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Belite Bio’s Tinlarebant Cuts Stargardt Lesion Growth 36% in Phase 3 Trial

If cleared, the RBP4-lowering pill would be the first treatment for Stargardt disease.

Overview

  • The 104-patient, 24-month DRAGON study met its primary endpoint with a 35.7% reduction in lesion-growth rate versus placebo (p=0.0033).
  • Statistically significant effects were also seen in the fellow eye and on decreased autofluorescence lesion growth based on imaging analyses.
  • Tinlarebant was generally well tolerated, with four treatment-related discontinuations and mostly mild, resolving eye-related side effects.
  • The 5 mg dose lowered serum RBP4 by about 80% from baseline, consistent with its mechanism to curb toxic vitamin A byproducts.
  • Belite plans regulatory discussions and targets New Drug Application submissions in the first half of 2026, and the stock rose by double digits premarket.