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

See which workplaces made this year's list.

2025_GPTW_BiotechnologyPharmaceuticals_Large_Vertex Pharmaceuticals Inc_US1__685d500b023fa8a02634231b_4

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."

Best Large Workplaces in Biotechnology and Pharmaceuticals

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.

GPTW_100BestCompaniesToWorkFor_2025-Vertex-Pharmaceuticals-Inc_US1__20241210214006_2.jpg

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."

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

GPTW_Women_Large_2024-Vertex Pharmaceuticals Inc_US1__20240830224000_5

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."

100 Best Large Workplaces for Women

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.

Great-Places-to-Work-in-Biotechnology-Large-2022-Vertex-Pharmaceuticals-Inc_7007527-6

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 Workplaces For Women

Industry Vertical

Founded

# Work Sites

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

Best Workplaces In Biopharma

Sites

Key Financials (last Fiscal Year)

Revenues ($M)

Profits ($M)

Assets ($M)

Total Stockholder Equity ($M)

Profit Ratios

Profit as % of Revenues

Profits as % of Assets

Profits as % of Stockholder Equity

Earnings Per Share (last Fiscal Year)

Earnings Per Share ($)

EPS % Change (from prior fiscal year)

EPS % Change (5 year annual rate)

EPS % Change (10 year annual rate)

Total Return

Total Return to Investors (last year)

Total Return to Investors (5 year, annualized)

Total Return to Investors (10 year, annualized)

Best Companies To Work For Europe

CEO/Top executive

Group headquarters

HQ location

GPTW national list

Woman CEO

Number of Years on List

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.

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 First Quarter twenty twenty five Earnings Call. All participants will be in a listen only mode. After today's presentation, there will be an opportunity to ask questions. Please note this event is being recorded. I would now like to turn the conference over to Ms.

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 first quarter twenty twenty five financial results conference call. On tonight's call, making prepared remarks, we have Doctor. Reshma Kewalramani, Vertex's CEO and President Stuart Arbuckle, Chief Operating Officer Charlie Wagner, Chief Financial Officer and Duncan McKechnie, SVP, North America Commercial Operations and come July 1, Chief Commercial Officer. 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, without limitation, those regarding Vertex's marketed medicines for cystic fibrosis, sickle cell disease, beta thalassemia, and moderate to severe acute pain are pipelined 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.

Thanks, Susie. Good evening all, and thank you for joining us on the call today. Continuing the momentum from 2024, we've kicked off 2025 with another quarter of strong performance across the board, growing and diversifying revenue as we execute on multiple launches, accelerating programs in pivotal development and advancing the R and D pipeline. We continue to reach more patients with more products and delivered $2,770,000,000 in revenue in the first quarter, representing 3% growth versus Q1 twenty twenty four. This year, we are keenly focused on commercialization, and we are pleased with the early launch dynamics and physician and patient feedback on Alivtrec, our fifth CF medicine, and Gernavix, the first oral non opioid for moderate to severe acute pain in more than two decades, both of which were approved in The U.

S. In just the last few months. With these approvals and the continued global launch of Kashgevi, our gene edited therapy for sickle cell disease in beta thalassemia, we are significantly expanding the number of patients we serve. We are also sharply focused on advancing the four programs currently in pivotal development: sousetrogene in diabetic peripheral neuropathy, zamylosel in type one diabetes, inaxaplin in APOL1 mediated kidney disease, and povi in IgA nephropathy. Importantly, three of these phase three programs are on track to complete enrollment of the interim analysis cohort, or the full study, this year, setting up a series of potential filings in 2026.

And as we approach the one year anniversary of the acquisition of Alpine Immune Sciences, I wanted to highlight two big recent povatacept related milestones. First, we completed enrollment in the interim analysis cohort in the phase three RENEER IGAN trial. And second, we reached agreement with the FDA to advance POV to pivotal development in a second indication, primary membranous nephropathy. This is a notable milestone as POV continues to deliver on its promise as a pipeline and a product with best in class potential. The start of the POBE membranous study will also mark our fifth program in pivotal development.

