• Tinlarebant (aka LBS-008) continues to be safe and well tolerated in adolescent Stargardt Disease (STGD1) subjects at the 18-month time point
• A continued trend of slowing expansion of autofluorescence was observed
• The growth rate of incident atrophic retinal lesions was reduced compared to a natural history study of the disease (“ProgStar”)
• Visual acuity was stabilized with no significant loss, and no clinically significant changes in retinal thickness observed over 18 months of treatment

SAN DIEGO, April 25, 2023 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE), a San Diego based clinical stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which have significant unmet medical needs, today presented 18-month data from its ongoing two-year, open-label Phase 2 clinical study of Tinlarebant in adolescent STGD1 patients (“LBS-008-CT02”) as part of the poster presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting (Presentation: April 25^th 8:45 am – 10:45 am, CST).

Professor John Grigg, the study’s Principal Investigator and Head of Specialty Clinical Ophthalmology at the University of Sydney and Consultant Ophthalmologist at the Sydney Children’s Hospitals Network at Westmead and Sydney Eye Hospital was the presenter of this interim study data. “It is great to observe that the 18-month interim data showed a consistent safety profile in patients treated with Tinlarebant,” said Dr. John Grigg. “Belite Bio’s current Phase 2 data also continued to demonstrate a promising trend toward slowing the disease progression in the study cohort.”

To date, twelve patients* have completed 18 months of treatment in the ongoing two-year Phase 2 study of Tinlarebant. Routine assessments were performed to evaluate safety and tolerability of Tinlarebant. Retinal imaging data have been collected for the evaluation of disease progression in all subjects. At Month 18, data from fundus autofluorescence (FAF) imaging revealed that nearly 60% of subjects (seven out of 12) showed no incident atrophic retinal lesions. Mean visual acuity was stabilized in the study cohort throughout the 18-month treatment period. Nine of 12 patients experienced mild xanthopsia/chromatopsia and delayed dark adaptation and one of 12 patients experienced night vision impairment, all of which were mild in severity and well-tolerated. A copy of the poster is available here (LINK).

“It is important to note that when compared with ProgStar study participants with only questionably decreased autofluorescence lesions at baseline, LBS-008-CT02 subjects exhibited a smaller increase (0.2 ± 0.1 mm²) in definitely decreased autofluorescence (DDAF) lesion size compared to ProgStar subjects (0.4 ± 0.3 mm²) after 18 months on study,” said Dr. John Grigg.

“We are very encouraged by the 18-month treatment results from our Phase 2 study as a majority of the subjects showed no transition to atrophic (DDAF) lesions, and in those subjects with transition to DDAF lesions, the DDAF lesion progression rate was slowed compared to a study of the natural history of disease,” said Dr. Nathan L. Mata, Chief Scientific Officer of Belite Bio. “The Phase 2 data presented at ARVO continue to reinforce that this investigational therapy could be a promising oral treatment for STGD1 patients.”

Belite Bio is currently conducting a two-year Phase 2 study and is enrolling patients in a two-year Phase 3 study (DRAGON) of Tinlarebant in adolescent STGD1 subjects and plans to begin enrolling patients in a two-year Phase 3 study (PHOENIX) of Tinlarebant in Geographic Atrophy (GA) in mid 2023. Belite Bio expects the next data readout in the Phase 2 STGD1 study to occur during the fourth quarter of 2023 when all subjects are expected to complete two years of treatment.