Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Q1 2023 Earnings Presentation
39 slides from Pharming Group Nv's earnings presentation, May 11, 2023
Slide 1: 1Q 2023 Financial Results
Pharming Group N.V. 1Q 2023 Financial Results May 11, 2023 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 Stephen Toor Jeroen Wakkerman Chief Executive Officer Chief Medical Officer Chief Commercial Officer Chief Financial Officer
Slide 4: Introduction
CEO Sijmen de Vries, MD Chief Executive Officer Introduction
Slide 5: Building a sustainable rare disease business
Building a sustainable rare disease business Market RUCONEST® Global approvals and Ongoing pipeline development in all major international commercialization of and management of rare markets Joenja® (leniolisib) disease assets Positive cash flow Successful Advance internal from RUCONEST® helps comme
Slide 6: Pipeline – multiple commercial stage rare disease products
Pipeline – multiple commercial stage rare disease products 6
Slide 7: RUCONEST (rhC1INH): durable commercialized asset
RUCONEST® (rhC1INH): durable commercialized asset RUCONEST® sales >US$200m Outlook of single digit revenue (trailing 12 months) growth for 2023 The only recombinant treatment Well-tolerated and effective that targets the root cause of HAE treatment option for acute by replacing missing or hereditary
Slide 8: Strong rare disease product commercial infrastructure
Strong rare disease product commercial infrastructure Dedicated sales force and marketing in US, EU, 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 educational KOL
Slide 9: Joenja is now approved
- Joenja® is now approved % Pharming 3 NOW Introducing Joenja, the first and only FDA-approved treatment for APDS, a rare primary immune deficiency (P1)'* Sign Up for Updates )) |
Slide 10: APDS Joenja (leniolisib)
CMO Anurag Relan, MD Chief Medical Officer APDS Joenja® (leniolisib)
Slide 11: 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 12: 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 13: Genetic defect leads to PI3Kδ hyperactivity, disrupting immune cell balance
Genetic defect leads to PI3Kδ hyperactivity, disrupting immune cell balance Hyperactive PI3Kδ results in dysregulated Immune imbalance leads to diverse signs B and T cell development1-3 and symptoms1,4-6 Severe, recurrent, persistent infections The PI3Kδ enzyme • Sinopulmonary p110δ • Herpesvirus (e
Slide 14: Management for APDS prior to Joenja
Management for APDS1,2 prior to Joenja® 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 stem cell transpla
Slide 15: 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 16: 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 17: Joenja (leniolisib) US label/packaging
Joenja® (leniolisib) US label/packaging Overview of Prescribing Information Indication JOENJA is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta Statement (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Contraindications
Slide 18: Phase 3 co-primary endpoints
Joenja® addresses the underlying cause of APDS to help restore immune balance – Phase 3 co-primary endpoints At 12 weeks Joenja® decreased lymphadenopathy and increased naïve B cells Change from baseline in index nodes* Change from baseline in naïve B cells† Log10-transformed SPD of index lesions Jo
Slide 19: Open-label extension interim analysis of infections and IRT reduction
Open-label extension interim analysis of days spent with infections and IRT reduction Annual Rate of Infection Days* Physician-reported IRT Reduction‡ 45 40 42.4 Of 27 patients receiving IRT: Days with infection per patient (m/N) Baseline† Baseline† 35 39.0 38.5 30 25 Baseline† 26.7 20 Baseline† 22.
Slide 20: Joenja – looking ahead
Joenja® ‒ looking ahead Joenja® launched & Europe – CHMP opinion on reimbursed commercial MAA expected 2H23 shipments to patients (approval ~ 2 months later) commenced early April UK – MHRA filing expected Initiation of Japan clinical 2H23 (approval ~2 months study in 1H23 later) Development ongoing
Slide 21: HAE gene therapy (OTL-105)
HAE gene therapy (OTL-105) Partnership with Orchard Therapeutics to develop ex vivo autologous hematopoietic stem cell gene therapy for HAE Progress continues in preclinical study Good progress on developing the lentiviral vector to enhance C1- inhibitor expression, now testing in preclinical HAE di
Slide 22: Commercial update
CCO Stephen Toor Chief Commercial Officer Commercial update
Slide 23: RUCONEST commercial update
RUCONEST® commercial update Strong underlying in-market Disruptions have since demand for RUCONEST® resolved, but impacted including high, double digit, FEB 2023 February sales new patient enrollments in 1Q23 Disruptions in reimbursement for some patients on U.S. Pharming has since seen a government
Slide 24: 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 25: 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 26: Joenja commercial update
Joenja® commercial update Strong start to our early April product launch Pharming continues to engage with both national and regional payers First commercial shipment of Joenja®, with full reimbursement, ~two weeks after FDA approval To date, we have 23 U.S. patients on paid therapy with Joenja® The
Slide 27: Financials
CFO Jeroen Wakkerman Chief Financial Officer Financials
Slide 28: Financial highlights: 1Q 2023 vs 1Q 2022
Financial highlights: 1Q 2023 vs 1Q 2022 TOTAL TOTAL REVENUES US$46.6 million REVENUES US$42.5 million 1Q 2022 1Q 2023 GROSS GROSS PROFIT US$41.7 million PROFIT US$38.5 million 1Q 2022 1Q 2023 OPERATING OPERATING COSTS US$39.8 million COSTS US$52.7 million 1Q 2022 1Q 2023 OPERATING OPERATING PROFIT
Slide 29: Continued investment in the launch of Joenja
Continued investment in the launch of Joenja Cost category breakdown 1Q22 – 1Q23 60 in US$ million TOTAL: 57.5 TOTAL: 52.7 50 TOTAL: 44.7 TOTAL: 42.4 29 TOTAL: 39.8 40 27.1 20.4 19.3 30 17.2 20 17.6 10 7.7 8.7 12 10 14.9 14.4 15.6 12.3 10.9 0 1Q 22 2Q 22 3Q 22 4Q 22 1Q 23 R&D G&A M&S 29
Slide 30: 1Q 2023: Cashflow January 1, 2023 – March 31, 2023
1Q 2023: Cashflow January 1, 2023 – March 31, 2023 (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, 2023 (used in) operating (used in) investing (used in)
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 Positive CHMP opinion in 2H 2023, marketing authorization in Europe ~2 months later* File leniolisib with UK’s MHRA following ECDRP route* Conti
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
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} mounts in US$ ‘000 March 31, 2023) December 31, 2022 Non-current assets 4 Intangible assets 75,606 75,121 Property, plant and equipment 10,403 10,392 Right-of-use assets 30,369 28,753 Long-term prepayments 233 228 Deferred tax assets 29,350 22,973 Investment ac
Slide 37: Balance sheet – liabilities
Balance sheet – liabilities 37
Slide 38: Cash flow (1/2)
Cash flow (1/2) % Pharming |35} Amounts in USS’ YTD 2023 YTD 2022 “ Profit (loss) before tax (16,674) 4,233 Non-cash adjustments: Depreciation, amortization, impairment of non-current assets 2,306 2,190 Equity settled share based payments 1,558 1,070 Other finance income (123) (3,228) Other finance
Slide 39: Cash flow (2/2)
Cash flow (2/2) % Pharming |35} Interest received (paid) 117 (52)} Income taxes paid (440) - Net cash flows generated from (used in) operating activities (22,785) 609 Capital expenditure for property, plant and equipment (215) (208)| Investment intangible assets - (167) Net cash flows used in invest