Once-Daily Gildeuretinol Acetate Shows Promise in Halting Early-Stage Stargardt Disease Progression

New data from the 42nd American Society of Retina Specialists (ASRS) Annual Scientific Meeting suggests that gildeuretinol acetate could pause disease progression in patients with early-stage Stargardt disease. The TEASE-3 trial, the first clinical trial in early-stage Stargardt disease, detailed the effects of gildeuretinol among 3 patients who had been receiving the once-daily oral agent for at least 2 years.

Principal investigator Michael B. Gorin, MD, PhD, highlighted the unique opportunity to evaluate gildeuretinol in presymptomatic children with a family history of Stargardt disease. The study compared the medication's ability to stall vision loss in these children against age-matched data of their genetically-matched Stargardt siblings, showing a dramatic delay in vision and photoreceptor loss.

The TEASE program by Alkeus Pharmaceuticals includes four clinical trials, with TEASE-3 focusing on early-stage Stargardt disease. The trial's primary endpoint is disease progression over 2 years, assessed by retinal imaging and functional outcome measures. Data presented at ASRS 2024 from 3 patients within the trial showed that mean retinal sensitivity declined during the first year of treatment but improved in the second year. Two patients experienced a reduction in photoreceptor area initially, followed by an increase in photoreceptor density.

Investigators noted that the cohort's mean visual acuity was maintained at 88 ETDRS letters during the 2-year trial, with visual acuity preserved for an additional 4 years in two patients who continued in the open-label extension trial. No serious adverse events were reported during the trial.

Seemi Khan, MD, MPH, MBA, chief medical officer of Alkeus Pharmaceuticals, emphasized the potential value of treating patients with confirmed ABCA4 genetic mutations as early as possible to prevent progressive loss of central vision. The TEASE-3 study's interim results highlight the unmet need in this disease area and the potential of gildeuretinol acetate as the first therapy for Stargardt disease.