Record ORLADEYO Full-Year Revenue
Full-year ORLADEYO revenue of $601.8M, up 38% year-over-year and up 43% when excluding the European business sold in October 2025.
Strong Overall Revenue and Profitability
Non-GAAP total revenue increased 45% year-over-year in 2025; non-GAAP ORLADEYO revenues rose by approximately $169M (about 43% YoY). Non-GAAP operating profit jumped to $214M, a 198% increase YoY and the highest in company history.
Robust Balance Sheet and Financing Optionality
Ended 2025 with $337.5M in cash and investments and secured a $400M financing facility with Blackstone Life Sciences to support growth and optionality.
Pediatric Approval for ORLADEYO Pellets
FDA approved ORLADEYO pellets for children aged 2 to <12 in December 2025; pediatric launch included in 2026 guidance (expected to be a small contribution in 2026 but with substantial long-term upside).
Navenibart Clinical Efficacy and Development Progress
Phase II ALPHA-SOLAR data showed mean attack rate reductions of 92% (every 3 months) and 90% (every 6 months); mean attack rate decreased from 2.23/month to 0.16/month (fewer than two attacks per year). Median attack rate reduction was 97%. Pivotal Phase III enrollment (145 patients) is recruiting well and expected to complete around mid-2026.
Clear Regulatory & Development Timeline for Navenibart
Company plans a BLA submission by end of 2027 (driven by the need for 12 months of safety data) with a pathway to approval by late 2028, assuming trial timelines are met.
BCX17725 Early-Stage Results and Patient Study Initiation
Healthy volunteer Phase I (single/multiple ascending doses) completed with BCX17725 safe and well-tolerated; observed epidermal distribution and linear exposure; half-life ~12–19 days. Patient cohorts (Part 4 up to 12 patients) prioritized to generate data by year-end 2026.
Commercial Execution and Patient Persistence
Real-world persistence strong: pivotal trial showed just over 50% of starters stayed on ORLADEYO for 2 years with a 91% reduction from baseline; real-world 60% persistence at 12 months and nearly 50% of U.S. patients who tried ORLADEYO over the past 5 years remain on therapy. Sales & marketing generated ~4x return on ORLADEYO net sales.
Conservative, Disciplined Financial Guidance
2026 ORLADEYO revenue guidance of $625M–$645M (midpoint ≈13% growth vs. 2025 adjusted for Europe); 2026 non-GAAP OpEx guidance $450M–$470M that includes Astria expenses, with management emphasizing R&D discipline and focus on capital allocation.