Pfizer, Inc. · NYSE:PFE
Pfizer — Investor Day (September 2020)
25 slides from Pfizer's investor day presentation, Sep 15, 2020
Slide 1: Rare Disease
Rare Disease Pfizer Confidential 1
Slide 2: Forward-Looking Statements and Other Notices
Forward-Looking Statements and Other Notices Our discussions during Pfizer’s Investor Day include forward-looking statements about our anticipated future operating and financial performance, business plans and prospects; expectations for our product pipeline, in-line products and product candidates,
Slide 3: Rare Disease Leadership Team
Rare Disease Leadership Team Suneet Varma Brenda Cooperstone, MD Seng Cheng, PhD Global President, Chief Development Officer, Chief Scientific Officer, Rare Disease Rare Disease Rare Disease 3
Slide 4: Leading Innovation in Rare Disease Through Focused Strategy
Leading Innovation in Rare Disease Through Focused Strategy A Leading In-Line Portfolio Expand through Launch Scale to Innovate & Accelerate Rare Hematology Rare Cardiology End-to-End Capabilities Rare Endocrine/Metabolic Rare Neurology in Gene Therapy 3 Gene Therapy Manufacturing Sites 7 Products i
Slide 5: Robust Rare Disease Pipeline With New Molecular Entities in Phase 3 by Year End 2020
Robust Rare Disease Pipeline With 6 New Molecular Entities in Phase 3 by Year End 2020 Rare Endocrine/ Rare Hematology Rare Neurology Rare Cardiology Metabolic In line Growth Hormone Duchenne Muscular LMNA-related Dilated Hemophilia B Deficiency (Somatrogon) Dystrophy Cardiomyopathy (p38 MAPK Inhibi
Slide 6: Robust Rare Disease Pipeline With New Molecular Entities in Phase 3 by Year End 2020
Robust Rare Disease Pipeline With 6 New Molecular Entities in Phase 3 by Year End 2020 Rare Endocrine/ Rare Hematology Rare Neurology Rare Cardiology Metabolic In line Growth Hormone Duchenne Muscular LMNA-related Dilated Hemophilia B Deficiency (Somatrogon) Dystrophy Cardiomyopathy (p38 MAPK Inhibi
Slide 7: Growing Leadership in Rare Cardiology
Growing Leadership in Rare Cardiology Brenda & Harvey 7
Slide 8: Growing Leadership in Rare Cardiology
Growing Leadership in Rare Cardiology • Vyndaqel / Vyndamax for ATTR-Cardiomyopathy In line • Approved US Q2 2019 / EU Q1 2020 • 25 global submissions completed; 9 more planned through 2021 • PF-07265803 for Dilated Cardiomyopathy with lamin A/C gene mutation Phase 3 • Phase III study enrolling Pre-
Slide 9: Driving Treatment and Disease Understanding, Early Diagnosis to Solidify Leadership in ATTR-CM
Driving Treatment and Disease Understanding, Early Diagnosis to Solidify Leadership in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Tafamidis 80 mg Demonstrated 30% Greater Commitment to Understanding Prevalence Survival Benefit Compared with 20 mg1 and Increasing Early Diagnosis Phase 3 ATTR-ACT
Slide 10: PF-07265803 – p38 MAPK Inhibitor in Development for LMNA-related DCM
PF-072658031 – p38 MAPK Inhibitor in Development for LMNA-related Dilated Cardiomyopathy (DCM) • Loss of functional lamin proteins in the setting of Disease Overview LMNA DCM associated with p38 MAPK activation4 • A potent and selective, oral small molecule inhibitor 100+ known mutations in lamin A/
Slide 11: Phase 3 Study Initiated in Patients with LMNA-related DCM Based on Encouraging Phase 2 Results
Phase 3 Study Initiated in Patients with LMNA-related DCM Based on Encouraging Phase 2 Results Phase 21 Demonstrated Sustained Improvement Ongoing Phase 3 Study on 6-Minute Walk Test Distance (6MWD) Primary Endpoint: 69 meters mean absolute change • Randomized, placebo-controlled 24-week study from
Slide 12: An Industry Leading End-to-End Gene Therapy Platform
An Industry Leading End-to-End Gene Therapy Platform Brendan & Colleen 12
Slide 13: An Industry Leading End-to-End Gene Therapy Platform
An Industry Leading End-to-End Gene Therapy Platform Positioned to Launch Three Transformational Gene Therapies by 2023 Discover Develop Deliver • 10 preclinical rAAV programs in • Three Phase 3 programs • $800M investment in in-house rare cardiology, hematology, expected in 2020 manufacturing which
