Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Oppenheimer Movers in Rare Disease Summit 2025
38 slides from Pharming Group Nv's conference presentation, Dec 11, 2025
Slide 1: Pharming Group N.V. Oppenheimer Movers in Rare Disease Summit
Pharming Group N.V. Oppenheimer Movers in Rare Disease Summit December 11, 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 Leniolisib for PIDs / CVID > $1B with immune Significant near-term catalysts revenu
Slide 4: Vision for the future
Vision for the future Develop a leading global rare disease company with a diverse portfolio and presence in large markets, leveraging proven and efficient clinical development, supply chain, and commercial infrastructure 4
Slide 5: Strong third quarter 2025 performance
Strong third quarter 2025 performance Revenues RUCONEST Joenja Amounts in US$ million 97.3 • Total revenues up 30% 15.1 74.8 11.2 • Significant growth in operating profit 82.2 63.6 • Significant cash flow from operations – US$32 million 3Q 2024 3Q 2025 • High double-digit Joenja® and RUCONEST® reven
Slide 6: Executing on high value rare disease pipeline
Executing on high value rare disease pipeline Leniolisib sNDA for 4-11 yo APDS patients – FDA Priority Review, Jan. 26 PDUFA APDS Japan, EMA and other regulatory reviews on track for 2026 approvals PIDs with immune Genetic PID and CVID Phase II POC trials on track for 2H 2026 read-outs dysregulation
Slide 7: Diverse rare disease portfolio and pipeline
Diverse rare disease portfolio and pipeline 7
Slide 8: RUCONEST for HAE
% Pharming’ RUCONEST® for HAE
Slide 9: RUCONEST unique value proposition and positioning
RUCONEST® unique value proposition and positioning Type 1, Type 2, and Normal C1-INH HAE patients rely on RUCONEST® • Only recombinant C1-INH protein replacement therapy • Targets the root cause of HAE across all pathways • IV administration – rapid onset, high dose Time of taking 4 hours after 24 h
Slide 10: RUCONEST strong growth continues in acute HAE market
RUCONEST® strong growth continues in acute HAE market RUCONEST® revenues RUCONEST® U.S. new prescribers • Strong U.S. in-market demand ₋ Continuing to add prescribers and patients Amounts in US$ million 82.2 26 ₋ New patient enrollments 24 remain high (~60) 22 22 63.6 20 20 ₋ Increase in more severe
Slide 11: Joenja (leniolisib) APDS & PID indications
Joenja® (leniolisib) APDS & PID indications
Slide 12: 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 13: 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. 13
Slide 14: Joenja strong double-digit growth in 12y+ APDS segment
Joenja® strong double-digit growth in 12y+ APDS segment Joenja® revenues US patients on Joenja® 116 • Strong YoY increase in APDS 114 patients on therapy in the US Amounts in US$ million 15.1 102 ➢ 116 patients (+25% vs 3Q24) 96 11.2 91 93 • Acceleration of U.S. APDS patient 83 identification ➢ +13
Slide 15: Joenja 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 >13,000 U.S. pati
Slide 16: Joenja APDS pediatric label expansion – go to market strategy
Joenja® APDS pediatric label expansion – go to market strategy Children 4-11 years old with APDS FDA Priority Review with PDUFA date of Jan 31, 2026* FDA filing based on Phase III data consistent with the improvements and safety seen in the previously reported randomized controlled trial in adolesce
Slide 17: Activities surrounding expanded APDS prevalence
Activities surrounding expanded APDS prevalence Findings >100 new PI3Kδ gain of function (GOF) variants identified in Cell paper Carriers of these variants were found in population databases with prevalence up to 100X higher than current APDS estimates Associated patient phenotypes more diverse than
Slide 18: 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 19: Three PID 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 Additional PIDs linked to PI3Kδ CVID w/immune dysregulation Prevalence 1.5 7.5 39 per million population Genetic Diagnosis Yes (PIK3CD, PIK3R1) Yes
Slide 20: KL1333 for mtDNA Mitochondrial Disease
KL1333 for mtDNA Mitochondrial Disease
Slide 21: 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 22: KL1333 Phase 1b demonstrated significant activity vs. placebo
KL1333 Phase 1b demonstrated significant activity vs. placebo Fatigue Reduction Muscle Function Improvement Changes from baseline to day 10 Changes from baseline to day 10 KL1333 showed efficacy in patients diagnosed with mtDNA PMD after 10 days using 50 mg/day • Fatigue reduction (NeuroQoL fatigue
Slide 23: 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 endpoints passing futility 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) Promising differences
Slide 24: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 25: Financial highlights: 3Q 2025 vs 3Q 2024
Financial highlights: 3Q 2025 vs 3Q 2024 Revenues Adjusted (Non-GAAP) Cash flow from operations RUCONEST Joenja Operating profit (loss)* Amounts in US$ million Amounts in US$ million Amounts in US$ million 97.3 15.1 32.0 74.8 11.2 16.0 82.2 9.7 63.6 4.1 3Q 2024 3Q 2025 3Q 2024 3Q 2025 3Q 2024 3Q 202
Slide 26: Financial highlights: 9M 2025 vs 9M 2024
Financial highlights: 9M 2025 vs 9M 2024 Revenues Adjusted (Non-GAAP) Cash flow from operations RUCONEST Joenja Operating profit (loss)* Amounts in US$ million Amounts in US$ million Amounts in US$ million 269.6 44.0 38.4 29.7 204.5 31.9 231.2 172.6 (11.1) (15.3) 9M 2024 9M 2025 9M 2024 9M 2025 9M
Slide 27: 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$365 - 375 million 23 - 26% growth • Assumes constant currency Operating Expenses • Includes $10.2 million non-recurring Abliva- US$304 - 308 million related transaction and
Slide 28: Driving strong commercial, financial and pipeline momentum
Driving strong commercial, financial and pipeline momentum Strong Q3 growth Upgraded High value Building a leading momentum financial outlook pipeline rare disease co. High dbl-digit growth for Raised 2025 revenue Joenja® (leniolisib) for Proven commercial RUCONEST® and Joenja® guidance to $365-375M
Slide 29: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 30: Appendix
Pharming Group N.V. Appendix
Slide 31: 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 32: 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 33: 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 34: 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 35: 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 36: 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 37: 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 38: 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
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