Ken Gorson, MD, Tufts University School of Medicine, discusses topline results from real-world evidence study evaluating ANX005 in patients with Guillain-Barré syndrome (GBS).
GBS is an acute, rapidly progressive neurological disease consisting of a spectrum of rare post-infectious neuropathies that usually occur in otherwise healthy patients. The peripheral nerve damage progresses rapidly, causing acute neuromuscular paralysis that can lead to significant morbidity, disability and mortality.
ANX005 is an intravenous, selective, rapid-acting agent that targets C1q and prevents its activity in the nervous system, leading to reduced inflammation and nerve damage. The ANX005 clinical trial is a phase 3, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of ANX005 in patients with GBS. Results from this study demonstrated statistically significant effects of 30 mg/kg ANX005 over placebo including a higher likelihood of being in a better state of health at week one, expedited recovery, reduced days on mechanical ventilation, and with a well-tolerated safety profile.
In the real-world evidence study (IGOS), patients were matched 1:1 on key prognostic factors of disease severity such as muscle strength and GBS disability score prior to treatment. In this global, prospective, observational, multicenter cohort study, 79 real-world patients were observed in comparison to 79 patients treated with ANX005 30 mg/kg from the previous study.
Patients treated with ANX005 were observed to have a faster and greater improvement in muscle strength by week one. These patients were also about twice as likely to be in a better state of health at multiple points in the study, with less required mechanical ventilation and fewer days spent in the ICU.
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To learn more about GBS and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/