Slide 1: Third quarter 2024 financial results and business update
Pharming Group N.V. Third quarter 2024 financial results and business update October 24, 2024 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Speakers
SPEAKERS Sijmen de Vries, MD 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 Sijmen de Vries, MD Chief Executive Officer Introduction
Slide 5: Building a leading global rare disease biopharma company
Building a leading global rare disease biopharma company Market RUCONEST® Global approvals and Ongoing pipeline development for acute HAE attacks in key commercialization of and management of rare markets – U.S. focus Joenja® (leniolisib) for APDS disease assets Positive cash flow from RUCONEST® Suc
Slide 6: Pipeline – multiple commercial stage rare disease products
Pipeline – multiple commercial stage rare disease products 6
Slide 7: C1-INH targets the root cause of HAE
C1-INH targets the root cause of HAE Addresses patient needs unmet by other therapies TARGET BRAND GENERIC NAME STATUS TYPE NAME C1 Inhibitor Overview of Marketed and In-Development Therapies and Their Ruconest C1 esterase inhibitor (recombinant) Marketed OD Targets Within the Three Known Cascades L
Slide 8: Commercial update
CCO Stephen Toor Chief Commercial Officer Commercial update
Slide 9: RUCONEST (rhC1INH): trusted treatment cornerstone for HAE
RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE The only recombinant treatment 2nd most prescribed product for that targets the root cause of HAE acute attacks by replacing missing or Typical patient: failed icatibant dysfunctional C1-INH (BK inh) and on prophy Tx (K inh) Well-tolerated a
Slide 10: Joenja (leniolisib) – strong execution and growth opportunity
Joenja® (leniolisib) – strong execution and growth opportunity Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) Revenues: • Marketed (12+) • Found >870 • Phase II POC trial in • U.S. commercial sales patients globally PIDs with immune • Europe / RoW access progr
Slide 11: Joenja (leniolisib) for APDS; leniolisib for PIDs
CMO Anurag Relan, MD Chief Medical Officer Joenja® (leniolisib) for APDS leniolisib for PIDs
Slide 12: Hiding in plain sight: Patient finding strategy
Hiding in plain sight: Patient finding strategy Medical education to raise awareness of APDS Genetic testing VUS resolution and share leniolisib data Conferences and congresses Sponsored, no-cost Validation studies with various Abstracts testing program laboratories to confirm which Assistance from
Slide 13: Joenja – geographic / pediatric / indication expansion
Joenja® ‒ geographic / pediatric / indication expansion Europe – CHMP review extended to January 2026 Israel marketing authorization Single outstanding CMC request received April 30, 2024 Positive clinical benefit and safety concluded CAN, AUS submissions U.K. marketing authorization under regulator
Slide 14: Leniolisib for PIDs with immune dysregulation
Leniolisib for PIDs with immune dysregulation Primary Immunodeficiencies (PIDs) are a broad group of disorders with key potential features: • Genetic basis • Immune dysfunction → increased risk of infection • Immune dysregulation → lymphoproliferation and autoimmunity PIDs • High morbidity and morta
Slide 15: PIDs linked to PI3Kδ signaling – Phase II study design
PIDs linked to PI3Kδ signaling – Phase II study design Phase II proof of concept clinical trial – single arm, open- label, dose range-finding study (N=12) 10mg 30mg 70mg • Patients with PIDs linked to PI3Kδ signaling, e.g. Lead Investigator: Gulbu Uzel, M.D., Senior ALPS-FAS1, CTLA4 haploinsufficien
Slide 16: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 17: Financial highlights: 3Q 2024 vs 3Q 2023
Financial highlights: 3Q 2024 vs 3Q 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 74.8 68.0 64.7 66.7 11.2 56.8 6.5 58.4 28.7 25.1 60.2 63.6 10.9 15.3 20.8 20.7 3Q 2023 3Q 2024 3Q 2023 3Q 2024 3Q 2023 3Q
Slide 18: Financial highlights: 9M 2024 vs 9M 2023
Financial highlights: 9M 2024 vs 9M 2023 Revenues Gross profit OpEx development RUCONEST® Joenja® R&D G&A M&S Amounts in US$ million Amounts in US$ million 204.5 Amounts in US$ million 181.3 198.7 31.9 175.3 164.1 146.0 10.3 91.9 86.1 153.8 172.6 31.9 46.0 57.3 60.8 9M 2023 9M 2024 9M 2023 9M 2024 9
Slide 19: OpEx development – 2024 quarterly
OpEx development – 2024 quarterly Operating Costs 1Q24 – 3Q24 (R&D, G&A, M&S) 70.1 63.8 64.7 Amounts in US$ million 1Q24 2Q24 3Q24 19
Slide 20: 2024 Financial guidance
2024 Financial guidance FY 2024 Revenue Guidance % Growth vs. FY 2023 Total Revenues US$280 - 295 million 14-20% Joenja® significant driver of revenue growth, continued RUCONEST® growth Joenja® revenue assumptions: • Continued growth in patients on paid therapy • Continued high (monthly) adherence (
Slide 21: Pharming 2024 Outlook
Pharming 2024 Outlook Total revenues between US$280 and US$295 million (14% to 20% growth). Joenja® (leniolisib) U.S.: Continued progress finding additional APDS patients, supported by family testing and VUS validation efforts, and subsequently converting patients to paid therapy. Leniolisib ex-U.S.
Slide 22: Q&A
Q&A Sijmen de Vries, MD Stephen Toor Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Commercial Officer Chief Medical Officer Chief Financial Officer
Slide 23: Presentation, recording and transcript available on website
This presentation, a recording and a transcript of this call will be made available on the company’s website www.pharming.com | investor@pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM Bloomberg: PHAR.AS
Slide 24: Appendix
Pharming Group N.V. Appendix
Slide 25: Statement of profit and loss
Statement of profit and loss 25
Slide 26: Balance sheet – assets
Balance sheet — assets Pharming’ September 30,2024 December 31, 2023 Non-current assets Intangible assets 67,096 71,267 Property, plant and equipment 8,692 9,689 Right-of-use assets 21,975 23,777 Long-term prepayments 93 92 Deferred tax assets 36,752 29,761 Investment accounted for using the equity
Slide 27: Balance sheet – liabilities
Balance sheet – liabilities 27
Slide 28: Cash flow (1/2)
Cash flow (1/2) Pharming’ 3Q 2024 3Q 2023) 9M2024 9M 2023 Profit (loss) before tax 1,514 3,307 (15,170)| (9,981) Adjustments to reconcile net profit (loss) to net cash used in operating activities: Depreciation, amortization, impairment of non-current assets 2,743 2,902 8,371 8,370 Equity settled sh
Slide 29: Cash flow (2/2)
Cash flow (2/2) Pharming’ Interest received 1,784 260 4,154 1,059 Income taxes received (paid) (9,117) _ (13,864) _ Net cash flows generated from (used in) operating activities 9,742 3,527 (11,126) (28,879) Capital expenditure for property, plant and equipment (366) (147) (660) (1,133) Proceeds on P