Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX

Vertex Pharmaceuticals — J.P. Morgan Healthcare Conference 2026

21 slides from Vertex Pharmaceuticals's conference presentation, January 2026

  1. Slide 1: J.P. Morgan Healthcare Conference

    J.P. Morgan Healthcare Conference Reshma Kewalramani, M.D. CEO and President January 2026 ©2026 Vertex Pharmaceuticals Incorporated ©2026 Vertex Pharmaceuticals Incorporated

  2. Slide 2: Safe Harbor Statement & Non-GAAP Financial Information

    This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding future financial and operating performance and statements regarding (i) expectations, goals, development and com

  3. Slide 3: Differentiated R+D strategy and business model continue to deliver

    Differentiated R+D strategy and business model continue to deliver Three established disease areas, renal vertical emerging and more to follow Near Term Additional Phase 3 Marketed medicines opportunities Results* CYSTIC MID- AND HEME ACUTE RENAL LATE-STAGE Investment of majority FIBROSIS PAIN of Op

  4. Slide 4: Significant growth from established and emerging disease areas

    Significant growth from established and emerging disease areas Cystic fibrosis portfolio CASGEVY Continue to expand market Leverage established global ATC leadership network to drive patient infusions JOURNAVX Povetacicept Build on momentum Submit for U.S. Accelerated of first year launch Approval;

  5. Slide 5: Potential to serve patients across 10+ disease areas

    Potential to serve patients across 10+ disease areas, each with multi-$B market potential ~110K DM1 ~300K ADPKD ~35K wAIHA Next wave of ADDRESSABLE PATIENT POPULATION* >4M ~65K T1D growth >300K >300K gMG >600K >600K pMN ~2.5M ~2.5M DPN Prepare for ~150K ~250K AMKD emerging renal franchise, starting

  6. Slide 6: CF: Continued market leadership, with patent protection through ~2040

    CF: Continued market leadership, with patent protection through ~2040 Transformative Serial Patient Reimbursement impact innovation connectivity expertise • ~15 years of real-world safety • 5 globally commercialized • ~85% U.S. CF patients in VRTX • Access in >60 countries on and efficacy data with

  7. Slide 7: CF: Multiple factors drive sustained growth

    CF: Multiple factors drive sustained growth •Best CFTR protein function, as measured by sweat chloride, plus once-daily dosing ALYFTREK •Approved for additional rare mutations launch •Expect majority of patients to switch to ALYFTREK from TRIKAFTA over time •Increases eligible population Younger •TR

  8. Slide 8: CASGEVY: Acceleration continues into 2026+ towards multi-$B potential

    CASGEVY: Acceleration continues into 2026+ towards multi-$B potential Exceeded goal for $100M CASGEVY revenue in FY 2025 Accelerate patient infusions through existing global ATC network Patients initiated 1st Cell collections Cell infusions Expand to younger patients Broad coverage U.S.: ~90% Medica

  9. Slide 9: Pain: 2025 execution positions JOURNAVX for significant growth

    Pain: 2025 execution positions JOURNAVX for significant growth in 2026 and beyond PAYERS PRESCRIBERS PATIENTS 2025 >200M >30K >500K covered lives prescribers prescriptions All 3 national PBMs* contracted Excellent breadth of prescribers Strong early success and ~900 hospitals with access and adoptio

  10. Slide 10: Pain: Committed to long-term leadership, starting with JOURNAVX

    Pain: Committed to long-term leadership, starting with JOURNAVX in acute pain RESEARCH PHASE 1 PHASE 2 PHASE 3 APPROVED PHASE 4 JOURNAVX NaV1.8 inhibitor: mod/severe acute JOURNAVX: laparoscopy, arthroscopy procedures JOURNAVX: aesthetic, reconstructive procedures JOURNAVX: real-world evidence* Suze

  11. Slide 11: Povetacicept: Potential best-in-class asset in IgAN

    Povetacicept: Potential best-in-class asset in IgAN Specifically engineered to RUBY-3 Phase 2 data demonstrated Differentiated monthly dosing and achieve improved… potential best-in-class profile patient-centric features  Binding affinity for BAFF + APRIL  Reductions in  At-home administration 

