ARVO 2023: Belite Bio presents 18-month interim data from Phase 2 study of LBS-008 in adolescent Stargardt disease

Belite Bio is currently conducting a two-year Phase 2 study and is enrolling patients in a two-year Phase 3 study (DRAGON) of in adolescent STGD1 subjects and plans to begin enrolling patients in a two-year Phase 3 study (PHOENIX) of LBS-008 in Geographic Atrophy (GA) in mid 2023. The copany presented data at ARVO 2023 in New Orleans. (Image courtesy of Adobe Stock)

Belite Bio Inc. presented 18-month data from its ongoing two-year, open-label Phase 2 clinical study of LBS-008 (Tinlarebant) as part of the poster presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in New Orleans.

The company noted John Grigg, MD, PhD, 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,” Grigg said. “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, 12 patients* have completed 18 months of treatment in the ongoing two-year Phase 2 study of LBS-008. Routine assessments were performed to evaluate safety and tolerability of LBS-008. 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.

“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,” Grigg explained.

“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 Nathan L. Mata, PhD, 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 in adolescent STGD1 subjects and plans to begin enrolling patients in a two-year Phase 3 study (PHOENIX) of LBS-008 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.

About DRAGON Study
The two-year DRAGON Phase 3 study (NCT05244304) is a Multi-Center, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of LBS-008.

DRAGON is designed to evaluate the safety and efficacy of LBS-008 in adolescent STGD1 patients. Approximately 90 patients are targeted for enrollment in this study with a 2:1 randomization (active:placebo). To date, Belite Bio has commenced this Phase 3 study in the U.S., the United Kingdom, Germany, Netherlands, France, Belgium, Switzerland, China, Hong Kong, Taiwan, and Australia.

About LBS-008
LBS-008 is a novel oral therapy which is intended to reduce the accumulation of toxins in the eye that cause STGD1 and contribute to GA, or advanced dry AMD. These toxins are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. The therapeutic works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, LBS-008 reduces the formation of these toxins. LBS-008 has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., and Orphan Drug Designation in the U.S. and Europe for the treatment of STGD1.

* The Phase 2 clinical trial of Tinlarebant in adolescent STGD1 initially enrolled 13 subjects in Australia and Taiwan. Among the 13 subjects, one subject in Australia was lost to follow up and therefore 12 subjects with complete 18-month data were evaluated.