Tonight, I'll limit my R and D comments on the pipeline programs with the most significant new information to share, specifically CF, pain, type one diabetes, and the kidney programs. Starting with CF. Following our December U. S. Approval of AlifTrex, we have gained MHRA approval in The UK and a positive CHMP opinion in The EU.

As a result, we expect potential approval from the European Commission for a Lift Truck in the second half of this year, along with potential approvals in Canada, Australia, and Switzerland. These approvals are in addition to the European Commission's early April approval of Caf Trio for rare mutations, which followed similar approvals for Trikafta rare mutations in The U. S. And Canada late last year, adding hundreds of additional eligible patients in North America and thousands in Europe. These approvals are a direct result of the team's decades long, painstaking work to establish and verify the hypothesis that the three unique binding sites of our CFTR modulators results in overall protein stabilization and have the potential to transform the lives of nearly ninety five percent of patients with CF.

Stuart will share more on The US Elephtrak launch shortly. Next on the horizon for our CF small molecule program is the next gen or NG3.0 CFTR regimen. With this program, we seek to reach our longstanding goal of bringing most, if not all, patients with CF to normal levels of CFTR function. The backbone of this NG3.0 combination is VX-eight twenty eight, the most efficacious CFTR corrector that we have ever studied in vitro. It is completing Phase I development, and we remain on track to initiate a study with VX-eight twenty eight in patients with CF before the end of this year.

For the ongoing Phase III study of VX-five twenty two for the approximately 5,000 or so patients who cannot benefit from our CFTR modulators, we have recently implemented a temporary pause to the study as we assess a tolerability issue. Given that this remains active clinical trial, we won't be providing any additional details at this time so as to maintain study integrity. We will update you when we know more. Moving next to the pain programs. First, the phase three study of sousetrogene in diabetic peripheral neuropathy, a chronic peripheral neuropathic pain condition that affects over two million Americans annually, is well underway with ongoing enrollment and dosing.

As a reminder, susetralgene has Fast Track designation for peripheral neuropathic pain and breakthrough designation for diabetic peripheral neuropathy. Next, I'm very pleased to share that the study of oral VX-nine ninety three, another Nav1.8 inhibitor, in acute pain post bunionectomy is on track to complete this quarter, and we expect to report results from this trial in the second half of this year. VX-nine ninety three has fast track designation for acute pain in both the oral and IV formulations. Lastly, we continue to make solid progress with additional Nav1.8 inhibitors beyond VX-nine ninety three, as well as in our NAV1.7 pain signal inhibitor program that may be used alone or in combination with NAV1.8 inhibitors. Transitioning now to type one diabetes.

Cimilacel remains on track to complete enrollment and dosing of its pivotal study this quarter, positioning us for global regulatory submissions in 2026, if the data are supportive. Recall, we expect about sixty thousand severe type one diabetics who may potentially benefit from this first zamylosel submission. Based on the high unmet need in T1D and the transformative nature of this therapy, Zamylosel has multiple global regulatory designations, including RMAT and Fast Track in The US, PRIME in The EU, and the Innovation Passport in The UK. In our other T1D work, following the recent data from VX264 or the Cells Plus Device program, we have returned this approach to the research stage. We continue to make preclinical progress on our other approaches to cloak the VX-eight 80 cells from the immune system.

These cells have already demonstrated transformative efficacy. These approaches include alternative immunosuppressive regimens and gene editing to make hypoimmune islet cells, and we look forward to updating you as these programs advance. Finally, a few updates on our kidney portfolio, which now has clinical stage programs in four renal diseases: IgA nephropathy, AMKD, membranous nephropathy, and ADPKD, or autosomal dominant polycystic kidney disease, starting with povitacept, a potential best in class dual antagonist of the BAFF and APRIL cytokines, which play a key role in the pathogenesis of B cell mediated autoimmune diseases. First, in IgAN, as mentioned earlier, I am very pleased to share that we have completed enrollment in the interim analysis cohort of the RAINIER Phase III trial. Once this cohort completes thirty six weeks of treatment, we will conduct the interim analysis, and if positive, it will support filing in the first half of twenty twenty six for potential accelerated approval in The U.

