Monday, July 24, 2023
Belite Bio, Inc (NASDAQ: BLTE) has successfully completed enrollment in its pivotal global Phase 3 "DRAGON" trial for Stargardt Disease (STGD1). Belite Bio is a clinical stage biopharmaceutical drug development company focusing on novel therapeutics for retinal degenerative eye diseases with significant unmet medical needs. The trial evaluates the efficacy of oral Tinlarebant, a retinol binding protein 4 (RBP4) antagonist designed to lower ocular vitamin-A based toxins linked to STGD1.
The Phase 3 DRAGON trial enrolled 90 adolescent subjects from 11 countries worldwide and follows a randomized, double-masked, placebo-controlled design. The study aims to assess the safety and efficacy of Tinlarebant in STGD1 patients, with the primary endpoint being the slowing of lesion growth rate. Tinlarebant has received Fast Track Designation and Rare Pediatric Disease Designation in the U.S., along with Orphan Drug Designation in both the U.S. and Europe for STGD1.
Belite Bio's CEO, Dr. Tom Lin, expressed satisfaction with the completion of enrollment and highlighted the trial's potential to demonstrate a treatment benefit for STGD1 patients. The company plans to share interim safety and efficacy data from the DRAGON trial in mid-2024.
Professor Michel Michaelides, the Principal Investigator and Chief Investigator of the DRAGON trial from Moorfields Eye Hospital in the United Kingdom, praised the promising Phase 2 trial results for STGD1 patients, supporting Tinlarebant's transformative potential as a potential first FDA-approved treatment for the disease.
Apart from STGD1, Belite Bio plans to initiate a Phase 3 study (PHOENIX) of Tinlarebant for Geographic Atrophy (GA) in the third quarter of 2023. GA shares similar pathophysiology with STGD1, characterized by the accumulation of cytotoxic bisretinoids, retinal cell death, and progressive vision loss.
Stargardt Disease (STGD1) is the most common inherited retinal dystrophy, causing blurring or loss of central vision in adults and children. Currently, there are no FDA-approved treatments for this condition, making the DRAGON trial and Tinlarebant's potential success crucial for addressing the unmet medical needs in this area.