Belite Bio (BLTE) Deutsche Bank ADR Virtual Investor Conference summary

Key developments and pipeline updates

• Focused on developing Tinlarebant, an oral therapeutic for Stargardt's disease and geographic atrophy, both linked to toxic vitamin A byproducts.

• Completed a two-year open-label phase II study in adolescent Stargardt's patients, showing promising safety and efficacy; phase III enrollment completed with 104 subjects, interim analysis expected late 2024 to early 2025.

• Launched DRAGON II phase 2/3 study in Japan, U.K., and U.S., leveraging Pioneer Drug Designation for potential accelerated registration.

• PHOENIX phase III study in geographic atrophy is over halfway enrolled, with interim data expected Q1 2026 and top-line results in 2027.

• Added Dr. Hendrik Scholl, a leading expert in retinal diseases, to the management team.

Scientific rationale and mechanism of action

• Tinlarebant targets retinol binding protein 4 (RBP4) to reduce vitamin A delivery to the eye, aiming to slow toxic bisretinoid accumulation.

• Bisretinoids are implicated in early retinal pathology in both Stargardt's and geographic atrophy; reducing their formation may slow disease progression.

• No FDA-approved treatments exist for Stargardt's or oral therapies for geographic atrophy, highlighting significant unmet need.

• Strong patent portfolio with 14 active families, some expiring as late as 2040.

Clinical trial results and data insights

• Phase II Stargardt's study showed 80% reduction in RBP4, with 42% of subjects showing no conversion to atrophic lesions over two years.

• Lesion growth rate in treated subjects was significantly lower than natural history controls from the PROGSTAR study.

• AI-based image analysis revealed additional macular lesions and demonstrated halted lesion growth after 16 months of treatment.

• Visual acuity stabilized, with mean annual loss of 2.5 letters versus typical 5–6 letter loss.