Belite Bio (BLTE) investor relations material

Disease background and unmet need

• Stargardt disease is a recessively inherited retinal disorder caused by ABCA4 mutations, leading to blindness and affecting about 53,000 people in the U.S., with a global prevalence of 1 in 7,000.

• No approved therapies exist; current management is limited to lifestyle advice such as avoiding vitamin A supplementation and protecting eyes from sunlight.

• The disease presents a significant market opportunity due to its prevalence and lack of treatments.

Product mechanism and early development

• Tinlarebant is an oral RBP4 antagonist that reduces vitamin A delivery specifically to the eye, lowering toxic bisretinal accumulation in photoreceptors.

• Preclinical mouse models showed that reducing RBP4 by 80% halted photoreceptor degeneration; early clinical trials in adolescents stabilized vision over two years.

• The main clinical endpoint is the progression rate of DDAF lesions, an FDA-approved measure.

Phase III DRAGON trial results

• The DRAGON trial enrolled 104 patients globally, randomizing them 2:1 to Tinlarebant or placebo, with a primary endpoint of DDAF lesion growth rate.

• Tinlarebant reduced DDAF progression by 36% over two years, with high statistical significance (P=0.0033; <0.0001 with covariance matrix).

• Secondary endpoints showed a 34% reduction in DAF lesion progression, with similar efficacy in both eyes.