Positive Phase III DRAGON trial of tinlarebant in Stargardt disease

Shares of San Diego, USA-based Belite Bio (Nasdaq: BLTE) shot up 16.4% to $160.00 in pre-market activity today, after it announced top-line results from the global Phase III DRAGON trial of tinlarebant.

Belite noted that this marked the first successful pivotal trial in patients with Stargardt disease type 1 (STGD1). 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 III 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 (DDAF) by fundus autofluorescence imaging, compared with placebo. Statistical significance was reached when applying the pre-specified analysis (p-value = 0.0033). 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 < 0.0001.