Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q4 2022 Earnings Presentation
21 slides from Vertex Pharmaceuticals's earnings presentation, Feb 7, 2023
Slide 1: Fourth Quarter and Full Year 2022 Financial Results
FOURTH QUARTER AND FULL YEAR 2022 FINANCIAL RESULTS FEBRUARY 7, 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 future financial and operating performance, full
Slide 4: Strong Progress in 2022 Sets Up Promise for Expanded Leadership in CF
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 PROGRESS IN • Continue to build unparalleled portfolio of real-world and long-term data 2022
Slide 5: Differentiated Vertex Business Model and R&D Strategy
DIFFERENTIATED VERTEX BUSINESS MODEL AND R&D STRATEGY HAVE DELIVERED TRANSFORMATIVE CYSTIC FIBROSIS MEDICINES AND 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
Slide 6: Continuing to Serially Innovate in Cystic Fibrosis
CONTINUING TO SERIALLY INNOVATE IN CYSTIC FIBROSIS Vanzacaftor Triple VX-522 • Next-in-class CFTR modulator triple therapy • CFTR mRNA approach in development for more than 5,000 CF patients who cannot benefit from • Holds potential for greater clinical benefit CFTR modulators • Convenient, once-dai
Slide 7: Near-Term Launch Potential: Exa-cel
NEAR-TERM LAUNCH POTENTIAL: EXA-CEL DELIVERING A POTENTIAL ONE-TIME, FUNCTIONAL CURE FOR SICKLE CELL DISEASE (SCD) AND TRANSFUSION-DEPENDENT BETA THALASSEMIA (TDT) PATIENTS Sickle Cell Disease and Beta Thalassemia Exa-cel holds potential for one-time, functional cure Genetic diseases caused by mutat
Slide 8: Near-Term Launch Potential: VX-548
NEAR-TERM LAUNCH POTENTIAL: VX-548 ADDRESSING CRITICAL GAP IN TREATMENT OF MODERATE TO SEVERE ACUTE PAIN Significant Pivotal Near-Term Validated Unmet Program Commercial Needs Target Opportunity Ongoing • Millions in the U.S. each year • NaV1.8 is genetically and • Phase 3 program similar to Phase 2
Slide 9: Inaxaplin Is in Pivotal Development for AMKD
INAXAPLIN IS IN PIVOTAL DEVELOPMENT; POTENTIAL FIRST MEDICINE TO TREAT THE UNDERLYING CAUSE OF APOL1-MEDIATED KIDNEY DISEASE (AMKD) Phase 2B (n=66) Phase 3 (n=400) Enrolling patients Small molecule targeting the Dose 1 underlying cause of AMKD Selected dose of inaxaplin (VX-147) • Single, adaptive P
Slide 10: Type 1 Diabetes: Advancing Potentially Curative Treatments
TYPE 1 DIABETES: ADVANCING POTENTIALLY CURATIVE TREATMENTS ~2.5 MILLION PEOPLE LIVING WITH T1D IN NORTH AMERICA AND EUROPE HYPOIMMUNE CELLS Stem cell-derived islets with hypoimmune gene editing In preclinical development VX-264: CELLS + DEVICE Stem cell-derived islets with encapsulation Canadian CTA
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 Initiated 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 INCREASING EPIDEMIOLOGY ESTIMATES TO 88,000 PATIENTS WITH CF 88,000 PATIENTS WITH CF DRIVERS OF GROWTH vs. 83,000 estimated in 2021 U.S., Europe, Australia and Canada 1. More people with CF, living longer Median predicted age of surviv
Slide 13: Near-Term Launch Potential: Exa-cel Specialty Model
NEAR-TERM LAUNCH POTENTIAL: MULTI-BILLION $ OPPORTUNITY, EXA-CEL SPECIALTY MODEL THE POTENTIAL FOR A ONE-TIME, FUNCTIONAL ~24 States with ~90% of SCD/TDT Patients CURE IS A COMPELLING OPTION FOR PATIENTS Severe, symptomatic diseases • Highly symptomatic with frequent hospitalizations due to vaso-occ
Slide 14: VX-548: Potential Key Role in Broad Treatment of Acute Pain
VX-548: POTENTIAL FOR KEY ROLE IN BROAD TREATMENT OF ACUTE PAIN, WITHOUT LIMITATIONS OF OPIOIDS, INCLUDING ABUSE POTENTIAL SIGNIFICANT UNMET NEED Opioids VX-548 Many patients unable to tolerate NSAIDs, acetaminophen Effective pain relief Highly addictive Without abuse potential Constipation Ineffect
Slide 15: Near-Term Launch Potential: VX-548 in Acute Pain
NEAR-TERM LAUNCH MULTI-$B MARKET OPPORTUNITY ACCESSIBLE POTENTIAL: VX-548 IN VIA VERTEX SPECIALTY MODEL ACUTE PAIN Large existing U.S. market, despite being highly generic • Acute pain ~$4B market today (with 90% of prescriptions generic) • 1.5B treatment days Specialty market given concentration of
Slide 16: Q4 and Full Year 2022 Financial Highlights
Q4 AND FULL YEAR 2022 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q4 21 FY 21 Q1 22 Q2 22 Q3 22 Q4 22 FY 22 Total CF product revenues $2.07B $7.57B $2.10B $2.20B $2.33B $2.30B $8.93B TRIKAFTA/KAFTRIO 1.69B 5.70B 1.76B 1.89B 2.01B 2.02B 7.69B SYMDEKO/SYMK
Slide 17: Full Year 2023 Financial Guidance
FULL YEAR 2023 FINANCIAL GUIDANCE FY 2023 FY 2022 Actuals FY 2023 Guidance Commentary Continued performance of TRIKAFTA in ages 6+, Total CF Product Revenue $8.9B $9.55 - $9.7B approval and launch of TRIKAFTA in ages 2-5 Combined GAAP R&D, Acquired IPR&D and SG&A $3.60B $4.35 - $4.6B Includes expect
Slide 18: Progress in 2022 Sets Up Meaningful Clinical Catalysts in 2023
PROGRESS IN 2022 SETS UP MEANINGFUL CLINICAL CATALYSTS IN 2023 Recent Highlights Anticipated Key Milestones Submitted global regulatory filings of TRIKAFTA in patients with CF ages 2 to 5 Priority Review granted; PDUFA April 28 Fully enrolled vanzacaftor/tezacaftor/deutivacaftor Phase 3 studies Comp
Slide 19: Fourth Quarter and Full Year 2022 Financial Results
FOURTH QUARTER AND FULL YEAR 2022 FINANCIAL RESULTS FEBRUARY 7, 2023 ©2023 Vertex Pharmaceuticals Incorporated
Slide 20: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q4 21 FY 21 Q1 22 Q2 22 Q3 22 Q4 22 FY 22 Combined R&D, Acquired IPR&D, and SG&A GAAP 950 3.89B 818 877 921 984 3.60B Non-GAAP 830 3.44B 687 750 758 872 3.07B Operating income GAAP 878 2.7
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
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