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.