The companies on this annual list make a conscious effort to be supportive, inclusive workplaces.

2026_GPTW_best-companies-for-women-Large-Vertex_Pharmaceuticals_Inc

Employees say:"Vertex is amazing when it comes to benefits and also taking care of its employees, especially when they have dealt with the loss of a family member. I cannot thank everyone enough—from HR to my colleagues, direct management all the way to upper management—who reached out to help and/or check on me during this difficult time. I feel work is more than work and is an extension of my family. Where else could I go to find a company that cares about helping patients and also cares about my well-being?"

100 Best Large Workplaces for Women

See which workplaces made this year's list.

2026_GPTW_best-places-by-industry-BioPharma-Vertex_Pharmaceuticals_Inc

Employees say:"I love working at Vertex because of how hard they go above and beyond to make everyone feel welcome and to help them understand what it is that we do. In my experience, everyone has the shared goal of putting patients first and they have the desire to go above and beyond to bring transformative treatments to patients. &nbsp;It's not just a 'mission statement' but feels like it is the actual foundation of what we do."

Best Large Workplaces in Biotechnology and Pharmaceuticals

Companies that foster an environment that supports professional growth are more likely to thrive and successfully adopt digital innovation. Here are the 100 best places to work in Europe—and how they stay that way.

Vertex Pharmaceuticals ranks 94 on Fortune 100 Best Companies to Work For – Europe 2026 list.

Fortune 100 Best Companies To Work For – Europe

With a combined revenue of $21 trillion and $2.1 trillion in profits, the 2026 Fortune 500 is the definitive list of America's largest companies.

Fortune 500

This year's 100 Best Companies to Work For are rebuilding the social contract at work so people feel supported, trusted, and future-ready as they undergo that transition.

100 Best Places to Work 2026_Vertex Pharmaceuticals Inc

Employees say:"Vertex is a company that truly cares, from the top down, about the mission for bringing transformative therapies to patients and about its employees. The benefits offered here are unparalleled, as are the opportunities for growth and development. This is a truly special place to be."

Fortune 100 Best Companies to Work For

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Employees say:&nbsp;"The Vertex 101 and other scientific and commercial learning series is awesome! The 'leading edge' is also a great connection point for the average employee that is appreciated and 'humanizes' the C-Suite. Finally, the continued learning series that provides development in management and job skills is one of a kind and shows the investment Vertex makes for its employees as workers and people in general."

Almost $20 trillion in revenue—and a record-breaking $1.87 trillion in profits. Introducing the 2025 Fortune 500, the definitive list of the biggest companies in America.

The top companies on our list have one thing in common: Staff largely trust leadership to see them as well-rounded people and reward them for a job well done.

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Employees say: "This organization is like none other. Patients are at the center of everything we do. Secondly, the care and compassion we have for each other, both personal and professional, is unprecedented in the industry. The executive team and management are always seeking ideas and ways to innovate process to better serve the patients and the team."

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Employees say:"I love the professional, mutual respect, and care for the employees that is demonstrated on a daily basis. The commitment to patients and focus on innovation across all departments, make it very easy to be committed to and passionate about our work. In addition, the truly inclusive and diverse culture, is very modern and refreshing."

GPTW BioPharma Large 2024-Vertex Pharmaceuticals Inc

Employees say:"This organization is like none other. Patients are at the center of EVERYTHING we do. Secondly, the care and compassion we have for each other, both personal and professional is unprecedented in the industry. The executive team and management is always seeking ideas and ways to innovate process to better serve the patients and the team."

Fortune partners with global people analytics firm Great Place to Work to analyze survey feedback representing employees from companies across the U.S.

GPTW Millennials-2024-Vertex Pharmaceuticals Inc_US1__20240522184224_4

Employees say:"Vertex makes products that are truly life-changing for patients. People here recognize the importance of what we do, work hard, and are always looking for ways to do things better. Leadership recognizes the value of the employees and goes the extra mile to give recognition and ensure that the benefits we receive are at the top of our industry."

100 Best Large Workplaces for Millennials

Industries included in the sector are insurance and managed care, medical products and facilities, pharmacy services, and pharmaceutical companies.

Fortune Sector Leaders: Health Care

The Fortune 500, in its 70th year in 2024, ranks the biggest U.S. companies by revenue.

Creating a more empathetic workplace looks like staying loyal to workers, emphasizing D&I initiatives, and paying well.

100 Best Companies 2024-Vertex Pharmaceuticals Inc

Vertex, a global biotechnology company, invests in the scientific pursuit of medicines and treatments for serious diseases. It currently has ongoing clinical and research programs that treat underlying causes of cystic fibrosis, and recently <a href="https://www.nytimes.com/2024/01/30/health/vertex-pain-medicine-non-opioid.html">announced</a> progress toward a nonopioid investigational drug to treat moderate to severely acute pain.&nbsp; &nbsp;Flexible remote work, two weeks off in addition to paid vacation time, and commuting benefits are on the list of employee benefits. The company also encourages employees to get involved in volunteer opportunities by organizing an annual Global Day of Service, through which the company matches up to $5,000 per year to nonprofits of an employees’ choosing. The group’s CEO, Reshma Kewalramani, hosts small gatherings of employees through a meeting she calls Lemonade and Pretzels, to answer employee questions.&nbsp;

The 200 companies on this list are transforming industries from the inside out.

