Ryoncil: which tox species, and why?

“The nonclinical program utilized multiple surrogate animal-derived ceMSC manufactured similarly to the clinical product.”

“This approach was taken to model administration of allogeneic ceMSCs in humans and evaluate the interactions between the cell product and recipient immune system.”

“Nonclinical studies were conducted using human bone marrow-derived ceMSC and surrogate animal-derived ceMSC produced using an early manufacturing process.”

“In a 90-day GLP repeat dose toxicity study, rats received 2×106, 10×106 or 20×106 rat ceMSC/kg administered in a 5mL/kg dosing volume by tail vein injection over a two-minute period”

“Genotoxicity, carcinogenicity and reproductive and developmental toxicity studies were not performed for remestemcel-L. These studies are not warranted based on the product characteristics and safety profile.”

“In a tumorigenicity study in athymic mice, administration of human ceMSC from five separate donors was evaluated at dose levels up to 50x106 cells/kg. There was no evidence of neoplastic tissue formation attributable to ceMSC over the six-week duration of the study.”

“The nonclinical program utilized multiple surrogate animal-derived ceMSC manufactured similarly to the clinical product.”