Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Q4 2024 Earnings Presentation
33 slides from Pharming Group Nv's earnings presentation, Mar 13, 2025
Slide 1: Fourth quarter and full year 2024 financial results and business update
Pharming Group N.V. Fourth quarter and full year 2024 financial results and business update March 13, 2025 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Speakers
SPEAKERS Fabrice Chouraqui 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 Fabrice Chouraqui Chief Executive Officer Introduction
Slide 5: History of growth and innovation at Pharming
History of growth and innovation at Pharming UK MHRA approves Joenja® FDA accepts priority Pharming completes Pharming acquires Start of Phase II trial of review of NDA acquisition of Abliva leniolisib for APDS leniolisib for PIDs with for leniolisib immune dysregulation Start of Phase II trial of P
Slide 6: Vision for the future
Vision for the future Develop a leading global rare disease company with a diverse portfolio and presence in large markets, leveraging proven and efficient clinical development, supply chain, and commercial infrastructure 6
Slide 7: Strong fourth quarter and full year 2024 performance
Strong fourth quarter and full year 2024 performance reinforces growth foundation for the future Revenues: FY24: US$297 million (+21%) 4Q24: US$93 million (+14%) EURONEXT AMS: PHARM Nasdaq: PHAR Operating profit and positive operating cash flow in 3Q-4Q 2024 Growing commercial RUCONEST® for acute HA
Slide 8: Commercial update
CCO Stephen Toor Chief Commercial Officer Commercial update
Slide 9: Growing our commercial portfolio
Growing our commercial portfolio RUCONEST® Joenja® Strong U.S. in-market demand Increasing APDS patients on therapy U.S. physician prescriber base +11% in FY24 Found >240 in the U.S. and >880 globally New enrollments up 24% in FY24 Paid therapy: 96 patients + 5 pending (U.S.) Additional 188 patients
Slide 10: RUCONEST: 2nd most prescribed therapy for acute HAE attacks in the US
RUCONEST® (rhC1INH): 2nd most prescribed therapy for acute HAE attacks in the US Only recombinant treatment that targets the root cause of HAE by replacing C1-INH Only recombinant treatment that acts at multiple points in the cascades leading to HAE attacks 97% patients needed just 1 dose1 93% acute
Slide 11: RUCONEST addresses patient needs unmet by other therapies
RUCONEST® addresses patient needs unmet by other therapies Type 1, Type 2, and Normal C1-INH HAE patients rely on RUCONEST RUCONEST patients experience moderate to severe Time of taking 24 hours after 4 hours after attacks, and attack more frequently RUCONEST RUCONEST patients switching from other a
Slide 12: Joenja (leniolisib) – Reaching more APDS patients and expanding addressable population
Joenja® (leniolisib) – Reaching more APDS patients and expanding the addressable patient population Potential total prevalence ~40 patients / million Genetic PIDs prevalence +7.5 patients / million APDS prevalence ~1.5 patients / million Joenja for CVID Joenja for PIDs with immune VUS resolution wit
Slide 13: R&D Update
CMO Anurag Relan, MD Chief Medical Officer R&D Update
Slide 14: Expanding rare disease pipeline
Expanding rare disease pipeline 14
Slide 15: Finding more APDS patients - VUS focus
Finding more APDS patients - VUS focus Variants of Uncertain VUS study results Significance VUSs: insufficient data to High throughput screening (MAVE) determine if variant is disease study, completed in December, causing identified novel variants leading to PI3Kδ hyperactivity >1200 patients in the
Slide 16: Pediatric APDS clinical trial results support regulatory filings
Pediatric APDS clinical trial results support regulatory filings Pediatric Phase III trial for children 4-11 years old with APDS Positive topline data announced December 2024 21 patients enrolled in U.S., Europe, and Japan Both co-primary endpoints show improvement consistent with the RCT in adolesc
