Belite Bio (BLTE) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Leadership and expertise

• Dr. Scholl appointed as Chief Medical Officer, bringing unmatched clinical and research expertise in Stargardt disease and geographic atrophy.

• Dr. Scholl previously chaired the phase III Stargardt trial and led the largest natural history study in the field.

Clinical trial progress and timelines

• Phase III DRAGON trial for Stargardt disease is progressing well, with most patients past the 12-month visit; interim analysis expected late 2024 or early 2025.

• Top-line phase III DRAGON data anticipated in early 2026.

• DRAGON II trial in Japan focuses on PK/PD data first, with full data expected after the main DRAGON trial.

• PHOENIX phase III trial for geographic atrophy is enrolling globally, aiming to close enrollment in Q1 2025, with top-line data expected in Q1 2027.

Efficacy, safety, and competitive landscape

• Tinlarebant is an oral, once-daily treatment targeting lesion growth in Stargardt and GA, with strong safety and efficacy signals in phase II open-label studies.

• No systemic drug-related adverse events observed; mild, transient ocular AEs indicate intended biological effect.

• Competing oral therapies exist, but Tinlarebant is positioned for early intervention, especially in juveniles and adolescents.