Patients were randomized to receive an intravitreal dose of 5mg ANX007 monthly (n=89), 5mg ANX007 every other month (n=92) or sham monthly or every other month (pooled n=89) for a treatment period of 12 months, followed by a six-month off-treatment period. The primary outcome measure of the study was the rate of change in GA lesion growth (slope) from baseline as measured by fundus autofluorescence (FAF) through 12 months for the study eye. The study included multiple pre-specified visual function measures to assess the effects of ANX007 on vision: change from baseline in BCVA, change from baseline in low-luminance best corrected visual acuity (LLVA) and change in baseline from low-luminance visual acuity deficit (LLVD). Top line results from the study were reported in May 2023.
About ANX007
GA is a disease of vision loss driven by the loss of photoreceptor cells, a type of neuron. Annexon was founded on the discovery that C1q, the initiating molecule of the classical complement cascade, is a driver of neurodegenerative disease. C1q binds to synapses (neuronal connections) in disease, triggering activation of the classical complement cascade with immune cell recruitment, neuroinflammation, synapse loss, and neuronal damage. In GA, C1q anchors classical pathway activation on photoreceptor cell synapses and outer segments to cause inflammation and photoreceptor cell loss. Inhibiting C1q blocks all downstream components of the classical cascade to protect synapses and photoreceptors, providing a unique neuroprotective mechanism. Annexon’s neuroprotective mechanism is distinct from inhibition of C3 or C5, which do not inhibit upstream components of the classical cascade that contribute to photoreceptor damage. Further, selective inhibition of the classical cascade leaves the lectin and alternative complement pathways in place for normal homeostatic and immune functions. Preclinical models have demonstrated that inhibition of C1q protected photoreceptor synapses and cells and importantly, photoreceptor cell function.
About Geographic Atrophy
Geographic atrophy (GA), also known as atrophic age-related macular degeneration (AMD) or dry AMD, has a genetic link to aberrant complement activity and can lead to blindness caused by damaged and dying retinal cells. It is estimated that one million people in the United States and five million people globally suffer from GA.
About Annexon
Annexon (Nasdaq: ANNX) is a clinical-stage biopharmaceutical company seeking to bring game-changing medicines to patients with classical complement-mediated diseases of the body, brain and eye. The classical complement pathway within the immune system, when overactivated, drives inflammation in a host of autoimmune, neurodegenerative and ophthalmic diseases. Annexon is advancing a new class of complement medicines targeting the early classical cascade and all downstream pathway components that contribute to disease, while selectively preserving the beneficial immune functions of other complement pathways. Annexon is rigorously developing a pipeline of diversified product candidates across multiple mid- to late-stage clinical trials, with clinical data anticipated throughout 2023 and beyond.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “suggest,” “target,” “on track,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press
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