Core Insights - BioCryst Pharmaceuticals will present new findings on the burden of current treatments for hereditary angioedema (HAE) on pediatric patients under 12 and their caregivers at the 2025 US HAEA National Summit [1][2] - The company emphasizes its commitment to the HAE patient community and its efforts to develop effective treatments, including the first oral, once-daily prophylactic therapy for HAE [2] Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases [12] - The company has commercialized ORLADEYO (berotralstat), the first oral plasma kallikrein inhibitor for HAE, and is advancing a pipeline of small-molecule and protein therapies [12] Product Information - ORLADEYO is indicated for the prophylaxis of HAE attacks in adults and pediatric patients aged 12 years and older [5] - The product works by decreasing the activity of plasma kallikrein, preventing HAE attacks with a once-daily capsule [4] Research Presentation - BioCryst will present three abstracts at the summit, focusing on patient perspectives, treatment burdens for pediatric patients, and interim analysis results of prophylactic berotralstat in pediatric patients aged 2 to under 12 years [3][10]

Core Viewpoint - BioCryst Pharmaceuticals has entered into a definitive agreement to sell its European ORLADEYO business to Neopharmed Gentili for up to $264 million, which includes $250 million upfront and up to $14 million in future milestones [1][8] Financial Impact - BioCryst plans to use the proceeds from the transaction to retire all remaining term debt of $249 million, eliminating approximately $70 million in future interest payments [2][8] - The company expects to end 2027 with approximately $700 million in cash and no term debt, an increase of $400 million from prior net cash guidance [2][8] Strategic Rationale - The transaction aligns with BioCryst's strategy to make ORLADEYO available to patients in Europe while providing capital to retire debt, resulting in a higher margin business [3] - Neopharmed Gentili aims to leverage this acquisition to enhance its position in the rare disease field and strengthen its growth trajectory as a leading European specialty pharmaceutical company [4] Operational Efficiency - The acquisition will enable commercial continuity for ORLADEYO and is expected to result in at least $50 million in annual operating expense savings for BioCryst [8] Advisory and Legal Support - BofA Securities, Inc. and TD Cowen are serving as financial advisors to BioCryst, while Centerview Partners UK LLP is acting as exclusive financial advisor to Neopharmed Gentili [6]

Core Viewpoint - BioCryst Pharmaceuticals has received approval from INVIMA in Colombia for ORLADEYO (berotralstat) as a prophylactic treatment for hereditary angioedema (HAE) in patients aged 12 and older, marking a significant advancement in treatment options for HAE in the pan-Latin America region [1][2]. Group 1: Product Information - ORLADEYO is the first and only oral therapy specifically designed to prevent HAE attacks in adults and pediatric patients aged 12 years and older, administered as one capsule daily [4]. - The drug functions by inhibiting plasma kallikrein, which is involved in the pathophysiology of HAE [4]. - ORLADEYO has already received regulatory approvals in several Latin American countries, including Chile, Argentina, Brazil, Mexico, and Peru [3]. Group 2: Commercial Strategy - BioCryst has an exclusive collaboration with Pint Pharma GmbH for the registration and promotion of ORLADEYO in the pan-Latin America region, with Pint responsible for obtaining marketing authorizations and commercialization [3]. - Following the approval from INVIMA, BioCryst is working with Pint Pharma to ensure the availability of ORLADEYO to patients in Colombia [2]. Group 3: Safety and Usage Information - ORLADEYO is indicated for the prophylaxis of HAE attacks but is not approved for the treatment of acute HAE attacks [5][6]. - The recommended dosage is 150 mg once daily, with a reduced dosage of 110 mg for patients with moderate or severe hepatic impairment [7]. - Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease, occurring in 10% or more of patients [7].

