Emapalumab-LZSG

Sobi® (STO: SOBI), a global biopharmaceutical company dedicated to delivering innovative treatments for patients with rare disease, will be presenting new data across its immunology portfolio at the annual American College of Rheumatology (ACR) Convergence 2025 meeting in Chicago, October 24-29. Sobi is presenting a total of 15 scientific abstracts, with six oral presentations featuring new clinical data from completed and ongoing studies of NASP, pacritinib, and emapalumab-lzsg. "The data Sobi is presenting highlight the distinct clinical benefit of emapalumab-lzsg in MAS in Still’s disease, the ongoing study of pacritinib in VEXAS Syndrome, and the potential for NASP to redefine the treatment of uncontrolled gout. The results underscore the strength of our portfolio in advancing care for complex and rare inflammatory diseases where progress is urgently needed,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Summary of full Sobi data to be presented at ACR 2025: NASP Nanoencapsulated Sirolimus plus Pegadricase Reduced Disease Burden in Patients with Uncontrolled Gout: Results from the Phase 3 DISSOLVE Trials Presenting Author: Puja P. Khanna, MD, MPH, Division of Rheumatology, University of Michigan, Ann Arbor, MI Oral Presentation Session: Abstracts: Metabolic & Crystal Arthropathies – Basic & Clinical Science Date: Tuesday 28 October Session time: 1:00 PM - 2:30 PM CT Presentation time: 1:30 PM - 1:45 PM CT Nanoencapsulated Sirolimus plus Pegadricase Demonstrates a Reduction in Gout Flares: Results from the Phase 3 DISSOLVE Studies*) Presenting Author: Angelo Gaffo, MD, Division of Rheumatology and Clinical Immunology, University of Alabama, Birmingham, AL Oral Presentation Session: Abstracts: Metabolic & Crystal Arthropathies – Basic & Clinical Science Date: Tuesday 28 October Session time: 1:00 PM - 2:30 PM CT Presentation time: 1:45 PM - 2:00 PM CT Nanoencapsulated Sirolimus plus Pegadricase Demonstrates Long Term Efficacy and Safety in Patients with Uncontrolled Gout: Results from the 24-week Double-blind Extension of the Phase 3 DISSOLVE I Study Presenting Author: Alan Kivitz, MD, Altoona Center for Clinical Research, Duncansville, PA Poster Presentation Session: Metabolic & Crystal Arthropathies – Basic & Clinical Science Poster I Date: Monday 27 October Presentation time: 10:30 AM - 12:30 PM CT Characterisation of Infusion Reactions Within 1 Hour of Treatment with Nanoencapsulated Sirolimus Plus Pegadricase: Pooled Results from the Phase 3 DISSOLVE I and DISSOLVE II Trials Presenting Author: Herbert S.B. Baraf, MD, FACP, MACR, The Center for Rheumatology and Bone Research, Wheaton, MD Poster Presentation Session: Metabolic & Crystal Arthropathies – Basic & Clinical Science Poster II Date: Tuesday 28 October Presentation time: 10:30 AM - 12:30 PM CT Reduction in Tophi Observed in Patients with Uncontrolled Gout Treated with NASP: Results from Phase 3 DISSOLVE Studies* Presenting Author: Herbert S.B. Baraf, MD, FACP, MACR, The Center for Rheumatology and Bone Research, Wheaton, MD Poster Presentation Session: Poster Session C, (1990–2014) Metabolic & Crystal Arthropathies – Basic & Clinical Science Poster II Date: Tuesday 28 October Presentation time: 10:30 AM - 12:30 PM CT Patient and Caregiver Perspectives on the Burden of Disease in Uncontrolled Gout: A Cross-Sectional Survey Study Presenting Author: Angelo Gaffo, MD, Division of Rheumatology and Clinical Immunology, University of Alabama, Birmingham, AL Poster Presentation Session: Patient Outcomes, Preferences, & Attitudes Poster II Date: Monday 27 October Presentation time: 10:30 AM - 12:30 PM CT A Real-World Survey on Physicians' Perspectives of Uncontrolled Gout and Gout Management Practices Presenting Author: John Botson, MD, RPh,CCD, Orthopedic Physicians, Anchorage, AK Poster Presentation Session: Health Services Research Poster III Date: Tuesday 28 October Presentation time: 10:30 AM - 12:30 PM CT The Care Pathway and Treatment Patterns in Patients with Uncontrolled Gout: A Real-World Survey of Physicians in the United States Presenting