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Business Wire
9 hours ago
- Business
- Business Wire
Vertex Presents Positive Data for Zimislecel in Type 1 Diabetes at the American Diabetes Association 85th Scientific Sessions
BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced simultaneous presentation and publication of updated data from the Phase 1/2 portion of the Phase 1/2/3 FORWARD-101 clinical trial of zimislecel (VX-880), an investigational stem cell-derived, fully differentiated islet cell therapy, in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). The data were featured in an oral presentation at the American Diabetes Association (ADA) annual conference in Chicago as part of the symposium, 'Innovation and Progress in Stem Cell-Derived Islet-Cell Replacement Therapy,' from 6:15-6:30 p.m. CT (abstract 2025-A-1921) and published online by the New England Journal of Medicine. The data are from 12 patients who received the full dose of zimislecel as a single infusion and were followed for at least one year, as of October 2024. Results from the study to date continue to demonstrate the transformative potential of zimislecel with consistent and durable patient benefit with longer follow-up. All 12 participants: Demonstrated engraftment with glucose-responsive endogenous C-peptide production, which was durable through one year of follow-up. Achieved the ADA targets of HbA1c <7% and time in range of >70%. Were free of SHEs from day 90 onwards. Had a reduction in exogenous insulin use (mean reduction in daily insulin dose: 92%). 10/12 (83%) no longer required exogenous insulin at Month 12. Achieved the Phase 1/2 primary endpoint of elimination of SHEs with HbA1c <7%. Zimislecel continues to be generally well tolerated. Most adverse events (AEs) were mild or moderate, and there were no serious AEs related to zimislecel treatment. As previously reported, two patient deaths occurred, both unrelated to treatment with zimislecel. The safety profile is generally consistent with the immunosuppressive regimen used in the study, the infusion procedure, and complications from long-standing diabetes. 'These data on the first fully differentiated, stem cell-derived, off-the-shelf islet cell therapy continue to be unprecedented. The magnitude, consistency and durability of the results from all 12 patients with more than one year of follow-up reinforce the transformative potential of zimislecel for people living with T1D complicated by severe hypoglycemia,' said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. 'We are excited to complete enrollment and dosing in the Phase 1/2/3 Program and look forward to potential regulatory submissions next year.' 'It's remarkable to see 12 out of 12 patients with baseline HbA1c above 7% and multiple severe hypoglycemic events reach consensus targets for glycemic control by both HbA1c and time in range as well as elimination of severe hypoglycemic events,' said Michael R. Rickels, M.D., M.S., Medical Director, Pancreatic Islet Cell Transplant Program, Willard and Rhoda Ware Professor in Diabetes and Metabolic Diseases, Presenting Author and Steering Committee Co-Chair for the zimislecel clinical program, and author on the New England Journal of Medicine paper. 'As I think about my patients and the unmet need in the type 1 diabetes community, the results we've seen so far for restoring endogenous insulin secretion with a stem cell-derived islet therapy bring me hope and confidence for a transformative treatment option for individuals with type 1 diabetes in the not-so-distant future.' About Type 1 Diabetes T1D results from the autoimmune destruction of insulin-producing beta cells in pancreatic islets. Insulin deficiency results in hyperglycemia and can lead to acute life-threatening complications such as diabetic ketoacidosis. People with T1D are reliant on lifelong treatment with exogenous insulin that requires careful monitoring of blood glucose levels. Even with the availability of advanced exogenous insulin delivery and glucose monitoring systems, people with T1D can have periods of very low and very high blood sugar levels. Exogenous insulin has a narrow therapeutic range and carries an inherent risk of causing low blood sugar levels or hypoglycemic events, which can potentially result in arrhythmias, seizures, coma and even death. Due to the limitations and complexities of exogenous insulin treatment, it can be difficult for people with T1D to achieve and maintain good glucose control. Exposure to prolonged periods of high blood glucose levels, or hyperglycemia, can lead to long-term complications such as nerve damage, kidney disease/failure, eye disease (including vision loss), cardiovascular disease, stroke and even death. HbA1c is a measure of average blood glucose over the most recent ~2-3 months, and the consensus guidance is to maintain an HbA1c of <7% to reduce the risk of long-term complications; only ~1 in 4 people with T1D globally meet this clinical target. Current standards of care do not address the underlying cause of the disease and leave people with T1D susceptible to both hypo- and hyperglycemia and their associated morbidity and mortality. There is no cure for T1D. About Zimislecel Zimislecel (VX-880) is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. Zimislecel is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. Zimislecel has the potential to restore the body's ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose-responsive insulin production. Zimislecel is delivered by an infusion into the hepatic portal vein and requires chronic immunosuppressive therapy to protect the islet cells from immune rejection. The zimislecel trial has expanded to additional sites that are currently active and enrolling in the U.S., Canada and Europe. Zimislecel has been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration, Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), and has secured an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency (MHRA). Zimislecel is investigational and has not been approved by health authorities globally. 