Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Jefferies Global Healthcare Conference 2025
55 slides from Pharming Group Nv's conference presentation, Jun 4, 2025
Slide 1: Pharming Group N.V. Jefferies Global Healthcare Conference
Pharming Group N.V. Jefferies Global Healthcare Conference June 4-5, 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: History of growth and innovation at Pharming
History of growth and innovation at Pharming Pharming completes UK MHRA approves Joenja® acquisition of Abliva FDA accepts priority Pharming acquires Start of Phase II trial of Start of Phase II trial of review of NDA leniolisib for APDS leniolisib for PIDs with leniolisib for CVID with for leniolis
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: Building a leading global rare disease biopharma company
Building a leading global rare disease biopharma company Strong start to 2025 High value pipeline Significant catalysts • 1Q25 revenues up 42% • 2 assets with >$1B sales • Joenja® for APDS – • Strong RUCONEST® potential each VUSs reclassification, growth and acceleration pediatric label, of Joenja®
Slide 6: Strong first quarter 2025 performance
Strong first quarter 2025 performance Revenues Amounts in US$ million 79.1 55.6 • Continued strong RUCONEST® growth • Acceleration in Joenja® patient uptake 1Q 2024 1Q 2025 • Raised 2025 revenue guidance to Adjusted (Non-GAAP) US$325-340 million Operating profit (loss)* Amounts in US$ million 0.8 •
Slide 7: RUCONEST strong growth continues in acute HAE market
RUCONEST® strong growth continues in acute HAE market RUCONEST® revenues • Strong U.S. in-market demand Amounts in US$ million ₋ Continuing to add prescribers and patients 68.6 ₋ New patient enrollments remain high (>90) 46.0 • Continued robust U.S. volume growth ₋ Quarterly growth +37% ₋ 1Q25 boost
Slide 8: Joenja growth continues in 12y+ APDS segment
Joenja® growth continues in 12y+ APDS segment Joenja® revenues • Increasing APDS patients on therapy ₋ 102 U.S. patients (+23% vs 1Q24) Amounts in US$ million ₋ Acceleration in patient uptake (+6 in 1Q25, most since 2Q24) 10.5 9.6 • 18% volume growth • Launched in U.K. (England and Wales) in April •
Slide 9: Diverse rare disease pipeline
Diverse rare disease pipeline 9
Slide 10: RUCONEST for HAE
% Pharming’ RUCONEST® for HAE
Slide 11: RUCONEST: 2nd most prescribed therapy for acute HAE attacks in the US
RUCONEST® (rhC1INH): 2nd most prescribed therapy for acute HAE attacks in the US Only recombinant treatment that targets the root cause of HAE by replacing C1-INH Only recombinant treatment that acts at multiple points in the cascades leading to HAE attacks 11
Slide 12: RUCONEST unique value proposition
RUCONEST® unique value proposition Type 1, Type 2, and Normal C1-INH HAE patients rely on RUCONEST 97% patients needed just 1 dose1 Time of taking 4 hours after 24 hours after 93% acute attacks stopped for at least 3 days2 RUCONEST RUCONEST® mostly used by patients experiencing moderate to severe at
Slide 13: Joenja (leniolisib) for APDS; leniolisib for PIDs with Immune Dysregulation
Joenja® (leniolisib) for APDS leniolisib for PIDs with Immune Dysregulation
Slide 14: Joenja: immune modulator that targets the root cause of APDS
Joenja®: immune modulator that targets the root cause of APDS Helps address immune deficiency and immune dysregulation Joenja® facilitates a balanced PI3Kδ pathway JOENJA WAS DESIGNED TO to support proper immune function6 TARGET THE ROOT CAUSE OF APDS TO HELP NORMALIZE THE HYPERACTIVE PI3Kδ PATHWAY1
Slide 15: 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. 15
Slide 16: Joenja Lifecycle to realize $1Bn+ sales potential
Joenja® (leniolisib) Lifecycle to realize $1Bn+ sales potential Potential total prevalence ~40 patients / million Genetic PIDs prevalence +7.5 patients / million APDS prevalence ~1.5 patients / million Joenja® for CVID Targeted geographic Joenja® for PIDs with immune expansion with immune dysregulat
Slide 17: Joenja: Reclassification of VUS patients
Joenja®: Reclassification of VUS patients Variants of Uncertain VUS Resolution Steps Significance >1300 patients in the US with VUS High throughput screening (MAVE) test result study completed, identifying many new variants causing PI3Kδ hyperactivity VUSs: insufficient data to determine if variant
Slide 18: Joenja development status
Joenja® development status Expanding the addressable patient population Geographic expansion Pediatric expansion Indication expansion (APDS) (APDS) (additional PIDs) Europe – review extended to Jan. 2026 4 to 11 years – Positive data presented PIDs with immune dysregulation Single outstanding CMC re
Slide 19: Leniolisib for PIDs with immune dysregulation
Leniolisib for PIDs with immune dysregulation Patient Population • PID patients with clinical manifestations similar to APDS, including early mortality • Significant unmet clinical need, no approved therapies • Large group: Prevalence 5-26x APDS All PIDs Rationale • Critical role of PI3Kδ in lymphoc
