Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Jefferies London Healthcare Conference 2024
22 slides from Pharming Group Nv's conference presentation, Nov 20, 2024
Slide 1: Pharming Group N.V. Jefferies London Healthcare Conference
Pharming Group N.V. Jefferies London Healthcare Conference November 20, 2024 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: Building a leading global rare disease biopharma company
Building a leading global rare disease biopharma company Market RUCONEST® Global approvals and Ongoing pipeline development for acute HAE attacks in key commercialization of and management of rare markets – U.S. focus Joenja® (leniolisib) for APDS disease assets Positive cash flow from RUCONEST® Suc
Slide 4: Pipeline – multiple commercial stage rare disease products
Pipeline – multiple commercial stage rare disease products 4
Slide 5: RUCONEST®
% Pharming’ RUCONEST®
Slide 6: C1-INH targets the root cause of HAE
C1-INH targets the root cause of HAE There are 3 known cascades that can lead to an HAE attack Complement System1-6 Contact Activation System1,7-10 Fibrinolysis System8,14 Classical Lectin C1-INH therapy regulates Trigger(s)12 Pathway Pathway FXII multiple pathways11 Immune Complexes • Trauma Plasmi
Slide 7: C1-INH targets the root cause of HAE: addresses unmet patient needs
C1-INH targets the root cause of HAE Addresses patient needs unmet by other therapies TARGET BRAND GENERIC NAME STATUS TYPE NAME C1 Inhibitor Overview of Marketed and In-Development Therapies and Their Ruconest C1 esterase inhibitor (recombinant) Marketed OD Targets Within the Three Known Cascades L
Slide 8: RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE
RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE The only recombinant treatment 2nd most prescribed product for that targets the root cause of HAE acute attacks by replacing missing or Typical patient: failed icatibant dysfunctional C1-INH (BK inh) and on prophy Tx (K inh) Well-tolerated a
Slide 9: Joenja® (leniolisib)
% Pharming’ Joenja’ (leniolisib)
Slide 10: U.S. launch of Joenja®: first and only approved therapy for APDS
U.S. launch of Joenja®: first and only approved therapy for APDS, corrects the underlying immune defect Joenja® (leniolisib) is a prescription FDA approval (March 2023) based on medicine that is used to treat randomized pivotal study and OLE study activated phosphoinositide 3-kinase U.S. launch (Apr
Slide 11: Joenja® (leniolisib) – strong execution and growth opportunity
Joenja® (leniolisib) – strong execution and growth opportunity Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) Revenues: • Marketed (12+) • Found >870 • Phase II POC trial in • U.S. commercial sales patients globally PIDs with immune • Europe / RoW access progr
Slide 12: Hiding in plain sight: Patient finding strategy
Hiding in plain sight: Patient finding strategy Medical education to raise awareness of APDS Genetic testing VUS resolution and share leniolisib data Conferences and congresses Sponsored, no-cost Validation studies with various Abstracts testing program laboratories to confirm which Assistance from
Slide 13: VUS by the numbers
VUS by the numbers VUSs frustrate patients and doctors, limiting diagnosis of genetic diseases such as APDS Pharming is aware of ~1,200 US patients harboring PIK3CD/R1 VUSs • This figure will continue to grow over time • VUS are identified at ~4x the rate of likely pathogenic/pathogenic (LP/P) varia
Slide 14: Joenja® ‒ geographic / pediatric / indication expansion
Joenja® ‒ geographic / pediatric / indication expansion Europe – CHMP review extended to January 2026 Israel marketing authorization Single outstanding CMC request received April 30, 2024 Positive clinical benefit and safety concluded CAN, AUS submissions U.K. marketing authorization under regulator
Slide 15: Leniolisib for PIDs with immune dysregulation
Leniolisib for PIDs with immune dysregulation Primary Immunodeficiencies (PIDs) are a broad group of disorders with key potential features: • Genetic basis • Immune dysfunction → increased risk of infection • Immune dysregulation → lymphoproliferation and autoimmunity PIDs • High morbidity and morta
Slide 16: PIDs linked to PI3Kδ signaling – Phase II study design
PIDs linked to PI3Kδ signaling – Phase II study design Phase II proof of concept clinical trial – single arm, open- label, dose range-finding study (N=12) 10mg 30mg 70mg • Patients with PIDs linked to PI3Kδ signaling, e.g. Lead Investigator: Gulbu Uzel, M.D., Senior ALPS-FAS1, CTLA4 haploinsufficien
Slide 17: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 18: Financial highlights: 3Q 2024 vs 3Q 2023
Financial highlights: 3Q 2024 vs 3Q 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 74.8 68.0 64.7 66.7 11.2 56.8 6.5 58.4 28.7 25.1 60.2 63.6 10.9 15.3 20.8 20.7 3Q 2023 3Q 2024 3Q 2023 3Q 2024 3Q 2023 3Q
Slide 19: Financial highlights: 9M 2024 vs 9M 2023
Financial highlights: 9M 2024 vs 9M 2023 Revenues Gross profit OpEx development RUCONEST® Joenja® R&D G&A M&S Amounts in US$ million Amounts in US$ million 204.5 Amounts in US$ million 181.3 198.7 31.9 175.3 164.1 146.0 10.3 91.9 86.1 153.8 172.6 31.9 46.0 57.3 60.8 9M 2023 9M 2024 9M 2023 9M 2024 9
Slide 20: 2024 Financial guidance
2024 Financial guidance FY 2024 Revenue Guidance % Growth vs. FY 2023 Total Revenues US$280 - 295 million 14-20% Joenja® significant driver of revenue growth, continued RUCONEST® growth Joenja® revenue assumptions: • Continued growth in patients on paid therapy • Continued high (monthly) adherence (
Slide 21: Pharming 2024 Outlook
Pharming 2024 Outlook Total revenues between US$280 and US$295 million (14% to 20% growth). Joenja® (leniolisib) U.S.: Continued progress finding additional APDS patients, supported by family testing and VUS validation efforts, and subsequently converting patients to paid therapy. Leniolisib ex-U.S.
Slide 22: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
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