Vertex Pharmaceuticals Inc (VRTX)

Vertex Announces US FDA Approval for Expanded Use of CASGEVY® for the Treatment of People Ages 2 Years and Older With Sickle Cell Disease or Transfusion-Dependent Beta ThalassemiaJuly 1, 2026 7:58 PM
Business Wire - First and only approved genetic therapy to treat children as young as 2 years for both severe sickle cell disease and transfusion-dependent beta thalassemia - - Approximately 5,500 additional children in the U.S. are now eligible for this established one-time therapy, expanding upon the prior FDA approval in people 12 years and older - - Regulatory review for label expansion underway in the Kingdom of Saudi Arabia and United Kingdom - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved expanded use of CASGEVY® (exagamglogene autotemcel) for the treatment of people ages 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first approved genetic therapy indicated for children as young as 2 years for both SCD and TDT. “Just as we redefined what is possible in cystic fibrosis, our ambition is to transform the future for people living with sickle cell disease and transfusion-dependent beta thalassemia. The remarkable consistency of results across age groups reinforces the potential of CASGEVY to deliver durable, transformative benefits to those who have historically had limited options,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We’re deeply grateful to the patients, families and investigators who participated in the clinical trials that led to this historic approval, and we are ready to bring CASGEVY to children and their families across the U.S.” “Today’s approval offers renewed hope for children living with sickle cell disease or transfusion-dependent beta thalassemia,” said Haydar Frangoul, M.D., M.S., Medical Director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, investigator with Sarah Cannon Research Institute (SCRI) and Member of Vertex’s SCD Program Steering Committee. “Earlier access to the transformative potential of this therapy will allow clinicians and families to consider treatment before years of cumulative damage from these life-shortening diseases take hold.” Vertex has established a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to eligible patients through existing access and reimbursement pathways. Today, there are more than 75 activated ATCs in the U.S. The full list can be accessed at CASGEVY.com. About Sickle Cell Disease (SCD) Sickle cell disease (SCD) is a rare serious, inherited blood disease that is progressive and life-shortening. The disease causes red blood cells to become rigid and misshapen, restricting blood flow and oxygen delivery to vital organs. Recurrent vaso-occlusive crises (VOCs), unpredictable episodes of severe pain caused by blocked blood vessels, are a defining feature of SCD and frequently require hospitalization. Many patients experience these complications early in life, and over time, repeated VOCs and chronic anemia lead to progressive and irreversible organ damage, including damage to the brain, lungs, kidneys and heart. SCD places a substantial burden on patients and their families, who must manage frequent medical visits, hospitalizations, school and work disruptions, and the emotional toll of chronic pain and life-threatening complications. Despite lifelong treatment, people with SCD and recurrent VOCs in the U.S. face shortened life expectancy, with a median age of death of approximately 45 years, report quality-of-life scores far below the general population and the estimated lifetime healthcare costs of managing the disease are $4–6 million. About Transfusion-Dependent Beta Thalassemia (TDT) Transfusion-dependent beta thalassemia (TDT) is a rare serious, inherited blood disease that is progressive and life-shortening. The disease impairs the body’s ability to produce sufficient hemoglobin, limiting oxygen delivery to tissues and organs. People with TDT do not have enough functional hemoglobin in their red blood cells and require regular, lifelong blood transfusions, often beginning early in childhood, along with ongoing iron chelation therapy. While transfusions are necessary for survival, many of the long-term complications of TDT are exacerbated by chronic transfusion therapy and iron overload and cumulative damage to the heart, liver and endocrine system, as well as bone abnormalities and delayed growth and puberty. TDT places a significant and ongoing burden on patients and their families, requiring frequent medical visits and complex lifelong treatment. Despite lifelong treatment, people with TDT in the U.S. face shortened life expectancy, with a median age of death of approximately 37 years, reduced quality of life and productivity, and the estimated lifetime healthcare costs of managing the disease are $5–5.7 million. About CASGEVY® (exagamglogene autotemcel) CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. 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 in clinical trials to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. In the United States, CASGEVY was approved using the Commissioner’s National Priority Voucher. Vertex has also recently completed regulatory submissions in the Kingdom of Saudi Arabia and United Kingdom to expand the use of CASGEVY to children as young as five. About the CLIMB Studies The completed Phase 1/2/3 open-label studies, CLIMB-111 and CLIMB-121, were designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12-35 years with TDT or with SCD and recurrent VOCs. Patients were followed for approximately two years after CASGEVY infusion in these studies. CLIMB-141 and CLIMB-151 are ongoing Phase 3 open-label studies, designed to assess the safety and efficacy of a single dose of exagamglogene autotemcel in patients ages 2-11 years with TDT or with SCD and recurrent VOCs. Enrollment and dosing are complete for the 5–11-year-old cohort in both studies. Each patient in these studies is asked to participate in the ongoing long-term, open-label study, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients with up to 15 years of follow up after CASGEVY infusion. U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY INDICATION CASGEVY is indicated for the treatment of patients aged 2 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) transfusion-dependent ß-thalassemia (TDT) IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Neutrophil Engraftment Failure There is potential risk of neutrophil engraftment failure after treatment with CASGEVY. In the clinical trials, all treated patients achieved neutrophil engraftment and no patients received rescue CD34+ cells. Monitor absolute neutrophil counts (ANC) and manage infections according to standard guidelines and medical judgement. In the event of neutrophil engraftment failure, patients should be infused with rescue CD34+ cells. Granulocyte colony-stimulating factor (G-CSF) is not recommended for 21 days after CASGEVY infusion. Delayed Platelet Engraftment Delayed platelet engraftment has been observed with CASGEVY treatment. There is an increased risk of bleeding until platelet engraftment is achieved. Monitor patients for bleeding according to standard guidelines and medical judgement. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise. Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis can occur due to dimethyl sulfoxide (DMSO) or dextran 40 in the cryopreservation solution. Monitor patients for hypersensitivity reactions during and after infusion. Off-Target Genome Editing Risk The risk of unintended, off-target editing in an individual’s CD34+ cells cannot be ruled out due to genetic variants. The clinical significance of potential off-target editing is unknown. ADVERSE REACTIONS The most common Grade 3 or 4 non-laboratory adverse reactions (occurring in ≥ 25%) were mucositis and febrile neutropenia in patients with SCD and patients with TDT, and decreased appetite in patients with SCD. All (100%) of the patients with TDT and SCD experienced Grade 3 or 4 neutropenia and thrombocytopenia. Other common Grade 3 or 4 laboratory abnormalities (≥ 50%) include leukopenia, anemia, and lymphopenia. DRUG INTERACTIONS No formal drug interaction studies have been performed. CASGEVY is not expected to interact with the hepatic cytochrome P450 family of enzymes or drug transporters. Use of Granulocyte-Colony Stimulating Factor (G-CSF): G-CSF must not be used for CD34+ HSC mobilization of patients with SCD. Use of Hydroxyurea: Discontinue the use of hydroxyurea at least 8 weeks prior to start of each mobilization cycle and conditioning. There is no experience of the use of hydroxyurea after CASGEVY infusion. Use of Crizanlizumab: Discontinue the use of crizanlizumab at least 8 weeks prior to start of mobilization and conditioning, as their interaction potential with mobilization and myeloablative conditioning agents is not known. Use of Iron Chelators: Discontinue the use of iron chelators at least 7 days prior to initiation of myeloablative conditioning, due to potential interaction with the conditioning agent. Some iron chelators are myelosuppressive. If iron chelation is required, avoid the use of non-myelosuppressive iron chelators for at least 3 months and use of myelosuppressive iron chelators for at least 6 months after CASGEVY infusion. Phlebotomy can be used instead of iron chelation, when appropriate. USE IN SPECIFIC POPULATIONS Pregnancy/Lactation: CASGEVY must not be administered during pregnancy and breastfeeding should be discontinued during conditioning because of the risks associated with myeloablative conditioning. Pregnancy and breastfeeding after CASGEVY infusion should be discussed with the treating physician. Females and Males of Reproductive Potential: A negative serum pregnancy test must be confirmed prior to the start of each mobilization cycle and reconfirmed prior to myeloablative conditioning. Women of childbearing potential and men capable of fathering a child should use effective methods of contraception from start of mobilization through at least 6 months after administration of CASGEVY. Advise patients of the risks associated with conditioning agents. Infertility has been observed with myeloablative conditioning, therefore, advise patients of fertility preservation options before treatment, if appropriate. Please see full Prescribing Information for CASGEVY. 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 IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, 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 Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Reshma Kewalramani, M.D., and Haydar Frangoul, M.D., M.S., in this press release, statements regarding the expected clinical benefits of CASGEVY, expectations regarding the eligible patient population, and expectations for patient access to CASGEVY. 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 factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include the risks listed under the heading “Risk Factors” in Vertex's annual report and in subsequent filings 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. 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/20260701379449/en/ Vertex Pharmaceuticals Incorporated

