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VRTXVertex Pharmaceuticals

Health care · Pharmaceuticals · Nasdaq

We are a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. 10-K, Item 1

Employees
6,400
Headquarters
Boston, MA
CEO
Reshma Kewalramani
Next results
around Feb 11, 2027
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What VRTX's management wrote with each quarter's results: the press release (8-K item 2.02, exhibit 99) and, when the company files them, its prepared remarks or the call transcript. The call itself is not filed with SEC, so most quarters have the release only.

Press release Nov 2, 2026 · EX-99.1

Vertex Reports Third Quarter 2026 Financial Results

Open on SEC ↗

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

— 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 —

-- 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.”

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 du e 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. 1

were 16.6% and 18.0%, respectively, compared to 16.6% and 17.6%, respectively, for the third quarter of 2025.

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.

was $1.2 billion in each of the third quarters of 2026 and 2025, as increased product revenue was of fset 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. 2

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.

Current FY Previous FY 2026 2026 Total $13.15 to $13.1 to revenue $13.25 $13.2 billion billion Legacy non-CF product Unchanged $0.5 billion revenue or greater (CASGEVY, JOURNAVX) PALSONIFY $40 to $50 revenue million Combined GAAP R&D, $7.15 to $6.3 to AIPR&D, and $7.2 billion $6.45 SG&A billion expenses * Combined non-GAAP $5.65 to R&D, Approximate… $5.75 AIPR&D, and $6.0 billion billion SG&A expenses* Non-GAAP 19.5% to effective Unchanged 20.5% tax rate *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 compensati… expense, and approximat… $500 million of compensati… and transaction costs related to the Crinetics Pharmaceut… acquisitio… **Combined GAAP and non-GAAP R&D, AIPR&D and SG&A expenses guidance includes approximat… $100 million of AIPR&D expenses.

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. 3 • 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 is a first-in-class, oral, selective, non-opioid Na V 1.8 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. 4 • As of August 2026, JOURNAVX has become the most prescribed branded oral pain medicine in the U.S.

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.

• 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. 5 • 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.

• 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 V 1.7 for use alone or in combination with a Na V 1.8 inhibitor in acute and neuropathic pain.

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. 6 • 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.

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.

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 7 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.

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 enrollin g and dosing a Ph ase 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.

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. 8 • 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).

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.

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

• 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.

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.” 9 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. 10

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. 11

2: In the three and nine months ended September 30, 2026, “Acquisition-related costs” primarily related to compensation expense associated with cash-settled unvested Crinetics Pharmaceuticals equity awards and transaction costs directly related to the Crinetics Pharmaceuticals acquisition.

3: In the three and nine months ended September 30, 2026 and 2025, “Tax adjustments” included the estimated income taxes related to non-GAAP adjustments to the company's pre-tax income and excess tax benefits related to stock-based compensation. 15

September 30, 2026 December 31, 2025 ────────────────────────────────────────────────────────────────────────────── Assets Cash, cash equivalents and marketable $ 6,303.3 $ 6,608.1 securities Accounts receivable, net 2,311.0 2,052.8 Inventories 2,245.3 1,686.8 Prepaid expenses and other current 826.7 853.3 assets Total current assets 11,686.3 11,201.0 Property and equipment, net 1,711.5 1,520.3 Goodwill and other intangible assets, 10,941.2 1,512.2 net Deferred tax assets 1,952.9 2,897.9 Operating lease assets 1,617.2 1,562.7 Long-term marketable 3,517.9 5,712.3 securities Other long-term assets 1,309.3 1,236.6 Total assets $ 32,736.3 $ 25,643.0 Liabilities and Shareholders' Equity Accounts payable and $ 3,707.7 $ 3,432.9 accrued expenses Short-term debt 999.6 — Other current liabilities 293.0 428.3 Total current liabilities 5,000.3 3,861.2 Long-term debt 3,497.0 — Long-term operating lease 1,981.8 1,846.5 liabilities Other long-term liabilities 1,349.8 1,269.5 Shareholders' equity 20,907.4 18,665.8 Total liabilities and shareholders' $ 32,736.3 $ 25,643.0 equity Common shares outstanding 253.5 254.0

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, acute pain and acromegaly, 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, congenital adrenal hyperplasia, ACTH-dependent Cushing's syndrome, 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 17 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 www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X.

