Pharming Group NV · EURONEXT:PHARM
Pharming Group NV — Conference Presentation (January 2026)
36 slides from Pharming Group NV's conference presentation, Jan 12, 2026
Slide 1: Pharming Group N.V. Corporate Overview
Pharming Group N.V. Corporate Overview January 12, 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 Differentiated value proposition Highly specific manufacturing process PIDs Joenja® for APDS Leniolisib for PIDs / CVID > $1B with immune Significant near-term catalysts revenue dy
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 commercial and financial momentum
Strong commercial and financial momentum • Announced preliminary 2025 revenues* of $376M (+ 27%) – above latest guidance Revenues • Results reflects continued growth of RUCONEST® and acceleration in Joenja® APDS uptake Amounts in US$ million 376.0 • Significant operating profit $30M and operating ca
Slide 6: Executing on high value rare disease pipeline
Executing on high value rare disease pipeline Leniolisib sNDA for 4-11 yo APDS patients – FDA Priority Review, Jan. 26 PDUFA APDS Japan, EMA and other regulatory reviews on track for 2026 approvals PIDs with immune Genetic PID and CVID Phase II POC trials on track for 2H 2026 read-outs dysregulation
Slide 7: Diverse rare disease portfolio and pipeline
Diverse rare disease portfolio and pipeline 7
Slide 8: RUCONEST for HAE
% Pharming’ RUCONEST® for HAE
Slide 9: RUCONEST poised to remain a cornerstone on-demand treatment for HAE
RUCONEST® poised to remain a cornerstone on-demand treatment for difficult to treat HAE patients Differentiated value proposition • Only recombinant C1-INH protein replacement therapy • Targets the root cause of HAE across all pathways • IV administration – rapid onset, high dose Unique patient popu
Slide 10: RUCONEST Q3 update – resilient demand in acute HAE
RUCONEST® Q3 update – resilient demand in acute HAE RUCONEST® revenues RUCONEST® U.S. new prescribers • Strong U.S. in-market demand ₋ Continuing to add prescribers and patients Amounts in US$ 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 Q3 update – strong double-digit growth in 12y+ APDS
Joenja® Q3 update – strong double-digit growth in 12y+ APDS Joenja® revenues US patients on Joenja® • Strong YoY increase in APDS patients 116 on therapy in the US 114 ➢ 116 patients (+25% vs 3Q24) Amounts in US$ million 15.1 102 • Acceleration of U.S. APDS patient 96 identification 11.2 91 93 ➢ +13
Slide 15: 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 PIDs with immune dysregulation dysregulation linked APDS* to PI3Kδ signaling >13,000 U.S. patients ~500
Slide 16: Joenja APDS pediatric label expansion
Joenja® APDS pediatric label expansion 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 adolescent and adult APDS patien
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: Three primary immunodeficiency with immune dysregulation indications
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 19: Leniolisib compassionate use experience in PID/CVID
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 • Pat
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: Growth acceleration across the commercial portfolio
Growth acceleration across the commercial portfolio Revenues (US$ million) 376 297 Total revenues in $* millions 45 245 18 376 253 212 227 192 199 206 (Re-acquisition 160 from Valeant Dec. 2016) 101 18 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 RUCONEST® Joenja® Total • From FY 2016 – FY 2020
Slide 26: Positive rolling 12-month financial trends
Positive rolling 12-month financial trends Revenues Operating profit (loss) Operating Cash Flow 376* Amounts in US$ million 53 Amounts in US$ million Amounts in US$ million 362 340 321 51 286 297 48 31 46 26 40 45 15 6 376 1 0 292 311 275 -2 246 252 -9 -14 3Q24 FY24 1Q25 2Q25 3Q25 FY25 3Q24 FY24 1Q2
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 FY 2025 Results Notes Total Revenues US$365 - 375 million US$376 million • Preliminary, unaudited Operating Expenses US$304 - 308 million • Includes $10.2 million non-recurring Abliva- related tran
Slide 28: Building momentum across commercial, financial and pipeline
Building momentum across commercial, financial and pipeline Strong growth Strategic growth High value Building a leading momentum priorities pipeline rare disease co. 2025 revenue ~$376M Sustained growth of Joenja® (leniolisib) for Growth-oriented vs. $365-375M guidance commercial portfolio PIDs/CVI
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 infection days 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.
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