Slide 1: Third Quarter 2024 Financial Results
THIRD QUARTER 2024 FINANCIAL RESULTS NOVEMBER 4, 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 that are subject to risks, uncertainties and other factors. All statements other than statements of historical fact are statements that could be deemed forward-looking statements, including all
Slide 4: Third Quarter Performance Extended Our Strong Momentum This Year
Expand our leadership and raise the bar in CF • Continue to reach more people living with cystic fibrosis • Vanzacaftor triple: Jan. 2, 2025 PDUFA; MAAs validated in EU and U.K. • VX-522: for >5K pts who can’t benefit from CFTRm; MAD portion of study ongoing THIRD QUARTER Drive multiple near-term co
Slide 5: Clinical Portfolio Is Broad, Diverse and Rapidly Advancing
CLINICAL PORTFOLIO IS BROAD, DIVERSE AND RAPIDLY ADVANCING ON TRACK TO MEET GOAL OF FIVE LAUNCHES OVER FIVE YEARS (2028) Select, Next Wave Phase 1 Phase (1)/2 Pivotal Development Regulatory Submissions Approved Research-stage Programs in Healthy Volunteers in Patients Completed or Underway Hypoimmun
Slide 6: CF: Expanding Leadership & Raising the Bar with Serial Innovation
CF: EXPANDING LEADERSHIP & RAISING THE BAR WITH SERIAL INNOVATION VANZACAFTOR TRIPLE: U.S. PDUFA: JANUARY 2, 2025 AND SUBMISSIONS COMPLETE IN MULTIPLE GEOGRAPHIES Vanzacaftor Triple VX-522 • Positive Phase 3 results met high expectations • CFTR mRNA therapy in development for >5,000 CF patients who
Slide 7: VX-880 in Type 1 Diabetes: EASD Data Demonstrate Curative Potential
VX-880 IN TYPE 1 DIABETES: DATA FROM EASD* DEMONSTRATE CURATIVE POTENTIAL OF VX-880 AND SUPPORT ADVANCEMENT TO PIVOTAL DEVELOPMENT • All 12 participants achieved a reduction in HbA1c to <7% • All 12 participants had elimination of severe hypoglycemic events (SHEs) during the evaluation period (day 9
Slide 8: Povetacicept: Global Phase 3 in IgAN Underway
POVETACICEPT: GLOBAL PHASE 3 IN IGAN UNDERWAY SIGNIFICANT UNMET NEED WITH ~130K PATIENTS IN THE U.S. ALONE • Best-in-class potential: Pove is a dual APRIL/BAFF inhibitor; holds promise as a transformative approach for IgA nephropathy (IgAN), a serious, Randomized, double-blind, placebo-controlled pi
Slide 9: Pove IgAN: Updated RUBY-3 Data at ASN
POVE IGAN: UPDATED RUBY-3 DATA AT ASN CONTINUE TO DEMONTRATE BEST-IN-CLASS POTENTIAL At 48 weeks, pove 80mg SC Q4W: • 66% mean reduction in UPCR • Stable renal function as assessed by eGFR • 63% achievement of clinical remission, defined as UPCR < 0.5 g/g, negative hematuria, and stable renal functi
Slide 10: Pove PMN: Emerging RUBY-3 Data at ASN in Primary Membranous Nephropathy
POVE PMN: EMERGING RUBY-3 DATA AT ASN IN PRIMARY MEMBRANOUS NEPHROPATHY ALSO SHOW BEST-IN-CLASS POTENTIAL a Samples collected at Week 24 for two participants were not able to be processed; value shown represents data for one participant only. Pove 80mg SC Q4W: • 62% reduction from baseline in UPCR a
Slide 11: Vertex Is Committed to Transforming the Treatment of Pain
VERTEX IS COMMITTED TO TRANSFORMING THE TREATMENT OF PAIN SERIAL INNOVATION, BROAD/DEEP PIPELINE FOR LEADERSHIP IN MULTIPLE PAIN STATES FORMULATION RESEARCH PHASE 1 PHASE 2 PHASE 3 APPROVED VX-548 NaV1.8 inhibitor* - Acute Oral VX-548 NaV1.8 inhibitor - DPN Oral VX-548 NaV1.8 inhibitor - LSR Oral VX
