Alkeus publishes latest interim data from Stargardt disease therapy trial

Alkeus Pharmaceuticals has reported interim data from the TEASE-3 trial showing that gildeuretinol acetate effectively halts early-stage Stargardt disease progression.

This open-label study, involving six patients with early-stage Stargardt and their siblings who have irreversible vision loss, measures disease progression over two years using retinal imaging and functional tests. Following this, patients continue to receive daily gildeuretinol doses in a long-term study.

Results indicate that those on gildeuretinol remained asymptomatic for up to six years. Gildeuretinol acetate (ALK-001), a deuterated form of vitamin A, aims to reduce vitamin A dimerization without affecting vision. Preclinical trials show it normalizes vitamin A dimerization and prevents retinal degeneration in Stargardt models.

The FDA has granted breakthrough therapy and orphan drug designations for gildeuretinol. A Phase III SAGA study is also underway to assess its effectiveness for geographic atrophy from age-related macular degeneration, with results due this year.