Ivacaftor/Lumacaftor

– Total revenue of $2.99 billion, an 8% increase compared to first quarter 2025 – – Povetacicept program continues rapid advancement: Completed rolling BLA submission for U.S. accelerated approval for povetacicept in IgA nephropathy, following positive Phase 3 interim analysis data; initiated Phase 3 portion of Phase 2/3 study in primary membranous nephropathy and Phase 2 proof-of-concept study in generalized myasthenia gravis – – Continued progress across broad clinical-stage pipeline, including label expansion of eligible U.S. patient populations for ALYFTREK and TRIKAFTA and completion of U.S. regulatory submission for approval of CASGEVY in children ages 5 to less than 12 years old with SCD or TDT– BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the first quarter ended March 31, 2026, and reiterated its full year 2026 financial guidance. “Vertex is off to a strong start in 2026, driven by leadership in cystic fibrosis; growth in sickle cell disease, beta thalassemia, and acute pain; as well as rapid pipeline progress,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “CASGEVY and JOURNAVX delivered more than 25 percent of our growth this quarter, underscoring the strength of the increasingly diversified revenue base. As we execute across the commercial portfolio and pipeline and build our fourth franchise in nephrology, Vertex is poised to continue to deliver for patients and create long-term value.” First Quarter 2026 Results Total revenue increased 8% to $2.99 billion compared to the first quarter of 2025, primarily driven by the continued performance of cystic fibrosis (CF) therapies and growth from diversification into additional disease areas. In the U.S., total revenue increased 7% to $1.78 billion due to continued strong CF patient demand, including from new initiations of ALYFTREK; higher realized net prices in CF versus the prior year; and contributions from CASGEVY and JOURNAVX. Outside the U.S., total revenue increased 9% to $1.21 billion due to strong CF performance across multiple geographies, including ALYFTREK uptake, increased CASGEVY revenue, and a favorable impact from foreign exchange. Combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses were $1.5 billion and $1.3 billion, respectively, in the first quarter of 2026, compared to $1.4 billion and $1.2 billion, respectively, for the first quarter of 2025. These increases were primarily due to commercial investment to support the launch of JOURNAVX in acute pain and the build-out of the renal franchise, led by povetacicept in IgAN. GAAP effective tax rate was 17.7% compared to 11.5% for the first quarter of 2025, primarily due to higher excess tax benefits related to stock-based compensation and lower pre-tax book income in the first quarter of 2025 due to an intangible asset impairment charge. Non-GAAP effective tax rate was 19.6% compared to 18.8% for the first quarter of 2025. GAAP net income was $1.0 billion compared to $646 million for the first quarter of 2025, as a result of increased product revenue, partially offset by increased operating expenses and income tax expenses. In addition, first quarter 2025 results included a $379.0 million intangible asset impairment charge. Non-GAAP net income was $1.1 billion, an increase of $93 million compared to the first quarter of 2025, primarily due to increased product revenue, partially offset by increased operating and income tax expenses in the first quarter of 2026. Cash, cash equivalents, and total marketable securities as of March 31, 2026, were $13.0 billion, compared to $12.3 billion as of December 31, 2025. The increase was primarily due to cash flows from operating activities, partially offset by repurchases of Vertex’s common stock pursuant to its share repurchase programs. Full Year 2026 Financial Guidance Vertex today reiterated full year 2026 financial guidance. Vertex’s total revenue guidance of $12.95 billion to $13.1 billion includes expectations for continued growth in CF, including the ongoing U.S. rollout and ex-U.S. launches of ALYFTREK, as well as $500 million or more in revenue from non-CF products, including increased patient infusions of CASGEVY through Vertex’s global ATC network and growth in prescriptions and revenue from the second year of the launch of JOURNAVX. Vertex’s guidance for both combined GAAP and non-GAAP R&D, AIPR&D, and SG&A expenses includes expectations for continued investment in multiple mid- and late-stage clinical development programs and commercialization capabilities, and approximately $100 million of currently anticipated AIPR&D expenses. This guidance also includes an immaterial cost impact from tariffs in 2026 based on currently known tariff rates and regulations. Vertex’s financial guidance is summarized below: Current FY 2026 Previous FY 2026 Total revenue Unchanged $12.95 to $13.1 billion Non-CF product revenue Unchanged $0.5 billion or greater Combined GAAP R&D, AIPR&D and SG&A expenses * Unchanged $6.3 to $6.45 billion Combined non-GAAP R&D, AIPR&D and SG&A expenses* Unchanged $5.65 to $5.75 billion Non-GAAP effective tax rate Unchanged 19.5% to 20.5% *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 $650 million to $700 million of stock-based compensation expense. **Combined GAAP and non-GAAP R&D, AIPR&D and SG&A expenses guidance includes approximately $100 million of AIPR&D expenses. Key Business Highlights Marketed Products Cystic Fibrosis (CF) Portfolio Vertex has worked for more than 20 years to discover and develop medicines to treat the underlying cause of CF. Vertex CFTR modulators can treat approximately 95 percent of all people living with CF in core markets, including patients as young as one month old. ALYFTREK, the newest marketed CFTR modulator, is approved in the U.S., the United Kingdom (U.K.), the European Union (EU), Canada, New Zealand, Switzerland, Australia, and Israel for the treatment of patients 6 years and older. Vertex anticipates that the number of CF patients taking its medicines will continue to grow through new approvals and reimbursement agreements, treatment of younger patients, increased survival, and expansion into additional geographies. Recent progress includes: The U.S. Food and Drug Administration (FDA) recently approved label extensions for ALYFTREK and TRIKAFTA, expanding availability of these medicines to approximately 95% of all people with CF in the United States. This label expansion was supported by clinical and/or in vitro data from 564 variants demonstrating response to ALYFTREK and 521 variants demonstrating response to TRIKAFTA. With this approval, approximately 800 more people with CF in the U.S. are now eligible for the first time for a medicine that treats the underlying cause of their disease. Vertex recently secured reimbursement agreements for ALYFTREK in Scotland, Spain, Sweden, Switzerland, New Zealand, Israel, and Finland and is working to secure access for eligible patients in additional countries. Following recently reported positive results from the study of ALYFTREK in children ages two to five years, Vertex is on track to submit for global regulatory approvals in the first half of 2026. Vertex continues to enroll and dose the pivotal study of ALYFTREK in children ages one to less than two years. Following recently reported positive results from the study of TRIKAFTA in children ages one to less than two years, Vertex has begun submissions for global regulatory approvals for TRIKAFTA in this age group. CASGEVY for the treatment of severe 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 U.S., the U.K., the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain, Qatar, Canada, Switzerland, the United Arab Emirates (UAE), and Kuwait for patients 12 years and older with SCD or TDT. In total, there are more than 60,000 eligible patients in these countries, including approximately 37,000 in North America and Europe and more than 23,000 in the Middle East. Recent highlights include: Vertex recorded first quarter 2026 CASGEVY revenue of $43 million. Vertex recently secured a pricing agreement for CASGEVY for eligible patients with SCD or TDT in Germany. Vertex is now working through final implementation to provide long-term reimbursed access to patients at a sustainable price. Vertex completed the regulatory submission in the U.S. for approval of CASGEVY in children ages 5 to less than 12 years old with SCD or TDT. The FDA awarded Vertex a Commissioner’s National Priority Voucher for this pediatric submission, indicating an accelerated timeline for review once the submission is accepted. JOURNAVX (suzetrigine) for the treatment of moderate-to-severe acute pain JOURNAVX is a first-in-class, oral, selective, non-opioid Na V 1.8 pain signal inhibitor, approved in the U.S. for the treatment of moderate-to-severe acute pain. Since the launch of JOURNAVX in March 2025, more than 1 million prescriptions have now been filled for JOURNAVX across the hospital and retail settings for a broad range of acute pain conditions. In the first quarter of 2026, more than 350,000 prescriptions were filled, and Vertex recorded revenue of $29 million. The Centers for Medicare and Medicaid Services (CMS) have approved the inclusion of JOURNAVX in the NOPAIN Act separate payment list, with a retroactive payment date of January 23, 2026. Addition to the NOPAIN list provides a separate payment for non-opioid medicines such as JOURNAVX in the hospital outpatient and ambulatory surgical center settings. Vertex has reached an agreement with a major pharmacy benefit manager for Medicare Part D coverage for JOURNAVX, effective May 1. The agreement adds approximately 10 million lives covered under Part D. Twenty-two states now provide coverage for JOURNAVX via Medicaid. In total, approximately 240 million individuals now have reimbursed access to JOURNAVX across a wide range of commercial and government payers. Select R&D Pipeline Programs Cystic Fibrosis Consistent with its commitment to serial innovation and bringing as many patients as possible to normal levels of CFTR function, Vertex is evaluating VX-828, the first of the next-generation 3.0 CFTR corrector class, in a proof-of-concept study in people with CF. Vertex is on track to complete dosing in this study in the first half of 2026 and share results in the second half. Vertex is enrolling and dosing first-in-human studies with VX-581 and VX-272, additional next-generation 3.0 CFTR correctors. Vertex has ended the Phase 1/2 study of VX-522 after observing persistent tolerability issues in the study. The early termination precludes assessment of efficacy and full safety and prevents further development of the VX-522 program. Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia Vertex continues to advance preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population. Acute and Peripheral Neuropathic Pain (PNP) Vertex is on track to complete enrollment in both Phase 3 studies of suzetrigine in diabetic peripheral neuropathy (DPN), a form of peripheral neuropathic pain (PNP), by the end of 2026. Vertex also continues to enroll and dose people with DPN in a Phase 2 study of VX-993. Vertex continues to advance preclinical assets that inhibit Na V 1.7 for use alone or in combination with a Na V 1.8 inhibitor in acute and neuropathic pain. IgA Nephropathy (IgAN) and Other B Cell-Mediated Diseases Vertex is developing povetacicept for multiple diseases. Povetacicept is a dual inhibitor of the BAFF and APRIL cytokines, which play key roles in the pathogenesis of multiple B cell-mediated autoimmune diseases. Povetacicept has pipeline-in-a-product potential and represents a potentially best-in-class approach to control B cell activity in IgAN, primary membranous nephropathy (pMN), and generalized myasthenia gravis (gMG). In March, Vertex reported positive Week 36 interim analysis results for the primary and all secondary endpoints in the RAINIER Phase 3 trial of povetacicept in adults with IgAN. Based on these results, Vertex completed the submission of its rolling biologics license application (BLA) to the FDA in March for potential accelerated approval in the U.S. Vertex is using a Priority Review Voucher and therefore expects the FDA review of povetacicept’s BLA to be expedited to six months from the date of the FDA’s acceptance of the BLA. Vertex recently completed enrollment in the Phase 2 portion of the Phase 2/3 OLYMPUS pivotal study of povetacicept in people with pMN and initiated the Phase 3 portion. Enrollment and dosing in the trial are ongoing. The FDA has granted Fast Track and Orphan Drug designations for povetacicept in pMN, and the EMA has granted Priority Medicines (PRIME) designation. Vertex has initiated a placebo-controlled, Phase 2 dose-ranging proof-of-concept study evaluating povetacicept for the treatment of gMG. APOL1-Mediated Kidney Disease (AMKD) Vertex has discovered and advanced multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that targets the underlying cause of this genetic kidney disease. In the second half of 2025, Vertex completed enrollment in the interim analysis cohort of the AMPLITUDE Phase 2/3 trial of inaxaplin in people with primary AMKD and will conduct the pre-planned interim analysis for potential accelerated approval after this cohort reaches 48 weeks of treatment. Vertex expects to share data from the interim analysis in early 2027. The AMPLITUDE study is on track to complete full enrollment in the second half of 2026. Vertex has completed enrollment in the AMPLIFIED Phase 2 study of inaxaplin. AMPLIFIED is a study of people with AMKD with moderate proteinuria, and people with AMKD and Type 2 diabetes — populations not being studied in the AMPLITUDE trial. Vertex is on track to complete dosing and share data from the AMPLIFIED study in the second half of 2026. Type 1 Diabetes (T1D) Vertex is evaluating stem cell-derived, fully differentiated islet cell therapies for patients suffering from T1D, with the goal of developing a potential one-time functional cure for this disease. Vertex has completed the internal manufacturing analysis for the Phase 1/2/3 study of zimislecel in people with T1D and has resumed dosing in the study. Multiple patients have been treated since the resumption of dosing. The company expects to provide updated timelines for study completion later this year. Autosomal Dominant Polycystic Kidney Disease (ADPKD) Vertex is developing small molecule correctors that restore function to polycystin 1 (PC1) protein variants, with the goal of addressing the underlying cause of ADPKD. Vertex is enrolling and dosing AGLOW, a Phase 2 study of VX-407 in patients with a subset of variants in the PKD1 gene, which encodes the PC1 protein, estimated to be up to approximately 30,000 (or up to approximately 10%) of the overall patient population living with ADPKD. AGLOW is a 24-patient, single-arm, 52-week, Phase 2 proof-of-concept study that will evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV). AGLOW is on track to complete enrollment in the second half of 2026. Myotonic Dystrophy Type 1 (DM1) Vertex is evaluating multiple approaches that target the underlying cause of DM1. Vertex’s lead approach, VX-670, is an oligonucleotide linked to a cyclic peptide, which holds the potential to promote effective delivery into cells and address the causal biology of DM1. Vertex continues to enroll and dose the MAD portion of the GALILEO global Phase 1/2 clinical trial of VX-670 in people with DM1; the study is assessing both safety and efficacy. Vertex is on track to complete enrollment and dosing in the trial and share results in the second half of 2026. Additional Earlier Stage R&D Programs Consistent with its overall strategy, Vertex takes a serial innovation approach to all of its programs, with additional assets or approaches across its portfolio. Non-GAAP Financial Measures In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (i) stock-based compensation expense, (ii) intangible asset amortization expense, (iii) gains or losses related to the fair value of the company's strategic investments, (iv) increases or decreases in the fair value of contingent consideration, (v) an intangible asset impairment charge, and (vi) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company’s calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information. 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. Vertex Pharmaceuticals Incorporated Consolidated Statements of Income (unaudited, in millions, except per share amounts) Three Months Ended March 31, 2026 2025 Revenues: Product revenues, net $ 2,986.9 $ 2,760.2 Other revenues — 10.0 Total revenues 2,986.9 2,770.2 Costs and expenses: Cost of sales 392.8 363.0 Research and development expenses 961.6 979.7 Acquired in-process research and development expenses 0.5 19.8 Selling, general and administrative expenses 493.7 396.4 Intangible asset impairment charge — 379.0 Change in fair value of contingent consideration 0.2 2.2 Total costs and expenses 1,848.8 2,140.1 Income from operations 1,138.1 630.1 Interest income, net 114.8 117.9 Other expense, net — (17.6 ) Income before provision for income taxes 1,252.9 730.4 Provision for income taxes 221.5 84.1 Net income $ 1,031.4 $ 646.3 Net income per common share: Basic $ 4.06 $ 2.52 Diluted $ 4.02 $ 2.49 Shares used in per share calculations: Basic 254.1 256.9 Diluted 256.3 259.5 Vertex Pharmaceuticals Incorporated Total Revenues (unaudited, in millions) Three Months Ended March 31, 2026 2025 TRIKAFTA/KAFTRIO $ 2,354.7 $ 2,535.5 ALYFTREK 424.4 53.9 Other CF product revenues (1) 135.9 155.3 Total CF product revenues, net 2,915.0 2,744.7 CASGEVY 42.9 14.2 JOURNAVX 29.0 1.3 Product revenues, net 2,986.9 2,760.2 Other revenues — 10.0 Total revenues $ 2,986.9 $ 2,770.2 1: Includes KALYDECO, ORKAMBI, and SYMDEKO/SYMKEVI Vertex Pharmaceuticals Incorporated Reconciliation of GAAP to Non-GAAP Financial Information (unaudited, in millions, except percentages) Three Months Ended March 31, 2026 2025 GAAP cost of sales $ 392.8 $ 363.0 Stock-based compensation expense (3.2 ) (2.6 ) Intangible asset amortization expense (5.0 ) (5.0 ) Non-GAAP cost of sales $ 384.6 $ 355.4 GAAP research and development expenses $ 961.6 $ 979.7 Stock-based compensation expense (101.7 ) (100.1 ) Intangible asset amortization expense (0.6 ) (0.6 ) Non-GAAP research and development expenses $ 859.3 $ 879.0 Acquired in-process research and development expenses $ 0.5 $ 19.8 GAAP selling, general and administrative expenses $ 493.7 $ 396.4 Stock-based compensation expense (61.5 ) (63.4 ) Non-GAAP selling, general and administrative expenses $ 432.2 $ 333.0 Combined non-GAAP R&D, AIPR&D and SG&A expenses $ 1,292.0 $ 1,231.8 GAAP other expense, net $ — $ (17.6 ) Decrease in fair value of strategic investments 2.0 15.0 Non-GAAP other income (expense), net $ 2.0 $ (2.6 ) GAAP provision for income taxes $ 221.5 $ 84.1 Tax adjustments (2) 58.6 160.1 Non-GAAP provision for income taxes $ 280.1 $ 244.2 GAAP effective tax rate 17.7 % 11.5 % Non-GAAP effective tax rate 19.6 % 18.8 % Vertex Pharmaceuticals Incorporated Reconciliation of GAAP to Non-GAAP Financial Information (continued) (unaudited, in millions, except per share amounts) Three Months Ended March 31, 2026 2025 GAAP operating income $ 1,138.1 $ 630.1 Stock-based compensation expense 166.4 166.1 Intangible asset impairment charge — 379.0 Intangible asset amortization expense 5.6 5.6 Increase in fair value of contingent consideration 0.2 2.2 Non-GAAP operating income $ 1,310.3 $ 1,183.0 GAAP net income $ 1,031.4 $ 646.3 Stock-based compensation expense 166.4 166.1 Intangible asset impairment charge — 379.0 Intangible asset amortization expense 5.6 5.6 Decrease in fair value of strategic investments 2.0 15.0 Increase in fair value of contingent consideration 0.2 2.2 Total non-GAAP adjustments to pre-tax income 174.2 567.9 Tax adjustments (2) (58.6 ) (160.1 ) Non-GAAP net income $ 1,147.0 $ 1,054.1 Net income per diluted common share: GAAP $ 4.02 $ 2.49 Non-GAAP $ 4.47 $ 4.06 Shares used in diluted per share calculations: GAAP and Non-GAAP 256.3 259.5 2: In the three months ended March 31, 2026 and 2025, “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. Vertex Pharmaceuticals Incorporated Condensed Consolidated Balance Sheets (unaudited, in millions) March 31, 2026 December 31, 2025 Assets Cash, cash equivalents and marketable securities $ 7,246.7 $ 6,608.1 Accounts receivable, net 1,996.1 2,052.8 Inventories 1,766.7 1,686.8 Prepaid expenses and other current assets 720.8 853.3 Total current assets 11,730.3 11,201.0 Property and equipment, net 1,608.4 1,520.3 Goodwill and other intangible assets, net 1,506.5 1,512.2 Deferred tax assets 2,947.8 2,897.9 Operating lease assets 1,685.1 1,562.7 Long-term marketable securities 5,749.9 5,712.3 Other long-term assets 1,256.4 1,236.6 Total assets $ 26,484.4 $ 25,643.0 Liabilities and Shareholders' Equity Accounts payable and accrued expenses $ 3,473.5 $ 3,432.9 Other current liabilities 407.1 428.3 Total current liabilities 3,880.6 3,861.2 Long-term operating lease liabilities 1,986.5 1,846.5 Other long-term liabilities 1,255.4 1,269.5 Shareholders' equity 19,361.9 18,665.8 Total liabilities and shareholders' equity $ 26,484.4 $ 25,643.0 Common shares outstanding 254.2 254.0 Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com or 617-961-7163 Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 Read full story here