America's Most Innovative Companies

Building a culture of trust and transparency is essential to help everyone thrive, especially in rocky economic times.

100 BEST COMPANIES 2023-Vertex Pharmaceuticals Inc

The global biotechnology company invests in innovation in order to develop medicines for people with serious diseases. The company culture values recognizing the efforts of its employees. 
<ul> 
 	<li>When a major milestone is achieved, such as the FDA approving one of its medicines, Vertex brings in patients who will benefit from the drug, along with their families, to share their stories and ring a giant antique fire bell to commemorate the occasion.</li> 
 	<li>New managers take part in Lead, a 12-week immersive leadership development program designed to better help them manage a team.</li> 
 	<li>The company offers global synchronous days off, where all employees are off at the same time, ideally meaning no overflowing inbox or missed deadlines when they return, letting them truly enjoy the time away. Additionally, all of Vertex shuts down for two weeklong periods each year, letting employees focus on themselves, spend time with loved ones, and recharge.</li> 
 	<li>The company’s mentoring program uses a matching tool to facilitate pairings based on how closely aligned a mentee’s development goals are to a mentor’s strengths. As a result, people with different lived experiences and perspectives are connected, and mentors grow from the experience as well.</li> 
</ul>

GPTW 2023 Best Workplaces Pharma Large-Vertex Pharmaceuticals Inc_3

Employees say: 
 
"The company goes out of their way to ensure employees are allowed to have non-work time—like creating 'synchronous days' so that people taking a day off will not be bothered by others still working that day. They also make sure to engage the employees with all major accomplishments and have regular formal and informal discussions across employee groups to ensure all voices are heard."

GPTW Women 2023-Vertex Pharmaceuticals Inc

Employees say: 
 
"Vertex is nothing like anywhere I've ever worked. When you walk into the building, you can just feel that something special is happening there. It has a 'pulse' of people passionate about helping patients and innovating at high speeds. It is a privilege to work here!"

Fortune partnered with Great Place to Work to analyze survey feedback representing nearly half a million U.S. employees.

GPTW Millennial Large 2023-Vertex Pharmaceuticals Inc

Employees say: 
 
"People care about each other. The management cares about employees with great benefits about work-life balance! The work we do means a great deal to a lot of people/patients, and I'm so proud to work here!"

The Fortune 500, in its 69th year in 2023, ranks the biggest U.S. companies by revenue.

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Employees say: 
 
“This company puts our own personal career development as a higher priority than any other place I have worked. I have never done an individual development plan at any other company, never worked at a place that offered gigs and rotations like Vertex does, or had some many opportunities to work on side projects that align with my own interests. Our benefits are also unmatched in my experience, the stock offerings, opportunities for bonuses, health coverage (HSA particularly), and maternity leave as well as commuter benefits are incredibly generous.”

GPTW Women 2022-Vertex Pharmaceuticals Inc

Employees say: 
 
“This company puts our own personal career development as a higher priority than any other place I have worked. I have never done an individual development plan at any other company, never worked at a place that offered gigs and rotations like Vertex does, or had some many opportunities to work on side projects that align with my own interests.”

This year’s Fortune 500 marks the 68th running of the list.

This year’s Fortune 500 marks the 67th running of the list.

The biopharmaceutical company makes its debut on the Fortune 500 this year, riding the wave of a 49% year-over-year revenue increase in 2020—driven primarily by its cystic fibrosis drugs Kaftrio and Trikafta.

Collectively, the ranked companies produced a three-year average shareholder return of 33%, vs. 19% for the S&P 500.

FGC-2021-Vertex-Pharmaceuticals

100 Fastest-Growing Companies

Explore this year's ranking of the 50 companies with the best long-term growth potential.

Future-50-2020-Vertex-Pharmaceuticals

In the fall of 2019, Vertex launched Trikafta, the latest of its breakthrough therapies for people with cystic fibrosis. Capable of treating up to 90% of those who suffer from the progressive, life-threatening disease, the drug has already been used to treat thousands of patients in the U.S. and recently won approval in the EU. The company’s sales—$4.2 billion in 2019—have averaged 35% annual growth over the past three years. One setback: The biotech’s stock fell 23% in October after it scrapped a new drug in Phase II clinical trials.

The Future 50

This year's Fortune 500 marks the 66th running of the list.

Collectively, the ranked companies produced a three-year average shareholder return of 19%, vs. 11% for the S&P 500.

Fastest Growing Companies 2020-06-Vertex Pharmaceuticals

Fortune's proprietary methodology allows us to zero in on the 50 global firms that are best poised to deliver—and continue delivering—above average gains.

This year's Fortune 500 marks the 65th running of the list.

Welcome to the inaugural Fortune Future 50, our new ranking of companies best positioned for breakout growth.

This year's Fortune 500 marks the 64th running of the list.

