Ivacaftor

— 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)--Feb. 12, 2026-- 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 NaV1.8 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 NaV1.7 for use alone or in combination with a NaV1.8 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. Vertex Pharmaceuticals Incorporated Reconciliation of GAAP to Non-GAAP Financial Information (unaudited, in millions, except percentages) Three Months Ended December 31 , Twelve Months Ended December 31 , 2025 2024 2025 2024 GAAP cost of sales $ 466.0 $ 423.4 $ 1,651.3 $ 1,530.5 Stock-based compensation expense (3.2 ) (2.0 ) (11.1 ) (7.5 ) Intangible asset amortization expense (5.1 ) (5.1 ) (20.2 ) (20.2 ) Non-GAAP cost of sales $ 457.7 $ 416.3 $ 1,620.0 $ 1,502.8 GAAP research and development expenses $ 973.7 $ 998.7 $ 3,909.5 $ 3,630.3 Stock-based compensation expense (99.7 ) (98.3 ) (415.4 ) (425.8 ) Intangible asset amortization expense (0.7 ) (0.6 ) (2.6 ) (1.5 ) Acquisition-related costs (2) — — — (172.3 ) Non-GAAP research and development expenses $ 873.3 $ 899.8 $ 3,491.5 $ 3,030.7 Acquired in-process research and development expenses $ 56.5 $ 87.5 $ 133.0 $ 4,628.4 GAAP selling, general and administrative expenses $ 487.0 $ 377.6 $ 1,753.1 $ 1,464.3 Stock-based compensation expense (54.7 ) (67.5 ) (259.4 ) (265.2 ) Acquisition-related costs (2) — — — (36.5 ) Non-GAAP selling, general and administrative expenses $ 432.3 $ 310.1 $ 1,493.7 $ 1,162.6 Combined non-GAAP R&D, AIPR&D and SG&A expenses $ 1,362.1 $ 1,297.4 $ 5,118.2 $ 8,821.7 GAAP other income (expense), net $ 6.5 $ (14.9 ) $ (7.7 ) $ (86.1 ) (Increase) decrease in fair value of strategic investments (7.2 ) 7.2 5.0 57.7 Non-GAAP other expense, net $ (0.7 ) $ (7.7 ) $ (2.7 ) $ (28.4 ) GAAP provision for income taxes $ 139.9 $ 223.5 $ 690.0 $ 784.1 Tax adjustments (3) 61.0 56.2 301.0 340.0 Non-GAAP provision for income taxes $ 200.9 $ 279.7 $ 991.0 $ 1,124.1 GAAP effective tax rate 10.5 % 19.7 % 14.9 % 315.5 % Non-GAAP effective tax rate 13.5 % 21.3 % 17.3 % 91.0 % Vertex Pharmaceuticals Incorporated Reconciliation of GAAP to Non-GAAP Financial Information (continued) (unaudited, in millions, except per share amounts) Three Months Ended December 31 , Twelve Months Ended December 31 , 2025 2024 2025 2024 GAAP operating income (loss) $ 1,205.9 $ 1,026.0 $ 4,173.3 $ (232.9 ) Stock-based compensation expense 157.6 167.8 685.9 698.5 Intangible asset impairment charge — — 379.0 — Intangible asset amortization expense 5.8 5.7 22.8 21.7 Increase (decrease) in fair value of contingent consideration 0.9 (1.2 ) 2.1 (0.5 ) Acquisition-related costs (2) — — — 208.8 Non-GAAP operating income $ 1,370.2 $ 1,198.3 $ 5,263.1 $ 695.6 GAAP net income (loss) $ 1,191.1 $ 913.0 $ 3,953.2 $ (535.6 ) Stock-based compensation expense 157.6 167.8 685.9 698.5 Intangible asset impairment charge — — 379.0 — Intangible asset amortization expense 5.8 5.7 22.8 21.7 (Increase) decrease in fair value of strategic investments (7.2 ) 7.2 5.0 57.7 Increase (decrease) in fair value of contingent consideration 0.9 (1.2 ) 2.1 (0.5 ) Acquisition-related costs (2) — — — 208.8 Total non-GAAP adjustments to pre-tax income 157.1 179.5 1,094.8 986.2 Tax adjustments (3) (61.0 ) (56.2 ) (301.0 ) (340.0 ) Non-GAAP net income $ 1,287.2 $ 1,036.3 $ 4,747.0 $ 110.6 Net income (loss) per diluted common share: GAAP $ 4.65 $ 3.50 $ 15.32 $ (2.08 ) Non-GAAP $ 5.03 $ 3.98 $ 18.40 $ 0.42 Shares used in diluted per share calculations: GAAP and Non-GAAP 256.1 260.5 258.0 257.9 Estimated effect of potentially dilutive securities not used in GAAP diluted per share calculation (4) — — — 3.0 Non-GAAP 256.1 260.5 258.0 260.9 2: In the twelve months ended December 31, 2024 , “Acquisition-related costs” were primarily related to compensation expense associated with cash-settled unvested Alpine equity awards. 3: In the three and twelve months ended December 31, 2025 and 2024, “Tax adjustments” included the estimated income taxes related to non-GAAP adjustments to the company's pre-tax income (loss) and excess tax benefits related to stock-based compensation. “Tax adjustments” for the twelve months ended December 31, 2024 also included a discrete benefit related to prior tax years resulting from a R&D tax credit study. 