clinical pipeline
for diseases of the body, brain & eye
Scientific Co-Founder Dr. Ben Barres
STOP COMPLEMENT-MEDIATED DISEASES AT THE START
Annexon is pioneering a class of new complement medicines for patients with classical complement-mediated disorders of the body, brain and eye. We are conducting ongoing clinical trials in multiple serious autoimmune, neurodegenerative and ophthalmic diseases.
Late-stage Pipeline of Drug Candidates for Neuroinflammatory Diseases
Targeting Both Rare & Large Patient Populations
| Candidate | Design | Franchise/Indication | Preclinical | Phase 1 | Phase 2 | Phase 3 | Anticipated Milestones | |
|---|---|---|---|---|---|---|---|---|
| FLAGSHIP PROGRAMS | ||||||||
| Tanruprubart (ANX005) |
IV mAb |  |
Guillain-Barré Syndrome (GBS) | Guillain-Barré Syndrome (GBS) is a severe disease resulting from an autoantibody attack on the peripheral nerves, triggering the complement cascade (C1q) and causing neurodegenerative and cognitive impairment. Annexon’s clinical-stage investigational monoclonal antibody, tanruprubart (ANX005), is intended to treat patients with GBS. This novel therapy is formulated for intravenous (IV) administration and is designed to inhibit C1q and the entire classical complement pathway. Tanruprubart has received both Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration, and Orphan Drug Designation from the European Medicines Agency, for the treatment of GBS.
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MAA filed Jan 2026; FDA discussions ongoing |
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| Vonaprument (ANX007) |
IVT Fab |  |
Geographic Atrophy (GA) | Geographic atrophy (GA) is an advanced form of dry, age-related macular degeneration (AMD) of the retina and is a chronic, progressive disease of the macula that results in loss of central vision. Excess classical complement activity in the retina is a key driver of GA. Annexon’s vonaprument (ANX007) is a clinical-stage investigational monoclonal antibody antigen-binding fragment (Fab) formulated for intravitreal (IVT) administration. Vonaprument involves a differentiated neuroprotective approach designed to protect photoreceptor cells and retinal function by blocking C1q and the entire classical pathway, while allowing for normal immune activity of the lectin and alternative complement pathways. Vonaprument has received Priority Medicine (PRIME) designation from the European Medicines Agency for the treatment of GA.
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ARCHER II enrollment completed; Ph3 data 2H26 |
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| ANX1502 | Oral small molecule | |
Autoimmune Indications | ANX1502 is a first-in-kind oral small molecule inhibitor of the classical complement pathway in development for the treatment of complement-mediated autoimmune diseases. Annexon is advancing ANX1502 into a proof-of-concept study in patients with cold agglutinin disease (CAD).
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Update upon CAD study completion in 2026 |
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| NEXT WAVE PROGRAMS | ||||||||
| ANX005 | IV mAb |  |
Huntington’s Disease (HD) | Huntington’s disease (HD) is a progressive movement disorder, resulting in dementia and psychosis driven by the activation of the classical complement pathway. C1q is recognized as a major driver of synaptic loss and neurodegeneration. Annexon’s clinical-stage investigational monoclonal antibody, ANX005, targets aberrant C1q activity in complement-mediated neurodegenerative disorders like HD. This novel therapy is formulated for intravenous (IV) administration and is designed to inhibit C1q and the entire classical complement pathway.
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Poised for late-stage Phase 2b/3 development | |||
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Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the progressive weakness of the limb and respiratory muscles. Aberrant C1q activity potentially drives synaptic loss, resulting in this disability. Annexon’s clinical-stage investigational monoclonal antibody, ANX005, targets both central and peripheral nervous system aspects of ALS. This novel therapy is formulated for intravenous (IV) administration and is designed to inhibit C1q and the entire classical complement pathway.
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Poised for late-stage Phase 2b/3 development | |||||
| ANX009 | Subcutaneous Fab | |
Lupus Nephritis (LN) | Lupus nephritis (LN) is a severe and life-threatening nephritic disease characterized by autoantibody-driven activation of C1q and the classical complement pathway. Endogenous pathogenic anti-C1q antibodies (PACAs) occur in the majority of patients with LN and have been shown to correlate with classical complement activation and disease activity. Annexon’s clinical-stage investigational drug candidate, ANX009, is a subcutaneously administered antigen-binding fragment (Fab) that disrupts autoantibody complement activation. It is being developed for the treatment of antibody-mediated autoimmune diseases of blood and vascular tissues.
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Evaluating options for future development | |||
| Autoimmune Neuro Ophthalmology |
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