Vertex Reports First Quarter 2025 Financial Results
— Total revenue of
— Strong progress with CASGEVY ® , ALYFTREK™ and JOURNAVX™ launches —
— Povetacicept (pove) IgAN Phase 3 interim analysis (IA) cohort fully enrolled and zimislecel Phase 3 program to complete dosing this quarter, setting up potential filings in 2026; inaxaplin Phase 3 IA cohort on track to complete enrollment in the second half of 2025 —
— Four programs already in pivotal development, with pivotal study of pove in primary membranous nephropathy (pMN) to start this year —
“Vertex delivered a strong start to 2025 with notable execution across the business as we grow and diversify the revenue base, progress multiple launches and advance the R&D pipeline. We continued to expand our leadership in CF and build global momentum for CASGEVY, and we launched JOURNAVX in moderate-to-severe acute pain,” said
First Quarter 2025 Results
Total revenue increased 3% to
Combined GAAP and Non-GAAP R&D, Acquired IPR&D and SG&A expenses were
Intangible asset impairment charge of
GAAP effective tax rate was 11.5% compared to 14.0% for the first quarter of 2024.
Non-GAAP effective tax rate was 18.8% compared to 17.4% for the first quarter of 2024. The difference between the GAAP and non-GAAP effective tax rates was primarily due to tax benefits related to stock-based compensation.
GAAP net income was
Non-GAAP net income was
Cash, cash equivalents and total marketable securities as of
Full Year 2025 Financial Guidance
Vertex’s financial guidance is summarized below:
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Current FY 2025 |
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FY 2025 |
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Total revenue |
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Combined GAAP R&D, AIPR&D and SG&A expenses * |
Unchanged |
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Combined Non-GAAP R&D, AIPR&D and SG&A expenses * |
Unchanged |
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Non-GAAP effective tax rate |
Unchanged |
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20.5% to 21.5% |
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*The difference between the combined GAAP R&D, AIPR&D and SG&A expenses and the combined non-GAAP R&D, AIPR&D and SG&A expenses guidance relates primarily to |
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**Combined GAAP and Non-GAAP R&D, AIPR&D and SG&A expenses guidance includes approximately |
Key Business Highlights
Cystic Fibrosis (CF) Portfolio
Marketed Products
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ALYFTREK, the once-daily, next-in-class combination CFTR modulator for the treatment of people with CF ages 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene, is now approved in the
U.S. and theU.K. Vertex is working with the National Health Service (NHS ) in theU.K. to secure coverage for eligible patients. -
In April, the European Medical Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ALYFTREK for the treatment of CF patients ages 6 years and older.
European Commission approval is expected in the second half of 2025. Regulatory reviews are underway for ALYFTREK inCanada ,Switzerland ,Australia and New Zealand . -
Vertex securedEuropean Commission approval for the label expansion of KAFTRIO in combination with ivacaftor for CF patients ages 2 years and older who have at least one non-class I mutation in the CFTR gene. With this approval, approximately 4,000 people in theEuropean Union are newly eligible for a medicine that treats the underlying cause of their disease. Work to ensure access for all eligible patients is underway.
CASGEVY for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved in the
-
In
Europe ,Vertex secured a formal reimbursement agreement withNHS England for SCD patients to access CASGEVY, following the agreement reached inAugust 2024 for eligible TDT patients to access CASGEVY.Vertex recently entered into a similar agreement inWales for both eligible SCD and TDT patients. Following a positive assessment,Vertex also finalized national reimbursement inAustria . In theMiddle East , following regulatory approval in theUAE ,Vertex secured reimbursement in the majority of emirates. -
Since launch through
May 1st ,Vertex has activated more than 65 authorized treatment centers (ATCs) globally and approximately 90 patients have had their first cell collection. -
Vertex recently filed a manufacturing license submission with the FDA and expects to begin manufacturing CASGEVY inPortsmouth, NH , in the second half of 2025. This submission is part of the planned ramp of CASGEVY manufacturing capacity as demand for the therapy increases.
