Slide 1: Second Quarter 2024 Financial Results
SECOND QUARTER 2024 FINANCIAL RESULTS AUGUST 1, 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: Continued Momentum and Outstanding Execution Across the Business in Q2
Expand our leadership and raise the bar in CF • Continue to reach more people living with cystic fibrosis • Vanzacaftor triple: NDA accepted with Priority Review, Jan. 2, 2025 PDUFA • MAAs validated in EU and U.K. • VX-522: MAD portion of study underway; for >5K pts who can’t benefit from CFTRm CONT
Slide 5: 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 U.K. & EU REGULATORY SUBMISSIONS VALIDATED Vanzacaftor Triple VX-522 • Positive Phase 3 results met high expectations: • CFTR mRNA therapy in development for • non-inferior to TRIKAF
Slide 6: Novel, Effective, Non-Opioid Therapies for Moderate to Severe Acute Pain
NOVEL, EFFECTIVE, NON-OPIOID THERAPIES FOR MODERATE TO SEVERE ACUTE PAIN First novel mechanism for acute pain in over 20 years Suzetrigine (VX-548) is an oral, selective Nav1.8 pain signal inhibitor with the potential to treat acute pain types across multiple care settings Origin of pain Compellin
Slide 7: Lumbosacral Radiculopathy: Suzetrigine Phase 2 Study Completed Enrollment Early
LUMBOSACRAL RADICULOPATHY (LSR): SUZETRIGINE PHASE 2 STUDY COMPLETED ENROLLMENT EARLY AND RESULTS EXPECTED BY END OF YEAR Phase 2 study n = ~200 Suzetrigine (69mg qd) • Primary Endpoint: within-group change Run-in to from baseline to week 12 in the weekly Safety Screening establish average of daily
Slide 8: Diabetic Peripheral Neuropathy: Suzetrigine Phase 3 Pivotal Program
DIABETIC PERIPHERAL NEUROPATHY (DPN): SUZETRIGINE PHASE 3 PIVOTAL PROGRAM TO BEGIN IN Q3 Two Identical Randomized, Double-Blind, Placebo Controlled Trials n = ~1,100 Efficacy Endpoints Suzetrigine (70mg qd) • Based upon change from baseline at week 12: • Primary: SUZ vs placebo on weekly average of
Slide 9: Type 1 Diabetes: Advancing Potentially Curative Treatments
TYPE 1 DIABETES: ADVANCING POTENTIALLY CURATIVE TREATMENTS FOR ~3.8M PATIENTS IN NORTH AMERICA & EUROPE ALONE UPDATED VX-880 PHASE 1/2 TRIAL DATA FROM ADA CONTINUE TO DEMONSTRATE POTENTIAL AS A FUNCTIONAL CURE • All patients dosed demonstrated islet cell engraftment and glucose-responsive insulin pr
Slide 10: Povetacicept: Successful EOP2 Meetings & On Track for Phase 3 in IgAN
POVETACICEPT: SUCCESSFUL EOP2 MEETINGS & ON TRACK TO INITIATE GLOBAL PHASE 3 IN IGAN LATER THIS MONTH • Best-in-class potential: Pove is a dual SIGNIFICANT UNMET NEED WITH ~130K PATIENTS IN THE U.S. ALONE APRIL/BAFF inhibitor; holds promise as a transformative approach for IgA nephropathy (IgAN), a
Slide 11: 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 12: 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 13: CASGEVY Represents a Potential Multi-$B Opportunity for Vertex
CASGEVY REPRESENTS A POTENTIAL MULTI-$B OPPORTUNITY FOR VERTEX STRONG PROGRESS WITH ATC ACTIVATION, PHYSICIAN & PATIENT ENGAGEMENT, AND ACCESS Patients Providers Payers ~35,000 Severe patients in U.S., Europe >35 ATCs activated; expect to activate ~75 ATCs U.S. Strong payer support across commercial
Slide 14: Acute Pain Is a Multi-Billion Dollar Market, with Significant Unmet Need
ACUTE PAIN IS A MULTI-BILLION DOLLAR MARKET, WITH SIGNIFICANT UNMET NEED Anti-Inflammatory/ TODAY Opioids Analgesics Pain signal inhibitors, FUTURE starting with suzetrigine • 80M patients are prescribed a medicine for moderate to severe acute • Completed hiring of field team and strategic account l
Slide 15: Q2 2024 Financial Highlights
Q2 2024 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q2 23 FY 23 Q2 24 Total product revenues $2.49B $9.87B $2.65B TRIKAFTA/KAFTRIO 2.24B 8.94B 2.45B Other CF products 253 925 196 Combined non-GAAP R&D and SG&A expenses 928 3.71B 978 Acquired IPR&D expens
Slide 16: 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 well as contribution in the second half from the Total Product Revenues $10.65 - $10.85 $10.55 - $10.75B launch of CASGEVY in approved ind
Slide 17: Multiple Catalysts Throughout 2024 and Beyond
MULTIPLE CATALYSTS THROUGHOUT 2024 AND BEYOND RECENT HIGHLIGHTS ANTICIPATED KEY MILESTONES • KALYDECO received European Commission approval in infants with CF ages 1 mo to <4 mo • Continue to reach more eligible patients; expand into younger age groups • Vanzacaftor triple: NDA accepted with Priorit
Slide 18: Second Quarter 2024 Financial Results
SECOND QUARTER 2024 FINANCIAL RESULTS AUGUST 1, 2024 ©2024 Vertex Pharmaceuticals Incorporated
Slide 19: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q2 23 FY 23 Q2 24 Combined R&D and SG&A GAAP 1.05B 4.30B 1.34B Non-GAAP 928 3.71B 978 Operating income GAAP 1.03B 3.83B (3.51)B Non-GAAP 1.15B 4.37B (3.15)B Operating Margin %: GAAP 41% 39