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1 H.C. Wainwright Global Investment Conference NASDAQ: PHAR EURONEXT Amsterdam: PHARM SEPTEMBER 14 -16, 2026
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2 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. Examples of 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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3 • APDS: Geographic, pediatric and prevalence1 expansion • PIDs / CVID: Two Phase II studies (readout Q4 2026) • Cornerstone on-demand therapy for high-burden HAE patients • mtDNA mitochondrial disease: FALCON pivotal study with positive futility analysis (readout 2027) 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 2026 Revenue guidance US$375–395M
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4 4 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
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5 5 RUCONEST® for HAE
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6 RUCONEST – durable market position in high-burden HAE Overwhelming majority of RUCONEST patients remain on therapy one year into oral competitor launch Differentiated value proposition • Targets the root cause of HAE across all pathways • Efficacy and reliability of IV self administration Mostly used by patients experiencing more severe / frequent attacks Highly specific manufacturing process
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7 17 new prescribers 84 new patient enrollments Improvement in underlying demand RUCONEST Q2 performance Revenue: $72.3M -10% vs. Q2 2025 +24% vs. Q1 2026 Stabilization with return to revenue growth expected by year-end Financial performance Resilient patient base Active RUCONEST Patients Dynamic Patients 93% YoY New Starts Restarts Switches Drop Offs
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8 8 Joenja® (leniolisib) APDS & PID indications
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9 Joenja Q2 update – strong global momentum in APDS ahead of geographic and pediatric expansion expected this year 11.8 15.4 1.0 2.5 2Q 2025 2Q 2026 Amounts in US$ million Joenja revenue (global) U.S. EU & RoW 96 102 114 116 120 127 132 4Q24 1Q25 2Q25 3Q25 4Q25 1Q26 2Q26 U.S. patients on Joenja Revenue +40% 132 patients on therapy in US (+16% vs 2Q25) +16 U.S. patients identified Geographic expansion accelerating +150%12.8 17.9 +31%
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10 Joenja® for APDS - pediatric label expansion U.S. FDA approval received September 11, 2026 Children aged 4 to 11, at least 27 kg PDUFA date – October 24, 2026 sNDA approved – September 11, 2026 New 40 mg and 50 mg twice-daily dosing Available to eligible patients in October Children aged 4 to 11, 13 kg to less than 27 kg Separate sNDA filed July 30, 2026 Covers lower doses 60 eligible patients identified in the U.S.
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11 11 Established APDS Commercial Base • 132 APDS Patients on Therapy (U.S.)* U.S. APDS Expansion • Pediatric (4-11 yrs) launch • VUS reclassification International APDS Expansion • Focused geo- expansion that doubles addressable opportunity 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: 298 patients in the U.S. (198 eligible 12+, 60 eligible 4-11), 1042 globally (as of June 30, 2026). **Previously launched in U.K. ***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 +16% (Q2’26 vs ’25) Recent approvals**: Germany launch July ‘26 Japan launch Q3’26 PhII trial readouts Q4’26
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12 PI3Kẟ is a master regulator of the immune system and imbalance contributes to immune dysregulation PI3Kẟ is a master regulator of the immune system Immune dysregulation pathology Lymphoproliferation Autoimmunity GI Disease Pulmonary Disease p110δ p85ɑ AKT/PKB FOXO mTOR PI3Kδ Cell proliferation Cell Differentiation Cell trafficking Apoptosis inhibition/survival Cell Growth
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13 PI3Kδ inhibition validated in APDS Joenja (leniolisib) APPROVED for APDS Sustained, durable efficacy Lymph-node and spleen volume reduced, sustained through multi-year OLE† Established safety and tolerability* Clinically meaningful improvements** Reduced infections, fewer hospitalizations, and lower treatment burden Primarily mild-to-moderate adverse events; no treatment- related SAEs with up to 7 years of therapy *Based on Phase III clinical trial and open label extension **Based on post-hoc analysis and real-world evidence †OLE: Open Label Extension
