BioCryst Pharmaceuticals (NASDAQ:BCRX) reported second-quarter results marked by growth in ORLADEYO revenue, the start of shipments for its pediatric oral-pellet formulation, and completion of enrollment in its pivotal navenibart trial for hereditary angioedema, or HAE.
President and CEO Charlie Gayer said the company completed the integration of Astria Therapeutics during the quarter and increased cash generation through revenue growth and spending discipline. BioCryst also decided in June to discontinue its internal drug-discovery programs and close its Birmingham research facility by the end of 2026, shifting its research strategy toward externally sourced innovation.
ORLADEYO Revenue and Pediatric Launch
Ghias said total revenue also included $55.7 million recognized from the upfront payment under the company’s European navenibart licensing agreement with Neopharmed Gentili.
BioCryst began shipping ORLADEYO oral pellets for pediatric patients during the week of the call. Gayer said the company had received 47 prescriptions through July 31, despite a manufacturing delay, a figure that exceeded its previous expectation for total pediatric prescriptions during 2026. Prior authorizations had been completed for about half of those patients, with what Gayer described as a strong early approval rate.
The company said the initial pediatric prescription count represented close to 10% of an estimated 500 diagnosed children in the applicable age range. Gayer added that physicians have expressed enthusiasm for prescribing the oral formulation and that the availability of an oral prophylactic option could expand use among pediatric HAE patients. He said BioCryst believes there may be as many as 1,200 diagnosed children in the age population, though the company expects it will take several quarters to assess market development.
For patients age 12 and older using ORLADEYO capsules, new prescription demand during the quarter remained consistent with historical trends. Gayer said BioCryst continued to add slightly more than its historical average of about 60 new prescribing physicians per month.
BioCryst’s paid rate for ORLADEYO reached 84% at quarter-end, compared with 83% a year earlier. The figure applied only to the capsule population because pediatric pellets were not yet available during the second quarter. Gayer said the company typically sees the rate decline by one or two percentage points in the second half as new patients enter treatment, before recovering during the following year’s reauthorization season.
Management said approximately half of ORLADEYO’s year-over-year revenue growth reflected volume, while the other half reflected price and reimbursement. The company had previously discussed a 9% price increase, of which it said it nets about 4.5%.
Specialty Pharmacy Transition and Competition
BioCryst selected CareMed as its sole-source specialty pharmacy for ORLADEYO shipments. The pediatric pellet prescriptions are already shipping through CareMed, while patients 12 and older are expected to transition during August.
Gayer said the company selected CareMed because of its ability to scale as BioCryst’s HAE portfolio and patient population grow. He acknowledged that a pharmacy transition can involve operational challenges but said the company does not expect the change to affect ORLADEYO’s long-term performance.
On competition in the HAE prophylaxis market, Gayer said newer injectable products have primarily affected use of existing injectable therapies, particularly TAKHZYRO. He said ORLADEYO retention has remained in line with historical patterns, with patients who respond well to the treatment generally remaining on therapy.
Pipeline Updates
Chief Research and Development Officer Dr. Sandeep Menon said BioCryst completed enrollment in June for ALPHA-ORBIT, its pivotal trial of navenibart, an investigational long-acting injectable plasma kallikrein inhibitor for HAE prophylaxis. The company expects to report top-line data in the third quarter of 2027.
The study will remain blinded to collect one year of safety and efficacy data, as agreed with the Food and Drug Administration. Menon said this design will allow BioCryst to evaluate efficacy at both six and 12 months, reflecting navenibart’s potential every-six-month dosing regimen.
Management said the trial was expanded because of patient and investigator interest, rather than concerns about statistical power or patient discontinuations. Menon said the study is already overpowered.
BioCryst also said its proof-of-concept study of BCX17725 in Netherton syndrome is enrolling well and remains on track to produce early patient data from Part 4 of the Phase 1b study by the end of 2026. Gayer said the company expects enrollment to reach up to 12 patients by year-end.
Menon described BCX17725 as a potential first systemic, targeted treatment for Netherton syndrome, a severe skin condition with no approved therapies. The drug targets KLK5, which BioCryst identified as implicated in the disease. He noted that Netherton syndrome can be variable and involve waxing and waning symptoms, and said the company will need the full data set to evaluate pharmacokinetics, pharmacodynamics and clinical efficacy.
Guidance and Financial Position
BioCryst reported non-GAAP operating profit of $113.2 million for the quarter and ended the period with more than $350 million in cash, cash equivalents and investments. Ghias said the company generated positive cash flow even excluding the upfront proceeds from the Neopharmed Gentili agreement.
The company reduced its 2026 non-GAAP operating-cost guidance to $420 million to $440 million, from a previous range of $454 million to $470 million. Ghias attributed the lower outlook to the planned shutdown of internal discovery operations and the Birmingham facility closure.
- 2026 total revenue guidance: Raised to $690 million to $715 million.
- 2026 ORLADEYO revenue guidance: Maintained at $625 million to $645 million.
- 2026 non-GAAP operating-cost guidance: Maintained at $420 million to $440 million.
Ghias said BioCryst intends to pursue external business-development opportunities focused on high-value, clinically validated assets, particularly early- to mid-stage programs. He said the company does not intend to pursue large acquisitions that would unnecessarily strain its balance sheet, while maintaining a commitment to profitable growth and positive cash flow.
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.
