Vertex Reports Second Quarter 2025 Financial Results

— Total revenue of $2.96 billion, a 12% increase compared to Q2 2024; reiterated full year 2025 financial guidance, including revenue guidance of $11.85 to $12 billion —

— Continued strong execution of CASGEVY®, ALYFTREK® and JOURNAVX® launches —

— Rapid advancement of next wave of clinical programs through pivotal development, with suzetrigine in DPN, zimislecel in T1D, povetacicept in IgAN and pMN, and inaxaplin in AMKD —

— David Altshuler, M.D., PhD., Chief Scientific Officer (CSO), announces intent to retire August 1, 2026; as part of planned transition, Mark Bunnage, D.Phil., SVP of Global Research, to assume role of CSO effective February 1, 2026 —

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

“Vertex delivered a strong quarter of revenue growth with each of our three product launches — ALYFTREK, JOURNAVX, and CASGEVY — contributing, as well as continued advancement of our clinical programs,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “As we enter the second half of the year, we are focused on expanding leadership in cystic fibrosis, executing the launches, advancing the pipeline, and preparing for submissions and commercialization in additional disease areas.” Dr. Kewalramani added, “I would like to express my deep gratitude to David for his exceptional scientific vision and patient impact over the last 13 years. As CSO, David has been at the helm through the discovery, development and approval of four CF medicines, our groundbreaking CRISPR/Cas9 gene-edited therapy, and our novel non-opioid pain medicine. As part of the planned transition, I am delighted that Mark Bunnage, current SVP and Head of Global Research, will be the next CSO. He is a world-class scientist with long tenure at the company who has contributed enormously to our R&D success, working alongside David since 2016 and heading up discovery research since March 2024. Mark is the ideal leader to drive the next wave of innovation.”

Second Quarter 2025 Results

Total revenue increased 12% to $2.96 billion compared to the second 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 14% to $1.85 billion due to continued strong patient demand and favorable gross-to-net versus prior year. Outside the U.S., total revenue increased 8% to $1.12 billion due to strong performance across multiple geographies.

Combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses were $1.4 billion and $1.2 billion, respectively, compared to $5.8 billion and $5.4 billion, respectively, in the second quarter of 2024. The decreases were primarily due to $4.4 billion of Acquired IPR&D (AIPR&D) expenses associated with Vertex’s acquisition of Alpine Immune Sciences incurred in the second quarter of 2024, partially offset by continued 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 19.5% and 19.4%, respectively, compared to (6.0)% and (10.0)%, respectively, for the second quarter of 2024. In the second quarter of 2024, Vertex reported a pre-tax loss, due to the impact of non-deductible AIPR&D expenses related to the Alpine acquisition.

GAAP and non-GAAP net income were $1.0 billion and $1.2 billion, respectively, compared to net losses of $3.6 billion and $3.3 billion, respectively, for the second quarter of 2024, given the impact of the Alpine AIPR&D expense in the second quarter of 2024.

Cash, cash equivalents, and total marketable securities as of June 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 and income tax payments.

Full Year 2025 Financial Guidance

Vertex today reiterated its full year 2025 financial guidance, including revenue guidance of $11.85 billion to $12 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 also reiterated its guidance for both combined GAAP and non-GAAP R&D, AIPR&D, and SG&A expenses, which includes expectations for continued investment in multiple mid- and late-stage clinical development programs and commercial capabilities, and AIPR&D expenses of approximately $100 million. In addition, Vertex reiterated non-GAAP effective tax rate guidance of 20.5-21.5%. Vertex does not currently expect a material change to its 2025 non-GAAP effective tax rate, following the enactment of the recent tax legislation under H.R.1. 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.), Europe and Canada 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 and anticipated progress includes:

• Vertex secured European Commission approval of ALYFTREK, the once-daily, next-in-class combination CFTR modulator, for the treatment of people with CF ages 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. Eligible patients in Germany and Denmark already have access to ALYFTREK, and Vertex expects that eligible patients in Ireland will have access in the coming weeks. Vertex continues to work with reimbursement bodies across additional EU member states to ensure access for all eligible patients as quickly as possible. In addition, Vertex entered into a reimbursement agreement with the National Health Service (NHS) England for eligible CF patients to access ALYFTREK.
• In July, Vertex received approval from Health Canada 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. Vertex is working to secure reimbursement for eligible patients in Canada.
• Regulatory reviews are underway for ALYFTREK in Switzerland, Australia, and New Zealand.