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14 Shared clinical manifestations across all three populations Autoimmunity GI Disease Pulmonary DiseaseLymphoproliferation Leniolisib opportunity: Targeting shared PI3Kδ-driven immune dysregulation across primary immunodeficiencies (PIDs) Genetic PIDs with immune dysregulation linked to PI3Kẟ CVID with immune dysregulation Two Phase II trials Expanding the addressable market Joenja (leniolisib) APDS APPROVED
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15 Leniolisib Phase II trials in CVID and PIDs: Fully enrolled, readouts 4Q 20265 CVID with immune dysregulation N = 20 Open-label, single-arm, multi-center NCT06897358 Genetic PIDs with immune dysregulation N = 12 Open-label, single-arm basket trial at NIH (ALPS-FAS, CTLA4, NFKB1, PTEN, SOCS1, NRAS/KRAS RALD) NCT06549114 E N D P O I N T S Lymph-node & spleen size Blood cell counts Lung & liver (end-organ) Novel and indication- specific patient / clinician outcomes Biomarkers (CXCL13, B cells) W H A T W E A I M T O D E M O N S T R A T E Clinically meaningful reductions in lymphoproliferation and cytopenias, end-organ stabilization and supportive biomarker shifts — establishing proof of concept and confirming dose. Fully enrolled Topline Q4 2026 Dose-ranging: 10 mg / 30 mg / 70 mg twice daily · shared 3-year open-label extension
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16 16 Napazimone (KL1333) mtDNA Mitochondrial Disease
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17 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. Napazimone (KL1333) targets underlying pathology • Normalizes 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 disorders 1 Registrational clinical study underway • Clinically-relevant Fatigue, Sit-to-Stand endpoints supported by FDA • Positive interim analysis – both endpoints cleared futility • Over 45 sites actively recruiting • On track to complete enrollment in 2026 and for trial readout in late 2027 DYSFUNCTIONAL MITOCHONDRIA 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+
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18 Napazimone (KL1333) – FALCON study positive interim analysis Pivotal FALCON Study WAVE 1 – Fully enrolled • 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) • Interim analysis at 24 weeks WAVE 2 – Enrolling • 180 total patients treated for 48 weeks • Over 45 active sites, with significant expansion in US • Readout anticipated 2027 Interim Futility Analysis Positive outcome achieved, with both primary endpoints passing futility Promising differences favoring the active arm vs. placebo for both primary efficacy endpoints; if trends continue consistently, we expect a successful result at the completion of this trial Data monitoring committee (DMC) concluded: • Safety and tolerability profile acceptable • No changes to study design • 180 total patients confirmed in the study
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19 19 Financials and Outlook
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20 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$159.5M) 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$375 - 395 million • 0 - 5% growth • COGS: ~11% Operating Expenses US$315 - 320 million • 1 – 3% growth • +US$40 million R&D to advance pipeline • -US$9 million from G&A structural cost reductions (as announced in October 2025)
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21 • RUCONEST resilience – durable cash engine • Joenja sales momentum 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: Two Phase II readouts with leniolisib for PIDs/CVID 2027: FALCON pivotal study readout with napazimone in mtDNA mitochondrial disease • More disciplined operating model • Scalable organization • Value creation focus • Aligning shareholder base + vision
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22 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!
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23 23 Appendix
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24 Joenja®: Targeting the root cause of APDS to help restore immune balance Treatment with Joenja® in a randomized, controlled-trial led to: • Significant improvements in immune dysregulation (e.g, lymph node and splenomegaly reductions) • Significant improvements in immunophenotype Favorable Safety Profile • No serious AEs were related to Joenja treatment • No patients withdrew from the clinical trials due to an adverse drug reaction • The most common adverse reactions (incidence >10%) in the Phase III trial were headache, sinusitis, and atopic dermatitis Long-term open-label study • Median duration of Joenja® exposure was ~2 years • Reduction in infections (see right) 42.4 13.8 8.7 5.5 0 5 10 15 20 25 30 35 40 45 ≥ 1 year of follow-up ≥ 2 years of follow-up ≥ 3 years of follow-up ≥ 4 years of follow-up ≥ 5 years of follow-up Baseline† 26.7 Baseline† 38.5 Baseline† 22.9 Baseline† 39.0 N=28 N=14 N=7 N=4 0 N=3 Patient-reported Annual Rate of Infection Days* Days with infection per patient (m/N)
