Belite Bio Inc. presented one-year data from their ongoing two-year Phase 2 clinical study of Tinlarebant in STGD1 as part of the oral presentation series at the Annual Meeting of the American Academy of Ophthalmology (AAO) held during September 30 - October 3, 2022 at McCormick Place, Chicago. To date, all 13 patients have completed one-year of treatment in the ongoing two-year Phase 2 study of Tinlarebant. The results for safety and tolerability assessments, and retinal imaging data have been collected for the evaluation of disease progression.

Images from spectral-domain optical coherence tomography imaging, an imaging modality that permits visualization of the retinal anatomy, have shown a stabilization of retinal thickness in many subjects. Fundus autofluorescence imaging shows no autofluorescence expansion (QDAF) in 7 of 13 (53.8%) subjects, and 12 of 13 (92.3%) subjects also show no atrophic lesion (DDAF) after one year of treatment. More importantly, 9 of 13 (69.2%) subjects show a stabilization or improvement in visual acuity throughout the one-year treatment period.

According to the international prospective study of STGD1 (the ProgStar Study), childhood-onset patients with no atrophic lesion (DDAF) at baseline experienced an average lesion growth rate of 0.66 mm(2) for the left eyes and 0.74 mm(2) for the right eyes at one year. In addition, the Prospective Cohort Study of Childhood-Onset Stargardt Disease by Georgiou et al. reported an average atrophic lesion growth rate (DDAF) of 0.69 mm(2) /year for children.

Belite Bio's one-year data from ongoing Phase 2 study showed an average lesion growth rate of 0.03 mm(2) /year, demonstrating a promising trend toward halting or slowing the disease progression in the study cohort. Belite Bio expects the next data readout in its Phase 2 STGD1 study to occur during the second quarter of 2023 when all subjects will complete 18 months of treatment.