JOURNAVX (suzetrigine) for the treatment of moderate-to-severe acute pain
JOURNAVX is a first-in-class, oral, selective, non-opioid NaV1.8 pain signal inhibitor, approved in the
-
On
January 30, 2025 , the FDA approved JOURNAVX for the treatment of adults with moderate-to-severe acute pain, and it is now available and stocked at pharmacies across theU.S. , including major national and regional retail pharmacy chains. -
Since JOURNAVX became available in early March through
April 18th , more than 20,000 prescriptions have been written and filled across the hospital and retail settings in different acute pain conditions, consistent with JOURNAVX’s broad label. -
As of
May 1st , across commercial and government payers, approximately 94 million lives already have covered access to JOURNAVX, and approximately 42 million have unrestricted access (i.e., without the need for prior authorization or step edits).Vertex has reached a formal coverage agreement with a large national Pharmacy Benefit Manager (PBM) to make JOURNAVX available to their customers, representing approximately 22 million commercial lives. A total of ten state Medicaid plans are also providing unrestricted access to JOURNAVX and 20 more are currently evaluating their policies. - More than 50 large healthcare systems have taken steps to initiate pharmacy and therapeutics (P&T) committee reviews of JOURNAVX, and some have already added it on formularies.
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Vertex recently initiated two Phase 4 studies in various moderate-to-severe acute pain conditions to provide additional data on the effectiveness and safety of JOURNAVX as part of real-world clinical practice, in both inpatient and outpatient settings. -
The Alternatives to Pain Act has been reintroduced in both the
House and Senate of the newU.S. Congress , while nearly 35 U.S. states to date have passed or introduced new legislation in support of non-opioid options.Vertex also expects JOURNAVX to be added to the list of treatments eligible for an add-on payment under the NOPAIN Act, which became effective onJanuary 1, 2025 .
Select Clinical-Stage R&D Pipeline
Cystic Fibrosis
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Vertex is enrolling and dosing studies in younger age groups — a Phase 3 study of TRIKAFTA/KAFTRIO in 1- to 2-year-olds and a Phase 3 study of ALYFTREK in 2- to 5-year-olds — to expand the labels and enable earlier treatment of children with CF. -
Consistent with its commitment to serial innovation and bringing as many patients as possible to normal levels of CFTR function,
Vertex continues to advance new oral small molecule combination therapies through preclinical and clinical development.Vertex expects to advance the once-daily, next-generation 3.0 VX-828 combination into a clinical trial in people with CF this year. -
Vertex has implemented a temporary pause to the multiple ascending dose (MAD) portion of the Phase 1/2 study of VX-522, a nebulized CFTR mRNA therapy, in order to assess a tolerability issue.
Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
-
Vertex has completed enrollment of children 5 to 11 years of age with SCD or TDT in two global Phase 3 studies of CASGEVY. -
Vertex continues to advance preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population.
Acute Pain
-
Vertex expects to complete the Phase 2 study for an oral formulation of VX-993 this quarter. VX-993 is a next-generation selective NaV1.8 pain signal inhibitor, for the treatment of moderate-to-severe acute pain following bunionectomy surgery.Vertex expects to report results from the Phase 2 study in the second half of 2025. VX-993 has Fast Track designation for moderate-to-severe acute pain. -
Vertex has completed the Phase 1 study of an intravenous formulation of VX-993 in healthy volunteers. The FDA has granted the intravenous formulation of VX-993 Fast Track designation for moderate-to-severe acute pain.
Peripheral Neuropathic Pain (PNP)
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Vertex continues to enroll and dose patients with diabetic peripheral neuropathy (DPN) in a Phase 3 pivotal trial of suzetrigine. The FDA has granted suzetrigine Fast Track designation in PNP and Breakthrough Therapy designation in DPN. -
Vertex continues to enroll and dose patients in a Phase 2 study for the oral formulation of VX-993 for the treatment of DPN.
Type 1 Diabetes (T1D)
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Zimislecel (VX-880), fully differentiated islet cells with standard immunosuppression:
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Vertex continues to enroll and dose patients in the Phase 3 portion of the Phase 1/2/3 study of zimislecel in patients with T1D with severe hypoglycemic events and impaired awareness of hypoglycemia in theU.S. ,Canada ,U.K. , and EU.Vertex expects to complete enrollment and dosing of the pivotal study in the second quarter of 2025 and submit marketing applications to global regulators in 2026. -
Zimislecel has been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the
U.S. Food and Drug Administration , Priority Medicines (PRIME) designation from the EMA, and has secured an Innovation Passport under theInnovative Licensing and Access Pathway (ILAP) from theUK Medicines and Healthcare products Regulatory Agency (MHRA). -
Vertex will present updated clinical data from the 12 participants who received the full dose of zimislecel as a single infusion with over one year of follow-up in the Phase 1/2 portion of the study in an oral presentation at theAmerican Diabetes Association 85th Scientific Sessions.Vertex will host an in-person investor event at theADA conference onFriday, June 20, 2025 at7:15 p.m. CT /8:15 p.m. ET . The investor event will also be webcast live, and a link to the webcast can be accessed through Vertex’s website at www.vrtx.com in the “Investors” section.
