Belite Bio – Phase 2/3 Trial for Adolescents with Stargardt disease

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.

Recruitment Criteria:

The trial is currently open to recruitment, however this is expected to close soon. Please note that recruitment at Moorfields is currently only open to those based in the UK, aged 12-20 years old with genetically confirmed Stargardt disease. Further eligibility criteria also apply.

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.

For participation enquiries UK-based patients can contact the study team at Moorfields by emailing [email protected].