Author: John Albert, MD, Rheumatic Disease Center, Glendale, WI Poster Presentation Session: Health Services Research Poster II Date: Monday 27 October Presentation time: 10:30 AM - 12:30 PM CT Management of Uncontrolled Gout Among Rheumatologists: Findings from a Medical Chart Audit Presenting Author: Hyon Choi, MD, MPH, DrPH, Massachusetts General Hospital, Boston, MA Poster Presentation Session: ARP Posters I Date: Tuesday 28 October Presentation time: 10:30 AM - 12:30 PM CT Gamifant (emapalumab-lzsg) Baseline Pharmacodynamic Markers and Response to emapalumab in Children and Adults with Macrophage Activation Syndrome (MAS) in Still’s Disease: Results from a Pooled Analysis of Two Prospective Trials Presenting Author: Ed Behrens, MD, Chief, Division of Rheumatology, Children’s Hospital of Philadelphia Oral Presentation Session: Abstracts: Miscellaneous Rheumatic & Inflammatory Diseases I: Focus on Auto-Inflammatory Diseases Date: Sunday 26 October Session time: 1:00 PM - 2:30 PM CT Presentation time: 1:45 PM - 2:00 PM CT Emapalumab Treatment for Patients with Differing Presentations of Macrophage Activation Syndrome (MAS) Secondary to Still’s Disease: Results from a Pooled Analysis of Two Prospective Trials Presenting Author: Alexei Grom, MD, Research Director, Division of Rheumatology, Cincinnati Children’s Hospital Oral Presentation Session: Abstracts: Pediatric Rheumatology – Clinical I Date: Monday 27 October Session time: 1:00 PM - 2:30 PM CT Presentation time: 1:45 PM - 2:00 PM CT Economic Burden of Macrophage Activation Syndrome (MAS) in Patients with Still’s Disease (Systemic Juvenile Idiopathic Arthritis (sJIA) and Adult-onset Still’s Disease (AOSD): Analysis of a US National Administrative Claims Database Presenting Author: Michael Marrone, Director of Epidemiology and Biostatistics, Sobi Poster Presentation Session: Health Services Research Poster III Date: Tuesday 28 October Presentation Time: 10:30 AM - 12:30 PM CT Vonjo (Pacritinib) Development of a Disease Activity Index for the Assessment of VEXAS Syndrome (VEXAS-DAI)* Presenting Author: Dr. Matthew J. Koster, Assistant Professor of Medicine, Mayo Clinic Rochester Oral Presentation Session: Abstracts: Miscellaneous Rheumatic & Inflammatory Diseases I: Focus on Auto-Inflammatory Diseases Date: Sunday 26 October Session time: 1:00 PM - 2:30 PM CT Presentation time: 1:00 PM - 1:15 PM CT PAXIS: A Randomised, Double-Blind, Placebo-Controlled, Dose Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome* Presenting Author:Dr. David B. Beck, MD, PhD, Division of Rheumatology, NYU Langone Oral Presentation Session: Abstracts: Miscellaneous Rheumatic & Inflammatory Diseases III: End Organ Focus on Heart, Lung and Eye Date: Wednesday 29 October Session time: 11:30 AM - 1:00 PM CT Presentation time: 11:30 AM - 11:45 AM CT Development of a Consensus Definition of VEXAS Flare for Use in Clinical Research* Presenting Author: Dr. Marcela A. Ferrada, MD, Clinical Associate Professor, University of Maryland School of Medicine Poster Presentation Session: Miscellaneous Rheumatic & Inflammatory Diseases Poster I Date: Sunday 26 October Presentation time: 10:30 AM - 12:30 PM CT *Signifies encore data presentation About NASPNASP is a novel investigational medicine designed to reduce serum uric acid (sUA) levels in people living with uncontrolled gout, potentially reducing harmful tissue urate deposits which when left untreated can lead to debilitating gout flares and joint deformity. NASP is administered every 4-weeks as a sequential, two-component, infusion therapy consisting of tolerogenic nanoencapsulated sirolimus (NAS) which mitigates the formation of anti-drug antibodies (ADAs) and a uricase, pegadricase (P), which reduces serum uric acid. ADAs develop due to unwanted immune responses to biologic medicines, reducing their efficacy and tolerability, which remains an issue across multiple therapeutic modalities and disease states including uncontrolled gout. About Uncontrolled GoutGout is the most common form of inflammatory arthritis with more than 8.3 million people in