Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Needham Virtual Healthcare Conference 2023
39 slides from Pharming Group Nv's conference presentation, April 2023
Slide 1: Pharming Group N.V. Needham Virtual Healthcare Conference
Pharming Group N.V. Needham Virtual Healthcare Conference April 17-20, 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: Joenja is now approved
Joenja® is now approved % Pharming’ 35: A NOW Introducing Joenja, the first and only FDA-approved treatment for APDS, a rare primary immune deficiency (P1)'* Sign Up for Updates ))
Slide 7: Joenja – looking ahead
Joenja® ‒ looking ahead Joenja® (leniolisib) is the first and only FDA-approved treatment for APDS Targeted treatment of APDS (activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome) PI3Kδ inhibitor with demonstrated efficacy, safety and tolerability in a 12-week randomized placebo-controlled tr
Slide 8: APDS Overview
APDS Overview
Slide 9: APDS is a rare, primary immunodeficiency first characterized in 2013
APDS is a rare, primary immunodeficiency (PI) first characterized in 2013 APDS affects ~1500 Until now, treatments for APDS have patients* addressed the symptoms of the disease To date, Pharming has which manifest early in childhood, but identified >500 of these not the root cause of APDS patients W
Slide 10: 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 11: 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 p110δ The PI3Kδ enzyme • Sinopulmonary • Herpesvirus (e
Slide 12: Current Management for APDS
Current Management for APDS1,2 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 stem cell transplant APDS,
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)
Joenja® (leniolisib)
Slide 15: FDA approval of Joenja: another win for Pharming
FDA approval of Joenja®: another win for Pharming and a much-needed treatment for patients with APDS 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: 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 17: Joenja addresses the underlying cause of APDS – 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 18: Joenja significantly reduced splenomegaly
Joenja® significantly reduced splenomegaly Secondary endpoint: Significant reductions in at week 12 spleen size by 2D and 3D analysis compared to placebo 27% reduction in 3D spleen volume* • The adjusted mean difference in bidimensional spleen size between Joenja® (n=19) and placebo (n=9) was -13.5
Slide 19: An exploratory end point showed Joenja reduced IgM levels
An exploratory end point showed Joenja® reduced IgM levels Mean serum IgM rapidly reduced to within normal limits • In the Joenja® arm, IgM was 600 elevated above normal limits in 6 patients at baseline, and by week 12 was reduced in all, with 50% returning to 400 within normal limits IgM, mg/dL • I
Slide 20: 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† 37%
Slide 21: 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 22: 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 23: RUCONEST
RUCONEST®
Slide 24: RUCONEST (rhC1INH): durable commercialized asset
RUCONEST® (rhC1INH): durable commercialized asset RUCONEST® sales Return to growth in 2022, US$205.6 million +3% over 2021 The only recombinant treatment Well-tolerated and effective that targets the root cause of HAE treatment option for acute by replacing missing or hereditary angioedema (HAE) - d
Slide 25: C1-INH targets the root cause of HAE
C1-INH targets the root cause of HAE There are 3 known cascades that can lead to an HAE attack Complement System1-6 Contact Activation System1,7-10 Fibrinolysis System8,14 Classical Lectin C1-INH therapy regulates Trigger(s)12 Pathway Pathway FXII multiple pathways11 Immune Complexes • Trauma Plasmi
Slide 26: Strong commitment to HAE community
Strong commitment to HAE community Strong patient organization support since 2000 Over 700 physicians have prescribed RUCONEST® since 2014 >2,000 patients with HAE have been prescribed RUCONEST® 26
Slide 27: Financials and Outlook
Financials and Outlook
Slide 28: Pharming grew sales & investments in leniolisib in 2022
Pharming grew sales & investments in leniolisib in 2022 Revenue in US$ million Gross Profit and Other Income in US$ million Operating costs in US$ million 14.5 33.9 17.6 205.6 2.6 149.2 150.5 188.1 198.9 177.7 2021 2022 2021 2022 2021 2022 Gross Profit Other Income Operating costs leniolisib out of
Slide 29: Outlook 2023
Outlook 2023 Continued low single-digit growth in RUCONEST® revenues Joenja® approved by US FDA March 24, 2023, launch and commercialization April 2023 Positive CHMP opinion in 2H 2023, marketing authorization in Europe ~2 months later* File leniolisib with UK’s MHRA following ECDRP route* Continued
Slide 30: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 31: Appendix
Pharming Group N.V. Appendix
Slide 32: Heterogeneous, evolving symptomology can often lead to missed diagnoses
Heterogeneous, evolving symptomology can often lead to missed diagnoses Timeline of the most common pathologies* seen in APDS1-4 Median age at diagnosis: 12 years (7-year median diagnosis delay) <1 year 3 years 5 years 10.5 years 11.2 years† 18 years (range, 1 month-10 years) (range, 1-6 years) (ran
Slide 33: Joenja clinical trial designs
Joenja® clinical trial designs Pivotal Trial - Pivotal Trial - Part 2: Open-label Part 1: Efficacy extension Dose- & Safety study4,5 finding1,2 Evaluation3 Nonrandomized, open-label, Randomized, triple-blinded, Nonrandomized, open-label, dose-escalating placebo-controlled long-term study 31 patients
Slide 34: Joenja safety profile
Joenja® safety profile Phase 3 Trial1,2 Open-label Extension Study3 Data cutoff for interim analysis: December 13, 2021 Adverse reactions reported by ≥2 patients treated with Joenja and more frequently than placebo • 32/37 patients Most common AEs n Joenja (n=21) Placebo (n=10) n (%) n (%) reported
Slide 35: Joenja commercial launch strategy
Joenja® commercial launch strategy SI 1: Identify SI 2: Educate SI 3: Differentiate Joenja® SI 4: Establish Access Continue to identify HCPs and Build knowledge of APDS and Differentiate Joenja® as a Establish access that enables patients to expand networks with belief in PI3Kδ inhibitor benefits we
Slide 36: Facilitating access through APDS Assist
Facilitating access through APDS Assist Program Offerings Dedicated, Experienced Support Team APDS Assist Care Coordinators Welcomes patient families/caregivers to APDS Assist and Cost & insurance helps navigate coverage, access, and support options Providing insurance coverage assistance and financ
Slide 37: Commitment to rapid access for eligible patients
Commitment to rapid access for eligible patients Starter Program Bridge Program • Available to all newly • Available for patients in which insurance has been enrolled patients verified • Up to 30-day supply within • Provides continuation of one week of enrollment for therapy when there is a gap most
Slide 38: Beginning treatment with JOENJA
Beginning treatment with JOENJA® Commercial Product Available mid-April 2023 APDS Assist patient support services now active www.joenja-hcp.com/APDSAssist 1-877-796-2737 (APDS) Joenja® NDC 71274-170-60 Supplied as 60-count bottle (30-day supply) Wholesale acquisition cost (WAC) Per tablet $750.00 Pe
Slide 39: 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
Pharming Group — Needham Virtual Healthcare Conference 2023 on 6ix