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Additional approaches:
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Vertex previously announced that it has discontinued development of the VX-264 (“cells plus device” program), as the Phase 1/2 study did not meet its efficacy endpoint. -
Vertex is pursuing research-stage programs to evaluate additional approaches that could provide transformative benefit to people with T1D and reduce or eliminate the need for standard immunosuppressive regimens. These approaches include alternative immunosuppressives, gene editing, and novel immunoprotection to encapsulate the islet cells.
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IgA Nephropathy (IgAN), Primary Membranous Nephropathy (pMN) and Other B Cell-Mediated Diseases
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The global Phase 3 RAINIER trial of povetacicept in patients with IgAN has completed enrollment of the interim analysis cohort. The interim analysis will be conducted once this cohort reaches 36 weeks of treatment, with the potential to file for Accelerated Approval in the
U.S. in the first half of 2026, if results are supportive. Studies to support the launch of povetacicept for home administration are underway. -
Based on positive results of povetacicept in primary membranous nephropathy (pMN) in the RUBY-3 study,
Vertex has reached agreement with the FDA to advance povetacicept into pivotal development for this disease.Vertex will initiate a single, adaptive Phase 2/3 trial of povetacicept vs. standard-of-care this year. In the Phase 2 portion, both 80 mg and 240 mg of povetacicept will be assessed and the selected dose will advance to Phase 3, where the primary endpoint of complete clinical remission at 72 weeks of treatment will be evaluated. -
Vertex plans to present updated data from the IgAN and pMN cohorts of the RUBY-3 study at upcoming medical congresses. -
Vertex expects to share data and next steps from other RUBY-3 renal diseases and the hematologic conditions in the RUBY-4 study later this year.
APOL1-Mediated Kidney Disease (AMKD)
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Vertex expects to complete enrollment in the interim analysis cohort of the Phase 3 portion of the AMPLITUDE trial of inaxaplin in the second half of 2025.Vertex will conduct the pre-planned interim analysis once this cohort has been treated for 48 weeks, with potential to file for accelerated approval in theU.S. if the results are supportive. -
Vertex continues to enroll and dose patients in the AMPLIFIED Phase 2 study of inaxaplin in people with AMKD and diabetes or other co-morbidities.
Myotonic Dystrophy Type 1 (DM1)
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Vertex continues to enroll and dose the MAD portion of the global Phase 1/2 clinical trial of VX-670 in people with DM1, which will assess both safety and efficacy.
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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Vertex has completed the Phase 1 study of VX-407 in healthy volunteers. -
Vertex expects to advance VX-407 into a Phase 2 proof-of-concept study this year in patients with a subset of variants in the PKD1 gene, which encodes the PC1 protein, estimated to be up to ~30,000 (or ~10%) of the overall patient population.
Non-GAAP Financial Measures
In this press release,
The company provides guidance regarding combined R&D, AIPR&D and SG&A expenses and effective tax rate on a non-GAAP basis. Unless otherwise noted, the guidance regarding combined R&D, AIPR&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.