Slide 12: Lumbosacral Radiculopathy: Suzetrigine Phase 2 Study Completed
LUMBOSACRAL RADICULOPATHY (LSR): SUZETRIGINE PHASE 2 STUDY HAS COMPLETED AND RESULTS EXPECTED BY END OF YEAR Phase 2 study n = 218 Run-in to Suzetrigine (69mg qd) Primary Endpoint: within-group Safety change from baseline to week 12 Screening establish follow- in the weekly average of daily leg base
Slide 13: Expanding Leadership in CF and Raising the Bar with Serial Innovation
EXPANDING LEADERSHIP IN CF AND RAISING THE BAR WITH SERIAL INNOVATION ~92,000 GROWTH DRIVERS Treating younger patients Patients living longer Best-in-class Goal: carrier levels patients with CF* Serial CFTRm innovation medicines of CFTR function mRNA for last >5,000 patients for all patients
Slide 14: Creating Strong Foundation for CASGEVY to Achieve Multi-$B Potential
CREATING STRONG FOUNDATION FOR CASGEVY TO ACHIEVE MULTI-$B POTENTIAL CONTINUED STRONG PROGRESS WITH ATC ACTIVATION, PHYSICIAN & PATIENT ENGAGEMENT, AND ACCESS Patients Providers Payers ~35,000 Severe patients in U.S., Europe 45 ATCs activated; expect to activate ~75 ATCs U.S. Strong payer support ac
Slide 15: Suzetrigine in Acute Pain: Launch Preparedness Well Underway
SUZETRIGINE IN ACUTE PAIN: LAUNCH PREPAREDNESS WELL UNDERWAY PDUFA TARGET ACTION DATE 1/30/25; HIGH LEVELS OF ENTHUSIASM FOR POTENTIAL NEW CLASS OF PAIN MEDICINE • Experienced team of strategic account leads and territory Launch focus account managers are fully trained and engaged • Hospitals & hosp
Slide 16: Survey Indicates Significant Need for New Options to Treat Acute Pain
SURVEY INDICATES SIGNIFICANT NEED FOR NEW OPTIONS TO TREAT MODERATE-TO-SEVERE ACUTE PAIN 40M acute pain patients* COSTS OF OPIOID USE DISORDER (OUD) REMAIN STUBBORNLY HIGH receive an opioid Rx annually1 10% of acute pain patients treated initially with an opioid will go on to have prolonged Healthca
Slide 17: Q3 2024 Financial Highlights
Q3 2024 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q3 23 FY 23 Q3 24 Total product revenues $2.48B $9.87B $2.77B TRIKAFTA/KAFTRIO 2.27B 8.94B 2.59B Other products 209 925 187 Combined non-GAAP R&D and SG&A expenses 942 3.71B 1.06B Acquired IPR&D expense
Slide 18: Raising Full Year 2024 Product Revenue Guidance
RAISING FULL YEAR 2024 PRODUCT REVENUE GUIDANCE Current Previous Commentary FY 2024 Guidance FY 2024 Guidance Includes expectations for continued growth in CF as Total Product Revenues $10.8 - $10.9B $10.65 - $10.85B well as for the launch of CASGEVY in approved indications and geographies. Combined
Slide 19: Multiple Catalysts Throughout 2024 and Beyond
MULTIPLE CATALYSTS THROUGHOUT 2024 AND BEYOND RECENT HIGHLIGHTS ANTICIPATED KEY MILESTONES • Vanzacaftor triple: Global reviews underway in U.S., EU, U.K., Canada, Australia, • Vanza triple: Jan. 2, 2025 PDUFA; Execute launch upon approval Switzerland and New Zealand • VX-522 CFTR mRNA study: SAD co
Slide 20: Third Quarter 2024 Financial Results
THIRD QUARTER 2024 FINANCIAL RESULTS NOVEMBER 4, 2024 ©2024 Vertex Pharmaceuticals Incorporated
Slide 21: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q3 23 FY 23 Q3 24 Combined R&D and SG&A GAAP 1.07B 4.30B 1.25B Non-GAAP 942 3.71B 1.06B Operating income GAAP 1.04B 3.83B 1.12B Non-GAAP 1.17B 4.37B 1.31B Operating Margin %: GAAP 42% 39%