Belite Bio (BLTE) investor relations material

Company initiatives and clinical progress

• Oral once-daily tinlarebant targets Stargardt disease and geographic atrophy, both with significant unmet needs.

• NDA for Stargardt disease submitted in the U.S. and Japan; FDA granted priority review with a PDUFA date of February 12 next year.

• Phase III study for geographic atrophy completed enrollment and is in execution globally.

Regulatory review and endpoints

• FDA has been supportive, recognizing the unmet need and is familiar with the trial design and endpoints.

• Primary endpoint based on imaging to measure photoreceptor preservation is accepted by the FDA for both indications.

• Advisory committee meeting is not anticipated due to clear understanding of endpoints.

Label expectations and patient population

• Data from patients aged 12–20 is believed to be generalizable to all ages with Stargardt disease.

• Genetic confirmation of ABCA4 mutations is standard for diagnosis and likely to be required for treatment eligibility.