- Gildeuretinol acetate demonstrated less loss in ellipsoid zone (EZ) area compared to historical sibling controls.
- Early-stage Stargardt disease patients treated with gildeuretinol for two to seven years exhibited relatively stable disease over the course therapy.
- Gildeuretinol was well tolerated and had a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate.
CAMBRIDGE, Mass., Aug. 07, 2025 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc. today announced the presentation of an interim data update from its TEASE-3 study demonstrating that five early-stage Stargardt disease patients treated with investigational oral gildeuretinol acetate experienced less loss in ellipsoid zone (EZ) area compared to historical sibling controls and overall disease remained relatively stable while on therapy ranging between two to seven years. The study update was presented during the 43rd American Society of Retina Specialists (ASRS) Annual Scientific Meeting, held July 30 – Aug. 2 in Long Beach, Calif.
“We are encouraged by this new interim data from the TEASE-3 study of gildeuretinol in early-stage Stargardt patients with confirmed disease-causing ABCA4 genetic mutations,” said Seemi Khan, M.D., M.P.H., M.B.A., Chief Medical Officer of Alkeus Pharmaceuticals. “These additional data further support the potential of investigational gildeuretinol to slow disease progression and stabilize vision in individuals living with Stargardt disease. We are excited to continue advancing our efforts towards delivering a treatment for Stargardt disease.”
TEASE-3, the first clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol in genetically confirmed patients who exhibit early retinal changes on imaging but have not yet developed meaningful symptoms of vision loss. TEASE-3 study participants each have a sibling who was previously diagnosed with Stargardt disease.
The disease progression over two years is assessed by retinal imaging and functional outcome measures. After the initial two-year treatment period, patients continue to receive gildeuretinol while enrolled in an open label long-term extension study. In the TEASE-3 study, five patients have completed two years of treatment with once-daily oral gildeuretinol.
Gildeuretinol was well tolerated and demonstrated a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate in severity.
“Time is of the essence for patients losing vision from early-onset Stargardt disease,” said Kenneth Fan, M.D., M.B.A., Retina Consultants of Texas. “With no approved treatments available, these data inform our understanding of retinal health in early-stage disease and underscore the importance of ongoing clinical evaluation.”
About Stargardt Disease
Stargardt disease is a serious cause of blindness in children and adults, with an estimated 30,000 people affected in the U.S. and more than 150,000 worldwide. There is no approved treatment. In individuals with Stargardt disease, the ABCA4 protein is defective. This defect in the protein results in the accelerated dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss.
About Alkeus Pharmaceuticals
Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases. Based in Cambridge, Mass., Alkeus is backed by institutional investors led by Bain Capital Life Sciences. Alkeus is developing therapies for serious diseases of the eye with high unmet need. Alkeus’ breakthrough-designated lead candidate, oral gildeuretinol acetate (ALK-001), is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration.
About Gildeuretinol Acetate (ALK-001)
Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. Gildeuretinol is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. Gildeuretinol has received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for Stargardt disease from the U.S. Food and Drug Administration.
About the TEASE Program
The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol acetate (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in 50 patients with advanced Stargardt disease and is complete. The TEASE-2 trial is a randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025. TEASE-3, a clinical trial in early-stage Stargardt disease, is an ongoing open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing meaningful symptoms of vision loss. TEASE-4 is an ongoing open-label extension study.
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