CASGEVY for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved in the 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:

• Through reimbursement agreements, Vertex has secured access for eligible SCD and TDT patients in 10 countries. Countries with recent reimbursement agreements include Northern Ireland, Scotland and Denmark. Vertex continues to work with government and reimbursement authorities globally to secure access for eligible patients.
• Vertex has met its goal of activating more than 75 authorized treatment centers (ATCs) globally. Globally, since launch through June 30th, 2025, approximately 115 patients have had their first cell collection, and 29 patients have received infusions of CASGEVY, including 16 infused in the second 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-July more than 110,000 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-July, across commercial and government payers, nearly 150 million individuals already have covered access to JOURNAVX, representing almost half of U.S. covered lives. This includes formal coverage agreements with two of the three large national pharmacy benefit managers (PBMs) and unrestricted access within 16 state Medicaid plans. Vertex expects access to JOURNAVX to continue to expand over the course of 2025.
• More than 50 of Vertex’s targeted 150 large healthcare systems and more than 500 individual hospitals of the 2,000 targeted institutions have added JOURNAVX to formularies, protocols or order sets. Vertex now has national group purchasing agreements in place with two of the largest group purchasing organizations in the U.S.

Select Clinical-Stage R&D Pipeline

Cystic Fibrosis

• Vertex is completing Phase 3 studies in younger age groups to expand the TRIKAFTA/KAFTRIO and ALYFTREK labels and enable earlier treatment of children with CF. Vertex recently completed enrollment in a global study of ALYFTREK in children 2 to 5 years of age.
• Vertex is on track to complete the healthy volunteer study of VX-828, the once-daily, next-generation 3.0 corrector, and advance the VX-828 combination into a CF cohort this year.
• The Independent Data Monitoring Committee (IDMC) has completed its review of VX-522, a nebulized CFTR mRNA therapy, and endorsed restart of the Phase 1/2 study. Vertex expects to resume dosing in the multiple ascending dose (MAD) portion of the study in the near term.

Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

• Vertex has completed enrollment of children 5 to 11 years of age with SCD or TDT in two global Phase 3 studies of CASGEVY and is on track to complete dosing in the second half of 2025.
• Vertex continues to advance preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population.

Acute Pain

• Vertex announced results from its Phase 2 placebo-controlled dose-ranging study evaluating the safety and efficacy of VX-993, an investigational selective NaV1.8 pain signal inhibitor, in treating acute pain after bunionectomy surgery. Treatment with VX-993 did not result in a statistically significant improvement on the primary endpoint of the time-weighted sum of the pain intensity difference from 0 to 48 hours (SPID48) compared to placebo. VX-993 was generally safe and well-tolerated. Based on these results, Vertex will not further advance VX-993 as monotherapy in acute pain.

Peripheral Neuropathic Pain (PNP)

• Vertex continues to enroll and dose patients with diabetic peripheral neuropathy (DPN) in a Phase 3 pivotal trial of suzetrigine. The U.S. Food and Drug Administration (FDA) has granted suzetrigine Fast Track designation in PNP and Breakthrough Therapy designation in DPN.
• As part of an End of Phase 2 discussion with the FDA, the Agency indicated they do not see a path to a broad PNP label at this time. As such, Vertex will not initiate a Phase 3 lumbosacral radiculopathy (LSR) study but instead will prioritize DPN as the first PNP indication and begin a second DPN Phase 3 study in the near term. Vertex expects to complete enrollment in both DPN Phase 3 studies by the end of next year. Vertex will continue to work with the FDA to expand the DPN indication over time to include additional neuropathic pain conditions and assess potential pathways to secure a broad PNP label.
• Vertex continues to enroll and dose patients in a Phase 2 study for the oral formulation of VX-993 for the treatment of DPN.

Type 1 Diabetes (T1D) 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 is on track to complete enrollment and dosing in the Phase 3 portion of the Phase 1/2/3 global study of zimislecel in patients with T1D with severe hypoglycemic events and impaired awareness of hypoglycemia in the near term. Vertex expects global regulatory submissions for zimislecel in 2026.
• Zimislecel has been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration, Priority Medicines (PRIME) designation from the EMA, and has secured an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency (MHRA).
• Vertex announced positive results from the Phase 1/2 portion of the Phase 1/2/3 clinical trial of zimislecel in an oral presentation at the American Diabetes Association 85th Scientific Sessions and simultaneously published these data in the New England Journal of Medicine. All 12 participants who received the full dose in a single infusion and were followed for at least one year:
  • Demonstrated engraftment with glucose-responsive endogenous C-peptide production, which was durable through the period of follow-up;
  • Achieved the ADA/EASD-recommended targets of HbA1c 70% and were free of SHEs from day 90 onwards;
  • Had a reduction in exogenous insulin use (mean reduction in daily insulin dose: 92%), and 10/12 (83%) no longer required exogenous insulin at Month 12; and
  • Achieved the Phase 1/2 primary endpoint of elimination of SHEs with HbA1c