Cincinnati Children's Hospital Medical Center

STOCKHOLM, Oct. 30, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) today announced that new research showing the effect of emapalumab in patients with macrophage activation syndrome (MAS) in Still's disease, including systemic juvenile idiopathic arthritis (sJIA) and adult-onset still's disease (AOSD) who had an inadequate response to high-dose glucocorticoids (GCs) will be presented at the American College of Rheumatology (ACR) Convergence in Washington D.C. The data were pooled from two open-label, single-arm interventional studies, the phase 3 NI-0501-14 study [EMERALD; NCT05001737] and NI-0501-06 [NCT03311854], including 39 patients with MAS. Key results: Complete response at week 8: At week 8, 21 patients (53.8%) achieved a complete response (95% confidence interval (CI): 37.2–69.9%). Complete response at any time: 33 patients (85%) achieved a complete response at any time during the studies. Glucocorticoid tapering: Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. By week 8, glucocorticoids were tapered to a clinically meaningful dose of ≤1 mg/kg/day in 28 patients (72%) and to ≤0.5 mg/kg/day in 17 patients (44%). Safety: No new safety concerns were identified during the studies. "MAS in Still's disease is a severe condition characterised by intense hyperinflammation and multiple organ failure. Those affected, including young children, often experience high fevers, liver and spleen enlargement, severe cytopenias, and neurological symptoms," said Lydia Abad-Franch, MD, Head of Research & Development and Medical Affairs and Chief Medical Officer at Sobi. "The results to be presented at ACR indicate that emapalumab could potentially provide a new therapeutic option, offering effective control of MAS symptoms and possibly reducing the need for high-dose glucocorticoids. This represents an important development in the treatment landscape for MAS." Additional key results: Overall response (OR) by week 8: 32 patients (82.4%) achieved an overall response by week 8. OR was observed as early as day 5, with a median time to first OR of 2.3 weeks. Clinical MAS remission: 32 patients (82.1%) achieved investigator-assessed clinical MAS remission at any time , with a median time to clinical remission of 3.3 weeks. Survival rate: 37 patients (94.9%) were alive at week 8. Emapalumab is a monoclonal antibody that neutralises interferon gamma (IFNγ), a key cytokine which contributes to the inflammation and tissue damage seen in MAS. The purpose of these two prospective studies is to assess the efficacy and safety of emapalumab in controlling MAS in patients with Still's disease, including sJIA and AOSD, who had an inadequate response to high-dose glucocorticoids. In May 2024, the US FDA granted emapalumab fast track designation as a potential therapeutic option in patient with MAS. Sobi plans to submit an sBLA for this indication to the FDA in 2024. The data will be presented by Alexei A. Grom, MD, Professor of Paediatrics, Research Director Division of Rheumatology, Cincinnati Children's Hospital Medical Centre, in a session entitled "Efficacy and Safety of emapalumab in children and adults with macrophage activation syndrome (MAS) in Still's disease: Results from a phase 3 study and a pooled analysis of two prospective trials" on Tuesday, 19 November at ACR Convergence. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH). About emapalumab Emapalumab (Gamifant®) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, emapalumab is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The health system ranked #1 for Cancer, Gastroenterology & GI Surgery, and Pulmonology & Lung Surgery. CINCINNATI, Oct. 8, 2024 /PRNewswire/ -- Cincinnati Children's has once again been named to U.S. News & World Report's Best Children's Hospitals Honor Roll for the 2024-25 rankings. The Honor Roll is comprised of the top 10 pediatric hospitals in the nation out of 108 considered. This year, Cincinnati Children's ranked No. 1 in three specialties and placed in the top 10 for all 10 ranked specialties, making it the only health system in the 13-state Midwest region—and one of just two nationwide—to achieve this distinction. Continue Reading U.S. News Specialties Badge In a significant change, Honor Roll hospitals are no longer ranked in a specific order, a shift that began with adult hospitals in 2023 and now applies to children's hospitals. Additionally, Pediatric & Adolescent Behavioral Health has been added to the rankings for the first time in more than 15 years. The initial list includes the top 50 hospitals, without a specific order, with Cincinnati Children's among those recognized. "To be named one of the best pediatric hospitals in the nation for more than 15 years is a distinct honor that each and every one of our team members should celebrate," says Steve Davis, MD, MMM, president and CEO of Cincinnati Children's. "Their relentless commitment to our patients and families has made Cincinnati Children's a national leader where the sickest kids around the world can come for the best possible outcomes. I'm so proud to walk the halls of our health system and see researchers, caregivers and support staff collaborating with a 'how might we?' mindset to providing the highest quality care and researching the latest breakthroughs in medicine. These rankings are a reminder for us to continue pushing the boundaries of what is possible so that all kids can live their healthiest lives." Hospitals were ranked based on key clinical data that includes measures such as patient outcomes, resources devoted directly to patient care, and patient safety. The goal of these rankings is to identify hospitals that provide the highest quality care for children with the most serious or complex medical issues. Cincinnati Children's Specialty Rankings: *Reflects the Joint Pediatric and Congenital Heart Program ranking. The Joint Pediatric and Congenital Heart Program is a collaboration between Cincinnati Children's and Kentucky Children's Hospital, part of UK HealthCare in Lexington, KY. The full report can be found here. B-roll of Cincinnati Children's can be downloaded here. About Cincinnati Children's Cincinnati Children's is ranked among the nation's best pediatric health systems by U.S. News & World Report, including No. 1 in pediatric cancer, gastroenterology and pulmonology, as well as being recognized on its list of top hospitals for pediatric and adolescent behavioral health. Nearly one-third of the organization's 19,500 employees are engaged in research, and Cincinnati Children's is recognized as one of America's Most Innovative Companies by Fortune. Established in 1883, Cincinnati Children's is a nonprofit that cares for