Summary 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.

Financial Data and Key Metrics Changes - The company currently has USD 157 million available, which is sufficient to complete all ongoing Phase III clinical trials [32] Business Line Data and Key Metrics Changes - The lead candidate, telarabant, is being evaluated in two Phase III trials named Dragon and Dragon II, with the first Dragon trial expected to complete in Q4 2025 [13][18] - The Dragon II trial is currently enrolling patients, with 16 out of a target of 60 patients already enrolled [19] Market Data and Key Metrics Changes - The estimated prevalence of Stargardt disease in the U.S. is between 43,000 and 59,000 patients, indicating a significant market opportunity [12] - The prevalence of Stargardt disease is approximately 1 in 6,500 for populations of European descent and 1 in 11,000 to 12,000 for East Asians [11] Company Strategy and Development Direction - The company aims to expedite the approval process for telarabant, having received breakthrough designation from the FDA based on interim data [15][17] - The company is also exploring opportunities in Japan, with the PMDA showing interest in telarabant's market authorization [21] Management's Comments on Operating Environment and Future Outlook - Management expressed high expectations for the final study data from the Dragon trial, which is anticipated by the end of the year [15] - The management is closely monitoring the regulatory environment and market conditions, particularly in relation to pricing policies [24] Other Important Information - Telarabant's mechanism of action is unique as it addresses vitamin A availability in photoreceptors, which is crucial for treating Stargardt disease [7][9] - The company is aware of competitors in the geographic atrophy space but believes telarabant could be transformative due to its oral administration [31] Q&A Session Summary Question: Does BELIBA currently have sufficient capital to complete all ongoing Phase III trials? - The company confirmed it has USD 157 million available, which is sufficient to complete all clinical trials currently running [32]