Core Insights - Pharming Group reported strong financial performance for 2025, with significant revenue growth and a return to operating profitability, driven by its products RUCONEST and Joenja [6][5][4] Financial Performance - Total revenue for 2025 reached EUR 376.1 million, reflecting a 27% year-over-year increase, with fourth-quarter revenue of EUR 106.5 million, up 15% from the previous year [4][5] - Adjusted operating profit for 2025 was EUR 36.4 million, a turnaround from a loss of EUR 8.6 million in 2024, while operating cash flow improved to EUR 54.7 million [2][5] - Cash and marketable securities at year-end totaled EUR 181.1 million, despite EUR 68 million used for the Abliva acquisition [2] Product Performance - RUCONEST experienced a 26% year-over-year revenue growth in 2025, with a 9% increase in the fourth quarter, while Joenja's revenue grew 29% for the year and 53% in Q4 [1][4] - Joenja's global revenue for the fourth quarter was $19.8 million, with full-year revenue reaching $58 million, and the number of patients on paid therapy in the U.S. increased by 25% to 120 [9][10] Market Dynamics - The U.S. hereditary angioedema market is seeing new treatment options, but RUCONEST remains resilient, focusing on high-attack patients [7][8] - Pharming noted competitive impacts in Q4 as patients trialed new therapies, with some returning to RUCONEST, indicating a potential for patient switching back to the product [8] Future Guidance - For 2026, Pharming expects revenue growth of EUR 405 million to EUR 425 million, representing an 8% to 13% increase compared to 2025, driven by continued growth in RUCONEST and accelerated growth in Joenja [18] - Operating expenses for 2026 are projected to be between EUR 330 million and EUR 335 million, including over EUR 60 million in R&D investments [21] Pipeline and Regulatory Updates - Pharming is expanding Joenja beyond APDS into additional primary immunodeficiencies, with ongoing phase II trials expected to yield data in the second half of 2026 [13] - The company is addressing regulatory challenges for Joenja's pediatric label expansion and expects to align with the FDA on a resubmission path [15][16]

Financial Data and Key Metrics Changes - Total revenues grew by 15% in Q4 2025 and by 27% for the full year, reaching EUR 376.1 million [2][25] - Operating profit for 2025 was $26 million, a significant improvement from a loss in 2024 [2][28] - Operating cash flow was $55 million in 2025, with cash position at year-end above that of 2024 [3][28] - Adjusted operating profit was EUR 36.4 million, compared to a loss of EUR 8.6 million in 2024 [28] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 26% year-over-year and 9% in Q4 2025, with U.S. volume growth of 20% [3][9] - Joenja revenue increased by 29% year-over-year and 53% in Q4 2025, reaching $19.8 million globally [4][12] - Joenja annual revenues exceeded EUR 50 million for the first time, triggering a EUR 5 million sales milestone payment [25] Market Data and Key Metrics Changes - Joenja's growth was fueled by new patient acceleration in the U.S. and increased demand in international markets, including the U.K. [4][14] - The number of identified APDS patients in the U.S. increased by 40 in 2025, more than double the increase in 2024 [14] Company Strategy and Development Direction - The company aims to maintain strong financial discipline while investing in growth drivers, focusing on becoming a leading rare disease company [6][8] - RUCONEST is positioned as a cornerstone treatment for severe HAE patients, while Joenja is expected to generate a significant portion of future revenues [32][34] - The company is exploring geographic expansion and regulatory approvals for Joenja, with a focus on pediatric indications [15][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth for RUCONEST and Joenja, with expectations for 2026 revenue between EUR 405 million and EUR 425 million [6][29] - The company anticipates operating expenses to increase at a slower pace, despite higher R&D investments [30] - Management highlighted the importance of upcoming clinical data readouts as potential value inflection points [32][34] Other Important Information - The company completed the enrollment of two leniolisib phase II trials and expects top-line data readout in the second half of 2026 [7][19] - The company is committed to optimizing capital allocations to support growth and shareholder value [8][30] Q&A Session Summary Question: Joenja growth expectations from U.S. vs. international markets - Management expects around 70%-75% of Joenja's growth in 2026 to come from the U.S. market [41][43] Question: RUCONEST patient return dynamics - Patients tend to return to RUCONEST quickly after trialing other treatments that do not control their crises effectively [37][38] Question: Timeline for getting patients on paid therapy in international markets - The company plans to submit pricing for reimbursement in Japan shortly, with launch timelines planned for summer [60][61] Question: Variants of uncertain significance (VUS) project timeline - New experiments are being planned, but timelines for results are not yet clear [76] Question: Inventory-related effects on RUCONEST sales - The company expects a higher inventory drawdown in Q1 due to market dynamics settling [78]

