Latest news with #Zimislecel
Yahoo
5 hours ago
- Business
- Yahoo
Vertex Pharmaceuticals Presents 'Unprecedented' Data for T1D Cell Therapy Zimislecel, Shows Durable Benefit
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of the best biotech stocks to invest in now. On June 20, Vertex Pharmaceuticals announced the 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). Zimislecel is an investigational stem cell-derived, fully differentiated islet cell therapy for individuals with type 1 diabetes (T1D) who experience impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). The data were presented at the American Diabetes Association/ADA annual conference in Chicago, specifically during the symposium 'Innovation and Progress in Stem Cell-Derived Islet-Cell Replacement Therapy' and were simultaneously published online by the New England Journal of Medicine. The presented data included 12 patients who received a full dose of zimislecel as a single infusion and were followed for at least one year, as of October 2024. A pharmacist delivering a specific medication to a patient in a specialty pharmacy. The results consistently demonstrated the transformative potential of zimislecel, showing durable patient benefit with longer follow-up. All 12 participants exhibited engraftment with glucose-responsive endogenous C-peptide production, which remained durable throughout one year of follow-up. Zimislecel was generally well-tolerated, with most adverse events being mild or moderate. Type 1 diabetes (T1D) is an autoimmune disease where the body's insulin-producing beta cells in pancreatic islets are destroyed, leading to insulin deficiency and hyperglycemia. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that develops and commercializes therapies for treating cystic fibrosis/CF. While we acknowledge the potential of VRTX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Daily Mail
a day ago
- Health
- Daily Mail
Major breakthrough as 10 patients have their diabetes CURED with new drug
Ten people have been effectively cured of their type 1 diabetes after a breakthrough infusion of stem cells on the path toward FDA approval. One year after being treated, 10 of the 12 patients who took the drug, called Zimislecel, no longer needed insulin, while the other two needed much smaller doses. The groundbreaking therapy's foundation uses stem cells that researchers manipulated to become pancreatic islet cells, tiny clusters of specialized cells scattered throughout the pancreas that produce hormones to regulate blood sugar. The cells were injected into the patients, traveling through the liver, implanted there, and began producing insulin where their bodies had previously produced none. Their blood sugar spikes were less severe after meals. Their insulin production also kept improving, and their time spent in a healthy glucose range went from about 50 percent at baseline to over 93 percent at one year. The study's participants are among the 30 percent of type 1 diabetes patients with a complication that makes it impossible for them to tell when their blood sugar is low or high, lacking the normal signs like shakiness or sweating. All of the patients in the study had this subtype of type 1, known as hypoglycemic unawareness. The condition can also cause patients to pass out, have seizures, or even die. Researchers behind the study believe their drug paves the way toward a cure for type 1 diabetes overall. The condition, which is due to a combination of genetics and environmental factors like childhood viral infections, affects roughly 1.6 million Americans. Trevor Reichman, a study co-author and surgeon at University Health Network in Toronto, told STAT: 'This study represents for the first time that biologic replacement can be administered to patients with type 1 diabetes in a single safe and effective procedure with minimal risk to the recipient.' 'This study has the potential to get us one step closer to a 'functional cure' for patients with type 1 diabetes,' Reichman added. Researchers expect to apply for approval of this drug with the FDA within the next five years. Patients had to have a history of hypoglycemic unawareness to participate in the study, causing seizures, coma, loss of consciousness, or hospital stays. Once they started taking Zimislecel, made by Vertex Pharmaceuticals of Boston, the patients also had to take immune-suppressing drugs to prevent the body from attacking the foreign islet cells. One study enrollee, Amanda Smith, 36, from London, told the New York Times that she jumped at the chance to join the groundbreaking trial. Six months after receiving the infusion, she no longer needed insulin. 'It's like a whole new life,' she said. Traditionally, stem cells are isolated from the pancreas of a deceased organ donor. However, the cells in the latest research were grown in the lab rather than taken from cadavers, offering a scalable, renewable source of islet cells without having to rely on a limited supply of donors. Type 1 is less common than type 2 diabetes, which affects 32 million Americans and typically comes on later in life due to a confluence of lifestyle factors and genes. Without insulin, type 1 diabetics' bodies have no way to regulate blood sugar, which can build up in the bloodstream and skyrocket. The body starts breaking down fat for fuel, creating ketones, or acidic byproducts. A buildup of ketones in the blood can cause diabetic ketoacidosis, a condition that causes nausea, vomiting, rapid breathing, dehydration, and confusion. Without proper treatment with insulin and fluids, diabetic ketoacidosis can cause a laundry list of potentially fatal effects, including brain swelling, kidney failure, cardiac arrest, and death. This therapy is 25 years in the making, pioneered by the father of a baby who was diagnosed with type 1 diabetes, followed by his teen daughter. He pledged to find a cure for the disease. Their findings were published in the New England Journal of Medicine. The first patient to receive this therapy was Brian Sheton, who got it in 2021. He