ChemoCentryx has enrolled 30 percent of the 300 ANCA-associated vasculitis patients it hopes to recruit for a Phase 3 trial assessing the effectiveness of its therapy avacopan.
Avacopan inhibits a protein known as the complement 5a receptor (C5aR) that is involved in inflammatory signaling in ANCA-associated vasculitis, also known as AAV. A vasculitis is a disease characterized by inflamed blood vessels. If the inflammation is left untreated, it can destroy vessels.
AAV’s full name is anti-neutrophil cytoplasmic antibody-associated vasculitis.
Both the U.S. Food and Drug Administration and European Medicines Agency have designated avacopan an orphan drug. This status is designed to speed up its development and regulatory approval.
ChemoCentryx recently announced that avacopan has received orphan drug designation in Switzerland for the treatment of two AAV subtypes, microscopic polyangiitis and granulomatosis with polyangiitis.
“Our pursuit of new and better medicines for people with orphan diseases has been relentless,” Dr. Thomas J. Schall, ChemoCentryx’s president and CEO, said in a press release. “Dedicated to creating value for patients and shareholders alike, we at CCXI started with basic science in the discovery of novel molecules that selectively inhibit chemo-attractant receptors, which are the molecular guidance systems of destructive inflammatory cells involved in a wide range of diseases and conditions.”
The 12-week Phase 2 CLEAR trial (NCT01363388) showed that avacopan could replace high-dose glucocorticoids as a treatment for vasculitis. Glucocorticoids are steroids that can counter the inflammation in vasculitis but can damage organs if used for a long time.
The main objective of the Phase 3 ADVOCATE trial is to see if avacopan can put AAV into remission. ADVOCATE will also look at whether oral avacopan can prevent the disease from returning — something other therapies are unable to do.
Trial participants will receive either avacopan or another therapy, prednisone, for up to 12 months. Both treatments will be given in combination with Genentech’s Rituxan (rituximab) or cyclophosphamide, followed by azathioprine.
In addition to seeing whether avacopan can put patients’ vasculitis into remission, researchers will look for any adverse events the drug generates, at glucocorticoid-triggered toxicity that patients experience, their health-related quality of life, and other measures.