S. In addition, our program to support the launch of PoV with a subcutaneous autoinjector for monthly at home administration is well underway, and for full approval, we are making strong progress towards our goal of enrolling the complete cohort of four eighty patients, in whom we will assess eGFR through week one hundred four. Second, based on the positive results from the RUBI-three basket study, we have reached agreement with the FDA to advance PoV to pivotal development in membranous nephropathy. Beginning in the second half of this year, we are planning to initiate a single phase twothree adaptive study of povy versus standard of care with the primary endpoint of complete remission at week seventy two. Next, two highlights on enaxaplin for APOL1 mediated kidney disease or AMKD.

First, we remain on track to complete enrollment in the interim analysis cohort of the AMPLITUDE pivotal trial this year. AMPLITUDE is a study of primary AMKD, that is to say, patients with two APOL1 variants and no additional renal related comorbidities. After completing enrollment, when this cohort reaches forty eight weeks of treatment, we will conduct an interim analysis. If positive, we will be poised to file for potential accelerated approval in The U. S.

Second, based on the positive proof of concept results of naxaplan in primary AMKD, the momentum in the Phase III study, and interest from the community, we recently initiated the AMPLIFIED study. AMPLIFIED is a phase two proof of concept study of enaxaplin in patients with AMKD and other comorbidities, including type two diabetes. This study is enrolling and dosing patients. To close on our kidney pipeline, a few comments on VX407 in autosomal dominant polycystic kidney disease or ADPKD. VX407 is a first in class small molecule protein folding corrector that is designed to target the underlying cause of ADPKD by restoring PC1 protein function, thereby reducing total kidney volume and preventing progression to kidney failure.

As a reminder, by way of its mechanism of action, VX407 addresses up to ten percent of ADPKD patients, and as in CF, we will seek to expand the eligible patient population with serial innovation over time. We have completed the phase I trial of VX407, and the PK and safety are supportive of advancement. The phase II proof of concept study is designed as a fifty two week, single arm study of 24 patients that will evaluate the efficacy of VX-four zero seven as measured by the height adjusted total kidney volume, and we are on track to initiate this study in the second half of this year. For five years now, at the end of my remarks, I've turned the call over to Stuart. I'll do so for the final time tonight.

Let me acknowledge and thank Stuart once again for the incredible run at Vertex and wish him the very best in retirement. With that, I'll now turn the call over to Stuart and Duncan for a commercial update.

Thanks very much, Reshma. I'll focus my comments tonight on the CF franchise, including the launch of a Lift Truck and the continuing global launch of Kashyvi, building on the foundation we established in 2024. I'll then turn it over to Duncan to provide an update on The US launch of Genavix in acute pain. Starting with CF. With our existing portfolio of CF medicines, Kalydeco, Orkambi, Symdeko, Trikafta, we have continued to grow the number of eligible patients taking our CFTR modulators.

As expected, we also continue to make regulatory and reimbursement progress that enables us to expand to younger patients. KALYDECO is now approved down to one month olds. Patients with rare mutations. Trikafta recently secured US and EMA approvals to expand the label for additional mutations, which means that the triple combination is now approved for mutations present in nearly ninety five percent of all CF patients in our core markets, and patients in new geographies, such as Brazil. In addition, as a result of better patient care, including the availability of our CFTR modulators, people with CF are now living longer than ever before.

Now turning to the Elephthrex launch, our fifth CFTR modulator approved to treat the underlying cause of CF. In phase three studies, when compared head to head with Trikafta, Elephtrex demonstrated non inferiority on lung function and further improvements in CFTR function as measured by sweat chloride. Elyftrex was also approved by the FDA for an additional 31 mutations not covered by the Trikafta label and offers the convenience of once daily dosing. Recall too that Aliftrec carries a meaningfully lower royalty burden for Vertex, and extends our composition of matter patent protection from 2037 for Trikafta into 2039 for Aliftrec. We're pleased with the early US launch progress, and we're seeing uptake in all of the patient groups eligible for Elephtrex.

