Core Insights - KalVista Pharmaceuticals reported strong momentum in the US launch of EKTERLY®, achieving $13.7 million in net product revenue for the third quarter of 2025, with 937 patient start forms received by October 31, 2025 [1][3][9] - The company has successfully launched EKTERLY in Germany, with initial orders indicating positive demand, and has received regulatory approval in Australia, expanding its global footprint [2][3] - As of September 30, 2025, KalVista has approximately $309 million in cash, expected to fund operations until profitability [1][9] Commercial Progress - The US commercial launch of EKTERLY began on July 7, 2025, with 423 unique prescribers activated [3] - EKTERLY is the first and only oral on-demand treatment for hereditary angioedema (HAE) approved in the US, EU, Switzerland, and Australia [6][9] - The European Commission and Swissmedic approved EKTERLY for treating acute HAE attacks in adults and adolescents aged 12 and older [3] Financial Performance - For the three months ended September 30, 2025, KalVista reported a net product revenue of $13.7 million, with a cost of revenue of $1.2 million [9][12] - Research and development expenses decreased to $12 million from $18.7 million in the same period of 2024, primarily due to reduced clinical trial costs [9][12] - Selling, general and administrative expenses increased to $46.5 million from $24.8 million, attributed to commercialization efforts for EKTERLY [9][12] Organizational Updates - The company appointed Bilal Arif as Chief Operating Officer and Linea Aspesi as Chief People Officer [9] - Bethany L. Sensenig joined KalVista's Board of Directors and the Audit Committee [9] Clinical Highlights - Interim results from the KONFIDENT-KID trial showed a median time to dosing of 30 minutes and a median time to symptom relief of 1.5 hours for pediatric patients [9] - In the KONFIDENT-S trial, 84% of treated attacks were rated as satisfied by patients who switched from injectable treatments [9]

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 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 is acquiring Astria Therapeutics for an implied $13 per share, resulting in an enterprise value of approximately $700 million, with the transaction expected to close in Q1 2026 [1][2]. Group 1: Acquisition Details - The acquisition will be financed through a combination of BioCryst's available cash and a strategic financing facility of up to $550 million from Blackstone-managed funds [3][4]. - Astria's CEO, Jill Milne, will join BioCryst's board of directors upon completion of the acquisition [1]. Group 2: Strategic Benefits - The acquisition is aimed at expanding BioCryst's portfolio with a late-stage product candidate, navenibart, which is in Phase III clinical development for hereditary angioedema (HAE) [2][5]. - Navenibart is expected to offer advantages over current treatments due to its extended administration schedule of every three to six months [2]. Group 3: Future Prospects - BioCryst's existing commercialization infrastructure and expertise in HAE will enhance the distribution of navenibart [3]. - The company is also acquiring Astria's early-stage atopic dermatitis program, STAR-0310, and is exploring strategic options for this asset [3].

Hereditary Angioedema (HAE) - Navenibart - Navenibart aims to be the first-choice therapy for preventative treatment of HAE, with potential for quarterly (Q3M) and semi-annual (Q6M) administration[15, 6] - Phase 1b/2 data suggests Navenibart's efficacy could match or exceed existing treatments while reducing treatment burden[16] - The Phase 3 program, including ALPHA-ORBIT and ORBIT-EXPANSE trials, is designed to support global registration, with topline data expected in early 2027[18] - ALPHA-STAR and SOLAR trials showed a mean attack rate reduction of 91-95% across all doses, with an overall mean of 92%[24] - The ALPHA-SOLAR trial demonstrated consistent efficacy with a longer median follow-up of 9.1 months, showing a 92% attack rate reduction from baseline[28, 29] - Market research indicates that offering both Q3M and Q6M dosing options could increase market adoption, with 53% of patients initiating preventative therapy and 46% switching preventative therapy preferring Q3M & Q6M options[32] Atopic Dermatitis (AD) - STAR-0310 - STAR-0310 targets the OX40 pathway and has the potential to become the first-choice OX40 therapy for Atopic Dermatitis[45] - The U S commercial opportunity for moderate-to-severe AD is projected to reach $22 billion by 2030[35, 36, 44] - Phase 1a data demonstrates a best-in-class 68-day half-life, supporting potential for every-six-month dosing[51, 71, 77] - Phase 1a data showed broad and durable pharmacodynamic effect across Type 1, 2, and 3 inflammation, with maximal suppression achieved within one week and sustained for at least 16-20 weeks[63, 68, 77]

