Vertex Pharmaceuticals Reports Robust Third Quarter 2025 Results and Refines Full-Year Outlook as Pipeline Momentum Accelerates
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX), a global leader in genetic and transformative medicine, today announced financial results for the third quarter ended September 30, 2025. The company delivered strong operational and pipeline performance, reinforcing its leadership in cystic fibrosis (CF), expanding the global reach of its groundbreaking gene therapy CASGEVY, and accelerating the commercial rollout of JOURNAVX, its novel non-opioid acute pain therapy.
Vertex also refined its full-year 2025 guidance to reflect sustained revenue growth, robust global launches, and greater investment in advancing pivotal programs, including povetacicept in kidney disease.
Strong Performance Across All Business Segments
“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 Pharmaceuticals. “We also made meaningful progress across our R&D pipeline, completing enrollment in the Phase 3 study of povetacicept in IgA nephropathy, initiating a pivotal trial in primary membranous nephropathy, and advancing several earlier-stage programs. For the remainder of 2025, our focus is on flawless execution — completing the povetacicept BLA submission, accelerating pipeline development, and preparing for new launches across additional disease areas.”
Third Quarter 2025 Financial Highlights
Revenue Growth Driven by Multiple Launches
Total revenue for the third quarter reached $3.08 billion, representing an 11% increase compared to $2.77 billion in Q3 2024. Growth was primarily fueled by continued expansion of Vertex’s CF franchise and initial commercial contributions from its three ongoing launches — ALYFTREK, CASGEVY, and JOURNAVX.
- U.S. revenue rose 15% to $1.98 billion, driven by robust patient uptake of CF therapies, the successful launch of ALYFTREK, and contributions from CASGEVY and JOURNAVX.
- International revenue grew 4% to $1.10 billion, supported by strong uptake across key geographies and recently approved indications.
Operating Results and Expenses
Combined GAAP and non-GAAP R&D, Acquired IPR&D (AIPR&D), and SG&A expenses were $1.5 billion and $1.3 billion, respectively, up from $1.3 billion and $1.1 billion in the prior-year quarter. The increases primarily reflect higher R&D spending to advance multiple mid- and late-stage clinical programs, commercial investments related to the JOURNAVX launch, and increased AIPR&D costs.
Vertex reported GAAP net income of $1.1 billion and non-GAAP net income of $1.2 billion, up from $1.0 billion and $1.1 billion, respectively, in Q3 2024. Net income growth was driven by higher product sales, partially offset by increased operating expenditures.
Effective tax rates were 16.6% (GAAP) and 17.6% (non-GAAP), compared to 14.6% and 19.8% last year, with both periods benefiting from enhanced R&D tax credits.
As of September 30, 2025, Vertex held $12.0 billion in cash, cash equivalents, and marketable securities, up from $11.2 billion at year-end 2024. The increase reflects strong operating cash flow, partially offset by ongoing share repurchases.
Refined 2025 Financial Guidance
Vertex refined its full-year 2025 outlook, highlighting its confidence in continued revenue expansion and disciplined investment strategy.
- Total revenue is now expected to range between $11.9 billion and $12.0 billion, up slightly from the previous forecast of $11.85–$12.0 billion.
- Combined GAAP operating expenses are projected at $5.65–$5.8 billion, and non-GAAP operating expenses at $5.0–$5.1 billion, reflecting higher R&D and commercialization spending tied to JOURNAVX and povetacicept advancement.
- The non-GAAP effective tax rate is now expected to be 17–18%, reduced from the prior 20.5–21.5% range, incorporating one-time R&D tax benefits from the Alpine transaction and deferred tax recognitions anticipated in Q4.
Vertex’s updated guidance also accounts for minor tariff-related cost impacts expected in 2025 based on current trade conditions.
Business Highlights and Portfolio Updates
Cystic Fibrosis (CF) Franchise: Sustaining Global Leadership
For more than two decades, Vertex has led innovation in CF, addressing the root cause of disease for nearly 95% of all CF patients in core markets. Its suite of CFTR modulators — including TRIKAFTA, KAFTRIO, and now ALYFTREK — continues to expand across age groups and geographies.
Key Updates:
- The U.S. launch of ALYFTREK is progressing strongly across eligible age cohorts.
- Regulatory approvals were secured in 2025 from the European Commission, Health Canada, Medsafe New Zealand, and Swissmedic for CF patients aged 6 years and older with at least one F508del mutation or another responsive mutation in the CFTR gene.
- Regulatory review in Australia is underway, while England, Ireland, Germany, Denmark, and Northern Ireland have secured reimbursement access for eligible patients.
- At the North American Cystic Fibrosis Conference (NACFC) in October, Vertex presented new ALYFTREK data demonstrating superior reductions in sweat chloride compared to TRIKAFTA — correlating with improved clinical outcomes and enhanced quality of life.
CASGEVY: Expanding Access to Transformative Gene Therapy
CASGEVY, a first-in-class ex vivo CRISPR/Cas9 gene-edited cell therapy, continues to redefine the standard of care for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Approved across major global markets, including the U.S., U.K., EU, Canada, Saudi Arabia, Bahrain, Qatar, and UAE, the therapy is accessible to over 60,000 eligible patients worldwide.
Recent Milestones:
- In September, Vertex finalized a reimbursement agreement in Italy, home to Europe’s largest TDT population.
- As of September 30, 2025, approximately 165 patients have undergone their first cell collection for CASGEVY, with 39 having received infusions — reflecting steady ramp-up since launch.
- Vertex remains committed to expanding patient access through strategic partnerships, manufacturing scale-up, and payer collaborations worldwide.
