Pharming Group NV · EURONEXT:PHARM
Pharming Group NV — Van Lanschot Kempen Life Sciences Conference 2024
40 slides from Pharming Group NV's conference presentation, Apr 16, 2024
Slide 1: Van Lanschot Kempen Life Sciences Conference
Pharming Group N.V. Van Lanschot Kempen Life Sciences Conference April 16-17, 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: RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE
RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE The only recombinant treatment that targets the root cause of HAE Second most prescribed product by replacing missing or for acute attacks dysfunctional C1-INH Well-tolerated and effective 97%: needed just 1 dose of treatment option for acut
Slide 8: Strong commitment to HAE community
Strong commitment to HAE community # of unique U.S. physicians prescribing Strong patient organization support 800 since 2000 700 600 # of HCPs 500 More than 720 U.S. physicians (and growing) prescribing RUCONEST® 400 729 300 647 584 508 200 411 >2,000 patients with HAE have been 295 prescribed RUCO
Slide 9: APDS Overview
% Pharming’ APDS Overview
Slide 10: APDS is a rare, primary immunodeficiency (PID) first characterized in 2013
APDS is a rare, primary immunodeficiency (PID) first characterized in 2013 Activated phosphoinositide 3- kinase delta (PI3Kδ) syndrome A genetic test can provide a definitive (APDS) diagnosis of APDS Global prevalence estimated at 1.5 patients per million population* To date, Pharming has identified
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® (leniolisib)
% Pharming’ Joenja’ (leniolisib)
Slide 14: 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 15: U.S. launch of Joenja®: a much-needed treatment for APDS patients
U.S. launch of Joenja®: a much-needed treatment for APDS patients and another achievement for Pharming Joenja® (leniolisib) is a prescription There were no drug-related serious adverse medicine that is used to treat events or study withdrawals in Joenja® trials activated phosphoinositide 3-kinase de
Slide 16: Joenja® U.S. launch: strong commercial execution
Joenja® U.S. launch: strong commercial execution Strong commercial execution 9 months into U.S. launch Continue to enroll patients and add patients on paid therapy in 4Q23 92 enrollments, of which 81 patients on paid therapy at end 4Q23 APDS Assist program ensures eligible patients have access to th
Slide 17: Joenja® ‒ looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval Europe – awaiting CHMP UK – submitted MAA to MHRA opinion on MAA* on March 12, 2024** Japan clinical study: Patient CAN, AUS, ISR submissions enrollment is now complete under regulatory review PMDA filing following completion of Approvals in 2024*** appropriate
Slide 18: Hiding in plain sight: Patient finding strategy
Hiding in plain sight: Patient finding strategy Medical education to raise awareness of APDS Genetic testing Family testing and share leniolisib data Conferences and congresses Sponsored, no-cost testing program Inherited disease* but most APDS Abstracts patients do not have diagnosed family members
Slide 19: Helping diagnose APDS patients: VUS resolution
Helping diagnose APDS patients: Variant of Uncertain Significance (VUS) resolution Genetic testing frequently leads to inconclusive results - previously unseen genetic variants: Patients have clinical symptoms Need to determine if Variant of compatible with APDS, but genetic Frustrating for patients
Slide 20: Primary Immunodeficiencies (PIDs) with immune dysregulation
Primary Immunodeficiencies (PIDs) with immune dysregulation PIDs are a broad group of disorders1 with key features: PIDs Genetic basis, i.e., not secondarily caused by another disease ‘Inborn Errors of Immunity’ (IEI) is used interchangeably with PID An increased risk of infection may be the predomi
Slide 21: PI3Kδ signaling and immune dysregulation in PIDs beyond APDS
Given importance of PI3Kδ in B & T cells, immune dysregulation in PIDs can occur via alterations in PI3Kδ signaling Altered PI3Kδ signaling can occur in multiple Clinical manifestations, disease onset PID genetic disorders beyond APDS and severity similar to APDS 5-8 (ex: ALPS-FAS, CTLA4, PTEN) 1-4
Slide 22: Leniolisib development for PIDs with immune dysregulation
Leniolisib development for PIDs with immune dysregulation Based on APDS experience, leniolisib has potential to be an effective & tolerable chronic treatment approach for PIDs with immune dysregulation Leniolisib, by reducing PI3Kδ activity, should help rebalance immune dysregulation in PIDs, positi
Slide 23: PIDs linked to PI3Kδ signaling – Phase 2 study design
PIDs linked to PI3Kδ signaling – Phase 2 study design Phase 2 proof of concept clinical trial – single arm, open- label, dose range-finding study (N=12) 10mg 30mg 70mg Lead Investigator: Gulbu Uzel, M.D., Senior Research Physician • Patients with PIDs linked to PI3Kδ signaling, e.g. ALPS-FAS, CTLA4
Slide 24: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 25: Financial highlights: FY 2023 vs FY 2022
Financial highlights: FY 2023 vs FY 2022 Revenue Gross profit Other Income 220.1 Other Income 245.3 Grants Divestments in associates 0.2 205.6 188.1 1.8 in US$ million in US$ million PRV sale 0.5 1.8 21.3 12.2 2022 2023 2022 2023 2022 2023 OpEx development Operating profit Net profit (loss) R&D G&A
Slide 26: RUCONEST® and Joenja® driving revenue growth
RUCONEST® and Joenja® driving revenue growth 300 Pharming revenues FY 2016 – FY 2023 250 245.3 18.2 Total revenues in $* millions 200 150 227.1 100 212.2 205.6 189.4 198.9 159.6 50 101.3 17.5 0 2016 2017 2018 2019 2020 2021 2022 2023 RUCONEST® Joenja® • • From FY 2016 – FY 2020 Pharming Group report
Slide 27: Investment in Joenja® launch and leniolisib development
Investment in Joenja® launch and leniolisib development 80 Cost category breakdown 4Q22 – 4Q23 TOTAL: 73.6 70 in US$ million TOTAL: 65.8* 60 TOTAL: 57.4 10.0* TOTAL: 56.8 TOTAL: 52.7 37.9 50 33.9 29.0 23.9 25.1 40 27.1 30 11.0 10.9 24.0 20 17.6 10.0 10 20.9 20.8 15.6 10.9 11.6 0 4Q22 1Q23 2Q23 3Q23
Slide 28: 2024 Revenue guidance
2024 Revenue guidance FY 2024 Revenue Guidance % Growth vs. FY 2023 Total Revenues US$280 - 295 million 14-20% Assumptions Quarterly fluctuations expected Joenja® significant driver of revenue growth, continued RUCONEST® growth Joenja® assumptions: • Continued growth in patients on paid therapy • U.
Slide 29: Pharming 2024 Outlook
Pharming 2024 Outlook Total revenues between US$280 and US$295 million (14% to 20% growth), with quarterly fluctuations expected. Joenja® (leniolisib) U.S.: Continued progress finding additional APDS patients, supported by family testing and VUS validation efforts, and subsequently converting patien
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® 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 34: 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 35: 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 36: 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 37: 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 38: 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 IgM, mg/dL 400 within normal limits • I
Slide 39: 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 Joenja (n=21) Placebo (n=10) • 32/37 patients Most common AEs n n (%) n (%) reported
Slide 40: Recent medical conference presentations (selected)
Recent medical conference presentations (selected) AMCP Nexus - Academy of Managed Care Pharmacy (October 2023) • A Real-world Comparison of Health Care Resource Utilization and Health Care Costs Among Patients With Activated PI3K-Delta Syndrome Versus a Control Cohort of Patients Without Activated
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