Belite Bio (BLTE) investor relations material

Key presentations and clinical updates

• Tinlarebant, an oral RBP4 antagonist, demonstrated a 36% reduction in lesion growth in Stargardt disease over two years in the phase III DRAGON trial, with statistically significant results in both primary and secondary endpoints.

• The therapy showed a sustained 80% reduction in RBP4 levels and was well-tolerated, with most adverse events being mild and resolving during the study.

• No significant change in visual acuity was observed, aligning with the natural history of Stargardt disease, but structural endpoints showed robust improvement.

• The PHOENIX phase III trial in geographic atrophy is fully enrolled, targeting early intervention in patients with small lesions.

• Tinlarebant has received breakthrough therapy, fast track, orphan drug, and pioneer designations in multiple regions, with a rolling FDA submission underway.

Market opportunity and strategic positioning

• Stargardt disease affects over 50,000 in the U.S. and more than 100,000 in China and Europe; geographic atrophy impacts about 1 million in the U.S. and 5 million globally.

• No FDA-approved treatments exist for Stargardt disease or oral therapies for geographic atrophy, representing significant unmet needs.

• Tinlarebant’s mechanism targets bisretinoid-mediated toxicity, addressing the root cause of retinal degeneration.

• The company holds 14 active patent families, with composition of matter protection until at least 2040.

• Commercial strategy considers pricing differences between rare and common indications, aiming to maximize opportunities in both Stargardt and geographic atrophy.

Regulatory and future directions

• The FDA has accepted DDAF lesion growth as a primary endpoint for Stargardt trials, and the company has initiated a rolling NDA submission.

• Early intervention, especially before significant visual acuity loss, is anticipated to yield even greater benefits, though long trial durations are challenging.

• Tinlarebant is the first oral therapy to show a clinically meaningful slowdown in neurodegeneration for retinal diseases.

• The company’s approach is supported by robust natural history data and standardized imaging endpoints validated in prior studies.

• Future trials may explore earlier-stage patients, but statistical significance would require extended follow-up.