Achillion Pharmaceuticals to Support a Natural History Study of C3 Glomerulopathy, a Rare Renal Disorder, Conducted by Expert...
February 28 2017 - 6:05AM
Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
announced that it has entered into an agreement with Imperial
College London to conduct a natural history study of C3
glomerulopathy (C3G), a rare renal disorder which includes dense
deposit disease (DDD) and C3 glomerulonephritis (C3GN). C3G affects
an estimated 8,000 people across Europe and the United States.
“Currently there is no available cure for C3G,
nor any approved treatments to prevent its progression toward renal
failure,” said Dr. David Apelian, M.D., Ph.D., and Chief Medical
Officer at Achillion. “This natural history study will provide
important insights into both C3G as a disease and the experiences
of patients. We expect this information to be valuable as we
continue to progress development of ACH-4471, our first-in-class
oral factor D inhibitor.”
This study, funded by Achillion, is being conducted by a team of
researchers led by Dr. Matthew Pickering and Dr. H. Terry Cook,
both of Imperial College. The title of the study is “Natural
History Study of C3 Glomerulopathy (C3G): Discovery of Histological
Predictors of Outcome.” The study is expected to be three years in
length. It will include up to 400 participants.
“C3G is a devastating illness for which a treatment or cure is
urgently needed. Approximately 10 years after diagnosis, about half
of all patients will progress to renal failure, ultimately
requiring dialysis or transplantation,” said Dr. Pickering. “This
natural history study will help to more clearly characterize this
disease, and its results will be shared publicly with the hope that
its findings will support research and development of a
treatment.”
A natural history study tracks the course of a disease over
time. The aim of such studies is to collect data on disease
progression, and this knowledge can inform and support product
development and approval. Without a natural history study, this
type of information is often not available, or is incomplete, for
many rare diseases, including C3G.
C3 glomerulopathy (C3G) describes a rare renal disease
characterized by the presence of fragments of a protein called C3
in the filtering units (glomeruli) of the kidney. C3 fragment
accumulation results from over-activation of part of the immune
system known as the complement alternative pathway. This
accumulation results in inflammation in the glomeruli
(glomerulonephritis) and subsequent permanent damage (need for
dialysis or transplant) in 30-50% of patients within 10 years of
diagnosis. C3G affects persons of all ages, although the mean age
appears to be lower in DDD patients as compared to C3GN patients.
The incidence of C3G is estimated at 2-3 per 1,000,000 people.
Achillion researchers have designed a library of nearly 2,000
small molecule inhibitors of complement factor D. Factor D occupies
a critical position in the complement alternative pathway.
Dysregulation of the alternative pathway can induce inflammation
and tissue damage and is associated with a variety of diseases,
including PNH and C3G. Achillion's lead candidate, ACH-4471,
entered the clinic last year and is reported to be the first factor
D inhibitor to demonstrate complement alternative pathway
inhibition in humans after oral dosing.
About the Achillion Complement Factor D
Platform
Achillion has leveraged its internal discovery capabilities and
a novel complement-related platform to develop small molecule drug
candidates that are oral inhibitors of complement factor D. Factor
D is an essential serine protease involved in the complement
pathway, a part of the innate immune system. Achillion's complement
platform is focused on seeking to advance small molecule compounds
that inhibit factor D and can potentially be used in the treatment
of immune-related diseases in which complement alternative pathway
plays a critical role. Potential indications being evaluated for
these compounds include paroxysmal nocturnal hemoglobinuria (PNH),
C3 glomerulopathy (C3G), and dry age-related macular degeneration
(dry AMD).
About Achillion
Pharmaceuticals
Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is
a science-driven, patient-focused company seeking to leverage its
strengths across the continuum from discovery to commercialization
in its goal of providing better treatments for people with serious
diseases. The company employs a highly-disciplined discovery and
development approach that has allowed it to pursue best-in-class
oral antiviral therapy for chronic hepatitis C (HCV) and build a
platform of potent and specific complement inhibitors. Achillion is
rapidly advancing its efforts to become a fully-integrated
pharmaceutical company with a goal of bringing life-saving
medicines to patients with rare diseases. More information is
available at http://www.achillion.com.
Cautionary Note Regarding
Forward-Looking Statements
This press release includes forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995 that are subject to risks, uncertainties and
other important factors that could cause actual results to differ
materially from those indicated by such forward-looking statements.
Achillion may use words such as “expect,” “anticipate,” “project,”
“target,” “intend,” “plan,” “aim,” “believe,” “seek,” “ estimate,”
“can,” “could” “focus,” “will,” “look forward,” “goal,” “may,”
“potential,” and similar expressions to identify such
forward-looking statements. These forward-looking statements also
include statements about: the expected benefits of the C3G natural
history study and the information to be derived from such study;
the potential benefits of, and potential indications for,
Achillion’s compounds that inhibit factor D; and statements
concerning Achillion’s strategic goals, efforts, plans, and
prospects. Among the important factors that could cause actual
results to differ materially from those indicated by such
forward-looking statements are risks relating to, among other
things the ability of researchers at Imperial College to
successfully enroll, complete and extract relevant data from the
C3G natural history study, as well as Achillion’s ability to:
advance the preclinical and clinical development of its complement
factor D inhibitors under the timelines it projects in current and
future preclinical studies and clinical trials; obtain and maintain
patent protection for its drug candidates and the freedom to
operate under third party intellectual property; demonstrate in any
current and future clinical trials the requisite safety, efficacy
and combinability of its drug candidates; obtain and maintain
necessary regulatory approvals; establish commercial manufacturing
arrangements; identify, enter into and maintain collaboration
agreements with third-parties, including the current collaboration
with Janssen; compete successfully in the markets in which it seeks
to develop and commercialize its product candidates and future
products; manage expenses; manage litigation; raise the substantial
additional capital needed to achieve its business objectives; and
successfully execute on its business strategies. These and other
risks are described in the reports filed by Achillion with the U.S.
Securities and Exchange Commission, including its Annual Report on
Form 10-K for the fiscal year ended December 31, 2016, and any
subsequent SEC filings.
In addition, any forward-looking statement in
this press release represents Achillion's views only as of the date
of this press release and should not be relied upon as representing
its views as of any subsequent date. Achillion disclaims any duty
to update any forward-looking statement, except as required by
applicable law.
Investors & Corporate:
Glenn Schulman
Executive Director, Investor Relations
Achillion Pharmaceuticals, Inc.
Tel. (203) 752-5510
gschulman@achillion.com
Media:
Liz Power
Senior Director, Public Relations
Achillion Pharmaceuticals, Inc.
Tel: (203) 752-5509
lpower@achillion.com
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