MOUNTAIN VIEW, Calif., June 01, 2016 (GLOBE NEWSWIRE) -- ChemoCentryx, Inc., (Nasdaq: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.
About EMA PRIME Designation
PRIME is a program launched by the EMA to enhance support for the development of medicines that target an unmet medical need. This voluntary scheme is based on enhanced interaction and early dialogue with developers of promising medicines, to optimize development plans and speed up evaluation so these medicines can reach patients earlier. To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on early clinical data. Through PRIME, the EMA offers early and proactive support to medicine developers to optimize the generation of robust data on a medicine’s benefits and risks and enable accelerated assessment of medicines applications. More information about PRIME can be found on the EMA website.
About ANCA-Associated Vasculitis
Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, or AAV, is a type of rare autoimmune inflammation caused by auto-antibodies. AAV encompasses granulomatosis with polyangiitis (GPA, formerly known as Wegener's granulomatosis), microscopic polyangiitis (MPA), eosinophilic polyangiitis (formerly Churg-Strauss syndrome) and renal limited vasculitis.
AAV represents a severe and often fatal autoimmune disease that is characterized by inflammation that can destroy different organ systems. AAV is the lead indication in the Company's orphan and rare disease program which has the objective of eliminating chronic high dose steroids, which are associated with significant safety issues including death, from the standard of care (SOC) regimen in AAV and replacing steroids with CCX168.
AAV affects approximately 40,000 people in the U.S. (with approximately 4,000 new cases each year) and greater than 75,000 people in Europe (with at least 7,500 new cases each year), and is currently treated with courses of immuno-suppressants (cyclophosphamide or rituximab) combined with high dose steroid administration. Following initial treatment, up to 30 percent of patients relapse within six to 18 months, and approximately half of all patients will relapse within three to five years.
Current SOC for AAV is associated with significant safety issues. First year mortality is approximately 11 to 18 percent. The single major cause of premature mortality is not disease-related adverse events, but rather infection that is thought largely to be a consequence of steroid administration. Indeed, the multiple adverse effects of courses of steroid treatment (both initial courses and those that are repeated as a consequence of relapse) are major causes of both short-term and long-term disease and death. Such therapy related adverse events contribute significantly to patient care costs, as well as to the diminution of quality of life for patients.
By damaging the body's small blood vessels, AAV affects many organ systems, mostly the kidneys, eyes, lungs, sinuses and nerves. This damage is caused by the destructive activity of inflammatory leukocytes in the body, with neutrophils considered to be the terminal effector cell. In AAV, neutrophils are attracted to sites of vascular destruction as well as activated at those sites by the activity of the complement system product known as C5a and its receptor, C5aR, which is the target of CCX168. By blocking the C5aR, CCX168 is thought to reduce vasculitis by reducing neutrophil activation, accumulation, and adhesion, as well as vascular permeability.
CCX168 is an orally-administered complement inhibitor, specifically targeting the C5a receptor (C5aR), which binds the complement fragment C5a. CCX168 is the lead drug candidate in the Company's orphan and rare disease program and is being developed for various autoimmune disorders including ANCA-associated vasculitis (AAV), atypical hemolytic uremic syndrome (aHUS) and immunoglobulin A nephropathy, or IgA nephropathy.
ChemoCentryx, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing orally-administered therapeutics that target the chemokine and chemoattractant systems in order to treat autoimmune diseases, inflammatory disorders and cancer. The chemokine system is a biological network that regulates inflammation via a collection of secreted chemokine molecules, or ligands, and their specific cell surface receptors. Based on its proprietary drug discovery and drug development platform, ChemoCentryx has generated multiple clinical and preclinical-stage programs, each targeting distinct chemokine and chemoattractant receptors with different small molecule compounds. CCX168, a C5aR inhibitor, is in Phase II development for the treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV). CCX168 appears to be safe, well tolerated and successful in allowing reduction and elimination of high-dose steroids, part of standard of care for AAV patients, without compromising efficacy or safety in clinical studies to date. CCX168 is also in Phase II studies for the treatment of atypical hemolytic uremic syndrome (aHUS) and immunoglobulin A nephropathy, or IgA nephropathy (IgAN). ChemoCentryx has licensed exclusive rights to Vifor Pharma to commercialize CCX168 in Europe and certain other markets outside of the U.S. and most of Asia. CCX872, a CCR2 inhibitor, successfully completed Phase I development and is in development for the treatment of non-resectable pancreatic cancer. CCX140, a distinct CCR2 inhibitor, successfully completed a Phase II clinical trial where it was shown to be safe and well tolerated while demonstrating statistically significant improvement in albuminuria in patients with diabetic nephropathy. Other clinical programs include CCX507, a next generation CCR9 inhibitor, which has successfully completed Phase I development, vercirnon (also known as Traficet-EN or CCX282) a specific CCR9 inhibitor for the treatment of inflammatory bowel disease, and CCX354, a CCR1 inhibitor which successfully completed a Phase II clinical trial for the treatment of rheumatoid arthritis. ChemoCentryx also has several programs in advanced preclinical development.
ChemoCentryx cautions that statements included in this press release that are not a description of historical facts are forward-looking statements. Words such as "may," "could," "will," "would," "should," "expect," "plan," "anticipate," "believe," "estimate," "intend," "predict," "seek," "contemplate," "potential," "continue" or "project" or the negative of these terms or other comparable terminology are intended to identify forward-looking statements. These statements include the Company's statements regarding whether CCX168 will be able to replace chronic high dose steroids and when and if CCX168 will obtain regulatory approval and be commercialized for the treatment of AAV. The inclusion of forward-looking statements should not be regarded as a representation by ChemoCentryx that any of its plans will be achieved. Actual results may differ from those set forth in this release due to the risks and uncertainties inherent in the ChemoCentryx business and other risks described in the Company's filings with the Securities and Exchange Commission ("SEC"). Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and ChemoCentryx undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. Further information regarding these and other risks is included under the heading "Risk Factors" in ChemoCentryx's periodic reports filed with the SEC, including ChemoCentryx's Annual Report on Form 10-K filed with the SEC March 14, 2016 and its other reports which are available from the SEC's website (www.sec.gov) and on ChemoCentryx's website (www.chemocentryx.com) under the heading "Investors." All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
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