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

See which workplaces made this year's list.

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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."

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.

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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."

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

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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."

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>

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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.

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100 Fastest-Growing Companies

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

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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

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Best Workplaces For Women

Industry Vertical

Founded

# Work Sites

Best Companies

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U.S. employees

U.S. job openings

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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)

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Total Stockholder Equity ($M)

Profit Ratios

Profit as % of Revenues

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Earnings Per Share (last Fiscal Year)

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Best Companies To Work For Europe

CEO/Top executive

Group headquarters

HQ location

GPTW national list

Woman CEO

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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

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Q4 2024

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

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Vertex Pharmaceuticals

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Good day, and welcome to the Vertex Pharmaceuticals Second 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 that this event is being recorded. I would now like to turn the conference over to Susie Lisa. Please go ahead, ma'am.

Good evening, My name is Suzie Lisa. And as the Senior Vice President of Investor Relations, it is my pleasure to welcome you to our second 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 Duncan McKechnie, Chief Commercial Officer and Charlie Wagner, Chief Operating and Financial 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, including without limitation, regarding Vertex's marketed medicines for cystic fibrosis, sickle cell disease, beta thalassemia, and moderate to severe acute pain, our pipeline, 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 will now turn the call over to Reshma.

Thanks, Susie. Good evening all and thank you for joining us on the call today. As anticipated, momentum accelerated and we executed with very strong performance across the board, growing and diversifying revenue with multiple new product launches, driving advancement of programs in pivotal development and progressing the earlier stage R and D pipeline. We continue to reach more patients with more products and delivered $2,960,000,000 in revenue in the second quarter, representing 12% growth versus Q2 twenty twenty four. We remain sharply focused on commercialization across multiple disease areas and expansion of our patient reach.

We are pleased with the first six months of launch performance as well as physician and patient feedback on both Elephtrak in CF and GERNAVEX in acute pain. As well as the building global momentum for KASJEBI, our gene edited therapy for sickle cell disease and beta thalassemia. In addition to this commercialization focus, research and clinical progress remain paramount. And to that end, we continue to rapidly advance the four programs in pivotal development and are working with urgency to begin our fifth in primary membranous nephropathy. Before I cover R and D highlights for the quarter, I would like to acknowledge our CSO, David Alschuler's retirement a year from now and the planned CSO transition to Mark Bunnich, our current SVP and Global Head of Research.

David will be retiring from Vertex effective 08/01/2026 after an incredible thirteen year career with the company. David initially joined Vertex as a member of the Board of Directors in 2012 and became Chief Scientific Officer in 2015. During David's tenure, multiple CF medicines have advanced from research through commercialization including ORKAMBI, SYMDEKO, TRIKAFTA and Alluvtrac. Our disease sandbox has broadened and we successfully advanced the scientific breakthroughs that became KASJEVY and JERNAVICS. In an industry with notoriously low R and D success rates, David's teams delivered remarkable achievements year after year.

David is a world renowned physician scientist who's known for his creative and critical thinking, penchant for debate, and commitment to building teams. On a more personal level, I know him as a deeply passionate Vertexian, dedicated physician and valued member of the executive team. As mentioned in our earnings release, as part of this planned transition, I am delighted to announce that Mark Bonage will assume the role of EVP and Chief Scientific Officer effective 02/01/2026, after which David will work with Mark to ensure a smooth transition. Mark joined Vertex and has worked alongside David since 2016. Since that time, Mark has held increasing senior leadership roles across research starting as SVP Site Head Boston Research and most recently as SVP Head of Global Research overseeing all five of Vertex's research sites.

Under Mark's leadership, we advanced many molecules to the clinic including Kaschevy and Gynavix, inaxiplin for AMKD, VX828 for CF, VX670 for DM1 and VX47 for autosomal dominant polycystic kidney disease. Mark was also intimately involved in the diligence that led to the Alpine acquisition, which brought povitacicept to Vertex. I look forward to celebrating David's retirement and welcoming Mark as CSO when the time gets closer. Returning then to R and D highlights. I'll limit my comments to the pipeline programs with the most significant new information to share, specifically CF, pain, type one diabetes and the renal programs.

Starting with CF. As of July, we've gained approval for AlifTrek in The U. S, U. K, EU and Canada. We've also secured reimbursement for AlifTrek in England and will add Ireland shortly.

Patients in Germany and Denmark already have reimbursed access due to existing agreements and provisions. And across other EU member nations in Canada, we are working with reimbursement bodies to secure access for eligible patients as quickly as possible. As we continue to expand our existing CFTR modulator portfolio to younger age groups, we also continue to develop new CFTR regimens with the aim of reaching our long standing objective of bringing most, if not all people with CF to normal levels of CFTR function. Our Next Gen three point zero or NG three point zero regimen is next on deck. The backbone of the NG three point zero combination is VX-eight twenty eight, the most efficacious CFTR corrector that we have ever studied in vitro and brought to the clinic.

We are nearing completion of the healthy volunteer study and we remain on track to initiate a cohort of people with CF with the VX-eight twenty eight regimen before the end of this year. Finally in CF, on VX-five twenty two I am pleased to note that the Data Safety Monitoring Committee has completed its review and is endorsed restarting trial. We're now in the process of working to resume dosing in the MAD portion of the Phase onetwo for the 5,000 or so patients who cannot benefit from our CFTR modulators and expect to do so in the near term. Now shifting to pain. First in peripheral neuropathic pain or PNP.

We had a productive end of phase two meeting with the FDA on our PNP program. While a broad PNP label remains our goal, at this time the FDA does not see a path to a broad indication. As such, we will not be initiating an LSR trial at present. Instead, in light of the discussions and clear agreement with the FDA regarding approval requirements for diabetic peripheral neuropathy or DPN, we will begin a second DPN Phase three study shortly in order to secure DPN as our first PNP indication for sousetrigene. Recall we have breakthrough therapy designation for the DPN indication.

Also note that our first phase three study of sousetrogene in DPN is already well underway And with the near term initiation of the second DPN study, our goal is to complete enrollment of both of these trials by the 2026. We look forward to working with the agency to secure DPN as our first PNP indication, to expand the indication over time and to continue to discuss a potential pathway to a broad PNP label. Turning now to acute pain and VX-nine ninety three, another Nav1.8 inhibitor. This afternoon we shared top line results from the Phase two trial of this molecule in the post bunionectomy setting. To recap, as part of our serial innovation strategy, we developed VX-nine ninety three with three main goals: First, to have an IV option.

Second, to provide additional Nav1.8 inhibitor candidates for potential use as co formulation with future Nav1.7 inhibitors. And third, to further refine dose response relationships including whether higher clinical exposure might result in greater clinical efficacy. Focusing on this last point of efficacy, based on the predicted clinical potency and exposure of nine ninety three, we powered the phase two trial with the goal of detecting a treatment effect higher than previously achieved. This phase two trial included a placebo group, three VX993 dosage arms and a hydrocodone reference arm. The primary endpoint was SPID-forty eight compared to placebo.

The results showed that VX-nine ninety three was safe and well tolerated with no related SAEs and an overall profile consistent with the placebo arm. The placebo effect was well controlled and desired nine ninety three exposures were achieved with adequate separation between doses. On efficacy, VX-nine ninety three did not meet the primary endpoint and did not show statistical significance at the point zero five level. The SPIT-forty eight treatment effect was similar at both the mid and high doses and both were numerically better versus placebo. The outcome of the study combined with the totality of evidence from our preclinical models and previous Nav1.8 inhibitor clinical studies in acute pain suggest we are at the high end of the Nav1.8 dose response curve for acute pain in the post bunionectomy setting.

As such, we do not plan to advance VX993 as monotherapy in acute pain because we do not expect that it will be superior to our Nav1.8 inhibitors. We do plan to complete the ongoing VX993 study of DPN to further define the exposure response relationship and maximal efficacy of Nav1.8 inhibitors in this chronic pain indication. To close out on pain, a quick word on our Nav1.7 inhibitor program. We're very encouraged by our strong preclinical progress in this program and look forward to advancing candidates for use alone or in combination with Nav1.8 inhibitors. Transitioning now to type one diabetes.

Zemila Cell will soon complete enrollment and dosing of its pivotal study, positioning us for global regulatory submissions in 2026, if the data are supportive. Recall we expect about sixty thousand severe type one diabetic patients may potentially benefit from this first Zemila Cell submission. In June at the ADA meeting and concurrently in the New England Journal of Medicine, positive data for Zamyla cell in type one diabetes were presented that continue to demonstrate this cell therapy's transformative potential. All twelve patients with at least one year of follow-up who received a full dose of Zamylacil as a single infusion achieved ADA recommended target hemoglobin A1c levels less than 7%. Freedom from severe hypoglycemic events during the evaluation period, and greater than 70% time in range.

