MOUNTAIN VIEW, Calif., April 28, 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 that the U.S. Food and Drug Administration (FDA) has awarded the Company a one-year grant of $500,000 to assist in the clinical development of CCX168, the Company’s lead drug candidate for the treatment of patients with anti-neutrophil cytoplasmic antibody (ANCA) -associated vasculitis, or AAV. CCX168 is an orally-administered small molecule that is a selective inhibitor of the complement C5a receptor, or C5aR.
The goal of the FDA's Orphan Products Development grant program is to support the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA provides grants for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products.
“We are pleased with the FDA’s recognition of the potential of CCX168 to shift the current treatment paradigm in ANCA Associated Vasculitis, by eliminating the use of steroids and their noxious side effects from the current standard of care,” said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. “ANCA associated vasculitis is a rare, severe, and often fatal autoimmune disease that affects approximately 40,000 people in the U.S. Premature death within a year of AAV diagnosis is unacceptably high, and most do not realize that the single biggest cause of premature death is not the disease itself, but rather the steroid-containing standard therapy. The FDA’s grant will be put to good use as we prepare to initiate our Phase III clinical trial with CCX168 for the treatment of ANCA associated vasculitis by the end of the year.”
CCX168 is an orally-administered complement inhibitor which specifically targets the receptor for the complement fragment C5a receptor (C5aR). This receptor is known to activate destructive cells in certain autoimmune diseases including AAV. CCX168 is the lead drug candidate in the Company's orphan and rare disease program. In January 2016, the Company reported positive top-line data from the Phase II CLEAR trial with CCX168 in 63 evaluable patients with AAV. The objective of the trial was to eliminate chronic high dose steroids, which are associated with significant safety issues including death, from the standard of care (SOC) regimen in AAV and replace steroids with CCX168. The Company plans to initiate a Phase III clinical trial with CCX168 for the treatment of AAV by the end of 2016. CCX168 is also being developed for other autoimmune disorders including atypical hemolytic uremic syndrome (aHUS) and potentially other rare disease indications.
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. By damaging the body’s small blood vessels, AAV can affect any organ system, but mostly involves the kidneys, lungs, and ear/nose/throat. 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, C5a and its receptor, the target of CCX168, are involved in priming and activation of neutrophils, chemoattraction and vascular endothelial adhesion of these neutrophils and other leukocytes, and increased vascular permeability. By blocking the C5aR, CCX168 is thought to reduce vasculitis by reducing neutrophil activation, accumulation, and adhesion, as well as vascular permeability.
AAV affects approximately 40,000 people in the United States (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 non-specific immuno-suppressants (cyclophosphamide or rituximab) combined with high dose corticosteroid 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.
The current standard of care 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 treatment-induced, including infection that is thought largely to be a consequence of corticosteroid administration. Indeed, the multiple adverse effects of courses of corticosteroid 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.
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). 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 the achievement of anticipated milestones in 2016 or whether CCX168 will be shown to be safe and effective in ongoing or future clinical trials. 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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