Alkeus Pharmaceuticals, Inc., announced positive data from its TEASE-1 study, a randomized, double-masked, placebo-controlled clinical trial investigating the effects of gildeuretinol (ALK-001) in patients with Stargardt disease. Christine Kay, M.D., of Vitreo Retinal Associates Florida, will present the trial results during the 2023 American Academy of Ophthalmology (AAO) Retina Subspecialty Day.
Dr. Kay’s presentation is titled “Gildeuretinol (ALK-001) Slows the Growth of Atrophic Lesions in ABCA4-Related Stargardt Disease Results of a Randomized, Placebo-Controlled Clinical Trial (“TEASE-1” study).”
Gildeuretinol slowed the growth rate of atrophic retinal lesions by 21% during the two-year study (square root transformation analysis) or 28% using untransformed atrophic areas. The growth rates of atrophic retinal lesions were 0.19 mm/year (0.90 mm²/year untransformed area) with gildeuretinol and 0.24 mm/year (1.25 mm²/year) in the untreated arm, mean difference 0.05 mm/year (0.35 mm²/year) (95% confidence interval, 0.03 to 0.07, p<0.001, R² = 0.9996). In a post-hoc comparison, gildeuretinol-treated patients had an 80% slower rate of retinal sensitivity loss compared to the 330 eyes ProgStar natural history (0.4 dB/year loss in gildeuretinol vs. 2.2 dB/year in ProgStar). In total, over 150 patients have been treated with gildeuretinol for over a year in several studies. The longest treatment duration with gildeuretinol has been five years. In these studies, gildeuretinol has been well tolerated.
“These results underscore the potential of gildeuretinol to transform the lives of patients living with Stargardt disease, a genetic eye condition that leads to significant vision loss and currently has no approved treatment,” said Leonide Saad, Ph.D., Co-Founder, President and CEO of Alkeus Pharmaceuticals.
Stargardt disease is a leading genetic cause of blindness in children and young adults, with an estimated 30,000 people affected in the U.S. and more than 150,000 worldwide. In individuals with Stargardt disease, the ABCA4 protein is defective. Loss of the protein results in the accelerated dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss.
“I have been involved as a principal investigator with the Alkeus TEASE trials since their inception,” said Dr. Kay. “It is extremely satisfying and humbling to have the opportunity to provide positive data at the Academy regarding the TEASE-1 trial. I think gildeuretinol has the capacity to benefit patients with Stargardt disease in a clinically-meaningful way. I am hopeful gildeuretinol will be our first FDA-approved therapy for Stargardt disease, one of the most common inherited retinal diseases.”
The TEASE trials consist of four independent clinical trials evaluating gildeuretinol in Stargardt disease. Gildeuretinol addresses the underlying cause of Stargardt disease, by selectively reducing the rate of vitamin A dimerization in the eye. In the TEASE-1 trial, 50 patients with Stargardt disease were randomized 3:2 to gildeuretinol or placebo once daily. After one year of treatment, 50% of placebo patients were randomly selected to cross over to gildeuretinol. The placebo arm was augmented with 59 natural history cases. The primary outcome measure was the growth rate of atrophic retinal lesions.
“BrightFocus Foundation provided early funding for this important research and is hopeful for the life-enhancing option it may provide for people living with Stargardt disease,” said Stacy Pagos Haller, president and CEO of BrightFocus Foundation, a global nonprofit organization that funds eye and brain research.