Annexon Expands Classical Complement Platform into Neurodegenerative Diseases of the Brain with Initiation of Huntington’s Disease Clinical Program

– First patient dosed in Phase 2 study of ANX005 C1q targeted mAb –

SOUTH SAN FRANCISCO, Calif., Nov. 12, 2020 (GLOBE NEWSWIRE) -- (“Annexon”) (Nasdaq: ANNX), a clinical stage biopharmaceutical company developing a pipeline of novel therapies for patients with classical complement-mediated disorders of the brain, body and eye, today announced that it has initiated a Phase 2 study, dosing the first patient with its full-length monoclonal antibody ANX005 in Huntington’s Disease (HD). The Phase 2 trial in HD expands Annexon’s classical complement platform into neurodegenerative diseases of the brain and highlights the pioneering research of the company’s co-founder, the late Dr. Ben Barres, former member of the National Academy of Sciences and Chair of Neurobiology, Stanford University. Aberrant activation of C1q plays a significant role in the neurodegenerative process by causing synapse loss, chronic neuroinflammation and eventual neuronal death.

“Huntington’s Disease is a devastating, progressive movement disorder with no cure and no approved therapeutic options available to patients and their families,” commented Sanjay Keswani, MBBS, BSc, FRCP, Chief Medical Officer of Annexon. “In neurodegenerative conditions like HD, our goal is to disrupt the disease course by inhibiting harmful classical complement activity, including synapse loss, that leads to neurodegeneration and cognitive impairment. We are excited to advance ANX005 and look forward to initial results from our Phase 2 trial in the second half of 2021.”

“Annexon targets the initiating protein of the classical complement pathway, C1q, which uniquely binds to synapses in the brain and appears to cause inappropriate synapse elimination during chronic neurodegenerative disease, such as HD,” stated Beth Stevens, PhD, Associate Professor of Neurology, Children’s Hospital Boston and former postdoctoral scholar in Dr. Barres’ lab. “Inhibiting C1q and protecting functioning synapses may benefit patients with neurodegenerative conditions.”

About the Clinical Trial and ANX005

The Phase 2 open-label trial is enrolling up to 24 patients and is designed to assess safety, tolerability and biomarkers of target engagement and impact on neurodegeneration.

ANX005 is an IV formulated monoclonal antibody designed to inhibit C1q and the entire classical complement pathway. ANX005 is designed to treat patients with antibody-mediated autoimmune and complement-mediated neurodegenerative disorders. In addition to the HD indication, Annexon has completed a Phase 1b clinical trial of ANX005 in Guillain-Barré Syndrome (GBS) and has received fast track and orphan drug designations from the U.S. Food and Drug Administration for the treatment of GBS.

More information can be found at or , identifier NCT04514367, or the HD Coalition for Patient Engagement .        

About Annexon, Inc.



Annexon is a clinical-stage biopharmaceutical company developing a pipeline of novel therapies for patients with classical complement-mediated disorders of the brain, body and eye. The company’s pipeline is based on its platform technology addressing well-researched classical complement-mediated autoimmune and neurodegenerative disease processes, both of which are triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon is deploying a disciplined, biomarker-driven development strategy designed to establish that its product candidates are engaging the target at a well-tolerated therapeutic dose in the intended tissue compartments. For more information, visit .

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