ChemoCentryx Reports Improvement in Renal Physiology and Stabilization of Kidney Function Following Treatment with Orally Adm...
October 27 2016 - 8:30AM
ChemoCentryx, Inc., (Nasdaq:CCXI), today announced that its orally
administered complement 5a receptor inhibitor CCX168 (newly
designated “avacopan”) has shown a beneficial effect on disease in
a patient with C3 glomerulopathy (C3G). C3G is a rare disease of
the kidney characterized by deposition of the protein known as C3
(a component of the body’s complement system) in the filtration
units (the glomeruli) of the kidney, leading to profound kidney
damage and eventual renal failure. There is currently no approved
effective standard therapy for C3G.
Under the Special Needs program in the United Kingdom (similar
to compassionate use protocols in the United States), a C3G renal
transplant recipient with deteriorating kidney function has
responded well to treatment with the orally administered complement
inhibitor CCX168 (avacopan). After only one month of initial
treatment with avacopan, renal function (based on estimated
glomerular filtration rate, or eGFR) stabilized. Moreover,
sequential kidney biopsies taken after the patient had been on
avacopan for two and seven months showed continued improvement in
kidney histology based on a decrease in glomerular endocapillary
proliferation and a marked reduction in the number of glomerular
inflammatory macrophages, as compared to the pre-treatment
biopsy.
Prior to receiving treatment with CCX168 (avacopan), the C3G
patient had received treatment with a wide spectrum of
immunosuppressant drugs including rituximab, cyclophosphamide,
mycophenolate mofetil, tacrolimus and glucocorticosteroids.
All of these previous treatments had failed to prevent
disease recurrence and progression, including new disease in the
patient’s transplanted kidney. The patient has now had over 13
months of treatment with avacopan and continues to enjoy stabilized
kidney function, and to tolerate the agent well with no serious
adverse events. These findings provide the first evidence that
avacopan may be effective in treating patients with this rare and
debilitating disorder.
“This devastating disease, frequently targeting young people, is
called ‘C3’ glomerulopathy, but there is strong evidence that the
production of C5a in the terminal complement pathway is a major
driver of the pathology,” said Thomas J. Schall, Ph.D., President
and Chief Executive Officer of ChemoCentryx. “Accordingly,
there is a clear scientific rationale to treat C3G patients with
the C5aR inhibitor avacopan with the goal of offering an effective
treatment option otherwise not available to these individuals. This
is particularly encouraging since we recently announced positive
Phase II clinical data for avacopan in another rare disease that
typically involves the kidney, ANCA associated vasculitis, or
AAV. The positive results in AAV provide additional
validation of the use of avacopan in important rare diseases for
which current care approaches are simply not satisfactory.
Based on these findings, we plan to initiate a multi-center
clinical endpoint study to further investigate avacopan in the
treatment of C3G in the first half of 2017.”
About CCX168 (avacopan)
CCX168 (avacopan) is an orally-administered small molecule that
is a selective inhibitor of the complement C5a receptor, or C5aR,
and is the lead drug candidate in the Company's orphan and rare
disease program. The U.S. Food and Drug Administration
granted orphan-drug designation for CCX168 for the treatment of
patients with AAV, (which includes Wegener's granulomatosis,
microscopic polyangiitis, and Churg-Strauss syndrome) and also for
the treatment of patients with atypical hemolytic uremic syndrome
(aHUS). The European Commission has granted orphan medicinal
product designation for CCX168 for the treatment of microscopic
polyangiitis and granulomatosis with polyangiitis (formerly known
as Wegener’s granulomatosis). Both conditions are forms of
ANCA-associated vasculitis (AAV). CCX168 was also granted
access to the European Medicines Agency’s (EMA) PRIority MEdicines
(PRIME) initiative, which supports accelerated
assessment of investigational therapies addressing unmet medical
need.
About Complement 3 Glomerulopathy (C3G)
C3 glomerulopathy is characterized by evidence of alternative
complement activation based on C3 deposition in the glomeruli.
There are two forms of the disease: dense deposit disease (DDD,
formerly called membranoproliferative glomerulonephritis (MPGN)
Type II) and C3 glomerulonephritis (C3GN, formerly called
idiopathic MPGN). Genetic lesions leading to defective complement
regulation, including mutations in complement factor H (CFH) have
been described in these patients. Patients with C3 glomerulopathy
often have high proteinuria and progressive deterioration in renal
function. Without treatment, C3G invariably leads to kidney
failure, and kidney transplant is frequently the only option.
Even after transplantation, the new kidney will frequently manifest
the disease. There is no approved effective standard therapy for
C3G; non-specific immunosuppressants are frequently employed. The
estimated prevalence of C3G is two-to-three per one million
people.
Phase II study data in patients with ANCA-associated vasculitis
showed a significant anti-proteinuric effect of CCX168 in the
majority of treated patients in addition to marked improvements in
the Birmingham Vasculitis Activity Score, or BVAS. This treatment
effect was observed in patients receiving CCX168 plus
glucocorticoids, but also in patients receiving CCX168 with no
glucocorticoids.
About ChemoCentryx
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 (avacopan), 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 during a 12-week treatment
period. 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 improvements in kidney function 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 which has been tested in Crohn’s
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.
Forward-Looking Statements
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 whether CCX168 (avacopan) will be shown to be safe and
effective in in the treatment of C3 glomerulopathy and other rare
diseases and the Company’s statement regarding the timing of
initiating additional clinical trials to further investigate CCX168
in the treatment of C3G. 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 13, 2015 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.
CCXI-G
Contacts:
Susan M. Kanaya
Senior Vice President, Finance and
Chief Financial Officer
investor@chemocentryx.com
Media:
Denise Powell
denise@redhousecomms.com
510.703.9491
Investors:
Steve Klass, Burns McClellan
212.213.0006
sklass@burnsmc.com
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