Exagamglogene Autotemcel

ZUG, Switzerland and BOSTON, Aug. 03, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today reported financial results for the second quarter ended June 30, 2026. "The second quarter reflected strong execution across CRISPR Therapeutics' portfolio and platform," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "CASGEVY's momentum continued to build, highlighted by the FDA's approval for children as young as 2 years old, while we expanded our in vivo pipeline with the initiation of Phase 1 clinical trials for CTX340 for refractory hypertension and CTX460 for alpha-1 antitrypsin deficiency. We enter the second half of 2026 well positioned, with a number of important milestones ahead across our pipeline." Recent Highlights and Outlook Hemoglobinopathies and CASGEVY ® (exagamglogene autotemcel) ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); CASGEVY is a non-viral, ex vivo , CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved in 39 countries across North America, Europe, and the Middle East. CASGEVY generated second quarter 2026 revenue of $76 million, representing 78% growth quarter-over-quarter and 151% growth year-over-year. The U.S. FDA recently approved CASGEVY in children 2 years of age and older with SCD or TDT, the first genetic therapy indicated for children as young as 2 years for both SCD and TDT. With this approval, achieved in just 53 days post filing, approximately 5,500 patients with SCD or TDT may be eligible for treatment with CASGEVY for the first time. Regulatory submissions have also been completed for CASGEVY in the Kingdom of Saudi Arabia (KSA) and the United Kingdom (U.K.) for the treatment of children 5 to 11 years of age. In May, Vertex secured reimbursement for CASGEVY for eligible patients 12 years and older with SCD or TDT in Germany. Efforts remain underway with government and reimbursement authorities globally to ensure sustainable access for eligible patients. CRISPR Therapeutics continues to advance its in vivo hematopoietic stem cell editing approach using lipid nanoparticle (LNP)-mediated delivery. This approach has the potential to expand the addressable patient populations for SCD and TDT. In Vivo Liver Editing CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary liver-directed LNP delivery platform. Development of CTX310 ® , an investigational therapy targeting angiopoietin-related protein 3 (ANGPTL3), continues in a Phase 1b clinical trial, with the Company prioritizing indications in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. U.S. trials have been initiated, with ex-U.S. trials ongoing. CRISPR Therapeutics announced that a late-breaking abstract entitled " Durability of Effects of CTX310, a CRISPR-Cas9 Gene Editing Targeting ANGPTL3 " has been accepted for presentation at the European Society of Cardiology (ESC) Congress 2026 in Munich, Germany, on August 28 at 4:30 p.m. CET to discuss a clinical update of the ongoing Phase 1a trial. The Company also expects to provide a Phase 1b clinical update in the second half of 2026. CTX340™, targeting angiotensinogen (AGT), has received IND clearance from the FDA. The Company has initiated a Phase 1 clinical trial for patients with refractory hypertension. CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate generated from the Company's SyNTase™ editing platform. The Company has initiated a Phase 1 clinical trial for CTX460. CRISPR Therapeutics' pipeline of preclinical in vivo gene editing candidates includes CTX321™, the Company's next-generation LPA program, which is progressing through IND/CTA-enabling studies. The candidate incorporates an optimized guide RNA that delivered approximately two-fold greater potency in preclinical models, paired with the same LNP delivery system used previously. An Lp(a) program update is anticipated in 2026. siRNA-based Programs CRISPR Therapeutics' small interfering RNA (siRNA)-based portfolio includes clinical-stage programs targeting cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics. CTX611 (SRSD107), a long-acting siRNA therapeutic targeting Factor XI (FXI), is advancing through a Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026. CTX611 has the potential to address a broad range of thromboembolic and clotting-related indications, including atrial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), thrombosis in chronic kidney disease (CKD), peripheral vascular disease (PVD), and chronic coronary artery disease (CAD), collectively representing a multi-billion-dollar market opportunity. CRISPR Therapeutics is expected to lead global Phase 3 development, with Sirius Therapeutics overseeing development activities in greater China. CRISPR Therapeutics has the option to nominate up to two additional siRNA