BioCryst Pharmaceuticals (NASDAQ:BCRX) outlined its full-year 2025 performance and priorities for 2026 during its fourth-quarter conference call, highlighting strong growth for its hereditary angioedema (HAE) franchise, continued profitability, and advancing late-stage and early-stage pipeline programs.
2025 results driven by ORLADEYO growth
President and CEO Charlie Gayer said the company closed 2025 with “strong momentum,” reporting full-year ORLADEYO revenue of $601.8 million, up 38% year-over-year and 43% excluding the European business that BioCryst sold in October. Gayer framed HAE as a “structurally segmented market,” arguing that patient needs, preferences, and real-world experience drive treatment choices rather than a “winner-take-all efficacy race.”
Pediatric expansion: ORLADEYO Pellets approved
BioCryst highlighted the FDA’s approval in December 2025 of ORLADEYO oral pellets for children ages 2 to less than 12. Chief Development Officer Dr. Bill Sheridan said the company is continuing marketing authorization applications in other major regions. Management described the unmet need as significant, noting that HAE and related attacks are underdiagnosed in younger children and that prophylaxis usage is about half that of adults.
Gayer said the pediatric launch is included in 2026 guidance but is expected to be a small near-term contributor, with the company taking a conservative stance on how quickly diagnosis rates and prophylaxis adoption could change. He added that BioCryst’s claims-data review found roughly 500 identified HAE patients under age 12, versus an estimated 1,200 based on epidemiology.
Navenibart: Phase 3 enrollment underway; Phase 2 data highlighted
BioCryst also focused on navenibart, describing it as a late-stage asset intended to complement—not replace—ORLADEYO by addressing patients who prefer high-efficacy injectables with infrequent dosing. Sheridan said the pivotal Phase 3 Alpha Orbit trial continues to recruit “very well,” with the company expecting to complete enrollment of the required 145 patients around mid-2026. The primary efficacy analysis will compare each dosing regimen versus placebo, based on the time-normalized rate of investigator-confirmed HAE attacks through six months.
At the same time, Sheridan previewed Phase 2 findings from the Alpha Solar trial being presented at the AAAAI meeting, describing results in 29 patients through up to 24 months of dosing with “no safety signals.” He cited mean reductions from baseline in attack rates of 92% for every-three-month dosing and 90% for every-six-month dosing, with the overall mean attack rate decreasing from 2.23 per month at baseline to 0.16 per month during treatment. For both regimens, he said the median attack-rate reduction was 97%.
In the Q&A, management addressed regulatory timing. Sheridan said HAE prophylactic therapies require 12 months of safety data, which he said should be delivered in mid-2027, driving the timing of the BLA submission. Gayer added the company is “on track for filing by the end of next year” and “on track for approval by late 2028.”
On positioning, Gayer said BioCryst sees the primary opportunity for navenibart among roughly 5,000 U.S. patients doing well on injectable prophylaxis who may be attracted to dosing 2 to 4 times per year. He also said navenibart could serve patients who try ORLADEYO and determine it is not the right therapy, keeping treatment decisions “within the BioCryst portfolio.” Management said there is no reliable way to predict ORLADEYO “super responders” other than having patients try therapy.
Netherton syndrome program advances into patient cohorts
BioCryst provided an update on BCX17725, its KLK5 inhibitor Fc-fusion protein in development for Netherton syndrome. Sheridan said the Phase 1 healthy-volunteer single- and multiple-ascending dose portions have been completed using subcutaneous and intravenous administration and were safe and well tolerated, with no discontinuations, no dose-related adverse findings, and no safety signals. He noted the estimated half-life of roughly 12–19 days supports every-two-week administration schedules.
The company said patient cohorts are open label, with a short-term cohort (Part 3, four weeks) and a longer-term cohort (Part 4, 12 weeks). BioCryst said it is prioritizing Part 4 recruitment and expects no more than three patients to enter Part 3, with plans to release the data together. Sheridan said BioCryst expects to recruit up to 12 patients in Part 4 and generate results by the end of the year, with primary efficacy based on change from baseline in the Ichthyosis Area and Severity Index (IASI) score and secondary endpoints including Investigator Global Assessment and Worst Itch Numerical Rating Scale.
Profitability, balance sheet, and 2026 guidance
Chief Financial Officer Anthony Doyle (introduced on the call as “Babar”) said 2025 delivered “record profitability” and a strengthened balance sheet. Discussing non-GAAP results adjusted for items including the European ORLADEYO business sale, stock-based compensation, workforce reduction costs, and transaction-related costs, he said non-GAAP total revenue increased 45% year-over-year. He highlighted non-GAAP ORLADEYO revenue growth of approximately $169 million, or 43%.
Doyle said non-GAAP operating profit rose to $214 million, up 198% year-over-year, which he described as the highest in company history. He said R&D declined slightly in 2025 but is expected to increase in 2026 as the company completes the Phase 3 trial and BLA-enabling CMC activities for navenibart. Non-GAAP sales and marketing expense was $144 million for the year, which he attributed to reallocation methodology, pediatric pre-launch costs, higher specialty distribution fees, and incentive compensation tied to strong growth.
BioCryst ended 2025 with $337.5 million in cash and investments. Doyle also said that concurrent with closing the Astria acquisition, BioCryst entered into a $400 million financing facility with Blackstone Life Sciences.
For 2026, BioCryst maintained guidance for:
- ORLADEYO revenue of $625 million to $645 million
- Non-GAAP OpEx of $450 million to $470 million, including Astria-related expenses
Management also discussed seasonality and reimbursement dynamics, noting that first-quarter revenue is typically pressured by reauthorization activity, higher free product, and higher commercial co-pay support, with revenue expected to be slightly down versus the fourth quarter before rebounding in the second quarter.
On pricing, Gayer said the company implemented a 9% price increase in January and expects to net about half of that, characterizing it as a catch-up move rather than a long-term reliance on pricing to reach its longer-term revenue targets.
About BioCryst Pharmaceuticals (NASDAQ:BCRX)
BioCryst Pharmaceuticals, Inc is a clinical?stage biotechnology company headquartered in Durham, North Carolina, that focuses on the discovery and development of novel, oral small?molecule medicines for rare and serious diseases. Since its founding in 1986, the company has leveraged structure?based drug design to advance a pipeline of targeted therapeutics designed to address underlying disease mechanisms rather than just treat symptoms.
The company’s first commercial product, Orladeyo (berotralstat), is an oral kallikrein inhibitor approved for the prophylactic treatment of hereditary angioedema (HAE) in both the United States and Europe.
