pharmaceutical) for Stargardt disease. The TEASE trials consist of 4 independent clinical studies of gildeuretinol in Stargardt disease, TEASE-1, TEASE-2, TEASE-3, and TEASE-4. TEASE-3 is an open-label study assessing gildeuretinol in early-stage Stargardt patients.
Dr. Gorin presented data on 3 patients from the TEASE-3 study of a small group of 6 patients who are asymptomatic younger siblings of individuals with Stargardt disease identified in a natural history study. The older siblings’ data served as control. The patients were treated with gildeuretinol and monitored every 6 months. The area of photoreceptor damage was significantly smaller in these patients than in their older siblings. The progression of photoreceptor damage was vastly smaller than what their older siblings experienced, said Dr. Gorin. The older siblings had experienced loss of vision at the age of their younger siblings, but the patients in TEASE-3 maintained 20/20 vision or better. There were no serious or severe adverse events and no reports of dark adaptation delays or low-luminance issues.
“The TEASE-3 study has been a unique opportunity to evaluate gildeuretinol in a small group of presymptomatic children for whom we had clinical history information regarding the course of Stargardt disease in older siblings,” Dr. Gorin told Retinal Physician. “We were able to assess the ability of the medication to reduce vision loss in these children as compared to the age-matched data of their genetically matched Stargardt siblings. The dramatic delay of vision and photoreceptor loss in this small cohort is an exciting advancement in the therapy for ABCA4-related Stargardt disease and suggests that the clinical benefits may be greater at the earliest stages of this condition.”
Gildeuretinol acetate is a specialized form of deuterated vitamin A designed to reduce the dimerization of vitamin A without disrupting vision. In addition to the TEASE trials, a phase 3 study of gildeuretinol in 200 patients with geographic atrophy secondary to age-related macular degeneration has recently been completed and is expected to read out topline data in 2024. Gildeuretinol has received breakthrough therapy designation and orphan drug designation by the US Food and Drug Administration.
