Belite Bio – Phase 2/3 Trial for Adolescents with Stargardt disease
*Update - Belite Bio has announced that enrolment in the DRAGON II clinical trial of tinlarebant is complete. 60 adolescents aged 12-20 with genetically confirmed Stargardt disease have been enrolled in DRAGON II across Japan, the United States, and the United Kingdom.
Belite Bio are running a phase 2/3 trial, known as DRAGON II, for tinlarebant – a treatment approach in development for Stargardt disease. This trial is for those aged 12-20 years old only, with genetically confirmed Stargardt disease and is taking place across multiple sites including at Moorfields Eye Hospital in London. Please note that recruitment for this trial is now closed.
“We are pleased to complete enrolment in the DRAGON II clinical trial and sincerely thank the investigators, the adolescent participants, and their families who made this study possible. 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.” Dr. Tom Lin, Chairman and CEO of Belite Bio.
What is Stargardt Disease?
Stargardt disease is a progressive inherited retinal condition in which vision loss is caused by the death of photoreceptor cells (light-sensing cells) in the central portion of the retina called the macula. It is the most common form of juvenile macular dystrophy.
The condition is most commonly caused by mutations in the ABCA4 gene, which provides the instructions needed to make a protein found in the photoreceptor cells. This protein is responsible for transporting toxic byproducts, made by the regular visual cycle, away from photoreceptor cells. Without this protein, these toxins can accumulate over time on the macula, which we use for seeing fine detail in high definition, destroying light-sensing cells and central vision.
The toxins that form during the visual cycle depend on vitamin A (retinol) being transported to the eye from the liver. This is why individuals with Stargardt disease are advised against taking vitamin A supplements. There are currently no treatments available for the condition, however, there are several therapeutic approaches in development, including tinlarebant by Belite Bio.
What is tinlarebant?
Tinlarebant is an orally administered, once-a-day tablet which aims to slow vision loss by addressing some of the damaging toxin accumulation that occurs in the retina as a result of Stargardt disease.
How Does it Work?
Tinlarebant works by lowering the amount of the main protein, known as RBP4, that transports vitamin A through the bloodstream. By reducing RBP4, less vitamin A reaches the eye, which helps to decrease the production of harmful toxins, protect retinal tissue and slow vision loss. Unlike the retina, most tissues in the body do not rely on RBP4 to receive vitamin A, meaning that tinlarebant does not cause major vitamin A shortages elsewhere in the body.
The Trial:
Belite Bio have previously announced positive findings from their trials including a two-year phase 2 study of tinlarebant in adolescent patients. The goal of this DRAGON II clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in individuals aged 12-20 with Stargardt Disease. This trial has 2 components – a phase 1b trial in Japan only and a phase 2/3 trial across Japan, the US and UK. The UK site is at Moorfields Eye Hospital in London.
The phase 2/3 part of the study is randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months. This means that participants are randomly assigned to receive either 5 mg of tinlarebant or a placebo. Neither the participants or the researchers know who is getting which treatment. The medicine is taken once a day for 24 months to evaluate how safe, tolerable, and effective tinlarebant is for individuals with Stargardt disease.
What happens now?
Tinlarebant has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation in the US, Orphan Drug Designation in the US, Europe and Japan, and Sakigake Designation in Japan for the treatment of STGD1. The results of this registration-enabling study, along with encouraging results from their global phase 3 trial of tinlarebant, will provide crucial evidence to support Belite Bio’s application to medicine regulators who will decide whether to license tinlarebant for wider availability. Please note that different regulatory bodies operate in the US, UK, Europe and elsewhere, and obtaining regulatory approval can take a long time, even if Belite Bio seeks to work with all of them. Reaching agreement for NHS availability is a further, separate process. As a result, there may be a significant wait before the treatment becomes widely available.
If you would like more information about Belite Bio or tinlarebant, please visit their website here: Belite Bio |
If you wish to read more about the trial itself, please visit this website Study Details | NCT06388083 | A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease | ClinicalTrials.gov.