Belite Bio(US:BLTE)2025-12-01 14:02Summary of Belite Bio's Phase Three DRAGON Study Conference Call Company and Industry - Company: Belite Bio - Industry: Biotechnology, specifically focusing on treatments for retinal diseases, particularly Stargardt disease Core Points and Arguments 1. Study Overview: The DRAGON Study is a phase three trial evaluating Tinlarebant for treating Stargardt disease, the largest interventional trial conducted in adolescent patients with this condition [2][4][6] 2. Significant Results: Tinlarebant demonstrated a statistically significant and clinically meaningful reduction in the annualized rate of lesion growth in Stargardt disease, achieving a treatment effect size of 36% compared to placebo [17][24] 3. Mechanism of Action: Tinlarebant is an orally administered small molecule that targets retinal binding protein 4 (RBP4) to reduce vitamin A delivery to the eye, thereby slowing disease progression [5][24] 4. Patient Impact: The slowing of lesion growth is expected to lead to a longer preservation of vision, allowing patients to maintain daily activities longer [26][32] 5. Safety Profile: The treatment maintained an excellent safety and tolerability profile over the two-year study period, with no serious treatment-related adverse events reported [22][23][33] 6. Regulatory Pathway: Belite Bio plans to submit the full data package to regulatory agencies, with confidence that the DDAF (definitely decreased autofluorescence) endpoint will be accepted for approval [41][48] Additional Important Content 1. Epidemiology: Stargardt disease affects approximately 53,000 people in the U.S. and over 100,000 in Europe and China, making it one of the most prevalent inherited retinal diseases [8][57] 2. Visual Acuity Findings: Best-corrected visual acuity did not show significant changes over the study duration, which aligns with the natural history of Stargardt disease [20][21] 3. Fellow Eye Data: Statistically significant treatment effects were also observed in the fellow eye, providing strong confirmatory evidence for the treatment's efficacy [18][52] 4. Market Potential: The estimated patient population for Stargardt disease is around 50,000 in the U.S., with expectations of increased diagnosis rates following the introduction of a treatment [56][57] 5. Future Directions: The company aims to transform the management of Stargardt disease by shifting focus to earlier diagnosis and treatment, potentially before significant vision loss occurs [35][36] This summary encapsulates the key findings and implications from the conference call regarding Belite Bio's Tinlarebant and its potential impact on the treatment of Stargardt disease.