Slides
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1 Third quarter 2026 financial results NASDAQ: PHAR EURONEXT Amsterdam: PHARM OCTOBER 27, 2026
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2 2 Kenneth Lynard Interim Co-CEO & Chief Financial Officer Co-CEO & CFO Introduction
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3 Interim Co-CEO & Chief Commercial Officer Interim Co-CEO & Chief Financial Officer Chief Medical Officer Leverne Marsh Kenneth Lynard Anurag Relan, MD Speakers
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4 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 results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as “aim”, “ambition”, ‘‘anticipate”, ‘‘believe”, ‘‘could”, ‘‘estimate”, ‘‘expect”, ‘‘goals”, ‘‘intend’’, ‘‘may”,“milestones”, ‘‘objectives”, ‘‘outlook”, ‘‘plan’’, ‘‘probably”, ‘‘project”, ‘‘risks”, “schedule”, ‘‘seek”, ‘‘should”, ‘‘target”, ‘‘will” and similar terms and phrases. Examplesof forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments.In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2025 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2025, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby.All forward-looking statements contained in this presentation are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this presentation and are based on information available to Pharming as of the date of this presentation. Pharming does not undertake any obligation to publicly update or revise any forward- looking statement as a result of new information, future events or other information. Forward-looking statements
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5 • APDS: Geographic, pediatric and prevalence1 expansion • PIDs / CVID: Two Phase II studies – readouts Oct and Nov • Cornerstone on-demand therapy for high-burden HAE patients • Returned to growth in Q3 2026 • mtDNA mitochondrial disease: FALCON pivotal study with positive futility analysis (readout H1 2028) Strong commercial foundation with high-value pipeline HAE Hereditary Angioedema RUCONEST® Joenja® (leniolisib) PIDs Primary Immunodeficiencies with immune dysregulation napazimone (KL1333) PMD Primary Mitochondrial Disease These product candidates are under investigation, and their safety and efficacy have not been established. There is no guarantee that these products will receive health authority approval or become commercially available for the uses being investigated. HAE: Hereditary Angioedema, PIDs: Primary Immunodeficiencies, PMD: Primary Mitochondrial Disease, CVID: Common Variable Immunodeficiency 1. Walsh et al., Scalable generation and functional classification of genetic variants in inborn errors of immunity to accelerate clinical diagnosis and treatment, Cell (2025), https://doi.org/10.1016/j.cell.2025.05.037 >$1B revenue potential>$1B revenue potentialDurable cash flow CLINICAL APPROVED for acute HAE for APDSAPPROVED Tightened 2026 Revenue guidance US$380–390M
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6 Q3 2026 financial and commercial update RUCONEST stabilization and return to growth • U.S. demand stabilized with vial volume returning to PY levels • 14% revenue growth Joenja growth momentum • 58% revenue growth, geographic expansion key growth driver • Continued strong YoY U.S. patient growth 82.2 93.8 15.1 23.8 Amounts in US$ million Revenues RUCONEST Joenja 117.6 97.3 Full-year 2026 financial guidance • Revenue – US$380-390M (vs. prior US$375-395M) • Operating Expenses – US$310-315M (lowered by $5M) Q3 2025 Q3 2026 +21%
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7 Progress on near-term milestones Joenja (leniolisib) for additional PIDs Positive genetic PIDs Phase II readout (October) RUCONEST for HAE Revenues returned to growth and demand stabilized Joenja for APDS Geographic expansion – Germany and Japan launches Pediatric label expansion (U.S.) Phase II trial readout for broader CVID population – November 2026
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8 8 Leverne Marsh Interim Co-CEO & Chief Commercial Officer Commercial update Co-CEO & CCO
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9 RUCONEST Q3 update – returned to revenue growth 82.2 93.8 3Q 2025 3Q 2026 Amounts in US$ million RUCONEST revenue (global) Revenue +14% vs. Q3 2025 US demand returned to PY level in volume terms Early seasonal inventory increase contributed 9% of quarterly revenue growth Added 43 new enrollments, 14 new prescribers +14% 68.6 80.4 82.2 58.4 72.3 93.8 Q1 Q2 Q3 Amounts in US$ million RUCONEST quarterly revenue (global) 2025 2026