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 revenue from CASGEVY and JOURNAVX combined, and $40 million to $50 million in revenue from PALSONIFY, (ii) expectations for continued growth in CF, including the continued global uptake of ALYFTREK, plans for the studies evaluating each of VX-581 and VX-272, and plans for potential advancement of next-generation 3.0 regimens once proof-of-concept results are available for all next-generation 3.0 programs, and expectations for continued progress in next-generation 4.0 programs, (iii) beliefs regarding the expected clinical benefits of CASGEVY and eligible patient population, (iv) expectations to complete enrollment in both Phase 3 studies of suzetrigine in DPN by the end of 2026 and share results mid-2027, expectations to complete enrollment in the Phase 2 study of VX-993 in DPN by the end of 2026 and share results in mid-2027, and plans for advancement of preclinical assets that inhibit Na V 1.7, (v) beliefs regarding the expected clinical benefits of PALSONIFY, expectations for the eligible patient population, and expectations to begin launches in European markets in the second half of 2027, (vi) 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 for the Phase 2/3 OLYMPUS pivotal study in pMN, and plans to share results from the Phase 2 gMG study in the first half of 2027, (vii) expectations regarding the AMPLITUDE study of inaxaplin in AMKD, including plans to conduct the interim analysis for AMPLITUDE for potential accelerated approval in the U.S. and to share data in early 2027, (viii) beliefs regarding the potential benefits of atumelnant for the treatment of CAH and ACTH-dependent Cushing’s Syndrome, including with respect to the treatable patient population and potential clinical 17 benefits, plans for the Phase 3 trial of atumelnant in adults with CAH, including expectations to complete enrollment in the first quarter of 2027, and plans for the Phase 2 dose-ranging portion of the Phase 2/3 study of atumelnant in children with CAH, (ix) expectations regarding the T1D program, including plans for the study evaluating VX-017, beliefs regarding the potential of VX-017 to reach more patients than zimislecel, and expectations to advance VX-017 into pivotal development in 2027, and with respect to progress in pre-clinical development of advanced methods to protect the manufactured islet cells as well as on hypoimmune islet cells, (x) expectations regarding the ADPKD program and the Phase 2 study evaluating VX-407, (xi) beliefs regarding the potential benefits and clinical status of VX-670 for the treatment in people with DM1 and expectations to complete dosing in the trial and share interim results in the second half of 2026, and (xii) the company’s beliefs with respect to additional assets or approaches 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 not be realized (including because one or more of the company's assumptions underlying its expectations may be incorrect), that the company may be unable to continue increasing revenues from its commercialized medicines, 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 acquisitions or 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, that anticipated commercial launches may be delayed, if they occur at all, that external factors may have different or more significant impacts on the company's business or operations than the company currently expects, and other risks listed under the heading “Risk Factors” in Vertex’s annual report on Form 10-K, and subsequent quarterly reports on Form 10-Q filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. 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.

Full text with the tables
                                                                                
              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                                                                        
                                                                                
-- 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 du e 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.
                                       1                                        
                                                                                
  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 of fset 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.
                                       2                                        
                                                                                
  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:
                                                                                