Remarkably, 10 of the 12 patients at twelve months were insulin free, a testament to the potential transformative benefit and durability of this therapy. We also continue to make preclinical progress on our approaches to cloak the same VX-eight 80 cells from the immune system, including improved immunosuppressive regimens, gene editing to produce hypo immune islet cells and novel immunoprotection to encapsulate these cells. We look forward to updating you as these programs advance. Finally, few updates on our kidney portfolio, which now has clinical stage programs in four diseases: IgA nephropathy, AMKD, membranous nephropathy, ADPKD or autosomal dominant kidney disease. Starting with povitacept, a number of autoimmune diseases are driven by uncontrolled B cells.

By controlling B cells, POV is designed to restore immune balance for patients and holds the potential to have transformative benefit across multiple disease states. POV was specifically engineered for better tissue penetration and to deliver optimized targeted dual inhibition of the BAF and APRIL cytokines, which both play a key role in the pathogenesis of B cell mediated autoimmune diseases. First up for POVY is IGAN. We disclosed previously that we completed enrollment of the interim analysis cohort in the RAINIER Phase III trial. Today we are pleased to share that we are on track to complete enrollment of the full RAINIER study by the end of this year.

With regard to the interim analysis cohort, once this group completes thirty six weeks of treatment, we will conduct the interim analysis and if positive will file for potential accelerated approval in The U. S. In the 2026. To close out on IgAN, studies to support the launch of POV for at home self administration with a subcutaneous auto injector are also well underway. Next and consistent with its pipeline and a product potential, the second disease state where we believe POV can provide B cell control is primary membranous nephropathy.

Based on strong emerging data from the RUBI-three study, we completed our end of phase two meeting with the FDA and reached agreement on a phase twothree adaptive study for traditional approval of povi versus standard of care with the primary endpoint of complete remission at seventy two weeks. This Phase twothree study will begin later this year. And third, we have prioritized additional diseases in which we believe povi also holds best in class promise. Based on emerging data, potential patient impact, the treatment landscape and commercial opportunity, the next two autoimmune diseases in focus for us are generalized myasthenia gravis and warm autoimmune hemolytic anemia or WAHA. So to summarize, our current priority disease areas are IgAN, membranous nephropathy, generalized myasthenia gravis and WAHA.

And we are deprioritizing other indications at this time. Next on enaxaplin for APOL1 mediated kidney disease. We remain on track to complete enrollment in the interim analysis cohort of the AMPLITUDE pivotal trial in primary AMKD this year. Of note, while we are able to enroll more adolescent patients, we have hit an important milestone in this study, where the target number of ten-seventeen year old AMKD patients has now been achieved. After completing enrollment in the IA cohort, these patients will be followed for forty eight weeks of treatment, at which point we will conduct the interim analysis and if positive, will be poised to file for potential accelerated approval in The U.

S. The AMPLIFIED study, a phase two proof of concept study in patients with AMKD and comorbidities including type two diabetes is also underway. And this trial is on track to complete enrollment by the 2025. To close on our kidney pipeline is VX407 for ADPKD, a first in class small molecule protein folding corrector that is designed to treat the underlying cause of ADPKD by restoring PC1 protein function, thereby reducing total kidney volume and preventing progression to kidney failure. As a reminder, there are approximately three hundred thousand patients with ADPKD and there are no approved therapies that treat the underlying cause of this disease.

We believe about ten percent of patients with ADPKD may be eligible for treatment with VX407. This quarter we'll begin the VX407 proof of concept trial which is a fifty two week single arm study in twenty four patients that will evaluate the efficacy of VX407 as measured by height adjusted total kidney volume. In closing, Vertex has multiple Phase III programs well underway that are poised for accelerated or traditional approval. These trials have either already completed enrollment or are on track to do so in 2025. They each serve a disease with high unmet need.

And they've secured multiple regulatory designations including Fast Track, Breakthrough, Regenerative Medicine or RMAT, PRIME amongst others. All of which positions us for multiple regulatory submissions in 2026 and early twenty twenty seven with potential approvals and launches to follow. Accordingly, as we drive to achieve our R and D milestones, we're executing on the concurrent work of preparing for commercialization of these potential launches. With that, I'll now turn over the call to Duncan for a commercial update.

