Vertex Reports Third Quarter 2026 Financial Results

— Achieved total revenue of $3.44 billion, a 12% increase compared to third quarter 2025 —

— Raising full-year revenue guidance to $13.15 billion to $13.25 billion —

— Established fourth commercial pillar in rare endocrine diseases with closing of Crinetics Pharmaceuticals acquisition and preparing for fifth in renal medicine with povetacicept PDUFA date on November 30^th —

— R&D pipeline continues to advance with multiple additional near-term milestones —

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the third quarter ended September 30, 2026, and raised its full year 2026 revenue guidance.

“Vertex delivered excellent third quarter results marked by strong revenue growth across the increasingly diversified portfolio, continued advancement of the R&D pipeline, and the successful close of the Crinetics Pharmaceuticals acquisition,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “With the potential povetacicept approval in IgAN coming later this month, we are well positioned to establish nephrology as our fifth commercial franchise and expand the reach of our transformative medicines to even more patients.”

Third Quarter 2026 Results

Total revenue increased 12% to $3.44 billion compared to the third quarter of 2025, primarily driven by the continued performance of cystic fibrosis (CF) therapies and growth from diversification into additional disease areas. In the U.S., total revenue increased 10% to $2.17 billion from continued strong CF patient demand, including from new initiations of ALYFTREK; higher realized net prices in CF versus the prior year; and contributions from CASGEVY, JOURNAVX, and PALSONIFY. Outside the U.S., total revenue increased 16% to $1.27 billion from strong CF performance across multiple geographies, including ALYFTREK uptake; increased CASGEVY infusions; and a favorable impact from foreign exchange.

GAAP and non-GAAP combined R&D, Acquired IPR&D, and SG&A expenses were $2.2 billion compared to $1.5 billion for the third quarter of 2025 and $1.5 billion compared to $1.3 billion for the third quarter of 2025, respectively. The increases were primarily due to commercial investment to support build-out of the renal franchise, led by povetacicept in IgAN; the addition of Crinetics Pharmaceuticals; and investment to support the ongoing launch of JOURNAVX in acute pain. Combined GAAP R&D, acquired IPR&D, and SG&A expenses also reflected approximately $500 million of compensation and transaction-related expenses associated with the Crinetics Pharmaceuticals acquisition.

GAAP and non-GAAP effective tax rates were 16.6% and 18.0%, respectively, compared to 16.6% and 17.6%, respectively, for the third quarter of 2025.

GAAP net income was $654 million compared to $1.1 billion for the third quarter of 2025. The year-over-year decline is primarily due to expenses related to the Crinetics Pharmaceuticals acquisition.

Non-GAAP net income was $1.2 billion in each of the third quarters of 2026 and 2025, as increased product revenue was offset by increased operating expenses to fund pipeline and commercial opportunities, as well as higher interest expense and lower interest income as a result of the Crinetics Pharmaceuticals acquisition.

Cash, cash equivalents, and total marketable securities as of September 30, 2026, were $9.8 billion, compared to $12.3 billion as of December 31, 2025, primarily due to $9.0 billion net cash used to acquire Crinetics Pharmaceuticals and to repurchase Vertex common stock pursuant to its share repurchase program, partially offset by $4.5 billion borrowed pursuant to a term loan executed in the third quarter of 2026 and cash flows provided by Vertex’s operating activities.

Full Year 2026 Financial Guidance

Vertex today raised its full year 2026 total revenue guidance to $13.15 billion to $13.25 billion from $13.1 billion to $13.2 billion previously. Embedded in this guidance are expectations for continued growth in CF, $500 million or more in revenue from CASGEVY and JOURNAVX combined, and $40 million to $50 million in revenue from PALSONIFY following the closing of the Crinetics Pharmaceuticals transaction on September 1. Total revenue guidance continues to reflect a year-over-year benefit of approximately 150 basis points from changes in foreign exchange rates, net of Vertex’s foreign exchange hedging program. Vertex is also increasing full year 2026 combined GAAP and non-GAAP R&D, AIPR&D, and SG&A expense guidance to reflect expenses primarily related to the operations and integration of Crinetics Pharmaceuticals for the remainder of 2026. Vertex GAAP guidance also includes approximately $500 million of compensation and transaction expenses noted above.