4: In 2024, the company had a GAAP net loss and non-GAAP net income. Therefore, the impact of potentially dilutive securities was excluded from the calculation of GAAP weighted-average common shares outstanding (“WASO”) but was included in the calculation of non-GAAP WASO. Vertex Pharmaceuticals Incorporated Condensed Consolidated Balance Sheets (unaudited, in millions) December 31, 2025 December 31, 2024 Assets Cash, cash equivalents and marketable securities $ 6,608.1 $ 6,115.9 Accounts receivable, net 2,052.8 1,609.4 Inventories 1,686.8 1,205.4 Prepaid expenses and other current assets 853.3 665.7 Total current assets 11,201.0 9,596.4 Property and equipment, net 1,520.3 1,227.8 Goodwill and other intangible assets, net 1,512.2 1,913.9 Deferred tax assets 2,897.9 2,331.1 Operating lease assets 1,562.7 1,356.8 Long-term marketable securities 5,712.3 5,107.9 Other long-term assets 1,236.6 999.3 Total assets $ 25,643.0 $ 22,533.2 Liabilities and Shareholders' Equity Accounts payable and accrued expenses $ 3,432.9 $ 3,201.6 Other current liabilities 428.3 363.0 Total current liabilities 3,861.2 3,564.6 Long-term operating lease liabilities 1,846.5 1,544.4 Other long-term liabilities 1,269.5 1,014.6 Shareholders' equity 18,665.8 16,409.6 Total liabilities and shareholders' equity $ 25,643.0 $ 22,533.2 Common shares outstanding 254.0 256.9 About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston , with international headquarters in London . Additionally, the company has research and development sites and commercial offices in North America , Europe , Australia , Latin America , and the Middle East . Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit at www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X. 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 Vertex and members of the Vertex senior management team. Forward-looking statements are not purely historical and may be accompanied by words such as “anticipates,” “may,” “forecasts,” “expects,” “intends,” “plans,” “potentially,” “believes,” “seeks,” “estimates,” and other words and terms of similar meaning. Such statements include, without limitation, Dr. Kewalramani's statements in this press release, the information provided regarding future financial performance and operations, the section captioned “Full Year 2026 Financial Guidance” and statements regarding (i) expectations for $500 million or more in non-CF product revenue, (ii) expectations for continued growth in CF, including by increasing the number of CF patients taking its medicines through new approvals and reimbursement agreements, treatment of younger patients, increased survival, and expansion into additional geographies, (iii) beliefs regarding the clinical benefits of ALYFTREK and Vertex’s work to secure access in additional countries, (iv) beliefs regarding the anticipated benefits, eligible patient population, and access to CASGEVY, (v) expectations regarding the potential benefits and commercial launch progress for JOURNAVX, including expectations with respect to expanding access to JOURNAVX in 2026, (vi) expectations to submit for approval with global regulators for ALYFTREK in children 2 to 5 years of age in the first half of 2026, and to begin global regulatory submissions for TRIKAFTA/KAFTRIO in children 1 year to less than 2 years of age in the first half of 2026, (vii) expectations to complete enrollment and dosing in the clinical trials evaluating VX-828 in the first half of 2026, study plans with respect to VX-581, and plans to complete dosing in the MAD portion of the Phase 1/2 study of VX-522 and share data in the second half of 2026, (viii) expectations for CASGEVY, including with respect to beginning global regulatory submissions for CASGEVY to treat children ages 5 to 11 years old in the first half of 2026, potential accelerated timelines for review of the submission to the FDA, and advancing preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population, (ix) expectations to complete enrollment in both Phase 3 studies of suzetrigine in DPN by the end of 2026, advance additional preclinical assets that inhibit NaV1.7, and regarding plans for the Phase 2 study of VX-993 in DPN, (x) expectations with respect to povetacicept, including beliefs about its potential benefits and therapeutic scope, its potential to be a best-in-class approach to control B cell activity in IgAN, pMN and gMG, and its potential to be a pipeline-in-a-product, expectations regarding povetacicept in IgAN, including plans to release interim analysis data in the first half of 2026, expectations to complete the BLA filing for U.S. accelerated approval in the first half of 2026, and the anticipated expedited review of the BLA, expectations for povetacicept in pMN, including with respect to completion of the Phase 2 portion of the study and initiation of the Phase 3 portion in mid-2026, and expectations to initiate a Phase 2 study evaluating povetacicept in gMG in the first half of 2026, (xi) expectations regarding the AMPLITUDE Phase 2/3 trial of inaxaplin in AMKD, including expectations regarding the interim analysis, plans to share data in late 2026 or early 2027, and expectations to complete