Thanks very much Reshma. I will focus my comments tonight on the CF franchise including the launch of ElifTrex in The US, the continuing global launch of Kasjevi and The US launch of Genavix in moderate to severe acute pain. Starting with CF. Our CF franchise continues to deliver strong results as we grow the number of eligible patients taking our CFTR modulators.

We continue to make progress with younger patients, patients with rare mutations and patients in new geographies. The overall market outlook is also supported by the fact that patients are living longer. Now turning to The US launch of AlifTrek, our fifth therapy approved to treat the underlying cause of CF. We believe AlifTrek is the best CF modulator available for eligible patients. As we've discussed on prior calls, versus Trikafta, ElefTrex provides further improvements in CFTR function as measured by sweat chloride, is indicated for additional mutations not covered by the Trikafta label, and offers the convenience of once daily dosing.

In The US, the early launch of ElefTrek is progressing well across all patient groups. We have seen particularly rapid uptake in those patients who are naive to CFTR modulators and thus newly eligible for ElefTrek, as well as those who previously discontinued one of our other CFTR modulators. For patients currently on Trikafta many have been on therapy for multiple years, have experienced incredible clinical benefit and therefore have considerable brand loyalty. In this context the pace of transitions is steady as these patients continue their conversations with physicians and make decisions that balance the short term logistics of augmented liver monitoring with the Liftrec against a lifetime of potential benefits including greater CFTR protein function and once daily dosing. Overall, we're very pleased with the reports of a LiftRec clinical success that physicians and patients are sharing, whether they were naive to a CFTR modulator, are returning to therapy, or have recently transitioned from TRIKAFTA.

We continue to expect the majority of patients who are currently on CFTR modulator therapy will transition to ElefTrek over time given its multiple benefits. We're also now in the process of launching Elefrec in England, following our recent reimbursement agreement with NHS England, as well as in Germany and Denmark. Transitioning to Casjevi, our transformative, one time treatment for patients with sickle cell disease and beta thalassemia. The CASJEVY launch is building momentum and we're pleased with the progress we're making in 2025 as well as the growth it positions us for in 2026. In quarter two, we've seen an acceleration across the board in patient initiations, cell collections and infusions.

This progress is a reflection of the foundational work initiated in 2024 to build our ATC network, expand our geographic footprint to include The Middle East and secure reimbursement for Kashyvi worldwide. Regarding reimbursement, 10 countries now provide access to Kashyvi, including The US and several European and Middle Eastern countries. In terms of our CASJEVY launch metrics, I'm pleased to report that since launch and through the 2025, firstly, we have met our authorised treatment centre goal with more than 75 ATCs now activated globally. We are now dosing patients with sickle cell disease or TDT in multiple regions including The Middle East, Europe and The US. And secondly, nearly two fifty patients have been referred by their physicians to an ATC to initiate the treatment process.

Approximately one hundred and fifteen patients have had their first cell collections, including thirty five first cell collections in quarter two twenty twenty five. And finally, a total of twenty nine patients have completed their treatment journey and received their infusions of CasJevi edited cells, including 16 patients in the 2025. Now shifting to the launch of Genavix in moderate to severe acute pain. Genavix received FDA approval on January 30 and has been available in channel since March. We continue to see a very positive reaction to this novel, non opioid option for the treatment of moderate to severe acute pain.

We're pleased with the rapid pace of payer coverage, P and T committee reviews, formulary adoption, breadth of usage and hospital uptake. We're also very encouraged by the broad range of positive Genavix experiences reported by both physicians and patients. I'll detail several key elements of our ongoing launch. One, we continue to make great progress with payers, which is a testament to their appreciation for the Genavix clinical profile and the importance of offering a novel non opioid option in the treatment of acute pain. As of mid July, across commercial and government payers, approximately one hundred and fifty million lives, or roughly half of all lives covered in The US, have reimbursed access to Genovix.

With commercial payers, our negotiations continue to progress very favourably. We recently reached a formal coverage agreement with a second large national pharmacy benefit manager to make Genavix available to their customers, representing an incremental 22,000,000 commercial lives. As a result, we now have formal coverage from two of the three large national PBMs and anticipate adding the third before year end. In addition, we now have national agreements in place with the two largest hospital group purchasing organisations to make Genavix available to acute pain patients in their network member hospitals. In Medicare, we continue to engage with plans to secure coverage.

