Alkeus Pharmaceuticals announced positive interim data from its TEASE-3 Stargardt disease study. Two additional participants who completed 24 months of therapy with oral gildeuretinol acetate showed no disease progression or decline in visual acuity, adding to the initial three participants who have now been on treatment for up to seven years with continued stability. This is the first clinical trial demonstrating such prolonged stability in early-stage Stargardt patients.
This news is crucial because Stargardt disease, a currently untreatable genetic disorder, leads to progressive and irreversible vision loss in children and young adults. Demonstrating that gildeuretinol acetate can halt disease progression, especially in early-stage patients, offers significant hope for preserving vision long-term and potentially altering the trajectory of this devastating condition. The extended follow-up data, showing sustained efficacy over seven years, further strengthens the drug’s potential to become a first-in-class treatment.
The TEASE-3 trial is an open-label study focusing on early-stage Stargardt patients who have not yet experienced vision loss symptoms. The data demonstrate that gildeuretinol acetate effectively prevented disease progression, measured by fundus autofluorescence imaging and other metrics, over two years and beyond. Seven patients have been enrolled in TEASE-3 to date, with five completing the initial two-year period and continuing treatment in an extension study. Alkeus also has other ongoing trials within its TEASE program, including TEASE-2, a placebo-controlled study in patients with moderate Stargardt disease.
This positive interim data reinforces the promise of gildeuretinol acetate as a potential treatment for Stargardt disease. Alkeus plans to submit a New Drug Application to the FDA in 2025 based on the combined results from its clinical trials. If approved, gildeuretinol acetate could become the first therapy for this serious unmet medical need, significantly impacting the lives of patients and families affected by Stargardt disease. Further research and continued monitoring of patients in the extension studies will be essential to fully understand the long-term benefits and safety profile of this promising therapy.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
