After positive topline results in a phase 3 trial, Belite Bio has announced that it will be filing an NDA with the FDA in the first half of 2026 for tinlarebant, an oral drug to treat Stargardt disease type 1 (STGD1) in adolescents.
In the Dragon trial, tinlarebant demonstrated a reduction in lesion growth of 36%, as measured by decreased autofluorescence by fundus autofluorescence imaging. These results were statistically significant and clinically meaningful compared to a placebo. The drug was also well tolerated in the trial, leading to only four discontinuations, with a safety profile consistent with what Belite previously reported.
STGD1 is a disease of the eye that typically presents in childhood or the early years of adulthood and leads to progressive vision loss. Tinlarebant is the first therapeutic candidate to show clinical efficacy for a disease that currently has no approved treatment.