Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Investor Presentation (November 2026)
40 slides from Pharming Group Nv's investor presentation, November 2026
Slide 1: Corporate Overview November 2026
PHARMING GROUP N.V. Corporate Overview NASDAQ: PHAR November 2026 EURONEXT Amsterdam: PHARM 1
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: Strong commercial foundation with high-value pipeline
Strong commercial foundation with high-value pipeline HAE PIDs PMD Hereditary Angioedema Primary Immunodeficiencies Primary Mitochondrial with immune dysregulation Disease RUCONEST® Joenja® (leniolisib) napazimone (KL1333) APPROVED for acute HAE APPROVED for APDS CLINICAL • Cornerstone on-demand • A
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: Progress on near-term milestones
Progress on near-term milestones RUCONEST for HAE Revenues returned to growth and demand stabilized Geographic expansion – Germany and Japan launches Joenja for APDS Pediatric label expansion (U.S.) Joenja (leniolisib) Positive genetic PIDs Phase II readout (October) for additional PIDs Phase II tri
Slide 6: Diverse rare disease portfolio and pipeline
Diverse rare disease portfolio and pipeline PRECLINICAL PHASE I PHASE II PHASE III / REGULATORY COMMERCIAL REGISTRATIONAL SUBMISSION STUDY Hereditary angioedema (HAE) RUCONEST® acute HAE Primary immunodeficiencies (PIDs) with immune dysregulation Joenja® (leniolisib) activated phosphoinositide 3-kin
Slide 7: RUCONEST for HAE
RUCONEST® for HAE @ Pharming’
Slide 8: RUCONEST Q3 update – returned to revenue growth
RUCONEST Q3 update – returned to revenue growth RUCONEST revenue RUCONEST quarterly revenue (global) (global) 93.8 Revenue +14% vs. Q3 2025 93.8 82.2 80.4 US demand returned to PY level 72.3 Amounts in US$ million 68.6 in volume terms Amounts in US$ million 82.2 +14% 58.4 Early seasonal inventory in
Slide 9: RUCONEST – resilient market position in high-burden HAE
RUCONEST – resilient market position in high-burden HAE Differentiated value proposition Overwhelming majority of • Targets the root cause of HAE across all pathways • Median time to complete attack resolution of 4.5hrs RUCONEST patients remain on therapy one year into oral competitor launch Mostly
Slide 10: RUCONEST – rapid, reliable, complete attack resolution
RUCONEST – rapid, reliable, complete attack resolution Stopped 97% of acute attacks 93% Median time to complete attack needed just one dose of attacks for at resolution with of RUCONEST1 least 3 days2 RUCONEST3 1 3 4.5 DOSE DAYS HOURS References: 1. RUCONEST. Prescribing information. Pharming Health
Slide 11: RUCONEST Post Hoc Analysis: Rapid Attack Relief & Resolution
RUCONEST® Post Hoc Analysis: Rapid Attack Relief & Resolution Key takeaways* • Severe to very severe baseline attack severity: All patients in rhC1-INH and saline trial arms had severe attacks at baseline (12-23% severe and 77-88% very severe attacks). • Rapid onset of symptom relief: Median time to
Slide 12: rhC1-INH shorter time to complete resolution vs sebetralstat
rhC1-INH demonstrated significantly shorter time to complete resolution compared to sebetralstat Time to Complete Resolution of rhC1-INH vs Sebetralstat Patients on rhC1-INH were 93.4% 4.5x % of HAE Attacks Resolved rhC1-INH more likely 49.5% to achieve complete symptom Sebetralstat resolution than
Slide 13: Joenja (leniolisib) APDS & PID indications
Joenja® (leniolisib) APDS & PID indications 13
Slide 14: Joenja Q3 update – strong global momentum in APDS
Joenja Q3 update – strong global momentum in APDS Continued U.S. growth and international launches Joenja revenue (global) U.S. patients on Joenja Revenue +58% U.S. EU & RoW 136 132 127 136 patients on therapy in US 120 (+17% vs 3Q25) 23.8 114 116 Amounts in US$ million 7.9 102 Significant increase
Slide 15: Joenja for APDS – pediatric label expansion
