Belite Bio (BLTE) Investor update summary

Regulatory and Clinical Development Milestones

• FDA accepted the NDA for tinlarebant in Stargardt disease type 1, granting priority review with a PDUFA date of February 12, 2027; NDA was submitted in June 2026 and accepted in August 2026.

• Tinlarebant could become the first and only approved treatment for Stargardt disease type 1, marking a transition to a commercial-stage company.

• The DRAGON phase III trial demonstrated a 36% reduction in lesion growth rate (DDAF) and a 34% reduction in total decreased autofluorescence (DAF) compared to placebo, with a strong safety profile.

• Tinlarebant is the first oral therapy to show a clinically meaningful slowdown of neurodegeneration in a retinal disease, with visual acuity remaining stable over two years.

• Achieved sustained 80% reduction in RBP4, exceeding the 70% target.

Safety Profile

• Tinlarebant demonstrated a well-tolerated safety profile over two years, with most adverse events mild and resolving during the study.

• No serious ocular adverse events were reported; non-ocular serious events were rare and mostly unrelated to treatment.

Unmet Need and Patient Impact

• Stargardt disease type 1 is the most common inherited retinal disease, leading to progressive vision loss and legal blindness, with no approved therapies.

• The disease profoundly affects daily living, independence, and mental health, as highlighted by patient testimonials.

• Approximately 53,000 people in the U.S. are estimated to have Stargardt disease, with 20,000 clinically diagnosed and about half genetically confirmed.

• ABCA4 gene mutations are the largest cause of inherited retinal disease globally, representing a major rare disease opportunity.