New York University

Phase 1b study supports proof-of-concept in DOK7 congenital myasthenic syndromes Decision informed by favorable safety profile and consistent functional improvement over time across multiple efficacy measures Advancing ARGX-119 further validates strong track record of Immunology Innovation Program (IIP), argenx’s collaborative discovery model AMSTERDAM, The Netherlands I June 30, 2025 I argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its plan to advance the clinical development of ARGX-119, a first-in-class agonist antibody to muscle-specific kinase (MuSK), to a registrational study in patients with congenital myasthenic syndromes (CMS) following the analysis of topline data from the Phase 1b study. Detailed results will be presented at a future medical meeting. “The results of our Phase 1b ARGX-119 study in congenital myasthenic syndromes, an ultra-rare disorder that affects patients from birth, builds on our experience and understanding of myasthenic disorders and aligns with our aspiration to serve even more patients living with these debilitating diseases,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “ARGX-119 is the sixth molecule developed through our Immunology Innovation Program to show proof-of-concept, reflecting the strength of our innovation model where our deep knowledge of the biology and expertise in antibody engineering come together to push the boundaries of what’s possible. argenx remains focused on uncovering new biological insights into misunderstood diseases to meaningfully change the lives of patients who have long-been underserved,” said Peter Ulrichts, Ph.D., Chief Scientific Officer of argenx. The decision to advance the development of ARGX-119 in CMS is supported by the results of the Phase 1b study. ARGX-119 demonstrated a favorable safety and tolerability profile, which was the primary endpoint. Efficacy was evaluated across multiple secondary and exploratory endpoints, including Six-Minute Walk Test (6MWT), Quantitative Myasthenia Gravis (QMG) score and Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Consistent improvements were observed in treated DOK7-CMS patients through the 12-week study across multiple efficacy scores. Phase 1b CMS Study Design The Phase 1b, multicenter, randomized, double-blinded, placebo-controlled clinical trial assessed safety, tolerability, PK, immunogenicity, and preliminary efficacy of ARGX-119 in participants with DOK7-CMS. The study was also designed to demonstrate proof-of-biology through preliminary efficacy assessments, including muscle weakness, fatigability, daily activities and patient-reported global health outcomes. The clinical trial spanned approximately 11 months across a screening period (up to 28 days), a 12-week treatment period and a follow-up period of nearly seven months. At baseline, eligible participants were randomized 4:1 to receive intravenous ARGX-119 or placebo. The primary objective was to evaluate safety and tolerability; secondary objectives included PK, immunogenicity and preliminary efficacy of ARGX-119 in participants with DOK7-CMS. All but one of the patients enrolled in the Phase 1b study also participated in an observational natural history study initiated by argenx in 2024 to better understand the CMS patient journey and disease burden, helping to inform future development plans. About Congenital Myasthenic Syndromes Congenital Myasthenic Syndromes (CMS) are an ultra-rare and heterogenous group of congenital neuromuscular disorders caused by genetic defects that are essential for the integrity of the neuromuscular junction. Early age of onset and fatigable muscle weakness are considered clinical hallmarks of CMS. Muscle weakness can be debilitating and life-threatening causing difficulties in speaking or swallowing, impaired or absent mobility, proximal arm and leg weakness, and respiratory insufficiency. DOK7 variations are one of the more frequent and severe causes of CMS, accounting for approximately 24% of CMS cases. There are no approved treatments. The prevalence of CMS is estimated to be 5 per 1M (DOK7-CMS estimated to be 1.2 per 1M). About ARGX-119 ARGX-119 is a first-in-class humanized agonist monoclonal antibody (mAb) that specifically targets and activates muscle-specific tyrosine kinase (MuSK) to promote maturation and stabilization of the neuromuscular junction (NMJ). It is a mAb derived from llamas and discovered using the argenx SIMPLE Antibody™ platform technology. ARGX-119 is being developed for patients with neuromuscular disease, including congenital myasthenic syndromes (CMS), amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). ARGX-119 was developed through argenx’s IIP program in collaboration with the world’s leading key opinion leaders on MuSK and the NMJ, Professor Steven J. Burden from MGH, Professor Shohei Koide from NYU and Professor Jan Verschuuren and Associate Professor Maartje Huijbers from LUMC. About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn , Instagram , Facebook , and YouTube . SOURCE: argenx