Financial Data and Key Metrics Changes - The company is in phase 3 for two products, with the first phase 3 readout expected by the end of the year and the second in the first half of 2026 [2] - The prophylaxis market is anticipated to grow significantly, with 60% of patients currently on prophylaxis representing 80% of the market value [4][5] Business Line Data and Key Metrics Changes - The on-demand market is expected to grow due to the introduction of oral products, which could lead to more attacks being treated [4] - The prophylaxis market is viewed as a blue ocean for oral treatments, with the company aiming to capture a significant share of new patients transitioning from injectables [5][6] Market Data and Key Metrics Changes - The U.S. market currently has about 150 to 250 new HAE patients each year, with a significant portion expected to transition to prophylaxis [5] - The company expects the overall prophylactic market to grow to 70% to 80% over time, which may impact the on-demand market [35] Company Strategy and Development Direction - The company aims to position its oral product as a leading option in the prophylaxis market, leveraging its efficacy and tolerability compared to injectables [20][21] - The strategy includes building relationships within the HAE community to facilitate product adoption and trust [51][52] Management's Comments on Operating Environment and Future Outlook - Management noted that 65% of patients on prophylaxis are dissatisfied with their current treatment, indicating a potential market opportunity for their oral product [10] - The company is optimistic about the upcoming phase 3 data and believes it will confirm the efficacy seen in phase 2 trials [12][19] Other Important Information - The company is exploring opportunities in the acquired angioedema segment, which could unlock additional patient populations [36][39] - The management is focused on building a strong organizational infrastructure in preparation for product launches [52] Q&A Session Summary Question: How likely is the company to translate phase 2 data into competitive phase 3 results? - Management expressed confidence in the consistency of trial designs and endpoints, suggesting that the phase 3 results should align with phase 2 findings [13][19] Question: What drives the stickiness of current treatments among patients? - Management indicated that patient satisfaction and the availability of alternatives are key factors, with many patients expressing a desire for better options [9][10] Question: How does the company plan to differentiate its on-demand product? - The company believes that the rapid onset of symptom relief and the ability to achieve complete resolution with a single dose will set its product apart [28][29] Question: What is the expected impact of payers on market share? - Currently, payers have not significantly influenced the prophylaxis market, but a broader label could lead to preferential treatment from payers [25][26] Question: How does the company plan to prepare for the launch of its products? - The company is focused on hiring experienced personnel and building relationships within the HAE community to support the launch [51][52]

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 demonstrating the long-term efficacy and safety of ORLADEYO (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) across all age groups [1][2] Group 1: Efficacy and Safety - Data from clinical trials and real-world settings indicate that ORLADEYO is an effective and well-tolerated long-term prophylactic treatment for HAE, with patients experiencing fewer and less severe attacks [2] - The ongoing APeX-P study shows that berotralstat significantly reduces the number of HAE attacks requiring treatment or professional care in pediatric patients aged 2 to 11 years [4] - In the Berolife study, patients aged over 12 years reported a mean baseline attack rate of 1.1 attacks per month, which decreased significantly during treatment [7][10] Group 2: Quality of Life Improvements - Patients reported an 86% reduction in attacks requiring professional care, with the mean adjusted monthly attack rate decreasing from 1.28 to 0.38 during the initial weeks of treatment [10] - Significant improvements in quality of life were observed, with 60% of patients achieving the Minimal Clinically Important Difference (MCID) in the Angioedema Quality of Life Questionnaire (AE-QoL) total score at week 24 [20] - Participants noted reduced frequency and severity of HAE attacks, leading to improved disease management and a greater sense of normalcy in daily life [21] Group 3: Patient Experience - A qualitative study revealed that 71% of participants had switched to berotralstat from previous treatments due to long-term tolerability concerns [14] - Patients described berotralstat as less burdensome and easier to incorporate into daily routines compared to prior therapies, contributing to enhanced quality of life [21] Group 4: Product Information - ORLADEYO is the first and only oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [16][17] - The recommended dosage is 150 mg once daily, with specific recommendations for patients with hepatic impairment [22]