VRTX

- Vertex is on track for the U.S. FDA Prescription Drug User Fee Act (PDUFA) date of November 30 for potential accelerated approval of povetacicept – - Data published in NEJM and presented at ASN highlight povetacicept's unique trifecta of efficacy, favorable safety, and the simplicity of dosing at-home every 4 weeks with a low-volume autoinjector – - Data presented at ASN include a qualitative study examining patient perspectives on current and emerging IgAN therapies, showing that 96% of participants preferred an autoinjector over a prefilled syringe, and 80% of participants preferred dosing every 4 weeks versus weekly dosing - - New clinical studies of povetacicept underway including a study evaluating patient experience on povetacicept administered via autoinjector - BOSTON, October 23, 2026--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the publication of Phase 3 RAINIER results for povetacicept in the New England Journal of Medicine (NEJM), presentation of these data at the American Society of Nephrology (ASN) Kidney Week 2026 in Denver, Colorado, and advancement of the povetacicept clinical research program in IgAN. Vertex will also host an investor event during ASN on October 23 at 6:15-7 p.m. MDT (8:15-9 p.m. EDT). Further details are below. Data being presented include a pre-specified Week 36 interim analysis of the ongoing Phase 3 RAINIER trial of povetacicept, an engineered fusion protein and dual inhibitor of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, in immunoglobulin A nephropathy (IgAN). RAINIER met its primary endpoint, with povetacicept achieving a 52.0% reduction from baseline in proteinuria at Week 36 and a statistically significant and clinically meaningful 49.8% reduction versus placebo (P<0.0001), while also meeting both secondary endpoints. The Week 36 interim analysis of RAINIER demonstrated that the depth of proteinuria reduction from baseline was consistent across demographic, clinical and regional subgroups, including an unprecedented 54.2% reduction in urine protein to creatinine ratio (UPCR) versus placebo in North America. Additionally, findings showed that 42.2% of povetacicept-treated participants reached 24-hour UPCR <0.5 g/g, the threshold aligned with current Kidney Disease: Improving Global Outcomes (KDIGO) guidelines, compared with only 6.2% on placebo. Importantly, these findings were observed on top of optimized supportive care in both treatment arms, including sodium-glucose co-transporter 2 (SGLT2) inhibitor use in 65.8% of participants, which is among the highest reported of any contemporary IgAN trial. "At just 36 weeks of treatment, povetacicept has delivered impressive efficacy results, together with favorable safety and convenient dosing at-home every 4 weeks in a low-volume auto-injector that can fit into the lives of IgAN patients, underscoring the potential of povetacicept to substantially impact and redefine expectations for disease control in IgAN," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "We are grateful to the patients, investigators, and study teams who made RAINIER the largest and fastest-enrolling contemporary IgAN trial to date and look forward to the November 30th PDUFA target action date." "What stands out from the RAINIER results is the important clinical validation of simultaneously targeting BAFF and APRIL to address upstream drivers and kidney injury in IgA nephropathy. Povetacicept was specifically designed as a high-potency, high-affinity TACI fusion protein with broad tissue distribution to deliver this dual inhibition in order to target upstream pathogenic immune complex production," said Richard Lafayette, M.D., Professor of Medicine (Nephrology) at Stanford University Medical Center and RAINIER Steering Committee Co-Chair, who presented the data at ASN Kidney Week. "The results observed with povetacicept are among the most compelling I have seen in IgAN and highlight the potential of povetacicept to become an important new treatment option for patients living with this chronic progressive disease." Vertex also presented findings from a qualitative study examining patient perspectives on the device design and administration of current and emerging IgAN therapies, "Patient Perceptions of Injectable Therapies Among Adults with IgAN: A Qualitative Study" (#TH-PO0473). The study included in-depth, one-on-one interviews with 25 U.S. adults living with IgAN. Participants were shown pictures and short videos of the devices and asked to assess key treatment administration-related attributes. Findings showed that 96% of participants (24 of 25) preferred the characteristics of an autoinjector over a prefilled syringe, citing features such as the needle not being visible and elimination of manual needle insertion. Additionally, 80% of patients (20 of 25) preferred dosing every 4 weeks versus weekly dosing, with the top reasons being convenience and travel or time away from home considerations. Vertex also announced a new clinical trial underway for povetacicept: - PAVANT IgAN Patient Experience Study (NCT07890909), A Phase 3b, Open-label, Single-arm, Patient Experience Study of Povetacicept Injection (via Autoinjector) in Adults With Immunoglobulin A Nephropathy Visit news.vrtx.com/kidney-congress for more information about Vertex's presence at ASN Kidney Week 2026. Investor Event Vertex will host an investor event and webcast on October 23 at 6:15-7 p.m. MDT (8:15-9 p.m. EDT) in Denver, Colorado, to discuss the data for povetacicept in IgAN, the Company's program in APOL1- mediated kidney disease (AMKD), and other highlights across its kidney disease portfolio. A live webcast of the presentation and Q&A portions can be accessed through the Investor Relations section of Vertex's website at investors.vrtx.com. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website in the "Investors" section. About RAINIER RAINIER (NCT06564142) is a global Phase 3 randomized, double-blind, placebo-controlled pivotal trial of povetacicept 80 mg administered subcutaneously every 4 weeks versus placebo on top of standard of care in 605 adults with IgAN (N=557 in main cohort, N=48 in the exploratory cohort). The trial was designed to have a pre-planned interim analysis evaluating the percent change from baseline in UPCR for the povetacicept arm versus placebo after a pre-specified number of patients reach 36 weeks of treatment. Final analysis will occur at two years of treatment, with a primary endpoint of total estimated glomerular filtration rate (eGFR) slope through Week 104. RAINIER is the largest trial conducted in IgAN and achieved full enrollment faster than any contemporary IgAN trial. As previously reported, patients treated with povetacicept achieved a 52.0% reduction from baseline in 24-hour UPCR at Week 36, with a statistically significant and clinically meaningful reduction of 49.8% in 24-hour UPCR compared to placebo (P<0.0001). The trial also met its secondary objective. For the first secondary endpoint, patients treated with povetacicept demonstrated a 77.4% reduction from baseline in serum galactose deficient IgA1 (Gd-IgA1) compared to an increase of +9.1% in the placebo group, yielding a reduction of 79.3% compared to placebo (P<0.0001). For the second secondary endpoint, in patients with baseline hematuria, 85.1% achieved hematuria resolution in the povetacicept treatment group compared to 23.4% in the placebo group, resulting in hematuria resolution of 61.7% compared to placebo (P<0.0001). Povetacicept was generally safe and well tolerated. The majority of adverse events (AEs) were mild to moderate. The most common events (≥10% of participants in either treatment group) for the povetacicept group versus the placebo group were upper respiratory tract infection (18.3% vs 11.4%), nasopharyngitis (9.7% vs 12.4%), and injection site reactions (14.5% vs 2.2%). There were no serious adverse events (SAEs) related to povetacicept and no deaths in the trial. There were no opportunistic infections, and no discontinuations due to infections. As expected, anti-drug antibodies (ADAs) were observed; these ADAs had no impact on efficacy or the risk profile. Results from the RAINIER trial support the Biologics License Application (BLA) for povetacicept, which has been accepted by the U.S. Food and Drug Administration (FDA) for accelerated approval in adults with IgAN. The Prescription Drug User Fee Act (PDUFA) target action date is November 30, 2026. The RAINIER trial continues in a blinded manner until the last patient enrolled completes 104 weeks. Patients in RAINIER who have completed the Week 104 visit are eligible to enroll in the open-label extension study (NCT07807995), "A Phase 3 Open-Label Study of Long-term Treatment With Povetacicept in Adults With Immunoglobulin A Nephropathy," and receive an additional 104 weeks of treatment and follow-up. About Povetacicept Povetacicept is a dual inhibitor of the BAFF and APRIL cytokines, which promote B cell activation, differentiation and/or survival, and provides B cell control by inhibiting the ability of BAFF and APRIL to drive the pathogenesis of multiple immune-mediated diseases. Due to its engineered TACI domain, povetacicept has demonstrated improved binding affinity, potency, pharmacokinetics, and/or tissue distribution compared to other APRIL, BAFF, and dual BAFF+APRIL inhibitors in preclinical studies. If povetacicept is approved by the FDA, Vertex plans to launch povetacicept as a low-volume (<0.5 mL) subcutaneous autoinjector delivered once every 4 weeks via at-home administration. IgAN is the first of multiple indications that Vertex is exploring for povetacicept given its pipeline-in-a-product potential. There are ongoing studies including the Phase 2/3 OLYMPUS trial (NCT07204275) in primary membranous nephropathy (pMN) and the Phase 2 ETNA trial (NCT07501702) in generalized myasthenia gravis (gMG). About IgA Nephropathy (IgAN) IgAN is a serious, progressive, life-threatening kidney disease driven by uncontrolled autoreactive B cell activity and is the most common cause of primary glomerulonephritis, affecting approximately 330,000 people in the United States and Europe and more than 1.5 million globally. IgAN results from the deposition of circulating immune complexes, consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1), in the renal glomerular mesangium, triggering kidney injury and fibrosis. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia, acute pain and acromegaly, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, congenital adrenal hyperplasia, adrenocorticotropic hormone (ACTH)-dependent Cushing's syndrome, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 17 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements made by Carmen Bozic, M.D., and Richard Lafayette, M.D., and statements regarding expectations for the potential accelerated approval of povetacicept in the U.S., expectations for new clinical trials planned for povetacicept, expectations for the RAINIER trial, expectations for the launch of povetacicept if approved by the FDA, expectations for the anticipated benefits of povetacicept, including its best-in-class potential, and the clinical status of and expectations for the Phase 2/3 OLYMPUS trial in pMN and the Phase 2 ETNA trial in gMG. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's clinical programs may not support registration or further development in a timely manner, or at all, due to safety, efficacy, and other risks, that the potential accelerated approval and/or the anticipated commercial launch of povetacicept may be delayed, if it occurs at all, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on businesswire.com: https://www.businesswire.com/news/home/20261023342443/en/ Contacts Vertex Pharmaceuticals Incorporated Investors: [email protected] or +1 617-341-6108 Media: [email protected]

Vertex Pharmaceuticals Incorporated (VRTX), with a market capitalization of approximately $123.2 billion, is a prominent biotechnology company headquartered in Boston. It discovers, develops, and commercializes innovative medicines for serious diseases, focusing on specialty markets through scientific research and development. VRTX is set to report its Q3 earnings on Monday, November 2, 2026, after the market closes. Wall Street anticipates diluted EPS of $4.28, a 1.4% increase from $4.22 in the same quarter last year. Notably, the company has topped analysts' EPS expectations in each of the past four quarters. - Dear Intel Fans, Mark Your Calendars for October 29 - Bill Gates Says Despite Elon Musk Being Mean to Him, He's Still 'In Awe' of His Work — 'He's Brilliant, He's Rich, He's Influential' - At 97, Charlie Munger Borrowed $37 Million to Go All-In on Alibaba — 2 Years Later He Called It One of the 'Worst Mistakes' of His Life - Markets move fast. Keep up by reading our FREE midday Barchart Brief newsletter for exclusive charts, analysis, and headlines. For fiscal 2026, analysts expect VRTX to report EPS of $16.62, up only marginally from $16.47 in fiscal 2025. Moreover, earnings growth is expected to remain robust in fiscal 2027, with EPS forecast to climb another 10.1% year over year to $18.29. Over the past 52 weeks, VRTX stock has climbed 20.9%, outperforming the S&P 500 Index ($SPX), which advanced 16.2%. However, the stock has trailed the State Street Health Care Select Sector SPDR ETF (XLV), which gained 22.2% over the same period. Vertex Pharmaceuticals' solid price performance over the past year has been supported by stellar revenue growth, with sales increasing at an annual rate of 13.5% over the past five years. Operating profits also improved over the past two years as the company gained operating leverage and became more efficient, while its market-beating returns on capital reflect management's ability to invest in profitable growth opportunities. Adding to this momentum, on Sept. 22, Vertex reported promising Phase 2b results for inaxaplin, its investigational treatment for APOL1-mediated kidney disease. The drug reduced proteinuria by 42.7% in patients with modest proteinuria and 17.3% in those with type 2 diabetes, and was generally well tolerated, with no treatment-related serious adverse events. Vertex also completed enrollment in the pivotal Phase 2/3 AMPLITUDE trial and expects interim data in early 2027. Positive results could support potential accelerated approval in the U.S., opening a new growth opportunity in a kidney disease with no currently approved therapies. Analysts remain highly bullish on VRTX, with the stock holding a consensus "Strong Buy" rating. Of the 33 analysts covering the stock, 24 recommend a "Strong Buy," two assign a "Moderate Buy," six rate it a "Hold," and one suggests a "Strong Sell." Meanwhile, the average analyst price target of $572.69 suggests potential upside of 14.9% from the current share price. On the date of publication, Kritika Sarmah did not have (either directly or indirectly) positions in any of the securities mentioned in this article. All information and data in this article is solely for informational purposes. This article was originally published on Barchart.com

