Belite Bio Reports Positive Topline Results From Global Phase 3 DRAGON Trial Of Tinlarebant, Achieving First Successful Pivotal Study In Stargardt Disease Type 1

Belite Bio, Inc. announced topline results from its global Phase 3 DRAGON trial of Tinlarebant, marking the first successful pivotal trial in patients with Stargardt disease type 1 (STGD1), a progressive eye disorder that typically begins in childhood or young adulthood and currently has no approved treatment worldwide. The DRAGON trial enrolled 104 patients with STGD1 and achieved its primary efficacy endpoint. 

The study showed a statistically significant and clinically meaningful 36% reduction in the growth rate of retinal lesions, measured as definitely decreased autofluorescence (DDAF) using fundus autofluorescence imaging, compared with placebo. Statistical significance was observed in the pre-specified analysis (p = 0.0033), and post-hoc analyses confirmed the treatment effect remained consistent (p < 0.0001), despite the progressive nature of STGD1.

Tom Lin, Chairman and CEO of Belite Bio, stated that the results represent a historic breakthrough, potentially offering the first treatment for Stargardt disease. He highlighted that Tinlarebant is the first oral therapy to demonstrate a clinically meaningful outcome in retinal degenerative disease. With these data, Belite Bio is advancing regulatory interactions globally and moving closer to delivering a treatment option for patients with STGD1. He also expressed gratitude to the patients, families, and investigators involved in the trial.

Nathan Mata, Chief Scientific Officer at Belite Bio, noted that the significant reduction in lesion growth, combined with a favorable safety profile, validates both the therapeutic approach and the mechanism of Tinlarebant. He emphasized the potential of the therapy to meaningfully improve quality of life for patients living with Stargardt disease. As expected, overall visual acuity changes were minimal over the 24-month study period in both treatment and placebo groups, consistent with the disease’s natural history. Tinlarebant was well tolerated, with only four treatment-related discontinuations. Belite Bio plans to share additional data at upcoming medical meetings once the full analysis is complete.

Leading investigators involved in the trial also commented on the results. Professor Michel Michaelides of Moorfields Eye Hospital, the UK’s top enrolling investigator in DRAGON, described the findings as deeply encouraging, signaling that an approved treatment option may soon be available for patients with this devastating disease. Professor Quan Dong Nguyen of Stanford University added that Tinlarebant’s reduction in lesion growth is expected to translate into meaningful benefits in visual function over time.

Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio, highlighted that the DRAGON trial provides the most compelling evidence to date that an oral therapy can alter the course of Stargardt disease. He noted that Tinlarebant’s mechanism reducing the accumulation of toxic retinal byproducts significantly slowed disease progression, producing a clear treatment effect in both eyes. The therapy also showed statistically significant benefits in key secondary endpoints and demonstrated a favorable safety and tolerability profile. Collectively, these results reinforce Tinlarebant’s potential to change the treatment landscape for Stargardt disease and set a new benchmark for future research in inherited retinal disorders.