New data for emapalumab in the treatment of macrophage activation syndrome to be presented at the ACR conference
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
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
The data will be presented by
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
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
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