Pemphigus, Benign Familial

Fresenius Medical Care achieves growth milestone of more than 14,000 U.S.-based patients using the Company's NxStage systems to perform Home Hemodialysis therapy This growth has generated greater adoption from a range of dialysis providers and delivers on the company's commitment to grow access to and use of home dialysis therapy The newest NxStage® Versi®HD with GuideMe Software home dialysis machine is designed to simplify treatment, increase ease of learning, and improve user experience WALTHAM, Mass., Sept. 4, 2024 /PRNewswire/ -- Fresenius Medical Care (FME), a global leader in kidney care, has achieved a growth milestone of more than 14,000 U.S.-based patients using the Company's NxStage systems to perform Home Hemodialysis (HHD) therapy through both Fresenius Kidney Care clinics and other providers. The company has in parallel launched the newest version of its home dialysis machine, the NxStage® Versi®HD with GuideMe Software, featuring enhancements designed to simplify treatment, increase ease of learning, and improve user experience. These achievements highlight the efficacy and reliability of the NxStage system in delivering safe, high-quality care to patients in the comfort of their homes. Continue Reading Fresenius Medical Care Grows Number of U.S. Patients Using Company’s Home Hemodialysis Machines, Launches NxStage® Versi®HD with GuideMe Software Between January and April 2024, the number of new HHD patients starting treatment with NxStage in the United States grew by 18% compared to the same period in 2023. For example, April saw 927 new patients initiating HHD therapy in the U.S. "We are proud to announce these achievements in our Home Therapies division," said Joseph Turk, Executive Vice President and Global Head of Home Therapies at Fresenius Medical Care. "These accomplishments underscore our relentless commitment to continuous innovations that enable us to improve the lives of patients seeking the safety, convenience, and flexibility of home dialysis." Since the FDA clearance in 2023, the newest NxStage HHD System, Versi®HD with GuideMe Software, has garnered significant interest in the United States. Large, mid-sized, and several independent dialysis providers are moving forward with the adoption of Versi®HD with GuideMe Software. Versi®HD with GuideMe Software's advanced technology is designed to simplify HHD treatment and improve ease of learning, skill retention, and user experience for patients and care partners. The machine's pictorial-based guidance was designed to meet diverse patient needs throughout the setup, treatment and troubleshooting, while improving training time and skill retention. It provides graphical walk-through guidance that aims to enhance ease of use and confidence for both patients and nurses. The interface is designed to accommodate a wide variety of learning styles and improve skill retention from initial training, while static picture instructions allow patients to linger and study any step at their own pace. "The patient training on Versi®HD with GuideMe-Software at our clinic catch on quickly and feel comfortable by the second week of training. Even better, they are able to complete their training seven to 10 days sooner," said Sandra Flores, Home Therapies Registered Nurse in North Central Oklahoma City. Currently, NxStage Versi®HD with GuideMe Software is being fully launched in the U.S. with planned global rollouts over the coming years. For more information about Fresenius Medical Care's Home Therapies, please visit © 2024 Fresenius Medical Care. All Rights Reserved. Fresenius Medical Care, the triangle logo, NxStage, and Versi are trademarks of Fresenius Medical Care Holdings, Inc. or its affiliated companies. All other trademarks are the property of their respective owners. About Fresenius Medical Care: Fresenius Medical Care is the world's leading provider of products and services for individuals with renal diseases of which around 4.1 million patients worldwide regularly undergo dialysis treatment. Through its network of 3,757 dialysis clinics, Fresenius Medical Care provides dialysis treatments for approx. 311,000 patients around the globe. Fresenius Medical Care is also the leading provider of dialysis products such as dialysis machines or dialyzers. Fresenius Medical Care is listed on the Frankfurt Stock Exchange (FME) and on the New York Stock Exchange (FMS). For more information visit the Company's website at . Disclaimer: This release contains forward-looking statements that are subject to various risks and uncertainties. Actual results could differ materially from those described in these forward-looking statements due to various factors, including, but not limited to, changes in business, economic and competitive conditions, legal changes, regulatory approvals, impacts related to the COVID-19 pandemic results of clinical studies, foreign exchange rate fluctuations, uncertainties in litigation or investigative proceedings, and the availability of financing. These and other risks and uncertainties are detailed in Fresenius Medical Care's reports filed with the U.S. Securities and Exchange Commission. Fresenius Medical Care does not undertake any responsibility to update the forward-looking statements in this release. Media contact Emanuela Cariolagian T +1 213 706 0051 [email protected] Christine Peters T +49 160 60 66 770 [email protected] Contact for analysts and investors Dr. Dominik Heger T +49 6172 609 2601 [email protected] SOURCE Fresenius Medical Care Holdings, Inc.