Joenja for APDS – pediatric label expansion U.S. FDA approval received September 11, 2026 >60 Eligible patients (ages 4–11) identified in the U.S. APPROVED UNDER FDA REVIEW Children aged 4 to 11, at least 27 kg Children aged 4 to 11, 13 kg to less than 27 kg • sNDA approved – September 11, 2026 • Se
Slide 16: Joenja expansion from APDS foundation into broad immunology opportunity
Joenja expansion from APDS foundation into broad blockbuster immunology opportunity Broad PIDs/CVID Opportunity*** International APDS Expansion • Genetic PIDs with U.S. APDS immune dysregulation Expansion • Focused geo- (>2,500 U.S. patients) Established APDS expansion that Commercial Base • Pediatr
Slide 17: 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 the Immune dysregulation pathology immune system Lymphoproliferation p110δ PI3Kδ p85ɑ AKT/PKB Autoimmunity FOXO mTOR GI Disease Cell trafficking Cell Growth Cell prolifer
Slide 18: PI3Kδ inhibition validated in APDS
PI3Kδ inhibition validated in APDS Sustained, durable efficacy Lymph-node and spleen volume reduced, sustained through multi-year OLE† Joenja (leniolisib) Established safety and tolerability* APPROVED for APDS Primarily mild-to-moderate adverse events; no treatment- related SAEs with up to 7 years o
Slide 19: Joenja addresses the underlying cause of APDS – Phase III endpoints
Joenja® addresses the underlying cause of APDS to help restore immune balance – Phase III 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
Slide 20: Joenja significantly reduced splenomegaly
Joenja® significantly reduced splenomegaly Secondary endpoint: Significant Secondary measure: spleen volume scan results of reductions in spleen size by 2D and actual patient illustrate average improvement 3D analysis compared to placebo documented for patients taking Joenja® • The adjusted mean dif
Slide 21: Reports of Joenja changing patients' lives
Reports of Joenja® changing patients’ lives 24-year-old male with APDS Since starting Joenja Before study enrollment whose progress was followed treatment in the Joenja® open-label extension study for 6 years Infections and • Experienced fatigue from IRT • Stopped IRT infusions and treatment infusio
Slide 22: Leniolisib opportunity across primary immunodeficiencies
Leniolisib opportunity: Targeting shared PI3Kδ-driven immune dysregulation across primary immunodeficiencies (PIDs) E X P A N D I N G T H E A D D R E S S A BL E M A R K E T ONE MECHANISM: PI3Kδ Shared clinical manifestations across all three populations Up to 26× the APDS Genetic PIDs + CVID populat
Slide 23: Leniolisib Phase II trials in genetic PIDs and CVID
Leniolisib Phase II trials in genetic PIDs and CVID Positive data presented at ESID 2026 N = 13 Topline results expected November N = 20 Genetic PIDs with immune dysregulation CVID with immune dysregulation Open-label, single-arm basket trial at NIH Open-label, single-arm, multi-center dose-confirma
Slide 24: Genetic PIDs study: well-tolerated, improved immune dysregulation measures
Genetic PIDs study: leniolisib was well-tolerated and improved multiple measures of immune dysregulation First clinical data for leniolisib in a larger group of PID participants beyond APDS Improvement across objective and clinician-reported measures, consistent with PI3Kδ mechanism LUNG FUNCTION –
Slide 25: Leniolisib reduced lymphoproliferation
Leniolisib reduced lymphoproliferation U.S. patients on Joenja SPLEEN VOLUME INDEX LYMPH NODES −26.4% −33.7% 132 Revenue +40% 127 17.9 120 114 116 132 patients on therapy in US mean change from mean change from baseline at end of 102 baseline in SPD of index (+16% vs 2Q25) treatment (SD 16.2;+150% 9
Slide 26: Trends toward improvement in lung and liver end organ disease
Trends toward improvement in lung and liver end organ disease LUNG FUNCTION – GAS EXCHANGE LUNG IMAGING (CT) +8.0% 4 of 5 mean increase in DLCO % predicted participants with a baseline ILD (SD 8.9) in participants with DLCO composite scoring improved <80% at baseline (n=7) LIVER (FIBROSCAN) 2 of 3 C
Slide 27: Safety: well-tolerated, with no new safety signals