                 Current FY                      Previous FY                    
                 2026                            2026                           
  Total                          $13.15 to                        $13.1 to      
  revenue                        $13.25                           $13.2         
                                 billion                          billion       
  Legacy                                                                        
  non-CF                                                                        
  product                        Unchanged                        $0.5 billion  
  revenue                                                         or greater    
  (CASGEVY,                                                                     
  JOURNAVX)                                                                     
  PALSONIFY                      $40 to $50                                     
  revenue                        million                                        
  Combined                                                                      
  GAAP R&D,                      $7.15 to                         $6.3 to       
  AIPR&D, and                    $7.2 billion                     $6.45         
  SG&A                                                            billion       
  expenses *                                                                    
  Combined                                                                      
  non-GAAP                                                        $5.65 to      
  R&D,                           Approximate…                     $5.75         
  AIPR&D, and                    $6.0 billion                     billion       
  SG&A                                                                          
  expenses*                                                                     
  Non-GAAP                                                        19.5% to      
  effective                      Unchanged                        20.5%         
  tax rate                                                                      
  *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                                                                   
  compensati…                                                                   
  expense,                                                                      
  and                                                                           
  approximat…                                                                   
  $500                                                                          
  million of                                                                    
  compensati…                                                                   
  and                                                                           
  transaction                                                                   
  costs                                                                         
  related to                                                                    
  the                                                                           
  Crinetics                                                                     
  Pharmaceut…                                                                   
  acquisitio…                                                                   
  **Combined                                                                    
  GAAP and                                                                      
  non-GAAP                                                                      
  R&D, AIPR&D                                                                   
  and SG&A                                                                      
  expenses                                                                      
  guidance                                                                      
  includes                                                                      
  approximat…                                                                   
  $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 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.
                                       3                                        
• 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 V 1.8 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.
                                       4                                        
• 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.
                                       5                                        
• 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 V 1.7 for use alone or in combination with a Na V 1.8 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.
                                       6                                        
• 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
                                       7                                        
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 enrollin g and dosing a Ph ase 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.
                                       8                                        
• 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.”
                                       9                                        
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.
                                       10                                       
                                                                                
  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.
                                       11                                       
                                                                                
                      Vertex Pharmaceuticals Incorporated                       
                                                                                
                                                                                
                       Consolidated Statements of Income                        
                                                                                
               (unaudited, in millions, except per share amounts)               
                                                                                
           Three                               Nine                             
           Months                              Months                           
           Ended                               Ended                            
           Septem…                             Septe…                           
           30,                                 30,                              
  Reve…    2026               2025             2026               2025          
 ────────────────────────────────────────────────────────────────────────────── 
  Prod…                                                                         
  reve…    $        3,440…    $     3,076.4    $       9,761.6    $     8,780…  
  net                                                                           
  Other                  —                —                  —            30.7  
  reve…                                                                         
  Total             3,440…          3,076.4            9,761.6          8,811…  
  reve…                                                                         
  Costs                                                                         
  and                                                                           
  expe…                                                                         
  Cost                                                                          
  of                 526.1            414.8            1,408.1          1,185…  
  sales                                                                         
  Rese…                                                                         
  and               1,320…            977.7            3,276.0          2,935…  
  deve…                                                                         
  expe…                                                                         
  Acqu…                                                                         
  in-p…                                                                         
  rese…               40.6             54.5               62.5            76.5  
  and                                                                           
  deve…                                                                         
  expe…                                                                         
  Sell…                                                                         
  gene…                                                                         
  and                844.1            445.1            1,920.0          1,266…  
  admi…                                                                         
  expe…                                                                         
  Inta…                                                                         
  asset                  —                —                  —           379.0  
  impa…                                                                         
  char…                                                                         
  Chan…                                                                         
  in                                                                            
  fair                                                                          
  value               -3.5             -1.9               -2.9             1.2  
  of                                                                            
  cont…                                                                         
  cons…                                                                         
  Total                                                                         
  costs             2,727…          1,890.2            6,663.7          5,843…  
  and                                                                           
  expe…                                                                         
  Inco…                                                                         
  from               712.9          1,186.2            3,097.9          2,967…  
  oper…                                                                         
  Inte…              118.4            125.7              360.1           369.0  
  inco…                                                                         
  Inte…              -36.0             -3.3              -42.3           -10.0  
  expe…                                                                         
  Other                                                                         
  (exp…              -11.9             -9.8               12.4           -14.2  
  inco…                                                                         
  net                                                                           
  Inco…                                                                         
  befo…                                                                         
  prov…              783.4          1,298.8            3,428.1          3,312…  
  for                                                                           
  inco…                                                                         
  taxes                                                                         
  Prov…                                                                         
  for                129.8            215.9              643.3           550.1  
  inco…                                                                         
  taxes                                                                         
  Net      $         653.6    $     1,082.9    $       2,784.8    $     2,762…  
  inco…                                                                         
  Net                                                                           
  inco…                                                                         
  per                                                                           
  comm…                                                                         
  shar…                                                                         
  Basic    $          2.58    $        4.24    $         10.98    $      10.77  
  Dilu…    $          2.56    $        4.20    $         10.90    $      10.68  
  Shar…                                                                         
  used                                                                          
  in                                                                            
  per                                                                           
  share                                                                         
  calc…                                                                         
  Basic              253.5            255.6              253.7           256.4  
  Dilu…              255.2            257.6              255.5           258.6  
                                                                                
