Belite Bio says tinlarebant met primary efficacy endpoint in Phase 3 trial

Belite Bio (BLTE) announced topline results from the global Phase 3 “DRAGON” trial of Tinlarebant, marking the first successful pivotal trial in patients with Stargardt disease type 1. STGD1 is an eye disease that leads to progressive vision loss, usually beginning in childhood or young adulthood, and currently has no approved treatment worldwide. The Phase 3 DRAGON trial enrolled 104 patients with STGD1 and met its primary efficacy endpoint, demonstrating a statistically significant and clinically meaningful 36% reduction in the growth rate of retinal lesions, measured as definitely decreased autofluorescence by fundus autofluorescence imaging, compared with placebo. Statistical significance was reached when applying the pre-specified analysis. Considering the progressive nature typically seen in STGD1, a further post-hoc analysis providing a specific data correlation showed that the treatment effect remained consistent with a p-value less than 0.0001. “The significant lesion growth reduction observed in the DRAGON study, along with the favorable safety profile, provide important validation of our therapeutic approach and the mechanism of Tinlarebant. These results underscore the team’s commitment to addressing the unmet need in Stargardt disease and the potential to meaningfully improve the quality of life for those affected,” said Dr. Nathan Mata, Chief Scientific Officer at Belite Bio. As expected, the overall change in visual acuity was minimal over the period of 24 months in both study groups, consistent with natural history data. The safety profile remains consistent with what the Company previously reported, and Tinlarebant was well tolerated with only four treatment-related discontinuations. After the full analysis is complete, the Company plans to share additional data at upcoming medical meetings.