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25 Joenja® clinical trial designs bid, twice a day; PI3Kδ, phosphoinositide 3-kinase delta; SPD, sum of product diameters 1. Rao VK, et al. Blood. 2017;130(21):2307-2316. 2. NCT02435173. ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02435173. Updated May 6, 2015. Accessed March 13, 2023. 3. Rao VK, et al. Blood. 2023;141(9):971-983. 4. NCT02859727. ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02859727. Updated October 31, 2022. Accessed March 3, 2023. 5. Data on file. Pharming Healthcare Inc; 2022. Pivotal Trial - Part 1: Dose-finding 1,2 Pivotal Trial - Part 2: Efficacy & Safety Evaluation 3 Open-label extension study 4,5 Nonrandomized, open-label, dose-escalating 6 patients with APDS 12 weeks 10 mg, 30 mg, 70 mg bid (4 weeks each dose) 70 mg bid selected for Part 2 Randomized, triple-blinded, placebo-controlled 31 patients with APDS (21 Joenja® , 10 placebo) 12 weeks 70 mg bid Co-primary efficacy end points • Change from baseline in log10-transformed SPD of index lesions - Also assessed as % change • Change from baseline in percentage of naïve B cells out of total B cells Secondary and exploratory end points Safety Nonrandomized, open-label, long- term study • 35 patients with APDS from Parts 1 and 2 • 2 patients with APDS previously treated with investigational PI3Kδ inhibitors Ongoing 70 mg bid Long-term safety, tolerability, efficacy, and pharmacokinetics
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26 Joenja® addresses the underlying cause of APDS to help restore immune balance – Phase III co-primary endpoints Data were analyzed using an ANCOVA model with treatment as a fixed effect and baseline as a covariate. Use of glucocorticoids and IRT at baseline were both included as categorical (Yes/No) covariates. Baseline is defined as the arithmetic mean of the baseline and D1 values when both are available, and if either baseline or the D1 value is missing, the existing value is used. P-value is 2-sided. Least square means are graphed. Error bars are standard error of the mean. *The analysis excluded 2 patients from each treatment group due to protocol deviations and 1 Joenja patient having complete resolution of the index lesion identified at baseline. †Out of 27 patients in the PD analysis set, 13 patients met the analysis requirements, including having a percentage of <48% of naïve B cells at baseline, to form the B-PD analysis set. Joenja [package insert]. Leiden, The Netherlands: Pharming Technologies B.V.; 2023. Please see Important Safety Information and full Prescribing Information available at joenja.com Change from baseline in naïve B cells † At 12 weeks Joenja® decreased lymphadenopathy and increased naïve B cells Log10-transformed SPD of index lesions Joenja (n=18) Placebo (n=8) Baseline mean (SD) 3.03 (0.42) 3.05 (0.39) Change from baseline, LS mean (SE) -0.27 (0.04) -0.02 (0.05) Difference vs placebo (95% CI) -0.25 (-0.38, -0.12) Immune Dysregulation Immune Deficiency Change from baseline in index nodes* p=0.0006 Joenja Placebo -46% -5% 21 x 16 mm 12 x 9 mm Prior to Treatment After 12 Weeks -10 0 10 20 30 40 50 CFB at D85 in Naïve B Cells/Total B Cells, % Joenja Placebo p=0.000237% 0.1% n=5
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27 Joenja® significantly reduced splenomegaly Rao VK, et al. Blood. 2023;141(9):971-983. *In the PD analysis set, the mean (SD) percentage change from baseline to week 12 in 3D spleen volume (mm3) was -26.68% (12.137) with Joenja® (n=19) and -1.37% (24.238) with placebo (n=9). The ANCOVA model was used with treatment as a fixed effect and log10-transformed baseline as a covariate for index and non-index lesions. The use of both glucocorticoids and IV Ig at baseline was included as categorical (yes/no) covariates. This analysis excluded 2 patients in each treatment group. In the Joenja® group, 1 patient with a complete index lesion response was excluded, and 3 patients were excluded for no non-index lesion at baseline. PD, pharmacodynamics. Secondary endpoint: Significant reductions in spleen size by 2D and 3D analysis compared to placebo • The adjusted mean difference in bidimensional spleen size between Joenja® (n=19) and placebo (n=9) was -13.5 cm 2 (95% CI: -24.1, -2.91), P=0.0148 • The adjusted mean difference in 3D spleen volume between Joenja® (n=19) and placebo (n=9) was -186 cm 3 (95% CI: -297, -76.2), P=0.0020 Secondary measure: spleen volume scan results of actual patient illustrate average improvement documented for patients taking Joenja® Prior to treatment: 491 mL At week 12: 314 mL Actual patient images of a 17-year-old male. As individual results vary, images may not be representative of all patients. At week 12 27% reduction in 3D spleen volume*
