Slide 1: Second quarter and first half 2024 financial results and business update
Pharming Group N.V. Second quarter and first half 2024 financial results and business update August 1, 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: Disease overview
Disease overview 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 Leading to HAE Attacks Berinert C1 esterase inhibitor (human) Marketed
Slide 7: 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 APDS global prevalence: access/ named PIDs with immune ~1.5 patients / million • Significan
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 U.S. launch: strong commercial execution
Joenja® U.S. launch: strong commercial execution Strong commercial execution 15 months into U.S. launch Continue to enroll and add patients on paid therapy in 2Q24 91 patients on paid therapy at end 2Q24, with 2 additional enrollments pending authorization 2Q24 revenue US$11.1M (+16% vs. 1Q24) 1H24
Slide 11: Joenja (leniolisib) franchise – strong 3-5 year growth potential
Joenja® (leniolisib) franchise – strong 3-5 year growth potential Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) • Marketed (12+) • Found >870 • Phase II POC trial in Joenja® U.S. and Europe / RoW patients globally PIDs with immune • Found >230 of dysregulatio
Slide 12: Joenja (leniolisib) for APDS, leniolisib for PIDs
CMO Anurag Relan, MD Chief Medical Officer Joenja® (leniolisib) for APDS leniolisib for PIDs
Slide 13: U.S. launch of Joenja: first and only approved therapy for APDS
U.S. launch of Joenja®: first and only approved therapy for APDS, corrects the underlying immune defect Joenja® (leniolisib) is a prescription FDA approval (March 2023) based on medicine that is used to treat randomized pivotal study and OLE study activated phosphoinositide 3-kinase U.S. launch (Apr
Slide 14: 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 15: VUS by the numbers
VUS by the numbers VUSs frustrate patients and doctors, limiting diagnosis of genetic diseases such as APDS Pharming is aware of ~1,200 US patients harboring PIK3CD/R1 VUSs • This figure will continue to grow over time • VUS are identified at ~4x the rate of likely pathogenic/pathogenic (LP/P) varia
Slide 16: Joenja – looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval 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 MHRA decision expected in under regulatory review the four
Slide 17: 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 18: 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 19: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 20: Financial highlights: 2Q 2024 vs 2Q 2023
Financial highlights: 2Q 2024 vs 2Q 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 74.1 65.8 70.1 66.1 11.1 54.9 32.9 33.9 3.8 49.2 63.0 11.0 15.6 51.1 20.9 21.6 2Q 2023 2Q 2024 2Q 2023 2Q 2024 2Q 2023 2Q
Slide 21: Financial highlights: 1H 2024 vs 1H 2023
Financial highlights: 1H 2024 vs 1H 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 129.7 134.0 20.7 113.3 118.5 97.4 63.2 3.8 87.6 61.0 109.0 21.0 30.7 93.6 36.5 40.1 1H 2023 1H 2024 1H 2023 1H 2024 1H 20
Slide 22: Joenja revenue breakdown
Joenja® revenue breakdown Quarterly Revenue Half Year Revenue 20.7 2.0 Amounts in US$ million Amounts in US$ million 14.4 0.3 11.1 0.9 18.7 14.1 10.2 3.8 3.8 2Q 2023 2Q 2024 1H 2023 2H 2023 1H 2024 US revenues EU and RoW revenues US revenues EU and RoW revenues 22
Slide 23: 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 adherence (compliance
Slide 24: 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 25: 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 26: 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 27: Appendix
Pharming Group N.V. Appendix
Slide 28: Statement of profit and loss
Statement of profit and loss 28
Slide 29: Balance sheet – assets
Balance sheet — assets Pharming™ Non-current assets Intangible assets 66,572 71,267 Property, plant and equipment 8,617 9,689 Right-of-use assets 22,107 23,777 Long-term prepayments 90 92 Deferred tax assets 13 39,049 29,761 Investment accounted for using the equity method 12 1,404 2,285 Investments
Slide 30: Balance sheet – liabilities
Balance sheet – liabilities 30
Slide 31: Cash flow (1/2)
Cash flow (1/2) Pharming’ 1H 2024 1H 2023 Profit (loss) before tax (16,684) (13,288)| Adjustments to reconcile net profit (loss) to net cash used in operating activities: Depreciation, amortization, impairment of non-current assets 5,628 5,468 Equity settled share based payments 5,687 3,970 Fair val
Slide 32: Cash flow (2/2)
Cash flow (2/2) Pharming’ Capital expenditure for property, plant and equipment (294) (986) Proceeds on PRV sale = 21,080 Disposal of investment designated as at FVOCI 1,964 — Purchases of marketable securities (112,453) (87,347) Proceeds from sale of marketable securities 147,841 - Net cash flows g