— Full year total revenue of $12.0 billion, a 9% increase compared to full year 2024; fourth quarter total revenue of $3.19 billion, a 10% increase compared to fourth quarter 2024 — — Company provides full year 2026 total revenue guidance of $12.95 billion to $13.1 billion, with non-CF products expected to contribute $500 million or more in revenue — — Broad mid- and late-stage clinical pipeline accelerates with multiple proof-of-concept and pivotal programs advancing; on track to complete BLA filing for U.S. accelerated approval of povetacicept in IgAN in the first half of 2026 — BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the fourth quarter and full year ended December 31, 2025, and provided its full year 2026 financial guidance. “2025 marked a year of strong revenue growth, commercial diversification, and pipeline advancement,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “Our focus in 2026 remains on executing across the CF franchise, bringing CASGEVY to more patients around the globe and continuing to launch JOURNAVX, as we also prepare for the anticipated near‑term commercialization of povetacicept in IgAN. With expanding leadership in CF, exciting commercial momentum, and multiple mid- and late-stage programs advancing, Vertex is well positioned to deliver long‑term value for patients and shareholders.” Fourth Quarter 2025 Results Total revenue increased 10% to $3.19 billion compared to the fourth quarter of 2024, primarily driven by the continued performance of cystic fibrosis (CF) therapies and additional growth from diversification into additional disease areas. In the U.S., total revenue increased 12% to $2.06 billion due to continued strong CF patient demand, including for ALYFTREK; a modest benefit from CF channel inventory; higher realized net prices in CF versus the prior year; and contributions from CASGEVY and JOURNAVX. Outside the U.S., total revenue increased 5% to $1.13 billion due to solid CF performance across multiple geographies and increased CASGEVY revenue. Combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses were $1.52 billion and $1.36 billion, respectively, in the fourth quarter of 2025, compared to $1.46 billion and $1.30 billion, respectively, for the fourth quarter of 2024. These increases were primarily due to commercial investment to support the launch of JOURNAVX in acute pain. GAAP and non-GAAP effective tax rates were 10.5% and 13.5%, respectively, compared to 19.7% and 21.3%, respectively, for the fourth quarter of 2024. In the fourth quarter of 2025, the tax rates incorporated a one-time benefit from recognition of previously deferred tax credits and a change in estimated prior-year liabilities. GAAP and non-GAAP net income were $1.2 billion and $1.3 billion, respectively, compared to $913 million and $1.0 billion, respectively, for the fourth quarter of 2024, primarily driven by increased product revenue. Full Year 2025 Results Total revenue of $12.0 billion increased 9% compared to 2024, primarily driven by the continued performance of CF therapies and early contributions from the three ongoing launches. In the U.S., total revenue increased 13% to $7.55 billion due to continued strong CF patient demand, including for ALYFTREK, and higher realized net prices in CF versus the prior year; as well as contributions from CASGEVY and JOURNAVX. Outside the U.S., total revenue increased 3% to $4.45 billion due to solid CF performance across multiple geographies and contributions from CASGEVY. Combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses were $5.8 billion and $5.1 billion, respectively, compared to $9.7 billion and $8.8 billion, respectively, in 2024. The decreases were primarily due to $4.4 billion of AIPR&D expenses associated with Vertex’s acquisition of Alpine Immune Sciences incurred in the second quarter of 2024, partially offset by increased R&D investment in support of multiple mid- and late-stage clinical development programs and increased commercial investment to support the launch of JOURNAVX. GAAP and non-GAAP effective tax rates were 14.9% and 17.3%, respectively, compared to 315.5% and 91.0%, respectively, in 2024. In 2025, the effective tax rates were lower than U.S. statutory rates primarily due to one-time benefits from recognition of previously deferred tax credits and Alpine-related R&D tax credits. The GAAP effective tax rate also included excess tax benefits related to stock-based compensation. GAAP and Non-GAAP net income were $4.0 billion and $4.7 billion, respectively, compared to a GAAP net loss of $(536) million and non-GAAP net income of $111 million, respectively, for 2024, reflecting the Alpine AIPR&D in 2024 and increased product revenue partially offset by increased operating expenses. Cash, cash equivalents, and total marketable securities as of December 31, 2025, were $12.3 billion, compared to $11.2 billion as of December 31, 2024. The increase was primarily due to cash flows from operating activities, partially offset by repurchases of Vertex’s common stock pursuant to its share repurchase programs. Full Year 2026 Financial Guidance Vertex today provided full year 2026 financial guidance. Vertex’s total revenue guidance of $12.95 billion to $13.1 billion includes expectations for continued growth in CF, including