Slide 1: First quarter 2024 financial results and business update
Pharming Group N.V. First quarter 2024 financial results and business update May 8, 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: Joenja (leniolisib) franchise – multi-year growth potential
Joenja® (leniolisib) franchise – multi-year growth potential Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) • Marketed (12+) • Patients on early • Phase II POC trial in access/ named PIDs with immune • Significant portion patient programs dysregulation linked
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: Joenja U.S. launch: strong commercial execution
Joenja® U.S. launch: strong commercial execution Strong commercial execution 12 months into U.S. launch Continue to enroll and add patients on paid therapy in 1Q24 83 patients on paid therapy at end 1Q24, with 5 additional enrollments pending authorization >50 diagnosed patients (12+) not yet enroll
Slide 10: Joenja (leniolisib) franchise – multi-year growth potential
Joenja® (leniolisib) franchise – multi-year growth potential Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) • Marketed (12+) • Global expansion / • Phase II POC trial in regulatory reviews PIDs with immune Joenja® U.S. and Europe / RoW • 83 patients on paid dy
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: 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 13: 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 14: Joenja – looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval Europe – awaiting CHMP Israel marketing authorization opinion on MAA received April 30, 2024 Japan clinical study: Patient U.K., CAN, AUS submissions enrollment is now complete under regulatory review PMDA filing following completion of Approvals in 2024-25* **
Slide 15: 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 16: 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 17: 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 18: PIDs linked to PI3Kδ signaling – patient prevalence
PIDs linked to PI3Kδ signaling – patient prevalence Epidemiology of PIDs linked to PI3K signaling suggests treatable population of ~5/million1 Patients identified to date included in table below Genetic PID Type Publication/cohort/registry Cohort Size NIH protocol cohort ~500 ALPS-FAS ESID registry2
Slide 19: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 20: Financial highlights: 1Q 2024 vs 1Q 2023
Financial highlights: 1Q 2024 vs 1Q 2023 Revenues Gross profit OpEx development RUCONEST® Joenja® R&D G&A M&S Amounts in US$ million Amounts in US$ million 63.8 Amounts in US$ million 55.6 52.7 9.6 47.2 30.2 38.5 27.1 15.1 42.5 46.0 10.0 15.6 18.5 1Q 2023 1Q 2024 1Q 2023 1Q 2024 1Q 2023 1Q 2024 Oper
Slide 21: Revenue breakdown by product and geographic segment
Revenue breakdown by product and geographic segment Amounts in US$ millions 1Q 2024 1Q 2023 RUCONEST® Joenja® Total RUCONEST® Joenja® Total Revenues US 44.8 8.5 53.3 40.9 - 40.9 Europe and RoW 1.2 1.1 2.3 1.6 - 1.6 Total Revenues 46.0 9.6 55.6 42.5 - 42.5 21
Slide 22: Investment in Joenja launch and leniolisib development
Investment in Joenja® launch and leniolisib development 80 Cost category breakdown 1Q23 – 1Q24 in US$ million TOTAL: 73.5 70 TOTAL: 65.8* TOTAL: 63.8 60 10.0* TOTAL: 56.8 TOTAL: 52.7 37.9 50 33.9 30.2 23.9 25.1 40 27.1 30 11.0 10.9 15.1 24.0 20 10.0 10 20.9 20.8 18.5 15.6 11.6 0 1Q23 2Q23 3Q23 4Q23
Slide 23: 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 24: 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 25: 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 26: Appendix
Pharming Group N.V. Appendix
Slide 27: Statement of profit and loss
Statement of profit and loss 27
Slide 28: Balance sheet – assets
Balance sheet — assets Pharming’ Amounts in USS ‘0 cember 31, 2023 March 31, 2024 Non-current assets Intangible assets 68,299 71,267 Property, plant and equipment 9,013 9,689 Right-of-use assets 22,849 23,777 Long-term prepayments 90 92 Deferred tax assets 35,686 29,761 Investment accounted for usin
Slide 29: Balance sheet – liabilities
Balance sheet – liabilities 29
Slide 30: Cash flow (1/2)
Cash flow (1/2) Amounts in $’000 102024 10.2023 loss) before tax (16,624) (16,674) Adjustments to reconcile net profit (loss) to net cash used in operating activities: Depreciation, amortization, impairment of non-current assets 5,921 2,306 Equity settled share based payments 2,427 1,553 Other finan
Slide 31: Cash flow (2/2)
Cash flow (2/2) Pharming’ Amounts in $’000 1Q 2023 Interest received 117 Income taxes received (paid) - (440) Net cash flows generated from (used in) operating activities (7,647) (22,785) Capital expenditure for property, plant and equipment (80) (215) Disposal of investment designated as at FVOCI 1