Core Insights - BioCryst Pharmaceuticals has secured reimbursement approval for ORLADEYO (berotralstat) in the Netherlands, enabling routine prevention of hereditary angioedema (HAE) attacks for patients aged 12 and older [1][2] - This approval signifies national reimbursement for ORLADEYO across all major European countries, enhancing access to the first oral, once-daily preventive therapy for HAE patients [1][3] Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases, utilizing expertise in structure-guided drug design [11] - The company has commercialized ORLADEYO, the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies [11] Product Information - ORLADEYO is indicated for the prophylaxis of hereditary angioedema attacks in adults and pediatric patients aged 12 years and older, with one capsule taken daily to reduce plasma kallikrein activity [4][5] - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it is not recommended for such use [6][10]

Core Insights - New data presented at the 14th C1-inhibitor Deficiency & Angioedema Workshop supports the use of berotralstat (ORLADEYO) for hereditary angioedema (HAE) across all age groups, demonstrating significant efficacy and safety [1][2] Group 1: Efficacy and Safety of Berotralstat - The APeX-P trial showed a reduction in the percentage of days with angioedema symptoms among young children, with a drop from a mean of 11% to 4% over 12 weeks, sustained up to 48 weeks [4] - In the Italian Expanded Access Program, median Angioedema Control Test scores improved from 11.5 to 16.0 over six months, indicating better disease control [6] - The Berolife study reported a decrease in the median monthly attack rate from 2.25 at baseline to 0.55 after six months of treatment in adolescents [10][11] Group 2: Patient Demographics and Treatment Cohorts - The APeX-P study involved children aged 2 to 11 years, with a median age of 8 years and a mean of 18 missed school days annually due to HAE prior to treatment [4] - The Italian Expanded Access Program included 22 patients receiving berotralstat free of charge, with 15 transitioning to commercially available ORLADEYO upon program closure [5][7] Group 3: Treatment Administration and Mechanism - ORLADEYO is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, administered as one capsule daily [12] - The treatment works by decreasing the activity of plasma kallikrein, which is involved in the pathophysiology of HAE [12]

Core Insights - BioCryst Pharmaceuticals announced significant real-world evidence demonstrating the effectiveness of ORLADEYO (berotralstat) in reducing hereditary angioedema (HAE) attack rates in adolescents and individuals with severe HAE over an 18-month follow-up period [1][3][7] Group 1: Study Findings - The study presented at the ISPOR 2025 conference showed that patients experienced significantly lower HAE attack rates while on ORLADEYO, with a reduction from a baseline of 7.78-8.23 attacks/month to 1.24-1.90 attacks/month [10] - Specifically, patients had 6.25 fewer attacks/month at 12 months and 6.43 fewer attacks/month at 18 months after starting ORLADEYO [10] - Adolescents aged 12-17 years also showed significant reductions, with 1.56 fewer attacks/month at 12 months and 1.85 fewer attacks/month at 18 months [10] Group 2: Patient Population - The findings were based on a retrospective pre-post study involving 56 U.S. patients with C1-inhibitor deficiency and 99 U.S. adolescents with HAE [5][6] - The results indicate that ORLADEYO is effective across different age groups and severity levels of HAE, providing confidence to both physicians and patients [3][7] Group 3: Product Information - ORLADEYO is the first and only oral therapy specifically designed for the prophylaxis of HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [8][9] - The therapy is administered as a once-daily capsule, which works by decreasing the activity of plasma kallikrein to prevent HAE attacks [8]

Core Insights - BioCryst Pharmaceuticals has appointed Steve Frank, a leader in healthcare investment banking, to its board of directors, enhancing its strategic capabilities in the biopharma sector [1][3]. Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on developing innovative therapies for rare diseases, leveraging expertise in structure-guided drug design [6]. - The company has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies [6]. Leadership and Expertise - Steve Frank has extensive experience in healthcare investment banking, previously serving as chairman of global healthcare investment banking at J.P. Morgan and leading Bear Stearns' healthcare investment banking before its acquisition [2]. - His background includes directing State Farm's multi-billion-dollar life science equity portfolio in the 1980s and 1990s, showcasing his investment acumen [2]. Strategic Positioning - BioCryst is positioned for growth with a commercial product nearing $1 billion in peak sales and a strong cash position that allows independence from capital markets [4]. - The company is focused on accelerating towards profitability while advancing its pipeline of potential best-in-class medicines [4].