Core Insights - BioCryst Pharmaceuticals reported a transformative year in 2025, driven by strong demand for ORLADEYO and successful business maneuvers, including the sale of its European ORLADEYO business and the proposed acquisition of Astria Therapeutics [2] Financial Performance - Preliminary, unaudited ORLADEYO net revenue for full year 2025 was $601 million, reflecting a 37% year-over-year increase, surpassing prior guidance of $590 million to $600 million [4] - Excluding European ORLADEYO revenue, the net revenue for 2025 was $563 million, marking a 43% year-over-year growth on a comparable basis [4] - For the fourth quarter of 2025, ORLADEYO net revenue was $151 million, representing a 22% year-over-year increase, and a 36% increase on a comparable basis [3] Revenue Guidance - The company anticipates full year 2026 global net ORLADEYO revenue to be between $625 million and $645 million [5] - Total revenue for 2026, including RAPIVAB®, is expected to range from $635 million to $660 million [5] Operating Expenses - BioCryst expects full year 2026 non-GAAP operating expenses to be between $380 million and $390 million, excluding stock-based compensation and other costs [6] - After the expected acquisition of Astria, additional non-GAAP operating expenses in 2026 are projected to be between $70 million and $80 million [7] Cash Position - As of December 31, 2025, the company reported preliminary cash, cash equivalents, restricted cash, and investments totaling $338 million [5]

Core Viewpoint - BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) is highlighted as a promising small-cap stock with significant upside potential, particularly following the FDA approval of Orladeyo for pediatric patients with hereditary angioedema [1][2]. Group 1: FDA Approval and Product Details - On December 12, BioCryst Pharmaceuticals announced that the FDA approved its New Drug Application for an oral pellet formulation of ORLADEYO (berotralstat) for pediatric patients aged 2 to <12 years with hereditary angioedema [2]. - ORLADEYO is noted as the first and only targeted oral prophylactic therapy for this age group, which previously had limited treatment options that were burdensome [2]. - The company also reported that a capsule formulation of ORLADEYO was approved in December 2020 for patients aged 12 years and older, and it is now approved in over 45 countries [3]. Group 2: Investment Outlook - H.C. Wainwright raised the price target for BioCryst Pharmaceuticals from $30 to $32 and reaffirmed a Buy rating, citing the approval of Orladeyo as a significant factor for this update [1]. - The company is recognized for its innovative approach in biotechnology, focusing on structure-guided drug design to develop therapies for rare diseases [3].

–ORLADEYO now first and only targeted oral prophylactic therapy for patients with HAE aged 2 and older–-Oral pellet formulation provides child-friendly method of administration- –Showed early and sustained reductions in monthly attack rates in APeX-P– RESEARCH TRIANGLE PARK, N.C., Dec. 12, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for the use of an oral pellet formulation ...

Core Insights - BioCryst Pharmaceuticals is under review by the U.S. FDA for the oral granule formulation of ORLADEYO for pediatric patients aged 2 to <12 years, with a decision expected by December 12, 2025, which would make it the first targeted oral prophylactic therapy for this age group [1][2][4] Clinical Trial Data - The APeX-P clinical trial has shown early and sustained reductions in monthly attack rates for pediatric patients with hereditary angioedema (HAE) treated with ORLADEYO over one year [1][6] - At month 1, 65.5% of patients were attack-free, increasing to 70.4% by month 12, with a median attack rate of 0 for 11 out of 12 months [7] - The trial demonstrated high continuation rates, with 93.1% of patients completing at least 48 weeks of treatment [7] Psychosocial Impact - HAE attacks have a significant negative psychosocial impact on pediatric patients and their caregivers, affecting mental health and quality of life [4][11] - The majority of young patients experience symptoms before age 6, with a median age at diagnosis of 2 years [7][13] - Caregivers report that HAE attacks occur approximately every 3 months and are rated as moderately severe [13] Emergency Department Experiences - Over three-quarters of adolescents and nearly half of caregivers reported at least one emergency department (ED) or hospital visit before age 12, often characterized by treatment delays and heightened stress [23] - HCPs express concern about inadequate care and the long-term mental health impacts of these ED visits on young patients [23] Safety and Tolerability - ORLADEYO was found to be safe and well-tolerated, with no new safety signals identified during the trial [7][8] - The most commonly reported treatment-emergent adverse event was nasopharyngitis [7]

