Slide 1: 9M 2022 Financial Results Analyst Call
Pharming Group N.V. 9M 2022 Financial Results Analyst Call October 27, 2022 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: 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 3: Speakers
SPEAKERS Sijmen de Vries, MD Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Medical Officer Chief Financial Officer
Slide 4: Strategic and operational highlights
CEO Sijmen de Vries, MD Chief Executive Officer Strategic and operational highlights
Slide 5: Pharming's strategic objectives
Pharming’s strategic objectives Building a sustainable business by focusing on RUCONEST® sales Focus on market approval, launch and commercialization of leniolisib in key markets of US, UK and EEA Ongoing pipeline development and management of rare disease assets 5
Slide 6: Continuing to build a sustainable business in rare diseases
Continuing to build a sustainable business in rare diseases Building a sustainable business Anticipated approval and Ongoing pipeline development and by focusing on RUCONEST® sales commercialization of leniolisib management of rare disease assets Positive cash flow from RUCONEST® Successful commerci
Slide 7: Strategic highlights: leniolisib progress (1/2) USA
Strategic highlights: leniolisib progress (1/2) USA Filing and acceptance for Priority Review of New Drug Application to the FDA. Assigned a Prescription Drug User Fee Act (PDUFA) SEP 28 goal date of March 29, 2023 International Classification of Diseases, 10th Revision, Clinical Modification (ICD-1
Slide 8: Strategic highlights: leniolisib progress (2/2) EEA UK
Strategic highlights: leniolisib progress (2/2) EEA UK MHRA granted Promising Pharming receives positive EMA Innovative Medicine (PIM) decision on pediatric investigation designation for the treatment of plan (PIP) for leniolisib in Europe APDS in children 1 year of age to JAN 6 APR 26 less than 18
Slide 9: Strategic highlights: OTL-105 & Pompe
Strategic highlights: OTL-105 & Pompe Progress continues in preclinical studies OTL-105 POMPE Good progress on developing Study into the development of a the lentiviral vector to enhance next-generation alpha-glucosidase C1-inhibitor expression, now therapy for the treatment of testing in preclinica
Slide 10: Pipeline at a glance
Pipeline at a glance % Pharming’ “Ms Commericalized asset - rhC1INH | %, USE Sa Activated Phosphoniositide 3-kinase % leniolisib Delta Syndrome (APDS) In-licensed 7 H Programs SS | 8 Z 10. OTL-105 Additional : Platform % a-Glucosidase Pompe Program - + | eee eee eee
Slide 11: Operational highlights: RUCONEST (conestat alfa)
Operational highlights: RUCONEST® (conestat alfa) Stable revenues. RUCONEST® sales of Will allocate resources US$151.0 million for to leniolisib with view the first 9 months of of accelerating future 2022 growth Safe and effective acute treatment Single digit growth of option for hereditary revenues
Slide 12: APDS and leniolisib
CMO Anurag Relan, MD Chief Medical Officer APDS and leniolisib
Slide 13: Clinical Features of APDS
Clinical Features of APDS Overt Non-malignant Lymphoproliferation Malignant Lymphadenopathy – peripheral, Hepatosplenomegaly Nodular lymphoid hyperplasia of the airway and GI mucosa Lymphoproliferation mediastinal, mesenteric Lymphoma – diffuse large B cell, Hodgkin’s, marginal zone B cell, MALToma,
Slide 14: 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 15: 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 *** 65.80 66.52 Leniolisib Total CD19+ B Cells (%) 61.81 CFB at D85 in Naïve B 40 Ce
Slide 16: Leniolisib over three months was well tolerated
Leniolisib over three months 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 3 6
Slide 17: Leniolisib Reduced Lymphadenopathy
Leniolisib Reduced Lymphadenopathy Indicates start of leniolisib treatment Mean Index Node SPD Part 1 & OLE: Leniolisib 800 Index Lymph Node SPD, cm3 600 400 SCR: 23 x 8mm D85: 20 x 7mm ED252: 16 x 4mm 200 RCT: Placebo 0 Screen End of First OLE Parts 1/2 Readout (Wk 0) (Wk 12) (Wk 36/48) n 24 24 19
