Belite Bio (BLTE) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Company overview and leadership

• Focuses on oral therapeutics for degenerative retinal diseases, specifically Stargardt disease and geographic atrophy (GA).

• Recently appointed Dr. Hendrik Scholl, a leading expert in Stargardt disease and AMD, as CMO.

Clinical development and trial updates

• Completed a two-year phase II open-label study in 13 adolescent Stargardt subjects, focusing on early intervention.

• Phase III studies (DRAGON I & II) are underway, with DRAGON II targeting Japan, U.K., and U.S., and leveraging a pioneer drug designation in Japan.

• PHOENIX phase III trial for GA is halfway through enrollment, aiming for 429 subjects.

Mechanism of action and disease biology

• Tinlarebant is an oral, once-daily small molecule that antagonizes the carrier protein for vitamin A, reducing its delivery to the eye.

• The approach targets early-stage disease by reducing toxic vitamin A byproducts (bisretinoids) that drive retinal degeneration.

• The drug's action is site-specific, affecting vitamin A homeostasis only in the eye.