Vonaprument

Mixed gender (female-only in single-dose study; male and female in repeat-dose studies), 2-4 years old, 2.1-3.7 kg^[1]

1 mg/eye; 2.5 mg/eye; 5 mg/eye

i.v.t.; single bilateral dose, or two bilateral doses on days 1 and 29

Measured vitreous free drug levels through 4 weeks following single doses of 1 or 5 mg/eye, with an approximate 3-day vitreous half-life.
Recorded vitreous Cmax values of 468,000 ng/mL (1 mg/eye) and 2,010,000 ng/mL (5 mg/eye), with AUClast values of 46,500,000 h*ng/mL (1 mg/eye) and 247,000,000 h*ng/mL (5 mg/eye).
Detected free drug in perfused retina and choroid 4 weeks post-last dose, and up to 2 weeks post-last dose in optic nerve following repeat doses of 5 mg/eye.
Achieved near complete inhibition of free-C1q levels in all sampled ocular fluids and tissues at 2 weeks post-last dose, with suppression maintained in retina, choroid, vitreous, and aqueous humor at 4 weeks post-last dose.
Demonstrated a strong correlation between aqueous humor free-C1q suppression and vitreous and retina free-C1q suppression, and a weaker but significant correlation with choroid free-C1q suppression.
No drug-related adverse changes were observed across all dose levels.