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10 RUCONEST – resilient market position in high-burden HAE Differentiated value proposition • Targets the root cause of HAE across all pathways • Median time to complete attack resolution of 4.5hrs Mostly used by patients with more severe / frequent attacks • Where reliability, rapid onset and confidence matter most Highly specific manufacturing process • Limits the potential for direct biosimilar competition Overwhelming majority of RUCONEST patients remain on therapy one year into oral competitor launch Particularly strong retention in high-burden patients Continue to add new patients and prescribers
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11 Joenja Q3 update – strong global momentum in APDS Continued U.S. growth and international launches 13.4 15.9 1.7 7.9 3Q 2025 3Q 2026 Amounts in US$ million Joenja revenue (global) U.S. EU & RoW 102 114 116 120 127 132 136 1Q25 2Q25 3Q25 4Q25 1Q26 2Q26 3Q26 U.S. patients on Joenja +365%15.1 23.8 +19% Revenue +58% 136 patients on therapy in US (+17% vs 3Q25) Significant increase in international demand – 75 patients on therapy (30 launch markets and 45 paid access) Increased identified patients to 304 in U.S. (243 eligible) and 751 international
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12 Joenja for APDS – pediatric label expansion U.S. FDA approval received September 11, 2026 Eligible patients (ages 4–11) identified in the U.S. APPROVED Children aged 4 to 11, at least 27 kg • sNDA approved – September 11, 2026 • New 40 mg and 50 mg twice-daily dosing • 38 eligible patients identified (September 30) • All eligible EAP patients approved by insurance • Began shipping to patients in October UNDER FDA REVIEW Children aged 4 to 11, 13 kg to less than 27 kg • Separate sNDA filed July 30, 2026 – covers lower doses • FDA granted Priority Review • PDUFA date – January 30, 2027 >60
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13 13 Established APDS Commercial Base • 136 APDS Patients on Therapy (U.S.)* U.S. APDS Expansion • Pediatric (4-11 yrs) launch • Continued patient finding International APDS Expansion • Focused geo- expansion that doubles addressable opportunity • Approved in all target launch markets** Broad PIDs/CVID Opportunity*** • Genetic PIDs with immune dysregulation (>2,500 U.S. patients) • CVID with immune dysregulation (>13,000 U.S. patients) Joenja expansion from APDS foundation into broad blockbuster immunology opportunity *APDS prevalence est. ~1.5 patients / million in U.S. and Core 8 ex-US markets. Patients identified: 304 patients in the U.S. (243 eligible, including 38 aged 4-11 >27 kg), 1056 globally (as of September 30, 2026). **Launched in U.K., Germany (first launched EU market), Japan ***Prevalence overlap: CVID prevalence est. ~39 patients / million. Genetic PIDs prevalence est. ~7.5 patients / million (4.5 patients / million of which are included in CVID prevalence) Sequential Growth Drivers to Blockbuster Potential Pediatric sNDA approved (higher doses) Filed lower dose sNDA CVID expanded access clinical experience U.S. patients +17% (Q3’26 vs ’25) 243 eligible patients Germany launch July ‘26 Japan launch August ’26 PhII trial readouts: PIDs at ESID – October CVID - November
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14 14 Anurag Relan Chief Medical Officer R&D update CMO
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15 Leniolisib opportunity: Targeting shared PI3Kδ-driven immune dysregulation across primary immunodeficiencies (PIDs) APDS APPROVED Joenja (leniolisib) Genetic PIDs + CVID with immune dysregulation Two Phase II trials ↓ One potential registrational trial ONE MECHANISM: PI3Kδ Shared clinical manifestations across all three populations Lymphoproliferation Pulmonary disease Autoimmunity GI disease The two Phase II studies test whether the same targeted approach can benefit these broader populations. E X P A N D I N G T H E A D D R E S S A B L E M A R K E T Up to 26× the APDS population
