Belite Bio Begins Phase 2/3 DRAGON II Trial of Tinlarebant for Stargardt Disease

Belite Bio, Inc, a clinical-stage biopharmaceutical company, has announced the commencement of the Phase 2/3 portion of its DRAGON II clinical trial at Tokyo Medical Center. This trial explores the efficacy, safety, and tolerability of Tinlarebant in approximately 60 adolescents with Stargardt Disease type 1 (STGD1) across the U.S., U.K., and Japan. Tinlarebant is an innovative oral therapy aimed at decreasing the accumulation of vitamin A-based toxins that contribute to retinal diseases.

Dr. Tom Lin, Chairman and CEO of Belite Bio, expressed satisfaction at dosing the first patient in the trial, marking a pivotal step towards addressing the unmet needs of those with Stargardt Disease. The Phase 1b study of Tinlarebant was recently concluded at the Tokyo Medical Center, and the continued collaboration with the center is seen as beneficial for the ongoing trial. Enrollments are also in progress in the U.S. and U.K., reflecting significant strides towards providing a transformative oral therapy for affected individuals.

Professor Kaoru Fujinami, Principal Investigator at the National Hospital Organization, Tokyo Medical Center, highlighted the significance of DRAGON II as the first global Stargardt disease trial in Japan. This trial offers Japanese patients a chance to access a potential treatment for a disease that has historically lacked therapeutic options. The interest in the trial underscores the urgent need for treatments for STGD1. With Tinlarebant receiving the Sakigake designation from Japan's Ministry of Health, the trial is positioned to advance swiftly.

The DRAGON II trial consists of a Phase 1b open-label study in Japan to assess Tinlarebant's pharmacokinetics and pharmacodynamics, followed by a Phase 2/3 multicenter, double-masked, placebo-controlled, randomized study designed to evaluate its efficacy, safety, and tolerability over 24 months in adolescent STGD1 subjects. The goal is to enroll approximately 60 subjects aged 12 to 20 years, including around 10 Japanese participants, with a 1:1 randomization between Tinlarebant and a placebo. Data from the Japanese cohort will support future New Drug Applications (NDA) in Japan.

Tinlarebant aims to reduce vitamin A-based toxins, known as bisretinoids, which cause retinal disease in STGD1 and contribute to disease progression in Geographic Atrophy (GA), an advanced form of Dry Age-related Macular Degeneration (AMD). Bisretinoids are by-products of the visual cycle and depend on vitamin A (retinol) supply to the eye. Tinlarebant functions by lowering serum retinol binding protein 4 (RBP4), the carrier protein for retinol, thus reducing the formation of bisretinoids. Tinlarebant has received multiple designations, including Fast Track and Rare Pediatric Disease in the U.S., Orphan Drug designation in the U.S., Europe, and Japan, and Sakigake designation in Japan for STGD1 treatment.

STGD1 is the most common inherited retinal dystrophy, leading to central vision loss and is caused by mutations in the ABCA4 gene. This gene mutation results in the accumulation of bisretinoids, causing retinal cell death and vision impairment. The fluorescent properties of bisretinoids have aided ophthalmologists in diagnosing and monitoring the disease. Currently, there are no FDA-approved treatments for STGD1.

Both STGD1 and GA share similar pathological features, characterized by excessive bisretinoid accumulation, retinal cell death, and progressive vision loss. Vision loss progresses slowly until it affects the macula, the central part of the eye. Belite Bio is also assessing Tinlarebant's safety and efficacy in GA patients through a two-year Phase 3 study known as PHOENIX, with over 200 participants enrolled globally.

Belite Bio specializes in developing novel therapeutics for degenerative retinal diseases that present significant unmet medical needs, including Stargardt Disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), along with specific metabolic diseases. Tinlarebant, its leading candidate, is under evaluation in multiple clinical trials aimed at reducing ocular toxins and improving patient outcomes.