BOSTON, October 07, 2026--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its third quarter 2026 financial results on Monday, November 2, 2026, after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the "Vertex Pharmaceuticals Third Quarter 2026 Earnings Call." The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia, acute pain and acromegaly, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, congenital adrenal hyperplasia, ACTH-dependent Cushing's syndrome, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X. (VRTX-WEB) View source version on businesswire.com: https://www.businesswire.com/news/home/20261007119271/en/ Contacts Vertex Pharmaceuticals Incorporated Investor Relations: [email protected]

In people with modest proteinuria, albumin in the urine fell 42.7% from baseline, and total protein fell 44.7% in the study. A second group — people who also have type 2 diabetes — saw a smaller drop: 17.3% for albumin and 25.4% for total protein. Truist called the data a validation of APOL1 inhibition in a potential market of more than 100,000 patients and said the opportunity is large enough to support blockbuster sales for Maze’s MZE829 even if it only matches inaxaplin. Shares of Vertex Pharmaceuticals (VRTX) rose 3% on Tuesday after the company said its experimental pill inaxaplin cut protein leakage into urine in a study in two extra groups of patients with APOL1-mediated kidney disease (AMKD), a genetic condition with no approved treatments. Rival Maze Therapeutics (MAZE), which is developing its own drug for the condition called MZE829, jumped more sharply, by about 25%, on the read-through. See what 10M+ investors are talking about. Get the Stocktwits Daily Rip for what retail is watching right now, free to your inbox The mid-stage AMPLIFIED study tested a once-daily 45 mg dose of inaxaplin for 13 weeks on top of optimized standard care. In people with modest proteinuria, urinary albumin fell 42.7% from baseline, and total protein fell 44.7%. That matched an earlier mid-stage study in patients with a scarring form of the disease known as Focal Segmental Glomerulosclerosis (FSGS). Most people in the new modest-proteinuria group did not have an FSGS diagnosis. A second group — people who also have type 2 diabetes — saw a smaller drop: 17.3% for albumin and 25.4% for total protein. Vertex called that effect encouraging but more modest. The drug was generally well tolerated. No serious side effects were tied to inaxaplin; headache was the most common complaint. AMKD is a rapidly progressing disease caused by two variants in the APOL1 gene and occurs mainly in people of African ancestry. Vertex estimates it affects about 150,000 people in the United States and Europe. Left untreated, it can lead to dialysis, transplant, or death. Vertex also said it has finished enrollment in AMPLITUDE, the larger trial in patients with more severe proteinuria and no other major kidney-damaging conditions. An interim look is due in early 2027 and, if positive, could support a U.S. accelerated-approval filing. The company plans to discuss the AMPLIFIED results with regulators alongside that program. Chief medical officer Carmen Bozic said the findings add to evidence that inaxaplin could become a first- and best-in-class therapy aimed at the disease’s root cause. Truist analyst Danielle Brill called the data a meaningful validation of APOL1 inhibition in classic AMKD and estimated an addressable population of more than 100,000 patients. Truist sees “a significant positive readthrough” for Maze. While it is too early to compare the two drugs, the firm said the opportunity is large enough to support blockbuster sales for MZE829 even if efficacy and safety prove only comparable to inaxaplin. Truist rates Maze a Buy with a $64 price target. The new price target implies a potential upside of about 186% from the stock’s last closing price on Monday. On Stocktwits, retail sentiment around VRTX stock stayed in ‘bearish’ territory over the past 24 hours, while message volume stayed ‘low.’ Meanwhile, sentiment around MAZE jumped from ‘neutral’ to ‘bullish’ over the past 24 hours, while retail chatter jumped to ‘high’ levels. VRTX stock is up about 15% year to date. MAZE is down about 35% over the same stretch, leaving Tuesday’s pop against a steep 2026 drawdown. Read More: Michael Burry’s Memory Stocks Thesis Gets Fresh Support From Acer CEO’s Supply Warning For updates and corrections, email newsroom[at]stocktwits[dot]com Anan Ashraf has no position in any of the stocks mentioned in this article. StockTwits' news team content is for informational purposes only and is not intended as investment advice. For more, see our editorial policy. This article was originally published on StockTwits. Related: Marc Benioff Says Microsoft Shut Salesforce Out Of OpenAI — So He Bet On Anthropic Instead, And It Could Pay 'Tens Of Billions' Marc Benioff Says Microsoft Shut Salesforce Out Of OpenAI — So He Bet On Anthropic Instead, And It Could Pay 'Tens Of Billions' MSTR Stock Climbs Overnight: This Analyst Sees Upside Of 16.5% From Current Levels

– In people with AMKD with modest proteinuria, treatment with inaxaplin led to a reduction in urine albumin to creatinine ratio (UACR) of -42.7% at Week 13 compared to baseline – - In people with AMKD and type 2 diabetes, treatment with inaxaplin led to a reduction in UACR of -17.3% at Week 13 compared to baseline – - Inaxaplin was generally safe and well tolerated in both cohorts – - Vertex has completed enrollment in the AMPLITUDE trial and remains on track for interim analysis results in early 2027, which, if positive could support potential accelerated approval in the US - BOSTON, September 22, 2026--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from the Phase 2b AMPLIFIED study of inaxaplin in people with APOL1-mediated kidney disease (AMKD) and modest proteinuria and in people with AMKD and type 2 diabetes. In this study, inaxaplin was evaluated as a 45 mg once-daily dose on top of optimized standard of care. Forty-one people were enrolled and dosed in two cohorts: 1) people with AMKD with modest proteinuria (UACR ≥0.1 g/g to <0.42 g/g) were eligible for cohort 1 (N=23), and 2) people with AMKD, type 2 diabetes and proteinuria (UACR ≥0.1 g/g to <6 g/g) were eligible for cohort 2 (N=18). The primary endpoint in the study was mean percent change in UACR at Week 13 compared to baseline, evaluated separately for each cohort. The other endpoints included mean percent change in urine protein to creatinine ratio (UPCR) at Week 13 compared to baseline and safety. In cohort 1, treatment with inaxaplin administered on top of optimized standard of care led to a reduction of -42.7% (95% CI -58.3%, -21.1%) in UACR at Week 13 compared to baseline. UPCR decreased by -44.7% from baseline at Week 13. These results are in line with the treatment effect previously reported in the Phase 2a study of inaxaplin in AMKD with focal segmental glomerulosclerosis (FSGS), which demonstrated a reduction in UACR of -43.4% and a reduction in UPCR of -47.6% at Week 13 compared to baseline. In this modest proteinuria cohort, most people were not diagnosed with FSGS. In cohort 2, treatment with inaxaplin administered on top of optimized standard of care led to a reduction in UACR at Week 13 of -17.3% (95% CI -36.3%, 7.2%) compared to baseline. UPCR decreased by -25.4% from baseline at Week 13. Efficacy Results for AMPLIFIED Phase 2b Study by Cohort Inaxaplin was generally safe and well tolerated across both cohorts. There were no serious adverse events (SAEs) related to inaxaplin. All adverse events (AEs) were mild or moderate in severity. The most common AE (occurring in >5% of subjects) was headache (7.3%). Five people had isolated, asymptomatic transaminase elevations, which resolved. "These results add to the evidence base for inaxaplin and its potential as a first- and best-in-class treatment targeting the underlying cause of AMKD. The modest proteinuria cohort, which included patients with and without FSGS, delivered remarkable reductions in proteinuria consistent with the original Phase 2a study in patients with AMKD and FSGS," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "We are also excited with the treatment effect, though smaller in magnitude, in the type 2 diabetes cohort. We look forward to discussing the AMPLIFIED results with regulators in the context of the AMPLITUDE pivotal study." "These results are very convincing," said Glenn Chertow, M.D., M.P.H., Professor of Medicine, Stanford University School of Medicine, and Chair of the Vertex APOL1 Program Steering Committee. "These patients were already well treated with foundational chronic kidney disease therapies and treatment with inaxaplin led to additional reductions in proteinuria. As someone who treats this rapidly progressing disease where unmet need is high, I am looking forward to the AMPLITUDE interim analysis and am hopeful that we’ll soon have a potentially disease-specific and disease-modifying therapy to offer to patients living with AMKD." Vertex has completed full enrollment in the Phase 2/3 AMPLITUDE study of inaxaplin in people with severe proteinuria and no other kidney disease-causing comorbidities. Vertex is on track to share data from the pre-planned interim analysis (IA) of AMPLITUDE in early 2027 once the IA cohort reaches 48 weeks of treatment. If positive, the data could form the basis for potential accelerated approval in the U.S. About APOL1-Mediated Kidney Disease (AMKD)AMKD is a rapidly progressing, proteinuric kidney disease caused by two variants in the APOL1 gene. It occurs in people of African ancestry. AMKD occurs when inherited APOL1 genetic variants cause kidney cell injury, cell death and damage to the glomeruli, which filter blood in the kidney. This leads to protein in the urine (known as "proteinuria") and decreased ability of the kidney to function, which can lead in turn to dialysis, transplant or death. AMKD affects approximately 150,000 people in the U.S. and Europe. There are no therapies currently approved for AMKD. About AMPLIFIEDAMPLIFIED is a Phase 2, single-arm, open-label study evaluating the effect of treatment with a 45 mg once-daily dose of inaxaplin for 13 weeks to reduce proteinuria in people with AMKD with modest proteinuria, and people with AMKD and type 2 diabetes — populations not being studied in the AMPLITUDE trial. The primary endpoint is the percent change of urine albumin to creatinine ratio (UACR) from baseline. About AMPLITUDEAMPLITUDE is a global Phase 2/3 clinical trial designed to assess the impact of inaxaplin on kidney function and proteinuria for people living with AMKD. Inaxaplin is being evaluated as a 45 mg once-daily oral dose compared to placebo, on top of standard of care. The primary efficacy endpoint for the final analysis is estimated glomerular filtration rate (eGFR) slope in patients receiving inaxaplin compared to placebo after two years of treatment. About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia, acute pain and acromegaly, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, congenital adrenal hyperplasia, adrenocorticotropic hormone (ACTH)-dependent Cushing's syndrome, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements made by Carmen Bozic, M.D., and Glenn Chertow, M.D., M.P.H., and statements about sharing data from the pre-planned interim analysis of AMPLITUDE in early 2027, expectations that, if positive, the AMPLITUDE data could form the basis for potential accelerated approval in the U.S., and expectations regarding the eligible patient population. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on businesswire.com: https://www.businesswire.com/news/home/20260921995768/en/ Contacts Investors:[email protected] or+1 617-341-6108Media:[email protected] or+1 617-341-6992