targets for research and development. An update is expected in 2026. Autoimmune Disease and Immuno-Oncology Zugocabtagene geleucel (zugo-cel; formerly CTX112™) continues to advance across both autoimmune disease and hematologic malignancies. In autoimmune disease, zugo-cel is currently being evaluated in two ongoing Phase 1 basket trials: a rheumatology basket including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis (IM); and a hematology basket in immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA). In addition, a third Phase 1 trial has been initiated in autoimmune neurologic diseases. The trial, includes progressive multiple sclerosis (PMS), neuromyelitis optica spectrum disorder (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated Disease (MOGAD), N-methyl-D-aspartate receptor (NMDAR) and leucine-rich glioma-inactivated Protein 1 (LGI1) autoimmune encephalitis (AIE), and stiff person syndrome (SPS). Enrollment across the zugo-cel autoimmune clinical program continues in SSc, IIM, SLE, ITP and wAIHA with multiple clinical sites activated globally. The Company expects to provide further updates in the second half of 2026. In immuno-oncology, the Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing, with updates anticipated in the second half of 2026. The Company has also initiated a combination study evaluating zugo-cel with pirtobrutinib in aggressive B-cell lymphomas, under the Company's existing collaboration with Lilly. The Company's autoimmune and immuno-oncology programs are supported by a wholly-owned GMP manufacturing facility in Framingham, Massachusetts. The facility provides end-to-end production capabilities across the cell therapy portfolio, supports both clinical and future commercial supply and enables an industry-leading cost of goods. CRISPR Therapeutics is also advancing a proprietary in vivo CAR-T platform with potential applications across autoimmune disease and oncology. The Company is pursuing two complementary modalities, supported by an antibody-conjugated LNP delivery system that enables targeted delivery to immune cells: a transient, re-dosable CAR-T leveraging engineered mRNA, and a non-viral, integrating CAR-T employing next-generation site-specific integration technologies. Both programs are currently in the IND/CTA-enabling phase, focused on a best-in-class profile. Regenerative Medicine CRISPR Therapeutics continues to advance its regenerative medicine program in diabetes. The Company is developing CTX213™, a deviceless beta cell replacement candidate for Type 1 diabetes, consisting of unencapsulated precursor islet cells derived from edited induced pluripotent stem cells (iPSCs). CTX213 has demonstrated compelling preclinical efficacy through direct administration and is progressing toward the clinic. The Company expects to provide additional updates as development progresses. Second Quarter 2026 Financial Results Cash Position : Cash, cash equivalents, and marketable securities were $2,364.4 million as of June 30, 2026, compared to $1,975.8 million as of December 31, 2025. The increase in cash was primarily driven by net proceeds of $585.4 million from the issuance of convertible senior notes in March 2026, offset by operating expenses. R&D Expenses : R&D expenses were $67.2 million for the second quarter of 2026, compared to $69.9 million for the second quarter of 2025. The decrease in R&D expense was primarily attributable to a decrease in employee-related costs and facility-related expenses, offset by an increase in license fees. Acquired In-Process R&D Expenses : Acquired in-process R&D expenses were $2.5 million for the second quarter of 2026, compared to $96.3 million for the second quarter of 2025. Acquired in-process R&D expenses for the second quarter of 2026 were not material. Acquired in-process R&D expenses for the second quarter of 2025 were related to costs incurred upon entering the Sirius Agreement in 2025. G&A Expenses : General and administrative expenses were $17.6 million for the second quarter of 2026, compared to $18.9 million for the second quarter of 2025. The decrease in G&A expense was primarily attributable to a decrease in employee-related costs, including stock-based compensation expenses. Collaboration Expense : Collaboration expense, net, was $40.3 million for the second quarter of 2026, compared to $45.2 million for the second quarter of 2025. The decrease was primarily attributable to an increase in the Company's share of CASGEVY revenue. Net Loss : Net loss was $91.2 million for the second quarter of 2026, compared to a net loss of $208.5 million for the second quarter of 2025. About CASGEVY ® (exagamglogene autotemcel [exa-cel]) CASGEVY ® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CRISPR Therapeutics – Vertex Collaboration for CASGEVY CRISPR Therapeutics and Vertex established a strategic research collaboration in 2015 to discover and develop therapies leveraging CRISPR/Cas9 technology to address the underlying genetic causes of human disease. CASGEVY is the first approved therapy to emerge from this collaboration. Under an amended agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY, and Vertex and CRISPR Therapeutics share profits and program costs on a 60/40 basis. Vertex is the manufacturer and exclusive license holder of CASGEVY. About In Vivo Liver Editing Programs CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technology. The Company's in vivo portfolio includes three cardiovascular programs: CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), in development for heterozygous and homozygous familial hypercholesterolemia, mixed dyslipidemias, and severe hypertriglyceridemia; CTX340, targeting angiotensinogen (AGT), in development for refractory hypertension; and CTX321, targeting LPA, in development for patients with elevated lipoprotein(a) [Lp(a)]. In addition, the Company's disclosed development candidates also include CTX460™, targeting SERPINA1 using SyNTase editing, for the treatment of alpha-1 antitrypsin deficiency (AATD). About Zugocabtagene Geleucel (zugo-cel; formerly CTX112) Zugocabtagene geleucel (zugo-cel) is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications. The off-the-shelf therapy leverages CRISPR/Cas9 for targeted gene knockout and CAR insertion, enabling immune evasion and enhanced T effector cell potency, and is administered following a standard lymphodepletion regimen without the need for human leukocyte antigen (HLA) matching. Zugo-cel is being investigated in ongoing clinical trials in adult patients with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), inflammatory myositis (IM), immune thrombocytopenic purpura (ITP), warm autoimmune hemolytic anemia (wAIHA), progressive multiple sclerosis (PMS), neuromyelitis optica spectrum disorder (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated Disease (MOGAD), N-methyl-D-aspartate receptor (NMDAR) and leucine-rich glioma-inactivated Protein 1 (LGI1) autoimmune encephalitis (AIE), and stiff person syndrome (SPS), as well as in adult patients with relapsed or refractory B-cell malignancies. About CTX611 (SRSD107) CTX611 is a novel double-stranded, long-acting siRNA, designed to target the human coagulation factor XI, or FXI, messenger RNA and inhibit FXI protein expression. Through modulation of the intrinsic coagulation pathway, CTX611 is intended to provide anticoagulant and antithrombotic effects with a decreased risk of bleeding compared to other anti-thrombotics. Supported by clinical experience conducted by Sirius Therapeutics in two Phase 1 clinical trials, the Company and Sirius Therapeutics are developing CTX611 as a long-acting FXI inhibitor with the potential to support infrequent, including semi-annual, subcutaneous administration. About the CRISPR Therapeutics – Sirius Therapeutics Collaboration for siRNA CRISPR Therapeutics and Sirius Therapeutics entered into a strategic collaboration in 2025 to develop and commercialize siRNA therapies for thromboembolic disorders and other serious diseases. The lead program, CTX611, is a long-acting siRNA targeting FXI, which the companies will co-develop on an equal cost-and-profit-sharing basis. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will lead commercialization in greater China. The collaboration also provides CRISPR Therapeutics with the option to license up to two additional siRNA programs. About CRISPR Therapeutics CRISPR Therapeutics is a leading biopharmaceutical company focused on developing transformative gene-based medicines for serious human diseases. Founded over a decade ago as an early pioneer in CRISPR/Cas9 gene editing, the Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY ® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. Today, CRISPR Therapeutics is advancing a broad, diversified pipeline spanning hemoglobinopathies, cardiovascular disease, autoimmune disease, oncology, regenerative medicine and rare diseases. The Company is also expanding its gene editing toolkit through SyNTase™ editing, its novel, proprietary platform designed to enable precise, efficient, and scalable gene correction. To accelerate its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit . CRISPR THERAPEUTICS ® standard character mark and design logo, CTX112™, CTX213™, CTX310 ® , CTX321™, CTX340™, CTX460™, CTX611™ and SyNTase™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY ® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. CRISPR Special Note Regarding Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned studies and clinical trials for the design and initiation of further studies and clinical trials; (iii) CRISPR Therapeutics strategy, goals, anticipated financial performance and the sufficiency of its cash resources; (iv) plans and expectations for the commercialization of and anticipated benefits of CASGEVY, including anticipated patient access to CASGEVY; (v) regulatory submissions and authorizations, including timelines for and expectations regarding regulatory agency decisions; (vi) the expected benefits of its collaborations; and (vii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9 and SyNTase, as well as other technologies. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This press release also contains information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. This press release discusses investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. Investor Contact: +1-617-307-7503 ir@crisprtx.com Media Contact: +1-617-315-4493 media@crisprtx.com CRISPR Therapeutics AG Condensed Consolidated Statements of Operations (Unaudited, In thousands except share data and per share data) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Revenue: Collaboration revenue $ 10,000 $ — $ 11,000 $ — Grant revenue 181 892 639 1,757 Total revenue 10,181 892 11,639 1,757 Operating expenses: Research and development 67,152 69,894 135,726 142,378 Acquired in-process research and development 2,473 96,253 2,473 96,253 General and administrative 17,553 18,916 34,736 38,212 Collaboration expense, net 40,272 45,153 86,221 102,662 Total operating expenses 127,450 230,216 259,156 379,505 Loss from operations (117,269 ) (229,324 ) (247,517 ) (377,748 ) Total other income, net 27,075 22,067 35,231 35,604 Net loss before income taxes (90,194 ) (207,257 ) (212,286 ) (342,144 ) Provision for income taxes (960 ) (1,292 ) (1,799 ) (2,401 ) Net loss (91,154 ) (208,549 ) (214,085 ) (344,545 ) Foreign currency translation adjustment 6 80 (26 ) 121 Unrealized (loss) gain on marketable securities (2,694 ) (174 ) (9,424 ) 2,080 Comprehensive loss $ (93,842 ) $ (208,643 ) $ (223,535 ) $ (342,344 ) Net loss per common share — basic $ (0.94 ) $ (2.40 ) $ (2.22 ) $ (3.98 ) Basic weighted-average common shares outstanding 96,514,102 87,069,690 96,283,944 86,507,330 Net loss per common share — diluted $ (0.94 ) $ (2.40 ) $ (2.22 ) $ (3.98 ) Diluted weighted-average common shares outstanding 96,514,102 87,069,690 96,283,944 86,507,330 CRISPR Therapeutics AG Condensed Consolidated Balance Sheets Data (Unaudited, in thousands) As of June 30, 2026 December 31, 2025 Cash and cash equivalents $ 291,337 $ 347,559 Marketable securities 2,073,015 1,628,269 Working capital 2,238,874 1,836,551 Total assets 2,650,770 2,265,243 Total shareholders' equity 1,746,551 1,921,813

— Total revenue of $3.33 billion, a 12% increase compared to second quarter 2025 — — Raising full-year revenue guidance to $13.1 billion to $13.2 billion — — Continued progress across research and development pipeline; povetacicept PDUFA date November 30th — — Entered into agreement to acquire Crinetics Pharmaceuticals, with closing anticipated in the third quarter of 2026 — ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the second quarter ended June 30, 2026, and updated its full year 2026 revenue guidance. “Vertex delivered excellent second quarter results, expanding our leadership in cystic fibrosis; delivering strong revenue growth in sickle cell disease, beta thalassemia, and acute pain; and with the pending acquisition of Crinetics, adding rare endocrine diseases as our fifth pillar, further diversifying our portfolio,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “With this breadth of commercial and clinical opportunities, we look forward to bringing more medicines to more patients around the globe and in so doing, creating long-term value.” Second Quarter 2026 Results Total revenue increased 12% to $3.33 billion compared to the second quarter of 2025, primarily driven by the continued performance of cystic fibrosis (CF) therapies and growth from diversification into additional disease areas. In the U.S., total revenue increased 11% to $2.06 billion due to continued strong CF patient demand, including from new initiations of ALYFTREK; higher realized net prices in CF versus the prior year; and contributions from CASGEVY and JOURNAVX. Outside the U.S., total revenue increased 14% to $1.28 billion due to strong CF performance across multiple geographies, including ALYFTREK uptake; increased CASGEVY infusions; and a favorable impact from foreign exchange. Combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses were $1.6 billion and $1.4 billion, respectively, in the second quarter of 2026, compared to $1.4 billion and $1.2 billion, respectively, for