New findings presented at the 42nd American Society of Retina Specialists (ASRS) Annual Scientific Meeting suggest that gildeuretinol acetate may effectively pause disease progression in patients with early-stage Stargardt disease. The data, unveiled from the TEASE-3 trial by Alkeus Pharmaceuticals, marks a significant advancement in the pursuit of treating this genetic disorder.
Principal investigator Michael B. Gorin, MD, PhD, highlighted the study’s unique approach in evaluating gildeuretinol among three presymptomatic children with a family history of Stargardt disease. The trial monitored these patients over two years and observed a notable delay in vision and photoreceptor loss compared to genetically-matched siblings who were symptomatic.
The TEASE program, consisting of four trials (TEASE-1, -2, -3, and -4), focuses on assessing the efficacy of gildeuretinol acetate in treating Stargardt disease. TEASE-1, presented previously, demonstrated promising results in reducing the progression of retinal lesions, laying the groundwork for subsequent trials.
TEASE-3 specifically targets early-stage Stargardt disease and has enrolled six patients to date, aiming to assess disease progression through retinal imaging and functional outcomes over a two-year period. Interim data from three patients aged 13 and 17 years indicated a maintained mean visual acuity throughout the trial, with promising trends in retinal sensitivity and photoreceptor density over time.
Seemi Khan, MD, MPH, MBA, Chief Medical Officer of Alkeus Pharmaceuticals, emphasized the high unmet need in Stargardt disease treatment and the potential of gildeuretinol acetate to become the first therapeutic option. Khan underscored the importance of early intervention in managing ABCA4-related mutations before significant vision loss occurs, highlighting the study’s ongoing contributions to advancing treatment options for this challenging condition.
As the TEASE-3 trial progresses and additional data becomes available, researchers remain optimistic about the future implications of gildeuretinol acetate in reshaping the therapeutic landscape for Stargardt disease.