ChemoCentryx Reports Received EMA PRIME Designation for Accelerated Assessment of CCX168 for Treatment of Patients with ANCA-Associated Vasculitis

ChemoCentryx, Inc., CCXI, a clinical-stage biopharmaceutical company developing orally-administered therapeutics to treat autoimmune diseases, inflammatory disorders, and cancer, today announced it received PRIority MEdicines (PRIME) designation from the European Medicines Agency (EMA) for CCX168 for the treatment of ANCA-Associated Vasculitis, or AAV. As described by the EMA, PRIME aims to bring promising innovative medicines to patients faster by optimizing and supporting medicine development. To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on clinical data. ChemoCentryx had previously received orphan drug status in Europe for CCX168 for use in the two main forms of AAV, namely granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). In the newly conferred PRIME designation, EMA acknowledged that GPA and MPA are highly severe diseases, and that the safety profiles of the existing treatments are not optimal. Mortality at one year exceeds 15% and the predominant causes of early death are infection and active vasculitis. This, together with the severe toxicity and only partial efficacy of the currently used regimens indicate an unmet medical need for improved treatment options for patients with AAV. Furthermore, the EMA confirmed its view that CCX168 provides a new mechanism of action for the treatment of GPA and MPA, and the potential of the product to significantly address the unmet medical need is supported by nonclinical and clinical data. It was noted that nonclinical proof-of-principle has been demonstrated in two relevant disease models and the nonclinical safety profile appeared reassuring. The EMA also found that, while duration of the clinical exposure is currently limited, the magnitude of effect, measured using a valid endpoint of Birmingham Vasculitis Activity Score (BVAS) response, is promising and suggests steroid sparing potential. "The EMA's PRIME designation provides further validation of the potential of CCX168 to fulfill a high unmet medical need and change the current treatment paradigm in AAV," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "The use of steroid-containing standard treatment for AAV is thought to be the single biggest cause of premature death among these patients. Our goal with CCX168 is to eliminate or limit the use of steroids along with their deleterious side effects, while controlling the disease, and possibly, more rapidly. The PRIME designation will allow us to accelerate the regulatory review process, potentially allowing CCX168 to reach the market sooner than previously expected." Earlier this year, ChemoCentryx announced positive results from the Phase II CLEAR trial with CCX168 in patients with AAV. Chronic high dose steroid administration in the current standard of care (SOC) is associated with premature death and a spectrum of other harmful side effects in AAV therapy. The objective of the CLEAR trial was to replace chronic high dose steroids with CCX168 and to eliminate the safety issues associated with steroids, from the SOC regimen in AAV. Results from the CLEAR trial showed that CCX168 has the potential to safely replace chronic high dose steroids, and patients receiving CCX168 showed improvement within four weeks of starting treatment based on BVAS, urinary albumin excretion, and health-related quality of life outcomes. Based on the results from the CLEAR trial, the Company plans to initiate the Phase III development program with CCX168 for the treatment of AAV by the end of 2016.