full enrollment in the second half of 2026, and expectations to complete the AMPLIFIED Phase 2 study of inaxaplin and share data in mid-2026, (xii) expectations regarding the clinical benefits and goals for zimislecel in T1D, including expectations regarding the temporary postponement in the Phase 1/2/3 study of zimislecel and plans for the ongoing internal manufacturing analysis, (xiii) expectations regarding the ADPKD program and the Phase 2 proof-of-concept study of VX-407, including expectation to complete enrollment in the AGLOW study by the end of 2026, (xiv) beliefs regarding the potential benefits and clinical status of VX-670 for the treatment in people with DM1 and expectations to complete enrollment and dosing mid-2026, and (xv) the company’s beliefs with respect to additional assets across its portfolio. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the company's expectations regarding its 2026 full year revenues, expenses and effective tax rates may be incorrect (including because one or more of the company's assumptions underlying its expectations may not be realized), that we may be unable to further successfully commercialize ALYFTREK as a treatment for CF, JOURNAVX as a treatment for acute pain, and CASGEVY as a treatment for SCD and TDT, that external factors may have different or more significant impacts on the company's business or operations than the company currently expects, that data from preclinical testing or clinical trials, especially if based on a limited number of patients, may not be indicative of final results or available on anticipated timelines, that patient enrollment in the company’s trials may be delayed, that the company may not realize the anticipated benefits from collaborations with third parties, that data from the company's development programs may not support registration or further development of its potential medicines in a timely manner, or at all, due to safety, efficacy or other reasons, that regulatory submissions or approvals may not occur on the anticipated timeline, or at all, that interactions with regulators may cause delays in the company’s pipeline programs, and that anticipated commercial launches may be delayed, if they occur at all. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Vertex’s business, particularly those risks listed under the heading “Risk Factors” and the other cautionary factors discussed in Vertex’s periodic reports filed with the SEC , including Vertex’s annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, all of which are filed with the Securities and Exchange Commission (SEC) and available through the company's website at www.vrtx.com and on the SEC’s website at www.sec.gov. You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. Conference Call and Webcast The company will host a conference call and webcast at 4:30 p.m. ET . To access the call, please dial (833) 630-2124 ( U.S. ) or +1(412) 317-0651 (International) and reference the “Vertex Pharmaceuticals Fourth Quarter 2025 Earnings Call.” The conference call will be webcast live and a link to the webcast can be accessed through Vertex 's website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website. (VRTX-E) View source version on businesswire.com: https://www.businesswire.com/news/home/20260212596146/en/ 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 Source: Vertex Pharmaceuticals Incorporated

Monday will feature JPM presentations from Big Pharmas, including Bristol Myers Squibb, Novartis and Pfizer. After a flurry of Big Pharma deals in the first week of the new year, the life sciences industry is heading to San Francisco for the industry's biggest investor event of the year. Monday, we'll be covering J.P. Morgan Healthcare Conference presentations from Bristol Myers Squibb, Johnson & Johnson, Novartis and many other companies. Apart from the scheduled conference presentations, our on-the-ground reporters and editors will be sharing dispatches from their JPM interactions. Be sure to check back regularly today and during the rest of the week for the latest from JPM 2026. UPDATE: 11:25 p.m. ETWhile Amgen made headlines on Monday night at the J.P. Morgan Healthcare Conference by revealing the success (PDF) of a phase 2 trial of its closely watched investigational GLP-1 treatment MariTide, the company also pointed to three growth drivers that currently are boosting its top line.The trio are osteoporosis drug Evenity, heart disease treatment Repatha and severe asthma therapy Tezspire. All are generating rapidly growing sales and are still “underpenetrated medicines,” CEO Robert Bradway said.Sales of Evenity, which are annualizing at $2 billion, were up 33% year over year in the third quarter of 2025. Repatha, which is annualizing at $3 billion, was also up 33% in the period, while AstraZeneca-partnered Tezspire, which is annualizing at $1 billion, was up 49%.