Those naive to CFTR modulators, or with newly approved rare mutations, where we have seen the fastest initial uptake. Patients who've discontinued one of our other CFTR modulators, and patients switching from Trikafta who seek greater improvement in CFTR function and or the convenience of once daily dosing. Elephantrex prescriptions are off to a strong start, as patients and physicians familiarize themselves with the Elephantrex clinical data, including statistically significant lower sweat chloride than Trikafta, the liver monitoring requirements when initiating therapy, and the convenience of once daily dosing. We continue to expect the majority of patients in The US who are currently on CFTR modulator therapy will switch to Elephtrec over time. We also look forward to launching Elefthrex later this year in The UK and other countries pending ongoing regulatory approvals.

Transitioning now to Kashyvi, our transformative one time treatment for patients with sickle cell disease and beta thalassemia. Since regulatory approvals in late twenty twenty three and early twenty twenty four, the rollout of Kashyvi is progressing as we expected and gathering momentum across all regions. ATC activations and patient initiations continue to increase, as we now have more than 65 authorized treatment centers, nearing our goal to activate approximately 75 total ATCs globally. We're also encouraged to see many ATCs have now collected cells from multiple patients. As to specifics on the other important marker of our progress, since launch, approximately ninety patients have now had their first cell collections, meaning they have begun the patient treatment journey.

Encouragingly, more than twice that number of patients has been referred by their physicians to ATCs to initiate the treatment process. And in Q1, '8 patients completed their treatment journey and received their infusions of Casjevi edited cells. With regard to access and reimbursement, we continue to make progress on the Kashyvi payer front. In The US, formal commercial coverage is either in place or provided through single case agreements. For Medicaid patients, who represent about 45% of total patients, the majority of states have joined the CMMI demonstration project for cell and gene therapy access model, enabling fertility coverage for these patients and providing an alternative, seamless approach for ATCs and states to the existing case by case coverage and state agreements.

In Europe, we have now secured reimbursed access for both sickle cell disease and beta thalassemia patients in England, Wales, Denmark, Austria, and Luxembourg. And in The Middle East, we have reimbursement in Bahrain, Saudi Arabia, and recently added coverage in the majority of Emirates in The UAE. The interest in Kashyvi continues to be incredibly high in the sickle cell disease and beta thalassemia patient and physician communities globally, and uptake is accelerating as access and reimbursement are secured and familiarity with the process for collecting cells and infusing this truly transformative treatment grows. The impact of Kashyavi is best captured by the real world feedback from patients, caregivers, and physicians. It's been inspiring to hear that Kashyavi patients now feel able to live their lives in ways they never have before, Whether that means having the energy to play with their kids, taking up snowboarding without fear that the cold might bring on a pain crisis, or investing in their education and careers, given expectations now for a longer and healthier life.

It is a privilege to be part of their journey. I'll close my comments today by saying what a true honor it has been to serve patients, employees, and shareholders as the chief commercial officer and chief operating officer at Vertex. The company has never been better positioned from a scientific, commercial, financial, or people perspective, and I look forward to following its continued success, including with Duncan as the new chief commercial officer. I'll now hand over to Duncan to provide an update on the latest chapter in our commercial diversification with the exciting launch of Genavix in moderate to severe acute pain.

It has been a privilege to know you for over thirty five years and work with

you at Vertex for the last twelve. I wish you every happiness in your retirement. Genavix received FDA approval on January 30, and has been available at retailers since mid March. Although it's early days, we are seeing for a novel non opioid option for the treatment of moderate to severe acute pain. We are pleased with the early launch, including broad retail pharmacy stocking, progress of reimbursement discussions and payer coverage, P and T committee reviews, the breadth of usage to date, and media coverage.

