“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.”
✓ VerifiedFDA multi-discipline review, p. 38Source ↗
Why that species
Binds human and monkey C5 with high affinity but not mouse, rat or rabbit C5
“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.”
✓ VerifiedFDA multi-discipline review, p. 37Source ↗
Surrogate molecule
No surrogate in toxicology; C5 humanized transgenic mice used only for pharmacology; clinical antibody tested in monkeys
“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.”
✓ VerifiedFDA multi-discipline review, p. 36Source ↗