Belite Bio Inc. announced that, following discussions with the FDA, it has finalized the study design for its planned Phase 3 clinical trial to evaluate efficacy and safety of Tinlarebant (a/k/a LBS-008) in patients with geographic atrophy (GA) associated with Dry AMD. Tinlarebant is a novel oral therapy intended as an early intervention to prevent the accumulation of vitamin A-based toxins (bisretinoids) that cause STGD1 and contribute to pathogenesis in Dry AMD. Bisretinoids are formed as by-products of vitamin A in the visual cycle.

Tinlarebant works by reducing the level of serum RBP4, the carrier protein which transports the retinol to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids to preserve the health of retinal tissues. 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 a dysfunctional retina-specific protein (ABCA4) which causes an early, aberrant accumulation of cytotoxic byproducts of vitamin A in the retina leading to retinal cell death and progressive loss of vision. There are currently no approved treatments available for STGD1. Dry AMD is a leading cause of vision loss in the U.S. Dry AMD has a heterogenous etiology but shows a pathophysiology that is similar to that of STGD1 in which the excessive accumulation of cytotoxic bisretinoids leads to retinal cell death and loss of vision.

There are an estimated 11 million patients with dry AMD in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of USD 255 billion. An advanced vision-threatening form of dry AMD is Geographic Atrophy (GA), which is characterized by progressive atrophy of the retinal pigment epithelium and underlying choriocapillaris, followed by loss of photoreceptors within the retina, and ongoing visual impairment. Over 8 million people are affected worldwide with GA, which is approximately 20% of all individuals with AMD.

The incidence of GA is expected to rise as the age-burden of developed countries is increasing. There are currently no approved treatments available for Dry AMD.