“Tezspire is performing very well and has all the hallmarks of a medicine that will go on and make a big difference for many, many patients in a number of different important diseases where the unmet medical needs remain very high,” Bradway said of the treatment, which earned an FDA nod to two months ago to treat chronic rhinosinusitis with nasal polyps (CRSwNP) and is being studied to treat COPD and eosinophilic esophagitis.   UPDATE: 9:34 p.m. ETWith Takeda at a “pivotal moment” in its growth outlook, the company’s Monday presentation at the  J.P. Morgan Healthcare Conference revolved around the pipeline of eight “highly innovative” programs that will help foster growth beyond the end-of-decade loss of exclusivity coming for immunology med Entyvio, CEO Christophe Weber said.The company expects multibillion-dollar sales from each of the eight new medicines, three of which are on track to launch over the next 18 months. Those late-stage assets include narcolepsy type 1 med oveporexton, polycyemia vera prospect rusfertide and once-daily oral psoriasis candidate zasocitinib. Takeda outlined high hopes for the trio, with zasocitinib positioned to “transform and expand” the advanced oral therapy market, rusfertide said to “revolutionize” treatment outcomes and oveporexton touted as a paradigm-changing option that can “rapidly capture” patient share.“That’s our agenda for the next three years,” Weber summed up. By June, Weber is scheduled to pass the CEO title to Julie Kim, who currently serves as head of the global portfolio division.UPDATE: 9:20 p.m. ETRoche expects its HER2 breast cancer franchise to peak this year at 9 billion Swiss francs ($11.3 billion) sales, CEO of Roche Pharmaceuticals, Teresa Graham, said Monday at the J.P. Morgan Healthcare Conference. After that, the franchise will “maintain a very healthy tail through the decade,” she said.Roche has been busy converting patients who take its Herceptin and Perjeta separately to the fixed-dose combination of Phesgo.In November, the FDA approved Organon and Henlius Biotech’s Poherdy as the first biosimilar to Perjeta. When that copycat launches, which Roche has projected will happen this year, the Swiss pharma expects it will have converted 60% of patients to Phesgo, Graham said.Roche is counting on up to 19 new medicines launches by 2030. One filing for the oral SERD med giredestrant in previously treated ER-positive, HER2-negative breast cancer was already delivered to regulators at the end of 2025, and Roche is working on a submission in the adjuvant setting following the positive lidERA readout.The company is awaiting readout from a third phase 3 study of fenebrutinib in the first half of this year to file the BTK inhibitor in multiple sclerosis. In another pair of potential submissions this year, Roche has confirmed with the FDA that existing data support filings for vamikibart in uveitic macular edema and for satralizumab in thyroid eye disease, according to Graham.“Our on-market portfolio will deliver growth through 2028,” Graham said. “We do not see a patent cliff ahead for Roche.”UPDATE: 8:20 p.m. ETParticipating in the J.P. Morgan Healthcare Conference is much more an opportunity for Regeneron to schmooze investors than to scout out business development opportunities.The New York drugmaker is much more focused on internal investment than other companies in its orbit. On Monday, CEO Len Schleifer explained why Regeneron historically uses roughly 95% of its resources on internal R&D, while other companies in the industry dedicate almost half of their R&D investment to external initiatives.“We do this for two reasons. First, we have the benefit of an extraordinarily productive R&D organization,” Schleifer said. “Second, our internal analysis shows that the overall return on external business development throughout the industry has been modest, with only a small percentage of deals leading to successful approvals and even fewer still to commercial success. Large M&A deals, in particular, have often resulted in value destruction.”Regeneron revealed one bit of news, reporting fourth-quarter sales of closely watched eye disease treatment Eylea. While the company’s high-dose version of the injected drug generated sales of $506 million in the period for an 18% sequential and 66% year-over-year increase, the boost didn’t provide an increase in overall revenue from the Eylea franchise. It remained at $1.1 billion indicating that the success of Eylea HD is compensating for the decline in sales of its aging forerunner.   