Vonaprument

Vonaprument (ANX-007) is an intravitreally injected C1q antibody fragment (with a molecular weight of approximately 48 kDa) and a human monoclonal antibody. Vonaprument inhibits the activation of the classical complement cascade by specifically recognizing the globular head of C1q and blocking substrate binding. After intravitreal injection, Vonaprument distributes to the retina, choroid and optic nerve, and achieves complete C1q target binding in the aqueous humor and retinal tissues. Vonaprument can be used in research related to neurodegenerative eye diseases such as age-related macular degeneration and glaucoma ^{[1] [2] [3]}.

In Vivo, Vonaprument (1-5 mg/eye; i.v.t.; single bilateral dose, or two bilateral doses on days 1 and 29) distributes to relevant ocular tissues in healthy cynomolgus monkeys, achieves sustained C1q target engagement (near complete inhibition in ocular fluids and tissues for up to 4 weeks post-repeat dosing), and is well tolerated at doses of 1, 2.5, and 5 mg/eye^[1].

In Vitro, Vonaprument (ANX007/ANX-007) (8-3000 ng/mL) binds with similar high affinity to human and cynomolgus monkey C1q, with EC50 values of 136 pM, 147 pM, and 179 pM for purified human C1q, human serum C1q, and cynomolgus monkey serum C1q, respectively^[1].

References:
[1]. Grover A, et al. Pharmacokinetic and Target Engagement Measures of ANX007, an Anti-C1q Antibody Fragment, Following Intravitreal Administration in Nonhuman Primates. Investigative ophthalmology & visual science. 2023 Feb 01;64(2):3.
[2]. Sun Y, et al. Safety and Target Engagement of Complement C1q Inhibitor ANX007 in Neurodegenerative Eye Disease: Results from Phase I Studies in Glaucoma. Ophthalmol Sci. 2023 Feb 24;3(2):100290.
[3]. Packer J S, et al. Genetic analysis of age-related macular degeneration highlights precision therapy opportunities for patients with high polygenic risk[J]. 2026.