Belite Bio announced that Tinlarebant, its experimental oral treatment for Stargardt disease type 1 (STGD1), has met the main goal in a global, late-stage Phase 3 trial. The trial enrolled 104 adolescents aged 12–20 and demonstrated that Tinlarebant reduced the growth rate of retinal lesions by about 36% compared with placebo, a statistically significant and clinically meaningful result.
STGD1 is a rare inherited eye condition that typically leads to progressive vision loss in childhood or adolescence, and until now has had no approved therapy worldwide.
Safety and tolerability appeared acceptable: only four participants discontinued due to treatment-related adverse events. Most side effects — including some ocular events were described as mild and resolved during the trial.
With this success, Belite Bio plans to file for regulatory approval in the U.S. and other markets, aiming for formal approval in the first half of 2026.
If approved, Tinlarebant would become the first treatment available for STGD1 offering hope to thousands living with a previously untreatable retinal disease.