Core Insights - BioCryst Pharmaceuticals reported a strong Q3 2025 performance, with ORLADEYO net revenue reaching $159.1 million, a 37% increase year-over-year [1][6][8] - The company raised its FY 2025 revenue guidance for ORLADEYO to between $590 million and $600 million, while lowering non-GAAP operating expense guidance to between $430 million and $440 million [1][17] - BioCryst completed the sale of its European ORLADEYO business, using the proceeds to retire all remaining Pharmakon term debt [1][15] - A definitive agreement was signed to acquire Astria Therapeutics, expected to close in Q1 2026, which will enhance BioCryst's HAE portfolio [1][15] Financial Performance - Total revenues for Q3 2025 were $159.4 million, up 36% from $117.1 million in Q3 2024 [8] - Operating profit for Q3 2025 was $29.6 million, a significant increase of 285% year-over-year, with non-GAAP operating profit at $51.7 million, up 107% [1][11] - Net income for Q3 2025 was $12.9 million, or $0.06 per share, compared to a net loss of $14.0 million, or $0.07 per share, in Q3 2024 [13][27] Market Dynamics - ORLADEYO demand remained strong, with new patient prescriptions slightly exceeding those in Q3 2024 despite new competitors entering the market [4][6] - The U.S. market contributed 89% of global ORLADEYO net revenues in Q3 2025, indicating strong domestic performance [6] Strategic Moves - The sale of the European ORLADEYO business has strengthened the company's financial position and allowed for debt repayment [2][15] - The acquisition of Astria Therapeutics is anticipated to provide opportunities for sustainable growth and profitability in the HAE market [2][15] Research and Development - R&D expenses for Q3 2025 increased to $44.6 million, primarily due to advancements in clinical programs [9] - The company is progressing with clinical trials for BCX17725 and avoralstat, with initial data expected by early 2026 [15]

Core Insights - BioCryst Pharmaceuticals will present four abstracts at the ACAAI Annual Scientific Meeting, focusing on the APeX-P trial results for ORLADEYO in pediatric patients with hereditary angioedema [1][2] Group 1: Presentation Details - The abstracts will include a second interim analysis of the APeX-P trial evaluating long-term prophylaxis with ORLADEYO in children aged 2 to <12 years [1] - Additional findings will address the psychosocial impact of hereditary angioedema on young patients and their caregivers [1] Group 2: Product Information - ORLADEYO (berotralstat) is the first oral therapy specifically designed to prevent hereditary angioedema attacks in patients aged 12 years and older [4] - The drug functions as a plasma kallikrein inhibitor, with a recommended dosage of one capsule per day [4][5] Group 3: Safety and Efficacy - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it should not be used for this purpose [6][10] - Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease [7]

Core Viewpoint - BioCryst Pharmaceuticals will report its third quarter 2025 financial results on November 3, 2025, and will host a conference call to discuss these results and provide a corporate update [1]. Group 1: Financial Reporting - The third quarter 2025 financial results will be announced on November 3, 2025 [1]. - A conference call and webcast will take place at 8:30 a.m. ET on the same day to discuss the financial results [1]. Group 2: Access Information - Domestic callers can access the live call by dialing 1-844-481-2942, while international callers can dial 1-412-317-1866 [2]. - A live webcast and replay of the call will be available on the investors section of the company website [2]. Group 3: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases [3]. - The company specializes in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics [3]. - BioCryst has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of therapies [3].

Core Insights - BioCryst Pharmaceuticals has successfully completed the sale of its European ORLADEYO business to Neopharmed Gentili for $250 million upfront, with potential future milestones of up to $14 million based on sales in Central and Eastern Europe [1][2][3] - The divestiture is expected to improve BioCryst's operating margin as the European business was approximately breakeven on a direct basis [2] - Following the sale, BioCryst will concentrate on increasing ORLADEYO sales in the US market while Neopharmed Gentili will manage commercialization in Europe [2][3] - Despite the loss of European revenue, BioCryst is projected to meet the upper half of its 2025 revenue guidance range of $580 million to $600 million [3] Financial Impact - The transaction is valued at $250 million upfront, which will enhance BioCryst's financial position [1] - Future milestones could add an additional $14 million based on performance in specific European markets [1] Strategic Focus - BioCryst will shift its focus to the US market for ORLADEYO sales, indicating a strategic realignment post-divestiture [2][3] - Neopharmed Gentili will retain the European commercial organization established by BioCryst, ensuring operational continuity in Europe [2]

Core Insights - BioCryst Pharmaceuticals has completed the sale of its European ORLADEYO business to Neopharmed Gentili for $250 million, with potential future milestones of up to $14 million [1][8] - The transaction allows BioCryst to focus on its core U.S. market, significantly improving its operating margin and cash flow generation [2][8] - Neopharmed Gentili will manage the commercialization of ORLADEYO in Europe, retaining the existing European commercial organization built by BioCryst [8] Transaction Details - The sale price of $250 million reflects a multiple of approximately 5.4 times sales over the last twelve months ending June 2025 [8] - BioCryst intends to use the proceeds to retire a $199 million Pharmakon term loan, resulting in a cleaner balance sheet for future strategic activities [8] Strategic Advantages - The divestiture of the European business, which was approximately breakeven on a direct basis, provides an immediate improvement to BioCryst's operating margin [8] - The transaction enhances BioCryst's strategic optionality and positions the company to reach the upper half of its 2025 revenue guidance range of $580 million to $600 million, even without European revenue [2][8]

Core Insights - BioCryst Pharmaceuticals announced the departure of Dr. Helen Thackray, the chief research and development officer, effective September 1, 2025, transitioning to an advisory role until the end of the year [1][2] - Dr. Thackray joined BioCryst in 2019 as a board member and became the chief research and development officer in 2021, being a finalist for the CEO succession process [2] - The CEO of BioCryst, Jon Stonehouse, expressed gratitude for Dr. Thackray's contributions, including the launch of a new protein therapeutics platform and advancements in various clinical programs [3] Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases [5] - The company utilizes structure-guided drug design to develop innovative oral small-molecule and protein therapeutics targeting challenging diseases [5] - BioCryst has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of therapies [5]