Financial Data and Key Metrics Changes - Total revenues grew by 15% in Q4 2025 and by 27% for the full year, reaching EUR 376.1 million [2][25] - Operating profit for 2025 was $26 million, a significant improvement from a loss in 2024 [2] - Operating cash flow was $55 million in 2025, with cash position at year-end above 2024 levels [3][28] - Adjusted operating profit was EUR 36.4 million, compared to a loss of EUR 8.6 million in 2024 [28] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 26% year-over-year and 9% in Q4 2025, with U.S. volume growth of 20% [4][9] - Joenja revenue increased by 29% year-over-year and 53% in Q4 2025, reaching $19.8 million globally [12][25] - Joenja's annual revenues exceeded EUR 50 million for the first time, triggering a EUR 5 million sales milestone payment [25] Market Data and Key Metrics Changes - Joenja's growth was fueled by new patient acceleration in the U.S. and increased demand in international markets, including the U.K. [4][14] - The number of identified APDS patients in the U.S. increased by 40 in 2025, more than double the increase in 2024 [14] Company Strategy and Development Direction - The company aims to maintain strong financial discipline while optimizing capital allocations to growth drivers [6][8] - Pharming is transitioning from a single asset company to a high-growth biotech with two commercial products and a late-stage pipeline [4] - The company expects 2026 revenue between EUR 405 million and EUR 425 million, representing 8%-13% growth [6][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth for RUCONEST and accelerating growth for Joenja, particularly in the U.S. pediatric market [7][30] - The company is focused on addressing FDA feedback regarding the pediatric label expansion for Joenja [22] - Management highlighted the importance of upcoming clinical data readouts as potential value inflection points [33] Other Important Information - The company completed the enrollment of two leniolisib phase 2 trials for lower prevalence PIDs, with data readouts expected in the second half of 2026 [7][19] - The company is committed to exploring M&A opportunities that are value-accretive and complementary to its mission [67] Q&A Session Summary Question: Joenja growth expectations from U.S. vs. international markets - Management expects approximately 70%-75% of Joenja's growth in 2026 to come from the U.S. market [41][43] Question: Dynamics of RUCONEST and patient returns - Patients tend to return to RUCONEST quickly after trialing new treatments that do not adequately control their crises [38] Question: Timeline for getting patients on paid therapy in international markets - The company plans to submit pricing for reimbursement in Japan shortly, with launch timelines planned for summer [60] Question: Variants of uncertain significance (VUS) and their potential reclassification - New experiments are planned to generate data needed for reclassification of VUS, but timelines are still uncertain [76] Question: Inventory-related effects on RUCONEST sales - The company anticipates a higher inventory drawdown in Q1 due to market dynamics settling, which is a common quarterly fluctuation [78]

Financial Data and Key Metrics Changes - Total revenues grew by 15% in Q4 2025 and by 27% for the full year, reaching EUR 376.1 million [3][26] - Operating profit for 2025 was $26 million, a significant improvement from a loss in 2024 [3][29] - Operating cash flow was $55 million in 2025, with cash position at year-end above that of 2024 [4][29] - Adjusted operating profit was EUR 36.4 million, compared to a loss of EUR 8.6 million in 2024 [29] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 26% year-over-year and 9% in Q4 2025, with U.S. volume growth of 20% [4][9] - Joenja experienced a 29% year-over-year growth and a 53% growth in Q4 2025, reaching $19.8 million globally [5][12] - Joenja's annual revenues exceeded EUR 50 million for the first time, triggering a EUR 5 million sales milestone payment [26] Market Data and Key Metrics Changes - Joenja's growth was fueled by new patient acceleration in the U.S. and increased demand in international markets, particularly in the U.K. [5][13] - The number of identified APDS patients in the U.S. increased significantly, reflecting successful educational efforts [13][16] Company Strategy and Development Direction - The company aims to maintain strong financial discipline while optimizing capital allocations to growth drivers [6][8] - Pharming is transitioning from a single asset company to a high-growth biotech with two commercial products and a late-stage pipeline [5][6] - The company is focused on expanding Joenja's market presence and preparing for pediatric approvals in the U.S. [7][15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth for RUCONEST and Joenja, with expectations for 2026 revenue between EUR 405 million and EUR 425 million [6][30] - The company anticipates challenges in early 2026 due to inventory drawdowns but expects mid-single-digit growth for RUCONEST [30][31] - Management highlighted the importance of upcoming clinical data readouts as potential value inflection points [33] Other Important Information - The company completed the enrollment of two leniolisib phase 2 trials and expects data readouts in the second half of 2026 [7][19] - The company is actively pursuing regulatory approvals in multiple regions, including Europe and Japan, for its pipeline products [24][25] Q&A Session Summary Question: Joenja growth expectations from U.S. vs. international markets - Management expects approximately 70%-75% of Joenja's growth in 2026 to come from the U.S. market [42][43] Question: Dynamics of RUCONEST and patient returns - Patients tend to return to RUCONEST quickly after trying other treatments that do not adequately control their crises [38][50] Question: Access to paid therapies in international markets - The reimbursement process in international markets is slower initially but becomes efficient once negotiated [54] Question: Timeline for additional trials for Joenja - Management anticipates a phase 3 randomized study following the results of ongoing trials [56] Question: Variants of uncertain significance (VUS) and their potential - New experiments are planned to better understand VUS, but timelines for results are still uncertain [76]

