Belite Bio (NasdaqCM:BLTE) FY Conference Transcript

Summary of the Conference Call on Belite Bio and Tinlarebant Company Overview - Company: Belite Bio - Product: Tinlarebant - Indication: Stargardt disease Key Points Development Status - The Dragon trial is a two-year international registration trial involving 104 patients with Stargardt disease, with top-line data readout expected by the end of Q4 2023 and an NDA submission planned for the first half of 2026 [3][6] Efficacy and Safety Data - Tinlarebant has shown efficacy in preventing the development of atrophic lesions (DDAF) and slowing their growth by almost 50% compared to natural history controls [4] - The overall withdrawal rate due to ocular adverse events is less than 4%, indicating an excellent safety profile [5] Regulatory Strategy - The FDA granted breakthrough designation based on interim analysis data, allowing for expedited regulatory review [6] - Full approval will be sought in the U.S., Europe, Japan, and other markets, with a second registration trial (Phoenix trial) for geographic atrophy (GA) already fully enrolled with 530 patients [14][22] Market Potential - The estimated prevalence of Stargardt disease is approximately 1 in 7,000, translating to about 47,000 to 59,000 patients in the U.S. alone, with a potential market penetration of around 40% [8][9] - The company anticipates that Tinlarebant will be beneficial for both pediatric and adult patients, with no age restrictions on the label [8] Competitive Landscape - NanoScope Therapeutics' gene therapy is viewed as a complementary approach rather than a direct competitor, as it targets different aspects of vision restoration [10][11][13] Future Catalysts - Key upcoming milestones include: - Q4 2025: Full enrollment of the Dragon 2 trial - End of Q4 2025: Top-line data readout for the Dragon trial - First half of 2026: NDA submission anticipated [23] Financial Position - Belite Bio has approximately $160 million in cash, providing a four-year runway for ongoing programs, bolstered by a recent private placement financing of up to $275 million [22] Patent Protection - The company holds 14 active patent families, primarily composition of matter patents, which will last until at least 2040 [21] Go-to-Market Strategy - The company plans to prioritize the commercialization of Tinlarebant for Stargardt disease due to its orphan indication status, which allows for higher pricing and easier access to patients through specialized networks [18] Correlation with Geographic Atrophy - There is a belief that Tinlarebant's efficacy in Stargardt disease may correlate positively with its efficacy in GA, as both conditions share common pathological features [16][17] Additional Insights - The company is strategically positioned to leverage its connections with retinal specialists and inherited retinal disease specialists to facilitate the commercialization of Tinlarebant [18]