Alkeus Pharmaceuticals Reports Positive Interim Results Showing No Disease Progression in Early-Stage Stargardt Patients Treated with Gildeuretinol

Alkeus Pharmaceuticals, Inc. has unveiled promising interim results from its TEASE-3 study, underscoring the potential of gildeuretinol acetate in treating early-stage Stargardt disease. The findings were presented at the ARVO 2024 Annual Meeting in Seattle. The data revealed that patients with early-stage Stargardt disease who were treated with gildeuretinol acetate showed no disease progression and remained symptom-free for the duration of the therapy, which spanned between two and six years.

Dr. Leonide Saad, President, and CEO of Alkeus Pharmaceuticals, expressed optimism about the results, noting that without treatment, patients would likely experience vision loss similar to their siblings with the same ABCA4 genetic mutations. The company is committed to advancing research to positively impact patients living with vision loss and plans to seek regulatory review for gildeuretinol later this year.

The TEASE-3 study is a pioneering clinical trial for early-stage Stargardt disease. This open-label study involves genetically confirmed patients who exhibit early signs of the disease visible through retinal imaging but have not yet shown symptoms of vision loss. The study uses retinal imaging and functional outcome measures to assess disease progression. Thus far, five patients have been enrolled, with each receiving a daily pill of gildeuretinol.

Dr. Michael B. Gorin from UCLA's David Geffen School of Medicine, who is part of the Departments of Ophthalmology and Human Genetics, praised the interim results for demonstrating the clinical benefits of gildeuretinol in early-stage Stargardt disease patients. He highlighted the agent’s potential to significantly slow disease progression and delay vision loss in young patients as a notable advancement.

Stargardt disease predominantly affects children and young adults and is a major cause of blindness. It affects approximately 30,000 individuals in the U.S. and over 150,000 worldwide, with no approved treatment currently available. The disease is characterized by a defective ABCA4 protein, which leads to the rapid formation of toxic vitamin A by-products that damage the retina, resulting in progressive vision loss.

The TEASE trials consist of four studies (TEASE-1, TEASE-2, TEASE-3, and TEASE-4) investigating gildeuretinol acetate (ALK-001) in Stargardt disease. TEASE-1 was a randomized, double-masked, placebo-controlled trial in 50 patients, showing a 21% reduction in the growth rate of retinal lesions. The ongoing TEASE-2 trial involves 80 patients and aims to provide topline data by 2025. TEASE-3 is focused on early-stage patients, while TEASE-4 is an open-label extension study.

Gildeuretinol acetate is a novel molecule designed to reduce vitamin A dimerization without affecting vision. Preclinical studies have shown that it can normalize the dimerization rate and prevent retinal degeneration and vision loss in animal models of Stargardt disease. The molecule has received breakthrough therapy and orphan drug designations from the U.S. FDA. Besides the TEASE trials, a Phase 3 (SAGA) study is assessing gildeuretinol in 200 patients with geographic atrophy secondary to age-related macular degeneration, with results expected in 2024.

Alkeus Pharmaceuticals, founded in 2010 and headquartered in Cambridge, Mass., is a private biopharmaceutical company focused on developing treatments for serious eye diseases with high unmet medical needs. Supported by Bain Capital Life Sciences, Alkeus is advancing its lead candidate, gildeuretinol acetate, through clinical trials for Stargardt disease and geographic atrophy secondary to age-related macular degeneration.