ChemoCentryx Granted EU Orphan Drug Designation for Avacopan in the Treatment of Debilitating Kidney Disease C3 Glomerulopath...
May 23 2017 - 8:30AM
ChemoCentryx, Inc., (Nasdaq:CCXI), a biopharmaceutical company
developing new medications targeted at inflammatory and autoimmune
diseases and cancer, announced today that the European
Medicines Agency (EMA) has granted orphan medicinal product
designation for avacopan (formerly CCX168) in the treatment of
patients with C3 glomerulopathy (C3G).
In March, ChemoCentryx announced that the U.S. Food and Drug
Administration (FDA) had granted orphan drug designation for
avacopan in the treatment of C3G. Orphan drug designation can help
expedite a drug candidate’s path to market and provide certain
exclusive marketing advantages after approval. ChemoCentryx plans
to initiate a multi-center clinical endpoint study of avacopan for
the treatment of C3G in mid-2017.
C3G is a severe kidney disease characterized by deposits of
proteins from the body's complement system into and around the
kidney's filtration units, the glomeruli. The deposition of
complement protein disrupts kidney function and also triggers a
profoundly destructive inflammation of the kidney. The disease
process ultimately leads to renal failure, with the need for
dialysis and kidney transplant.
There is currently no approved treatment for C3G, and relapse is
common even after kidney transplant.
Avacopan targets the complement C5a receptor (C5aR) which drives
the destructive inflammatory cells in C3G and other kidney
diseases. In this way, avacopan is designed to arrest the
ongoing process of kidney damage in C3G, allowing kidney function
to stabilize.
"Securing both U.S. and EU orphan drug designations in quick
succession for avacopan in the treatment of C3G provides further
validation of the drug candidate’s potential,”
said Thomas J. Schall, Ph.D., President and Chief
Executive Officer of ChemoCentryx. "There are no approved
treatments for C3G, a disorder caused by dysregulation of the
complement system. We believe the ability to selectively target the
C5a receptor with a patient-friendly small molecule such as
avacopan could be a significant breakthrough in the treatment of
C3G and other kidney disorders.”
About Avacopan (CCX168)
Avacopan (CCX168) is an orally-administered small molecule that
is a selective inhibitor of the complement C5a receptor, or C5aR.
Avacopan is in Phase III development for the treatment of
anti-neutrophil cytoplasmic auto-antibody-associated vasculitis
(AAV). In clinical studies to date, avacopan was shown to be safe,
well tolerated and provided effective control of the disease, while
in addition, successfully allowing elimination of high-dose
steroids, part of the standard of care for AAV patients. Avacopan
is also being developed in patients with atypical hemolytic uremic
syndrome (aHUS) and C3 glomerulopathy (C3G). In C3G, avacopan
targets the C5a receptor, blocking the effects of C5a which
contributes to the inflammatory hypercellularity in the glomeruli,
a main feature of C3G.
The FDA has granted avacopan orphan-drug designation for
all three of these diseases: C3G, AAV, and aHUS. The EMA has
now granted orphan medicinal product designation for avacopan for
the treatment of two forms of AAV: microscopic polyangiitis and
granulomatosis with polyangiitis (formerly known as Wegener's
granulomatosis) and C3G.
Avacopan was also granted access to the EMA’s 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. This
C3 deposition is a manifestation of inappropriate complement
activation which also leads to complement 5a (C5a) production.
Patients with C3 glomerulopathy often have high levels of protein
in the urine (proteinuria) and experience progressive deterioration
in renal function. Without treatment, C3G leads to kidney failure,
with the need for dialysis and kidney transplant. 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 million people.
About ChemoCentryx
ChemoCentryx is a biopharmaceutical company developing new
medications targeted at inflammatory and autoimmune diseases, and
cancer. ChemoCentryx targets the chemokine and chemoattractant
systems to discover, develop and commercialize orally-administered
therapies. ChemoCentryx is currently focusing on its late stage
drug candidates for patients with rare kidney diseases. Besides
avacopan (described above), the Company’s other late stage drug
candidate is CCX140, an inhibitor of the chemokine receptor known
as CCR2, which is currently being developed for patients with focal
segmental glomerulosclerosis (FSGS), a debilitating kidney
disease.
ChemoCentryx’s Kidney Health Alliance with Vifor Pharma provides
Vifor Pharma with exclusive rights to commercialize avacopan and
CCX140 in markets outside of the U.S. and China.
ChemoCentryx also has an immuno-oncology program, which
includes a distinct CCR2 inhibitor, CCX872, currently in
development for the treatment of advanced non-resectable pancreatic
cancer.
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 avacopan (CCX168) will be shown to be safe and
effective in the treatment of C3 glomerulopathy and other rare
diseases, whether orphan drug designation will expedite a drug
candidate’s path to market and provide certain exclusive marketing
advantages after approval and the Company’s statement regarding the
timing of initiating additional clinical trials to further
investigate avacopan 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 14, 2017 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.
Contacts:
Susan M. Kanaya
Executive Vice President, Finance and Chief Financial and Administrative 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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