Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q4 2023 Earnings Presentation
27 slides from Vertex Pharmaceuticals's earnings presentation, Feb 5, 2024
Slide 1: Fourth Quarter and Full Year 2023 Financial Results
FOURTH QUARTER AND FULL YEAR 2023 FINANCIAL RESULTS FEBRUARY 5, 2024 ©2024 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, including, without limitation, the information provided regarding and expectations for future financial and operating performa
Slide 4: Vertex Delivered Excellent Q4:23 Results
Expand our leadership and raise the bar in CF • Increased epidemiology estimates for people living with cystic fibrosis from ~88,000 to ~92,000 • North America, Europe, Australia VERTEX • Shared positive pivotal data for vanzacaftor triple from two head-to-head studies (SKYLINE 102 and 103) in patie
Slide 5: Vanzacaftor Triple Pivotal Program Results
VANZACAFTOR TRIPLE PIVOTAL PROGRAM RESULTS ©2024 Vertex Pharmaceuticals Incorporated 2
Slide 6: SKYLINE 102, 103: Met Primary Endpoint, Non-Inferiority on ppFEV1
SKYLINE 102, 103 (12+ YEARS): FOLLOWING 4-WEEK RUN-IN WITH TRIKAFTA TO ESTABLISH BASELINE (BL), PATIENTS RANDOMIZED TO TRIKAFTA OR VANZA TRIPLE → MET PRIMARY ENDPOINT: NON-INFERIORITY ON PPFEV1 ∆ FROM BL SKYLINE 102 SKYLINE 103 TRIKAFTA Vanza Triple TRIKAFTA Vanza Triple N=202 N=196 N=289 N=284 67.2
Slide 7: 1st Key Secondary Endpoint: Change in Sweat Chloride
1ST KEY SECONDARY ENDPOINT: ABSOLUTE CHANGE IN SWEAT CHLORIDE (SwCl) →TREATMENT WITH VANZA TRIPLE WAS SUPERIOR TO TRIKAFTA SKYLINE 102 SKYLINE 103 TRIKAFTA Vanza Triple TRIKAFTA Vanza Triple N=202 N=196 N=289 N=284 54.3 53.6 42.1 43.4 Baseline SwCl; mean (SD) (18.2) (17.0) (17.9) (18.5) Absolute cha
Slide 8: 2nd Key Secondary Endpoint: Patients SwCl <60 mmol/L
2ND KEY SECONDARY ENDPOINT: PROPORTION PATIENTS SwCl <60 mmol/L →TREATMENT WITH VANZA TRIPLE WAS SUPERIOR TO TRIKAFTA Pooled Endpoint SKYLINE 102 and SKYLINE 103 TRIKAFTA Vanza Triple N=491 N=480 Baseline SwCl <60 mmol/L, proportion, % patients 74% 76% SwCl <60 mmol/L through Week 24* Proportion, %
Slide 9: 3rd Key Secondary Endpoint: Patients SwCl <30 mmol/L
3RD KEY SECONDARY ENDPOINT: PROPORTION PATIENTS SwCl <30 mmol/L →TREATMENT WITH VANZA TRIPLE WAS SUPERIOR TO TRIKAFTA Pooled Endpoint SKYLINE 102 and SKYLINE 103 TRIKAFTA Vanza Triple N=491 N=480 Baseline SwCl <30 mmol/L, proportion, % patients 21% 19% SwCl <30 mmol/L through Week 24* Proportion, %
Slide 10: RIDGELINE (6-11 Years) Study Results
RIDGELINE (6-11 YEARS) STUDY: FOLLOWING 4-WEEK RUN-IN WITH TRIKAFTA TO ESTABLISH BASELINE, PATIENTS TREATED WITH VANZA TRIPLE; 95% OF CHILDREN ACHIEVED SwCl <60 mmol/L AND 53% ACHIEVED CARRIER LEVELS OF SwCl WITH VANZA TRIPLE SwCl <60 mmol/L RIDGELINE SwCl <30 mmol/L RIDGELINE Vanza Triple Vanza Tri
Slide 11: Common TEAEs Consistent with Underlying CF Across Treatment Groups
