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NASDAQ: PHAR | EURONEXT Amsterdam: PHARM Pharming Group N.V. Corporate Overview January 12, 2026
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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 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 2024 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2024, 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. 2
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Commercial 3 Combination of commercial and pipeline assets poised to deliver strong value creation Leniolisib for PIDs / CVID Phase II trials HAE PMD KL1333 Registrational Phase II trial Positive interim analysis Pipeline PIDs with immune dysregulation > $1B revenue potential > $1B revenue potential RUCONEST® Differentiated value proposition Highly specific manufacturing process Joenja® for APDS Significant near-term catalysts Up to 100x current prevalence
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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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• Announced preliminary 2025 revenues* of $376M (+ 27%) – above latest guidance • Results reflects continued growth of RUCONEST® and acceleration in Joenja® APDS uptake • Significant operating profit $30M and operating cash flow $44M in 9M 2025 • Reiterated $304-308M operating expense guidance for 2025 – committed to cost discipline and deploying capital to high growth initiatives • February 3rd Investor Day to cover PID and PMD clinical programs and 2026 financial guidance 5 Strong commercial and financial momentum FY 2024 FY 2025* Amounts in US$ million Revenues 297.2 376.0 * 2025 revenues are preliminary and unaudited. Final results may differ and will be reported in the financial results for the fourth quarter and full year 2025, to be published in March 2026.
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Executing on high value rare disease pipeline 6 Leniolisib sNDA for 4-11 yo APDS patients – FDA Priority Review, Jan. 26 PDUFA Japan, EMA and other regulatory reviews on track for 2026 approvalsAPDS Genetic PID and CVID Phase II POC trials on track for 2H 2026 read-outsPIDs with immune dysregulation KL1333 pivotal trial – 20+ sites actively enrolling with additional 20+ being opened, on track for late 2027 read-outPMD
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7 Diverse rare disease portfolio and pipeline
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RUCONEST® for HAE
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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 population and positioning • Type 1, Type 2, and Normal C1-INH HAE patients • Mostly used by patients experiencing more severe / frequent attacks, who have failed other on-demand medications Highly specific manufacturing process RUCONEST® poised to remain a cornerstone on-demand treatment for difficult to treat HAE patients 9References: 1. RUCONEST®. Prescribing information. Pharming Healthcare Inc; 2020. 2. Bernstein JA, et al. Ann Allergy Asthma Immunol. 2017;118(4):452-453. 3. Data on file. Pharming Healthcare Inc; 2019 The most common adverse reactions (incidence ≥2%) were headache, nausea and diarrhea. The most serious adverse reaction reported in clinical trials was anaphylaxis. Time of taking RUCONEST 4 hours after 24 hours after
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22 20 24 20 26 22 2Q24 3Q24 4Q24 1Q25 2Q25 3Q25 RUCONEST® U.S. new prescribers RUCONEST® Q3 update – resilient demand in acute HAE 10 • Strong U.S. in-market demand ₋ Continuing to add prescribers and patients ₋ New patient enrollments remain high (~60) ₋ Increase in more severe / frequent attack patients • Continued robust U.S. volume growth ₋ +24% in 3Q25 ₋ +28% in 9M25 63.6 82.2 3Q 2024 3Q 2025 Amounts in US$ million RUCONEST® revenues
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Joenja® (leniolisib) APDS & PID indications
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12 APDS is a rare primary immunodeficiency (PID) Genetic defect leads to PI3Kδ hyperactivity Hyperactive PI3Kδ results in dysregulated B and T cell development1-3 Bcell / T cell PI3Kδ p110δ p85ɑ AKT/PKB FOXO mTOR Growth Survival Differentiation Proliferation Apoptosis inhibition Note: Illustration does not include all steps in the signaling pathway. Immune imbalance leads to diverse signs and symptoms1,4-6 Severe, recurrent, persistent infections • Sinopulmonary • Herpesvirus (especially EBV and CMV) Lymphoproliferation • Lymphadenopathy • Splenomegaly/hepatomegaly • Nodular lymphoid hyperplasia Enteropathy Autoimmunity • Cytopenias • Autoimmune disorders • Autoinflammatory disorders Lymphoma Bronchiectasis FOXO, forkhead box O; mTOR, mammalian target of rapamycin; PI3Kδ, phosphoinositide 3-kinase delta; PKB, protein kinase B. 1. Lucas CL, et al. Nat Immunol. 2014;15(1):88-97. 2. Fruman DA, et al. Cell. 2017;170(4):605-635. 3. Okkenhaug K, Vanhaesebroeck B. Nat Rev Immunol. 2003;3(4):317-330. 4. Coulter TI, et al. J Allergy Clin Immunol. 2017;139(2):597-606. 5. Elkaim E, et al. J Allergy Clin Immunol. 2016;138(1):210-218. 6. Jamee M, et al. Clin Rev Allergy Immunol. 2020;59(3):323-333.