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16 Leniolisib Phase II trials in genetic PIDs and CVID Positive data presented at ESID 2026 N = 13 Genetic PIDs with immune dysregulation Open-label, single-arm basket trial at NIH (ALPS-FAS, CTLA4, NFKB1, SOCS1) NCT06549114 Topline results expected November N = 20 CVID with immune dysregulation Open-label, single-arm, multi-center dose-confirmation study NCT06897358 Intra-patient dose escalation: 10 mg / 30 mg / 70 mg twice daily · Followed by separate 3-year open-label extension ENDPOINTS Spleen size & index lymph nodes Blood cell counts Lung & liver (end-organ) Novel and indication- specific patient & clinician outcomes Biomarkers (CXCL13, B cells) Together, these studies will inform our plans for a potential registrational study in the broader CVID population Presented as a late-breaker ePoster at the European Society for Immunodeficiency (ESID) biannual meeting 2026 Aim to demonstrate clinically meaningful reductions in lymphoproliferation and cytopenias, end-organ stabilization and supportive biomarker shifts — establishing proof of concept and confirming dose
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17 Genetic PIDs study: leniolisib was well-tolerated and improved multiple measures of immune dysregulation +150%12.8 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 SPLEEN VOLUME −26.4% mean reduction from baseline at end of treatment (n=11) LYMPH NODE SIZE −33.7% mean reduction in index lesion size (n=4) LUNG FUNCTION – GAS EXCHANGE +8.0% mean change in DLCO % predicted (n=7) LIVER STIFFNESS 2 of 3 participants with elevated baseline stiffness improved Well tolerated, with no new safety signals • Infections were the most common adverse event (7 participants), as expected in a PID population • Two participants experienced SAEs, all considered unrelated to leniolisib • All 12 study completers continued into a separate 3-year open-label extension study POPULATION 13 participants (12 study completers); mean age 32.5 years; 77% male GENETIC DRIVERS CTLA4 (6), SOCS1 (3), NFKB1 (2), FAS (2) CVID OVERLAP 9 of 13 also had a CVID diagnosis
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18 Leniolisib Phase II trials – CVID results expected in November Positive data presented at ESID 2026 N = 13 Genetic PIDs with immune dysregulation Open-label, single-arm basket trial at NIH (ALPS-FAS, CTLA4, NFKB1, SOCS1) NCT06549114 Topline results expected November N = 20 CVID with immune dysregulation Open-label, single-arm, multi-center dose-confirmation study NCT06897358 Intra-patient dose escalation: 10 mg / 30 mg / 70 mg twice daily · Followed by separate 3-year open-label extension ENDPOINTS Spleen size & index lymph nodes Blood cell counts Lung & liver (end-organ) Novel and indication- specific patient & clinician outcomes Biomarkers (CXCL13, B cells) Together, these studies will inform our plans for a potential registrational study in the broader CVID population Presented as a late-breaker ePoster at the European Society for Immunodeficiency (ESID) biannual meeting 2026 Aim to demonstrate clinically meaningful reductions in lymphoproliferation and cytopenias, end-organ stabilization and supportive biomarker shifts — establishing proof of concept and confirming dose
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19 Napazimone (KL1333) for mtDNA-driven primary mitochondrial disease Aiming for the first disease-modifying treatment 1. In US, EU4 and UK. Diagnoses can include MELAS-MIDD and KSS-CPEO spectrum disorders as well as MERRF syndrome. NADH ETC Mutation Impaired ETC protein expression Low NAD+/NADH ratio Decreased energy production and decreased mitochondrial biogenesis Debilitating fatigue and muscle weakness significantly impairing activities of daily living NAD+ Targets the underlying pathology Normalizes the NAD+/NADH ratio and mitochondrial function, with evidence from in vitro data, animal models, and in patients treated with KL1333 >30,000 diagnosed patients with mtDNA disorders¹ Registrational clinical study underway Clinically-relevant Fatigue and Sit-to-Stand endpoints supported by FDA Positive interim analysis – both endpoints cleared futility Over 50 sites actively recruiting Expect to complete enrollment 1H 2027 with trial readout 1H 2028 DYSFUNCTIONAL MITOCHONDRIA
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20 20 Kenneth Lynard Interim Co-CEO & Chief Financial Officer Financial results and outlook Co-CEO & CFO