To give you a sense of Genavix's progress thus far, I'll detail several key elements of our launch plan. One, let me start with retail pharmacy stocking, which is crucial given the acute nature of pain. By mid March, Genavix was available at approximately 33,000 pharmacy locations, including nearly every location for the three largest pharmacy chains in the country, as well as over a dozen regional chains nationwide. Two, I'm also happy to report that we've made rapid progress with payers, which is a testament to their appreciation for the Genavix clinical profile and the importance of a novel non opioid option in the treatment of acute pain. As of May 1, across commercial and government payers, ninety four million lives already have covered access to Genavix, and forty two million have unrestricted access without the need to complete prior authorizations or step edits.

With commercial payers, our negotiations continue to progress favorably. We've recently reached a formal coverage agreement with one of the large national pharmacy benefit managers to make Genavix available to their customers, collectively representing twenty two million commercial lives. In Medicare, we continue to engage with Medicare plans to secure off cycle coverage in 2025 and 2026 coverage in line with Medicare bid cycle timing. For Medicaid patients, 10 state Medicaid plans are now providing unrestricted access to Genovix, meaning no prior authorization or step edit requirements and copays as low as $3 to $5 as is common practice for Medicaid. We expect the coverage across commercial, Medicare and Medicaid payers will expand through 2025.

'3, a key area of focus is hospital P and T committees. As a reminder, we are prioritising approximately 2,000 hospitals, many of which will add up to 150 healthcare systems or integrated delivery networks. More than a third of these target healthcare systems have already taken steps to initiate P and T reviews of Genavix, and some have already added it onto their formulary. Four, turning to patients, our patient support programmes are working as designed, to provide a smooth and positive patient experience so that eligible patients who are prescribed Genavix for their acute pain get access to the medicine for an interim period while payer coverage decisions are made. Five, shifting to the policy landscape.

We are encouraged by the continued momentum and interest by federal and state policymakers to provide equal access to non opioids. The critical need for non opioid options was underscored by the presentation of new research at the American Academy of Pain Medicine in April. Our health economics analysis indicates that replacing just twenty five percent of current acute pain prescriptions for opioids with non opioids, like Genavix, could deliver annual cost savings of $4,500,000,000 to the health care system and could prevent up to two hundred and sixty thousand cases of opioid use disorder and approximately nine thousand overdose deaths over the next fifteen years. With respect to state legislation, to date, nearly 35 states have already either enacted or proposed legislation to support the use of non opioids. At the federal level, I'll highlight the No Pain Act, which in January began providing an add on payment for non opioids used in Medicare patients in the hospital outpatient or ambulatory surgery center settings.

We continue to expect Genavix to be added near term to the list of medicines approved for this add on payment. Lastly, an important indicator of our launch progress and a reflection of the unmet need in moderate to severe acute pain is that more than 20,000 prescriptions were successfully filled for Genavix as of April. While it is early days in the launch, we are very encouraged by the breadth of physician types writing prescriptions for Genavix, as well as the wide range of pain types being treated, which is aligned with Genavix's broad label. We continue to execute on the opportunity to transform the treatment of pain, while also creating another multibillion dollar franchise for Vertex. To conclude, we are in a new era of commercial diversification at Vertex, and we look forward to bringing our transformative therapies to more and more patients and to keeping you updated on our progress.

I'll now turn the call over to Charlie to review the financials.

Thanks, Duncan. Vertex's Q1 twenty twenty five results demonstrate our consistent strong performance and attractive growth profile. First quarter twenty twenty five total revenue increased 3% year over year to $2,770,000,000 U. S. Revenue growth of 9% year over year was driven by ongoing patient demand, higher net realized pricing and the early launch of AlifTrex.

As expected, ex U. S. Revenue in the quarter declined and was down 5% year on year. Recall that outside The U. S, Q1 Twenty Twenty Four benefited from increased channel inventory due to the majority of Russia shipments occurring early in that year.

