Belite Bio Completes Tinlarebant Phase 3 for STGD1

Belite Bio has announced the completion of its Phase 3 DRAGON clinical trial (NCT05244304), investigating the efficacy and safety of tinlarebant in patients with Stargardt disease type 1 (STGD1). Topline results are expected in Q4 2025, with new drug applications (NDAs) planned for submission in the first half of 2026.

Study Design and Objectives

The DRAGON trial is a Phase 3, multicenter, randomized, double-masked, placebo-controlled study, designed with a 2:1 randomization ratio in favor of tinlarebant over placebo.

Primary Objectives:

• Assess the rate of atrophic lesion growth

• Evaluate the tolerability and safety of tinlarebant

Tinlarebant works by reducing serum retinol-binding protein 4 (RBP4) levels, which limits the transport of retinol from the liver to the eye, a mechanism aimed at slowing disease progression in STGD1.

Patient Enrollment and Eligibility

A total of 104 participants, aged 12 to 20 years, were enrolled across 11 global regions, including Australia, Belgium, United States, United Kingdom, Germany, France, Switzerland, China, Netherlands, Hong Kong and Taiwan.

Inclusion Criteria:

• Clinically diagnosed Stargardt disease type 1 (STGD1)

• At least one ABCA4 gene mutation

• Aggregate atrophic lesion size within three disc areas in the study eye, measured by fundus autofluorescence (FAF)

Tinlarebant: Mechanism and Therapeutic Potential

Tinlarebant is an oral investigational therapy developed to reduce the accumulation of vitamin A-based toxins known as bisretinoids. These toxins are implicated in the pathogenesis of STGD1 and have also been associated with the progression of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

“With no approved therapies available today, tinlarebant has the potential to be the first treatment for this devastating inherited macular degeneration,” said Tom Lin, MMed, PhD, MBA, CEO and Chairman of Belite Bio.

“We are deeply grateful to the patients, families, investigators, and study teams worldwide who made this clinical trial possible.”

Regulatory Status

Tinlarebant has received multiple regulatory designations recognizing its potential to treat a rare and serious condition. These include Fast Track Designation and Rare Pediatric Disease Designation in the United States, as well as Orphan Drug Designation in the United States, Japan, and Europe. Additionally, the therapy has been granted Sakigake Designation in Japan, supporting expedited development and review processes across multiple regulatory jurisdictions.

These designations may facilitate expedited development and review pathways.

“This is an important milestone in our mission to bring a treatment to patients living with Stargardt disease,” Lin added.