Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Investor Presentation (February 2023)
36 slides from Pharming Group Nv's investor presentation, February 2023
Slide 1: Corporate Overview
Pharming Group N.V. Corporate Overview February 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: Company Overview
Company Overview One commercialized asset: Commercialization reach: RUCONEST® (rhC1INH) for the active in over 30 markets, treatment of acute hereditary including the US, the EEA, the angioedema (HAE) UK and MENA Anticipated approvals & launch of Development of rare disease leniolisib, a PI3Kδ inhib
Slide 4: Leadership: Executive Committee
Leadership: Executive Committee Sijmen de Vries, MD MBA Jeroen Wakkerman Executive Director & CEO Chief Finance Officer CFO Chief Executive Officer Anurag Relan MD Mireille Sanders MSc Chief Medical Officer CMO Chief Operations Officer COO Ruud van Outersterp Stephen Toor Chief Ethics & CECO Chief C
Slide 5: Building a sustainable rare disease business
Building a sustainable rare disease business Market RUCONEST® Anticipated approval and Ongoing pipeline development in all major international commercialization of and management of rare markets leniolisib disease assets Positive cash flow Successful Advance internal from RUCONEST® helps commerciali
Slide 6: Pipeline of rare disease assets
Pipeline of rare disease assets 6
Slide 7: 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 KO
Slide 8: RUCONEST®
RUCONEST®
Slide 9: RUCONEST® (rhC1INH): sustainable commercialized asset
RUCONEST® (rhC1INH): sustainable commercialized asset RUCONEST® sales of US$198.9 Continued single digit growth million (FY2021), US$151.0 million of revenues expected for 2022 (first 9M of 2022) The only recombinant treatment Well-tolerated and effective that targets the root cause of HAE treatment
Slide 10: Importance of C1-INH in Hereditary Angioedema
Importance of C1-INH in Hereditary Angioedema 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 • Trau
Slide 11: Strong commitment to HAE community
Strong commitment to HAE community Strong patient organization support since 2000 Over 700 physicians have prescribed RUCONEST® since 2014 >2,000 patients with HAE have been prescribed RUCONEST® 11
Slide 12: leniolisib
leniolisib
Slide 13: Genetic defect leads to PI3Kδ hyperactivity, causing APDS symptoms
Genetic defect leads to PI3Kδ hyperactivity, causing APDS symptoms Hyperactive Excess of immature Deficit of Common Symptoms of APDS[4,5] PI3Kδ activity or senescent cells functional cells Severe, Recurrent, Persistent Infections: • Sinopulmonary • Herpesvirus (especially EBV and CMV) Lymphoprolifer
Slide 14: 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 15: Current Management for APDS
Current Management for APDS Current APDS Management1,2 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 ste
Slide 16: Leniolisib: a targeted disease modifying treatment for APDS
Leniolisib: a targeted disease modifying treatment for APDS leniolisib[3] [1,2] Leniolisib balances PI3Kδ enzyme activity Addressing immune deficiency and dysregulation 1. Fruman DA, et al. Cell. 2017;170(4):605-635. 2. Okkenhaug K, Vanhaesebroeck B. Nat Rev Immunol. 2003;3(4):317-330. 3. Rao VK, et
Slide 17: Leniolisib clinical development program
Leniolisib clinical development program Completed Ph2/3 DBPC Registrational Trial Dose-finding, Placebo-controlled, Part 1: Part 2: New Patients3 12-week1,2 12-week2 Leniolisib Leniolisib Placebo No previous 10, 30 and 70 mg bid 70 mg bid bid leniolisib trial N=6 N=21 N=10 N=2 2 did not enroll in Ex
Slide 18: Leniolisib clinical summary – RCT and long-term extension study
Leniolisib clinical summary – RCT and long-term extension study Highly effective therapy addresses underlying cause of APDS Primary Outcomes Other Efficacy Outcomes Increased Decreased Decreased Improved naïve B cells lymphadenopathy spleen size cytopenias Met both primary endpoints (p=0.0002, p=0.0
Slide 19: Safety Data from RCT: Leniolisib was well-tolerated
Safety Data from RCT: Leniolisib 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