In Q1 twenty twenty five, Russia revenue was negatively impacted by the availability of an illegal copy product. Excluding the impact of the revenue decline in Russia, ex U. S. CF revenue growth would have increased in the low single digits. Included in Q1 total revenue was $14,000,000 from CASJEVI and $10,000,000 of collaboration revenue.

First quarter twenty twenty five combined non GAAP R and D, acquired IPR and D and SG and A expenses were $1,230,000,000 an increase of 21 compared to $1,020,000,000 in the first quarter of twenty twenty four. The most significant increases in R and D and SG and A expenses versus prior year were due to rapid advancement of our broad pipeline, including clinical trials for IGAN, pain and type one diabetes, as well as the build out of commercial capabilities in pain. First quarter twenty twenty five non GAAP acquired IPR and D expenses were $20,000,000 compared to $77,000,000 in the first quarter of twenty twenty four. First quarter '20 '20 '5 non GAAP operating income was $1,180,000,000 compared to $1,340,000,000 in non GAAP operating income in the first quarter of twenty twenty four. First quarter '20 '20 '5 non GAAP effective tax rate was 18.8%.

First quarter twenty twenty five non GAAP earnings per share were $4.06 compared to $4.76 in the first quarter of twenty twenty four, primarily due to increased operating expenses as well as lower interest income. We ended the quarter with $11,400,000,000 in cash and investments after deploying approximately $425,000,000 to repurchase more than 930,000 shares in the first quarter. Overall, our priorities for cash deployment remain unchanged. Now switching to guidance. Given the strong start and clear line of sight to the balance of the year, we are raising the low end of our 2025 total revenue guidance from $11,750,000,000 to a revised range of 11,850,000,000.00 to $12,000,000,000 representing growth of approximately 8% at the midpoint at current exchange rates.

This outlook reflects our expectation for continued growth from our portfolio of CF medicines, including the ongoing launch of AlifTrek in The U. S. Followed by other regions later this year. We believe the illegal copy issue is isolated to Russia and is fully included in our outlook. Guidance also includes a continued ramp up in CASJEVI revenue, as we treat more patients in geographies where we have secured regulatory approval and reimbursement.

In addition, guidance reflects a revenue contribution from Gernavix primarily in the second half of twenty twenty five. As a reminder, we expect volumes will ramp ahead of revenue due to financial assistance programs that are designed to provide eligible patients with immediate access while we work to secure broad sustainable payer coverage. Recently announced positive coverage decisions are included in our revenue guidance. As a result of these positive trends and as implied in our guidance range, we expect growth to accelerate over the remainder of the year, delivering another strong year for Vertex in 2025. For combined non GAAP R and D, acquired IPR and D expenses and SG and A, there is no change to our guidance range of 4,900,000,000.0 to $5,000,000,000 for the full year 2025.

Consistent with prior guidance, this includes approximately $100,000,000 in projected IPR and D charges. We will continue to invest a majority of our operating expenses into R and D, given the momentum in our multiple mid and late stage clinical development programs with four and soon to be five Phase III studies ongoing and multiple Phase IIs. The planned increase in commercial costs in 2025 supports our increasingly diversified commercial portfolio, a full year of investments to support the launch of DERNAVIX and potential near term launches. Given our differentiated business model and focus on specialty markets, we can make these targeted investments while maintaining attractive profitability and cash flow. We expect an immaterial cost impact from tariffs based on what we know today due to our low exposure to China and a geographically diverse supply chain.

Additionally, much of our intellectual property is either in The U. S. Or The UK. Of course, given the dynamic nature of the tariff situation, including the potential for sector specific tariffs, this outlook is subject to change. And finally on guidance, there is no change expected full year 2025 non GAAP effective tax rate in the range of 20.5% to 21.5%.

