Belite Bio announces Positive Results from Phase 3 DRAGON Trial for Stargardt Disease Treatment
September 15, โ2025 – Belite Bio today โannounced the successful completion of its Phase 3 โDRAGON trial evaluating tinlarebant for the treatment of Stargardt disease (STGD1), a rare inherited form of macular degenerationโ causing vision loss in children and young adults.โ This marks a notable milestone as tinlarebant potentially represents the first-ever treatment โfor this โคdevastating condition, for which no approved therapies currently exist.
the multicenter, randomized, double-masked, placebo-controlled DRAGON study enrolled 104 participants aged 12 too 20 years old across โข11 regions – Australia, Belgium, the US, the UK, Germany, France, Switzerland, China, the Netherlands, Hong Kong, and Taiwan. Participants were โrequired to have a clinical diagnosis of STGD1 with at least one identified mutationโ in the ABCA4 gene and a defined aggregate atrophic lesion size within 3 disc areas asโค imaged โbyโ fundus autofluorescence (FAF). The โขstudy utilized a 2:1 randomization ratio, favoring tinlarebant over placebo.
Tinlarebantโค works by minimizing โขand regulating levels of retinol binding protein 4 (RBP4), โthe primary carrier of retinol from the liver to the eye, ultimately decreasing the buildup of vitamin A-based toxins called bisretinoids. These โbisretinoids are believed to contribute to the progression of STGD1,and โalso โขgeographic atrophy (GA) associated with age-related macular degeneration (AMD). The primary goal of the DRAGON trial was to measure the rate of โatrophic lesion growth, alongside assessing the tolerability and safety โฃof tinlarebant.
“This is an important milestone in our mission to bring a treatment to patients living with โขStargardt disease,” said Belite Bio CEO โand chairman Tom Lin, MMed, PhD, MBA. “With no approved therapies available today, tinlarebant has theโ potential to be the first treatment for this devastating inherited macularโค degeneration.”
Tinlarebant has received fast-track designation and rare pediatric disease designation in the US, โorphan drug designation in the US, Japan, and Europe, and the Sakigake designationโ in Japan for the treatment of STGD1.
References:
- Belite Bio wraps โคup Phase III trial of Stargardt disease โขtreatment. โคClinical Trials Arena.Published September 15,2025. Accessedโฃ September 15, 2025. https://www.clinicaltrialsarena.com/news/belite-bio-stargardt-disease/
- Clinicaltrials.gov.Published 2025. Accessed September 15, 2025. https://clinicaltrials.gov/study/NCT05244304?term=DRAGON%20phase%203%20stargardt&rank=1