Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — IEX Beleggersdag June 2023
23 slides from Pharming Group Nv's conference presentation, June 2023
Slide 1: Pharming Group N.V. IEX Beleggersdag
Pharming Group N.V. IEX Beleggersdag June 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: 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 4: Pipeline – multiple commercial stage rare disease products
Pipeline – multiple commercial stage rare disease products 4
Slide 5: 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 6: RUCONEST®
% Pharming’| 35} RUCONEST®
Slide 7: 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 8: 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 9: APDS Overview
% Pharming’|35! APDS Overview
Slide 10: 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 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: 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 13: 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 14: Joenja® (leniolisib)
% Pharming’| 35} Joenja’ (leniolisib)
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: Phase 3 co-primary endpoints: Immune Dysregulation
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 17: Open-label extension interim analysis of infection days 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 18: 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 Initiation of Japan clinical UK – MHRA filing expected study in 1H23 (Orphan Drug 2H23 (approval ~2 months Designation O
Slide 19: 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 20: Financials and Outlook
% Pharming’| 35} Financials and Outlook
Slide 21: Continued investment in the launch of Joenja
Continued investment in the launch of Joenja in US$ million TOTAL: 57.5 TOTAL: 52.7 TOTAL: 44.7 TOTAL: 42.4 TOTAL: 39.8 1Q 22 2Q 22 3Q 22 4Q 22 1Q 23 21
Slide 22: 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 23: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM