Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q2 2023 Earnings Presentation
20 slides from Vertex Pharmaceuticals's earnings presentation, Aug 1, 2023
Slide 1: Second Quarter 2023 Financial Results
SECOND QUARTER 2023 FINANCIAL RESULTS AUGUST 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: Continued Strong Momentum in the Second Quarter
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 • Continue to build unparalleled portfolio of real-world and long-term data Prepare for potential ne
Slide 5: Executing on Vertex Business Model and R&D Strategy with Rapid Progress on a Robust Pipeline
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 STUDIES TO COMPLETE AT END OF 2023 WITH DATA IN EARLY 2024 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 • Now expec
Slide 7: Near-Term Launch Potential: Exa-cel
NEAR-TERM LAUNCH POTENTIAL: EXA-CEL FDA HAS GRANTED PRIORITY REVIEW FOR EXA-CEL BLA IN SICKLE CELL DISEASE Exa-cel holds potential for one-time, functional cure The first CRISPR-based gene- New EHA data, which were the basis for SCD PDUFA: December 8, 2023 editing treatment potentially to EMA and MH
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 ON TRACK TO COMPLETE PIVOTAL PROGRAM BY THE END OF 2023 Significant Pivotal Near-Term Validated Unmet Program Commercial Target Needs Ongoing Opportunity • Millions in the U.S. each year • NaV1.8 is genetically and • Phase 3 pivota
Slide 9: Type 1 Diabetes: ADA Data Demonstrate Confidence in Foundational VX-880 Cells
TYPE 1 DIABETES: ADA DATA DEMONSTRATE CONFIDENCE IN FOUNDATIONAL VX-880 CELLS 3 PROGRAMS TO ADDRESS ~2.5M T1D PATIENTS IN NORTH AMERICA & EUROPE EDITED, FULLY DIFFERENTIATED, HYPOIMMUNE CELLS • The same cells as VX-880 • Research program continues to progress • $70M milestone to CRSPR Q2 2023 VX-264
Slide 10: Positive VX-880 Results from ADA
POSITIVE VX-880 RESULTS FROM ADA INITIATED PART C WITH CONCURRENT DOSING AT FULL TARGET DOSE Efficacy Highlights Participant 1: Participant 2: • All 6 participants demonstrated: 10 9.9 Participant 3: • Production of endogenous insulin (C- 7.8 Participant 4: 9 8.6 7.3 Participant 5: peptide) HbA1c (%
Slide 11: Inaxaplin: First Potential Medicine to Target the Underlying Cause of AMKD
INAXAPLIN: FIRST POTENTIAL MEDICINE TO TARGET THE UNDERLYING CAUSE OF AMKD 100,000 patients in the U.S. and Europe Image TBD APOL1-MEDIATED PIVOTAL TRIAL RAISING DISEASE AWARENESS KIDNEY DISEASE UNDERWAY AND ONGOING GENETIC TESTING EFFORTS • Two APOL1 variants • On track to complete Phase 2B portion
Slide 12: Clinical Portfolio Is Broad, Diverse and Rapidly Advancing
CLINICAL PORTFOLIO IS BROAD, DIVERSE AND RAPIDLY ADVANCING; RESEARCH PIPELINE PROGRESSING TO DELIVER NEXT WAVE OF INNOVATION Next Wave Phase 1 Phase (1)/2 Pivotal Regulatory Submissions Launched Discovery Research In Healthy Volunteers in Patients Development Completed Vertex hypoimmune Follow-on VX
Slide 13: Sustaining and Expanding Leadership in CF with Serial Innovation
SUSTAINING AND EXPANDING LEADERSHIP IN CF WITH SERIAL INNOVATION RECEIVED EU APPROVAL FOR ORKAMBI FOR AGES 1-2 EXPECT TRIKAFTA/KAFTRIO APPROVALS IN EU, U.K. AND CANADA FOR AGES 2-5 BY END OF 2023 88,000 PATIENTS WITH CF DRIVERS OF GROWTH vs. 83,000 estimated in 2021 1. Treating younger patients and
Slide 14: Near-Term Launch Potential: Exa-cel Access and Reimbursement
NEAR-TERM LAUNCH POTENTIAL: EXA-CEL PROGRESS WITH ACCESS AND REIMBURSEMENT: STRONG INTEREST IN EXA-CEL AND INTENT TO PROVIDE COVERAGE QUICKLY UPON APPROVAL ~24 States with ~90% of SCD/TDT Patients 4 Countries in Europe with ~75% of SCD/TDT Patients COMMERCIAL • Government payers (~65% of U.S. payer
Slide 15: Q2 2023 Financial Highlights
Q2 2023 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q2 22 FY 22 Q2 23 Total CF product revenues $2.20B $8.93B $2.49B TRIKAFTA/KAFTRIO 1.89B 7.69B 2.24B Other CF products 303 1.24B 253 Combined non-GAAP R&D, acquired IPR&D and SG&A expenses 750 3.07B 1.04
Slide 16: Full Year 2023 Updated Financial Guidance
FULL YEAR 2023 UPDATED FINANCIAL GUIDANCE Current Previous FY 2023 Guidance FY 2023 Guidance Commentary Increase primarily reflects the strong uptake of TRIKAFTA/KAFTRIO in multiple countries Total CF Product Revenues $9.7 - $9.8B $9.55 - $9.7B internationally and continued performance of TRIKAFTA i
Slide 17: A Strong Cadence of Catalysts Through Early 2024
A STRONG CADENCE OF CATALYSTS THROUGH EARLY 2024 Recent Highlights Anticipated Key Milestones Expect TRIKAFTA/KAFTRIO approvals in EU, U.K. and Canada in patients ages Received FDA approval in U.S. for TRIKAFTA in patients with CF ages 2 to 5 2-5 years by end of 2023 Fully enrolled vanzacaftor/tezac
Slide 18: Second Quarter 2023 Financial Results
SECOND QUARTER 2023 FINANCIAL RESULTS AUGUST 1, 2023 ©2023 Vertex Pharmaceuticals Incorporated
Slide 19: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q2 22 FY 22 Q2 23 Combined R&D, Acquired IPR&D and SG&A GAAP 877 3.60B 1.16B Non-GAAP 750 3.07B 1.04B Operating income GAAP 1.11B 4.31B 1.03B Non-GAAP 1.19B 4.79B 1.15B Operating Margin %:
Slide 20: R&D Strategy Designed to Deliver Serial Innovation; Clinical-Stage Pipeline
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 — Q2 2023 Earnings Presentation on 6ix