the second quarter of 2025. These increases were primarily due to commercial investment to support the launch of JOURNAVX in acute pain and the build-out of the renal franchise, led by povetacicept in IgAN. GAAP and non-GAAP effective tax rates were 21.0% and 21.1%, respectively, compared to 19.5% and 19.4%, respectively, for the second quarter of 2025. GAAP and non-GAAP net income were $1.1 billion and $1.2 billion, respectively, compared to $1.0 billion and $1.2 billion, respectively, for the second quarter of 2025, as a result of increased product revenue, partially offset by increased operating expenses. Cash, cash equivalents, and total marketable securities as of June 30, 2026, were $13.6 billion, compared to $12.3 billion as of December 31, 2025. The increase was primarily due to cash flows from operating activities, partially offset by repurchases of Vertex’s common stock pursuant to its share repurchase program. Full Year 2026 Financial Guidance Vertex today raised its full year 2026 total revenue guidance to $13.1 billion to $13.2 billion from $12.95 billion to $13.1 billion previously. Embedded in this guidance are expectations for continued growth in CF, including the continued global uptake of ALYFTREK and TRIKAFTA, as well as $500 million or more in revenue from non-CF products, namely CASGEVY and JOURNAVX. Total revenue guidance continues to reflect a year-over-year benefit of approximately 150 basis points in growth from foreign exchange rates, net of Vertex’s foreign exchange hedging program. Vertex reiterated its full year 2026 guidance for combined GAAP and non-GAAP R&D, AIPR&D, and SG&A expenses of $6.3 billion to $6.45 billion and $5.65 billion to $5.75 billion, respectively. This guidance also continues to include an immaterial cost impact from tariffs in 2026 based on currently known tariff rates and regulations. Vertex’s financial guidance for 2026 does not reflect the impact of the pending acquisition of Crinetics Pharmaceuticals. Vertex continues to anticipate completion of the acquisition in the third quarter of 2026, subject to customary closing conditions, and will provide updated guidance for 2026 following transaction close. Vertex’s financial guidance is summarized below: Current FY 2026 Previous FY 2026 Total revenue $13.1 to $13.2 billion $12.95 to $13.1 billion Non-CF product revenue Unchanged $0.5 billion or greater Combined GAAP R&D, AIPR&D and SG&A expenses * Unchanged $6.3 to $6.45 billion Combined non-GAAP R&D, AIPR&D and SG&A expenses* Unchanged $5.65 to $5.75 billion Non-GAAP effective tax rate Unchanged 19.5% to 20.5% *The difference between the combined GAAP R&D, AIPR&D and SG&A expenses and the combined non-GAAP R&D, AIPR&D and SG&A expenses guidance relates primarily to $650 million to $700 million of stock-based compensation expense. **Combined GAAP and non-GAAP R&D, AIPR&D and SG&A expenses guidance includes approximately $100 million of AIPR&D expenses. Key Business Highlights Marketed Products Cystic Fibrosis (CF) Portfolio Vertex has worked for more than 20 years to discover and develop medicines to treat the underlying cause of CF. Vertex CFTR modulators can treat approximately 95 percent of all people living with CF in core markets, including patients as young as one month old. Our CF medicines are accessible in more than 60 countries across six continents. Recent highlights include: Vertex presented new data on ALYFTREK at the European Cystic Fibrosis Conference demonstrating the potentially transformative impact of treating cystic fibrosis with ALYFTREK in children 2 to 5 years of age. The Phase 3 data set shows that 65% of children in the study, including those with F/F and F/MF genotypes, reached carrier sweat chloride levels of < 30 mmol/L, with a mean reduction in sweat chloride of 9.6 mmol/L from a baseline on TRIKAFTA. ALYFTREK was generally safe and well tolerated. Vertex recently initiated global regulatory submissions for this age group. In the second quarter of 2026, Vertex secured reimbursement for ALYFTREK in four additional countries, including Spain, bringing the total number of countries where ALYFTREK is reimbursed to 25. Vertex also signed a letter of intent with the Pan-Canadian Pharmaceutical Alliance for reimbursement of ALYFTREK for eligible patients 6 years and older in Canada. Vertex has submitted numerous abstracts for presentation at the North American Cystic Fibrosis Conference in October, including data on reductions in sweat chloride with CFTR modulators, reduced need for pancreatic enzyme replacement therapy with ALYFTREK, and evidence of the positive impact of ALYFTREK on quality of life, treatment burden, treatment experience and overall health collected through patient-reported outcomes. CASGEVY