Investors:

Vertex Presents New Data on CASGEVY®, Including First European Presentation of Data in Children Ages 5–11, at the European Hematology Association Congress and Announces Additional Global Regulatory SubmissionsJune 11, 2026 2:01 AM
Business Wire - Data from pivotal studies of CASGEVY in children ages 5–11 with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrate transformative potential in younger patients, consistent with the durable benefits established in patients 12 years and older - - Data simultaneously published in the New England Journal of Medicine - - Regulatory review underway in the United States to expand the use of CASGEVY, and Vertex has recently completed submissions in the Kingdom of Saudi Arabia and United Kingdom - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data demonstrating the clinical benefits of CASGEVY® (exagamglogene autotemcel) in people ages 5 years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, from pivotal studies in children ages 5–11, show that the efficacy and safety outcomes in this age group are consistent with the transformative profile established in adult and adolescent patients. These data were presented at the European Hematology Association (EHA) Congress and simultaneously published in the New England Journal of Medicine (NEJM). “The data presented at EHA and published in NEJM underscore the consistent, durable and transformative benefits CASGEVY can provide to people living with sickle cell disease or transfusion-dependent beta thalassemia from early in life,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “Despite optimized supportive therapy, children living with sickle cell disease and transfusion-dependent beta thalassemia carry a significant disease burden from a very young age, with progressive complications leading to the irreversible and life-shortening consequences of these diseases,” said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital, Chair of Vertex’s TDT Program Steering Committee, and presenting author of the 5–11 years old CASGEVY data at EHA. “These data represent a profoundly important step forward, and I look forward to the possibility of providing earlier intervention to prevent complications in children and for families who have had limited potentially curative options to date.” CASGEVY clinical data for children ages 5–11 presented at EHA and published in NEJM Data from an interim analysis of the CLIMB-151 and CLIMB-141 studies highlight the transformative potential CASGEVY can provide to children ages 5–11 and are consistent with the durable clinical profile established in adult and adolescent patients. Collectively, these findings highlight the potential benefits of addressing vaso-occlusive crises (VOCs) and transfusion burden earlier in life, which can begin in childhood and are associated with cumulative, long-term complications in SCD and TDT including organ damage. In the Phase 3 CLIMB-151 clinical study of children with severe SCD, all 11 patients dosed are free from VOCs and all 8 out of 8 (100%) patients with sufficient follow-up achieved the primary endpoint of being free from VOCs for at least 12 consecutive months (VF12). Of children achieving VF12, the mean (min, max) duration VOC-free was 19.0 (13.2, 30.1) months. In the Phase 3 CLIMB-141 clinical study of children with TDT, 15 patients have been dosed with CASGEVY, and all 8 out of 8 (100%) patients with sufficient follow-up achieved the primary endpoint of transfusion independence for at least 12 consecutive months while maintaining a weighted average hemoglobin of at least 9 g/dL (TI12). All children who achieved TI12 remained so throughout the follow-up; the mean (min, max) duration transfusion independence was 23.4 (13.3, 28.5) months. The safety profile of CASGEVY in younger patients is consistent with myeloablative conditioning and autologous transplant, as established in clinical studies in older patients with SCD and TDT. As previously disclosed, there was one death, not related to CASGEVY, in a child with TDT who developed severe veno-occlusive disease from busulfan conditioning. Consistent with studies in older patients, children with severe SCD and TDT treated with CASGEVY have durable and clinically relevant increases in fetal hemoglobin (HbF) and stable allelic editing. Global regulatory submissions to expand use of CASGEVY CASGEVY is currently approved for eligible people 12 years and older with SCD with recurrent VOCs or TDT in several countries around the world. In the United States the regulatory review is underway with the FDA to expand the use of CASGEVY to younger children after Vertex was awarded the Commissioner’s National Priority Voucher. Vertex has also recently completed regulatory submissions in the Kingdom of Saudi Arabia and United Kingdom to expand the use of CASGEVY to younger children. Upon availability, there is an established network of activated authorized treatment centers in these countries prepared to support patients. The use of CASGEVY in children ages 5–11 years is investigational. About Sickle Cell Disease (SCD) Sickle Cell Disease (SCD) is a rare serious, inherited blood disease that is progressive and life-shortening. The disease causes red blood cells to become rigid and misshapen, restricting blood flow and oxygen delivery to vital organs. Recurrent vaso-occlusive crises (VOCs), unpredictable episodes of severe pain caused by blocked blood vessels, are a defining feature of SCD and frequently require hospitalization. Many patients experience these complications early in life, and over time, repeated VOCs and chronic anemia lead to progressive and irreversible organ damage, including damage to the brain, lungs, kidneys and heart. SCD places a substantial burden on patients and their families, who must manage frequent medical visits, hospitalizations, school and work disruptions, and the emotional toll of chronic pain and life-threatening complications. Despite lifelong treatment, people with SCD and recurrent VOCs in Europe face shortened life expectancy, with a mean age of death of around 40 years, and report quality-of-life outcomes far below the general population. About Transfusion-Dependent Beta Thalassemia (TDT) Transfusion-dependent beta thalassemia (TDT) is a rare serious, inherited blood disease that is progressive and life-shortening. The disease impairs the body’s ability to produce sufficient hemoglobin, limiting oxygen delivery to tissues and organs. People with TDT do not have enough functional hemoglobin in their red blood cells and require regular, lifelong blood transfusions, often beginning early in childhood, along with ongoing iron chelation therapy. While transfusions are necessary for survival, many of the long-term complications of TDT are exacerbated by chronic transfusion therapy and iron overload and cumulative damage to the heart, liver and endocrine system, as well as bone abnormalities and delayed growth and puberty. TDT places a significant and ongoing burden on patients and their families, requiring frequent medical visits and complex lifelong treatment. Despite lifelong treatment, people with TDT face shortened life expectancy, with a mean age of death of approximately 50–55 years in Europe, reduced quality of life and productivity, and significant use of health care resources. About CASGEVY® (exagamglogene autotemcel) CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. 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 in clinical trials to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. About the CLIMB Studies The completed Phase 1/2/3 open-label studies, CLIMB-111 and CLIMB-121, were designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12–35 years with TDT or with SCD and recurrent VOCs. Patients were followed for approximately two years after CASGEVY infusion in these studies. CLIMB-141 and CLIMB-151 are ongoing Phase 3 open-label studies, designed to assess the safety and efficacy of a single dose of exagamglogene autotemcel in patients ages 2–11 years with TDT or with SCD and recurrent VOCs. Enrollment and dosing are complete for the 5–11-year-old cohort in both studies. Each patient in these studies is asked to participate in the ongoing long-term, open-label study, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients with up to 15 years of follow-up after CASGEVY infusion. U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY WHAT IS CASGEVY? CASGEVY is a one-time therapy used to treat people ages 12 years and older with: • sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs • beta thalassemia (ß-thalassemia) who need regular blood transfusions CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia. IMPORTANT SAFETY INFORMATION What is the most important information I should know about CASGEVY? After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels. Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of platelet cells: severe headache abnormal bruising prolonged bleeding bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of white blood cells: fever chills infections You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects. How will I receive CASGEVY? Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It’s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment. After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment. STEP 1: Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week. During this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY. STEP 2: After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider. STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY. STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time. After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home. What should I avoid after receiving CASGEVY? Do not donate blood, organs, tissues, or cells at any time in the future What are the possible or reasonably likely side effects of CASGEVY? The most common side effects of CASGEVY include: Low levels of platelet cells, which may reduce the ability of blood to clot and may cause bleeding Low levels of white blood cells, which may make you more susceptible to infection Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms: fever chills infections severe headache abnormal bruising prolonged bleeding bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. General information about the safe and effective use of CASGEVY Talk to your healthcare provider about any health concerns. Please see full Prescribing Information including Patient Information for CASGEVY. 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 IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, 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. 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, statements made by Carmen Bozic, M.D., and Franco Locatelli, M.D., Ph.D., and statements regarding expectations for the transformative potential of CASGEVY in this age group, and expectations for the global regulatory submissions for younger children. 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 potential medicines in a timely manner, or at all, due to safety, efficacy or other reasons, that CASGEVY may not receive regulatory approval for this age range on the expected timeline, or 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/20260610130971/en/ Vertex Pharmaceuticals Incorporated Investors:

