Mesoblast Limited announced key Day 100 survival outcomes of its Phase 3 trial for remestemcel-L, an allogeneic mesenchymal stem cell product candidate, in children with steroid refractory acute Graft Versus Host Disease (aGVHD). The results are being presented today at the 2018 annual meeting of the International Society for Stem Cell Research (ISSCR), being held in Melbourne from June 20-23.
The open-label Phase 3 trial enrolled 55 children with steroid-refractory aGVHD (aged between two months and 17 years) in 32 sites across the United States, with 89% of patients suffering from the most severe form, grade C/D aGVHD. The trial was performed under an United States Food and Drug Administration (FDA) Investigational New Drug Application.
This trial previously met the primary endpoint of Day 28 overall response rate (69% versus 45% historical control rate, p=0.0003). Top line Day 100 results demonstrated 87% survival rate for Day 28 responders to remestemcel-L treatment (33/38), and an overall survival rate of 75% (41/55). The multi-infusion regimen of remestemcel-L was well tolerated.
Acute GVHD is associated with significant morbidity and is a leading cause of mortality after allogeneic hematopoietic stem cell transplantation for blood cancers or other conditions. Severe aGVHD (determined by grade C/D, liver or gut involvement, or high risk stratification) has the highest risk of failure to first-line corticosteroids and high transplant-related mortality1. Day 100 mortality can reach 70% in patients who fail to respond to initial steroid therapy2-4 , and 12-month mortality approaches 90%5 .
Mesenchymal stem cells have anti-inflammatory and immunomodulatory biological activity that supports their investigational use in aGVHD6 . The immunomodulatory actions of these cells are triggered through receptor activation by inflammation, resulting in regulation of multiple cellular arms of the immune system that are central to aGVHD pathogenesis
Based on interactions with the FDA, Mesoblast believes that successful results from the completed Phase 3 trial, together with Day 180 safety, survival and quality of life parameters in these patients, may provide sufficient clinical evidence to file for accelerated approval of remestemcel-L in the United States, where there are currently no approved products for steroid-refractory aGVHD.
Mesoblast Chief Executive Dr Silviu Itescu said, “It is wonderful to see that our unique cellular therapy has shown such promising survival rates at Day 100 in children suffering from this devastating disease. Our objective is to bring this new therapy to market, and make it available to patients who are in desperate need with this life-threatening complication of an allogeneic bone marrow transplant.”
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