Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q1 2023 Earnings Presentation
21 slides from Vertex Pharmaceuticals's earnings presentation, May 1, 2023
Slide 1: First Quarter 2023 Financial Results
FIRST QUARTER 2023 FINANCIAL RESULTS MAY 1, 2023 ©2023 Vertex Pharmaceuticals Incorporated
Slide 2: Agenda
AGENDA Introduction Susie Lisa, CFA, Senior Vice President, Investor Relations CEO Perspective and Pipeline Update Reshma Kewalramani, M.D., Chief Executive Officer and President Commercial Update Stuart Arbuckle, Executive Vice President and Chief Operating Officer Financial Results Charlie Wagner,
Slide 3: Safe Harbor Statement & Non-GAAP Financial Measures
SAFE HARBOR STATEMENT & NON-GAAP FINANCIAL MEASURES This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the information provided regarding expectations for future financial and operating
Slide 4: Strong Start to 2023: Delivering on Our Goals
Continue the journey in cystic fibrosis (CF) • Serially innovate to bring highly efficacious CFTRm to all eligible patients • Reach the last ~5,000 patients (ineligible for a CFTRm) with mRNA therapy STRONG START TO 2023: • Continue to build unparalleled portfolio of real-world and long-term data DE
Slide 5: Executing on Vertex Business Model and R&D Strategy
EXECUTING ON VERTEX BUSINESS MODEL AND R&D STRATEGY WITH RAPID PROGRESS ON A ROBUST PIPELINE FIVE POTENTIAL LAUNCHES IN THE NEXT FIVE YEARS We focus on 4 Approved • Validated targets that medicines in Investment of majority address causal human cystic fibrosis of OpEx in R&D and biology BD/external
Slide 6: Continuing Our Serial Innovation in Cystic Fibrosis
CONTINUING OUR SERIAL INNOVATION IN CYSTIC FIBROSIS Vanzacaftor Triple VX-522 • Next-in-class CFTR modulator triple therapy • CFTR mRNA therapy in development for ~5,000 CF patients who cannot benefit from CFTR • Completed enrollment in pivotal program in patients ages modulators 12+ in late 2022, a
Slide 7: Near-Term Launch Potential: Exa-cel
NEAR-TERM LAUNCH POTENTIAL: EXA-CEL COMPLETED ROLLING BLA SUBMISSIONS IN THE U.S. FOR SICKLE CELL DISEASE AND TRANSFUSION- DEPENDENT BETA THALASSEMIA Sickle Cell Disease and Beta Thalassemia • Causal human biology well understood Genetic diseases caused by mutation in the beta-globin gene • Severe,
Slide 8: Near-Term Launch Potential: VX-548 for Moderate to Severe Acute Pain
NEAR-TERM LAUNCH POTENTIAL: VX-548 FOR MODERATE TO SEVERE ACUTE PAIN ANTICIPATE COMPLETING THE PHASE 3 PIVOTAL PROGRAM BY LATE 2023/EARLY 2024 Significant Pivotal Near-Term Validated Unmet Program Commercial Target Needs Ongoing Opportunity • Millions in the U.S. each year • NaV1.8 is genetically an
Slide 9: Inaxaplin: First Potential Medicine for APOL1-Mediated Kidney Disease
INAXAPLIN: THE FIRST POTENTIAL MEDICINE TO TREAT THE UNDERLYING CAUSE OF APOL1-MEDIATED KIDNEY DISEASE (AMKD) RECENT NEJM ARTICLES UNDERSCORE SIGNIFICANCE OF INAXAPLIN DATA 100,000 patients in the U.S. and Europe APOL1-MEDIATED FIRST PUBLICATION FOR INAXAPLIN PIVOTAL TRIAL UNDERWAY KIDNEY DISEASE •
Slide 10: Type 1 Diabetes: Advancing Fully Differentiated Cells as Curative Treatments
TYPE 1 DIABETES: ADVANCING FULLY DIFFERENTIATED CELLS AS CURATIVE TREATMENTS FOR ~2.5 MILLION PATIENTS IN NORTH AMERICA AND EUROPE EDITED, FULLY DIFFERENTIATED, HYPOIMMUNE CELLS • The same cells as VX-880 • Licensing agreement with CRISPR will enable use of CRISPR/Cas9 editing of cells • Research pr
Slide 11: Clinical Portfolio Is Broad, Diverse and Rapidly Advancing
