Slide 1: Second quarter and first half 2023 financial results
Pharming Group N.V. Second quarter and first half 2023 financial results August 3, 2023 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Speakers
SPEAKERS Sijmen de Vries, MD Anurag Relan, MD Stephen Toor Jeroen Wakkerman Chief Executive Officer Chief Medical Officer Chief Commercial 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 sustainable rare disease business 1H23 updates
Building a sustainable rare disease business 1H23 updates Market RUCONEST® Global approvals and Ongoing pipeline development in all key international commercialization of and management of rare markets – U.S. focus Joenja® (leniolisib) disease assets Positive cash flow from RUCONEST® Advance interna
Slide 6: Pipeline – multiple commercial stage rare disease products
Pipeline – multiple commercial stage rare disease products 6
Slide 7: Pharming strengthens leadership with new Chairman and Chief Business Officer
Pharming strengthens leadership with new Chairman of the Board (nominated) and Chief Business Officer CHAIR Dr. Richard Peters Start date: Upon appointment at upcoming EGM. Nominated as Non-Executive Experience: Over 30 years of experience in the healthcare industry and academia director and new Cha
Slide 8: RUCONEST®: Established commercial business
RUCONEST®: Established commercial business
Slide 9: Strong rare disease product commercial infrastructure
Strong rare disease product commercial infrastructure Dedicated sales force and marketing in U.S., Europe, and MENA Market access teams Patient support and reimbursement teams Disease educators and specialists for APDS and HAE Medical Affairs teams High conference penetration & Support for education
Slide 10: RUCONEST® established commercial business
RUCONEST® established commercial business GLOBAL HQ: LEIDEN, NL UK EEA USA USA HQ: WARREN, NY MENA OFFICE LOCATIONS KEY MARKETS ACCESS MARKETS 10
Slide 11: RUCONEST® (rhC1INH): durable commercialized asset
RUCONEST® (rhC1INH): durable commercialized asset 2Q23: RUCONEST® returned to RUCONEST® sales >US$200m growth (trailing 12 months) Outlook of low single digit revenue growth for 2023 The only recombinant treatment Well-tolerated and effective that targets the root cause of HAE treatment option for a
Slide 12: APDS Joenja® (leniolisib)
CMO Anurag Relan, MD Chief Medical Officer APDS Joenja® (leniolisib)
Slide 13: APDS is a rare, primary immunodeficiency first characterized in 2013
APDS is a rare, primary immunodeficiency (PI) first characterized in 2013 Activated phosphoinositide Until now, treatments for APDS have 3-kinase delta (PI3Kδ) addressed the symptoms of the disease syndrome (APDS) affects which manifest early in childhood, but >1500 patients* not the root cause of A
Slide 14: Joenja®: immune modulator that targets the root cause of APDS
Joenja®: immune modulator that targets the root cause of APDS Joenja® facilitates a balanced PI3Kδ pathway JOENJA WAS DESIGNED TO to support proper immune function6 TARGET THE ROOT CAUSE OF APDS TO HELP NORMALIZE THE HYPERACTIVE PI3Kδ PATHWAY1-5 p85α p110δ Note: Illustration does not include all ste
Slide 15: FDA approval of Joenja®: a much-needed treatment for patients with APDS
FDA approval of Joenja®: a much-needed treatment for patients with APDS and another win for Pharming Joenja® (leniolisib) is a prescription medicine Joenja® reported additional findings that is used to treat activated phosphoinositide from an ongoing long-term open- 3-kinase delta (PI3Kδ) syndrome (
Slide 16: APDS patient finding – genetic testing and VUS resolution
APDS patient finding – genetic testing and VUS resolution All patients with IEI/PID ~200 patients identified with Potential APDS patients with APDS in the U.S. Undiagnosed APDS patients gene VUS Disease state awareness A.I. methods to i.d. APDS Variant of Uncertain Significance patients seeing Immun
Slide 17: Joenja® ‒ looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval Europe – CHMP opinion on UK – MHRA filing expected MAA expected 4Q23 4Q23 (approval ~ 2 months later) (approval ~2 months later) Japan clinical study open for enrollment Regulatory submissions filed in additional markets: First patient expected in CAN, AUS, ISR
Slide 18: EMA update
EMA update May: responses submitted for CHMP Day 120 list of questions July: receives the CHMP’s Day 180 list of outstanding issues CHMP will consult an Ad-hoc Expert Group (AEG) given the rarity of the disease and the unmet medical need for the treatment of APDS patients AEG will be consulted at a
Slide 19: Commercial update
CCO Stephen Toor Chief Commercial Officer Commercial update
Slide 20: RUCONEST® - 1H23 commercial updates
