Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q4 2024 Earnings Presentation
19 slides from Vertex Pharmaceuticals's earnings presentation, Feb 10, 2025
Slide 1: Fourth Quarter and Full Year 2024 Financial Results
FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS FEBRUARY 10, 2025 ©2025 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: Vertex Delivered Strong Q4:24 Results
Expand CF leadership ✓ ALYFTREK: approved in the U.S. for patients with CF ages 6+, launch underway • Increased epidemiology estimates for people with CF to 109,000 due primarily to patients living longer, plus additional geographies • Next gen 3.0 CFTRm regimen (VX-828, VX-118, tez triple) expected
Slide 5: Recent Achievements and Ongoing Programs to Sustain Long Term Growth
RECENT ACHIEVEMENTS AND ONGOING PROGRAMS TO SUSTAIN LONG TERM GROWTH 2 3 4 5 2 recent approvals 3 key anticipated 4 ongoing 5 launches in 5 years enrollment milestones pivotal trials (by 2028) in pivotal studies • Zimislecel (T1D) • Zimislecel (T1D) ✓ CASGEVY (SCD) For patients 6+ with CF • Povetaci
Slide 6: Expanding Leadership in CF: ALYFTREK Is Approved in the U.S.
EXPANDING LEADERSHIP IN CF: ALYFTREK IS APPROVED IN THE U.S. RAISING THE BAR WITH SERIAL INNOVATION Patients 1 month and older Patients 1 year and older Patients 6 years and older NG 1.0 regimen, ages 2+ ✓ Now approved for ages 6+ in U.S.; potential to set new standard of CF care • Serial innovation
Slide 7: Renal: Broad Pipeline of Potentially Transformative Medicines
RENAL: BROAD PIPELINE OF POTENTIALLY TRANSFORMATIVE MEDICINES IN MULTIPLE SERIOUS RENAL DISEASES PATIENTS1 RESEARCH PHASE 1 PHASE 2 PHASE 3 APPROVED Inaxaplin – Primary AMKD ~150K APOL1 mediated Inaxaplin – AMKD with ~100K kidney disease comorbidities2 (AMKD) ~250K Additional APOL1 inhibitors3 (150K
Slide 8: Povetacicept: Global Phase 3 Clinical Trial in IgAN Underway
POVETACICEPT: GLOBAL PHASE 3 CLINICAL TRIAL IN IGAN UNDERWAY POTENTIAL BEST-IN-CLASS APPROACH TO TREAT IGAN; ENROLLING AND DOSING PATIENTS IN NA, EUROPE, ASIA Best-in-class potential RAINIER global trial structure Enrollment/dosing underway Strong preclinical profile: • Dual BAFF/APRIL inhibitor wit
Slide 9: VX-407 for ADPKD Is a First-in-Class PC1 Corrector
VX-407 FOR ADPKD IS A FIRST-IN- CLASS PC1 CORRECTOR FOR A SUBSET OF VARIANTS IN PKD1 GENE Over time, kidney cysts lead to kidney function (eGFR) decline and kidney failure • ~300,000 people in the U.S. & Europe diagnosed with Normal Hyperfiltration Impairment Failure ADPKD • No currently available t
Slide 10: 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 Regulatory Submissions Approved Research-stage Programs in Healthy Volunteers in Patients Development Completed or Underway Improved
Slide 11: ALYFTREK: Approved for Ages 6+ in U.S.
ALYFTREK: APPROVED FOR AGES 6+ IN U.S. WITH POTENTIAL TO SET NEW STANDARD OF CF CARE POTENTIAL APPROVALS IN UK, EU, AUSTRALIA, NEW ZEALAND, CANADA, SWITZERLAND IN 2025 ALYFTREK: A highly efficacious, once-daily CFTR modulator delivering equivalent improvement in lung function* and greater CFTR funct
Slide 12: CASGEVY: Foundational 2024 Builds Momentum for 2025
CASGEVY: FOUNDATIONAL 2024 BUILDS MOMENTUM FOR 2025 AND BEYOND FOR MULTI-$B OPPORTUNITY Rapid pace of global approvals* underscores high unmet need and transformative potential of Casgevy >50 ATCs activated and >50 cell collections initiated across U.S., Europe, Middle East** OUS Payer Highlights Re
Slide 13: JOURNAVX: Landmark U.S. Approval for First Non-Opioid in >20 Years
JOURNAVX: LANDMARK Launch progress U.S. APPROVAL FOR 1ST Establish conditions for patient access and long-term commercial success • Healthcare providers & physicians: salesforce engaging broadly NON-OPIOID IN >20 YRS. • Institutions: focus on ~2,000 high-volume hospitals/~150 related health systems;
Slide 14: Q4 and Full Year 2024 Financial Highlights
Q4 AND FULL YEAR 2024 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q4 23 FY 23 Q1 24 Q2 24 Q3 24 Q4 24 FY 24 Total product revenues $2.52B $9.87B $2.69B $2.65B $2.77B $2.91B $11.02B TRIKAFTA/KAFTRIO 2.33B 8.94B 2.48B 2.45B 2.59B 2.72B 10.24B Other product
Slide 15: 2025 Financial Guidance
2025 FINANCIAL GUIDANCE FY 2025 FY 2024 Actuals FY 2025 Guidance Commentary Includes expectations for continued growth in CF, including the U.S. launch of ALYFTREK; Total Revenue $11.02B $11.75 - $12.0B continued uptake of CASGEVY in multiple regions; and a contribution from the launch of JOURNAVX,
Slide 16: Multiple Catalysts Throughout 2025
MULTIPLE CATALYSTS THROUGHOUT 2025 ANTICIPATED KEY MILESTONES ALYFTREK (CF) Drive U.S. launch; secure additional regulatory approvals and execute global launches VX-522 (CF) Complete MAD portion of the Phase 1/2 study and share data in 2025 Next-generation 3.0 (CF) Triple combo: VX-828 (CFTR correct
Slide 17: Fourth Quarter and Full Year 2024 Financial Results
FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS FEBRUARY 10, 2025 ©2025 Vertex Pharmaceuticals Incorporated
Slide 18: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION (in millions except per share amounts and percentages) Q4 23 FY 23 Q1 24 Q2 24 Q3 24 Q4 24 FY 24 Combined R&D and SG&A GAAP 1.19B 4.30B 1.13B 1.34B 1.25B 1.38B 5.09B Non-GAAP 984 3.71B 942 978 1.06B 1.21B 4.19B Operating income GAAP 989 3.83B 1.14B (3.
Slide 19: Vertex Targeted Disease Area Epidemiology Estimates
VERTEX TARGETED DISEASE AREA EPIDEMIOLOGY ESTIMATES DISEASE STATE ASSET APPROACH/MODALITY PATIENT OPPORTUNITY 5 approved, incl. Cystic fibrosis Small molecules ~109,000 COMMERCIALIZED ALYFTREK Sickle cell disease + TDT CASGEVY Cell and gene therapy ~60,000 severe NEAR-TERM Small molecule NaV1.8 Acut
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