Fontolizumab

Results showed that MAS remission was seen in 13 of 14 patients with sHLH/MAS receiving emapalumab who had an inadequate response to high-dose glucocorticosteroidsWALTHAM, Mass., April 04, 2023 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®) (STO:SOBI), today announced that the Annals of Rheumatic Diseases has published results from an open-label, single-arm, multicenter phase 2 study evaluating the safety and efficacy of emapalumab, an anti-interferon-gamma monoclonal antibody, being investigated in patients with Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) who developed secondary hemophagocytic lymphohistiocytosis (sHLH)/Macrophage Activation Syndrome (MAS) following an inadequate response to high-dose glucocorticosteroids. In the published study, 14 patients with sJIA or AOSD and sHLH/MAS who did not respond to high-dose glucocorticosteroids received emapalumab. MAS remission was seen in 13 of the 14 patients by week 8, at a median time of 25 days, based on clinical and laboratory criteria. All 14 patients completed the trial, entered long-term follow-up and were alive at the end of follow-up. Based on the results of this study, Sobi decided to continue to evaluate emapalumab in this patient population and initiated the EMERALD phase 3 study, which is ongoing. "I believe that the results of this study represent an important milestone for the identification of a potential novel targeted treatment for patients with sJIA or AOSD with sHLH/MAS who have failed high-dose glucocorticoids," said Dr. Fabrizio De Benedetti, Head of the Division of Pediatric Rheumatology and Head of the Laboratory of ImmunoRheumatology at Bambino Gesù Children's Hospital in Rome, Italy. "We are eager to collect more data so that emapalumab becomes part of the therapeutic armamentarium for this rare disease and its high unmet medical need." sHLH/MAS is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on a background of rheumatologic diseases such as sJIA. It is classified as a secondary form of HLH and is caused by excessive activation and expansion of T cells and macrophages. A body of scientific evidence has been accumulated suggesting interferon gamma (IFNγ) is a major driver of hyperinflammation in diseases such as sHLH/MAS. “The results published in the Annals of Rheumatic Diseases are encouraging for patients with sJIA or AOSD developing sHLH/MAS, who have had an inadequate response to high dose glucocorticoid treatment,” said Dr. Alexei Grom, Research Director, Division of Rheumatology and Professor of Pediatrics at Cincinnati Children's Hospital Medical Center, in Cincinnati, Ohio. “The ability of emapalumab to achieve MAS remission is meaningful as there is a high unmet need for these patients.” “We are pleased that this newly published clinical data shows the potential benefit of emapalumab in patients with secondary HLH,” said Tony Hoos, Head of Research & Development and Medical Affairs, Chief Medical Officer at Sobi. “We remain committed to evaluating emapalumab as a potential new treatment option for patients affected by this severe condition. If our ongoing EMERALD phase 3 study in sHLH/MAS confirms the benefit, we are intending to file a supplemental Biologics License Application.” Emapalumab is marketed in the United States as Gamifant® and is indicated 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 has not been reviewed or approved by the U.S. Food and Drug Administration for sHLH/MAS.ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.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 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 emapalumab-lzsgEmapalumab-lzsg is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. In the US, emapalumab-lzsg is indicated for pediatric (newborn and older) and adult primary hemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, 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 in 2018 was based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). The efficacy and safety of emapalumab are currently being evaluated in a phase 3 study in patients with MAS in Still’s disease or systemic lupus erythematosus (SLE) (EMERALD; NCT05001737).U.S. Indication for Gamifant® (emapalumab-lzsg)Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated 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. Important Safety InformationInfectionsBefore initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay. During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated. Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration. Increased Risk of Infection with Use of Live VaccinesDo not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied. Infusion-Related ReactionsInfusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion. Adverse ReactionsIn the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%). Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema. Please see full US prescribing information for Gamifant. Sobi North AmericaAs the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of providing access to innovative treatments that make a significant difference in the lives of individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at www.sobi-northamerica.com or at www.sobi.com. Sobi® Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East, Asia and Australia. In 2022, revenue amounted to SEK 18.8 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Media Contact:matt.tooth@sobi.com

