Slide 1: Fourth quarter and full year 2023 financial results
Pharming Group N.V. Fourth quarter and full year 2023 financial results March 14, 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: Commercial update
CCO Stephen Toor Chief Commercial Officer Commercial update
Slide 8: RUCONEST (rhC1INH): trusted treatment cornerstone for HAE
RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE The only recombinant treatment that targets the root cause of HAE Second most prescribed product by replacing missing or for acute attacks dysfunctional C1-INH Well-tolerated and effective 97%: needed just 1 dose of treatment option for acut
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 729 U.S. physicians (and growing) prescribing RUCONEST® 400 729 300 647 584 508 200 411 >2,000 patients with HAE have been 295 prescribed RUCO
Slide 10: Joenja U.S. launch: strong commercial execution
Joenja® U.S. launch: strong commercial execution Strong commercial execution 9 months into U.S. launch Continue to enroll patients and add patients on paid therapy in 4Q23 92 enrollments, of which 81 patients on paid therapy at end 4Q23 APDS Assist program ensures eligible patients have access to th
Slide 11: APDS Joenja (leniolisib) Second indication
CMO Anurag Relan, MD Chief Medical Officer APDS Joenja® (leniolisib) Second indication
Slide 12: APDS is a rare primary immunodeficiency first characterized in 2013
APDS is a rare, primary immunodeficiency (PID) first characterized in 2013 Activated phosphoinositide 3- kinase delta (PI3Kδ) syndrome A genetic test can provide a definitive (APDS) diagnosis of APDS Global prevalence estimated at 1.5 patients per million population* To date, Pharming has identified
Slide 13: U.S. launch of Joenja: a much-needed treatment for APDS patients
U.S. launch of Joenja®: a much-needed treatment for APDS patients and another achievement for Pharming Joenja® (leniolisib) is a prescription There were no drug-related serious adverse medicine that is used to treat events or study withdrawals in Joenja® trials activated phosphoinositide 3-kinase de
Slide 14: Joenja ‒ looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval Europe – awaiting CHMP UK – submitted MAA to MHRA opinion on MAA* on March 12, 2024** Japan clinical study: Patient CAN, AUS, ISR submissions enrollment is now complete under regulatory review PMDA filing following completion of Approvals in 2024*** appropriate
Slide 15: 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 16: 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 17: Recent medical conference presentations (selected)
Recent medical conference presentations (selected) AMCP Nexus - Academy of Managed Care Pharmacy (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
Slide 18: Primary Immunodeficiencies (PIDs) with immune dysregulation
Primary Immunodeficiencies (PIDs) with immune dysregulation PIDs are a broad group of disorders1 with key features: PIDs Genetic basis, i.e., not secondarily caused by another disease ‘Inborn Errors of Immunity’ (IEI) is used interchangeably with PID An increased risk of infection may be the predomi
Slide 19: Importance of PI3Kδ in B & T cells and immune dysregulation in PIDs
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 5-8 (ex: ALPS-FAS, CTLA4, PTEN) 1-4
Slide 20: Leniolisib development for PIDs with immune dysregulation
Leniolisib development for PIDs with immune dysregulation Based on APDS experience, leniolisib has potential to be an effective & tolerable chronic treatment approach for PIDs with immune dysregulation Leniolisib, by reducing PI3Kδ activity, should help rebalance immune dysregulation in PIDs, positi
Slide 21: PIDs linked to PI3Kδ signaling – Phase 2 study design
PIDs linked to PI3Kδ signaling – Phase 2 study design Phase 2 proof of concept clinical trial – single arm, open- label, dose range-finding study (N=12) 10mg 30mg 70mg Lead Investigator: Gulbu Uzel, M.D., Senior Research Physician • Patients with PIDs linked to PI3Kδ signaling, e.g. ALPS-FAS, CTLA4
