Belite Bio Inc. announced the approval from the National Medical Products Administration (NMPA) of China to initiate the Phase 3 clinical trial of LBS-008 in adolescent STGD1 in China. Belite Bio's DRAGON trial is a 2-year Phase 2 trial and a 2-year Phase 3 trial of LBS-008 in adolescent STGD1 subjects which is currently underway. The Phase 2 trial has enrolled a total of 13 subjects at clinical sites in Australia and Taiwan.

Preliminary data from the Phase 2 trial at the first 6-month interval shows that 8 of the 13 patients (or 61.5%) recorded a gain in best-corrected visual acuity (BCVA) in at least one eye, including 2 patients who recorded a BCVA gain in both eyes. In addition, there were no atrophic lesions in any of the 13 subjects at study start and only 1 subject showed evidence of a retinal lesion (0.3mm(2) in size) at 6-months. Belite expects the next data readout of this Phase 2 trial to occur in the last quarter of 2022 when all subjects have completed 12 months of treatment.

The DRAGON trial is a phase 3, randomized, double-masked, placebo-controlled, global and multi-center study, designed to evaluate the safety and efficacy of LBS-008 in adolescent STGD1 patients. To date, the Company has commenced the DRAGON trial in the U.S., the United Kingdom, Germany, Belgium, Switzerland, Hong Kong, Taiwan, and Australia. The accumulation of toxic bisretinoids have also been implicated in the progression of Dry AMD, a disease with a huge unmet need, which primarily affects the elderly and shows a pathophysiology that is similar to that of STGD1.

This finding has led to the sponsorship and endorsement of LBS-008 by the NIH Blueprint program as a promising first-in-class oral medication to slow or halt the progression of Dry AMD. Belite believes that LBS-008 has the potential to be an effective early intervention treatment to maintain the health of retinal tissues in Dry AMD. Belite plans to initiate a Phase 2/3 clinical trial for Dry AMD in the fourth quarter of 2022.

About LBS-008 LBS-008 is a novel oral therapy intended as an early intervention to prevent the buildup of toxins in the eye that cause STGD1 and contribute to Dry AMD. These toxins are by-products of vitamin A in the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. LBS-008 works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for transport of retinol into the eye.

By modulating the amount of retinol entering the eye, LBS-008 reduces the formation of vitamin A-based toxins which have been implicated in STGD1 and Dry AMD in order to maintain the health of retinal tissues. LBS-008 has been granted Fast Track Designation, Rare Pediatric Disease Designation in the U.S., and Orphan Drug Designation in the U.S. and Europe for the treatment of STGD1. Stargardt Disease 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 gene (ABCA4) which results in massive accumulation of toxic vitamin A byproducts (known as 'bisretinoids') in the retina leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging have helped ophthalmologists identify and monitor disease progression. STGD1 and Dry AMD share a similar pathophysiology characterized by excessive accumulation of cytotoxic bisretinoids, retinal cell death, and loss of vision.

Vision loss occurs slowly, despite peripheral expansion of 'dead retina', until the disease reaches the center of the eye (the macula). Dry Age-related Macular Degeneration Dry AMD is a leading cause of vision loss in the U.S. There are no approved treatments available for Dry AMD. There are an estimated 11 million Dry AMD patients in the U.S. and over 196 million patients worldwide with an estimated global direct healthcare cost of US$255 billion.