Belite Bio has concluded the final participant visit in its Phase III DRAGON study, which is focused on assessing the Tinlarebant in treating Stargardt disease type 1 (STGD1).
The trial saw the enrolment of 104 adolescent participants from 11 different regions, including Australia, Belgium, China, France, the Netherlands, and Taiwan.
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It was designed with a 2:1 randomisation ratio, favouring Tinlarebant over a placebo. A total of 94 subjects completed the trial.
The trial’s primary efficacy goal is to measure the rate of growth of atrophic lesions in the retina, while also evaluating the tolerability and safety of the therapy.
The company is anticipating the release of the top-line outcomes from this trial in the fourth quarter of 2025 and is preparing to submit new drug applications in the first half of 2026.
Belite Bio CEO and chairman Dr Tom Lin said: “We are very pleased to announce the successful completion of the DRAGON trial. This is an important milestone in our mission to bring a treatment to patients living with Stargardt disease.
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By GlobalData“With no approved therapies available today, Tinlarebant has the potential to be the first treatment for this devastating inherited macular degeneration. We are deeply grateful to the patients, families, investigators, and study teams worldwide who made this clinical trial possible.”
According to the company, tinlarebant is an oral medication aimed at decreasing the build-up of vitamin A-based toxins, known as bisretinoids. These are implicated in retinal diseases such as STGD1 and are also known to contribute to the progression of geographic atrophy (GA) in advanced dry age-related macular degeneration (AMD).
These bisretinoids are said to be the by-products of the visual cycle, which relies on vitamin A being transported to the eye.
Tinlarebant’s mechanism of action involves the minimising and regulation of serum retinol binding protein 4 (RBP4) levels, which is the primary carrier protein for retinol from the liver to the eye.
By controlling retinol entry into the eye, the therapy aims to decrease the formation of bisretinoids.
The therapy has received fast-track designation and rare paediatric disease designation in the US, orphan drug designation in the US, Japan, Europe, and the Sakigake designation in Japan for the STGD1 treatment.
In July 2025, Belite Bio completed the enrolment for its 24-month PHOENIX Phase III trial, assessing the tolerability and safety of Tinlarebant in those with GA in dry AMD.