for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved in 39 countries across North America, Europe, and the Middle East. Recent highlights include: Vertex recorded second quarter 2026 CASGEVY revenue of $76 million, representing 78% quarter-over-quarter sequential growth and 151% growth compared to the second quarter of 2025. The U.S. FDA recently approved CASGEVY in children 2 years of age and older with SCD or TDT, making it the first genetic therapy indicated for children as young as 2 years for both SCD and TDT. With this approval, achieved in just 53 days post filing, approximately 5,500 patients with SCD or TDT may be eligible for treatment with CASGEVY for the first time. Vertex has also completed regulatory submissions in KSA and the U.K. for the treatment of children 5 to 11 years of age. In May, Vertex secured reimbursement for CASGEVY for eligible patients 12 years and older with SCD or TDT in Germany. Vertex remains committed to working with government and reimbursement authorities globally to ensure sustainable access for eligible patients. JOURNAVX (suzetrigine) for the treatment of moderate-to-severe acute pain JOURNAVX is a first-in-class, oral, selective, non-opioid Na V 1.8 pain signal inhibitor, approved in the U.S. for the treatment of adults with moderate-to-severe acute pain. Recent highlights include: Vertex recorded second quarter 2026 JOURNAVX revenue of $50 million, representing 71% quarter-over-quarter sequential growth and more than quadrupling compared to $12 million in the second quarter of 2025. In the second quarter and first six months of 2026, approximately 535,000 and approximately 900,000 prescriptions, respectively, have been filled for JOURNAVX across the hospital and retail settings. Vertex has reached agreements with two additional major pharmacy benefit managers (PBMs) for Medicare Part D coverage of JOURNAVX. As a result, seniors covered by three of the four major Medicare Part D PBMs now have reimbursed access. Twenty-three states provide coverage for JOURNAVX via Medicaid. In total, approximately 260 million individuals in the U.S. now have reimbursed access to JOURNAVX across a wide range of commercial and government payers. During the second quarter, Health Canada accepted Vertex’s new drug submission for suzetrigine for the treatment of moderate-to-severe acute pain, and review is underway. Real world evidence in support of JOURNAVX (suzetrigine) continues to build, including the recent publication of “Suzetrigine as Part of Multimodal Therapy Enables Opioid-Free Recovery After Aesthetic or Reconstructive Procedures,” in the journal Plastic and Reconstructive Surgery . Select R&D Pipeline Programs Cystic Fibrosis Consistent with its commitment to serial innovation and bringing as many patients as possible to carrier levels of CFTR function, Vertex is evaluating VX-828, the first of the next-generation 3.0 CFTR corrector class, in a proof-of-concept study in people with CF. Vertex completed dosing in this study and is on track to share results in the second half of 2026. Vertex is enrolling and dosing first-in-human studies in healthy volunteers with VX-581 and VX-272, additional next-generation 3.0 CFTR correctors. Vertex expects to study all three correctors in early-stage trials in people with CF and advance the best asset into further development. Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia Vertex continues to make progress on advancing preclinical assets for gentler conditioning for CASGEVY toward clinical trials. Gentler conditioning could broaden the eligible patient population for CASGEVY. Acute and Peripheral Neuropathic Pain (PNP) Vertex is on track to complete enrollment in both Phase 3 studies of suzetrigine in diabetic peripheral neuropathy (DPN), a form of peripheral neuropathic pain (PNP), by the end of 2026. Vertex is on track to complete enrollment in a Phase 2 study of VX-993 in people with DPN by the end of 2026. Vertex continues to advance toward clinical trials preclinical assets that inhibit Na V 1.7 for use alone or in combination with a Na V 1.8 inhibitor in acute and neuropathic pain. IgA Nephropathy (IgAN) and Other B Cell-Mediated Diseases Vertex is developing povetacicept for multiple diseases. Povetacicept is a dual inhibitor of the BAFF and APRIL cytokines, which play key roles in the pathogenesis of multiple B cell-mediated autoimmune diseases. Povetacicept has pipeline-in-a-product potential and represents a potentially best-in-class approach to control B cell activity in IgAN, primary membranous nephropathy (pMN), and generalized myasthenia gravis (gMG). The U.S. FDA accepted the BLA submission for accelerated approval of povetacicept for adults with IgAN and assigned a PDUFA target action date of November 30, 2026. The