Belite Bio(US:BLTE) Globenewswire·2025-12-01 11:00Core Insights - Belite Bio announced successful topline results from the Phase 3 "DRAGON" trial of Tinlarebant, the first pivotal trial for treating Stargardt disease type 1 (STGD1), which currently has no approved treatments globally [1][3] Trial Results - The DRAGON trial enrolled 104 patients and met its primary efficacy endpoint, showing a statistically significant 36% reduction in the growth rate of retinal lesions compared to placebo, with a p-value of 0.0033 [2][6] - A post-hoc analysis indicated a consistent treatment effect with a p-value of <0.0001, reinforcing the efficacy of Tinlarebant [2][6] - The treatment also demonstrated a significant reduction in lesion growth in the fellow eye, with a 33.6% reduction (p = 0.041) [13] Safety Profile - Tinlarebant was well tolerated throughout the trial, with only four treatment-related discontinuations reported [6][20] - The safety profile was consistent with previous trials, with mild ocular adverse events such as xanthopsia and delayed dark adaptation [20] Regulatory Plans - Belite Bio plans to file a New Drug Application (NDA) with the US FDA in the first half of 2026 [6][9] - The drug has received multiple designations including Breakthrough Therapy and Orphan Drug Designation in the U.S., Europe, and Japan [9][17] Company Overview - Belite Bio is focused on developing novel therapeutics for degenerative retinal diseases, with Tinlarebant as its lead candidate [18] - The company is also evaluating Tinlarebant in ongoing trials for other retinal conditions, indicating a commitment to addressing significant unmet medical needs [18]