Belite Bio concluded enrollment in Pivotal Global Phase 3 DRAGON Trial

Belite Bio has completed patient enrollment for a trial evaluating Tinlarebant, which is the first ever treatment for Stargardt Disease. (Image Credit: ©Przmek Klos - Adobe.Stock.com)

Belite Bio Inc announced¹ that enrollment is completed for its global Phase 3 DRAGON trial, which is for patients with Stargardt Disease (STGD1). The trial evaluated Tinlarebant, a once daily, oral tablet designed to slow disease progression in patients with STGD1 and geographic atrophy (GA) in advanced dry age-related macular degeneration (dry AMD).

Tinlarebant, also known as LBS-008, is a retinol binding protein 4 (RBP4) antagonist intended to reduce the level of ocular vitamin-A based toxins that cause STGD1 and contribute to GA, or advanced dry AMD. Tinlarbant works by modulating the amount of retinol entering the eye and reducing the formation of these toxins.

Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S. and Europe for the treatment of STGD1.¹

The Phase 3 DRAGON trial is a randomized, double-masked, placebo-controlled, and global and multi-center study. It included 90 patients across 11 different countries. All patients have been enrolled in the study with a 2:1 randomization (active:placebo).

Dr Tom Lin, CEO of Belite Bio, stated in a press release, “completing enrollment of all 90 adolescent subjects across 11 countries worldwide in the Phase 3 DRAGON trial marks an important milestone for our late-stage program in STGD1. Importantly, we are pleased to see the embrace of this much needed therapeutic opportunity by the STGD1 patient community. The DRAGON trial has potential to be the first global Phase 3 clinical trial to demonstrate a treatment benefit in patients with STGD1 and we look forward to sharing the interim safety and efficacy data in mid-2024.”

Professor Michel Michaelides, the Principal Investigator and the Chief Investigator of the DRAGON trial from Moorfields Eye Hospital in the United Kingdom added that, “It has been a pleasure and privilege to be one the recruiting sites for this promising Phase 3 DRAGON trial. The sustained slowing of disease progression demonstrated in the 18-month Phase 2 trial for patients with STGD1 is highly encouraging, and further reinforces our belief in the transformative potential of Tinlarebant to be the first FDA-approved treatment option for STGD1.”

Topline data for Tinlarebant in the Phase 2 STGD1 study is expected in the fourth quarter of 2023 and the interim data for the Phase 3 DRAGON trial is expected by mid-2024.

References

1. Belite bio completes enrollment in pivotal global phase 3 dragon trial evaluating oral tinlarebant for stargardt disease. Belite Bio, Inc. July 24, 2023. Accessed July 26, 2023. https://investors.belitebio.com/news-releases/news-release-details/belite-bio-completes-enrollment-pivotal-global-phase-3-dragon.