Vertex Presents New Data on ALYFTREK® at European Cystic Fibrosis ConferenceJune 5, 2026 12:00 PM
Business Wire - ALYFTREK Phase 3 data on children with cystic fibrosis ages 2 to 5 with vanzacaftor/tezacaftor/deutivacaftor-responsive genotypes including F/F and F/MF shows 65% reached sweat chloride levels of

Vertex to Participate in the Goldman Sachs 47th Annual Global Healthcare Conference on June 10June 3, 2026 4:00 PM
Business Wire Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in the Goldman Sachs 47th Annual Global Healthcare Conference. Dr. Reshma Kewalramani, President and Chief Executive Officer will participate in a fireside chat on Wednesday, June 10, 2026, at 11:20 a.m. ET. A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived 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 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 IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, 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/20260603629281/en/ Investors:
InvestorInfo@vrtx.com Original: Vertex to Participate in the Goldman Sachs 47th Annual Global Healthcare Conference on June 10

Vertex Announces New Drug Submission for Suzetrigine Has Been Accepted for Review by Health Canada for the Treatment of Moderate-to-Severe Acute Pain in AdultsMay 21, 2026 12:56 PM
PR Newswire (Canada) – Suzetrigine, a selective pain signal inhibitor, has the potential to treat millions of Canadians who suffer from moderate-to-severe acute pain each year –TORONTO, May 21, 2026 /CNW/ - Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that Health Canada has accepted for review a New Drug Submission (NDS) for suzetrigine for the treatment of moderate-to-severe acute pain in adults. Suzetrigine is a selective NaV1.8 pain signal inhibitor that represents a new class of oral pain medicine; it is not an opioid or a nonsteroidal anti-inflammatory drug (NSAID). If approved by Health Canada, suzetrigine has the potential to be the first new class of medicine in Canada to treat acute pain in over twenty years. Each year, approximately 8 million Canadian adults are prescribed pain medication to manage their acute pain. "Today's acceptance of our NDS for suzetrigine represents an important step in our mission to transform pain management in Canada," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "Canadians experiencing acute pain deserve access to innovative treatment options. After more than two decades without a new class of acute pain medicine, suzetrigine has the potential to address this significant unmet need and expand the options available to health care providers."Vertex intends for the regulatory review of suzetrigine to be part of an aligned review with Canadian Health Technology Assessment (HTA) organizations: Canada's Drug Agency (CDA-AMC) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Quebec. Aligned reviews provide an opportunity for information sharing between Health Canada and HTA bodies, leading to greater coordination to support Canadians' timely access to effective new therapies.Suzetrigine was approved by the U.S. Food and Drug Administration under the trade name JOURNAVX® in January 2025. The U.S. application received breakthrough therapy, fast track, and priority review designations.About SuzetrigineSuzetrigine (JOURNAVX) is an investigational, oral, selective NaV1.8 pain signal inhibitor. NaV1.8 is a voltage-gated sodium channel that is selectively expressed in peripheral pain-sensing neurons (nociceptors), where its role is to transmit pain signals (action potentials). NaV1.8 is not expressed in the human brain and is not related to addiction.NaV1.8 is a genetically validated target for the treatment of pain, and suzetrigine has demonstrated a favorable benefit/risk profile in three Phase 3 studies and two Phase 2 studies in patients with moderate-to-severe acute pain.The U.S. Food and Drug Administration approved twice-daily JOURNAVX for the treatment of adults with moderate-to-severe acute pain, including postoperative pain.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 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 IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, 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/en-ca. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Michael Siauw in this press release, statements regarding the expected benefits of suzetrigine, including its potential to be the first new class of medicine in Canada to treat acute pain in over twenty years, and statements regarding expectations for the aligned review with the HTA, CDA-AMC and the INESSS. 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 factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities in Canada may not approve suzetrigine on a timely basis or at all, and other risks listed under the heading "Risk Factors" in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov. 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) Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.comMedia: 
mediainfo @sofaman-1600SOURCE Vertex Pharmaceuticals (Canada) Inc. Original: Vertex Announces New Drug Submission for Suzetrigine Has Been Accepted for Review by Health Canada for the Treatment of Moderate-to-Severe Acute Pain in Adults

Vertex annonce l'acceptation par Santé Canada de la présentation de drogue nouvelle pour la suzétrigine, un médicament utilisé comme traitement de la douleur aiguë modérée ou sévère chez les adultesMay 21, 2026 12:57 PM
PR Newswire (Canada) - La suzétrigine, un inhibiteur sélectif du signal de la douleur, pourrait offrir un traitement à plusieurs millions de personnes annuellement qui présentent des douleurs aiguës modérées ou sévères au Canada -TORONTO, le 21 mai 2026 /CNW/ - Vertex Pharmaceuticals (Nasdaq : VRTX) a annoncé aujourd'hui que Santé Canada a accepté d'étudier sa présentation de drogue nouvelle pour la suzétrigine, un médicament destiné au traitement de la douleur aiguë modérée ou sévère chez les adultes. La suzétrigine est un inhibiteur sélectif du signal de la douleur transmis par le canal NaV1.8 qui représente une nouvelle classe d'analgésiques oraux; elle ne fait pas partie de la famille des opioïdes ni de celle des anti-inflammatoires non stéroïdiens (AINS). Si elle est homologuée par Santé Canada, la suzétrigine pourrait être la première nouvelle classe de médicaments pour le traitement de la douleur aiguë à voir le jour au Canada depuis plus de vingt ans. Chaque année, environ 8 millions d'adultes canadiens se voient prescrire des analgésiques pour traiter des douleurs aiguës. « Avec l'acceptation de notre présentation de drogue nouvelle pour la suzétrigine annoncée aujourd'hui, nous franchissons une étape majeure dans notre mission consistant à transformer la prise en charge de la douleur au Canada, a souligné Michael Siauw, directeur général de Vertex Pharmaceuticals (Canada) Incorporated. Les Canadiens et les Canadiennes présentant des douleurs aiguës devraient avoir accès à des traitements novateurs. Aucune nouvelle classe de médicaments contre la douleur aiguë n'a été introduite depuis plus de 20 ans. Il s'agit là d'un besoin de grande importance qui pourrait être comblé grâce à la suzétrigine, laquelle donnerait de surcroît plus d'options aux professionnels de la santé. »Vertex souhaite que l'examen réglementaire de la suzétrigine s'inscrive dans un processus d'examen harmonisé avec les organismes canadiens d'évaluation des technologies de la santé (ETS) : l'Agence canadienne des médicaments (AMC) et l'Institut national d'excellence en santé et en services sociaux (INESSS) du Québec. Un examen harmonisé est un processus dans le cadre duquel Santé Canada et les organismes d'ETS s'échangent de l'information afin d'améliorer la coordination de l'examen. Les nouveaux traitements efficaces peuvent ainsi être mis sur le marché plus rapidement, pour le bien de la population canadienne.La suzétrigine a été homologuée par la Food and Drug Administration aux États-Unis en janvier 2025, sous l'appellation commerciale JOURNAVX®. Aux États-Unis, la demande s'est vu accorder le statut de traitement révolutionnaire, a été inscrite au processus accéléré et son examen a été désigné prioritaire.À propos de la suzétrigineLa suzétrigine (JOURNAVX) est un médicament expérimental administré par voie orale qui inhibe de façon sélective le signal de la douleur transmis par le canal sodique dépendant du voltage NaV1.8. Ce canal est exprimé sélectivement dans les neurones sensoriels de la douleur périphérique (nocicepteurs), où il transmet les signaux douloureux (potentiels d'action). Il n'est pas exprimé dans le cerveau humain et n'est pas associé à la dépendance.Le canal NaV1.8 est une cible validée sur le plan génétique pour le traitement de la douleur, et un rapport favorable entre les bienfaits et les risques a été observé avec la suzétrigine dans trois essais cliniques de phase 3 et dans deux essais cliniques de phase 2 chez des patients présentant des douleurs aiguës modérées ou sévères.Aux États-Unis, la Food and Drug Administration a approuvé l'administration de JOURNAVX deux fois par jour pour le traitement de la douleur aiguë modérée ou sévère, y compris en contexte postopératoire, chez les adultes.À propos de VertexVertex est une société mondiale de biotechnologie qui investit dans l'innovation scientifique afin de mettre au point des médicaments transformateurs pour les personnes atteintes de maladies graves. La société a des traitements approuvés pour la fibrose kystique, l'anémie falciforme, la bêta-thalassémie dépendante des transfusions et la douleur aiguë; elle continue à mener des programmes cliniques et des programmes de recherche dans ces domaines. Vertex dispose également d'une solide gamme de traitements expérimentaux utilisant diverses modalités thérapeutiques pour d'autres maladies graves où elle possède une connaissance approfondie des processus biologiques humains en cause, notamment la néphropathie à immunoglobulines A (IgA), la douleur neuropathique, la néphropathie médiée par l'APOL1, la glomérulonéphrite extramembraneuse primitive, la maladie rénale polykystique autosomique dominante, le diabète de type 1, la myasthénie grave généralisée et la dystrophie myotonique de type 1.Vertex a été fondée en 1989. Son siège social mondial est à Boston et son siège social international est à Londres. De plus, la société possède des centres de recherche et développement et des bureaux commerciaux en Amérique du Nord, en Europe, en Australie, en Amérique latine et au Moyen-Orient. Vertex est constamment reconnue comme l'un des meilleurs milieux de travail de l'industrie; elle a figuré 16 années consécutives sur la liste des meilleurs employeurs du magazine Science et est l'un des 100 meilleurs employeurs selon le magazine Fortune. Pour connaître les dernières actualités sur l'entreprise et pour en apprendre davantage sur l'histoire de l'innovation chez Vertex, visitez www.vrtx.com/fr-ca. Remarque concernant les déclarations prospectives Ce communiqué de presse contient des déclarations prospectives, comme défini dans la loi sur la réforme des litiges en matière de valeurs mobilières privées (Private Securities Litigation Reform Act) de 1995, y compris, sans s'y limiter, les déclarations faites par Michael Siauw dans ce communiqué de presse, les déclarations relatives aux bienfaits attendus de la suzétrigine, notamment la possibilité qu'elle constitue la première nouvelle classe de médicaments pour le traitement de la douleur aiguë à voir le jour au Canada depuis plus de 20 ans, ainsi que les déclarations concernant les attentes liées à l'examen harmonisé avec les organismes d'ETS, soit l'AMC et l'INESSS. Bien que Vertex estime que les déclarations prospectives contenues dans ce communiqué de presse sont exactes, ces déclarations prospectives représentent les opinions de la société uniquement à la date de ce communiqué de presse et il existe un certain nombre de facteurs qui pourraient faire en sorte que les événements ou les résultats réels diffèrent sensiblement de ceux indiqués par ces déclarations prospectives. Parmi ces risques et ces incertitudes, citons entre autres la possibilité que la suzétrigine ne soit pas homologuée par les autorités réglementaires canadiennes ou qu'elle ne le soit pas en temps voulu, et d'autres risques énumérés sous la rubrique Risk Factors (facteurs de risque) dans le rapport annuel de Vertex et les documents ultérieurs déposés auprès de la Securities and Exchange Commission et disponibles sur le site Web de la société aux adresses www.vrtx.com et www.sec.gov. Vous ne devez pas vous fier indûment à ces déclarations. Vertex décline toute obligation de mettre à jour les renseignements contenus dans ce communiqué de presse à mesure que de nouveaux renseignements deviennent disponibles. (VRTX-GEN) Vertex Pharmaceuticals Incorporated
Investisseurs :
InvestorInfo@vrtx.comMédias : 
mediainfo @sofaman-1600SOURCE Vertex Pharmaceuticals (Canada) Inc. Original: Vertex annonce l'acceptation par Santé Canada de la présentation de drogue nouvelle pour la suzétrigine, un médicament utilisé comme traitement de la douleur aiguë modérée ou sévère chez les adultes

