Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — H.C. Wainwright 27th Annual Global Investment Conference 2025
46 slides from Pharming Group Nv's conference presentation, September 2025
Slide 1: H.C. Wainwright 27th Annual Global Investment Conference
Pharming Group N.V. H.C. Wainwright 27th Annual Global Investment Conference September 2025 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 Unique value proposition/positioning Highly specific manufacturing process PIDs Joenja® for APDS PID / CVID > $1B with immune Significant near-term catalysts revenue dysregulation
Slide 4: Vision for the future
Vision for the future Develop a leading global rare disease company with a diverse portfolio and presence in large markets, leveraging proven and efficient clinical development, supply chain, and commercial infrastructure 4
Slide 5: Strong second quarter 2025 performance
Strong second quarter 2025 performance Revenues RUCONEST Joenja Amounts in US$ million 93.2 12.8 74.1 • Total revenues up 26% 11.1 63.0 80.4 • Meaningful operating profit and cash generated from operations 2Q 2024 2Q 2025 • Continued double-digit RUCONEST® revenue growth Adjusted (Non-GAAP) Operatin
Slide 6: RUCONEST for HAE
% Pharming’ RUCONEST® for HAE
Slide 7: RUCONEST (rhC1INH) targets the root cause of HAE across all pathways
RUCONEST® (rhC1INH) Targets the root cause of HAE across all pathways Only recombinant treatment that targets the root cause of HAE by replacing C1-INH Only recombinant treatment that acts at multiple points in the cascades leading to HAE attacks 7
Slide 8: RUCONEST unique value proposition and positioning
RUCONEST® unique value proposition and positioning Type 1, Type 2, and Normal C1-INH HAE patients rely on RUCONEST 97% attacks treated with just 1 dose1 Time of taking 4 hours after 24 hours after RUCONEST 93% acute attacks stopped for at least 3 days2 RUCONEST® mostly used by patients experiencing
Slide 9: RUCONEST strong growth continues in acute HAE market
RUCONEST® strong growth continues in acute HAE market RUCONEST® revenues RUCONEST® U.S. new prescribers • Strong U.S. in-market demand in 2Q 2025 ₋ Continuing to add Amounts in US$ million prescribers and patients 80.4 26 22 24 ₋ New patient enrollments 63.0 20 20 remain high (~90) 15 • Continued ro
Slide 10: Joenja (leniolisib) APDS & PID indications
Joenja® (leniolisib) APDS & PID indications
Slide 11: APDS is a rare primary immunodeficiency (PID)
APDS is a rare primary immunodeficiency (PID) Genetic defect leads to PI3Kδ hyperactivity Hyperactive PI3Kδ results in dysregulated Immune imbalance leads to diverse signs B and T cell development1-3 and symptoms1,4-6 Severe, recurrent, persistent infections • Sinopulmonary p110δ • Herpesvirus (espe
Slide 12: 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. 12
Slide 13: Joenja acceleration in patient growth in 12y+ APDS
Joenja® acceleration in patient growth in 12y+ APDS Joenja® revenues US patients on Joenja® 114 • Acceleration in APDS patients on therapy Amounts in US$ million 12.8 102 ₋ 114 U.S. patients 11.1 96 (+25% vs 2Q24) 93 91 ₋ 1H25 increase exceeded 83 total for 2024 81 • Launched in the U.K. in April •
Slide 14: Joenja near-term growth catalysts – APDS
Joenja® near-term growth catalysts – APDS Reclassification of Pediatric Geographic VUS* patients 2H25 label expansion 1H26 Expansion ongoing • >1400 patients in the US with • FDA filing for 4-11 years • Launched and reimbursed in VUS results pediatrics in July 2025 the UK • June 2025 publication in
Slide 15: Joenja (leniolisib) lifecycle to realize $1Bn+ sales potential
