Clinical Trial SuccessThe Phase 3 DRAGON trial of tinlarebant in adolescent Stargardt disease met the primary efficacy endpoint and demonstrated a significant reduction in the growth rate of retinal lesions compared with placebo.
Financial PositionThe company has a strong cash position, which should provide funding through the likely commercial launch of tinlarebant.
Regulatory ApprovalsTinlarebant has been granted Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., which should warrant accelerated approval in all territories.