Safety and Target Engagement of Complement C1q Inhibitor ANX007 in Neurodegenerative Eye Disease: Results from Phase I Studies in Glaucoma.

ABSTRACT:

Purpose

Complement C1q, the initiating molecule of the classical complement cascade, is involved in synapse elimination and neuronal loss in neurodegenerative diseases including glaucoma. Here we report an evaluation of the safety, tolerability, and ocular pharmacokinetics (PK) and pharmacodynamics of intravitreal (IVT) injections of ANX007, an anti-C1q monoclonal antibody fragment that blocks activation of the classical complement cascade.

Design

An open-label, single-dose-escalation phase Ia study followed by a double-masked, randomized, sham-controlled, repeat-injection phase Ib study.

Participants

A total of 26 patients with primary open-angle glaucoma.

Methods

Nine patients with primary open-angle glaucoma (mean Humphrey visual field deviation between -3 a

SUBMITTER: Sun Y 

PROVIDER: S-EPMC10130689 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature