- Conditional marketing authorization application for
treatment of patients with ANCA-associated vasculitis validated for
start of procedure by EMA
- Significant milestone further strengthens Kidney Health
Alliance
- ChemoCentryx to receive regulatory milestone payment
from VFMCRP
VIFOR FRESENIUS MEDICAL CARE RENAL PHARMA AND CHEMOCENTRYX
ANNOUNCE THAT THE EUROPEAN MEDICINES AGENCY HAS ACCEPTED FOR REVIEW
THE REGISTRATION DOSSIER IN SUPPORT OF A CONDITIONAL MARKETING
AUTHORIZATION FOR AVACOPAN IN THE TREATMENT OF PATIENTS WITH
ANCA-ASSOCIATED VASCULITIS.
Vifor Fresenius Medical Care Renal Pharma (VFMCRP) and
ChemoCentryx, Inc., (Nasdaq:CCXI), announced today a significant
milestone in their Kidney Health Alliance: ChemoCentryx’s
application for avacopan in the treatment of patients with
anti-neutrophil cytoplasmic antibody associated vasculitis
(ANCA-associated vasculitis or ANCA vasculitis) for regulatory
review of its Conditional Marketing Authorization (CMA) application
was accepted by the European Medicines Agency (EMA). The EMA’s
Committee for Medicinal Products for Human Use (CHMP) will now
start to assess the CMA application. Under the terms of its kidney
health alliance with VFMCRP, ChemoCentryx will receive a milestone
payment triggered by this validation of the avacopan CMA
application by the EMA.
Avacopan is an orally-administered small molecule that is a
highly selective inhibitor of the terminal effector complement C5a
receptor (C5aR). Avacopan is currently in late-stage clinical
development for the treatment of orphan and rare renal diseases,
including ANCA vasculitis. In a randomized, double-blind,
placebo-controlled Phase II study in ANCA vasculitis patients,
known as the CLEAR trial, avacopan demonstrated that blocking C5aR
at the terminal effector pathway of the complement cascade provides
therapeutic efficacy and a favorable risk/benefit profile with a
rapid onset of action. Avacopan is currently being studied in the
pivotal Phase III ADVOCATE trial for the treatment of ANCA
vasculitis and is on track to complete enrollment by mid-2018.
ChemoCentryx, which is responsible for the discovery and
development of avacopan, owns and retains the commercial rights to
the drug in the United States and China, and VFMCRP has licensed
the rights to commercialize the drug in all other countries.
“The EMA’s validation of the Conditional Marketing Authorization
application for avacopan represents another critical step in Europe
toward realizing our vision of becoming a global leader in
nephrology therapies. Avacopan is a much more selective, targeted
means of treating ANCA than the current standard of care,” said
Stefan Schulze, Vifor Pharma President of the Executive Committee
and COO. “We believe this treatment has real potential for
transforming the way that ANCA vasculitis is treated and that it
will be of tremendous value to patients who have to live with this
rare renal inflammatory disease.”
ANCA vasculitis is a systemic disease in which over-activation
of the complement pathway further activates neutrophils to lead to
inflammation and destruction of small blood vessels. This results
in organ damage and failure, with the kidney as the major target,
and is fatal if not treated. Currently, treatment for ANCA
vasculitis consists of courses of non-specific immuno-suppressants
(cyclophosphamide or rituximab), combined with high-dose
corticosteroid administration for prolonged periods of time, which
can be associated with significant clinical risk including death
from infection. The CLEAR data show that avacopan may markedly
reduce the reliance on glucocorticoids in the treatment of ANCA
vasculitis. Furthermore, avacopan by targeting the underlying
inflammatory disease process while permitting otherwise normal
functioning of other components of the immune system, may provide a
basis for a totally new of way of improving ANCA vasculitis
outcomes.