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Rao VK, et al. Blood Adv. 2024;8(12):3092-3108. doi:10.1182/bloodadvances.2023011000. 24-year-old male with APDS whose progress was followed in the Joenja® open-label extension study for 6 years Reports of Joenja® changing patients’ lives 13
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11.2 15.1 3Q 2024 3Q 2025 Amounts in US$ million Joenja® revenues 83 91 93 96 102 114 116 1Q24 2Q24 3Q24 4Q24 1Q25 2Q25 3Q25 US patients on Joenja® Joenja® Q3 update – strong double-digit growth in 12y+ APDS 14 • Strong YoY increase in APDS patients on therapy in the US ➢ 116 patients (+25% vs 3Q24) • Acceleration of U.S. APDS patient identification ➢+13 in Q3, +36 YTD • FDA priority review of 4-11 yr ped filing (Jan. 31, 2026 PDUFA) • Additional 180 APDS patients in access programs and clinical studies globally
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15 Unlocking Joenja® (leniolisib) growth to realize $1Bn+ potential Expanding addressable patient population and indications *Initial APDS prevalence estimate ~1.5 patients / million. 270 patients currently identified in the U.S. (73 pediatric), 990 identified globally. (Data as of September 30, 2025) **Estimate: 20% of >1,400 U.S. patients with a variant of uncertain significance, or VUS, in the PIK3CD and PIK3R1 genes implicated in APDS could ultimately be diagnosed with APDS. CVID with immune dysregulation >13,000 U.S. patients (PID/CVID) APDS* ~500 U.S. patients (30% ped) + >250 potential VUS patient reclassifications** Potential 100x prevalence Cell publication (June 2025) PIDs with immune dysregulation linked to PI3Kδ signaling >2,500 U.S. patients New indications in Phase II POC trialsCommercial U.S. patient numbers ONLY shown to illustrate prevalence
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16 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 patients Identified 54 patients in the U.S., many already on drug Launch readiness of track Joenja® APDS pediatric label expansion * Assuming a positive FDA decision
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>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 “classic” APDS Findings Next steps Global advisory board to discuss how these variants may cause disease (Nov. 2025) Identify individuals who may benefit from PI3Kδ inhibition – build predictive, AI-driven model • Apply AI-based clustering and PheWAS* to link GOF variants to patient phenotypes in large biobanks • Generate data supporting expansion of APDS clinical definition • Apply predictive model to identify patients in large health system EMRs Identify additional GOF variants Activities surrounding expanded APDS prevalence *PheWAS – Phenome Wide Association Study 17
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Prevalence per million population Three primary immunodeficiency with immune dysregulation indications driven by dysfunctional B and T cells under the influence of the PI3Kδ pathway Yes (PIK3CD, PIK3R1) No. Clinical Dx (75% no genetic drivers) CVID w/immune dysregulation Genetic Diagnosis Recurrent viral and bacterial infections Autoantibodies: Autoimmune cytopaenias Lymphoproliferation: lymphadenopathy splenomegaly Lymphocytes infiltrate end-organs: lung, GI tract, liver, Malignancy: Lymphomas Yes (6 different mutations in study) N/A Joenja controls B and T cell dysregulation via PI3Kδ pathway, correcting the abnormal immunophenotype Link to PI3Kδ pathway PI3Kδ Lock & KEY mutation linked to PI3Kδ hyperactivity Cluster of clinical manifestations driven by B & T Cell dysfunction APDS Additional PIDs linked to PI3Kδ 7.5 39 Generally well controlled with Ig, antibiotics Current SoC Poor disease control (Steroids, immunosuppressants, and immunomodulators) Current SoC Poor disease control (Steroids, immunosuppressants, and immunomodulators) 1.5 18Development status Approved PhII POC trial (2H26 readout)PhII POC trial (2H26 readout)
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19 • 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 • Patients treated for 6 to 29 months Leniolisib compassionate use experience in PID/CVID with immune dysregulation
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KL1333 for mtDNA Mitochondrial Disease
