RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), PRIME (European Union), Conditional marketing approval (China), Priority Review (China)

Structure/Sequence

Sequence Code 25650568H

Source: *****

Sequence Code 25650570L

Source: *****

Clinical Trials associated with Emapalumab-LZSG

NCT06996119

/ Not yet recruitingPhase 1IIT

Pilot Study of Emapalumab With Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis for Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation

This phase I trial tests the safety, side effects and effectiveness of emapalumab with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil in preventing graft-versus-host disease (GVHD) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after reduced-intensity donor (allogeneic) hematopoietic cell transplant (HCT). Giving chemotherapy, such as fludarabine, melphalan, or busulfan, before a donor [peripheral blood stem cell] transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When healthy stem cells for a donor are infused into a patient (allogeneic HCT), they may help the patient's bone marrow make more healthy cells and platelets. Allogeneic HCT is an established treatment, however, GVHD continues to be a major problem of allogeneic HCT that can complicate therapy. GVHD is a disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Emapalumab binds to an immune system protein called interferon gamma. This may help lower the body's immune response and reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus is a drug used to help reduce the risk of rejection by the body of organ and bone marrow transplants. Mycophenolate mofetil is a drug used to prevent GVHD after organ transplants. It is also being studied in the prevention of GVHD after stem cell transplants for cancer, and in the treatment of some autoimmune disorders. Mycophenolate mofetil is a type of immunosuppressive agent. Giving emapalumab with post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil may be safe, tolerable and/or effective in preventing GVHD in patients with AML or MDS after a reduced-intensity allogeneic HCT.

Start Date25 May 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT06694701

/ RecruitingPhase 2IIT

Emapalumab Treatment For Anticipated Clinical Benefit In Sepsis Driven By The Interferon-Gamma Endotype (The EMBRACE Trial)

EMBRACE is a double-blind, randomized, placebo-controlled, phase IIa study that will be conducted in multiple Intensive Care Units (ICUs) and departments of Internal Medicine across Greece. It aims to investigate if treatment with emapalumab, a monoclonal antibody which blocks IFNγ, may improve the outcome of patients with sepsis driven by the IDS (endotype of IFNγ-driven sepsis) endotype. EMBRACE also aims to identify the best dosing regimen of emapalumab for the management of IDS.

Start Date22 Mar 2025

Sponsor / Collaborator

Hellenic Institute for the Study of Sepsis

NCT06550141

/ RecruitingPhase 2IIT

A Phase 2 Trial of Emapalumab for the Prevention of CAR-T Cell Associated Toxicities

This research study involves assessing the impact of emapalumab as preventative management of CAR-T related cytokine release syndrome in participants with Non-Hodgkin's lymphoma (NHL).
The research study involves the following study interventions:
* Fludarabine and cyclophosphamide (Lymphodepleting Chemotherapy)
* Axicabtagene Ciloleucel
* Emapalumab

Start Date18 Sep 2024

Sponsor / Collaborator

The Massachusetts General Hospital

[+1]

100 Clinical Results associated with Emapalumab-LZSG

100 Translational Medicine associated with Emapalumab-LZSG

100 Patents (Medical) associated with Emapalumab-LZSG

Literatures (Medical) associated with Emapalumab-LZSG

01 May 2025·ANNALS OF THE RHEUMATIC DISEASES

Refractory MDA-5 dermatomyositis rapidly progressive interstitial lung disease successfully treated with emapalumab

Letter

Author: Concepcion, Jennifer ; Corbitt, Kelly ; Vehar, Susan ; Marti, Miriam

01 Apr 2025·CLINICAL RHEUMATOLOGY

Salvage treatment of AOSD-associated macrophage activation syndrome with bariticinib following conventional immunosuppressive therapy

Article

Author: Xiang, TianDan ; Xu, Ying ; Sun, Wei ; Huang, Xiaoping ; Huang, Yan ; Zhou, Jin ; Yin, Yufeng ; Li, Wenting ; Xu, Chuancai ; Wang, Yan

OBJECTIVES:

Adult-onset Still's disease (AOSD)-associated macrophage activation syndrome (MAS) is a highly inflammatory condition. Currently, the main treatment method for AOSD-associated MAS is high-dose glucocorticoid shock therapy, but the effect is not satisfactory, and new therapeutic methods or drugs are urgently needed. The aim of this study was to evaluate the efficacy and safety of salvage baricitinib therapy for AOSD-associated MAS following conventional immunosuppressive therapy.