Vertex Pharmaceuticals stock has delivered strong multi year gains, and current valuation checks suggest the shares now look closer to fairly priced than clearly cheap or clearly expensive. Vertex Pharmaceuticals has returned 184.9% over the past 5 years, which puts extra focus on whether the current price still offers an appealing entry point for new money. Expectations for continued revenue and cash flow growth from its existing treatment portfolio can support today’s valuation, while any setback in clinical execution or pricing pressure may weigh on how durable those cash flows look. The broader valuation checks are mixed rather than decisive, with a value score of 3 that does not flag Vertex Pharmaceuticals as either a clear bargain or an obvious outlier on price. The issue now is whether the current share price of Vertex Pharmaceuticals offers enough potential upside to justify the risks after such a strong 5 year run. Compare Vertex Pharmaceuticals’ strong 5 year run with other stocks that pair solid balance sheets and fundamentals by scanning the hand picked solid balance sheet and fundamentals stocks screener (53 results). The P/E ratio is a useful check for a profitable company like Vertex Pharmaceuticals because it ties the share price directly to current earnings. Vertex Pharmaceuticals trades on a P/E of about 31.5x, which is higher than the broader biotechs industry average of roughly 16.8x but below the peer group average around 39.4x. That suggests investors are already paying a premium relative to the overall sector, while still not assigning the highest multiples seen among closer peers. A fair P/E level that takes account of Vertex Pharmaceuticals’ size, margins, industry and risk profile is estimated at about 31.3x. The current 31.5x multiple is very close to this mark, so the shares are not screening as clearly cheap or clearly expensive on earnings alone. For you as an investor, this points to a market view that current earnings power is largely reflected in the price. On the P/E multiple, Vertex Pharmaceuticals stock currently appears roughly fairly valued. See what the numbers say about this price — find out in our valuation breakdown. This is where Simply Wall St Narratives come in for Vertex Pharmaceuticals, since they spell out which views on future growth, margins and earnings would need to prove accurate for the stock to be worth materially more or materially less than today's price on the Community page. Each one links its number to a clear view of how Vertex Pharmaceuticals' growth, profitability and risk profile might evolve, which you can revisit as new information comes through. The community is split on Vertex Pharmaceuticals, with one camp seeing meaningful upside and the other worried that expectations already run too hot. Bull case: 19% undervalued Read the full Bull Case to see why Vertex Pharmaceuticals could be undervalued Bear case: 29% overvalued Read the full Bear Case to see why Vertex Pharmaceuticals could be overvalued Do you think there's more to the story for Vertex Pharmaceuticals? Head over to our Community to see what others are saying! Vertex Pharmaceuticals now looks priced roughly in line with what its current earnings power and peer comparisons imply, rather than clearly cheap or clearly expensive. That leaves less room for valuation alone to drive returns, so the debate shifts to how durable its cash flows prove and how well the late stage pipeline performs. For you as an investor, the key question is whether Vertex Pharmaceuticals can sustain the kind of growth and profitability the market is already paying for, without unexpected setbacks on execution, regulation or pricing. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include VRTX. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email [email protected]

It has been about a month since the last earnings report for Vertex Pharmaceuticals (VRTX). Shares have added about 14.4% in that time frame, outperforming the S&P 500. Will the recent positive trend continue leading up to its next earnings release, or is Vertex due for a pullback? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its latest earnings report in order to get a better handle on the important catalysts. Vertex reported adjusted earnings of $4.73 per share for the second quarter of 2026, missing the Zacks Consensus Estimate of $4.79. Earnings, however, rose around 4.6% year over year on higher product revenues, partially offset by higher operating expenses.Second-quarter total revenues of $3.33 billion beat the Zacks Consensus Estimate of $3.23 billion. Total revenues rose 12% year over year, primarily driven by higher sales of CF drugs Trikafta/Kaftrio and Alyftrek, as well as meaningful contributions from other new products, Journavx and Casgevy. Its total revenues rose 11% year over year in the United States to $2.06 billion, driven by strong demand for CF drugs, higher realized net prices in CF and Casgevy and Journavx sales. Outside the U.S. market, sales increased 14% to $1.28 billion, driven by strong CF growth, contribution from Casgevy and a favorable impact from foreign exchange. Trikafta generated sales worth $2.50 billion, down around 2% year over year. The product’s sales slightly beat the Zacks Consensus Estimate of $2.45 billion.Alyftrek generated sales worth $573.6 million in the second quarter, up 35% on a sequential basis. The drug surpassed $1 billion in global revenues in the first half of 2026. Growth was driven primarily by patients switching from Trikafta, along with new-to-therapy patients and strong uptake in Europe. Expansion into rare mutations and younger patients should support further growth.Revenues from other CF products (including Kalydeco, Orkambi, and Symdeko/Symkevi) decreased 29.2% year over year to $137.1 million. Vertex expects incremental patients from the label expansions for Alyftrek and Trikafta, along with launches of Alyftrek in additional geographies and for treating younger patients, to drive CF growth through the rest of the year.Casgevy’s sales were $76.4 million in the second quarter, up 78% on a sequential basis and 151% on a year-over-year basis due to an increase in patient infusions. Casgevy recorded more than 100 patient initiations in the second quarter as the launch continues to progress. First-half 2026 infusions already exceeded the total for 2025, supported by improved reimbursement and growing patient uptake across key markets.In 2026, Vertex expects continued quarter-to-quarter variability in Casgevy infusions.Journavx (suzetrigine) generated $49.6 million in sales in the second quarter, up 71% on a sequential basis. Journavx sales in the second quarter benefited from both strong underlying prescription growth and inventory restocking by distributors, following a reduction in channel inventory during the first quarter.More than 535,000 prescriptions were written for Journavx across both hospital and retail settings in the quarter, compared with approximately 350,000 in the first quarter, showing that uptake is accelerating. In 2026, Vertex expects Journavx prescriptions to triple compared to 550,000 written in 2025, supported by a larger commercial field force, wider payer coverage and improving gross-to-net economics. Vertex is reasonably confident of exceeding the goal.Journavx’s reimbursement trends are also improving, with coverage now reaching approximately 260 million lives. Vertex expanded access further by securing agreements with two additional Medicare Part D plans effective July 1. Journavx is now covered by three out of four of the major Medicare Part D PBMs. In addition, 23 states now cover Journavx through Medicaid. Adjusted research and development (R&D) expenses increased 1.2% year over year to $888.7 million.Adjusted selling, general and administrative (SG&A) expenses rose 44.7% to $520.2 million in the reported quarter, primarily to support the launch of Journavx and the upcoming launches in renal.During the quarter, Vertex recorded acquired in-process research and development (AIPR&D) costs of $21.4 million compared with $2.2 million in the year-ago quarter.Adjusted operating income rose 7.5% year over year to $1.42 billion in the quarter. Vertex increased its full-year 2026 revenue guidance backed by a strong first-half performance.The company now expects total revenues to be in the range of $13.10-$13.20 billion for 2026 compared with the previous expectation of $12.95-$13.10 billion. Global CF revenues grew 8% in the first half of 2026, helped by prior-year U.S. price increases and favorable foreign exchange. However, Vertex expects these benefits to fade in the second half.Vertex expects its non-CF product revenues to be more than $500 million in 2026, representing more than 185% year-over-year growth from 2025, reflecting higher patient infusions for Casgevy and a ramp-up in Journavx prescriptions.The revenue guidance also includes an expected 150 basis point benefit from foreign exchange.Combined adjusted R&D, AIPR&D and SG&A expense guidance for 2026 was maintained in the range of $5.65-$5.75 billion. However, the company now expects to be at the high end of that range. Adjusted gross margin is expected to be under 86%. The adjusted tax rate is expected to be in the range of 19.5%-20.5%. Both remain unchanged from the previous expectation.The guidance does not reflect the impact of the pending Crinetics acquisition, which is expected to be closed in the third quarter. It turns out, estimates revision have trended downward during the past month. At this time, Vertex has a average Growth Score of C, though it is lagging a lot on the Momentum Score front with an F. Charting a somewhat similar path, the stock has a grade of D on the value side, putting it in the bottom 40% for this investment strategy. Overall, the stock has an aggregate VGM Score of D. If you aren't focused on one strategy, this score is the one you should be interested in. Estimates have been broadly trending downward for the stock, and the magnitude of these revisions indicates a downward shift. Notably, Vertex has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months. Vertex is part of the Zacks Medical - Biomedical and Genetics industry. Over the past month, Biogen Inc. (BIIB), a stock from the same industry, has gained 3.6%. The company reported its results for the quarter ended June 2026 more than a month ago. Biogen reported revenues of $2.74 billion in the last reported quarter, representing a year-over-year change of +3.4%. EPS of $3.60 for the same period compares with $5.47 a year ago. Biogen is expected to post earnings of $2.31 per share for the current quarter, representing a year-over-year change of -52%. Over the last 30 days, the Zacks Consensus Estimate has changed -19.4%. Biogen has a Zacks Rank #3 (Hold) based on the overall direction and magnitude of estimate revisions. Additionally, the stock has a VGM Score of C. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report Biogen Inc. (BIIB) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

ANT survey delivers a 3D subsurface view that reinforces the Robinsons River geological model and de-risks the next stage of technical work VANCOUVER, British Columbia, Aug. 18, 2026 (GLOBE NEWSWIRE) -- Vortex Energy Corp. (CSE: VRTX) (OTC: VTECF) (FSE: AA3) (“Vortex” or the “Company”) is pleased to announce the final results of the Ambient Noise Tomography (“ANT”) survey completed at the Company's 100%-owned Robinsons River Salt Project (the “Project”), located approximately 35 kilometres south of Stephenville, Newfoundland and Labrador. Further to the Company's September 16, 2025 announcement that field acquisition had been completed, the final ANT results add a 3D shear-wave velocity dataset to the geological, geophysical and drilling information being considered within the Company's ongoing staged technical review of the Project. The ANT survey successfully mapped broad subsurface velocity domains and lateral velocity changes that may reflect structural or lithological variation across the survey area. The computational model extends to approximately 3.8 kilometres and is considered to image the subsurface to at least 2.5 kilometres depth. The resulting model provides regional and structural context for the existing drilling, seismic and gravity information and is being considered as part of the Company's ongoing integrated review. However, it does not sharply or uniquely resolve the boundaries of the interpreted salt structures, likely because of overlap between the shear-wave velocities of halite and the consolidated clastic sedimentary rocks that comprise much of the local stratigraphic sequence. Accordingly, the ANT results are being used as one component of the integrated interpretation and not as a standalone method for delineating salt. 3D Shear-Wave Velocity Model The resulting shear-wave velocity model covers an area approximately 17.5 kilometres east-west by 9.24 kilometres north-south. It extends to a maximum depth of approximately 3.8 kilometres and is considered to image the subsurface to at least 2.5 kilometres depth. Modelled shear-wave velocities range from approximately 1,100 metres per second near surface to approximately 4,450 metres per second at depth. Three broad velocity domains were identified. The upper few hundred metres are generally characterized by velocities below approximately 1,500 metres per second. An intermediate, laterally variable domain extends to approximately 2.0 to 2.5 kilometres depth and generally ranges from approximately 2,000 to 4,000 metres per second. A deeper, more homogeneous domain is generally characterized by velocities above approximately 4,000 metres per second. A depth-normalized velocity-contrast model further highlights lateral velocity variation within the survey area. One of the most persistent features is a northeast-trending velocity discontinuity along the eastern part of the model that may represent a major structural or lithological boundary. Persistent higher-velocity zones along the eastern survey margin and below approximately two kilometres depth may reflect metasedimentary and granitic rocks similar to those exposed southeast of the survey area. These features remain geological interpretations of velocity variations and have not been confirmed as specific contacts or rock types. Figure 1. 