Annexon Inc (NASDAQ: ANNX) has announced encouraging final data from the open-label Phase 2 clinical study of ANX005 in individuals with Huntington’s disease (HD).
Annexon targets C1q in treating HD
HD, a deadly degenerative mobility disorder, affects 80,000 people worldwide. To reduce or stop alternative complement illnesses, Annexon’s novel strategy for treating HD targets C1q, the starting molecule of the classic complement system. By activating and increasing complement elements that induce neuroinflammation, synapse destruction, and eventually synapse loss, C1q improperly detects and tags functional synapses required for proper brain health in neurodegenerative illnesses like HD. In order to preserve functional synapses and slow or stop neurodegeneration, ANX005 is made to block C1q and the whole alternative complement pathway completely.
UCL Huntington’s Disease Centre associate director and neurology professor at University College London Edward Wild said, “Huntington’s disease is a devastating condition, with no cure or approved disease-modifying treatments available. The apparent continued stabilization of clinical function over nine months is notable, and generally not expected in a progressive disease like HD.”
Additionally, the sustained advancement seen in patients with higher baseline supplement activity and the favorable benefit-risk profile shown by ANX005 over the course of the nine-month study support the idea that complement inhibition could be a useful therapeutic strategy for this challenging disease.
ANX005 administered intravenously in subjects with early HD
In individuals with early evident HD or at risk for developing it, ANX005 was delivered intravenously for a six-month dosage period, accompanied by a three-month follow-up time frame, in Phase 2 multi-site, open-label clinical research. The tolerability and safety of ANX005, its pharmacokinetics (PK), as determined by serum and CSF concentrations, and its pharmacodynamics (PD) impacts, as determined by C1q, C4a, and NfL serum and CSF concentrations, were the study’s key outcome measures.
Around 28 patients in total participated in the research, and 23 of them finished the full six months of treatment as well as the subsequent three months of follow-up. Efficacy statistics determined by the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) and Total Functional Capacity are included in the Phase 2 results published.