Urinary Calculi

Abbott's world-leading1 FreeStyle Libre 2 and 3 systems for people living with diabetes are now the only continuous glucose monitoring sensors 2 in the U.S. that users do not need to remove during common imaging procedures like X-rays, CT scans and MRIs* ABBOTT PARK, Ill. – Oct. 30, 2024 – Abbott today announced that its FreeStyle Libre 2 and 3 systems can now be worn during common imaging procedures, such as X-rays, CT scans and MRIs*. The U.S. Food and Drug Administration (FDA) cleared the removal of the imaging contraindication, making Abbott’s systems the first and only patient-applied continuous glucose monitoring (CGM) sensors approved for these screenings. People who use FreeStyle Libre 2 and 3 systems rely on their CGM sensors to make important decisions about their diabetes, including when to take insulin. They will no longer have to remove and discard their CGM sensor before its wear time has ended, providing them with increased convenience and the potential to save money.  "For people with diabetes, especially those using insulin, removing a CGM sensor for long periods can be problematic," said Carol Wysham, M.D., clinical professor of medicine at the University of Washington School of Medicine and section head of the department of diabetes and endocrinology at Rockwood Clinic in Spokane. "Previously, patients had to remove their sensors during these procedures, resulting in several hours without critical data, especially if they didn’t have a replacement sensor. The removal of the imaging contraindication from Abbott’s FreeStyle Libre 2 and 3 systems is a big win for patients, allowing them to keep their sensors on and avoid lost data." Diabetes can cause complications like cardiovascular disease, neuropathy, and infections3. Imaging procedures are often part of diabetes care: X-rays detect bone injuries, CT scans identify kidney stones and blood clots, and MRIs provide detailed images of organs and tissues to identify issues such as brain injuries, ischemic heart disease and fatty liver disease4,5. Abbott rigorously tested its FreeStyle Libre 2 and 3 systems sensors to ensure they remain effective after radiologic procedures*. This testing led the FDA to clear the removal of the contraindication requirement, with no changes made to the sensor. "Continuous glucose monitoring is essential for diabetes management," said Anila Bindal, M.D., associate medical director for Abbott's diabetes care business. "We’ve seen first-hand how consistent use of Libre technology benefits people living with diabetes. This contraindication removal aligns with Abbott’s commitment to provide easy, convenient, and affordable technology for diabetes management." About Abbott:  Abbott is a global healthcare leader that helps people live more fully at all stages of life. Our portfolio of life-changing technologies spans the spectrum of healthcare, with leading businesses and products in diagnostics, medical devices, nutritionals and branded generic medicines. Our 114,000 colleagues serve people in more than 160 countries. Connect with us at www.abbott.com and on LinkedIn, Facebook, Instagram, X and YouTube.  Important Safety Information: Product for prescription only, for Important Safety Information, please visit https://www.freestyle.abbott/us-en/safety-information.html. *MRI Safety Conditions: Sensor readings may be compromised during the MRI, but System function returns fully back to normal after 1 hour. Scanning between the pelvis and sternum (3T scanners) should be limited to 12 minutes maximum with a cooling period of 2 minutes in between scans. All other areas (1.5T scanners) can scan for up to 1 hour continuously without a cooling period.  1Data on file. Abbott Diabetes Care. Data based on the number of patients assigned to each manufacturer. 2Among patient applied sensors 3American Heart Association, Diabetes Complications and Risks, accessed August 22, 2024. 4Radiopaedia, Diabetes mellitus: Radiology Reference Article , accessed August 22, 2024. 5The Diabetes Council, X-ray and Diabetes: How can it help, accessed August 22, 2024.

