Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Oppenheimer 36th Annual Healthcare Life Sciences Conference (February 2026)
31 slides from Pharming Group Nv's conference presentation, Feb 25, 2026
Slide 1: Pharming Group N.V. Oppenheimer 36th Annual Healthcare Life Sciences Conference
Pharming Group N.V. Oppenheimer 36th Annual Healthcare Life Sciences Conference February 25, 2026 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Forward-looking statements
Forward-looking statements This presentation may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual resu
Slide 3: Combination of commercial and pipeline assets poised to deliver strong value creation
Combination of commercial and pipeline assets poised to deliver strong value creation Commercial Pipeline RUCONEST® HAE (recombinant C1 esterase inhibitor) Differentiated value proposition Highly specific manufacturing process PIDs Joenja® (leniolisib) for APDS > $1B leniolisib for PIDs / CVID reven
Slide 4: Vision for the future
Vision for the future Develop a leading global rare disease company with a diverse portfolio and presence in large markets, leveraging proven and efficient clinical development, supply chain, and commercial infrastructure 4
Slide 5: Strong commercial and financial momentum
Strong commercial and financial momentum Revenues • Announced preliminary 2025 revenues* of $376M RUCONEST® Joenja® (+ 27%) – above latest guidance 376* • Results reflects continued growth of RUCONEST® and acceleration in Joenja® APDS uptake Amounts in US$ million 58 297 45 • Significant operating p
Slide 6: Diverse rare disease portfolio and pipeline
Diverse rare disease portfolio and pipeline Phase III / registrational Regulatory Approval and Preclinical Phase I Phase II study submission commercialization Hereditary angioedema (HAE) RUCONEST® acute HAE Primary immunodeficiencies (PIDs) with immune dysregulation (Marketed in the U.S., U.K. - Eng
Slide 7: RUCONEST for HAE
% Pharming’ RUCONEST® for HAE
Slide 8: RUCONEST poised to remain a cornerstone on-demand treatment for difficult to treat HAE patients
RUCONEST® poised to remain a cornerstone on-demand treatment for difficult to treat HAE patients Differentiated value proposition • Only recombinant C1-INH protein replacement therapy • Targets the root cause of HAE across all pathways • IV administration – rapid onset, high dose Unique patient popu
Slide 9: Joenja (leniolisib) APDS & PID indications
Joenja® (leniolisib) APDS & PID indications
Slide 10: PI3Kδ is a master regulator of the immune system and imbalance contributes to immune dysregulation
PI3Kẟ is a master regulator of the immune system and imbalance contributes to immune dysregulation PI3Kẟ is a master regulator of Immune Shared pathology under the immune system and influences dysregulation pathology the influence of PI3K Lymphoproliferation p110δ APDS p85ɑ PI3Kδ AKT/PKB Autoimmuni
Slide 11: Reports of Joenja changing patients' lives
Reports of Joenja® changing patients’ lives 24-year-old male with APDS whose progress was followed in the Joenja® open-label extension study for 6 years Rao VK, et al. Blood Adv. 2024;8(12):3092-3108. doi:10.1182/bloodadvances.2023011000. 11
Slide 12: Unlocking Joenja (leniolisib) growth to realize $1Bn+ potential
Unlocking Joenja® (leniolisib) growth to realize $1Bn+ potential Expanding addressable patient population and indications U.S. patient numbers ONLY shown to illustrate prevalence CVID with immune PIDs with immune dysregulation dysregulation linked APDS* to PI3Kδ signaling >13,000 U.S. patients ~500
Slide 13: Leniolisib compassionate use experience in PID/CVID with immune dysregulation
Leniolisib compassionate use experience in PID/CVID with immune dysregulation • 6 patients treated in Expanded Access Program • Leniolisib has been generally well-tolerated with signs of improvements: • Biomarkers (immunophenotype) • Lymphoproliferation • End-organ disease • Fatigue/well-being • Med
Slide 14: Three primary immunodeficiency with immune dysregulation indications driven by dysfunctional B and T cells
Three primary immunodeficiency with immune dysregulation indications driven by dysfunctional B and T cells under the influence of the PI3Kδ pathway APDS Genetic PIDs linked to PI3Kδ CVID w/immune dysregulation Prevalence per million population 1.5 7.5 39 Genetic Diagnosis Yes. (PIK3CD, PIK3R1) Yes.
