Christine N. Kay, MD: TEASE Trials Show Promise of Gildeuretinol for Stargardt

At the 128th Annual American Academy of Ophthalmology (AAO) Meeting, Christine N. Kay, MD, a retina specialist specializing in inherited retinal diseases at Vitreo Retinal Associates in Gainesville, Florida, presented encouraging findings from the TEASE clinical trial program investigating gildeuretinol (ALK-001) for Stargardt disease.

Stargardt disease, a genetic form of juvenile macular degeneration, commonly affects young people, leading to severe vision loss and potential legal blindness over time. With no current US Food and Drug Administration (FDA)-approved treatments for Stargardt, gildeuretinol—an oral, vitamin A-derived drug—could offer new hope by aiming to slow disease progression and reduce retinal degeneration.

"Patients with this disease do develop that central blind spot and tend to lose their ability to drive in the state they live in, meeting criteria for legal blindness," Kay told HCPLive. "It would be an important target if we can develop a therapy to either halt progression or slow progression. There's currently no FDA-approved treatment for Stargardt disease and hopefully that will change the next few years."

The TEASE program comprises four studies, with TEASE-1 focusing on patients with advanced Stargardt disease marked by large atrophic retinal lesions. In this randomized study of 50 participants, patients receiving Gildeuretinol experienced a significant 21% reduction in the growth of these atrophic lesions compared with placebo. This effect is achieved by replacing natural vitamin A with Gildeuretinol’s modified form, which decreases toxic byproducts in the retina, thus preserving visual function and minimizing further damage.

Safety data from TEASE-1 were also positive, showing minimal adverse effects aside from a single case of papilledema in a patient on a high dose, which resolved without lasting complications. Kay indicated gildeuretinol’s impact on Stargardt disease progression was evident even without including natural history cases in the statistical analysis, strengthening the results' robustness. Additional trials within the TEASE program are investigating early intervention for younger patients, microperimetry testing in initial-stage patients, and pharmacokinetics in a broader cohort, with further data anticipated in 2025.

With US Food and Drug Administration (FDA) orphan drug status and breakthrough designation, Gildeuretinol is a promising candidate for addressing Stargardt disease's significant unmet needs. Kay expressed excitement about the trial’s outcomes and the potential for Gildeuretinol to become a viable treatment option pending FDA approval, marking a hopeful advancement in managing this debilitating disease.

"It was exciting to be able to give the first randomized, controlled trial that has shown efficacy in Stargardt presentation today," Kay told HCPLive. "This drug has orphan drug status and breakthrough designation status from the FDA, and the company is moving forward with the FDA pathway shortly, so we'll be excited to follow along and see how things go."

Disclosures: Relevant disclosures for Kay include Atsena Therapeutics, Alkeus Therapeutics, EyePoint, RegenxBio, and others.

References

Kay CN. Deuterated Vitamin A Preserves Vision in Stargardt’s Disease (TEASE Study). Symposium presented at the American Academy of Ophthalmology (AAO) 2024 Meeting. Chicago, Illinois. October 18-21, 2024.