Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q4 2017 Earnings Presentation
24 slides from Vertex Pharmaceuticals's earnings presentation, Jan 31, 2018
Slide 1: Business Update & Full-Year and Q4'17 Financial Results
Business Update & Full-Year and Q4’17 Financial Results January 31, 2018 ©2016 Vertex Pharmaceuticals Incorporated
Slide 2: Agenda
Agenda Introduction Michael Partridge, Vice President, Investor Relations Key Progress and Next Steps Jeff Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer Data Highlights Jeff Chodakewitz, M.D., Executive Vice President and Chief Medical Officer Fourth-Quarter and Full-Year 2017
Slide 3: Safe Harbor Statement & Non-GAAP Financial Measures
Safe Harbor Statement & Non-GAAP Financial Measures This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided in the slide captioned "2018 Financial Guidance” and statements regard
Slide 4: Developing Medicines for All People with CF
Developing Medicines for All People with CF Any CF patient with at least one F508del mutation 68,000 à 75,000 Gene Editing 44,000 à 68,000 mRNA Potential to treat Investigational Triple all people with CF 34,000 à 44,000 Combination Regimens F508del/ 2018 Growth Drivers Minimal CFTR Function 34,000
Slide 5: Key Progress and Next Steps
Key Progress and Next Steps We have selected VX-659 and VX-445 to advance into Phase 3 development as part of two separate triple combination regimens, one of which may be a once-daily regimen • In Phase 2, both VX-659 and VX-445 triple combination regimens were well-tolerated and have a favorable s
Slide 6: Key Milestones and Goals
Key Milestones and Goals 2017 2018 Phase 2 data for triple combinations in CF patients Approval of KALYDECO in residual function mutations Initiation of pivotal development of up to two triple ACHIEVE combination regimens OUR Phase 3 tezacaftor/ivacaftor data in VISION IN multiple mutations Approval
Slide 7: VX-659 & VX-445 Phase Evaluations in Cystic Fibrosis Patients
ENTIRE DECK IS DRAFT and NOT QC’D VX-659 & VX-445 Phase 2 Evaluations in Cystic Fibrosis Patients Jeff Chodakewitz, M.D., Executive Vice President and Chief Medical Officer ©2018 Vertex Pharmaceuticals Incorporated
Slide 8: VX-659 Dosing in CF Patients with F508del/Minimal Function Mutations
VX-659 Dosing in CF Patients with F508del/Minimal Function Mutations START WEEK 4 N = 11 VX-659 80 mg QD + TEZ/IVA N = 20 VX-659 240 mg QD + TEZ/IVA N = 22 VX-659 400 mg QD + TEZ/IVA N = 10 Triple Placebo • Primary Objectives: Safety, tolerability and efficacy as assessed by mean absolute change in
Slide 9: VX-659 Safety Summary
VX-659 Safety Summary VX-659: F508del/Minimal Function • VX-659 in combination with tezacaftor and ivacaftor was generally well tolerated and the overall safety profile was favorable • Majority of adverse events were mild or moderate • No discontinuations due to adverse events o One interruption in
Slide 10: VX-659 Absolute Change in Lung Function Over Time
VX-659 Absolute Change in Lung Function Over Time VX-659: F508del/Minimal Function 20 Absolute Change in ppFEV1, LS Mean (+/- SE) Through Day 29* 15 +13.3 +11.6 (Percentage Points) 10 +10.2 5 +0.3 0 -5 Day 1 Day 15 Day 29 Study Visit 10 Placebo VX-659 80 mg QD + TEZ/IVA VX-659 240 mg QD + TEZ/IVA VX
Slide 11: VX-659 Sweat Chloride Significantly Reduced
VX-659 Sweat Chloride Significantly Reduced VX-659: F508del/Minimal Function Through 10 Day 29* Mean Absolute Change in Sweat Chloride, +2.9 0 LS Mean (+/- SE) (mmol/L) -10 -20 -30 -40 -43.7 -45.8 -50 -51.4 -60 Day 1 Day 15 Day 29 Study Visit Placebo VX-659 80 mg QD + TEZ/IVA VX-659 240 mg QD + TEZ/
Slide 12: VX-659 Significant Improvements in Respiratory Symptoms
VX-659 Significant Improvements in Respiratory Symptoms VX-659: F508del/Minimal Function Mean Absolute Change in Respiratory Domain Treatment Group of CFQ-R at Day 29 (mean, points) Triple Placebo +4.7 VX-659 80mg + TEZ/IVA +24.6 VX-659 240mg + TEZ/IVA +19.8 VX-659 400mg + TEZ/IVA +21.8 CFQ-R result
Slide 13: VX-445 Dosing in CF Patients with F508del/Minimal Function Mutations