Slide 15: Napazimone (KL1333) mtDNA Mitochondrial Disease
Napazimone (KL1333) mtDNA Mitochondrial Disease
Slide 16: Napazimone (KL1333) for mtDNA-driven primary mitochondrial disease
Napazimone (KL1333) for mtDNA-driven primary mitochondrial disease Aiming for the first disease-modifying treatment Napazimone (KL1333) targets underlying pathology Dysfunctional mitochondria • Normalizes NAD+/NADH ratio and mitochondrial function, with evidence from in vitro data, animal models, Im
Slide 17: Phase Ia/b study of napazimone (KL1333) demonstrated positive changes in outcome measures
In a Phase Ia/b study, napazimone (KL1333) demonstrated positive changes in outcome measures Reduction of fatigue Improvement in muscle function Biomarker changes Changes from baseline to day 101 Changes from baseline to day 101 Lactate: Pyruvate ratio1 Improvement Improvement 1. Pizzamiglio C et al
Slide 18: Napazimone (KL1333) – FALCON study positive interim analysis
Napazimone (KL1333) – FALCON study positive interim analysis Pivotal FALCON Study Interim Futility Analysis WAVE 1 – Fully enrolled Positive outcome achieved, with both primary endpoints passing futility 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) Promising
Slide 19: Executing on high value rare disease pipeline
Executing on high value rare disease pipeline Leniolisib sNDA for 4-11 yo APDS patients – requested Type A meeting APDS Responded to CHMP (EMA) questions – expect March opinion with potential 1H 2026 approval Japan and other regulatory reviews on track for 2026 approvals PIDs Genetic PID and CVID ph
Slide 20: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 21: Positive rolling 12-month financial trends
Positive rolling 12-month financial trends Revenues Operating profit (loss) Operating Cash Flow 376* Amounts in US$ million 53 Amounts in US$ million Amounts in US$ million 362 340 321 51 286 297 48 31 46 26 40 45 15 6 376 1 0 292 311 275 -2 246 252 -9 -14 3Q24 FY24 1Q25 2Q25 3Q25 FY25 3Q24 FY24 1Q2
Slide 22: 2026 financial guidance and long-term capital outlook
2026 financial guidance and long-term capital outlook Revenue and operating expenses (in constant currency): FY 2026 Guidance Notes Total Revenues US$405 - 425 million • 8 - 13% growth • US$60 million incremental R&D investments to advance pipeline Operating Expenses US$330 - 335 million • US$9 mill
Slide 23: Building momentum across commercial, financial and pipeline
Building momentum across commercial, financial and pipeline Strong growth Strategic growth High value Building a leading rare momentum priorities pipeline disease company 2025 revenue ~$376M: Sustained growth of Joenja® (leniolisib) for Growth-oriented • High dbl-digit growth for commercial portfoli
Slide 24: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 25: Appendix
Pharming Group N.V. Appendix
Slide 26: Joenja: Targeting the root cause of APDS to help restore immune balance
Joenja: Targeting the root cause of APDS to help restore immune balance Treatment with Joenja in a randomized, controlled-trial led to: Patient-reported Annual Rate of 45 • Significant improvements in immune dysregulation Infection Days* (e.g, lymph node and splenomegaly reductions) 40 42.4 Days wit
Slide 27: Joenja clinical trial designs
Joenja® clinical trial designs Pivotal Trial - Pivotal Trial - Part 2: Open-label Part 1: Efficacy extension Dose- & Safety study4,5 finding1,2 Evaluation3 Nonrandomized, open-label, Randomized, triple-blinded, Nonrandomized, open-label, dose-escalating placebo-controlled long-term study 31 patients
Slide 28: Joenja Phase 3 co-primary endpoints
Joenja® addresses the underlying cause of APDS to help restore immune balance – Phase 3 co-primary endpoints At 12 weeks Joenja® decreased lymphadenopathy and increased naïve B cells Change from baseline in index nodes* Change from baseline in naïve B cells† Log10-transformed SPD of index lesions Jo
Slide 29: Joenja significantly reduced splenomegaly
Joenja® significantly reduced splenomegaly Secondary endpoint: Significant reductions in at week 12 spleen size by 2D and 3D analysis compared to placebo 27% reduction in 3D spleen volume* • The adjusted mean difference in bidimensional spleen size between Joenja® (n=19) and placebo (n=9) was -13.5
Slide 30: Joenja safety profile
Joenja® safety profile Phase 3 Trial1,2 Open-label Extension Study3 Data cutoff for interim analysis: December 13, 2021 Adverse reactions reported by ≥2 patients treated with Joenja and more frequently than placebo • 32/37 patients Most common AEs n Joenja (n=21) Placebo (n=10) n (%) n (%) reported
Slide 31: Open-label extension interim analysis of days spent with infections and IRT reduction
Open-label extension interim analysis of days spent with infections and IRT reduction Annual Rate of Infection Days* Physician-reported IRT Reduction‡ 45 40 42.4 Of 27 patients receiving IRT: Days with infection per patient (m/N) Baseline† Baseline† 35 39.0 38.5 30 25 Baseline† 26.7 20 Baseline† 22.
Pharming Group — Oppenheimer 36th Annual Healthcare Life Sciences Conference (February 2026) on 6ix