–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 - Saturn V Capital Management has initiated a new position in Pharvaris N.V. (PHVS), acquiring 886,332 shares valued at approximately $22.1 million during the third quarter, indicating strong interest from a major hedge fund in the biotech sector [1][2]. Company Overview - Pharvaris N.V. is a clinical-stage biotechnology company focused on developing novel oral therapies for hereditary angioedema (HAE), a rare genetic disorder [6][9]. - The company has three pivotal programs underway: PHA121 (Phase II), PHVS416 (Phase II), and PHVS719 (Phase I), targeting both acute and prophylactic treatment needs [9][10]. - As of market close on the reporting date, Pharvaris shares were priced at $25.35, reflecting a 33% increase over the past year, outperforming the S&P 500, which rose by 12% in the same period [3][4]. Financial Metrics - Pharvaris has a market capitalization of $1.6 billion and reported a net income of -$163.7 million for the trailing twelve months (TTM) [4]. - The company had €329 million in cash on hand as of September 30, which is expected to finance operations into the first half of 2027, covering major data readouts [10]. Investment Implications - Saturn V's stake in Pharvaris represents about 4.9% of its 13F assets, aligning with the fund's focus on biotech investments and high-conviction clinical risks [3][11]. - Recent positive topline data from the RAPIDe-3 Phase 3 trial showed a median time to onset of symptom relief of 1.3 hours, highlighting the potential for future growth despite the company being pre-revenue [11][12].

Core Insights - Pharvaris (PHVS) shares increased nearly 22% following positive results from the phase III RAPIDe-3 study for its drug deucrictibant, aimed at treating hereditary angioedema (HAE) attacks in adults and adolescents aged 12 and older [2][8] Study Results - The RAPIDe-3 study achieved its primary endpoint, with patients experiencing symptom relief within 1.28 hours after taking deucrictibant, compared to over 12 hours with placebo [3] - All secondary endpoints were met, including a median time to substantial symptom relief of under 3 hours with deucrictibant versus over 12 hours with placebo, and complete symptom resolution in 11.95 hours compared to more than 24 hours for placebo [3] - 83% of HAE attacks were managed with a single dose of deucrictibant, and over 93% of attacks were treated without the need for backup treatment [4] Regulatory Plans - Pharvaris plans to submit a regulatory filing for deucrictibant in the first half of 2026, which could position it as the second oral on-demand therapy for HAE after KalVista Pharmaceuticals' Ekterly [5] Competitive Landscape - Comparisons between deucrictibant and KalVista's Ekterly highlight potential advantages for deucrictibant, as it showed faster symptom relief in the RAPIDe-3 study compared to the KONFIDENT study supporting Ekterly's approval [6] - Year-to-date, Pharvaris stock has risen 53%, outperforming the industry growth of 20% [7] Product Development - Pharvaris is developing two formulations of deucrictibant: an immediate-release capsule for acute treatment and an extended-release tablet for prophylactic treatment [10] - The extended-release tablet is being evaluated in the phase III CHAPTER-3 study for HAE prophylaxis, with top-line data expected in the second half of 2026 [11] - Additionally, the extended-release formulation is under evaluation in the CREAATE study for treating acquired angioedema with C1-inhibitor deficiency [12] Market Positioning - If approved, deucrictibant will compete in the on-demand treatment space against KalVista's Ekterly, with advantages over older injectable treatments like Firazyr and Kalbitor due to its oral administration [14] - In the prophylactic treatment segment, competitors include BioCryst Pharmaceuticals and Ionis Pharmaceuticals, with BioCryst's Orladeyo and Ionis' Dawnzera being notable products [15]

Core Insights - Pharvaris N.V. is focused on developing treatments for rare diseases, particularly Hereditary Angioedema (HAE) [1][6] - Morgan Stanley has an "Overweight" rating for Pharvaris, raising the price target from $37 to $41, with the current stock price at $29.37 [1][6] Study Results - The RAPIDe-3 Phase III study of Deucrictibant showed a median time to symptom relief of 1.28 hours, significantly faster than placebo [2][3][6] - The study involved 134 participants aged 12 and older, demonstrating the efficacy of Deucrictibant in treating HAE attacks [2] - 83% of attacks were treated with a single capsule, and 93.2% of participants did not require rescue medication within 12 hours [3] Future Plans - Pharvaris plans to use the RAPIDe-3 study data for marketing authorization applications, expected to be submitted in the first half of 2026 [3][6] - The positive results from the study have contributed to an increase in PHVS stock value by 21.75% [6] Market Performance - Pharvaris' current stock price is $29.37, reflecting a change of $5.25, with a market capitalization of approximately $1.91 billion [5][6] - The stock has fluctuated between $23.80 and $29.68 today, with a 52-week high of $29.67 and a low of $11.51 [5]

Core Insights - Pharvaris N.V. announced topline data from the RAPIDe-3 Phase 3 trial of deucrictibant for treating hereditary angioedema (HAE) attacks, indicating positive results for the on-demand treatment [1][3] Study Results - The RAPIDe-3 trial enrolled 134 participants aged 12 years and older, evaluating the oral deucrictibant immediate-release (IR) capsule for HAE attacks [2] - Deucrictibant showed a faster median time to treatment response compared to placebo, with shorter median times for substantial symptom relief and complete symptom resolution [3] - The data indicated fewer attacks required a second dose or rescue medication within 12 hours [4] - 83.0% of attacks were treated with a single capsule of deucrictibant IR, and 93.2% of treated attacks did not require rescue medication [9] Safety and Tolerability - Deucrictibant was well tolerated, with no serious treatment-related adverse events reported, and no participants discontinued treatment due to adverse events [5] Future Plans - The data from the RAPIDe-3 study will support marketing authorization applications planned for submission in the first half of 2026 [3] Competitive Landscape - The FDA approved Ionis Pharmaceuticals' Dawnzera for HAE prophylaxis, which significantly reduced the monthly attack rate by 81% compared to placebo over 24 weeks [6] - KalVista Pharmaceuticals' Ekterly was also approved for acute HAE attacks, being the first oral on-demand treatment for HAE [7] Market Reaction - Pharvaris shares increased by 12.32% to $27.09 following the announcement of the trial results [7]

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]