Slide 1: Full Year 2022 Financial Results
Pharming Group N.V. Full Year 2022 Financial Results March 16, 2023 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Forward-looking statements
Forward-looking statements This press release 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 res
Slide 3: Speakers
SPEAKERS Sijmen de Vries, MD Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Medical Officer Chief Financial Officer
Slide 4: Introduction
CEO Sijmen de Vries, MD Chief Executive Officer Introduction
Slide 5: Pharming – strong base with potential for growth
Pharming – strong base with potential for growth One commercialized asset: Commercialization reach: RUCONEST® (rhC1INH) for the active in over 30 markets, treatment of acute hereditary including the US, the EEA, the angioedema (HAE) UK and MENA Anticipated approvals & launch of Development of rare d
Slide 6: Building a sustainable rare disease business
Building a sustainable rare disease business Market RUCONEST® Anticipated approval and Ongoing pipeline development in all major international commercialization of and management of rare markets leniolisib disease assets Positive cash flow Successful Advance internal from RUCONEST® helps commerciali
Slide 7: Pipeline of rare disease assets
Pipeline of rare disease assets 7
Slide 8: RUCONEST (rhC1INH): durable commercialized asset
RUCONEST® (rhC1INH): durable commercialized asset RUCONEST® sales Return to growth in 2022, US$205.6 million +3% over 2021 The only recombinant treatment Well-tolerated and effective that targets the root cause of HAE treatment option for acute by replacing missing or hereditary angioedema (HAE) - d
Slide 9: APDS, leniolisib
CMO Anurag Relan, MD Chief Medical Officer APDS, leniolisib
Slide 10: Genetic defect leads to PI3Kδ hyperactivity, causing APDS symptoms
Genetic defect leads to PI3Kδ hyperactivity, causing APDS symptoms Hyperactive Excess of immature Deficit of Common Symptoms of APDS[4,5] PI3Kδ activity or senescent cells functional cells Severe, Recurrent, Persistent Infections: • Sinopulmonary • Herpesvirus (especially EBV and CMV) Lymphoprolifer
Slide 11: APDS can impact many facets of life
APDS can impact many facets of life Physical1,2 Frequent infections Social3,4 Swollen glands Missing school, work, or Shortness of breath daily activities Coughing/wheezing Chest or joint pain Fatigue Treatment Burden1-4 Inability to exercise Frequent hospitalizations Hearing loss Surgeries Diarrhea
Slide 12: Current Management for APDS
Current Management for APDS Current APDS Management1,2 Immune Deficiency Immune Dysregulation • Antimicrobial prophylaxis • Corticosteroids • Immunoglobulin • Other immunosuppressants replacement therapy • mTOR inhibitors None of these therapies are FDA- approved for APDS treatment Hematopoietic ste
Slide 13: Leniolisib: a targeted disease modifying treatment for APDS
Leniolisib: a targeted disease modifying treatment for APDS leniolisib[3] [1,2] Leniolisib balances PI3Kδ enzyme activity Addressing immune deficiency and dysregulation 1. Fruman DA, et al. Cell. 2017;170(4):605-635. 2. Okkenhaug K, Vanhaesebroeck B. Nat Rev Immunol. 2003;3(4):317-330. 3. Rao VK, et
Slide 14: Leniolisib: randomized clinical program for a targeted therapy
Leniolisib: randomized clinical program for a targeted therapy Completed Ph2/3 DBPC Registrational Trial Dose-finding, Placebo-controlled, Part 1: Part 2: New Patients3 12-week1,2 12-week2 Leniolisib Leniolisib Placebo No previous 10, 30 and 70 mg bid 70 mg bid bid leniolisib trial N=6 N=21 N=10 N=2
Slide 15: Leniolisib clinical summary – RCT and long-term extension study
