Scientific Co-Founder Dr. Ben Barres
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.
Targeting Both Rare & Large Patient Populations
|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, 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. ANX005 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.
Report Phase 3 trial results in 2Q 2024
Prepare BLA submission in 2H 2024
|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 ANX007 is a clinical-stage investigational monoclonal antibody antigen-binding fragment (Fab) formulated for intravitreal (IVT) administration. ANX007 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. ANX007 has received Priority Medicine (PRIME) designation from the European Medicines Agency for the treatment of GA.
Initiate Phase 3 ARCHER II trial in mid 2024
Initiate Phase 3 ARROW trial in 2H 2024
|Oral small molecule
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).
Initiate CAD proof-of-concept trial in 2Q 2024
Report CAD proof-of-concept trial results in 2H 2024
|NEXT WAVE PROGRAMS
|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.
|Poised for late-stage Phase 2b/3 development
|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.
|Poised for late-stage Phase 2b/3 development
|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.
|Evaluating options for future development
At Annexon Biosciences, we are committed to bringing medicines to patients with complement-mediated diseases of the body, brain and eye. At present time, our therapeutic candidates are investigational, meaning that they have not been approved as safe effective for their intended use(s) by regulatory health authorities, such as the United States Food and Drug Administration (FDA).
Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to certain investigational drugs for serious or life-threatening diseases or conditions. Providing access to investigational therapies through an expanded access program is a complex matter. We are currently evaluating the safety of our investigational therapies across multiple clinical trials, and before we provide patients with access to these therapies, we want to ensure that doing so will not inadvertently cause harm to those patients or potentially compromise our broader clinical development program, which is shaped by our data and by insight from the FDA and other regulatory authorities.
We believe that diligent adherence to the formal clinical development and regulatory approval processes offers the greatest potential for us to have a positive impact on the patients for whom our investigational therapies are designed to benefit. With these goals in mind, our focus is on completing our clinical trials in an effort to generate the necessary evidence of safety and efficacy required to obtain regulatory approval.
For these reasons, Annexon is not currently offering access to its investigational therapies outside of a clinical trial setting. We encourage those interested in gaining access to our investigational therapies to consult their physician regarding the possibility of participating in one of our clinical trials. A listing of our clinical trials can be found on publicly accessible databases, such as clinicaltrials.gov.
Additional questions can be submitted to: