Veopoz: which tox species, and why?

“Thirteen- and 26-week repeat-dose toxicity studies were conducted with pozelimab in cynomolgus monkeys. As pozelimab is not pharmacologically active in rat or mouse, it was not evaluated in a rodent species.”

“Pozelimab (REGN3918) is a human monoclonal antibody that binds with high affinity to complement factor 5 (C5) from human and monkey but not mouse, rat, or rabbit.”

“serum samples from pozelimab-treated C5 humanized mice (i.e., transgenic mice expressing human C5) and monkeys resulted in > 80% inhibition of classical pathway-induced hemolysis.”

“Thirteen- and 26-week repeat-dose toxicity studies were conducted with pozelimab in cynomolgus monkeys. As pozelimab is not pharmacologically active in rat or mouse, it was not evaluated in a rodent species.”

“Pozelimab is not pharmacologically active in rat or mouse. Thus, an enhanced pre- and postnatal development study was conducted with pozelimab in pregnant cynomolgus monkeys, with juvenile dosing of the offspring for three months post-weaning.”

“The Executive Carcinogenicity Assessment Committee concurred with the Applicant’s proposal that nonclinical carcinogenicity studies with pozelimab are not necessary to support a BLA for this product.”

“serum samples from pozelimab-treated C5 humanized mice (i.e., transgenic mice expressing human C5) and monkeys resulted in > 80% inhibition of classical pathway-induced hemolysis.”