Belite Bio (BLTE) Piper Sandler Virtual Ophthalmology Day summary

Company and pipeline overview

• Focus on severe retinal diseases with Tinlarebant as lead compound, targeting Stargardt disease and geographic atrophy secondary to AMD.

• Tinlarebant completed a phase III trial (DRAGON) for Stargardt disease and is being developed for geographic atrophy.

• Both diseases share similar pathophysiology involving toxic bisretinoid accumulation due to vitamin A metabolism.

• Tinlarebant acts as a retinol binding protein 4 antagonist, reducing vitamin A availability in the eye by 80%.

• Oral administration and favorable safety profile distinguish Tinlarebant from previous therapies.

Clinical development and regulatory strategy

• FDA involved early in endpoint selection, with DDAF (definitely decreased autofluorescence) as primary endpoint.

• Breakthrough designation received for Tinlarebant in Stargardt disease; priority review is anticipated.

• EMA review may be more favorable due to oral administration, higher efficacy, and safety compared to prior GA treatments.

• Structure-function correlation data from ProgStar and other studies support regulatory submissions.

• Visual acuity decline is slow in Stargardt, making structural endpoints more practical for trials.

Ongoing and future trials

• DRAGON II trial initiated to enable approval in Japan, with similar design to DRAGON1 and full enrollment.

• DRAGON II includes patients from Japan, U.S., and U.K., with 1:1 randomization for greater statistical power.

• Consistent dosing and trial design across all studies, including PHOENIX for geographic atrophy.

• DRAGON II data will provide additional support for global regulatory filings.