And for Medicaid patients, through mid July, we added six state plans since our quarter one earnings call for a total of 16 states with legislation that ensures access is available without prior authorization or step edit requirements. We continue expect the coverage across commercial, Medicare and Medicaid payers will continue to expand through 2025. Two, we are prioritizing the P and T committees at approximately 150 health care systems and roughly 2,000 hospitals. The majority of these 2,000 hospitals ladder up to one of the 150 health care systems. Last quarter we disclosed that more than a third of these target health care systems had taken steps to initiate the P and T review of Genavix.

I'm very happy to report that over a third of these priority target healthcare systems have now added Genavix to their formularies, protocols or order sets and many more have Genavix under active consideration. In addition, at the hospital level, about 500 of the 2,000 hospitals we're targeting have also added Genavix to their formularies, protocols or order sets. Three, prescribing patterns are encouraging for the near and long term outlook for Genavix with excellent breadth of usage to date across a wide range of inpatient and outpatient settings, pain conditions and physician specialties in line with the broad label. Lastly, more than 110,000 prescriptions were successfully filled for Genavix across the retail and hospital settings as of mid July. We remain tremendously excited about our opportunity to transform the treatment of pain and will continue to invest as we see warranted.

Given the rapid contracting and formulary progress we have made, as well as the prescriber and patient feedback, we believe now is the time to make additional investments in our commercial activities behind Genavix. This includes additional marketing activities in Q3 as well as field support as we continue to secure more access and hospital formulary wins over the coming months. We have high confidence that we are in the early days of creating another multibillion dollar franchise for Vertex. To conclude, we are well on our way to establishing a new era of commercial diversification at Vertex as we execute on multiple launches in CF, sickle cell disease, TDT and acute pain and begin the build out for the next wave of launches in a number of disease areas with high unmet need. We look forward to bringing our transformative therapies to millions 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 Q2 twenty twenty five revenue growth accelerated as expected and our results demonstrate our consistent strong performance and attractive growth profile. Second quarter twenty twenty five total revenue increased 12% year over year to $2,960,000,000 U. Revenue growth of 14% year over year was driven in CF by ongoing patient demand and favorable gross to net versus prior year, and also included contributions from CASJEVY, GERNAVIX, and collaboration revenue.

As expected, revenue outside The US rebounded this quarter and was up eight percent year on year, including healthy CF growth and a contribution from CASJEVY. Included in total revenue and the regional growth rates was $30,000,000 of CASJEVY revenue, 12,000,000 from Gernavix, and $21,000,000 of collaboration revenue. Second quarter twenty twenty five combined non GAAP R and D, acquired IPR and D, and SG and A expenses were $1,240,000,000 compared to $5,430,000,000 in the 2024. Excluding ALPN related acquired IPR and D, non GAAP operating expenses increased 24% year on year, driven primarily by the continued 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. Second quarter twenty twenty five acquired IP R and D expenses were $2,000,000 compared to $4,400,000,000 in the 2024, which included the acquisition of Alpine Immune Sciences.

Second quarter twenty twenty five non GAAP operating income was 1,330,000,000.00 compared to a non GAAP operating loss of $3,150,000,000 in the 2024. Second quarter twenty twenty five non GAAP effective tax rate was 19.4%. Second quarter twenty twenty five net income was $1,200,000,000 compared to a net loss of $3,300,000,000 in '24. Second quarter twenty twenty five non GAAP earnings per share were $4.52 compared to a loss per share of $12.83 in the 2024, primarily due to higher revenue and disciplined operating spend as well as the impact of the Alpine AIP R and D expense in the 2024. We ended the quarter with $12,000,000,000 in cash and investments after deploying approximately $395,000,000 to repurchase more than 8 and 65,000 shares in the second quarter.

In May, we announced a new $4,000,000,000 share repurchase program, building upon our existing $3,000,000,000 share repurchase program, which was authorized in 2023 and had $570,000,000 remaining as of June 30. Our priorities for cash deployment remain unchanged: innovation and growth fueled by investment, both internal and external, followed by share repurchases. Now switching to guidance. We are reiterating all elements of our financial guidance, including our 2025 total revenue guidance range of $11.85 to $12,000,000,000 representing growth of approximately eight percent 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 AlifTrex in The U.

S, followed by other regions later this year. Recall that Aliftrec carries a meaningfully lower royalty burden than Trikafta and extends our composition of matter patent protection to 2,039. Revenue guidance also includes Kasgevi revenue, as we treat more patients in geographies where we have secured regulatory approval and reimbursement. Given the duration of the patient journey, we have high visibility into Kasgevi revenue. As patient initiations and cell collections continue to ramp, we expect commensurate increases in infusions, but note that because the timing of infusions is predicated on patient scheduling choices, there may be revenue variability from quarter to quarter.