METHOD:

Data from nine patients with MAS treated in the Department of Infection and the Department of Rheumatology, First Affiliated Hospital of Soochow University, from January 2020 to December 2022, were retrospective analysed. Salvage therapy with baricitinib was administered, with an initial dose of 8 mg/day. The patients' clinical symptoms and MAS indices (blood counts and triglyceride, fibrinogen, lactate dehydrogenase, sCD25, and Fer levels) were observed. After a complete response was achieved, the baricitinib dose was gradually reduced to 2-4 mg/day and maintained.

RESULTS:

The MAS blood test results of all nine patients improved to varying degrees within 1 week post-treatment. The MAS indices gradually returned to normal approximately 8 weeks after discharge; at the 12-month outpatient follow-up, seven patients did not develop MAS again, but two patients (patients 5 and 9) had MAS recurrence. The follow-up period ended with salvage treatment with emapalumab.

CONCLUSIONS:

Baricitinib can effectively inhibit the inflammatory response and improve outcomes in the treatment of MAS secondary to rheumatic immunity-related diseases; moreover, this drug treatment is safe. Our study provides a new strategy for MAS salvage therapy. Key Points • Bariticinib may be a palliative treatment option for recurrent/refractory MAS.

01 Apr 2025·RHEUMATOLOGY

Acute respiratory distress syndrome after emapalumab treatment in a child with severe Still’s disease and lung involvement: a case report

Article

Author: Despert, Véronique ; Koné-Paut, Isabelle ; Rossi-Semerano, Linda ; Anselmi, Federica ; Dusser, Perrine ; Terzic, Arnaud

News (Medical) associated with Emapalumab-LZSG

27 Jun 2025

·www.prnewswire.com

FDA approves Gamifant® (emapalumab-lzsg) as first-ever treatment for adults and children with Macrophage Activation Syndrome in Still's disease

Approval based on the pooled analysis of our pivotal EMERALD and NI-0501-06 studies, showing 54% of patients treated with Gamifant achieved complete response at week 8. STOCKHOLM, June 28, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) today announced that the U.S. Food and Drug Administration (FDA) approved Gamifant ® (emapalumab-lzsg) 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, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. "With our expertise in primary hemophagocytic lymphohistiocytosis, we understand the urgency of managing MAS quickly to improve patient outcomes," said Guido Oelkers, Chief Executive Officer at Sobi. "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 approval is based on results of the pooled data from two pivotal studies, the Phase 3 study (NCT05001737) and NI-0501-06 (NCT03311854). 54 percent (21/39) of patients had a complete response at Week 8, and 82% (32/39) achieved clinical MAS remission (VAS ≤1 cm) at Week 8. Safety and tolerability were consistent with previous clinical studies. In patients with HLH/MAS in Still's disease, the most common adverse events (≥20%) were viral infections, including cytomegalovirus infection or reactivation, and rash. "MAS in Still's disease is a serious and potentially life-threatening complication, marked by severe hyperinflammation and, in some cases, multi-organ failure," said Alexei A. Grom, MD, Professor of Pediatrics, Research Director Division of Rheumatology, Cincinnati Children's Hospital Medical Center. "Many patients affected by MAS—both young children and adults—face significant unmet medical needs. With Gamifant now as the first FDA-approved treatment for MAS, we have a new therapeutic option that helps control hyperinflammation and reduce our reliance on high-dose glucocorticoids." MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still's disease including systemic juvenile idiopathic arthritis and adult-onset Still's disease. HLH/MAS is a rare systemic disorder of interferon gamma (IFNy) driven hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly. Gamifant, an interferon gamma (IFNγ)–blocking antibody, is the first and only FDA approved treatment for adult and pediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. U.S. Indication for Gamifant® (emapalumab-lzsg) Please see the full Prescribing Information for Gamifant . 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 Gamifant® (emapalumab-lzsg) Gamifant® (emapalumab-lzsg) is the only approved anti-interferon gamma (IFNγ) monoclonal antibody. Gamifant works by binding to and neutralizing IFNγ. When IFNγ 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 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. This information is information that Sobi is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, on 28 June 2025 at 00:55 CEST. Gerard Tobin Head of Investor Relations 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