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28 A patient with multiple occurrences of an AE is counted only once in the AE category. Only AEs occurring at or after first drug intake are included. *Includes dermatitis atopic and eczema. †Includes tachycardia and sinus tachycardia. AEs, adverse events; ALT, alanine aminotransferase; AST, aspartate aminotransferase; SAE, serious adverse event. 1. Rao VK, et al. Blood. 2023;141(9):971-983. 2. Joenja [package insert]. Leiden, The Netherlands: Pharming Technologies B.V.; 2023. 3. Data on file. Pharming Healthcare Inc; 2022. Please see Important Safety Information and full Prescribing Information available at joenja.com Joenja® safety profile Adverse reactions reported by ≥2 patients treated with Joenja and more frequently than placebo • 32/37 patients reported ≥1 AE • 78.4% of AEs were grade 1, 48.6% grade 2, 27.0% grade 3, 0% grade 4 • No SAEs related to Joenja® Phase III Trial1,2 Open-label Extension Study3 Data cutoff for interim analysis: December 13, 2021 • Study drug-related AEs occurred in 8 patients; the incidence was lower in the Joenja® arm (23.8%) than in the placebo arm (30.0%) • No AEs led to discontinuation of study treatment Joenja® (n=21) n (%) Placebo (n=10) n (%) Headache 5 (24) 2 (20) Sinusitis 4 (19) 0 Dermatitis atopic* 3 (14) 0 Tachycardia† 2 (10) 0 Diarrhea 2 (10) 0 Fatigue 2 (10) 1 (10) Pyrexia 2 (10) 0 Back pain 2 (10) 0 Neck pain 2 (10) 0 Alopecia 2 (10) 0 Most common AEs n Upper respiratory tract infection 8 Headache 6 Pyrexia 6 Otitis externa 5 Weight increase 5 COVID-19, positive/negative 5/14 One patient with significant baseline cardiovascular comorbidities suffered cardiac arrest resulting in death at extension Day 879; determined by investigator not to be related to study drug Across all trials2 • 38 patients had a median exposure of ~2 years • 4 patients had >5 years of exposure
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29 Open-label extension interim analysis of days spent with infections and IRT reduction Patient-reported Annual Rate of Infection Days* Physician-reported IRT Reduction‡ *Infections that developed during the study were reported as adverse events. Investigators were requested to inquire about signs and symptoms of infections at each visit, with a particular focus on bacterial enterocolitis. Patients were not provided an infection diary to document infections occurring between visits. One patient was excluded from the analysis due to an incorrect year that was recorded for an infection. †Baseline infections are each group’s year 1 annual rate of infections. N values changed because patients were in the OLE for different lengths of time. ‡Data on concomitant medication usage was reported at each patient visit. §One patient had a subsequent one-time dose. ||One patient achieved IRT freedom for 3 months but subsequently restarted IRT. IRT, immunoglobulin replacement therapy; m, number of infection days; N, number of patients in follow-up category. Rao VK, et al. Poster presented at: 64th Annual American Society of Hematology Annual Meeting; December 10-13, 2022; New Orleans, LA. Please see Important Safety Information and full Prescribing Information available at joenja.com 42.4 13.8 8.7 5.5 0 5 10 15 20 25 30 35 40 45 ≥ 1 year of follow-up ≥ 2 years of follow-up ≥ 3 years of follow-up ≥ 4 years of follow-up ≥ 5 years of follow-up N=28 N=14 N=7 N=4 0 N=3 6 achieved IRT freedom§ 3 reduced use 50%|| 1 reduced use 30% At the data cutoff, 4 of the patients who were IRT free had been so for 1 – 2.5 years Although safety was the primary objective of the open-label study, this post hoc analysis from the open-label study was not powered to provide any statistical significance of efficacy and therefore no conclusions should be drawn. 37% of patients receiving IRT reduced or discontinued dose N=4 Days with infection per patient (m/N) Baseline† 26.7 Baseline† 38.5 Baseline† 22.9 Baseline† 39.0 Of 27 patients receiving IRT: