Belite Bio Trial update

The Dragon Study

Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects.

The closing date for recruitment to the Phase 3 Belite Bio Trial is due to finish end of June 2023 and the age range to extend up to 20 years pending final regulatory approval.

Belite Bio is sponsoring a Phase 3 clinical research study in adolescents with a clinical diagnosis of Stargardt disease (STGD1) and at least one mutation identified in the ABCA4 gene. The primary objective of this trial is to assess the efficacy of Tinlarebant in slowing the rate of growth of atrophic lesions. In patients with STGD1, a mutation in the transporter gene ABCA4 interferes with normal protein trafficking in the visual cycle. This leads to an accumulation of cytotoxic metabolites which causes the retinal pigment epithelium and the photoreceptors to undergo profound structural changes. These metabolites are products of retinol derivates, which are transported into the retina via Retinal Binding Protein 4 (RBP4). Tinlarebant is an antagonist of RBP4 which is supposed to reduce the concentration of retinal derivates inside the retina.

Eligibility criteria:

• Male or female patients 12 to 18 years old, inclusive.
• Clinically diagnosed Stargardt disease with at least 1 mutation identified in the responsible gene (ABCA4).
• Patients must have an atrophic lesion of a certain size as determined by ocular imaging
• Patients must present with a visual acuity score of at least 20/200 in one eye

A screening visit is necessary to determine eligibility. Eligible participants will be randomly assigned in a 2:1 ratio to receive either Tinlarebant or placebo. The treatment period is two years, with follow-up four weeks after final treatment. In total, there will be 13 visits on site over 26 months.

The study will be enrolling approximately 60 participants across multiple sites in Australia, Asia, Europe, and North America. In the UK, the study is being hosted at Moorfields Eye Hospital with Professor Michel Michaelides as Principal Investigator. They are currently recruiting for the trial. For further information contact [email protected]. Or if you are outside the UK you can contact Belite Bio at [email protected].