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21 32.0 32.4 Amounts in US$ million Cash flow from operations Financial highlights: Q3 2026 vs Q3 2025 * Adjusted operating profit for 3Q 2025 excludes US$0.2 million of non-recurring Abliva acquisition-related expenses. For 3Q 2026 adjusted operating profit excludes US$5.1 million in one-time manufacturing-related impairments of inventory. • Revenues of US$117.6 million (+21%). • Strong Joenja revenue growth (+58%). RUCONEST returned to growth +14% YoY and up 30% sequentially. • Strong Adjusted Operating Profit growth, impacted by one-time manufacturing inventory impairments. • Cash and Marketable Securities US$190.9M, up US$31.4M vs. Q2 2026, primarily due to net profit. 82.2 93.8 15.1 23.8 Amounts in US$ million Revenues RUCONEST Joenja 16.0 35.0 Amounts in US$ million Adjusted (Non-GAAP) Operating profit* +21% Q3 2025 Q3 2026 Q3 2025 Q3 2026 Q3 2025 Q3 2026 97.3 117.6
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22 44.0 24.7 Amounts in US$ million Cash flow from operations 29.7 38.0 Amounts in US$ million Adjusted (Non-GAAP) Operating profit* Financial highlights: 9M 2026 vs 9M 2025 * Adjusted operating profit for 9M 2025 excludes US$10.1 million of non-recurring Abliva acquisition-related expenses. For 9M 2026 adjusted operating profit excludes US$10.0 million in one- time manufacturing-related impairments of inventory and US$1.7 million in expenses associated with the planned closure of the production-support site in Évry, France. • Revenues of US$280.2 million (+4%). Strong Joenja revenue growth (+45%). RUCONEST lower (-3%). • Adjusted Operating Profit growth driven by an increase in revenue and lower operating expenses (-2.8% vs. PY). • Cash and Marketable Securities increased by US$9.8M to US$190.9M in 9M, primarily due to net profit, partially offset by -US$12.3M for DSP lease termination. 231.2 224.5 38.4 55.7 Amounts in US$ million Revenues RUCONEST Joenja 9M 2025 9M 2026 9M 2025 9M 2026 269.6 280.2 +4% 9M 2025 9M 2026
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23 Significant and accelerating Joenja growth, and RUCONEST revenue stabilization and return to growth during 2H 2026. Strong financial discipline, and prioritized investments to drive near- and long-term value creation. Available cash (US$190.9M) and future cash flows expected to cover current pipeline and pre-launch costs. Revenue and operating expenses (in constant currency): 2026 financial guidance and long-term capital outlook FY 2026 Guidance Notes Total Revenues US$380 - 390 million • 1 - 4% growth • COGS: ~11% Operating Expenses US$310 - 315 million • 0 - 1% growth • +US$35 million R&D to advance pipeline
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24 • Durable cash engine Advancing Pharming as a leading global rare disease company • Geographic, pediatric and prevalence expansion Joenja - commercial Significant growth opportunities Building a leading rare disease company Strong commercial foundation Pipeline - blockbuster potential Q4 2026: leniolisib Ph II readouts in PIDs (October) and CVID (November) H1 2028: napazimone FALCON pivotal study readout in PMD • Disciplined operating model • Scalable organization • Value creation focus • Aligning shareholder base + vision RUCONEST Joenja • Global sales momentum
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25 Interim Co-CEO & Chief Commercial Officer Interim Co-CEO & Chief Financial Officer Chief Medical Officer Leverne Marsh Kenneth Lynard Anurag Relan, MD Q&A
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26 The Netherlands Pharming Group Vondellaan 47 2332 AA Leiden The Netherlands United States Pharming Healthcare, Inc. 10 Independence Blvd Suite 401, Warren NJ 07059 United States WWW.PHARMING.COM Thank you! Joenja® and RUCONEST® are registered trademarks owned by or licensed to Pharming Group N.V. or its affiliates. Other trademarks and trade names are the property of their respective owners.
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27 27 Appendix
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28 Statement of profit and loss
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29 Balance sheet – assets
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30 Balance sheet – liabilities
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31 Cash flow (1/2) 1 Comparative presentation updated for consistency.
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32 Cash flow (2/2) 2 Money market funds are classified as cash equivalents from April 1, 2026