Belite Bio (NASDAQ:BLTE), a clinical-stage biopharmaceutical company focused on developing therapies for degenerative retinal diseases, today announced positive topline results from its pivotal Phase 3 DRAGON trial evaluating tinlarebant, an oral retinol binding protein 4 (RBP4) antagonist, for the treatment of adolescent Stargardt disease.
The trial met its primary endpoint, demonstrating a 35.7% reduction in the growth rate of macular lesion area (atrophic lesion progression) compared with placebo over the 24-month treatment period, with the result achieving statistical significance.
Tinlarebant was generally well tolerated, consistent with prior studies, and the company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration in the second quarter of 2026.
To support late-stage development, regulatory submissions and potential commercialization, Belite Bio completed a $402 million public offering in early 2026.
As of December 31, 2025, the company reported cash and cash equivalents of $352.9 million and total investments of $419.7 million, providing a strong financial position to advance its pipeline through key milestones.
Clinical program updates include the DRAGON II trial (a global Phase 3 study in adolescent Stargardt patients) reaching its targeted enrollment of 72 subjects as of February 27, 2026, and the PHOENIX Phase 3 trial (evaluating tinlarebant in geographic atrophy secondary to age-related macular degeneration) completing enrollment with 530 subjects.
For the full year 2025, Belite Bio reported a GAAP net loss of $77.6 million, primarily reflecting increased research and development expenses associated with the ongoing pivotal trials and manufacturing scale-up for tinlarebant, partially offset by interest income on cash holdings.