Christine N. Kay, MD: Results of TEASE-1 Study on Stargardt Disease


At AAO 2023, Kay shares insight into the topline results of the TEASE-1 study investigating the effect of gildeuretinol (ALK-001) on the inherited retinal disease.

New topline data from the TEASE-1 study revealed atrophic lesion growth reduction among patients with ABCA4-related Stargardt Disease treated with gildeuretinol (ALK-001), a novel, selectively modified version of vitamin A.1

The findings, presented at the 127th Annual American Academy of Ophthalmology (AAO) Meeting in San Francisco, California, showed a 21% reduction in the growth of atrophic lesions in individuals treated with gildeuretinol, as opposed to the placebo-treated control arm. Among the untransformed areas, there was a 28% reduction in patients treated with gildeuretinol.

“We have an unmet need with no current US Food and Drug Administration (FDA) approved therapy yet for Stargardt Disease. These patients rapidly go blind to a visually legally blind status over a 10-year period, usually in late childhood, early adolescence,” said Christine N. Kay, MD, the director of electrophysiology, retinal genetics, and clinical trials at Vitreoretinal Associates in Gainesville, Florida, in an interview with HCPLive. “It is a visually disabling disease that we would like to be able to treat and halt progression, if possible.”

Stargardt disease affects more than 30,000 people in the United States and is one of the most common genetic causes of blindness in children and young adults without a current treatment.2 In patients with Stargardt, the ABCA4 protein is defective or absent allowing excess vitamin A dimerization, resulting in toxic by-products that damage the retinal pigment epithelium layer and lead to progressive vision loss.

The TEASE-1 study is a randomized-controlled, double-masked clinical trial evaluating oral gildeuretinol (ALK-001) and the growth of atrophic lesions among patients with Stargardt Macular Dystrophy. The therapy is in clinical development to selectively address the underlying mechanism of Stargardt disease and reduce the rate of vitamin A dimerization in the eye.

In TEASE-1, 30 patients were randomized to gildeuretinol treatment and 20 patients to the control arm, with 10 of these patients switching to active treatment at 12 months.1 The trial augmented the control arm by including 59 natural history cases with Stargardt macular dystrophy.

Safety signals revealed the drug was well-tolerated, with effects expected from a Vitamin A drug. A post-hoc analysis investigating microperimetry to determine visual function also revealed an 80% reduction in the loss of retinal sensitivity in individuals treated with gildeuretinol, as opposed to placebo, suggesting a benefit in function.

The FDA has granted Breakthrough Therapy Designation and Orphan Drug Designation to gildeuretinol for the treatment of Stargardt Disease.

For more insight into the TEASE-1 results, watch the full interview with Kay in the video above.


  1. PA025 ALK-001 (C20-D3-Vitamin A) Slows the Growth of Atrophic Lesions in ABCA4-Related Stargardt Disease: Results of a Randomized, Placebo-Controlled Clinical Trial, the TEASE Study. Presented at the 2023 American Academy of Ophthalmology Annual Meeting, November 3 – 6, 2023.
  2. Transformation in Sight. Alkeus Pharmaceuticals Inc. Accessed November 17, 2023.
Related Videos
What Makes JAK Inhibitors Safe in Dermatology
Potential JAK Inhibitor Combination Regimens in Dermatology
Therapies in Development for Hidradenitis Suppurativa
"Prednisone without Side Effects": The JAK Inhibitor Ceiling in Dermatology
Discussing Changes to Atopic Dermatitis Guidelines, with Robert Sidbury, MD, MPH
Ghada Bourjeily, MD: Research Gaps on Sleep Issues During Pregnancy
John Winkelman, MD, PhD: When to Use Low-Dose Opioids for Restless Legs Syndrome
Bhanu Prakash Kolla, MBBS, MD: Treating Sleep with Psychiatric Illness
How Will Upadacitinib, Povorcitinib Benefit Hidradenitis Suppurativa?
© 2024 MJH Life Sciences

All rights reserved.