Aside from entrenched rivals like AstraZeneca’s Ultomiris, Vyvgart is now facing pressure from a pair of myasthenia gravis biologics newcomers introduced by UCB. As argenx continues to scoop up biologics market share with its antibody fragment Vyvgart in generalized myasthenia gravis (gMG), the Dutch immunology outfit is confident that it’s “just at the beginning of the growth curve” in its inaugural indication, executives said on an analyst call Thursday. Not content to rest on its laurels, either, argenx is leveraging the commercial insights it’s garnered over the past two years as it preps for the launch of Vyvgart’s next potential approval in chronic inflammatory demyelinating polyneuropathy (CIDP), slated for an FDA decision by June 21. Argenx this morning reported 83% year-over-year and 6% quarter-over-quarter revenue growth to $398 million in the first three months of 2024. The autoimmune company’s sales currently hinge on Vyvgart and the drug’s Halozyme-partnered subcutaneous formulation Vyvgart Hytrulo, both of which are approved in gMG in the U.S. and many other countries. Vyvgart also boasts a single approval for the rare bleeding disorder immune thrombocytopenia (ITP) in Japan. The company’s sales growth this quarter reflects the “strong medical need in myasthenia gravis,” as well as the “quality” of Vyvgart and the “excellent execution” of the drug’s commercial rollout, analysts with ODDO BHF wrote in a note to clients Thursday. The analysts stuck by their thesis that the products remain well positioned to hit peak sales of $11 billion in 2035—an estimate that accounts for future indications like CIDP, too. Aside from the opportunities in gMG and CIDP, argenx also aims to broaden Vyvgart’s reach with the introduction of a prefilled syringe. The company said in an earnings release that it’s on track to submit a U.S. application for that delivery format in 2024’s second quarter. Still, Vyvgart is far from the only gMG biologic in town. Aside from entrenched rivals like AstraZeneca’s Ultomiris, Vyvgart is now facing pressure from a pair of newcomers introduced by UCB. UCB won back-to-back nods for two separate gMG therapies last year, starting with a green light for its subcutaneous monoclonal antibody Rystiggo in July. In October, the company’s med Zilbrysq—a targeted C5 inhibitor with a different mechanism of action than Rystiggo—snared FDA backing as the first subcutaneous gMG-targeted therapy patients can administer themselves, according to UCB. But despite those new challengers, argenx’s chief operating officer, Karen Massey, said the company isn’t seeing a significant impact from competition.  In fact, argenx continues to see Vyvgart’s market share grow quarter over quarter among biologics, she said during the call. She credited much of that growth to the product’s subcutaneous version, which saw a 34% uptick in U.S. patients this quarter. Massey said the company continues to see many new patient starts on Vyvgart, with more than 50% of those novel prescriptions coming from people who are switching over from an oral gMG therapy. “I think the more innovation that comes to market, the more the biologic share overall is going to grow and the more we’ll be able to help lead within that biologic share,” Massey said. “I’m very confident that we’re just at the beginning of the growth curve with MG,” the COO added. Building on Massey’s point, argenx CEO Tim Van Hauwermeiren noted on the company’s call that argenx recently kicked off a Vyvgart study in seronegative myasthenia gravis as well, which has the potential to become a label-expanding study. Roughly 15% of gMG patients are estimated to be seronegative, which could provide a meaningful addition to the drug’s current green light, the CEO said. All told, there are now more than 10,000 gMG patients on argenx’s treatment globally, the company noted (PDF) in a slide deck accompanying its earnings presentation. As for Vyvgart’s upcoming opportunity in CIDP, argenx has been busy establishing a “comprehensive launch plan” to help educate both neurologists and patients around the benefits of switching over to the med, Massey said. With the CIDP therapies on tap today—typically steroids and intravenous immunoglobulin—roughly 88% of patients still experience residual symptoms despite ongoing treatment, argenx’s research has shown. Given the drug’s successful phase 3 trial in CIDP last month and its clean safety profile, argenx figures patients and doctors will be enticed to make the switch to Vyvgart because they won’t “have to balance that trade-off between efficacy and treatment burden,” Massey said. Argenx plans to adopt the same pricing approach it used in gMG for Vyvgart’s potential green light in CIDP, including through value-based arrangements with payers, Massey said, noting access talks for that potential launch are “on track.” Since its commercial debut in late 2021, argenx has touted the potential for Vyvgart as a pipeline in a product. But while Vyvgart is faring well in many disease areas, the drug has shown its limitations in recent months, too. Alongside earnings Thursday, argenx revealed that it’s discontinuing planned development of the FcRn inhibitor in ANCA-associated vasculitis, which describes a group of rare autoimmune diseases marked by inflammation and damage to the small blood vessels. The decision comes after a risk assessment of ongoing studies following recent clinical misfires in ITP and the chronic blistering disease pemphigus.