had been living with low blood sugar that often plunged him into a state of unconsciousness, even crashing his motorcycle into a wall at one point. The infusion cured him, but Vertex said he died afterward due to dementia symptoms that were present before he was treated. Stem cell therapy is the newest frontier in disease research, starting with niche conditions, such as hypoglycemic unawareness. Still, it has the potential to be scaled up to cover a broader umbrella of diseases. After 25 years of taking insulin shots, Illinois mom Marlaina Goedel finally said what she had longed to say for years: 'I am cured.' The 30-year-old was diagnosed with type 1 diabetes at five, and became one of a handful of people to have received an islet cell transplant. Her blood sugar normalized within a month after the infusion, and she no longer needed insulin injections. Now, she's chasing long-postponed dreams: riding her horse, going back to school, and soaking in the sweetness of a life no longer dictated by blood sugar numbers. 'We hope in the next five to 10 years that this therapy will have the potential to be given with minimal or zero immunosuppression, further minimizing the risk for patients long-term,' Dr Reichman said, adding that more research is still needed on a larger population.
Yahoo
28-03-2025
- Business
- Yahoo
Vertex Announces Program Updates for Type 1 Diabetes Portfolio
- VX-264 Phase 1/2 enrollment and dosing complete in Parts A and B: VX-264 was generally safe and well tolerated; efficacy data are not supportive of further clinical advancement - - Zimislecel (VX-880) pivotal trial on track to complete enrollment and dosing in H1 2025; Vertex expects to submit marketing applications to global regulators in 2026 - - Continue to progress multiple novel, research-stage immunoprotective approaches - BOSTON, March 28, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced several updates on the Company's type 1 diabetes (T1D) portfolio. VX-264 UpdateVertex has completed enrollment and dosing in Parts A and B of the Phase 1/2 VX-264 (cells + device) study and the planned analysis at Day 90 for Part B. In Part B of the study, participants received the full dose of the investigational fully differentiated pancreatic islet cell therapy encapsulated in a proprietary immunoprotective device. There were two primary endpoints in Part B, safety and change in peak C-peptide during a mixed-meal tolerance test (MMTT) from baseline at Day 90. VX-264 was generally safe and well tolerated; however, the study did not meet the efficacy endpoint. Increases in C-peptide, a marker of insulin production, were not observed at levels necessary to deliver benefit. Therefore, VX-264 will not be advancing further in clinical trials. Vertex plans to conduct further analyses, including of explanted devices, to better understand these findings. Zimislecel UpdateZimislecel (formerly VX-880), Vertex's investigational fully differentiated islet cell therapy with standard immunosuppression, is in the Phase 3 portion of the Phase 1/2/3 study in patients with T1D with severe hypoglycemic events (SHEs) and impaired awareness of hypoglycemia. This pivotal trial is well underway and on track to complete enrollment and dosing in the first half of 2025, setting up global regulatory submissions in 2026. Zimislecel has previously 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). Consistent with this progress, Vertex is investing in expanding its manufacturing and commercial capabilities to ensure launch readiness. If approved, eligible patients across the U.S. and Europe with recurrent SHEs despite best available care could benefit from zimislecel, and Vertex anticipates that initial approval could serve approximately 60,000 people with severe T1D. T1D Research UpdateVertex is also pursuing research-stage T1D programs to evaluate additional approaches that could provide transformative benefit to people with T1D and reduce or eliminate the need for standard immunosuppressive regimens. These approaches include alternative immunosuppressive regimens, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel devices to encapsulate islet cells. "We'd like to thank the patients, physicians and T1D community who participated in the VX-264 study. Today's data show that more work needs to be done to advance the 'cells plus device' program, and we are committed to doing so," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex Pharmaceuticals. "Equally, we are very pleased with the rapid progress of our zimislecel program, which is on track to complete enrollment and dosing in the Phase 3 study this summer, positioning us for global regulatory submissions in 2026. We're excited for the opportunity to bring the promise of zimislecel to patients as quickly as possible." 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 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, statements by Carmen Bozic, M.D., and statements regarding Vertex's plans and expectations for the clinical trial evaluating VX-264, including plans to conduct further analyses, expectations for the clinical trial evaluating zimislecel, including expectations for the trial to complete enrollment and dosing in the first half of 2025 and expectations for global regulatory submissions in 2026, plans to invest in manufacturing and commercial capabilities to ensure zimislecel launch readiness, expectations for the patient population that could benefit from zimislecel, and plans to progress multiple novel, research-stage immunoprotective approaches. 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 development or registration of its compounds due to safety, efficacy or other reasons, that clinical trial data might not be available on the expected timeline, and other risks listed under Risk Factors in Vertex's most recent annual report 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 IncorporatedInvestors:InvestorInfo@ Media:mediainfo@ Sign in to access your portfolio
Yahoo
26-01-2025
- Business
- Yahoo
Is Vertex Pharmaceuticals Stock a Buy in 2025?