RUCONEST® - 1H23 commercial updates Revenues increased 20% in 2Q23 (US$51.1m) vs 1Q23 2% revenue increase 2Q23 v 2Q22 Performed well in leading revenue indicators in the U.S. including active patients, vials shipped, and number of physicians prescribing Strong U.S. in-market demand – over 70 quarter
Slide 21: U.S. physicians prescribing RUCONEST® continues to grow
U.S. physicians prescribing RUCONEST® continues to grow # of unique U.S. physicians prescribing # of HCPs 2016 2017 2018 2019 2020 2021 2022 2023* *Data thru June 30, 2023 21
Slide 22: Joenja® set up for commercial success
Joenja® set up for commercial success Commercial Field Team Patient Identification Support Services Patient Access Rare Disease Team of 27 • Work with HCPs • Dedicated support, education • Partnered exclusively with focused on to further identify and resources for patients PANTHERx Specialty Allergy
Slide 23: Joenja® value proposition
Joenja® value proposition Innovation: Precision medicine targeting rare and • Pharming is committed to providing patients with genetically-defined patient population rare disease the solutions they need Value: • APDS is a progressive disease • Joenja® designed to treat the root cause of APDS First a
Slide 24: Joenja® commercial updates
Joenja® commercial updates MAR: FDA approval APR: First commercial shipment to patients Strong start to U.S. launch in 2Q23: 60 enrollments, of which 43 patients on paid therapy 19 of ~25 U.S. EAP/OLE patients are now on paid therapy. 24 patients on paid therapy were previously untreated patients or
Slide 25: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 26: Financial highlights: 2Q 2023 vs 2Q 2022
Financial highlights: 2Q 2023 vs 2Q 2022 TOTAL TOTAL REVENUES US$50.1 million REVENUES US$54.9 million 2Q 2022 2Q 2023 GROSS GROSS PROFIT US$46.1 million PROFIT US$49.2 million 2Q 2022 2Q 2023 OPERATING OPERATING COSTS US$(42.4) million COSTS US$(65.8) million 2Q 2022 2Q 2023 OPERATING OPERATING PRO
Slide 27: Financial highlights: 1H 2023 vs 1H 2022
Financial highlights: 1H 2023 vs 1H 2022 TOTAL TOTAL REVENUES US$96.8 million REVENUES US$97.4 million 1H 2022 1H 2023 GROSS GROSS PROFIT US$87.9 million PROFIT US$87.6 million 1H 2022 1H 2023 OPERATING OPERATING COSTS US$(82.2) million COSTS US$(118.5) million 1H 2022 1H 2023 OPERATING OPERATING PR
Slide 28: Quarterly revenue breakdown by product
Quarterly revenue breakdown by product in US$ million TOTAL: 54.9 1Q 22 2Q 22 3Q 22 4Q 22 1Q 23 2Q 23 28
Slide 29: Investment in Joenja® launch and leniolisib development
Investment in Joenja® launch and leniolisib development in US$ million TOTAL: 65.8* TOTAL: 57.5 10.0* TOTAL: 52.7 TOTAL: 44.7 33.9 TOTAL: 42.4 2Q 22 3Q 22 4Q 22 1Q 23 2Q 23 *2Q23 marketing and sales expenses includes US$10M milestone payments paid 29
Slide 30: 2Q 2023: Cashflow March 31, 2023 – June 30, 2023
2Q 2023: Cashflow March 31, 2023 – June 30, 2023 (US$ Million) Cash and cash Net cash flows Net cash flows Net cash flows Exchange rate Cash and cash equivalents on used in operating generated from used in financing effects equivalents on March 31, 2023 activities investing activities activities Jun
Slide 31: Outlook 2023
Outlook 2023 Continued low single digit growth in RUCONEST® revenues Joenja® approved by FDA March 24, 2023, commercializing in U.S. since early April 2023 CHMP opinion in 4Q23, marketing authorization in Europe ~2 months later* File leniolisib with UK’s MHRA following ECDRP route* Continued operati
Slide 32: Q&A
Q&A Sijmen de Vries, MD Anurag Relan, MD Stephen Toor Jeroen Wakkerman Chief Executive Officer Chief Medical Officer Chief Commercial Officer Chief Financial Officer
Slide 33: Presentation, recording and transcript will be made available on the 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 34: Appendix
Pharming Group N.V. Appendix
Slide 35: Statement of profit and loss
Statement of profit and loss 35
Slide 36: Balance sheet – assets
Balance sheet — assets % Pharming |35} June 30,2023) December 31, 2022 Non-current assets Intangible assets 73,413 75,121 Property, plant and equipment 9,910 10,392 Right-of-use assets 29,436 28,753 Long term prepayments 91 228 Deferred tax assets 23 27,010 22,973 Investments accounted for using the
Slide 37: Balance sheet – liabilities
Balance sheet – liabilities 37
Slide 38: Cash flow (1/2)
Cash flow (1/2) % Pharming |35} ts in $’0C 1H 2023 1H 2022 Profit (loss) before tax (13,288) 23,790 Adjustments to reconcile net profit (loss) to net cash used in operating activities: Depreciation, amortization, impairment 5,468 4,263 Equity settled share-based payments 3,970 2,879 Gain on disposal
Slide 39: Cash flow (2/2)
Cash flow (2/2) % Pharming |35} ut 1H 2023 1H 2022 Net cash flows generated from (used in) operating activities (32,406) 4,607 Capital expenditure for property, plant and equipment (986) (729) Proceeds on PRV sale 21,080 - Investment intangible assets - (829) Investment in associate - 7,578 Net cash