Belite Bio's Q4 2024 Earnings Call: Discrepancies in Regulatory Strategies, Discontinuation Rates, and Trial Design

These are the key contradictions discussed in Belite Bio's latest 2024Q4 earnings call, specifically including: DSMB recommendations and regulatory strategies, discontinuation rates in clinical trials, study population and trial design, and regulatory strategy:



Phase 3 Trials Progress:
- Belite Bio has made significant progress in its Phase 3 trials for Tinlarebant, with the DRAGON trial completing an interim analysis and the DSMB recommending no sample size increase.
- The progress is attributed to the strong trial design and promising interim results, which indicate potential efficacy.

Interim Analysis and Regulatory Review:
- The Data Safety Monitoring Board (DSMB) recommended submission of interim data for further regulatory review and drug approval for Tinlarebant in Stargardt disease.
- This positive outcome is a result of the DSMB's confidence in the interim analysis and possible efficacy signals.

Sample Size Increase in GA Trial:
- Belite Bio plans to increase the sample size in the Phase 3 PHOENIX trial for Geographic Atrophy from approximately 450 to 500 subjects.
- This decision is to further boost the chances of success, leveraging the smooth enrollment rate, and maintaining feasibility within the expected timeline.

Adolescent Stargardt Disease Population:
- The current trial population for Stargardt disease consists of adolescents, which represents a significant portion of the real-world diagnosed population.
- The decision to focus on this age group is based on the typical onset of the disease and the potential to approve the drug for a broader patient population with similar disease progression.