Slide 17: Leniolisib for PIDs with immune dysregulation beyond APDS
Leniolisib for PIDs with immune dysregulation beyond APDS PI3Kδ activity drives immune dysregulation in PIDs, providing strong rationale for development of leniolisib All PIDs PIDs with immune dysregulation linked to PI3Kδ signaling PIDs with immune dysregulation • Multiple genetically identifiable
Slide 18: Joenja development status
Joenja® development status Expanding the addressable patient population Geographic expansion Pediatric expansion Indication expansion (APDS) (APDS) (additional PIDs) Europe – CHMP review extended to PIDs with immune dysregulation January 2026 4 to 11 years – Positive topline data Single outstanding
Slide 19: KL1333 for mtDNA mitochondrial disease (Abliva acquisition)
KL1333 for mtDNA mitochondrial disease (Abliva acquisition) Primary mitochondrial diseases – rare disorders impairing mitochondrial energy production • Severe fatigue, myopathy (muscle weakness), and reduced life expectancy • Poor quality of life (e.g., loss of job, social isolation, depression) KL1
Slide 20: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 21: Financial highlights: 4Q 2024 vs 4Q 2023
Financial highlights: 4Q 2024 vs 4Q 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 80.5 92.7 74.1 81.2 13.1 73.6 73.9* 7.9 27.0 37.9 73.3 79.6 24.6 24.1 22.3 11.6 4Q 2023 4Q 2024 4Q 2023 4Q 2024 4Q 2023 4Q
Slide 22: Financial highlights: FY 2024 vs FY 2023
Financial highlights: FY 2024 vs FY 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 261.8 297.2 220.1 272.6 245.3 45.0 248.8 18.2 118.8 124.0 227.1 252.2 70.6 55.9 68.9 83.2 FY 2023 FY 2024 FY 2023 FY 2024
Slide 23: Abliva acquisition completed
Abliva acquisition completed Acquisition Terms Financial Details Timing Acquisition through a public Acquisition of shares with Announced ownership exceeding tender offer under Swedish available cash 90% on February 20 and initiated Takeover Act and Nasdaq delisting activities KL1333 in-licensed by
Slide 24: 2025 Financial guidance
2025 Financial guidance Revenue and operating expenses: FY 2025 Guidance Notes Total Revenues US$315 - 335 million 6 - 13% growth Operating Expenses Flat vs. FY 2024 (pre-Abliva impact) Operating Expenses Preliminary estimate including R&D and non- ~US$30 million (Abliva-related) recurring transacti
Slide 25: Building a leading global rare disease biopharma company
Building a leading global rare disease biopharma company Growing commercial RUCONEST for acute HAE attacks portfolio Joenja (leniolisib) for APDS – U.S. and EU/Rest of World Additional patient finding Reaching more APDS Targeted geographic expansion patients with Joenja Pediatrics label expansion Pi
Slide 26: Q&A
Q&A Fabrice Chouraqui 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’ jecember 31, 2024 December 31, 2023 Non-current assets Intangible assets 61,039 71,267 Property, plant and equipment 7,752 9,689 Right-of-use assets 16,382 23,777 Long-term prepayments 90 92 Deferred tax assets 31,090 29,761 Investment accounted for using the equity
Slide 31: Balance sheet – liabilities
Balance sheet – liabilities 31
Slide 32: Cash flow (1/2)
Cash flow (1/2) Pharming’ f 1 $'000 2024 2023 Profit (loss) before tax (8,513)| (12,012) Adjustments to reconcile net profit (loss) to net cash used in operating activities: Depreciation, amortization, impairment of non-current assets 16,070 15,925 Equity settled share based payments 11,248 9,251 Fa
Slide 33: Cash flow (2/2)
Cash flow (2/2) Pharming’ Interest received 5,201 2,883 Income taxes received (paid) (15,584)| (655) Net cash flows generated from (used in) operating activities (1,793)| (17,302) Capital expenditure for property, plant and equipment (790) (1,437) Proceeds on PRV sale - 21,279 Investment intangible