Belite Bio (BLTE) H.C. Wainwright 27th Annual Global Investment Conference summary

Tinlarebant clinical development and regulatory progress

• DRAGON trial for Stargardt disease completed last patient visit; top-line data expected by end of Q4 2024, with NDA submission planned for H1 2026.

• Interim analysis showed Tinlarebant is safe and well tolerated, with less than 4% withdrawal due to ocular adverse events.

• FDA granted breakthrough designation based on interim data; full approval will be sought in the U.S. and other major markets.

• DRAGON 2 trial is underway, mainly to meet Japanese regulatory requirements; not required for U.S. submission.

• PHOENIX trial in geographic atrophy (GA) fully enrolled; top-line data expected Q3 2027.

Efficacy, safety, and market potential

• Phase II data showed Tinlarebant slowed lesion growth by nearly 50% compared to natural history controls.

• Safety profile remains excellent through end of treatment.

• Estimated U.S. Stargardt prevalence is 47,000–59,000, with potential market penetration of over 20,000 patients.

• Tinlarebant expected to be effective in both adolescent and adult patients.

• Label anticipated to include all age groups.

Competitive landscape and positioning

• Nanoscope’s gene-agnostic therapy targets late-stage, low-vision patients; Tinlarebant aims to prevent vision loss earlier, making the approaches complementary.

• Tinlarebant’s oral administration offers a significant advantage over monthly intravitreal injections for GA.

• If efficacy is comparable to complement inhibitors, Tinlarebant could become first-line therapy for GA.