CLINICAL PORTFOLIO IS BROAD, DIVERSE AND RAPIDLY ADVANCING; RESEARCH PIPELINE PROGRESSING TO DELIVER NEXT WAVE OF INNOVATION Phase 1 Phase 1/2 Pivotal Regulatory Submissions Next Wave Launched in Healthy Volunteers in Patients Development Completed Vertex hypoimmune Follow-on VX-880 VX-548 Exa-cel c
Slide 12: Sustaining and Expanding Leadership in CF with Serial Innovation
SUSTAINING AND EXPANDING LEADERSHIP IN CF WITH SERIAL INNOVATION RECENTLY RECEIVED FDA APPROVAL FOR TRIKAFTA IN PATIENTS AGES 2-5 YEARS IN THE U.S. 88,000 PATIENTS WITH CF DRIVERS OF GROWTH vs. 83,000 estimated in 2021 U.S., Europe, Australia and Canada 1. Treating younger patients and securing addi
Slide 13: Near-Term Launch Potential: Exa-cel Launch Preparedness
NEAR-TERM LAUNCH POTENTIAL: EXA-CEL LAUNCH PREPAREDNESS WORK FOCUSES ON KEY STAKEHOLDERS Patients Providers Payers ATC • Initial launch will focus on • Clear recognition of different • Commercial payers (~35% of U.S. payer ~32,000 most severe patients genetic therapy approaches mix2): working to ens
Slide 14: Q1 2023 Financial Highlights
Q1 2023 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q1 22 FY 22 Q1 23 Total CF product revenues $2.10B $8.93B $2.37B TRIKAFTA/KAFTRIO 1.76B 7.69B 2.10B Other CF products 336 1.24B 278 Combined non-GAAP R&D, acquired IPR&D and SG&A expenses 687 3.07B 1.21
Slide 15: Strategic Collaborations Augment Our Internal Innovation
STRATEGIC COLLABORATIONS AUGMENT OUR INTERNAL INNOVATION TRANSACTIONS COMPLETED IN Q1:23 Discovery and development of intracellular Research collaboration for new tRNA-based therapies Endosomal Escape Vehicle (EEV™) therapeutics for for patients with Duchenne muscular dystrophy myotonic dystrophy ty
Slide 16: Reiterate Full Year 2023 Financial Guidance
REITERATE FULL YEAR 2023 FINANCIAL GUIDANCE Current FY 2023 FY 2023 Guidance Commentary Continued performance of TRIKAFTA in Total CF Product Revenue $9.55 - $9.7B ages 6+, approval and launch of TRIKAFTA in ages 2-5 Combined GAAP R&D, Acquired $4.35 - $4.6B IPR&D and SG&A Expenses No change to full
Slide 17: Industry Leading Culture of Innovation and Inclusion
INDUSTRY LEADING CULTURE OF INNOVATION AND INCLUSION WILL SUSTAIN LONG-TERM SUCCESS Improve the Lives of People Foster an Ethical Culture Carefully Manage Our Make a Positive Impact With Serious Diseases That Embraces Innovation, Operations and in the Communities Where Inclusion, Diversity and Equit
Slide 18: Strong Start to 2023 Sets Up Cadence of Clinical Catalysts
STRONG START TO 2023 SETS UP CADENCE OF CLINICAL CATALYSTS Recent Highlights Anticipated Key Milestones in 2023 Received FDA approval in U.S. for TRIKAFTA in patients with CF ages 2 to 5 TRIKAFTA/KAFTRIO global launch in patients ages 2-5 years Fully enrolled vanzacaftor/tezacaftor/deutivacaftor Pha
Slide 19: First Quarter 2023 Financial Results
FIRST QUARTER 2023 FINANCIAL RESULTS MAY 1, 2023 ©2023 Vertex Pharmaceuticals Incorporated
Slide 20: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q1 22 FY 22 Q1 23 Combined R&D, Acquired IPR&D and SG&A GAAP 818 3.60B 1.33B Non-GAAP 687 3.07B 1.21B Operating income GAAP 1.04B 4.31B 779 Non-GAAP 1.17B 4.79B 902 Operating Margin %: GAA
Slide 21: R&D Strategy Designed to Deliver Serial Innovation
R&D STRATEGY DESIGNED TO DELIVER SERIAL INNOVATION WITH HIGH PROBABILITY OF SUCCESS; CLINICAL-STAGE PIPELINE IS BROAD, DEEP AND ADVANCING RESEARCH PHASE 1 PHASE 2 PHASE 3 APPROVED KALYDECO / ORKAMBI / SYMDEKO / TRIKAFTA vanzacaftor/tezacaftor/deutivacaftor Cystic Fibrosis Additional Small Molecules
Vertex Pharmaceuticals — Q1 2023 Earnings Presentation on 6ix