Belite Bio concludes phase 3 DRAGON study for Stargardt disease

Belite Bio announced the conclusion of its phase 3 DRAGON study (NCT05244304), which is investigating tinlarebant in the treatment of adolescent patients with Stargardt disease type 1 (STGD1).¹ The company anticipates the release of the topline outcomes in the fourth quarter of 2025 and plans to submit new drug applications in the first half of 2026.¹

The phase 3, multicenter, randomized, double-masked, placebo-controlled study was randomized 2:1, favoring tinlarebant over placebo.^1,2 The primary efficacy goal is to measure the rate of atrophic lesion growth, as well as evaluate the tolerability and safety of tinlarebant.¹ Tinlarebant’s mechanism of action involves minimizing and regulating the serum retinol binding protein 4 (RBP4) levels, which is the primary carrier of retinol from the liver to the eye.¹

“We are pleased to announce the successful completion of the DRAGON trial,” Belite Bio CEO and Chairman Tom Lin, MMed, PhD, MBA, said in a news release. “This is an important milestone in our mission to bring a treatment to patients living with Stargardt disease.”

DRAGON enrolled 104 participants aged 12 to 20 years from 11 regions, including the US, Australia, Belgium, United Kingdom, Germany, France, Switzerland, China, Netherlands, Hong Kong, and Taiwan.^1,2 Participants must have clinically diagnosed STGD1 with at least 1 mutation identified in the ABCA4 gene, as well as a defined aggregate atrophic lesion size within 3 disc areas as imaged by fundus autofluorescence in the study eye.²

Tinlarebant is an oral medication aimed at decreasing the buildup of vitamin A–based toxins, known as bisretinoids.¹ Bisretinoids are implicated in retinal diseases such as STGD1 and are also known to contribute to the progression of geographic atrophy (GA), secondary to age-related macular degeneration.¹

“With no approved therapies available today, tinlarebant has the potential to be the first treatment for this devastating inherited macular degeneration,” Lin said. “We are deeply grateful to the patients, families, investigators, and study teams worldwide who made this clinical trial possible.”

Tinlarebant has received fast-track designation and rare pediatric disease designation in the US; orphan drug designation in the US, Japan, and Europe; and Sakigake designation in Japan for the treatment of patients with STGD1.¹

References

1. Belite Bio wraps up phase III trial of Stargardt disease treatment. Clinical Trials Arena. September 15, 2025. Accessed September 15, 2025. https://www.clinicaltrialsarena.com/news/belite-bio-stargardt-disease/

2. Phase 3, randomized, placebo-controlled study of tinlarebant to explore safety and efficacy in adolescent Stargardt disease (DRAGON). ClinicalTrials.gov. Updated June 7, 2024. Accessed September 15, 2025. https://clinicaltrials.gov/study/NCT05244304?term=DRAGON%20phase%203%20stargardt&rank=1