Joenja® (leniolisib) lifecycle to realize $1Bn+ sales potential U.S. patient numbers ONLY shown to illustrate prevalence Geo expansion – UK, EU4, Japan, Australia, Canada + access programs CVID with immune PIDs with immune dysregulation dysregulation linked APDS* to PI3Kδ signaling ~500 U.S. patient
Slide 16: Leniolisib development in PIDs with immune dysregulation
Leniolisib development in PIDs with immune dysregulation: Significant expansion of addressable patient population Patient Population • PID patients with clinical manifestations similar to APDS • Significant unmet clinical need, no approved therapies All PIDs • Prevalence 5-26x APDS Rationale PIDs wi
Slide 17: KL1333 for mtDNA Mitochondrial Disease
KL1333 for mtDNA Mitochondrial Disease
Slide 18: KL1333 for mtDNA-driven primary mitochondrial disease
KL1333 for mtDNA-driven primary mitochondrial disease Aiming for the first disease-modifying treatment KL1333 targets underlying pathology Dysfunctional mitochondria • Normalizes NAD+/NADH ratio and mitochondrial function, with evidence from in vitro data, animal models, Impaired ETC protein Mutatio
Slide 19: KL1333 – FALCON study positive interim analysis
KL1333 – FALCON study positive interim analysis Pivotal FALCON Study Interim Futility Analysis WAVE 1 – Fully enrolled Positive outcome achieved, with both primary 40 patients recruited across six countries (U.S., UK, endpoints passing futility France, Spain, Belgium, Denmark) Promising differences
Slide 20: Outlook
% Pharming Outlook >
Slide 21: 2025 financial guidance and long-term capital outlook
2025 financial guidance and long-term capital outlook Revenue and operating expenses: FY 2025 Guidance Notes Total Revenues US$335 - 350 million 13 - 18% growth Assumes constant currency, Operating Expenses US$304 - 308 million Includes $10.2 million non-recurring Abliva- related transaction and int
Slide 22: Building a leading global rare disease biopharma company
Building a leading global rare disease biopharma company Strong start to Updated High value Significant 2025 2025 guidance pipeline catalysts Strong RUCONEST® Raised revenue Joenja® (leniolisib) for Joenja® for APDS: growth and acceleration guidance to PIDs/CVID with VUSs, pediatric label, of Joenja
Slide 23: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 24: Appendix
Pharming Group N.V. Appendix
Slide 25: Diverse rare disease portfolio and pipeline
Diverse rare disease portfolio and pipeline 25
Slide 26: APDS: Heterogeneous, evolving symptomology can often lead to missed diagnoses
APDS: Heterogeneous, evolving symptomology can often lead to missed diagnoses Timeline of the most common pathologies* seen in APDS1-4 Median age at diagnosis: 12 years (7-year median diagnosis delay) <1 year 3 years 5 years 10.5 years 11.2 years† 18 years (range, 1 month-10 years) (range, 1-6 years
Slide 27: Newly published study expands characterization of APDS
Newly published study expands characterization of APDS VUS patients: Inconclusive genetic test due to insufficient data to determine if variant is disease causing Study uncovered >100 new variants leading to PI3Kδ hyperactivity (GOF variants) Leniolisib restored / improved PI3Kδ signaling defects an
Slide 28: 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) 42.4 Days with i
Slide 29: 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 30: 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 31: 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 32: 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 Joenja (n=21) Placebo (n=10) • 32/37 patients Most common AEs n n (%) n (%) reported
Slide 33: 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.