                                       12                                       
                                                                                
                      Vertex Pharmaceuticals Incorporated                       
                                                                                
                                                                                
                                 Total Revenues                                 
                                                                                
                            (unaudited, in millions)                            
                                                                                
                     Three                               Nine                   
                    Months                             Months                   
                     Ended                              Ended                   
                   Septem…                           Septemb…                   
             2026      30,    2025             2026       30,    2025           
 ────────────────────────────────────────────────────────────────────────────── 
  TRIKAF…    $     2,436.9    $     2,653.6    $      7,288.8    $     7,740.2  
  ALYFTR…            705.8            247.0           1,703.8            457.7  
  Other                                                                         
  CF                                                                            
  product            130.5            139.3             403.5            488.3  
  revenu…                                                                       
  (1)                                                                           
  Total                                                                         
  CF                                                                            
  product          3,273.2          3,039.9           9,396.1          8,686.2  
  revenu…                                                                       
  net                                                                           
  CASGEVY             99.2             16.9             218.5             61.5  
  JOURNA…             58.3             19.6             136.9             32.9  
  PALSON…             10.1                —              10.1                —  
  Product                                                                       
  revenu…          3,440.8          3,076.4           9,761.6          8,780.6  
  net                                                                           
  Other                  —                —                 —             30.7  
  revenu…                                                                       
  Total      $     3,440.8    $     3,076.4    $      9,761.6    $     8,811.3  
  revenu…                                                                       
  1:                                                                            
  Includ…                                                                       
  KALYDE…                                                                       
  ORKAMB…                                                                       
  and                                                                           
  SYMDEK…                                                                       
                                                                                
                                       13                                       
                                                                                
                      Vertex Pharmaceuticals Incorporated                       
                                                                                
                                                                                
            Reconciliation of GAAP to Non-GAAP Financial Information            
                                                                                
                  (unaudited, in millions, except percentages)                  
                                                                                