“A new treatment paradigm for ANCA vasculitis has been a goal
for clinicians and patients for decades,” said Professor David
Jayne, Vasculitis Director, Addenbrooke’s Hospital, University of
Cambridge, UK. “For half a century, we have used high-dose
glucocorticoids as part of the treatment of ANCA vasculitis,
despite their well-documented toxicities that contribute to the
long term morbidity and incapacity associated with vasculitis. In
the Phase II CLEAR study, it was demonstrated that inhibition of
C5aR may make the chronic use of these toxic steroids obsolete,
because the target of avacopan, the C5aR, is an important and
specific driver of the destructive inflammation of ANCA vasculitis.
The current data are very promising and we look forward to the
results from the ADVOCATE Phase III trial.”
“The value and promise of avacopan for patients with ANCA
vasculitis has today been reinforced by the EMA validation of the
avacopan CMA application,” said Thomas J. Schall, Ph.D., President
and Chief Executive Officer of ChemoCentryx. “We at ChemoCentryx,
together with our outstanding partners at Vifor Pharma, are
unswerving in our devotion to creating new medicines for patients
suffering from devastating kidney diseases. To ANCA patients, we
say this: your cause is our cause; we are determined to succeed.
Achieving this pivotal regulatory milestone is a big step toward
our ultimate success.”
In June 2016, avacopan was granted access to the EMA's Priority
Medicines (PRIME) regulatory initiative for the treatment of
patients with ANCA vasculitis. Access to the PRIME initiative is
granted by the EMA to support the development and accelerate the
review of new therapies to treat patients with unmet medical
need.
Anti-neutrophil cytoplasmic antibody (ANCA)-associated
vasculitis, or ANCA vasculitis, is a group of highly
inflammatory autoimmune and progressive rare diseases caused by the
over-activation of the complement system, which activates
neutrophils to destroy blood vessels through inflammation. Patients
with granulomatosis with polyangiitis (GPA; formerly Wegener's) or
microscopic polyangiitis (MPA), two forms of ANCA vasculitis have
various presentations from asymptomatic abnormal lab results to
relapsing-remitting states characterized with recurring flares,
accruing into irreversible organ system damage, failure and
death.
ANCA vasculitis is the lead indication in ChemoCentryx's orphan
and rare disease program which has the objective of making chronic
high dose glucocorticoids irrelevant. In addition to being at major
risk of severe necrotizing inflammation of vessel walls leading to
multiple organ system failure, in particular end stage renal
insufficiency as well as mortality, newly diagnosed patients as
well as those in remission suffer from impaired quality of life,
which amongst several general health-reported parameters, fatigue
is major determinant. The disease affects people of working age and
significantly impacts multiple aspects of their physical function,
emotional well-being, and overall productivity.
ANCA vasculitis affects approximately 40,000 people in the U.S.
(with approximately 4,000 new cases each year) and less than 50,000
people in Europe (with an estimated 5,000 new cases each year), and
is currently treated with courses of immunosuppressants
(cyclophosphamide or rituximab) combined with high dose steroid
administration. These induction protocols achieve only partial
sustained remission rates of approximately 70%, with up to 30
percent of patients relapsing within six to 18 months, and
approximately half of all patients relapsing within three to five
years. Patients with renal involvement have a worse prognosis than
patients without renal involvement, and 23% of ANCA vasculitis
patients who require dialysis or transplant at time of diagnosis
die within six months. As early as six months after diagnosis, 8%
of patients develop end-stage renal disease and a total of 42% of
patients with renal involvement die or develop end-stage renal
disease at two years.
The current standard of care for ANCA vasculitis is associated
with significant safety issues, underscoring the need for new
therapies that specifically target disease mechanisms more
selectively. First year mortality is approximately 11% to 18%, with
most of the deaths occurring within the first three months, during
the time when glucocorticoids are used at high doses. The single
major cause of premature mortality is not disease-related, 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 morbidity and mortality. 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, ANCA vasculitis
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 ANCA vasculitis,
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 avacopan. By blocking the C5aR, avacopan is thought to
reduce vasculitis by reducing neutrophil activation, accumulation,
and adhesion, as well as vascular permeability.
