Rare Inflammatory Disease Drug Beats Immunosuppressants in Phase III

lupus autoimmune disease neutrophil citryll

A small molecule drug developed by the US company ChemoCentryx and its Swiss partner Vifor Fresenius Medical Care Renal Pharma (VFMCRP) was more effective at treating a rare inflammatory vascular disease than current treatments in a phase III trial.

The drug avacopan was trialed in 331 patients with anti-neutrophil cytoplasmic antibody-associated vasculitis. This life-threatening condition is also known as ANCA vasculitis, and happens when inflammatory immune cells called neutrophils attack small blood vessels and cause organ damage, fever and skin lesions.

The phase III trial met its two primary goals, which were to reduce symptoms of the condition within six months, and to maintain this reduction over one year. After a year of treatment with avacopan, over 65% of patients had their symptoms reduced compared to only 55% of patients treated with glucocorticoid immunosuppressants, the current standard of care. Avacopan also showed fewer harmful effects than the current standard of care, which can cause progressive organ damage.

Avacopan is designed to block C5a, a protein that activates inflammatory neutrophils. This stops neutrophils from attacking the blood vessels and leaves the rest of the immune system intact. In contrast, current immunosuppressants can weaken the whole immune system and leave the patient vulnerable to infections. In addition, avacopan is designed to reduce the need for glucocorticoid immunosuppressants in ANCA vasculitis, whose harmful effects are responsible for around half of all ANCA vasculitis patient deaths in the first year of therapy.

This day we have for the first time demonstrated that a highly targeted therapy aimed at the very center of the ANCA disease process is superior to the traditional approach of broad immune suppression therapy; a therapy which the present findings may make obsolete,” stated Thomas Schall, CEO of ChemoCentryx.

The partners expect to apply for FDA and EMA approval in the rare condition in 2020. 

ChemoCentryx is responsible for the development of avacopan, and owns the rights to commercialize the drug in the US and China. Its partner VFMCRP has an exclusive license to commercialize avacopan outside these regions.

ANCA vasculitis is also a target disease of the German biotech InflaRx. The company launched a phase II trial of an antibody that also targets the C5a protein in the condition earlier this year. However, the same antibody failed earlier this year in a phase IIb trial on another inflammatory condition, so it remains to be seen if its drug will be able to compete with avacopan.

Images from Shutterstock

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