Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Oppenheimer 34th Annual Healthcare Life Sciences Conference (February 2024)
28 slides from Pharming Group Nv's conference presentation, Feb 13, 2024
Slide 1: Oppenheimer 34th Annual Healthcare Life Sciences Conference
Pharming Group N.V. Oppenheimer 34th Annual Healthcare Life Sciences Conference February 13, 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® Adv
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) for HAE: still growing after 10+ years
RUCONEST® (rhC1INH) for HAE: still growing after 10+ years The only recombinant treatment that targets the root cause of HAE Second most prescribed product by replacing missing or detailed for acute attacks dysfunctional C1-INH Well-tolerated and effective 97%: needed just 1 dose of treatment option
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 first characterized in 2013
APDS is a rare, primary immunodeficiency (PID) first characterized in 2013 Activated phosphoinositide 3- Until now, treatments for APDS have kinase delta (PI3Kδ) syndrome addressed the symptoms of the disease (APDS) Global prevalence estimated at 1.5 which manifest early in childhood, but patients p
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 patients with APDS
U.S. launch of Joenja®: a much-needed treatment for patients with APDS and another win 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 delta
Slide 16: Joenja® launch update: continued strong commercial execution
Joenja® launch update: continued 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
Slide 17: Joenja® ‒ looking beyond FDA approval
Joenja® ‒ looking beyond FDA approval Europe – CHMP opinion on UK – MHRA filing expected MAA expected 1Q24 1Q24** (approval ~ 2 months later)* AUS, CAN, ISR submissions Japan clinical study – under regulatory review 1st patient enrolled Aug 2023 CAN & AUS approval 2Q24*** ISR approval 1H24*** Pediat
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 alterations 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 (ex: ALPS-FAS, CTLA4, PTEN) Lymphopr
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 Ph2 (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, CT
Slide 24: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 25: Updated FY 2023 Guidance (preliminary and unaudited)
Updated FY 2023 Guidance (preliminary and unaudited)* FY 2023 Revenue Guidance % Growth (preliminary and unaudited)* vs. FY 2022 RUCONEST® ~ US$227 million 10% Joenja® ~ US$18 million N/A Total ~ US$245 million 19% RUCONEST® guidance increased from previous low single digit revenue growth Cash and c
Slide 26: RUCONEST® and Joenja® driving revenue growth
RUCONEST® and Joenja® driving revenue growth ~245 ~ ~ • From FY 2016 – FY 2020 Pharming Group reported earnings in EUR. Revenues during this time frame have been converted to USD. In 2021, Pharming Group began reporting earnings in USD. • 4Q 2020 and 1Q 2021 quarterly fluctuations and volatility fro
Slide 27: Pharming summary and outlook
Pharming summary and outlook Significantly increased RUCONEST® 2023 revenue guidance to ~US$227M (10% growth) Joenja® launched early April 2023 – 81 patients on paid therapy & expect ~US$18M revenues in 2023 Leniolisib CHMP opinion expected in 1Q24, marketing authorization in Europe ~2 months later*
Slide 28: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
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