Slide 1: First quarter 2025 financial results and business update
Pharming Group N.V. First quarter 2025 financial results and business update May 8, 2025 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Speakers
SPEAKERS Fabrice Chouraqui Stephen Toor Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Commercial Officer Chief Medical Officer Chief Financial Officer
Slide 3: 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 4: Introduction
CEO Fabrice Chouraqui Chief Executive Officer Introduction
Slide 5: 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 5
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 • Raising 2025 revenue guidance to Adjusted (Non-GAAP) US$325-340 million Operating profit (loss)* Amounts in US$ million 0.8 •
Slide 7: Diverse rare disease pipeline
Diverse rare disease pipeline 7
Slide 8: Commercial update
CCO Stephen Toor Chief Commercial Officer Commercial update
Slide 9: 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 10: 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 11: 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 12: 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 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 (leniolisib) Expanding the addressable patient population
Joenja® (leniolisib) Expanding the addressable patient population Potential total prevalence ~40 patients / million Genetic PIDs prevalence +7.5 patients / million APDS prevalence ~1.5 patients / million Joenja® for CVID Joenja® for PIDs with immune Targeted geographic with immune dysregulation expa
Slide 15: R&D Update
CMO Anurag Relan, MD Chief Medical Officer R&D Update
Slide 16: Joenja: Finding more APDS patients - VUS focus
Joenja®: Finding more APDS patients - VUS focus 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 va
Slide 17: 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 18: 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 19: KL1333 for primary mitochondrial disease
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, “The fatigue is almost with evidence from in vitro data, animal models, and in patients treated with KL1333 impos
Slide 20: Diverse rare disease pipeline
Diverse rare disease pipeline 20
Slide 21: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 22: 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 23: 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) (
Slide 24: 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 25: 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% • Joenja® (leniolisib) for • Joenja® for APDS – • Strong RUCONEST® PIDs with immune VUSs, pediatric label, growth and increased dysregulation geo expansion (
Slide 26: Q&A
Q&A Fabrice Chouraqui Stephen Toor Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Commercial Officer Chief Medical Officer Chief Financial Officer
Slide 27: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 28: Appendix
Pharming Group N.V. Appendix
Slide 29: Statement of profit and loss
Statement of profit and loss 29
Slide 30: Balance sheet – assets
Balance sheet — assets Pharming’ December 31, 2024 Non-current assets Intangible assets 138,863 61,039 Property, plant and equipment 7,770 7,752 Right-of-use assets 16,457 16,382 Long-term prepayments 94 90 Deferred tax assets 18,390 30,544 Investment accounted for using the equity method 672 466 In
Slide 31: Balance sheet – liabilities
Balance sheet – liabilities 31
Slide 32: Cash flow (1/2)
Cash flow (1/2) Pharming’ 1Q 2025 1Q 2024 Profit (loss) before tax (11,788) (16,624) Adjustments to reconcile net profit (loss) to net cash used in operating activities: Depreciation, amortization, impairment of non-current assets 2,582 5,921 Equity settled share based payments 2,576 2,427 Loss (gai
Slide 33: Cash flow (2/2)
Cash flow (2/2) Pharming’ Capital expenditure for property, plant and equipment (282) (80) Investment intangible assets (6) - Disposal of investment designated as at FVOCI - 1,971 Investment in associates using the equity method (411) _ Purchases of marketable securities = (94,778), Proceeds from sa