Belite Bio, Inc Q4 FY2025 earnings call

2025 was a year of significant progress with key milestones like Phase III Pivotal Dragon Trial top-line result showing 36% reduction in ocular lesion growth rate. Dragon 2 study had 72 enrolled subjects as of Feb 27 with target of 60. Phase 3 Phoenix trial in GA completed enrollment with 130 subjects. Completed a $402 million public offering. 2026 will be transformative with top priority being NDA submission to FDA in Q2 and commercialization preparation for Stargardt disease. Hired key leadership for sales, market access, etc. to support launch preparation

For the fourth quarter, R&D expenses were 14.6 million compared to 7.3 million in Q4 2024. Non-GAAP R&D expenses (excluding share-based compensation) were 12.2 million vs 5.7 million in 2024. SG&A expenses were 13.5 million vs 4.2 million in 2024. Non-GAAP SG&A expenses were 4.2 million vs 1.5 million in 2024. Net loss was 25.3 million vs 10.1 million in 2024. Non-GAAP net loss was 13.6 million vs 5.9 million in 2024. For the full year, R&D expenses were 45.4 million vs 29.9 million in 2024. Non-GAAP R&D expenses (excluding share-based compensation) were 36.2 million vs 26.2 million in 2024. SG&A expenses were 38.9 million vs 10.1 million in 2024. Non-GAAP SG&A expenses were 9.1 million vs 4.8 million in 2024. Net loss was 77.6 million vs 36.1 million in 2024. Non-GAAP net loss was 38.7 million vs 27.2 million in 2024. Cash at end of 2025 was 772.6 million vs 145.2 million in 2024

Expect NDA submission to FDA in Q2 2026. For next three years, R&D related to existing pipeline including NDA submission expected to cost about 150 million. Commercialization for next three years expected to be between 200-250 million. Expect to launch StarverDZ by Q1 2027

Q: On the NDA submission, is it a rolling submission and role of Dragon 2?

A: It will be a rolling submission, on track for Q2 submission. Dragon 2 is for Japan only as Japanese authorities want data on Japanese patients.

Q: Guidance on pricing and key gating items before NDA submission?

A: Still early to set price. Key gating item is waiting for clinical study report to finalize and submit.

Q: Timing of launch and launch team?

A: Expect to launch by Q1 2027 with sales team of 25-30. Challenges include getting patients, physicians aware, genetic testing and insurance coverage.

Q: Filing plans OUS and timing of interim look for GA work?

A: Priority is FDA US, then EMA, Japan, etc. Interim look for GA work expected in second half of the year.

Q: Threshold for GA trial success and capital allocation for LBS 009?

A: For GA, if reach 15-20% lesion growth inhibition like injectable drugs would be success. No plans for LBS 009 yet, prioritizing Tadaraban.

Q: Label for Stargardt and ex-US regulatory strategy?

A: Pressing for full label for subjects 12 and older as it's the same disease. Ex-US regulatory strategy has FDA US as top priority then EMA, Japan, etc.

Q: Prevalence of Stargardt and impact of treatment?

A: True prevalence estimated at 53,000 in US with ABCO4 mutated retinal disease. Having a treatment could help build awareness and uncover undiagnosed patients.

Q: Pediatric expansion for Stargardt?

A: Have approved pediatric investigational plan starting in April 2026 to study 3-11 year olds