Belite Bio, Inc, a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical need, has announced that following a pre-specified interim analysis of the pivotal global phase 3 DRAGON trial data of Tinlarebant in adolescent Stargardt disease patients, the Data Safety Monitoring Board (DSMB) has recommended the trial proceed without any modifications. The Interim Analysis was performed when all subjects completed the one-year assessment.
Tinlarebant has been granted Fast Track and Rare Pediatric Disease Designations in the US, Orphan Drug Designation in the US, Europe, and Japan, and the Pioneer Drug Designation in Japan for Stargardt disease, for which there are no approved treatments.
The study design for the DRAGON trial included an adaptive sample size re-estimation that would determine the need for an increase in sample size in order to enhance power, based on a treatment effect observed at the Interim Analysis. The recommendation by the DSMB that the trial should proceed without modifications indicates that a sample size increase is not warranted. In addition, the DSMB recommended to submit the data for further regulatory review for drug approval.
According to the DSMB, Tinlarebant is well-tolerated and the safety profile remains consistent with previously observed data and the mechanism of action for Tinlarebant. In addition, visual acuity was stabilized in the majority of subjects, with mean change from baseline of less than three letter scores under both standard and low luminance, throughout the two-year study.
“We are pleased to have reached this important trial milestone and are excited by the safety profile that we continue to observe for Tinlarebant,” said Dr. Hendrik Scholl, chief medical officer of Belite Bio. “Following the DSMB’s recommendation to continue the trial with the current sample size, we remain on track to complete the trial by Q4 2025, including a three-month follow-up period, and look forward to building on the promising efficacy results observed in our completed phase 2 trial for Tinlarebant.”
The pivotal phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, global and multi-center study, designed to evaluate the safety and efficacy of Tinlarebant in adolescent Stargardt disease patients. The DRAGON trial has sites in 11 jurisdictions, including the US, the United Kingdom, Germany, France, Belgium, Switzerland, Netherlands, China, Hong Kong, Taiwan, and Australia. The study enrolled 104 subjects with a 2:1 randomization (active:placebo). The primary efficacy endpoint is the growth rate of atrophic lesion, along with the assessment of safety and tolerability of Tinlarebant.
