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Segment Information
 9 Months Ended
Sep. 30, 2025
Segment Reporting [Abstract]  
Segment Information Segment Information
The Company operates as one reportable and operating segment, centered around its commercialized product, ORLADEYO, and its pipeline with the goal of developing first-in-class or best-in-class oral small-molecule and injectable protein therapeutics to target difficult-to-treat rare diseases. The determination of a single segment is consistent with the consolidated financial information regularly provided to the Company’s chief operating decision maker (“CODM”). The Chief Executive Officer, as the CODM, uses consolidated, single-segment financial information for purposes of evaluating performance, making operating decisions, allocating resources, and planning and forecasting for future periods.
The CODM assesses performance and decides how to allocate resources based on consolidated net income (loss). This measure is used to monitor budget versus actual results to evaluate the performance of the segment. The CODM uses consolidated cash, cash equivalents and investments as the measure of segment assets. As of September 30, 2025 the Company’s cash, cash equivalents, and investments were $267,382, of which $14,840 was considered held for sale (see
Note 2 - Assets Held for Sale”). As of December 31, 2024 the Company’s cash, cash equivalents, and investments were $341,173.
The following table illustrates information about segment revenues, significant segment expenses, and segment net income (loss) for the three and nine months ended September 30, 2025 and 2024 (in thousands):
Three Months Ended September 30,Nine Months Ended September 30,
2025202420252024
Revenues$159,395 $117,085 $468,282 $319,178 
Less1:
Cost of product sales2,187 3,211 9,553 6,175 
Research and development
Berotralstat2,575 2,954 9,214 10,504 
BCX177257,574 1,154 13,346 6,591 
Avoralstat1,800 2,104 7,584 4,960 
Factor D Program35 1,193 454 7,299 
Research, discovery and preclinical programs5,457 3,309 11,641 8,325 
Compensation and related personnel costs13,313 14,257 38,025 42,630 
Stock-based compensation6,520 8,530 24,374 20,923 
Other non-program specific and indirect costs7,329 7,580 20,621 23,965 
Selling, general and administrative83,014 65,101 252,866 185,841 
Interest income(2,238)(3,591)(7,778)(11,176)
Interest expense19,661 24,828 64,737 74,067 
Foreign currency (gains) losses, net(35)(98)27 37 
Loss on extinguishment of debt2,740 — 6,911 — 
Other income(2,667)— (2,667)— 
Income tax (benefit) expense(769)586 1,358 1,123 
Segment net income (loss)12,899 (14,033)18,016 (62,086)
Reconciliation of segment profit or loss:
Adjustments and reconciling items— — — — 
Consolidated net income (loss)$12,899 $(14,033)$18,016 $(62,086)
1 The significant expense categories and amounts align with the segment-level information that is regularly provided to the CODM.
All material long-lived assets of the Company reside in the U.S. For geographic information about the Company’s product revenues, see “Note 3—Revenue”.