Belite Bio, Inc. (NASDAQ: BLTE) has announced the dosing of the first patient in the Phase 2/3 portion of its DRAGON II clinical trial at the Tokyo Medical Center. This trial is designed to evaluate Tinlarebant for the treatment of Stargardt disease type 1 (STGD1) in adolescent subjects.
The Phase 2/3 study is a multicenter, double-masked, placebo-controlled, randomized trial that will assess the efficacy, safety, and tolerability of Tinlarebant. Approximately 60 adolescent STGD1 subjects, aged 12 to 20 years, will be enrolled across sites in the U.S., U.K., and Japan. The primary endpoint is to evaluate the drug's impact on the progression of Stargardt disease.
Trial Design and Objectives
The DRAGON II trial combines a Phase 1b open-label study in Japan, which evaluated the pharmacokinetics (PK) and pharmacodynamics (PD) of Tinlarebant, with the Phase 2/3 study. The Phase 2/3 portion will administer Tinlarebant daily for 24 months. A 1:1 randomization (Tinlarebant:placebo) will be employed. Data from the Japanese subjects will be used to facilitate future NDA applications in Japan.
"We are pleased to have successfully dosed the first subject in the Phase 2/3 portion of our DRAGON II trial. This milestone is a significant step forward in our mission to address the unmet needs of people living with Stargardt Disease," said Dr. Tom Lin, Chairman and CEO of Belite Bio.
About Tinlarebant
Tinlarebant (a/k/a LBS-008) is an oral therapy designed to reduce the accumulation of vitamin A-based toxins (bisretinoids) that cause retinal disease in STGD1 and contribute to disease progression in geographic atrophy (GA), or advanced Dry AMD. The drug works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), which is responsible for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids.
Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S. Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1.
Stargardt Disease (STGD1)
STGD1 is the most common inherited retinal dystrophy, causing blurring or loss of central vision in both adults and children. It is caused by mutations in the ABCA4 gene, leading to the progressive accumulation of bisretinoids, which results in retinal cell death and vision loss. Currently, there are no FDA-approved treatments for STGD1.
Professor Kaoru Fujinami, Principal Investigator at National Hospital Organization, Tokyo Medical Center, noted, "DRAGON II is the first global Stargardt disease trial in Japan... The level of interest we have received in this trial speaks to the urgency of the unmet need for patients living with STGD1."
