Annexon’s ANX-005 in combination with intravenous immunoglobulin (IVIg) has entered into an open-label multi-centre Phase Ib clinical trial for the treatment of Guillain-Barré syndrome (GBS), and recruitment for the study is now ongoing.
This study will involve a cohort of 12 patients and use an ANX-005 dose shown to be safe and well-tolerated as monotherapy in another trial and will assess the safety, tolerability, and drug-drug interactions of ANX005 and IVIg.
The subjects will be followed for six months after treatment for observation and evaluation of outcomes based on a disability score ranking.
GBS is a rare autoimmune neurological disorder in which the immune system attacks the peripheral nervous system, and is often triggered by an antecedent illness such as influenza, Campylobacter jejuni infection, mycoplasma pneumonia, cytomegalovirus (CMV) infection, or Epstein-Barr virus (EBV) infection. The clinical presentation of GBS is highly variable, resulting in difficulty diagnosing the syndrome. GBS patients progress in weakness with about 25% requiring artificial ventilation, 20% becoming unable to walk independently after six months, and 2–5% dying. About 3–5% of patients will experience a relapse in their lifetimes.
The two main treatments for GBS are IVIg and plasma exchange, which can reduce the severity and shorten the duration of the illness. Few recent advances have been made in treating the syndrome, though some recent experimental studies have shown that complement inhibition combined with IVIg could improve outcomes.
ANX-005 was chosen as a potential treatment for GBS as it is a humanized immunoglobulin G4 recombinant antibody against C1q that inhibits its function as the initiating molecule of the classical complement cascade. It has been developed by Annexon, a pharmaceutical company that focuses on therapeutic products and pathway inhibitors for the treatment of neurological disorders.
In view of the recent Fast Track designation given to ANX-005 by the FDA for the treatment of GBS, observers can only hope for successful safety and efficacy outcomes from this and any additional trials of ANX-005 in order to make this drug available for patients with GBS as quickly as possible.