Slide 34: Pediatric APDS clinical trial results support regulatory filings
Pediatric APDS clinical trial results support regulatory filings Pediatric Phase III trial for children 4-11 years old with APDS Positive topline data announced December 2024 21 patients enrolled in U.S., Europe, and Japan Both co-primary endpoints show improvement consistent with the RCT in adolesc
Slide 35: Joenja development status
Joenja® development status Expanding the addressable patient population Geographic expansion Pediatric expansion Indication expansion (APDS) (APDS) (additional PIDs) Europe – review extended to Jan. 2026 4 to 11 years – Positive data presented PIDs with immune dysregulation Single outstanding CMC re
Slide 36: Leniolisib for PIDs with immune dysregulation – Phase II studies
Leniolisib for PIDs with immune dysregulation – Phase II studies Genetically defined PIDs with immune Common variable immunodeficiency (CVID) dysregulation linked to PI3Kδ with immune dysregulation • Single arm, open-label, dose range-finding (N=12) • Single arm, open-label, dose range-finding (N=20
Slide 37: Primary mitochondrial disease (PMD): Heavy patient burden with no approved therapies
Primary mitochondrial disease (PMD) Heavy patient burden with no approved therapies Presentation and Diagnosis Patients present to their primary care doctor and then often get referred to a neurologist for musculoskeletal issues Either the neurologist or a referral to a metabolic geneticist will res
Slide 38: Dysfunctional mitochondria produce less ATP
Dysfunctional mitochondria produce less ATP Primary Mitochondrial Disease (PMD) Mitochondria, often described as the “powerhouses” Dysfunctional Mitochondria of cells, are crucial for energy production Mutation Impaired ETC protein expression PMD is a group of disorders caused by mutations in mitoch
Slide 39: KL1333 corrects the underlying pathophysiology in PMD
KL1333 corrects the underlying pathophysiology in PMD KL1333 normalizes conversion of NADH to NAD+ via NQO1 Normalizes the NAD+/NADH Ratio Restored Energy Metabolism Electron Transport Chain Complex III Complex IV Mitochondrial Membrane Q Synthase ATP Complex I Complex II X NADH NAD+ ADP + Pi ATP Re
Slide 40: KL1333: Phase 1b demonstrated significant activity vs. placebo
KL1333: Phase 1b demonstrated significant activity vs. placebo The placebo-controlled Phase 1b study demonstrated that KL1333 reduced patients’ fatigue and myopathy after only 10 days, 50 mg/day Fatigue Reduction Muscle Function Improvement Changes from baseline to day 10 Changes from baseline to da
Slide 41: KL1333 pivotal FALCON study
KL1333 pivotal FALCON study Design informed by regulatory and patient advocacy input Study Schematic Regulatory Feedback Study Design Screening Both FDA and EMA accepted Methodology study as registrational • Randomized, double-blind, parallel-group, placebo- FDA said achieving one of the controlled
Slide 42: Significant revenue opportunity for KL1333
Significant revenue opportunity for KL1333 PMD Prevalence 1 per 4,3001 US 78,200; EU4 and UK 76,400 Diagnosed mtDNA 9.6 per 100,0002 US 32,300; EU4 and UK 31,600 FALCON Trial Inclusion Criteria* 51% US 16,500; EU4 and UK 16,100 >30,000 diagnosed mtDNA mitochondrial disease patients addressable in th
Slide 43: Majority of mitochondrial disease patients diagnosed and treated in US Centers of Excellence
Majority of mitochondrial disease patients diagnosed and treated in US Centers of Excellence or academic institutions Mitochondrial Care Network – Centers: www.mitonetwork.org/centers 43
Slide 44: Financial highlights: 2Q 2025 vs 2Q 2024
Financial highlights: 2Q 2025 vs 2Q 2024 Revenues Gross profit OpEx development* RUCONEST Joenja R&D G&A M&S Amounts in US$ million Amounts in US$ million Amounts in US$ million 93.2 12.8 84.2 74.1 70.1 75.2 11.1 66.1 31.0 32.9 80.4 20.5 63.0 15.6 21.6 23.7 2Q 2024 2Q 2025 2Q 2024 2Q 2025 2Q 2024 2Q
Slide 45: Financial highlights: 1H 2025 vs 1H 2024
Financial highlights: 1H 2025 vs 1H 2024 Revenues Gross profit OpEx development* RUCONEST Joenja R&D G&A M&S Amounts in US$ million Amounts in US$ million Amounts in US$ million 172.3 23.3 155.0 153.4 129.7 134.0 20.7 113.3 65.6 63.2 149.0 109.0 43.0 30.7 40.1 44.8 1H 2024 1H 2025 1H 2024 1H 2025 1
Slide 46: Financial highlights: FY 2024 vs FY 2023
Financial highlights: FY 2024 vs FY 2023 Revenues Gross profit OpEx development RUCONEST Joenja R&D G&A M&S Amounts in US$ million Amounts in US$ million Amounts in US$ million 261.8 297.2 220.1 272.6 245.3 45.0 248.8 18.2 118.8 124.0 227.1 252.2 70.7 55.9 68.9 83.1 FY 2023 FY 2024 FY 2023 FY 2024
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