Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Investor Presentation (May 2024)
39 slides from Pharming Group Nv's investor presentation, May 2024
Slide 1: Corporate Presentation
Pharming Group N.V. Corporate Presentation May 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: 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 access/ named PIDs with immune • Significant portion patient programs dysregulation linked
Slide 6: RUCONEST
% Pharming’ 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): 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 9: Joenja (leniolisib)
% Pharming’ Joenja’ (leniolisib)
Slide 10: 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 11: APDS is a rare, primary immunodeficiency (PID)
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 The PI3Kδ enzyme • Sinopulmonary p110δ •
Slide 12: 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 13: 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+) • Global expansion / • Phase II POC trial in regulatory reviews PIDs with immune Joenja® U.S. and Europe / RoW • Found >220 of dysregul
Slide 14: Joenja U.S. launch: strong commercial execution
Joenja® U.S. launch: strong commercial execution Strong commercial execution 12 months into U.S. launch Continue to enroll and add patients 83 patients on paid therapy at end 1Q24, with 5 additional enrollments pending authorization >50 diagnosed patients (12+) not yet enrolled and >50 pediatric FY2
Slide 15: 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 16: Joenja ‒ looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval Europe – awaiting CHMP Israel marketing authorization opinion on MAA received April 30, 2024 Japan clinical study: Patient U.K., CAN, AUS submissions enrollment is now complete under regulatory review PMDA filing following completion of Approvals in 2024-25* **
Slide 17: 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 18: Immune dysregulation in PIDs can occur via alterations in PI3Kδ signaling
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 19: 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 20: 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
Slide 21: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 22: 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 $* millions 200 150 227.1 100 212.2 205.6 189.4 198.9 159.6 50 101.3 17.5 0 2016 2017 2018 2019 2020 2021 2022 2023 RUCONEST® Joenja® • • From FY 2016 – FY 2020 Pharming Group report
Slide 23: Financial highlights: 1Q 2024 vs 1Q 2023
Financial highlights: 1Q 2024 vs 1Q 2023 Revenues Gross profit OpEx development RUCONEST® Joenja® R&D G&A M&S Amounts in US$ million Amounts in US$ million 63.8 Amounts in US$ million 55.6 52.7 9.6 47.2 30.2 38.5 27.1 15.1 42.5 46.0 10.0 15.6 18.5 1Q 2023 1Q 2024 1Q 2023 1Q 2024 1Q 2023 1Q 2024 Oper
Slide 24: Revenue breakdown by product and geographic segment
Revenue breakdown by product and geographic segment Amounts in US$ millions 1Q 2024 1Q 2023 RUCONEST® Joenja® Total RUCONEST® Joenja® Total Revenues US 44.8 8.5 53.3 40.9 - 40.9 Europe and RoW 1.2 1.1 2.3 1.6 - 1.6 Total Revenues 46.0 9.6 55.6 42.5 - 42.5 24
Slide 25: 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 26: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 27: Appendix
Pharming Group N.V. Appendix
Slide 28: RUCONEST - Strong commitment to HAE community
RUCONEST® - 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 740 U.S. physicians (and growing) prescribing RUCONEST® 400 729 744 300 647 584 508 200 411 >2,000 patients with HAE have been 295
Slide 29: 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 30: 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 31: 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 32: 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 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: 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 35: 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 36: 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 37: 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 38: 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 39: 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