Slide 14: Fidanacogene elaparvovec: Over 4 Years of Follow-up Gene Therapy for Hemophilia B
Fidanacogene elaparvovec: Over 4 Years of Follow-up Gene Therapy for Hemophilia B Patients WW prevalence ~88K; US/JP/EU ~17K1 ABR = 0.4 ABR = 0.8 ABR = 0.4 ABR = 0.0 Projected 2022 Global Market Size ~$2B2 FDA Breakthrough, RMAT & EMA Prime designations Phase 1/2a and Long-Term Follow-Up (LTFU) • 15
Slide 15: Giroctocogene fitelparvovec: Potential Best-in-Class Gene Therapy for Hemophilia A
Giroctocogene fitelparvovec: Potential Best-in-Class Gene Therapy for Hemophilia A Patients WW prevalence ~400K; US/JP/EU ~78K1 # Projected 2023 Global Market Size ~$11B2 FDA Fast Track & RMAT designations Phase 1/2 # • 11 patients across 4 doses with up to 85 weeks of follow-up at Phase 3 dose • Me
Slide 16: DMD Gene Therapy: No SAEs Observed in Additional 9 Boys Dosed
DMD Gene Therapy: No SAEs Observed in Additional 9 Boys Dosed Low Dose High Dose WW prevalence ~140K; US/EU ~30K1 Projected 2023 Global Market Size ~$4B2 Phase 1b • Dosed additional 9 boys at high dose (2E14 vg/kg3) since ASGCT4 (18 total patients), 3 with drug from commercial manufacturing process
Slide 17: Gene Therapy for Wilson Disease IND Planned in 2020
Gene Therapy for Wilson Disease IND Planned in 2020 Widespread Decrease in Copper Diagnosed prevalence ~21K US/EU1 in VTX-801-treated Liver Projected 2026 Global Market Size ~$500M2 • Wilson disease is a chronic, life-threatening disease caused by aberrant accumulation of copper in liver, brain and
Slide 18: Leader in Gene Therapy Manufacturing – Building for Growth
Leader in Gene Therapy Manufacturing – Building for Growth ~300,000 sq ft with Capacity to Fully Supply All Gene Therapy Programs in Pipeline, including DMD at Launch Grade Scale Kit Creek, NC Research & Development GLP 10-250L Early Clinical ~80,000 sq ft GMP 2 x 500L Durham, NC Pivotal Clinical &
Slide 19: Framing the Future Opportunity
Framing the Future Opportunity Michael 19
Slide 20: Significant Clinical and Commercial Opportunities: Rare Cardiology
Significant Clinical and Commercial Opportunities: Rare Cardiology LMNA-related Dilated Cardiomyopathy Vyndaqel/Vyndamax (p38 MAPK Inhibitor) 50K US total prevalence1 100K US total prevalence3 Peak Diagnosis Rate Current Diagnosis Rate ~35% (7-10% at launch) 15% (1% at launch, >35% peak) 40-45% Tre
Slide 21: Significant Clinical and Commercial Opportunities: Gene Therapies
Significant Clinical and Commercial Opportunities: Gene Therapies Hemophilia B Hemophilia A ~17K US / JP / EU total prevalence1 ~78K US / JP / EU total prevalence1 60-65% Target Population (moderate & severe) 60-65% Target Population (moderate & severe) ~30% Eligibility2 ~30% Eligibility2 >80% Acces
Slide 22: Significant Clinical and Commercial Opportunities: Gene Therapies
Significant Clinical and Commercial Opportunities: Gene Therapies Duchenne Muscular Dystrophy Wilson Disease 30K US / EU total prevalence1 21K US / EU total prevalence4 Target Population (failing therapy plus 56% Target Population (boys ages 4-18)2 15-20% those transitioning from stable-to-fail) 75
Slide 23: Our Robust Pipeline Aims to Make a Profound Impact on Pfizer and Patients
Our Robust Pipeline Aims to Make a Profound Impact on Pfizer and the Lives of the Patients We Serve 2019/2020 2021 2022 2023 2024 2025 Vyndaqel Pfizer Rare Disease 1H 2020 Revenue of $1.3B, +36%G* * Operational growth excluding FX 23
Slide 24: Our Robust Pipeline Aims to Make a Profound Impact on Pfizer and Patients
Our Robust Pipeline Aims to Make a Profound Impact on Pfizer and the Lives of the Patients We Serve 2019/2020 2021 2022 2023 2024 2025 Growth Hormone Duchenne Pan Hemophilia LMNA-related Vyndaqel Deficiency Hemophilia B Muscular (Marstacimab) Dilated (Somatrogon) Dystrophy Cardiomyopathy (p38 MAPK I
Slide 25: Q&A
Q&A 25