3D shear-wave velocity model generated from the Robinsons River ANT survey, viewed looking north. Cool colours represent lower shear-wave velocities and warm colours represent higher shear-wave velocities. The model identifies three broad velocity domains and extends computationally to approximately 3.8 kilometres depth; CAUR concludes that the model images the subsurface to at least 2.5 kilometres depth. Source: CAUR Technologies Inc., Vortex Energy - Robinsons River Salt, CAU-25-023 - Ambient Noise Tomography Survey, Final Report. Integration with Existing Technical Information Comparison of the previously interpreted salt outlines derived from active seismic modelling with the ANT model shows that those interpretations coincide approximately with areas of lower shear-wave velocity. However, lower shear-wave velocity is not unique to salt, and the ANT results alone cannot determine the boundaries, thickness, composition or continuity of the interpreted salt structures. Because the prior outlines were used in the comparison itself, this spatial association should not be considered independent confirmation of those structures. The ANT results were also compared with the FALCON airborne gravity gradiometry data and the 3D density inversion available at the time of the analysis. This comparison showed moderate to low density variation and subtle density contrasts spatially associated with the previously interpreted salt structures. The Company is also considering the ANT velocity model alongside legacy seismic data and recent drilling information and core data within its ongoing staged technical review. Figure 2. Plan-view slices from the 3D ANT shear-wave velocity model at approximately 500, 900, 1,500 and 1,900 metres below surface. Black contours show the previously interpreted salt isopachs derived from active-seismic modelling, while solid and dashed black lines show mapped and inferred faults, respectively. The spatial association between the pre-existing salt interpretations and lower-velocity zones is approximate, and the ANT model did not independently resolve the boundaries of the interpreted salt structures. Source: CAUR Technologies Inc., Vortex Energy - Robinsons River Salt, CAU-25-023 - Ambient Noise Tomography Survey, Final Report. Ongoing Technical Review The ANT velocity and contrast models are being considered as an additional dataset within the Company's ongoing geological, geophysical and commercial review. The current stage remains focused on review and analysis of existing core and technical information, including detailed review and analysis of the VTX-24-W-02 core, representative density measurements, determination of salt percentage by volume, desktop evaluation of available information concerning source-water and disposal requirements, refinement of the integrated geological and geophysical model, and land-package optimization. "Completing the ANT survey is another meaningful step forward for Robinsons River, adding a property-scale 3D view of the subsurface to our growing body of technical data," said Paul Sparkes, Chief Executive Officer of Vortex. "By mapping broad velocity changes and highlighting potential structural and lithological boundaries, this dataset sharpens the geological framework and adds real value to our integrated interpretation. Layered together with our drilling, seismic, gravity and core data, these results give us growing confidence in the model as we advance the project through its staged technical review and work toward unlocking the full potential of this district-scale salt opportunity." Collaboration and Acknowledgements The ANT program was conducted in collaboration with the University of Alberta. Specialized survey design, instrumentation and data processing were provided by CAUR Technologies. The research collaboration was supported in part by a Mitacs grant. The Company also acknowledges the financial support of the Junior Exploration Assistance (JEA) Program, administered by the Department of Industry, Energy and Technology, Government of Newfoundland and Labrador. Qualified Person The scientific and technical content of this news release has been reviewed and approved by Jared Suchan, Ph.D., P.Geo., Vice President of Exploration of the Company and a “Qualified Person” as defined by National Instrument 43-101. The Qualified Person verified the ANT information disclosed in this news release through review of the survey documentation provided by CAUR Technologies, including the acquisition and instrument-recovery summaries, data-quality assessments, processing methodology, model outputs and interpretations. Quality-control measures included checks of instrument operating duration and recovery, assessment of cross-correlation signal-to-noise behaviour and stability, and evaluation of the dominant directional noise source from the Gulf of St. Lawrence, which was accounted for during processing and inversion. The Qualified Person did not independently reprocess the raw passive-seismic dataset or recreate the 3D inversion and has relied on CAUR for those specialist technical components. No sampling or laboratory analytical work was involved in the ANT survey. For additional information concerning the Project and the Company’s data-verification and quality-assurance procedures, readers are referred to the Company’s technical report entitled Independent Technical Report on the Robinsons River Salt Property, dated July 31, 2023, available under the Company’s profile on SEDAR+. About Vortex Energy Corp. Vortex Energy Corp. is an exploration stage company engaged principally in the acquisition, exploration, and development of mineral properties in North America. The Company is currently advancing its Robinson River Salt Project comprised of a total of 942 claims covering 23,500 hectares located approximately 35 kilometres south of the town of Stephenville in the Province of Newfoundland & Labrador. The Robinson River Salt Project is prospective for both salt and hydrogen salt cavern storage. The Company is also currently advancing its Fire Eye Uranium Property in the Athabasca Basin, a region renowned for its uranium deposits. On Behalf of the Board of Directors Paul Sparkes Chief Executive Officer, Director +1 (778) 819-0164 [email protected] Cautionary Note Regarding Forward-Looking Statements Certain statements contained in this press release constitute forward-looking information. These statements relate to future events or future performance. The use of any of the words “could”, “intend”, “expect”, “believe”, “will”, “projected”, “estimated” and similar expressions and statements relating to matters that are not historical facts are intended to identify forward-looking information and are based on the Company’s current beliefs or assumptions as to the outcome and timing of such future events. In particular, this press release contains forward-looking information relating to, among other things, the Company’s exploration plans at the Project. Various assumptions or factors are typically applied in drawing conclusions or making the forecasts or projections set out in forward-looking information, including, in respect of the forward-looking information included in this press release, assumptions regarding the Company’s ability to execute on its exploration plans, including that it will be successful in carrying out the planned drilling and that such drilling will yield the expected information and the desired outcomes. Although forward-looking information is based on the reasonable assumptions of the Company’s management, there can be no assurance that any forward-looking information will prove to be accurate. Forward-looking information involves known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking information. Such factors include, among other things, the risk that exploration at the Project does not proceed in the manner and on the timeline currently contemplated, or at all; risks inherent in the exploration and development of mineral deposits, including risks relating to receiving requisite permits and approvals, changes in project parameters or delays as plans continue to be redefined, that mineral exploration is inherently uncertain and that the results of mineral exploration may not be indicative of the actual geology or mineralization of a project; and that mineral exploration may be unsuccessful or fail to achieve the results anticipated by the Company, including as a result of factors beyond the Company’s control, such as geological conditions. The forward-looking information contained in this release is made as of the date hereof, and the Company not obligated to update or revise any forward-looking information, whether as a result of new information, future events or otherwise, except as required by applicable securities laws. Because of the risks, uncertainties and assumptions contained herein, investors should not place undue reliance on forward-looking information. The foregoing statements expressly qualify any forward-looking information contained herein. The Canadian Securities Exchange has not reviewed, approved, or disapproved the contents of this press release. Photos accompanying this announcement are available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/fab8ba50-09f2-4a22-bfbd-5e2e1eae3663 https://www.globenewswire.com/NewsRoom/AttachmentNg/484fe765-4ea9-4930-a6b4-352edcbfec4d

Vertex Pharmaceuticals’ second quarter reflected strong commercial momentum, with management highlighting the performance of its cystic fibrosis (CF) portfolio and accelerating uptake of newer products like CASGEVY and JOURNAVX. CEO Reshma Kewalramani attributed revenue growth to “the strength of our cystic fibrosis portfolio and the growing contributions from our newer products,” while also emphasizing progress in regulatory and clinical milestones. Management specifically noted that ALYFTREK and TRIKAFTA continued to drive CF revenue, and that European launches benefitted from fewer monitoring requirements. Is now the time to buy VRTX? Find out in our full research report (it’s free). Revenue: $3.33 billion vs analyst estimates of $3.19 billion (12.5% year-on-year growth, 4.6% beat) Adjusted EPS: $4.73 vs analyst expectations of $4.75 (in line) Adjusted Operating Income: $1.42 billion vs analyst estimates of $1.37 billion (42.7% margin, 3.9% beat) The company slightly lifted its revenue guidance for the full year to $13.15 billion at the midpoint from $13.03 billion Operating Margin: 37.4%, down from 38.8% in the same quarter last year Market Capitalization: $125.7 billion While we enjoy listening to the management’s commentary, our favorite part of earnings calls is the analyst questions. Those are unscripted and can often highlight topics that management teams would rather avoid or topics where the answer is complicated. Here is what has caught our attention. Salveen Richter (Goldman Sachs): Asked about the Phase II/III OLYMPUS study dose selection for Pove in membranous nephropathy and bottlenecks in JOURNAVX’s payer dynamics. CEO Reshma Kewalramani clarified the Data Safety Monitoring Board’s role in dose selection and Duncan McKechnie highlighted ongoing efforts to secure broader access for JOURNAVX. Geoffrey Meacham (Citibank): Inquired about desired clinical outcomes for next-generation CF therapies and payer feedback on JOURNAVX in diabetic peripheral neuropathy. Kewalramani said efficacy and safety are the main goals, with further data and payer perspectives expected in coming quarters. Jessica Fye (JPMorgan): Asked about expected differentiation on eGFR endpoints for new IgAN treatments. Kewalramani explained that long-term benefits will depend on time to end-stage renal disease and the ability to achieve deeper reductions in proteinuria and hematuria. Cory Kasimov (Evercore ISI): Sought clarity on interim analysis criteria for inaxaplin in AMPLITUDE and accelerated approval. Kewalramani said the FDA agreement focuses on 1-year GFR as the primary endpoint for the interim analysis. Mohit Bansal (Wells Fargo): Queried prescription duration trends for JOURNAVX. McKechnie noted average durations have remained stable at around 10–11 days per prescription, split between hospital and retail settings. In coming quarters, the StockStory team will be watching (1) the pace of ALYFTREK adoption and label expansions in CF, (2) launch execution and patient uptake for CASGEVY and JOURNAVX, and (3) regulatory progress and launch timing for Pove in IgAN and the integration of Crinetics’ assets. Progress in clinical data readouts for next-generation CF and renal assets will also be key signposts. Vertex Pharmaceuticals currently trades at $496.00, up from $470.72 just before the earnings. At this price, is it a buy or sell? Find out in our full research report (it’s free for active Edge members). ONE MORE THING: Top 5 Growth Stocks. The biggest stock winners almost always had one thing in common before they ran. Revenue growing like crazy. Meta. CrowdStrike. Broadcom. Our AI flagged all three. They returned 315%, 314%, and 455%, respectively. Find out which 5 stocks it’s flagging this month — FREE. Get Our Top 5 Growth Stocks for Free HERE. Stocks that have made our list include now familiar names such as Nvidia (+1,460% between June 2020 and June 2025) as well as under-the-radar businesses like the once-micro-cap company Kadant (+214% between June 2020 and June 2025). Find your next big winner with StockStory today.