- New data on povetacicept 80 mg SC Q4 weeks in IgA nephropathy shows mean UPCR reduction from baseline of 66% observed at 48 weeks, associated with stable renal function (eGFR) and 63% achieving clinical remission -- - First proteinuria data on povetacicept in primary membranous nephropathy shows mean UPCR reduction from baseline of 62% at 24 weeks – - Global Phase 3 RAINIER trial of povetacicept in IgA nephropathy now underway – – Enrollment and dosing ongoing in Phase 3 portion of the global Phase 2/3 pivotal clinical trial of inaxaplin for the treatment of APOL1-mediated kidney disease – BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today provided updates on multiple kidney diseases in its pipeline including IgA nephropathy (IgAN), primary membranous nephropathy (pMN), and APOL1-mediated kidney disease (AMKD). These updates demonstrate the transformative potential of Vertex’s investigational therapies in multiple serious kidney diseases, and include positive new data on povetacicept, a dual inhibitor of the BAFF and APRIL pathways, in IgAN and pMN, presented at the American Society of Nephrology’s (ASN) Kidney Week Congress on October 23-27 in San Diego, California. “We are very pleased with the broadening of our innovative pipeline in renal medicine which now spans programs in AMKD, IgAN, pMN and polycystic kidney disease,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “The new data in IgAN and pMN we shared at this year’s ASN congress continue to reinforce povetacicept as a potential best-in-class therapy and demonstrate its potential as a pipeline-in-a-product. We also continue to make progress in AMKD and autosomal dominant polycystic kidney disease (ADPKD) where we are advancing the first potential treatments to address the underlying cause of these diseases.” Povetacicept in IgAN Vertex presented data on 54 patients with IgAN who received povetacicept 80 mg or 240 mg subcutaneously every 4 weeks (SC Q4W). Treatment with povetacicept 80 mg SC Q4W demonstrated a clinically meaningful decrease in proteinuria, with a mean 66% reduction from baseline in urine protein to creatinine ratio at 48 weeks (UPCR; n=8) associated with stable renal function over 48 weeks as assessed by estimated glomerular filtration rate (eGFR). By 48 weeks, 63% (5 out of 8) of study participants achieved clinical remission, defined as UPCR 50% reduction in UPCR from baseline. Anti-PLA2R1 autoantibodies, which are a marker of disease activity and associated with clinical outcomes, decreased from baseline by a mean of 87% at week 20. Povetacicept was well tolerated in patients with pMN, with AEs that were mild or moderate in severity. There were no SAEs related to povetacicept. A poster presentation #TH-PO589 entitled “Updated Results with Povetacicept, an Enhanced Dual BAFF/APRIL Antagonist, in Primary Membranous Nephropathy (RUBY-3 Study)” was presented during the poster session on October 24 from 10:00 a.m. to 12:00 p.m. PDT. AMKD Vertex is developing inaxaplin, a potential first-in-class, investigational, oral small molecule inhibitor of APOL1, with the goal of targeting the underlying cause of AMKD. Enrollment and dosing are ongoing in the Phase 3 portion of the global Phase 2/3 pivotal AMPLITUDE clinical trial of inaxaplin. Vertex has the following AMKD poster presentations at ASN: Poster presentation #TH-P01203 entitled “AMPLITUDE: A Phase 2/3 Adaptive Trial of Inaxaplin in APOL1-mediated Kidney Disease” was presented during the poster session on October 24 from 10:00 a.m. to 12:00 p.m. PDT. Poster presentation #SA-PO701 entitled “Small Molecule APOL1 Channel Inhibitor Reduces Proteinuria, Rescues Podocyte Injury, and Reverses eGFR Decline in an APOL1-Mediated Kidney Disease Mouse Model” will be presented during the poster session on October 26 from 10:00 a.m. to 12:00 p.m. PDT. Poster presentation #SA-PO700 entitled “Small Molecule Inhibition of APOL1 Channel Activity Protects Podocytes from Mitochondrial Dysfunction, Cell Death and Barrier Disruption Induced by APOL1 Risk Variants” will be presented during the poster session on October 26 from 10:00 a.m. to 12:00 p.m. PDT. All accepted abstracts are available online on the ASN website . About IgA Nephropathy (IgAN) IgAN is a serious, progressive, life-threatening, B cell-mediated chronic kidney disease that is the most common cause of primary (idiopathic) glomerulonephritis, affecting people worldwide including approximately 130,000 people in the U.S. IgAN results from deposition of circulating immune complexes consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1) in the renal glomerular mesangium, triggering kidney injury and fibrosis. A high percentage of people with IgAN progress to end-stage renal disease. There are no approved therapies that specifically target the underlying cause of IgAN. About RAINIER RAINIER is a global Phase 3 pivotal trial of povetacicept 80 mg vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating UPCR for the povetacicept arm versus placebo after a certain number of patients reach 36 weeks of treatment. