Tinlarebant is the first therapeutic candidate to demonstrate clinical efficacy in a global Phase 3 trial for Stargardt disease, achieving a statistically significant p-value of 0.0033Tinlarebant met the primary efficacy endpoint, demonstrating clinical benefit by significantly reducing the lesion growth rate by 36% compared to placebo, as measured by retinal imagingTinlarebant was well tolerated throughout the trial Stargardt disease impacts more than 50,000 patients in the U.S. Belite Bio plans to file an ...

Core Insights - Belite Bio, Inc. has received acceptance from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) for a Conditional Marketing Authorization application for Tinlarebant, aimed at treating Stargardt disease based on interim analysis results from the Phase 3 DRAGON trial [1][3][8] Company Overview - Belite Bio is a clinical-stage drug development company focused on novel therapeutics for degenerative retinal diseases with significant unmet medical needs, including Stargardt disease and geographic atrophy in advanced dry age-related macular degeneration [6] - The lead candidate, Tinlarebant, is an oral therapy designed to reduce the accumulation of vitamin A-based toxins in the eye, which are implicated in retinal diseases [5][6] Clinical Trial Details - The Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled study evaluating the safety and efficacy of Tinlarebant in 104 adolescent patients with Stargardt disease across 11 jurisdictions [4] - The primary efficacy endpoint of the trial is the growth rate of atrophic lesions, alongside safety and tolerability assessments [4] Regulatory Progress - The interim analysis results from the DRAGON trial met the criteria for the Conditional Marketing Authorization application, with final topline data expected to be reported in Q4 2025 [3][8] - The company is optimistic about the global submissions and potential approvals based on consistent feedback from major regulatory agencies [2]