Image source: The Motley Fool. Monday, Aug. 3, 2026 at 4:30 p.m. ET Chief Executive Officer and President - Dr. Reshma Kewalramani Chief Operating Officer and Chief Financial Officer - Charles Wagner Chief Commercial Officer - Duncan J. McKechnie Senior Vice President of Investor Relations - Susie Lisa Operator: Good day, and welcome to the Vertex Pharmaceuticals Second Quarter 2026 Earnings Call. [Operator Instructions] 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: 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 Second 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, the proposed acquisition of Crinetics Pharmaceuticals and the expected benefits of that transaction and Vertex's future financial performance, are based on management's current assumptions. Actual outcomes and events could differ materially. I would also note that select financial results and guidance that we will review on the call this evening are presented on a non-GAAP basis. I'll now turn the call over to Reshma. Reshma Kewalramani: Thanks, Susie. Good evening, all, and thank you for joining us on the call today. Vertex's second quarter performance was excellent, with strong momentum in the commercial portfolio, rapid progress across our R&D pipeline, and the announcement of the definitive agreement to acquire Crinetics Pharmaceuticals, which brings rare endocrine diseases as a fifth pillar to Vertex. Second quarter total revenue grew 12% year-on-year, driven by the strength of our cystic fibrosis portfolio and the growing contributions from our newer products, CASGEVY and JOURNAVX. As I've previously highlighted, this is a year of execution for Vertex across commercial, clinical and regulatory. And on each of those fronts, we advanced significantly in the second quarter. Commercially, we delivered strong revenue growth across all diseases, made meaningful progress in reimbursed access and continue to execute on near-term launch planning to drive the next phase of growth. Clinically, we continue to make significant progress in advancing our pipeline, including completing enrollment in the AGLOW Phase II study of VX-407 in ADPKD, tracking to complete enrollment in the AMPLITUDE Phase III study in AMKD by the end of this year and reporting results from the interim analysis cohort of AMPLITUDE in the beginning of 2027. We also remain on track to release results later this year from a proof-of-concept study in DM1 and an expanded population for AMKD in the AMPLIFIED trial as well as the initial patient data from VX-828 in CF. On the regulatory front, the BLA for Pove in IgAN was accepted in the U.S. with a November 30 PDUFA date. We achieved expanded labeling in record time for CASGEVY in patients ages 2 to 11 in the U.S. And I'm very pleased to share that as we continue to dose the Phase I/II/III study of zimislecel in type 1 diabetes, the IND was cleared for the blood type O islet cells in our T1D program, VX-017. We expect initiation of the VX-017 Phase I/II study in the near term. Finally, with the announced acquisition of Crinetics Pharmaceuticals, we look forward to multiple benefits of the deal, establishing a fifth pillar in rare endocrine diseases, adding to our innovative R&D pipeline, accelerating revenue growth and enhancing long-term earnings. Tonight, I'll limit my R&D comments to new news in CF, renal and type 1 diabetes and close with some additional remarks regarding the Crinetics acquisition. Let me start with CF, where we continue to extend our market leadership. Data we presented at ECFS reinforced that ALYFTREK best restores CFTR function amongst the available CFTR modulators. In particular, among children with CF under 12 years of age, the majority across all eligible genotypes achieve a sweat chloride less than 30 millimoles, which is the median among CF carriers. This is remarkable because at these sweat chloride levels, CF carriers do not exhibit manifestations of disease. In addition, we have initiated global regulatory submissions for ALYFTREK in children, ages 2 to 5. Global regulatory submissions for TRIKAFTA in patients ages 1 to 2 are also in progress. Turning to our next wave in CF and VX-828, our next-generation 3.0 CFTR modulator recently completed dosing in the patient cohort and data are expected in the second half of this year. Behind VX-828, we continue to advance additional correctors in the NextGen 3.0 family and both VX-581 and VX-272 are in healthy volunteer studies. Let me close on CF with this. Our ultimate goal has been consistent for 2-plus decades to bring patients to carrier levels of sweat chloride. Frankly, ALYFTREK's remarkable results, where nearly 2/3 of younger patients achieved sweat chloride levels less than 30 millimole per liter and for patients ages 12 plus more than 75% achieved sweat chloride levels within the carrier range of CFTR function means we are very close to that goal. Given the improvements in sweat chloride, ppFEV1, pulmonary exacerbations, hospitalizations, lung transplant and survival that we have seen in patients in clinical trials and/or the real world, we recognize that the unmet need is far lower today and the bar for any medicine to beat ALYFTREK is very, very high. Thus, as we develop our next-gen 3.0 and Beyond programs, we will evaluate multiple regimens in Phase I in cohorts of patients with CF. However, we will only advance assets into Phase II in Beyond that show promise to beat ALYFTREK. In other words, to bring even more patients to sweat chloride levels less than 30 millimoles across all genotypes with once-daily dosing and excellent drug-like properties, including drug-drug interactions. Anything less would not be competitive. Moving now to our renal franchise, where we have 4 programs in mid- and late-stage development, povetacicept in IgAN and primary membranous nephropathy, inaxaplin in APOL1-mediated kidney disease and VX-407 in ADPKD, or autosomal dominant polycystic kidney disease. Let me start with the most advanced program and significant milestone. In late May, the FDA accepted our BLA for Pove in IgAN and assigned a PDUFA date of November 30 of this year. As a reminder, the RAINIER Phase III interim analysis was a home run, delivering statistically significant and clinically meaningful results across the primary and all secondary endpoints with a favorable safety profile and consistency in the primary endpoint of change from baseline in proteinuria across all groups. We are in the final stages of launch readiness. Duncan will provide more details regarding our approach and excitement to go to market with Pove's differentiated profile of potentially best-in-class efficacy, a well-tolerated safety profile and patient-centric administration through small volume once-monthly dosing via an auto-injector at home. We are also advancing Pove internationally. We have completed the regulatory submission for accelerated approval of Pove in IgAN in Saudi Arabia, where Pove has received breakthrough designation. Turning to Pove in membranous nephropathy, our OLYMPUS Phase II/III pivotal trial is well underway. The Phase II portion is complete and the Phase III portion initiated last quarter. I'm pleased to share that the IDMC has completed its review and selected the Phase III dose, 80 milligrams subcutaneously every 4 weeks. We hold fast track, orphan drug designation and EMA PRIME designations for Pove in membranous. Stepping briefly outside of renal, on Pove in myasthenia gravis, I'm also pleased to share that the 30-patient Phase II proof-of-concept study is on track to complete enrollment by the end of this year. Recall this study evaluates 80 milligrams and 240-milligram doses of Pove versus placebo for 12 weeks. Turning now to inaxaplin in AMKD. On AMPLITUDE, our pivotal Phase II/III study in AMKD, we completed enrollment of the interim analysis cohort in September of last year and are on track to complete full enrollment by the end of this year. The interim analysis will be conducted following 48 weeks of treatment, and we remain on track to share these IA results in early 2027. If positive, we would be positioned to file for potential accelerated approval in the U.S. thereafter. AMPLIFIED is our Phase IIb basket study of inaxaplin in AMKD patients with either lower proteinuria or AMKD patients with diabetes, expanded patient populations not studied in AMPLITUDE. The AMPLIFIED study has completed enrollment and dosing and we expect to share results this fall. Lastly, in the renal portfolio is VX-407 in ADPKD, or autosomal dominant polycystic kidney disease. Our AGLOW Phase II study has completed enrollment. This is a proof-of-concept study with up to 52 weeks of treatment. We are excited about the potential for VX-407 in ADPKD and look forward to sharing more information as dosing continues and the data matures. Let me now touch on type 1 diabetes. We had very constructive meetings with the FDA following our voluntary pause in order to conduct a manufacturing analysis of zimislecel. As we shared on our Q1 call, we have resumed dosing patients in the zimislecel Phase I/II/III study. The new news today is that the FDA has cleared the IND for VX-017, our Type O or universal donor cell product. VX-017 has a similar target product profile to zimislecel, but is designed for people of all blood types and we expect the VX-017 Phase I/II study to initiate in the near term. By designing and bringing to market VX-017, another allogeneic, off-the-shelf, glucose responsive insulin producing, fully differentiated islet cell therapy, in this case, for any blood type, we anticipate doubling our market opportunity from about 60,000 to about 120,000 patients. A silver lining to the pause we took in the zimislecel Type A program is that the Type O program time differential versus zimislecel has shortened. Type O is making rapid progress. And thus, we are considering options to further streamline our regulatory strategy and commercialization approach. We expect to provide updated T1D plans, including time lines later this year. We also continue to progress our serial innovation work focused on improved immunosuppression and hypoimmune programs to make our potentially one-and-done curative therapy available to even more patients with type 1 diabetes. Let me close with a few words on our announced acquisition of Crinetics Pharmaceuticals, which we detailed in a separate call last month. Crinetics is an excellent strategic fit for Vertex with its focus on serious endocrine diseases, high unmet need, validated targets and well-understood causal biology as well as a strong people and culture fit. We believe the 2 lead assets, PALSONIFY and Atumelnant, together represent a peak sales opportunity of about $5 billion. Both are small molecules that address serious diseases for patients treated by a concentrated group of specialized endocrinologists. This fits directly within Vertex's proven, efficient, specialty commercial model. We enter this transaction from a position of strength. We view CF as a long-duration franchise with sustained growth. We continue to expect both CASGEVY and JOURNAVX to be multibillion-dollar assets and we anticipate our emerging renal franchise could one day rival CF in revenue. In addition, we have a broad and deep pipeline in earlier stages of development. The Crinetics acquisition will add to this innovation pipeline and enhance our revenue growth and long-term earnings profile by adding a fifth commercial pillar in rare endocrine diseases. The transaction is expected to close in the third quarter, and we really look forward to welcoming the talented Crinetics team to Vertex. With that, I'll turn the call over to Duncan for a commercial update. Duncan J. McKechnie: Thanks very much, Reshma. Our commercial story this quarter is one of building momentum across each of our franchises, supported by the appropriate investments to drive growth. We are very excited for the close of the Crinetics acquisition and for Vertex to establish a new pillar in specialty endocrine diseases like acromegaly, CAH and Cushing's syndrome. Crinetics Q2 results were excellent, with strong growth in PALSONIFY revenue and patients treated, but I will hold any further comments until after the deal closes. So tonight, let me start with CF. CF continues to perform very well. Global CF revenue grew 11% year-over-year in the second quarter with balanced growth across the U.S. and internationally and continued strength from both ALYFTREK and TRIKAFTA. ALYFTREK performance has been excellent and crossed another significant milestone, exceeding $1 billion in revenue in the first half of 2026. In the U.S., we continue to see patients initiating ALYFTREK who are new to therapy, returning to therapy and patients switching from TRIKAFTA. The majority of ALYFTREK revenue continues to come from these TRIKAFTA switch patients, which reflects the benefits of ALYFTREK and our success establishing ALYFTREK as the new standard of care. We're pleased with the pace at which physicians and patients are embracing ALYFTREK given its improved sweat chloride profile and once daily dosing. We've seen accelerated uptake of ALYFTREK from the recent approvals in rare mutations as well as patients rolling off our open-label extension studies. Outside the U.S., the ALYFTREK European launches remain very strong with no requirement for augmented liver monitoring in the EU, we are seeing rapid