Mesoblast Limited announced the primary endpoint success of a phase three trial on February 21st. The drug tested aims to help treat steroid refractory acute graft versus host disease. No products currently exist in the United States to treat this specific condition. Keep reading to learn more about his development, or follow the original news brief here.
The phase three trial focused on Mesoblast Limited’s new allogenic mesenchymal stem cell product. The company refers to the product as MSC-100-IV (remestemcell-L). Researchers with the company studied the product’s effects on steroid refractory acute graft versus host disease in children. Reaching primary endpoint for the study involved Day 28 overall response.
55 children enrolled in the Mesoblast phase three trial. The trial took place in 32 sites across the United States. Researchers recorded the Day 28 overall response at 69%. This represents a sizable improvement over standards. Defined by protocol, the standard control rate has been 45%.
Other significant results include the reduction of mortality rates. When steroid therapy proves ineffective, the day 100 mortality rate for patients can reach as high as 70%. Patients in the study, however, who received at least one infusion of the new treatment faced much lower rates. After 100 days of observation, the mortality rate for this group reached only 22%.
Furthermore, patients tolerated the treatment with remestemcel-L well. The occurrence of negative side effects matched estimates by researchers. Researchers based these expectations on previous experiences with remestemcell-L, and what can be expected from graft versus host disease.
Dr. Joanne Kurtzberg, senior investigator of the Mesoblast phase three trial, said the following regarding the trial:
“These children are a very challenging patient population as they suffer from a particularly aggressive and life-threatening disease for which there are currently no available treatments. We are now seeing that children who receive remestemcel-L can have significant overall response rates and reduced early mortality. We are delighted that Mesoblast has attained such an important milestone towards delivering a potentially effective treatment for this very serious and life threatening condition.”
The United States Food and Drug Administration designated Mesoblast for fast tracking in 2017. This comes as a result of there being no current treatments for steroid-refractory acute graft versus host disease.
With this sort of relationship with the FDA, Mesoblast hopes to use their successful trial results to file for accelerated approval.
Dr. Silviu Itescu, Mesoblast’s chief executive underlined the huge impact of the study’s results. He continues to express a belief that Mesoblast’s cell therapies, expertise, and practices, will make a “substantial difference” in the lives of patients with “serious, and life threatening diseases.”