STOCKHOLM, Nov. 11, 2022 /PRNewswire/ -- Sobi® will present new data at the 64th Annual Meeting of the American Society of Hematology (ASH) taking place from 10 to 13 December 2022. Sobi's commitment to developing innovative treatments for people living with rare diseases is highlighted in studies spanning several rare disorders, including haemophilia, paroxysmal nocturnal haemoglobinuria (PNH), cold agglutinin disease (CAD), immune thrombocytopenia (ITP) and primary haemophagocytic lymphohistiocytosis (pHLH). Results from the long-term extension study of Aspaveli®/Empaveli™ (pegcetacoplan) in adults with PNH will be presented, demonstrating robust and sustained improvements for approximately two years in key markers of disease across a broad population of PNH patients. In haemophilia A, new data on efanesoctocog alfa will be presented. Efanesoctocog alfa, an investigational new class of factor therapy for people with haemophilia A, has the potential to elevate standards of care by providing high sustained factor VIII activity into the near-normal range for the majority of the week with once-weekly prophylaxis. New data also will be presented from the REAL-HLH study, which is the first study describing the clinical characteristics, treatment patterns and treatment outcomes of a larger cohort of patients who received Gamifant® (emapalumab) in a real-world clinical setting. New information will also be presented supporting the role of Doptelet® (avatrombopag) in the treatment of ITP and describing the design of a study with Aspaveli/Empaveli for the treatment of CAD. "The breadth of data being presented at ASH this year showcases our ongoing commitment to providing innovative treatments for those affected by rare, ultra-rare and life-threatening conditions," said Anders Ullman, Head of Research & Development and Chief Medical Officer at Sobi. "We look forward to collaborating and connecting in person at this year's meeting." ADC Therapeutics to present new data on loncastuximab tesirine Sobi collaboration partner ADC Therapeutics will present eight abstracts at ASH. Multiple presentations will highlight the clinical utility of loncastuximab tesirine in diffuse large B-cell lymphoma. For more information please visit: ir.adctherapeutics.com/press-releases. Key Sobi data to be presented at ASH 2022 All abstracts can be accessed via the official ASH website. About efanesoctocog alfa (BIVV001) Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to potentially extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been reviewed by any regulatory authority. Efanesoctocog alfa was granted orphan drug designation by the US Food & Drug Administration (FDA) in August 2017 and the European Commission in June 2019. The FDA has accepted for priority review the Biologics License Application for efanesoctocog alfa for the treatment of haemophilia A. The target action date for the FDA decision is 28 February 2023. Regulatory submission in the EU will follow availability of data from the ongoing XTEND-Kids paediatric study, expected in 2023. About the Sobi and Sanofi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, an investigational factor VIII therapy with the potential to provide high sustained factor activity levels with once-weekly dosing for people with haemophilia A. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory. About Aspaveli®/Empaveli™ Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. It is approved as Aspaveli for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months in the European Union and the United Kingdom and as Empaveli for treatment of adult patients with PNH in the United States and Saudi Arabia. Empaveli is also approved in Australia for treatment of adult patients with PNH who have an inadequate response to, or are intolerant of, a C5 inhibitor. About the Sobi and Apellis Collaboration Sobi and Apellis collaborate to develop and commercialize systemic Aspaveli/Empaveli. Sobi has exclusive ex-US commercialisation rights for systemic Aspaveli/Empaveli. Apellis has exclusive US commercialisation rights for systemic Empaveli. The companies have global co-development rights for systemic Aspaveli/Empaveli. About Doptelet® Doptelet (avatrombopag) is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved by the European Medicines Agency and the US Food & Drug Administration for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure and for the treatment of thrombocytopenia in adult patients with primary chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets. The incidence of primary ITP in adults is 3.3/100 000 adults per year with a prevalence of 9.5 per 100 000 adults1. About Gamifant® Gamifant (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant 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 loncastuximab tesirine Loncastuximab tesirine (US brand name ZYNLONTA®) is a CD19-directed antibody-drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalised by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumour cell death. The US Food and Drug Administration (FDA) has approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including DLBCL not otherwise specified, DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The LOTIS-2 study included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory study. ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy. 1 Lambert et al. Blood 2017 Sobi® Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East and Asia. In 2021, revenue amounted to SEK 15.5 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Contacts Thomas Kudsk Larsen Head of Communication and Investor Relations thomas.kudsklarsen@sobi.com +44 7443 191 773 For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. The following files are available for download: View original content: SOURCE Swedish Orphan Biovitrum AB Company Codes: Bloomberg:SOBI@SS, ISIN:SE0000872095, RICS:SOBI.ST, Stockholm:SOBI