Vitamin A replacement may slow atrophic lesion growth in Stargardt disease
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CHICAGO — ALK-001, a vitamin A replacement, helped slow the growth rate of atrophic lesions in patients with Stargardt disease, according to a study presented at the American Academy of Ophthalmology meeting.
Christine Kay, MD, said supply of vitamin A is critical for vision, but it can form toxic by-products as a result of the visual cycle in patients with Stargardt disease.
“Eliminating or slowing vitamin A is not a good option as it leads to night blindness, delayed dark adaptation and possibly long-term retinal degeneration or other ocular and systemic side effects,” Kay said. “ALK-001 is a vitamin A replacement and retains vitamin A’s natural functions while allowing the visual cycle to continue normally.”
In the TEASE study, 50 patients with Stargardt disease were randomly assigned to undergo treatment with either one pill of ALK-001 per day (Alkeus Pharmaceuticals) (30 patients) or one placebo pill per day (20 patients) for 24 months. At 1 year, 10 patients in the placebo group were randomly selected to switch to the treatment arm.
The primary efficacy endpoint of the study was the growth rate of well-defined atrophic lesions.
Kay said patients in the treatment arm experienced a 21% slower growth rate than patients in the placebo arm (P < .001), which equated to a difference in mean area estimate of 0.35 mm2 per year between the two groups. Patients in the ALK-001 group also had a 34% slower growth rate of atrophic lesions toward the foveal center (P < .001).
ALK-001 had the same safety profile as natural vitamin A, and no dark adaptation delays, night blindness or significant changes in visual acuity were observed.
Kay said ALK-001 is the first treatment to show efficacy in Stargardt disease.
“ALK-001 shows safe slowing of the growth of atrophic lesions in ABCA4 Stargardt,” she said. “We may have our first promising therapeutic for the treatment of Stargardt.”
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Kay C. ALK-001 (C20-D3-vitamin A) slows the growth of atrophic lesions in ABCA4-related Stargardt disease. Presented at: American Academy of Ophthalmology meeting; Sept. 30-Oct. 3, 2022; Chicago.
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