BioCryst Pharmaceuticals NASDAQ: BCRX reported what executives described as a strong start to 2026, driven by continued growth of hereditary angioedema (HAE) therapy ORLADEYO and progress across its development pipeline, while also flagging a manufacturing issue that will delay initial fulfillment of its newly launched pediatric formulation.
ORLADEYO sales, demand trends, and pediatric launch update
President and CEO Charlie Gayer said ORLADEYO net revenue was $148.3 million in the first quarter, which he said was “right in line with our expectations.” Chief Financial Officer Babar Ghias added that ORLADEYO revenue increased 21% year-over-year, excluding the European divestiture impact, and that the company continues to see growth in prescribers “even in the sixth year of launch.”
Gayer said monthly new patient prescriptions tracked “slightly ahead of 2025 averages,” and emphasized that demand from patients ages 12 and older has remained consistent “despite new competition.” Addressing competitive dynamics further in Q&A, Gayer said recent injectable launches have competed mainly with the “existing market leader, TAKHZYRO,” and “it’s not affecting ORLADEYO.” He also said retention has remained consistent with prior trends, noting that “60% of them get to a year and then very slow, or very sticky after that.”
On ORLADEYO’s pediatric indication launch, Gayer said early signals support “the need for oral prophylaxis for kids with HAE,” and that BioCryst has received prescriptions across all four product strengths of ORLADEYO pellets. However, he disclosed the company “recently discovered a manufacturing issue that will delay the first product fulfillment.” He characterized the issue as “not an FDA issue at all” and “not a safety issue,” but rather “a batch problem in the specifications.” Gayer said the pellets are made at a different plant and by a different manufacturer than the capsule product.
Management said it expects to provide more information on product readiness later in the quarter and does not expect the delay to affect 2026 revenue guidance. In response to analyst questions about reimbursement workstreams during the delay, Gayer said BioCryst can proactively continue prior authorization and reimbursement activities, and noted that the pellets “slot right into our existing contracts for ORLADEYO capsules.”
Pipeline: navenibart enrollment ahead of plan; Netherton syndrome program dosing begins
BioCryst highlighted progress for navenibart, its injectable plasma kallikrein inhibitor for HAE prophylaxis. Gayer said enrollment in the pivotal phase III ALPHA-ORBIT trial “has exceeded our expectations” and is expected to be completed by the end of the following month. He said that at about 145 enrolled patients, ALPHA-ORBIT “will be the largest pivotal trial ever for HAE prophylaxis.”
Chief R&D Officer Sandeep Menon, who joined the company the prior month, said completion of enrollment by the end of June keeps BioCryst “on track to submit a regulatory filing in the U.S. by the end of next year.” He cited data from the long-term, open-label ALPHA-SOLAR study, where navenibart “recently demonstrated 92% and 90% mean attack reductions in the three-month and the six-month dosing regimens respectively.”
In Q&A, Gayer attributed rapid enrollment to patient and physician appetite for less frequent dosing, calling three- and six-month administration “meaningful and transformative.” Discussing expectations for phase III outcomes, he said the precise percent reduction matters less than the resulting attack rate, pointing again to open-label data where mean attack rates were “0.16 per month,” which he described as “functionally attack free for patients.”
BioCryst also discussed BCX17725, a KLK5 inhibitor being studied for Netherton syndrome. Menon said patients have started dosing in Part 4 of the phase I study, which will enroll up to 12 patients with three months of dosing, and that BioCryst expects to report data by year-end. Gayer said the company has “a high level of confidence that the U.S. market is 3,000+ patients,” while noting the importance of correct diagnosis and identification and that having an approved therapy can be a catalyst for finding patients in rare diseases.
Astria integration, portfolio decisions, and European licensing agreement
Ghias said BioCryst has been “very pleased with the overall integration of Astria Therapeutics,” adding the process has been running ahead of expectations. He also explained that the acquisition was classified as an asset acquisition for accounting purposes, leading to an in-process R&D charge of $698 million in Q1, along with other transaction-specific charges.
Ghias said BioCryst continues to evaluate internal programs that do not make viable business sense and disclosed that the company decided in the first quarter to discontinue development of avoralstat in DME.
Separately, Ghias said BioCryst finalized a license agreement with Neopharmed Gentili to commercialize navenibart in Europe. BioCryst expects to receive $70 million in cash upfront and up to $275 million in future regulatory and sales milestones, plus tiered royalties on net sales “ranging from 18%–30% overall.” In Q&A, management declined to break out milestone amounts tied to regulatory versus commercial thresholds.
Financial results, liquidity, and 2026 guidance
Ghias said BioCryst posted a first-quarter non-GAAP operating profit of $54 million, up 25% year-over-year, and noted non-GAAP total revenue increased about 17% year-over-year. He said sales and marketing expense was $37 million on a non-GAAP basis, “down slightly” from the prior-year period, while non-GAAP G&A expense increased by $1.8 million, primarily due to incremental overhead tied to the Astria closing. He also said R&D costs rose versus the prior year as the company consolidated costs related to navenibart, and reiterated expectations for higher R&D spending in 2026 as BioCryst completes phase III and BLA-enabling CMC work.
BioCryst ended the quarter with approximately $261 million in cash and investments, Ghias said, despite funding part of the Astria purchase price from the balance sheet. He added the company also closed a $400 million senior credit facility to fund the remaining cash portion of the Astria acquisition and issued about 37 million shares to Astria shareholders.
Including net proceeds from the European navenibart license agreement, Ghias said pro forma liquidity stood at $331 million as of March 31, 2026.
Management maintained 2026 guidance, including:
- ORLADEYO revenue: $625 million to $645 million
- Non-GAAP operating expenses: $450 million to $470 million
Gayer closed the call by saying BioCryst is executing well across commercial and development programs and that he believes “the best is yet to come” for the company.
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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