the ongoing U.S. rollout and ex-U.S. launches of ALYFTREK; as well as $500 million or more in revenue from non-CF products, including increased patient infusions of CASGEVY through Vertex’s global ATC network and growth in prescriptions and revenue from the second year of the launch of JOURNAVX. Vertex’s guidance for both combined GAAP and non-GAAP R&D, AIPR&D and SG&A expenses includes expectations for continued investment in multiple mid- and late-stage clinical development programs and commercial and manufacturing capabilities, and approximately $100 million of currently anticipated AIPR&D expenses. This guidance also includes an immaterial cost impact from tariffs in 2026 based on currently known tariff rates and regulations. Vertex’s financial guidance is summarized below: FY 2026 Total revenue $12.95 to $13.1 billion Non-CF product revenue $0.5 billion or greater Combined GAAP R&D, AIPR&D and SG&A expenses * $6.3 to $6.45 billion Combined non-GAAP R&D, AIPR&D and SG&A expenses * $5.65 to $5.75 billion Non-GAAP effective tax rate 19.5% to 20.5% *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 $650 million to $700 million of stock-based compensation expense. **Combined GAAP and non-GAAP R&D, AIPR&D and SG&A expenses guidance includes approximately $100 million of AIPR&D expenses. Key Business Highlights Marketed Products Cystic Fibrosis (CF) Portfolio Vertex has worked for more than 20 years to discover and develop medicines to treat the underlying cause of CF. Vertex CFTR modulators can treat nearly 95 percent of all people living with CF in core markets and are approved for patients as young as one month old. ALYFTREK, the newest marketed CFTR modulator, is approved in the U.S., the United Kingdom (U.K.), the European Union (EU), Canada, New Zealand, Switzerland, Australia, and Israel for the treatment of patients 6 years and older. Vertex anticipates that the number of CF patients taking its medicines will continue to grow through new approvals and reimbursement agreements, treatment of younger patients, increased survival, and expansion into additional geographies. Recent progress includes: ALYFTREK is reimbursed for eligible patients in the U.S., England, Ireland, Germany, Denmark, Northern Ireland, Norway, Wales, Italy, Australia, New Zealand, and Luxembourg. Vertex is working to secure access for eligible patients in additional countries. In January, Vertex completed the pivotal study of ALYFTREK in children 2 to 5 years of age. The data showed that ALYFTREK was generally safe and well tolerated, consistent with the established safety profile. Treatment with ALYFTREK resulted in a clinically meaningful improvement in CFTR function with a mean reduction in sweat chloride from a TRIKAFTA baseline of -9.6 mmol/L (95% CI -12.1 to -7.0) through Week 24, with 65% of children reaching a normal sweat chloride value of <30mmol/L. These are unprecedented results for this age group. Vertex expects to submit for approval with global regulators in the first half of 2026. Vertex recently initiated a pivotal study of ALYFTREK in children 1 to less than 2 years of age. Following positive results from the study of TRIKAFTA in patients one year to less than two years of age, reported in November 2025, Vertex expects to begin submissions for global regulatory approvals in this age group in the first half of 2026. CASGEVY for the treatment of severe 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 U.S., the U.K., the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain, Qatar, Canada, Switzerland, the United Arab Emirates (UAE), and Kuwait for patients 12 years and older with SCD or TDT. In total, there are more than 60,000 eligible patients in these countries, including approximately 37,000 in North America and Europe and more than 23,000 in the Middle East. Recent highlights include: Vertex recorded fourth quarter 2025 CASGEVY revenue of $54 million and full year 2025 revenue of $116 million. This reflects 64 patients receiving infusions of CASGEVY in 2025, including 30 people infused in the fourth quarter of 2025. Globally, in 2025, 147 people with SCD or TDT had their first cell collection for CASGEVY. As of the end of 2025, approximately 90 percent of patients in the U.S. have reimbursed access to CASGEVY, which is also reimbursed in the U.K., Italy, Austria, Denmark, Luxembourg, KSA, Bahrain, UAE, and Kuwait. Most recently, in January, Vertex secured reimbursed access to CASGEVY for eligible patients with SCD in Scotland, consistent with the reimbursement agreement reached in 2025 for patients with TDT. At the American Society of Hematology (ASH) annual meeting in December 2025, Vertex presented positive data from the pivotal studies of CASGEVY in children ages 5 to 11 years old with SCD or TDT, which was highlighted by the conference in the “Best of ASH” presentations. Vertex expects to begin global regulatory submissions for approvals in this age group in the first half of 2026. The U.S. Food and Drug Administration (FDA) awarded Vertex a Commissioner’s National Priority Voucher for this pediatric submission, indicating an accelerated timeline for review once the submission is complete. JOURNAVX (suzetrigine) for the treatment of moderate-to-severe acute pain JOURNAVX is a first-in-class, oral, selective, non-opioid Na V 1.8 pain signal inhibitor, approved