Fastest-growing Companies

Revenue 3 Yr Growth Rank

Revenue 3 Yr Annual Growth Rate

EPS 3 Yr Growth Rank

EPS 3 Yr Annual Growth Rate

Total Return 3 Yr Rank

Total Return 3 Yr Annual Rate

Beat S&P 500 3 Yr Return

Current Streak

Years on List

Sector

Future 50

Overall Score

Culture rank

Process rank

Product rank

Best Companies

Rank last year

Years on list

U.S. employees

U.S. job openings

Year founded

Benefits

Subsidized childcare

Compressed work weeks

Option to work remotely

Minimum requirement in-office attendance (days per week)

Mental health coverage

Free mental health sessions (per year)

Free backup childcare time off (days)

Maximum $ amount covered for fertility services

Fully paid maternal leave (days)

Fully paid paternal leave (days)

Fully paid adoptive leave (days)

Maternal leave in addition to regular time off (days)

Paid days off for volunteering

Key Financials (last Fiscal Year)

Revenues ($M)

Profits ($M)

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Total Stockholder Equity ($M)

Profit Ratios

Profit as % of Revenues

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Profits as % of Stockholder Equity

Earnings Per Share (last Fiscal Year)

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Total Return

Total Return to Investors (last year)

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Best Companies To Work For Europe

CEO/Top executive

Group headquarters

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Certified countries

Woman CEO

Number of Years on List

Best Workplaces In Biopharma

Founded

Sites

Industry Vertical

Best Workplaces For Women

# Work Sites

Vertex Pharmaceuticals, a biotechnology company, develops and commercializes therapies for serious chronic conditions such as cystic fibrosis, which damages the lungs, digestive system, and other organs. Vertex also developed Casgevy, one of two gene therapies approved for the treatment of sickle cell anemia, a rare and painful blood disorder. Casgevy is also the first FDA-approved therapy that utilizes CRISPR/Cas9 genome editing technology.

Q3 2026

Q2 2026

Q1 2026

Q4 2025

Q3 2025

Q2 2025

Q1 2025

Q4 2024

Vertex Pharmaceuticals (VRTX): Company Profile, Stock Price, News, Rankings

legacy

Vertex Pharmaceuticals

vertex pharmaceuticals

Good day, and welcome to the Vertex Pharmaceuticals third quarter 2026 earnings call. All participants will be in a listen-only mode. Should you need assistance, please signal conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star then one on your touch-tone phone. To withdraw your question, please press star then two. Please note this event is being recorded. I would now like to turn the conference over to Ms. Susie Lisa. Please go ahead.

Good evening all. My name is Susie Lisa, and as the Senior Vice President of Investor Relations, it is my pleasure to welcome you to our third quarter 2026 financial results conference call. On tonight's call, making prepared remarks, we have Dr. Reshma Kewalramani, Vertex's CEO and President, Charlie Wagner, Chief Operating Officer and Chief Financial Officer, and Duncan McKechnie, Chief Commercial Officer. Given the January 1 transition by Charlie to the COO role solely, please note that this is his last quarterly earnings call. His successor, Jonathan Poole, our current SVP of Finance, will join us for our Q4 2026 call in February. We recommend that you access the webcast slides as you listen to this call. The call is being recorded, and a replay will be available on our website.

We will make forward-looking statements on this call that are subject to the risks and uncertainties discussed in detail in today's press release and in our filings with the Securities and Exchange Commission. These statements, including without limitation those regarding Vertex's marketed medicines for cystic fibrosis, sickle cell disease, beta thalassemia, moderate to severe acute pain, and acromegaly, our pipeline, the recently completed acquisition of Crinetics Pharmaceuticals and the expected benefits of that transaction, and Vertex's future financial performance are based on management's current assumptions. Actual outcomes and events could differ materially. I would also note that select financial results and guidance that we will review on the call this evening are presented on a non-GAAP basis. I'll now turn the call over to Reshma.

Thanks, Susie. Good evening all, and thank you for joining us on the call today. I'm pleased to note that Vertex achieved another quarter of excellent commercial results and pipeline advancement. Third quarter total revenue grew 12% year-on-year. Revenue growth was nicely balanced, coming from both the U.S. and international, from CF, and from our two more recently launched medicines, CASGEVY and JOURNAVX, as well as a contribution from PALSONIFY for the month of September, post-close of the Crinetics acquisition. Rare endocrine is now our fourth commercial pillar, and with the PDUFA date later this month for povetacicept in IgA nephropathy, or IgAN, we plan to soon establish our fifth in renal. Before turning to the R&D update, I want to acknowledge Charlie's final and 31st earnings call as CFO. Charlie's leadership, integrity, judgment, and intellectual rigor are unparalleled.

He has deftly helped lead our transition from a CF-only company with $4 billion in revenue in 2019 to a company soon to be commercialized in five disease areas with more than triple the revenue, with our guidance for over $13 billion in 2026. I look forward to continuing to work with Charlie in his role as COO, driving forward the Crinetics integration and our key priorities as we grow and scale the company. I can think of no better successor to Charlie than Jonathan Poole, our Senior Vice President and current head of finance, who will take over as CFO January 1, 2027. Moving now to the R&D update for the quarter. I will cover four disease areas: CF, endocrinology, both rare endocrine disorders and type 1 diabetes, and renal in detail. First, let me start with quick highlights from four other programs.