COMMON TEAES (≥10% IN ANY TREATMENT GROUP) (POOLED) WERE CONSISTENT WITH UNDERLYING CF AND ACROSS TREATMENT GROUPS 445-102/105 SKYLINE 102 AND 103 52 wks TRIKAFTA Vanza Triple TRIKAFTA N=491 N=480 N=403 n (%) n (%) Subjects with any TEAEs 469 (95.5) 459 (95.6) 399 (99.0) Infective PEx 158 (32.2) 133
Slide 12: Expanding Leadership in CF and Raising the Bar with Serial Innovation
EXPANDING LEADERSHIP IN CF AND RAISING THE BAR WITH SERIAL INNOVATION • Delivered positive results in all three Phase 3 studies: • SKYLINE 102 and 103 studies in ages 12+ Vanzacaftor triple combination • RIDGELINE study in ages 6+ • On track to file in U.S., Europe, Canada by mid-2024 for ages 6+ VX
Slide 13: CASGEVY (exa-cel): Beginning of New Era of Diversification
CASGEVY (EXA-CEL): BEGINNING OF NEW ERA OF DIVERSIFICATION The first precise, durable, CRISPR/Cas9 gene-edited therapy, delivering a potential one-time functional cure for patients with SCD and TDT Rapid pace of global approvals: • Approved for eligible patients ages 12+ with sickle cell disease or
Slide 14: VX-548 Acute Pain Pivotal Program Results
VX-548 ACUTE PAIN PIVOTAL PROGRAM RESULTS ©2024 Vertex Pharmaceuticals Incorporated 9
Slide 15: VX-548 Showed Stat Sig Pain Relief Compared to Placebo
VX-548 FOR ACUTE PAIN: TREATMENT WITH VX-548 SHOWED STAT SIG PAIN RELIEF COMPARED TO PLACEBO IN BOTH RANDOMIZED CONTROLLED TRIALS Primary endpoint: SPID48 VX-548 vs. placebo 548-105 ABDOMINOPLASTY 548-104 BUNIONECTOMY PLACEBO VX-548 PLACEBO VX-548 100 mg, 50 mg q12h 100 mg, 50 mg q12h N=223 N=447 N=
Slide 16: Reduction in Acute Pain with VX-548 Rapid, Sustained and Clinically Meaningful
REDUCTION IN ACUTE PAIN WITH VX-548 WAS RAPID, SUSTAINED AND CLINICALLY MEANINGFUL IN BOTH TRIALS 548-105 ABDOMINOPLASTY 548-105 ABDOMINOPLASTY Placebo (N=223) PLACEBO HB/APAP VX-548 MEAN CHANGE FROM BASELINE 0.0 HB/APAP (N=448) 5 mg / 325 mg q6h 100 mg, 50 mg q12h VX-548 (N=447) N=223 N=448 N=447 -
Slide 17: VX-548 Safe and Well Tolerated in >2,400 Patients
VX-548 WAS SAFE AND WELL TOLERATED IN THESE TRIALS WITH >2,400 PATIENTS WITH MODERATE TO SEVERE ACUTE PAIN Results support broad moderate to severe acute pain label • Across all three studies, the majority of AEs with VX-548 were mild or moderate in severity VX-548 • Across all three studies, there
Slide 18: Next Steps for Our Pain Program
NEXT STEPS FOR OUR PAIN PROGRAM • Results from pivotal program support a broad moderate-to-severe acute pain label • On track to file NDA in the U.S. by mid-2024 Acute Pain • Advance VX-993 into Phase 2 study in acute pain – oral formulation • Initiate VX-993 Phase 1 study in acute pain – intravenou
Slide 19: Clinical Portfolio Is Broad, Diverse and Rapidly Advancing