On the sidelines of a high-level US visit focused on healthcare innovationABU DHABI, UAE, June 18, 2025 /PRNewswire/ -- The Department of Health – Abu Dhabi (DoH), the regulator of the healthcare sector in Abu Dhabi, has signed a Memorandum of Understanding (MoU) with Sanofi, a research and development-driven, AI-powered healthcare biopharma company at the BIO International Convention 2025, in Boston, USA. The collaboration will leverage the Emirate's health-tech ecosystem and advanced research infrastructure to drive the development of new global vaccines and strengthen regional capabilities in vaccine manufacturing. Continue Reading The Department of Health – Abu Dhabi and Sanofi Forge Strategic Alliance to Accelerate the Development of New Global Vaccines in Abu Dhabi (PRNewsfoto/The Department of Health – Abu Dhabi) In the presence of H.E. Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi and Laurie Gery. Global Head of Business Development, Vaccines at Sanofi, the agreement was signed during the BIO International Convention in Boston, USA, by Dr. Asma Al Mannaei, the Executive Director of the Health Life Sciences Sector at the DoH and Baptiste de Clarens, General Manager Greater Gulf, Vaccines at Sanofi. Dr. Asma Al Mannaei, the Executive Director of the Health Life Sciences Sector at the DoH, said: "DoH is partnering with Sanofi to expedite the development of new global vaccines. This initiative aims to shorten the timeline from early research to public availability by utilising advanced technologies, real-time data analysis, and conducting parallel clinical trial phases. By combining Sanofi's global scientific expertise with Abu Dhabi's state-of-the-art infrastructure, we set new benchmarks for resilience, innovation and international partnerships. Abu Dhabi offers a globally connected, purpose-driven environment for leading research and together we will drive a transformative change that will safeguard the health and well-being of our community while contributing to global health security." Both entities will work together to streamline regulatory processes, enhance manufacturing preparedness, and promote knowledge sharing between local and international experts. This partnership reflects Abu Dhabi's strategic goal to establish itself as a leading center for bio- and pharmaceutical innovation and underscores the emirate's commitment to advancing proactive, technology-driven healthcare solutions. Baptiste de Clarens, General Manager Greater Gulf, Vaccines, Sanofi, said : "The Memorandum of Understanding signed with the Department of Health – Abu Dhabi marks an encouraging step towards advancing global health security while reflecting our shared interest in addressing global health challenges through scientific partnership. This agreement allows us to examine how Sanofi's experience in vaccine development could complement Abu Dhabi's growing R&D ecosystem. Through this initial framework, we look forward to exploring opportunities for public-private collaboration in healthcare innovation."In addition, both the Department and Sanofi will advance clinical research and development planning, optimise resource allocation and establish the foundational terms for funding and access. These efforts are designed to enhance healthcare resilience and sustainability across Abu Dhabi's healthcare sector and beyond, expanding access to quality care for patients locally and globally.Led by DoH, a high-level delegation has embarked on a strategic mission to the United States from June 15 to 21, 2025.The delegation will conduct over 20 strategic meetings and visits with public and private sector leaders across the U.S., aimed at knowledge exchange, investment opportunities and the signing of new agreements that accelerate the adoption of advanced health solutions.Representing Abu Dhabi's innovation ecosystem, the delegation includes key stakeholders such as the Abu Dhabi Investment Office , Mubadala BIO , M42 , Masdar City , KEZAD , PureHealth , and Etihad Cargo , New York University Abu Dhabi (NYUAD), Khalifa University , startAD and Mohamed bin Zayed University of Artificial Intelligence (MBZUAI) .Photo - Logo - SOURCE The Department of Health - Abu Dhabi WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Advanced Dental Center Expands Full-Mouth Restoration and Gum Disease Treatment with Modern Tools Norwalk, CT / USA, June 16, 2025 (GLOBE NEWSWIRE) -- Norwalk, CT — Advanced Dental Center in Norwalk, Connecticut, led by Dr. Elad Yossefi and Dr. Tal Yossefi, is introducing new technology-driven services to improve patient care. Located on Main Avenue, the practice is focusing on procedures such as full-mouth restoration, laser-assisted periodontal therapy, and Invisalign, with an emphasis on clinical precision and patient experience. Drs. Elad and Tal Yossefi, honored as America’s Best Dentists 2023 at Advanced Dental Center in Norwalk, CT. Dr. Tal Yossefi, a graduate of the University of Pennsylvania, and Dr. Elad Yossefi, who earned his degree from NYU and the Lithuanian University of Health Sciences, bring international training to their practice. Their approach integrates digital diagnostics and design tools to customize treatment plans for each individual. Among the expanded services is full-mouth restoration, a procedure combining crowns, veneers, implants, and bite realignment. The center employs digital scans, facial mapping, and smile design software to support outcomes that are both functional and aesthetic. The clinic has also adopted LANAP® (Laser-Assisted New Attachment Procedure), an FDA-cleared technique used to treat advanced gum disease. This method utilizes laser technology to reduce discomfort and preserve healthy tissue, offering an alternative to traditional surgical procedures. To increase accessibility and reduce patient wait times, the center uses CEREC equipment, enabling same-day crown production without temporary fittings. For orthodontic treatment, the practice has achieved Gold Provider status with Invisalign®, using 3D planning and clear aligners to guide dental realignment. From pediatric care to complex oral rehabilitation, Advanced Dental Center is expanding its offerings with a focus on clinical transparency and patient-centered care. For more information, visit www.smilenorwalk.com or call (203) 364-5084. Media Contact: Eva Kavanagh Advanced Dental Center Norwalk adcnorwalk@gmail.com 1(203) 364-5084 Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.