Belite Bio completes enrollment in phase 2/3 DRAGON II trial for STGD1

Belite Bio has completed enrollment in its phase 2/3 DRAGON II clinical trial evaluating tinlarebant for the treatment of Stargardt disease type 1 (STGD1).

Tinlarebant is a novel oral therapy intended to reduce the accumulation of vitamin A-based toxins (bisretinoids) that cause retinal disease in STGD1 and contribute to disease progression in geographic atrophy (GA), or advanced dry age-related macular degeneration (AMD). The company notes that tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye, reducing the formation of bisretinoids.

DRAGON II is a global, 24-month, randomized, double-masked, placebo-controlled study designed to evaluate the efficacy, safety, and tolerability of tinlarebant in adolescent patients with STGD1. It has enrolled 60 patients aged 12 to 20 years old across Japan, the United States, and the United Kingdom. Patients have been randomized 1:1 to receive either tinlarebant or placebo.¹

Hendrik Scholl, MD, chief medical officer of Belite Bio commented on the enrollment, saying, “Completing enrollment in DRAGON II clinical trial represents continued execution of our tinlarebant clinical development program. We are pleased to have implemented a registration-enabling study that aligns with the requirements of the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), supporting Belite Bio’s ability to pursue potential approval in Japan while advancing research for adolescents living with Stargardt disease globally.”

At the end of 2025, Belite Bio released topline results from the global phase 3 DRAGON trial of tinlarebant in patients with STGD1.²

The DRAGON trial was a 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal phase 3 trial in adolescent STGD1 patients. A total of 104 patients ranging from ages 12 to 20 were enrolled (n=69 in the tinlarebant arm and n=35 in the placebo arm). All patients had been diagnosed with STGD1 with at least 1 mutation identified in the ABCA4 gene, an atrophic lesion size within 3 disc areas (7.62 mm²), and a best corrected visual acuity (BCVA) of 20/200 or better.²

According to the company, the DRAGON trial 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 an unstructured covariance matrix under the Mixed Model for Repeated Measures (MMRM) (p-value = 0.0033). Additionally, a post-hoc analysis providing a specific data correlation showed the treatment effect remained consistent with a p-value < 0.0001.²

Belite Bio noted it plans to discuss potential next steps and submit a New Drug Application for tinlarebant in the first half of 2026. Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the US.^1,2

References:

1. Belite Bio Completes Enrollment in the DRAGON II Clinical Trial of Tinlarebant for Stargardt Disease (STGD1). Published January 27, 2026. Accessed January 27, 2026. https://ir.opusgtx.com/press-releases/detail/516/opus-genetics-launches-gene-therapy-clinical-trial-for-mertk-related-retinitis-pigmentosa

2. Harp MD. Belite Bio releases topline results from phase 3 DRAGON trial of Tinlarebant for STGD1. Published December 1, 2025. Accessed January 27, 2026. https://www.ophthalmologytimes.com/view/belite-bio-releases-topline-results-from-phase-3-dragon-trial-of-tinlarebant-for-stgd1