Belite Bio Scores First Successful Phase 3 Trial For Rare Vision-Loss Disease

Core Insights - Belite Bio Inc. announced successful topline results from the Phase 3 DRAGON trial of Tinlarebant for treating Stargardt disease type 1 (STGD1), marking a significant milestone in the development of therapies for this condition [1] Group 1: Trial Results - The Phase 3 DRAGON trial enrolled 104 patients and demonstrated a statistically significant reduction in lesion growth rate of 35.7% compared to placebo (p-value of 0.0033) [3] - A treatment effect was also observed in the fellow eye, with a 33.6% reduction in lesion growth (p = 0.041) [3] - Tinlarebant slowed decreased autofluorescence (DAF) lesion growth by 33.7% in the study eye (p = 0.027) and 32.7% in the fellow eye (p = 0.017) [4] Group 2: Mechanism of Action - Tinlarebant reduces the accumulation of vitamin A-based toxins (bisretinoids) by modulating retinol transport to the eye, specifically by reducing serum retinol binding protein 4 (RBP4) levels [2] - The 5 mg daily dose achieved an approximate 80% reduction in RBP4 levels relative to baseline, with levels returning to 84% of baseline at the end of the study [4] Group 3: Safety and Future Plans - The safety profile of Tinlarebant was consistent with previous reports, with only four treatment-related discontinuations noted [6] - The company plans to engage regulatory authorities and submit New Drug Applications for Tinlarebant in the first half of 2026 [6] Group 4: Market Reaction - Following the announcement, Belite Bio Inc.'s stock rose by 11.3%, reaching $153.05 during premarket trading [6]