Summary of Belite Bio Conference Call Company Overview - **Company**: Belite Bio - **Ticker Symbol**: BLTE (NASDAQ) - **Focus**: Development of therapeutics for Stargardt disease and age-related macular degeneration (AMD) Key Points Industry and Market Opportunity - **Stargardt Disease**: Most common inherited retinal dystrophy, affecting approximately 1 in 8,800 people, with an estimated 53,000 cases in the US and a larger population in China [9][10] - **Geographic Atrophy (GA)**: Advanced form of dry AMD, with around 1 million patients in the US [11] - **Market Gap**: No FDA-approved treatments for Stargardt disease and no orally approved treatments for GA [7] Drug Development - **Drug Name**: Timlerapat (also referred to as teleribat) - **Mechanism**: Designed to reduce toxic byproducts of vitamin A implicated in disease progression by binding to retinal binding protein 4 [7][18] - **Phase 3 Trials**: - **Dragon Study**: Completed with 104 subjects aged 12-20, focusing on Stargardt disease; top-line data expected in 3-4 weeks [5][39] - **PHOENIX Study**: Ongoing phase 3 study for GA with 530 subjects enrolled [6][55] Clinical Data and Efficacy - **Phase 2 Study Results**: Showed a 50% reduction in lesion growth in treated subjects compared to natural history data [37][38] - **Interim Analysis of Dragon Study**: Indicated a statistically significant treatment effect, leading to breakthrough therapy designation from the FDA [43] - **Safety Profile**: Withdrawal rate less than 10%, with ocular adverse events at less than 4% [44] Drug Design and Advantages - **Advantages of Timlerapat**: - Purpose-built antagonist of retinal binding protein 4, with improved bioavailability and potency compared to previous drugs like phenretinide [31][30] - Designed to be taken without regard to food, addressing compliance issues seen in earlier studies [30] Adverse Events - **Common Adverse Events**: - Xanthopsia (color perception changes) and delayed dark adaptation, both mild and transient [46][48] - No significant systemic adverse events reported, except for mild acne in some subjects [50] Future Outlook - **Positive Expectations**: Success in the Dragon study is expected to predict similar outcomes in the Dragon 2 and PHOENIX trials, reducing risk for investors [55] Conclusion Belite Bio is advancing its drug timlerapat through critical phase 3 trials for Stargardt disease and geographic atrophy, addressing significant unmet medical needs in the ophthalmology space. The company has demonstrated promising clinical data and a favorable safety profile, positioning itself for potential regulatory approval and market entry.

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.