uptake by patients in Europe, transitioning from TRIKAFTA or one of our other CFTR modulators. In fact, in Germany and the U.K., more than 1 in 3 eligible CF patients are now benefiting from ALYFTREK. Globally, the CF growth drivers for the remainder of 2026 are clear: continued ALYFTREK uptake, the label expansion into rare mutations, younger patients and additional geographies. Shifting to heme and CASGEVY, where the momentum continues to build. During the second quarter, we delivered $76 million in CASGEVY revenue, reflecting approximately 75% sequential growth versus quarter 1, 2026 and over 150% year-over-year growth. This was in line with our expectations based on our visibility into patient scheduling patterns. The strength of the CASGEVY franchise continues to build. New data on CASGEVY at EHA with simultaneous publication in the New England Journal of Medicine demonstrated its transformative potential in pediatric patients as well as durable benefits, reinforcing the importance of early intervention to prevent the complications of sickle cell disease and beta thalassemia in children. Stemming from this compelling data, last month CASGEVY became the first and only gene therapy FDA approved to treat children as young as 2 years old in both sickle cell disease and beta thalassemia. CASGEVY received supplemental approval in the U.S. in a record 53 days post-filing, and our first pediatric patient has already initiated therapy and conducted cell collection. Outside the U.S., CASGEVY regulatory submissions in the 5 to 11 age group are now complete in Saudi Arabia and the United Kingdom. On the reimbursement front, we are seeing strong trends in initiations in Germany after reaching a historic reimbursement agreement there as well as continued strong uptake in the U.K., Italy and the Middle East following the negotiations of sustainable access agreements. The CASGEVY story continues to be one of an increasingly robust pipeline of patients initiating the treatment journey. There were more CASGEVY infusions in the first half of 2026 than in all of 2025. Second quarter 2026 was also the third sequential quarter with more than 100 patient initiations, which enhances our visibility to continued growth for the rest of this year and early 2027 as patients continue to move through cell collection, editing and infusion. Quarter-to-quarter variability in CASGEVY revenue will continue and reflects the timing of patient infusions as people choose to receive their infusions when it best suits them. As we look forward, we expect continued CASGEVY momentum with the pipeline of patients at every stage continuing to build. CASGEVY is well positioned to contribute meaningfully to our $500 million non-CF revenue goal this year and to achieve its stand-alone multibillion-dollar potential. Turning to JOURNAVX in moderate-to-severe acute pain, where our launch continues to gain traction. In the second quarter, JOURNAVX generated $50 million in revenue, reflecting sequential revenue growth of approximately 70% and sequential prescription growth of approximately 45% versus quarter 1, 2026. Unpacking Q2 performance, revenue was positively impacted by channel build after we've seen a drawdown in quarter 1. At this stage in an acute product launch, we continue to expect some quarterly volatility in inventory build and drawdown as full-line wholesalers and retail channel buying patterns normalize to reflect formulary adoption, physician awareness and seasonality in elective surgeries. We are building a pain franchise for the long term and are focused on the following 4 critical markers of success: prescription growth, breadth and depth of prescribers, the addition of JOURNAVX to hospital and IDN pathways and broad payer coverage. These are the building blocks of a sustainable, long-term, multibillion-dollar business. We are extremely pleased with the prescription growth we continue to build, which is ahead of our forecast for 2026. The breadth and depth of prescriptions across a wide range of settings of care as well as the clinical impact of JOURNAVX continue to be very strong and all bode well for the long-term growth of JOURNAVX in acute pain. The consequence of this rapid prescription growth is that we are seeing greater use of the PSP program than we forecast as securing unrestricted payer access and physician education catches up with prescription growth. Let me break down what I mean by that. At this point, we have a total of 260 million lives covered out of a total possible of approximately 320 million. Of the 260 million covered lives, 180 million of them have unrestricted coverage. This means that there are 60 million lives yet to be covered and about 80 million lives who have coverage, but with some form of restriction, making some of them eligible for the PSP program. These restrictions are usually very minor in nature, such as a 14-day quantity limit or a prior authorization to indication. As we continue to educate physicians and their office staff about the quantity limits and prior authorizations, we expect the PSP to be triggered less frequently and therefore, more revenue to be recognized. Let me now provide you with some more details on prescriptions, prescribers and access before concluding our thinking on the PSP program and gross-to-net. In terms of prescriptions, quarter 2, 2026 JOURNAVX prescriptions totaled approximately 535,000 and just over 900,000 for the first half of 2026. The prescriptions continue to be split roughly 50-50 between the hospital and retail channels. In both channels, monthly prescriptions were approximately 50,000 in January and doubled to approximately 100,000 in each channel in June. In terms of prescribers, we added approximately 18,000 new HCP prescribers to JOURNAVX in Q2 '26 and are pleased that JOURNAVX is now on 1,400 hospital and 130 IDN pathways in terms of formulary, protocol or order sets. These are important metrics as we seek to convert practices and continue to embed the use of JOURNAVX among our target physicians. We've also made further progress with respect to access. We recently signed agreements to expand reimbursed access to JOURNAVX with 2 additional Medicare Part D plans, effective from July 1. With these additions, 3 of the big 4 Medicare Part D plans now provide covered access alongside the 3 large commercial PBMs. As mentioned, this brings the total covered lives for JOURNAVX to approximately 260 million out of a total possible of 320 million and within that, approximately 180 million lives with unrestricted access. Our goal continues to be to ensure the prescribing experience for physicians and patients is as seamless as possible in a market where the delivery of the medicine is highly time-sensitive. We will continue to work to educate physicians to navigate the minimal quantity limits and prior authorizations that exist and secure ever broader coverage. In the meantime, we will maintain the PSP program so that patients who are prescribed JOURNAVX can get it. We continue to see this as a strategic choice as we seek to convert physician practices away from decades of reliance on opioids to ongoing and sustained use of JOURNAVX for many years to come. As a result, we continue to expect gross to net to normalize in line with other branded oral medicines, but now in the first half of 2027. To conclude on pain, we also continue to be on track to exceed our goal of more than tripling the 550,000 prescriptions and more than tripling revenue from 2025 into 2026 as well as delivering more than $500 million in revenue from CASGEVY and JOURNAVX combined in 2026. Let me conclude with an update on our commercial readiness in renal and specifically Pove in IgAN. With the FDA's acceptance of our BLA and the November 30 PDUFA date, we are in the final stages of commercial launch preparation. We're investing in our renal franchise and the nephrology community for the long term, given our innovative pipeline of multiple potentially transformative kidney disease medications that address the underlying causes of serious renal conditions. Our goal is for Pove to be physicians' first choice among disease-modifying therapies for IgAN and we know from our market research and from nephrologist feedback that physicians are looking for treatments that meaningfully and rapidly reduce proteinuria, have a favorable tolerability profile and offer a seamless treatment experience from access through patient support to convenient dosing. We believe Pove has the winning trifecta of efficacy, tolerability and ease of use for patients and physicians alike. With dual BAFF + APRIL inhibition, Pove has clear best-in-class potential and delivers effectively on all the needs we've heard from the community in research and advisory boards making it the ideal first choice after baseline therapy with ACEi, ARBs and SGLT2s. We have completed the hiring of our renal field force of whom about 90% have nephrology experience and was built with the breadth of our renal pipeline in mind. We anticipate that we will have the largest field force among the novel APRIL or APRIL + BAFF therapies for IgAN. Our payer conversations are also proceeding well. In the U.S., approximately 70% of patients with IgAN have commercial coverage. From our engagements with payers, their awareness of IgAN and the new BAFF + APRIL inhibitors is high. Payers understand the unmet need, have a good understanding of the KDIGO guidelines and how the new therapies fit into treatment pathways. Payers are also very aware of the strength of the Pove Phase III interim analysis data and Pove's November 30 PDUFA date. Our market access teams continue to actively engage with payers to prepare for the upcoming launch of Pove. Additionally, we will provide robust patient support programs drawing on our decades of experience in CF. Pove in IgAN is the first component of our emerging renal franchise, and we're excited to bring it to nephrologists and to their patients. We believe Pove's trifecta of efficacy, tolerability and ease of use delivers exactly what nephrologists are seeking. And just as we've done for over a decade in CF, Pove's success will be driven by a field force delivering a high-science sell, fueled by a potentially best-in-class product, broad reimbursement and robust high-quality patient programs. We are very excited to commercialize Pove in IgAN and begin building a multibillion-dollar renal franchise at Vertex. I'll now turn the call over to Charlie to review the financials. Charles Wagner: Thanks, Duncan. As Reshma noted, Vertex's second quarter results demonstrate our consistent strong performance and attractive growth profile. Second quarter 2026 total revenue of $3.3 billion increased 12% year-over-year with growth balanced between the U.S. and international markets. As expected, Q2 2026 revenue growth reflects an approximate 170 basis point benefit from foreign exchange rates. Q2 '26 Global CF revenue grew 11% year-over-year, and new disease areas also contributed with CASGEVY delivering $76 million compared to $30 million in Q2 of 2025 and JOURNAVX revenue of $50 million compared to $12 million in Q2 of 2025. As a reminder, Q2 '25 results also included $21 million of collaboration revenue. Q2 '26 U.S. CF revenue grew 9% year-over-year, led by strong volume growth from ALYFTREK uptake, continued performance from TRIKAFTA and higher realized net price. Outside the U.S., CF revenue grew 12% year-over-year, driven by strong ALYFTREK launches, timing of orders in certain geographies as well as the benefit from FX. Note that global CF revenue growth for the first half of 2026 was 8%, including the benefit of prior year U.S. price increases and foreign exchange. We expect both of these factors to contribute less to growth in the second half of the year. Our second quarter 2026 gross margin was 85.6%, an expected sequential step-down from Q1 of '26. This step down reflects the impact of product mix as well as manufacturing network investments in various products. As our new products, particularly CASGEVY, increase in revenue contribution with higher cost of goods sold than our small-molecule CF products, we continue to expect full year gross margin of just under 86%, roughly in line with this quarter's result. The impact from product mix and manufacturing network investment costs will be more pronounced in the second half than they were in the first half of 2026. Turning to operating expenses. We continue to invest appropriately given the attractive opportunity presented by our ongoing and near-term launches as well as our attractive mid- and late-stage pipeline. Second quarter non-GAAP R&D expense of $889 million increased 1% year-over-year with steady progress across multiple Phase III studies and the earlier-stage pipeline. Non-GAAP SG&A expense of $520 million increased 45% year-over-year, driven primarily by commercial investment split roughly evenly between pain and renal. We also recorded $21 million in acquired IPR&D expense in the quarter. Note that while R&D continues to account for nearly 2/3 of our operating expenses, the modest growth rate reflects that we are in a period where we can redeploy dollars from programs that wind down to fund programs that are new or scaling up. In contrast, much of our commercial spending is to build new businesses and thus is incremental, as reflected in the higher year-over-year growth rates