Drug ApprovalPhase 3Clinical Result

12 Jun 2025

·www.sobi.com

Sobi share new clinical data across multiple hematologic diseases at EHA 2025

Sobi® (STO: SOBI) will present data at the 30th EHA (European Haematology Association) hybrid congress, in Milan, Italy (12-15 June). The congress will feature the latest advances in the treatment of diffuse large B-cell lymphoma (DLBCL), immune thrombocytopenia (ITP), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and primary hemophagocytic lymphohistiocytosis (pHLH). An extensive programme of poster presentations will showcase Sobi’s commitment to helping patients with rare diseases by advancing treatment options. In addition, Sobi will host several scientific symposiums at the congress including: 1.Advancing Therapeutic Knowledge in Paroxysmal Nocturnal Haemoglobinuria: Reshaping disease management to unlock new norms, Thursday, 12 June, 10:00am - 11:30am CEST, at Amber Hall 3 & 4. 2. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. 3. Dissecting Treatment Sequencing in relapsed/refractory DLBCL, from laboratory to real life. Saturday, 14 June, 8:00 am – 9:30 am, CEST at Coral Hall 1. “The breadth of data that we share at this year’s EHA congress demonstrates Sobi’s comprehensive approach to addressing rare conditions in haematology. We are proud to contribute to advancing the science in several indications from early clinical phases in diffuse large B-cell lymphoma to clinical and real-world evidence in myelofibrosis, primary hemophagocytic lymphohistiocytosis, immune thrombocytopenia and paroxysmal nocturnal haemoglobinuria,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Key data to be presented at EHA 2025 DLBCL PS1911: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large BCell Lymphoma (DLBCL) Presenting Author: Juan Pablo Alderuccio Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1957: Updated Safety Run-in Results from LOTIS-5: A Phase 3, Randomized Trial of Loncastuximab Tesirine with Rituximab Versus Immunochemotherapy in Patients With R/R DLBCL Presenting Author: Carmelo Carlo-Stella Immune Thrombocytopenia (ITP) PF1236: Platelet Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia Who Switched from Eltrombopag or Romiplostim: the REAL-AVA 2.0 Real-World Study Presenting Author: Shruti Chaturvedi Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1239: Durability of Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia: The REAL-AVA 2.0 Real-World Study Presenting Author: Srikanth Nagalla PF1251: Clinically Meaningful Response to Avatrombopag: A Phase 3B Trial for Treatment of Children with ITP Presenting Author: Rachael F. Grace PS2231: Effectiveness and safety of avatrombopag for the treatment of adults with newly diagnosed, persistent, or chronic immune thrombocytopenia: Interim results from the phase 4 ADOPT study Presenting Author: Waleed Ghanima Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS2234: Efficacy and safety of avatrombopag for the treatment of pediatric immune thrombocytopenia in the open-label extension of a phase 3, randomized, double-blind, placebo-controlled trial Presenting Author: Rachael F. Grace PS2239: Real-World Treatment Patterns & Clinical Outcomes in Thrombopoietin Receptor Agonist Naive Patients with Immune Thrombocytopenia Treated with Avatrombopag: Interim Results from the REAL-AVA 3.0 Study Presenting Author: Sandhya Panch PS2242: Effectiveness and safety of avatrombopag for treatment of immune thrombocytopenia in older patients and those with comorbidities or prior TPO-RA exposure: Interim results from the phase 4 ADOPT study