UPDATE: 7:20 p.m. ETWhen Madrigal Pharmaceuticals grows up, it wants to be just like Vertex.According to CEO Bill Sibold Monday at the J.P. Morgan Healthcare Conference, that’s the model Madrigal is mimicking as it attempts to not only retain its leadership in treating metabolic dysfunction-associated steatohepatitis (MASH), but double down on it. While its MASH treatment Rezdiffra is on its way to blockbuster status, the company is focused solely on maximizing its opportunity in the indication instead of looking to diversify. Madrigal’s first two business development deals—with Pfizer and China’s CSPC—are designed to find cardiometabolic agents that can be used to enhance the effectiveness of Rezdiffra.Before branching out, Vertex used the same strategy in building its dominance of the cystic fibrosis (CF) market after the 2012 approval of Kalydeco. The company, which generated $11 billion in revenue in 2024, initially focused its M&A strategy on capitalizing on its expertise in the indication.“Vertex is the unmitigated leader in CF,” Sibold said. “We see no reason why we can’t be the unmitigated leader in MASH. You’ve seen how that’s worked out for them. Our focus is MASH. Let’s win here.”Madrigal is winning with its launch of Rezdiffra, which generated sales of $287 million in the third quarter of 2025, which was up from $213 million in the previous period. The company sees a doubling of the market potential for Rezdiffra if it can expand the treatment to patients with stage 4 MASH. It is currently approved for those in stage 2 and 3. A trial in patients with stage 4 is expected to read out in 2027.“It’s very rare when you are at the start of something that is such a big opportunity,” Sibold said. “It’s even rarer that you have a great asset to start to tackle that with. I’ve been in the industry for over 30 years now and the setup for this is better than anything I’ve seen. What some companies have done is they squander an opportunity to extend leadership in a disease or an indication and try to diversify into something else prematurely. That’s not what we’re doing. Before the table is set by somebody else, we want to be the one who sets the table.”  UPDATE: 6:32 p.m. ETCommercialization—of franchises established, new and anticipated—was the dominant theme of Vertex Pharmaceuticals’ Monday presentation at the J.P. Morgan Healthcare Conference.In practice, that means continuing to invest in the company’s bread-and-butter cystic fibrosis (CF) medications, supporting gene therapy Casgevy’s “path to become a blockbuster medicine,” aiming to more than triple the prescriptions of its non-opioid pain medicine Journavx from 2025 levels and laying the groundwork for a potentially CF-rivaling renal franchise, beginning with lead nephrology asset povetacicept in IgA nephropathy (IgAN), Vertex CEO Reshma Kewalramani, M.D., said during her company’s presentation.Kewalramani gave special attention in her prepared remarks to Journavx, which secured FDA approval roughly a year ago with the promise of ushering in a new era of nonaddictive pain treatment.Through the end of 2025, more than 30,000 prescribers wrote over half a million prescriptions for Vertex’s pain med, and now the company aims to triple that number of scripts in 2026, Kewalramani said. To get there, Vertex will bulk up its force of sales reps from about 150 currently to roughly 300 “in the coming months,” the CEO explained.Meanwhile, preparing for the potential launch of Vertex’s renal franchise, Kewalramani noted that her company initiated its submission for an accelerated approval of povetacicept in IgAN in 2025’s fourth quarter. Vertex intends to complete its filing within the first half of 2026, the CEO said.The Boston-based biopharma believes povetacicept has both best-in-class and pipeline-in-a-product potential, although the company sports a number of other renal candidates in its pipeline as well.“We’re investing in renal for the long term to improve the lives of patients with kidney diseases, to go after the underlying cause of disease [and] to bring transformative medicines,” Kewalramani said. UPDATE: 5:29 p.m. ET 2026 is the year of “strategic realization” for BioMarin, following up on 2025’s “operational implementation” and the “refreshed strategy” at the company that came in 2024 with new CEO Alexander Hardy, he said at the J.P. Morgan Healthcare Conference Monday. One defining point of 2025 was the “exceptional strategic fit” of the company’s $4.8 billion purchase of Amicus Therapeutics—the largest deal in BioMarin's 28-year history. Amicus makes for a “perfect match with what BioMarin does really, really well,” Hardy said. The deal, which has yet to close, brings oral Fabry disease treatment Galafold and Pompe disease combination