in the U.S. for the treatment of moderate-to-severe acute pain. Since JOURNAVX became available at pharmacies in early March, through year-end 2025, more than 550,000 prescriptions for JOURNAVX have been written and filled across the hospital and retail settings in different acute pain conditions, consistent with JOURNAVX’s broad label. Vertex secured commercial coverage for JOURNAVX with the remaining large national pharmacy benefit manager (PBM), effective January 1, 2026, and now has secured access for JOURNAVX with all three national PBMs. As of January 2026, over 200 million individuals across commercial and government payers have coverage, representing two-thirds of U.S. covered lives. In addition, 21 states now provide coverage via Medicaid. More than 100 of Vertex’s targeted 150 healthcare systems and more than 950 individual hospitals of the 2,000 targeted institutions have added JOURNAVX to formularies, protocols or order sets. Select R&D Pipeline Programs Cystic Fibrosis Consistent with its commitment to serial innovation and bringing as many patients as possible to normal levels of CFTR function, Vertex has advanced VX-828, the first of the next-generation 3.0 CFTR corrector class, into a proof-of-concept study in people with CF. Vertex expects to complete enrollment and dosing in the first half of 2026. Vertex is enrolling and dosing a Phase 1 study of VX-581, another corrector in the next-generation 3.0 class, in healthy volunteers. Vertex is targeting completion of dosing in the multiple ascending dose (MAD) portion of the Phase 1/2 study of VX-522 and disclosure of the data in the second half of 2026. VX-522 is a CFTR mRNA therapeutic that Vertex is developing in collaboration with Moderna for the approximately 5,000 people with CF who cannot benefit from CFTR modulators. Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia Vertex continues to advance preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population. Peripheral Neuropathic Pain (PNP) Vertex continues to expect to complete enrollment in both Phase 3 studies of suzetrigine in diabetic peripheral neuropathy (DPN), a form of peripheral neuropathic pain (PNP), by the end of 2026. Vertex also continues to enroll and dose people with DPN in a Phase 2 study of VX-993. Vertex continues to advance preclinical assets that inhibit Na V 1.7 for use alone or in combination with a Na V 1.8 inhibitor in acute and neuropathic pain. IgA Nephropathy (IgAN) and Other B Cell-Mediated Diseases Vertex is developing povetacicept for multiple diseases. Povetacicept is a dual inhibitor of the BAFF and APRIL cytokines, which play key roles in the pathogenesis of multiple B cell-mediated autoimmune diseases. Povetacicept has pipeline-in-a-product potential and represents a potentially best-in-class approach to control B cell activity in IgAN, primary membranous nephropathy (pMN), and generalized myasthenia gravis (gMG). In the fourth quarter of 2025, Vertex initiated the rolling biologics license application (BLA) filing for U.S. accelerated approval of povetacicept in IgAN with submission of the first module. The FDA has granted Breakthrough Therapy Designation for this indication. Vertex remains on track to release interim analysis data in the first half of 2026 and also complete the submission in the first half of 2026, if data from the interim analysis are supportive. Vertex is using a priority review voucher to expedite the review of the povetacicept BLA from ten months to six months. Vertex continues to enroll and dose patients in the adaptive Phase 2/3 OLYMPUS pivotal study of povetacicept in people with pMN. Vertex anticipates completing the Phase 2 portion of the study and initiating the Phase 3 portion in mid-2026. The FDA has granted Fast Track and Orphan Drug designations for povetacicept in pMN, and the EMA has granted Priority Medicines (PRIME) designation. Vertex expects to initiate a placebo-controlled, Phase 2 dose-ranging proof-of-concept study evaluating povetacicept for the treatment of gMG in the first half of 2026. APOL1-Mediated Kidney Disease (AMKD) Vertex has discovered and advanced multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that targets the underlying cause of this genetic kidney disease. In September, Vertex completed enrollment in the interim analysis cohort of the AMPLITUDE Phase 2/3 trial of inaxaplin in people with primary AMKD and will conduct the pre-planned interim analysis once this cohort reaches 48 weeks of treatment. Vertex expects to share data from the interim analysis in late 2026 or early 2027. The AMPLITUDE study is on track to complete full enrollment in the second half of 2026. Vertex expects to complete the AMPLIFIED Phase 2 study of inaxaplin and share data in mid-2026. AMPLIFIED is a study of people with AMKD with moderate proteinuria, and people with AMKD and Type 2 diabetes — populations not being studied in the AMPLITUDE trial. Type 1 Diabetes (T1D) Vertex is evaluating stem cell-derived, fully differentiated islet cell therapies for patients suffering from T1D, with the goal of developing a potential one-time functional cure for this disease. Vertex has completed enrollment in the Phase 1/2/3 study of zimislecel in people with T1D and has temporarily postponed completion of dosing in the study, pending an ongoing internal manufacturing