Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q3 2023 Earnings Presentation
20 slides from Vertex Pharmaceuticals's earnings presentation, Nov 6, 2023
Slide 1: Third Quarter 2023 Financial Results
THIRD QUARTER 2023 FINANCIAL RESULTS NOVEMBER 6, 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 Third Quarter: Continue to Drive Execution Across the Company
Continue the journey in cystic fibrosis (CF) • Serially innovate to bring highly efficacious therapies to all CF patients Prepare for potential near-term commercialization opportunities • Exa-cel: SCD PDUFA: December 8, 2023; TDT PDUFA: March 30, 2024; regulatory decisions in the EU and U.K. expecte
Slide 5: Continuing Our Serial Innovation in Cystic Fibrosis
CONTINUING OUR SERIAL INNOVATION IN CYSTIC FIBROSIS ON TRACK TO COMPLETE VANZACAFTOR TRIPLE STUDIES BY THE 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 CFT
Slide 6: Near-Term Launch Potential: Exa-cel
NEAR-TERM LAUNCH POTENTIAL: EXA-CEL FDA ADVISORY COMMITTEE COMPLETED FOR SCD, NOW APPROACHING REGULATORY DECISIONS FROM U.S., U.K., AND EU IN THE NEAR TERM Exa-cel holds potential for one-time, functional cure The first CRISPR-based SCD PDUFA: December 8, 2023 Updated clinical data in TDT gene-editi
Slide 7: VX-548 for Acute and Neuropathic Pain
VX-548 FOR ACUTE AND NEUROPATHIC PAIN HOLDS THE PROMISE OF EFFECTIVE PAIN RELIEF WITHOUT THE SIDE EFFECTS OR ADDICTIVE PROPERTIES OF OPIOIDS ACUTE PAIN: PIVOTAL PROGRAM TO COMPLETE BY END OF 2023 WITH DATA IN EARLY 2024 PNP: PHASE 2 DPN TRIAL COMPLETE, PHASE 2 LSR TRIAL TO INITIATE BY END OF 2023 •
Slide 8: Type 1 Diabetes: Advancing Potentially Curative Treatments
TYPE 1 DIABETES: ADVANCING POTENTIALLY CURATIVE TREATMENTS FOR ~2.5M PATIENTS IN NORTH AMERICA & EUROPE PART C FULLY ENROLLED IN VX-880 TRIAL EDITED, FULLY DIFFERENTIATED, HYPOIMMUNE CELLS • The same cells as VX-880 • Research program continues to progress VX-264: FULLY DIFFERENTIATED CELLS + DEVICE
Slide 9: 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 • Phase 2B dose-ranging portion of the
Slide 10: Clinical Portfolio Is Broad, Diverse and Rapidly Advancing
CLINICAL PORTFOLIO IS BROAD, DIVERSE AND RAPIDLY ADVANCING Select, Next Wave Phase 1 Phase (1)/2 Pivotal Regulatory Submissions Launched Research-stage Programs in Healthy Volunteers in Patients Development Completed Vertex hypoimmune VX-880 VX-548 Exa-cel Follow-on cells Type 1 diabetes Acute Pain
Slide 11: Sustaining and Expanding Leadership in CF with Serial Innovation
SUSTAINING AND EXPANDING LEADERSHIP IN CF WITH SERIAL INNOVATION RECEIVED TRIKAFTA APPROVAL IN CANADA FOR AGES 2-5 EXPECT KAFTRIO APPROVALS IN EU AND U.K. 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 securing addi
Slide 12: Exa-cel Market Represents a Potential Multi-$B Opportunity
EXA-CEL MARKET REPRESENTS A POTENTIAL MULTI-$B OPPORTUNITY 32,000 eligible patients with severe disease in the U.S. and Europe Additional opportunity ~24 States with ~90% of 4 Countries in Europe with ~75% of The Kingdom of Saudi Arabia SCD/TDT Patients SCD/TDT Patients COMM COMMERCIAL ERCIAL • On t
Slide 13: 2024 Will Be a Foundational Year for Exa-cel
2024 WILL BE A FOUNDATIONAL YEAR FOR EXA-CEL GOAL TO DELIVER TRANSFORMATIVE PATIENT OUTCOMES WITH THE POSSIBILITY OF A LIFETIME OF BENEFIT Each stage can take several months Stage 1: Stage 2: Stage 3: Patient referral & Cell collection & Treatment preparation manufacturing • Patient referred to • Mo
Slide 14: VX-548 Has Potential to Play a Key Role in Acute and Peripheral Neuropathic Pain Markets
VX-548 HAS POTENTIAL TO PLAY A KEY ROLE IN ACUTE AND PERIPHERAL NEUROPATHIC PAIN MARKETS ACUTE PAIN AND PNP ARE EACH MULTI-$B MARKETS TODAY 80 MILLION PATIENTS 10 MILLION PATIENTS every year in the U.S. every year in the U.S. Acute pain Peripheral neuropathic pain • More than 2/3 of patients receive
Slide 15: Q3 2023 Financial Highlights
Q3 2023 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q3 22 FY 22 Q3 23 Total CF product revenues $2.33B $8.93B $2.48B TRIKAFTA/KAFTRIO 2.01B 7.69B 2.27B Other CF products 324 1.24B 209 Combined non-GAAP R&D, acquired IPR&D and SG&A expenses 758 3.07B 993
Slide 16: Raising Full Year 2023 Product Revenue Guidance
RAISING FULL YEAR 2023 PRODUCT REVENUE GUIDANCE Current Previous FY 2023 Guidance FY 2023 Guidance Commentary Includes expectations in the U.S. for continued performance of TRIKAFTA in ages 6+ and the Total CF Product Revenues ~$9.85B $9.7 - $9.8B launch of TRIKAFTA in the 2-5 age group, as well as
Slide 17: Multiple Catalysts Through Early 2024
MULTIPLE CATALYSTS THROUGH EARLY 2024 Recent Highlights Anticipated Key Milestones Received positive CHMP opinion in patients with CF ages 2 to 5 TRIKAFTA/KAFTRIO approvals in EU and U.K. in ages 2-5 years by end of 2023 Received approval from Health Canada for TRIKAFTA in ages 2 to 5 Secure reimbur
Slide 18: Third Quarter 2023 Financial Results
THIRD QUARTER 2023 FINANCIAL RESULTS NOVEMBER 6, 2023 ©2023 Vertex Pharmaceuticals Incorporated
Slide 19: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q3 22 FY 22 Q3 23 Combined R&D, Acquired IPR&D and SG&A GAAP 921 3.60B 1.13B Non-GAAP 758 3.07B 993 Operating income GAAP 1.13B 4.31B 1.04B Non-GAAP 1.29B 4.79B 1.17B Operating Margin %: G
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
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