Summary of Belite Bio (BLTE) FY Conference - August 13, 2025 Company Overview - **Company**: Belite Bio - **Focus**: Development of oral treatments for retinal diseases, specifically Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD) [12][31] Key Points and Arguments 1. **Leadership Team**: - Doctor Tom Lin, Chairman and COO, has over 10 years of executive management experience in biotech and has been involved in over 10 new drug developments [4] - Doctor Hendrick Scholl, Chief Medical Officer, has 25 years of experience in treating retinal diseases and has led significant research initiatives [5][6] - Doctor Nathan Mata, Chief Science Officer, has over 15 years of expertise in ophthalmic drug development [7] 2. **Pipeline Overview**: - **Tinlariband**: A novel once-daily oral tablet targeting serum retinal binding protein 4 (RBP4) to reduce retinol delivery to the eye, aimed at slowing or halting disease progression in Stargardt disease and geographic atrophy [11][17] - Ongoing clinical trials include: - **Dragon Trial**: A global registration trial for Stargardt disease with 104 subjects, focusing on slowing atrophic lesions [9][25] - **PHOENIX Trial**: A phase three trial for geographic atrophy, recently completed enrollment of 500 subjects [10][32] 3. **Market Opportunity**: - Significant unmet medical need for Stargardt disease, with no FDA-approved treatments available [12] - Breakthrough therapy, Fast Track, and orphan drug designations received in the US and EU for Stargardt disease [12] 4. **Clinical Trial Results**: - Phase two trial showed that tinlariband slowed lesion growth in Stargardt disease, with a significant reduction in RBP4 levels [21][24] - Interim analysis of the Dragon trial indicated that tinlariband is safe and well-tolerated, with a withdrawal rate of less than 10% [27] 5. **Safety Profile**: - Common adverse events included mild xanthopsia and delayed dark adaptation, with no severe treatment-related adverse events reported [29] - Overall, the treatment was well tolerated with no significant findings related to vital signs or organ functions [29] 6. **Visual Acuity Data**: - No significant decline in visual acuity was observed over the two-year duration of the Dragon trial, with average changes being minimal [30] 7. **Future Directions**: - Focus on early intervention for patients with small lesions to potentially slow disease progression [31] - Plans for further regulatory submissions based on interim analysis data [28] Additional Important Information - **Pathophysiology**: Stargardt disease and geographic atrophy share similar mechanisms involving the accumulation of toxic bisretinoids leading to retinal cell death [16][17] - **Regulatory Engagement**: Data from the interim analysis has been presented to various regulatory agencies, including the PMDA in Japan and the FDA [28] This summary encapsulates the critical insights from the conference, highlighting Belite Bio's strategic focus on innovative treatments for retinal diseases and the promising results from ongoing clinical trials.