submission is supported by positive data from a pre-specified Week 36 interim analysis of the ongoing Phase 3 RAINIER trial of povetacicept in IgAN. If approved, povetacicept will become the first commercialized therapy in Vertex’s emerging nephrology pillar. Vertex has completed its regulatory submission for accelerated approval of povetacicept in adults with IgAN in the Kingdom of Saudi Arabia, and the Saudi Food and Drug Authority has granted povetacicept Breakthrough Designation. Vertex completed the Phase 2B portion of the Phase 2/3 OLYMPUS pivotal study of povetacicept in people with pMN and confirmed the dose selection of 80 mg subcutaneously every four weeks for the Phase 3 portion, which is underway. The FDA has granted Fast Track and Orphan Drug designations for povetacicept in pMN, and the EMA has granted Priority Medicines (PRIME) designation. Vertex continues to enroll and dose the placebo-controlled, Phase 2 dose-ranging proof-of-concept study evaluating povetacicept for the treatment of gMG. Vertex is on track to complete enrollment in this study by the end of 2026. APOL1-Mediated Kidney Disease (AMKD) Vertex has discovered and advanced multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that targets the underlying cause of this genetic kidney disease. The AMPLITUDE study is on track to complete full enrollment in the second half of 2026. Vertex is on track to share data from the interim analysis of the AMPLITUDE study in early 2027. Vertex will conduct the pre-planned interim analysis for potential U.S. accelerated approval after the interim analysis cohort reaches 48 weeks of treatment. Vertex has completed enrollment and dosing in the AMPLIFIED Phase 2 study of inaxaplin and is on track to share data this fall. AMPLIFIED is a study of inaxaplin in people with AMKD with moderate proteinuria, and people with AMKD and Type 2 diabetes — populations not being studied in the AMPLITUDE trial. Type 1 Diabetes (T1D) Vertex is evaluating stem cell-derived, fully differentiated islet cell therapies for patients suffering from T1D, with the goal of developing a potential one-time functional cure for this disease. The Phase 1/2/3 study of zimislecel in people with T1D continues to enroll and dose patients. The IND application for VX-017 has been cleared by the FDA, and Vertex plans to initiate a Phase 1/2 trial to evaluate the safety and efficacy of VX-017 in people with T1D in the near term. VX-017 is a stem cell-derived, fully differentiated islet cell therapy designed to treat all eligible patients with T1D, regardless of blood type. Following recent positive regulatory interactions, the company expects to provide updated timelines for the zimislecel and VX-017 programs later this year . Autosomal Dominant Polycystic Kidney Disease (ADPKD) Vertex is developing small molecule correctors that restore function to polycystin 1 (PC1) protein variants, with the goal of addressing the underlying cause of ADPKD. Vertex has completed enrollment in AGLOW, a Phase 2 study of VX-407 in patients with a subset of variants in the PKD1 gene, which encodes the PC1 protein, estimated to be up to approximately 30,000 (or up to approximately 10%) of the overall patient population living with ADPKD. AGLOW is a 26-patient, single-arm, 52-week, Phase 2 proof-of-concept study that will evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV). Myotonic Dystrophy Type 1 (DM1) Vertex is evaluating multiple approaches that target the underlying cause of DM1. Vertex’s lead approach, VX-670, is an oligonucleotide linked to a cyclic peptide, which holds the potential to promote effective delivery into cells and address the causal biology of DM1. Vertex recently completed enrollment and continues to dose people with DM1 in the multiple ascending dose portion of the GALILEO global Phase 1/2 clinical trial of VX-670. The study is assessing both safety and preliminary efficacy, as measured by the change from baseline in the splicing index on muscle biopsy as well as other endpoints, including those that evaluate muscle function and strength, such as video hand opening time (vHOT) and quantitative muscle testing (QMT) score . Vertex is on track to complete dosing in the trial and share results in the second half of 2026. Additional Earlier Stage R&D Programs Consistent with its overall strategy, Vertex takes a serial innovation approach to all of its programs, with additional assets or approaches across its portfolio. Investments in External Innovation In July, Vertex and Crinetics Pharmaceuticals entered into a definitive agreement under which Vertex will acquire Crinetics for $85.00 per share in cash, for a total equity value of approximately $10.0 billion, or approximately $8.8 billion net of estimated cash acquired. The