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 approximately 95 percent of all people living with CF in core markets, including patients as young as one month old. Vertex’s CF medicines are accessible in more than 65 countries across six continents. Recent highlights include:

• In the third quarter of 2026, Vertex secured reimbursement for ALYFTREK in 7 additional countries, bringing the total number of countries where ALYFTREK is reimbursed to 32.
• Vertex has initiated global regulatory submissions for ALYFTREK for the treatment of children with CF ages 2 to 5 years.

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 patients 12 years and older in 36 countries across North America, Europe, and the Middle East; CASGEVY is also approved for the treatment of patients 2 years and older in the U.S., Bahrain, and the United Arab Emirates, and patients 5 years and older in the U.K. Recent highlights include:

• Vertex recorded third quarter 2026 CASGEVY revenue of $99 million, representing 30% quarter-over-quarter growth, and nearly six times the $17 million of CASGEVY sales reported in the third quarter of 2025.
• In July, the U.S. FDA approved CASGEVY for the treatment of children 2 years of age and older with SCD and TDT, making it the first genetic therapy for SCD and TDT indicated for children as young as 2 years old. Since approval, multiple children have initiated treatment. In October, the United Arab Emirates and Bahrain also approved CASGEVY for the treatment of children 2 years of age and older, and the United Kingdom approved CASGEVY for the treatment of children 5 years of age and older. Vertex has also completed regulatory submissions in the Kingdom of Saudi Arabia for the treatment of children with SCD and TDT.

JOURNAVX (suzetrigine) for the treatment of moderate-to-severe acute pain

JOURNAVX is a first-in-class, oral, selective, non-opioid Na_V1.8 pain signal inhibitor, approved in the U.S. for the treatment of adults with moderate-to-severe acute pain. Recent highlights include:

• In the third quarter, more than 750,000 prescriptions were filled for JOURNAVX across the hospital and retail settings, representing more than 40% quarter-over-quarter growth and more than four times the 170,000 JOURNAVX prescriptions that were filled in the third quarter of 2025.
• Vertex recorded third quarter 2026 JOURNAVX revenue of $58 million, representing 18% quarter-over-quarter growth, and nearly tripling the $20 million in revenue reported in the third quarter of 2025.
• As of August 2026, JOURNAVX has become the most prescribed branded oral pain medicine in the U.S.

PALSONIFY (paltusotine) for the treatment of acromegaly

PALSONIFY is a first-in-class, small molecule, once-daily oral somatostatin receptor type 2 (SST2) agonist, approved in the U.S. for the treatment of adults with acromegaly who had an inadequate response to surgery or for whom surgery is not an option. Acromegaly is a rare and debilitating condition caused by a benign pituitary tumor that secretes excess growth hormone. There are an estimated 12,000 adults with acromegaly in the U.S. for whom PALSONIFY is an option.

• Vertex recorded PALSONIFY revenue of $10 million in September 2026, following the closing of the Crinetics Pharmaceuticals acquisition on September 1, 2026.
• More than 245 million individuals have access to PALSONIFY, representing approximately 80% of U.S. covered lives.
• An estimated 3,200 endocrinologists treat the majority of people with acromegaly in the United States. As of the end of the third quarter of 2026, approximately 500 of those target specialty endocrinologists are prescribing PALSONIFY.
• Among patients starting or switching therapy, PALSONIFY has the leading brand share at more than 40%.
• PALSONIFY was approved in the EU in April 2026, and Vertex expects to begin launches in European markets in the second half of 2027. The regulatory submission for PALSONIFY in Japan was completed in April 2026, and the Ministry of Health, Labor and Welfare (MHLW) has granted orphan drug designation in Japan.

Mid- and Late-Stage Pipeline Programs

Cystic Fibrosis

• Data from the Phase 1 study of next-generation 3.0 CFTR corrector VX-828 in people with CF with the F/MF genotype showed that the triple combination therapy containing VX-828 was safe and well tolerated. Treatment with the VX-828 triple combination therapy resulted in a 6.6 mmol/L mean reduction in sweat chloride at 4 weeks of treatment and a 9.4 mmol/L mean reduction through 4 weeks of treatment, compared to a baseline on TRIKAFTA.
• Vertex continues to enroll and dose first-in-human studies in healthy volunteers with VX-581 and VX-272, additional next-generation 3.0 CFTR correctors. As part of the Phase 1 program, Vertex expects to study both correctors in people with CF to establish proof of concept.
• Once proof-of-concept results are available from all three next-generation 3.0 programs, Vertex will make the decision on which, if any, of the next-generation 3.0 regimens should be advanced into further development. Next-generation 4.0 programs also continue to make progress in pre-clinical development.

Acute and Peripheral Neuropathic Pain (PNP)

• Vertex remains on track to complete enrollment in both Phase 3 studies of suzetrigine in diabetic peripheral neuropathy (DPN), a form of peripheral neuropathic pain (PNP), by the end of 2026 and to share results in mid-2027.
• Vertex is also on track to complete enrollment in a Phase 2 study of VX-993 in people with DPN by the end of 2026 and also to share results in mid-2027.
• Vertex continues to advance toward clinical trials of preclinical assets that inhibit Na_V1.7 for use alone or in combination with a Na_V1.8 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).

• The BLA submission for accelerated approval of povetacicept in IgAN is under priority review by the U.S. FDA, with an assigned PDUFA target action date of November 30, 2026. The submission is supported by positive data from a pre-specified Week 36 interim analysis of the ongoing Phase 3 RAINIER trial of povetacicept in IgAN. If approved, povetacicept will become the first commercialized therapy in Vertex’s emerging nephrology franchise.
• Povetacicept is also under review for accelerated approval in IgAN in the Kingdom of Saudi Arabia, and the Saudi Food and Drug Authority has granted povetacicept Breakthrough Designation.
• Vertex is enrolling and dosing the Phase 3 portion of the Phase 2/3 OLYMPUS pivotal study in people with pMN with the 80mg dose selected in the Phase 2b portion. The FDA has granted Fast Track and Orphan Drug designations for povetacicept in pMN, and the EMA has granted Priority Medicines (PRIME) designation.
• Vertex has completed enrollment in the placebo-controlled, Phase 2 dose-ranging proof-of-concept study evaluating povetacicept for the treatment of gMG. Vertex is on track to share results from the study in the first half of 2027.

APOL1-Mediated Kidney Disease (AMKD)

Vertex has discovered and advanced multiple oral, small molecule inhibitors of APOL1 function, including inaxaplin, pioneering a new class of medicines that targets the underlying cause of this genetic kidney disease.

• Vertex announced positive results from the Phase 2b AMPLIFIED study of inaxaplin in people with AMKD with modest proteinuria (improvement in urine albumin to creatinine ratio [UACR] of -42.7%, and urine protein to creatinine ratio [UPCR] of -44.7%) and people with AMKD and Type 2 diabetes (improvement in UACR of -17.3%, UPCR of -25.4%) — populations not being studied in the AMPLITUDE trial. Inaxaplin was generally safe and well tolerated in both cohorts.
• Having completed enrollment in the interim analysis (IA) cohort of the AMPLITUDE pivotal study last year, Vertex has now completed full enrollment. Vertex remains on track to conduct the pre-planned IA for potential U.S. accelerated approval after the IA cohort reaches 48 weeks of treatment and to share data from the IA in early 2027. The FDA has granted Breakthrough Therapy designation to inaxaplin, and the European Medicines Agency (EMA) has granted PRIME designation.

Rare Endocrine Disorders

Vertex is developing atumelnant, a once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist, for the treatment of classic congenital adrenal hyperplasia (CAH), which affects 17,000 people in the U.S. Classic CAH is a rare, chronic, genetic condition in which the adrenal glands are unable to make enough cortisol. Current therapies force a choice between androgen control and steroid burden, representing a significant unmet need. Through Phase 2, atumelnant has shown transformative potential to both normalize androgen levels and decrease glucocorticoid requirements, the true goal of CAH treatment. Atumelnant has also demonstrated therapeutic potential in patients with ACTH-dependent Cushing’s Syndrome, another serious endocrine disease, which affects more than 10,000 people in the U.S.