Slide 20: KL1333 for mtDNA Mitochondrial Disease
KL1333 for mtDNA Mitochondrial Disease
Slide 21: KL1333 for primary mitochondrial disease: Potential first standard of care
KL1333 for primary mitochondrial disease: Potential first standard of care KL1333 targets underlying pathology of low NAD+ / NADH • Normalizes NAD+/NADH ratio and mitochondrial function, with evidence from in vitro data, animal models, “The fatigue is almost and in patients treated with KL1333 • >30
Slide 22: KL1333 – FALCON study positive interim analysis
KL1333 – FALCON study positive interim analysis Pivotal FALCON Study Interim Futility Analysis: Positive outcome achieved, with both primary endpoints having WAVE 1 – Fully enrolled passed futility 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) Promising diffe
Slide 23: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 24: Financial highlights: FY 2024 vs FY 2023
Financial highlights: FY 2024 vs FY 2023 Revenues Gross profit OpEx development RUCONEST Joenja R&D G&A M&S Amounts in US$ million Amounts in US$ million Amounts in US$ million 261.8 297.2 220.1 272.6 245.3 45.0 248.8 18.2 118.8 124.0 227.1 252.2 70.7 55.9 68.9 83.1 FY 2023 FY 2024 FY 2023 FY 2024
Slide 25: Financial highlights: 1Q 2025 vs 1Q 2024
Financial highlights: 1Q 2025 vs 1Q 2024 Revenues Gross profit OpEx development* RUCONEST Joenja R&D G&A M&S Amounts in US$ million Amounts in US$ million Amounts in US$ million 70.8 78.2 79.1 10.5 63.8 34.6 55.6 47.2 30.2 9.6 68.6 22.5 15.1 46.0 18.5 21.1 1Q 2024 1Q 2025 1Q 2024 1Q 2025 1Q 2024 1Q
Slide 26: 2025 Financial guidance
2025 Financial guidance Revenue and operating expenses: FY 2025 Guidance Notes Total Revenues US$325 - 340 million 9 - 14% growth Operating Expenses Flat vs. FY 2024 (pre-Abliva impact) Operating Expenses Includes R&D and non-recurring transaction ~US$30 million (Abliva-related) and integration cost
Slide 27: Building a leading global rare disease biopharma company
Building a leading global rare disease biopharma company Strong start to 2025 High value pipeline Significant catalysts • 1Q25 revenues up 42% • 2 assets with >$1B sales • Joenja® for APDS – • Strong RUCONEST® potential each VUSs reclassification, growth and acceleration pediatric label, of Joenja®
Slide 28: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 29: Appendix
Pharming Group N.V. Appendix
Slide 30: 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 The PI3Kδ enzyme • Sinopulmonary p110δ •
Slide 31: APDS can impact many facets of life
APDS can impact many facets of life Physical1,2 Frequent infections Social3,4 Swollen glands Missing school, work, or Shortness of breath daily activities Coughing/wheezing Chest or joint pain Fatigue Treatment Burden1-4 Inability to exercise Frequent hospitalizations Hearing loss Surgeries Diarrhea
Slide 32: Heterogeneous, evolving symptomology can often lead to missed diagnoses
Heterogeneous, evolving symptomology can often lead to missed diagnoses Timeline of the most common pathologies* seen in APDS1-4 Median age at diagnosis: 12 years (7-year median diagnosis delay) <1 year 3 years 5 years 10.5 years 11.2 years† 18 years (range, 1 month-10 years) (range, 1-6 years) (ran
Slide 33: Joenja: First and only approved therapy for APDS
Joenja®: First and only approved therapy for APDS Joenja® (leniolisib) is an oral medication No drug-related serious adverse events or used to treat activated phosphoinositide study withdrawals in Joenja® trials 3-kinase delta (PI3Kδ) syndrome (APDS) Clinical data and tolerability for long term in a
Slide 34: Management for APDS prior to Joenja
Management for APDS1,2 prior to Joenja® Immune Deficiency Immune Dysregulation • Antimicrobial prophylaxis • Corticosteroids • Immunoglobulin • Other immunosuppressants replacement therapy • mTOR inhibitors None of these therapies are FDA- approved for APDS treatment Hematopoietic stem cell transpla
Slide 35: 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 36: Joenja addresses the underlying cause of APDS – 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 37: 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 38: Joenja reduced IgM levels
An exploratory endpoint showed Joenja® reduced IgM levels Mean serum IgM rapidly reduced to within normal limits • In the Joenja® arm, IgM was 600 elevated above normal limits in 6 patients at baseline, and by week 12 was reduced in all, with 50% returning to IgM, mg/dL 400 within normal limits • In
Slide 39: 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 Joenja (n=21) Placebo (n=10) • 32/37 patients Most common AEs n n (%) n (%) reported
Slide 40: Open-label extension interim analysis of 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 41: 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 42: VUS by the numbers