Leniolisib clinical summary – RCT and long-term extension study Highly effective therapy addresses underlying cause of APDS Primary Outcomes Other Efficacy Outcomes Increased Decreased Decreased Improved naïve B cells lymphadenopathy spleen size cytopenias Met both primary endpoints (p=0.0002, p=0.0
Slide 16: Safety Data from RCT: Leniolisib was well-tolerated
Safety Data from RCT: Leniolisib was well-tolerated 100 Patients with AEs 90.0 Percentage of Patients, % 85.7 80.0 Grade 1 80 71.4 Grade 2 60 Grade 3 50.0 42.9 Grade 4 40 Study drug related AEs 30.0 30.0 23.8 SAEs 20.0 20 14.3 9.5 9.5 10.0 0 Leniolisib (n=21) Placebo (n=10) Number of AEs 92 65 19 3
Slide 17: Actively developing APDS market opportunity
Actively developing APDS market opportunity Market opportunity with an estimated Partnership with Invitae – a compressive genetic ~1,500 APDS patients* platform – used to find APDS patients in the US >500 patients identified by Pharming to date (as of December 2022 for Australia, Canada, Europe, Jap
Slide 18: Regulatory status: on track for approvals in major markets
Regulatory status: on track for approvals in major markets USA EEA UK Announced FDA accepted NDA Positive EMA decision MHRA grants PIM filing with Priority Review for SEP 28 JAN 6 on Pediatric Investigation APR 6 designation for patients adults and adolescents aged 12 2022 and older 2022 Plan (PIP)
Slide 19: Anticipated milestones for leniolisib
Anticipated milestones for leniolisib* SEP 2022: Initiation of Initiation of FDA accepted NDA FDA regulatory MHRA filing in pediatric studies Japanese filing with priority approval the UK enrollment clinical trial review 2022 1H 2023 2H 2023 OCT 2022: Positive US commercial EMA CHMP MAA submission l
Slide 20: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 21: Financial highlights: FY 2022 vs FY 2021
Financial highlights: FY 2022 vs FY 2021 TOTAL TOTAL REVENUES US$198.9 million REVENUES US$205.6 million FY 2021 FY 2022 GROSS GROSS PROFIT US$177.7 million PROFIT US$188.1 million FY 2021 FY 2022 OPERATING OPERATING COSTS US$166.8 million COSTS US$184.4 million FY 2021 FY 2022 OPERATING OPERATING P
Slide 22: Pharming grew sales & investments in leniolisib
Pharming grew sales & investments in leniolisib Revenue in US$ million Gross Profit and Other Income in US$ million Operating costs in US$ million 14.5 17.6 33.9 205.6 2.6 149.2 150.5 188.1 198.9 177.7 2021 2022 2021 2022 2021 2022 Gross Profit Other Income Operating costs leniolisib out of pocket C
Slide 23: FY 2022: Profit before tax
FY 2022: Profit before tax Dec 31, 2021 – Dec 31, 2022 (US$ Million) Profit One off Increased Increased Increased BioConnection Decrease Other Profit Growth in before tax costs R&D G&A M&S transaction Financial before tax gross profit FY 2021 2021 expenditure expenditure expenditure result FY 2022 2
Slide 24: FY 2022: Cashflow
FY 2022: Cashflow January 1, 2022 – December 31, 2022 (US$ Million) Cash and cash Net cash flows Net cash flows Net cash flows Exchange rate Cash and cash equivalents on generated from generated from generated from effects equivalents on January 1, 2022 (used in) operating (used in) investing (used
Slide 25: Outlook 2023
Outlook 2023 Continued low, single digit growth RUCONEST® revenues US FDA commercial approval 1Q 2023, US launch and commercialization 1H 2023* Positive CHMP opinion in 2H 2023, marketing authorization in Europe ~2 months later* File leniolisib with UK’s MHRA following ECDRP route* To accelerate fut
Slide 26: Q&A
Q&A Sijmen de Vries, MD Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Medical Officer Chief Financial Officer
Slide 27: 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 28: Appendix: leniolisib clinical trial data