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21 KL1333 for mtDNA-driven primary mitochondrial disease Aiming for the first disease-modifying treatment 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 Significant patient population • >30,000 diagnosed patients with mtDNA disorders1 • Majority of patients treated in centers of excellence2 Registrational clinical study underway • Clinically-relevant Fatigue, Sit-to-Stand endpoints supported by FDA • Positive interim analysis – both endpoints cleared futility • Expect readout in 2027 and FDA approval end of 2028 1. In US, EU4 and UK. Diagnoses can include MELAS-MIDD and KSS-CPEO spectrum disorders as well as MERRF syndrome. 2. UNITED MITOCHONDRIAL DISEASE FOUNDATION, Voice of the Patient Report, 2019. NADH NAD+ ETC Mutation Impaired ETC protein expression Low NAD+/NADH ratio Dysfunctional mitochondria Decreased energy production and decreased mitochondrial biogenesis Debilitating fatigue and muscle weakness significantly impairing activities of daily living
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22 KL1333 showed efficacy in patients diagnosed with mtDNA PMD after 10 days using 50 mg/day • Fatigue reduction (NeuroQoL fatigue change) • Muscle function improvement (30 seconds sit-to- stand) KL1333 Phase 1b demonstrated significant activity vs. placebo Fatigue Reduction Changes from baseline to day 10 Muscle Function Improvement Changes from baseline to day 10 Source: Pizzamiglio C et al., Optimizing rare disorder trials: a phase 1a/1b randomized study of KL1333 in adults with mitochondrial disease, Brain, 2024. Lactate:Pyruvate Ratio Changes from baseline to day 10 Improved lactate/pyruvate ratio, reflecting target engagement No serious adverse events reported
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23 KL1333 – FALCON study positive interim analysis 180 total patients treated for 48 weeks All Wave 1 sites + three new sites active (n=20) Planning 40+ total sites, with significant expansion in the US Readout anticipated 2027 WAVE 1 – Fully enrolled 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 Data monitoring committee (DMC) concluded: • Safety and tolerability profile acceptable • No changes to study design • 180 total patients confirmed in the study WAVE 2 – Enrolling 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) Interim analysis at 24 weeks Pivotal FALCON Study
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Financials and Outlook
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25 Growth acceleration across the commercial portfolio • From FY 2016 – FY 2020 Pharming Group reported earnings in EUR. Revenues during this time frame have been converted to USD. In 2021, Pharming Group began reporting earnings in USD. • 4Q 2020 and 1Q 2021 quarterly fluctuations and volatility from COVID-19. • 2025 revenues are preliminary and unaudited. Final results may differ and will be reported in the financial results for the fourth quarter and full year 2025, to be published in March 2026. 18 101 160 192 212 199 206 227 253 18 45 376 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 Total revenues in $* millions Revenues (US$ million) RUCONEST® Joenja® Total 245 297 376 (Re-acquisition from Valeant Dec. 2016)
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246 252 275 292 311 376 40 45 46 48 51 3Q24 LTM FY24 1Q25 LTM 2Q25 LTM 3Q25 LTM FY25 Amounts in US$ million Revenues RUCONEST Joenja 26 Positive rolling 12-month financial trends * 2025 revenues are preliminary and unaudited. Final results may differ and will be reported in the financial results for the fourth quarter and full year 2025, to be published in March 2026. 362 376* 286 297 321 340 -14 -9 1 15 26 Amounts in US$ million Operating profit (loss) 3Q24 LTM FY24 1Q25 LTM 2Q25 LTM 3Q25 LTM 0 -2 6 31 53 Amounts in US$ million Operating Cash Flow 3Q24 LTM FY24 1Q25 LTM 2Q25 LTM 3Q25 LTM
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27 2025 financial guidance and long-term capital outlook 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 transaction and integration expenses • Excludes ~$7M restructuring costs in Q4 Revenue and operating expenses: RUCONEST® well positioned to provide continued strong cash flows Available cash and future cash flows expected to cover current pipeline and pre-launch costs
