BioCryst Pharmaceuticals (NASDAQ:BCRX) outlined a multi-pronged growth strategy at the JPMorgan Healthcare Conference, highlighting continued momentum for its hereditary angioedema (HAE) franchise, a pending acquisition of Astria Therapeutics, and progress on an early-stage program for Netherton syndrome.
2025 described as a “transformative year”
CEO Charlie Gayer said 2025 marked a turning point for the company, pointing to several milestones including growth in ORLADEYO, the sale of BioCryst’s European business, first-time profitability, and a new pediatric approval for ORLADEYO.
BioCryst ended the year with nearly $340 million in cash, Gayer said. He added that the company achieved profitability for the first time in its history and expects to remain profitable going forward.
2026 outlook and expense framework
Management provided 2026 expectations, including ORLADEYO revenue of $625 million to $645 million. Including RAPIVAB, total revenue is expected to be $635 million to $660 million.
On expenses, the company guided to non-GAAP base operating expenses of $380 million to $390 million prior to the Astria acquisition. Management said it expects to add $70 million to $80 million in operating expenses after closing the Astria deal to fund ongoing operations and completion of the navenibart clinical trial.
In Q&A, CFO Babar Ghias said the base business expense structure is “steady state,” noting that sales and marketing increases are primarily tied to variable costs such as specialty pharmacy fees as volumes rise. He also said the company expects R&D to remain relatively stable, describing rare disease development as not requiring “several hundred million dollars” of annual R&D spend.
ORLADEYO growth thesis and competitive dynamics
Gayer said ORLADEYO continues to grow in its sixth year on the market, which he characterized as unusual for a rare disease launch. He cited 2025 as the year with the most new U.S. patient prescriptions since the first year of launch and said BioCryst had approximately 1,600 patients on therapy in the U.S. despite increased competition in HAE.
He also discussed U.S. reimbursement trends, saying BioCryst ended the year with an ORLADEYO paid rate of 81%, compared with a long-term goal of 85%. He said the paid rate was 84% at the end of the first quarter and improved during the year.
On the broader market, Gayer noted that HAE has become crowded, with “10 or 11 products approved” and recent approvals including an oral acute therapy and two injectable prophylaxis therapies. However, he said ORLADEYO remains differentiated as the only oral prophylaxis product and that new injectable options moving from every two weeks to once a month are “a nice to have” but have not changed ORLADEYO’s trajectory.
Gayer reiterated a long-term target for ORLADEYO to reach $1 billion in peak sales in 2029, supported by a projected mid-teens revenue CAGR through that period. He said the company sees a path to that goal primarily through U.S. growth, improvements in paid rates, modest price increases, and contributions from Japan, Canada, and other regions. He also highlighted ORLADEYO’s patent runway, citing composition-of-matter protection through May 2040.
In response to questions about achieving $1 billion in peak sales after divesting European rights, Gayer said BioCryst’s updated outlook relies on the pace of U.S. growth plus the newly approved pediatric population down to age two. He described the pediatric opportunity as underdiagnosed and undertreated, adding that ORLADEYO offers capsules for patients 12 and up and oral pellets for younger children in a market where pediatric prophylaxis alternatives are largely injectable.
Astria acquisition and the navenibart opportunity
Gayer said BioCryst sold its European business to Neopharmed Gentili for $250 million in upfront cash, describing Europe as not profitable for BioCryst as a single-product operation. He said the divestiture helped streamline the company and enabled a pivot to business development, including the pending acquisition of Astria Therapeutics.
Management said BioCryst is acquiring Astria for $700 million in total enterprise value and expects to close “in the next couple of weeks,” pending an Astria shareholder vote. The deal would add navenibart, a late-stage HAE prophylaxis candidate that Gayer described as potentially dosed every three to six months.
Gayer highlighted Phase Ib proof-of-concept data showing a “very competitive efficacy profile” at both the three- and six-month dosing regimens, and said the injections would be relatively small (2 mL for every-three-month dosing, with every-six-month dosing double that in auto-injectors). He also cited Phase Ib observations of no injection site pain, contrasting that with patient experiences seen with the current market leader, lanadelumab.
Management framed ORLADEYO and navenibart as complementary, with ORLADEYO positioned for patients seeking oral prophylaxis and navenibart aimed largely at switching patients currently on injectable prophylaxis. Gayer said there are about 5,000 U.S. patients on injectable prophylaxis, and that the overall prophylaxis population is growing 3% to 5% annually.
Pipeline focus: BCX17725 for Netherton syndrome; avoralstat partnering plans
Beyond HAE, BioCryst highlighted BCX17725 for Netherton syndrome as a key internal R&D priority. Gayer described Netherton as a severe genetic disorder characterized by uncontrolled skin turnover, infections, and atopic conditions, and said there are no approved therapies.
Management discussed efforts to size the market, noting there is currently no ICD-10 code for the disease. Gayer said earlier work using claims data (including mentions of “bamboo hair,” a feature associated with the condition) suggested about 1,600 U.S. patients, while newer work applying natural language processing to electronic medical record data suggests the U.S. population could be 3,000+.
On development, management said BCX17725 has completed healthy volunteer work, which showed multi-dose safety signals and evidence the drug reaches the epidermis. The company is moving into patient dosing, including an open-label component with up to a dozen patients dosed every two weeks for three months. Chief Development Officer Bill Sheridan said the program will focus on clinical outcomes such as pain, itch, redness, and overall well-being, since there is no plasma biomarker comparable to HAE. Management said the goal is to generate enough evidence of a “big treatment effect” to support regulatory discussions and potentially move directly into a pivotal study.
BioCryst also addressed avoralstat, which is in a Phase I proof-of-concept study in diabetic macular edema. Gayer said the program is not core to BioCryst’s rare-disease strategy and that the company plans to finish the Phase I study and seek a partner to out-license it. He said early cohort work includes three planned dose levels, with the first cohort completed at 100 micrograms and additional cohorts at 250 and 500 micrograms. BioCryst recently opened the IND in the U.S. after conducting work in Australia, and Gayer said additional data is expected around the middle of the year, with disclosure more likely alongside a partnering announcement.
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.
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