Belite Bio's Phase 3 DRAGON trial investigating Tinlarebant for adolescent Stargardt disease has reached a significant milestone, with the independent Data Safety Monitoring Board (DSMB) recommending trial continuation without modifications following a planned interim efficacy analysis.
The DSMB's assessment, based on one-year data from all participants, confirmed that the original sample size of 104 subjects remains statistically appropriate, eliminating the need for additional enrollment. Notably, the board has recommended submitting the data for regulatory review, marking a potential advancement toward drug approval.
Safety and Efficacy Signals
The interim analysis revealed encouraging safety and efficacy indicators. "We are pleased to have reached this important trial milestone and are excited by the safety profile that we continue to observe for Tinlarebant," stated Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. The drug demonstrated consistent tolerability with its known mechanism of action and previously observed safety data.
Visual function outcomes have been particularly promising, with most subjects showing stabilized visual acuity. Throughout the two-year study period, mean changes from baseline remained minimal, with less than three letter scores difference under both standard and low luminance conditions.
Trial Design and Global Reach
The DRAGON trial employs a randomized, double-masked, placebo-controlled design across multiple international centers. The study features a 2:1 randomization ratio between active treatment and placebo groups, with sites established in 11 jurisdictions including the United States, European countries, and Asia-Pacific regions.
The trial's primary efficacy endpoint focuses on measuring the growth rate of atrophic lesions, alongside comprehensive safety and tolerability assessments. The study is projected to conclude by Q4 2025, including a three-month follow-up period.
Regulatory Status and Recognition
Tinlarebant has garnered significant regulatory support globally, reflecting its potential as a breakthrough therapy for Stargardt disease, a condition currently lacking approved treatments. The drug has secured:
- Fast Track and Rare Pediatric Disease Designations in the United States
- Orphan Drug Designation in the U.S., Europe, and Japan
- Pioneer Drug (Sakigake) Designation in Japan
These designations underscore the urgent need for effective treatments in Stargardt disease and could potentially expedite the regulatory review process upon trial completion.