In severe sickle cell disease and transfusion-dependent beta thalassemia, we were pleased to have recently received approval for CASGEVY in patients ages two to 11 in the U.S., U.A.E., Bahrain, as well as in patients ages five to 11 in the U.K. In pain, along with the strong growth in prescriptions, we are also seeing fast growth in the body of compelling JOURNAVX real-world data emerge, such as a recent paper detailing 80% or greater freedom from opioid use following total knee replacement when the pain management regimen included JOURNAVX. In chronic pain, both phase III studies in painful diabetic peripheral neuropathy remain on track to complete enrollment by the end of this year, and for us to be able to share results by mid-2027.

In myotonic dystrophy type 1, or DM1, we remain on track to share interim results from the GALILEO global phase I/II clinical trial of VX-670 before the end of the year. In generalized myasthenia gravis, we have completed enrollment of the ETNA phase II dose-ranging study of povetacicept and expect to share proof-of-concept data in the first half of 2027. Moving on to the more detailed R&D review, starting with cystic fibrosis. We see ALYFTREK as the most effective medicine approved for CF. With once-daily dosing, ALYFTREK can treat up to 95% of people with CF, and in our trials, more than 75% of ALY-treated patients ages 12 and older achieve sweat chloride levels in the range of CF carriers. That is to say, ALYFTREK-treated patients are achieving sweat chloride levels associated with no manifestation of CF disease nor reduced life expectancy.

On the regulatory front, we continue our work to expand to treating younger patients and have initiated global submissions for ALYFTREK approval in children ages two to five. Lastly, in CF, an update on our next-generation 3.0 correctors. We studied VX-828 as part of a triple combination in a cohort of eight people with CF and as a dual therapy in another cohort of eight. All 16 patients had F/MF mutations, which is a more challenging population to treat than patients with F/F mutations. In this F/MF population, the VX-828 dual combination did not alter sweat chloride relative to the TRIKAFTA baseline. In the triple combination versus a baseline on TRIKAFTA, VX-828 delivered a 6.6 mmol per liter reduction in sweat chloride at day 28, and a 9.4 mmol reduction through day 28. The VX-828 regimen is oral, once daily, with a good safety, tolerability, and DDI profile.

In short, these are excellent results. Given the very high bar set by ALYFTREK, we intend to wait for all the data from the NG 3.0 family of molecules before determining next steps, namely advancement into pivotal development. VX-581 and VX-272, the other NG 3.0 correctors, are currently in first-in-human studies, and the next-gen 4.0 molecules are in late preclinical development. Moving to our newest pillar, rare endocrinology. With the Crinetics acquisition now closed, PALSONIFY in acromegaly is our eighth commercialized medicine, and Duncan will provide details on the ongoing U.S. launch. On the R&D side, the most advanced molecule after PALSONIFY is atumelnant in congenital adrenal hyperplasia, or CAH, which has orphan drug designation in the U.S., E.U., and Japan, and where the phase III pivotal trial is well underway.

The primary efficacy endpoint for this phase III study is the proportion of patients achieving the combination of normalization of androgens and physiologic glucocorticoid dose levels. This is the true goal in CAH therapy, and it is what was demonstrated in the phase II trial. It is also what no other CAH medicine to date has attempted to achieve. In adult CAH, we are targeting completion of enrollment in the phase III study in Q1 2027. The study then calls for 32 weeks of dosing. In pediatric CAH, atumelnant is in a phase II dose-ranging study. Our goal is to rapidly advance the pediatric program into pivotal development following planned regulatory interactions. In addition, the adaptive phase II/III global study of atumelnant in ACTH-dependent Cushing's syndrome is already underway outside the U.S. and was recently granted orphan drug designation in the U.S.

We have planned engagements with the FDA to confirm their expectations for this program and look forward to updating you in the coming months. Let me now turn to T1D. After careful analysis, we have chosen to prioritize acceleration of VX-017 as our lead program. Recall, VX-017 is an allogeneic, off-the-shelf, glucose-responsive, insulin-producing, fully differentiated islet cell therapy. It has a similar target product profile to zimislecel, but is designed for people with all blood types. This doubles the eligible patient population to about 120,000 people. The potential to bring transformative benefit to even more patients is the key reason to focus on VX-017, given the resource intensity, complexity, and manufacturing demands of cell therapy programs. We have already begun dosing patients with VX-017 in a phase I/II study.

Our near-term goals are to demonstrate proof of concept and to scale up manufacturing, with the goal of moving to pivotal development next year. Finally, we continue to progress our serial innovation work in T1D, focused on improved immunosuppression and on generating hypoimmune islets, with the goal of making this potentially curative therapy available to even more people. Turning now to renal, where we have four programs in mid and late-stage development. Starting with pove and IgAN, we are both excited and ready for our PDUFA date on November 30th, just weeks away. Duncan will address our launch readiness. We just wrapped up a sparkling ASN annual meeting with the RAINIER phase III interim analysis presentation and publication in the New England Journal of Medicine, supporting what we believe is a differentiated, potentially best-in-class profile for pove in IgAN.