treatment Pombiliti-Opfolda under BioMarin’s umbrella, with each representing a potential $1 billion opportunity.The company has already mapped a route to help each franchise reach its blockbuster potential, including a focus on boosting diagnosis and treatment rates for Galafold and executing on the “tremendous opportunities” for label expansions for Pombiliti-Opfolda, Hardy noted. Meanwhile, BioMarin has now gathered the post-marketing data needed to convert the 2021 accelerated approval of its drug Voxzogo into a full one and will soon file its request with the FDA, according to the company. Voxzogo, approved in the hereditary growth condition achondroplasia, is “only at the beginning of its journey,” Hardy said. He pointed to four potential new indications indications for the therapy, including one that the company hopes to pick up in 2027 for the genetic disorder hypochondroplasia. Expected 2025 revenues for Voxzogo total some $920 million, while BioMarin’s preliminary revenue estimate for the full year sits at $3.2 billion. UPDATE: 5:05 p.m. ETSeveral years into his run as Biogen’s chief exec, Chris Viehbacher took the chance Monday to sing the praises of the “New Biogen” he’s created through a series of strategic decisions.Sales gains from Biogen’s recent launches in Friedreich’s ataxia, Alzheimer’s disease, postpartum depression and amyotrophic lateral sclerosis are offsetting declines in the company’s stalwart multiple sclerosis franchise, the CEO said during his presentation, which helps to “put a floor under the company” as it prepares for key upcoming readouts.Among Biogen’s expected readouts, the drugmaker is anticipating its Topaz-1 and Topaz-2 studies of litifilimab in systemic lupus erythematosus to generate topline data by the end of the year, according to an investor presentation (PDF) shared in conjunction with the conference.On the business development front, the CEO cited a desire to “find one more near-term growth asset,” but he added that Biogen is confident in its own R&D capabilities. Besides a potential near-term growth driver, early-stage research is one area where Biogen could stand to bulk up, Viehbacher said. UPDATE: 4:00 p.m. ETGilead’s current opportunity to “bend the arc" of the HIV epidemic is unmatched in the company’s history, CEO Daniel O’Day told an audience during the first day of the J.P. Morgan Healthcare Conference. Gilead tallied up more than $20 billion in HIV sales last year and is plotting 7 additional product launches in the space of the next 7 years, the CEO said. In addition, after the groundbreaking launch of twice-yearly pre-exposure prophylaxis therapy Yeztugo in July, the company met its guidance of at least $150 million in sales for the drug last year, O’Day said during his prepared remarks. Plus, after CVS confirmed coverage of Yeztugo as of Jan. 1, all major U.S. payers “now have a coverage plan” for the drug, he added.Gilead’s efforts aren’t solely focused on HIV, as the company sees the potential for up to 10 new product launches or label expansions this year and next. Besides its HIV portfolio, those launches could include Livdelzi in primary biliary cholangitis, Hepcludex in hepatitis D and Arcellx-partnered anito-cel in fourth-line relapsed/refractory multiple myeloma, according to Gilead’s presentation.As for business development, Gilead has been involved in some of the behind-the-scenes discussions surrounding recent deals in the industry, CFO Andy Dickinson said during the Q&A portion of Gilead’s presentation. Those talks didn’t materialize for one reason or another, but Gilead continues to explore “small to medium” deals to bolster its current portfolio, the CFO said. UPDATE: 3:35 p.m. ETWhen it comes to the politically-fueled distrust of vaccines in the United States and the narrowing of recommendations for their use in the U.S., Sanofi is playing the long game.“It’s clear this administration has a particular sensitivity around vaccination and indeed pediatric vaccination. Preferring to give parents the choice, we know that is a very delicate line to walk because not everybody’s well informed,” Sanofi CEO Paul Hudson said Monday at the J.P. Morgan Healthcare Conference.In the third quarter, while sales of Sanofi’s respiratory syncytial virus (RSV) vaccine Beyfortus swelled by 228% year over year around the rest of the world, they fell by 25% in the U.S.“I’m asked all the time, ‘What are you going to do to fix this?’ And the truth is, we just need to stay extremely objective and keep presenting the evidence,” Hudson said. “We will have to maintain a steely focus on the long-term future of vaccines and deal with any uncertainty around vaccine coverage rates in the short term based on misinformation, Facebook posts and statements from the top. We’re just gonna have to deal with it and try to provide patients and parents the very best advice that we can.”As for its business development efforts, which have been extensive in recent months, Sanofi will look to fuel its pipeline with more early-stage assets, Hudson added. “I think we’re light in phase 1,” Hudson said, adding that the company is looking to add “eight to a dozen high-quality programs” and that conversations taking place this week at the conference could address that need.In addition to its $9.1 billion buyout of Blueprint and its potential blockbuster Ayvakit in June of last year—which was the company’s largest transaction in seven years—Sanofi struck a $2.2 billion deal last month to acquire Dynavax Technologies and its shingles vaccine Z-1018, which could challenge GSK’s powerhouse Shingrix. Earlier this month, Sanofi also paid $160 million upfront as part of a collaboration with AI biotech Earendil to discover bispecific antibodies for autoimmune diseases.“We think we have enough in mid- to late-stage but I think we have to accept that to underwrite the mid/late-stage pipeline we should continue to add externally,” Hudson said. “It’s not an easy thing to do. It’s a very competitive space with many companies with huge LOEs ahead of us rushing to add their mechanisms. We’re looking for things that launch in the sort of ‘28, ’29, and ’30 horizon.” UPDATE: 1:45 p.m. ETPfizer’s priorities for 2026 include maximizing the value of its recent transactions, delivering on expected R&D milestones, investing for future growth and continuing to incorporate AI across its business, CEO Albert Bourla said during a fireside chat with JPM’s Chris Schott. The CEO is expecting 2026 to be “very rich” in catalysts for the drugmaker, with several anticipated regulatory decisions and trial readouts penciled in for the year, according to a brief investor presentation (PDF) prepared for the event.  In the closely watched obesity market, Bourla expects “very fast” growth to $150 billion in annual sales in 2030. The competitive, consumer-driven obesity market will require differentiated assets and commercial reach, which “plays to the strength of Pfizer,” the CEO said. UPDATE: 1:38 p.m. ETFollowing a turbulent 2025, Sarepta Therapeutics is doubling down on the potential of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys, which was at the center of a safety controversy and regulatory back-and-forth involving two patient deaths last year.“We thought 2025 was going to be an easy year,” Sarepta CEO Doug Ingram said at the J.P. Morgan Healthcare Conference Monday, referring to his company’s strong positioning with four commercial assets last January.“Well, it wasn’t. It was obviously not only a challenging year, but a few times there were absolutely heartbreaking moments,” he admitted. “But this team that works for me never lost sight of their mission. They never failed to execute, and as a result, thousands of boys live better lives because of them.”While much of Sarepta’s time has been occupied discussing Elevidys’ safety in recent months—and rightly so, according to Ingram— “one needs both sides of the equation,” the CEO explained, pointing to the need to reinforce the efficacy benefits of Sarepta’s gene therapy in ambulatory DMD patients, for whom the treatment is still approved. By Sarepta’s reckoning (PDF), roughly 80% of the ambulatory DMD population—defined as those who can walk independently—remains untreated.To capitalize on the “enormous opportunity” that remains in ambulatory DMD, Sarepta is upsizing its commercial field force, sharpening its healthcare professional outreach and providing deeper dives into Elevidys data, the CEO explained. Plus, Sarepta is plotting fresh initiatives among the patient community to discuss the status of Elevidys in DMD patients who cannot walk independently, Ingram said.Sarepta is awaiting a data readout on cohort 8 of its Endeavor program, expected toward the end of 2026, to shed more light on the potential path back to treating non-ambulatory patients, he added.Also on Monday, Sarepta reported its preliminary performance in the fourth quarter and for all of 2025. For the full year, Sarepta’s sales were $1.86 billion, with $898.7 million of that coming from Elevidys, according to the preliminary numbers.UPDATE: 1:35 p.m. ETApellis has reported that sales of its geographic atrophy (GA) drug Syfovre came in at $587 million for the year, which is a 4% decline from 2024. With the result, the share price of the San Francisco-based company tumbled by 14%. On the plus side, Apellis reported more patent starts than expected for kidney disease drug Empaveli, which should boost 2026 sales well