Slide 1: Third quarter 2023 financial results
Pharming Group N.V. Third quarter 2023 financial results October 26, 2023 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 sustainable rare disease business 3Q23 updates
Building a sustainable rare disease business 3Q23 updates Market RUCONEST® Global approvals and Ongoing pipeline development in all key international commercialization of and management of rare markets – U.S. focus Joenja® (leniolisib) disease assets Positive cash flow from >$200 million Advance int
Slide 6: Pipeline – multiple commercial stage rare disease products
Pipeline – multiple commercial stage rare disease products 6
Slide 7: Commercial update
CCO Stephen Toor Chief Commercial Officer Commercial update
Slide 8: RUCONEST - 3Q23 commercial updates
RUCONEST® - 3Q23 commercial updates Revenues increased 11% in 3Q23 (US$60.2m) vs 3Q22 Revenues increased 2% in 9M23 (US$153.8m) vs 9M22 Performed well in leading revenue indicators in the U.S. including active patients, vials shipped, and number of physicians prescribing Strong U.S. in-market demand
Slide 9: Strong commitment to HAE community
Strong commitment to HAE community # of unique U.S. physicians prescribing Strong patient organization support 800 since 2000 700 600 # of HCPs 500 More than 700 U.S. physicians (and growing) prescribing RUCONEST® 400 704 300 647 584 508 200 411 >2,000 patients with HAE have been 295 prescribed RUCO
Slide 10: Joenja set up for U.S. commercial success
Joenja® set up for U.S. commercial success Patient Identification Patient Access • Work with HCPs to further identify • Dedicated support and education resources through the patients and get them tested APDS Assist patient support program • APDS clinical educators assist with • APDS Assist to help p
Slide 11: Joenja 3Q23 launch update: continued strong commercial execution
Joenja® 3Q23 launch update: continued strong commercial execution Strong commercial execution 6 months into U.S. launch Continue to add enrollments 76 enrollments, of which 63 patients on paid therapy at end 3Q23 All but one pre-existing OLE/EAP patients enrolled or are on paid therapy 37 patients o
Slide 12: APDS Joenja (leniolisib)
CMO Anurag Relan, MD Chief Medical Officer APDS Joenja® (leniolisib)
Slide 13: APDS is a rare, primary immunodeficiency first characterized in 2013
APDS is a rare, primary immunodeficiency (PI) first characterized in 2013 Activated phosphoinositide Until now, treatments for APDS have 3-kinase delta (PI3Kδ) addressed the symptoms of the disease syndrome (APDS) affects which manifest early in childhood, but >1500 patients* not the root cause of A
Slide 14: U.S. launch of Joenja: a much-needed treatment for patients with APDS
U.S. launch of Joenja®: a much-needed treatment for patients with APDS and another win for Pharming Joenja® (leniolisib) is a prescription medicine Joenja® reported additional findings that is used to treat activated phosphoinositide from an ongoing long-term open- 3-kinase delta (PI3Kδ) syndrome (A
Slide 15: Joenja – looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval Europe – CHMP opinion on UK – MHRA filing expected MAA expected 4Q23 4Q23 (approval ~ 2 months later)* (approval ~2 months later)** AUS, CAN, ISR submissions Japan clinical study – processing as anticipated 1st patient enrolled Aug 2023 CAN & AUS approval 2Q24**
Slide 16: Hiding in plain sight: Patient finding strategy
Hiding in plain sight: Patient finding strategy Medical education to raise awareness of APDS Genetic testing Family testing and share leniolisib data Conferences and congresses Sponsored, no-cost testing program Inherited disease* but most APDS Abstracts patients do not have diagnosed family members