CLINICAL PORTFOLIO IS BROAD, DIVERSE AND RAPIDLY ADVANCING STRONG PROGRESS TOWARDS OUR GOAL OF FIVE LAUNCHES OVER FIVE YEARS (2028) Select, Next Wave Phase 1 Phase (1)/2 Pivotal Regulatory Submissions Approved Research-stage Programs in Healthy Volunteers in Patients Development Completed or Underwa
Slide 20: Expanding Leadership in CF and Raising the Bar with Serial Innovation
EXPANDING LEADERSHIP IN CF AND RAISING THE BAR WITH SERIAL INNOVATION ~92,000 DURABLE GROWTH DRIVERS Best-in-class patients with CF* Treating younger patients Goal: carrier levels Patients living longer medicines of CFTR function Serial CFTRm innovation ~20,000 mRNA for last >5,000 patients
Slide 21: CASGEVY Represents a Potential Multi-$B Opportunity
CASGEVY REPRESENTS A POTENTIAL MULTI-$B OPPORTUNITY FOR VERTEX 2024 A FOUNDATIONAL YEAR; STRONG PROGRESS ACROSS ALL REGIONS WITH PAYERS, PATIENTS, PHYSICIANS 35,000 eligible patients with severe disease in the U.S. and Europe Additional opportunities ~24 States in U.S. 4 Countries in Europe with ~75
Slide 22: VX-548 in Acute Pain Fills the Gap/Unmet Need for ~80 Million Patients
VX-548 IN ACUTE PAIN FILLS THE “GAP”/UNMET NEED FOR ~80 MILLION PATIENTS; LAUNCH PREP UNDERWAY Actively planning for a potential Anti-Inflammatory/ near-term launch TODAY Opioids Analgesics • Specialty market due to concentration of prescribing Pain signal inhibitors, FUTURE • Focus on ~2,000 hospit
Slide 23: Q4 and Full Year 2023 Financial Highlights
Q4 AND FULL YEAR 2023 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q4 22 FY 22 Q1 23 Q2 23 Q3 23 Q4 23 FY 23 Total CF product revenues $2.30B $8.93B $2.37B $2.49B $2.48B $2.52B $9.87B TRIKAFTA/KAFTRIO 2.02B 7.69B 2.10B 2.24B 2.27B 2.33B 8.94B Other CF pro
Slide 24: 2024 Financial Guidance
2024 FINANCIAL GUIDANCE FY 2024 FY 2023 Actuals FY 2024 Guidance Commentary Includes expectations for continued growth in CF Total Product Revenue $9.87B $10.55 - $10.75B as well as the launch of CASGEVY in approved indications and geographies Combined GAAP R&D, Acquired IPR&D and SG&A $4.83B $4.9 -
Slide 25: Multiple Catalysts Throughout 2024 and Beyond
MULTIPLE CATALYSTS THROUGHOUT 2024 AND BEYOND RECENT HIGHLIGHTS ANTICIPATED KEY MILESTONES • Received approvals for TRIKAFTA in EU, U.K. and Canada in patients with CF ages 2 to 5 • Advance launch of TRIKAFTA/KAFTRIO OUS in ages 2-5 years • Reported positive Phase 3 results with the vanzacaftor trip
Slide 26: Fourth Quarter and Full Year 2023 Financial Results
FOURTH QUARTER AND FULL YEAR 2023 FINANCIAL RESULTS FEBRUARY 5, 2024 ©2024 Vertex Pharmaceuticals Incorporated
Slide 27: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q4 22 FY 22 Q1 23 Q2 23 Q3 23 Q4 23 FY 23 Combined R&D, Acquired IPR&D, and SG&A GAAP 984 3.60B 1.33B 1.16B 1.13B 1.21B 4.83B Non-GAAP 872 3.07B 1.21B 1.04B 993 1.00B 4.24B Operating incom
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