when compared to R&D spending. Our second quarter 2026 non-GAAP effective tax rate was 21.1%, including some one-time expenses. Year-to-date, our non-GAAP effective tax rate was 20.4%, within our guidance range of 19.5% to 20.5%. Our second quarter 2026 non-GAAP earnings per share of $4.73 represents 5% growth versus prior year, reflecting strong revenue growth as well as investments in our pipeline and commercial capabilities. Turning to the balance sheet. We ended the quarter with approximately $13.6 billion in cash and investments. During the second quarter, we deployed approximately $455 million to repurchase roughly 1 million shares. This activity reflects our ongoing commitment to returning value to shareholders while maintaining the flexibility to act on strategic growth opportunities. Of course, our top priority for capital deployment remains investing in innovation as evidenced by our recent announcement to acquire Crinetics for approximately $8.8 billion net of cash acquired. Now turning to guidance. Given our strong first half performance and the momentum across the business, we are raising our full year 2026 total revenue guidance to a range of $13.1 billion to $13.2 billion, 2026 revenue guidance reflects continued strong performance from the CF franchise, including ALYFTREK and TRIKAFTA as well as growing year-over-year contributions from CASGEVY and JOURNAVX. We continue to expect revenue of $500 million or greater from our non-CF products, and our outlook also continues to include an expected 150 basis point benefit from foreign exchange net of our hedging program. As I previously mentioned, we continue to expect full year gross margin of just under 86%. On operating expenses, we are reiterating our combined non-GAAP operating expense guidance of $5.65 billion to $5.75 billion, though we now expect to be at the high end of that range. This reflects continued investment in our late-stage clinical pipeline and the commercial infrastructure and activities that support our new launches and revenue diversification. We continue to expect our non-GAAP effective tax rate to be in the range of 19.5% to 20.5% for the full year 2026. I would note that today's guidance does not yet reflect the pending Crinetics acquisition, which is expected to close in the third quarter. Given the anticipated timing, we expect the impact to 2026 revenue and non-GAAP operating expenses to be relatively modest, and we will provide updated guidance for 2026 around the time of closing. As a reminder, we expect to fund the transaction through a combination of cash on hand and proceeds from a $4.5 billion term loan, and we expect the transaction to become accretive to non-GAAP operating income in 2029. In summary, Vertex delivered strong second quarter results. Our commercial launches and diversification are gaining momentum, and we continue to invest with discipline in both innovation and commercialization. Overall, our financial performance and outlook remain compelling, with expanding CF leadership, heme and pain scaling, renal on the doorstep of launch and the addition of a fifth pillar in specialty endocrine through the pending Crinetics acquisition, Vertex is exceptionally well positioned for continued growth. Our high success rate in R&D and our disciplined specialty commercial model allow us to maintain industry-leading margins even as we step up investments to support our launches and pipeline. With this unique profile, we are well positioned to continue expanding our impact for patients, investors and all stakeholders. We look forward to updating you on our continued progress across multiple disease areas with key upcoming milestones detailed on Slide 19. I'll now ask Susie to begin the Q&A period. Operator: [Operator Instructions] And our first question for today will come from Salveen Richter with Goldman Sachs. Salveen Richter: Two for me. One is you announced that the Phase II/III OLYMPUS study for Pove in pMN is going to move to Phase III with an 80-milligram dose every 4 weeks. Can you frame what signal this was based on and whether you or the DSMB or what you or the DSMB saw on the Phase IIb portion to move forward? And then on the pain front, it was really nice to see the progress here. Maybe help us understand where the bottlenecks lie now or what needs to be worked on with regard to formulary as well as the payer dynamics as you look at co-pay, et cetera. Reshma Kewalramani: Sure thing, Salveen. Let me kick us off with the first question, which is about Pove in membranous. The Phase II is complete, the Phase III was already initiated, you might recall, a couple of months ago as we designed it as a seamless Phase II/III. The DSMB was asked to base their decision and it was their decision because we do not have access to the unblinded data to look at on efficacy PLA2R, which is the biomarker equivalent in membranous as Gd-IgA1 is to IgAN. Of course, they had full access to the safety results as they made their decision. I suppose in many ways, it's not surprising that they picked the 80-milligram dose given the RUBY-3 results, where you could see that the 80 milligrams had a very nice reduction in PLA2R, but that's how the decision was made. Study is well on its Phase III portion, and we look forward to getting that study enrolled and completed. Duncan, I'm going to turn it over to you for a little commentary on JOURNAVX scripts and what more we're working on. Duncan J. McKechnie: Salveen, so as you know, our goal with JOURNAVX is to fundamentally transform how pain is treated and to move physician practices away from decades of reliance on opioids. In terms of our progress, we're very pleased with the prescription numbers that we're seeing. We are also very pleased with the increased number of hospitals that have adopted JOURNAVX, now 1,400 or so with 130 IDNs, having it on formularies and we have also now secured 2 additional Medicare Part D plans to cover JOURNAVX starting from July 1. So overall, our progress is very well and going very well. And I would add that those prescriptions are coming from a broad range of physician types and being used in a broad range of pain types consistent with our label. In terms of the payer side and access, we're very pleased with the coverage that we've secured to date, 200 -- 260 million lives, and that's ahead of those 2 Medicare Part D plans coming in. And I would say we have obviously more work to do to secure the final elements of access for JOURNAVX. And we also have to make sure that those patients whose physicians might have, say, a quantity limit are able to navigate that in order to ensure the patient can secure access. In the meantime, we have the PSP program in place and anticipate that we'll continue to see prescriptions transition to increasing growth in revenue in the second half of 2026. And indeed, as we've communicated before that our gross to net will ultimately normalize at the same level as our oral branded medicines in the pharmaceutical arena. So we're very happy with the progress. We have a little bit more work to do, but we are very happy with where we're at right now in terms of physician adoption, payer coverage and hospital usage. Operator: The next question will come from Geoff Meacham with Citibank. Geoffrey Meacham: Have 2 quick ones. The first one is CF on 828 or the other assets in Phase I, what are some of the clinical attributes you're looking for? I wasn't sure if you're looking for perhaps a not only better treatment effect or if there is a potential to not need liver monitoring, for example, in future combos. Second question on JOURNAVX. You guys have had substantial discussions with payers, hospital systems, physicians on acute pain. But in these conversations, have you gotten any perspectives or context on DPN like what the clinical profile needs to show as we look to the data end of the year, beginning of next year, what the access and reimbursement could look like in this setting? Reshma Kewalramani: Sure thing, Geoff. Let me take the second question first. On JOURNAVX, we have been hyper-focused on JOURNAVX in acute pain to make sure that we get all of those reimbursement contracts done and get access. So I think it would be just very fair to say, we've spent all of our time hyper focused on acute pain. We'll have more to say on where we are with DPN, the data, what payers are looking for, what doctors are looking for, et cetera, in the coming months. But for here and now, it's acute pain. On VX-828 and the next-gen molecule, so just to give you all of the numbers, VX-828 is the first of the next-gen. The second and third are 581, VX-581 and VX-272. We are looking for potential improvement in efficacy, i.e., more people who can get down to less than 30 millimoles. And of course, we're looking for safety as well. So the monitoring will depend on what the results in the clinical trial are. So sure, there's opportunity for monitoring to be different with this VX-828 program. It just depends on what the actual results are through the clinical trial program. Last thing to say, once daily dosing, really good-looking DDIs as well as other drug-like properties remain really important as I mentioned in my prepared remarks. Operator: The next question will come from Jessica Fye with JPMorgan. Jessica Fye: Maybe for Reshma. I'm curious if you expect to see material differentiation on eGFR across the new IgAN products like Pove and its competitors? And if so, over what time horizon do you think any differentiation on that endpoint would become apparent? Reshma Kewalramani: Sure thing, Jess. As we've discussed before, in IgAN in particular, but you could say this for homogeneous proteinuric kidney diseases in general. Good reductions in proteinuria should, based on everything we know, result in stabilization of GFR. I expect that to be the case with APRIL-BAFF inhibitors as well. I think so that your question is asking a very important second point. And to me, the most important point. What is the differentiation we can expect between various molecules if you have more reduction in proteinuria or hematuria or in the case of IgA nephropathy, Gd-IgA1, these inciting antibodies. And I think for that, the answer is it's really about time to ESRD. That's to say time to dialysis, transplantation or death. And I do expect that the medicine that has the stronger reductions in proteinuria, the medicine that gets more patients to less than 0.5 or 0.3, better improvements in hematuria and Gd-IgA1 are more likely to have an improved profile when it comes to that ultimate endpoint. Proteinuria, 1-year GFR, 2-year GFR, these are all endpoints on the way to the ultimate endpoint. And I think that's where you'll see the real differentiation. Operator: The next question will come from Cory Kasimov with Evercore ISI. Cory Kasimov: Wanted to ask about inaxaplin in the AMPLITUDE study? And what kind of data would be necessary in that interim analysis to file for accelerated approval? Basically, like what constitutes the win with this first data look. Reshma Kewalramani: Sure thing. Cory, I think you're asking about AMPLITUDE. So the core study that's now in Phase III in patients with 2 APOL1 alleles, moderate-to-heavy proteinuria and depressed GFR. We were really pleased and remain very pleased that the agency has provided and we have an agreement with the agency for a potential accelerated approval based on the primary endpoint at the time of the IA, which is 1 year GFR. So that's what our agreement is based on. Obviously, we're also going to look at the proteinuria, but the agreement with the agency for the potential to file for accelerated approval based on the interim analysis is 1-year GFR. Operator: Next question will come from Brian Abrahams with RBC Capital Markets. Brian Abrahams: Congrats on the quarter. On pain, we've seen some data published recently from another NaV1.8. And I'm just curious how you see the acute pain dynamics playing out with additional entrants into the market potentially? And then secondarily, just on zimi. Just wondering if you could talk about the potential impact to launch timing if you do end up syncing the filing with VX-017. Reshma Kewalramani: Yes. Thanks for the kind words, Brian. Maybe I'll do the pain one first and then come on to type 1 diabetes. I did see the publication. And maybe, Brian, what I'd say is that ever since Vertex published VX-150, which you'll remember was the molecule circa 2017 or so. We saw a spike in others following our footsteps and pursuing NaV1.8 as a target. And what I'll say is that we decided not to advance 150 -- VX-150 because we didn't think it had, as I described at the time, the perfect drug-like molecule, properties that we were looking for and we bypassed 150 in favor of what is now suzetrigine or VX-548. So we know the space very well. We know the molecule well, and we know that every time we publish a patent, there is a slew of followers. Maybe if you say, well, what's the takeaway from that, I think that there is a high appetite in the biopharma industry to make nonopioids. There is high unmet need for non-opioid effective pain medicines that have not only the right efficacy, but the right safety tolerability, drug-like properties profile. And I really like where we are well on the market with JOURNAVX, and I'm very much looking forward to the possibility of NaV1.7/NaV1.8 combination. And