Initiation of pivotal phase III clinical Trial of LBS 008 in Stargardt disease in the U.S.

“STGD1 is the most common inherited retinal dystrophy causing blurring and/or loss of central vision in both adults and children,” said Dr. Tom Lin, Belite Bio’s Chairman and CEO. “With over 30,000 STGD1 patients alone in the U.S., we have a treatment with the potential to address a large unmet need with a clear clinical pathway to bring hope to patients afflicted with this debilitating disease.”

Belite Bio has completed multiple randomized, double-blind, placebo-controlled, Phase 1 trials of LBS 008 in healthy adult subjects, including a single ascending dose, or SAD trial in 40 subjects in the U.S., and a SAD trial in 39 subjects and a multiple ascending dose, or MAD, trial in 32 healthy adult subjects in Australia. These trials were conducted to confirm the safety, toxicity, PK, and PD of LBS 008 on a range of SAD (10-50 mg in the U.S.; 25-400 mg in Australia) / MAD (5-25 mg in Australia) levels in healthy adult subjects in fasted / fed conditions. All dose levels were well tolerated.

Belite Bio is currently conducting a 2-year Phase II trial and a 2-year Phase III (DRAGON) trial of LBS 008 in adolescent STGD1 subjects. The Phase II trial has enrolled a total of 13 subjects at clinical sites in Australia and Taiwan. Preliminary data from the Phase II 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 (~0.3mm2 in size) at 6 months. Belite Bio expects the next data readout of this Phase II trial to occur in the last quarter of 2022 when all subjects have completed 12 months of treatment.

The Phase 3 (DRAGON) trial a randomized, double-masked, placebo-controlled, global and multi-center study, is designed to evaluate the safety and efficacy of LBS 008 in adolescent STGD1 patients. To date, the Company has commenced the Phase III clinical trial in the U.S., the United Kingdom, Germany, Belgium, Switzerland, Hong Kong, Taiwan, and Australia. Approximately 60 patients are targeted for enrollment in this study with a 2:1 randomization (active:placebo).

.Additionally, because the accumulation of toxic bisretinoids has also been implicated in the progression of Dry AMD, Belite Bio believes that LBS 008 has the potential to be effective for the treatment of Dry AMD as well. Belite Bio plans to initiate a Phase II/III clinical trial for Dry AMD in the 4th quarter of 2022.