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Consolidated Statements of Income |
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(unaudited, in millions, except per share amounts) |
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Three Months Ended |
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2025 |
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2024 |
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Revenues: |
|
|
|
||||
Product revenues, net |
$ |
2,760.2 |
|
|
$ |
2,690.6 |
|
Other revenues |
|
10.0 |
|
|
|
— |
|
Total revenues |
|
2,770.2 |
|
|
|
2,690.6 |
|
Costs and expenses: |
|
|
|
||||
Cost of sales |
|
363.0 |
|
|
|
342.6 |
|
Research and development expenses |
|
979.7 |
|
|
|
789.1 |
|
Acquired in-process research and development expenses |
|
19.8 |
|
|
|
76.8 |
|
Selling, general and administrative expenses |
|
396.4 |
|
|
|
342.7 |
|
Intangible asset impairment charge |
|
379.0 |
|
|
|
— |
|
Change in fair value of contingent consideration |
|
2.2 |
|
|
|
(0.1 |
) |
Total costs and expenses |
|
2,140.1 |
|
|
|
1,551.1 |
|
Income from operations |
|
630.1 |
|
|
|
1,139.5 |
|
Interest income |
|
120.9 |
|
|
|
181.2 |
|
Interest expense |
|
(3.0 |
) |
|
|
(10.4 |
) |
Other expense, net |
|
(17.6 |
) |
|
|
(31.2 |
) |
Income before provision for income taxes |
|
730.4 |
|
|
|
1,279.1 |
|
Provision for income taxes |
|
84.1 |
|
|
|
179.5 |
|
Net income |
$ |
646.3 |
|
|
$ |
1,099.6 |
|
|
|
|
|
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Net income per common share: |
|
|
|
||||
Basic |
$ |
2.52 |
|
|
$ |
4.26 |
|
Diluted |
$ |
2.49 |
|
|
$ |
4.21 |
|
Shares used in per share calculations: |
|
|
|
||||
Basic |
|
256.9 |
|
|
|
258.2 |
|
Diluted |
|
259.5 |
|
|
|
261.1 |
|
|
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Total Revenues |
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(unaudited, in millions) |
|||||
|
Three Months Ended |
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|
2025 |
|
2024 |
||
TRIKAFTA/KAFTRIO |
$ |
2,535.5 |
|
$ |
2,483.6 |
ALYFTREK |
|
53.9 |
|
|
— |
Other product revenues (1) |
|
170.8 |
|
|
207.0 |
Product revenues, net |
|
2,760.2 |
|
|
2,690.6 |
Other revenues |
|
10.0 |
|
|
— |
Total revenues |
$ |
2,770.2 |
|
$ |
2,690.6 |
1: In the first quarter of 2025, "Other product revenues" included |
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|||||||
Reconciliation of GAAP to Non-GAAP Financial Information |
|||||||
(unaudited, in millions, except percentages) |
|||||||
|
Three Months Ended |
||||||
|
|
2025 |
|
|
|
2024 |
|
GAAP cost of sales |
$ |
363.0 |
|
|
$ |
342.6 |
|
Stock-based compensation expense |
|
(2.6 |
) |
|
|
(1.8 |
) |
Intangible asset amortization expense |
|
(5.0 |
) |
|
|
(5.0 |
) |
Non-GAAP cost of sales |
$ |
355.4 |
|
|
$ |
335.8 |
|
|
|
|
|
||||
GAAP research and development expenses |
$ |
979.7 |
|
|
$ |
789.1 |
|
Stock-based compensation expense |
|
(100.1 |
) |
|
|
(119.4 |
) |
Intangible asset amortization expense |
|
(0.6 |
) |
|
|
— |
|
Non-GAAP research and development expenses |
$ |
879.0 |
|
|
$ |
669.7 |
|
|
|
|
|
||||
Acquired in-process research and development expenses |
$ |
19.8 |
|
|
$ |
76.8 |
|
|
|
|
|
||||
GAAP selling, general and administrative expenses |
$ |
396.4 |
|
|
$ |
342.7 |
|
Stock-based compensation expense |
|
(63.4 |
) |
|
|
(70.7 |
) |
Non-GAAP selling, general and administrative expenses |
$ |
333.0 |
|
|
$ |
272.0 |
|
|
|
|
|
||||
Combined non-GAAP R&D, AIPR&D and SG&A expenses |
$ |
1,231.8 |
|
|
$ |
1,018.5 |
|
|
|
|
|
||||
GAAP other expense, net |
$ |
(17.6 |
) |
|
$ |
(31.2 |
) |
Decrease in fair value of strategic investments |
|
15.0 |
|
|
|
27.0 |
|