Summary of Pharming Group FY Conference Call Company Overview - **Company**: Pharming Group (NasdaqGM:PHAR) - **Date of Conference**: February 25, 2026 - **Key Participants**: Anurag Relan (Chief Medical Officer), Kenneth Lynard (CFO), Michael Levitan (IR team) Core Points and Arguments Financial Performance - **2025 Sales**: $376 million, representing a **27% growth** compared to 2024 [6] - **2026 Revenue Guidance**: Expected to be between **$405 million and $425 million**, indicating an **8%-13% growth** [7] - **Operating Expenses for 2025**: Projected to be between **$304 million and $308 million** [7] - **Operating Profit**: Generated **$30 million** in operating profit for the first nine months of the year [8] - **Operating Cash Flow**: Achieved **$44 million** in operating cash flow [8] Product Highlights - **RUCONEST**: - Approved for treating acute attacks of hereditary angioedema (HAE) and has been in the market for about **10 years** [4] - Unique value proposition as the only recombinant C1 inhibitor replacement therapy [9] - Demonstrated efficacy with **97%** of attacks treated successfully in clinical trials [10] - Sustained response in **93%** of treated attacks lasting at least three days [10] - **Joenja**: - Launched in 2023 for treating activated PI3K delta syndrome (APDS) [5] - Considered a significant growth catalyst with potential for higher prevalence than previously observed [5] - Ongoing Phase II trials for leniolisib in new indications [5] - **Napazimone (formerly KL1333)**: - Targeting primary mitochondrial disease with a significant patient population of over **30,000** diagnosed in the U.S. and Europe [21] - Phase 2 program underway with positive interim analysis indicating potential for disease-modifying treatment [22] Strategic Developments - **Acquisition of Abliva**: Enhances the pipeline with KL1333 (napazimone) making good progress in trials [5] - **Commercial Strategy**: Pharming has withdrawn from commercial activities related to RUCONEST outside the U.S. but retains infrastructure to support Joenja's growth in Europe [40] - **Business Development**: Active scouting for in-licensing and M&A opportunities to enhance the pipeline [42] Additional Important Information - **Regulatory Updates**: - Received a Complete Response Letter for the supplemental NDA application to expand Joenja's use to pediatric patients aged 4-11 [26] - Anticipated approval from EMA in March, with potential approval in Japan later this year [27] - **Clinical Trials**: - Two Phase II proof-of-concept studies for Joenja expected to read out in the second half of the year [27] - Napazimone study (FALCON Study) enrolling **180 patients** with results expected by the end of next year [25] - **Market Position**: Pharming aims to develop into a leading global rare disease company with a diverse portfolio and strong market presence [6] This summary encapsulates the key points discussed during the Pharming Group FY Conference Call, highlighting the company's financial performance, product developments, strategic initiatives, and regulatory updates.

Core Insights - Pharming Group forecasts 2026 revenue between $405 million and $425 million, representing an 8% to 13% growth compared to 2025, exceeding analyst expectations of approximately $398 million [1][10] - The company emphasizes continued revenue growth, disciplined spending, and multiple clinical readouts expected over the next two years [4] Financial Guidance - For 2026, Pharming expects operating expenses to be between $300 million and $335 million, with a gross margin of approximately 90% [1][12] - The company reported preliminary 2025 revenue of around $376 million, a 27% increase from 2024, with Joenja revenue surpassing $50 million [7][9] Commercial Portfolio - RUCONEST is projected to generate $318 million in 2025, reflecting a 26% year-over-year growth, while Joenja is expected to reach $58 million, up 29% [9] - CEO Fabrice Chouraqui highlighted RUCONEST's durability and its role as a reliable treatment for hereditary angioedema patients, noting its complex manufacturing process [5][14] Regulatory and Pipeline Updates - Pharming plans to request a Type A meeting with the FDA in March regarding pediatric dosing for Joenja, which has been excluded from 2026 revenue guidance due to regulatory uncertainties [2][6][11] - Management anticipates two phase II readouts for leniolisib (Joenja) in H2 2026 and an EMA decision in H1 2026, alongside a pivotal trial for napazimone (KL1333) with results expected in late 2027 [3][16][21] Market Dynamics - RUCONEST's growth is expected to be driven by U.S. volume growth, with a 3% price increase implemented in line with CPI [14] - The company has not included potential revenue from reclassification of VUS patients in its guidance due to timing uncertainties [15]

