Qalsody: which tox species, and why?

“From the PK point of view, the monkey was the most relevant species for non-clinical efficacy and safety studies.”

“Furthermore, a single base-pair mismatch to the Cynomolgus monkey gene suggests it may be active in NHPs, thereby enabling assessment of possible on-target effects, while wild-type animals of all other species appear not to be suitable due to at least four or more mismatches in the target sequence.”

“No ASO specifically targeting rat and mouse SOD1 was used in toxicology studies, due to the lack of sequence homology of tofersen in rodents.”