                     Three                               Nine                   
                    Months                             Months                   
                     Ended                              Ended                   
                   Septem…                           Septemb…                   
             2026      30,    2025             2026       30,    2025           
 ────────────────────────────────────────────────────────────────────────────── 
  GAAP                                                                          
  cost of    $       526.1    $       414.8    $      1,408.1    $     1,185.3  
  sales                                                                         
  Acquir…                                                                       
  invent…                                                                       
  fair                -5.4                —              -5.4                —  
  value                                                                         
  step-up                                                                       
  expense                                                                       
  Stock-…                                                                       
  compen…             -4.2             -2.8             -11.2             -7.9  
  expense                                                                       
  Intang…                                                                       
  asset              -20.3             -5.0             -30.4            -15.1  
  amorti…                                                                       
  expense                                                                       
  Non-GA…                                                                       
  cost of    $       496.2    $       407.0    $      1,361.1    $     1,162.3  
  sales                                                                         
  GAAP                                                                          
  resear…                                                                       
  and        $     1,320.6    $       977.7    $      3,276.0    $     2,935.8  
  develo…                                                                       
  expens…                                                                       
  Stock-…                                                                       
  compen…           -102.0           -116.0            -308.1           -315.7  
  expense                                                                       
  Intang…                                                                       
  asset               -0.6             -0.6              -1.9             -1.9  
  amorti…                                                                       
  expense                                                                       
  Acquis…                                                                       
  costs             -282.4                —            -282.4                —  
  (2)                                                                           
  Non-GA…                                                                       
  resear…                                                                       
  and        $       935.6    $       861.1    $      2,683.6    $     2,618.2  
  develo…                                                                       
  expens…                                                                       
  Acquir…                                                                       
  in-pro…                                                                       
  resear…    $        40.6    $        54.5    $         62.5    $        76.5  
  and                                                                           
  develo…                                                                       
  expens…                                                                       
  GAAP                                                                          
  sellin…                                                                       
  general    $       844.1    $       445.1    $      1,920.0    $     1,266.1  
  and                                                                           
  admini…                                                                       
  expens…                                                                       
  Stock-…                                                                       
  compen…            -68.2            -76.1            -191.7           -204.7  
  expense                                                                       
  Acquis…                                                                       
  costs             -219.6                —            -219.6                —  
  (2)                                                                           
  Non-GA…                                                                       
  sellin…                                                                       
  general    $       556.3    $       369.0    $      1,508.7    $     1,061.4  
  and                                                                           
  admini…                                                                       
  expens…                                                                       
  Combin…                                                                       
  non-GA…                                                                       
  R&D,                                                                          
  AIPR&D     $     1,532.5    $     1,284.6    $      4,254.8    $     3,756.1  
  and                                                                           
  SG&A                                                                          
  expens…                                                                       
  GAAP                                                                          
  other                                                                         
  (expen…    $       -11.9    $        -9.8    $         12.4    $       -14.2  
  income,                                                                       
  net                                                                           
  (Incre…                                                                       
  decrea…                                                                       
  in fair                                                                       
  value               -2.1              2.6             -37.3             12.2  
  of                                                                            
  strate…                                                                       
  invest…                                                                       
  Non-GA…                                                                       
  other      $       -14.0    $        -7.2    $        -24.9    $        -2.0  
  expens…                                                                       
  net                                                                           
  GAAP                                                                          
  provis…                                                                       
  for        $       129.8    $       215.9    $        643.3    $       550.1  
  income                                                                        
  taxes                                                                         
  Tax                                                                           
  adjust…            137.1             47.8             227.4            240.0  
  (3)                                                                           
  Non-GA…                                                                       
  provis…                                                                       
  for        $       266.9    $       263.7    $        870.7    $       790.1  
  income                                                                        
  taxes                                                                         
                                                                                
                                                                           
  GAAP effective tax rate        16.6  %    16.6  %    18.8  %    16.6  %  
  Non-GAAP effective tax rate    18.0  %    17.6  %    19.6  %    18.6  %  
                                                                           
                                       14                                       
                                                                                
                      Vertex Pharmaceuticals Incorporated                       
                                                                                
      Reconciliation of GAAP to Non-GAAP Financial Information (continued)      
                                                                                
               (unaudited, in millions, except per share amounts)               
                                                                                
