BELITE BIO, INC Q1 FY2025 earnings call

• Clinical progress: Tinlarebant's Phase 3 DRAGON trial for Stargardt's disease had DSMB recommend trial proceed without sample size change and submit data for regulatory review; DRAGON 2 trial enrolled 16 of ~60 subjects. PHOENIX trial for geographic atrophy had enrolled 464 subjects, expected full enrollment of 500 in Q3 2025.
• Safety/tolerability: DRAGON trial had <10% withdrawal rate, <3.8% due to ocular adverse events, visual acuity stabilized in majority of subjects.
• Financials: Cash position strong with $157.4M in cash, liquidity funds, etc. at end of Q1, expecting four years of cash runway.

There are no distinct product segments with revenue contributions. Instead, focus is on clinical progress of Tinlarebant. Financials: Q1 2025 had R&D expenses of $9.4 million (up from $6.8M same period prior), G&A expenses of $6.1 million (up from $1.6M same period prior), net loss of $14.3 million (up from $7.9M same period prior). Cash increased by $12.3 million to $157.4 million with 4 years of cash runway.

• Expect four years of cash runway with current cash. - Operating expenses expected to be slightly higher in remainder of 2025 and next year, then decrease. - Expect to complete current clinical trials with existing cash.

• Regulatory risks: Uncertainty around potential regulatory changes, but data from trials is positive. - PHOENIX trial dropout rate around 20%, but lower than some previous studies for similar trials.

Q: Could you provide update about the discontinuation rates, and how the enrollment is ongoing in the PHOENIX trial? And any update on regulatory meetings for Stargardt disease trial requirements?

A: PHOENIX dropout rate ~20%, lower than some previous studies; enrollment ongoing. Still scheduling meetings with regulators for Stargardt disease trial requirements.

Q: Recent interactions with the FDA regarding Stargardt disease and thoughts on regulatory risk given agency changes? And goalpost for Phase 3 data?

A: Haven't met with FDA yet but scheduled to; no major regulatory risk concerns as data speaks for itself. Phase 3 DRAGON study powered to detect 35% treatment effect between placebo and active, expecting close to anticipated effect size.

Q: Comment on potential strategy regarding approval and launching of drug in ex U.S. territories, particularly Japan, and impact on U.S. pricing?

A: Tinlarebant not launched yet, monitoring impact of policy but no immediate answer.

Q: Will operating expenses continue to rise during remainder of 2025?

A: Operating expenses expected to be slightly higher than Q1 in remainder of 2025 and next year, then decrease.

Q: On manufacturing, inventory and supply chain structure?

A: Tinlarebant manufactured in U.S. and other geographies, tariff not affecting.

Q: Timing and conditions for interim geographic atrophy trial? Any additional sample size reestimations for PHOENIX study?

A: Meeting with regulators, no plan for additional sample size reestimation in PHOENIX as enrollment going smoothly