Slide 20: Actively developing APDS market opportunity
Actively developing APDS market opportunity Market opportunity with an estimated Partnership with Invitae – a compressive genetic ~1,500 APDS patients* platform – used to find APDS patients in the US >500 patients identified by Pharming to date (as of December 2022) Disease educators and patient fin
Slide 21: Regulatory status: on track for approvals in major markets
Regulatory status: on track for approvals in major markets USA EEA UK Announced FDA accepted NDA MHRA grants PIM Positive EMA decision filing with Priority Review for on Pediatric Investigation designation for patients SEP 28 adults and adolescents aged 12 JAN 6 APR 6 2022 and older 2022 Plan (PIP)
Slide 22: Anticipated milestones for leniolisib
Anticipated milestones for leniolisib* SEP 2022: Initiation of FDA accepted NDA FDA regulatory US commercial MHRA filing in pediatric studies filing with priority approval launch the UK enrollment review 2022 1H 2023 2H 2023 OCT 2022: Initiation of Positive MAA submission Japanese EMA CHMP to EMA cl
Slide 23: HAE gene therapy (OTL-105) & Pompe disease programs
HAE gene therapy (OTL-105) & Pompe disease programs 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
Slide 24: Financials and Outlook
Financials and Outlook
Slide 25: Financial highlights (9M 2022)
Financial highlights (9M 2022) US$198.9 million total revenues for FY2021. Revenues for first 9M of 2022 increased by 3% compared to the first 9M 2021 Gross profit increased by 7% to US$139.7 million (9M 2022), driven by growth in revenues, production efficiencies, and favorable tailwind from curren
Slide 26: Total revenues in $ millions
Pharming Group revenues since reacquiring RUCONEST® rights from Valeant Pharmaceuticals Pharming revenues FY 2016 - 9M 2022 250 200 Total revenues in $* millions 150 100 50 0 FY 2016 FY 2017 FY 2018 FY 2019 FY 2020 FY 2021 9M 2022 Reporting Year • • From FY 2016 – FY 2020 Pharming Group reported ear
Slide 27: Outlook
Outlook Single digit growth Group revenues from RUCONEST® sales in 2022 Commercial approval of leniolisib from FDA in 1Q 2023, with an anticipated launch and commercialization in US in 1H 2023.* Positive opinion of leniolisib from the CHMP in 2H 2023, followed by approval of MAA by European Commissi
Slide 28: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 29: Appendix
Pharming Group N.V. Appendix leniolisib clinical trial data
Slide 30: 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 31: 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 *** 66.52 65.80 Leniolisib Total CD19+ B Cells (%) 61.81 CFB at D85 in Naïve B Cells
Slide 32: Leniolisib continued to reduce lymphadenopathy
Leniolisib continued to reduce lymphadenopathy Indicates start of leniolisib treatment Mean Index Node SPD Part 1 & OLE: 20 Index Lymph Node SPD (cm2) Leniolisib 15 10 SCR: 23 x 8mm D85: 20 x 7mm ED252: 16 x 4mm 5 RCT: Placebo 0 Screen End of First OLE Parts 1/2 Readout (Wk 0) (Wk 12) (Wk 36/48) n 2
Slide 33: Extension study: continued improvement in spleen size
Extension study: continued improvement in 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/
Slide 34: Extension study: continued reduction in IgM levels
Extension study: continued reduction in IgM levels 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 include
Slide 35: Extension study: continued reductions in annual infections
Extension study: continued reductions in annual infections despite decreased IRT use in 37% of patients on IRT 45 Annual Rate of Infection Days 40 42.4 Days of infection per patient (m/N) Baseline* Baseline* 35 39.0 38.5 30 Baseline* Baseline* 26.7 25 22.9 20 15 13.8 10 8.7 5 5.5 0 N=28 N=14 N=7 N=4
Slide 36: Long term leniolisib results (N=6)
Long term leniolisib results (N=6) Leniolisib is an investigational new drug that has not been approved for any use. Patients have stopped (*) or decreased (§) immunoglobulin supplementation as a reflection of the normalization of their B cell function. Dashed lines indicate patient not on treatment
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