Slide 17: Helping diagnose APDS patients: VUS resolution
Helping diagnose APDS patients: Variant of Uncertain Significance (VUS) resolution Genetic testing frequently leads to inconclusive results - previously unseen genetic variants: Patients have clinical symptoms Need to determine if Variant of compatible with APDS, but genetic Frustrating for patients
Slide 18: Medical conference presentations (selected)
Medical conference presentations (selected) AMCP Nexus (October 2023) • A Real-world Comparison of Health Care Resource Utilization and Health Care Costs Among Patients With Activated PI3K-Delta Syndrome Versus a Control Cohort of Patients Without Activated PI3K-Delta Syndrome in the United States A
Slide 19: Recent publications
Recent publications Rao VK, et al. (Sep 2023) Interim Analysis: Open-Label Extension Study of Leniolisib for Patients with APDS. https://doi.org/10.1016/j.jaci.2023.09.032 Cant AJ, et al. (Sep 2023) PI3Kδ Pathway Dysregulation and Unique Features of Its Inhibition by Leniolisib in Activated PI3Kδ Sy
Slide 20: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 21: Financial highlights: 3Q 2023 vs 3Q 2022
Financial highlights: 3Q 2023 vs 3Q 2022 TOTAL TOTAL REVENUES US$54.2 million REVENUES US$66.7 million 3Q 2022 3Q 2023 GROSS GROSS PROFIT US$51.9 million PROFIT US$58.4 million 3Q 2022 3Q 2023 OPERATING OPERATING COSTS US$(44.7) million COSTS US$(56.8) million 3Q 2022 3Q 2023 OPERATING OPERATING PRO
Slide 22: Financial highlights: 9M 2023 vs 9M 2022
Financial highlights: 9M 2023 vs 9M 2022 22
Slide 23: RUCONEST and Joenja driving revenue growth
RUCONEST® and Joenja® driving revenue growth Revenue breakdown RUCONEST® and Joenja® 1Q22 – 3Q23 70 in US$ million TOTAL: 66.7 6.5 60 TOTAL: 54.9 3.8 50 40 30 60.2 54.2 54.6 50.1 51.1 46.6 20 42.5 10 0 1Q 22 2Q 22 3Q 22 4Q 22 1Q 23 2Q 23 3Q 23 RUCONEST® Joenja® 23
Slide 24: Investment in Joenja launch and leniolisib development
Investment in Joenja® launch and leniolisib development Cost category breakdown 3Q22 – 3Q23 in US$ million TOTAL: 65.8* 60 TOTAL: 57.5 10.0* TOTAL: 56.8 TOTAL: 52.7 50 TOTAL: 44.7 33.9 29.0 23.9 25.1 40 27.1 20.4 30 11.0 10.9 20 17.6 10.0 12.0 10 20.9 20.8 15.6 12.3 10.9 0 3Q 22 4Q 22 1Q 23 2Q 23 3Q
Slide 25: Outlook 2023
Outlook 2023 On track for low single digit growth in RUCONEST® revenues Joenja® approved by FDA March 24, 2023, commercializing in U.S. since early April 2023 CHMP opinion in 4Q23, marketing authorization in Europe ~2 months later* File leniolisib with UK’s MHRA following ECDRP route* Continued oper
Slide 26: 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 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’|35i 30, December 31, Amounts i 2022 Non-current assets Intangible assets 69,849 75,121 Property, plant and equipment 9,648 10,392 Right-of-use assets 27,834 28,753 Long-term prepayments 88 228 Deferred tax assets 26,608 22,973 Investments accounted for using the eq
Slide 31: Balance sheet – liabilities
Balance sheet – liabilities 31
Slide 32: Cash flow (1/2)
Cash flow (1/2) 9M 2023 IM 2022 Profit before tax (9,981) 33,069 Adjustments to reconcile net profit (loss) to net cash used in operating activities: Depreciation, amortization, impairment 8,370 6,216 Equity settled share based payments 5,935 4,522 Gain on disposal of investment in associate = (12,3
Slide 33: Cash flow (2/2)
Cash flow (2/2) Amounts in $’000 9M 2023 Net cash flows generated from (used in) operating activities (28,879) 17,223 Capital expenditure for property, plant and equipment (1,133) (1,071) Proceeds on PRV sale 21,080 — Investment intangible assets 23 (591) Investment in associate = 7,384 Purchases of