Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Investor Presentation (October 2026)
41 slides from Pharming Group Nv's investor presentation, October 2026
Slide 1: Corporate Overview October 2026
PHARMING GROUP N.V. Corporate Overview NASDAQ: PHAR October 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: RUCONEST for HAE
RUCONEST® for HAE @ Pharming’
Slide 6: RUCONEST – durable market position in high-burden HAE
RUCONEST – durable market position in high-burden HAE Differentiated value proposition • Targets the root cause of HAE across all pathways • Efficacy and reliability of IV self administration Overwhelming majority of Mostly used by patients experiencing more RUCONEST patients remain severe / frequen
Slide 7: 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 8: RUCONEST Q2 performance
RUCONEST Q2 performance Improvement in Financial performance Resilient patient base underlying demand Revenue: $72.3M 84 new patient Dyn amic Patients -10% vs. Q2 2025 enrollments Active +24% vs. Q1 2026 RUCONEST New Starts Patients Switches Restarts Drop Offs Stabilization with return to 93% revenu
Slide 9: RUCONEST Post Hoc Analysis: Rapid Attack Relief & Resolution
RUCONEST® Post Hoc Analysis: Rapid Attack Relief & Resolution Key takeaways* • Severe to very severe baseline attack severity: All patients in rhC1-INH and saline trial arms had severe attacks at baseline (12-23% severe and 77-88% very severe attacks). • Rapid onset of symptom relief: Median time to
Slide 10: rhC1-INH vs sebetralstat time to complete resolution
rhC1-INH demonstrated significantly shorter time to complete resolution compared to sebetralstat Time to Complete Resolution of rhC1-INH vs Sebetralstat Patients on rhC1-INH were 93.4% 4.5x % of HAE Attacks Resolved rhC1-INH more likely 49.5% to achieve complete symptom Sebetralstat resolution than
Slide 11: Joenja (leniolisib) APDS & PID indications
Joenja® (leniolisib) APDS & PID indications 11
Slide 12: Joenja Q2 update – strong global momentum in APDS
Joenja Q2 update – strong global momentum in APDS ahead of geographic and pediatric expansion expected this year Joenja revenue (global) U.S. patients on Joenja U.S. EU & RoW 132 Revenue +40% 127 17.9 120 114 116 132 patients on therapy in US Amounts in US$ million 2.5 12.8 +150% 102 (+16% vs 2Q25)
Slide 13: Joenja for APDS - pediatric label expansion
Joenja® for APDS - pediatric label expansion U.S. FDA approval received September 11, 2026 60 eligible patients identified in the U.S. Children aged 4 to 11, at least 27 kg Children aged 4 to 11, 13 kg to less than 27 kg PDUFA date – October 24, 2026 Separate sNDA filed July 30, 2026 sNDA approved –
Slide 14: Joenja expansion from APDS 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 15: 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 16: 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 17: Joenja Phase III co-primary 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 18: 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 19: 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 20: Leniolisib opportunity across primary immunodeficiencies
Leniolisib opportunity: Targeting shared PI3Kδ-driven immune dysregulation across primary immunodeficiencies (PIDs) Two Phase II trials Genetic PIDs with immune dysregulation APPROVED Joenja (leniolisib) Expanding the addressable market linked to PI3Kẟ APDS CVID with immune dysregulation Shared clin
Slide 21: 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 22: Genetic PIDs study: well-tolerated, improved immune dysregulation
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 23: 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 24: 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 25: 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 26: 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 27: Napazimone (KL1333) mtDNA Mitochondrial Disease
Napazimone (KL1333) mtDNA Mitochondrial Disease 27
Slide 28: 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 Napazimone (KL1333) targets underlying pathology • Normalizes NAD+/NADH ratio and mitochondrial function, with Impaired ETC protein Mutation evidence from in
Slide 29: Napazimone Phase Ia/b 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 30: 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 31: Financials and Outlook
31 Financials and Outlook @ Pharming’
Slide 32: 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 • 0 - 5% growth Total Revenues US$375 - 395 million • COGS: ~11% • 1 – 3% growth • +US$40 million R&D to advance pipeline Operating Expenses US$315 - 320 million • -US$
Slide 33: 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 resilience – • Geographic, pediatric and • More disciplined operating model durable cash engine prevalence
Slide 34: 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, Warren NJ 07059 United States 34
Slide 35: Appendix
35 Appendix @ Pharming’
Slide 36: Joenja: Targeting the root cause of APDS
Joenja®: Targeting the root cause of APDS to help restore immune balance Treatment with Joenja® in a randomized, Patient-reported Annual Rate of Infection Days* controlled-trial led to: • Significant improvements in immune dysregulation 45 42.4 Days with infection per patient (m/N) (e.g, lymph node
Slide 37: Joenja clinical trial designs
Joenja® clinical trial designs Pivotal Trial - Part 1: Pivotal Trial - Part 2: 4,5 1,2 3 Open-label extension study Dose-finding Efficacy & Safety Evaluation Nonrandomized, open-label, Randomized, triple-blinded, Nonrandomized, open-label, long- dose-escalating placebo-controlled term study 6 patien
Slide 38: Joenja Phase III co-primary 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 39: 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 40: Joenja safety profile
Joenja® safety profile Phase III Trial1,2 Open-label Extension Study3 Data cutoff for interim analysis: December 13, 2021 Adverse reactions reported by ≥2 patients treated with Joenja and • 32/37 patients Most common AEs n more frequently than placebo reported ≥1 AE Upper respiratory tract infection
Slide 41: Open-label extension interim analysis of infection days and IRT reduction
Open-label extension interim analysis of days spent with infections and IRT reduction 37% of patients receiving IRT reduced or discontinued dose Patient-reported Annual Rate of Infection Days* Physician-reported IRT Reduction‡ Of 27 patients receiving IRT: Days with infection per patient (m/N) 45 42
Pharming Group — Investor Presentation (October 2026) on 6ix