Belite Bio announced positive top-line results from its 104-patient Phase 3 Dragon study: oral tinlarebant met the primary endpoint by significantly slowing the growth rate of retinal lesions in Stargardt disease type 1 versus placebo. The trial measured lesion progression by fundus autofluorescence and reported a clinically meaningful reduction. Tinlarebant operates by lowering serum retinol-binding protein 4 to reduce toxic vitamin A cycle intermediates implicated in Stargardt pathophysiology. Belite plans to use the Phase 3 data to pursue regulatory submissions and a commercial strategy for the first disease-modifying oral therapy in a previously untreatable inherited retinal disorder. The readout shifts attention onto regulatory review timelines, potential label claims, and payers’ assessment of long-term benefit in a rare ophthalmology indication.
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