Overview
- The 24-month DRAGON trial enrolled 104 patients aged 12 to 20 with genetically confirmed STGD1 and met its primary endpoint (p=0.0033).
- Treatment reduced lesion growth by 35.7% in the study eye and by 33.6% in the fellow eye, with additional slowing on decreased autofluorescence measures.
- Tinlarebant was generally well tolerated, with four treatment-related discontinuations and mostly mild, resolving ocular side effects such as xanthopsia and night-vision issues.
- The 5 mg dose lowered serum RBP4 by about 80% from baseline, with levels trending back toward baseline after drug cessation.
- If cleared, tinlarebant could become the first approved therapy for Stargardt disease, and the company is also running a Phase 3 study in geographic atrophy.