VUS by the numbers VUSs frustrate patients and doctors, limiting diagnosis of genetic diseases such as APDS Pharming is aware of ~1,300 US patients harboring PIK3CD/R1 VUSs • This figure will continue to grow over time • VUS are identified at ~4x the rate of likely pathogenic/pathogenic (LP/P) varia
Slide 43: Immune dysregulation in PIDs can occur via alterations in PI3Kδ signaling
Given importance of PI3Kδ in B & T cells, immune dysregulation in PIDs can occur via alterations in PI3Kδ signaling Altered PI3Kδ signaling can occur in multiple Clinical manifestations, disease onset PID genetic disorders beyond APDS and severity similar to APDS 5-10 (ex: ALPS-FAS, CTLA4, NFKB1, PT
Slide 44: Leniolisib for PIDs with immune dysregulation – Phase II studies
Leniolisib for PIDs with immune dysregulation – Phase II studies Genetically defined PIDs with immune Common variable immunodeficiency (CVID) dysregulation linked to PI3Kδ with immune dysregulation • Single arm, open-label, dose range-finding (N=12) • Single arm, open-label, dose range-finding (N=20
Slide 45: Dysfunctional mitochondria produce less ATP
Dysfunctional mitochondria produce less ATP Primary Mitochondrial Disease (PMD) Mitochondria, often described as the “powerhouses” Dysfunctional Mitochondria of cells, are crucial for energy production Mutation Impaired ETC protein expression Mitochondrial diseases are a group of genetic disorders c
Slide 46: Heavy patient burden with no approved therapies
Heavy patient burden with no approved therapies Presentation and Diagnosis Patients present to their primary care doctor and then often get referred to a neurologist for musculoskeletal issues Either the neurologist or a referral to a metabolic geneticist will result in a diagnosis Many patients are
Slide 47: KL1333 corrects the underlying pathophysiology
KL1333 corrects the underlying pathophysiology KL1333 normalizes conversation of NADH to NAD+ via NQO1 Normalizes the NAD+/NADH Ratio Restored Energy Metabolism Electron Transport Chain Complex III Complex IV Mitochondrial Membrane Q Synthase ATP Complex I Complex II X NADH NAD+ ADP + Pi ATP Restore
Slide 48: KL1333: First-in-disease small molecule with unique MOA
KL1333: First-in-disease small molecule with unique MOA Attributes Outcomes Directly increases the NAD+/NADH ratio Improved energy regulation and ETC via NQO1 function Unique MoA works upstream from all competing MoA in PMD Stimulation of mitochondria biogenesis Oral, small molecule, BID dosing Fati
Slide 49: Phase 1b demonstrated significant activity vs. placebo
Phase 1b demonstrated significant activity vs. placebo The placebo-controlled Phase 1b study demonstrated that KL1333 reduced patients’ fatigue and myopathy after only 10 days, 50 mg/day Fatigue Reduction Muscle Function Improvement Changes from baseline to day 10 Changes from baseline to day 10 KL1
Slide 50: Pivotal study design based on regulatory and patient advocacy input
Pivotal study design based on regulatory and patient advocacy input Study Schematic Regulatory Feedback Study Design Screening Both FDA and EMA accepted Methodology study as registrational • Randomized, double-blind, parallel-group, placebo- FDA said achieving one of the controlled pivotal study 3:2
Slide 51: FALCON study – positive interim analysis
FALCON study – positive interim analysis Pivotal FALCON Study Interim Futility Analysis: Positive outcome achieved, with both primary endpoints having WAVE 1 – Fully enrolled passed futility 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) Promising differences
Slide 52: Significant revenue opportunity for KL1333
Significant revenue opportunity for KL1333 PMD Prevalence 1 per 4,3001 US 78,200; EU4 and UK 76,400 Diagnosed mtDNA 9.6 per 100,0002 US 32,300; EU4 and UK 31,600 FALCON Trial Inclusion Criteria* 51% US 16,500; EU4 and UK 16,100 >30,000 diagnosed mtDNA mitochondrial disease patients addressable in th
Slide 53: Majority of patients treated in US Centers of Excellence or academic institutions
Majority of patients diagnosed and treated in US Centers of Excellence or academic institutions Mitochondrial Care Network – Centers: www.mitonetwork.org/centers 53
Slide 54: Other programs focus on different patient population or failed with different MOA
Other programs focus on different patient population or failed with different MOA Previous programs failed due to old mechanisms of action or evaluating the wrong endpoints Asset Type MOA / ROA Stage Patient Group Comments mtDNA mutations (e.g., NAD+/NADH ▪ Ongoing potentially registrational phase 2
Slide 55: Abliva acquisition – first quarter financial impact
Abliva acquisition – first quarter financial impact Income statement Amounts in US$m 1Q 2025 1Q 2024 Revenues 79.1 55.6 Cost of Sales (8.3) (8.4) Gross profit 70.8 47.2 Other income 0.4 0.3 Research and development* (21.1) (18.5) General and administrative* (22.5) (15.1) Marketing and sales (34.6) (
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