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Building momentum across commercial, financial and pipeline 28 2025 revenue ~$376M vs. $365-375M guidance High dbl-digit growth for RUCONEST® and Joenja® US$30M operating profit (9M 2025) US$44M operating cash flow (9M 2025) Strong growth momentum Sustained growth of commercial portfolio Significant Joenja® APDS growth catalysts: • Pediatric label, VUSs, targeted geo expansion, prevalence expansion Enhanced capital allocation driving growth Strategic growth priorities Joenja® (leniolisib) for PIDs/CVID with immune dysregulation • PhII readouts (2026) KL1333 for mtDNA mitochondrial disease • Pivotal study readout (2027) High value pipeline Growth-oriented leadership team Proven commercial and development capabilities Scalable organization Building a leading rare disease co.
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NASDAQ: PHAR | EURONEXT Amsterdam: PHARM www.pharming.com
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Pharming Group N.V. Appendix
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31 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 3 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) Joenja: Targeting the root cause of APDS to help restore immune balance 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 Baseline† 26.7 Baseline† 38.5 Baseline† 22.9 Baseline† 39.0 Days with infection per patient (m/N) Patient-reported Annual Rate of Infection Days*
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32 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- finding1,2 Pivotal Trial - Part 2: Efficacy & Safety Evaluation3 Open-label extension study4,5 6 patients with APDS 12 weeks 10 mg, 30 mg, 70 mg bid (4 weeks each dose) 70 mg bid selected for Part 2 31 patients with APDS (21 Joenja®, 10 placebo) 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, dose-escalating Randomized, triple-blinded, placebo-controlled • 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 Nonrandomized, open-label, long-term study 12 weeks 70 mg bid
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33 Joenja® addresses the underlying cause of APDS to help restore immune balance – Phase 3 co-primary endpoints -10 0 10 20 30 40 50 CFB at D85 in Naïve B Cells/Total B Cells, % 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† n=8 n=5 Joenja Placebo p=0.0002 Change from baseline in index nodes* Joenja Placebo n=18 n=8 Immune Deficiency Immune Dysregulation 21 x 16 mm 12 x 9 mm Prior to Treatment After 12 Weeks p=0.0006 -46% -5% 37% 0.1% 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) At 12 weeks Joenja® decreased lymphadenopathy and increased naïve B cells
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34 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 cm2 (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 cm3 (95% CI: -297, -76.2), P=0.0020 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. at week 12 27% reduction in 3D spleen volume* 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.
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35 Joenja® safety profile 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 • 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 Phase 3 Trial1,2 Adverse reactions reported by ≥2 patients treated with Joenja and more frequently than placebo 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 Open-label Extension Study3 Data cutoff for interim analysis: December 13, 2021 • 38 patients had a median exposure of ~2 years • 4 patients had >5 years of exposure • 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 Across all trials2 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
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36 Open-label extension interim analysis of days spent with infections and 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 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. 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 Baseline† 26.7 Baseline† 38.5 Baseline† 22.9 Baseline† 39.0 Days with infection per patient (m/N) Annual Rate of Infection Days* Physician-reported IRT Reduction‡ 6 achieved IRT freedom§ Of 27 patients receiving IRT: 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 37% OF PATIENTS RECEIVING IRT REDUCED OR DISCONTINUED DOSE