Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q1 2025 Earnings Presentation
20 slides from Vertex Pharmaceuticals's earnings presentation, May 5, 2025
Slide 1: First Quarter 2025 Financial Results
FIRST QUARTER 2025 FINANCIAL First Quarter 2025 RESULTS Financial Results May 5, 2025 ©2025 Vertex Pharmaceuticals Incorporated ©2025 Vertex Pharmaceuticals Incorporated ©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 Duncan McKechnie, SVP, North Amer
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 a strong start to 2025
Expand CF leadership • ALYFTREK: Approved in the U.S. and U.K. for patients with CF ages 6+; strong U.S. launch • VX-828 combo (next gen 3.0 CFTRm regimen): on track to initiate clinical trial in CF patients by EoY • VX-522 (mRNA): Implemented pause to MAD portion of Ph 1/2 study to assess a tolerab
Slide 5: Expanding CF leadership: ALYFTREK approved in U.S. & U.K.
Expanding CF leadership: ALYFTREK approved in U.S. & U.K. and Positive CHMP Opinion in EU Vertex CFTR modulators have the potential to transform the lives of nearly 95% of patients with CF Patients 1 month and older Patients 1 year and older Patients 6 years and older N.G 1.0 regimen, ages 2+ • Seri
Slide 6: Vertex is committed to long-term leadership in pain
Vertex is committed to long-term leadership in pain with a broad and deep pipeline FORMULATION RESEARCH PHASE 1 PHASE 2 PHASE 3 APPROVED JOURNAVX NaV1.8 inhibitor - Acute Oral Suzetrigine NaV1.8 inhibitor - DPN Oral Suzetrigine NaV1.8 inhibitor - LSR Oral VX-993 NaV1.8 inhibitor - Acute Oral VX-993
Slide 7: Zimislecel: Pivotal trial will complete enrollment and dosing in Q2:25
Zimislecel: Pivotal trial will complete enrollment and dosing in Q2:25, with global regulatory Total Daily Insulin submissions planned for 2026 EASD 2024 Data presented on 12 patients who completed Day 180 visit: Patient population • Initial launch targets ~60,000 severe T1D patients in U.S. and Eur
Slide 8: Kidney: Broad pipeline of potentially transformative medicines
Kidney: Broad pipeline of potentially transformative medicines in multiple serious renal diseases PATIENTS1 RESEARCH PHASE 1 PHASE 2 PHASE 3 APPROVED Povetacicept – IgAN2 ~300K Enrollment complete (>750K China) in IA cohort Moving to pivotal B cell mediated Povetacicept – pMN ~150K Development H2:25
Slide 9: Povetacicept: IgAN - completed enrollment of interim analysis cohort
Povetacicept: IgAN - completed enrollment of interim analysis cohort in global Phase 3 study; pMN - advancing to pivotal development Best-in-class potential RAINIER Phase 3 trial in IgAN Phase 2/3 trial in pMN Strong preclinical profile: • Reached agreement with FDA to • Dual antagonist of BAFF/APRI
Slide 10: VX-407: First-in-class PC1 corrector for ADPKD
VX-407: First-in-class PC1 corrector for ADPKD advancing to Phase 2 in H2 2025 Over time, kidney cysts lead to kidney function (eGFR) decline and • ~300,000 people in the U.S. & Europe diagnosed with kidney failure ADPKD • No treatments address the underlying cause of disease Normal Hyperfiltration
Slide 11: Clinical portfolio is broad, diverse, and rapidly advancing
Clinical portfolio is broad, diverse, and rapidly advancing On track to meet goal of 5 launches over 5 years (by 2028) Select, Next Wave Phase 1 Phase (1)/2 Pivotal Approved Research-stage Programs in Healthy Volunteers in Patients Development Improved conditioning Follow-on Suzetrigine Zimislecel C
Slide 12: ALYFTREK: Approved for ages 6+ in U.S. and U.K.
ALYFTREK: Approved for ages 6+ in U.S. and U.K; Uptake across all eligible patient groups in U.S. Reimbursement discussions underway in UK Potential approvals in EU and other geographies in 2025 ALYFTREK: A highly efficacious, once-daily CFTR modulator delivering equivalent improvement in lung funct
Slide 13: CASGEVY: Launch gathering momentum in all regions
CASGEVY: Launch gathering momentum in all regions Now approved in U.S., UK, EU, Kingdom of Saudi Arabia, Bahrain, Canada, Switzerland, and United Arab Emirates >65 ATCs activated and ~90 patients have had first cell collections* 8 CASGEVY infusions in Q1 2025 U.S. Payer Highlights Commercial – conti
Slide 14: JOURNAVX: FDA approved Jan 30, 2025
JOURNAVX: FDA approved Wide availability, strong early uptake Jan 30, 2025 • Retail pharmacy stocking: JOURNAVX was broadly available at ~33,000 Strong early launch retail locations by mid-March signals • >20,000 prescriptions filled through April 18th Rapid progress with payers • ~94 million covere
Slide 15: Q1 2025 financial highlights
Q1 2025 financial highlights ($ in millions except where noted or per share data and percentages) Q1:24 FY:24 Q1:25 Total revenue $2.69B $11.02B $2.77B TRIKAFTA/KAFTRIO 2.48B 10.24B 2.54B ALYFTREK - - 54 Other product revenue* 207 782 171 Combined non-GAAP, Acquired IPR&D and SG&A expenses 1.02B 8.8
Slide 16: Updated full year 2025 financial guidance
Updated full year 2025 financial guidance Current Previous Commentary FY 2025 Guidance FY 2025 Guidance Includes expectations for continued growth in CF, including the launch of ALYFTREK; continued Total Revenue $11.85 - $12.0B $11.75 - $12.0B uptake of CASGEVY; and a contribution from the launch of
Slide 17: Multiple catalysts throughout 2025
Multiple catalysts throughout 2025 ANTICIPATED KEY MILESTONES ALYFTREK (CF) Drive U.S. launch, secure reimbursement in U.K. and secure regulatory approvals in EU and other geographies VX-522 (CF) Temporary pause implemented to assess a tolerability issue Next-generation 3.0 (CF) VX-828 (next-generat
Slide 18: First Quarter 2025 Financial Results
FIRST QUARTER 2025 FINANCIAL First Quarter 2025 RESULTS Financial Results May 5, 2025 ©2025 Vertex Pharmaceuticals Incorporated ©2025 Vertex Pharmaceuticals Incorporated ©2025 Vertex Pharmaceuticals Incorporated
Slide 19: Appendix A: GAAP to non-GAAP Financial Information
Appendix A GAAP to non-GAAP Financial Information ($ in millions except as noted, per share data and percentages) Q1:24 FY:24 Q1:25 Combined R&D, Acquired IPR&D and SG&A GAAP 1.21B 9.72B 1.40B Non-GAAP 1.02B 8.82B 1.23B Operating income GAAP 1.14B (233) 630 Non-GAAP 1.34B 696 1.18B Operating Margin
Slide 20: 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 ALYFTREK COMMERCIALIZED Sickle cell disease + TDT CASGEVY Cell and gene therapy ~60,000 severe Small molecule NaV1.8 Acute Pain JOU
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