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 and acute pain, 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 neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes 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 15 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 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, (i) statements by Carmen Bozic, M.D., and Michael R. Rickels, M.D., M.S., in this press release, (ii) plans, expectations for, and the potential benefits of zimislecel, (iii) expectations for the Phase 1/2/3 clinical trial for zimislecel, including expectations for the trial to complete enrollment and dosing, and (iv) plans for potential regulatory submissions next year. 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 a limited number of patients may not be indicative of final clinical trial results, that data from the company's research and development programs may not support registration or further development of its potential medicines in a timely manner, or at all, due to safety, efficacy, that timelines for regulatory submissions may be longer than anticipated, 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 and available through the company's website at 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) Vertex will host an investor event on Friday, June 20, 2025, at 7:15 p.m. CT/8:15 p.m. ET, in Chicago, to discuss the positive zimislecel data in type 1 diabetes. A live webcast of the presentation and Q&A portions can be accessed through the Investor Relations section of Vertex's website at An archived webcast will be available on the company's website.
Yahoo
07-03-2025
- Business
- Yahoo
Vertex Announces UK MHRA Approval of ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor), a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis
- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including additional mutations not previously approved with other CFTR modulator therapies - - In head-to-head clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior on ppFEV1 and further decreased sweat chloride compared to ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor - LONDON, March 07, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals (Nasdaq: VRTX) announced today that the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor), a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatment for people living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. "For more than 20 years we have been focused on discovering medicines that treat the underlying cause of the disease with the goal of helping people live longer and better lives. The approval of ALYFTREK, our fifth CFTR modulator regimen, represents another significant milestone in that journey for people with CF in the UK," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "The deutivacaftor/tezacaftor/vanzacaftor Phase 3 trial results showed that it is possible to further improve CFTR protein function with this once-a-day, more flexible and less burdensome regimen," said Professor Alex Horsley, Professor of Respiratory Medicine at the University of Manchester, UK. "Children and adults taking the new triple combination therapy were more likely to have carrier levels of sweat chloride compared to those on the ivacaftor/tezacaftor/elexacaftor regimen, which we hope will translate to reduced risk of developing CF-related complications in the long term." Vertex is working with the National Institute for Health and Care Excellence (NICE) and the NHS to ensure eligible patients can access this new therapy, which treats the underlying cause of CF, as soon as possible. This medicine was approved for patients ages 6 years and older by the U.S. FDA in December 2024 and Marketing Authorization Applications are ongoing with the European Medicines Agency (EMA) and the regulatory authorities in Canada, Switzerland, Australia and New Zealand in the same age group. About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving. Today Vertex CF medicines are treating over 68,000 people with CF across more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). Higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better and more stable lung function, fewer pulmonary exacerbations, better quality of life and improved survival. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease. About ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane. ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) is approved in the UK for the treatment of CF in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. For complete product information, please see the Summary of Product Characteristics that can be found on 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 and acute pain, 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 neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes 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 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. Vertex 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 by Carmen Bozic, M.D., and Professor Alex Horsley, in this press release, and statements regarding expectations for and the anticipated benefits of ALYFTREK, Vertex's work with NICE and the NHS to ensure eligible patients can access this new therapy as soon as possible, and the status of the Marketing Authorization Applications for ALYFTREK with global regulatory authorities. While we believe 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 development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, 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 and available through the company's website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@ Media: mediainfo@ orInternational: +44 20 3204 5275 Sign in to access your portfolio