JOURNAVX (Suzetrigine): Redefining Acute Pain Treatmen
JOURNAVX, an oral, non-opioid NaV1.8 pain signal inhibitor, represents a paradigm shift in acute pain management. Approved in the U.S. for moderate-to-severe acute pain, JOURNAVX continues its strong commercial rollout.
Since its launch in March 2025, over 300,000 prescriptions have been filled across hospital and retail channels, spanning a range of acute pain indications. By mid-October:
- More than 170 million covered lives had access to JOURNAVX across commercial and government payers, including major PBMs and 19 state Medicaid plans.
- Approximately 90 of 150 targeted healthcare systems and 750 hospitals have added JOURNAVX to their formularies and clinical protocols.
Vertex anticipates continued expansion of access and utilization through 2026 as healthcare systems integrate JOURNAVX into pain management protocols.
Pipeline Progress: Advancing Multiple Late-Stage Programs
Vertex continues to invest deeply in R&D, advancing an expansive portfolio of transformative therapies targeting rare and serious diseases.
Cystic Fibrosis: Expanding Treatment to Younger Populations
- Completed pivotal study of TRIKAFTA in children aged 12 months to under 24 months, demonstrating strong efficacy and safety, including a 71.8 mmol/L mean reduction in sweat chloride. Global regulatory submissions are expected in H1 2026.
- Completed enrollment for ALYFTREK in children aged 2–5 years, with results expected in early 2026.
- Advanced VX-828, a once-daily next-generation CFTR corrector, into patient cohorts.
- Resumed dosing in the VX-522 nebulized mRNA therapy study for patients ineligible for CFTR modulators.
Sickle Cell Disease and TDT: Pediatric Trials Progressing
Vertex completed enrollment of children aged 5–11 years in two global Phase 3 CASGEVY trials for SCD and TDT. Full dosing is expected by year-end 2025, with emerging data to be presented at the American Society of Hematology (ASH) meeting on December 6, 2025.
Pain Portfolio: Expanding Beyond Acute Pain
Vertex initiated its first Phase 3 trial of suzetrigine in diabetic peripheral neuropathy (DPN), a major form of peripheral neuropathic pain (PNP), and expects to launch a second pivotal trial by November 2025. Enrollment for both studies should complete by 2026.
Additionally, Vertex is advancing VX-993, an oral therapy for painful DPN, currently in Phase 2 development.
Type 1 Diabetes: Toward a Functional Cure
Vertex’s zimislecel, a stem cell-derived, fully differentiated islet cell therapy, aims to provide a potential one-time functional cure for Type 1 diabetes (T1D).
The company completed enrollment in the Phase 1/2/3 study but temporarily paused dosing pending manufacturing analysis. Zimislecel holds RMAT, Fast Track, PRIME, and ILAP designations across major regulatory agencies, underscoring its potential global impact.
Vertex is also developing next-generation immunoprotection and gene-editing approaches to minimize or eliminate immunosuppressive requirements.
Kidney Diseases: Expanding Renal Leadership
Vertex’s povetacicept continues to demonstrate significant promise as a dual BAFF/APRIL inhibitor for multiple B cell-mediated diseases, including IgA nephropathy (IgAN) and primary membranous nephropathy (pMN).
- The FDA granted Breakthrough Therapy Designation and a rolling review for the BLA submission in IgAN.
- Vertex completed full enrollment in the Phase 3 IgAN study and plans to submit the first BLA module by year-end 2025.
- The Phase 2/3 pivotal pMN study has commenced, supported by Fast Track designation.
- Updated Phase 1/2 data will be presented at the American Society of Nephrology (ASN) Kidney Week on November 8, 2025, followed by a live investor event.
Vertex also made progress in APOL1-mediated kidney disease (AMKD) with its small molecule inhibitor inaxaplin:
- Enrollment completed for the AMPLITUDE Phase 2/3 interim cohort, positioning the program for potential accelerated approval.
- Enrollment in the AMPLIFIED Phase 2 trial remains on track for completion by year-end.
For autosomal dominant polycystic kidney disease (ADPKD), Vertex initiated AGLOW, a Phase 2 proof-of-concept trial evaluating VX-407 in 24 patients with specific PKD1 gene variants. Results will assess changes in height-adjusted total kidney volume (htTKV) — a key disease progression biomarker.
Myotonic Dystrophy Type 1 (DM1): Precision RNA Therapeutics
Vertex continues to dose participants in its Phase 1/2 study of VX-670, an innovative oligonucleotide–cyclic peptide conjugate designed for enhanced cellular delivery. Completion of enrollment is anticipated in H1 2026.
Innovation Pipeline: Sustained Investment in Next-Generation Scienc
Consistent with its “portfolio-in-a-platform” R&D model, Vertex maintains multiple programs across discovery and early clinical development in CF, pain, diabetes, genetic kidney disease, and rare disorders. Emerging preclinical initiatives include:
- Novel NaV1.7 inhibitors for pain.
- Improved immunosuppressive regimens to enhance zimislecel outcomes.
- Gentler conditioning regimens for CASGEVY to improve patient tolerability.
Strategic Execution and Long-Term Growth
As Vertex advances toward the close of 2025, the company remains strategically positioned to sustain double-digit revenue growth, supported by the strength of its CF franchise, diversification through new therapeutic launches, and continued investment in its high-value pipeline.
Dr. Kewalramani concluded, “Vertex’s third quarter results reflect the continued success of our innovation-driven business model. We are executing with precision — delivering breakthrough medicines to more patients worldwide while advancing the next wave of transformative therapies across multiple disease areas.”
About Vertex Pharmaceuticals
Vertex Pharmaceuticals (Nasdaq: VRTX) is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. Headquartered in Boston, with global operations across North America, Europe, and Asia, Vertex continues to expand its leadership in genetic therapies, cell and molecular medicine, and precision drug discovery. For more information, visit www.vrtx.com.
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