I've never felt better in Vertex history for the fact that may come to pass for us to be able to bring that to the clinic. Switching then to the type 1 diabetes program. So let me just say what I said in my prepared remarks. I may have gotten a little quick there. The zimislecel program is in Phase I/II/III, back up in dosing. And because it's a type A program, it serves about 60,000 people in the U.S. and Europe. The 017 program because it is Type O, has the potential to serve 120,000 people because it's the universal donor type O. And now what we're trying to do is see if we can get the type O program to go even faster and bring that program out either first or very close behind. That's what we're working on in terms of both the regulatory approach and the commercial approach. I don't have a time line for you today, but we should be able to tell you our exact plans with time lines in the back half of this year. But I am very excited about the opportunity to perhaps bring type O out first or very, very close behind. Operator: Your next question will come from Evan Seigerman with BMO. Evan Seigerman: Congrats on the progress, so you've maintained the expectation for at least $500 million of non-CF revenue this year, while CASGEVY and JOURNAVX delivered roughly $125 million this quarter. So as you think about the path to this target, should we expect that the majority of the upside comes from accelerating patient starts with CASGEVY, continued growth with JOURNAVX or kind of a relatively balanced contribution from both franchises. Reshma Kewalramani: Evan, I'll ask Charlie, if he wants to make any additional comments on our guidance on the $500 million. Charles Wagner: Yes, Evan, thanks. As you pointed out, so far in the first half of the year, CASGEVY and JOURNAVX combined delivered about $200 million in revenue. So we're well on our way to achieving our target of $500 million-plus in that first half. CASGEVY has been a bigger contributor than JOURNAVX, but I'm not willing to give further color on the balance of the year other than to say we're very confident in getting to that $500 million plus. Operator: The next question will come from Michael Yee with UBS. Michael Yee: I guess the IgAN competitor data to your eGFR data is hot off the press, and it's out there on the tape, and you can see that the approved product has essentially a stabilization of eGFR, if not slightly above the baseline. So to what extent Reshma, given that you have an approval coming up soon, should we think about comparing the 2, either from a launch perspective or perhaps given the strong numbers that they're putting up, it speaks to the significant market opportunity and you can get equivalent share? Maybe just talk a little bit about the data that the competitor is putting up and how you think about your launch? Reshma Kewalramani: Sure thing. Mike, the -- I did just see the eGFR data, but I've just seen the top-line number. And as you say, it shows a stabilization right around 0. That is what we should expect given the proteinuria reduction. So that seems very much in line. With regard to what it means for the povetacicept IgAN program, I would say all the more reason if anybody needed a little bit more conviction, you can certainly look at these data that were presented today, look at the proteinuria reduction, look at the GFR and reconfirm for yourself that significant reductions in proteinuria should and have resulted in GFR stabilization. So it makes a lot of sense to me. For what I see for Pove, I see us putting up very strong numbers on proteinuria, numerically, the best out there, 52% change from baseline in terms of proteinuria reduction, 70-plus percent reductions in hematuria and 70-plus percent reductions in Gd-IgA1. That bodes very well for Pove. And then I'll emphasize, Mike, the patient-centric attributes of delivery once-monthly, small-volume, 0.46 ml via an auto-injector. And I think when you put all of that together, real excitement for me for what Pove may bring to patients once the PDUFA date comes and goes, and we have the opportunity to launch. Operator: The next question will come from Tazeen Ahmad with Bank of America. Tazeen Ahmad: Are you still planning on presenting additional data from the RAINIER study this year? And if so, what level of data? And where could that be? And then secondly, for Pove in gMG, it's becoming an increasingly competitive space. So how are you thinking about what additional benefit your drug could provide into this space either with efficacy, safety or dosing frequency? Reshma Kewalramani: Yes, on RAINIER. We are planning to present data. The conferences don't like it when we suggest the name when submissions have been made, but acceptances haven't come through yet. So maybe I'll just leave it at, yes, we plan to present the full RAINIER IA data set. We're looking forward to do so. I'll say at a fall conference, and I'll leave it to your imagination for which one. On gMG and Pove, this one is really exciting. And you gave me 3 options for why we're excited about Pove in gMG: efficacy, safety or patient benefits administration, all 3. This is another one of those trifectas that Duncan has talked about. On efficacy, there is another molecule, a wild-type TACI, so not engineered for optimal potency, binding affinity or tissue distribution. That has already shown substantial efficacy benefit. And remember, that's a wild-type TACI compared to Pove, which is an engineered TACI. So that's on efficacy. On safety, Pove does not need to have a cycle on and a cycle off. That gives real benefit on safety, but that also has the secondary benefit on efficacy because you don't have that off period where the autoantibodies are allowed to return. And the third is same thing, auto-injector. We have to figure out whether it's the 80 milligrams or 240 milligrams, but in either case, it will be auto-injector, once-monthly at-home low-volume dosing. So of your options, I expect Pove to be better across the board on all 3 dimensions. Operator: The next question will come from Philip Nadeau with TD Cowen. Philip Nadeau: There's a lot of focus on the upcoming data from one of your competitors where we're going to get incremental sweat chloride reductions above TRIKAFTA. We're curious to hear Vertex's opinion on how you're going to interpret that data? Is there a level of sweat chloride reduction that would get your attention or Reshma, as you've suggested in the prepared remarks, is it more about simply the proportion of patients who get to less than 30 millimoles per liter and the exact reduction maybe isn't as meaningful because it can be influenced by things like baseline characteristics? Reshma Kewalramani: Yes. Phil, I think you have it right on our perspective. Where we sit today with ALYFTREK. We already know we can get 2/3 of patients to less than 30 millimoles. That's that normal or carrier threshold. And furthermore, if you think about as all physiologic parameters do, there is a Gaussian distribution around that median sweat chloride 30 millimoles. If you superimpose across all age groups, the ALYFTREK data on the carrier data, more than 75% of people across age groups overlap that distribution. So with those kind of data, I think that the bar is exceptionally high and rests on getting more patients to less than 30 millimoles. That is the mark and that's the mark that we or anyone else has to hit in order to have a competitive medicine. And of course, it goes without saying it has to be safe, it has to be well tolerated. It has to have good DDIs, it has to be once daily. But on pure efficacy, it has to be a molecule that gets more patients to less than 30 millimoles in terms of sweat chloride. Operator: The next question will come from Terence Flynn with Morgan Stanley. Terence Flynn: I have another one on inaxaplin. I was just wondering if you can help set expectations for the upcoming AMPLIFIED Phase II trial? And then how to think about any read-through to AMPLITUDE? Reshma Kewalramani: Yes. So AMPLIFIED is the study that's Phase II. It's the expanded AMKD population. By that, I mean, it's the population with 2 APOL1 alleles. And in one arm of the basket study, it's 2 APOL1 alleles diabetes and in the other arm, it's 2 APOL1 alleles, and let's call it, modest proteinuria, so low-grade proteinuria. The way I would frame it up is the study is completed. We are on track for us to be able to share results this fall. And what I'd be looking for and looking to understand is can we derive benefit on proteinuria when you have very modest proteinuria to start with. So this is 0.2 grams to 0.7 grams of protein as opposed to 0.7 grams and above. And of course, it all comes down to what the mean entry baseline level of protein is. Or in the case of diabetes, can we alter the proteinuria when you have second kidney disease involved. These are questions worth studying, but there are clearly different populations than AMPLITUDE, which is why we specifically did not include them in the Phase II original study of inaxaplin and equally why we didn't include them in the Phase III study called AMPLITUDE. So we're super excited to look at these results. We're going to learn a lot and I'm very, very, very happy, and I think you'll see the wisdom of our approach, given what has happened in the field for others to keep these populations, which are expanded populations separate and look at each one individually in this basket AMPLIFIED study. Operator: The next question will come from Mohit Bansal with Wells Fargo. Mohit Bansal: Just maybe a question for Duncan, if you want to help with the prescription trends here for JOURNAVX, so obviously, prescription growth is very strong. But how should we think about the prescribing behavior in terms of how many days of therapy physicians are writing? Has it changed at all in last few quarters or so because it does seem like you have good access, you have good prescription, but probably -- this is probably a missing piece, which could improve here. Duncan J. McKechnie: To answer your question specifically, as I think we've communicated before, in hospitals, the prescription duration is around about 5 days or so. In retail, it's around about 12, 14 days. So on average, you net out at around about 10 or 11 days or so for each JOURNAVX prescription. And candidly, that dynamic has not changed since the launch because it's really driven by the dynamics of the institution that the patient is in rather than anything else. So to answer your question simply, those are the numbers, and it has not changed over the last few months. Operator: The last question will come from Ellie Merle with Barclays. Eliana Merle: So in terms of the DM1 program, what would be good data at the data update in the second half? And how are you thinking about it in the context of the broader competitive landscape in DM1? Reshma Kewalramani: Sure, Ellie. Maybe I can take that one. In DM1, as you know, there hasn't been a clear correlation between the various endpoints that others in the field have looked at, albeit with different approaches. What people have tended to do in their Phase II studies to get an early read is look at splicing, a functional endpoint called vHOT and another functional endpoint called QMT, vHOT is sort of how long does it take to open/close your hand and QMT is a measure of muscle function. And what I would say is that of all of those, splicing is an important one, and we certainly are looking at splicing and these measures of muscle function are also something that we're looking at. The reason I like this approach compared to anything else has more to do with mechanism of action. And that has to do with the fact that it's an oligo, which others are also trying but it's an oligo linked to a circular peptide to a nuclear localizing domain peptide which we believe will allow it to get into the cell and get into the nucleus where it has to do its work. So that is a plus. And I would also say some of the other programs in order to get into the cell have used mechanisms that have some safety tolerability concerns, and that has not been a concern through the circular peptide program that we use. So for the efficacy endpoints in Phase II splicing, and we will also look at these QMT and vHOT endpoints, albeit in small numbers of patients. Operator: And that will conclude our question-and-answer session as well as our conference call for today. Thank you for your participation. A replay will be available shortly after the call concludes by dialing 1 (855) 669-9658 or 1 (412) 317-0088 using replay access code 10208186. Thank you for attending today's presentation. You may now disconnect. Ever feel like you missed the boat in buying the most successful stocks? Then you’ll want to hear this. On rare occasions, our expert team of analysts issues a “Double Down” stock recommendation for companies that they think are about to pop. If you’re worried you’ve already missed your chance to invest, now is the best time to buy before it’s too late. 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Please see our Terms and Conditions for additional details, including our Obligatory Capitalized Disclaimers of Liability. The Motley Fool has positions in and recommends Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. Vertex (VRTX) Q2 2026 Earnings Call Transcript was originally published by The Motley Fool