Annexon, Inc. Reports Promising Interim Phase 2 Data Showing Improvement in Clinical Measures with ANX005
January 04, 2022 at 09:05 pm
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Annexon, Inc. announced interim data from its ongoing, open-label Phase 2 clinical trial of ANX005 in patients with Huntington's disease (HD) who completed the 24-week treatment period. Annexon is developing ANX005, its lead monoclonal antibody candidate, for the treatment of a range of complement-mediated disorders, including HD. HD is a fatal, progressive movement disorder involving the activation of the classical complement pathway. C1q,
the initiator of the classical pathway, is recognized as a major driver of a destructive immune response that leads to synapse loss and neurodegeneration. ANX005 is designed to disrupt the disease course, stopping the start of damaging complement activation by blocking C1q and the entire classical complement pathway. Interim data from the ongoing Phase 2 trial show that treatment with ANX005 has been generally well-tolerated, with full target engagement of C1q in both serum and cerebrospinal fluid (CSF) observed in evaluable patients through the dosing period. Evaluable patients maintained clinical function, as measured by changes in mean Composite Unified Huntington's Disease Rating Scale (cUHDRS), relative to baseline after six months of treatment, and improvement in cUHDRS was observed in more than half of all evaluable patients and in 75% of evaluable patients who showed excess complement activity at baseline. NfL levels observed after six months of treatment remained generally consistent and were comparable to NfL levels described in published natural history data for HD patients. Overall, these interim findings appear to build on the scientific hypothesis of Annexon scientific founder, the late Ben Barres, who believed that blocking C1q protects synaptic loss and can lead to rapid functional impact on clinical outcomes in neurodegenerative diseases.
Annexon, Inc. is a clinical-stage biopharmaceutical company. The Company is engaged in advancing a late-stage clinical platform of therapies for people living with classical complement-mediated neuroinflammatory diseases of the body, brain, and eye. Using its platform, it is identifying and characterizing the role of the complement pathway in three therapeutic areas: autoimmune, neurodegeneration and ophthalmology. Its lead candidate, ANX005, is an investigational monoclonal antibody (mAb), formulated for intravenous administration in a pivotal Phase III clinical trial for the treatment of patients with Guillain-Barre Syndrome (GBS). It is evaluating ANX007, an antigen-binding fragment (Fab), formulated for intravitreal administration, for the potential treatment of patients with geographic atrophy (GA). It is developing ANX1502, which is an oral small molecule for the treatment of autoimmune indications. It has developed ANX009, a C1q-blocking Fab formulated for subcutaneous delivery.