Pharming Group N.V. Appendix leniolisib clinical trial data
Slide 29: Leniolisib reduced lymphadenopathy
Leniolisib reduced lymphadenopathy Primary Outcome Analysis* Individual Index Lesion Sizes Change from baseline in index lesions Safety analysis set 0.1 7000 SPD of Index Lymph Node Lesions CFB at D85 in log10 – Transformed 6500 0.0 n=18 n=8 6000 SPD (mm2) 4000 -0.1 -0.02 3000 2000 -0.2 -0.27 1000 0
Slide 30: Leniolisib increased the percentage of naïve B cells out of total B cells
Leniolisib increased the percentage of naïve B cells out of total B cells Primary Outcome Analysis* Mean Percentage of Naïve B Cells Over Time Change from baseline in naïve B cells Safety analysis set p=0.0002 50 80 *** 66.52 65.80 Leniolisib Total CD19+ B Cells (%) 61.81 CFB at D85 in Naïve B Cells
Slide 31: Leniolisib continued to reduce lymphadenopathy
Leniolisib continued to reduce lymphadenopathy Indicates start of leniolisib treatment Mean Index Node SPD Part 1 & OLE: 20 Index Lymph Node SPD (cm2) Leniolisib 15 10 SCR: 23 x 8mm D85: 20 x 7mm ED252: 16 x 4mm 5 RCT: Placebo 0 Screen End of First OLE Parts 1/2 Readout (Wk 0) (Wk 12) (Wk 36/48) n 2
Slide 32: Extension study: continued improvement in spleen size
Extension study: continued improvement in spleen size Indicates start of leniolisib treatment Mean 3D Spleen Volume 700 Part 1 & OLE: Spleen 3D Volume, cm3 Leniolisib 600 500 SCR: 782 mL D85: 501 mL ED252: 467 mL 400 300 RCT: Placebo 0 Screen End of First OLE Parts 1/2 Readout (Wk 0) (Wk 12) (Wk 36/
Slide 33: Extension study: continued reduction in IgM levels
Extension study: continued reduction in IgM levels Mean IgM* 600 All patients IgM, mg/dL 400 Previous leniolisib No previous leniolisib 200 0 ED1 ED84 ED168 ED252 Extension Study Date 23 24 21 21 n 15 17 15 16 8 7 6 5 *Excluded 1 patient due to extremely low B-cell count. Previous Leniolisib include
Slide 34: Extension study: continued reductions in annual infections
Extension study: continued reductions in annual infections despite decreased IRT use in 37% of patients on IRT 45 Annual Rate of Infection Days 40 42.4 Days of infection per patient (m/N) Baseline* Baseline* 35 39.0 38.5 30 Baseline* Baseline* 26.7 25 22.9 20 15 13.8 10 8.7 5 5.5 0 N=28 N=14 N=7 N=4
Slide 35: Long term leniolisib results (N=6)
Long term leniolisib results (N=6) Leniolisib is an investigational new drug that has not been approved for any use. Patients have stopped (*) or decreased (§) immunoglobulin supplementation as a reflection of the normalization of their B cell function. Dashed lines indicate patient not on treatment
Slide 36: Statement of profit and loss
Statement of profit and loss 36
Slide 37: Balance sheet – assets
Balance sheet — assets Pharming’ 4 Non-current assets Intangible assets 75,121 83,834 Property, plant and equipment 10,392 13,222 Right-of-use assets 28,753 19,943 Long-term prepayments 228 194 Deferred tax assets 22,973 21,216 > Investment accounted for using the equity method 2,501 7,201 Investmen
Slide 38: Balance sheet – liabilities
Balance sheet – liabilities 38
Slide 39: Cash flow (1/2)
Cash flow (1/2) Pharming’ 4 Profit before tax 14,987 23,078 Non-cash adjustments: Depreciation, amortization, impairment of non-current assets 13,188 19,610 Equity settled share based payments 6,392 9,056 Gain on disposal of investment in associate (11,057) - Fair value gain (loss) loss on revaluati
Slide 40: Cash flow (2/2)
Cash flow (2/2) Interest received 85 53 Income taxes paid (2,372) - Net cash flows generated from (used in) operating activities 22,897 37,842 Capital expenditure for property, plant and equipment (1,376) (10,739) Investment intangible assets (601) (3,447) Investment associate 7,300 — Investment in