Voydeya: animal efficacy model or human cells?

“In serum derived from cynomolgus monkeys administered single oral or multiple oral doses of danicopan, an ex vivo analysis revealed that significant (80% to 90%, at concentrations greater than or equal to 258nM) and concentration-dependent inhibition of AP activity was achieved in monkeys.”

“Based on the pharmacology data (and the lack of cross reactivity to rodent species), the cynomolgus monkey, beagle dog, and New Zealand white (NZW) rabbit were selected as the nonclinical efficacy species.”

“The significant variability noted inter- and intra-species was attributed to the intrinsic AP activity for each individual. This aspect should be considered for the interpretation of the pharmacological data, considering the potential uncertainty when results are interpreted.”