  12. Slide 12: We plan to win in renal medicine, starting with pove in IgAN

    We plan to win in renal medicine, starting with pove in IgAN Expertise in high-science sales informed by BIC therapies in CF, Heme, and moderate-to-severe Acute Pain; field force hiring well underway • Proven capabilities in securing rapid & broad patient access • Completed engagement with 60+ payer

  13. Slide 13: Rapid progress across renal portfolio, with multiple near-term catalysts

    Rapid progress across renal portfolio, with multiple near-term catalysts PATIENTS1 CURRENT PHASE 2026 MILESTONES ~330K Phase 3 trial • On track to complete submission H1:26 Povetacicept – IgAN (>1.5M enrollment for U.S. AA, if results are supportive B cell driven globally) complete • Prepare for U.S

  14. Slide 14: R+D portfolio is broad, deep, and rapidly advancing

    R+D portfolio is broad, deep, and rapidly advancing Tracking ahead of goal for 5 launches over 5 years (2028) Select, Next Wave Phase 1 Phase (1)/2 Pivotal Development Approved Research-stage Programs in Healthy Volunteers in Patients Improved conditioning Follow-on VX-407 Povetacicept CASGEVY – SCD

  15. Slide 15: Sustained revenue growth, attractive pipeline & multiple launch opportunities

    Sustained revenue growth, attractive pipeline & multiple launch opportunities Focus remains on investing in innovation, driving the late-stage pipeline & delivering on launches Non-GAAP OpEx ($M) Capital Allocation Total Revenue ($M) Ex-AIPR&D2 Strategy Sales & marketing investment increasing as a %

  16. Slide 16: Goals for 2026

    Goals for 2026 Expand CF leadership Execute commercial Advance broad and deep Deliver financial diversification R&D pipeline performance  Reach more CF patients  Drive CASGEVY patient infusions  Rapidly progress pivotal  Drive CF product revenue  Submit for approval for through global ATC netwo

  17. Slide 17: Appendix

    Appendix ©2026 Vertex Pharmaceuticals Incorporated ©2026 Vertex Pharmaceuticals Incorporated

  18. Slide 18: Anticipated Key Milestones

    Anticipated Key Milestones TRIKAFTA (CF) Completed pivotal study for 12 to <24 months of age; file for approval in H1 2026 Continue to drive adoption in U.S. and execute ongoing OUS launches in 6+ year olds ALYFTREK (CF) Share data from Phase 3 study in children ages 2-5 years and submit for regulat

  19. Slide 19: Appendix A: Reconciliation of GAAP to non-GAAP Financial Information

    Appendix A: Reconciliation of GAAP to non-GAAP Financial Information Full Year 2023 GAAP Selling, General and Administrative (SG&A) Expenses $1,137M Stock-based compensation expense (219M) Non-GAAP SG&A Expenses $918M Combined GAAP Research and Development (R&D) and SG&A Expenses $4,300M Combined No

  20. Slide 20: Vertex epidemiology estimates for targeted disease areas

    Appendix B Vertex epidemiology estimates for targeted disease areas DISEASE STATE ASSET APPROACH/MODALITY PATIENT OPPORTUNITY Cystic fibrosis 5 approved, incl. ALYFTREK Small molecules ~112,000 COMMERCIALIZED Sickle cell disease + TDT CASGEVY Cell and gene therapy >60,000 severe Acute Pain JOURNAVX

  21. Slide 21: Povetacicept IgAN offers best-in-class potential across both clinical and patient factors

    Povetacicept IgAN offers best-in-class potential across both clinical and patient factors Goal How pove achieves goal Potential BIC  Disease-modifying and pathogenic Directly reduces Gd-IgA1 MoA/ Preclinical  Highly specific and potent IC50 less than 10 nM  Inhibits BAFF + APRIL Dual inhibitor of

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