Slide 18: Leniolisib Reduced Spleen Size
Leniolisib Reduced 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 36/48) n 25 25 20 SCR: 120
Slide 19: Leniolisib Decreased Elevated IgM
Leniolisib Decreased Elevated IgM 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 includes patients who re
Slide 20: Upcoming milestones for leniolisib
Upcoming milestones for leniolisib* SEP 28: Initiation of Anticipated Anticipated Anticipated FDA accepted NDA pediatric studies FDA regulatory US commercial MHRA regulatory filing with priority recruitment approval launch approval review H2 2022 H1 2023 H2 2023 Anticipated Anticipated OCT 11: EMA r
Slide 21: Financial highlights
CFO Jeroen Wakkerman Chief Financial Officer Financial highlights
Slide 22: Financial highlights
Financial highlights Total revenues up 3% compared to the first 9M 2021 Gross profit increased by 7% to US$139.7 million, driven by growth in revenues, production efficiencies, and favorable tailwind from currency translation effects Net profit increased by 104% compared to first 9M 2021, driven by
Slide 23: Financial highlights: 9M 2022 vs 9M 2021
Financial highlights: 9M 2022 vs 9M 2021 TOTAL TOTAL REVENUES US$146.1 million REVENUES US$151.0 million 9M 2021 9M 2022 GROSS GROSS PROFIT US$130.6 million PROFIT US$139.7 million 9M 2021 9M 2022 OPERATING OPERATING PROFIT US$15.3 million PROFIT US$28.4 million 9M 2021 9M 2022 NET NET PROFIT US$13.
Slide 24: Financial highlights: Revenues 2022 vs 2021
Financial highlights: Revenues 2022 vs 2021 (US$ million) 151.0 146.1 93.2 96.8 43.6 46.6 Q1 2021 H1 2021 9M 2021 Q1 2022 H1 2022 9M 2022 24
Slide 25: First 9M 2022: Profit before tax
First 9M 2022: Profit before tax Sep 30, 2021 – Sep 30, 2022 (US$ Million) (0.3) Profit Growth in Increased Increased Increased 2021 BioConnection Other Profit before tax business R&D G&A M&S OTL-105 sale of shares before tax YTD Q3 2021 gross profit expenditure expenditure expenditure investment YT
Slide 26: First 9M 2022: Cashflow
First 9M 2022: Cashflow January 1, 2022 – September 30, 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
Slide 27: Outlook
Outlook Single digit growth Group revenues from RUCONEST® sales in 2022 Commercial approval of leniolisib from FDA in Q1 2023, with an anticipated launch and commercialization in US in H1 2023. *subject to positive outcomes of the FDA review Positive opinion of leniolisib from the CHMP, followed by
Slide 28: Q&A
Q&A Sijmen de Vries, MD Anurag Relan, MD Jeroen Wakkerman Chief Executive Officer Chief Medical Officer Chief Financial Officer
Slide 29: This presentation, a recording and a transcript will be made available
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 30: Statement of profit and loss
Statement of profit and loss 30
Slide 31: Balance sheet – assets
Non-current assets Intangible assets Property, plant and equipment Right-of-use assets Long-term prepayments Deferred tax assets Investments accounted for using the equity method Investment in equity instruments designated as at FVTOCI Investment in debt instruments designated as at FVTPL Restricted
Slide 32: Balance sheet – liabilities
Balance sheet – liabilities 32
Slide 33: Cash flow (1/2)
Cash flow (1/2) Amounts in $’000 YTD 2022 YTD 2021 Profit before tax 33.069 21.345 Non-cash adjustments: Depreciation, amortization, impairment 6.216 6.867 Equity settled share based payments 4.522 5.706 Fair value gain (loss) on revaluation of derivatives 0 (59) Gain on disposal of investment in as
Slide 34: Cash flow (2/2)
Cash flow (2/2) Pharming’ Capital expenditure for property, plant and equipment (1.071) (7.451) “4 Investment intangible assets (591) (1.544) Investment in equity instruments designated as at FVTOCI 0 (4.589) Investment in associate 7.384 0 Acquisition of license 0 (1.593) Net cash flows used in inv