Vertex Reports Third Quarter 2025 Financial Results

— Total revenue of $3.08 billion, an 11% increase compared to Q3 2024 —

— Refined full year financial guidance: total revenue guidance now $11.9 to $12.0 billion and total combined non-GAAP R&D, AIPR&D, and SG&A expense guidance now $5.0 to $5.1 billion —

— R&D pipeline continues to make progress: five programs in pivotal development and povetacicept Phase 3 IgAN trial full enrollment complete; on track to submit first module of povetacicept IgAN BLA to FDA by end of 2025 —

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the third quarter ended September 30, 2025, and refined full year 2025 financial guidance.

“Vertex delivered strong results across the board in the third quarter, extending our leadership in CF, continuing to build global momentum for CASGEVY, and advancing the launch of JOURNAVX in acute pain,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “We also delivered strong progress across the R&D pipeline, with completion of enrollment in the Phase 3 study of povetacicept in IgAN, initiation of the Phase 2/3 study of povetacicept in primary membranous nephropathy, as well as advancement of several programs in research and earlier-stage clinical development. For the remainder of 2025, we are focused on executing the ongoing launches, initiating the povetacicept BLA submission in IgAN for potential U.S. accelerated approval, advancing the pipeline, and preparing for new launches in additional disease areas.”

Third Quarter 2025 Results

Total revenue increased 11% to $3.08 billion compared to the third quarter of 2024, primarily driven by the continued performance of cystic fibrosis (CF) therapies and early contributions from the three ongoing launches. In the U.S., total revenue increased 15% to $1.98 billion due to continued strong CF patient demand, including for ALYFTREK; contributions from CASGEVY and JOURNAVX; and favorable net pricing in CF versus the prior year. Outside the U.S., total revenue increased 4% to $1.10 billion due to solid performance across multiple geographies.

Combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses were $1.5 billion and $1.3 billion, respectively, compared to $1.3 billion and $1.1 billion, respectively, in the third quarter of 2024. The increases were primarily due to increased R&D investment in support of multiple mid- and late-stage clinical development programs, increased commercial investment to support the launch of JOURNAVX in acute pain, and higher AIPR&D.

GAAP and non-GAAP effective tax rates were 16.6% and 17.6%, respectively, compared to 14.6% and 19.8%, respectively, for the third quarter of 2024. In each of the third quarters of 2025 and 2024, the tax rate benefited from increased R&D tax credits.

GAAP and non-GAAP net income were $1.1 billion and $1.2 billion, respectively, compared to $1.0 billion and $1.1 billion, respectively, for the third quarter of 2024, primarily driven by increased product revenue partially offset by increased operating expenses.

Cash, cash equivalents, and total marketable securities as of September 30, 2025, were $12.0 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 2025 Financial Guidance

Vertex today refined its full year 2025 revenue guidance with total revenue now expected to be $11.9 to $12.0 billion, which assumes continued growth in CF, including the global launch of ALYFTREK; continued uptake of CASGEVY in multiple regions; and early contributions from the U.S. launch of JOURNAVX. Vertex is also revising full year 2025 guidance for combined GAAP and non-GAAP operating expenses. Combined GAAP and non-GAAP R&D, AIPR&D, and SG&A expenses are now expected to be approximately $5.65 to $5.8 billion and $5.0 to $5.1 billion, respectively. This revised outlook accounts for acceleration of povetacicept programs and increased investment in sales and marketing initiatives for JOURNAVX in acute pain. In addition, Vertex lowered its prior non-GAAP effective tax rate guidance of 20.5% to 21.5% to a new range of 17% to 18% to incorporate several one-time tax benefits. These include one-time tax benefits recognized in the third quarter of 2025 from Alpine-related R&D tax credits, as well as anticipated recognition in the fourth quarter of 2025 of previously deferred tax benefits. This financial guidance also includes an immaterial cost impact from tariffs in 2025 based on currently known tariff rates and regulations.

Vertex’s financial guidance is summarized below:

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, and Switzerland 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. launch of ALYFTREK is progressing well across all eligible patient groups, and launches outside the U.S. are showing a strong start in multiple markets.
• Vertex received approval from the European Commission and Health Canada in July and from Medsafe New Zealand and Swissmedic in October for ALYFTREK for the treatment of people with CF ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Regulatory review is underway for ALYFTREK in Australia.
• Eligible patients in England, Ireland, Germany, Denmark and Northern Ireland currently have reimbursed access to ALYFTREK, and Vertex is working to secure access for eligible patients in additional countries.
• In October at the North American Cystic Fibrosis Conference (NACFC), Vertex presented multiple abstracts on ALYFTREK as well as the association between lower sweat chloride with CFTR modulator use and improved clinical outcomes. ALYFTREK has shown greater reductions in sweat chloride compared to TRIKAFTA in Phase 3 trials, suggesting that ALYFTREK has the potential for even greater improvements in quality of life and other health-related outcomes.

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., Great Britain, the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain (Bahrain), Qatar, Canada, Switzerland, and the United Arab Emirates (UAE) for the treatment of both SCD and 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:

• In September, Vertex announced a reimbursement agreement for patients with SCD and TDT in Italy. Italy has the largest population of people living with TDT in Europe, with approximately 5,000 people 12 years and older with TDT and around 2,300 with SCD.
• Globally, since launch through September 30th, 2025, approximately 165 people with SCD or TDT have had their first cell collection for CASGEVY, including 50 people in the third quarter of 2025; and 39 people with SCD or TDT have received infusions of CASGEVY, including 10 people infused in the third quarter of 2025.

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 mid-October more than 300,000 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.
• As of mid-October, across commercial and government payers, more than 170 million individuals already have covered access to JOURNAVX, representing more than half of U.S. covered lives. This includes formal coverage with two of the three large national pharmacy benefit managers (PBMs) and unrestricted access within 19 state Medicaid plans. Vertex expects access to JOURNAVX to continue to expand over the remainder of 2025 and into 2026.
• Approximately 90 of Vertex’s targeted 150 large healthcare systems and more than 750 individual hospitals of the 2,000 targeted institutions have added JOURNAVX to formularies, protocols or order sets.

Select Clinical-Stage Pipeline Programs

Cystic Fibrosis

• In October, Vertex completed the pivotal study of TRIKAFTA in children 12 months to