Annexon Biosciences has reported positive topline results from its Phase III clinical trial of ANX005, a C1q blocking antibody, in treating Guillain-Barré syndrome (GBS).
The placebo-controlled, randomised trial enrolled 241 patients in Bangladesh and the Philippines to assess two 30mg/kg and 75mg/kg doses of of ANX005.
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According to the findings, the trial met its primary endpoint at week eight for the 30mg/kg dose, demonstrating a 2.4-fold improvement on the GBS-disability scale.
The 30mg/kg dose also showed significant improvements on secondary endpoints, such as muscle strength and reduced dependency on artificial ventilation.
Patients treated with ANX005 30mg/kg experienced a median reduction of 28 days on artificial ventilation by week 26, and were able to walk independently 31 days sooner than those taking the placebo.
In addition, an early reduction in serum levels of neurofilament light chain, a biomarker of nerve damage, was observed.
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By GlobalDataBoth doses of ANX005 showed well-tolerated safety and tolerability profiles, without any new safety concerns identified.
Adverse events were predominantly mild to moderate, with infusion-related reactions being the most common.
There were no autoimmune adverse events, drug-related deaths, or serious infections reported.
Annexon expects to submit real-world evidence data and a biologics license application in the first half of next year.
ANX005 has received fast track and orphan drug designations from the US Food and Drug Administration (FDA) and orphan drug designation from the EMA for GBS treatment.
Annexon president and CEO Douglas Love said: “With the potential to be the first targeted treatment for GBS in the US, ANX005 demonstrated consistent improvement and functional benefits on key primary and secondary endpoints.
“Additionally, we observed in our Phase III trial that early treatment with ANX005 resulted in rapid neuroprotection that stopped the advancement of disease and helped GBS patients get better sooner. These results reinforce Annexon’s founding thesis that C1q inhibition is a powerful mechanism of action to stop the progression of neuroinflammation and underscore the potential of ANX005 and our classical complement platform to treat GBS and a host of other diseases of the body, brain and eye.”
