Pharming Group N.V. Fourth quarter and full year 2025 financial results March 12, 2026 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM CEO Fabrice Chouraqui Chief Executive Officer Introduction SPEAKERS Fabrice Chouraqui Chief Executive Officer Leverne Marsh Chief Commercial Officer Anurag Relan, MD Chief Medical Officer Kenneth Lynard Chief Financial Officer Forward-looking statements This presentation may contain forward-looking statements. Forward-looking statements are statements of future expectations that ar ...

Core Viewpoint - Pharming Group N.V. reported preliminary, unaudited revenues for 2025, estimating approximately US$376 million, which exceeds previous guidance and indicates a 27% growth compared to 2024 [2][5] Financial Performance - Total revenues for 2025 are estimated at US$376 million, surpassing the revised guidance of US$365 to US$375 million [2] - This revenue growth is attributed to the continued success of RUCONEST® and increased demand for Joenja®, particularly in the U.S. market [2] - Full-year operating expenses are expected to be between US$304 million and US$308 million, reflecting the company's commitment to cost discipline [2] Future Outlook - The company anticipates sustained revenue growth and further advancements in its clinical pipeline for 2026 [2] - A complete financial report for Q4 and full year 2025 is scheduled for release on March 12, 2026 [2] Investor Day - Pharming will host a virtual Investor Day on February 3, 2026, to provide updates on its clinical pipeline and financial guidance for 2026 [1][3] - The agenda will include discussions on leniolisib and KL1333, focusing on their clinical trials and the unmet needs in related diseases [3][4] - Presentations will feature leading clinical experts discussing immune dysregulation and mitochondrial diseases, providing context for Pharming's development programs [4]

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 Viewpoint - Pharming Group N.V. announced that the FDA has accepted its supplemental New Drug Application for leniolisib, aimed at treating children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), with a target action date of January 31, 2026 [1][7]. Group 1: FDA Approval Process - The supplemental New Drug Application is based on positive results from a Phase III study showing improvements in lymphadenopathy and naïve B cells over 12 weeks [2]. - The FDA has granted Priority Review status to the application, indicating that the treatment could offer significant improvements in effectiveness or safety for serious conditions [3]. Group 2: Disease Background - APDS is a rare primary immunodeficiency characterized by immune dysregulation and recurrent infections, often leading to severe complications such as lung damage and lymphoma [5]. - The condition affects approximately 1 to 2 individuals per million globally, and patients often experience a median diagnostic delay of 7 years [5]. Group 3: Treatment Details - Leniolisib is an oral PI3Kδ inhibitor, previously approved for patients aged 12 and older, and is the first targeted treatment for APDS [6][4]. - The drug has shown statistically significant improvements in clinical trials, and its safety and tolerability have been supported by long-term data [6]. Group 4: Future Developments - Leniolisib is currently under regulatory review in multiple regions, including the European Economic Area, Japan, and Canada, for its use in APDS [6]. - The company is also evaluating leniolisib in additional clinical trials for other primary immunodeficiencies with immune dysregulation [8].

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]