Belite Bio reported positive top-line results from its 104-patient Phase III DRAGON study of oral tinlarebant in Stargardt disease type 1, meeting the primary endpoint with a statistically significant reduction in retinal lesion growth versus placebo. The company said it will add the U.S. to its planned regulatory filings. The oral small molecule reduces serum retinol-binding protein 4 to slow retinal degeneration; the trial showed a 36% slowing of lesion growth. Belite positions tinlarebant as the first potential therapeutic for STGD1 and is preparing regulatory engagement and submission planning.
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