Belite Bio (BLTE) investor relations material

Executive summary

• FDA accepted the New Drug Application for tinlarebant with priority review for Stargardt disease type 1 (STGD1), setting a PDUFA date of February 12, 2027, based on strong Phase 3 DRAGON study data.

• Phase 3 DRAGON trial results showed tinlarebant stabilized or reduced QAF values, indicating efficacy in halting or slowing retinal degeneration.

• Enrollment completed for DRAGON II (STGD1) and PHOENIX (GA) Phase 3 trials; commercial launch preparations are underway with high engagement from medical and patient communities.

Financial highlights

• R&D expenses for Q2 2026 were $18.2M, up from $11M in Q2 2025, mainly due to a milestone royalty payment; SG&A expenses rose to $16.7M from $6.5M.

• Non-GAAP R&D expenses were $17.2M, up from $8.6M year-over-year; non-GAAP SG&A was $10.9M vs. $1.3M.

• GAAP net loss was $28.4M, compared to $16.3M in Q2 2025; non-GAAP net loss was $21.6M, up from $8.7M.

• Cash and cash equivalents were $279.9M as of June 30, 2026, with investments in U.S. Treasury bills and notes rising to $500.1M; total liquidity at quarter-end was $780M.

• Other income for Q2 2026 was $6.6M, mainly from interest income.

Outlook and guidance

• Focus remains on FDA approval, with European and Japanese filings planned after or in parallel with U.S. approval.

• Pediatric study (ages 3–11) initiated in London to support future label expansion below 12 years old.

• Interim analysis for the PHOENIX (GA) trial expected in Q1 2027, after the PDUFA date.

• Commercial Day event planned for September to disclose U.S. patient population data.

• Management expects to be well positioned for a strong launch and long-term success if tinlarebant is approved.