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]

Sebetralstat Overview - Sebetralstat, if approved, is poised to be the first oral on-demand treatment for Hereditary Angioedema (HAE), potentially transforming the global treatment landscape[20, 22] - Clinical data shows the safety profile of Sebetralstat is no different from placebo, with robust efficacy demonstrated across all attack types, severities, and locations[18, 129] - The median time to beginning of symptom relief was 161 hours for 300 mg and 179 hours for 600 mg of sebetralstat, compared to 672 hours for placebo in Phase III trials[67] - In an open-label extension study, the median time to treatment was 10 minutes overall, 20 minutes for abdominal attacks, and 115 minutes for laryngeal attacks[72] Market Opportunity - The current global HAE market is valued at $13 billion, with a $3 billion global on-demand opportunity[23] - The on-demand market is projected to increase by 70%, driving the global sales to over $4 billion by 2030[89] - In the U S, approximately 84,000 on-demand doses are utilized, with 70% of attacks treated with Firazyr or icatibant[98, 99] Commercialization Strategy - KalVista plans to launch Sebetralstat in the US by June 2025, followed by Germany in 2H 2025, and the UK and Japan in 1H 2026[20, 123] - The company anticipates broad payor access, primarily through commercial coverage, with pricing comparable to existing branded therapies, ranging from $11,000 to $16,000 per attack[17, 110] - KalVista is targeting the top 1,000 HCPs and is focusing on patients currently using Firazyr and icatibant to drive early demand and accelerate adoption[126]

Core Viewpoint - CSL has received FDA approval for ANDEMBRY®, a novel treatment for hereditary angioedema (HAE), marking a significant advancement in the management of this rare genetic disorder [1][3][11] Group 1: Product Overview - ANDEMBRY is the first monoclonal antibody developed entirely by CSL, targeting factor XIIa to prevent HAE attacks in patients aged 12 and older [1][3][11] - The treatment offers once-monthly subcutaneous self-injection, providing a convenient administration method [1][3] - ANDEMBRY has shown a median reduction of more than 99% in HAE attacks compared to placebo, with a least squares mean reduction of 89.2% [6][7] Group 2: Clinical Trial Data - The approval is based on data from the pivotal Phase 3 VANGUARD trial, which demonstrated the efficacy and safety of ANDEMBRY [3][12] - In the pivotal trial, 62% of patients treated with ANDEMBRY remained attack-free throughout the treatment period [7] - The most common adverse reactions reported were nasopharyngitis and abdominal pain, with injection-site reactions occurring in 14% of patients [4][7] Group 3: Market Impact and Availability - ANDEMBRY's approval expands CSL's HAE franchise and reinforces the company's commitment to innovation in the HAE community [6][8] - The product is set to launch commercially immediately, with availability expected before the end of June 2025 [9] - ANDEMBRY has also received approvals in multiple regions, including Australia, the UK, the EU, Japan, Switzerland, and the UAE [8]

Core Insights - BioCryst Pharmaceuticals announced new data on ORLADEYO (berotralstat) for hereditary angioedema (HAE) treatment to be presented at the EAACI meeting in Glasgow from June 13 to 16, 2025 [1][2][3] Group 1: Presentation Details - Four abstracts will be presented at the EAACI meeting, including a pooled analysis of the APeX-2 and APeX-J trials focusing on the impact of berotralstat on quality of life for HAE patients [1] - A focus group study reported positive perceptions from patients following berotralstat treatment [2] - Interim results from the APeX-P study indicated that berotralstat use reduced the number of HAE attacks requiring treatment in pediatric patients [3] Group 2: Product Information - ORLADEYO (berotralstat) is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [4][5] - The recommended dosage is one capsule per day, with caution advised for dosages higher than 150 mg due to potential QT prolongation [6][7] - The safety and effectiveness of ORLADEYO in pediatric patients under 12 years have not been established, and there is limited data on its use during pregnancy [9][10] 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, utilizing structure-guided drug design for developing therapeutics [11]

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]

Financial Data and Key Metrics Changes - The company reported having €5 million in cash as of September 24, which is expected to provide a cash runway into the third quarter of 2026, beyond the readout of the on-demand phase three study [43]. Business Line Data and Key Metrics Changes - The company is developing ducriptaban, a novel oral bradykinin B2 receptor antagonist, for both on-demand and prophylactic treatment of hereditary angioedema (HAE) [2][4]. - Phase two data showed that ducriptaban achieved an 85% reduction in attacks compared to placebo, with over 90% reduction in moderate to severe attacks requiring rescue medication [27][30]. Market Data and Key Metrics Changes - In the U.S. market, approximately 65% of HAE patients are currently on prophylaxis, with expectations that this could increase to 70-80% with the introduction of an oral option [32]. - The European market is slower in adopting prophylactic therapies, but countries like Germany and France are expected to catch up soon [10][11]. Company Strategy and Development Direction - The company aims to provide innovative oral therapies for both on-demand and prophylactic treatment, addressing the need for convenience and efficacy in HAE management [7][14]. - The strategy includes capturing a broader patient base globally, beyond just the U.S. and Western Europe, by offering oral therapies [11]. Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming oral therapies penetrating the market, highlighting the significant unmet need in the HAE community [13][14]. - The company anticipates growth in both prophylactic and on-demand markets, driven by the ease of administration and efficacy of their oral product [33]. Other Important Information - The company is also exploring opportunities in acquired angioedema, which is a different patient population but shares similar physiological responses to HAE [48][49]. - The company plans to release more data regarding efficacy, safety, and quality of life from ongoing studies in 2025 [45]. Q&A Session Summary Question: What is the opportunity in acquired angioedema? - The acquired angioedema population is estimated to be about 10% on top of type one and type two HAE, with a high unmet medical need and potential for commercial opportunity [48][49]. Question: How does the company plan to manage recruitment for the acquired angioedema study? - The company is optimistic about recruitment, noting a strong desire among patients to join clinical trials due to the lack of available treatments [58].