BioCryst Pharmaceuticals Conference: Profitability Pivot, ORLADEYO Growth, Navenibart Phase 3 Timeline

BioCryst Pharmaceuticals (NASDAQ:BCRX) executives highlighted the company’s transition to profitability, continued growth of its hereditary angioedema (HAE) franchise, and progress on newly acquired and earlier-stage pipeline programs during a recent investor discussion hosted by a biotech team.

Recent financial performance and “new BioCryst” profile

CEO Charlie Gayer said the company generated $601 million in revenue last year from ORLADEYO, or $563 million excluding the impact of the company’s sale of its European business in October. Gayer also cited $214 million in non-GAAP operating profits, characterizing BioCryst as “a profitable company and growing.”

Management also pointed to the completed acquisition of Astria Therapeutics, which added the late-stage HAE asset navenibart to BioCryst’s portfolio.

ORLADEYO: “Super-responders,” persistence, and market dynamics

Gayer discussed what the company calls ORLADEYO “super-responders,” emphasizing that while the terminology is newer, the concept has been communicated to physicians for years. He referenced two-year clinical trial data showing that just over 50% of patients who started ORLADEYO made it to two years, and that cohort had a 91% reduction in attacks versus baseline. In real-world use, he said about 60% of patients who try ORLADEYO remain on therapy at one year, and “most of them stay on after that.”

In discussing competitive dynamics, Gayer said ORLADEYO remains differentiated as the only oral prophylactic option currently on the market. He said that since launch, close to half of patients starting ORLADEYO have switched from injectable prophylaxis, while the other half are new to prophylaxis, including newly diagnosed patients and those previously using acute-only treatment.

He also described continued expansion in the ORLADEYO prescriber base, attributing part of that growth to data analytics used to identify new potential prescribers. BioCryst ended last year with more than 1,500 ORLADEYO prescribers, and Gayer said that figure is still increasing, driven largely by community allergists rather than academic centers.

Pediatric launch: pellets, pricing, and early expectations

Management discussed the pediatric opportunity following approval in December for use in younger patients. Gayer said BioCryst believes pediatric HAE is underdiagnosed and undertreated, estimating that while roughly 500 kids are diagnosed today, epidemiology suggests 1,100–1,200 should be diagnosed. He added that among diagnosed pediatric patients, only about 40% are treated with prophylaxis.

He outlined prior pediatric prophylaxis options as C1 inhibitor therapies down to age six and Takhzyro (lanadelumab) for ages two and up. BioCryst has now introduced ORLADEYO pellets for kids age two and up, which Gayer said was formally launched at the AAAAI meeting. He said the company expects product to be in its pharmacy “by about the end of this month, early April,” and that BioCryst is already taking prescriptions.

On adoption, management said its 2026 forecasting is “relatively conservative,” citing uncertainty about how quickly parents will bring children in and how quickly awareness builds, while still describing pediatrics as a meaningful long-term value driver. Gayer added the pediatric prescriber base is largely the same as the adult HAE prescriber base and that BioCryst does not expect to add field personnel for the launch.

He also said the pellets will be treated as ORLADEYO under existing contracts, and that BioCryst plans to launch at flat pricing relative to ORLADEYO capsules.

Guidance context, mix shift, and payer “paid rate” tailwinds

Gayer addressed questions about the company’s 2026 outlook, noting that year-over-year comparisons should account for the October sale of the European business, which he described as roughly $50 million of annualized revenue. He said the midpoint of BioCryst’s guidance range ($625 million to $645 million) represents about 13% growth versus the $563 million figure excluding Europe.

He also cited a prior-year payer tailwind: as provisions of the Inflation Reduction Act took effect, BioCryst saw a sharp improvement in Medicare affordability. According to Gayer, BioCryst’s “paid rate” in Medicare—representing about 20% of patients—improved from about 50% to close to 90% “kind of overnight.” He said the company does not expect that same tailwind this year, shifting the focus to steady patient growth and incremental paid-rate improvement.

On geographic mix, management said Europe had contributed about 10%–12% of revenue in recent quarters before the divestiture. Following the sale, Gayer said the U.S. is now “way over 90%” of revenue, with continued growth in Canada, Japan, and distributor markets.

Looking longer term, the company linked paid-rate improvements to revenue potential. Gayer said BioCryst ended last year with just over 1,600 patients on therapy and expects about 2,200 by the end of 2029. He stated that in 2029, each 1% of patients would be worth about $12 million in annualized revenue, and that improving the paid rate from 81% to 85% would equate to roughly $50 million in annualized revenue.

Navenibart and pipeline updates: phase 3 path and Netherton program

BioCryst executives described navenibart as intended to complement—rather than replace—ORLADEYO. Gayer said ORLADEYO’s durable oral prophylaxis segment is expected to continue growing, while navenibart targets a different segment: the “5,000 plus” patients doing well on injectable prophylaxis today, generally dosed every two or four weeks. He said navenibart could offer similar efficacy with two or four injections per year via an autoinjector and cited “zero incidence of injection site pain” as a differentiator versus the market leader.

Gayer referenced data presented at AAAAI from the phase 1b/2 program with patients followed to roughly 12 months on average. He said the three-month dosing arm showed a 92% reduction versus baseline and the six-month dosing arm showed a 90% reduction. He said BioCryst believes the profile offers flexibility, with comparable efficacy across three- and six-month regimens.

On phase 3 timing, Gayer said the key near-term milestone is enrolling the last patient in the pivotal study, expected “around the middle of this year.” He outlined a timeline of six months from that point to the six-month endpoint and another six months to the 12-month endpoint, noting that 12-month data are important for FDA safety expectations and for the six-month dosing arm. He said this timing would position BioCryst to file with the FDA by the end of next year, while the company has not decided what it will announce and when during 2027.

Beyond HAE, management provided an update on its KLK5 inhibitor program for Netherton syndrome. Gayer said the phase 1 study began in healthy volunteers before moving into Netherton patients. From the single-ascending-dose and multiple-ascending-dose portions in healthy volunteers, he highlighted two findings: the drug appeared safe “as best as you can measure” in that setting, and BioCryst demonstrated the drug reaches the epidermis, which he said was particularly encouraging given KLK5’s activity in the epidermis.

He said part 4 of the study will dose up to 12 patients every two weeks for three months, and that BioCryst has “high confidence” it will have that information by the end of the year, citing what the company is hearing from investigators and enrolling sites, including sites expected to open in Europe.

On market sizing for Netherton, Gayer said BioCryst’s work is complicated by the lack of an ICD-10 code. He referenced a claims analysis and additional work using LLM models, stating the company believes it has identified 3,000-plus patients and that patients may be underdiagnosed due to the lack of available treatment.

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.