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Belite Bio (BLTE) investor relations material

Belite Bio Study Result summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Study Result summary1 Dec, 2025

Study background and design

  • The DRAGON Phase 3 trial was a 24-month, multicenter, randomized, double-masked, placebo-controlled study in adolescents aged 12–20 years with Stargardt disease and at least one ABCA4 mutation, enrolling 104 patients globally.

  • Participants were randomized 2:1 to receive 5 mg/day oral Tinlarebant or placebo, with balanced baseline characteristics between groups.

  • The primary endpoint was the annualized rate of lesion growth in definitely decreased autofluorescence (DDAF) at 25 months, with key secondary endpoints including total decreased autofluorescence (DAF), best-corrected visual acuity (BCVA), and safety.

  • Tinlarebant is an oral, once-daily small molecule targeting RBP4 to reduce vitamin A delivery to the eye, aiming to slow retinal degeneration.

Efficacy results

  • Tinlarebant achieved a statistically significant 36% reduction in DDAF lesion growth rate versus placebo (p=0.0033), with consistent results in sensitivity analyses.

  • The treatment effect was confirmed in the fellow eye, showing a 33.6% reduction in lesion growth (p=0.041).

  • DAF lesion growth, a key secondary endpoint, was slowed by 33.7% in the study eye (p=0.027) and 32.7% in the fellow eye (p=0.017), both statistically significant.

  • Visual acuity (BCVA) remained stable in both groups over two years, consistent with disease natural history.

  • Tinlarebant reduced RBP4 levels by about 80% from baseline, exceeding the 70% target associated with clinical benefit.

Safety and tolerability

  • Tinlarebant demonstrated an excellent safety profile, with no serious treatment-related adverse events or deaths over two years.

  • Most adverse events were mild and included nasopharyngitis, headache, acne, xanthopsia, and delayed dark adaptation; most resolved during treatment.

  • Four treatment-emergent adverse events led to drug discontinuation, with about half likely related to ocular events.

  • No discontinuations were due to non-ocular adverse events; six serious non-ocular AEs occurred, none related to the drug.

  • The safety profile supports long-term compliance and chronic use in adolescents.

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Frequently asked questions

Belite Bio, Inc. is a clinical-stage biopharmaceutical company engaged in developing novel therapeutics targeting retinal degenerative eye diseases and certain metabolic diseases. The company's primary focus includes conditions such as atrophic age-related macular degeneration (AMD) and autosomal recessive Stargardt disease type 1 (STGD1), which can lead to permanent blindness. Additionally, Belite Bio is exploring treatments for metabolic disorders like non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), type 2 diabetes (T2D), and gout. Belite Bio, Inc. was founded in 2016 and is headquartered in San Diego, California. The company's shares are listed on the NASDAQ.

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