BioCryst presented new pediatric data for ORLADEYO (berotralstat) at the American College of Allergy, Asthma & Immunology conference on November 6, 2025. The presentation covered the second interim analysis of the APeX‑P trial, which enrolled 29 children aged 2 to <12 years with hereditary angioedema (HAE). The study reported a mean exposure of 63.9 weeks and showed a rapid, sustained reduction in monthly attack rates, with 65.5 % of patients attack‑free at month 1 and 70.4 % at month 12. Ninety‑three percent of participants completed at least 48 weeks of therapy, and the safety profile was consistent with the adult program, with nasopharyngitis the most common treatment‑emergent adverse event.
The pediatric data reinforce the case for a new indication that could lead to FDA approval by the target action date of December 12, 2025. Approval would expand ORLADEYO’s addressable market to younger patients, potentially adding hundreds of new prescriptions and positioning the drug as the first oral prophylactic for HAE in children. The data also support the company’s broader strategy of offering both oral and injectable options, complementing the recently announced acquisition of Astria Therapeutics, which will add the injectable navenibart to BioCryst’s portfolio.
On November 3, 2025, BioCryst released its Q3 2025 earnings. Net revenue for ORLADEYO rose 37 % year‑over‑year to $159.1 million, driven by strong demand in the U.S. market and a 12‑month growth in prescription volume. Earnings per share of $0.16 beat consensus estimates of $0.05 by $0.11, a 220 % upside, largely due to disciplined cost management and the absence of one‑time charges. However, total revenue of $159.1 million fell short of the $162.97 million expected by analysts, a miss of $3.87 million or 2.4 %. The revenue shortfall was attributed to a modest decline in the European market, where the company had recently divested its European ORLADEYO business, and to a temporary slowdown in new patient onboarding during the quarter.
The Q3 results also highlighted a strategic shift. BioCryst used proceeds from the European divestiture to retire remaining Pharmakon term debt, improving its balance sheet and freeing cash for future growth. The company raised its full‑year 2025 revenue guidance to $590–$600 million from the prior $580–$590 million range, reflecting confidence in continued uptake of both oral and injectable HAE therapies. Management emphasized that the combination of a robust pediatric data set, a strengthened balance sheet, and the planned Astria acquisition positions BioCryst to capture a larger share of the HAE market and to accelerate its path to profitability.
The convergence of clinical, financial, and strategic milestones suggests that BioCryst is on a trajectory to solidify its leadership in the HAE space. The pediatric data provide a critical regulatory lever, the Q3 earnings demonstrate operational efficiency, and the acquisition of Astria expands the product portfolio. Together, these developments are expected to enhance the company’s long‑term revenue potential and to reinforce its competitive advantage over existing injectable therapies.
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