Vertex Pharmaceuticals (NASDAQ:VRTX) held its first-quarter earnings conference call on Monday. Below is the complete transcript from the call.
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Summary
Vertex Pharmaceuticals reported Q1 2026 total product revenue of $2.99 billion, reflecting an 8% year-over-year increase, driven by growth across its portfolio, particularly from new disease areas.
The company’s strategic focus includes expanding its cystic fibrosis (CF) treatments, progressing its nephrology franchise, and advancing its pipeline with multiple regulatory submissions and trials in areas such as sickle cell disease and beta thalassemia.
Vertex Pharmaceuticals reiterated its 2026 revenue guidance of $12.95 to $13.1 billion, with significant contributions expected from non-CF products like Casgevy and Journavix.
Operational highlights include the rapid regulatory submission for Povi in IGAN and label expansions for ALIFTREC and Trikaftor, enhancing patient eligibility and market reach.
Management emphasized the potential for its renal franchise to rival CF in size, with promising interim results for Povi in IGAN and planned studies in other B-cell mediated diseases.
Full Transcript
OPERATOR
Good day and welcome to the Vertex Pharmaceuticals first quarter 2026 earnings call. All participants will be in a listen only mode. Should you need assistance, please signal conference specialist by pressing the star key followed by zero. After today’s presentation there will be an opportunity to ask questions. Please note this event is being recorded. I would now like to turn the conference over to Ms. Susie Lisa. Please go ahead.
Susie Lisa (Senior Vice President of Investor Relations)
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 2026 financial results conference call. On tonight’s call, making prepared Remarks, we have Dr. Reshma Kewalramani, Vertex’s CEO and President, Charlie Wagner, Chief Operating Officer and Chief Financial officer and Duncan McKechnie, chief commercial officer. We recommend that you access the webcast slides as you listen to this call. The call is being recorded and a replay will be available on our website. We will make forward looking statements on this call that are subject to the risks and uncertainties discussed in detail in today’s press release and in our filings with the securities and Exchange Commission. These statements including without limitation, those regarding Vertex’s marketed medicines for cystic fibrosis, sickle cell disease, beta thalassemia 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’ll now turn the call over to Reshma. Thanks Suzy. Good evening all and thank you for joining us on the call today. Vertex is off to a terrific start in 2026, which we see as a year defined by execution. Q1 revenue growth was strong across the portfolio as we reach more patients with more products and delivered total product revenue of 2.99 billion, reflecting 8% growth year on year. Importantly, we achieved key commercial milestones for each of the newer products since launch through end of Q1, Aliftrek exceeded 1 billion in cumulative revenue, more than 500 people have initiated their Kashgevi treatment journey and over 1 million prescriptions have been written for Journavix. Another highlight in Q1 was that products from the new disease areas, namely Kashevi and Journavix, drove approximately 25% of total product revenue growth. Execution in R and D was equally strong with multiple regulatory submissions recently completed and more anticipated. Combined with rapid progress across clinical trials and important advancement in research, let me spotlight a few accomplishments. First on POVI. The interim analysis results from the Phase 3 Rainier study in IGAN on efficacy and safety from top to bottom were sparkling and and further fueled our enthusiasm for POVI as a potentially best in class BAF April inhibitor. I was exceptionally pleased with the rapidity and quality of the recently submitted BLA filing for POVI in Igan. Indeed, at 27 days from database lock to regulatory submission, this was the fastest submission in Vertex history. Equally notable is the urgency with which the POVI primary membranous nephropathy and the POVI Myasthenia gravis programs are advancing in Membranous. The Phase two study has been fully enrolled and the Phase three program has already initiated. In addition, the Phase two Proof of concept Myasthenia gravis trial is underway. Second on Kasgevi I’m also very pleased with the rapidity and quality of this SBLA submission for Kashgevy in 5 to 11 year olds with sickle cell disease or beta thalassemia. The Kashgevi filing has been granted a Commissioner’s National Priority Voucher Review reflecting the importance of treating this younger age group before some of the most serious complications of the disease can begin. Overall, Vertex continues to extend its leadership in cf, drive growth with new product launches while building out our next disease area franchise in nephrology, accelerate programs in mid and late stage development and advance the earlier stage R and D pipeline. Tonight I’ll limit my R and D comments to CF as well as the pipeline programs with the most significant new information to share. Certain renal programs POVIne Myasthenia gravis and zamilacel in type 1 diabetes starting with CF.4 quick R& D updates for this quarter we recently reached a significant milestone in the US with label expansions for both ALIFTREC and trikaftor. With this expansion, patients with a clinical diagnosis of CF who have at least one variant in the CFTR gene they that is responsive based on clinical and or in vitro data are now covered by the ALIFTREC and Trikafta labels, reinforcing the impact of these medicines regardless of the location of the variant