the United States having been diagnosed. Gout is caused by high levels of uric acid in the body that accumulate around the joints and other tissues and can result in flares that cause intense pain. Approximately 200,000 people in the United States suffer from uncontrolled gout, with serum uric acid (sUA) levels above 6 mg/dL despite treatment with oral urate lowering therapies. This leads to debilitating flares and tophi. Elevated sUA levels have also been associated with diseases of the heart, vascular system, metabolism, kidney and joints. About Gamifant (emapalumab-lzsg) Gamifant is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralizing interferon gamma (IFNy). When interferon gamma (IFNy) is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour. Gamifant is approved in the US for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Gamifant is also approved in the US for the treatment of adult and pediatric (newborn and older) patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still’s disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently in Still’s disease including 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 characterized 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 Vonjo (pacritinib)Vonjo is a kinase inhibitor that is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). About VEXAS VEXAS syndrome is a disease that causes inflammatory and hematologic (blood) manifestations. The syndrome is caused by mutations in the UBA1 gene of blood cells and acquired later in life. The condition is not genetically inherited. About Sobi Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 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. We are a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Find out more about our business and financial performance. Here we present our most recent press releases, news articles, and images. Every day, we work actively to find better ways to understand and meet patient needs.

Sobi obtained FDA approval for Gamifant’s supplemental biologics licence application in February 2024. Credit: Roland Magnusson/Shutterstock. The US Food and Drug Administration (FDA) has approved Sobi’s Gamifant (emapalumab) to treat adults and children with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) associated with Still’s disease, including juvenile idiopathic arthritis (sJIA), where there is a reduced response or intolerance to glucocorticoids, or cases of recurrent MAS. Gamifant functions as an anti-interferon gamma (IFNγ) monoclonal antibody. It is offered in three strengths:10mg/2 ml, 50mg/10ml and 100mg/20ml vials, administered intravenously. Sobi CEO Guido Oelkers stated: “With our expertise in primary hemophagocytic lymphohistiocytosis, we understand the urgency of managing MAS quickly to improve patient outcomes. “Gamifant is already an established therapy making a meaningful difference for patients with primary HLH, and with this approval, we are excited about the opportunity to positively impact patients affected by MAS in Still’s disease.” The FDA approval was based on pooled data from two pivotal studies: the Phase III EMERALD trial and NI-0501-06. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence A complete response (CR) at week eight was observed in 54% of patients, while 82% achieved clinical MAS remission at the same interval. Safety and tolerability profiles were consistent with those seen in earlier clinical trials. HLH/MAS can be a severe complication within rheumatic diseases such as systemic sJIA and adult-onset Still’s disease. This rare disorder is characterised by hyperinflammation driven by IFNy, leading to symptoms like persistent high fever, elevated ferritin levels, coagulopathies, cytopenias and hepatosplenomegaly. In February 2025, the company obtained FDA approval for Gamifant’s supplemental biologics licence application . Gamifant is also authorised in the US for patients with primary HLH with refractory, persistent or progressive disease or intolerant to standard therapy. Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now