In addition, guidance reflects additional revenue contribution from Gernavix in the second half due to gains in sustainable payer coverage. Recently announced positive coverage decisions are included in our revenue guidance. Overall, we are confident in our ability to deliver another strong year of revenue growth for Vertex in 2025. We are also reiterating guidance for combined non GAAP R and D, acquired IPR and D and SG and A expenses in a range of 4,900,000,000.0 to $5,000,000,000 for the full year 2025, though we expect to be at the high end of this guidance range. Consistent with prior commentary, this range includes approximately 100,000,000 in projected IP R 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. In addition, given progress with respect to reimbursement and access and the size of the opportunity for Gernavix, as Duncan mentioned, we increasing our investment in marketing and commercial initiatives to support the launch in the second half of this year. We expect an immaterial cost impact from tariffs in 2025 based on what we know today due to our significant US presence and our geographically diverse supply chain. 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 to our expected full year 2025 non GAAP effective tax rate in the range of 20.5% to 21.5%, which implies a higher effective tax rate in the second half of the year.

We do not anticipate recent tax legislation to have a material impact on our expected effective tax rate in 2025. In closing, Vertex yet again delivered strong results in Q2 twenty five, growing and diversifying our revenue with the launch of two new products in The U. S, ElifTrac and Gernavix, continuing the global launch of Kasjevi and making significant pipeline progress across the portfolio. 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.

We will now begin the question and answer session. And your first question today will come from Jessica Fye with JPMorgan. Please go ahead.

Hey, guys. Good afternoon. Thanks for taking my questions. I had a couple on pain. So for the additional commercial efforts behind Geronavix, was that increase always planned as coverage came into place?

Or is that a reaction to what you're seeing as you kind of continue to launch so far? And then for Suzetra gene in DPN, I think you said you'd complete enrollment in both Phase 3s by the 2026. I'm just curious, can we expect enrollment completion for the first Phase three trial much sooner than that? Thank you.

Jess, this is Reshma. Let me split that into two parts. I'll take the second part first and then I'll ask Duncan to comment. Yes, I think that the first DPN might well enroll ahead of the second DPN because it started ahead of time and the enrollment is going very well. I will ask Duncan to comment on GERNAVIX.

As you know, it's really important to secure payer coverage as we ramp commercial efforts behind that. Duncan, any more comments on how you're thinking about commercialization?

Sure. And thank you for the question, Jess. So obviously, we're now about five months or so into the launch and I'd say there's sort of three factors that are really driving our thinking. Firstly, as Reshma just alluded to, we're very pleased with the progress we're making with payer coverage on hospital formularies, particularly considering how lengthy P and T processes can be. Secondly, we're receiving incredibly positive feedback from physicians and patients on how well Genavix is working for them clinically.

And thirdly, we've seen both in the face to face arena with our sales organization as well as in the digital arena, we've seen Genavix to be incredibly promotionally responsive. So based on those three factors, yes, we are thinking that now is the right time to augment our spend in marketing and field support. I would add the last comment that we do anticipate that field support would still sit within the specialty model.

And your next question today will come from Salveen Richter with Goldman Sachs. Please go ahead.

Good afternoon. Thanks for taking my questions. So with regard to the strategy in pain here, can you help us understand now that you won't be moving forward with a broad PNP label today, maybe what your plan is around running these DPN trials and then whether it's taking next generation drugs or so forth to move into LSR or any of the other smaller indications like small fiber neuropathy or some of the others? And then secondly, in light of the Joronevax launch that's progressing here, maybe help us understand how to think about gross to net over the balance of the year and beyond. Thank you.

Sure thing. Sami, let me take those questions. With regard to our plans in PNP, it remains our goal to get a broad PNP indication. Our conversations with the FDA at this recent meeting were very productive. They were open minded.

They were very open to ongoing discussions, but it was equally clear that at this time, they do not see a path to PNP. That's just not where they are. However, we have a clear agreement on DPN. So the first order of business for us is to secure the DPN indication, hence the, start of the second DPN study. We can broaden that indication and we had really good conversations of how that could occur.

Vertex Pharmaceuticals (VRTX) Q2 2025 Earnings Call Transcript

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