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Annexon Drug Shows Neuroprotective Effects

■ Annexon Biosciences has announced encouraging results from a phase 1b dose-ranging clinical trial to evaluate its anti-C1q antibody, ANX007, in 17 patients with glaucoma. ANX007 is an investigational monoclonal antibody antigen-binding fragment (Fab) formulated for intravitreal administration. In the phase 1b trial, ANX007 was well tolerated and resulted in full target engagement and inhibition of C1q in the eye for at least 4 weeks following a single intravitreal treatment.

“Inhibition of C1q may provide neuroprotective benefit by preventing the aberrant loss of functioning synapses in the retina in a variety of ophthalmic diseases,” said Jeffrey L. Goldberg, MD, professor and chair of ophthalmology at the Byers Eye Institute at Stanford University and member of Annexon’s scientific advisory board, in a news release. “There is a significant need to develop novel neuroprotective therapies that can slow damage to the optic nerve and prevent vision loss for patients with glaucoma and other ophthalmic diseases.”

In the phase 1b clinical trial of ANX007, glaucoma patients were treated with multiple doses of intravitreal ANX007. The trial objectives included safety, tolerability, pharmacokinetics, and target engagement. Top-line results demonstrated that ANX007 was well tolerated at all dose levels. At the 2 higher dose levels, a single intravitreal injection of ANX007 achieved complete suppression of the C1q target for at least 4 weeks, as measured in ocular fluid from aqueous humor taps.