Belite Bio (BLTE) H.C. Wainwright 28th Annual Global Investment Conference summary

Key clinical evidence and trial results

• FDA review of Tinlarebant for Stargardt disease is supported by DRAGON trial data and the large ProgStar natural history dataset, confirming treatment effects across similar patient stages.

• Fellow eye analysis in DRAGON showed a 33% effect size, statistically significant and consistent with the primary endpoint.

• Tinlarebant reduced DDAF lesion growth by 35.7% in DRAGON, with anatomical changes considered meaningful by clinicians due to established links between imaging and visual function.

• No significant differences in treatment effect were found across race, geography, sex, or age, supporting broad generalizability.

• Quantitative autofluorescence (qAF) analysis confirmed Tinlarebant's mechanism but is not yet clinically meaningful for individual patient monitoring.

Patient benefit, safety, and treatment considerations

• Slower atrophy progression is expected to translate to preserved vision over longer periods, though short-term trials cannot directly show this; imaging endpoints are FDA-accepted for this reason.

• Treatment is considered broadly applicable across all disease stages, including early and advanced cases, as supported by key opinion leaders.

• Tinlarebant showed excellent safety and tolerability, with mild, manageable side effects such as delayed dark adaptation and transient visual scene discoloration.

• High adherence is important for efficacy; Tinlarebant's half-life allows for occasional missed doses without significant loss of effect, but prolonged interruptions reduce benefit.

Commercial and financial outlook

• Approximately 20,000 U.S. patients are clinically diagnosed with Stargardt disease, with half confirmed by genetic testing and ready for treatment at launch.

• Payer research indicates strong support for coverage and rare disease pricing, with minimal restrictions expected if pricing aligns with expectations.

• Sufficient capital is available to support commercialization and ongoing development, with $780 million in cash and equivalents against projected $450 million in operating costs over three years.