analysis. Zimislecel has been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration, PRIME designation from the EMA, Breakthrough Medicine designation from the Kingdom of Saudi Arabia, and has secured an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). Autosomal Dominant Polycystic Kidney Disease (ADPKD) Vertex is developing small molecule correctors that restore function to polycystin 1 (PC1) protein variants, with the goal of addressing the underlying cause of ADPKD. Vertex is enrolling and dosing AGLOW, a Phase 2 proof-of-concept study of VX-407 in patients with a subset of variants in the PKD1 gene, which encodes the PC1 protein, estimated to be up to approximately 30,000 (or up to approximately 10%) of the overall patient population living with ADPKD. AGLOW is a 24-patient single-arm study that will evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV). Vertex expects to complete enrollment in the AGLOW study by the end of 2026. Myotonic Dystrophy Type 1 (DM1) Vertex is evaluating multiple approaches that target the underlying cause of DM1. Vertex’s lead approach, VX-670, is an oligonucleotide linked to a cyclic peptide, which holds the potential to promote effective delivery into cells and address the causal biology of DM1. Vertex continues to enroll and dose the MAD portion of the GALILEO global Phase 1/2 clinical trial of VX-670 in people with DM1; the study is assessing both safety and efficacy. Vertex is on track to complete enrollment and dosing in the trial in mid-2026. Additional Earlier Stage R&D Programs Consistent with its overall strategy, Vertex takes a serial innovation approach to all of its programs, with additional assets or approaches across its portfolio. External Innovation Consistent with its strategy to develop transformative medicines for serious diseases, Vertex entered into the following transaction: An exclusive global license agreement with WuXi Biologics to develop and commercialize a trispecific T cell engager for B cell-mediated autoimmune diseases, which is currently in preclinical development. Non-GAAP Financial Measures In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (loss) (i) stock-based compensation expense, (ii) intangible asset amortization expense, (iii) gains or losses related to the fair value of the company's strategic investments, (iv) increases or decreases in the fair value of contingent consideration, (v) acquisition-related costs, (vi) an intangible asset impairment charge, and (vii) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income (loss) described above and certain discrete items. These results should not be viewed as a substitute for the company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company’s calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information. 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. Vertex Pharmaceuticals Incorporated Consolidated Statements of Income (Loss) (unaudited, in millions, except per share amounts) Three Months Ended December 31, Twelve Months Ended December 31, 2025 2024 2025 2024 Revenues: Product revenues, net $ 3,190.0 $ 2,912.0 $ 11,970.6 $ 11,020.1 Other revenues — — 30.7 — Total revenues 3,190.0 2,912.0 12,001.3 11,020.1 Costs and expenses: Cost of sales 466.0 423.4 1,651.3 1,530.5 Research and development expenses 973.7 998.7 3,909.5 3,630.3 Acquired in-process research and development expenses 56.5 87.5 133.0 4,628.4 Selling, general and administrative expenses 487.0 377.6 1,753.1 1,464.3 Intangible asset impairment charge — — 379.0 — Change in fair value of contingent consideration 0.9 (1.2 ) 2.1 (0.5 ) Total costs and expenses 1,984.1 1,886.0 7,828.0 11,253.0 Income (loss) from operations 1,205.9 1,026.0 4,173.3 (232.9 ) Interest income 121.9 128.2 490.9 598.1 Interest expense (3.3 ) (2.8 ) (13.3 ) (30.6 ) Other income (expense), net 6.5 (14.9 ) (7.7 ) (86.1 ) Income before provision for income taxes 1,331.0 1,136.5 4,643.2 248.5 Provision for income taxes 139.9 223.5 690.0 784.1 Net income (loss) $ 1,191.1 $ 913.0 $ 3,953.2 $ (535.6 ) Net income (loss) per common share: Basic $ 4.69 $ 3.55 $ 15.46 $ (2.08 ) Diluted $ 4.65 $ 3.50 $ 15.32 $ (2.08 ) Shares used in per share calculations: Basic 253.9 257.5 255.7 257.9 Diluted 256.1 260.5 258.0 257.9 Vertex Pharmaceuticals Incorporated Total Revenues (unaudited, in millions) Three Months Ended December 31, Twelve Months Ended December 31, 2025 2024 2025 2024 TRIKAFTA/KAFTRIO $ 2,572.5 $ 2,720.8 $ 10,312.7 $ 10,238.6 ALYFTREK 380.1 — 837.8 — Other product revenues (1) 237.4 191.2 820.1 781.5 Product revenues, net 3,190.0 2,912.0 11,970.6 11,020.1 Other revenues — — 30.7 — Total revenues $ 3,190.0 $ 2,912.0 $ 12,001.3 $ 11,020.1 1: In the three and twelve months ended December 31, 2025, “Other product revenues” included $54.3 million and $115.8 million from CASGEVY, respectively, and $26.7 million and $59.6 million, respectively, from JOURNAVX. In the three and twelve months ended December 31, 2024, “Other product revenues” included $8.0 million and $10.0 million from CASGEVY, respectively, and no revenues from JOURNAVX. The remaining “Other product revenues” are related to KALYDECO, ORKAMBI, and SYMDEKO/SYMKEVI, our other CF products. Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com or 617-961-7163 Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 Read full story here