Belite Bio (BLTE) investor relations material

Company overview and lead program

• Focuses on ophthalmology and retinal diseases, spun out from Lin BioScience in 2018 and listed in 2022.

• Lead drug tinlarebant targets Stargardt disease, a severe unmet need with no current treatments.

• Clinical programs have shown strong data and smooth progression.

Stargardt disease and clinical results

• Stargardt is a monogenic, autosomal recessive disease affecting about 53,000 in the U.S., causing early vision loss.

• Tinlarebant, an oral once-daily tablet, reduced lesion growth by 36% over two years in phase III trials.

• Breakthrough Therapy status was granted after interim analysis and regulatory engagement.

• Physicians are highly enthusiastic about the efficacy and safety profile, with strong demand anticipated.

Regulatory and market strategy

• Rolling NDA submission began in April, with completion expected by mid-June and a decision six months later.

• Regulatory alignment is strong between U.S. and EU, with parallel submissions planned for rapid global launch.

• FDA's shift to single-study approval for rare diseases supports the current regulatory path.