                     Three                               Nine                   
                    Months                             Months                   
                     Ended                              Ended                   
                   Septem…                           Septemb…                   
             2026      30,    2025             2026       30,    2025           
 ────────────────────────────────────────────────────────────────────────────── 
  GAAP                                                                          
  operat…    $       712.9    $     1,186.2    $      3,097.9    $     2,967.4  
  income                                                                        
  Acquir…                                                                       
  invent…                                                                       
  fair                 5.4                —               5.4                —  
  value                                                                         
  step-up                                                                       
  expense                                                                       
  Stock-…                                                                       
  compen…            174.4            194.9             511.0            528.3  
  expense                                                                       
  Intang…                                                                       
  asset                  —                —                 —            379.0  
  impair…                                                                       
  charge                                                                        
  Intang…                                                                       
  asset               20.9              5.6              32.3             17.0  
  amorti…                                                                       
  expense                                                                       
  (Decre…                                                                       
  increa…                                                                       
  in fair                                                                       
  value               -3.5             -1.9              -2.9              1.2  
  of                                                                            
  contin…                                                                       
  consid…                                                                       
  Acquis…                                                                       
  costs              502.0                —             502.0                —  
  (2)                                                                           
  Non-GA…                                                                       
  operat…    $     1,412.1    $     1,384.8    $      4,145.7    $     3,892.9  
  income                                                                        
  GAAP                                                                          
  net        $       653.6    $     1,082.9    $      2,784.8    $     2,762.1  
  income                                                                        
  Acquir…                                                                       
  invent…                                                                       
  fair                 5.4                —               5.4                —  
  value                                                                         
  step-up                                                                       
  expense                                                                       
  Stock-…                                                                       
  compen…            174.4            194.9             511.0            528.3  
  expense                                                                       
  Intang…                                                                       
  asset                  —                —                 —            379.0  
  impair…                                                                       
  charge                                                                        
  Intang…                                                                       
  asset               20.9              5.6              32.3             17.0  
  amorti…                                                                       
  expense                                                                       
  (Incre…                                                                       
  decrea…                                                                       
  in fair                                                                       
  value               -2.1              2.6             -37.3             12.2  
  of                                                                            
  strate…                                                                       
  invest…                                                                       
  (Decre…                                                                       
  increa…                                                                       
  in fair                                                                       
  value               -3.5             -1.9              -2.9              1.2  
  of                                                                            
  contin…                                                                       
  consid…                                                                       
  Acquis…                                                                       
  costs              502.0                —             502.0                —  
  (2)                                                                           
  Total                                                                         
  non-GA…                                                                       
  adjust…            697.1            201.2           1,010.5            937.7  
  to                                                                            
  pre-tax                                                                       
  income                                                                        
  Tax                                                                           
  adjust…           -137.1            -47.8            -227.4           -240.0  
  (3)                                                                           
  Non-GA…                                                                       
  net        $     1,213.6    $     1,236.3    $      3,567.9    $     3,459.8  
  income                                                                        
  Net                                                                           
  income                                                                        
  per                                                                           
  diluted                                                                       
  common                                                                        
  share:                                                                        
  GAAP       $        2.56    $        4.20    $        10.90    $       10.68  
  Non-GA…    $        4.76    $        4.80    $        13.96    $       13.38  
  Shares                                                                        
  used in                                                                       
  diluted                                                                       
  per                                                                           
  share                                                                         
  calcul…                                                                       
  GAAP                                                                          
  and                255.2            257.6             255.5            258.6  
  Non-GA…                                                                       
                                                                                
2: In the three and nine months ended September 30, 2026, “Acquisition-related
costs” primarily related to compensation expense associated with cash-settled
unvested Crinetics Pharmaceuticals equity awards and transaction costs directly
related to the Crinetics Pharmaceuticals acquisition.

3: In the three and nine months ended September 30, 2026 and 2025, “Tax
adjustments” included the estimated income taxes related to non-GAAP adjustments
to the company's pre-tax income and excess tax benefits related to stock-based
compensation.
                                       15                                       
                                                                                
                      Vertex Pharmaceuticals Incorporated                       
                                                                                
                                                                                
                     Condensed Consolidated Balance Sheets                      
                                                                                
                            (unaudited, in millions)                            
                                                                                
                                    September 30, 2026       December 31, 2025  
 ────────────────────────────────────────────────────────────────────────────── 
  Assets                                                                        
  Cash, cash equivalents and                                                    
  marketable                     $             6,303.3    $            6,608.1  
  securities                                                                    
  Accounts receivable, net                     2,311.0                 2,052.8  
  Inventories                                  2,245.3                 1,686.8  
  Prepaid expenses and other                                                    
  current                                        826.7                   853.3  
  assets                                                                        
  Total current assets                        11,686.3                11,201.0  
  Property and equipment, net                  1,711.5                 1,520.3  
  Goodwill and other                                                            
  intangible assets,                          10,941.2                 1,512.2  
  net                                                                           
  Deferred tax assets                          1,952.9                 2,897.9  
  Operating lease assets                       1,617.2                 1,562.7  
  Long-term marketable                         3,517.9                 5,712.3  
  securities                                                                    
  Other long-term assets                       1,309.3                 1,236.6  
  Total assets                   $            32,736.3    $           25,643.0  
  Liabilities and                                                               
  Shareholders' Equity                                                          
  Accounts payable and           $             3,707.7    $            3,432.9  
  accrued expenses                                                              
  Short-term debt                                999.6                       —  
  Other current liabilities                      293.0                   428.3  
  Total current liabilities                    5,000.3                 3,861.2  
  Long-term debt                               3,497.0                       —  
  Long-term operating lease                    1,981.8                 1,846.5  
  liabilities                                                                   
  Other long-term liabilities                  1,349.8                 1,269.5  
  Shareholders' equity                        20,907.4                18,665.8  
  Total liabilities and                                                         
  shareholders'                  $            32,736.3    $           25,643.0  
  equity                                                                        
  Common shares outstanding                      253.5                   254.0  
                                                                                