Safety: well-tolerated, with no new safety signals TREATMENT-RELATED AES: ALL GRADE 1 No new safety signals Preferred term n (%) Findings consistent with the known disease profile; infections (7 participants; 9 events) were the most common AE type, followed by gastrointestinal AEs Alopecia* 4 (30.8)
Slide 28: Leniolisib Phase II trials – CVID results expected in November
Leniolisib Phase II trials – CVID results expected in November Positive data presented at ESID 2026 N = 13 Topline results expected November N = 20 Genetic PIDs with immune dysregulation CVID with immune dysregulation Open-label, single-arm basket trial at NIH Open-label, single-arm, multi-center do
Slide 29: Leniolisib expanded access in CVID derisks Phase II readout
Leniolisib expanded access in CVID derisks Improvement from baseline to last treatment visit No change or no progression from baseline upcoming Phase II readout to last treatment visit - Manifestation was not reported at baseline Clinician-reported patient outcomes across key clinical manifestations
Slide 30: Napazimone (KL1333) mtDNA Mitochondrial Disease
Napazimone (KL1333) mtDNA Mitochondrial Disease 30
Slide 31: Napazimone (KL1333) for mtDNA-driven primary mitochondrial disease
Napazimone (KL1333) for mtDNA-driven primary mitochondrial disease Aiming for the first disease-modifying treatment DYSFUNCTIONAL MITOCHONDRIA Targets the underlying pathology Normalizes the NAD+/NADH ratio and mitochondrial function, with evidence Impaired ETC protein from in vitro data, animal mod
Slide 32: Phase Ia/b study: 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 1 1 1 Changes from baseline to day 10 Changes from baseline to day 10 Lactate: Pyruvate ratio Improvement Improvement 1. Pizzamiglio C et
Slide 33: Napazimone – 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 • 40 patients recruited across six countries primary endpoints passing futility (U.S., UK, France, Spain, Belgium, Denmark) Promisi
Slide 34: Financials and Outlook
34 Financials and Outlook @ Pharming’
Slide 35: Q3 2026 financial and commercial update
Q3 2026 financial and commercial update Revenues RUCONEST stabilization and return to growth RUCONEST Joenja • U.S. demand stabilized with vial volume returning to PY levels 117.6 • 14% revenue growth Amounts in US$ million 97.3 +21% 23.8 15.1 Joenja growth momentum • 58% revenue growth, geographic
Slide 36: Financial highlights: Q3 2026 vs Q3 2025
Financial highlights: Q3 2026 vs Q3 2025 Revenues Adjusted (non-GAAP) Cash flow from operations RUCONEST Joenja operating profit* 117.6 Amounts in US$ million Amounts in US$ million Amounts in US$ million 97.3 35.0 23.8 32.0 32.4 15.1 +21% 16.0 93.8 82.2 Q3 2025 Q3 2026 Q3 2025 Q3 2026 Q3 2025 Q3 20
Slide 37: Financial highlights: 9M 2026 vs 9M 2025
Financial highlights: 9M 2026 vs 9M 2025 Revenues Adjusted (non-GAAP) Cash flow from operations RUCONEST Joenja operating profit* 280.2 Amounts in US$ million Amounts in US$ million Amounts in US$ million 269.6 44.0 38.4 55.7 38.0 +4% 29.7 24.7 231.2 224.5 9M 2025 9M 2026 9M 2025 9M 2026 9M 2025 9M
Slide 38: 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 • 1 - 4% growth Total Revenues US$380 - 390 million • COGS: ~11% • 0 - 1% growth Operating Expenses US$310 - 315 million • +US$35 million R&D to advance pipeline Signif
Slide 39: Advancing Pharming as a leading global rare disease company
Advancing Pharming as a leading global rare disease company Strong commercial Significant growth Building a leading rare foundation opportunities disease company Joenja - commercial RUCONEST • Geographic, pediatric and • Disciplined operating model prevalence expansion • Durable cash engine • Scalab
Slide 40: Thank you!
WWW.PHARMING.COM The Netherlands Pharming Group Thank you! Vondellaan 47 2332 AA Leiden The Netherlands United States Pharming Healthcare, Inc. 10 Independence Blvd Suite 401, Joenja® and RUCONEST® are registered trademarks owned by or licensed to Pharming Group N.V. Warren or its affiliates. Other
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