Slide 22: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 23: Financial highlights: 4Q 2023 vs 4Q 2022
Financial highlights: 4Q 2023 vs 4Q 2022 TOTAL TOTAL REVENUES US$54.6 million REVENUES US$81.2 million 4Q 2022 4Q 2023 GROSS GROSS PROFIT US$48.3 million PROFIT US$74.1 million 4Q 2022 4Q 2023 OPERATING OPERATING COSTS US$(57.4)million COSTS US$(73.6) million 4Q 2022 4Q 2023 OPERATING OPERATING PROF
Slide 24: Financial highlights: FY 2023 vs FY 2022
Financial highlights: FY 2023 vs FY 2022 Revenue Gross profit Other Income 220.1 Other Income 245.3 Grants Divestments in associates 0.2 205.6 188.1 1.8 in US$ million in US$ million PRV sale 0.5 1.8 21.3 12.2 2022 2023 2022 2023 2022 2023 OpEx development Operating profit Net profit (loss) R&D G&A
Slide 25: RUCONEST and Joenja driving revenue growth
RUCONEST® and Joenja® driving revenue growth 300 Pharming revenues FY 2016 – FY 2023 250 245.3 18.2 Total revenues in $* millions 200 150 227.1 100 212.2 205.6 189.4 198.9 159.6 50 101.3 17.5 0 2016 2017 2018 2019 2020 2021 2022 2023 RUCONEST® Joenja® • • From FY 2016 – FY 2020 Pharming Group report
Slide 26: Investment in Joenja launch and leniolisib development
Investment in Joenja® launch and leniolisib development 80 Cost category breakdown 4Q22 – 4Q23 TOTAL: 73.6 70 in US$ million TOTAL: 65.8* 60 TOTAL: 57.4 10.0* TOTAL: 56.8 TOTAL: 52.7 37.9 50 33.9 29.0 23.9 25.1 40 27.1 30 11.0 10.9 24.0 20 17.6 10.0 10 20.9 20.8 15.6 10.9 11.6 0 4Q22 1Q23 2Q23 3Q23
Slide 27: FY 2023: Cashflow including marketable securities
FY 2023: Cashflow including marketable securities January 1, 2023 – December 31, 2023 (US$ Million) Overall cash and Net cash flows Net cash flows Net cash flows Exchange rate Overall cash and marketable used in operating generated from used in financing effects marketable securities on activities i
Slide 28: 2024 Revenue guidance
2024 Revenue guidance FY 2024 Revenue Guidance % Growth vs. FY 2023 Total Revenues US$280 - 295 million 14-20% Assumptions Quarterly fluctuations expected Joenja® significant driver of revenue growth, continued RUCONEST® growth Joenja® assumptions: • Continued growth in patients on paid therapy • U.
Slide 29: Pharming 2024 Outlook
Pharming 2024 Outlook Total revenues between US$280 and US$295 million (14% to 20% growth), with quarterly fluctuations expected. Joenja® (leniolisib) U.S.: Continued progress finding additional APDS patients, supported by family testing and VUS validation efforts, and subsequently converting patien
Slide 30: Q&A
Q&A Sijmen de Vries, MD Stephen Toor Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Commercial Officer Chief Medical Officer Chief Financial Officer
Slide 31: Presentation and recording available on company website
This presentation, a recording and a transcript of this call will be made available on the company’s website www.pharming.com | investor@pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM Bloomberg: PHAR.AS
Slide 32: Appendix
Pharming Group N.V. Appendix
Slide 33: Statement of profit and loss
Statement of profit and loss 33
Slide 34: Balance sheet – assets
Balance sheet — assets Pharming™ Amounts in US$ ‘000 2023 2022 Non-current assets Intangible assets 71,267 75,121 Property, plant and equipment 9,689 10,392 Right-of-use assets 23,777 28,753 Long-term prepayments 92 228 Deferred tax assets 28,332 22,973 Investment accounted for using the equity meth
Slide 35: Balance sheet – liabilities
Balance sheet – liabilities 35
Slide 36: Cash flow (1/2)
Cash flow (1/2) mounts in $’000 2023 22 Profit (loss) before tax (12,012) 14,987 Adjustments to reconcile net profit (loss) to net cash used in operating activi Depreciation, amortization, impairment of non-current assets 15,925 13,188 Equity settled share based payments 9,251 6,392 Gain on disposal
Slide 37: Cash flow (2/2)
Cash flow (2/2) Pharming’ mounts in 2023 20: Interest received (paid) 2,883 85 Income taxes received (paid) (655) (1,235) Net cash flows generated from (used in) operating activities (17,524) 22,458 Capital expenditure for property, plant and equipment (1,437) (1,376) Proceeds on PRV sale 21,279 0 I