                                       16                                       
                                                                                
  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, acute pain and acromegaly, 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, congenital adrenal hyperplasia,
ACTH-dependent Cushing's syndrome, 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 17 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 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
revenue from CASGEVY and JOURNAVX combined, and $40 million to $50 million in
revenue from PALSONIFY, (ii) expectations for continued growth in CF, including
the continued global uptake of ALYFTREK, plans for the studies evaluating each
of VX-581 and VX-272, and plans for potential advancement of next-generation 3.0
regimens once proof-of-concept results are available for all next-generation 3.0
programs, and expectations for continued progress in next-generation 4.0
programs, (iii) beliefs regarding the expected clinical benefits of CASGEVY and
eligible patient population, (iv) expectations to complete enrollment in both
Phase 3 studies of suzetrigine in DPN by the end of 2026 and share results
mid-2027, expectations to complete enrollment in the Phase 2 study of VX-993 in
DPN by the end of 2026 and share results in mid-2027, and plans for advancement
of preclinical assets that inhibit Na V 1.7, (v) beliefs regarding the expected
clinical benefits of PALSONIFY, expectations for the eligible patient
population, and expectations to begin launches in European markets in the second
half of 2027, (vi) 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 for the Phase 2/3 OLYMPUS
pivotal study in pMN, and plans to share results from the Phase 2 gMG study in
the first half of 2027, (vii) expectations regarding the AMPLITUDE study of
inaxaplin in AMKD, including plans to conduct the interim analysis for AMPLITUDE
for potential accelerated approval in the U.S. and to share data in early 2027,
(viii) beliefs regarding the potential benefits of atumelnant for the treatment
of CAH and ACTH-dependent Cushing’s Syndrome, including with respect to the
treatable patient population and potential clinical
                                       17                                       
benefits, plans for the Phase 3 trial of atumelnant in adults with CAH,
including expectations to complete enrollment in the first quarter of 2027, and
plans for the Phase 2 dose-ranging portion of the Phase 2/3 study of atumelnant
in children with CAH, (ix) expectations regarding the T1D program, including
plans for the study evaluating VX-017, beliefs regarding the potential of VX-017
to reach more patients than zimislecel, and expectations to advance VX-017 into
pivotal development in 2027, and with respect to progress in pre-clinical
development of advanced methods to protect the manufactured islet cells as well
as on hypoimmune islet cells, (x) expectations regarding the ADPKD program and
the Phase 2 study evaluating VX-407, (xi) beliefs regarding the potential
benefits and clinical status of VX-670 for the treatment in people with DM1 and
expectations to complete dosing in the trial and share interim results in the
second half of 2026, and (xii) the company’s beliefs with respect to additional
assets or approaches 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 not be realized (including
because one or more of the company's assumptions underlying its expectations may
be incorrect), that the company may be unable to continue increasing revenues
from its commercialized medicines, 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 acquisitions or 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, that anticipated commercial launches may be delayed, if they occur at
all, that external factors may have different or more significant impacts on the
company's business or operations than the company currently expects, and other
risks listed under the heading “Risk Factors” in Vertex’s annual report on Form
10-K, and subsequent quarterly reports on Form 10-Q filed with the Securities
and Exchange Commission at www.sec.gov and available through the company's
website at www.vrtx.com. 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.

(VRTX-E)
                                                                                
  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
                                       18                                       

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