Simply put, pove will offer a trifecta of excellence in efficacy, tolerability, and patient-centric administration features. I will focus tonight just on the patient-centric features. The commercial presentation of pove is clearly differentiated, with a small volume, 0.46 mils, once every four weeks, subcutaneously dosed via auto-injector at home. On this point, this advantage is reflected in the poster presented by If et al. at the recent ASN of a qualitative survey study of about 25 adults with IgAN in the U.S., that revealed 96% of patients preferred an auto-injector relative to injection via a prefilled syringe, and 80% preferred dosing every four weeks versus weekly. Our ambition is to launch pove globally. To that end, we have already completed our filing for potential accelerated approval in the Kingdom of Saudi Arabia, where it has been granted breakthrough status. We estimate KSA represents an incremental 7,000-patient opportunity.

We are planning forward for our regulatory submissions in other regions, including Europe and Asia, when the study completes and the two-year eGFR data are available. On pove in membranous nephropathy, our OLYMPUS phase II/III pivotal trial is ongoing, with phase III enrollment and dosing well underway at the selected dose of 80 mg subcutaneously every four weeks. Recall, this study has a 104-week endpoint. On inaxaplin in AMKD, we have two pieces of news since our last earnings call. First, to recap the exciting results from the AMPLIFIED study. In cohort one of patients with AMKD and modest proteinuria, treatment with inaxaplin on top of optimized standard of care led to a 42.7% reduction in urine albumin-to-creatinine ratio, or UACR, at week 13 compared to baseline, with the UPCR down 44.7%.

This is remarkably consistent with the treatment effect previously shared in the phase II data in AMKD published in the New England Journal of Medicine. In cohort two of people with AMKD and type 2 diabetes, treatment with inaxaplin led to a 17.3% reduction in UACR and a 25.4% reduction in UPCR at week 13. Of note, the majority of patients in both cohorts did not have a diagnosis of FSGS. Inaxaplin was generally safe and well-tolerated with no serious adverse events related to inaxaplin. All adverse events were mild or moderate in severity. We look forward to discussing these results with regulators in the context of the AMPLITUDE study next year. The second key milestone was that we completed full enrollment in the pivotal AMPLITUDE phase II/III trial, and we are fully on track to share the results from the interim analysis of AMPLITUDE early next year.

If positive, this interim analysis will support potential accelerated approval in the U.S. Rounding out on renal enrollment in the VX-407-AGLOW phase II study in ADPKD completed enrollment, and we are now on track for study readout next year. With that, I will turn the call over to Duncan for a commercial update.

Thanks very much, Reshma. Our commercial story continues to be one of excellence in launch execution across CF, heme, pain, and most recently, rare endocrinology. Through the first nine months of the year, our ongoing launches of ALYFTREK, CASGEVY, and JOURNAVX have generated over $2 billion in revenue, led by over $1.7 billion in ALYFTREK sales and more than $350 million in combined CASGEVY and JOURNAVX sales. To give you a brief overview and put quarter three revenue, patient, and prescription growth in context, in CF, we delivered $3.3 billion in revenue with strong switching to ALYFTREK and over 18,000 patients benefiting from ALYFTREK around the world. In heme, CASGEVY achieved $99 million in quarterly revenue with approximately 200 infusions completed since launch and a fourth consecutive quarter with more than 100 patient initiations.

In moderate to severe acute pain, JOURNAVX reported over 750,000 prescriptions and $58 million in revenue. More than 1.6 million patients have now been treated with JOURNAVX in the U.S., and JOURNAVX is now the number one prescribed branded oral analgesic in the U.S. In rare endocrinology, PALSONIFY contributed $10 million in revenue for the month of September following the close of the acquisition. For the full third quarter, PALSONIFY revenue was approximately $24 million. I will now cover these disease areas in more detail, starting with CF. The Q3 $3.3 billion in global CF revenue represents 8% year-over-year growth. Global growth overall was led by ALYFTREK, which delivered $706 million in worldwide sales in Q3. In the U.S., ALYFTREK sales year-to-date have now surpassed $1 billion.

In both the U.S. and outside the U.S., ALYFTREK sales continue to build primarily from patients switching from TRIKAFTA and new patients with rare mutations. Outside the U.S., we now have ALYFTREK reimbursement in 31 countries. Looking ahead, our CF growth drivers remain consistent and clear. Label expansions in rare mutations, penetration into younger age groups, additional geographies, and by the underlying survival benefit of patients on CFTR modulators. Turning to CASGEVY in severe sickle cell disease and transfusion-dependent beta thalassemia, where $99 million in Q3 revenue reflects 30% sequential growth. We have infused twice as many patients in the first nine months of 2026 as all of 2025. Since launch, we have initiated over 750 patients worldwide on CASGEVY, and with four consecutive quarters of initiation numbers exceeding 100 patients, we now have patients at all stages of the treatment journey.

We have also continued to enhance the patient journey, shortening the overall time from referral to infusion and decreased the number of mobilizations. We have excellent visibility into the number of infusions, which is when we recognize revenue, for the rest of this year as well as into 2027. That said, infusion timing reflects patient choice with some expected seasonality in quarter four, so I would encourage you to focus on the strong annual performance rather than any single quarter. CASGEVY will contribute meaningfully to our greater than $500 million non-CF revenue goal this year and is on its way to achieving its own blockbuster potential.

