Core Insights - Pharming is presenting its financial guidance for the year and highlighting its clinical stage pipeline programs during the 2026 Investor Day [1][2] - The company has evolved from a single asset company to a fast-growing biotech with two commercial assets experiencing double-digit growth and a late-stage pipeline with two programs each having over $1 billion sales potential [3] Financial Update - A short business and financial update will be provided, focusing on the financial guidance for the year [2] - The company emphasizes its growth trajectory and the potential of its pipeline programs [3] Pipeline Overview - The presentation will include two longer sessions dedicated to the pipeline, specifically on leniolisib for high prevalence primary immunodeficiencies (PIDs) and KL1333, napazimone [2]

Summary of Pharming Group 2026 Investor Day Company Overview - **Company**: Pharming Group (NasdaqGM:PHAR) - **Event**: 2026 Investor Day held on February 3, 2026 - **CEO**: Fabrice Chouraqui Financial Performance - **2025 Revenue**: Approximately $376 million, a 27% increase from 2024, slightly exceeding previous guidance [5][6] - **Reconnect Revenue**: $318 million, a growth of 26% year-over-year [7] - **Joenja Revenue**: $58 million, a growth of 29% [7] - **Gross Margin**: Expected around 88%-89% for 2025, excluding a $5 million milestone payment to Novartis [8] - **Cash Position**: $181 million in cash and marketable securities at the end of 2025 [9] 2026 Financial Guidance - **Expected Revenue**: Between $405-$425 million, implying growth of 8%-13% over 2025 [9] - **Reconnect Growth**: Mid-single-digit growth expected, with a decline in ex-U.S. revenues due to market exit [9] - **Joenja Growth**: Anticipated to accelerate, with annual growth around 10 percentage points higher than in 2025 [9] - **Operating Expenses**: Expected between $300 million-$335 million, including over $60 million in R&D investments [10][11] Pipeline and Product Development - **Leniolisib**: Focused on high-prevalence primary immunodeficiencies (PIDs) and is in pivotal phase 2 trials [4][31] - **KL1333 (napazimod)**: Targeting primary mitochondrial disease, also a billion-dollar opportunity [4] - **Joenja**: Committed to pediatric label expansion despite FDA response; Type A meeting scheduled for March [3][10] - **Market Potential**: Both Reconnect and Joenja have over $1 billion sales potential each [2] Market Dynamics - **Reconnect**: Positioned as a cornerstone treatment for difficult-to-treat patients, with a mechanism that provides reliability during crises [2][30] - **Joenja**: Growth opportunities identified in APDS, with potential to reclassify about 250 VUS patients as APDS over time [3] - **CVID Expansion**: Plans to expand into larger PIDs and CVID, unlocking a much larger market [4] Clinical Insights - **Leniolisib's Role**: Investigated for treating immune dysregulation in PIDs, with a strong scientific rationale and patient need [55][56] - **CVID and APDS**: Clinical overlap noted, with a significant portion of CVID patients exhibiting similar characteristics to APDS [44][46] - **Patient Experience**: Anecdotal evidence suggests that leniolisib has provided hope and improved quality of life for patients with CVID [53] Regulatory and Approval Status - **FDA Engagement**: Plans to engage with the FDA regarding pediatric approval for Joenja and address concerns raised in the recent CRL [15][29] - **EMA Approval**: Expected approval for leniolisib in the first half of 2026 [32] Conclusion - Pharming Group is positioned for substantial growth with a strong pipeline and financial performance. The company is focused on expanding its market presence in rare diseases, particularly through innovative treatments like leniolisib and Joenja, while maintaining financial discipline and strategic investments in R&D.

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]

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].

Core Insights - Pharming Group N.V. is presenting Phase III clinical data for leniolisib, targeting pediatric patients aged 4-11 years with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) at the 2025 Annual Meeting of the Clinical Immunology Society [1][2] Presentations - The presentation titled "Primary and Safety Outcomes of a Phase 3 Open-Label, Single-Arm, 12-Week Study of Treatment With PI3Kδ Inhibitor Leniolisib in Pediatric Patients Aged 4-11 Years With Activated PI3Kδ Syndrome (APDS)" will be led by Dr. Shanmuganathan Chandrakasan on May 2, 2025 [2] - Additional presentations include a systematic literature review on immune dysregulation in common variable immunodeficiency and insights into APDS from patient perspectives [2] About Leniolisib - Leniolisib is an oral small molecule PI3Kδ inhibitor approved in several countries as the first targeted treatment for APDS in patients aged 12 and older [3] - The drug has shown statistically significant improvement in co-primary endpoints in a Phase III clinical trial, indicating a favorable impact on immune dysregulation [3] - Leniolisib is under regulatory review in the European Economic Area, Canada, and other regions, with plans for approval in Japan [3] About Pharming Group N.V. - Pharming Group N.V. is a global biopharmaceutical company focused on developing innovative medicines for rare and life-threatening diseases [4] - The company is headquartered in Leiden, the Netherlands, and operates in over 30 markets worldwide [4]