patients from all 50 states and dozens of countries, including kids with rare or complex disorders. More information: CincinnatiChildrens.org SOURCE Cincinnati Children's Hospital Medical Center WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CINCINNATI, Sept. 20, 2024 /PRNewswire/ -- As America grapples with a crisis in child mental health, Cincinnati Children's is pleased to announce that a top authority in pediatric healthcare and policy will become the first leader to direct the combined activities of the nation's largest provider of pediatric inpatient mental and behavioral health services. Continue Reading Laurel K. Leslie, MD, MPH Laurel K. Leslie, MD, MPH, currently vice president of research at the American Board of Pediatrics (ABP), will become the inaugural director of the new Cincinnati Children's Mental and Behavioral Health Institute (MBHI), effective Feb. 24, 2025. "Dr. Leslie's extensive pediatric leadership and proven experience in pediatric mental, behavioral and developmental health makes her the ideal choice to direct our Mental and Behavioral Health Institute," says Tina Cheng, MD, MPH, director of the Cincinnati Children's Research Foundation, chair of the Department of Pediatrics, and chief medical officer of the health system. "Her expertise and passion for improving pediatric mental healthcare will help us advance our aspirations as the leader in improving child health." What is the Mental and Behavioral Health Institute? Cincinnati Children's has been at the forefront of pediatric mental, behavioral and developmental healthcare and research for more than two decades. The new institute was announced in October 2023 at Cincinnati Children's annual Kaleidoscope fundraising event, which benefited the health system's mental and behavioral health services. The new institute integrates the divisions of Child and Adolescent Psychiatry, Behavioral Medicine and Clinical Psychology, and Developmental and Behavioral Pediatrics. This innovative structure allows for deeper collaboration among experts across specialty fields and among a network of community partners that will serve as a national model system for improving outcomes for children affected by mental, developmental, and behavioral challenges. The institute was launched with a generous $15 million lead gift from The James J. and Joan A. Gardner Family Foundation and has inspired additional giving to further expand access, research, community partnerships, and school services. Cincinnati Children's has three other major institutes that combine multiple divisions and reflect world-class leadership in care and research: the Perinatal Institute, the Heart Institute and the Cancer and Blood Diseases Institute. Who is Dr. Leslie? Leslie has held professorships at Tufts University School of Medicine since 2006, where she has focused on clinical, research and policy work to better integrate mental and behavioral care with clinical care for children in settings ranging from outpatient clinics and schools to child welfare and protective services. In addition to joining the American Board of Pediatrics in a leadership role in 2015, Leslie has co-chaired two National Academy of Medicine meetings focusing on healthcare workforce training and developing integrated care models for children's behavioral health. Leslie is board certified in general pediatrics and developmental-behavioral pediatrics. She holds degrees from Harvard University, UNC Chapel Hill and San Diego State University and completed residency and fellowship trainings at the University of California, San Francisco. "The mental health crisis among children, adolescents and young adults is the most critical pediatric public health emergency of our time," Leslie says. "I am honored to have the opportunity to work at Cincinnati Children's where the leadership, faculty and staff are committed to addressing this crisis in every setting from schools and primary care offices through inpatient care." ABP President and CEO Michael Barone, MD, MPH, congratulated Leslie as she takes on a new role. "While her departure will certainly be felt across our organization, this is a wonderful next step for Laurel as she follows her passion for improving the mental and behavioral health outcomes for children," Barone says. "Cincinnati Children's will greatly benefit from her leadership and vision in this important area. We wish her well in this very exciting next chapter." Moving forward With generous support from the Convalescent Hospital Fund for Children and other donors, Cincinnati Children's recently invested $108 million to renovate and expand its mental health care facilities at its College Hill campus. Experts here also have overhauled how children with mental and behavioral needs move through the health system, from emergency care to outpatient services. Under Leslie's leadership, the institute will prioritize: Reducing mental health-related emergency room visits and hospitalizations by strengthening outpatient care and early interventions. Improving mental health services in community settings such as schools and primary care clinics, embedding mental health providers in these environments to increase accessibility. Collaborating with families, educators and policymakers to ensure children's mental, behavioral and developmental health services are integrated with other healthcare needs. Investing in research and innovation to build early detection systems, digital health interventions, and collaborative models of care that empower families to take an active role in their children's mental health journey. "The creation and growth of the Mental and Behavioral Health Institute would not be possible without the support of families, healthcare professionals, community organizations, local schools and philanthropic partners, as we all work together to tackle the mental health crisis affecting youth here in our region and as a model for others," Cheng says. "Dr. Leslie and our entire MBHI team will continue to work tirelessly to ensure that every child receives the support and care they need to live their best possible life." About Cincinnati Children's Cincinnati Children's is a nonprofit, comprehensive pediatric health system that is internationally recognized for improving child health and transforming delivery of care. As a leader in research, education, patient care, advocacy and innovation, Cincinnati Children's is ranked #1 in U.S. News & World Report's 2023-2024 list of best children's hospitals in the nation and is the #2 recipient of pediatric research grant funding from the National Institutes of Health. SOURCE Cincinnati Children's Hospital Medical Center WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED