Alkeus Pharmaceuticals has reported the latest interim data from TEASE-3 trial where gildeuretinol acetate demonstrated to stall the progression of early-stage Stargardt disease.
The open-label study is designed to assess gildeuretinol’s impact on patients with early-stage Stargardt disease.
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These individuals, genetically confirmed to have the disease, exhibit early signs on retinal imaging but have not yet experienced vision loss symptoms.
Each participant has a sibling with the same genetic mutations who has already suffered irreversible vision loss due to the disease.
The primary endpoint of the study is to monitor disease progression over a two-year period, using retinal imaging and functional outcome measures.
Following this initial period, the six enrolled patients continue to receive daily doses of gildeuretinol as part of an open-label long-term extension study.
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By GlobalDataAccording to the findings, patients treated with this new molecule remained asymptomatic over periods ranging from two to six years.
Gildeuretinol acetate, also known as ALK-001, is a deuterated form of vitamin A, specifically engineered to reduce vitamin A dimerization without affecting vision.
Preclinical studies have shown that gildeuretinol reduces the rate of vitamin A dimerization to normal levels and prevents retinal degeneration in animal models of Stargardt disease.
The US Food and Drug Administration granted breakthrough therapy and orphan drug designations for the therapy earlier.
Additionally, a Phase III SAGA study is underway to evaluate gildeuretinol in patients with geographic atrophy secondary to age-related macular degeneration, with topline data expected this year.
Alkeus Pharmaceuticals chief medical officer Seemi Khan said: “Interim results from the TEASE-3 study of gildeuretinol in early-stage Stargardt patients indicate the potential value of treating patients with confirmed disease-causing ABCA4 genetic mutations as early as possible before the disease causes progressive loss of central vision.
“There is high unmet need in this disease area with no existing treatment, and we look forward to sharing additional results from this encouraging study in the future as we work to advance our development programme, which has the potential to be the first therapy for Stargardt disease.”