beyond the analyst consensus. (Story)UPDATE: 12:45 p.m. ETAfter a year dominated by policy headlines, Johnson & Johnson and other drugmakers can now turn the page to focus on “fundamentals,” CEO Joaquin Duato told JPM’s Chris Schott during a Monday fireside chat. As far as J&J is concerned, the company is focused on 6 core areas, including oncology, immunology and neuroscience in its innovative medicines business. After J&J in 2023 revealed a plan to deliver 5% to 7% annual sales growth during the back half of this decade, the CEO is now “increasingly bullish” on this forecast. Part of his confidence stems from a portfolio of about a dozen upcoming launches, including the oral IL-23 blocker icotrokinra, which is expected to debut this year. UPDATE: 11:45 a.m. ETKicking off the official schedule at the J.P. Morgan Healthcare Conference, Bristol Myers Squibb CEO Chris Boerner, Ph.D., touted six assets with multibillion-dollar potential, including neuroscience drug Cobenfy, Johnson & Johnson-partnered oral factor Xia inhibitor milvexian, pulmonary fibrosis candidate admilparant, BioNTech-partnered PD-L1xVEGF bispecific antibody pumitamig and two oral CELMoD agents for multiple myeloma. Together, these drugs are critical to strengthening the company's long-term trajectory, Boerner said.Several of these drugs are expected to deliver pivotal data this year. These include Cobenfy from three closely-watched Adept trials in Alzheimer’s disease psychosis, milvexian in atrial fibrillation and secondary stroke prevention, admilparant in idiopathic pulmonary fibrosis, and the two CELMoD drugs—iberdomide and mezigdomide—in previously treated myeloma.As for pumitamig, BMS is hoping that the bispecific can redefine the standard of care in multiple tumor types, Boerner said.“Our strategy with this asset is to be either first or second in each of the indications that we’re pursuing,” Boerner said. “By the end of this year, we expect to add three new registrational trials, including a broader reach across two non-small cell lung indications and a study in head and neck cancer.”The company and its partner BioNTech are also moving forward with trials in earlier-stage disease and novel combinations, including with antibody-drug conjugates and other cancer immunotherapies such as BioNTech’s mRNA vaccine and targeted therapies, he said.More broadly, BMS hopes to launch more than 10 new medicines by 2030, the CEO said.“Taken together, what emerges is a younger and more diversified portfolio that will provide an impressive foundation for sustained growth leading into 2030 and beyond,” Boerner said.UPDATE: 10:20 a.m. ETBudget-minded Moderna, busy with cost cuts over the last year, had some positive news to share on the revenue side of its business on Monday. Moderna's preliminary, unaudited sales for 2025, totaled $1.9 billion, or $100 million more than the midpoint of the company's most recent revenue guidance for the year. The vaccine specialist has been reeling from declining COVID-19 vaccine sales for years, and in 2025 it cut about $2 billion in annual operating costs. For 2026, Moderna expects sales growth reach up to 10% as it works to build a "large seasonal vaccine franchise" and advance its pipeline, CEO Stéphane Bancel said in a corporate update Monday. Moderna's presentation at JPM will take place later today. (Release)UPDATE: 8:50 a.m. ETWhile the J.P. Morgan Healthcare Conference won't officially start for a few more hours in San Francisco, some industry players are already sharing corporate updates tied to the event. Insmed kicked things off Friday by revealing $144.6 million in fourth-quarter Brinsupri sales, a result analysts at William Blair described as a "blowout sales performance." Various Wall Street analysts heaped praise on the ongoing launch, with the latest number reinforcing the multiblockbuster opportunity in front of the drugmaker, the analysts said. (Story)Alnylam, also underway with a key launch, Monday said it tallied nearly $3 billion in full-year 2025 sales. The company's transthyretin drug sales accounted for the vast majority of the sum, with Amvuttra sales last year reaching more than $2.3 billion. The numbers are preliminary and unaudited; Alnylam plans to share final results in February. Alnylam's Amvuttra won a key approval last year to enter the competitive transthyretin-mediated amyloidosis market, where it's clashing against rivals from Pfizer and BridgeBio. Also Monday, Alnylam touted a new plan called "Alnylam 2030," which is "focused on continuing to successfully scale the company’s operations," the drugmaker said in its corporate update ahead of its JPM presentation. As for 2026, Alnylam's guidance calls for more than $5 billion in sales. (Release)