Belite Bio (BLTE) Morgan Stanley 24th Annual Global Healthcare Conference summary

Company overview and pipeline

• Originated as a spin-off from Lin BioScience, focusing on rare retinal diseases.

• Lead asset tinlarebant targets Stargardt disease and geographic atrophy, with phase III trials completed or ongoing.

• NDA for Stargardt disease accepted by FDA with priority review; PDUFA date set for February 12.

• DRAGON II phase III trial underway for Japanese regulatory submission.

• PHOENIX phase III trial for geographic atrophy is fully enrolled.

Disease background and patient population

• Stargardt disease is the most prevalent inherited retinal disease, leading cause of blindness in youth.

• Prevalence is about 1 in 6,500 in European descent, lower in East Asia, higher in African descent.

• Estimated 47,000-59,000 affected in the U.S., with 20,000 clinically diagnosed.

• Diagnosis is straightforward with modern imaging; most patients will be managed by retinal specialists.

Tinlarebant mechanism and clinical efficacy

• Tinlarebant reduces toxic bisretinoid accumulation in the retina by targeting RBP4, lowering vitamin A influx.

• Phase III DRAGON trial used DDAF lesion size as the primary endpoint, recognized by FDA.

• Tinlarebant slowed lesion progression by 36% over two years compared to placebo.

• Fellow eye data showed similar efficacy, supporting systemic benefit.