The healthcare sector has struggled to deliver worthwhile returns for investors since 2021, weighed down by several headwinds. Rising interest rates, challenges in the U.S. healthcare system, and the inherent risks of drug development have contributed to the sector's underperformance relative to the broader markets during this period. However, some healthcare companies have bucked this downward trend in the post-pandemic era. Notably, Vertex Pharmaceuticals (NASDAQ: VRTX), which focuses on developing treatments for rare diseases, has significantly outpaced both its biotechnology peers and the S&P 500 since early 2022. With the biotech company's stock already posting impressive gains of over 9% in early 2025, it's worth examining whether Vertex stock remains a compelling buy right now. Vertex has built an empire in cystic fibrosis (CF) treatment, recently crowning its dominance with Alyftrek's approval last month. This latest CF drug adds even more firepower to a franchise already set to rake in approximately $10.9 billion last year. With patents locked up into the 2030s and rivals nowhere in sight, Vertex's CF fortress looks impregnable. The company has methodically built this position through years of innovation, leaving would-be competitors in the dust. But the real excitement lies in Vertex's push beyond CF, where years of investment are finally bearing fruit. All eyes are on Jan. 30, when the FDA weighs in on suzetrigine -- a potential game-changer for pain treatment that could offer an alternative to addictive opioids. If approved, Wall Street analysts think suzetrigine will eventually generate blockbuster-level sales (greater than $1 billion annually). The core reason is the drug's novel mechanism of action and potential to be a viable alternative to highly addictive opioids. The biotech also has a burgeoning gene therapy portfolio and pipeline. Working with CRISPR Therapeutics, Vertex has hit the ground running with Casgevy, its gene therapy for blood disorders. Moreover, its experimental cell therapy for type 1 diabetes, Zimislecel (formerly VX-880), has shown remarkable early results, setting the stage for the therapy to enter a pivotal-stage trial. At 23.8 times forward earnings, Vertex stock trades in line with the broader S&P 500 index, which is at 23.6 times earnings -- a surprising valuation for a company with a dominant market share and high barriers to entry. Wall Street sees plenty of growth ahead, projecting an 8.2% jump in sales for 2025 as new products gain traction. The balance sheet tells an equally compelling story: Vertex has $11.2 billion in cash and minimal debt, under $2 billion. This financial strength gives management ample room to pursue strategic deals or accelerate pipeline development as opportunities arise. The company's modest market multiple, combined with robust growth prospects and a fortress-like balance sheet, reflects management's disciplined approach to building long-term value. Few biotechs can match Vertex's combination of growth potential and financial stability. Vertex stands at an inflection point, with its CF franchise providing a rock-solid foundation while multiple late-stage programs could dramatically expand its reach. The next 12 months look particularly eventful, with decisions due on suzetrigine in acute pain, upcoming readouts in diabetes, and the continued rollout of Casgevy with partner CRISPR Therapeutics. This wouldn't be Vertex's first successful reinvention, having already transformed from a hepatitis C player into the dominant force in CF treatment. The company's methodical expansion into pain, diabetes, and kidney disease follows this proven playbook of tackling complex diseases with high unmet needs. With a bulletproof CF business generating a mountain of cash, minimal competition on the horizon in this key area, and multiple shots at billion-dollar markets, Vertex offers a compelling mix of stability and upside potential. The bottom line is few companies can match its combination of established leadership in rare diseases and realistic pathways to significant market expansion, making Vertex stock a worthwhile buy in 2025. Before you buy stock in Vertex Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Vertex Pharmaceuticals wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. 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