Presenting Author: María Eva Mingot-Castellano PS2244: Response to Avatrombopag Among Patients with Chronic and Persistent Primary Immune Thrombocytopenia: the REAL-AVA 2.0 Real-World Study Presenting Author: M Y Levy PS2250: Evaluation of Efficacy and Safety of Avatrombopag in Children with Immune Thrombocytopenia based on Disease Duration: Results from the Avatrombopag Phase 3-b Pediatric Trial Presenting Author: Rachael F. Grace PB3676: Baseline Correlates with Durability of Avatrombopag Response: A Phase 3B Trial for Treatment of Children with ITP Publication Only Published on May 14 at 15:30 CEST PB3684: Consistent Response to Avatrombopag across Various Baseline Characteristics: Results from the Phase 3-b Trial for the Treatment of Children with Immune Thrombocytopenia Myelofibrosis PF849: Hematologic improvement experienced by pacritinib-treated patients with myelofibrosis in real-world clinical settings Presenting Author: Michael Marrone Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1242: Efficacy of pacritinib vs momelotinib in patients with thrombocytopenic MF: a matched adjusted indicated treatment comparison Presenting Author: Koo Wilson PF1306: Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial Presenting Author: Abiola Oladapo PS1827: Real-world effectiveness of pacritinib in patients with myelofibrosis who have concurrent thrombocytopenia and anemia Presenting Author: Raajit Rampal Poster presentation Session title: Poster Session 2 Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1842: Real-World Treatment Patterns and Clinical Outcomes in Patients with Myelofibrosis Treated with Pacritinib (PAC) with platelets ≥50 x109/L at PAC initiation: Interim results from the MY-PAC Study Presenting Author: Doug Tremblay PS2295: Economic Burden of Cytopenia in Patients with Myelofibrosis: Analysis of a US National Administrative Claims Database Presenting Author: Lucia Marasova PB3079: Cytopenia is associated with real-world disease progression and diminished survival in patients with myelofibrosis: Analysis of a US national administrative claims database Publication Only Published on May 14 at 15:30 CEST Paroxysmal Nocturnal Hemoglobinuria PF672: Early response in complement inhibitor naïve patients with paroxysmal nocturnal hemoglobinuria treated with pegcetacoplan in the Phase 3 PRINCE trial Presenting Author: Austin Kulasekararaj Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF676: Interim analysis of the ongoing COMPLETE study on the real-world effectiveness of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH) Presenting Author: Regis Peffault de Latour PS1662: A benefit assessment of pegcetacoplan dose increase in the Phase 3 PEGASUS trial of PNH patients with difficult-to-control disease Presenting Author: Morag Griffin Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS1665: Benefit of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria irrespective of baseline transfusion status Presenting Author: Britta Höchsmann Primary Hemophagocytic Lymphohistiocytosis (pHLH) PF1036: Emapalumab in patients with primary hemophagocytic lymphohistiocytosis: Efficacy and safety outcomes from a multinational, open-label, single-arm study Presenting Author: Franco Locatelli Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Zynlonta® (loncastuximab tesirine)Zynlonta® (loncastuximab tesirine) is a CD19-directed antibody drug conjugate (ADC). Zynlonta as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy. 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 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.

Phase 3ImmunotherapyClinical ResultDrug ApprovalPhase 1

19 Mar 2025

·www.prnewswire.com

Sobi announces a new research collaboration on the development of Gamifant® (emapalumab) in sepsis, which is to be presented at the ISICEM congress

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. 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

Phase 2Drug ApprovalImmunotherapy

100 Deals associated with Emapalumab-LZSG

External Link

KEGG	Wiki	ATC	Drug Bank
-	Emapalumab-LZSG	L04AA39	DB14724

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Macrophage Activation Syndrome	United States	NovImmune SA	27 Jun 2025
Systemic onset juvenile chronic arthritis	United States	NovImmune SA	27 Jun 2025
Lymphohistiocytosis, Hemophagocytic	United States	NovImmune SA	20 Nov 2018

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Immune System Diseases	NDA/BLA	European Union	Swedish Orphan Biovitrum AB	23 Jul 2020
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	United States	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Japan	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Belgium	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Canada	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Czechia	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	France	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Germany	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Italy	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022
Polyarticular Juvenile Idiopathic Arthritis	Phase 3	Netherlands	Supi Pharmaceutical (Guangzhou) Co., Ltd.	14 Jan 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03311854 \| NCT05001737 (NI-0501-14, FDA_CDER) Manual	Not Applicable	Systemic onset juvenile chronic arthritis \| Lymphohistiocytosis, Hemophagocytic \| Macrophage Activation Syndrome	39	GAMIFANT	vvftsjjwob(rscxruaani) = bmyqnqbtql ugrwubaeem (cnixisttux, 37.2 - 69.9) View more	Positive	27 Jun 2025
PS2153 (EHA2025)	Not Applicable	Cytokine Release Syndrome IFN-γ \| TNF-α \| IL-2 ... View more	38	Emapalumab	vjafysbfvr(fusgnirhno) = 8 patients died (2 from disease relapse/progression, 4 from severe infections, and 2 from septic shock associated with CRS) aoufqtrgdj (feibjuwefq ) View more	Positive	14 May 2025
Tandem Meetings ASTCT and CIBMTR2024	Not Applicable	-	-	Emapalumab	jasotapoqy(qijnweqkmh) = admemrybwg rrwzeubkck (bnzsupeeor )	-	01 Feb 2024
NCT04765553 (CTgov)	Phase 1	Rare Diseases	8	NI-0501 (Emapalumab)	rnsqerkcag(wqnszkquvz) = zzwqqjfcbu sudoexyjpi (pjpzwmijvh, olqoaeutqw - bnpdeatsqj) View more	-	24 Nov 2023
NCT04765553 (CTgov)	Phase 1	Rare Diseases	8	Saline (Placebo)	jmlstreaab = qgzrhvcpui diabzkmwwh (jnuyjrgkah, kuvseaiwqg - cgvpisdjlx) View more	-	24 Nov 2023
NCT03985423 (CTgov)	Phase 2/3	Lymphohistiocytosis, Hemophagocytic	7	Emapalumab-Lzsg	gyupwraxoi(hgzwkjwccf) = xupyvlqypg cztrgpjrfh (azqowvxjfh, bshvkisifr - bzdlimujhl) View more	-	06 Oct 2023
P353 (EBMT2023)	Phase 2	IFNÎ³ \| CXCL9	1	Emapalumab 3 mg/kg	ozjueuwnzj(uwcnmqcryg) = hneibwxwok ovwtmehase (eadghlwgog ) View more	Positive	23 Apr 2023
NCT02069899 (NI-0501-04 and NI-0501-05 \| ###DELETE, CTgov)	Phase 2/3	Lymphohistiocytosis, Hemophagocytic	58	Emapalumab (Enrolled-04 Cohort)	ufrtdwrzkv = ljzmzljuma vwohyhldag (mmhgaekbab, qpffuybzvh - nprxnhenda) View more	-	28 Jun 2022
NCT02069899 (NI-0501-04 and NI-0501-05 \| ###DELETE, CTgov)	Phase 2/3	Lymphohistiocytosis, Hemophagocytic	58	emapalumab (Enrolled-06 Cohort)	ufrtdwrzkv = ywodygergv vwohyhldag (mmhgaekbab, dmoefgskxy - boyggnpcga) View more	-	28 Jun 2022
NCT04731298 (P449, EBMT2022)	Phase 2	CXCL9	10	Emapalumab	szbclzsoqc(thrrbpmvoj) = tqofowqbzx txjnqlfjku (upeazcyvbk ) View more	Positive	19 Mar 2022
NCT02069899 \| NCT01818492 (Tandem Meetings ASTCT and CIBMTR2021)	Phase 2/3	Lymphohistiocytosis, Hemophagocytic Second line	45	Emapalumab 1 mg/kg	huczkapilv(ixbwmzrmnd) = owzqshurvh hapzpoockm (pvuykvusjb, 68.3 - 97.6) View more	Positive	01 Mar 2021
	Phase 2/3	Lymphohistiocytosis, Hemophagocytic Second line	45	Background dexamethasone 5-10 mg/m2	huczkapilv(ixbwmzrmnd) = fdppocuzdr hapzpoockm (pvuykvusjb, 62.1 - 92.1) View more	Positive	01 Mar 2021
NCT02069899 \| NCT01818492 (NI-0501-04 \| NI-0501-05, Pubmed \| Br Dent J \| N Engl J Med) Manual	Phase 2/3	Lymphohistiocytosis, Hemophagocytic First line	34	Emapalumab (previously treated)	pjyruxioto(cthfzmimaa) = qboiecjmqf zjcopteumb (mxeowczgpp ) View more	Positive	07 May 2020
	Phase 2/3	Lymphohistiocytosis, Hemophagocytic First line	34	Emapalumab (previously untreated)	pjyruxioto(cthfzmimaa) = lxtuqfzdyu zjcopteumb (mxeowczgpp ) View more	Positive	07 May 2020

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