In closing, Vertex yet again delivered strong results in line with our expectations in Q1 twenty twenty five, growing and diversifying our revenue with the launch of two new products in The U. S, AlifTrac and Gernavix, continuing the global launch of KASJEVY and making significant pipeline progress across the portfolio. In addition, we now have five programs that are in Phase III or soon will be, and multiple additional programs with first in class and or best in class potential in the clinic in our early and mid stage pipeline. These and other anticipated milestones of continued progress in multiple disease areas are detailed on slide 17. We look forward to updating you on our progress on future calls.

Before turning the call to Susie to begin the Q and A period, let me also add my thanks and congratulations to Stuart. Stuart is a talented executive and team player who has contributed enormously to Vertex. He's also a friend. As Stuart passes the baton, we look forward to welcoming Duncan to the executive team and to future earnings calls. I'll now ask Susie to begin the Q and A.

Thanks, Charlie. Chuck, can you get us started, please?

We will now begin the question and answer session. And the first question will come from Geoff Meacham with Citibank. Please go ahead.

Afternoon, guys. Thanks for the question. Stuart, congrats again on the retirement, Duncan, looking forward to working together. I just had a couple of quick ones. One on Leaf Trek on the launch.

What's been the feedback on utilizing sweat chloride as a biomarker more in practice? I wasn't sure if the conversations that you guys are having with pulmonologists on switching from TRIKAFTA is more related to the dosing differential or the sweat chloride or maybe a combination? And then just on GERNAVICS, to get an update, maybe give us a sense for how you're thinking about the chronic pain indications and the design of those studies? I know you haven't had full FDA discussions, but as you look at the prior data, maybe give us a sense for where you are with implementing maybe more novel strategies for that study. Thank you.

Sure thing. Hey, Jeff, it's Rationale. Let me start with the Geronavix and chronic pain work and then I'll turn it over to Stuart to talk Elliptrex. Really no new news to report to you, Jeff. We're on track to have our end of Phase two meeting with the FDA this summer.

I really like the study designs the team has come up with. We've done a

work on innovating and optimizing the clinical trial designs. And I do expect that we'll have an update for you this summer. Separately, but on a related note, the DPN study, the diabetic peripheral neuropathy study with the X-five forty eight is well underway. Enrollment dosing in that study is going well. Stuart, Olyphtrex?

Yes, Jeff. On Olyftrex, I would say sweat chloride is not routinely used in clinical practice to assess CFTR function, although it's well understood as a measure of CFTR function, but it's not something that's being increasingly adopted in clinical practice despite the Elliptrex data. And in terms of what do people find compelling about the Elliptrex profile, would say it's really the sum of the parts, Jeff. So it's firstly, the fact that it's demonstrated in large, long, robust clinical trials that it's non inferior to TRIKAFTA, which as we all know sets a very high bar in terms of FEV1. And then it is the improvements in CFTR function that people have the potential to access as measured by sweat chloride.

Then reinforcing that improved potential efficacy, it's the additional 31 mutations in the Elephantrex label that the FDA granted us versus the TRIKAFTA label. And then lastly, but still very importantly for CF patients, it's the once a day versus the twice a day for TRIKAFTA. So I wouldn't say it's one thing more than the others. It's really that combination of benefits that I think people are finding attractive about the Elephtrak profile.

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

Hey, guys. Good afternoon. Thanks for taking my questions. Stuart, congrats again. First, when you maybe for Charlie, when you said you expect immaterial impact from tariffs, just to confirm, is that from the current tariffs?

Or is that if biopharma specific tariffs are implemented? And maybe just for our comfort, can you give us a little more detail on Vertex's manufacturing footprint, particularly for LiftTrak, Trikafta and DraNavix? And how you think about the company's potential exposure to tariffs if they are implemented? And then just separately on the CF business, can you recap how we should think about the impact of the Russia issue and just lapping last year's sales that included Russia? Is that impact largely contained to 1Q?

Or if it also impacts 2Q in subsequent quarters, can you just quantify what that headwind is? Thank you. Charlie?

Vertex Pharmaceuticals (VRTX) Q1 2025 Earnings Call Transcript

Company Participants