Belite Bio (BLTE) Study result presentation summary

Study design and methodology

• Phase 3, randomized, double-masked, placebo-controlled trial evaluated 5 mg/day oral tinlarebant in patients aged 12–20 years with Stargardt disease type 1 (STGD1).

• Inclusion required clinical diagnosis, at least one ABCA4 mutation, atrophic lesion size within 3 disc areas, and BCVA of 20/200 or better.

• Primary endpoint was reduction in DDAF lesion growth rate, assessed by FAF imaging.

Key efficacy results

• Sustained 80% reduction in serum RBP4 levels over two years with tinlarebant.

• Annualized DDAF growth rate was 0.38 mm²/year for tinlarebant vs. 0.59 mm²/year for placebo, a 35.7% treatment effect (P = 0.0033).

• BCVA remained stable in both treatment and placebo groups.

• qAF values remained stable or slightly decreased with tinlarebant, while placebo showed a 20% increase.

Safety and tolerability

• Most ocular and non-ocular treatment-emergent adverse events (TEAEs) were mild and resolved during the study.

• No serious ocular TEAEs; six serious non-ocular TEAEs reported, mostly unrelated to treatment.

• Tinlarebant was generally well tolerated, with a safety profile consistent with its mechanism of action.