Belite Bio reported positive topline results from its phase 3 DRAGON trial evaluating tinlarebant in adolescents with Stargardt disease type 1, marking what the company described as the first successful pivotal study for the inherited retinal disorder. The trial enrolled 104 patients globally and met its primary endpoint, showing a 36% reduction in the growth rate of definitely decreased autofluorescence lesions compared with placebo. The prespecified analysis reached statistical significance (P=.0033), and a post hoc analysis showed a consistent effect (P<.0001).

Investigators reported minimal changes in visual acuity over 24 months in both groups, aligning with known natural history patterns. Tinlarebant was generally well tolerated, with 4 treatment-related discontinuations. The company said safety findings were consistent with earlier studies.

Retina specialists involved in the trial noted the potential implications of the data. Michel Michaelides, MD, of Moorfields Eye Hospital, said the slowing of lesion growth represents an encouraging development for a condition long considered untreatable. Quan Dong Nguyen, MD, of Stanford University, said the results may eventually translate into functional benefits as lesion enlargement is strongly associated with long-term visual decline.

Belite Bio plans to submit an NDA to the FDA in the first half of 2026 and expects to share additional analyses at upcoming medical meetings. RP