Core Insights - Pharming Group N.V. has announced its financial guidance for 2026, projecting total revenues between US$405 million and US$425 million, representing an 8% to 13% growth [3][7][8] - The company will highlight its advancing clinical-stage pipeline, focusing on two major programs targeting primary immunodeficiencies and mitochondrial diseases during its Investor Day [2][4][6] Financial Guidance - Total revenue guidance for 2026 is set between US$405 million and US$425 million, driven by growth in its commercial products [3][8] - Total operating expenses are expected to be between US$330 million and US$335 million, primarily due to increased R&D expenses related to ongoing clinical trials [7][8] Pipeline Overview - Leniolisib is being developed for primary immunodeficiencies with immune dysregulation, with ongoing Phase II trials expected to yield top-line data in the second half of 2026 [4][5] - Napazimone (KL1333) is being developed for mtDNA-driven mitochondrial disease, with a pivotal clinical trial ongoing and results anticipated in 2027 [6][9] Clinical Programs - Leniolisib is currently approved as Joenja in the U.S. and is the first targeted treatment for activated PI3Kδ syndrome, with potential applicability across broader patient populations [5][6] - Napazimone (KL1333) aims to become the first standard of care for mtDNA-driven mitochondrial disease, addressing significant unmet medical needs [6][9] Investor Day Details - The Investor Day will feature presentations from clinical experts discussing disease biology and the potential impact of Pharming's programs [10][16] - The event is scheduled for February 3, 2026, from 10:00 a.m. to 12:00 p.m. EST, and will be available via webcast [11]

Summary of Pharming Group Conference Call Company Overview - Pharming Group is a Dutch-based biotech company primarily operating in the US, known for its recombinant protein platform and the drug RUCONEST, which has been on the market for 10 years [3][4] - The company has transitioned from a one-asset firm to a high-growth biotech with two market assets, RUCONEST and Joenja, both experiencing double-digit growth [3][5] Financial Performance - Pharming reported strong third-quarter results, raising revenue guidance due to the growth of RUCONEST (29% year-on-year in Q4) and Joenja (35% growth) [5][6] - The company aims to maintain double-digit growth while investing in high-growth assets from generated cash [10][32] Product Insights RUCONEST - RUCONEST serves a specific segment of the Hemophilia A (HA) market, targeting patients who are not controlled by prophylactic treatments and have severe crises [7][9] - The drug's unique value proposition includes its ability to replace the missing protein and provide a fast onset of action through IV administration [7][8] - Pharming does not foresee any new treatments that could significantly impact RUCONEST's market share, expecting continued growth [9][10] - The company has exited non-US markets due to financial unsustainability, with less than 1.5% of RUCONEST sales coming from those regions [11][12] Joenja - Joenja is positioned as a high-growth drug with potential to become Pharming's first billion-dollar product, currently indicated for APDS (Activated PI3K Delta Syndrome) [14][15] - The company is expanding Joenja's indications to pediatric patients, with FDA approval expected by the end of January, which could increase the addressable market by 30% [16][18] - There is also an opportunity to reclassify Variant of Uncertain Significance (VUS) patients as APDS patients, potentially increasing the patient base by 50% [17][18] Market Expansion - Joenja has launched successfully in the UK, with plans for further expansion into Europe and Japan, pending regulatory approvals [23][24] - The company is focused on ensuring appropriate pricing that reflects the drug's value in new markets [24] Acquisition and Pipeline - Pharming's acquisition of Abliva for KL1333, aimed at treating primary mitochondrial disease, is seen as a strategic addition to its rare disease portfolio [25][26] - KL1333 has a well-defined patient population and is in registrational phase 2 trials, with significant market potential [26][28] Operational Strategy - The company is focused on optimal capital allocation to drive growth and has recently cut 20% of G&A headcount to reallocate resources towards growth catalysts [32][33] - Pharming emphasizes the importance of disciplined management to enhance value creation in the long term [33] Conclusion - Pharming Group is positioned for continued growth through its existing products and strategic acquisitions, with a strong focus on expanding its market presence and optimizing operational efficiencies [32][33]