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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.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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