Belite Bio Completes DRAGON II Clinical Trial Enrollment Of Tinlarebant For Stargardt Disease

Belite Bio announced that it has completed enrollment in its Phase 2/3 DRAGON II clinical trial evaluating tinlarebant for adolescents with Stargardt disease type 1 (STGD1). The global study has enrolled 60 participants, including 15 patients from Japan, across trial sites in Japan, the United States, and the United Kingdom. Subjects aged 12 to 20 years were randomized equally to receive either tinlarebant or placebo and will be followed for 24 months in this double-masked, placebo-controlled trial.

DRAGON II is designed to assess the efficacy, safety, and tolerability of tinlarebant in slowing retinal degeneration in STGD1, a rare inherited macular disorder that leads to progressive vision loss in young people. Completion of enrollment marks a key operational milestone for the company as it advances the program toward late-stage development.

“We are pleased to complete enrollment in the DRAGON II clinical trial and sincerely thank the investigators, the adolescent participants, and their families who made this study possible,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “Their commitment reflects the strong engagement of the Stargardt disease community and supports our broader clinical development and commercialization efforts. Importantly, we remain on track to submit an NDA to the FDA for tinlarebant in the first half of 2026.”

Tinlarebant is an oral small-molecule therapy aimed at reducing the buildup of toxic vitamin A–derived by-products known as bisretinoids, which damage retinal cells in STGD1 and also play a role in geographic atrophy associated with advanced dry age-related macular degeneration. The drug works by lowering circulating levels of retinol-binding protein 4, thereby limiting the amount of vitamin A delivered to the eye and slowing the formation of these harmful compounds.

“Completing enrollment in DRAGON II clinical trial represents continued execution of our tinlarebant clinical development program,” said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. “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.”

Reflecting its potential in rare retinal diseases, tinlarebant has received multiple regulatory incentives worldwide, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations in the United States, Orphan Drug status in Europe and Japan, and Sakigake designation in Japan. Belite Bio is positioning the program as a cornerstone of its pipeline for degenerative eye disorders.