None Home hemodialysis (HHD) may offer improved health and quality of life for patients with end-stage kidney disease (ESKD), which affects more than 800,000 Americans.[i] However, due to patient fears and access concerns, nurse shortages, and the perception of extensive training time, HHD utilization remains low. Despite its association with improved patient outcomes,[ii] far too many patients are missing the opportunities to receive a more agile, flexible treatment modality.   Comprehensive and fresh approaches to HHD training are crucial to improve patient adoption rates. Kidney care providers can help increase the uptake of this underused but vital treatment through group training, an intriguing model of patient education.   Why Increasing HHD Uptake Matters   Home dialysis uptake has climbed in the past decade, yet HHD is lagging:   From 2011 to 2021, the percentage of incident patients on home dialysis increased to 13.4% – but just 0.4% started with HHD in 2021.iii Among new and preexisting patients, home dialysis use increased from 9.7% to 14.1%– but HHD only increased from 1.5% to 2.3%.[iii] Among the total patients with ESKD at Fresenius Kidney Care, HHD percentage increased from 1.2% in 2013 to 4.2% in 2022.[iv]   A group training model is key to increasing the number of home hemodialysis users – something a clinic in Clayton, North Carolina has seen first-hand.   Group Training in Action Fresenius Kidney Care Stallings Station serves patients on the outskirts of Raleigh. With the patient population generally having lower health literacy, many patients have not considered home dialysis since in-center treatment has been the default modality. In 2021, the clinic piloted a new model in which training is done in groups. Participants could see others learn the steps multiple times, reinforcing each individual’s knowledge. The training is also done in sight of in-center patients who are encouraged to join.   “It all begins with education and encouragement,” says Phyllis Merritt-James, a nurse practitioner at the clinic. “Once we’re able to educate patients about the program, they’ve been much more open to exploring it.”   This approach led to a spike in HHD patients and improved patient retention at the clinic. "Our HHD group training model, despite our dialysis staffing shortage, has led to a rapid and sustained HHD growth of 25% over 12 months," said Dr. Sejan Patel, the center’s nephrologist. There are now more home patients than in-center patients. The rate of patients continuing with HHD 120 days after training completion improved from 78% before the model’s introduction to nearly 84% afterward.   More People Means Less Time   Group training can allow more patients to train more quickly than individual training. Healthcare training teams will always ensure that patients are comfortable and can safely perform their treatments before going home. ​In a study of HHD patients, half were trained on a NxStage system in 10 sessions in 2 weeks or less.[v] While training times vary and depend on patient experience, being able to train quickly is a testament to NxStage systems being designed for simplicity and ease of use by patients at home.   The clinic provides comprehensive training including cannulation so patients can take on this new treatment at home – something patients at the center can attest to.   “This training model is second to none,” says Rickie Corey, an HHD patient at the clinic. “When my wife and I were told we could go home after 10 days of training, we couldn’t believe it, but they prepared us so well for doing this at home. I’m feeling worlds better.”   Addressing Staff Availability   The COVID-19 pandemic spurred increased interest in home dialysis. However, nursing shortages meant that there were fewer staff to train patients. The idea of group training was born. The 4:1 patient-to-nurse ratio in training sessions has proven to be successful, as the nephrologist, nurses, and other staff have created a sense of community while supporting individual patients.   Growing a Support System   The group training model offers a built-in support system. Patients can benefit from group discussion, learn from each other and reinforce what they’ve learned. For others, the group setting helped them overcome personal fears.   “Due to my extreme fear of needles, I was in tears when I had to stick myself,” says patient Christin Elrod. “Then, I watched the other patients in the group stick themselves and that gave me the courage to do it myself. I now do it myself every day.”   Groups As the Gold Standard   The group training model offers an easy and cost-effective way to help remove barriers that prevent HHD uptake – personal patient fears, concerns over training time, and staff shortages. This approach should be considered the gold standard for dialysis centers in training new patients.   For more information on HHD products, training and education resources, visit www.NxStage.com. Not all patients may experience these benefits. [i] USRDS 2023 ADR: United States Renal Data System. 2023 USRDS Annual Data Report: Epidemiology of kidney disease in the United States. National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD, 2023. [ii] https://www.nxstage.com/patients/benefits-of-home-hemodialysis/ [iii] https://usrds-adr.niddk.nih.gov/2023/end-stage-renal-disease/2-home-dialysis [iv] Data on file, Fresenius Medical Care [v] Cherukuri et al, doi: 10.1186/s12882-018-1059-2