Vertex to Participate in Upcoming May Investor ConferencesMay 11, 2026 4:00 PM
Business Wire Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced management participation in two upcoming investor conferences. Charlie Wagner, Chief Operating and Financial Officer, will participate in a fireside chat at the 2026 RBC Capital Markets Global Healthcare Conference on Tuesday, May 19, 2026, at 2:35 p.m. ET. Dr. Reshma Kewalramani, Chief Executive Officer and President, will participate in a fireside chat at Bernstein’s 42nd Annual Strategic Decisions Conference (SDC) on Friday, May 29, 2026, at 9:00 a.m. ET. 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 IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, 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 at www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and X. View source version on businesswire.com: https://www.businesswire.com/news/home/20260511401202/en/ Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com Original: Vertex to Participate in Upcoming May Investor Conferences

Halozyme signs licensing deal with Vertex for drug delivery technologyApril 7, 2026 10:11 AM
IH Market News
Halozyme Therapeutics (NASDAQ:HALO) said its subsidiary has entered into an exclusive collaboration and licensing agreement with Vertex Pharmaceuticals (NASDAQ:VRTX) covering the use of Halozyme’s Hypercon drug delivery technology.Under the terms of the agreement, Vertex has obtained rights to use the Hypercon microparticle platform for up to three drug targets. The technology is designed to concentrate drugs and biologics into smaller volumes while maintaining the same dosage, potentially enabling more convenient at-home administration.As part of the deal, Vertex will pay Halozyme $15 million upfront and may make additional payments tied to development and commercial milestones. Halozyme will also be eligible to receive royalties on net sales of any products developed using the Hypercon platform.“This collaboration with Vertex underscores the versatility and potential of our Hypercon technology to enable small volume, patient-delivered next generation biologics,” said Helen Torley, President and Chief Executive Officer of Halozyme.Mark Bunnage, Executive Vice President and Chief Scientific Officer of Vertex Pharmaceuticals, said the company is “pleased to add Halozyme’s Hypercon drug delivery technology to our toolbox as we pursue transformative therapies for patients with serious diseases.”Halozyme Therapeutics stock priceVertex Pharmaceuticals stock price

Original: Halozyme signs licensing deal with Vertex for drug delivery technology

Vertex to Announce First Quarter 2026 Financial Results on May 4thApril 6, 2026 4:00 PM
Business Wire
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its first quarter 2026 financial results on Monday, May 4, 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 First 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 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 IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, 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 at 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/20260406130837/en/
Vertex Pharmaceuticals Incorporated Investor Relations:

investorinfo@vrtx.com


Original: Vertex to Announce First Quarter 2026 Financial Results on May 4th

Vertex Announces US FDA Approval for Label Extensions of ALYFTREK® and TRIKAFTA®, Expanding Availability of These Medicines to ~95% of All People With CF in the United StatesApril 1, 2026 7:30 AM
Business Wire
-With this expansion, any variant that results in production of CFTR protein is now included in the indication for ALYFTREK and TRIKAFTA, reinforcing the impact these medicines have, regardless of the location of the variant in the CFTR protein-


-Approximately 800 more people with CF in the US are now eligible for a CFTR modulator for the first time-


Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved expanded use of ALYFTREK® (vanzacaftor/tezacaftor/ivacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 and older with a variant in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is either responsive based on clinical and/or in vitro data or results in production of CFTR protein. Additionally, the U.S. FDA has also expanded the indication statement for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in patients ages 2 and older.


This label expansion was supported by clinical and/or in vitro data from 564 variants demonstrating response to ALYFTREK and 521 variants demonstrating response to TRIKAFTA. As such, approximately 800 more people with a clinical diagnosis of CF in the U.S. are now eligible for a medicine that treats the underlying cause of their disease for the first time. This extension means approximately 95% of people with CF in the U.S. are now eligible for treatment with a CFTR modulator.


“This groundbreaking approval represents more than 20 years of innovation in CF, including testing over 600 variants in our laboratory, demonstrating clinical effects in large clinical trials, and studying younger people with CF so they can be treated as early as possible,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “With this label expansion, any variant that results in production of CFTR protein is now included in the ALYFTREK and TRIKAFTA labels, validating that these medicines can restore CFTR function and provide clinical benefit to patients regardless of where in the CFTR protein a variant is located. We thank the CF community and investigators for their trust and look forward to bringing ALYFTREK and TRIKAFTA to more patients than ever before.”


For more information on ALYFTREK or TRIKAFTA, patient assistance programs, or eligibility details, visit ALYFTREK.com, TRIKAFTA.com, VertexGPS.com or vertextreatments.com.


IMPORTANT SAFETY INFORMATION


WARNING: DRUG-INDUCED LIVER INJURY AND LIVER FAILURE


Elevated transaminases have been observed in patients treated with ALYFTREK.


TRIKAFTA can cause serious and potentially fatal drug-induced liver injury. Cases of liver failure leading to transplantation and death have been reported in both clinical trials and the postmarketing setting in patients with and without a history of liver disease taking TRIKAFTA, a fixed-dose combination drug containing elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA), the same or similar active ingredients as ALYFTREK. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of TRIKAFTA.


Assess liver function tests (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK or TRIKAFTA, then every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or liver function test (LFT) elevations at baseline.


Interrupt ALYFTREK or TRIKAFTA for significant elevations in LFTs or in the event of signs or symptoms of liver injury. Consider referral to a hepatologist. Follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If resolved, resume treatment only if benefit is expected to outweigh risk. Closer monitoring is advised after resuming treatment.


ALYFTREK or TRIKAFTA should not be used in patients with severe hepatic impairment (Child-Pugh Class C). ALYFTREK or TRIKAFTA is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B). ALYFTREK or TRIKAFTA should only be considered when there is a clear medical need and benefit outweighs risk. If ALYFTREK is used, monitor patients closely. If TRIKAFTA is used, use with caution at a reduced dosage and monitor patients closely.


WARNINGS AND PRECAUTIONS


DRUG-INDUCED LIVER INJURY AND LIVER FAILURE



Elevated transaminases have been observed in patients treated with ALYFTREK. TRIKAFTA can cause serious and potentially fatal drug-induced liver injury. Liver failure leading to transplantation and death has been reported in patients with and without a history of liver disease taking TRIKAFTA. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of TRIKAFTA



Assess LFTs in all patients prior to initiating ALYFTREK or TRIKAFTA, then every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or LFT elevations at baseline, or a history of elevated LFTs with drugs containing ELX, TEZ, and/or IVA



Interrupt ALYFTREK or TRIKAFTA in the event of signs or symptoms of liver injury, which may include:


Significant elevations in LFTs (e.g., ALT or AST >5x the upper limit of normal (ULN) or ALT or AST >3x ULN with bilirubin >2x ULN)



Clinical symptoms suggestive of liver injury (e.g., jaundice, right upper quadrant pain, nausea, vomiting, altered mental status, ascites)






Consider referral to a hepatologist and follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If resolved, and if benefit is expected to outweigh risk, resume treatment with close monitoring



ALYFTREK and TRIKAFTA should not be used in patients with severe hepatic impairment, are not recommended in patients with moderate hepatic impairment, and should only be considered when there is a clear medical need and benefit outweighs risk. If ALYFTREK is used, monitor patients closely. If TRIKAFTA is used, use with caution at a reduced dosage and monitor patients closely



HYPERSENSITIVITY REACTIONS, INCLUDING ANAPHYLAXIS



Hypersensitivity reactions, including cases of angioedema and anaphylaxis, have been reported in the postmarketing setting for TRIKAFTA. If signs or symptoms of serious hypersensitivity reactions develop during treatment, discontinue ALYFTREK or TRIKAFTA and institute appropriate therapy. Consider benefits and risks to determine whether to resume treatment



PATIENTS WHO DISCONTINUED OR INTERRUPTED ELX-, TEZ-, OR IVA-CONTAINING DRUGS DUE TO ADVERSE REACTIONS


ALYFTREK



There are no available safety data for ALYFTREK in patients who previously discontinued or interrupted treatment with drugs containing ELX, TEZ, or IVA due to adverse reactions. Consider benefits and risks before using ALYFTREK in these patients and if used, closely monitor for adverse reactions



INTRACRANIAL HYPERTENSION (IH)



IH has been reported in the postmarketing setting with TRIKAFTA, which contains the same or similar active ingredients as ALYFTREK. Clinical manifestations of IH include headache, blurred vision, diplopia, and potential vision loss; papilledema can be found on fundoscopy. If an unusual headache or visual disturbances occur during treatment, and IH is suspected, interrupt treatment and refer for prompt medical evaluation. Consider benefits and risks to determine whether to resume treatment. Patients should be monitored until IH resolution and for recurrence. Patients with elevated vitamin A levels may be at increased risk



NEUROPSYCHIATRIC EVENTS, INCLUDING SUICIDAL THOUGHTS AND BEHAVIORS



Serious neuropsychiatric events, including symptoms of anxiety, depression, suicidal ideation and behavior, and sleep disturbances, have been reported in the postmarketing setting in patients with and without a previous history of neuropsychiatric symptoms taking ALYFTREK or TRIKAFTA. Symptoms may occur within the first 3 months of treatment. Assess patients for baseline neuropsychiatric symptoms and monitor for new or worsening symptoms. Consider the benefits and risks to determine if treatment should be interrupted at symptom occurrence or resumed with symptom improvement



DRUG INTERACTIONS

Use With CYP3A Inducers



Following concomitant use of strong or moderate CYP3A inducers with ALYFTREK, exposures of vanzacaftor, TEZ, and deutivacaftor were decreased, which may reduce ALYFTREK effectiveness. Concomitant use with strong or moderate CYP3A inducers is not recommended



Exposure to IVA is significantly decreased and exposure to ELX and TEZ are expected to decrease with concomitant use of CYP3A inducers, which may reduce effectiveness of TRIKAFTA. Concomitant use with strong CYP3A inducers is not recommended



Use With CYP3A Inhibitors



Exposure to vanzacaftor, TEZ, and deutivacaftor or ELX, TEZ, and IVA are increased when used concomitantly with strong or moderate CYP3A inhibitors. The dose of ALYFTREK or TRIKAFTA should be reduced when used concomitantly with moderate or strong CYP3A inhibitors



CATARACTS



Non-congenital lens opacities have been reported in pediatric patients treated with TRIKAFTA, which contains IVA (similar to an active ingredient in ALYFTREK). Baseline and follow-up ophthalmological examinations are recommended in pediatric patients



ADVERSE REACTIONS

ALYFTREK



Serious adverse reactions that occurred more frequently with ALYFTREK than with ELX/TEZ/IVA in 2 or more patients (≥0.4%) were influenza (1.5%), increased AST (0.4%), increased GGT (0.4%), depression (0.4%), and syncope (0.4%)



The most common adverse reactions occurring in ≥5% of patients and at a frequency higher than ELX/TEZ/IVA by ≥1% were cough, nasopharyngitis, upper respiratory tract infection (URTI), headache, oropharyngeal pain, influenza, fatigue, increased ALT and AST, rash, and sinus congestion



TRIKAFTA



Serious adverse reactions that occurred more frequently in patients treated with TRIKAFTA compared to placebo included rash (1% vs

Anti-Aging Protein Research Takes a Step Forward with Cell CloningMarch 24, 2026 11:05 AM
PR Newswire (Canada)

Issued on behalf of Avaí Bio, Inc.VANCOUVER, BC, March 24, 2026 /CNW/ -- Equity-Insider.com — A protein called a-Klotho circulating in the bloodstream protects the brain, heart, kidneys, and the immune system. Peer-reviewed research has linked higher Klotho levels to reduced risk of Alzheimer's, cardiovascular disease, and certain cancers. Mayo Clinic research connects declining Klotho to arterial stiffness and vascular calcification.
The challenge is that the body cuts production of this protein by approximately 50% after the age of 40. The molecule that guards against the deadliest age-related diseases starts to decline just as the risk for these conditions starts to rises. Market projections highlight the scale of the issue: Alzheimer's alone is projected to reach $32.8 billion by 2033, cardiovascular disease remains the leading global cause of death, and kidney disease affects 850 million people worldwide.The global cell therapy market has surpassed $8.2 billion in 2026. The broader cell and gene therapy sector is forecast to surge from $10.4 billion to more than $45 billion by 2035, with more than 40 FDA-approved products now on the market. Regenerative medicine alone is projected to reach $578 billion by 2033. The science of reversing biological decline is no longer theoretical, it is an industry. And the companies building the cellular foundations for these therapies are at the forefront of market attention.Vertex Pharmaceuticals (NASDAQ: VRTX) demonstrated what that looks like at scale. The company's gene-edited cell therapy Casgevy — developed with CRISPR Therapeutics (NASDAQ: CRSP) — became the world's first approved CRISPR-based treatment, now available for sickle cell disease and transfusion-dependent beta-thalassemia. Vertex expects to file regulatory submissions for the 5–11 age group in the first half of 2026. CRISPR Therapeutics, meanwhile, reported Phase 1 data showing its in vivo gene-editing therapy CTX310 achieved mean reductions of 73% in ANGPTL3, 55% in triglycerides, and 49% in LDL cholesterol after a single intravenous infusion — a one-dose cardiovascular intervention that could reshape metabolic disease treatment.Altimmune (NASDAQ: ALT) is advancing pemvidutide, a GLP-1/glucagon dual receptor agonist for metabolic disease that targets the $65 billion metabolic syndrome opportunity through hormonal regulation of fat metabolism. Arrowhead Pharmaceuticals (NASDAQ: ARWR) dosed the first patients in a Phase 1/2a trial of ARO-DIMER-PA, the first clinical candidate designed to silence two genes simultaneously for atherosclerotic cardiovascular disease. These are platform-level interventions pulling institutional capital into longevity and regenerative medicine at unprecedented speed.But before any cell therapy can reach patients, it needs a starting point. And that's the step one this company just completed.Avaí Bio (OTCQB: AVAI) recently announced a critical early-stage milestone alongside joint venture partner Austrianova: beginning the creation of a Master Cell Bank (MCB) of genetically modified cells that overexpress the a-Klotho protein. An MCB is the process of taking a single genetically engineered cell and cloning it into tens of millions of identical copies, creating a standardized, GMP-compliant bank of cellular starting material. It's the essential foundation from which all future working cell banks and therapy development will proceed — the step that ensures consistency, quality, and scalability before any therapeutic product can be developed."We are excited to enter the first step in the production phase of a-Klotho producing cells as part of our commitment to deliver safe, effective treatments for aging associated diseases," said Chris Winter, CEO of Avaí Bio.The cells banked in the MCB will be used in conjunction with Austrianova's proprietary Cell-in-a-Box® encapsulation technology, which protects therapeutic cells inside a biocompatible shell to allow continuous protein secretion without triggering immune rejection. The technology is backed by over 50 peer-reviewed publications and decades of development. Avaí Bio's dual-program approach targets both the Klothonova anti-aging platform and the Insulinova diabetes program, each leveraging this same encapsulation technology. The company participated in the 15th European Pancreas and Islet Transplantation Association Symposium in January 2026, where Dr. Eva Maria Lilli Brandtner evaluated advanced cells for potential application in diabetes therapy.The protein that protects against the most serious age-related diseases is steadily disappearing from the bloodstream. The science to potentially restore it exists. And the company that just created the standardized cellular foundation to advance that science — Avaí Bio (OTCQB: AVAI) — is doing so in a market where every platform capable of developing cell-based therapeutics is being re-evaluated.For more information on Avaí Bio (OTCQB: AVAI) and its Klothonova and Insulinova programs, visit Equity-Insider.comRead this and more news for Avaí Bio at: Equity-Insider.comArticle Source: https://usanewsgroup.com/avai-profile/ CONTACT:
EQUITY INSIDER
info @acblanke1DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Avaí Bio, Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Avaí Bio, Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Avaí Bio, Inc. which were purchased in the open market. MIQ reserves the right to buy and sell, and will buy and sell shares of Avaí Bio, Inc. at any time thereafter without any further notice. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material disseminated by MIQ has been approved by the above mentioned company; this is a paid advertisement, and we own shares of the mentioned company that we will sell, and we also reserve the right to buy shares of the company in the open market, or through other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment.SOURCES:Straits Research, Global Cell Therapy Market 2026 — https://straitsresearch.com/report/cell-therapy-marketPrecedence Research, Cell and Gene Therapy Market Forecast — https://www.precedenceresearch.com/cell-and-gene-therapy-marketGrand View Research, CAR T-Cell Therapy Market — https://www.grandviewresearch.com/industry-analysis/car-t-cell-therapy-market-reportAstute Analytica, Regenerative Medicine Market 2025–2033 — https://www.globenewswire.com/news-release/2026/01/27/3226653/0/en/Regenerative-Medicine-Market-Review-2020-2024-and-Forecast-2025-2033-A-578-29-Bn-Opportunity-Says-Astute-Analytica.html Logo: https://mma.prnewswire.com/media/2840019/Equity_Insider_Logo.jpg  



View original content:https://www.prnewswire.com/news-releases/anti-aging-protein-research-takes-a-step-forward-with-cell-cloning-302723584.html

Original: Anti-Aging Protein Research Takes a Step Forward with Cell Cloning

Vertex Shares Jump After Positive Phase 3 Kidney Drug ResultsMarch 10, 2026 11:45 AM
IH Market News
Vertex Pharmaceuticals (NASDAQ:VRTX) shares climbed about 6% to $488.52 in premarket trading Thursday after the company reported encouraging results from a late-stage clinical trial of its kidney disease treatment povetacicept and said it plans to pursue U.S. regulatory approval.The drug is being developed to treat IgA nephropathy and showed a 52% reduction in proteinuria—a key indicator of kidney damage—after 36 weeks of treatment, compared with a 4.3% decline in patients receiving a placebo. The Phase 3 RAINIER study met its primary interim endpoint, delivering a placebo-adjusted proteinuria reduction of 49.8%.Vertex said the results from the trial, which enrolled 199 patients, will support a planned submission for U.S. approval. At the 36-week mark, 42% of patients treated with povetacicept reached KDIGO target proteinuria levels below 0.5 grams per day, compared with 6% of those in the placebo group.The company noted that upper respiratory infections were more common among patients receiving povetacicept, occurring in 18.3% of participants versus 11.4% in the placebo arm. Vertex also said the study population represented a more difficult-to-treat group, characterized by longer times since diagnosis and greater use of SGLT2 inhibitors.Analysts offered mixed reactions to the trial outcome.Barclays analysts said, “Though we note many expected the data to be positive, we think UPCR reduction (~50% pbo-adj) comes in at the higher end of investor expectations. We think totality of data and cross-trial comparisons suggest best-in-class efficacy.”However, analysts at Baird were more cautious, writing, “The Ph3 RAINIER readout of povetacicept in IgAN drove some upside in after-hours trading, but we continue to think the optimism is misplaced, with a UPCR reduction nearly identical to Otsuka’s (OTSKY, not covered) approved APRIL inhibitor, Voyxact. With Otsuka in the pole position and no meaningful differentiation for povetacicept, we continue to think there is too much hype around this program being the next major growth driver for Vertex when first and best-in-class is already out there.”Vertex Pharmaceuticals stock price

Original: Vertex Shares Jump After Positive Phase 3 Kidney Drug Results

Vertex Announces Positive Week 36 Interim Analysis Results for Primary and All Secondary Endpoints in the RAINIER Phase 3 Trial of Povetacicept in Adults With IgA NephropathyMarch 9, 2026 4:02 PM
Business Wire
- For the primary endpoint, patients treated with povetacicept achieved a 52.0% reduction from baseline in proteinuria as measured by 24-hour urine protein to creatinine ratio (UPCR) and achieved a statistically significant and clinically meaningful 49.8% reduction in UPCR versus placebo (P

Breaking Barriers: How 2026's Top Clinical Leaders Are Disrupting Chronic Disease MarketsJanuary 26, 2026 3:16 PM
PR Newswire (US)

Issued on behalf of Avant Technologies Inc.Equity Insider News CommentaryVANCOUVER, BC, Jan. 26, 2026 /PRNewswire/ -- The medical world is shifting as the global market for next-gen treatments heads toward $88.85 billion by 2030[1], driven by a surge in funding for high-tech cures for long-term illness. A major FDA move at the beginning of the year[2] is now making it much easier to build these advanced platforms, clearing a path for companies that can treat diseases internally without the harsh side effects of traditional drugs. This regulatory shift positions Avant Technologies, Inc. (OTCQB: AVAI), MannKind (NASAQ: MNKD), Vertex Pharmaceuticals (NASDAQ: VRTX), Fate Therapeutics (NASDAQ: FATE), and Ardelyx (NADSAQ: ARDX) at the center of a massive transformation in chronic healthcare.  Smart investors are preparing for a major 2026 rebound as capital flows into the most innovative and results-driven medical platforms[3]. With the total biotech sector projected to reach $9.06 trillion by 2035[4], the biggest opportunities are in companies solving massive issues like diabetes and kidney disease with better clinical execution. In 2026, the market is favoring 'ready-to-go' platforms over early-stage speculation, rewarding the companies that have built the infrastructure to dominate the next generation of medicine.Avant Technologies, Inc. (OTCQB: AVAI) is tackling some of healthcare's biggest challenges with a novel approach: genetically modified cells that produce therapeutic proteins inside the body, protected by a proprietary shield that keeps the immune system from attacking them. The company operates through two joint ventures targeting markets worth hundreds of billions of dollars.The core innovation is a cell encapsulation technology that solves a fundamental problem in regenerative medicine. When doctors transplant therapeutic cells into patients, the immune system typically destroys them within days or weeks. The traditional solution requires lifelong immunosuppressive drugs that cause serious side effects including infection risk, organ damage, and elevated cancer risk. Avant's technology eliminates this problem by creating a protective barrier around the cells while still allowing nutrients, oxygen, and therapeutic proteins to pass through freely.The first venture, Insulinova, Inc., partners with SGAustria Pte. Ltd. to develop treatments for type 1 diabetes and insulin-dependent type 2 diabetes. The approach uses genetically modified cells that produce, regulate and store insulin, essentially creating a bio artificial pancreas that restores natural glucose control without immunosuppressive drugs. The diabetes market opportunity is substantial: 589 million people globally live with type 1 and insulin-dependent type 2 diabetes, projected to reach 853 million by 2050 according to the International Diabetes Federation."Cell encapsulation is a game changer in the field of regenerative medicine," said Chris Winter, CEO of Avant Technologies. "By partnering with SGAustria, we're ensuring that genetically modified insulin-producing cells can thrive in the body long-term and offer the potential of restoring natural glucose control and dramatically improving patients' quality of life. This technology not only minimizes risks like immune rejection but also prevents potential complications such as cell escape or tumor formation, making it a cornerstone for safe and scalable diabetes therapies."Avant's second venture, Klothonova, partners with Singapore-based Austrianova to develop therapies for both age-related diseases and anti-aging therapies using cells that produce the Klotho protein. Research from the Mayo Clinic links declining Klotho levels to arterial stiffness, endothelial dysfunction, and vascular calcification.Both platforms are backed by over 50 peer-reviewed publications representing decades of development. The addressable markets span multiple areas: Alzheimer's disease ($32.8 billion by 2033), cardiovascular disease (32% of global deaths), and kidney disease (850 million affected worldwide).The strategic advantage lies in platform versatility. The same encapsulation technology that protects insulin-producing cells can theoretically protect cells producing other therapeutic proteins, potentially opening pathways into additional disease indications. This positions Avant at the intersection of multiple high-value healthcare markets with a single core technology that addresses the immune rejection challenge across different applications.CONTINUED... Read this and more news for Avant Technologies at:
https://equity-insider.com/2025/03/21/unlocking-the-trillion-dollar-ai-market-what-investors-need-to-know/ MannKind (NASDAQ: MNKD) provided business updates outlining anticipated growth drivers for 2026, including progress across commercial programs and clinical development initiatives following a record-setting fourth quarter surpassing $100 million in net revenue. The company expects two high-potential launches on the horizon with FDA decisions anticipated for Afrezza label updates and FUROSCIX ReadyFlow Autoinjector."MannKind closed 2025 on a high note, marked by milestones that reinforce our growth trajectory—including the acquisition of scPharmaceuticals and a record-setting fourth quarter surpassing $100 million in net revenue," said Michael Castagna, PharmD, CEO of MannKind Corporation. "With two high-potential launches on the horizon, 2026 is shaping up to be a catalyst-rich year that positions MannKind for long-term value creation."Major catalysts include an FDA decision on Afrezza dose conversion with PDUFA date of January 23, 2026, and review of the supplemental BLA for Afrezza in pediatric patients with PDUFA date of May 29, 2026. MannKind also continues advancing its pipeline including nintedanib DPI with first patient enrollment in the INFLO-1 Phase 1b study completed in December.Vertex Pharmaceuticals (NASDAQ: VRTX) provided pipeline and business updates ahead of the J.P. Morgan Healthcare Conference, highlighting strong commercial execution and rapid R&D progress setting up continued growth and important milestones for 2026. The company increased estimates for people with CF in all target markets to approximately 112,000 including approximately 97,000 in core markets, while CASGEVY realized greater than $100 million revenue in 2025 reflecting more than 60 patient infusions."2025 was a year of strong commercial execution and rapid R&D progress, setting up the company for continued growth and many important milestones in 2026," said Reshma Kewalramani, M.D., CEO and President of Vertex. "Building on this momentum, we are focused on expanding our commercial reach in multiple disease areas; advancing the emerging renal franchise, including the potential near-term launch of povetacicept; and progressing our mid- and late-stage clinical pipeline."Vertex expects to complete the rolling BLA filing for U.S. accelerated approval of povetacicept in IgAN in the first half of 2026 using a priority review voucher to expedite review from ten months to six months. The company also plans to complete enrollment in both Phase 3 studies of suzetrigine in diabetic peripheral neuropathy by end of 2026, with more than 500,000 JOURNAVX prescriptions written and filled in 2025.Fate Therapeutics (NASDAQ: FATE) presented updated Phase 1 clinical data of FT819 off-the-shelf CAR T-cell product candidate demonstrating meaningful decrease in disease and favorable safety profile with twelve systemic lupus erythematosus patients now treated and first systemic sclerosis patient dosed. The company continues to advance preparations for a pivotal study and is engaged in discussions with the FDA under its RMAT designation regarding plans to initiate registrational trial in 2026."We are very pleased with the accelerating patient enrollment, the expansion of U.S. clinical sites, and the addition of international clinical sites, which together are enabling broader access to FT819 for patients suffering with lupus," said Bob Valamehr, Ph.D., M.B.A., President and CEO of Fate Therapeutics. "The updated FT819 clinical data continue to demonstrate meaningful and durable responses with the use of less-intensive conditioning chemotherapy and a differentiated safety profile."Preliminary data in Regimen A showed mean SLEDAI-2K score decreased progressively from baseline with DL1 dropping 50% at month 3 and 70% at month 6, while DL2 decreased 65% at month 3 and 78% at month 6. Clinical SLEDAI-2K of 0 was achieved in 5 out of 10 patients with no Grade >2 CRS, ICANS, or GVHD reported.Ardelyx (NASDAQ: ARDX) presented real-world evidence studies of XPHOZAH (tenapanor) demonstrating patient satisfaction and reduction in serum phosphate at the American Society of Nephrology's Kidney Week. The first real-world study showed patients prescribed tenapanor experienced a reduction in serum phosphate of nearly 1 mg/dL on average, with 45.3% experiencing at least 1 mg/dL reduction."We are excited to present new data on XPHOZAH at ASN's Kidney Week, including the first results from our prospective, observational cohort study designed to evaluate the impact of an XPHOZAH-based regimen in a real-world setting," said Edward Conner, Chief Medical Officer. "Our results show the impact XPHOZAH can have in reducing serum phosphorus levels for these patients."Real-world survey data collected through the ArdelyxAssist patient services program showed that 63% of patients reported their phosphate levels were better since starting tenapanor. Among patients who reported a change in serum phosphate levels, 69% indicated their outlook on serum phosphate control was better, with improvements attributed to better control, improved bowel movements, and lower pill burden.Source: https://equity-insider.com/2025/03/21/unlocking-the-trillion-dollar-ai-market-what-investors- need-to-know/ CONTACT:
Equity Insider
info @acblanke1DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Avant Technologies Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares Avant Technologies Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Avant Technologies Inc. which were purchased in the open market. MIQ reserves the right to buy and sell, and will buy and sell shares of Avant Technologies Inc. at any time thereafter without any further notice. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material disseminated by MIQ has been approved by the above mentioned company; this is a paid advertisement, and we own shares of the mentioned company that we will sell, and we also reserve the right to buy shares of the company in the open market, or through other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment.SOURCES CITED:https://www.mordorintelligence.com/industry-reports/global-regenerative-medicines-market-industryhttps://www.fda.gov/news-events/press-announcements/fda-increases-flexibility-requirements-cell-and-gene-therapies-advance-innovationhttps://www.biospace.com/drug-development/biotech-investors-bet-on-a-2026-rebound-as-deal-activity-accelerateshttps://www.globenewswire.com/news-release/2026/01/20/3221521/0/en/Biotechnology-Market-Size-to-Surpass-USD-9-06-Trillion-by-2035.htmlLogo: https://mma.prnewswire.com/media/2840019/Equity_Insider_Logo.jpg





View original content to download multimedia:https://www.prnewswire.com/news-releases/breaking-barriers-how-2026s-top-clinical-leaders-are-disrupting-chronic-disease-markets-302670105.html

Original: Breaking Barriers: How 2026's Top Clinical Leaders Are Disrupting Chronic Disease Markets

$VRTX "CRISPR as if playing GOD with a gene editing adulterated and contaminated bio weapon passed off as a “vaccine” wasn’t enough" CRISPR

as if playing GOD with a gene editing adulterated and contaminated bio weapon passed off as a “vaccine” wasn’t enough

Let me offer you a solution to your vaxinjury

CRISPR

Their solution to the DOD fuck up of deploying a bioweapon and injecting billions of people with… pic.twitter.com/BivVv2miWz— Lyndsey, RN 💜🐭 (@HouseLyndseyRN) March 19, 2025

Finally making money here ..VRTX$ ...