in the CFTR protein. This is a significant expansion of eligibility that reflects decades of investment, effort and a relentless pursuit of the science. It is also a great example of innovation using using results from clinical trials complemented by in vitro data to expand the benefit of Vertex CFTR modulators to about 95% of people with CF, including those with rare and even N of 1 genotypes. As we expand the ALIFTREC and Trikafta labels to additional mutations. We’re also expanding the labels to younger patients. We will soon submit for approval for ALIFTREC in patients 2 to 5 years of age, where you may recall our pivotal trial demonstrated of a remarkable 65% of children reaching normal levels of CFTR function, and we also plan to submit for Trikafta in children 1 to 2 years of age in the near term. In addition, we continue to advance our Next Generation 3.0 CFTR modulators, including VX828, which is currently in a study of patients with CF. We are on track to complete the study and share results in the second half of this year. Following closely behind VX828 in the family of Next Gen 3.0 are VX581 and VX272, both of which are currently in the clinic in Phase 1 Healthy Volunteer Studies. As we have consistently said, if it is possible to do better in cf, we’re committed to being the ones who do so. And finally, on VX522, the MRNA therapy we’ve been developing for people who produce no CFTR protein and therefore cannot benefit from our modulators. We we previously disclosed tolerability issues in this program. Despite actions we have taken in the trial to overcome these issues, we have not been able to do so and as such we have chosen to discontinue the program. Given this early termination, we will not be able to assess the efficacy or full safety of VX522. We will be working with CITES to close out the study in the coming weeks, moving on to our renal franchise, which continues to make quick progress and is rapidly establishing itself as Vertex’s fourth franchise along cf, heme and pain. In total, we have four programs in mid and late stage development in renal POVI in Igan, POVI in primary membranous nephropathy, Enoxaplin in AMKD and VX407 in 80 PKD. Tonight I’ll cover the first three programs starting with POVI and Igan recall. POVI’s differentiated potential best in Class profile stems from its specific design as an engineered tachy fusion protein with binding affinity, potency and PK properties that deliver optimal dual BAF April inhibition. The dual inhibition and engineering advantage is evident in both the interim analysis data of the Rainier study where we saw rapid, deep and sustained improvement in proteinuria, a favorable safety profile and consistency across all subgroups, as well as in three Key patient dosing benefits once monthly dosing, small volume and subcutaneous administration via an auto injector. Overall, the phase three interim analysis data represent a home run in terms of study design, execution and the results with POVEY achieving statistically significant and clinically meaningful results across all primary and secondary endpoints. Patients in this trial received excellent standard of care with high rates of background medicines including the highest rates of of SGLT2s seen in any IGAN study. Baseline characteristics were well matched to real world IGAN patients in terms of age, renal function and degree of proteinuria. In addition, as a measure of study quality, it’s important to look at discontinuations. In this study, treatment discontinuations were low and trial discontinuations were even lower at a rate of 1.5% in the placebo group and 0.8% in the POVI group. To replay the top line primary and secondary efficacy results for the primary endpoint, POVI achieved a 52% reduction from baseline in peritoneuria as measured by 24 hour UPCR. That’s a 49.8% reduction versus placebo. For the first secondary endpoint, POVI treatment led to a 77.4% reduction from baseline in serum GDIGA1 levels. That’s a 79.3% reduction versus placebo for the second secondary endpoint. Of those patients with hematuria at baseline, 85.1% of POVI treated patients achieved hematuria resolution which is a 61.7% reduction versus placebo. In addition, 42.2% of patients reach the exploratory endpoint of 24 hour UPCR of less than 0.5 grams per gram, an important clinical threshold. These are remarkable results and particularly noteworthy considering that at the time of the interim analysis, patients had received just 36 weeks of POV treatment. On safety, POV was generally safe and well tolerated. The majority of adverse events were mild to moderate and there were no serious adverse events related to pov. Importantly, in terms of infections, most were mild to moderate. The rate of SAEs of infection was low at 0.5% observed in both the placebo and POV groups. There were no opportunistic infections and no discontinuations related to POV overall, including no discontinuations due to infections. Lastly, on antidrug antibodies or adas, adas were observed as expected with biologics but had no impact on povi’s efficacy or risk profile. We look forward to sharing more details of the interim analysis results and anticipate doing so at upcoming medical meetings this fall. Shifting to POVI and primary membranous nephropathy I am pleased to share we have completed enrollment of the Phase 2 portion of the Olympus Phase 2.3study and have already initiated the Phase 3 portion ahead of our previously announced Mid2026 goal and finally on POVI as part of its pipeline in the product potential for B cell mediated diseases beyond renal I’m also pleased to share that the Phase two proof of concept study of POVI in generalized myasthenia is underway. This is a 30 patient study of people with GMG evaluating both the 80 and 240 mg dose for 