transaction is expected to close in the third quarter of 2026. Conference Call and Webcast 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 Second 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 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. Non-GAAP Financial Measures In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (i) stock-based compensation expense, (ii) intangible asset amortization expense, (iii) gains or losses related to the fair value of the company's strategic investments, (iv) increases or decreases in the fair value of contingent consideration, (v) an intangible asset impairment charge, and (vi) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company’s calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information. The company provides guidance regarding combined R&D, AIPR&D and SG&A expenses and effective tax rate on a non-GAAP basis. Unless otherwise noted, the guidance regarding combined R&D, AIPR&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The guidance does not reflect the impact of the pending acquisition of Crinetics Pharmaceuticals. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material. Vertex Pharmaceuticals Incorporated Consolidated Statements of Income (unaudited, in millions, except per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Revenues: Product revenues, net $ 3,333.9 $ 2,944.0 $ 6,320.8 $ 5,704.2 Other revenues — 20.7 — 30.7 Total revenues 3,333.9 2,964.7 6,320.8 5,734.9 Costs and expenses: Cost of sales 489.2 407.5 882.0 770.5 Research and development expenses 993.8 978.4 1,955.4 1,958.1 Acquired in-process research and development expenses 21.4 2.2 21.9 22.0 Selling, general and administrative expenses 582.2 424.6 1,075.9 821.0 Intangible asset impairment charge — — — 379.0 Change in fair value of contingent consideration 0.4 0.9 0.6 3.1 Total costs and expenses 2,087.0 1,813.6 3,935.8 3,953.7 Income from operations 1,246.9 1,151.1 2,385.0 1,781.2 Interest income, net 120.6 118.7 235.4 236.6 Other income (expense), net 24.3 13.2 24.3 (4.4 ) Income before provision for income taxes 1,391.8 1,283.0 2,644.7 2,013.4 Provision for income taxes 292.0 250.1 513.5 334.2 Net income $ 1,099.8 $ 1,032.9 $ 2,131.2 $ 1,679.2 Net income per common share: Basic $ 4.34 $ 4.02 $ 8.39 $ 6.54 Diluted $ 4.31 $ 3.99 $ 8.33 $ 6.48 Shares used in per share calculations: Basic 253.7 256.7 253.9 256.8 Diluted 255.2 258.9 255.7 259.2 Vertex Pharmaceuticals Incorporated Total Revenues (unaudited, in millions) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 TRIKAFTA/KAFTRIO $ 2,497.2 $ 2,551.1 $ 4,851.9 $ 5,086.6 ALYFTREK 573.6 156.8 998.0 210.7 Other CF product revenues (1) 137.1 193.7 273.0 349.0 Total CF product revenues, net 3,207.9 2,901.6 6,122.9 5,646.3 CASGEVY 76.4 30.4 119.3 44.6 JOURNAVX 49.6 12.0 78.6 13.3 Product revenues, net 3,333.9 2,944.0 6,320.8 5,704.2 Other revenues — 20.7 — 30.7 Total revenues $ 3,333.9 $ 2,964.7 $ 6,320.8 $ 5,734.9 1: Includes KALYDECO, ORKAMBI, and SYMDEKO/SYMKEVI Vertex Pharmaceuticals Incorporated Reconciliation of GAAP to Non-GAAP Financial Information (unaudited, in millions, except percentages) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 GAAP cost of sales $ 489.2 $ 407.5 $ 882.0 $ 770.5 Stock-based compensation expense (3.8 ) (2.5 ) (7.0 ) (5.1 ) Intangible asset amortization expense (5.1 ) (5.1 ) (10.1 ) (10.1 ) Non-GAAP cost of sales $ 480.3 $ 399.9 $ 864.9 $ 755.3 GAAP research and development expenses $ 993.8 $ 978.4 $ 1,955.4 $ 1,958.1 Stock-based compensation expense (104.4 ) (99.6 ) (206.1 ) (199.7 ) Intangible asset amortization expense (0.7 ) (0.7 ) (1.3 ) (1.3 ) Non-GAAP research and development expenses $ 888.7 $ 878.1 $ 1,748.0 $ 1,757.1 Acquired in-process research and development expenses $ 21.4 $ 2.2 $ 21.9 $ 22.0 GAAP selling, general and administrative expenses $ 582.2 $ 424.6 $ 1,075.9 $ 821.0 Stock-based compensation expense (62.0 ) (65.2 ) (123.5 ) (128.6 ) Non-GAAP selling, general and administrative expenses $ 520.2 $ 359.4 $ 952.4 $ 692.4 Combined non-GAAP R&D, AIPR&D and SG&A expenses $ 1,430.3 $ 1,239.7 $ 2,722.3 $ 2,471.5 GAAP other income (expense), net $ 24.3 $ 13.2 $ 24.3 $ (4.4 ) (Increase) decrease in fair value of strategic investments (37.2 ) (5.4 ) (35.2 ) 9.6 Non-GAAP other (expense) income, net $ (12.9 ) $ 7.8 $ (10.9 ) $ 5.2 GAAP provision for income taxes $ 292.0 $ 250.1 $ 513.5 $ 334.2 Tax adjustments (2) 31.7 32.1 90.3 192.2 Non-GAAP provision for income taxes $ 323.7 $ 282.2 $ 603.8 $ 526.4 GAAP effective tax rate 21.0 % 19.5 % 19.4 % 16.6 % Non-GAAP effective tax rate 21.1 % 19.4 % 20.4 % 19.1 % Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com or 617-961-7163 Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 Read full story here