Removing COVID-19 vaccines from the market would eliminate a major driver of today’s chronic disease epidemic. Here’s why👇

🔹 Post-COVID-19 Vaccine Syndrome (PCVS) may affect 64.6% of vaccinated individuals for at least a year—rising to 83.9% in those who received boosters… https://t.co/KBJcBfYlf7 pic.twitter.com/73aV1lZQAD— Nicolas Hulscher, MPH (@NicHulscher) May 15, 2025 $VRTX

pic.twitter.com/UsO5ZV4oY3— Died Suddenly (@DiedSuddenly_) May 9, 2025 $VRTX

VRTX, 10Q due Monday 5/5

Great product expansion. :)

VRTX, 10Q due MONDAY

VRTX, new 52 week low

VRTX, 10Q due. 11/4

VRTX not far from a 52 week high

VRTX new 52 week high

VRTX new 52 week high

VRTX 10Q due 5/6 PM

PHARMA
Vertex Made a Fortune Curing Rare Diseases. Its Next Bet Is Developing a Non-Addictive Opioid Alternative.

img1
There’s more to pharma than just COVID vaccines and weight-loss medications. Thirty-five years ago, Vertex Pharmaceuticals (NASDAQ:VRTX) took a fresh approach to treating illnesses by targeting human cells and genes. Except it spent 20 years with bleak growth prospects before scoring its first blockbuster drug approval in 2012.
Since then, Vertex has generated over $20B in profits attacking cystic fibrosis at the source. And last year, it became the first company to bring a gene-editing therapy to market, a generational moment for the biopharma industry that pushed Vertex into the top 10 US pharma companies.
We feel heal your pain: Yesterday, Vertex announced positive clinical trial results from its experimental non-addictive alternative to opioid painkillers, VX-548, which blocks pain signals from reaching the brain. This opens the door for the “first new acute pain medicine in 25 years,” Vertex Chief Scientific Officer Dr. David Altshuler tells Barron’s.
While VX-548 might not match the effectiveness of Vicodin (opioid med), analysts predict it could replace addictive opioids for cases of “moderate-to-severe pain” once approved in early 2025.
The drug could cater to over 80M patients experiencing acute pain, a vast market that analysts at Leerink Partners forecast will help VX-548 generate $5.1B in sales by 2030.
Vertex’s next act
Altogether, Vertex has over a dozen treatments in research or studies — a strategy deemed “more important than ever” by RBC Capital Markets analyst Brian Abrahams, as it will drive the next leg of the company’s growth.
Vertex plans to expand its pain and blood disorder franchises but boasts a pipeline of gene-editing treatments with CRISPR Therapeutics (NASDAQ:CRSP), which could eventually treat Muscular Dystrophy and Type 1 Diabetes.
Focusing on rare diseases has also allowed Vertex to skip some costly drug filing fees and secure fast-track approval — all while charging hefty premiums for its orphan drugs.
Forward-looking: Unlike many other pharma giants facing patent expirations, Vertex doesn’t have any of its major drugs reaching a patent cliff anytime soon, leaving the company ample time to commercialize its new cures.

VRTX new 52 week high

VRTX new 52 week high

VRTX new 52 week high

Life is a journey

NICE. I was right about CRSP though, but VRTX as I said puzzled me. LOL

VRTX new 52 week high

Ok so maybe the FDA approval on Dec. 8th is already built into the price of CRSP during it's recent surge? That is kind of what I am thinking that if they do get the approval, that maybe it doesn't go up much more if any at all? But VRTX going down on the UK day of approval seems to me a puzzle?
By the way I am 76 years old and my favorite old car was the '57 Chevy.
My mom had a '58 though and my first car was an old '53 Ford. LOL.
Good luck with the book.
My only book can be bought on Kindle: "Making Music Money Memories"

Go VRTX and CRSP.

Profit taking
CRSP has a good chance for approval

Why do you think VRTX went down on the day they announced the FDA approval in the UK? And do you think this will go up along with CRSP on DEC. 8th if the FDA approves? These are new to me.

Does anyone think VRTX will get a boost as CRSP would if FDA approves on 12/8?

VRTX new 52 week high

VRTX new 52 week high

VRTX new 52 week high

VRTX new 52 week high

From Cathie Wood
Gene-Editing Therapies Could Rearrange Healthcare Expenditures In The US
ARK Invest_Illustration_Ali Urman_Final_Circle 400 px By Ali Urman | @aurmanARK
Analyst
Last week, Vertex Pharmaceuticals presented3 promising results from a Phase I/II trial focused on Type 1 Diabetes (T1D): VX-880, an allogeneic stem-cell derived islet cell therapy infused into the hepatic portal vein for T1D patients with severe hypoglycemia. Importantly, the six patients produced endogenous insulin, reducing the need for exogenous insulin and showing blood glucose control with a reduction of HbA1c.

In the US today, healthcare spending approximates $4.3 trillion,4 of which therapeutics accounts for ~15%, or $600 billion,5 as shown below.



In our view, novel gene-editing therapies could command premium pricing by shifting healthcare expenditures from chronic disease management to one-dose therapies like VX-880. Addressing diabetes, for example, therapeutics account for 30% of total direct and indirect costs, as shown below. If emerging drugs like VX-880 were to shift costs from chronic disease management to one-dose therapies, curative therapeutics could take share from chronic treatments.

FDA approval for Cystic Fibrosis drug for children.
Why isn't this moving up?

Cure for Diabetes Type 1 would be huge !!!

Vertex surprised by FDA halt of diabetes cell therapy study

https://www.biopharmadive.com/news/vertex-diabetes-clinical-hold-cell-therapy/623010/

Published May 2, 2022

Dive Brief:
The Food and Drug Administration has stopped Vertex from continuing a clinical trial testing a promising cell therapy for Type 1 diabetes, judging the company has “insufficient information” to study a higher dose of the treatment as planned.
The clinical hold, which Vertex announced in a statement Monday, comes after startlingly positive results from the first patient given the therapy, known as VX-880. Nine months after the treatment, that patient no longer needed to take synthetic insulin, while blood and laboratory testing indicated his body was producing its own.
Vertex treated the first and second patients in its trial with a half dose and, following the recommendation of independent trial monitors, treated a third with a full dose in the second phase of the study, which is meant to enroll four other participants. That progression seems to be what the FDA is questioning with its hold, which Vertex said it would work to address.
Dive Insight:
From Vertex’s perspective, the results it’s obtained from the first two patients in its study represent “proof of concept” for a treatment that’s designed to replace the insulin-producing cells destroyed by Type 1 diabetes with ones made from stem cells.

Initial results from the first patient, released last October, helped to validate the company’s approach, which was developed over decades by the laboratory of Doug Melton, a Harvard University professor who recently joined Vertex as a distinguished fellow. (Melton founded a biotech company called Semma Therapeutics, which Vertex bought in 2019 for nearly $1 billion.)

That patient no longer needs to inject himself with synthetic insulin, and further follow-up data indicates his body is now able to control blood sugar levels. According to Vertex, a second patient has been able to reduce insulin use by about a third and shows signs that treatment is restoring insulin production as well.

A third patient, who was given a full dose of VX-880, is only 29 days out from treatment and will be more comprehensively examined after three months.

No serious adverse reactions have been reported, while the most commonly reported side effects were judged unrelated to treatment.

However, the FDA, at least according to Vertex, doesn’t agree the company has enough information to proceed with “dose escalation,” an industry term for evaluating progressively higher doses of a treatment.

“We are surprised by the clinical hold placed on the study,” said Carmen Bozic, Vertex’s chief medical officer, in a statement.

The company said it will work with the agency to understand and address its concerns “so that the trial can resume at U.S. sites as soon as possible.” Nine of the 10 study sites are located in the U.S., while the 10th is in Montreal, according to a federal database of clinical trials.

Overall, Vertex intends to enroll 17 patients in the early-stage trial, which is divided into three phases.

VX-880 consists of pancreatic islet cells that are derived from stem cells and differentiated into their mature form. It’s administered by an infusion into a vein that conveys blood to the liver. Patients treated with VX-880 must take immunosuppressants so their immune system doesn’t destroy the islet cells.

Vertex is also working on another version of VX-880 “encapsulated” in an immunoprotective device that’s surgically implanted. The company aims to ask the FDA later this year for clearance to begin human testing.

The hold is an unexpected roadblock, however, and it’s unclear what impact the delay might have on Vertex’s plans. According to Michael Yee, an analyst at Jefferies, recent clinical holds for cell therapies have been lifted quickly, generally within four to five months.

“We’re hopeful for a speedy resolution and [management] also indicated they are being very transparent [with the] FDA,” Yee wrote in a May 2 note to clients.

Why is this going down??

News: $VRTX 3 Top Healthcare Stocks to Buy Now

Many things we spend on our money on are wants rather than needs. We could live without cars, smartphones, and streaming services if we had to. But what about healthcare? It's a need. What's more, demand for healthcare-related products and services is growing. An increasing senior population in...

In case you are interested VRTX - 3 Top Healthcare Stocks to Buy Now

News: $VRTX 3 Top Stocks to Buy in November

Trade tensions with China. A presidential impeachment inquiry underway in the U.S. House of Representatives. You might think that the stock market would be a scary place right now. Instead, major market indexes are at all-time highs. History has shown that the stock market often "rides a wall o...

Read the whole news VRTX - 3 Top Stocks to Buy in November

Great news out.

Not in this play but this can be long term payout with CF drug.

Maybe bring PPS near a normal evaluation instead of several times book value.

GLTYA especially the longs from the dip earlier this year.