With JOURNAVX in moderate to severe acute pain, we are building a franchise for the long term and are focused on the following critical markers of success: broad payer coverage, prescription growth, breadth and depth of prescribing, the addition of JOURNAVX to hospital pathways, and revenue. These are the building blocks of a sustainable, long-term, multi-billion dollar pain business. Let me start with prescriptions because that's the best indicator of underlying physician and patient demand. In the third quarter, JOURNAVX prescriptions totaled over 750,000, up more than 40% sequentially from quarter two 2026. At the end of quarter three 2026, we are well on our way to achieving our guidance of more than three times the number of prescriptions in 2026 versus 2025. Prescriptions continue to split roughly 50/50 between hospital and retail channels.

Third quarter JOURNAVX revenue of $58 million, up 18% quarter-over-quarter, reflects continued high gross-to-net with the patient support savings program remaining on. Let me walk you through the revenue growth dynamics. A significant driver of gross-to-net is continued utilization of our patient support program. We have chosen to leave the PSP in place and extend it into 2027 so that people who are prescribed JOURNAVX, but whose plans do not yet cover it, can receive the therapy. Accordingly, when we sunset the PSP mid-next year, we continue to expect gross-to-net to stabilize at a level close to other mass market brands in the range of 50%-60%. In addition, in quarter three, gross-to-net and revenue were both affected by the one-time impact of securing broad Medicare Part D access in July.

As that access came online, previously uncontracted prescriptions shifted to contracted, rebated pricing, resulting in a one-time reset in net revenue. This is in line with our overall strategy to secure broad, sustained access across commercial and government plans so we can successfully convert physician practices to JOURNAVX for all their patients. This quarter marks our first opportunity to speak about PALSONIFY as part of Vertex following the deal close at the beginning of September, and we are pleased with the launch trajectory to date. Let me briefly describe the disease, the unmet need, and frame the key metrics at this stage in the launch. Acromegaly is a symptomatic disease that affects approximately 20,000 patients in the U.S. and a similar number in the five largest European markets, plus Japan.

Although surgery is the first line of treatment, only 40%-50% of people achieve durable remission, so the majority of patients need lifelong treatment. In the U.S., they're treated by approximately 3,000 endocrinologists. Until the launch of PALSONIFY, the main option has been monthly injections with somatostatin receptor ligands, or SRLs, delivered using large gauge needles. These treatments have three limitations. Firstly, they can cause painful injection site reactions at the beginning of the month. Secondly, their efficacy tends to taper off in the latter part of the month, leading to breakthrough symptoms such as joint pain, swelling, and fatigue. Thirdly, the recurring logistical challenges and burden of chronic depot therapy. The unmet need is therefore significant.

The ideal treatment for patients would be a highly effective, easy to take, well-tolerated oral medicine that provides sustained control of the disease, and one that has a broad indication for both naive and switch patients. This is the exact profile of PALSONIFY as the first and only once-daily oral SST2 selective agonist. As we integrate the Crinetics business, there are three areas of focus for the success of PALSONIFY, building on the excellent base of 80% reimbursed coverage. Firstly, build a broad base of repeat HCP writers for PALSONIFY. Secondly, drive growth in both the new and switch settings. Thirdly, deliver on our revenue goals. We are very pleased with the progress that the Crinetics team, and now the integrated Vertex team, are making against these goals.

Approximately 500 endocrinologists are writing PALSONIFY prescriptions, and there was a significant increase in the number of repeat writers in quarter three compared to the first half of 2026. PALSONIFY now has the leading brand share at over 40% among patients initiating or switching therapy. Lastly, we see continued strong growth in patient enrollments, leading to $10 million in revenue in September, $24 million in quarter three, and approximately $60 million for the first three quarters of 2026. Our focus is on excellence in execution to further accelerate the U.S. performance and prepare for international launches. Let me conclude with an update on our commercial readiness for pove in IgAN. Reshma's already highlighted the trifecta of efficacy, safety, and patient-centric dosing of povetacicept.

Put simply, with the PDUFA date just days away now, we are 100% ready, and we are incredibly excited to bring pove to patients with IgAN. Our field team is fully hired, trained, and ready to go. Over 90% of the team have deep nephrology experience, and many of them have been engaging with nephrologists in IgAN disease educational activities since early this year. We also know that the patient experience is of supreme importance in biologics, and IgAN is no different. Based on years of providing comprehensive patient support to our CF patients, we have built a people-based and tech-enabled patient support program to facilitate patients to get their pove as fast and as easily as possible. In addition, our payer conversations continue to proceed very well.

Payers understand the significant unmet need, are very familiar with pove's clinical profile, and have responded positively to its differentiation and overall value proposition. We have had many engagements with nephrologists over the last few months, including at the excellent ASN Kidney Week meeting last month. They are looking for treatments that meaningfully and rapidly reduce proteinuria, have a favorable tolerability profile, and offer convenient dosing alongside a seamless treatment experience built on access and patient support. Our discussions with physicians over the last quarter have increasingly revealed the importance of dosing frequency, dose volume and viscosity, and method of administration. We continue to believe that, in addition to an excellent efficacy and safety profile, the low-dose, easy-to-use, at-home auto-injector will be an important differentiator for pove. I will now turn the call over to Charlie.

