Pharming Group NV · EURONEXT:PHARM
Pharming Group NV — H.C. Wainwright Global Investment Conference 2024
38 slides from Pharming Group NV's conference presentation, September 2024
Slide 1: H.C. Wainwright – Global Investment Conference
Pharming Group N.V. H.C. Wainwright – Global Investment Conference September 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: Joenja (leniolisib) franchise – multi-year growth potential
Joenja® (leniolisib) franchise – multi-year growth potential Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) • Marketed (12+) • Patients on early • Phase II POC trial in APDS global prevalence: access/ named PIDs with immune ~1.5 patients / million • Significan
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
C1-INH targets the root cause of HAE 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 Leading to HAE Attacks Berinert C1 esterase inhibi
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 U.S. launch: strong commercial execution
Joenja® U.S. launch: strong commercial execution Strong commercial execution 15 months into U.S. launch Continue to enroll and add patients on paid therapy in 2Q24 91 patients on paid therapy at end 2Q24, with 2 additional enrollments pending authorization 2Q24 revenue US$11.1M (+16% vs. 1Q24) 1H24
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 – looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval 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 MHRA decision expected in under regulatory review the four
Slide 15: Joenja (leniolisib) franchise – strong 3-5 year growth potential
Joenja® (leniolisib) franchise – strong 3-5 year growth potential Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) • Marketed (12+) • Found >870 • Phase II POC trial in Joenja® U.S. and Europe / RoW patients globally PIDs with immune • Found >230 of dysregulatio
Slide 16: 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 17: Given importance of PI3Kδ in B & T cells, immune dysregulation in PIDs can occur
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 18: 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 19: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 20: 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 $* millions 200 150 227.1 100 212.2 205.6 189.4 198.9 159.6 50 101.3 17.5 0 2016 2017 2018 2019 2020 2021 2022 2023 RUCONEST® Joenja® • • From FY 2016 – FY 2020 Pharming Group report
Slide 21: Financial highlights: 1H 2024 vs 1H 2023
Financial highlights: 1H 2024 vs 1H 2023 Revenues Gross profit OpEx development RUCONEST® Joenja® R&D G&A M&S Amounts in US$ million Amounts in US$ million 129.7 Amounts in US$ million 134.0 20.7 113.3 118.5 97.4 63.2 3.8 87.6 61.0 109.0 21.0 30.7 93.6 36.5 40.1 1H 2023 1H 2024 1H 2023 1H 2024 1H 20
Slide 22: Joenja revenue breakdown
Joenja® revenue breakdown Quarterly Revenue Half Year Revenue 20.7 2.0 Amounts in US$ million Amounts in US$ million 14.4 0.3 11.1 0.9 18.7 14.1 10.2 3.8 3.8 2Q 2023 2Q 2024 1H 2023 2H 2023 1H 2024 US revenues EU and RoW revenues US revenues EU and RoW revenues 23
Slide 23: 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 adherence (compliance
Slide 24: 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 25: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 26: Appendix
Pharming Group N.V. Appendix
Slide 27: Genetic defect leads to PI3Kδ hyperactivity
APDS is a rare, primary immunodeficiency (PID) Genetic defect leads to PI3Kδ hyperactivity 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 •
Slide 28: APDS is a rare, primary immunodeficiency 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 29: 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 30: 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 31: 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 32: 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 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: 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 35: 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 36: 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 37: Open-label extension interim analysis of days spent with 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† 37%
Slide 38: PIDs linked to PI3Kδ signaling – patient prevalence
PIDs linked to PI3Kδ signaling – patient prevalence Epidemiology of PIDs linked to PI3K signaling suggests treatable population of ~5/million1 Patients identified to date included in table below Genetic PID Type Publication/cohort/registry Cohort Size NIH protocol cohort ~500 ALPS-FAS ESID registry2
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