ChemoCentryx, Inc., has announced positive top-line results from its phase 2 CLASSIC study of CCX168 in patients with antineutrophil cytoplasmic auto-antibody (ANCA)-associated vasculitis (AAV). The study’s safety objective was achieved: The drug was found to be well tolerated in patients with AAV when added to the current standard-of-care (SOC) regimen of chronic high-dose steroids.
These results complement the positive efficacy results reported in May from the phase 2 CLEAR trial, which demonstrated that CCX168—in the absence of chronic high-dose steroids—induced rapid clinical benefits in AAV as measured by improvements in Birmingham Vasculitis Activity score, renal function parameters, and patient-reported outcomes.
Taken together, the findings of the CLEAR and CLASSIC trials of CCX168 suggest that the drug could potentially replace the steroids currently used in the SOC treatment of AAV, according to the company. Chronic steroid use is associated with significant safety issues, including death.
CCX168, an orally administered small molecule, is a selective inhibitor of the complement C5a receptor and is the lead drug candidate in ChemoCentryx’s orphan and rare disease program. CCX168 has been granted orphan drug designation for AAV in the U.S. and European Union and was also granted access to the European Medicines Agency's Priority Medicines initiative, which supports accelerated assessment of investigational therapies addressing unmet medical need.
AAV is a type of rare autoimmune inflammation caused by auto-antibodies that can destroy different organ systems, mostly the kidneys, eyes, lungs, sinuses, and nerves. AAV encompasses granulomatosis with polyangiitis (formerly known as Wegener's granulomatosis), microscopic polyangiitis, eosinophilic polyangiitis (formerly Churg-Strauss syndrome), and renal limited vasculitis. It affects approximately 40,000 people in the U.S. (with approximately 4,000 new cases each year) and more than 75,000 people in Europe (with at least 7,500 new cases each year). First-year mortality is approximately 11% to 18%.
Initiation of phase 3 development for CCX168 is expected by the end of this year.
Source: ChemoCentryx, Inc.; May 23, 2016; and ChemoCentryx, Inc.; June 16, 2016.