Financial Performance - Total revenues for Q3 2025 increased by 30% to US$97.3 million[13, 43] - RUCONEST® revenues in Q3 2025 reached US$82.2 million[13, 22] - Joenja® revenues in Q3 2025 amounted to US$15.1 million[13, 27] - Operating profit significantly grew to US$16.0 million in Q3 2025[13, 43] - Cash flow from operations was US$32 million in Q3 2025[14, 41] - Total revenues for 9M 2025 grew 33% to US$269.6 million[44, 45] Business Updates and Outlook - 2025 revenue guidance raised to US$365-375 million due to strong RUCONEST® performance[14, 51] - RUCONEST® U S volume growth was +24% in 3Q25 and +28% in 9M25[22] - There are 116 US patients on Joenja® representing a +25% increase vs 3Q24[27] - FDA filing for Leniolisib for 4-11 year old APDS patients is based on Phase III data and has a PDUFA date of Jan 31, 2026[28]

Core Insights - Pharming Group N.V. reported a strong financial performance for Q3 2025, with total revenues increasing by 30% to US$97.3 million compared to Q3 2024, driven by growth in both RUCONEST® and Joenja® [9][30][31] Financial Performance - Total revenues for Q3 2025 reached US$97.3 million, a 30% increase from US$74.8 million in Q3 2024 [30] - RUCONEST® revenue increased by 29% to US$82.2 million, reflecting sustained growth in patients and prescribers [9][10] - Joenja® revenue grew by 35% to US$15.1 million, driven by a significant increase in patients on paid therapy [13][31] - Operating profit surged by 285% to US$15.8 million compared to US$4.1 million in Q3 2024 [32] - The company generated US$32 million in cash flow from operations during the quarter [34] Strategic Developments - Pharming announced a significant reduction in general and administrative headcount to optimize capital deployment towards high-growth initiatives [5][23] - The company is focusing on expanding its pipeline, particularly for leniolisib in broader primary immunodeficiency populations and addressing unmet needs in primary mitochondrial disease with KL1333 [4][5] Market Position and Outlook - The company raised its full-year revenue guidance to between US$365 million and US$375 million, up from the previous range of US$335 million to US$350 million [6][44] - The U.S. market contributed 89% of Q3 revenue, while the EU and Rest of World contributed 11% [14] - Pharming plans to launch a pediatric formulation of leniolisib for children aged 4 to 11 years, with FDA priority review expected by January 2026 [20][44] Organizational Changes - Leverne Marsh has been appointed as Chief Commercial Officer, effective January 1, 2026, succeeding Stephen Toor [7][24] - Kenneth Lynard was appointed as Chief Financial Officer, effective October 1, 2025, enhancing the company's financial leadership [22] Corporate Highlights - Pharming was promoted from the Euronext AScX® to the AMX® index, reflecting its growing market capitalization and trading activity [25] - The company is working on options to mitigate the impact of recently announced U.S. tariffs, although it does not expect a material impact on its business [26]

Core Insights - Pharming Group N.V. announced that 12 abstracts have been accepted for presentation at the ACAAI 2025 Annual Scientific Meeting, highlighting the company's commitment to advancing research in hereditary angioedema (HAE) and activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) [1][3]. Summary by Category Clinical Data - Five posters will present positive clinical, economic, and comparative data for RUCONEST® in the treatment of on-demand hereditary angioedema (HAE) [2]. - Seven posters will focus on the real-world effectiveness of Joenja® (leniolisib), including results from the Phase III pediatric trial and insights into the burden faced by pediatric patients and caregivers dealing with APDS [2]. Presentation Details - Specific studies include an indirect treatment comparison of recombinant C1 inhibitor and Sebetralstat for HAE on-demand therapy, cost-effectiveness analysis, and patient-reported outcome measures across clinical trials [4][5]. - The presentations are scheduled for various dates and times during the ACAAI meeting, with ePosters accessible online for registered attendees [5]. Company Overview - Pharming Group is a biopharmaceutical company focused on developing innovative medicines for rare diseases, with a significant presence in both the U.S. and Europe [14].