Non-GAAP other expense, net |
$ |
(2.6 |
) |
|
$ |
(4.2 |
) |
|
|
|
|
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GAAP provision for income taxes |
$ |
84.1 |
|
|
$ |
179.5 |
|
Tax adjustments (2) |
|
160.1 |
|
|
|
81.6 |
|
Non-GAAP provision for income taxes |
$ |
244.2 |
|
|
$ |
261.1 |
|
GAAP effective tax rate |
11.5 |
% |
|
14.0 |
% |
Non-GAAP effective tax rate |
18.8 |
% |
|
17.4 |
% |
|
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Reconciliation of GAAP to Non-GAAP Financial Information (continued) |
|||||||
(unaudited, in millions, except per share amounts) |
|||||||
|
Three Months Ended |
||||||
|
|
2025 |
|
|
|
2024 |
|
GAAP operating income |
$ |
630.1 |
|
|
$ |
1,139.5 |
|
Stock-based compensation expense |
|
166.1 |
|
|
|
191.9 |
|
Intangible asset impairment charge |
|
379.0 |
|
|
|
— |
|
Intangible asset amortization expense |
|
5.6 |
|
|
|
5.0 |
|
Increase (decrease) in fair value of contingent consideration |
|
2.2 |
|
|
|
(0.1 |
) |
Non-GAAP operating income |
$ |
1,183.0 |
|
|
$ |
1,336.3 |
|
|
|
|
|
||||
GAAP net income |
$ |
646.3 |
|
|
$ |
1,099.6 |
|
|
|
|
|
||||
Stock-based compensation expense |
|
166.1 |
|
|
|
191.9 |
|
Intangible asset impairment charge |
|
379.0 |
|
|
|
— |
|
Intangible asset amortization expense |
|
5.6 |
|
|
|
5.0 |
|
Decrease in fair value of strategic investments |
|
15.0 |
|
|
|
27.0 |
|
Increase (decrease) in fair value of contingent consideration |
|
2.2 |
|
|
|
(0.1 |
) |
Total non-GAAP adjustments to pre-tax income |
|
567.9 |
|
|
|
223.8 |
|
Tax adjustments (2) |
|
(160.1 |
) |
|
|
(81.6 |
) |
Non-GAAP net income |
$ |
1,054.1 |
|
|
$ |
1,241.8 |
|
|
|
|
|
||||
Net income per diluted common share: |
|
|
|
||||
GAAP |
$ |
2.49 |
|
|
$ |
4.21 |
|
Non-GAAP |
$ |
4.06 |
|
|
$ |
4.76 |
|
Shares used in diluted per share calculations: |
|
|
|
||||
GAAP and Non-GAAP |
|
259.5 |
|
|
|
261.1 |
|
2: In the first quarter of 2025 and 2024, “Tax adjustments” included the estimated income taxes related to non-GAAP adjustments to the company's pre-tax income and excess tax benefits related to stock-based compensation. |
|||||||
|
|||||
Condensed Consolidated Balance Sheets |
|||||
(unaudited, in millions) |
|||||
|
|
|
|
||
Assets |
|
|
|
||
Cash, cash equivalents and marketable securities |
$ |
6,201.2 |
|
$ |
6,115.9 |
Accounts receivable, net |
|
1,805.1 |
|
|
1,609.4 |
Inventories |
|
1,359.7 |
|
|
1,205.4 |
Prepaid expenses and other current assets |
|
642.8 |
|
|
665.7 |
Total current assets |
|
10,008.8 |
|
|
9,596.4 |
Property and equipment, net |
|
1,295.9 |
|
|
1,227.8 |
|
|
1,529.2 |
|
|
1,913.9 |
Deferred tax assets |
|
2,544.3 |
|
|
2,331.1 |
Operating lease assets |
|
1,338.5 |
|
|
1,356.8 |
Long-term marketable securities |
|
5,156.5 |
|
|
5,107.9 |
Other long-term assets |
|
1,007.3 |
|
|
999.3 |
Total assets |
$ |
22,880.5 |
|
$ |
22,533.2 |
|
|
|
|
||
Liabilities and Shareholders' Equity |
|
|
|
||
Accounts payable and accrued expenses |
$ |
3,396.8 |
|
$ |
3,201.6 |
Other current liabilities |
|
386.4 |
|
|
363.0 |
Total current liabilities |
|
3,783.2 |
|
|
3,564.6 |
Long-term operating lease liabilities |
|
1,537.7 |
|
|
1,544.4 |
Long-term finance lease liabilities |
|
111.4 |
|
|
112.8 |
Other long-term liabilities |
|
951.9 |
|
|
901.8 |
Shareholders' equity |
|
16,496.3 |
|
|
16,409.6 |
Total liabilities and shareholders' equity |
$ |
22,880.5 |
|
$ |
22,533.2 |
|
|
|
|
||
Common shares outstanding |
|
257.0 |
|
|
256.9 |
About
Special Note Regarding Forward-Looking Statements
This press release 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 statements regarding the intent, belief, or current expectation of
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