ANX005 is a clinical-stage investigational monoclonal antibody intended to treat patients with antibody-mediated autoimmune and complement-mediated neurodegenerative disorders. This novel therapy is formulated for IV administration and is designed to inhibit C1q and the entire classical complement pathway.

We have completed a Phase 1b clinical trial of ANX005 in Guillain-Barré Syndrome (GBS). In this placebo controlled, dose escalation trial in 31 patients, ANX005 was well-tolerated at all dose levels. An IV infusion of ANX005 resulted in full and prolonged C1q engagement and classical cascade inhibition, measured in the blood and cerebrospinal fluid (CSF). Treated patients showed significantly reduced levels of neurofilament light chain (NfL), a well-accepted marker of nerve damage in neurodegenerative disease that has been shown to correlate with disease severity and clinical outcomes. While our Phase 1b trial was not powered for statistical significance, treated patients also demonstrated consistent positive trends across key GBS outcome measures, including an early impact on muscle strength, which correlates with prognosis for long-term functional recovery.

Based on these promising results, we plan to advance ANX005 into later-stage clinical trials in GBS. ANX005 has received both Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration for the treatment of GBS.