Avacopan is an orally-administered small
molecule that is a selective inhibitor of the terminal effector and
neutrophil chemoattractant complement C5a receptor, or C5aR. It
allows tempering of ANCA responses and thereby to prevent both
complement C5a activation while leaving other host defense
mechanisms (such as the membrane attack complex, being distinct
from C5b) of the immune system unaffected. Avacopan is in phase III
development for the treatment of anti-neutrophil cytoplasmic
auto-antibody-associated vasculitis (ANCA vasculitis). In clinical
studies to date, avacopan was shown to be safe, well tolerated and
provided effective control of the disease while successfully
allowing elimination of high-dose steroids, part of the standard of
care for patients with ANCA vasculitis. 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 U.S. Food and Drug Administration has granted avacopan
orphan-drug designation for all three of these diseases: C3G, ANCA
vasculitis, and aHUS. The European Commission has granted orphan
medicinal product designation for avacopan for the treatment of two
forms of ANCA vasculitis: microscopic polyangiitis and
granulomatosis with polyangiitis (formerly known as Wegener's
granulomatosis), and C3G. Avacopan 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.
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 early stage drug candidates that
target chemoattractant receptors in other Inflammatory and
autoimmune diseases and in cancer.
Vifor Pharma Group, formerly Galenica Group, is
a global specialty pharmaceuticals company. It aims to become the
global leader in iron deficiency, nephrology and cardio-renal
therapies. The company is the partner of choice for specialty
pharmaceuticals and innovative patient-focused solutions. Vifor
Pharma Group strives to help patients around the world with severe
and chronic diseases lead better, healthier lives. The company
develops, manufactures and markets pharmaceutical products for
precision patient care. Vifor Pharma Group holds a leading position
in all its core business activities and consists of the following
companies: Vifor Pharma; Vifor Fresenius Medical Care Renal Pharma,
a joint company with Fresenius Medical Care; Relypsa; and OM
Pharma. Vifor Pharma Group is listed on the Swiss Stock Exchange
(SIX Swiss Exchange, VIFN, ISIN: CH0364749348). For more
information, visit www.viforpharma.com.
Vifor Fresenius Medical Care Renal Pharma Ltd.,
a common company of Vifor Pharma Group and Fresenius Medical Care,
develops and commercialises innovative and high quality therapies
to improve the life of patients suffering from chronic kidney
disease (CKD) worldwide. The company was founded at the end of 2010
and is owned 55% by Vifor Pharma Group and 45% by Fresenius Medical
Care. For more information about Vifor Fresenius Medical Care Renal
Pharma and its parent companies, please visit www.vfmcrp.com,
www.viforpharma.com and www.freseniusmedicalcare.com.
ChemoCentryx 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"
or "continue" or the negative of these terms or other comparable
terminology are intended to identify forward-looking statements.
These statements include statements regarding whether avacopan will
be shown to be effective in the pivotal Phase III ADVOCATE trial in
the treatment of ANCA vasculitis and other rare renal diseases,
whether avacopan will receive conditional marketing approval by the
EMA for the treatment of ANCA vasculitis and whether patient
enrollment of the ADVOCATE Phase III trial will be completed by
mid-2018. 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
ChemoCentryx'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 14
March 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.
FURTHER INFORMATION
Media
Relations |
Investor
Relations |
|
Victoria Maier |
Julien Vignot |
|
Senior Manager External
Communications |
Head of Investor
Relations |
|
Tel.: +41 58 851 80
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Tel.: +41 58 851 66
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E-mail:
media@viforpharma.com |
E-mail:
investors@viforpharma.com |
|
ChemoCentryx Contacts:Susan M.
KanayaExecutive Vice President, Chief Financial and Administrative
OfficerE-mail: investor@chemocentryx.com
Media: Stephanie Tomei Tel.: +1
408-234-1279E-mail: media@chemocentryx.com
Investors:Steve KlassBurns
McClellanTel.: +1 212-213-0006E-mail: sklass@burnsmc.com
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