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Final analysis will occur at two years of treatment, with a primary endpoint of total eGFR slope through Week 104. About Primary Membranous Nephropathy (pMN) Primary membranous nephropathy is a serious, progressive, life-threatening B cell-mediated chronic kidney disease affecting people worldwide, with approximately 60,000 people diagnosed in the U.S. pMN causes a patient’s immune system to damage the glomeruli and may cause progressive loss of kidney function. There are no approved therapies that specifically target the underlying cause of pMN. About RUBY-3 RUBY-3 is an ongoing, multiple ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, pMN, lupus nephritis and ANCA-associated vasculitis with glomerulonephritis where povetacicept is being administered subcutaneously for up to 104 weeks. About Povetacicept Povetacicept is a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in pathogenesis of multiple autoimmune diseases via their roles in the activation, differentiation and/or survival of B cells, T cells and innate immune cells. Based upon an engineered TACI (transmembrane activator and CAML interactor) domain, povetacicept has higher binding affinity and greater potency in preclinical studies versus other inhibitors of BAFF and/or APRIL alone and has demonstrated potential best-in-class efficacy in a clinical trial in patients with IgA nephropathy and primary membranous nephropathy. Povetacicept is also in development for multiple serious diseases including other autoimmune kidney diseases and autoimmune cytopenias. About APOL1-Mediated Kidney Disease (AMKD) AMKD is a genetic kidney disease affecting approximately 100,000 people in the U.S. and Europe. AMKD is caused by two variants of the APOL1 gene, which exert a toxic effect on kidney cells, leading to cell injury, cell death and damage to glomeruli (which filter blood to the kidney). Even after treatment with currently available therapies, people with AMKD often progress to kidney failure. Kidney failure is treated with frequent, long-term dialysis or a kidney transplant. Both require lifelong treatment and follow-up and carry a high mortality risk . There are currently no approved treatments that address the underlying cause of AMKD. About Inaxaplin Inaxaplin is a potential first-in-class, investigational small molecule inhibitor of APOL1, and the first investigational therapy aimed at treating the underlying cause of AMKD. About AMPLITUDE AMPLITUDE is a global Phase 2/3 pivotal trial of inaxaplin for the treatment of AMKD, in which a 45 mg once daily oral dose is compared to placebo, on top of standard of care. The study is designed to have a pre-planned interim analysis at Week 48 evaluating estimated glomerular filtration rate (eGFR) slope, a measure of kidney function, supported by a percent change from baseline in proteinuria, in the inaxaplin arm versus placebo. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Enrollment and dosing are ongoing in the Phase 3 portion of the study. About Autosomal Dominant Polycystic Kidney Disease (ADPKD) ADPKD is the most common inherited kidney disease and one of the most common severe Mendelian genetic diseases, affecting approximately 250,000 diagnosed people in the U.S. and Europe. As an autosomal dominant disease, one affected parent can pass on the disease to their children. In most cases, ADPKD is caused by variants in the PKD1 and PKD2 genes, which express proteins known as polycystins. The majority of ADPKD patients (~80%) have a variant in the PKD1 gene, resulting in a loss of function of polycystin 1 (PC1). This leads to the proliferation of kidney epithelial cells, increased fluid secretion and the formation and expansion of numerous fluid-filled cysts. The progressive cyst formation causes an increase in kidney size and decline in kidney function. Around half of patients with ADPKD experience kidney failure by the age of 60. Kidney cysts can also lead to severe abdominal pain, cyst infection, blood in the urine and kidney stones, all of which significantly impair quality of life. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. 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 acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 15 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit or follow us on LinkedIn , Facebook , Instagram , YouTube and X . Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Carmen Bozic, M.D., in this press release, and statements regarding Vertex’s expectations and plans for the global Phase 3 RAINIER study of povetacicept, including the potential for an interim analysis to serve as the basis to seek accelerated approval in the U.S., and expectations and plans for the Phase 3 portion of the clinical trial of inaxaplin, including the potential for an interim analysis to serve as the basis to seek accelerated approval in the U.S. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company’s development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company’s website at . You should not place undue reliance on these statements, 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) Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607

Grant will lead to the creation of USDHub, a novel research tool bringing together research and clinical data from hundreds of thousands of kidney stone patients of all ages PHILADELPHIA, Oct. 15, 2024 /PRNewswire/ -- Researchers from Children's Hospital of Philadelphia (CHOP) and Vanderbilt University Medical Center were awarded a $7.37 million RC2 grant from the National Institutes of Health (NIH) to lead a five-year multisite project across the U.S. focused on kidney stone disease research. Continue Reading Dr. Gregory Tasian Led by CHOP pediatric urologist, Gregory Tasian, MD, MSc, MSCE and Vanderbilt University Medical Center urologist, Ryan Hsi, MD, the goal of this initiative is to establish a new research tool called the Urinary Stone Disease Hub (USDHub), which will aggregate data from across healthcare systems and research networks into one comprehensive platform drawing data from a cohort of more than 230,000 patients suffering from kidney stone disease. Urinary stone disease is a painful condition that can cause a substantial amount of suffering for affected patients. The prevalence of kidney stones is about 11% in Americans, and kidney stones send more than half a million patients to the emergency room each year, according to the National Kidney Foundation. Although urinary and kidney stones are traditionally thought of as an adult diagnosis, kidney stones in children are becoming more common, and CHOP clinicians encounter them daily. To address the unique needs of children with kidney stones, CHOP created the Pediatric Kidney Stone Center, a multidisciplinary program within its Divisions of Urology and Nephrology that is solely dedicated to the dietary, medical, and surgical management of kidney stones. There have been few advancements in the diagnosis and management of urinary stone disease, partly due to a lack of datasets that contain clinically important information about individuals with urinary stone disease across the lifespan. To help address these challenges, USDHub will draw from medical expertise across adult and pediatric urology and nephrology and combine informatics and AI to establish a novel data resource that includes clinically important information from CT images, clinical notes, stone composition, and 24-hour urine labs, which are not available in existing research databases. "We see this painful condition more frequently now, but strong evidence to support decisions about treating children is lacking because urinary stone disease was once rare," said Tasian, who holds the Cheers for CHOP Chair in Clinical Epidemiology of Pediatric Urological Disease. "Opportunities exist both for expanding the evidence base around the management of kidney and urinary stones and for improving the research process itself." In addition to the work being led by Drs. Tasian at CHOP and Hsi at Vanderbilt, USDHub will involve seven other sites involved are part of the STAR and PEDSnet, which are two PCORnet clinical research networks that have standardized electronic health record data. In addition to CHOP and Vanderbilt University Medical Center, USDHub sites include University of North Carolina, Medical University of South Carolina, Duke University, Texas Children's Hospital, Nationwide Children's Hospital, Lurie Children's Hospital of Chicago and Cincinnati Children's Hospital. "USDHub will be a collaborative effort to synergize efforts and expertise across adult and pediatric stone disease, and to engage patients and caregivers in the design process to ensure that our sustainable data access model meets their needs," Tasian said. "This program will finally provide us with longitudinal data and eliminate our reliance on single-center data sources that segregate pediatric and adult patients. We see the potential to significantly accelerate and improve urinary stone disease research." This research is funded by the National Institute of Diabetes and Digestive and Kidney Diseases (grant 1RC2DK140865-01). About Children's Hospital of Philadelphia: A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey, as well as the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia. In addition, its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit . Contact: Kaila Revello Children's Hospital of Philadelphia (610) 457-5916 [email protected] SOURCE Children's Hospital of Philadelphia WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED