Sobi, Inc.

STOCKHOLM, March 19, 2025 /PRNewswire/ -- Sobi ® (STO: SOBI), today announced a research collaboration involving a new Phase 2a clinical trial for Gamifant® (emapalumab) for the potential treatment of interferon-gamma (IFNγ)-driven sepsis (IDS) which is to be presented at the International Symposium on Intensive Care and Emergency Medicine (ISICEM) Congress by Prof. Giamarellos-Bourboulis, from the Hellenic Institute for the Study of Sepsis. The study underscores Sobi's commitment to advancing medicine through targeted therapies for severe and complex conditions such as sepsis, a leading cause of mortality globally. Key Highlights IDS is a new endotype representing approximately 20 percent of sepsis patients and is characterised by detection of IFNγ (above lower limit of detection) and elevated levels of the chemokine CXCL9. The EMBRACE phase 2a study will investigate the potential of Gamifant® (emapalumab) in treating a subgroup of sepsis patients driven by the IDS endotype and absence of sepsis-induced immunoparalysis. IDS patients with a low human leukocyte antigen DR (HLA-DR) expression on monocytes characteristic of immunoparalysis will not be included into the EMBRACE study. The potential inclusion of this IDS sub-population into further studies will be evaluated based on the results of EMBRACE. The EMBRACE phase 2a trial (NCT06694701) was approved in March 2025. The first trial sites have been initiated, and patient screening to identify eligible patients with IDS has started. The trial design is to be presented at ISICEM by Prof. Evangelos Giamarellos-Bourboulis, MD, PhD, FISAC, principal investigator and sponsor of the EMBRACE study, from the Hellenic Institute for the Study of Sepsis (HISS): "The EMBRACE Phase 2a study will adopt a precision immunotherapy approach for the treatment of sepsis driven by the IDS endotype, a patient group currently having limited therapeutic choices." "We are looking forward to this research collaboration on the use of Gamifant® (emapalumab) in the new endotype of interferon gamma-driven sepsis," said Lydia Abad-Franch, MD, Head of Research & Development and Medical Affairs and Chief Medical Officer at Sobi. "We are committed to advancing science and study Gamifant® (emapalumab) in indications with a high unmet need and where excessive interferon gamma production plays a key role in driving hyperinflammation." Presentation details Emapalumab treatment for anticipated clinical benefit in sepsis driven by the interferon-gamma endotype (the EMBRACE trial): O Konstantinidou, E Olntasi, H Florou, A Reininger, E Giamarellos-Bourboulis, F EMBRACE STUDY GROUP. ISICEM 2025; Abstract number A173; Poster number P249. About sepsis and IFNγ-driven sepsis (IDS) Sepsis is a serious condition in response to an infection that can lead to organ failure and is a leading global cause of mortality. A recent large study, published in eBioMedicine in 20241, describes different sepsis endotypes, suggesting varying endotypes require differentiated treatment strategies. Approximately 20% of the patients studied are of the newly described IFNγ-driven sepsis (IDS) endotype. IDS is marked by elevated levels of CXCL9 and detection of IFNγ and poor clinical outcomes, with a 28-day mortality rate ranging from 40 to 43%. About the EMBRACE study The EMBRACE study (NCT06694701) is a Phase 2a, double-blind, randomized controlled trial planned to be conducted at 24 sites in Greece. The trial investigates whether Gamifant® (emapalumab), an anti-IFNγ antibody, can improve clinical outcomes in patients with the interferon-gamma-driven sepsis (IDS) endotype and absence of sepsis-induced immunoparalysis. IDS is characterized by elevated levels of CXCL9 and detectable IFNγ and is associated with poor patient outcomes. By targeting this inflammation pathway, the EMBRACE trial aims to reduce mortality, improve organ function, and accelerate recovery. The trial design includes three arms, and plans to enrol 75 patients, two groups receiving Gamifant® (emapalumab), (low and high doses) alongside standard-of-care treatment, and one group receiving placebo alongside standard-of-care treatment. The primary endpoint is a ≥1.4-point decrease in the Sequential Organ Failure Assessment (SOFA) score from baseline to the end of treatment (28 days). Secondary endpoints include 28-day mortality, safety, pharmacokinetics, and changes in key inflammatory biomarkers such as CRP, IL-6, ferritin, IFNγ, and CXCL9. About Gamifant® (emapalumab) Gamifant® (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant® (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® (emapalumab) 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 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. 1: Interferon-gamma driven elevation of CXCL9: a new sepsis endotype independently associated with mortality Giamarellos-Bourboulis, Evangelos J. et al. eBioMedicine, Volume 109, 105414 Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. 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STOCKHOLM, Feb. 27, 2025 /PRNewswire/ -- Sobi® (STO: SOBI), today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Gamifant® (emapalumab-Izsg) for use in adult and paediatric patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. The application was granted Priority Review with a PDUFA date of June 27, 2025. HLH/MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still's disease including systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). HLH/MAS is a rare systemic disorder of hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly. "HLH/MAS in Still's disease is a serious and potentially fatal complication where patients can experience intense hyperinflammation and even multiple organ failure," said Lydia Abad-Franch, MD, MBA, Head of R&D and Chief Medical Officer at Sobi. "There is no approved therapy for HLH/MAS today. Gamifant® (emapalumab-Izsg) selectively neutralizes interferon gamma (IFN-γ), a key driver of hyperinflammation, and if approved, may also help reduce the need for high-dose glucocorticoids in these patients." The application is based on results from pooled data from two studies which enrolled a total of 39 patients, the EMERALD (NCT05001737) and the NI-0501-06 (NCT03311854) studies. Fifty-three percent of patients had a complete response at Week 8 and 85% had a complete response at any time during the studies. Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. Gamifant® (emapalumab-Izsg) a monoclonal antibody that binds and neutralises interferon gamma (IFN-y), was approved by the FDA in 2018 for the treatment of adult and paediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. About macrophage activation syndrome (MAS) Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinemia, possibly progressing to multiple organ failure and death. MAS is classified as a form of hemophagocytic lymphohistiocytosis (HLH). About Gamifant® (emapalumab-lzsg) Gamifant® (emapalumab-lzsg) is an anti-interferon gamma (IFN-γ) monoclonal antibody that binds to and neutralises IFN-γ. In the USA, Gamifant® (emapalumab-lzsg) is the first and only treatment approved by the FDA for the treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic 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® (emapalumab-lzsg) is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic 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 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. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Sobi® (STO: SOBI), today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Gamifant® (emapalumab-Izsg) for use in adult and paediatric patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still’s disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.The application was granted Priority Review with a PDUFA date of June 27, 2025. HLH/MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still’s disease (AOSD). HLH/MAS is a rare systemic disorder of hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly. “HLH/MAS in Still’s disease is a serious and potentially fatal complication where patients can experience intense hyperinflammation and even multiple organ failure,” said Lydia Abad-Franch, MD, MBA, Head of R&D and Chief Medical Officer at Sobi. “There is no approved therapy for HLH/MAS today. Gamifant® (emapalumab-Izsg) selectively neutralizes interferon gamma (IFN-γ), a key driver of hyperinflammation, and if approved, may also help reduce the need for high-dose glucocorticoids in these patients.” The application is based on results from pooled data from two studies which enrolled a total of 39 patients, the EMERALD (NCT05001737) and the NI-0501-06 (NCT03311854) studies. Fifty-three percent of patients had a complete response at Week 8 and 85% had a complete response at any time during the studies. Weekly mean glucocorticoid doses were reduced by 70.1% after 2 weeks of treatment. Gamifant® (emapalumab-Izsg) a monoclonal antibody that binds and neutralises interferon gamma (IFN-y), was approved by the FDA in 2018 for the treatment of adult and paediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. About macrophage activation syndrome (MAS)Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still’s disease (AOSD). MAS is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinemia, possibly progressing to multiple organ failure and death. MAS is classified as a form of hemophagocytic lymphohistiocytosis (HLH). About Gamifant® (emapalumab-lzsg)Gamifant® (emapalumab-lzsg) is an anti-interferon gamma (IFN-γ) monoclonal antibody that binds to and neutralises IFN-γ. In the USA, Gamifant® (emapalumab-lzsg) is the first and only treatment approved by the FDA for the treatment of adult and paediatric (newborn and older) patients with primary hemophagocytic 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® (emapalumab-lzsg) is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic 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 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. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. We provide access to innovative treatments that transform life for people. 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. Every day, we work actively to find better ways to understand and meet patient needs. Find out more about our business and financial performance. Here we present current and past media releases, images and videos.