Gildeuretinol Shows Lasting Visual Improvements in Stargardt Disease, with Kenneth Fan, MD, MBA

Based on recent data, oral gildeuretinol 14 mg is safe and well-tolerated for the treatment of Stargardt disease. Additionally, best-corrected visual acuity (BCVA) exhibited little to no decline during the 7 years of treatment, supporting gildeuretinol’s ability to slow disease progression.1

Presented at the 43rd Annual Scientific Meeting of the American Society of Retina Specialists in Long Beach, CA, by Kenneth Fan, MD, MBA, Retina Consultants of Texas, these interim data were gathered during the TEASE-3 trial. TEASE-3 is an ongoing open-label, 24-month study and the third of 4 in the TEASE series of studies.1

TEASE-1, the first study to show gildeuretinol’s disease-slowing capabilities, was a multicenter 2-year phase 2 trial randomizing 50 patients with Stargardt Disease to either gildeuretinol or placebo. Data from this study provided the backbone of the following research, as it confirmed that vitamin A dimers contribute to the pathophysiology of Stargardt disease.2

TEASE-3 has a target enrollment of 20 patients. To be included, patients must be ≥8 years old, have a clinical diagnosis of Stargardt disease with genetic confirmation, and exhibit early signs of maculopathy on fundus autofluorescence (FAF) or optical coherence tomography (OCT) without well-delineated atrophic lesions. As of January 2025, 7 patients have been enrolled in the study, with 5 having completed the entire 24-month period.1

Among these 5 patients, BCVA exhibited little to no decline over the course of the study. Gildeuretinol was well-tolerated, with all but 1 treatment-emergent adverse events designated mild or moderate. The 1 severe event, a case of pelvic inflammatory disease, was deemed unrelated to the study drug. At this stage, no discontinuations have occurred.1

Fan sat down with HCPLive to discuss the results of the study and of the collective 7-year TEASE program, as well as plans for the upcoming TEASE-4 study. Fan discussed the functional endpoints of the study, including FAF imaging, photoreceptor density by OCT, and microperimetry.

“I actually think most of these imaging modalities and functional endpoints showed pretty good signals over the study,” Fan told HCPLive. “The visual acuity and microperimetry data were very compelling, because the study showed patients being treated with gildeuretinol demonstrated stability of vision as compared to historical sibling data.”

Fan also discussed the long-term safety of gildeuretinol, given the 7-year treatment duration in some patients over the course of all 3 TEASE trial phases to date.

“Only positive takeaways from that,” Fan said. “Hopefully, these types of treatments, which are less invasive than intravitreal treatments, can be taken for a very long period of time, and especially with patients who are starting these treatments at an earlier age.”

Fan also advised ophthalmologists to be proactive in recognizing, identifying, and diagnosing Stargardt disease, particularly given how close so many treatments are to their endpoints.

“I think the biggest thing for ophthalmologists and frontline eye care providers is to know, whether it’s gildeuretinol or some other emerging treatment, that something is likely to be coming for the treatment of Stargardt disease, relatively soon,” Fan explained. “Having a workflow where you are identifying Stargardt disease in your patients is really important, whether that’s through multimodal imaging or genetic testing in your clinic, or having a referral pattern where you send patients to genetic counselors and IRD specialists for that testing, will be critical.”

Editor's Note: Fan reports disclosures with Beacon Therapeutics, Kiora, Boehringer Ingelheim, Bayer, Eyepoint, Janssen, and Alkeus.

References

1. Fan K, Ferrone P, Gorin M, Saad L, et al. Oral Gildeuretinol Slows Disease Progression in Early Stargardt Disease: Updates from the TEASE-3 Study. Abstract presented at the 43rd Annual Scientific Meeting of the American Society of Retina Specialists in Long Beach, CA, July 30-August 2, 2025.

2. Scholl H, DeBartolomeo G, Washington I, Leonide S. ALK-001 (C20-D3-Vitamin A) slows the growth of atrophic lesions in ABCA4-related Stargardt Disease: Results of a Phase 2 placebo-controlled clinical trial (TEASE study). Invest. Ophthalmol. Vis. Sci. 2022;63(7):38. Accessed August 2, 2025. https://iovs.arvojournals.org/article.aspx?articleid=2779035