Belite Bio (BLTE) Q4 2024 earnings summary

Executive summary

• Advanced Tinlarebant as a first-in-class oral therapy for Stargardt disease (STGD1) and geographic atrophy (GA), with global phase III trials (DRAGON, PHOENIX) and phase II/III trials (DRAGON II) underway.

• Tinlarebant received rare pediatric disease, fast-track, and orphan drug designations in the U.S., Europe, and Japan, highlighting significant unmet need.

• No FDA-approved treatments exist for STGD1 or oral treatments for GA, representing large unmet market opportunities.

• Interim analysis of the phase III DRAGON trial in STGD1 led to DSMB recommending continuation without changes and submission for regulatory review.

• PHOENIX phase III trial in GA increased enrollment to 500 subjects due to strong recruitment, with completion expected by Q3 2024.

Financial highlights

• R&D expenses were $29.9M in 2024, up from $24.8M in 2023, mainly due to increased royalty payments, share-based compensation, and trial milestones.

• G&A expenses rose to $10.1M in 2024 from $6.8M in 2023, driven by higher share-based compensation.

• Net loss was $36.1M in 2024, compared to $31.6M in 2023.

• Cash and investments totaled $145.2M at year-end 2024, up from $88.2M at year-end 2023.

• $15M raised in a registered direct offering in February 2025, with potential for an additional $15M from warrant exercises.

Outlook and guidance

• Four-year cash runway projected, not including costs for a potential second GA phase III study.

• Regulatory submissions for Tinlarebant in STGD1 are being prepared following DSMB recommendation.

• If positive interim results are seen in the PHOENIX GA trial, a second phase III trial may be expedited.

• DRAGON trial for STGD1 on track for completion by Q4 2025, including three-month follow-up.

• Company remains well positioned to reach key milestones with recent capital raise.