Thanks, Duncan. Vertex delivered strong third quarter results with total revenue growth of 12% year-over-year, reflecting revenue growth of 10% in the U.S. and 16% internationally. Results include an approximate 1 percentage point favorable impact from foreign exchange and $10 million of revenue from Crinetics following the close of the transaction. Globally, CF revenue grew 8% year-over-year with 5% growth in the U.S. and 12% outside the U.S., or 10% on a constant currency basis. On a non-GAAP basis, third quarter gross margin was 85.6%. On a GAAP basis, gross margin of 84.7% includes intangible asset and inventory step-up amortization resulting from the purchase accounting for the Crinetics acquisition. Turning to Q3 operating expenses, which include approximately $60 million of Crinetics operating and integration-related expenses in September. Third quarter non-GAAP R&D expense was $936 million, an increase of $74 million, or 9% year-over-year.

Approximately $30 million of the increase was attributable to Crinetics. Non-GAAP SG&A expense was $556 million, an increase of $187 million, or 51% year-over-year. Nearly 80% of the incremental spend was to enhance our commercial capabilities. Within the increase in commercial spend, the biggest drivers were pove and renal and JOURNAVX, and then PALSONIFY. Approximately $30 million of the incremental SG&A spend is related to Crinetics. We also recorded $41 million in acquired IPR&D expense in the quarter, including a $28 million upfront payment for new business development agreement with AbCellera. During Q3, we incurred approximately $50 million in non-operating costs associated with the Crinetics transaction financing, including higher interest expense due to the new bank loans and other related costs.

In addition, interest income was $15 million lower than it otherwise would have been as a result of lower cash balances after the acquisition. Our third quarter non-GAAP effective tax rate was 18%, up approximately 40 basis points compared to Q3 2025. Third quarter 2026 non-GAAP net income was $1.21 billion, and third quarter 2026 non-GAAP earnings per share were $4.76. Turning to the balance sheet. We ended the quarter with approximately $9.8 billion in cash and investments following the close of the Crinetics acquisition. Note that we raised $4.5 billion of term loans to fund a portion of the acquisition, currently bearing interest at approximately 4.8%. Even with a busy period for BD, we also deployed approximately $120 million during the quarter to repurchase roughly 250,000 shares. Year-to-date, we have repurchased approximately 2 million shares for $921 million.

Our strategy for capital deployment remains unchanged, with the priority on investing in innovation, including external innovation, and an additional allocation for share repurchases. Now turning to guidance. For total revenue guidance, we are raising our prior range of $13.1 billion-$13.2 billion to a revised range of $13.15 billion-$13.25 billion. The increase reflects the addition of Crinetics, which we expect to contribute $40 million-$50 million to full-year 2026 revenue. The underlying drivers of our revenue outlook are unchanged. Continued CF performance, including the ongoing ALYFTREK launch, together with growing contributions from heme, pain, and now rare endocrinology. We continue to expect more than $500 million in revenue from CASGEVY and JOURNAVX, with additional contribution from four months of PALSONIFY. Consistent with prior guidance, our outlook continues to include an expected full-year benefit from foreign exchange of roughly 150 basis points.

Given our visibility into the drivers of our business, and with just two months of the year remaining, we have a high degree of certainty in our revenue guidance range. We continue to expect full-year gross margin slightly below 86%. On operating expenses, we are revising our prior combined non-GAAP operating expense guidance of $5.65 billion-$5.75 billion to a revised estimate of approximately $6 billion. The standalone Vertex business remains at the high end of our prior range, and the increase in guidance is attributable to the inclusion of approximately $250 million of Crinetics operating expenses and integration-related costs. This updated operating expense guidance reflects continued investment in our late-stage clinical pipeline and the commercial infrastructure supporting our launches and revenue diversification.

For the full year of 2026, we expect total non-operating costs of approximately $105 million related to the financing of Crinetics, including higher interest expense and other related costs. In addition, interest income is projected to be approximately $60 million lower than it otherwise would have been as a result of lower cash balances after the acquisition. Please see slide 23 of the earnings presentation for full details on the impact of Crinetics upon Q3 results and full-year 2026 guidance. We continue to expect our full-year non-GAAP effective tax rate to be between 19.5%-20.5%. In summary, Vertex delivered strong third quarter results with 12% revenue growth and increasing revenue diversification beyond our leading CF business. The integration of Crinetics is well underway, and with the strong commitment of the Crinetics team, we are accelerating commercial, clinical, and valuable preclinical programs.

Our continued leadership in CF, the increasing contribution of CASGEVY and JOURNAVX, momentum in rare endocrinology, and with pove on the doorstep of launch, we are well-positioned for continued attractive growth and profitability for years to come. We look forward to updating you on our continued progress across multiple disease areas with key upcoming milestones detailed on slide 24. I'll now ask Susie to begin the Q&A period.