VX-445 Dosing in CF Patients with F508del/Minimal Function Mutations START WEEK 4 N = 10 VX-445 50 mg QD + TEZ/IVA TEZ/IVA N = 22 VX-445 100 mg QD + TEZ/IVA N = 21 VX-445 200 mg QD + TEZ/IVA Dual N = 12 Triple Placebo Placebo • Primary Objectives: Safety, tolerability and efficacy as assessed by mea
Slide 14: VX-445 Safety Summary
VX-445 Safety Summary VX-445: F508del/Minimal Function • VX-445 in combination with tezacaftor and ivacaftor was generally well tolerated and the overall safety profile was favorable • Majority of adverse events were mild or moderate • Two discontinuations due to adverse events in triple combination
Slide 15: VX-445 Absolute Change in Lung Function Over Time
VX-445 Absolute Change in Lung Function Over Time VX-445: F508del/Minimal Function 20 Absolute Change in ppFEV1, LS Mean (+/- SE) Through Day 29* 15 +13.8 (Percentage Points) +11.1 10 +7.8 5 0 0.0 -5 Day 1 Day 15 Day 29 Study Visit Placebo VX-445 50 mg QD + TEZ/IVA VX-445 100 mg QD + TEZ/IVA VX-445
Slide 16: VX-445 Sweat Chloride Significantly Reduced
VX-445 Sweat Chloride Significantly Reduced VX-445: F508del/Minimal Function 10 Through Day 29* Mean Absolute Change in Sweat Chloride, 0 -2.2 -10 (Mean +/- SE) (mmol/L) -20 -30 -33.2 -38.2 -40 -39.1 -50 -60 Day 1 Day 15 Day 29 Study Visit Placebo VX-445 50 mg QD + TEZ/IVA VX-445 100 mg QD + TEZ/IVA
Slide 17: VX-445 Significant Improvements in Respiratory Symptoms
VX-445 Significant Improvements in Respiratory Symptoms VX-445: F508del/Minimal Function Mean Absolute Change in Respiratory Domain Treatment Group of CFQ-R at Day 29 (mean, points) Triple Placebo +4.2 VX-445 50 mg + TEZ/IVA +20.8 VX-445 100 mg + TEZ/IVA +15.4 VX-445 200 mg + TEZ/IVA +25.7 CFQ-R res
Slide 18: Phase 3 Program Next Steps
Phase 3 Program Next Steps Focused on finalizing design of Phase 3 programs and remain on track to initiate first Phase 3 program for VX-659 in 1H 2018 upon completion of discussions with FDA Expect to conduct two separate studies for each VX-659 and VX-445 triple combination regimen - F508del/minim
Slide 19: Full-Year and Fourth-Quarter 2017 Financial Results
ENTIRE DECK IS DRAFT and NOT and Full-Year QC’D Fourth-Quarter 2017 Financial Results Ian Smith, Executive Vice President and Chief Operating Officer ©2018 Vertex Pharmaceuticals Incorporated
Slide 20: Full-Year and Q4 2017 Financial Highlights
Full-Year and Q4 2017 Financial Highlights FY FY ($ in millions except per share data and percentages) 2016 Q1 17 Q2 17 Q3 17 Q4 17 2017 ORKAMBI 980 295 324 336 365 1.32B KALYDECO 703 186 190 213 256 845 Total CF product revenues $1.68B 481 514 550 621 $2.17B Combined non-GAAP R&D and SG&A $1.20B 31
Slide 21: 2018 Financial Guidance
2018 Financial Guidance FY 2017 2018 2018 Guidance Actuals Guidance Commentary Year-over-year increase from: • Execution of Phase 3 studies for two separate triple Combined non-GAAP combination regimens $1.33B $1.50 - $1.55B R&D and SG&A • Supply chain investment for triple combination regimens • In
Slide 22: Significant Growth in Revenue Driving Operating Margin Expansion
Significant Growth in Revenue Driving Operating Margin Expansion 3 OperaXng Margin 2.5 2 Billions ($) 26% 1.5 17% 1 Total CF Revenues -17% Combined non-GAAP 0.5 R&D & SG&A Expenses 0 2015 2016 2017 2018 2014 2015 2016 2017 Operating margins reflect total CF revenues, combined non-GAAP R&D and SG&A e
Slide 23: Thank You
Thank You …to the hundreds of patients who took part in our clinical trials, and the physicians, nurses, families and others who care for them. …to our employees for their dedication to helping advance the treatment of CF. …and to the CF community for their support and commitment to changing the cou
Slide 24: Appendix A: Reconciliation of GAAP to non-GAAP Financial Information
Appendix A Reconciliation of GAAP to non-GAAP Financial Information FY Q1 Q2 Q3 Q4 FY ($ in millions except per share data and percentages) 2016 2017 2017 2017 2017 2017 GAAP total revenues $1,702 $715 $544 $578 $652 $2,489 Non-GAAP total revenues $1,701 $482 $517 $552 $623 $2,174 GAAP income (loss)
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