Belite Bio Inc. presented final data from a 24-month, Phase 2 study of Tinlarebant in adolescent STGD1 (?LBS-008-CT02?) at the American Association of Ophthalmology (AAO) Annual Meeting. A total of 12 adolescent STGD1 subjects aged 12-18 years completed 24 months of treatment in the Phase 2 study of Tinlarebant. Key study findings: Tinlarebant was safe and well-tolerated with no withdrawals due to adverse events; Retinal imaging showed that 5 of 12 subjects remained free of atrophic retinal lesions (referred to as definitely decreased autofluorescence or DDAF) after 24 months of Tinlarebant treatment; A comparison of the 24-month DDAF lesion growth between Tinlarebant-treated subjects and ProgStar participants possessing similar baseline characteristics (aged =18 years) showed a sustained lower DDAF lesion growth in Tinlarebant-treated subjects over the 24-month treatment period (p<0.001); Visual acuity was stabilized in majority of subjects during the study with a mean loss of five letters following 24 months of treatment (a loss of <10 letters is not considered clinically significant); The 2-year, Phase 3 study (DRAGON) is a Multi-Center, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of TinlaRebant in the Treatment of StArGardt Disease in AdOlesceNt Subjects.

This study has completed recruitment (104 subjects) and one-year interim data are expected during mid to late 2024. Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in GA, or advanced Dry AMD. Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye.

Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. 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.

STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression.

Currently, there are no FDA approved treatments for STGD1. Importantly, STGD1 and GA, or advanced Dry AMD, share a similar pathophysiology, which is characterized by the excessive accumulation of bisretinoids, retinal cell death, and progressive loss of vision. Vision loss occurs slowly, despite peripheral expansion of ?dead retina,?

until the disease reaches the center of the eye (the macula). Therefore, Belite Bio intends to evaluate safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). Dry AMD is a leading cause of vision loss in older adults.

Geographic Atrophy, or GA, is the advanced stage of Dry AMD. Currently, there are no FDA approved orally administered treatments for GA and no FDA approved therapies for the other stages of Dry AMD other than GA. There are an estimated 20 million AMD patients in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of USD 255 billion.