BioCryst highlighted the FDA’s approval in December 2025 of ORLADEYO oral pellets for children ages 2 to less than 12 . Chief Development Officer Dr. Bill Sheridan said the company is continuing marketing authorization applications in other major regions. Management described the unmet need as significant, noting that HAE and related attacks are underdiagnosed in younger children and that prophylaxis usage is about half that of adults.Gayer emphasized patient persistence and a “super responder” population f ...

Pharvaris (NasdaqGS:PHVS) FY Conference February 26, 2026 09:20 AM ET Company ParticipantsBerndt Modig - CEOMaggie Beller - Executive Director and Head of Corporate and Investor CommunicationsConference Call ParticipantsJeff Jones - Managing Director and Senior Analyst of BiotechnologyJeff JonesMorning, everyone, and welcome back to Oppenheimer's Healthcare Conference. I'm Jeff Jones, one of the analysts on the biotech team, and I'm delighted to welcome this morning, Berndt Modig, CEO of Pharvaris, as well ...

Core Insights - BioCryst Pharmaceuticals has completed the acquisition of Astria Therapeutics, enhancing its leadership in hereditary angioedema (HAE) and supporting long-term growth [1][5] Acquisition Details - The acquisition was valued at approximately $700 million, financed through cash on hand and a financing facility, with BioCryst issuing about 37.3 million shares to Astria's equity holders [5] Product Portfolio Enhancement - BioCryst adds navenibart, a late-stage plasma kallikrein inhibitor in Phase 3 development, which could be the first HAE therapy with every-three and every-six month dosing, improving patient treatment experiences [2][3] - The company now offers both the leading oral therapy (ORLADEYO®) and a potentially best-in-class injectable option, providing optimal choices for individualized patient care [3] Strategic Leadership Changes - Jill C. Milne, Ph.D., co-founder and CEO of Astria, has joined BioCryst's Board of Directors, while John Ruesch has been appointed as Chief Technical Operations Officer, bringing expertise in product development [6] Additional Programs - BioCryst has acquired Astria's early-stage program for atopic dermatitis, STAR0310, and plans to explore strategic alternatives for this program [4]

Core Insights - BioCryst Pharmaceuticals has received FDA approval for ORLADEYO® (berotralstat), the first and only targeted oral prophylactic therapy for pediatric patients with hereditary angioedema (HAE) aged 2 to less than 12 years [1][4][12] Group 1: Product Details - ORLADEYO is now available in an oral pellet formulation, designed for easier administration to children, allowing it to be swallowed directly or sprinkled over soft food [6][9] - The approval was based on positive interim data from the APeX-P clinical trial, which demonstrated that ORLADEYO was well tolerated and resulted in early and sustained reductions in monthly attack rates for pediatric patients [5][7] Group 2: Market Context - Approximately 40% of children with HAE experience their first attack by age 5, highlighting the need for effective treatment options that are less burdensome than intravenous or subcutaneous therapies [2] - The introduction of ORLADEYO oral pellets is expected to change the management of HAE in younger patients, providing a more convenient option for families and caregivers [9] Group 3: Company Commitment - BioCryst is committed to supporting patients through its Empower Patient Services, which offers comprehensive assistance for navigating insurance and treatment needs [10] - The company has plans for additional regulatory filings for ORLADEYO in other regions, including Europe and Japan, indicating a strategy for global market expansion [8]

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]