Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — 25th Annual Needham Virtual Healthcare Conference (April 2026)
27 slides from Pharming Group Nv's conference presentation, Apr 13, 2026
Slide 1: Pharming Group N.V. at 25th Annual Needham Virtual Healthcare Conference
Pharming Group N.V. 25th Annual Needham Virtual Healthcare Conference April 13, 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 > $1B leniolisib for PIDs / CVID reven
Slide 4: Vision for the future
Vision for the future Develop a leading global rare disease company with a diverse portfolio and presence in large markets, leveraging proven and efficient clinical development, supply chain, and commercial infrastructure 4
Slide 5: Strong 2025 performance
Strong 2025 performance Revenue growth and profitability inflection Revenues RUCONEST® Joenja® Amounts in US$ million 376 297 58 • Total revenues up 27% – exceeding guidance 45 • Continued growth of RUCONEST® and acceleration in 318 253 Joenja® APDS uptake 2024 2025 • Strong revenue growth and disci
Slide 6: 2026 Outlook – financial, commercial and pipeline progress
2026 Outlook – financial, commercial and pipeline progress Revenue guidance – US$405 million - US$425 million (8% to 13% growth) Operating expense guidance – US$330 million - US$335 million (6% to 8% growth) Joenja® APDS – geographic and pediatric label expansion ✓ Europe (12+) – CHMP positive opini
Slide 7: 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 8: RUCONEST® for HAE
% Pharming’ RUCONEST® for HAE
Slide 9: 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 Mostly used by pati
Slide 10: Indirect Treatment Comparison of rhC1-INH and sebetralstat for HAE on-demand therapy
Indirect Treatment Comparison (ITC) of rhC1-INH and sebetralstat for HAE on-demand therapy 10
Slide 11: rhC1-INH demonstrated significantly shorter time to complete resolution compared to sebetralstat
rhC1-INH demonstrated significantly shorter time to complete resolution compared to sebetralstat Time to Complete Resolution of rhC1-INH vs Sebetralstat 93.4% rhC1-INH Patients on rhC1-INH were % of HAE Attacks Resolved 4.5x more likely 49.5% to achieve complete symptom resolution than sebetralstat*
Slide 12: Joenja® (leniolisib) APDS & PID indications
Joenja® (leniolisib) APDS & PID indications
Slide 13: PI3Kδ is a master regulator of the immune system
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 14: PIDs with immune dysregulation under the influence of PI3K
PIDs with immune dysregulation under the influence of PI3K APDS Genetic PIDs Linked CVID With Immune (PIK3CD, PIK3R1) to PI3K Signaling Dysregulation 1 2 3 ✓ Joenja® (leniolisib) 14
Slide 15: 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. 15
Slide 16: 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 Genetic PIDs with dysregulation immune dysregulation APDS* linked to PI3Kδ >13,000 U.S. patients ~500 U.
Slide 17: Joenja® (leniolisib) – near-term APDS regulatory milestones
Joenja® (leniolisib) – near-term APDS regulatory milestones Pediatric label expansion – sNDA for patients aged 4-11 U.S. • FDA Type A meeting held March 26, 2026, awaiting meeting minutes MAA for patients aged 12+ Europe ✓ CHMP (EMA) positive opinion March 2026 • Potential EC approval Q2 ‘26 NDA for
Slide 18: 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 19: 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 20: Napazimone (KL1333) mtDNA Mitochondrial Disease
Napazimone (KL1333) mtDNA Mitochondrial Disease
Slide 21: 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 22: Phase Ia/b study of napazimone (KL1333) showed 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 23: 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 24: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 25: 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, with quarterly fluctuations • US$60 million incremental R&D investments to advance pipeline Operating Expenses US$
Slide 26: 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 RUCONEST® commercial portfolio P
Slide 27: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Pharming Group — 25th Annual Needham Virtual Healthcare Conference (April 2026) on 6ix