• The Phase 3 trial of atumelnant in adults with CAH is a global, 32-week, randomized, double blind, placebo-controlled trial of 150 patients, with a primary endpoint of percentage of patients who achieve physiologic doses of glucocorticoids and androgen levels at or below the upper limit of normal at week 32. Enrollment and dosing are well underway, and Vertex expects to complete enrollment in the trial in the first quarter of 2027. The FDA, the European Medicines Agency (EMA), and the MHLW in Japan have granted Orphan Drug designation to atumelnant in CAH.
• Vertex is also enrolling and dosing the Phase 2 dose-ranging portion of a Phase 2/3 study of atumelnant in children with CAH.
• Vertex has initiated a Phase 2/3 study of atumelnant in patients with ACTH-dependent Cushing’s syndrome. The FDA has granted Orphan Drug designation to atumelnant in ACTH-dependent Cushing’s syndrome.

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 enrolling and dosing a Phase 1/2 trial to evaluate the safety and efficacy of VX-017 in people with T1D. VX-017 is an allogeneic, stem cell-derived, fully differentiated islet cell therapy designed to treat the targeted patient population with T1D, regardless of blood type.
• Given the rapid progress with VX-017, Vertex has decided to focus on accelerating the development of VX-017, which has the potential to reach more patients than zimislecel, which is designed for patients with blood types A or A/B only. Vertex expects to advance VX-017 into pivotal development in 2027. The FDA has granted Fast Track designation to VX-017.
• Vertex continues to make progress in pre-clinical development on advanced methods to protect the manufactured islet cells as well as on hypoimmune islet cells. Both approaches would reduce or obviate the need for standard immunosuppression.

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 has completed enrollment and continues to dose patients in AGLOW, a Phase 2 study of VX-407 in patients with a subset of variants in the PKD1 gene, which encodes the PC1 protein, estimated to be up to approximately 30,000 (or up to approximately 10%) of the overall patient population living with ADPKD in the U.S. and Europe.
• AGLOW is a 26-patient, single-arm, 52-week, Phase 2 proof-of-concept study that will evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV).

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 completed enrollment and dosing in the multiple ascending dose portion of the GALILEO global Phase 1/2 clinical trial of VX-670. The study is assessing both safety and preliminary efficacy, as measured by the change from baseline in the splicing index on muscle biopsy as well as other endpoints, including those that evaluate muscle function and strength, such as video hand opening time (vHOT) and quantitative muscle testing (QMT) score.
• Vertex is on track to share interim results from the GALILEO study in the second half of 2026.

Additional Earlier Stage R&D Programs

Consistent with its overall strategy, Vertex takes a serial innovation approach to all of its programs, with additional assets or approaches across its portfolio.

Investments in External Innovation

• In September, Vertex completed its previously announced acquisition of Crinetics Pharmaceuticals for a total equity value of approximately $10.0 billion, or $9.0 billion net of cash, cash equivalents, and marketable securities acquired. The acquisition is accounted for as a business combination.
• In July, Vertex entered into a collaboration with AbCellera. Vertex made a $28 million upfront payment to AbCellera. 

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 Third Quarter 2026 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.

Non-GAAP Financial Measures

In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (i) stock-based compensation expense, (ii) intangible asset amortization expense, (iii) gains or losses related to the fair value of the company's strategic investments, (iv) increases or decreases in the fair value of contingent consideration, (v) acquisition-related costs, (vi) acquired inventory fair value step-up expense, (vii) an intangible asset impairment charge, and (viii) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company’s calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

The company provides guidance regarding combined R&D, AIPR&D and SG&A expenses and effective tax rate on a non-GAAP basis. Unless otherwise noted, the guidance regarding combined R&D, AIPR&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.

Vertex Pharmaceuticals Incorporated

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