Company: BLTE
Filing Date: 2025-10-22
Form Type: F-3ASR
Source: 0001104659-25-101403
Chunk: 9

Company: BELITE BIO, INC
Filing Date: 2025-10-22
Form: F-3ASR
Chunk 9
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1 SAD study in 39 healthy adult subjects and one Phase 1 multiple ascending dose, or MAD, study in 32 healthy adult subjects in Australia. These studies involved 111 healthy adult subjects in total and evaluated the safety, toxicity, pharmacokinetics, or PK, and pharmacodynamics, or PD, of Tinlarebant.

Following completion of the Phase 1 studies, an open-label, dose-finding Phase 1b/2 clinical trial in adolescent STGD1 subjects was initiated in Australia and Taiwan. The study design included two portions: the Phase 1b portion was a 1-month dose finding study which enrolled 11 adolescent STGD1 subjects; the Phase 2 portion was a 24 month extension of the Phase 1b portion in which the 11 STGD1 subjects completing in Phase 1b were enrolled.

Two additional adolescent STGD1 subjects were also enrolled, giving a total of 13 adolescent STGD1 subjects for the Phase 2 portion. The PD data from the Phase 1b portion revealed that during repeated daily dosing, Tinlarebant can achieve a sustained mean RBP4 reduction of >70%, relative to baseline values. A total of 12 subjects completed the Phase 2 portion of the study (1 subject was lost to follow up at Month 12). The 24-month data continued to support Tinlarebant’s safety profile and showed no growth of atrophic

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retinal lesions (referred to as definitely decreased autofluorescence or DDAF) in 5 of 12 subjects. Based on data from the Phase 1b/2 study, a Phase 3 clinical trial named “DRAGON” in adolescent STGD1 patients was initiated. This study, which is a global, multi-center, randomized, double masked, placebo-controlled study designed to evaluate the safety and efficacy of Tinlarebant in the treatment of adolescent STGD1 patients, has completed enrollment of 104 subjects. On February 26, 2025, an independent Data and Safety Monitoring Board (DSMB) conducted a pre-specified interim analysis of the DRAGON trial which included an adaptive sample size re-estimation that would determine the need for an increase in sample size in order to enhance power. The interim analysis was performed when all subjects completed the one-year assessment. Following the interim analysis, the DSMB recommended the trial proceed without any modifications, which indicates that