12 weeks with the primary endpoints of safety and the percent change from baseline in IgG at week 12. The rationale for studying POV and myasthenia is compelling and it’s a serious B cell mediated disease with high morbidity affecting approximately 175,000 people in the US and Europe. There is high unmet need as current therapies have meaningful limitations, which means there’s room for improved efficacy, a better benefit risk profile and more patient friendly dosing and administration, which we have discussed in the context of IGAN as being critically important when considering a chronic biologics market. We believe povi’s mechanism of action, striking at the heart of autoantibody production with an engineered protein format provides best in class promise in myasthenia and we are excited to develop this opportunity. Shifting back to renal to finish up with enoxaplin in Apol1 mediated kidney disease or AMKD. First on amplitude, the pivotal phase 3 study of primary AMKD, that is to say patients with two Apol1 variants for nurture kidney disease and no other renal related comorbidities. We are on track to conduct the interim analysis which occurs after 48 weeks of treatment, and to share data from this Cohort in early 2027. If positive, we will be poised to file for potential accelerated approval in the US thereafter. Second, on amplified our Phase 2b study of enoxapline in separate populations, patients with 2 APOL1 variants, modest peritoneuria and no other kidney disease and patients with two APOL1 variants, moderate to severe peritoneuria and a second disease, type 2 diabetes that could impact the kidney. These two populations are not being studied in amplitude. We recently completed enrollment in the Amplified study which is a study of 13 weeks in duration. Given the clear differences in these populations, we made the decision early on to study them in separate trials. Emerging data in the field confirm the wisdom of this decision. We are excited to learn from the Amplified study and look forward to sharing results in the second half of this year. Finally, on type 1 diabetes, a reminder that Zamylacel has very strong clinical results to date, as detailed in last year’s New England Journal of Medicine. Among patients who received a full dose and had at least one year of follow up, 10 out of 12 patients who were insulin free. These results are unprecedented and are particularly noteworthy given that these patients are those with 20 plus years of type 1 diabetes, undetectable endogenous insulin production at baseline, taking 40 plus units of exogenous insulin per day and with two or more severe hypoglycemic events per year despite best available care. You may recall that in the second half of last year we paused dosing of the phase 1, 2, 3 study in order to conduct a manufacturing analysis which we have now completed. I am pleased to report that dosing in the study has resumed and multiple patients have been dosed with dosing now restarted. We will update you in the coming months on the revised timelines for study completion and regulatory filings. With that, I’ll turn the call over to Duncan for for a commercial update.
Reshma Kewalramani
Thanks very much Reshma. I’ll start with cf, which continues to perform very well year over year. Revenue growth was 6% globally, balanced nicely between US growth of 5% and international growth of 8% in quarter one. Global growth reflects continued ALIFTREC uptake as its once daily dosing and improved sweat chloride profile continue to resonate with the clinical and patient communities. As mentioned, a Liftrek has now surpassed $1 billion in cumulative global revenue since its approval in the U.S. in late December 2024 and Europe in July 2025. Outside the U.S. we have signed reimbursement agreements in 11 countries for ALIFTREC in quarter one alone, building on the access generated in the second half of last year. The tremendous scientific and regulatory achievements represented by the label expansions for elliftrek and Trikafta also also represent a meaningful incremental commercial opportunity of approximately 800 people in CF who are newly eligible in the U.S. this broad labeling is one of several key CF growth drivers for the remainder of 2026, along with the global rollout of a LiftRec treating younger patients and expanding into additional geographies. We have worked closely with the CF population for two decades and remain focused on continuing to serve the CF community and and expand our leadership across all genotypes, age groups and geographies shifting to heme. The rollout of Kashgevi continues to gather momentum across all three regions and I’m pleased to highlight another significant commercial milestone. Since launch, over 500 patients have now initiated the Kashgevi treatment journey. Hundreds have had their first cell collection and many patients have had their cells edited and are ready for infusion. During the first quarter we delivered $43 million in Kashgevi revenue. Importantly, we worked on securing a pricing agreement for Kashgevi in Germany in quarter one and are currently working through the implementation steps. This is a historic moment and we’re excited that German patients with sickle cell disease and TDT may soon be benefiting from long term access to Kashgevi at a sustainable price. Overall, we are very encouraged by the robust flow of patients in the US in in Europe and the Middle east moving from referral to cell collection and infusion. First quarter revenue reflects expected variability quarter to quarter as patients choose the timing for their infusion that suits them best for the full year 2026. The Kashgevi Outlook is very promising as we have built our ATC network, secured reimbursements …
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