Financial Performance - Total revenues for Q2 2025 increased by 26% compared to Q2 2024 [16] - RUCONEST® revenues in Q2 2025 reached $80.4 million, up from $63.0 million in Q2 2024 [14, 23] - Joenja® revenues in Q2 2025 were $12.8 million, compared to $11.1 million in Q2 2024 [14, 28] - Adjusted operating profit for Q2 2025 was $12.9 million, compared to a loss of $3.1 million in Q2 2024 [14] - The company is raising its 2025 revenue guidance to $335-350 million [16] - Revenues for 1H 2025 increased by 33% to $172.315 million, compared to $129.679 million in 1H 2024 [55, 63] Commercial Updates - RUCONEST® U S volume growth increased by 27% in Q2 2025 and 31% in 1H 2025 [23] - The number of U S patients on Joenja® increased by 25% compared to Q2 2024, reaching 114 patients in Q2 2025 [28] Research and Development - A study uncovered >100 new variants leading to PI3Kδ hyperactivity (GOF variants) [38] - Data suggests that VUS patients with these GOF variants should be reclassified as APDS [39] - APDS may be up to 100x more prevalent than previously estimated [40]

Core Insights - Pharming Group N.V. reported a strong second quarter with a total revenue growth of 26% and positive operating profit, driven by significant growth in RUCONEST® and Joenja® [2][7][29] - The company raised its full-year revenue guidance to between US$335 million and US$350 million, up from the previous range of US$325 million to US$340 million [7][44] Financial Performance - Total revenues for Q2 2025 increased by 26% to US$93.2 million compared to Q2 2024, with RUCONEST® revenue growing by 28% to US$80.4 million and Joenja® revenue increasing by 15% to US$12.8 million [7][29][30] - Operating profit for Q2 2025 was US$10.8 million, a significant improvement from a loss of US$3.1 million in Q2 2024 [7][31] - For the first half of 2025, total revenues rose by 33% to US$172.3 million, with RUCONEST® revenues up 37% to US$149.0 million [35][36] Product Development and Market Expansion - Joenja® is showing accelerated uptake, with patient growth in the first half of 2025 surpassing the total for all of 2024 [2][11] - A recent study published in the journal Cell suggests that the prevalence of APDS may be up to 100 times higher than previously estimated, potentially expanding Joenja®'s addressable patient population [3][16] - The company launched Joenja® in the U.K. and submitted a regulatory filing for leniolisib in Japan, indicating ongoing efforts to expand market access [4][20] Strategic Initiatives - Pharming is working on mitigating the impact of U.S. tariffs, although it does not expect a material impact on its business [5] - The company is on track to reduce general and administrative expenses by 15% or US$10 million to optimize capital allocation for sustainable growth [26] - The acquisition of Abliva AB was completed, increasing Pharming's ownership to 100% [25] Cash Flow and Financial Position - Cash and marketable securities increased to US$130.8 million at the end of Q2 2025, up from US$108.9 million at the end of Q1 2025, primarily due to cash generated from operations [7][34] - The company reported a net profit of US$4.6 million for Q2 2025, compared to a net loss of US$1.2 million in Q2 2024 [33]

Core Insights - Pharming Group N.V. reported a strong performance in the first quarter of 2025, with total revenues increasing by 42% to US$79.1 million compared to the same period in 2024, driven primarily by a 49% increase in RUCONEST® revenue [6][33][37] - The company has raised its full-year revenue guidance to between US$325 million and US$340 million, reflecting confidence in continued growth [2][37] - Pharming is making significant progress in expanding the availability of Joenja® for additional patients and is preparing for regulatory submissions in multiple markets [3][12][18] Financial Performance - Total revenues for Q1 2025 were US$79.1 million, up from US$55.6 million in Q1 2024, with RUCONEST® contributing US$68.6 million, a 49% increase [6][33] - Joenja® revenues reached US$10.5 million, a 9% increase compared to Q1 2024, with an 18% increase in unit sales volume [6][9][33] - Operating loss improved to US$7.0 million from US$16.3 million in Q1 2024, with adjusted operating profit of US$0.8 million when excluding non-recurring expenses related to the Abliva acquisition [35][36] Product Developments - Joenja® was launched in England and Wales in April 2025 following a positive reimbursement decision from NICE, and the company is preparing to file for U.S. FDA approval for pediatric use in Q3 2025 [3][12][17] - The company is advancing its pipeline, including ongoing clinical trials for leniolisib in primary immunodeficiencies and KL1333 for mitochondrial diseases [4][21][25] Market Contributions - The U.S. market accounted for 97% of total revenues in Q1 2025, highlighting the strong demand for RUCONEST® and Joenja® in this region [7][8] - The company has identified approximately 250 APDS patients in the U.S. eligible for Joenja®, with ongoing efforts to find and transition more patients to paid therapy [11][13][14] Strategic Initiatives - Pharming completed the acquisition of Abliva AB for approximately US$66.1 million, which is expected to enhance future growth prospects [23][24] - The company is optimizing capital allocation with a target of reducing general and administrative expenses by 15% or US$10 million annually [2][6]