Thanks, Charlie. Chuck, can you please open it up to questions?

Will do. We will now begin the question-and-answer session. To ask a question, you may press star then one on your touchtone phone. If you are using a speakerphone, please pick up your handset before pressing the keys. If at any time your question has been addressed and you would like to withdraw your question, please press star then two. Today's first question will come from Cory Kasimov with Evercore. Please go ahead.

Great. Good afternoon, guys. Thanks for taking the question. Charlie, congrats on the new role. It's very well deserved. On pove, I wanted to ask about the recent citizen petition that raises the question of whether RAINIER's IgG monitoring and dose hold rules need to be carried into clinical practice. I guess, how frequently do patients actually fall below the IgG threshold or require treatment interruptions, and what do those data support or potentially indicate in terms of monitoring requirements in the commercial label? Thank you.

Hey, Cory, it's Reshma. Let me take that one. With regard to the citizens petition, there are about 200 or so of these that happen every year. About 80% or so of them are from named individuals or companies, and about 20% of them are anonymous. This one, as you know, was anonymous. If the question behind your question is how do we feel about the label, where are our conversations with the agency, everything that you would expect to be happening with the FDA is happening, and we're really looking forward to our PDUFA date. On the specific of how can we think about what values IgG levels can achieve, the bottom line is before this wave of B-cell modulating therapies, the way a lot of folks thought about IgG levels were from congenital diseases of immunologic basis, and that's where a lot of these values came from.

In this day and age, what numbers could IgG levels come to? I think the best examples to look at are the clinical trials. In our example, we studied over 500 people. We measured every four weeks. If the IgG was going to go down, we certainly would have found it because we looked, and we considered a single excursion to be worth noting. I would say the most important point to think about is infections. On that note, there are no opportunistic infections, as you know, in the pove program. The SAEs of infection are balanced. It's exactly 0.5% in pove and 0.5% in the placebo group, and the rest of the infections are mild to moderate. You put this together, and you can see why we're so excited about the PDUFA date.

That's very helpful, Reshma. I appreciate it.

The next question will come from Jessica Fye with JPMorgan. Please go ahead.

Hey, guys. Good afternoon. Thanks for taking my question. Can you help set expectations for what would represent a win coming out of the AMPLITUDE on both proteinuria and eGFR? Thank you.

Yeah. On the AMPLITUDE study, that is the one that is completed full enrollment, and it completed the interim analysis cohort enrollment last year, setting us up for the interim analysis early in 2027. A win would be a statistically significant result on the eGFR slope. I say that because, of course, we are going to measure proteinuria and eGFR, but our agreement with the FDA on what would be acceptable for accelerated approval is eGFR. That is where they are on the disease AMKD. I expect the phase III to look a whole hell of a lot like the phase II in terms of proteinuria, but the key element here is eGFR, and it is a statistically significant difference between the inaxaplin-treated group and the placebo on top of standard of care.

I will not share with you the power analysis and such, but that is what we are looking for because eGFR is what the accelerated approval agreement with the FDA is based on.

The next question will come from Salveen Richter with Goldman Sachs. Please go ahead.

Thank you. Good afternoon. Just a follow-up on Cory and Jess's questions. With regard to pove, can you speak to your comfort and history with the manufacturer? With regard to inaxaplin, can you just walk through further for the reasons for confidence into the 48-week interim data early next year, specifically how we get comfortable thinking about the separation between drug and placebo at that time point, and just the read-through from some of the data sets that you have generated to date?

You bet. Salveen, let's do pove first, and then let's get to inaxaplin. On pove, the specific question you had is, how is it going with manufacturing? We've been with the same manufacturer all through phase III and through to the lead up for our PDUFA date. We feel really good about where we are. As you know, in addition to the review of safety and efficacy clin pharm, there's also review of the CMC manufacturing package, and all of the interactions that you would expect us to be having with the FDA are occurring. Overall, I feel very good about where we are.

Moving then to inaxaplin, but staying within renal, one way to think about the inaxaplin program and GFR, because to date, in both the original phase IIa study that we published in the New England, that was the one that had the 47.6% reduction in proteinuria, and the more recent AMPLIFIED results, which were from the more cohort one, the more modest proteinuria, that one was a 44.7% reduction in UPCR. I think the best way to think about that in terms of GFR, which is the endpoint we've agreed upon with FDA, is in these two ways. One, substantial reductions in proteinuria, in glomerular proteinuric kidney disease, have time and again, been linked to improvements in GFR. That's one general statement. Maybe a second way to look at this is talking about IgA nephropathy.

We used to talk about IgA nephropathy as a fairly indolent disease, but as you can see from the placebo arm of IgAN trials, those patients are losing about 5 ccs per mil per year, right? AMKD is a rapidly progressive disease, so more than that, 6 ccs, 7 ccs, 8 ccs per mil per year. The FDA themselves have said in IgAN that they are going to be comfortable accepting one-year GFR differences. When I put that all together, it adds to my confidence level of what we're going to see with AMPLITUDE. But I'd say those are two ways to think about inaxaplin in AMKD.

Vertex Pharmaceuticals (VRTX) Q3 2026 Earnings Call Transcript

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